Roche's Investigational Hepatitis C Polymerase Inhibitor, R1626, Demonstrated Significant End-of-Treatment Response In Phase IIa Study

Roche's investigational therapy
for chronic hepatitis C virus (HCV) infection, R1626, has shown a
significant end-of-treatment response rate when given in combination with
PEGASYS(R) (peginterferon alfa-2a) and COPEGUS(R) (ribavirin). R1626 also
shows a high barrier to the development of resistance.


After four weeks of treatment with this triple combination, followed by
44 weeks of PEGASYS and COPEGUS, levels of HCV were undetectable in 84
percent of patients infected with genotype 1 virus. This was higher than
patients treated with PEGASYS and COPEGUS alone for the entire 48-week
treatment period (65 percent). These new data were presented in a
late-breaker oral session at the 43rd Annual Meeting of the European
Association for the Study of the Liver (EASL), being held in Milan, Italy.



R1626 was discovered and developed at Roche and belongs to a class of
antivirals called polymerase inhibitors, which are being investigated with
the current standard of care for hepatitis C combination therapy with
pegylated interferon and ribavirin.



"These results demonstrate that R1626 holds significant promise to
potentially increase the number of hepatitis C patients who can be
successfully treated," said Dr. David Nelson, Director of Hepatology and
Liver Transplantation at the University of Florida, Gainesville, Florida.
"Since most patients responded very early in treatment with R1626, we
expect sustained virological response (SVR) rates that improve
significantly on those achieved with the current standard of care. I look
forward to SVR data from this Phase IIa study, and to results of the
ongoing Phase IIb study."



Patients in this Phase IIa study will be followed for an additional 24
weeks with no treatment to determine the SVR rate, which indicates
successful treatment.



More About the Phase IIa Study and End-of-Treatment Results



The Phase IIa study is a multicenter trial that enrolled 104 patients
with genotype 1 HCV, who had not previously received treatment. Its primary
endpoint was to evaluate the four-week efficacy and safety of combining
R1626 with PEGASYS alone or with PEGASYS plus COPEGUS, in comparison to a
current HCV standard of care (SOC), pegylated interferon plus ribavirin.



Patients were randomized into the following treatment groups:



-- Group A: R1626 1,500 mg twice a day plus PEGASYS 180 mcg weekly for
four weeks



-- Group B: R1626 3,000 mg twice a day plus PEGASYS 180 mcg weekly for
four weeks



-- Group C: R1626 1,500 mg twice a day plus PEGASYS 180 mcg weekly plus
COPEGUS 1,000/1,200 mg daily for four weeks
















-- Group D (standard of care group): PEGASYS 180 mcg weekly plus COPEGUS
1,000/1,200 mg daily for four weeks



Following the four weeks of treatment in this study, all patients
received PEGASYS 180 mcg weekly plus COPEGUS 1,000/1,200 mg daily for an
additional 44 weeks to complete the 48-week trial.



The study found:



-- Data collected at four weeks showed that patients receiving the triple
combination (Group C) had a mean decrease in viral load of 5.2 log10
from baseline, indicating a robust and rapid virological response.



-- At week 48, HCV was undetectable in 84 percent of patients (26 of 31)
who received the triple combination of R1626 1,500 mg BID + PEGASYS +
COPEGUS compared with 65 percent of patients (13 of 20) treated with
the SOC.



-- A higher incidence of grade four neutropenia was reported in the R1626
treatment arms during the four-week treatment period; however, after
stopping treatment with R1626, absolute neutrophil counts returned to
the level typically seen with patients receiving the SOC alone.



R1626 - A High Barrier to the Development of Resistance



In a separate oral presentation at EASL, it was reported that R1626
continues to present a high barrier to the development of viral resistance.
This is a serious concern emerging in the development of small molecule
treatments for hepatitis C. Resistance to R1626 has not been yet been
identified, after either two weeks of R1626 monotherapy, or after four
weeks in patients treated with R1626 in combination therapy.



Rapid Development of R1626 - A Large Phase IIb Study is Now Fully
Enrolled



A large Phase IIb study with R1626 was initiated in November 2007 to
define the optimal dose of R1626, in combination with PEGASYS and COPEGUS.
This Phase IIb trial, called POLI 1, is now fully enrolled with
approximately 500 patients, all with genotype 1 hepatitis C.



About Hepatitis C



Hepatitis C is a blood-borne infectious disease of the liver and a
leading cause of cirrhosis, liver cancer and the need for liver
transplants. According to the Centers for Disease Control and Prevention
(CDC), an estimated 4.1 million Americans (1.6 percent) have been infected
with hepatitis C; 3.2 million are chronically infected. The number of new
infections per year has declined from an average of 240,000 in the 1980s to
about 26,000 in 2004. CDC estimates the number of hepatitis C-related
deaths could increase to 38,000 annually by the year 2010, surpassing
annual HIV/AIDS deaths.



About PEGASYS



PEGASYS, in combination with COPEGUS (ribavirin), are indicated for the
treatment of adults with chronic hepatitis C who have compensated liver
disease and have not previously been treated with interferon alpha.
Efficacy has been demonstrated in patients with compensated liver disease
and histological evidence of cirrhosis (Child-Pugh class A) and patients
with HIV disease that are clinically stable (e.g., antiretroviral therapy
not required or receiving stable antiretroviral therapy). In addition,
PEGASYS in combination with COPEGUS is the first and only FDA-approved
regimen for the treatment of chronic hepatitis C in patients coinfected
with hepatitis C and HIV. PEGASYS is the only pegylated interferon
indicated for the treatment of adult patients with chronic hepatitis B
(HBeAg positive and HBeAg negative chronic hepatitis B who have compensated
liver disease and evidence of viral replication and liver inflammation).



PEGASYS is dosed at 180mcg as a subcutaneous injection taken once a
week. COPEGUS is available as a 200mg tablet, and is administered orally
two times a day as a split dose. Roche has backed PEGASYS with the most
extensive clinical research program ever undertaken in hepatitis C, with
major studies initiated to advance treatment for hepatitis C patients with
unmet needs, including patients co-infected with HIV and HCV, African
Americans, patients with cirrhosis, and patients who have failed to respond
to previous therapy.



IMPORTANT SAFETY INFORMATION



PEGASYS, alone or in combination with COPEGUS, is indicated for the
treatment of adults with chronic hepatitis C virus infection who have
compensated liver disease and have not been previously treated with
interferon alpha. Patients in whom efficacy was demonstrated included
patients with compensated liver disease and histological evidence of
cirrhosis (Child-Pugh class A).



Alpha interferons, including PEGASYS(R) (Peginterferon alfa-2a), may
cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune,
ischemic, and infectious disorders. Patients should be monitored closely
with periodic clinical and laboratory evaluations. Therapy should be
withdrawn in patients with persistently severe or worsening signs or
symptoms of these conditions. In many, but not all cases, these disorders
resolve after stopping PEGASYS therapy (see CONTRAINDICATIONS, WARNINGS,
PRECAUTIONS and ADVERSE REACTIONS in complete product information).



Use with Ribavirin. Ribavirin, including COPEGUS(R), may cause birth
defects and/or death of the fetus. Extreme care must be taken to avoid
pregnancy in female patients and in female partners of male patients.
Ribavirin causes hemolytic anemia. The anemia associated with ribavirin
therapy may result in a worsening of cardiac disease. Ribavirin is
genotoxic and mutagenic and should be considered a potential carcinogen
(see CONTRAINDICATIONS, WARNINGS, PRECAUTIONS and ADVERSE REACTIONS in
complete product information).



PEGASYS is contraindicated in patients with hypersensitivity to PEGASYS
or any of its components, autoimmune hepatitis, and hepatic decompensation
(Child-Pugh score greater than 6; class B and C) in cirrhotic CHC
monoinfected patients before or during treatment. PEGASYS is also
contraindicated in hepatic decompensation with Child-Pugh score greater
than or equal to 6 in cirrhotic CHC patients coinfected with HIV before or
during treatment. PEGASYS is also contraindicated in neonates and infants
because it contains benzyl alcohol. Benzyl alcohol is associated with an
increased incidence of neurological and other complications in neonates and
infants, which are sometimes fatal. PEGASYS and COPEGUS therapy is
additionally contraindicated in patients with a hypersensitivity to COPEGUS
or any of its components, in women who are pregnant, men whose female
partners are pregnant, and patients with hemoglobinopathies (eg,
thalassemia major, sickle-cell anemia).



COPEGUS THERAPY SHOULD NOT BE STARTED UNLESS A REPORT OF A NEGATIVE
PREGNANCY TEST HAS BEEN OBTAINED IMMEDIATELY PRIOR TO INITIATION OF
THERAPY. Women of childbearing potential and men must use two forms of
effective contraception during treatment and during the 6 months after
treatment has concluded. Routine monthly pregnancy tests must be performed
during this time. If pregnancy should occur during treatment or during 6
months post-therapy, the patient must be advised of the significant
teratogenic risk of COPEGUS therapy to the fetus.



Chronic hepatitis C (CHC) patients with cirrhosis may be at risk of
hepatic decompensation and death when treated with alpha interferons,
including PEGASYS. During treatment, patients' clinical status and hepatic
function should be closely monitored, and PEGASYS treatment should be
immediately discontinued if decompensation (Child-Pugh score greater than
or equal to 6) is observed. Ischemic and hemorrhagic cerebrovascular events
have been observed in patients treated with interferon alfa-based
therapies, including PEGASYS. Events occurred in patients with few or no
reported risk factors for stroke, including patients less than 45 years of
age. Because these are spontaneous reports, estimates of frequency cannot
be made and causal relationship between interferon alfa-based therapies and
these events is difficult to establish.



The most common adverse events reported for PEGASYS and COPEGUS
combination therapy observed in clinical trials were fatigue/asthenia
(65%), headache (43%), pyrexia (41%), myalgia (40%),
irritability/anxiety/nervousness (33%), insomnia (30%), alopecia (28%),
neutropenia (27%), nausea/vomiting (25%), rigors (25%), anorexia (24%),
injection site reaction (23%), arthralgia (22%), depression (20%), pruritus
(19%) and dermatitis (16%).



