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East Coast Biotech Roundup: Editas, Hep C, Akashi, Tarveda & More

Xconomy Boston — 

January is just about over, and for biotech investors, that’s a good thing. The Nasdaq Biotechnology Index (NASDAQ: IBB) has plummeted more than 22 percent in January. Thursday alone the index fell more than 3.6 percent without any clear negative catalyst (see this Fortune article), prompting a series of notes from sell-side analysts trying to make sense of the situation. RBC Capital Markets’ Michael Yee, for instance, led a note with “Biotech—what the heck is going on?” cited a sentiment change for the sector, and added, “nobody seems to want to try to catch a falling knife.” What type of trouble does that spell out for the glut of biotechs that filed for IPOs at the start of the year? We’ll know soon, which brings us to our first roundup item…

—Cambridge, MA-based Editas Medicine inched closer to completing the first IPO of a company using the gene editing technology CRISPR-Cas9. The company aims to sell 5.9 million shares at $16 to $18 apiece and price the offering next week. Will the markets agree with Editas’ proposed value? It’s an open question, especially considering the rough start to 2016 for biotechs. Alex Lash has more on this, the evolving CRISPR landscape, and the high profile battle over who invented the landmark technology.

—The company formerly known as Blend Therapeutics raised $38 million and rebranded this week as Tarveda Therapeutics, a reflection of a strategic shift the Watertown, MA-based company first disclosed a year ago. Tarveda, led by former Clinical Data CEO Drew Fromkin, is now focused exclusively on developing miniature biologic conjugate drugs called “Pentarins,” with the first expected to begin human clinical testing this year. The company also spun the last remnant of its old strategy—a cisplatin-based chemotherapy drug—into a separate entity called Placon Therapeutics.

—Cambridge, MA-based Akashi Therapeutics suspended a clinical trial of its prospective Duchenne muscular dystrophy drug, HT-100, after a patient in the study began “experiencing serious, life threatening health issues.” Akashi didn’t disclose what those issues were, but it’s working with the FDA to find out. The news comes just two weeks after Akashi signed a partnership with Grunenthal Group on HT-100, and amidst a volatile few months for the Duchenne community. The FDA rejected one potential treatment from BioMarin Pharmaceutical (NASDAQ: BMRN); another, from Sarepta Therapeutics (NASDAQ: SRPT), awaits an advisory panel that was recently postponed because of inclement weather.

—Researchers at the Broad Institute of MIT and Harvard, Havard Medical School, and Boston Children’s Hospital published a study in Nature illustrating a genetic variant that could help cause schizophrenia. The New York Times has more on the study, which is a step forward in understanding the complicated neurological disease.

—Cambridge-based Codiak BioSciences raised a $61 million Series B round, adding to a $31 million Series A the startup closed in November. Codiak, which is led by former Biogen R&D chief Doug Williams, aims to use exosomes—tiny bubbles that shepherd genetic material in and out of cells—as drugs and diagnostics.

The Boston Globe first reported that Massachusetts attorney general Maura Healey sent a letter to Gilead Sciences, of Foster City, CA, threatening potential legal action against the company if it doesn’t reduce the price of its blockbuster hepatitis C medicines sofosbuvir (Sovaldi) and ledipasvir-sofosbuvir (Harvoni). Incidentally, potential pricing pressure immediately came from elsewhere, however. Merck (NYSE: MRK) won FDA approval of a new hepatitis C drug combination, grazoprevir/elbasvir (Zepatier) and priced it at $54,600 for a 12-week course of treatment. While that appears to be a big discount to the $94,500 list price of Harvoni and AbbVie/Enanta Pharmaceuticals’ $83,319 launch price for Viekira Pak, Evercore ISI analyst Mark Schoenebaum noted that the net price of Zepatier “likely won’t be known for several months,” so it’s “premature” to directly compare prices. Harvoni’s average net price, for instance, is $51,000 for 12 weeks, noted RBC’s Yee.

—Separately, Merck inked a partnership with the U.K.’s Cancer Research Technology to develop drugs that block protein arginine methyltransferase 5, which may have use treating blood disorders and cancer. CRT, the commercial arm of Cancer Research U.K., got $15 million up front and is eligible for another $500 million in downstream payments if the drugs progress.

—Scientists at Columbia University Medical Center and the University of Iowa published a study in Nature in which they used the gene editing technology CRISPR-Cas9 to repair a genetic mutation that causes retinitis pigmentosa, a condition that leads to blindness and has no cure. The study supports the notion that CRISPR’s first human therapeutic impact will be on eye diseases.

—New York-based Bristol-Myers Squibb (NYSE: BMY) stopped a Phase 3 study of its immuno-oncology drug nivolumab (Opdivo) in head and neck cancers early, for a good reason—an independent committee concluded that the study met its primary goal, demonstrating a clear survival benefit. Nivolumab was the first so-called checkpoint inhibitor to win a regulatory approval, and is already approved for melanoma, non-small cell lung cancer, and kidney cancer.

—New York-based Authorea raised $1.5 million in a round led by Lux Capital.  The firm has developed a GitHub-style collaboration platform researchers can use to share their findings. Joao-Pierre Ruth profiled how researchers are using Authorea to study the Ebola virus, for instance, in September.

—Boston-based Vertex Pharmaceuticals (NASDQ: VRTX) won approval in Canada of its cystic fibrosis combination drug, lumacaftor/ivacaftor (Orkambi). It also disclosed its fourth quarter and full 2015 earnings, which included $351 million in net sales for Orkambi, which the FDA approved in July. The drug is crucial in helping Vertex finally become profitable.

Photo “Bulle und Bär Frankfurt” courtesy of Eva K. via Commons.