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East Coast Biotech Roundup: Agios, Vertex, Epizyme, Celldex, & More

Xconomy Boston — 

One of the year’s biggest cancer meetings took place this week in Barcelona, and many biotechs from the East Coast were angling for the spotlight. Some wowed investors, others, not so much. We’ve got the ups and downs and the rest of the week’s news and tidbits below:

—Agios Pharmaceuticals (NASDAQ: AGIO) was one of the week’s big winners. Shares of the Cambridge, MA-based company jumped some 14.5 percent after it showed that the early data for AG-120, its second drug for a subset of patients with acute myeloid leukemia, mirrored the results for its first drug, AG-221—meaning, it, too, led to multiple complete remissions. I spoke with CEO David Schenkein about the data, and what’s next for Agios.

—Hampton, NJ- and Needham, MA-based Celldex Therapeutics (NASDAQ: CLDX) also had a big week on Wall Street. Shares surged almost 45 percent after its experimental brain cancer vaccine, rindopepimut, helped extend the lives of a small group of people in a mid-stage trial with recurrent glioblastoma. I spoke with Celldex executives about their plans for the vaccine, which include a discussion with the FDA about the idea of accelerated approval.

—Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD) released some more data from a phase 3 trial of migalastat, its pill for Fabry Disease, showing that the treatment helped reduce the rate of cardiac complications in patients with the disorder—a major cause of death for people with Fabry. Shares immediately climbed 18 percent, and Amicus quickly turned around and raised $90 million through a stock offering. For those unfamiliar with Amicus’s long road with migalastat, here’s a piece I wrote about the journey in August, and the results from the study that came out shortly thereafter.

—Cambridge-based Epizyme (NASDAQ: EPZM) saw about a 5 percent bump in share price following some updated data from a Phase 1 study of EPZ-6438, a drug it’s testing in patients with a variety of blood cancers or solid tumors. Eisai has worldwide rights to the drug, though Epizyme can exercise an option to co-commercialize and share profits in the U.S.

—Investors weren’t as thrilled with the Phase 2 data from Bind Therapeutics (NASDAQ: BIND) and its experimental nanoparticle-based cancer therapy, BIND-014. Bind touted the results as positive and noted some encouraging findings in tough-to-treat groups—like those with non-small cell lung cancer and KRAS mutations, as well as patients with squamous cell carcinoma. It’ll now start up some mid-stage trials for those specific patient groups. But shares sank more than 9 percent nonetheless on the data.

—The Cystic Fibrosis Foundation has invested $150 million in Vertex Pharmaceuticals (NASDAQ: VRTX) to help it develop drugs for the devastating lung disorder, and this week it made a huge return on that investment. The CF Foundation sold its royalty stream in Vertex’s ivacaftor (Kalydeco) and some other experimental drugs for CF, to Royalty Pharma for $3.3 billion.

—Shire (NYSE: SHPG) said this week that it plans to move more than 500 jobs from from its Chesterbrook, PA, site to Lexington, MA—which will be the new headquarters of its U.S. operations. Shire aims to finish the move by early 2016.

—Cambridge-based Dicerna Pharmaceuticals (NASDAQ: DRNA) cut a $24 million deal with Tekmira Pharmaceuticals (NASDAQ: TKMR) to use the company’s lipid-nanoparticle delivery system for its experimental RNA interference drug, DCR-PH1. Dicerna is devleoping the drug for primary hyperoxaluria type 1, a rare genetic disease that often leads to kidney failure. It’ll start testing DCR-PH1 in its first clinical trials next year.

—The FDA gave Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi a breakthrough therapy designation for their antibody drug dupilumab as a treatment for atopic dermatitis, commonly called eczema. A phase 3 study of dupilumab in eczema is currently underway, and the designation should speed the agency’s review of the drug..

—New York-based Pfizer (NYSE: PFE) cut a $2.85 billion deal with Germany’s Merck KGaA to co-develop a PD-L1 checkpoint inhibitor for multiple types of cancer. Pfizer paid $850 million up front and could shell out another $2 billion in milestone payments for the drug. The two companies will also work together to move Pfizer’s PD-1 antibody into Phase 1 testing.

—Cambridge-based Merrimack Pharmaceuticals (NASDAQ: MACK) got a fast-track designation from the FDA for MM-398, the nanoparticle-based drug it’s developing for pancreatic cancer. Merrimack aims to complete an application to approve the drug by early 2015.