Serious adverse events in hepatitis C trials included neuropsychiatric
disorders (homicidal ideation, suicidal ideation, suicide attempt, suicide,
psychotic disorder and hallucinations), serious and severe bacterial
infections (sepsis), bone marrow toxicity (cytopenia and rarely, aplastic
anemia), cardiovascular disorders (hypertension, supraventricular
arrhythmias and myocardial infarction), hypersensitivity (including
anaphylaxis), endocrine disorders (including thyroid disorders and diabetes
mellitus), autoimmune disorders (including idiopathic thrombocytopenic
purpura, thrombotic thrombocytopenic purpura, psoriasis, lupus, rheumatoid
arthritis and interstitial nephritis), pulmonary disorders (dyspnea,
pneumonia, bronchiolitis obliterans, interstitial pneumonitis and
sarcoidosis), colitis (ulcerative and hemorrhagic/ischemic colitis),
pancreatitis, and ophthalmologic disorders (decrease or loss of vision,
retinopathy including macular edema and retinal thrombosis/hemorrhages,
optic neuritis and papilledema). Adverse reactions reported during
post-approval use of PEGASYS therapy, with and without ribavirin, include
hearing impairment, hearing loss, serious skin reactions, including
erythema multiforme major, and infections (bacterial, viral and fungal).

NanoBio Corporation Presents Preclinical And Interim Clinical Data On Topical Treatments For Herpes Labialis And Onychomycosis At AAD 2008

NanoBio Corporation is
presenting safety and efficacy results on new topical treatments for herpes
labialis (cold sores) and onychomycosis (toe nail fungus) at the annual
meeting of the American Academy of Dermatology (AAD), being held February
1-5, 2008, in San Antonio.


The data being presented indicate that the company's topical lotions,
composed of nanometer-sized droplets, have potent antiviral and antifungal
effects without safety concerns or systemic absorption. Interim findings in
subjects with cold sores demonstrate faster healing without skin irritation
or safety concerns. Initial results in subjects with toe nail fungus
indicate an excellent safety profile with no systemic absorption coupled
with an ability to kill the fungi that cause onychomycosis, a common
condition affecting up to 36 million people.



"We are very encouraged by our preliminary results," said James R.
Baker Jr., M.D., founder and chairman of NanoBio Corporation. "Our human
and animal studies show that NanoBio's products are well tolerated and
without safety issues, adverse events or skin irritation concerns.
Pharmacokinetic studies show no systemic absorption, a factor that enhances
the safety profile."



Final results from a prior phase 2A study in herpes labialis, as well
as interim findings from an ongoing phase 2B study, indicate a significant
improvement in time to healing for subjects treated with NanoBio's
antiviral product, NB-001.



Results from a phase 1 study in onychomycosis, as well as preclinical
data being presented at the meeting, indicate that NanoBio's antifungal
product, NB-002, can be safely applied to the skin at doses more than 1,000
times higher than the minimum concentration required to kill fungi.



"NB-002 exerts its effect locally by penetrating the skin and diffusing
laterally underneath the nail plate to reach the dermatophytes and kill
both active hyphae and spores -- without irritating the epithelium or being
systemically absorbed," Baker said. "The absence of systemic involvement
could provide a significant advance for onychomycosis therapy in terms of
safety." Current oral (systemic) drugs for onychomycosis pose risks of
hepatic and cardiac toxicity, as well as the potential for drug
interactions, Baker added.



In addition to its safety and tolerability, NB-002 activity was
documented against numerous fungi involved in cutaneous infections,
including Candida albicans, Microsporum canis, Epidermophyton floccosum,
Trichophyton mentagrophytes and Trichophyton rubrum.



NanoBio is currently conducting a double-blind, placebo-controlled,
phase 2 study to further investigate the efficacy and safety of NB-002 in
443 subjects with onychomycosis. All subjects have been enrolled, and the
study is expected to be completed in February 2009.



The company's topical treatments are based on a novel anti-infective
platform comprising an oil-in-water emulsion and a surfactant. The
nanodroplets readily penetrate hair follicles and skin pores to reach the
site of infection without disrupting skin or mucous membranes. Upon contact
with the pathogen, the nanodroplets directly interact with the pathogen's
outer membranes to lyse the virus, fungus or bacteria.


Poster Titles -- Link to posters at nanobio



-- Safety, Tolerance, Pharmacokinetics, and Efficacy of Topical
Nanoemulsion NB-001 for the Treatment of Herpes Labialis



-- Development of a Novel Antiviral Drug (NB-001) for Topical Application
in Humans



-- Safety, Tolerance, and Pharmacokinetics of Topical Nanoemulsion (NB-
002) for the Treatment of Onychomycosis



-- Development of a Novel Antifungal Drug (NB-002) for Topical Application
in Humans




About NanoBio



NanoBio(R) Corporation is a privately held biopharmaceutical company
focused on developing and commercializing anti-infective products and
mucosal vaccines derived from its patented NanoStat(TM) technology
platform. The company's lead product candidates are treatments for herpes
labialis (cold sores), onychomycosis (nail fungus), MRSA and mucosal
vaccines for influenza and hepatitis B. The company's headquarters and
laboratory facilities are located in Ann Arbor, Mich.


NanoBio Corporation

nanobio

Repros' IND For The Commencement Of Phase III Studies Of Proellex(R) In The Treatment Of Anemia Associated With Uterine Fibroids Is Now Effective

Repros Therapeutics Inc. (NasdaqGM:RPRX) announced that the Company's Investigational New Drug application for the study of Proellex® in the treatment of anemia associated with uterine fibroids has become effective. The Company will open the IND with two identical registration quality Phase III studies. The work has been awarded to an appropriate CRO.


This IND is based on impressive clinical findings from a subset of anemic patients in a Phase IIb trial of Proellex in the treatment of uterine fibroid symptoms we completed last year. In that study, women with hemoglobin levels of less than 11.5 g/dl improved by up to 2 g/dl, or the equivalent of two pints of blood, after three months of treatment with Proellex. Even though only approximately 15-17 patients per active treatment arm were evaluable for anemia, the improvement of hemoglobin in Proellex-treated subjects was both clinically and statistically highly significant (p < 0.002) compared with placebo. Oral iron supplements for correction of anemia were used by a similar number of women in the placebo and 25 mg Proellex-treated subjects. A slightly higher percentage of women in the Proellex 12.5 mg group used oral iron supplements. This suggests that reversal of the anemia with Proellex treatment was largely due to the very effective prevention of severe blood loss experienced by these patients with symptomatic uterine fibroids.


One of the soon to start Phase III studies will be conducted in 15-20 sites in the US and the second study in several sites ex-US. The trials will consist of three parallel arms each consisting of placebo, 25 and 50 mg Proellex. Each study will enroll 65 patients unevenly distributed between placebo (15 patients) and 25 and 50 mg Proellex (25 patients each). Patients will be treated with Proellex or placebo for three months.


The primary endpoint of the study is improvement in hemoglobin concentration. The study is powered at an 85% level to achieve a significance of less than 0.05 to detect a difference of 1.01 g/dl of hemoglobin comparing 50 mg Proellex to placebo. Our previous study achieved a 2 g/dl hemoglobin difference between placebo and Proellex which suggests that this study is adequately powered to successfully meet its primary end point. All patients will receive a fixed dose of daily iron supplementation as well.


Joseph Podolski, President and CEO commented, "We are pleased with the progress we are making with Proellex. We will make every effort to rapidly enroll subjects into these registration studies. At the same time we will diligently complete the other ancillary work the FDA will require before we can submit our first Proellex NDA. Though our goal for NDA submission is aggressive we will make every effort to submit the dossier around year-end 2008." He further added, "We look forward to sharing what we believe will be exciting Proellex developments with our shareholders this year."















Additionally, Repros management has awarded contracts for the pivotal registration studies of Proellex for the indication of chronic treatment of symptomatic uterine fibroids, as well as the large open label safety studies necessary to support chronic use of the drug. These protocols will be submitted to the FDA for comment before initiation.


About Repros Therapeutics Inc.


Repros Therapeutics focuses on the development of oral small molecule drugs for major unmet medical needs. We have a proven track-record of efficient and rapid advancement of our therapeutic candidates through clinical development.


Our lead drug, Proellex®, is a selective blocker of the progesterone receptor and is targeted for the treatment of uterine fibroids, endometriosis and uterine fibroid induced anemia. We expect to initiate registration Phase 3 trials in the first quarter of 2008 with Proellex for the short-term treatment of uterine fibroid induced anemia and for the chronic treatment of uterine fibroids. Uterine fibroids, endometriosis and uterine fibroid induced anemia affect a significant number of women of childbearing age in the developed world. There is no currently-approved effective long-term drug treatment for uterine fibroids or endometriosis. In the United States alone, 300,000 women per year undergo a hysterectomy as a result of severe uterine fibroids. We have estimated the market opportunity addressed by Proellex to be in excess of $3 billion annually.


Our second drug, Androxal®, is a single isomer of clomiphene citrate. Androxal restores normal pituitary response in men resulting in normalization of testosterone and luteinizing hormone ("LH") levels. Androxal is entering two Phase 2b clinical trials in Q2, 2008. One of these trials will be in men with adult-onset idiopathic hypogonadism ("AIHH") with concomitant glycemic and lipid elevations ("Metabolic Syndrome"). Recent published studies in older men show a link of low testosterone with higher incidences of Metabolic Syndrome, diabetes and mortality rates. Based on a retrospective review of a recently completed six-month clinical trial of Androxal, our findings showed that Androxal therapy resulted in a significant reduction in mean glucose levels in men with a body mass index (BMI) >26 and glucose levels >99, an outcome not seen in the placebo or Androgel arms of this study. The second Phase 2b Androxal trial to begin in 2008 will be in men of reproductive age with low testosterone levels who want to improve or maintain fertility/sperm status. We believe Androxal will be superior to the existing drugs used to normalize testosterone as only Androxal has the property of restoring both LH and follicle stimulating hormone (FSH) levels. FSH is the pituitary hormone that stimulates testicular sperm production. It is estimated that 13 million men in the United States are testosterone deficient.


