PlasmaTech Announces Agreement to Acquire Abeona; Arrowhead Presents Data on Potential RNAi Candidate Targeting Factor Print E-mail
By William Kent   
Wednesday, 06 May 2015 19:43
Below is a look at some of the headlines for companies that made news in the healthcare sector on May 6, 2015. 
PlasmaTech Biopharmaceuticals, Inc. (PTBI), a biopharmaceutical company advancing protein biologic therapies and oncology supportive care products, announced today that it has entered into a definitive agreement to acquire Abeona Therapeutics, a company engaged in the development and commercialization of therapies for patients with lysosomal storage diseases. Under the terms of the agreement, the Company will issue to Abeona Therapeutic members a total of 3,979,761 common shares upon closing of the transaction, and up to an additional $9 million in performance milestones, in common stock or cash, at the Company's option.
"We believe this acquisition represents an exciting cornerstone of our strategy to build a world class cell and gene therapy company focused on rare diseases," noted Steven H. Rouhandeh, PlasmaTech's Executive Chairman. "Abeona's programs in MPS IIIB & IIIA address a compelling unmet medical need with breakthrough technology and fundamental core competencies to develop products and accelerate value for patients and shareholders. This acquisition will accelerate development and internationalization of both clinical programs due to increased access to capital and organizational capabilities. Importantly, the Abeona operating and scientific teams will remain intact and we believe Tim Miller's unique blend of experience and capabilities makes him ideal to move the company forward towards our goal of becoming a leader in rare diseases. Additionally, Abeona recently closed on $4.8 million in financing from leading Sanfilippo Foundations worldwide, and have an additional $1 million commitment for manufacturing."
In connection with the acquisition, Tim Miller, Ph.D., President & CEO of Abeona will succeed Scott Schorer. "Tim Miller brings significant experience and leadership in developing and commercializing novel cell and gene therapies," said Steven H. Rouhandeh, Executive Chairman of PlasmaTech. "We want to thank Scott Schorer for his service as PlasmaTech's Chief Executive Officer, and look forward to his continued contributions to the Alpha-1 program as a consultant to the Company."
"We are excited about building the future together, and believe this is just the first of many steps to building a comprehensive product pipeline in the rare disease space. PlasmaTech is currently developing a new generation plasma processing technology which enables and validates orphan proteins requiring periodic treatments," noted Dr. Tim Miller. "We will continue to build on these strengths and capabilities, while executing on our world class gene therapy platform to address pressing rare diseases such as Sanfilippo syndrome." To learn more about Abeona, please click on the following link for a Web Interview with Tim Miller:
Abeona Therapeutics is developing gene therapies for patients with rare genetic diseases, with initial therapies being developed for patients with Sanfilippo syndromes (MPS III). The Company was formed in early 2013 to help focus the search for a cure for Sanfilippo Syndrome and provide a unifying voice between patient advocate groups, researchers, clinicians and investors. Dr. Miller, a co-founder, has over 16 years of scientific research, product development, clinical operations and business development expertise, with a focus on transitioning novel biotherapeutics through pre-clinical development and into Phase 1 and 2 human clinical trials. Previously with Juventas Therapeutics, SironRX Therapeutics, and Copernicus Therapeutics, Dr. Miller has focused on gene therapy and regenerative medicine. During his career, he has worked on therapies in cystic fibrosis, cardiovascular disease, wound healing, scar prevention and Sanfilippo syndromes. Dr. Miller has managed all aspects of biotechnology business development, manufacturing of biologics, and multiple clinical programs from start-up and has direct experience engaging Food and Drug Administration (FDA) and NIH advisory agencies on multiple Investigational New Drug (IND) submissions.
"We want to thank our supporting foundations and patient advocates, who have demonstrated that their voice is a critical component of accelerating rare disease programs," said Dr. Miller. Abeona Investors include U.S.-based Cure Sanfilippo Foundation, Sanfilippo Research Foundation, Team Sanfilippo, the Abby Grace Foundation, the National MPS Society, The Children's Medical Research Foundation, Inc., and the Ryan Foundation. Abeona has strong international support from the international Sanfilippo community, with investments from Spain-based Stop Sanfilippo and Sanfilippo B Foundation, Geneva, Switzerland-based Fondation Sanfilippo, Australian-based Sanfilippo Children's Foundation and Mexico-based Red Sanfilippo Foundation. Support for the program has also been provided by the Canadian led Sanfilippo Children's Research Foundation (SCRF).
