|BioMarin Completes Rolling NDA Submission to FDA for Drisapersen; MannKind to Manufacture 12 Unit Cartridge Strength of AFREZZA(R)|
|By William Kent|
|Monday, 27 April 2015 19:10|
Below is a look at some of the headlines for companies that made news in the healthcare sector on April 27, 2015.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen, an investigational exon-skipping drug candidate for the treatment of the largest genetically defined subset of Duchenne muscular dystrophy (DMD). 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. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. The company intends to also submit an application for registration in the European Union in summer 2015.
"We believe drisapersen may offer a meaningful benefit to boys living with DMD whose mutations are amenable to exon 51 skipping. The totality of data on drisapersen contains three randomized, placebo-controlled, efficacy trials and two long term extension studies, which include some boys treated for approximately 3.4 years," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. "With this application, BioMarin continues in its long-standing tradition of developing important therapies for those who are most in need. The submission of the NDA represents a significant milestone for BioMarin, and we appreciate the strong, collaborative effort of many hard working employees, investigators, patients and their families. We look forward to working with the U.S. Regulatory Authorities to thoroughly understand the data generated for this heterogenous and critically ill patient population and hopefully to bring this treatment to patients expeditiously."
Drisapersen has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designation by the FDA.
DMD is caused by a mutation in the gene that encodes for dystrophin, a protein that is important in connecting the cytoskeleton of muscle fibers to the extracellular matrix. Its deficiency in DMD leads to progressive muscle weakness, loss of ambulation in early adolescence, and typically death due to pulmonary or cardiac insufficiency in the late twenties. Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures. There is currently no approved therapy in the United States for DMD.
"This is a first for the Duchenne community, and we are filled with hope that there could be a treatment for Duchenne in the United States," said Debra Miller, co-founder and CEO of CureDuchenne. "CureDuchenne has been supporting the development of drisapersen for more than a decade, and we are delighted that BioMarin has reached this important stage. We salute the researchers who have been working so hard, and we share their determination to find a cure for Duchenne."
MannKind Corporation (NASDAQ: MNKD) announced it will begin to manufacture a 12 unit cartridge strength of AFREZZA (insulin human) Inhalation Powder to improve glycemic control in adult patients with diabetes. The 12 unit cartridge was approved by the U.S. Food and Drug Administration on April 17, 2015 and is expected to be launched by Sanofi in the second half of 2015.
The new dosage strength complements the existing 4 unit and 8 unit cartridges and will provide patients with another option to receive their prescribed dose.
Aquinox Pharmaceuticals, Inc. (Nasdaq:AQXP), a clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology, announced today that David Main, Aquinox's President & CEO, will present a corporate overview at the upcoming 2015 Bloom Burton & Co. Healthcare Investor Conference on Tuesday, May 5th at 11:00 AM Eastern Time.
Cellular Biomedicine Group Inc. (Nasdaq:CBMG), a biomedicine firm engaged in the development of effective treatments for degenerative and cancerous diseases, today announced that management will present the Company's Chimeric Antigen Receptor T cell (CAR-T) therapy for cancer clinical development programs at the previously announced 10th Annual World Stem Cells & Regenerative Medicine Congress to be held at the Business Design Centre in London, UK on May 20-22, 2015.
Chembio Diagnostics, Inc. (Nasdaq:CEMI), which develops, manufactures, markets and licenses point-of-care diagnostic tests, announced today that the Company will release financial results for the first quarter of 2015, before the opening of the market on Thursday, May 7, 2015.
Coronado Biosciences, Inc., (Nasdaq:CNDO) today announced that it has changed its name to Fortress Biotech, Inc. The Company's new NASDAQ stock ticker symbol is FBIO, which will be effective at the open of market on Tuesday, April 28, 2015.
FutureWorld Corp. (OTC: FWDG), a leading provider of advanced technologies and solutions to the global cannabis industry, announces today that its subsidiary, FutureLand Corp, expands its corporate reach in the States of Washington, Oregon, California and Nevada for the purpose of facilitating land leases and building grow facilities in conjunction with its partner Second Sun LLC.