Any statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including Repros' ability to have success in the clinical development of its technologies, the timing of enrollment and release of data in such clinical studies and the accuracy of such studies, limited patient populations of clinical studies to date and the possibility that final data may not be consistent with interim data, Repros' ability to raise additional capital in a timely manner and on acceptable terms or at all and such other risks which are identified in the Company's Annual Report on Form 10-K for the year ended December 31, 2006 and Repros' Quarterly Reports on Form 10-Q for the quarters ended March 31, June 30, and September 30, 2007, as they may be updated by the Company's Exchange Act filings from time to time. These documents are available on request from Repros Therapeutics or at sec. Repros disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.


For more information, please visit the Company's website at reprosrx.




View drug information on AndroGel testosterone gel.

Data Published In Muscle And Nerve Validates The Six-Minute Walk Test As An Outcome Measure In Duchenne Muscular Dystrophy

Data published online in the medical journal Muscle and Nerve support the use of the six-minute walk test (6MWT) as an outcome measure in PTC Therapeutics, Inc.'s ongoing registration-directed clinical trial of ataluren in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDMD/BMD). The study results, which will also be published in an upcoming print issue of the journal, characterize the limitations on walking by patients with DMD relative to healthy boys and indicate that young boys can consistently and reliably perform the test. The data were obtained from an observational study conducted at the University of California Davis which was sponsored by PTC and supported by a grant from Parent Project Muscular Dystrophy (PPMD).


The 6MWT is an accepted and standardized measure of muscle, lung, and heart health in patients with other medical conditions. However, the test had not previously been used to evaluate boys with DMD. The observational study was designed to confirm that boys with DMD would have the stamina and focus required to successfully and consistently complete the test without injury.


"Advancing the validation of clinically meaningful endpoints for therapeutic and natural history studies in DMD has been a focus of the Duchenne clinical and advocacy communities for many years," said Craig McDonald, M.D., principal investigator of the observational study and director of the Rehabilitation Research and Training Center in Neuromuscular Diseases, University of California Davis. "The publication of these findings demonstrates that the 6MWT can serve as a clinically meaningful outcome measure for documenting the evolution of the disease and the treatment benefits of new therapies for DMD. The new test has rapidly become the favored primary outcome measure worldwide in clinical trials in ambulatory boys with Duchenne muscular dystrophy. This is an exciting moment for all of us involved in the care of boys with this progressively disabling condition."


The study was designed to evaluate the feasibility, safety and reproducibility of the 6MWT, comparing boys with DMD to healthy boys of the same age. The study evaluated 21 ambulatory boys with DMD and 34 healthy boys, ages four to 12 years old. Boys with DMD were tested twice, one week apart and healthy boys were tested once. This study demonstrated that ambulatory young boys can complete the 6MWT safely and consistently.


"PPMD is proud to have sponsored research that sets the foundation for developing drugs in DMD. The validation of the 6MWT as a clinical endpoint has significant implications for the DMD community," stated Patricia Furlong, Founding President & CEO of Parent Project Muscular Dystrophy. "The ability to study the efficacy of potential DMD treatments will facilitate the advancement of new therapies and ultimately benefit patients. An important part of what we do at PPMD is to help establish universal standards for boys and young men with DMD. It is particularly gratifying to have a validated clinical endpoint to expand the development of therapeutic options."















The study found that boys with DMD walked profoundly shorter distances in six minutes than healthy boys due to the muscular deficits that are characteristic of DMD, including markedly reduced stride length. The findings also suggest that the 6MWT is highly reproducible, as evidenced by a high degree of correlation (r=0.91) between the first and second test in boys with DMD. These findings directly support the use of the 6MWT in PTC's ongoing Phase 2b, double-blind, randomized trial of ataluren in boys with nmDMD/BMD, for which the 6MWT is the primary outcome measure. The trial is designed to determine whether ataluren taken orally can improve walking, muscle function and strength in patients with nmDMD/BMD and will also evaluate ataluren's long-term safety profile.


"We are encouraged that pretreatment data from 174 boys participating in our pivotal Phase 2b study of ataluren are consistent with the observational data and show that the 6MWT is safe and reproducible when performed repeatedly at multiple trial sites in many different countries," said Langdon Miller, M.D., Chief Medical Officer of PTC. "We look forward to sharing the data from the ataluren clinical trial in nmDMD/BMD patients in early 2010."


About DMD/BMD


Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle disorders that result in the loss of both muscle function and independence. Patients with DMD/BMD have increasing problems with walking as the disease progresses and eventually must depend upon assistive devices to maintain mobility. DMD/BMD is perhaps the most prevalent form of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children). It is estimated that one in ten DMD patients is likely to have a Becker presentation, a milder form of the disease that is associated with later manifestation of symptoms. Further information regarding DMD and BMD is available through the Muscular Dystrophy Association and Parent Project Muscular Dystrophy.


About Ataluren (PTC124®)


Ataluren is the first investigational new drug designed to restore the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nmDMD/BMD, nonsense mutation cystic fibrosis (nmCF) and nonsense mutation hemophilia A and B (nmHA/HB).


Ataluren has been granted orphan drug status for the treatment of nmCF and nmDMD/BMD by the U.S. Food and Drug Administration (FDA) and the European Commission. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing. The development of ataluren has been supported by the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, and the National Center for Research Resources.


Collaboration With Genzyme


PTC Therapeutics has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren. PTC will commercialize ataluren in the United States and Canada, while Genzyme will commercialize the product in other regions of the world.


About PTC Therapeutics


PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and that are the basis for collaborations with leading biopharmaceutical companies such as Celgene, Genzyme, Gilead, Merck, Pfizer and Roche.

Nitec Pharma Reports Positive And Highly Significant Phase III Results From Capra-2 Study Of Lodotra(TM) In RA

Nitec Pharma AG ("Nitec" or "Nitec Pharma"), a Switzerland-based specialty pharma company focused on the development and commercialization of medicines to treat chronic inflammation and pain-related diseases, announced positive results from the second pivotal phase III trial for its lead product, Lodotra(TM).


The CAPRA-2 study (Circadian Administration of Prednisone in Rheumatoid Arthritis-2) was a 12-week, multicentre, double-blind phase III trial evaluating the safety and efficacy of Lodotra(TM) for the treatment of rheumatoid arthritis ("RA"), a chronic, progressive and disabling autoimmune disorder. In total 350 patients, all inadequate responders to DMARD therapy, were randomised in one of two arms to receive either Lodotra(TM) (5mg once daily), or placebo in addition to their existing therapy. The primary efficacy endpoint was the ACR-20 response rate, which is defined as at least a 20% improvement in a number of disease-specific criteria. The key secondary endpoint was the change in the duration of morning stiffness of the joints. CAPRA-2-data will be used to file for US marketing approval with the FDA together with the data already available from the CAPRA-1 pivotal phase III trial which has shown the superiority of Lodotra(TM) over standard prednisone treatment.


Lodotra(TM)-treated patients showed an ACR-20 response of 49% compared to 29% in the placebo group. The difference was highly significant (p=0.0002; LOCF). The reduction of morning stiffness was 44% in the Lodotra(TM) group (21 % in the placebo group). This difference was also highly significant (p=0.0008). Lodotra(TM) was safe and well tolerated. The number of adverse events was low and comparable in both groups.


Dr. Anders Harfstrand, CEO of Nitec commented: "We are delighted to report successful and very positive outcomes of the CAPRA-2 study. These strongly underline the benefits of Lodotra(TM) in the treatment of RA. The study confirms clearly that the innovative delivery system of Lodotra(TM) is able to adapt the timing of glucocorticoid treatment to a patient's circadian rhythm in order to improve the efficacy and safety of prednisone. CAPRA-2 is the first pivotal phase III study to demonstrate the ACR-20 response of a very low dose of prednisone as a primary endpoint. Following the successful European launches of Lodotra(TM) and the positive feedback that we have received from RA specialists, we look forward to completing the regulatory process in the US."


LodotraTM, Nitec's novel single-pulse delayed-release ("SPDR") low-dose prednisone tablet, was launched in Germany by Merck KGaA, which holds the exclusive distribution rights for Germany and Austria. In April 2009 Mundipharma acquired the distribution rights to LodotraTM in the rest of Europe and Nitec retains all commercialization rights for the US and rest of the world.


About Nitec Pharma AG


Nitec Pharma is a Switzerland-based specialty pharmaceutical company focused on the development and commercialisation of innovative medicines and effective treatment solutions for chronic inflammation and pain-related diseases. Nitec's most advanced product is Lodotra(TM), a circadian cytokine modulator (CCM) for the treatment of rheumatoid arthritis (RA), which has received a recommendation for European regulatory approval and is launched in Germany. Nitec was originally founded in 2004 as a spin-out of Merck KGaA and is headquartered in Reinach in Switzerland. Nitec is financed by Atlas Venture, Global Life Science Ventures, NGN Capital, TVM Capital and a principal investment arm of Deutsche Bank AG, London.



About Lodotra(TM)


Lodotra(TM) is a circadian cytokine modulator (CCM), which can be taken at bedtime. Lodotra(TM)'s unique release mechanism releases the glucocorticoid prednisone during the night around 2am enabling suppression of the nocturnal proinflammatory cytokines. This results in an effective relief of the early morning symptoms of RA, in addition to the well established treatment effects of glucocorticoids.

Bayer Presents Positive Phase II Data With Florbetaben Potential To Visualize Beta-Amyloid Plaques In Patients With Alzheimer's Disease

Bayer Schering Pharma
AG, Germany, has presented positive data on a global Phase II study with
the novel positron emission tomography (PET) tracer florbetaben (BAY
94-9172) at the International Conference on Alzheimer's Disease (ICAD) in
Vienna, Austria. This study showed that patients with a clinical diagnosis
of Alzheimer??s disease could be differentiated from age-matched healthy
volunteers (HVs) on the basis of florbetaben uptake pattern in the brain.



