Biotech Roundup: Duchenne Delay, Forum’s Fate, Califf, Ionis & More

Xconomy National — 

Memorial Day weekend may be fast approaching, but don’t fire up those grills yet: there’s plenty of headlines to dig into first in our biotech trip around the Xconomy network.

In New York, the nation’s most prestigious science competition changed sponsors for just the second time in its 74-year history. In Boston, one company’s controversial quest to potentially get the first drug for Duchenne muscular dystrophy approved drags on, while the fate of another seems to hang by a thread.

And on the West Coast, the first implantable device for opioid addiction was approved—just as the FDA’s commissioner made some pointed remarks at a Stanford University chat about the future rate of drug approvals. Those stories and much more below.

—The multi-year saga of eteplirsen, a Duchenne muscular dystrophy drug from Cambridge, MA-based Sarepta Therapeutics—which could be the first ever approved for the deadly disease—will continue to drag on. The FDA said this week that it wouldn’t complete its view of the drug by the May 26 deadline, and didn’t give any timeline for when a decision would come. Shares of Sarepta (NASDAQ: SRPT) surged more than 25 percent as investors took the delay as a positive sign for eteplirsen’s approval chances. Forbes has more on the potential outcomes that might lie ahead.

—Deborah Dunsire, the former head of Millennium Pharmaceuticals and a well known Boston biotech veteran, told Xconomy that she’s no longer the CEO of Waltham, MA-based Forum Pharmaceuticals. Dunsire aims to head up another biotech after some time off. In the meantime, the fate of Forum—whose lead drug, encenicline, failed two Phase 3 trials in March—remains unclear.

—At a Stanford University conference, new FDA commissioner Robert Califf predicted that the high rate of drug approvals in recent years would continue, thanks to better understanding of biology and adoption of “big data” and other sophisticated technology.

—Titan Pharmaceuticals (NASDAQ: TTNP) and partner Braeburn Pharmaceuticals received FDA approval Thursday for an under-the-skin implant that treats opioid addiction. The implant, branded Probuphine, delivers the drug buprenorphine for up to six months, according to Titan.

—The agency rejected, meanwhile, a drug from AstraZeneca (NYSE: AZN) called ZS-9 for the kidney disease hyperkalemia, citing manufacturing issues. That’s positive news for Redwood City, CA-based Relypsa (NASDAQ: RLYP), which has a rival hyperkalemia drug on the market—Relypsa shares soared 37 percent Friday morning—and a setback for the British drugmaker, which acquired ZS-9 when it paid $2.7 billion for Coppell, TX-based ZS Pharma in November.

—The National Institutes of Health has awarded $142 million to the Mayo Clinic to build a repository for more than 30 million biological samples. The so-called biobank will be part of the Obama administration’s Precision Medicine Initiative, a long-term health study that aims to gather comprehensive health information from 1 million Americans.

—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) has been named the new sponsor of the nation’s oldest science competition, the “Science Talent Search” (formerly known as the Westinghouse Award), taking over for Intel, which gave up its sponsorship last year. Regeneron has pledged $100 million in a 10-year commitment to the competition. Here’s more from The New York Times.

—Watertown, MA-based Selecta Biosciences filed for an IPO this week and aims to trade on the Nasdaq under the ticker symbol “SELB.” Selecta is using nanotechnology to trick the immune system into calling off an unwanted attack on a particular target; its lead drug is for chronic refractory gout.

—Shares of Waltham-based Minerva Neurosciences (NASDAQ: NERV) more than tripled after the company reported positive results from a mid-stage clinical trial of an experimental drug for schizophrenia.

—Shares of Burlington, MA-based Flexion Therapeutics (NASDAQ: FLXN) climbed 50 percent as the FDA, according to the company, indicated that Flexion’s data for long-acting steroid injection FX006 are good enough to allow it to file an approval application.

—Theranos of Palo Alto, CA, is already in hot water with the DOJ, the SEC, and the folks who oversee Medicare. Now here come the personal injury lawyers after Theranos revealed last week it had to correct two years worth of blood test results. One lawsuit was filed in Northern California on behalf of an Arizona man, as the San Francisco Business Times reported.

