Amgen pushes for early FDA OK of leukemia drug blinatumomab

Bolstered by a breakthrough drug designation at the FDA, Amgen ($AMGN) is following through with a new drug application for its leukemia drug blinatumomab, hoping that promising Phase II data will be enough to push this program past the goal line on a greatly accelerated development schedule.

The FDA's cancer division under Richard Pazdur has been lowering the bar for new cancer drugs that may be able to help some very sick patients. In this case Amgen is focused on a bispecific T cell engager (BiTE) which, simply put, uses an antibody to redirect killer T cells to destroy tumor cells. And now it wants a green light to sell the treatment for adults with Philadelphia-negative elapsed/refractory B-precursor acute lymphoblastic leukemia.

Roger Perlmutter

New immuno-oncology therapies that spur an immune system attack on cancer have become a hot field in cancer, which has been attracting some heavy investments among the Big Pharma crowd. And former Amgen R&D chief Roger Perlmutter--now head of R&D at a resurgent Merck ($MRK)--had become clearly enamored with the drug when he decided to buy out the drug in the $1.2 billion Micromet buyout.

The Phase II study met its primary endpoint, with 43% of patients achieving complete remission or complete remission with partial hematologic recovery within two cycles of treatment with blinatumomab. The data were released at ASCO.

For Amgen the filing backs up its insistence that the company has turned things around on the R&D side of the business at the Big Biotech. Along with T-Vec and the PCSK9 drug evolocumab, it's one of several late-stage therapies angling for a near-term approval.

Amgen, which recently signaled that it will shutter a big research facility in Seattle as part of a major reorganization that will cost thousands of jobs, has received mixed messages from the investment community on just how effective it has been at drug development.

Bernstein's Geoff Porges has been one of Amgen's biggest critics, prodding Amgen repeatedly to join the Big Pharma breakup party, complaining that Amgen's stock price has languished as other companies of a similar size have done much, much better this year. He has criticized Amgen of having a portfolio of aging legacy products looking at biosimilar competition and a "scattered" pipeline that includes too many disparate programs, including blinatumomab.

Amgen, though is staying upbeat today.

"The filing for blinatumomab brings us a step closer to first realizing the potential of BiTE technology and represents our commitment to evaluating this novel approach in a broad range of difficult-to-treat cancers," said Sean E. Harper, M.D., executive vice president of R&D at Amgen.

- here's the release