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Bio Roundup: CMS Backs Cancer Tests, Right to Try Passes, AbbVie Sinks

Xconomy National — 

We’ll start this week’s roundup with several stories from Washington, DC, that could have a lasting effect on public health. The FDA continued its campaign against cigarettes, while the Centers for Medicare & Medicaid Services (CMS) gave a much-needed boost to the developers of cancer diagnostics. In its omnibus spending bill, Congress set aside more money to battle the opioid crisis, while lawmakers debated several other bills to help deal with the nationwide epidemic.

In other life sciences news, the first U.S. gene therapy was given to a patient in Boston, a $5.8 billion buyout looks like it could be a bust, and an obesity drugmaker just got a new lease on life. Let’s get right to it.

ON THE HILL

—CMS, the agency that oversees Medicare and Medicaid, said it finalized plans to cover FDA-approved tests that scan tumors for a range of genetic mutations, a boost for Foundation Medicine (NASDAQ: FMI), Thermo Fisher Scientific (NYSE: TMO), and others. The agency did not say how much it would reimburse for these tests. CMS did say, however, it would automatically reimburse for companion diagnostics once cleared by the FDA.

—The federal “All of Us” biomedical research program, which includes plans for a 1 million-volunteer biobank, is off to a slow start, the New York Times reported. Complexity and cost are two big issues. Data giant Google’s early involvement in the project raised eyebrows two years ago, as Xconomy reported.

—During two days of hearings, U.S. lawmakers discussed several bills to combat the opioid crisis. Cartier Esham, a top executive at the trade group Biotechnology Industry Organization, was one of several witnesses voicing support for new legislation, especially to help boost the development of new pain drugs.

—On its second attempt, the House passed so-called “right to try” legislation that would let extremely sick people try experimental drugs with less regulatory oversight. The vote overcame objections from the drug industry, bioethicists, and patient advocates. One critic told Politico the bill, now likely to become law, is a “first step” toward a “Wild West of medicine.”

—In a joint appearance to discuss the opioid crisis, Health and Human Services secretary Alex Azar and President Trump said a plan to deal with high drug prices is about a month away. Previous promises from Trump have gone nowhere, as Xconomy has noted. Meanwhile, a federal database to track the list price of pharmaceuticals has not been updated since Trump took office, CNN reported.

DATA UPS AND DOWNS

—AbbVie (NYSE: ABBV) disclosed it won’t seek accelerated approval of a cancer drug, Rova-T, after disappointing results from a mid-stage trial. The news dims the prospects of AbbVie’s $5.8 billion buyout of Stemcentryx in 2016. AbbVie shares fell 12.7 percent.

—A year after selling its weight-loss pill lorcaserin (Belviq) to focus elsewhere, Arena Pharmaceuticals (NASDAQ: ARNA) reported encouraging Phase 2 results for its experimental ulcerative colitis pill etrasimod. Arena shares climbed more than 34 percent, and the company quickly cashed in with a $353 million stock offering.

—The New England Journal of Medicine and Lancet Oncology published papers on a number of cancer drug regimens this week. Among them: Bristol-Myers Squibb’s nivolumab (Opdivo) and ipilimumab (Yervoy) in kidney cancer; venetoclax (Venclexta) and rituximab (Rituxan), from AbbVie and Roche/Genentech, in chronic lymphocytic leukemia; and encorafenib and binimetinib, from Array Biopharma, in melanoma.

—The NEJM also published a paper from scientists at the Regeneron Genetics Center identifying a variant of the gene HSD17B13 that may be associated with reduced risk of chronic liver diseases such as nonalcoholic steatohepatitis. Regeneron Pharmaceuticals (NASDAQ: REGN) will team with Alnylam Pharmaceuticals (NASDAQ: ALNY) to develop RNA interference medicines for NASH based on the discovery.

—Separately, Regeneron’s aflibercept (Eylea) succeeded in a Phase 3 study in non-proliferative diabetic neuropathy. The study is part of the Tarrytown, NY, company’s plans to expand the uses for aflibercept, which is already approved for three other eye diseases.

—Roche said a combination of its immunotherapy atezolizumab (Tecentriq) and chemotherapy met one of its goals in an ongoing Phase 3 study of squamous non-small cell lung cancer, but the Swiss drug giant did not release data. Roche is studying atezolizumab combinations and looking to gain ground on immunotherapy leaders Merck and Bristol-Myers Squibb.

RED LIGHT, GREEN LIGHT

—Radius Health (NASDAQ: RDUS) expects the Committee for Medicinal Products for Human Use, which advises the European Medicines Agency, to recommend against approving its osteoporosis drug abaloparatide (Tymlos) in Europe. Radius intends to appeal the ruling.

—Seattle Genetics’ (NASDAQ: SGEN) drug brentuximab vedotin (Adcetris) notched an additional approval as a first-line treatment for stage III or IV classical Hodgkin lymphoma, in combination with chemotherapy. It’s the fifth approval for a form of lymphoma for the Bothell, WA, company’s drug.

—Proteostasis Therapeutics (NASDAQ: PTI) withdrew a $61 million stock offering after investment manager Kerrisdale Capital issued a report casting doubt about the prospects of the Cambridge company’s cystic fibrosis drug PT1-428.

HELLOS & GOODBYES

—Massachusetts Eye and Ear dosed the first commercial patient to ever receive a U.S. gene therapy, Spark Therapeutics’ (NASDAQ: ONCE) voretigene neparvovec (Luxturna), for rare inherited vision loss. Here’s more from STAT.

—Cambridge, MA-based Rheos Medicines launched with $60 million in funding to develop cellular metabolism drugs for autoimmune and inflammatory disorders.

Alzheon filed for an IPO to take a once-failed Alzheimer’s disease drug, tramiprostate, through Phase 3 testing.

—Alder Biopharmaceuticals (NASDAQ: ALDR) abruptly parted ways with CEO Randy Schatzman, the Bothell company’s top executive since its 2004 founding, just as it prepares to file for FDA approval of its first drug.

DEALS & FUNDINGS

— TCR² Therapeutics of Cambridge raised $125 million to advance its pipeline of T cell receptor therapies, a kind of cousin of the higher-profile CAR-T cell treatments.

—Continuing its push into neurodegenerative disease, Celgene (NASDAQ: CELG) paid Prothena (NASDAQ: PRTA) $100 million upfront and invested $50 million more in an alliance to develop drugs that target Alzheimer’s, amyotrophic lateral sclerosis, and other diseases.

—Chicago health data firm Tempus Labs raised $80 million.

THIS WEEK IN THERANOS

—Last week the feds charged Theranos, its CEO Elizabeth Holmes, and its ex-president Ramesh “Sunny” Balwani with fraud, and said Theranos and Holmes decided to settle. Balwani, however, is pressing ahead with a court fight; STAT dug into his past this week to find out more about him.

EVENT NOTES

—This week we announced our latest New York life sciences event, “Bringing Back the Ex-Pats,” which will feature a candid discussion with biopharma leaders who are ex-New Yorkers—a group many see as critical to the development of New York biotech, if they can be lured back.

— CRISPR-Cas9 gene editing pioneer Feng Zhang is the latest to join up for our “What’s Hot in Boston Biotech” event at the Broad Institute on May 16.

Frank Vinluan and Alex Lash contributed to this report.