|Sarepta's Eteplirsen Shows Benefit on Walking Test in Phase IIb Study; Arena Initiates Phase 1 Clinical Trial of APD334|
|By Staff and Wire Reports|
|Friday, 05 April 2013 19:23|
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 74 weeks showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).
As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51. After 74 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) showed a statistically significant treatment benefit of 65.2 meters (p d 0.004) when compared to the placebo/delayed-treatment cohort (n=4). The eteplirsen-treated patients in the mITT population demonstrated less than a 5 percent decline (13.4 meters) from baseline in walking ability.
After experiencing a substantial decline earlier in the study, the placebo/delayed-treatment cohort also demonstrated stabilization in walking ability from week 36 through 74, the period in which meaningful levels of dystrophin were likely produced, with a less than 10 meter decline over this timeframe. "We are encouraged to see a continued stabilization of walking ability in patients treated with eteplirsen for nearly one and a half years," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "These data are particularly compelling when viewed in the context of published natural history studies, which showed substantial declines on the 6-minute walk test over this timeframe in a similar ambulatory DMD population. These results con tinue to support the potential of eteplirsen to be a major advance in the treatment of DMD in altering the course of this progressive and irreversible disease." Through 74 weeks, eteplirsen was well tolerated and there were no clinically significant treatment-related adverse events, serious adverse events, hospitalizations or discontinuations. As previously reported at 62 weeks, one patient had a transient elevation of urine protein on a laboratory urine dipstick test, which resolved and resulted in no clinical symptoms. The patient continued treatment without interruption and remained free of proteinuria through week 74.
Across both the eteplirsen (mITT) and placebo/delayed-treatment cohorts, there is evidence of continued stabilization on clinical laboratory tests, echocardiogram, pulmonary function tests and muscle strength.
Arena Pharmaceuticals, Inc. (NASDAQ: ARNA) announced the initiation of dosing in a Phase 1 clinical trial of APD334, a novel oral drug candidate that targets the sphingosine 1-phosphate subtype 1 (S1P) receptor for the potential treatment of autoimmune diseases.
This randomized, double-blind and placebo-controlled Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of single-ascending doses of APD334 in up to 64 healthy adult volunteers.
"We are pleased to expand our clinical-stage pipeline by initiating a Phase 1 trial of APD334, and look forward to advancing this novel compound through our validated development platform," said William R. Shanahan, M.D., Arena's Senior Vice President and Chief Medical Officer. "APD334's selectivity for the S1P receptor has the potential to improve upon the adverse event profile of currently available treatments for a spectrum of autoimmune diseases."
3DIcon Corporation (OTCBB: TDCP), a developer of volumetric, three-dimensional display technologies, announced today that Sidney Aroesty, currently a board member of 3DIcon Corporation ("3DIcon"), will be stepping down effective April 15, 2013.
Amarantus BioScience, Inc. (OTCQB: AMBS), a biotechnology company discovering and developing treatments and diagnostics for diseases associated with neurodegeneration and apoptosis, today announced it will hold a conference call on Tuesday, April 16, 2013 at 4:30 p.m. Eastern (1:30 p.m. Pacific) to provide an update on its business and conduct a question and answer session with investors.
Anika Therapeutics, Inc. (NASDAQ:ANIK) today announced that it plans to issue its first-quarter 2013 financial results after the close of the market on Wednesday, May 1, 2013.
Antares Pharma, Inc. (NASDAQ: ATRS) today announced that it has received a Notice of Allowance from the U.S. Patent Trade Office (USPTO) on a patent application for the VIBEX™ QuickShot (QS) device, the latest advancement in its proprietary line of VIBEX™ auto-injector systems.
Anthera Pharmaceuticals, Inc. (Nasdaq: ANTH), a biopharmaceutical company developing drugs to treat serious diseases associated with inflammation, today announced that it has secured a new $20.0 million debt financing facility through a syndicate led by MidCap Financial SBIC, LP ("MidCap Financial").
