East Coast Biotech Roundup: Amicus, Accelerator, Seventh Sense, & More

Xconomy Boston — 

New York’s biotech scene is getting a jumpstart, and a Seattle startup incubator is getting a second wind in the process. One of biotech’s high fliers in 2014 soared once again. And one local biotech is awaiting some critical data that could make or break its future. Those stories and much more below.

—A single clinical setback can often be the death knell for a small biotech. But despite failures with two separate drugs, Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD) has been able to survive and regroup. And now the company could be on the verge of actual success. Results are due any day now for a critical Phase 3 trial of its Fabry Disease drug, migalastat. The trial results are good, Amicus will start preparing an FDA application. If not, it will essentially become a preclinical company once again. With all that on the line, I spoke with CEO John Crowley about the highs and lows, and the series of moves Amicus has taken to stay alive as it awaits its latest day of reckoning.

—Sometime soon, Seattle’s Accelerator will open up a new office in New York. And when it does, it’ll mark an important moment both for the startup incubator and New York biotech. Despite all of its financial and research power, Gotham’s life sciences scene has been notoriously tough to negotiate for early-stage companies due to high prices and a lack of lab space. And Accelerator, CEO Thong Le concedes, has made its share of mistakes in the past—and now aims to change its investment strategy as a result. I spoke with Le about the move, the lessons Accelerator has learned over the course of its existence, and how he hopes to help energize New York biotech.

—People hate getting their blood drawn because of the big needles and finger pricks. Cambridge, MA-based Seventh Sense Biosystems is developing a quick and painless alternative, and just landed $16 million and the support of three big industry players—Novartis, LabCorp, and a unit of Siemens—to help get it to market. Many challenges remain, of course, but CEO Howard Weisman says that the company aims to start by introducing its so-called touch activated phlebotomy technology within the next year or two, and eventually give it the capability to perform diagnostic tests as well.

—Tokai Pharmaceuticals has been chasing after some of the big players in the prostate cancer field for some time. Now, as the Cambridge company prepares to ramp up its first Phase 3 trial, it’s looking to Wall Street for help. Tokai filed papers this week outlining plans to raise up to $75 million in an IPO. The company has been developing a drug called galeterone, which is meant to block the hormones that feed prostate cancer cells in three different ways, borrowing tactics used by two other successful prostate cancer drugs—Medivation/Astellas’ enzalutamide (Xtandi) and Johnson & Johnson’s abiraterone (Zytiga)—in the process. Tokai believes it’s identified a subgroup of prostate cancer patients that enzalutamide and abiraterone don’t effectively treat, but could benefit from its own drug, and plans to use the IPO cash in part to fund a Phase 3 trial testing that theory.

—It was another roller coaster week for New York-based Intercept Pharmaceuticals (NASDAQ: ICPT). Its shares surged almost 60 percent initially when more detailed data were released from the Phase 2 trial of its drug for nonalcoholic steatohepatitis (NASH), then settled in to a 25 percent total gain for the week as of early Friday. The additional data from Intercept’s trial appeared to alleviate some concerns about the cardiovascular effects tied to its drug, obeticholic acid (OCA), leading to a series of analyst upgrades for the stock. All eyes will be on Intercept, one of the darlings of biotech this year, as it brings OCA into Phase 3 studies for NASH in 2015. The company should file an FDA application for OCA in primary biliary cirrhosis next year as well.

—Cambridge-based Vertex Pharmaceuticals (NASDAQ: VRTX) revealed in a letter written to healthcare providers this week that it will stop selling telaprevir (Incivek)—its once high-flying hepatitis C drug—on Oct. 16. Telaprevir had one of the most successful drug launches in history, but its run was cut short by a wave of new all-oral, interferon-free regimens for the liver disease. Gilead Sciences’ sofosbuvir (Sovaldi) is the first of the group, and has already racked up close to $6 billion in sales in just its first half-year on the market. Vertex, meanwhile, has shifted all of its focus to its cystic fibrosis program, and has made a number of moves over the past year to phase out its work in hepatitis C.

—Lexington, MA-based Taris Biomedical this week brought in what could end up as a big haul for its investors, as Irvine, CA-based Allergan (NYSE: AGN) agreed to acquire worldwide rights to its drug-delivery device, known as LiRis, for $67.5 million up front and potentially another $525 million in milestone payments down the road. Taris has raised roughly $50 million from the likes of Flagship Ventures, Polaris Partners, Flybridge Capital Partners, Third Rock Ventures, and others. The company, founded by MIT professors Michael Cima and Bob Langer, developed an implantable device that delivers the common anesthetic lidocaine over a few weeks. Taris has been testing the device in Phase 2 studies in interstitial cystitis, also known as painful bladder syndrome.

—Cambridge-based Agios Pharmaceuticals (NASDAQ: AGIO) received a fast-track designation from the FDA for AG-221, a cancer metabolism drug it’s developing to treat a subgroup of patients with acute myeloid leukemia. The designation is supposed to speed up development and review of the drug, which is currently in early-stage testing. In case you missed it, I profiled the first clinical results that AG-221 produced back in April.

—Shares of Cambridge-based Epizyme (NASDAQ: EPZM) surged more than 12 percent this week off of early data it released at the American Society of Hematology’s meeting on Lymphoma Biology in Colorado. The company reported some preclinical and interim Phase 1 data results for its cancer drug prospect EPZ-6438, in patients with advanced solid tumors and B-cell lymphomas. Epizyme is trying to figure out the dose of EPZ-6438 it’ll take to a Phase 2 study, and said it has yet to hit the maximum tolerated dose yet. Epizyme aims to reveal its first full Phase 1 results later this year.

—Summit, NJ-based Celgene (NASDAQ: CELG) made an undisclosed equity investment in J. Craig Venter’s genomics startup, Human Longevity this week, and also handed the company rights to develop and co-market PSC-100, a placental stem cell treatment that Celgene has already tested in Phase 1 trials. Human Longevity will use its sequencing technology to complement those studies, and look into potential applications for PSC-100’s use in treating diseases such as sarcopenia, a degenerative muscle condition.