|Synthetic Biologics Issues UCLA Report of Positive Topline Efficacy of Trimesta; CryoLife Receives FDA Clearance for PerClot|
|By Staff and Wire Reports|
|Tuesday, 29 April 2014 20:20|
Synthetic Biologics, Inc. (NYSE: SYN), a developer of novel anti-infective biologic and drug candidates targeting specific pathogens that cause serious infections and diseases, reported today that the University of California, Los Angeles (UCLA) School of Medicine announced preliminary, positive topline data from the Phase II clinical trial evaluating Trimesta™ (oral estriol), the Company's oral, once-daily treatment for relapsing-remitting multiple sclerosis (RRMS) in women. The Phase II study, initiated and led by Rhonda Voskuhl, M.D., Professor, Department of Neurology, Jack H. Skirball Chair in Multiple Sclerosis Research and Director, Multiple Sclerosis Program at UCLA David Geffen School of Medicine, demonstrated that Trimesta given with first-line RRMS therapy Copaxone® (glatiramer acetate injection) resulted in reduced annualized relapse rate and improved cognitive function at 12 months of treatment as compared to placebo plus Copaxone. Dr. Voskuhl presented the Phase II topline data today at the 66th Annual Meeting of the American Academy of Neurology in Philadelphia as part of the Emerging Science Program.
Dr. Voskuhl commented, "Pregnancy is known to be a period of relative protection from MS relapses, and the therapeutic effects of pregnancy hormones, including estriol, include both anti-inflammatory and neuroprotective mechanisms as evidenced in animal models of MS. We are encouraged by the topline results from this first randomized placebo-controlled trial evaluating oral estriol due to the positive impact of adjunctive therapy with estriol on the patients in this study. We look forward to continued clinical evaluation of estriol, which we believe has the potential to slow disease progression and improve quality of life for women with RRMS."
The Phase II, double-blinded, placebo-controlled trial randomized 158 women with relapsing-remitting MS at 16 sites across the U.S. Among topline results, women receiving Trimesta plus Copaxone demonstrated a statistically significant 47 percent decrease in annualized MS relapse rate in the first 12 months of treatment as compared to women receiving placebo plus Copaxone. In addition, a significant improvement in cognitive function was observed at 12 months as measured by Paced Auditory Serial Addition Test (PASAT) scores. After 24 months of treatment, the reduction in relapse rate persisted in favor of the Trimesta plus Copaxone treatment group compared to the Copaxone plus placebo group (32 percent). Both treatment groups exhibited improvement in measures of cognitive function at 24 months. Treatment was safe and well tolerated with no evidence of adverse effects on breast or uterus.
"The topline data presented today are encouraging for women with MS, including the approximately 260,000 women with MS in the United States. We believe adjunctive therapy with Trimesta has the potential to improve therapeutic outcomes for women currently taking first-line RRMS treatments. Trimesta's differentiated mechanism of action, combined with the convenience of once-daily oral dosing, may potentially make it an ideal added therapy to address this underserved $14.1 billion worldwide market," stated Jeffrey Riley, Chief Executive Officer of Synthetic Biologics. "As Synthetic Biologics determines next steps for Trimesta, including potential plans for a Phase III clinical program, we look forward to feedback from the neurology community and from potential partners regarding the encouraging topline data presented by Dr. Voskuhl."
The investigator-initiated, Phase II, two-year, multi-center, randomized, double-blinded, placebo-controlled (1:1) study evaluated Trimesta as an oral, once-daily dose of 8 mg per day plus Copaxone in women with RRMS, aged 18-50 years. Dr. Voskuhl's work is supported by grants exceeding $8 million, which were awarded primarily by the National Multiple Sclerosis Society (NMSS) in partnership with the NMSS's Southern California chapter, and the National Institutes of Health (NIH).
CryoLife, Inc. (NYSE: CRY), a leading medical device and tissue processing company focused on cardiac and vascular surgery, announced today that it has received 510(k) clearance for PerClot Topical hemostatic powder from the U.S. Food and Drug Administration (FDA). This clearance allows CryoLife to begin commercialization of PerClot Topical hemostatic powder in the U.S. The Company plans to begin shipping PerClot Topical hemostatic powder in June of this year.
Cryolife logo. "We are pleased to have received 510(k) clearance for PerClot Topical in the U.S.," stated Steven G. Anderson, CryoLife president and chief executive officer. "We expect to begin commercialization efforts in June and to focus on the ENT specialty through a combination of our existing sales force and distributors experienced with a competitive product. Many of our existing hospital customers also have large ENT practices, providing a natural opportunity for PerClot Topical. Longer term, we believe we will be able to expand PerClot Topical to additional markets such as the emergency room."
PerClot Topical is a unique hemostat composed of polysaccharide granules and is intended for use as a topical dressing for the temporary treatment of mildly bleeding wounds such as surgical wounds (post-operative, donor sites, dermatological, etc.), cuts and lacerations and for the treatment of mild bleeding from topical ENT surgical wounds and nosebleeds. It is also indicated for control of bleeding from the skin at percutaneous needle access, vascular access and percutaneous catheter access sites.
