Target $160
Tuesday, June 18, 2019
Alnylam OKd in Japan of ONPATTRO for Hereditary ATTR Amyloidosis
– ONPATTRO is the First RNAi Therapeutic Approved in Japan –
− Approval Marks the Arrival of an Entirely New Treatment Approach for People Living with hATTR Amyloidosis with Polyneuropathy in Japan –
Alnylam Pharmaceuticals, Inc. (ALNY), the leading RNAi therapeutics company, today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved ONPATTRO® (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. ONPATTRO is the first approved RNAi therapeutic in Japan and Alnylam will launch and directly market it in the country.
“hATTR amyloidosis is a genetic disease that can cause a variety of debilitating symptoms and can often be fatal. Historically, treatment options for people living with this condition in Japan, where the V30M mutation of the condition is prevalent, were limited,” said Professor Yukio Ando, President of ISA (International Society of Amyloidosis) and JSA (Japan Society of Amyloidosis), Chairman and Professor/Department of Amyloidosis Research of Nagasaki International University and Professor Emeritus and Visiting Professor of Kumamoto University, Japan. “The approval of ONPATTRO, a treatment that has the potential to mitigate and potentially reverse symptoms of polyneuropathy and to improve quality of life, marks a paradigm shift in the way we approach and treat this serious disease.”
“We are very excited about the approval of ONPATTRO and are proud to be bringing this important new treatment to patients with hATTR amyloidosis with polyneuropathy in Japan,” said Masako Nakamura, Senior Vice President, Head of Asia, Alnylam. “With this approval, we look forward to working with the hATTR amyloidosis community to continue to raise disease awareness, increase diagnosis rates and ultimately provide treatment to patients suffering from this devastating disease. This is a significant milestone in our efforts to bring RNAi therapeutics to people around the world and we look forward to continuing to build our presence in Asia.”
Abeona Gets FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy
Abeona Therapeutics Inc. (ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its ABO-202 program. ABO-202, the Company’s novel one-time AAV9 gene therapy for CLN1 disease, is designed to deliver a functional copy of the PPT1 gene to the central nervous system and peripheral organs using a combination of intravenous and intrathecal administrations. Abeona is preparing to initiate a Phase 1/2 clinical trial evaluating ABO-202 in patients with CLN1 disease and will provide guidance on the timing of the trial later this year.
“Receiving Fast Track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” said João Siffert, M.D., Chief Executive Officer.
ABO-202 is administered as a one-time adeno-associated virus 9 (AAV9) gene therapy that delivers a functional copy of the PPT1 gene to cells of the central nervous system and peripheral organs. This enables cells to produce a functioning PPT1 enzyme, which is critical for proper metabolism in lysosomes. The absence of this enzyme in patients with CLN1 disease results in malfunctioning cells, including brain cells, neuroinflammation, and neurodegeneration. In preclinical studies, ABO-202 normalized survival and improved neurological function in CLN1 mice. These studies also showed that a combination of intravenous and intrathecal administrations of ABO-202 improved efficacy over either delivery route alone, and that early treatment significantly improved outcomes.
CymaBay OKd to Start Seladelpar Study on Liver Disease
CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on developing and providing access to innovative therapies for patients with liver and other chronic diseases, today announced FDA clearance of the company’s Investigational New Drug Application (IND) for seladelpar to treat primary sclerosing cholangitis (PSC). The company intends to initiate a Phase 2 study to evaluate the safety, tolerability, and efficacy of seladelpar in patients with PSC in the third quarter.
PSC is a rare, chronic cholestatic liver disease that is characterized by diffuse inflammation and fibrosis of the bile ducts. The disease predominantly affects the medium to large-sized bile ducts inside and outside the liver and is manifested by ongoing ductal destruction leading to cholestasis, advanced fibrosis, and cirrhosis. There are currently no FDA-approved treatments for PSC. Seladelpar is an orally administered, potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist that is also in development for primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). In clinical studies, seladelpar has demonstrated anti-cholestatic and anti-inflammatory activity, suggesting the potential of seladelpar as a therapeutic option for the treatment of PSC.
The planned Phase 2 study will be a randomized, placebo-controlled, dose-ranging study that will enroll approximately 100 patients at 60 sites globally. Seladelpar at doses of 5, 10, and 25 mg once daily will be studied versus placebo in a 1:1:1:1 randomization. The primary efficacy outcome will be the relative change in alkaline phosphatase (AP) from baseline at 24 weeks.
“PSC patients currently have significant unmet need for effective treatment options. I am encouraged by the effects of seladelpar observed in the PBC clinical program, such as the anti-cholestatic and anti-inflammatory activity as well as the favorable safety and tolerability profiles, which suggest a potential benefit for PSC patients,” said Dr. Cynthia Levy, Division of Hepatology, University of Miami.
Dr. Pol Boudes, Chief Medical Officer of CymaBay, commented, “The initiation of a Phase 2 study of seladelpar in PSC is a significant step forward in addressing the high unmet need that exists for PSC patients. As we continue to progress the Phase 3 development of seladelpar in PBC, it is important to expand the potential indications where the drug may have clinical activity. We are eager to commence this dose-ranging study to further evaluate our hypothesis that seladelpar can improve the clinical course for patients with PSC.”
ANI Expands Generic Pipeline, Acquiring 7 Coeptis Development Stage Products
ANI Pharmaceuticals, Inc. (“ANI”) (Nasdaq: ANIP) today announced that it has acquired a pipeline of seven development stage generic products from Coeptis Pharmaceuticals, Inc. for $2.3 million dollars in cash and up to $12.0 million in additional development and commercial milestones. As part of the agreement, ANI will pay a portion of gross profit generated from one of the injectable products to the commercial manufacturing partner. Included in the purchase price, ANI is also acquiring raw materials (API), manufacturing and packaging components, and reference drug valued at approximately $1.5M combined. The portfolio includes three oral and four injectable products and has a combined current annual U.S. market value of $1.02 billionaccording to IQVIA and ANI estimates.
Six of the products are currently in development at third parties with commercial manufacturing sites already established. The seventh product, a liposomal-based injectable, is in development at a third party and ANI will seek to identify a commercial manufacturing partner in the near-term.
Arthur S. Przybyl, ANI’s President and CEO stated, “We are excited to add these seven drugs to our generic product pipeline. Importantly, our growing injectable pipeline now stands at six drugs. With strong cash flows and a healthy balance sheet, ANI is well positioned to continue pursuing business development opportunities to further expand our brand, generic and contract manufacturing business segments.”
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