Bio Roundup: EASL Does It, Alnylam’s BFF, Gene Therapy Deals & More

Xconomy National — 

A major conference devoted to liver disease kicked off with updates from the race for approval of the world’s first drug to treat a form of advanced liver disease called nonalcoholic steatohepatitis, or NASH. The gene therapy field continued to attract venture cash and industry interest, and it was a busy week in Washington for healthcare policy and regulation. Congress grilled industry executives about drug prices, Bernie Sanders unveiled sweeping single-payer Medicare reform, and the FDA weighed in on several pharma pipelines.

It’s been a busy week, and the biggest biopharma news of the week could happen after you read this. Bristol-Myers Squibb shareholders are due to vote Friday on Bristol’s massive Celgene acquisition. Whatever happens, we’ve got you covered. They don’t call it a roundup for nothing.


—At the annual EASL International Liver Congress, Intercept Pharmaceuticals (NASDAQ: ICPT) of New York released a subset of data from its Phase 3 REGENERATE trial for its obeticholic acid to treat NASH, an advanced fatty liver disease. While likely to gain FDA approval, the first for a NASH drug, analysts questioned how broadly the drug would be used or reimbursed.

—Also at EASL, Viking Therapeutics (NASDAQ: VKTX) filled out details of a positive Phase 2 NASH study. The firm got a boost when the study abstract was released two weeks ago.

—At EASL this weekend, Alnylam Pharmaceuticals (NASDAQ: ALNY) will disclose details of the Phase 3 study of givosiran. It could become the first treatment for acute hepatic porphyria, a debilitating rare genetic disease. Without disclosing specifics, Alnylam touted success in March, but side effects dampened the outlook.

—Gilead Sciences (NASDAQ: GILD) had updates on its NASH, hepatitis B, and hepatitis C programs, but it was overshadowed by a Stat report that the firm is laying off 20 percent of its sales force.


—Pharmacy benefit manager (PBM) companies took their turn testifying before the Senate finance committee, which asked executives about the role their firms play in the high cost of prescription drugs.

—Three PBMs joined three drug companies at a House of Representatives subcommittee hearing on the rising cost of insulin that is forcing patients to ration their doses. Meanwhile, Sanofi (NYSE: SNY) announced it would charge $99 per month for any combination of 10 vials and injection pens, nearly $250 lower than it charges now.

—U.S. prosecutors charged British drug developer Indivior with a scheme to market opioid-addiction drugs, among them Suboxone, as safer and less addictive than cheaper generics. Here’s more from NPR.

—Presidential hopeful Sen. Bernie Sanders (I-VT) unveiled his Medicare-for-all plan. Politico digs into the details.


— This morning, Bristol-Myers Squibb (NASDAQ: BMY) shareholders will give either thumbs-up or thumbs-down to the company’s planned $74 billion buyout of Celgene (NASDAQ: CELG), a deal that would cause ripple effects through the biotech sector. Despite opposition, the deal has gained steam in recent weeks.

—Regeneron Pharmaceuticals (NASDAQ: REGN) invested $800 million in Alnylam as part of an alliance to develop RNA interference medicines for neurological and eye diseases.

—On the heels of recent multibillion dollar buyouts of AveXis and Endocyte, Novartis (NYSE: NVS) CEO Vas Narasimhan told Bloomberg that the company plans to spend more than $10 billion a year on acquisitions.

—Months after reporting a failed phase 3 study, cell therapy developer Histogenics (NASDAQ: HSGX) signed a reverse merger with Ocugen, a private developer of eye disease treatments.

—Audentes Therapeutics (NASDAQ: BOLD) cut a deal with Nationwide Children’s Hospital to develop treatments for two types of muscular dystrophy that combine elements of gene therapy and RNA-based medicines. Here’s more from Stat.

—AskBio of Research Triangle Park, NC, raised $235 million in funding to support its pipeline of experimental gene therapies.

—Inozyme of Boston reeled in $67 million to push its treatment for rare bone disorders into clinical trials.

—Philadelphia’s Scout Bio raised $20 million to develop gene therapies for chronic conditions in pets.

Trevi Therapeutics filed for an IPO to fund clinical trials for nalbuphine, an old opioid drug that it has developed into a treatment for tough-to-treat itching conditions.


—GlaxoSmithKline (NYSE: GSK) unit Viiv Healthcare won FDA approval of Dovato, the first two-drug, single-tablet regimen for HIV patients. The drug is a potential threat to Gilead Sciences’ (NASDAQ: GILD) flagship HIV franchise, which includes three-drug combination Biktarvy. GSK priced Dovato at a 25 percent discount to Biktarvy. Here’s more from Fierce Pharma.

—Amgen (NASDAQ: AMGN) got the FDA’s nod for romosozumab (Evenity), a treatment that builds bone strength in women with osteoporosis. Approval was delayed nearly two years by safety concerns.

—Merck (NYSE: MRK) received an expanded approval in non-small cell lung cancer for its immunotherapy pembrolizumab (Keytruda).

—Zogenix (NASDAQ: ZGNX) received a “refusal to file” letter from the FDA seeking more information about its marketing application for seizure drug fenfluramine hydrochloride (Fintepla). Shares sank more than 31 percent.

—AstraZeneca (NYSE: AZN) and Merck notched European approval of olaparib (Lynparza) as a monotherapy for certain breast cancer patients.

—Foundation Medicine and Flatiron Health, both units of Roche, published in JAMA a lung cancer study derived from their combined patient databases. The study comes more than two years after the companies first said they would work together.

—French firm Poxel and partner Sumitomo Dainippon Pharma reported positive Phase 3 results for its type 2 diabetes treatment imeglimin. The Japanese study could lead to market approval in that country. Poxel partner Roivant Sciences will run studies in the US and Europe.


—Andreas Busch, Shire’s former head of R&D and chief scientific officer, joined Cambridge, MA-based Cyclerion Therapeutics as chief innovation officer.

—Synlogic (NASDAQ: SYBX), which is developing engineered microbes to treat disease, named Scott Plevy its new chief scientific officer. Plevy was previously at J&J’s Janssen Research & Development.

—JDRF, a Type 1 diabetes philanthropy group, has promoted Aaron Kowalski to president and CEO.

Ben Fidler and Frank Vinluan contributed to this report.