The Board of Directors of PlasmaTech Biopharmaceuticals and the Managers of Abeona have unanimously approved the transaction. The transaction is expected to close in the second quarter of 2015, subject to customary closing conditions.
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today presented data at IBC's 17th Annual TIDES Conference in San Diego on the preclinical development of an RNAi therapeutic as a potential treatment for factor 12 (F12) mediated angioedemic and thromboembolic diseases. The presentation included data from in vitro screenings, in vivo evaluations, a disease model, and a multiple dose study in nonhuman primates. These data support advancement of ARC-F12 as a potential new candidate in Arrowhead's growing pipeline of RNAi-based therapeutics enabled by the company's Dynamic Polyconjugate (DPC) delivery platform. A copy of the presentation may be viewed on the Events and Presentations section of the company's website at
"We see factor 12 as an extremely attractive target to add to our pipeline. There is clear unmet need in thrombosis and angioedema and the biology of factor 12 as part of the coagulation cascade and the kinin-kallikrein system suggest that its reduction through RNAi may present opportunities in both disease areas," said Christopher Anzalone, Ph.D., president and chief executive officer. "We will be conducting additional studies in relevant disease models shortly to provide us with further data to decide on the advancement of ARC-F12 as a clinical candidate and initiation of IND enabling studies."
David Lewis, Ph.D., chief scientific officer, presented initial data from wild type mice, showing that various RNAi triggers selected from in vitro screening sets and co-administered with DPCs achieved significant and sustained knockdown of F12 levels of greater than 99% at nadir for most triggers. Strategic incorporation of various modifications to the most potent RNAi trigger increased the depth and duration of F12 knockdown activity as shown in dose response studies. In a study in mice, these modified triggers exhibited a dose-dependent increase in F12 knockdown. A single intravenous dose of 0.5 mg/kg reduced F12 by greater than 80%. When the dose was increased to 2 mg/kg, the reduction increased to greater than 95% at nadir, with greater than 70% knockdown observed at the one month time point. The lead RNAi trigger was also highly active in multiple dose nonhuman primate studies. With four intravenous doses of 2 mg/kg given once every four weeks, approximately 90% F12 knockdown was achieved after the first dose with even greater knockdown following subsequent doses. Knockdown was also highly durable with greater than 80% reduction maintained between monthly doses. The combination of RNAi trigger and DPC appeared to be generally well-tolerated and no drug-related changes in toxicity markers were observed as measured by clinical chemistry and hematologic parameters.
Dr. Lewis also presented data from a relevant disease model on the lead RNAi trigger and DPC combination. In this mouse model, thromboembolism is induced by exposure of carotid artery to ferric chloride. The time to blood flow occlusion is then measured as a clinically relevant indicator of physiological response to F12 knockdown. Animals were treated with saline or the lead RNAi trigger and DPC combination 15 days prior to ferric chloride challenge. Treated animals showed approximately 99% knockdown in serum F12 levels at Day 15 relative to baseline, while animals receiving saline showed no reduction. A dramatic increase in occlusion times as a measure of the inhibition of thrombus formation was observed in treated mice.
Arrowhead believes that ARC-F12 may present opportunities to target multiple diseases, including in thrombosis. The company is currently planning to investigate ARC-F12 in hereditary angioedema (HAE) as the first target indication. HAE is a rare genetic disorder with a prevalence of approximately 1/5,000-1/10,000 that is most commonly caused by mutations in the complement factor 1 esterase inhibitor gene (C1INH). Patients with HAE can experience recurrent and dangerous acute inflammatory attacks in multiple tissues, with attacks of laryngeal edema being particularly serious and potentially fatal. Current treatments seek to reduce the severity, duration, and frequency of acute HAE attacks, but frequent intravenous dosing of 1-3 times weekly is required and many patients do not respond adequately. Arrowhead believes the novel mechanism of ARC-F12 may fill an unmet need for patients and physicians who desire long term prophylaxis and may view intravenous dosing every 4-6 weeks as a significant advance.
The company is currently planning additional evaluation of ARC-F12 in relevant HAE disease models including C1INH knockout animals and captopril-induced vascular leak, among potential other studies to support advancement of ARC-F12 into IND enabling studies.
