East Coast Biotech Roundup: Concert, Merrimack, Vertex, & More

5/2/14Follow @benthefidler

A near-death experience for an IPO, new life for a cancer drug company, and plenty of clinical trial results dot the roundup this week. Those stories and more below.

—Lexington, MA-based Concert Pharmaceuticals (NASDAQ: CNCE) may have priced its IPO at the top of its range earlier this year, but not without a fair bit of scrambling. In fact, as CEO Roger Tung told me last week, that IPO was almost dead on arrival when the company got some disastrous data on its kidney disease drug, CTP-499 while it was drafting its S-1. I spoke with Tung at length about Concert’s rocky journey to an IPO, the study it had to overcome to get through it, and why Concert thinks that kidney drug still has a shot at the market

—Cambridge, MA-based Merrimack Pharmaceuticals (NASDAQ: MACK) has had a rough go on Wall Street since it went public in 2012. But Merrimack finally broke through with some good news this week when its pancreatic cancer drug, MM-398—a nanoparticle-based version of the chemo drug irinotecan—succeeded in a Phase 3 trial. Merrimack shares jumped more than 50 percent on the news. I spoke with Merrimack CEO Bob Mulroy about the study, and what lies ahead for MM-398.

—The changing hepatitis C landscape caused Cambridge-based Vertex Pharmaceuticals (NASDAQ: VRTX) to wave the white flag on its once high-flying hepatitis C drug, telaprevir (Incivek), a few months ago, and turn its attention to its cystic fibrosis franchise, led by ivacaftor (Kalydeco). This week it exited the hep C field altogether, announcing plans to out-license VX-135, the one remaining prospective hep C drug in its pipeline. Vertex gained rights to the drug via a 2011 deal with South San Francisco-based Alios Biopharma. Vertex’s CF efforts, meanwhile, gained more steam. The company released data Thursday from a mid-stage trial showing that VX-661 improved the lung function of patients with both a F508del mutation and a G551D mutation who were already taking ivacaftor. The news is the latest small step in Vertex’s plan to treat more and more subsets of the cystic fibrosis population by combining ivacaftor with other experimental drugs in its portfolio. A key moment for Vertex is coming later this year, for instance, when the company reveals the results of the so-called “Traffic” and “Transport” studies. These two Phase 3 trials are testing ivacaftor with another experimental CF drug, VX-809, in patients with two copies of the F580del gene mutation. There are about 30,000 CF patients worldwide with the double mutation, representing billions in potential sales for the company.

—Lightstone Ventures closed its first fund this week with $172 million to spend on early-stage investments in biotech and medtech startups. Lightstone’s name has showed up in some biotech and medtech financing rounds over the past few months, like those of Cambridge-based Catabasis, Dublin’s FIRE1, and Redwood City, CA-based EarLens. The firm is headed by life science VCs that hail from Advanced Technology Ventures and Morgenthaler Ventures.

—Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD) got a stock bump this week after it said that its oral Fabry disease drug, migalastat Hcl, posted some positive results in a Phase 3 trial. It’s been a challenging year for Amicus. Migalastat failed to meet its goals in a Phase 3 trial after six months. The company tabled its plan to file a new drug application, but didn’t give up on the drug, instead planning to see how it performed after 12- and 24-month periods. In the meantime, Amicus restructured, and its development partner on migalastat, GlaxoSmithKline, returned rights to the drug to the company. Amicus presented some 12- and 24-month data this week, and intends to discuss an approval pathway with the FDA after a second study wraps up.

—The FDA rejected Ardsley, NY-based Acorda Therapeutics’ (NASDAQ: ACOR) new drug application for a nasal spray formulation of epilepsy drug diazepam (Plumiaz), which Acorda acquired when it bought Morrisville, NC-based Neuronex in 2012. Most of the potential payouts for Neuronex equity holders in that deal—more than $100 million—were tied to future sales milestones, payments those shareholders may now never see. Acorda didn’t say why the FDA rejected the drug, but indicated it’s developing a response to the agency.

—Chelsea, MA-based Civitas Therapeutics reported positive results from a Phase 2b trial of CVT-301, an inhalable version of the Parkinson’s drug levodopa that is administered via a pen-sized device. Civitas’ drug-device combo is being developed as an adjunct therapy to levodopa pills, which are used by most Parkinson’s patients to manage symptoms, but tend to produce erratic levels of drug in the bloodstream. Civitas, formed around a drug delivery technology created by Alkermes (NASDAQ: ALKS), also changed its leadership in January. Co-founder Glenn Batchelder stepped down as CEO and was replaced by former Blend Therapeutics CEO Mark Iwicki.

—The FDA granted Princeton, NJ-based Advaxis (NASDAQ: ADXS) an orphan drug designation for ADXS-HPV as a potential immunotherapy for invasive cervical cancer. ADXS-HPV is the company’s most advanced drug candidate, and is being tested in a variety of human papillomavirus-associated cancers.

—Lexington, MA-based Agenus (NASDAQ: AGEN) this week parlayed its February acquisition of antibody discovery shop 4-Antibody into a collaboration deal with Merck to unearth new “checkpoint inhibitors” to treat cancer. Agenus didn’t get anything up front from the deal, though it could receive up to $100 million in milestone payments from Merck as well as royalty streams from any products that reach the market. Merck is looking for more candidates to pair up with its existing immuno-oncology prospects, led by the PD-1 inhibitor MK-3475.

—Lexington-based startup Avaxia Biologics’ oral anti-tumor necrosis factor (TNF) drug prospect, AVX-470, hit its marks in a Phase 1b trial in ulcerative colitis, and will move into Phase 2 testing. The drug got AbbVie’s attention last year—its VC arm, AbbVie Biotech Ventures, jumped into an $11.4 million Series B round for Avaxia and got a seat on the company’s board to get a closer look.

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

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