Arena Pharmaceuticals, Inc. (NASDAQ: ARNA) announced today the initiation of dosing in a Phase 1 clinical trial of APD334, a novel oral drug candidate that targets the sphingosine 1-phosphate subtype 1 (S1P1) receptor for the potential treatment of autoimmune diseases.
Decision Diagnostics Corp. (OTCBB: DECN), the exclusive world-wide sales, service and regulatory processes agent for the Shasta GenStrip, the revolutionary at-home glucose test strip specifically designed to work with the Johnson & Johnson's LifeScan family of glucose testing meters, announced today that on March 28, 2013 the company and its subsidiary PharmaTech Solutions, Inc. filed antitrust counterclaims against LifeScan, Inc. and LifeScan Scotland Ltd. (collectively, "LifeScan") in a patent action brought by LifeScan (2011cv04494) that is presently pending in the Northern District of California.
Emergent BioSolutions Inc. (NYSE: EBS) today announced its decision to expand the protocol for its ongoing Phase 1b, single arm, open label study (Protocol 16009) evaluating the safety and efficacy of TRU-016 in combination with rituximab in previously untreated patients with chronic lymphocytic leukemia (CLL).
IGI Laboratories, Inc. (NYSE Amex: IG), a New Jersey based generic topical pharmaceutical company will be presenting at the 12th Annual Needham & Company’s Annual Healthcare Conference.
JC Data Solutions (PINKSHEETS: JCDS) is a provider of innovative and cost-effective solutions for digital data processing and management focusing on the Oil & Gas, Legal and Healthcare industries.
Liberator Medical Holdings, Inc. (OTCBB: LBMH) today announced that on April 3, 2013, its Board of Directors approved a cash dividend of $0.02 per common share to its shareholders.
MMRGlobal, Inc. (OTCQB: MMRF) ("MMR") today announced that in an ONC Health IT Policy Committee report presented on April 3, 2013, it was discussed that the nationwide data exchange problem for health records could be solved through the use of unique identifiers including telephone numbers.
Navidea Biopharmaceuticals (NYSE MKT: NAVB), a biopharmaceutical company focused on precision diagnostic radiopharmaceuticals, announced today that it has retained Stern Investor Relations, Inc. to provide strategic advisory services and execute and enhance the Company’s investor relations program.
Neuralstem, Inc. (NYSE MKT: CUR) announced today that it received a notice of allowance for patent application 12/404,841.
Pacira Pharmaceuticals, Inc. (NASDAQ: PCRX) today announced that David Stack, president and chief executive officer of Pacira Pharmaceuticals, will close The NASDAQ Stock Market on Monday, April 8, 2013, in celebration of the one-year anniversary of the commercial launch of EXPAREL® (bupivacaine liposome injectable suspension).
PerkinElmer, Inc. (NYSE:PKI), a global leader focused on improving the health and safety of people and the environment, today announced that the Company will present at the Bio-IT World Conference at 11:00AM eastern time on Wednesday, April 10, 2013 at the Seaport World Trade Center located at 200 Seaport Boulevard in Boston, MA.
POZEN Inc. (NASDAQ: POZN), a pharmaceutical company committed to transforming medicine that transforms lives, announced the results of a POZEN sponsored study at the Academy of Managed Care Pharmacy’s 25th Annual Meeting and Expo on April 4, 2013.
Sirona (NASDAQ: SIRO), the dental technology leader, today announced that the Company will report its fiscal 2013 second quarter financial results on Friday, May 10th, 2013, before the opening of the financial markets.
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced it has entered into a non-exclusive agreement with Bristol-Myers Squibb Company (NYSE: BMY) to conduct Phase 2 studies of once-daily all-oral treatment regimens containing Vertex’s nucleotide analogue hepatitis C virus (HCV) polymerase inhibitor VX-135 and Bristol-Myers Squibb’s NS5A replication complex inhibitor daclatasvir for the treatment of hepatitis C.