Bio Roundup: Moderna’s IPO, Arena’s Comeback, E-Cig Survey & More

Thanksgiving is around the corner and this week delivered a bounty of deals that have a number of life science companies expressing their thanks a little early.

One deal saw a company land a whopping $800 million upfront for a rights to a drug still in clinical testing. Another biotech landed a $98 million investment from its European partner. And a secretive, closely watched company that has already raised billions from partners and investors has laid the groundwork for an IPO that could become the biggest in biotech history.

In other headlines this week, regulators moved to snuff out teen use of electronic cigarettes, new questions emerged from a huge study of a fish oil pill, and the race to treat hemophilia with gene therapy took another turn. Hungry for news? Let’s dig in to all of those stories in this week’s bio news roundup.

DOLLARS & DEALS

—Arena Pharmaceuticals (NASDAQ: [[ticker:ARNA]]) continued its revamp by netting $800 million upfront from United Therapeutics (NASDAQ: [[ticker:UTHR]]) for the rights to ralinepag, an experimental drug for pulmonary arterial hypertension currently in Phase 3 testing. Arena shares climbed 21 percent.

Ribometrix, of Durham, NC, raised $30 million in Series A financing, the latest startup to launch amongst an emerging group of companies developing chemical drugs that target RNA molecules.

—Celgene (NASDAQ: [[ticker:CELG]]) expanded its immunotherapy collaboration with Dragonfly Therapeutics in blood cancers, paying the startup $50 million to add solid tumors to the deal.

—Harpoon Therapeutics closed a $70 million Series C round to support its cancer immunotherapy R&D.

—Nestle Health Sciences made a $98 million equity investment in Aimmune Therapeutics (NASDAQ: [[ticker:AIMT]]), bringing the Swiss company’s stake in its Brisbane, CA, partner to approximately 19 percent.

Kymera Therapeutics raised $65 million in Series B financing as the biotech prepares to test its protein degradation drug in clinical trials.

Cadent Therapeutics raised $40 million in a Series B round for mid-stage tests of its experimental treatment for movement disorders. Cadent was formed from the merger of two Atlas Venture startups, Ataxion and Luc Therapeutics.

—On the heels of starting a $40 million drug discovery partnership with Eli Lilly (NYSE: [[ticker:LLY]]) last week, NextCure closed $93 million in Series B financing to support clinical testing of its two lead cancer drugs.

—Boehringer Ingelheim paid Epizyme (NASDAQ: [[ticker:EPZM]]) $15 million in cash and $5 million in research funding to co-develop two epigenetic drugs for cancers that “currently lack therapeutic options.”

—Roivant Sciences, the biotech holding company run by Vivek Ramaswamy, raised another $200 million at a $7 billion valuation. The cash continues Roivant’s strategy of plucking experimental drugs from pharma’s dustbin and forming new companies around them.

THIS WEEK IN HEALTH POLICY

—A national survey found that teen use of electronic cigarettes soared 78 percent in the past year, prompting FDA commissioner Scott Gottlieb to call for stricter rules for in-person and online sales of flavored e-cig products.

—Thanks to a campaign that focused intensely on healthcare, Democrats continued to gain seats in the House of Representatives. Now they must decide how strongly to push for a single-payer, Medicare-for-all system.

—At least 90 cases of the mysterious disease acute flaccid myelitis, almost entirely in young children, have been confirmed as of Nov. 9 by the Centers for Disease Control and Prevention, according to NPR.

DRUG DEVELOPMENTS

—Amarin (NASDAQ: [[ticker:AMRN]]) stunned the medical world in September, showing topline results that its prescription fish-oil treatment could significantly lower the risk of heart attack and stroke. But a deeper dive into the crucial REDUCE-IT study, released Saturday, surfaced questions about Amarin’s choice of placebo, the potential size of the risk reduction, and more. At Thursday’s close, Amarin shares were down 16 percent for the week.

—UniQure (NASDAQ: [[ticker:QURE]]) posted new data suggesting its hemophilia B gene therapy, AMT-061, may compete with a rival treatment from Pfizer (NYSE: [[ticker:PFE]]) and partner Spark Therapeutics (NASDAQ: [[ticker:ONCE]]). Both gene therapies have begun Phase 3, though UniQure CEO Matt Kapusta told Xconomy the Lexington, MA, firm may get to market first. UniQure shares surged 35 percent.

—Blueprint Medicines (NASDAQ: [[ticker:BPMC]]) provided updated results from an early-stage study of its experimental avapritinib in patients with advanced gastrointestinal stromal tumors. Blueprint aims to file for FDA approval of the drug next year.

—Eli Lilly (NYSE: [[ticker:LLY]]) filed for FDA approval of lasmiditan, a drug for acute treatment of migraines that it acquired through a 2017 buyout of CoLucid Pharmaceuticals. The FDA recently cleared Lilly to start selling galcanezumab (Emgality), a drug meant to prevent migraines.

—In a final look at a study called Mystic, AstraZeneca (NYSE: [[ticker:AZN]]) said its combination of immunotherapies durvalumab (Imfinzi) and tremelimumab didn’t help newly diagnosed advanced non-small cell lung cancer patients live longer than chemotherapy. The study had already failed its other main goal, preventing patients’ cancers from spreading, last year.

—AstraZeneca also sold U.S. rights to palivizumab (Synagis), a drug for respiratory syncytial virus, to Swedish Orphan Biovetrum for $1.5 billion in cash.

—Merck (NYSE: [[ticker:NYSE]]) racked up another approval for its immunotherapy pembrolizumab (Keytruda), which the FDA cleared for patients with liver cancer who have failed one treatment.

—Merck also said, without revealing details from a Phase 3 trial, that pembrolizumab helped extend the lives of some, but not all types of patients with advanced or metastatic esophageal cancer who have failed one treatment.

—An experimental Concert Pharmaceuticals (NASDAQ: [[ticker:CNCE]]) drug for sudden hair loss, alopecia areata, posted positive Phase 2 results. But as Vantage reports here, shares fell 20 percent as investors weighed the drug’s results against a rival treatment from Pfizer.

—Over the weekend, Nektar Therapeutics (NASDAQ: [[ticker:NKTR]]) gave an interim look at Phase 2 data from its immunotherapy combination of NKTR-214 and the blockbuster nivolumab (Opdivo) in first-line melanoma. Evaluate wrote that the results should “set off alarm bells.”

THIS WEEK IN IPOS

—Moderna, the high-flying, secretive developer of messenger RNA drugs, finally filed for its long-anticipated IPO. Moderna’s initial IPO target is $500 million, which would be the largest initial biotech stock offering in history.

—San Diego-based synthetic biology company Synthorx filed for an IPO targeting $100 million to finance clinical tests of its lead cancer drug and continue its autoimmune drug R&D.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user Tom Sackton via a Creative Commons license

Author: Frank Vinluan

Xconomy Editor Frank Vinluan is a business journalist with experience covering technology and life sciences. Based in Raleigh, he was a staff writer at the Triangle Business Journal covering technology, biotechnology and energy before joining MedCityNews.com as North Carolina bureau chief. Prior to moving to North Carolina’s Research Triangle in 2007 he held business reporting positions at The Des Moines Register and The Seattle Times.