East Coast Biotech Roundup: Raze, Retrophin, Alnylam, & More

Xconomy Boston — 

After a one-week hiatus, the roundup is back, and we’ve got plenty of news to take your mind off of Ebola, the plunging stock market, and the now-defunct AbbVie-Shire megamerger. Let’s get right to it:

—A month after leaving Epizyme for a return to the venture capital world, Jason Rhodes took the helm of a new startup, Cambridge, MA-based Raze Therapeutics, which emerged from stealth this past week with a $24 million Series A round from a group of VC and pharma backers. Raze aims to be a platform oncology company, based on insights from the Whitehead Institute, Massachusetts General Hospital, and Princeton University into a newly discovered set of biological pathways that appear to be a key driver of tumor growth. I spoke with Rhodes—now a partner at Atlas Venture—about the startup, which is similar, in many ways, to the one he just left.

—Last week, we hosted a biotech event, “Boston’s Life Science Disruptors,” giving people a behind-the-scenes look at how three local companies—Zafgen (NASDAQ: ZFGN), Epizyme (NASDAQ: EPZM), and Sage Therapeutics (NASDAQ: SAGE)—made their way from inception to successful biotech IPO. In case you missed it, here’s my wrap-up of the proceedings, including six takeaways from the founder-investor groups taking the stage that night.

—Watertown, MA-based Selecta Biosciences raised $20 million in new equity financing this week to advance its plan to use nanoparticle technology to help tamp down the unwanted immune reactions caused by some biologic drugs. The first test for that approach is a gout drug candidate Selecta is imbuing with its technology and putting into its first clinical trial next year. Existing investors (Polaris Partners, Flagship Ventures, OrbiMed Advisors, NanoDimension, Rusnano, and Leukon Investments) and some new backers (I2BF, Eminent Venture Capital, and one other undisclosed investor) provided the cash. The clinical push is part of a gradual strategic shift for Selecta; I discussed that strategy with CEO Werner Cautreels.

—Cambridge-based Moderna Therapeutics announced a long-term collaboration with the Karolinska Institutet and Karolinska University Hospital. Through the deal, Moderna—which has raised more than $400 million through various partnerships and venture financings—will sponsor research grants for scientists at both institutions to do preclinical work on potential messenger RNA (mRNA) therapies. Moderna will then run clinical trials on those candidates at a lab it’s building in Stockholm, Sweden, should the drugs pass through the preclinical phase. The deal is Moderna’s first academic partnership.

—Shares of Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) rocketed almost 20 percent on Monday after it posted positive results from a Phase 2 extension study of its experimental RNA drug, patisiran, in patients with a rare nerve-damaging disease called familial amyloidotic polyneuropathy, or FAP. The study showed that Alnylam’s drug helped keep the nerve damage of a small number of FAP patients in check after six months of treatment. (Historical data suggests the nerve damage gets progressively worse in untreated patients, as defined by a statistical measure of their nerve damage called “modified Neuropathy Impairment Score,” or mNIS +7.) Alnylam also reported the drug was still safe and well-tolerated, and that it continued to reduce the level of the disease-causing protein (TTR) that builds up in patients’ blood. A statistically significant improvement in mNIS +7 is the primary goal of Alnylam’s ongoing Phase 3 trial for patisiran—the company’s most advanced drug prospect.

—The messy divorce between New York-based Retrophin (NASDAQ: RTRX) and founding CEO Martin Shkreli concluded this past week. The company announced Shkreli’s resignation from its board of directors, and a deal to sell some of its non-core assets—an oxytocin nasal spray, mecamylamine HCl (Vecamyl), and ketamine—to Shkreli’s new startup, Turing Pharmaceuticals. (Retrophin acquired oxytocin and Vecamyl in two separate acquisitions Shkreli engineered.) Retrophin’s chief operating officer, Stephen Aselage, was named interim CEO of Retrophin on Sept. 30, ousting Shkreli—reportedly a result of missed development deadlines and a host of other reasons you can read about in TheStreet.com. The company is now focusing on the development of its candidates for a rare kidney disorder called focal segmental glomeruloscerosis (FSGS) and pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder.

—Cambridge-based Bluebird Bio (NASDAQ: BLUE) this week dosed the first patient in the sickle cell disease trial it’s running for gene therapy prospect, Lenti-Globin, in France. The company has also opened up a U.S. sickle cell study and aims to treat up to eight patients with the disorder.

—New York-based Bristol-Myers Squibb is putting another combination study together for its PD-1 checkpoint inhibitor, nivolumab (Opdivo). It’s working with Pharmacyclics (NASDAQ: PCYC) and Johnson & Johnson to test nivolumab in combination with their drug, ibrutinib (Imbruvica), in a variety of blood cancers.

—Cambridge-based Infinity Pharmaceuticals (NASDAQ: INFI) reported that its lead drug candidate, PI3 kinase inhibitor duvelisib, failed a Phase 2 study in allergic asthma. Infinity reported that the drug was well-tolerated and hit various “secondary and exploratory” endpoints, but didn’t produce a statistically significant improvement in patients’ lung function. Duvelisib is part of a big partnership with AbbVie; the two companies are testing the drug in a variety of blood cancers, though Infinity is also evaluating the drug in a mid-stage study for rheumatoid arthritis.

—Cambridge-based Ironwood Pharmaceuticals (NASDAQ: IRWD) began a Phase 2 study testing its drug linaclotide (Linzess) in patients with opioid-induced constipation. Ironwood aims to report data from the roughly 240-patient study in the second half of 2015. The FDA approved linaclotide as a treatment for chronic constipation and irritable bowel syndrome with constipation in August 2012.