Verastem bet on blood cancer drug pays off with trial success
- Boston biotech Verastem, Inc. plans to seek U.S. approval early next year for its lead blood cancer candidate duvelisib, unveiling Wednesday positive results from a Phase 3 study that the company believes could position the drug as new option to treat relapsed chronic lymphocytic leukemia (CLL).
- Successfully winning a regulatory OK would be a dramatic return on investment in duvelisib, which Verastem licensed from Infinity Pharmaceuticals, Inc. late last year for only $28 million in milestone payments.
- Treatment of CLL has evolved in recent years, moving away from chemotherapy to newer targeted therapies such as AbbVie, Inc.'s Imbruvica. Yet such drugs aren't curative and patients still relapse, creating a need for other options.
Verastem hopes to carve out a niche for itself as more and more CLL patients are treated with new targeted therapies rather than standard chemotherapy options.
These targeted drugs, from AbbVie's Imbruvica (ibrutinib) and Venclexta (venetoclax) to Gilead's Zydelig (idelalisib), have extended the treatment runway for CLL to include more lines of therapy.
As Imbruvica moves toward becoming standard of care in front-line treatment, Verastem said it expects the market to grow for secondary options to treat relapsed and refractory patients.
That's where duvelisib could come into play. In Verastem's DUO study, the PI3K inhibitor reduced the risk of disease progression or death by 48% compared to Novartis AG's Arzerra (ofatumumab) among patients with relapsed or refractory CLL as well as small lymphocytic lymphoma.
Median progression-free survival (PFS) for the duvelisib arm was 13.3 months — an improvement of 3.4 months over Arzerra's mark of 9.9 months. Duvelisib also improved median PFS for a subset of high-risk patients identified by a chromosomal abnormality known as 17p deletion.
Verastem says duvelisib has a more manageable safety profile than Venclexta, which is currently approved for only those high-risk patients. The company didn't specify any details on adverse event rates, but plans to disclose full data in an upcoming medical meeting.
The company will meet with regulators about the potential for an accelerated approval based on the DUO results as well as positive data from an earlier study in non-Hodgkin lymphoma. But primary focus will be on CLL and SLL.
"The DUO study in CLL/SLL is probably the most logical and straightforward indication to go forward with," said Verastem CEO Robert Forrester in an interview.
If duvelisib is approved, Forrester said Verastem plans to commercialize the drug in the U.S. on its own, while seeking partners for other markets. Those discussions are just getting started, but Forrester hopes to have at least one external collaboration in place by the middle of next year.
Shares in Verastem soared higher in value by more than 30% Wednesday morning to trade around $5 per share.
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