Bio Roundup: RNAi Drama, Immunotherapy Data, Lasker Winners & More

Xconomy National — 

The summer doldrums are over. Biotech news came at a breakneck pace this week both before and after Labor Day, starting with the surprise resignation of Novartis’s top executive just days after the Swiss company notched an historic FDA approval.

From there, the week featured big headlines, both good and bad, from several trials in cancer immunotherapy, gene therapy, RNA interference, and much more. New companies joined the IPO queue. Others raised cash from private investors. And we haven’t even mentioned anything going on in Washington, DC, yet. Let’s round it all up.

Inside the Beltway

—The drug industry’s Washington lobbyists BIO and PhRMA filed suit last Friday against the state of Nevada, which passed a law in June requiring diabetes drug price transparency.

—The Senate HELP committee and its leaders Lamar Alexander (R-TN) and Patty Murray (D-WA) held two days of healthcare hearings with state governors and insurance commissioners, headlined by a bipartisan reform proposal from the governors of Colorado and Ohio. The hearings are a reset button of sorts after the failure to repeal the Affordable Care Act. According to Alexander, the goal is to provide quick fixes by the end of the month to stabilize the individual insurance market.

—The Senate continues to buck the Trump administration’s goals of slashing NIH funding. A committee voted to increase the research agency’s budget by $2 billion next year.

—Furthering their ambitions to spur the biotechnology industry to speak out against policies it doesn’t agree with, CEOs Steve Holtzman and Jeremy Levin co-authored a letter—signed by 183 fellow industry veterans—urging the Trump Administration and Congress to preserve the Deferred Action for Childhood Arrivals Program (DACA). The White House wants to halt the program, which protects close to 800,000 “Dreamers,” undocumented immigrants brought to the U.S. as children, from deportation.

RNAi Drama

—Nearly 20 years after UMass Medical School professor Craig Mello co-discovered RNA interference, a way of switching off genes before they make proteins, the science is as close as ever to producing a drug—that is, if patisiran, a drug from Alnylam Pharmaceuticals (NASDAQ: ALNY), reports positive Phase 3 data soon. Xconomy spoke with Mello about the field’s roller-coaster history and what’s to come.

—While waiting on the patisiran data, Alnylam got bad news on a different drug. A patient on an experimental Alnylam RNAi hemophilia drug, fitusiran, died of brain swelling, causing the company to halt all ongoing studies. Alnylam expects to restart testing by the end of the year.

Immunotherapy Ups & Downs

—Cellectis (NASDAQ: CLLS) reported that the FDA has put a hold on its first CAR-T cancer program, UCART123, after the first patient to receive the therapy died of cytokine release syndrome, a common CAR-T side effect. Unlike most CAR-T therapies made from each patient’s own cells, the Cellectis CAR-T is “off the shelf” and uses donor cells.

— The FDA this week suspended, either fully or partially, clinical studies run by Celgene (NASDAQ: CELG) and Bristol-Myers Squibb (NYSE: BMY) that were testing immunotherapy  drug combinations. Most of the trials were in multiple myeloma. The FDA’s safety concerns stem from patient deaths that halted a multiple myeloma study testing pembrolizumab (Keytruda), a Merck (NYSE: MRK)  immunotherapy, in combination with other drugs.

—In a surprise win, meanwhile, Bristol-Myers said that a combination of its immunotherapies nivolumab (Opdivo) and ipilimumab (Yervoy) helped newly diagnosed kidney cancer patients live longer. The combination was not better than the standard of care sunitinib (Sutent) in preventing patients’ cancer from spreading, but Bristol still felt it could win approval based on the “totality of the…data.” This week’s news helped its case.

Pharma Moves

—Novartis (NYSE: NVS) announced on Labor Day that CEO Joe Jimenez plans to step down and return to Silicon Valley, where “biology comes together with technology.” Jimenez, a Stanford University alumnus, told Forbes that he sees solutions in the Valley for fixing “inefficiency in the U.S. healthcare system.”

—Indianapolis-based Eli Lilly announced plans to close R&D facilities in Bridgewater, NJ, and Shanghai, in a restructuring move to cut $500 million in yearly costs.

Big Money

—Capitalizing on a growing demand to manage and store vast amounts of genomic data, WuXi NextCODE closed a $240 million Series B round. It’s the latest chapter in a long-running story for NextCODE, whose executives were part of deCode Genetics, an Icelandic genomics project that began in the 1990s.

—Merck said it would acquire German immuno-oncology firm Rigontec for €115 million upfront and potentially €349 million more.

—Healthcare investment firm OrbiMed Advisors has raised its third Asia-focused fund, with $551 million available.

—Consumer DNA testing company 23andMe is lining up nearly $200 million in private financing, according to TechCrunch. Sequoia Capital will reportedly lead the round.

—Gritstone Oncology of  Emeryville, CA, notched $93 million in a Series B round. With the new cash, the cancer vaccine maker hopes to enter clinical studies next year for lung and stomach cancer.

—Replimune, of Woburn, MA, bagged a $55 million Series B round to continue developing oncolytic immunotherapies.

—Antibacterial firm Entasis Therapeutics of Waltham, MA, raised $32M in additional Series B funding.

—Two Boston-area biotechs filed for IPOs this week. Rhythm Pharmaceuticals hopes to finance the development of a drug for rare forms of obesity, while Deciphera Pharmaceuticals aims to fund its cancer drug work.

Data Dumps

—Boston-based Verastem’s (NASDAQ: VSTM) low-cost flier on a drug originally developed by Infinity Pharmaceuticals (NASDAQ: INFI) paid dividends this week as the drug succeeded in a Phase 3 trial in chronic lymphocytic leukemia. Verastem, whose shares climbed 28 percent on the news, will seek approval of duvelisib in 2018, while Infinity gets a $6 million milestone payment from the trial’s success.

—Cambridge-based Voyager Therapeutics (NASDAQ: VYGR) provided the latest update from an ongoing early-stage study of a gene therapy it’s developing to help Parkinson’s disease patients who no longer respond to their medication. The results remain promising, which keeps Voyager on track to start a pivotal trial next year. Shares rose 24 percent.

—Shares of Sarepta Therapeutics (NASDAQ: SRPT) also climbed 13 percent after a study showing one of its experimental Duchenne muscular dystrophy therapies, golodirsen, helped a small group of patients produce the muscle-boosting protein dystrophin. Here’s more from STAT.

Awards & Events

—Among the world’s top biomedical prizes, the Lasker Awards were announced this week. The clinical award went to the two scientists, Douglas Lowy and John Schiller, whose discoveries led to the development of a human papillomavirus vaccine.

—This week Corie Lok detailed the finalists for one of Xconomy’s own prizes, the “Newcomer” award, which featured some the people and companies new to Boston and making an impact in the biotech scene.

—Three cancer drug companies pursuing vastly different paths—Dragonfly Therapeutics, IFM Therapeutics, and Tesaro (NASDAQ: TSRO)—headline Xconomy’s latest biotech event, “Boston’s Life Science Disruptors,” on Sept. 28. Check out the details and the full agenda here.

Alex Lash contributed to this report.