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Bio Roundup: Trump’s Scalps, Policy Pressure, NASH Crash & More

Xconomy National — 

While politicians continue to debate what kind of healthcare system is best for the American people, the mostly U.S.-based pharmaceutical industry is trying to convince those same politicians—and anyone else who will listen—that what’s good for the industry is also good for America. Forcing drug prices lower, industry argues, would be bad for our health, because lower revenues mean less innovation. Those arguments might be losing traction, however, and officials and executives, like one of the drug industry’s longtime lobbying chiefs, are sounding alarm bells.

One direct outcome of the pressure on drug firms was evident this week: yet another drop in the price of a new type of anti-cholesterol drug. Those drugs, known as PCSK9 inhibitors, have spurred a cost-conscious backlash. Another price clash could come with the growing epidemic of NASH; that is, nonalcoholic steatohepatitis, a fatty liver disease that can lead to scarring, cancer, and transplants and has no approved pharma treatment. One drug racing to become the first approved fell flat this week; the next competitor to potentially emerge from the scrum should report data by midyear.

We’ve got all the main policy, regulation, clinical, and financial headlines from the week. Without further delay, you may enter the roundup.

POLICIES & POLITICS

—Biotechnology Innovation Organization CEO Jim Greenwood warned of “dangerous times” for the drug industry as politicians line up with investigations and regulations to rein in high drug prices. President Trump is “hell-bent on putting our scalps on the wall,” Greenwood told a conference in New York. But on a panel he moderated, speakers noted that, while one major administration proposal (an international price index) seemed terrible for the industry, a second one (elimination of rebates) might help them.

—Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi announced a 60 percent list-price cut to $5,850 a year for their cholesterol-lowering drug alirocumab (Praluent). The move, which matches an announcement from Amgen last year, is meant to lower out-of-pocket costs for Medicare beneficiaries.

—With Democrats in charge of the House, the fight over national health coverage continues on several levels. Some Democrats want Medicare for all, while one House bill is aiming for a more incremental Medicare for everyone over 50. Meanwhile, the Affordable Care Act lives on. Utah state legislators rolled back ACA-related Medicaid expansion that 53 percent of Utah voters approved in November, and GOP-led Georgia is now considering a partial expansion.

—The National Cancer Institute’s small-business program generated $9.1 billion in biotech product sales from the 690 grants issued between 1998 and 2010, according to an economic-impact report. The full report is available here.

—The FDA rejected the antibiotic iclaprim, telling owner Motif Bio (NASDAQ: MTFB) that it needs more data to evaluate the drug’s potential liver toxicity. Motif said it would need to raise cash soon.

—The FDA sent warnings and advisory letters to 17 companies—among them Earth Turns, Gold Crown Natural Products, and TEK Naturals—that are “illegally” claiming their supplements can help treat Alzheimer’s disease. The announcement came amidst a broader plan by the FDA to modernize regulation of dietary supplements.

DATA & STUDIES

—Gilead Sciences (NASDAQ: GILD) of Foster City, CA, said that its most advanced drug to treat the fatty liver disease NASH failed a major Phase 3 study. Eyes now turn to Intercept Pharmaceuticals (NASDAQ: ICPT) and Genfit (NASDAQ: GNFT) for late-stage data this year that could lead to the first approved drug to treat NASH.

—A study found that patients with glioblastoma, a deadly brain cancer, who take the Merck (NYSE: MRK) immunotherapy pembrolizumab (Keytruda) before and after surgery had longer survival rates than patients who only received the drug post-surgery.

—Partners Pfizer (NYSE: PFE) and Astellas filled in some details of the successful ARCHES prostate cancer study. They released topline results from the Phase 3 trial last December.

—An FDA advisory panel voted 14-2-1 in favor of approving the Johnson & Johnson (NYSE: JNJ) drug esketamine (Spravato), a fast-acting depression treatment delivered via nasal spray. The vote, which could pave the way for approval by March 4, comes despite mixed clinical results and the fact that esketamine is a chemical relative of the addictive party drug ketamine. Bloomberg has more.

—Immunotherapy rivals Merck and Bristol-Myers Squibb (NYSE: BMY) presented kidney cancer data at the ASCO Genitourinary Cancers Symposium. As Vantage reports, a combination of Merck’s pembrolizumab (Keytruda) and axitinib (Inyta, from Pfizer) looks to have bested Bristol’s FDA-approved nivolumab (Opdivo)/ipilimumab (Yervoy) regimen for newly diagnosed patients.

MONEY IN, MONEY OUT

—Johnson & Johnson (NYSE: JNJ) paid $3.4 billion to acquire Redwood City, CA, robotics startup Auris Health, a move that the company says is part of its “digital surgery” strategy.

—Passage Bio, co-founded by gene therapy pioneer James Wilson and longtime pharma executive Tachi Yamada, raised a $115.5 million Series A round. Yamada, now at Frazier Healthcare Partners, and Wilson have worked together in the past. Yamada helped keep Wilson’s work alive when Wilson was mired in controversy and the emerging field was in its darkest days.

TCR² Therapeutics raised $75 million in an IPO, which will fund clinical trials testing a T cell receptor therapy in solid tumors.

—AbbVie (NYSE: ABBV) paid $90 million upfront for rights to a Phase 1-ready multiple myeloma drug developed by Menlo Park, CA-based Teneobio.

—Shares of AstraZeneca (NYSE: AZN) climbed more than 9 percent on better-than-expected sales largely driven by cancer drugs, an area of focus in its transformation under CEO Pascal Soriot. The Wall Street Journal has more.

—Vor Biopharma of Boston has raised a $42 million Series A round, led by 5am Ventures and RA Capital Management, to push its lead product, a cell therapy for acute myeloid leukemia, toward human studies.

—Axovant Sciences (NASDAQ: AXON) is shuffling a quarter of its staff including two C-suite executives and what it calls “small-molecule expertise” into a new startup, Arvelle Therapeutics. Arvelle’s lead drug, an epilepsy candidate, will come from a third party, SK Biopharmaceuticals. Axovant is trying to pivot from its failed neurodegenerative disease portfolio into gene therapy.

—IFM Therapeutics launched a subsidiary called IFM Due that will develop drugs targeting the STING pathway, which is linked to a number of inflammatory and autoimmune diseases.

Neurogene emerged with $68.5 million in Series A financing to support development of gene therapies for rare genetic disorders affecting the brain.

—5AM Ventures closed two new life sciences funds totaling nearly $500 million. In addition, the firm unveiled a co-investment fund that will deploy cash to companies currently in 5AM’s portfolio.

PEOPLE ON THE MOVE

—Former Agios Pharmaceuticals (NASDAQ: AGIO) CEO David Schenkein surfaced at GV, where he will co-lead life science investments.

—Ameet Nathwani, executive vice president and chief medical officer of Sanofi (NYSE: SNY), is taking on additional responsibilities as the pharmaceutical giant’s chief digital officer.

—João Siffert was named CEO of Abeona Therapeutics (NASDAQ: ABEO), three months after taking the job on an interim basis.

Ben Fidler and Frank Vinluan contributed to this report.

Image by Kevin Dooley, Creative Commons 2.0.