|Ignyta Announces Collaboration with UCSF For Clinical Trial of Entrectinib; Strensiq Receives Marketing Approval for Hypophosphatasia|
|By Josh Gee|
|Monday, 06 July 2015 19:57|
Below is a look at some of the headlines for companies that made news in the healthcare sector on July 2, 2015.
Ignyta, Inc. (NASDAQ: RXDX), announced a clinical collaboration with the University of California, San Francisco (UCSF), under which UCSF will study entrectinib in a proof-of-concept clinical trial in cancer patients with metastatic melanoma that is positive for activating alterations to NTRK1/2/3 (encoding TrkA/TrkB/TrkC) or ROS1.
"We are excited to collaborate with UCSF, a world-renowned academic research institution," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "The focus on melanoma in this study will complement the broader range of indications on which we are focused in our own clinical trials, and we expect the findings to accelerate our understanding of the potential role of entrectinib in treating patients with NTRK-positive and ROS1-positive cancers."
Under the terms of the collaboration agreement, Ignyta will contribute $1 million toward the funding of the clinical trial, as well as per-patient fees based on enrollment of NTRK-positive or ROS1-positive patients and their participation in the trial. Ignyta will also provide UCSF with sufficient supply of entrectinib for use in the clinical trial. In addition to the safety and efficacy data from the trial, UCSF will provide Ignyta with tumor samples and genetic sequencing data for patients screened for inclusion in the trial for further genomic analysis.
The study is a multicenter, open label umbrella trial designed by UCSF to obtain proof-of-concept data in patients with metastatic melanoma that is positive for molecular alterations in specific tyrosine kinase receptors. UCSF will exclusively use entrectinib for patients enrolled in the clinical trial having activating molecular alterations to NTRK1/2/3 or ROS1.
Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced Japan's Ministry of Health, Labour and Welfare (MHLW) approved the Company's New Drug Application (NDA) for the use of Strensiq® (asfotase alfa) as a treatment for patients in Japan with hypophosphatasia (HPP), a life-threatening, ultra-rare metabolic disorder. Strensiq, a bone-targeted enzyme replacement therapy, is the first therapy approved in Japan for the treatment of patients with HPP. Alexion expects that initial patients with HPP in Japan will start commercial treatment with Strensiq by late Q3 2015.
"The rapid approval of the Strensiq NDA in Japan underscores the devastating nature of HPP and the life-transforming impact that Strensiq can provide to Japanese patients living with HPP," said David Hallal, Chief Executive Officer of Alexion. "We are delighted that this regulatory approval in Japan marks the first treatment option for patients with HPP, and we look forward to urgently working with the healthcare authorities to make Strensiq available to Japanese patients who can benefit from this therapy. I would also like to thank the investigators, patients, and their families in Japan who participated in the clinical trial that led to this approval."
HPP is a genetic, progressive, ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications. It is characterized by defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, seizures, pain, and respiratory failure leading to premature death in infants.1-5 As reflected in the prescribing information in Japan, infants with HPP treated with Strensiq had 84% overall survival, as estimated by Kaplan-Meier analysis, at 168 weeks.
"Hypophosphatasia is an ultra-rare disease with diverse clinical symptoms that may be difficult to diagnose. It can be a lethal disease in Japanese newborns and infants which has led to significant challenges since there have been no approved treatment options," said Professor Ozono, Department of Pediatrics Osaka University. "I am greatly delighted that the first treatment has been approved for HPP. The patients and physicians in Japan who participated in the Strensiq clinical trials have played a critical role in generating valuable data and we appreciate their contributions in enabling the approval of Strensiq. I look forward to using Strensiq in clinical practice and continuing to advance the understanding of HPP diagnosis and treatment."
"Today's approval marks a major turning point for patients and their families in Japan who have waited a long time for a treatment for hypophosphatasia," said Mr. Hara, Director of HypoPhosPhatasia Support Association of Japan. "The approval of Strensiq offers great hope to patients who previously suffered in the absence of an effective therapy, as well as to the healthcare professionals and families who care for and support them."
Alexion has submitted a Biologics License Application for Strensiq with the U.S. Food and Drug Administration, which was accepted for priority review, and received a positive CHMP opinion recommending marketing authorization for Strensiq for patients with pediatric-onset HPP in Europe. Regulatory decisions in the U.S. and Europe are expected in the second half of 2015.
Clinical Data -- The approval of Strensiq in Japan was based on clinical data from three pivotal prospective studies and their extensions, a retrospective natural history study in infants, and one investigator-sponsored study in Japan. The pivotal studies comprised 71 patients, including five Japanese patients, with infantile and juvenile-onset HPP (ages 1 day to 65 years). Study results showed that patients with infantile-onset HPP (ages ≤5 years at enrollment) treated with Strensiq demonstrated rapid and sustained improvements in bone mineralization, as measured by the Radiographic Global Impression of Change (RGI-C) scale, which evaluates the severity of rickets based on X-ray images. In addition, infants with HPP treated with Strensiq had 84% overall survival, as estimated by Kaplan-Meier analysis, at 168 weeks. Patients with juvenile-onset HPP treated with Strensiq demonstrated superior improvements in bone health compared to a control group of HPP patients selected from a natural history database, as well as improvements in ambulation, physical function and growth.