Until now, the clinical diagnosis of Alzheimer??s disease (AD) with current
methods such as cognitive tests is still limited. Currently, a definite
diagnosis of Alzheimer??s disease is only possible post mortem. The results
of this study showed PET images with a high specificity of over 90 percent:
More than 90 percent of the HVs had a negative florbetaben scan (i.e. no
tracer uptake) in the relevant brain regions. The results also show a
sensitivity of approximately 80 percent indicated by the clinical
diagnosis, meaning that about 80 percent of the clinically suspected
Alzheimer patients had positive florbetaben scans indicating the presence
of beta-Amyloid plaques. This is in line with the results of studies
comparing the clinical diagnosis with the definite post mortem
histopathological diagnosis.



"The data underline the potential of florbetaben as an important visual
adjunct to existing clinical parameters in the diagnosis of dementia," said
Kemal Malik, MD, member of the Board of Management and Head of Global
Development at Bayer Schering Pharma AG. "There is a high medical need for
an easy, non-invasive imaging technique that supports an early and precise
diagnosis of dementia and especially of Alzheimer's disease, allowing for
optimized care and treatment options." With the development of florbetaben,
Bayer Schering Pharma wants to contribute to diagnose AD in the future more
precisely and at an earlier time during the course of the disease.



In the blinded read of the images a high interreader agreement rate between
three independent readers was observed. This indicates that the scans with
the beta-Amyloid targeted PET tracer florbetaben are easy to visually
assess and that this assessment is highly reproducible.



Additional Phase II and pivotal Phase III global studies are under
preparation to validate the potential shown by florbetaben in this Phase II
setting. Start of the Phase III program is planned for end of 2009.



About the Phase II Study


This global Phase II, open-label, non randomized, multi-center study aims
to evaluate the efficacy of florbetaben PET in vivo imaging in the
detection/exclusion of cerebral beta-Amyloid plaques in patients with
mild-to-moderate, probable AD (older than 55 years of age) compared with
age-matched healthy volunteers. A total of 18 study centers in four
countries (Australia, Germany, USA, and Switzerland) screened 213
individuals of whom 150 individuals were imaged with florbetaben receiving
a single intravenous injection of the tracer. Reliable, high-quality images
were obtained across multiple centers and camera types over a long time
span. A 20-minute scan in this study provided optimal image quality.
















The florbetaben uptake pattern was visually assessed for all time points by
three independent, nuclear medicine physicians blinded to the clinical
diagnosis and all other clinical data.



The co-primary efficacy variables were the sensitivity and specificity of
the independent, blinded visual assessment of the images. As a secondary
endpoint, the standard uptake value ratios (SUVRs) were also calculated
using the cerebellar cortex as a reference region. The clinical diagnosis
of AD served as the standard of truth, and was performed by the referring
clinicians and based on internationally accepted and validated clinical
criteria. The HVs were also carefully examined to include only those with a
total lack of cognitive impairment. In addition, safety parameters were
evaluated and various technical parameters assessed to define the optimal
imaging procedure for further development (e.g. optimal imaging time
points).



About Florbetaben (18 F)


Florbetaben is an inlicensed 18F-labeled PET tracer that specifically binds
to beta-Amyloid plaques. These plaques consist of proteins that accumulate
in the brain and are a pathological hallmark of Alzheimer's disease. As the
aggregation of the beta-Amyloid protein in the brain is also a key target
for new therapeutic treatments under development, florbetaben might also be
able to support the development of these new treatment approaches.



About Alzheimer's Disease


Alzheimer's disease (AD) is a devastating neuro-degenerative disease and
the most common cause of dementia. Most cases of Alzheimer's disease affect
people over the age of 60. It is a progressive disease that can lead to
premature death. In 2006, estimates suggested that more than 26 million
people worldwide were suffering from Alzheimer's disease. By 2050, this
number could reach more than 100 million. At present there is no cure for
Alzheimer's disease, but treatments for symptoms, combined with the right
services and support, can make life better for the millions of people
living with Alzheimer's.



Source
Bayer HealthCare

First Implant Of DuraHeart(TM) Left Ventricular Assist System (LVAS) In Japanese Clinical Trial

Terumo Heart Inc. announced
that the first DuraHeart(TM) Left Ventricular Assist System (LVAS) was
implanted in October at the University of Tokyo Hospital in Japan. Terumo
Corporation will continue efforts toward an early application for approval
for manufacture and sales in Japan.



Terumo's LVAS is the world's first third generation left ventricular
assist system combining a centrifugal pump with a magnetically levitated
impeller. The product received the CE mark in Europe in 2007 and began its
U.S. Pivotal Trial in July 2008.



The "Mag-Lev" centrifugal pump technology was developed by Dr.
Akamatsu, former professor of the Faculty of Engineering, Kyoto University
in collaboration with NTN Corporation. The development base was moved to
the U.S. in 2000 to continue product realization and commercialization.



In Japan, the Ministry of Health, Labor and Welfare highlighted
implantable left ventricular assist devices in the "Council Regarding Early
Introduction of Medical Equipment with Medical Needs."



Terumo Heart, Inc. is a U.S. subsidiary of Terumo Corporation with
headquarters and manufacturing facilities in Ann Arbor, Michigan. The
company's focus is the innovation and introduction of products to improve
the quality of healthcare for heart failure patients. Terumo Corporation,
located in Tokyo, Japan, is a leading developer, manufacturer and global
marketer of a wide array of medical products. DuraHeart is limited to
investigational use only in the United States, and is CE marked in Europe.


Terumo Heart, Inc.

terumoheart

New Criteria For Measuring Tumour Size And Progression Will Help Ease Workloads In Clinical Trials

The first, formal revision of specific guidelines, known as RECIST, used by clinicians to measure tumour size and response to treatment, has been published today (Tuesday 20 January) in a special issue of the European Journal of Cancer (the official journal of ECCO - the European CanCer Organisation) [1]. The authors say that the revisions will ease the workload involved in running clinical trials, without compromising study outcomes.



RECIST (Response Evaluation Criteria in Solid Tumours) were first published in 2000 and are used by investigators in phase II and phase III clinical trials of new anti-cancer drugs as a way of measuring the efficacy of the treatment. Tumour shrinkage (objective response) and time to the development of disease progression are both important endpoints in trials, and, increasingly in recent years, trials have been using time to progression (or progression-free survival) as their main endpoint on which to base conclusions about the efficacy of a drug.



The new RECIST (RECIST 1.1 to distinguish them from the original RECIST) answer some of the questions and issues that have arisen since 2000 as a result of changing methodologies and available treatments.



Professor Jaap Verweij (Erasmus University Medical Centre, The Netherlands), the EJC's clinical oncology editor and a co-editor of the special issue on RECIST, said: "This special issue is very important for the cancer community as the updated criteria will affect all clinicians who are running clinical cancer trials. RECIST 1.1 describes a standard approach to solid tumour measurement and definitions for objective assessment of change in tumour size for use in adult clinical trials."



Co-editor, Professor Elizabeth Eisenhauer (National Cancer Institute of Cancer, Ontario, Canada), stressed that the goal of developing RECIST 1.1 was to improve the consistency and standardisation of trials. "Drug development and clinical cancer research is a global enterprise. The more consistent we are in describing what we have seen, and in using the same measures and endpoints, the more reliably we are able to interpret the results from a variety of sources," she said.



Key changes in RECIST 1.1 that will simplify, optimise and standardise the assessment of tumour burden in clinical trials are as follows:


- A reduction in the number of lesions to be assessed for response from a maximum of ten to five, and from five to a maximum of two per organ;


- New guidance on making robust measurements of lymph node involvement;


- Confirmation of response is required for trials with objective response as a primary endpoint, but is no longer required for randomised studies, since the control arm of these studies provides appropriate means for interpreting results of the experimental arm;















- The definition of disease progression has been refined so that it not only includes a 20% increase in the size of the lesion, but also a 5 mm absolute increase as well, so that changes of just a few mms in very small tumours, which may be within the range of measurement error, are not unnecessarily described as disease progression;


- Guidance on imaging, including its use in the detection of new lesions and the interpretation of FDG-PET scan assessment.


Prof Eisenhauer stressed that the recommendations are evidence-based: "They are grounded in the literature and where such information didn't exist - as was the case in some areas - we set about generating the evidence to guide and support the changes. The EORTC Data Centre, under the leadership of Jan Bogaerts and Patrick Therasse (co-authors of RECIST 1.1), undertook to create a warehouse of data from clinical trials of solid tumours from both industry and cooperative groups. We were able to test the recommendations on these data to look at the impact of proposed changes and decide whether the evidence supported the change or not."



The database consisted of over 6500 patients with more than 18000 target lesions, and was used to investigate the impact of a variety of questions, such as the number of lesions needing to be measured. "Our consequent recommendation that the number of lesions measured should be reduced from ten to five will have a big implication for the workload in clinical trials and we found that it will have no impact on described study outcomes," said Prof Eisenhauer. "This change can be made without any loss of information."



Professors Verweij, Eisenhauer and Therasse (who is also co-editor of the special issue and now based at GlaxoSmithKline Biologicals, Rixensart, Belgium) say that in the future it would be desirable to incorporate the use of modern functional imaging techniques in assessing benefit to treatment. "Although there are many exciting developments in this field, none of these techniques was considered as ready to substitute for anatomical endpoint assessment in clinical trials yet, since their use??¦.awaits validation in large datasets and their availability in trial centres is limited," they write in their introduction to the special issue. A discussion paper in the issue describes the process and the level of evidence that would be needed before functional imaging could be used in addition to, or instead of, anatomical imaging in clinical trials.



Professor Alexander M.M. Eggermont, president of ECCO (the European CanCer Organisation), welcomed the publication of RECIST 1.1. "This is an important and timely publication for all clinicians working in cancer clinical trials. I'm particularly pleased that some of the amendments will contribute to lessening the workload on trial investigators. This revision of the RECIST guidelines will enable researchers to work smarter and more effectively so that, ultimately, patients will benefit from improved treatments reaching them sooner."



