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