Also Wednesday:
Alphatec Holdings, Inc. (Nasdaq:ATEC), the parent company of Alphatec Spine, Inc., a global provider of spinal fusion technologies, announced today that the Company's President and Chief Executive Officer, Jim Corbett, will participate in the UBS 2015 Global Healthcare Conference on Tuesday, May 19, 2015 at 10:30 AM Eastern Time at the Sheraton New York – Times Square.
Avinger, Inc., (Nasdaq:AVGR) a developer and manufacturer of image-guided, catheter-based systems for the treatment of peripheral arterial disease (PAD) and pioneer of the lumivascular approach to treating vascular disease, today reported financial results for the first quarter ended March 31, 2015.
BioTelemetry, Inc. (Nasdaq:BEAT), the leading wireless medical technology company focused on the delivery of health information to improve quality of life and reduce cost of care, today reported results for the first quarter ended March 31, 2015.
ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company focused on autoimmune diseases, inflammatory disorders and cancer, today reported financial results for the first quarter ended March 31, 2015 and provided an update on the Company's corporate and clinical development activities.
Immunomedics, Inc., (Nasdaq:IMMU) today reported financial results for the third quarter ended March 31, 2015. The Company also highlighted recent key developments and planned activities for its clinical pipeline.
LifeVantage Corporation (Nasdaq:LFVN) today reported financial results for its third quarter and nine months ended March 31, 2015.
LHC Group, Inc. (Nasdaq:LHCG), a national provider of home health, hospice and community-based and comprehensive post-acute healthcare services, today announced its financial results for the three months ended March 31, 2015.
MacroGenics, Inc. (Nasdaq:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, today provided a corporate progress update and reported financial results for the first quarter ended March 31, 2015.
MannKind Corporation (Nasdaq:MNKD), focused on discovering, developing and commercializing therapeutic products for diabetes, announced today that it will present at the Bank of America Merrill Lynch 2015 Healthcare Conference on Wednesday, May 13, 2015 at 12:00 pm Pacific Time at the Encore at the Wynn Hotel in Las Vegas, Nevada.
NanoString Technologies, Inc. (Nasdaq:NSTG), a provider of life science tools for translational research and molecular diagnostic products, today reported financial results for the first quarter ended March 31, 2015.
PlasmaTech Biopharmaceuticals, Inc. (NASDAQ: PTBI), announced today that it has entered into a definitive agreement with one institutional investor in a private placement of approximately $10 million of common stock at a price of $8.00 per share, or 1,250,000 shares.
PRA Health Sciences, Inc. (Nasdaq:PRAH) today reported financial results for the quarter ended March 31, 2015.
Premier, Inc. (Nasdaq:PINC), a leading healthcare improvement company, today announced that its President and CEO Susan DeVore will present at the Bank of America Merrill Lynch 2015 Health Care Conference in Las Vegas on Wednesday, May 13, 2015, at 3:40 p.m. PDT.
SANUWAVE Health, Inc. (OTCQB:SNWV) today announced that the independent Data Monitoring Committee (DMC) has performed an interim analysis on the efficacy and safety results in the Phase III supplemental clinical trial using the dermaPACE® for treating diabetic foot ulcers.
Stereotaxis, Inc. (Nasdaq:STXS), a global leader in innovative technologies for the treatment of cardiac arrhythmias, today reported financial results for the first quarter ended March 31, 2015.
Transcat, Inc. (Nasdaq:TRNS), a leading provider of accredited calibration and compliance services and distributor of professional grade handheld test, measurement and control instrumentation, announced today that it will release its fourth quarter and full fiscal year 2015 results after the close of financial markets on Tuesday, May 19, 2015.
Versartis, Inc. (Nasdaq:VSAR), an endocrine-focused biopharmaceutical company that is developing VRS-317, a novel, long-acting form of recombinant human growth hormone (rhGH) for growth hormone deficiency (GHD), today provided an update on its clinical programs for VRS-317 and announced financial results for the first quarter ended March 31, 2015.
Versartis, Inc. (Nasdaq:VSAR), an endocrine-focused biopharmaceutical company that is developing VRS-317, a novel, long-acting form of recombinant human growth hormone (rhGH) for growth hormone deficiency (GHD), today announced that Mr. Jay Shepard, President and Chief Executive Officer, is scheduled to present at the Bank of America Merrill Lynch 2015 Health Care Conference on Wednesday, May 13, 2015 at the Encore at Wynn in Las Vegas, Nevada.

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