[1] European Journal of Cancer, Vol 45, issue 2 (January 2009), pages 225-310. "Response assessment in solid tumours (RECIST): Version 1.1 and supporting papers."



The European Journal of Cancer is the official journal of ECCO - the European CanCer Organisation.



ECCO - the European CanCer Organisation - exists to uphold the right of all European cancer patients to the best possible treatment and care and to promote interaction between all organisations involved in cancer research, education, treatment and care at the European level.

ECCO - the European CanCer Organisation

Asthmatx Pivotal Trial Manuscript For Bronchial Thermoplasty Published In The American Journal Of Respiratory And Critical Care Medicine

Asthmatx Inc., developer of bronchial thermoplasty with the Alair® System, announced the publication of positive results from its pivotal study, the Asthma Intervention Research 2 (AIR2) Trial, in the American Journal of Respiratory and Critical Care Medicine (AJRCCM). The publication, entitled "Effectiveness and Safety of Bronchial Thermoplasty in the Treatment of Severe Asthma: A Multicenter, Randomized, Double-Blind, Sham-Controlled Clinical Trial," demonstrated statistically significant reductions in asthma attacks (severe exacerbations) and emergency room visits for respiratory symptoms, as well as improvements in quality of life in adults with severe asthma who underwent bronchial thermoplasty with the Alair System.


"Bronchial thermoplasty decreases severe exacerbations and ER visits for respiratory symptoms and improves quality of life in patients with severe asthma," said Mario Castro, MD, Professor of Medicine and Pediatrics at the Washington University School of Medicine, a principal investigator in the AIR2 Trial and the lead author of the paper. "Bronchial thermoplasty with the Alair System offers clinicians a novel adjunctive therapy option, beyond the current use of inhaled corticosteroids and long acting beta agonists, to provide improvements in overall asthma control."


The AIR2 Trial was designed to evaluate the safety and effectiveness of bronchial thermoplasty in adult patients with severe asthma who remain symptomatic, despite treatment with standard of care medications (high dose inhaled corticosteroids and long-acting beta agonists). The study was a randomized, double-blind, sham-controlled trial that enrolled 297 patients at 30 sites in six countries. The primary effectiveness endpoint was the change from baseline in Asthma Quality of Life Questionnaire (AQLQ) score. Safety was assessed by comparing the short and long-term (out to one year) safety profiles for both the active and sham treatment groups.


In addition to an improvement in quality of life as measured by an increase in average Asthma Quality of Life Questionnaire (AQLQ) score at 6, 9, and 12 months in the active compared to the sham group, there were also improvements in overall asthma control as demonstrated by the following key statistically significant clinical findings of the AIR2 Trial during long-term follow-up:



-- 32% reduction in asthma attacks



-- 84% reduction in emergency room visits for respiratory symptoms



-- 66% reduction in days lost from work/school or other activities due to asthma



-- 36% reduction in patients reporting worsening of asthma due to multiple symptoms


In the period immediately following bronchial thermoplasty, there was an expected increase and worsening of respiratory-related symptoms, which were of the type expected following bronchoscopy in patients with asthma. These events typically occurred within a day of the procedure and resolved on average within seven days with standard care. In the long term after treatment, fewer bronchial thermoplasty treated patients reported respiratory adverse events. The report in AJRCCM concluded that the increased risk of adverse events in the short-term following the procedure is outweighed by the benefit of bronchial thermoplasty that persists for at least one year.















In October of 2009, the Anesthesiology and Respiratory Therapy Devices Panel of the Medical Devices Advisory Committee to the U.S. Food and Drug Administration (FDA) voted to recommend that the Alair® System be found approvable with conditions based on the results of this and other studies. The application for FDA approval of the Alair System is presently under review by FDA.


About Bronchial Thermoplasty Delivered by the Alair System


Bronchial thermoplasty is a bronchoscopic procedure for the treatment of severe persistent asthma in patients 18 years and older whose asthma is not well controlled with inhaled corticosteroids and long acting beta agonists. Bronchial thermoplasty is performed through the working channel of a standard flexible bronchoscope that is introduced through a patient's nose or mouth, and into their lungs. The tip of the small diameter Alair® catheter is expanded to contact the walls of targeted airways. Controlled thermal energy is then delivered to the airway walls to reduce the presence of airway smooth muscle that narrows the airways in patients with asthma. The minimally invasive procedure, like many other flexible endoscopy procedures, is done under moderate sedation, and the patient returns home the same day.


CAUTION: Alair System is an Investigational Device. It is limited by United States law to investigational use. To be used by Qualified Investigators only.


Alair, Asthmatx and the Asthmatx logo are registered trademarks of Asthmatx, Inc.


About Asthmatx


Asthmatx is developing catheter-based medical devices for the treatment of asthma. Asthmatx has developed the Alair System to perform an investigational outpatient procedure called bronchial thermoplasty. The Alair System has received a CE Mark for use in the European Union.

Independent Advisory Committee Recommends Continuation Of Phase III Oral Calcitonin Studies For Osteoporosis And Osteoarthritis

Unigene Laboratories, Inc. (OTCBB: UGNE) announced that an independent Data Monitoring Committee (DMC) has recommended that Novartis and its partner Nordic Bioscience proceed as planned with their ongoing oral calcitonin Phase III studies for osteoporosis and osteoarthritis.


Its recommendation is based on the committee's recently completed "futility" analysis of the data obtained from all patients enrolled for at least twelve months in these studies. That analysis included an assessment of both safety and efficacy parameters. It is the committee's opinion that there are no major or unexpected safety concerns and it unanimously recommends to proceed with the studies to evaluate the efficacy and safety profile of oral calcitonin as planned.


"The report of the DMC is an important benchmark in the conduct of these trials and it provides the first preliminary indication of the drug's performance," commented Dr. Ronald S. Levy, Executive Vice President of Unigene Laboratories, Inc. "Unigene has a vested interest in the success of these studies both from a milestone and a royalty perspective, and we are encouraged by this committee's recommendation."


Source

Unigene Laboratories, Inc.

Cancer Cell Division And Brain Tumor Progression Slowed In Electric Field Experiments

Low-intensity electric fields can disrupt the division of cancer cells and slow the growth of brain tumors, suggest laboratory experiments and a small human trial, raising hopes that electric fields will become a new weapon for stalling the progression of cancer. The research, performed by an international team led by Yoram Palti of the Technion-Israel Institute of Technology in Haifa, is explained in the August issue of Physics Today, the flagship magazine of the American Institute of Physics.



In the studies, the research team uses alternating electric fields that jiggle electrically charged particles in cells back and forth hundreds of thousands of times per second. The electric fields have an intensity of only one or two volts per centimeter. Such low-intensity alternating electric fields were once believed to do nothing significant other than heat cells. However, in several years' worth of experiments, the researchers have shown that the fields disrupt cell division in tumor cells placed on a glass dish (in vitro).



After intensively studying this effect in vitro and in laboratory animals, the researchers started a small human clinical trial to test its cancer-fighting ability. The technique was applied to ten human patients with recurrent glioblastoma multiforme (GBM), a form of brain cancer with a very low survival rate. All the patients had their earlier tumors treated by other methods, but the cancer had started to recur in all cases. Fitting the patients with electrodes that applied 200 kHz electric fields to the scalp at regular intervals for up to 18 hours per day, the researchers observed that the brain tumors progressed to advanced stages much slower than usual (taking a median time of 26 weeks), and sometimes even regressed. The patients also lived considerably longer, with a median survival time of 62 weeks. While no control group existed, the results compared favorably to historical data for recurrent GBM, in which the time for tumor progression is approximately 10 weeks and the typical survival time is 30 weeks. In addition, 3 of the 10 patients were still alive two years after the electrode therapy started. These results were announced in a recent issue of The Proceedings of the National Academy of Sciences (Kirson et al., PNAS 104, 10152-10157, June 12, 2007).



The Physics Today article explains these results in terms of the physical mechanisms that enable the electric fields to affect dividing cancer cells. In vitro, the electric fields were seen to have two effects on the tumor cells.



First, they slowed down cell division. Cells that ordinarily took less than an hour to divide were still not completely divided after three hours of exposure to an electrical field of 200 kHz. Another group consisting of Luca Cucullo, Damir Janigro and their colleagues at the Cleveland Clinic, slowed cell division by applying electric fields with a much lower frequency just 50 Hz. In addition, this protocol demonstrated the ability to decrease the intrinsic drug resistance of the cells.
















What causes cell division to slow down" In the 200-kHz case, the electric fields hamper the formation and function of a key cell structure known as the mitotic spindle. The spindle is composed of cell components known as microtubules. The microtubules in turn contain components that have a high electric dipole moment, in which there is a large separation of opposite electric charges. Therefore, parts of the mitotic spindle are greatly influenced, and apparently disrupted, by an electric field.



The second effect of the 200 kHz fields is that they sometimes disintegrated the daughter cells just before they split off from their partners. The dividing cells sometimes destruct because a high-electric-field region develops between the two daughter cells. This leads to a large slope, or gradient, in the electric field from each daughter cell to this region. This gradient may rip organelles (cell structures) and macromolecules (such as proteins) from the scaffolding of the cells.



The alternating electric fields are believed to have similar effects in the human glioblastomas. In contrast, the electric-field treatment poses little danger to normal brain tissue, because healthy brain cells do not divide. The electric fields were only observed to have disruptive effects on dividing cells. Based on the success of their initial human study, the researchers are working on another human clinical trial, this time with a control group receiving chemotherapy. The researchers are also investigating the possibility of combining the electric-field therapy with low-dose chemotherapy.







The Physics Today article, appearing in the magazine's Search and Discovery section and written by editor Johanna Miller, is freely available online, with full text and pictures, at ptonline.aip/journals/doc/PHTOAD-ft/vol_60/iss_8/19_1.shtml



The American Institute of Physics (AIP) is a not-for-profit membership corporation created for the purpose of promoting the advancement and diffusion of the knowledge of physics and its application to human welfare.

