East Coast Biotech Roundup: Regeneron, Sarepta, Jounce, & More

7/11/14Follow @benthefidler

Data that impressed. Data that disappointed. Regulatory strangleholds. Dramatic swings in stock prices. All of it made for just another wild week in East Coast biotech. We’ve got the details below.

—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) made a big splash last year with an experimental antibody drug, dupilumab, it believes could hit the root cause of several allergic diseases. The company and its partner Sanofi this week built the case that one of those diseases could be a chronic form of eczema called atopic dermatitis. Regeneron and Sanofi revealed positive results from a 380-patient, mid-stage trial of dupilumab, adding to the data the drug prospect has already produced in allergic asthma. I spoke with Gianluca Pirozzi, Sanofi’s global head for the dupilumab program, about the next steps for the program, which many analysts believe could be a mega-blockbuster if it continues to hold up in clinical trials.

—There was another twist in the ongoing saga for Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT), as the company released further data from the tiny, mid-stage trial of its Duchenne Muscular Dystrophy drug, eteplirsen. The results, which come after 144 weeks of testing, revealed that the ability of the boys in the study to walk has declined of late, rather than improved or held firm—as it has at previous data collection points. The news triggered a wild day of volatility, once again, in Sarepta’s stock. Shares sank more than 25 percent early on, and then rebounded to a roughly 13 percent loss by the end of the day, as investors tried to make sense of the news and what it means for Sarepta and the overall effectiveness of eteplirsen. (The study’s principal investigator, for instance, noted that the data show a significant improvement when compared to the natural history data on Duchenne—a crippling, progressive disorder that robs patients of their muscle function over time.) Sarepta will continue to track the progress of the boys in the trial as it winds its way towards a meeting with an FDA advisory panel next year—surely to be one of the closely-watched regulatory events in biotech in some time. Sarepta will file a new drug application with the FDA by the end of the year. You can read more about the data and its implications at Forbes, TheStreet.com, and FierceBiotech.

—Basking Ridge, NJ-based Regado Biosciences (NASDAQ: RGDO) made a massive gamble trying to run a 13,200-patient cardiovascular study on an anticoagulant drug without the help of a Big Pharma partner. That bet isn’t looking good these days. First, potential safety concerns raised by an independent data monitoring committee caused Regado to suspend its big trial. Then, the FDA stepped in a few days later and formally placed a clinical hold on the study—formally putting itself in the middle of any decision to restart enrollment or dosing of Revolixys Kit, Regado’s anticoagulant. Regado already had a rough time pricing its IPO last year due to its risky strategy, and the latest news sent many shareholders running—-the company’s stock has plummeted more than 60 percent since the news first broke on July 3.

—Cambridge-based Jounce Therapeutics is one of the more ambitious biotech startups to come out of Boston in recent years, and now it’s picked a former Merck executive to lead its plan to create a portfolio made up of diverse cancer immunotherapy approaches. Richard Murray was named Jounce’s first full-time CEO this week, moving on from Whitehouse Station, NJ-based Merck, where he was most recently the company’s senior vice president of biologics and vaccines. Jounce got started last year with a $47 million round led by Third Rock Ventures and support from experts at MD Anderson, Johns Hopkins University, the University of Chicago, and Georgetown University. I spoke with Murray this week, who immediately faces a big decision—which programs to move forward first.

—Summit, NJ-based Celgene’s (NASDAQ: CELG) plan to expand the potential use of its first inflammation drug, apremilast (Otezla), took a hit this week, as the company reported that the drug failed a late-stage trial for ankylosing spondylitis, a form of arthritis affecting the spine. Apremilast, a pill, is already approved to treat psoriatic arthritis, and Celgene is seeking to establish it as a treatment for other inflammatory disorders like psoriasis. Celgene, for its part, said that it still hasn’t given up in ankylosing spondylitis, pointing to a subgroup of patients who responded better than others. The company may run another Phase 3 trial after it collects more data.

—Newton, MA-based AesRx became a notable win for the National Institutes of Health’s National Center for Advancing Translational Sciences this week when it was acquired, for an undisclosed sum, by Baxter International (NYSE: BAX). AesRx was formed in 2008 by Stephen Seiler, who scooped up a sickle cell disease prospect from a now-defunct New Jersey company called Xechem and kept it on life support until he formed a key partnership with the NIH’s NCATS group in 2010. That deal enabled AesRx to nurture its drug prospect, now called AES-103, through the “Valley of Death”—a translational gap where projects can’t get the funding they need to become opportunities that venture firms or drugmakers want to advance—and ultimately into the hands of Baxter. NCATS director Christopher Austin noted in a statement that the deal “validates the NCATS model.” AES-103 is now in Phase 2 testing.

—Newly-named, Cambridge-based Akashi Therapeutics (formerly Dart Therapeutics), a startup founded by a group of disease foundations, presented the first clinical data on its prospective Duchenne Muscular Dystrophy treatment, HT-100. The drug candidate is meant to help remedy the fibrosis and inflammation associated with Duchenne. Akashi has received an orphan drug and fast-track designation from the FDA for HT-100.

—Capitalizing on the recent clinical success of one of its gene therapies, Cambridge-based Bluebird Bio (NASDAQ: BLUE) cashed in this week, raising about $95.6 million by pricing 3 million shares at $34.00 apiece. It’ll primarily use the cash for clinical studies of its gene therapy programs, like Lenti-Globin for beta-thalassemia and sickle cell disease, and Lenti-D for childhood cerebral adrenoleukodystrophy.

—Merck will likely be the first company to bring a PD-1 checkpoint inhibitor cancer treatment to market in the U.S., but New York-based Bristol-Myers Squibb may not be that far behind. Bristol announced this week that it plans to file an application with the FDA in the third quarter to approve nivolumab, its big cancer immunotherapy prospect, as a melanoma treatment. Nivolumab was approved in Japan earlier this week as well. Bristol also aims to file an application in lung cancer by the end of the year, and is testing nivolumab in a variety of other cancers as well.

—Long Island’s Cold Spring Harbor Laboratory got a big $50 million gift from Jim and Marilyn Simons to establish the Simons Center for Quantitative Biology, which will bring together experts in applied mathematics, computer science, physics, and engineering to advance research into diseases like autism, cancer, bipolar disorder, and depression. Cornell University professor Adam Siepel will leave his post to chair the Simons Center starting in September. Jim Simons is the billionaire mathematician and hedge fund manager who founded Renaissance Technologies. Marilyn Simons heads the Simons Foundation, a financier of basic scientific research.

—Fresh off its $96 million IPO, Cambridge-based Zafgen (NASDAQ: ZFGN) was awarded an orphan drug designation by the European Commission for its prospective fat-burning drug, beloranib, as a treatment for Prader-Willi Syndrome. Zafgen has already received that designation from the FDA. Zafgen has said it plans to use its IPO cash partly to fund a late-stage trial in Prader-Willi, a rare genetic disorder that causes severe overeating.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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