PerClot Topical is ready to use, requiring no mixing and/or other components and does not need special handling or storage conditions. Preclinical evaluations have shown the effectiveness of PerClot Topical to be comparable to the current popular choice of surgical hemostatic materials.
PerClot Topical is the same product as PerClot, which has received an Investigational Device Exemption (IDE) from the FDA for a pivotal clinical trial for use in surgical procedures as an adjunctive hemostatic device when control of capillary, venular and arteriolar bleeding by pressure, ligature and other conventional procedures is ineffective or impractical. The Company plans to begin enrollment in the pivotal clinical trial for PerClot in the second quarter of 2014, and could potentially receive pre-market approval from the FDA by the end of 2015.
Management estimates the U.S. hemostatic market for PerClot Topical in procedures included in the 510(k) clearance to be in excess of $100 million, while the U.S. hemostatic market for all procedures is estimated to have been $780 million in 2013, growing to approximately $915 million by 2016 and the European market is estimated to have been $395 million in 2013, growing to approximately $468 million by 2016.
Declaratory Judgment Action -- CryoLife also announced today that it has filed a declaratory judgment action against C.R. Bard, Inc. and certain of its subsidiaries (collectively, Bard) in federal court, requesting that the court confirm that CryoLife's anticipated sales of PerClot (once it has received final FDA approval) and certain of its derivative products (including PerClot Topical) will not infringe upon patents held by Bard. CryoLife filed the declaratory judgment action in the U.S. District Court for the District of Delaware.
Mr. Anderson noted, "We believe that our sales of PerClot Topical and PerClot (once approved by the FDA) will not infringe the Bard patent, and we are optimistic that the court will affirm our position."
Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced positive 12- and 24-month data from its first Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") monotherapy in Fabry patients with amenable mutations.
Anavex Life Sciences Corp. (OTCQB:AVXL), a clinical stage biopharmaceutical company developing novel drug candidates to treat Alzheimer's, other diseases of the central nervous system (CNS) and various types of cancer, today announced the findings of a research study published in the current issue of peer-reviewed scientific journal Brain.
AngioDynamics (Nasdaq:ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, surgery, peripheral vascular disease and oncology, today announced Howard W. Donnelly, 52, has been named Chairman of the Board of Directors, effective immediately.
Argos Therapeutics, Inc. (Nasdaq:ARGS) announced today that the Company's first quarter 2014 financial results will be released after market close on Tuesday, May 13, 2014.
Biota Pharmaceuticals, Inc. (Nasdaq:BOTA) announced today that management will host a conference call on Tuesday, May 6, 2014 at 4:30 p.m. EDT to review the Company's third quarter financial results and provide an update on recent corporate developments.
Biota Pharmaceuticals, Inc. (Nasdaq:BOTA) today announced that it has been notified by the U.S. Department of Health and Human Services (HHS) office of the Assistant Secretary for Preparedness and Response (ASPR) and Biomedical Advanced Research and Development Authority (BARDA) that pending a decision regarding the outcome of a recently completed In Process Review (IPR) of the Company's contract for the development of laninamivir octanoate, ASPR/BARDA has issued a Stop-Work Order notifying the Company to discontinue work on a number of activities under its contract.
Celladon Corporation (Nasdaq:CLDN), a clinical-stage biotechnology company focused on developing novel therapies by applying its leadership position in the field of SERCA enzymes, today announced that it will host an Investor and Analyst Day on Tuesday, May 6, 2014 at 8:00 a.m. in New York City.
ChemoCentryx, Inc. (Nasdaq:CCXI) announced today that the Company's first quarter 2014 financial results will be released after market close on Thursday, May 8, 2014.
Lipocine Inc. (Nasdaq:LPCN), a specialty pharmaceutical company, today announced that the Company has completed enrollment of its Study of Oral Androgen Replacement ("SOAR") pivotal Phase 3 clinical study (http://clinicaltrials.gov/show/NCT02081300).
Ocera Therapeutics (Nasdaq:OCRX), a clinical stage biopharmaceutical company focused on acute and chronic orphan liver diseases, today announced that it will release its first quarter financial results on May 8, 2014 at approximately 4:30 p.m. Eastern Time.
SIGA Technologies, Inc. (Nasdaq:SIGA) announced today that management will host a conference call and simultaneous webcast to provide a quarterly business update at 4:30 P.M. ET on Thursday, May 1, 2014.
Supernus Pharmaceuticals, Inc. (Nasdaq:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases, today announced that the Company expects to report the quarter ending March 31, 2014 financial results after the close of the U.S. financial markets Monday, May 12, 2014.
VIVUS, Inc. (Nasdaq:VVUS) today announced that it will report financial results for the first quarter ended March 31, 2014 after the NASDAQ Market closes on Monday, May 5, 2014.