Bio Roundup: Medicare Drug Prices, FDA Moves, Gene Therapy News & More

Xconomy National — 

Two imminent FDA rulings on two separate drugs aren’t just noteworthy for the patients they’ll help, but the long and winding road their developers have taken to get to this point.

First is Alnylam Pharmaceuticals, which has spent 16 years and over $2 billion to try to bring an unproven form of medicine, RNA interference (RNAi), to market. A positive FDA decision, which could come by Saturday, would mark RNAi as a bona fide drug-making method, and Alnylam as its standard-bearer.

Then there’s Amicus Therapeutics. The company’s on-again, off-again ambitions for FDA approval of a Fabry disease drug would make a fitting sequel to the 2010 Hollywood flick about its origins, Extraordinary Measures. The most recent twist: a drug approval made possible only because the FDA unexpectedly rescinded demands for an additional study that would have taken several years. Xconomy has documented several steps of the journey, which should wrap up by Monday with an FDA decision.

In other news, Medicare Advantage plans just got a new tool to negotiate drug prices, the hemophilia gene therapy race sped up, and a New York congressman faces insider trading charges. Time to wrap up this week’s biotech saga.


—Starting next year, The Centers for Medicare & Medicaid Services will give Medicare Advantage plans, which cover about 20 million seniors in the U.S., new tools to negotiate lower drug prices for the Medicare Part B drugs given in hospitals and doctor’s offices. One of them is to initiate “step therapy,” in which patients would have to fail a cheaper treatment before moving on to a more expensive one. Here’s more from Bloomberg.

—GOP Congressman Chris Collins (R-NY) and two others, including his son Cameron, were arrested and indicted on charges of insider trading in the Australian biotech Innate Immunotherapeutics. Collins denied any wrongdoing. CNBC has more.


—The FDA cleared a generic version of potassium chloride for hypokalemia, which causes dangerously low potassium levels. The drug, from Athenex (NASDAQ: ATNX), is the first to market after getting the FDA’s Competitive Generic Therapy designation, a new tool to speed the development of generics.

—The agency also approved mogamulizumab-kpkc (Poteligeo), from Kyowa Kirin, to treat rare non-Hodgkin lymphomas.

—The FDA also OK’d the cystic fibrosis medicine ivacaftor/lumacaftor (Orkambi), from Vertex Pharmaceuticals (NASDA; VRTX), for the estimated 1,300 children between two and five years old who have the most common form of the disease. The drug is already approved for adults.

—Ampio Pharmaceuticals (NASDAQ: AMPE) shares plunged 78 percent after the company disclosed that the FDA wants it to run another trial of its osteoarthritis drug Ampion before filing for U.S. approval.

—An FDA advisory panel voted to recommend U.S. approval of omadacycline, an experimental antibiotic from Paratek Pharmaceuticals’s (NASDAQ: PRTK), for bacterial skin infections and hospital-acquired pneumonia. The FDA could approve the drug by early October.

—Nearly two months after an FDA advisory panel panned an extended-release version of oxycodone (Remoxy) from Pain Therapeutics (NASDAQ: PTIE), the regulator rejected the drug. The Austin, TX-based will now restructure.


—Shares of Spark Therapeutics (NASDAQ: ONCE) fell nearly 30 percent after the latest update on the company’s experimental gene therapy for hemophilia. Spark plans to move its gene therapy into Phase 3 testing, but shares fell on investors’ concerns over how it stacks up to a rival therapy from BioMarin Pharmaceutical (NASDAQ: BMRN), STAT reports. Here’s more on the long-term questions surrounding hemophilia gene therapy.

—Sangamo Therapeutics (NASDAQ: SGMO) provided the first glimpse of human data from its experimental gene editing treatment for hemophilia A.

—An experimental gene therapy from RegenxBio (NASDAQ: RGNX) for age-related macular degeneration showed early, positive signs in a Phase 1 study. But an update from a second gene therapy for a genetic disease that causes high cholesterol was less encouraging. Here’s more from Forbes.

Nature published two editorials critiquing the controversial work of an Oregon researcher who used CRISPR technology to edit out a genetic mutation in human embryos. That researcher also updated the results this week. Here’s more from MIT Technology Review.


—Ambys Medicines, of Redwood City, CA, started up with a $60 million Series A and an unusual, broad alliance with Japanese pharma Takeda to develop an array of drugs for liver diseases. These early, wide-ranging partnerships between biotechs and pharma companies have previously proven too limiting for small biotechs; Xconomy spoke with CEO Jeff Tong about the pros and cons of such deals.

—Regeneron Pharmaceuticals (NASDAQ: REGN) invested $100 million in Bluebird Bio (NASDAQ: BLUE) as part of an alliance to co-develop a range of cell therapies for cancer.

—San Diego biotech Samumed, which is developing regenerative medicines for diseases like cancer and osteoarthritis, raised an eye-popping $438 million in its latest financing round. The privately held company is worth $12 billion. Results from a Phase 2 study in osteoarthritis will be released soon, the San Diego Union-Tribune reports.

—SQZ Biotechnologies closed a $72 million Series C round to continue advancing its experimental cell therapies for solid tumors and autoimmune diseases.

–Boston-based Akouos raised $50 million in a Series A financing round to support work on a gene therapy for hearing loss that it hopes to advance into human testing within three years.

—Sarepta Therapeutics (NASDSAQ: SRPT) invested $30 million in privately held Lacerta Therapeutics and got an option to license up to three of the startup’s experimental gene therapies for neurological diseases.

—Struggling women’s fertility company Ovascience (NASDAQ: OVAS), which trades at less than $1 a share, will merge with privately held Millendo Therapeutics, of Ann Arbor, MI, in an all-stock deal. The combined company will be 80 percent owned by Millendo shareholders, led by Millendo CEO Julia Owens, and focus on drugs for rare endocrine disorders.

—Citing disappointing “market test results,” Ironwood Pharmaceuticals (NASDAQ: IRWD) ended a 2016 deal with AstraZeneca (NYSE: AZN) for the struggling gout drug lesinurad (Zurampic) and returned its rights to the drug to the British pharma. Ironwood will ax 125 employees, mostly in sales.


—Shares of Ovid Therapeutics (NASDAQ: OVID) fell 30 percent on Phase 2 results for an experimental drug for the rare disease Angelman Syndrome. The study met its main goal, safety, but the benefits of the drug so far are unclear. Here’s more from Forbes.

—Protagonist Therapeutics (NASDAQ: PTGX) will continue developing its experimental ulcerative colitis drug PTGX-100 after an independent re-analysis of its Phase 2 data suggested “signals of clinical efficacy” and an error in the initial assessment in March. Shares climbed more than 30 percent.


—This week we profiled the finalists for Xconomy’s Newcomer, Young Innovator, and Commitment to Diversity awards.

Frank Vinluan contributed to this report.