The most frequently reported adverse events observed with Strensiq treatment in clinical studies were injection site reactions and injection-associated reactions. Most of these adverse events were mild to moderate in severity. Serious injection-associated reactions were reported in two patients, with neither patient discontinuing Strensiq treatment: one patient with infantile-onset HPP reported fever and chills, and one patient with juvenile-onset HPP reported numbness of lips, leg pain, chills, and headache.
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that Sue Washer, President and CEO will present at the Cantor Fitzgerald Healthcare Conference on July 8, 2015 at 10:15am in New York.
athenahealth, Inc. (NASDAQ:ATHN), a leading provider of cloud-based services and mobile applications for medical groups and health systems, today announced that it will hold a conference call for investors on Friday, July 24 2015, at 8:00 a.m. ET.
athenahealth, Inc. (Nasdaq:ATHN), a leading provider of cloud-based services and mobile applications for medical groups and health systems, today announced that Epocrates, athenahealth's well-loved source of clinical intelligence, topped the rankings.
BG Medicine, Inc. (Nasdaq:BGMD), the developer of the BGM Galectin-3® Test, today announced that the ARCHITECT® Galectin-3 assay is now available.
BioCorRx, Inc. (BICX), developer of the Start Fresh Program, announces update on the Company's pilot program with Myriad Medical Marketing (MMM).
BioRestorative Therapies, Inc. (OTCQB:BRTX), a life sciences company focused on adult stem cell-based therapies for various personal medical applications, today announced a 1-for-20 reverse split of its common stock in preparation for the planned listing of its common stock on the NASDAQ Capital Market.
Capnia, Inc. (NASDAQ:CAPN), focused on the development and commercialization of novel products based on its proprietary technologies for precision metering of gas flow, today announced that it has signed a definitive agreement to buy out CoSense® royalty rights originally held by BioMedical Drug Development, Inc. (BDDI).
After nearly two decades of collaboration to shape University of Missouri Health Care into a national leader in health information technology, the University of Missouri (MU) and Cerner (Nasdaq:CERN), a global leader in health care technology, today announced plans to extend their relationship through 2025.
Digital Power Corporation (NYSE MKT:DPW) announced the release of its new advanced capacitor chargers product family for powering laser systems in medical and industrial applications.
The Ensign Group, Inc. (NASDAQ:ENSG), the parent company of the Ensign™ group of skilled nursing, rehabilitative care services, home health care, hospice care, assisted living and urgent care companies, announced today that Cornerstone Healthcare, Inc., Ensign's home health and hospice portfolio subsidiary, had acquired Buena Vista Hospice, a Medicare and Medi-Cal certified hospice agency serving the Ventura County area from an office in Westlake Village, California.
Express Scripts Holding Company (Nasdaq:ESRX) announced today its intention to release its second quarter earnings on Tuesday, July 28, 2015 after the market closes and will hold its quarterly conference call to discuss second quarter results on Wednesday, July 29, 2015, at 8:30 a.m. Eastern Time (7:30 a.m. Central Time).
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics, today announced that Jozef Gecz, Ph.D., Professor of Human Genetics at the Department of Pediatrics, University of Adelaide, published data in the June issue of Human Molecular Genetics, supporting the role for neurosteroids in the pathophysiology of PCDH19 female epilepsy.
Merit Medical Systems, Inc. (NASDAQ:MMSI), a leading manufacturer and marketer of proprietary disposable medical devices used in interventional and diagnostic procedures, particularly in cardiology, radiology and endoscopy, announced today that the Company will release its financial results for the second quarter ended June 30, 2015, after the close of the stock market on Thursday, July 23, 2015.
Molina Healthcare, Inc. (NYSE:MOH) today announced that it will report its earnings for the second quarter and six months ended June 30, 2015, after the market closes on Thursday, July 30, 2015.
NewLink Genetics Corporation (NASDAQ:NLNK) today announced that it will present at the Cantor Fitzgerald Healthcare Conference on Wednesday, July 8, 2015 at 3:45 p.m. ET at the Le Parker Meridien in New York City.
OncoSec Medical Inc. (Nasdaq:ONCS), a company developing DNA-based intratumoral cancer immunotherapies, will visit the Nasdaq MarketSite in Times Square, Tuesday, July 7, 2015 – 9:15 a.m. to 9:30 a.m. ET, to celebrate the company's listing on the The Nasdaq Stock Market.
RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL), an Israeli biopharmaceutical company focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced that it has received regulatory authorization to commence patient enrollment in Australia and New Zealand for its ongoing Phase III study with RHB-104 for Crohn's disease (the MAP US study), and has commenced patient screening in New Zealand.
TearLab Corporation (NASDAQ:TEAR) (TSX:TLB) announced the appointment of Wes Brazell as its Chief Financial Officer, with overall responsibility for operational finance, financial reporting, budgeting and strategic planning, as well as helping to manage the Company's relationships and interactions with the investment community.
XBiotech Inc. (NASDAQ:XBIT), world's leading developer of next-generation True Human™ therapeutic antibodies, announced today treatment of first patient in its Phase 1/2 clinical study.
Zosano Pharma Corporation (Nasdaq:ZSAN) announced today that Novo Nordisk (NVO) has notified Zosano of its intention to discontinue the collaboration agreement related to development of a transdermal presentation of select NVO glucagon-like peptide-1 (GLP-1) analogues.