|Jazz Pharma Announces First Patients Enrolled in JZP-110; Carbylan Completes Enrollment for Hydros-TA|
|By Josh Gee|
|Monday, 08 June 2015 19:16|
Below is a look at some of the headlines for companies that made news in the healthcare sector on June 8, 2015.
Jazz Pharmaceuticals plc (NASDAQ: JAZZ) announced the first patients have been enrolled in a Phase 3 clinical development program evaluating the safety and efficacy of its investigational drug candidate, JZP-110, as a wake-promoting agent in the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy or with obstructive sleep apnea (OSA). The JZP-110 clinical development program includes three Phase 3 studies being conducted in the United States (U.S.), Canada and the European Union (EU). The program also includes an open-label extension study to evaluate the long-term safety of JZP-110.
"This R&D milestone represents another step forward in Jazz's development program for JZP-110 and in our efforts to develop new treatment options for people with narcolepsy and with other sleep disorders," said Karen Smith, M.D., Ph.D., Global Head of Research and Development and Chief Medical Officer of Jazz Pharmaceuticals. "We are excited to advance JZP-110 into Phase 3 clinical development to further evaluate it as a wake-promoting agent, and we look forward to continued collaboration with the FDA and key thought leaders as we advance development of JZP-110."
Clinical Program Study Design -- Approximately 880 patients in the aggregate are expected to be enrolled in the three Phase 3 studies to be conducted across 67 medical centers in the U.S., Canada and EU. The co-primary endpoints for all three Phase 3 clinical studies -- the Maintenance of Wakefulness Test (MWT) and the Epworth Sleepiness Scale (ESS) -- will measure patients' improvement in ability to stay awake and in sleepiness. Up to 450 patients are expected to be enrolled in the open-label, long-term safety extension study.
Carbylan Therapeutics (NASDAQ: CBYL), a specialty pharmaceutical company focused on the development of novel and proprietary combination therapies, today announced that it has completed enrollment of its 510 subject COR1.1 Phase 3 pivotal trial of Hydros-TA for the treatment of pain associated with osteoarthritis. Subjects were enrolled at 30 clinical sites in Canada, Australia, New Zealand, The Netherlands, Hungary and Curacao.
COR1.1 is the first of two pivotal trials of Hydros-TA as part of Carbylan's Phase 3 program design. The trial is comprised of subjects with Kellgren-Lawrence Grade 2 and 3 osteoarthritis (OA) of the knee. Subjects were randomized equally between three treatment arms: Hydros-TA, Hydros and TA (Triamcinolone Acetonide). The objective of the trial is to demonstrate the safety and efficacy of Hydros-TA and the contribution of each of the two components in this therapy. The primary endpoints are the change from baseline in WOMAC A (pain) subscale scores for Hydros-TA versus Hydros at two weeks and Hydros-TA versus TA at 26 weeks. Secondary endpoints include WOMAC C (function) changes, subject and physician global assessment changes and OMERACT_OARSI responder analysis. The Company expects top-line data from this trial in early 2016.
David Renzi, President and CEO of Carbylan Therapeutics, commented, "The completion of COR1.1 subject enrollment is a significant milestone for Carbylan and an important step toward our goal of bringing Hydros-TA to market. We believe this product can provide a potentially best in class alternative to the treatment options currently available for patients suffering from the pain of osteoarthritis by utilizing our proprietary crosslinking technology to combine a low dose steroid with our novel hyaluronan in order to offer both rapid and sustained pain relief."
Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has strengthened its executive leadership with the appointment of two new senior members of the commercial and executive team.
Amyris, Inc. (Nasdaq:AMRS), the industrial bioscience company, today announced the appointments of Caroline Hadfield as Senior Vice President Personal Care, effective June 15, 2015, and Cynthia ("Cindy") Bryant as Senior Vice President Corporate Development & Collaborations, effective June 22, 2015.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year.
Bio-Reference Laboratories, Inc. (Nasdaq:BRLI) announces its second quarter results. Except for per share data in quotations or where otherwise noted, numbers are in thousands.
Caladrius Biosciences, Inc. (Nasdaq:CLBS), a cell therapy leader with a late-stage clinical program for immuno-oncology, today announced that its Chief Executive Officer, Dr. David J. Mazzo, has issued a letter to shareholders.
Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug Application (IND) for the clinical development of CAP-1002 for the treatment of patients with Duchenne Muscular Dystrophy (DMD) related cardiomyopathy.
Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, announced today the successful enrollment of the targeted 450 patients in SUPPRESS, the Phase 3 trial evaluating brincidofovir for the prevention of clinically significant cytomegalovirus (CMV) infection in hematopoietic cell transplant (HCT), also known as bone marrow or stem cell transplant recipients.
Conatus Pharmaceuticals Inc. (Nasdaq:CNAT), a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease, today announced the appointment of Jean L. Chan, M.D., as Vice President, Clinical Development, reporting to David T. Hagerty, M.D., Executive Vice President, Clinical Development.
Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, today announced that the company has entered into a strategic clinical research alliance with The University of Texas MD Anderson Cancer Center to advance clinical development of intratumoral TLR9 agonist in combination with checkpoint inhibitors.
IPC Healthcare, Inc. (Nasdaq:IPCM), a leading national acute hospitalist and post-acute provider organization, announced today that it has appointed Beth Hawley, SFHM, MBA, FACHE to the newly created position of Senior Vice President of Strategic Initiatives.
Lion Biotechnologies, Inc. (Nasdaq:LBIO), a biotechnology company that is developing novel cancer immunotherapies based on tumor-infiltrating lymphocytes (TIL), today announced the appointment of Molly Henderson, CPA, as chief financial officer, effective immediately.
Lipocine Inc. (Nasdaq:LPCN), a specialty pharmaceutical company, today announced its development plans for LPCN 1107, the company's oral hydroxyprogesterone caproate ("HPC") product candidate. Recently the company received a Type C written response from the U.S. Food and Drug Administration ("FDA") that outlined next steps in the development of the product as a potential treatment for the prevention of preterm birth ("PTB").
Metabolix, Inc. (Nasdaq:MBLX), an advanced biomaterials company focused on sustainable solutions for the plastics industry, today announced it will be exhibiting at Profiles 2015, an international business and technology conference on polymer materials in exterior building applications.
MRI Interventions, Inc. (OTCQB:MRIC), a commercial stage medical device company focused on creating innovative platforms for performing the next generation of minimally invasive surgical procedures in the brain, today announced the first stereotactic laser ablation of the hippocampus (SLAH) procedure at Yale-New Haven Hospital.
Nanosphere, Inc. (Nasdaq:NSPH), a company enhancing medicine through targeted molecular diagnostics, today announced today that it has entered into a definitive agreement with a dedicated healthcare institutional investor in a registered direct offering for $4.4 million of convertible preferred stock (which are convertible into a total of 1,203,830 shares of common stock at a conversion price of $3.655) and warrants to purchase shares of common stock exercisable for up to 1,203,830 additional shares of common stock, in the aggregate.
Praxsyn Corporation's (OTCQB:PXYN) anticipated business plan for 2015 is to acquire a sister subsidiary retail community pharmacy and to purchase additional pharmacies.
Prothena Corporation plc (Nasdaq:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs, today announced that Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development of Prothena, will highlight clinical results from a Phase 1 single ascending dose study of PRX002, as part of the late breaker abstract track, during a poster presentation at the 19th International Congress of Parkinson's Disease and Movement Disorders to be held June 14 – 18, 2015 in San Diego, CA.
Repros Therapeutics Inc.® (Nasdaq:RPRX) today announced that the Division of Bone, Reproductive and Urologic Products of the U.S. Food and Drug Administration (FDA) has scheduled the advisory committee to review the Company's New Drug Application (NDA) for its enclomiphene product candidate, formerly known as Androxal®, on November 3, 2015.
Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has completed patient enrollment in the pivotal Phase 3 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome).
XBiotech (Nasdaq:XBIT), the developer of True Human™ therapeutic antibodies, announced today that Kelly R. Thornburg has joined the company as Senior Vice President of Operations.
Zealand Pharma A/S (Nasdaq Copenhagen: ZEAL) announces that today, in a keynote symposium at the 75th Scientific Sessions of the American Diabetes Association (ADA) in Boston, data from the ELIXA Cardiovascular Safety Outcome Trial on lixisenatide was reported and discussed.