- Following a discussion with the Food and Drug Administration, Verastem Inc. is putting together plans to file sometime in the first quarter of 2018 for U.S. approval of duvelisib, the drug it acquired from Infinity Pharmaceuticals Inc. last year.
- Buoyed by the September success of a Phase 3 study known as DUO, Verastem aims to win full approval of the PI3K dual inhibitor in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).
- In addition to those two indications, Verastem also hopes to secure a conditional approval for duvelisib in relapsed or refractory follicular lymphoma based on data from the Phase 2 DYNAMO study.
Verastem snagged exclusive rights to duvelisib a year ago from struggling biotech Infinity Pharmaceuticals for just $28 million in potential milestone payments, after some less-than-positive Phase 2 data triggered a swift exit by Infinity's partner AbbVie Inc.
That bet paid off earlier this fall when results from the DUO study showed treatment with duvelisib resulted in a median progression-free survival (PFS) of 13.3 months — 3.4 months longer than in patients treated with Novartis AG's Arzerra (ofatumumab) and meeting the threshold for statistical significance.
Risk of disease progression or death was lowered by 48%, according to the study results.
"With FDA guidance now in hand, we have a defined regulatory path forward for the duvelisib NDA," said Verastem CEO Robert Forrester in an Oct. 31 statement. "Our near-term focus will be on preparing the NDA, which we expect to submit during the first quarter of next year."
Entry of newer targeted therapies, such as AbbVie's Imbruvica (ibrutinib) and Venclexta (venetoclax), and Gilead Sciences Inc.'s Zydelig (idelalisib) have improved outcomes for CLL patients. By aiming at relapsed/refractory CLL/SLL patients, Verastem hopes to first carve out a niche for duvelisib as a secondary option.
Whether the FDA looks favorably on the drug's profile remains to be seen. Verastem offered little color on its meeting with the regulator, besides noting that it had also received written feedback. More details should be forthcoming at this year's annual meeting of the American Society for Hematology in December, where Verastem plans to present detailed results from the DUO study.
The news doesn't appear to have sparked investor interest, however. Shares in the company were little changed from Tuesday.