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Celgene Plots Speedy FDA Filing for Agios Blood Cancer Drug

Xconomy Boston — 

It typically takes about 14 years to discover a cancer drug, develop it, and get it to market. Agios Pharmaceuticals has a chance to beat that timeline.

Cambridge, MA-based Agios (NASDAQ: AGIO) said in a regulatory filing this morning that its partner Celgene (NASDAQ: CELG), of Summit, NJ, expects to file for FDA approval of a blood cancer drug they’re developing, enasidenib, by the end of the year. Celgene will discuss the planned application at a conference in Boston this morning.

The application would be filed on an “accelerated” basis, meaning Celgene would head to the FDA seeking approval on a thinner body of evidence than usually required for a drug. According to the statement, the application would be based on data from Agios’s ongoing early-stage trial of enasidenib (formerly AG-221), which is being studied in patients with the blood cancer acute myeloid leukemia who have a specific genetic mutation. Agios also has a Phase 3 trial of enasidenib underway, but Celgene’s application would be in the FDA’s hands before data from that trial are released.

Agios said that it intends to follow a similar path for another blood cancer drug in its portfolio, AG-120. That drug is slightly behind enaseidenib in development, meaning Agios could file for FDA approval next year. An update regarding AG-120’s regulatory path is expected at the end of the year.

Shares of Agios climbed almost 22 percent, to $45.45 apiece, in pre-market trading on Wednesday.

With enasidenib, Agios has been on one of the quicker development paths for a cancer drug in recent memory. It discovered the drug in 2009, filed papers with the FDA (a so-called investigational new drug application, or IND) to begin its first trial of the drug in June 2013, and dosed the first patient two months later. Should Celgene file for FDA approval of the drug this year and get it to market in 2017—still a big if—that would be a roughly four year timeline from IND to market, and eight years from discovery.

By comparison, it typically takes about eight years for a cancer drug to go from the start of its first trial to approval, and an average six years of development before that, according to the American Cancer Society. Some notable exceptions include Novartis’s imatinib (Gleevec), Pfizer’s crizotinib (Xalkori), and Millennium Pharmaceuticals’s bortezomib (Velcade).

There are still many questions yet to answer about enasidenib’s safety in the long term, effectiveness, and durability. But clearly Celgene has seen enough to take a shot. Agios has provided a variety of interim updates from the early-stage trial, and expanded the study a few times to include more and more patients at different stages of AML progression and of different ages. Another update on the trial will come later this year at the American Society of Hematology’s annual meeting in San Francisco.

Celgene holds worldwide rights to enasidenib, and is paying all of its development costs; Agios would get milestone payments and royalties on the drug if it continues forward. Agios regained full rights to AG-120 after the two companies revamped their partnership earlier this year.

Both drugs target enzymes that help generate energy within cells. When those enzymes—known as IDH2 and IDH1—are mutated in cancer, they create a byproduct that flips a genetic switch in immature bone marrow cells. Those cells then don’t develop properly, and start multiplying wildly, rather than becoming the blood cells they were supposed to become.

By binding to the mutated enzymes, enasidenib and AG-120 are supposed to block the byproduct and allow bone marrow cells to mature normally. As such, they work very differently than chemotherapy, which kills cancer and healthy cells alike. If they make it to market, the drugs could represent a potential paradigm shift in the treatment of AML—a fast-moving blood cancer whose only cure is a bone marrow transplant that is only available to a small number of patients. Between 9 and 13 percent of patients with AML have the IDH2 mutation. Aberrant IDH1 is found in about 6 to 10 percent of patients. AML affects about 18,000 people in the U.S. every year.

Here’s more on Agios, which went public in 2013 at $18 per share.