—-After struggling for years to convince the world it needs a drug for restless legs syndrome, XenoPort of Santa Clara, CA, has found a buyer. Arbor Pharmaceuticals of Atlanta bought XenoPort for gabapentil enacarbil (Horizant), which brought in $41 million in revenue last year, for $467 million.

—Shares of Carlsbad, CA-based Ionis Pharmaceuticals (NASDAQ: IONS) plunged by nearly 40 percent—eviscerating nearly $1 billion in market value—after partner GlaxoSmithKline decided to delay starting a Phase 3 study of its experimental RNA interference drug IONIS-TTRrx due to safety concerns. The FDA placed a clinical hold on a separate IONIS-TTRrx trial a few weeks ago, and according to a research note from Leerink Partners analyst Michael Schmidt, the company said on a conference call with analysts that cases of severe thrombocytopenia—dangerously low platelet counts—have been seen in the testing of two different Ionis treatments.

—Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) meanwhile, which has a rival drug to IONIS-TTRx—both are treatments for a rare condition called transthyretin amyloidosis—saw its shares jump 12 percent.

—Qualcomm Life is working with Medtronic to develop new systems to help people with type 2 diabetes monitor their blood sugar levels.

—Arrivo BioVentures of Morrisville, NC, raised $49 million from Jazz Pharmaceuticals and other investors to look for drugs already in development and ripe for licensing.

—There were a couple pharma executive shakeups in the news this week. Gilead Sciences brought back a familiar face, Kevin Young, as its COO, and Sanofi has named a new chief of its diabetes unit, Peter Guenter, as part of a larger reorganization.

—Sanofi is trying to shake up San Francisco biotech Medivation (NASDAQ: MDVN), too. The French pharma is one of a few suitors, so far unwanted, for the co-owner of prostate cancer drug enzalutamide (Xtandi). Sanofi this week took its bid hostile, aiming to oust some Medivation boardmembers. Medivation urged shareholders to reject Sanofi’s efforts.

—Clinical news: Seattle Genetics (NASDAQ: SGEN) began a pivotal Phase 3 trial of vadastuximab tailirine to treat newly diagnosed acute myeloid leukemia. Alkermes (NASDAQ: ALKS), stung by a recent clinical failure of its experimental depression treatment, is trying its hand at immuno-oncology, starting a Phase 1 trial in patients with solid tumors.

—U.K.-based Bicycle Therapeutics opened up an office in Kendall Square in Cambridge, and recruited former GSK executive Rosamond Deegan as its president and chief business officer. Deegan will hire the staff for Bicycle’s U.S. team.

—New York health informatics startup COTA got an unspecified investment from Celgene (NASDAQ: CELG), Novartis (NYSE: NVS), Foundation Medicine (NASDAQ: FMI), and consulting firm HealthScape Advisors. COTA has an analytics platform that helps drugmakers identify patients that might better respond to their treatments.

—Cambridge-based Spero Therapeutics announced a deal to grab rights to a group of experimental antibiotic compounds from Vertex Pharmaceuticals, among them a drug called VXc-486/VXc-100. The deal is the latest in a group of licensing agreements Spero has used to amass different ways of attacking troublesome drug-resistant bacteria, so-called “superbugs” that can resist common antibiotics.

—Avalon Ventures and GSK formed their eighth San Diego biotech—a cancer drug developer called PDI Therapeutics—under a collaboration established three years ago. As with the other startups in the Avalon/GSK partnership, PDI will get up to $10 million in Series A cash and research funding, and GSK has an exclusive option to buy the startup later on.

​—Struggling San Diego-based Sophiris ​Bio ​(NASDAQ: SPHS) has hired an investment banking firm to help evaluate strategic alternatives to advance its experimental prostate drug. Sophiris also cut its workforce ​from 10 to five, and has raised $4.6 million to extend its operations for at least one more year.

—An unexpected $70.5 million investment in Tribune Publishing from biotech billionaire Patrick Soon-Shiong has apparently thwarted Gannett’s hostile buyout bid for the media giant, which owns the The Los Angeles Times and The San Diego Union-Tribune. Soon-Shiong’s Culver City, CA-based firm NantHealth, meanwhile, is expected to go public next week.

—Alex Lash and Bruce Bigelow contributed to this report