Once-troubled Verastem wins FDA nod for AbbVie-abandoned lymphoma drug Copiktra

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Before winning an FDA nod, Copiktra was sold to Verastem for no upfront fees at a time when it showed weak efficacy data and the biotech was reeling from its own trial failure. (FDA)

Verastem Oncology has come a long way from the major failure of its lead candidate defactinib to its first FDA approval. And it's done it with a drug that itself had a troubled past.

Once an unloved child by Infinity Pharmaceuticals and AbbVie, duvelisib, to be marketed as Copiktra, has nabbed an FDA green light to treat patients with relapsed or refractory chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and follicular lymphoma (FL) after at least two prior therapies.

Versatem is ready to roll, having priced the twice-daily oral PI3K inhibitor at a monthly list price of $11,800, and it's expected to make the drug available within a week, the company’s chief commercial officer, Joe Lobacki, said on a conference call Monday.

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Now that Copiktra has become the first-approved dual inhibitor of PI3K-delta and PI3k-gamma, it will compete toe-to-toe against Gilead’s Zydelig, the first PI3K inhibitor, and Bayer’s Aliqopa, which just won an FDA accelerated approval in FL a year ago. For the first six months of this year, Zydelig registered sales of just $72 million.

RELATED: Bayer gears up to take on Gilead in lymphoma with Aliqopa green light

Copiktra’s label does carry a black-box warning of four potentially fatal toxicities—infections, diarrhea or colitis, cutaneous reactions and pneumonitis—though CEO Robert Forrester called the warning “appropriate and expected.”

With this approval, Verastem has finally entered the commercial stage. According to Lobacki, the company has assembled a 50-person oncology sales team, as well as medical affairs and patient advocacy teams, that are in place ready to roll. As of June 30, the company had $169 million in cash and cash equivalents at its disposal.

AbbVie had previously shelled out $275 million upfront to partner with Infinity on the PI3K inhibitor, but it walked away in 2016 after seeing weak, though primary endpoint-meeting, phase 2 data in indolent non-Hodgkin lymphoma. The termination of the collaboration triggered a massive layoff round at Infinity, which later licensed the drug to Verastem for no upfront payment and merely $28 million in milestones, as well as single-digit royalties.

While Infinity may have been eager to get rid of an asset that looked commercially unattractive, the deal was lifesaving for Verastem. The biotech was at the time reeling from a stock-crashing lung cancer flop for its lead drug defactinib that spurred the high-profile board resignations of Christoph Westphal and Henri Termeer.

RELATED: Novartis’ PI3K drug hits goal in phase 3 breast cancer trial, laying groundwork for talks with regulators

Copiktra’s clinical profile was apparently good enough to win plenty of FDA favor. In a disappointing phase 2, it showed an overall response rate of 46%. A 42% response rate among the FL subgroup led to the FDA accelerated approval in that indication, meaning Verastem will need to perform further studies to confirm its clinical benefit.

The phase 3 looks more like a win. In this study, Copiktra cut the risk of progression or death by 48% compared with Novartis’ Arzerra. Specifically, CLL/SLL patients who got Copiktra achieved median progression-free survival of 16.4 months, versus 9.1 months in the Arzerra arm.