Agios Taps Wall Street Off Early Data, Aims For $75M

4/10/14Follow @benthefidler

Agios Pharmaceuticals gave investors its first glimpse of encouraging, but early human clinical data a few days ago, and saw its shares soar as a result. Now, it’s looking to cash in.

Cambridge, MA-based Agios (NASDAQ: AGIO) wants to raise up to $75 million through a public stock offering, according to a regulatory filing. Agios had about $194 million in cash at the end of 2013, and is eyeing the extra dollars from the public markets to move its prospective cancer metabolism drugs—AG-221, AG-120, and AG-348—through their respective early-stage studies.

The offering comes during a week where Agios’ shares have climbed more than 36 percent. Its shares closed Wednesday at $48.42 apiece, the company’s highest closing price since debuting on the Nasdaq in July.

Investors have piled into the stock after Agios produced an interim look at its first clinical trial for AG-221, a potential treatment for cancers with the IDH2 mutation. Agios only has data from seven patients with acute myeloid leukemia so far, but six of them responded to AG-221, and three of them had no trace of cancer in their blood after 28 days of treatment.  This is despite the fact that those patients only received the lowest two of four planned dosages of Agios’s drug.

There’s still much data to accrue, and it’s unclear at this point how durable each of these responses are—or if Agios will even get the same type of responses out of the next batch of patients. But the results were a promising sign for Agios’s approach of going after cellular metabolism targets. The data, for instance, had analysts talking up streamlined regulatory approval paths for AG-221.

“While patient numbers are small, there appears to be more than enough activity to support a potential accelerated approval strategy based on a single arm Phase 2 trial assuming even a 2-3 month duration of response,” wrote Leerink Partners analyst Howard Liang in a note to investors.

Summit, NJ-based Celgene (NASDAQ: CELG) holds worldwide rights to AG-221 as part of the partnership it struck with Agios in 2009. About 10 to 15 percent of patients with AML, a fast-moving type of blood cancer, have the IDH2 mutation. AML patients are typically treated with chemotherapy, and often wind up needing bone marrow transplants.

You can read my wrap on the AG-221 study results here.

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

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