GW Pharmaceuticals plc (AIM:GWP) (Nasdaq:GWPH), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announced today that it intends to sell, subject to market and other conditions, 1,250,000 million American Depositary Shares ("ADSs") representing ordinary shares of GW on the NASDAQ Global Market in an underwritten U.S. public offering.
Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX), a biopharmaceutical company focused on bringing innovative therapies to market for patients with renal disease, today announced that a conference call will be held on Monday, May 4th, 2015 at 8:00 a.m. ET to discuss financial results for the first quarter 2015 and provide an update on the Auryxia™ (ferric citrate) launch.
NeoStem, Inc. (Nasdaq:NBS), a biopharmaceutical company developing novel cell-based individualized medicine therapies, announced today that the European Medicines Agency (EMA) has classified the Company's lead immuno-oncology product candidate, NBS20, as an Advanced Therapeutic Medicinal Product (ATMP). ATMP classification, which is approved by the Committee for Advanced Therapies (CAT), was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products and provide a benchmark for the level of quality compliance for pharmaceutical practices.
Otonomy, Inc. (Nasdaq:OTIC), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the inner and middle ear, today announced that results from two identical Phase 3 trials of AuriPro™ were presented at the annual American Society of Pediatric Otolaryngology (ASPO) meeting, held during the Combined Otolaryngology Spring Meetings in Boston.
Pacific Biosciences of California, Inc., (Nasdaq:PACB) provider of the PacBio® RS II Sequencing System, today announced the introduction of several new solutions for targeted sequencing applications.
Pazoo, Inc. (OTC Pinks:PZOO) (German WKN#:A1J3DK), is pleased to announce that as of today and more than a week ahead of schedule, the company has completely repaid the total of its obligations on its Convertible Promissory Note to Eastmore Capital LLC. Pazoo announced on April 15 its intentions to pay off Eastmore Capital within the next few weeks.
PetMed Express, Inc. (Nasdaq:PETS) will announce its financial results for the fiscal year ended March 31, 2015 on Monday, May 4, 2015 at 8:00 A.M. Eastern Time, then at 8:30 A.M. Eastern Time, Menderes Akdag, the Company's Chief Executive Officer and President, will host a conference call to review the financial results.
Premier, Inc. (Nasdaq:PINC) plans to report fiscal 2015 third-quarter financial results after the market closes on Monday, May 11, 2015, followed by a conference call at 5:00 p.m. ET to discuss the results.
Protea Biosciences Group, Inc. (OTCQB:PRGB) announced today that it has closed on total gross proceeds of $7,415,551 million from the private placement offering of preferred stock and 3-year warrants to existing and new accredited investors, which included the conversion of $2,146,977 of existing loans from shareholders.
ScripsAmerica, Inc. (OTCBB:SCRC), a leading provider of a range of specialty prescription and over the counter pharmaceuticals and medical supplies, today announced that SeeThruEquity, a leading independent equity research and corporate access firm focused on smallcap and microcap public companies, has issued an update to the coverage it initiated in 2014.
StemCells, Inc. (Nasdaq:STEM) announced today that Martin McGlynn, CEO of StemCells, Inc., will participate on a panel moderated by Alice Park, writer for TIME Magazine, at the inaugural World Medical Innovation Forum to be held in Boston, April 27-29, 2015.
Vital Therapies, Inc. (Nasdaq:VTL), a biotherapeutic company developing ELAD®, a cell-based therapy targeting the treatment of liver failure, today announced the presentation of two posters at the recent annual meeting of the European Association for the Study of the Liver (EASL), as well as the date and details of the Company's first quarter 2015 financial results conference call.
ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, today announced the initiation of a Phase 1b/2 study of Ad-RTS-hIL-12 + veledimex following standard chemotherapy for the treatment of patients with locally advanced or metastatic breast cancer.
Zogenix, Inc. (Nasdaq:ZGNX), a pharmaceutical company developing and commercializing products for the treatment of central nervous system (CNS) disorders, announced today that it is implementing its succession plan as the Company focuses on advancing its late-stage product development pipeline.