Long-Term Study Suggests Parkinson's Drug Rasagiline May Slow Progression Of Movement Disorder

There is hope that the drug rasagiline can do what no other medication for Parkinson's disease now does -- slow the progression of a devastating degenerative brain disease that eventually robs people of their ability to move and function.



Now a new study looking at the long-term effects of rasagiline (Azilect) on newly diagnosed patients indicates that people who began the drug earlier continued to do better than those for whom treatment was delayed six months. The study "Long-term Outcome of Early Versus Delayed Rasagiline Treatment in Early Parkinson's Disease" was recently published in the early online version of the journal Movement Disorders.



"Patients who received rasagiline right from the beginning rather than after a six-month delay experienced less progression of the clinical signs and symptoms of Parkinson's disease that interfere with activities of daily living such as eating, walking and dressing," said the study's lead author Robert A. Hauser, MD, director of the University of South Florida Parkinson's Disease and Movement Disorders Center. "This is potentially consistent with a slowing of underlying disease progression, although other possible mechanisms also need to be considered."



The study, sponsored by Teva Pharmaceutical Industries Ltd. (Israel), Teva Neuroscience, Inc. (USA) and H. Lundbeck A/S (Denmark), was a long-term open label extension of the multisite trial "TVP-1012 (rasagiline) in Early Monotherapy for Parkinson's Disease Outpatients" study, known as TEMPO. In TEMPO, more than 400 untreated patients with early Parkinson's disease were randomly assigned to rasagiline for a year (1 mg daily or 2 mg daily) or to placebo for six months followed by rasagiline for six months (2 mg daily). At the end of a year, patients receiving rasagiline from the start fared better as measured by the Unified Parkinson's Disease Rating Scale. They experienced less worsening of motor symptoms, such as rigidity and tremor, and had fewer problems with activities of daily living than patients who began rasagiline six months later.



The open-label extension study followed more than 300 patients from the TEMPO study for up to 6.5 years. In this extension study, all patients continued on rasagiline (1 mg. daily) and could take other Parkinson's disease medications as needed. The researchers found those who started rasagiline right from the beginning of the TEMPO study continued to fare better than patients in the delayed-start group. Over the course of the entire study, the early-start group had 16 percent less progression of the signs and symptoms of Parkinson's disease, and this greater clinical benefit was observed even as patients received conventional Parkinson's disease medications in addition to rasagiline. Rasagiline appeared to be well tolerated in this long-term study.



If the clinical outcomes from the TEMPO and extension study hold up under further scrutiny, it may indicate that early initiation of rasagiline confers a protective effect against disease progression, Dr. Hauser said. "If this is the case, it reinforces the importance of individuals being diagnosed and treated as soon as possible."



The study authors point out that early initiation of any drug to relieve symptoms of Parkinson's disease may lead to a better clinical outcome compared to delayed administration -- something that will be elucidated as more delayed-start studies are performed with other Parkinson's medications.







USF Health is dedicated to creating a model of health care based on understanding the full spectrum of health. It includes the University of South Florida's colleges of medicine, nursing, and public health; the schools of biomedical sciences as well as physical therapy & rehabilitation sciences; and the USF Physicians Group. With more than $360 million in research grants and contracts last year, USF is one of the nation's top 63 public research universities and one of 39 community-engaged, four-year public universities designated by the Carnegie Foundation for the Advancement of Teaching. For more information, visit health.usf

Synexus Increases Osteoporosis Clinical Trial Capacity

Synexus has significantly increased its capacity to recruit and run global osteoporosis trials by increasing the number of DEXA scanners at its hub sites by a third. According to the American College of Radiology the DEXA scan is the most reliable way of identifying osteoporosis and is the best test for measuring bone mineral density [ [1]].



Osteoporosis affects one in three women - over 50 - and one in five men around the world; increasing to one in two and one in three in the over 60s [ [2]]. It is estimated that the worldwide cost of osteoporosis will increase to USD 131.5billion by 2050 [ [3]].



Synexus has complemented its DEXA investment with the provision of heel scanners to hundreds of primary care practices to boost enrolment.



Dr Ian Smith Medical Director and founder commented; "The heel scan is a quick, easy and cost effective way of determining whether there is a likelihood of osteoporosis. All the patients who have an elevated reading are then offered a DEXA scan at one of our hub sites in order to make a definitive diagnosis."



Synexus has recently carried out tens of thousands of heel tests and followed up with thousands of DEXA scans to recruit patients onto osteoporosis studies.



Michael Fort CEO of Synexus said "We have dramatically increased our capacity in the area of osteoporosis due to demand from our clients. With our network of fourteen clinics in the UK, Eastern Europe, South Africa and India we are able to randomise thousands of patients."


References


[1]. American College of Radiology. ACR appropriateness criteria for osteoporosis and bone mineral density. 2001.

[2]. Osteoporosis Australia osteoporosis.au 2004

[3]. Lindsay R et al., 2001, JAMA 285:320



Synexus(R) background


Synexus(R) headquartered in Manchester, England, is the world's largest multi-national company dedicated to the recruitment and running of late stage clinical trials.



Synexus(R)recruits participants for clinical trials on behalf of pharmaceutical, biotech and CROs. The clinical trials are then run and managed by Synexus???' at 14 hub sites across the UK, Poland, Hungary, Bulgaria, India and South Africa.



Synexus(R) pioneered the running of clinical trials at hub sites as against the traditional method of contracting with primary care physicians, who on average only recruit and manage five patients each.



synexus

Spherix Phase 2 Trial With D-tagatose Determines Minimum Dose For HbA1c And Triglycerides

Spherix Incorporated (Nasdaq: SPEX), an innovator in biotechnology for therapy in diabetes, metabolic syndrome and atherosclerosis; and providers of technical and regulatory consulting services to food, supplement, biotechnology and pharmaceutical companies, announced that its Phase 2 diabetes clinical trial, designed to determine the minimum dose of D-tagatose capable of reducing HbA1c, found that the minimum dose capable of affecting HbA1c (7.5 g three-times daily, or TID) was within the range of doses tested (2.5, 5.0, and 7.5 g TID), with the 2.5 and 5.0 g doses producing similar responses to one another, and the 7.5 g dose producing a greater response.


In addition, by the end of the six-month trial, the 7.5 g dose reduced serum triglycerides vs. the 2.5 g dose by -42 mg/dl from a mean of 180 mg/dl in the Evaluable Efficacy (EE) population. The reduction in serum triglycerides became statistically significant in the Intent-To-Treat (ITT) population at three months of treatment (-31 mg/dl, p=0.03) and the reduction essentially held steady at the six-month end-of-study visit (-29 mg/dl).


In the single-blind study designed to establish the minimum dose capable of causing a beneficial effect, three different doses of D-tagatose were administered to patients orally with meals TID. The comparator was the 2.5 g dose. The study was designed with a minimum of 34 patients in each of the three groups for a total of 102 evaluable patients. The primary endpoint for the study was reduction in HbA1c after six months of treatment. In the minimum dose range, D-tagatose produced a -0.3% reduction in HbA1c in the 7.5 g dose group vs. the 2.5 g dose group in the EE population, from a mean randomization HbA1c of 7.4%. The reduction of the 7.5 g dose was 0.2% more than the 2.5 g dose in the ITT population.


Unlike other drugs, D-tagatose lowered triglycerides without elevating LDL. In fact, D-tagatose in the 7.5 g dose reduced LDL vs. the 2.5 g dose by -11 mg/dl by the third month of treatment, although the difference was not statistically significant. The reduction essentially held steady at the six-month end-of-study visit (-10 mg/dl). HDL was unchanged, increasing only between 0.3 and 1.4 mg/dl over the entire course of the study vs. comparator. Analysis of patient subgroups in the U.S. and India with elevated body mass and/or HbA1c is in progress.


"The decrease in triglycerides of more than 20% in patients with a mean serum triglycerides level that is not even in the High category supports our decision to begin an aggressive drug development program with D-tagatose in hypertriglyceridemia, atherosclerosis and the metabolic syndrome," said Dr. Claire L. Kruger, CEO of Spherix. "We are encouraged by the statistically significant reduction in triglycerides in the ITT population, which is usually difficult to achieve because patients in this population need only receive a single dose in the entire trial in order to qualify. However, this token dose requirement allows a larger number of patients into the ITT population, which in turn makes it easier to detect a smaller change with statistical significance in a minimum dose range finding trial."

Novexel's NXL104/ceftazidime Combination Commences Second Phase II Clinical Trial In Hospital Patients With Complicated Intra Abdominal Infections

Novexel, a speciality pharmaceutical company focused on the discovery and development of novel antibiotics designed to overcome the significant global problem of microbial resistance, announces today that its most advanced injectable antibacterial, which combines Novexel's broad spectrum beta lactamase inhibitor, NXL104, with the well established cephalosporin antibiotic, ceftazidime, has entered a second Phase II clinical trial.


This Phase II trial with NXL104/ceftazidime will be in hospitalized patients with complicated intra-abdominal infections (cIAIs). In 2007, Novexel estimates that more than 300,000 patients were treated for cIAIs in hospital in the seven major pharmaceutical markets. This estimate is based on national databases providing hospital patient discharge data.


NXL104/ceftazidime is being developed to treat hospital infections that are caused by Gram negative bacteria, including those resistant to many currently used antibiotics. This Phase II trial is a prospective, multicenter, double-blind, randomized study which is designed to evaluate the efficacy, safety, and tolerability of NXL104/ceftazidime plus metronidazole vs. meropenem (Merrem(R) or Meronem(R), AstraZeneca, NYSE: AZN) in the treatment of adults with cIAIs. Complicated intra-abdominal infections include those infections requiring surgical intervention and which extend beyond the hollow viscus into the peritoneal space. A total of approximately 200 patients will be enrolled, with 100 patients being included in each treatment arm.


The primary objective of the study is to evaluate the clinical response to NXL104/ceftazidime plus metronidazole in the treatment of adult patients with cIAIs as compared to meropenem. This evaluation will be based on the Test of Cure visit two weeks post-therapy. The total duration of antibiotic therapy for each patient should be 5 to 14 days. The results of this study are expected in early 2010.


