Biotech Roundup: Opioid News, ASH Preview, Boston Bio IPOs & More

Xconomy National — 

The rising death toll from opioids has prompted policymakers to try various ways to take on the problem. President Trump’s opioid commission released its final report this week, calling for expansion of drug courts, better physician training, and penalties for insurers that don’t cover treatment.

But the commission did not say how much its recommendations would cost, nor did it offer a way to pay for them. The absence of those details highlight the difficulties in addressing the crisis. One option is offering other, non-addictive medications. But as an Xconomy report noted, the pipeline of alternatives is thin due to various challenges developing new pain drugs. Some officials are pursuing legal remedies. New Jersey sued Stamford, CT-based Purdue Pharma, pinning blame for the state’s opioid crisis on the drug maker’s marketing tactics to doctors and patients.

In other news this week, companies gave an early look at data they plan to present at the annual conference of the American Society of Hematology, a pair of Boston drug developers went public, and a savvy Bay Area biotech entrepreneur used an unconventional way to snag the attention of high-profile investor Mark Cuban.


—The federal agency that oversees Medicare and Medicaid issued plans to reform so-called 340b payments, which lay out how much the government pays hospitals for in-patient drugs

—Digital health investor Rock Health issued its annual “Women in Healthcare” report. It included a survey of more than 300 women who were pessimistic about reaching gender equality anytime soon.

—A new digital health trade group, the Digital Therapeutics Alliance, launched to promote clinical and regulatory validation of technology-driven treatments.

—Liquid biopsy firm Grail has raised north of a billion dollars but is now dealing with a spate of executive departures that a spokeswoman told Buzzfeed News were part of “a reorganization to focus on nearer-term, unanticipated product opportunities from our R&D organization.”


—With a preview of new data from the upcoming American Society of Hematology (ASH) conference, Juno Therapeutics (NASDAQ: JUNO) gained momentum in the competition to treat non-Hodgkin lymphoma with cutting-edge CAR-T cell therapy. Rival Novartis (NYSE: NVS) also shed light on its lymphoma CAR-T, which is now before the FDA for an approval decision.

—The New England Journal of Medicine detailed the results of a Phase 1 trial of AVXS-101, an experimental gene therapy for spinal muscular atrophy from AveXis (NASDAQ: AVXS) in late-stage clinical testing.

—Bluebird Bio (NASDAQ: BLUE) will present several updates at ASH, among them the first proof of whether a new manufacturing process will help its gene therapy, LentiGlobin, produce better results in patients with the blood diseases beta-thalassemia and sickle cell disease. An ASH abstract showed signs that that might ultimately be the case, sending shares up about 10 percent, but Bluebird will disclose more significant details at the meeting.

—Regeneron Pharmaceuticals (NASDAQ: REGN) will seek FDA approval of dupilumab (Dupixent) as an asthma treatment after the drug—already approved for a form of eczema—succeeded in the second of two Phase 3 trials in asthma patients.

—At a medical meeting in Paris, Alnylam Pharmaceuticals (NASDAQ: ALNY) and Ionis Pharmaceuticals (NASDAQ: IONS) provided detailed results of their dueling prospective treatments for the rare disease transthyretin amyloidosis. Endpoints has more on the data here, and here’s more on the looming commercial battle between both drugs.


—Boston-area drugmakers Spero Therapeutics (NASDAQ: SPRO) and Allena Pharmaceuticals (NASDAQ: ALNA) raised $152 million combined, following a quarter in which life sciences offerings were responsible for some of the biggest gains across all sectors.

—Voyager Therapeutics (NASDAQ: VYGR) of Cambridge is preparing a key study of its experimental treatment for Parkinson’s disease without Sanofi’s (NYSE: SNY) help. The pharma giant gave up its rest-of-world rights to the gene therapy after failing to grab a share of U.S. rights, which Voyager holds under terms of the companies’ original 2015 pact.

—Frazier Healthcare Partners closed a $419 million fund, two thirds of which the Menlo Park, CA, venture capital firm says will be invested in seed and Series A rounds, including the financings of companies that the firm creates.

—Aisling Capital closed a $280 million fund that the New York firm plans to invest in clinical-stage companies.

—Kymera Therapeutics of Cambridge, MA, nabbed a $30 million Series A financing to develop drugs targeting the ubiquitin-proteasome system.

—Sarepta Therapeutics (NASDAQ: SRPT) reached a deal that gives the Cambridge biotech the option to license CRISPR/Cas9 technology that Duke University scientists have studied for potential applications treating Duchenne muscular dystrophy.

—Baebies, a Durham, NC, diagnostics startup, raised $10 million in a Series B financing round to support commercialization of its testing platform that screens newborns for inherited diseases.

—San Jose, CA-based VitalConnect raised a $38M Series C round to support the launch of its disposable, wearable sensor that monitors patient vital signs in hospitals and outpatient settings.

—Mallinckrodt Pharmaceuticals (NYSE: MNK) reached a deal to acquire Ocera Therapeutics for approximately $42 million up front, and up to $75 million more if the Redwood City, CA, company’s experimental treatment for hepatic encephalopathy—which failed in a Phase 2 trial—hits milestones.

—Dicerna Pharmaceuticals (NASDAQ: DRNA) got $10 million up front from Boehringer Ingelheim in a deal to develop RNA interference drugs for chronic liver diseases, starting with a therapy for nonalcoholic steatohepatitis.

—The Arnold Foundation has pledged nearly $14 million more to the Institute for Clinical and Economic Review, or ICER, a high-profile evaluator of drug prices and values.

—NBA owner turned TV personality Mark Cuban is putting a bit of his billions into San Francisco startup Perlara’s $7 million Series A round. Perlara CEO Ethan Perlstein initially contacted Cuban via Twitter. It’s not the first time he has gained an investor’s attention via 140 characters.

Alex Lash and Ben Fidler contributed to this report.

Photo by Flickr user ollagrafik via a Creative Commons license.