|Amgen R&D Chief Sean Harper|
Amgen has scored boasting rights to a breakthrough drug designation from the FDA for its promising mid-stage leukemia drug blinatumomab.
The coveted breakthrough title was won on promising Phase II data on adults with Philadelphia-negative relapsed or hard to treat B-precursor acute lymphoblastic leukemia--ALL--a lethal cancer of the blood and bone marrow.
Today's action takes Amgen ($AMGN) a big step closer to an NDA. The BTD title also comes as Amgen's R&D group is under increasing pressure to perform. Now it can advertise the near-term prospects of blinatumomab, joining key therapies like T-Vec and its PCSK9 drug evolocumab (AMG 145).
Unlike some of the earliest breakthrough titles, which included drugs already under review at the FDA, Amgen stands to benefit greatly from the FDA's open-door policy on its BTD portfolio. FDA cancer chief Richard Pazdur has made no secret of his willingness to hurry along new drugs that offer a better standard of care for patients--particularly in late-stage cases where patients have dwindling options to choose from. And Amgen R&D chief Sean Harper has already highlighted Amgen's interest in shooting for an accelerated approval of this drug based on Phase II data.
The drug is a bispecific T cell engager (BiTE) which, simply put, uses an antibody to redirect killer T cells to destroy tumor cells. New immuno-oncology therapies that spur an immune system attack on cancer have become a hot field in cancer, which has been attracting some heavy investments among the Big Pharma crowd. And Roger Perlmutter--now head of R&D at a resurgent Merck ($MRK)--had become clearly enamored with the drug when he decided to buy out the biotech in the $1.2 billion Micromet buyout.
The Phase II study met its primary endpoint, a spokesperson for Amgen tells FierceBiotech, 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.
"There is a high unmet need for new medicines to treat relapsed and refractory ALL patients, who have very few treatment options," said Harper in a statement. "The results from the Phase II trial evaluating blinatumomab in adult patients with relapsed or refractory ALL are encouraging and provide a strong basis for a regulatory filing later this year and potential approval in this serious disease."
- here's the release