This second Phase II trial in cIAIs, follows a Phase II clinical trial of NXL104/cetazidime in hospitalized patients with complicated urinary tracts infections (cUTIs). This study which began in November is due to complete in late 2009.


In completed Phase I studies in healthy subjects NXL104/ceftazidime was generally well tolerated and the pharmacokinetics of the two components were well matched. Additional studies in special populations are ongoing.


Iain Buchanan, Novexel's CEO, said, "The start of this second Phase II study demonstrates Novexel's commitment to developing NXL104 in combination with ceftazidime in a broad range of indications where resistant Gram negative bacteria are present or are suspected. NXL104 has the ability to inhibit the activity of a broader range of clinically important beta-lactamases than currently marketed beta-lactam inhibitors and we believe that this combination product could provide an important new treatment option in the hospital."















Under an independent development program, Forest Laboratories (NYSE : FRX) will combine NXL104 in a treatment regimen with Forest's ceftaroline, a novel, bactericidal injectable broad spectrum cephalosporin. Ceftaroline is being developed as a therapeutic agent for the treatment of gram-positive pathogens including methicillin resistant staphylococcus aureus (MRSA), and multi-drug resistant streptococcus pneumoniae (MDRSP), as well as common gram-negative organisms. Ceftaroline is being studied as a monotherapy in Phase III clinical trials by Forest. In January 2008, Novexel granted Forest a license to develop, manufacture and commercialise NXL104, only in combination with ceftaroline, in North America.


Novexel retains worldwide rights on the NXL104/ceftazidime combination.


About NXL104


NXL104 is a novel injectable non beta lactam, beta-lactamase inhibitor, which is being developed to address the increasing problem of microbial resistance to beta lactam antibiotics (penicillins, cephalosporins, carbopenems) mediated by beta-lactamase enzymes.


NXL104 is a significant advance, as it is able to inhibit a broader range of beta-lactamases than currently marketed inhibitors. Its spectrum includes class A (including ESBL and KPC) and class C enzymes. NXL104 in combination with ceftazidime will be targeting infections caused by Gram negative bacteria, including Enterobacteriaceae and Pseudomonas.


About Novexel


Novexel is a speciality pharmaceutical company focused on the discovery and development of novel antibiotics designed to overcome the significant global problem of microbial resistance. The ever increasing resistance to marketed antibiotics has led to a clear need for novel drugs that are active against multi-drug resistant bacteria. Novexel's products are targeting the global hospital antibiotic market, which was worth an estimated $15bn in 2007 according to information derived from the IMS Midas(R) database.


Novexel currently has two novel antibacterials in Phase II clinical development. These are the injectable beta-lactamase inhibitor, NXL104, which is being developed in combination with the cephalosporin antibiotic ceftazidime for serious Gram negative infections, and the oral streptogramin antibiotic, NXL103, for the treatment of Gram positive infections, with a focus on treatment in the hospital setting and intravenous (IV) to oral switch. Novexel has three further programmes in preclinical development, NXL105, a novel anti-Pseudomonal antibiotic, NXL201, a novel echinocandin antifungal agent, and NXL104 in combination with ceftaroline. This latter product is being developed by our partner, Forest Laboratories, solely for North American markets.


Novexel was created in December 2004 as an independent spin-out of the sanofi-aventis (Euronext Paris: SAN, NYSE: SNY) anti-infectives unit. Novexel has a team of 50 employees with significant experience in anti-infective research and development, who are located in Paris, France and Philadelphia, USA.


Novexel

novexel

Novel Program Underway To Increase Participation In Clinical Trials -- A Critical Step In Development Of Improved Medical Therapies

Before a new treatment for a disease like cancer becomes available, physician-researchers must recruit hundreds or thousands of patients to participate in clinical research trials. Yet, too often, finding these patients is difficult, as individuals may be unaware of clinical studies, or logistical barriers may preclude participation. The result is that many patients miss out on opportunities for novel treatment approaches, and the development of new and improved therapies is delayed.



Addressing this vital issue is a unique and innovative collaborative study, known as IMPACT, for "Improving Methods for Patient Accrual to Clinical Trials." Two institutions -- Weill Cornell Medical College in New York City and the Cornell University College of Agriculture and Life Sciences in Ithaca, NY -- have teamed to develop strategies to better understand and enhance such patient participation in clinical trials.



"Low patient accrual in clinical trials poses a serious problem for the advancement of medical science," observes Dr. John Leonard, a co-leader of the IMPACT study, who is also professor of medicine at Weill Cornell Medical College and attending physician at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. "The time required to conduct clinical trials is widely recognized as a rate-limiting step in moving novel treatments forward. As one example, less than 2 percent of patients choose to participate in clinical trials for cancer therapies across the United States. Even a modest increase of 2 to 3 percentage points would make a major impact, meaning the difference between completing a study in two years instead of three years -- and potentially resulting in thousands of lives saved if the standard of care is improved more rapidly. To this end, this unique collaborative study will seek to uncover new means for improving recruitment and better serving patients," he says.



"We're excited to strengthen the alliances between our two campuses while bringing together expertise in both medical and communication science to address important social issues. The IMPACT project is the start of what we envision as a long-term collaboration between our two groups. We envision that together we will use established communication research methods to promote health-related activities in numerous areas," says Dr. Geri Gay, another co-leader of the effort as well as professor and chairman of the Department of Communication in the College of Agriculture and Life Sciences at Cornell University.



IMPACT investigators will also collaborate with The Leukemia & Lymphoma Society, which has helped finance the first phase of the project, including a national survey on attitudes toward participation in clinical trials. The funding will also support a doctoral student in the Communication Department focusing in this area.



"Cancer clinical trials have brought enormous advances in the areas of cancer prevention, treatment and diagnosis. By encouraging broader and more rapid enrollment, the benefits of clinical research, including prevention and treatment, will be more quickly available to a greater number of patients," says Dr. Dwayne Howell, president and CEO of The Leukemia & Lymphoma Society.
















"Hundreds of studies have sought to identify and overcome barriers to enrollment. This project is the first to assess the problem from a socio-psychological perspective, using the specialized methods of risk communication," says Dr. Katherine McComas, principal leader of the IMPACT study and assistant professor in the Department of Communication at the College of Agriculture and Life Sciences at Cornell. "We will be using two proven approaches -- the model of Risk Information Seeking and Processing (RISP) and Theory of Planned Behavior. These will allow us to examine specific factors that influence how patients inform themselves about a clinical trial and decide whether to participate."



Previous research has identified several barriers to enrollment, including patients' fears of randomization and risk of side effects; distrust of physicians and researchers; time commitment and other logistical concerns; and lack of familiarity with clinical trials. On the flip side, patients may choose to participate in clinical trials because they perceive potential for a better treatment (or for refractory cases, any treatment where no good alternative is available), reduced treatment cost and feelings of altruism.



The IMPACT project is one of several recent efforts at Weill Cornell Medical College to foster clinical research programs across the medical spectrum, including the development of the Institute for Clinical Research under the direction of Dr. Ralph Nachman, Associate Dean for Clinical Research at Weill Cornell.



"Our aim is to provide data-supported recommendations for strategies to improve the accrual of patients in clinical trials," adds Dr. Andrew Dannenberg, also a co-leader of the IMPACT project, professor of medicine at Weill Cornell Medical College and attending physician at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. "Future phases of the study will develop specific tools to better inform patients, educate them as to the pros and cons of enrolling in clinical trials, as well as create strategies to facilitate participation so that new therapies for many disorders can be more rapidly designed and evaluated in order to deliver their maximal benefit."



Weill Cornell Medical College


Weill Cornell Medical College -- Cornell University's Medical School located in New York City -- is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Weill Cornell, which is a principal academic affiliate of NewYork-Presbyterian Hospital, offers an innovative curriculum that integrates the teaching of basic and clinical sciences, problem-based learning, office-based preceptorships, and primary care and doctoring courses. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research in such areas as stem cells, genetics and gene therapy, geriatrics, neuroscience, structural biology, cardiovascular medicine, AIDS, obesity, cancer, psychiatry and public health -- and continue to delve ever deeper into the molecular basis of disease in an effort to unlock the mysteries behind the human body and the malfunctions that result in serious medical disorders. The Medical College -- in its commitment to global health and education -- has a strong presence in such places as Qatar, Tanzania, Haiti, Brazil, Salzburg, and Turkey. With the historic Weill Cornell Medical College in Qatar, the Medical School is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances -- from the development of the Pap test for cervical cancer to the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial for gene therapy for Parkinson's disease, the first indication of bone marrow's critical role in tumor growth, and, most recently, the world's first successful use of deep brain stimulation to treat a minimally-conscious brain-injured patient.

med.cornell.



College of Agriculture and Life Sciences at Cornell University


The College of Agriculture and Life Sciences at Cornell University is committed to developing leaders to address the global challenges of the 21st century. With more than 3,000 students and 400 faculty, the preeminent college for research, teaching and extension of agriculture and the life sciences is the second largest undergraduate college at Cornell and the third largest college of its kind in the United States. A commitment to four priorities -- the land-grant mission, the applied social sciences, the environmental sciences and the new life sciences -- allows faculty and students to examine the world from multiple perspectives, and ensures that every student's education is geared to contemporary, real-world issues. Diverse strengths across the biological sciences, a commitment to biodiversity, a strong undergraduate business program, a long tradition of international work and the translation of groundbreaking discoveries into commercial practice and educational outreach ensures that faculty, staff and students make an important difference in the lives of stakeholders in New York state, the nation and the world.

cals.cornell.


The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society, headquartered in White Plains, N.Y., with 68 chapters in the United States and Canada, is the world's largest voluntary health organization dedicated to funding blood cancer research and providing education and patient services. The Society's mission: Cure leukemia, lymphoma, Hodgkin's disease and myeloma, and improve the quality of life of patients and their families. Since its founding in 1949, the Society has invested more than $550 million in research -- specifically targeting leukemia, lymphoma and myeloma. Last year alone, the Society made 5.1 million contacts with patients, caregivers and health-care professionals.

