The wait continues. An FDA ruling on a Duchenne muscular dystrophy drug from Sarepta Therapeutics—which could be the first ever approved for the disease—has been imminent for a while now, yet Labor Day is fast approaching and still the saga drags on. Speculation abounds, from analysts and pundits, each giving different opinions as to whether the delay means good or bad things for Sarepta and Duchenne patients. Will next week be the week a decision finally comes? Next month? Later today? Until then everyone sits, waits, and speculates. That story and the rest of the week’s biotech headlines below.
—The U.S. government’s Precision Medicine Initiative is an ambitious effort to collect the genomic data of 1 million Americans, and tech giant Alphabet (NASDAQ: GOOG) is deeply involved. Alex Lash examined the company’s role, via its biotech subsidiary Verily Life Sciences, in the massive project. In other Google-related news, Recode reported that Bill Maris, the founder and president of Google Ventures (now called GV), has left the firm and will be replaced by David Krane.
—David H. Koch Institute for Integrative Cancer Research founding director Tyler Jacks has spent decades making important contributions in cancer research, but until forming Cambridge, MA-based Dragonfly Therapeutics, he’d never spun a startup out of his own lab at MIT. Xconomy spoke with Jacks and CEO and longtime tech entrepreneur Bill Haney about the venture, a cancer immunotherapy startup that is relying on funding from family offices—among them the Disney family—rather than venture firms.
—Option-to-buy deals are by definition no guarantee, and an agreement between Boston-based Acetylon Pharmaceuticals and Celgene is the latest example. Celgene paid Acetylon $100 million in 2013 for an option to buy the company outright, but the deal recently expired without an acquisition, leaving Acetylon to chart a new course. Celgene remains an Acetylon investor, however, and the two are testing drugs together in a variety of trials.
—Indianapolis-based Eli Lilly (NYSE: LLY) said that a breast cancer drug it’s been developing called abemaciclib didn’t meet a predetermined interim efficacy bar in a Phase 3 trial. Lilly will continue the study, but the news puts distance between abemaciclib and two similar, rival cancer drugs from Pfizer (palbociclib (Ibrance)) and Novartis (ribociclib)). Both Pfizer and Novartis stopped Phase 3 trials of their drugs early because they were clearly benefitting breast cancer patients. And Pfizer’s is already on the market, and generated $514 million in the last quarter alone.
—The status of Sarepta Therapeutics’ (NASDAQ: SRPT) Duchenne muscular dystrophy drug eteplirsen remains in regulatory limbo, and the Wall Street Journal published this editorial speculating that a “differing-professional-opinions” proceeding—how the FDA clears up internal scientific disputes—might be holding it up. Meanwhile, the FDA agreed to review an approval application for deflazacort, a steroid that Marathon Pharmaceuticals, of Northbrook, IL, is advancing as a potential Duchenne treatment.
—Heron Therapeutics (NASDAQ: HRTX) won FDA approval of granisetron (Sustol), a drug it developed for the nausea and vomiting associated with certain chemotherapy regimens. The news was a relief for Redwood City, CA-based Heron, which has been waiting for word on the status of its drug since September 2015.
—Biogen (NASDAQ: BIIB) disclosed the name for its coming hemophilia-focused spinoff: Bioverativ. The new company, expected to launch in 2017, will trade on the Nasdaq under the symbol “BIVV” when the split is complete. Its financials were posted here.
—Inflammation drug developer Aldeyra Therapeutics (NASDAQ: ALDX) reported positive results from another mid-stage trial, this time in patients with a rare skin condition called Sjogren-Larsson Syndrome. Lexington, MA-based Aldeyra’s drug, NS2, has already succeeded in two Phase 2 trials for two different eye diseases.
—Kite Pharma (NASDAQ: KITE), of Santa Monica, CA, said that it could file for FDA approval of an experimental treatment known as KTE-C19 by the end of 2016—meaning there’s a chance Kite could be the first to market with a “CAR-T” product, a form of cellular immunotherapy for cancer.
—This week’s gene therapy news: MIT Technology Review reported that Strimvelis, the gene therapy recently approved in Europe and given a $665,000 price tag, has a money-back guarantee if it doesn’t work. In the U.S., Philadelphia-based Spark Therapeutics (NASDAQ: ONCE) bolstered the data it has for genetic blindness treatment voretigene neparvovec, which has a chance to become the first gene therapy approved by the FDA. Its application for approval won’t be completed until next year, however—a delay from previous expectations.
—Financings: AstraZeneca disclosed that it’s invested $140 million more into Cambridge-based Moderna Therapeutics and brought its stake into the privately held messenger RNA company up to 9 percent… Celgene took part in a $37 million round for Cleave Biosciences, a San Francisco Bay Area startup developing cancer drugs… The state of Texas added $3.5 million to San Antonio-based Fe3 Medical’s Series B funding, bringing the total to $14.5 million… Humacyte, of Durham, NC, got a $9.9 million grant from the California Institute for Regenerative Medicine to help fund the development of “off the shelf” bioengineered blood vessels.
—On the IPO trail: Northville, MI-based Gemphire Therapeutics (NASDAQ: GEMP) went public, but Gemphire had to settle for a $30 million raise, rather than the $45 million it previously hoped for. Milpitas, CA-based Protagonist Therapeutics (NASDAQ: PTGX) fared better, raising $90 million in an IPO priced in line with its projections.
—Deals, deals, and more deals: Allergan paid $95 million for privately held Menlo Park, CA-based ForSight VISION5 and its experimental, implantable glaucoma treatment, which dispenses the drug bimatoprost (Lumigan) into the eye over the course of several months… Versartis, of Menlo Park, CA, got $40 million from Tenjin for rights in Japan to its growth hormone deficiency treatment somvaratan… Swiss biopharma Helsinn paid $15 million up front for rights to pracinostat, an experimental acute myeloid leukemia drug from San Diego, CA-based MEI Pharma (NASDAQ: MEIP)… Danish biotech Genmab got $5 million up front from Gilead Sciences (NASDAQ: GILD) for rights to an experimental bi-specific antibody drug for HIV… Zymeworks, of Vancouver, Canada, paid an unspecified licensing fee to grab rights to an antibody discovery technology developed by Innovative Targeting Solutions.
—Cambridge-based Editas Medicine (NASDAQ: EDIT) teamed up with Adverum Biotechnologies (NASDAQ: ADVM) in a pact to develop methods of delivering gene editing therapies to the eye for a group of undisclosed retinal diseases. Menlo Park, CA-based Adverum got $1 million up front in the deal.
—Vedanta Biosciences, based in Cambridge, will work with scientists at NYU Langone Medical Center to develop microbiome-based cancer immunotherapies.
—In Boston-area people news, stem cell scientist George Q. Daley was named the new dean of Harvard Medical School. He’ll begin his tenure on Jan. 1.
— San Antonio, TX-based Inspyr hopes to find a partner to help fund the clinical trials of a drug it’s developing for liver cancer and glioblastoma, according to newly hired CEO Chris Lowe.
—The Metis Foundation, a San Antonio nonprofit focused on helping military researchers land Department of Defense funding, plans to raise $3 million in private funds for cancer vaccine research.
David Holley contributed to this report.