Bio Roundup: Senate Drama, A.I. Stories, Data Dives, Shkreli & More

Xconomy National — 

There is no Obamacare replacement, at least not as of this writing. The Senate Republicans are fractured, with a handful of conservatives and moderates each giving a cold shoulder to their chamber’s version of healthcare reform, the Better Care Reconciliation Act. The nonpartisan Congressional Budget Office report Monday put the bill’s potential effects in stark relief: $321 billion in deficit reduction because of tax cuts and a Medicaid rollback, and 22 million more people uninsured by 2026. An addendum from the CBO Thursday looked ahead an extra decade and estimated by 2036, the BCRA would blunt Medicaid spending 35 percent.

Speaking of legal drama, the Martin Shkreli circus is back. After the former hedge-fund manager and drug-price whipping boy spent a year refusing to keep a low profile, his securities fraud trial began this week after problems finding unbiased jurors.

In opening remarks, Shkreli’s lawyer painted a colorful picture of his client’s eccentricities—meeting clients while wearing bunny slippers, for example—while defending his investment strategies against the government’s accusations. Politicians, regulators, scientists, and business folks were all busy this week. Let’s round up the rest of the life sciences headlines.

MORE DOINGS IN D.C.

—As of late Thursday, Senate leaders were pitching last-minute woo to naysayers of the health bill with carrots like a $45 billion opioid-epidemic earmark for hard-hit states like Ohio and West Virginia. Ohio Gov. John Kasich called it “spitting in the ocean” compared to the Medicaid funding for treatment that the BCRA would slash. Politico’s Dan Diamond pointed out the irony of the proposed funds.

—The Trump administration won temporary victory when the Supreme Court said its travel ban on people from six majority-Muslim countries and refugees could take effect until the court rules on the ban in the fall. The court wrote that the ban could not apply to people already with visas, or with a “bona fide relationship” to a person or entity already in the U.S. The State Department was in charge of interpreting the court’s vague edict.

—The House seemed to defy the White House’s earlier call for deep FDA budget cuts, and the FDA said it would address price gouging of generic drugs. The agency highlighted lack of competition as one reason the generic system can be “gamed”—interesting timing, with the Shkreli trial underway—and promised to make changes. A public meeting is scheduled for July 18.

—The FDA also said it would move to clear the backlog of requests for orphan drug designation and try to rule on all future requests within 90 days.

—Former Vice President Joe Biden and his wife, physician Jill Biden, launched the Biden Cancer Initiative to build upon the Cancer Moonshot effort started under President Obama. Biden says the initiative will operate independently of the federal government.

—The FDA approved the anticoagulant pill betrixaban (Bevyxxa) from South San Francisco-based Portola Pharmaceuticals (NASDAQ: PTLA).

WHEN TECH MEETS HEALTH

—Xconomy began a new series on the use of artificial intelligence in healthcare.

—Last Friday, San Francisco editor Bernadette Tansey reported on a Silicon Valley A.I. hackathon that focused on the genome of a tech entrepreneur with a rare disease.

—Senior editor Jeff Engel examined the role of two tech titans, IBM and General Electric, in the race to “A.I.-ify” healthcare.

—Jeff Buchanan, our Wisconsin editor, unpacked the common assertion from developers that A.I. is meant to help people do their jobs, not to replace them.

—We also recently launched a new podcast, Xconomy Voices, hosted by Wade Roush. Wade’s first guest is former Google and Facebook executive Mary Lou Jepsen, who has turned her own experience with a brain tumor into a quest for better medical imaging.

—A small, randomized study called MedSeq reported that, when using healthy patients’ whole genome sequences in primary care, there was little connection between suspicious genes and actual disease. The study also looked at medical costs and other outcomes, calling the use of sequencing “of uncertain value.”

DID SOMEONE SAY DATA?

—Alkermes (NASDAQ: ALKS), which operates in Waltham, MA, released top line results from the first of two Phase 3 schizophrenia studies of its drug ALKS-3831. The four-week study met its primary and secondary efficacy goals, but notable weight gain among the patients was a side effect the company and investors did not want to see. Shares were down more than 3 percent in after-hours trading Thursday.

—Early Phase 3 data showed the migraine drug eptinezumab from Bothell, WA-based Alder BioPharmaceuticals (NASDAQ: ALDR) reduced the frequency of headaches. But the results failed to distinguish eptinezumab from other drugs ahead in the migraine-prevention race, and Alder shares dipped nearly 20 percent.

—Preliminary Phase 3 data for Roche’s hemophilia treatment emicizumab showed the once-a-week injectable drug reduced bleeding by 87 percent.

— Earlier this month, patient deaths halted a leukemia drug from Seattle Genetics (NASDAQ: SGEN) of Bothell, WA. The firm had better news this week as brentuximab vedontin, combined with chemotherapy, met its Phase 3 goal of improving survival in patients with the most common form of Hodgkin lymphoma.

—A topical pain drug from Xenon Pharmaceuticals (NASDAQ: XENE) failed a Phase 2 study, leaving the Burnaby, BC biotech to shift to earlier-stage neurology compounds in its pipeline.

DOLLARS AND JOBS

—Cancer drug developer Mersana Therapeutics of Cambridge, MA, held an IPO that raised $75 million and could bring in up to $11.25 million more. It was quietly a big week for biotech IPOs: Aileron Therapeutics, Dova Pharmaceuticals, and micro-cap Avenue Therapeutics all went public, as well.

Atlas Venture unveiled a $350 million fund, the Cambridge firm’s eleventh, which it plans to use to invest in more seed-stage biotech startups.

—Dimension Therapeutics of Cambridge said it would lay off a quarter of staff by end of year. Poor Phase 1/2 results from its experimental gene therapy for hemophilia B cut its share price in half five months ago, and the firm ended work on the program last month.

—Cambridge-based LogicBio reeled in a $45 million Series B round as it works toward clinical trials for gene therapies that could treat rare pediatric liver diseases.

—Vivace Therapeutics closed a $25 million Series B round that the San Mateo, CA-based company will use to continue development of new cancer drugs.

NEW NAMES AND FACES

—Sarepta Therapeutics (NASDAQ: SRPT) named Douglas Ingram its new CEO, its third in three years. Ingram, a former Allergan executive, replaces Ed Kaye, who announced in April his plan to leave. Kaye led Sarepta to a controversial approval last year of its Duchenne muscular dystrophy drug Exondys 51, which insurers have been slow to cover.

—Cambridge-based RaNA Therapeutics renamed itself Translate Bio.

—Dragonfly Therapeutics, also of Cambridge, hired Nicolai Wagtmann away from Innate Pharma to be its new chief scientific officer.

—At Regenacy Pharmaceuticals, Simon Jones was promoted to CEO, succeeding Walter Ogier, who remains on the Boston company’s board of directors.

Photo of U.S. Capitol by Kevin Dooley via a Creative Commons 2.0 license.

Frank Vinluan contributed to this report.