LLS.

Repligen Announces Completion Of Patient Treatment In Phase 3 Clinical Trial Of RG1068 In MRI Imaging Of The Pancreas

Repligen Corporation (Nasdaq: RGEN) reported that it has completed patient treatment in its Phase 3 clinical trial of RG1068, synthetic human secretin, in magnetic resonance imaging (MRI) of the pancreas. The study is designed to assess the sensitivity and specificity of RG1068 in conjunction with MRI for the detection of pancreatic duct abnormalities compared to MRI alone. Additional assessments include safety, physician confidence in the identification of structural abnormalities, the number of pancreatic duct segments visualized and the improvement in the quality of the MRI images. The study enrolled 258 patients at 23 clinical sites within the U.S. and Canada. Detailed visual assessment of the pancreatic ducts is important in the diagnosis and treatment of diseases such as acute and chronic pancreatitis.


Structural abnormalities of the pancreatic ducts were assessed by RG1068 used in conjunction with MRI and independently by endoscopy, a commonly used invasive procedure. There were no serious adverse events (SAEs) associated with the RG1068/MRI procedure compared to 68 patients with an SAE associated with endoscopy. The most commonly reported SAE following endoscopy was acute pancreatitis requiring hospitalization.


"We are very pleased to have completed the patient treatment phase in this study which confirmed the advantageous safety profile of RG1068," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We expect to release top-line results for the study later this year."


RG1068 is a synthetic version of human secretin, a natural gastrointestinal hormone involved in the process of digestion. Secretin has been used for many years by gastroenterologists in conjunction with endoscopy, an invasive procedure to evaluate and treat diseases of the pancreas and gallbladder. There are risks associated with the use of endoscopic retrograde cholangiopancreatography (endoscopy), which have generated interest in the development of safer non-invasive tests to diagnose gastrointestinal disorders. The use of secretin in combination with a non-invasive procedure such as MRI can improve the detection of abnormalities and increase the diagnostic quality of the MRI image of the pancreas. The FDA has granted RG1068 Orphan Drug status and Fast Track Designation, a process designed to facilitate the development and expedite the review of drugs that treat serious diseases and fill an unmet medical need based on the need for safer non-invasive tests to diagnose pancreatic disorders. There are more than 300,000 MRI procedures conducted in the U.S. and Europe each year that could directly benefit by the addition of RG1068.


About Repligen Corporation


Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for neurological disorders. In addition, we are the world's leading supplier of recombinant Protein A, the sales of which partially fund the advancement of our development pipeline while supporting our financial stability. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453.















This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

Study Reveals Neighborhood Asthma Risks

Mayo Clinic researchers recently released study data showing children who lived near major highway or railroad intersections have higher diagnoses of asthma. The researchers used this study to show how neighborhood environment is a risk factor in understanding the development of pediatric asthma. The study appears in a recent addition of The Journal of Allergy and Clinical Immunology.


"Using nearest propensity score, children who lived in census tracts facing the intersection with major highways or railways had about 40 to 70 percent increased risk of developing childhood asthma," says Young Juhn, M.D., of Mayo Clinic's Department of Community Pediatric and Adolescent Medicine. "What this tells us is that clinicians need to be concerned about neighborhood environment beyond home environment to understand the individual asthma case."


The study was a retrospective, population-based birth cohort where researchers studied 3,970 people born between 1976 and 1979 in Rochester, Minn. Of the 1,947 subjects living in census tracts that faced intersections, 6.4 percent developed asthma, while 4.5 percent of those living in census tracts not facing intersections developed asthma. Dr. Juhn and his colleagues are currently conducting research that looks at the influence of neighborhood environment on other disease outcomes.


Other study authors include Rui Qin, Ph.D., Department of Health Sciences Research, Mayo Clinic; Sanghwa Urm, M.D., Ph.D, Department of Preventive Medicine, School of Medicine, Inje University, South Korea; Slavica Katusic, M.D., Department of Health Sciences Research, Mayo Clinic; and Delfino Vargas-Chanes, Ph.D., Center of Sociological Studies, El Colegio de Mexico.


About Mayo Clinic


For more than 100 years, millions of people from all walks of life have found answers at Mayo Clinic. These patients tell us they leave Mayo Clinic with peace of mind knowing they received care from the world's leading experts. Mayo Clinic is the first and largest integrated, not-for-profit group practice in the world. At Mayo Clinic, a team of specialists is assembled to take the time to listen, understand and care for patients' health issues and concerns. These teams draw from more than 3,700 physicians and scientists and 50,100 allied staff that work at Mayo Clinic's campuses in Minnesota, Florida, and Arizona; and community-based providers in more than 70 locations in southern Minnesota, western Wisconsin and northeast Iowa. These locations treat more than half a million people each year. To best serve patients, Mayo Clinic works with many insurance companies, does not require a physician referral in most cases and is an in-network provider for millions of people.

Pfizer's Maraviroc To Receive Accelerated Regulatory Reviews In The U.S. And Europe

Pfizer announced today that
marketing authorization applications for maraviroc will receive accelerated
review in both the United States and Europe. Accelerated reviews are
granted to potential medicines that, if approved, would represent
significant improvements over current therapies.


If approved by the regulatory agencies, maraviroc will be the first in
a new class of HIV/AIDS treatments called CCR5 antagonists that work by
blocking viral entry. Rather than fighting HIV inside white blood cells,
CCR5 antagonists prevent the virus produced by infected cells from entering
uninfected cells by blocking its predominant entry route, the CCR5 co-
receptor.



"There is a profound global need for new medicines to help HIV/AIDS
patients," said John LaMattina, president, Pfizer Global Research and
Development. "We expect that CCR5 antagonists, like maraviroc, will become
critically important new treatment options for patients who are resistant
or intolerant to their current HIV/AIDS therapies."



The U.S. Food and Drug Administration (FDA) priority review process
takes place within a six-month period. Pfizer submitted the U.S. and EU
maraviroc marketing applications in December 2006. An FDA Advisory Panel is
scheduled for April 24. Pfizer has begun pursuing regulatory approval for
maraviroc in other countries to enable broad access to the drug.



Moving with Urgency



The discovery of maraviroc dates back to 1997 when Pfizer research
scientists in Sandwich, UK designed the molecule following the publication
of two significant research findings. A study was published in 1996 that
described resistance to HIV-1 infection in certain Caucasian subjects, and
in the same year, another journal reported the binding of HIV to the CCR5
receptor. Scientists noted that about one percent of Europeans who lacked
the genes for CCR5 receptors were the very ones who were resistant to
acquiring HIV infection. This finding suggested that blocking the virus's
entry through this gateway may lead to a breakthrough therapy. Based on
these emerging scientific insights and patient need, the maraviroc team
significantly accelerated development time.



"This is the kind of targeted science that underscores our commitment
to research and development in a range of infectious diseases where there
is high human cost due to drug resistance," said Dr. Ethan Weiner, senior
vice president, Pfizer Global Research and Development. "Maraviroc is an
outstanding example of rapid development and continuous innovation through
which Pfizer researchers quickly translated a scientific hypothesis into a
promising compound in this area of great medical need."



Maraviroc is the seventh Pfizer new drug application to receive
"Priority Review" status from the FDA over the past two years. Other
priority review FDA approvals include Sutent for advanced kidney cancer and
gastrointestinal stromal tumors, Chantix for smoking cessation, Revatio for
pulmonary arterial hypertension, and Macugen for age-related macular
degeneration which can lead to blindness in elderly patients.
















Pivotal Trials



The marketing applications follow Pfizer's review of efficacy and
safety data from two pivotal phase 3 trials. The trials, MOTIVATE-1 and 2
(Maraviroc plus Optimized Therapy In Viremic Antiretroviral
Treatment-Experienced patients), represent 24-week data comparing Optimized
Background Therapy, with or without maraviroc, in over 1,000 highly
treatment-experienced patients with CCR5-tropic HIV-1. These study results
have been accepted for presentation at an upcoming HIV conference.



In addition, the independent Data Safety Monitoring Board (DSMB) for
maraviroc met on January 15, 2007 and continues to monitor the ongoing
clinical program. The DSMB recommended that the maraviroc Phase 3
registrational trials, in both treatment-na??ve and treatment-experienced
patients, continue as currently designed.



Update on Expanded Access Program



In December 2006, Pfizer announced plans to establish a multi-national
Expanded Access Program to provide maraviroc to patients with limited
available treatment options based on its safety and efficacy observed in
clinical trials to date. The program is now open for enrollment with a
target to enroll patients from over 30 countries.



Through partnerships and focused philanthropic efforts, Pfizer strives
to support HIV prevention efforts, build improved healthcare
infrastructure, and further access to HIV/AIDS medicines. Current
initiatives include the U.S. Southern States HIV/AIDS Prevention
Initiative; the building of the Infectious Disease Institute in Kampala,
Uganda; the Pfizer Global Health Fellows Program; and the Diflucan(R)
Partnership Program. For more information on these and other Pfizer
initiatives, go to pfizer.



The information contained in this release is as of
February 13, 2007. Pfizer assumes no obligation to update any
forward-looking statements contained in this release as the result of new
information or future events or developments.



This release contains forward-looking information that involves
substantial risks and uncertainties regarding a product candidate,
including its potential benefits, that is under review by the United States
Food and Drug Administration (FDA), the European Medicines Evaluation
Agency (EMEA) and certain other regulatory authorities. Such risks and
uncertainties include, among other things, whether and when the FDA, the
EMEA and other regulatory authorities will approve the product candidate,
their decisions regarding labeling and other matters that could affect its
availability or commercial potential, as well as competitive developments.



A further list and description of risks and uncertainties can be found
in Pfizer's Annual Report on Form 10-K for the fiscal year ended December
31, 2005 and in its reports on Form 10-Q and Form 8-K.


Pfizer Inc

pfizer


View drug information on Chantix; Macugen; Sutent.