Curis takes aim at late-stage trials as FDA lifts final hold on leukemia, lymphoma med

The FDA has freed Curis from a remaining partial clinical hold, lifting the pause on a phase 1/2 leukemia study and giving the green light to launch using the recommended phase 2 dose.

In April 2022, the FDA placed a partial clinical hold on the trial, dubbed TakeAim Leukemia, evaluating emavusertib in patients with recurring or non-responsive acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), after a patient death occurred. A week later, the agency slapped a separate partial hold on the biotech’s TakeAim Lymphoma study examining the same drug in patients with relapsed or refractory B-cell malignancies.

The FDA implemented the partial holds so it could investigate the safety and efficacy of the IRAK4 kinase inhibitor following an information request about the death of an AML patient who experienced rhabdomyolysis and other conditions. Rhabdomyolysis—the breakdown of skeletal muscle—is a known dose-limiting toxicity of emavusertib.

The holds have remained in place since then, but in August the small molecule biotech received good news. The FDA lifted the partial hold on the lymphoma trial and allowed Curis to resume enrollment for the monotherapy dose-finding phase of its leukemia study.

Now, the partial hold has been lifted for the combo therapy portion of TakeAim Leukemia, in which Curis is evaluating doses of emavusertib with azacitidine or the AbbVie-Genentech drug Venclexta. The trial’s expansion portion—a phase 2a—was also included in the hold and subsequently is now free.

With the FDA's blessing, the Massachusetts-based biotech is now moving forward with the recommended phase 2 dose for emavusertib as a monotherapy: 300 mg twice daily in patients with AML or MDS. Curis is now working to enroll patients with a specific form of AML that have already received two or more treatments, said James Dentzer, president and CEO of Curis.

Future plans include a front-line combination study of emavusertib with azacitidine and venetoclax, Dentzer said in the July 6 release. Updated data from the TakeAim Leukemia monotherapy study, clarification of a monotherapy registrational study design, and initial data from an azacitidine and Venclexta combination study is expected in 2024. 

On the same day the FDA lift was announced, Curis also announced the sale of 18.4 million shares of common stock for 82 cents apiece in a registered direct offering. The shares were purchased by existing investors and gross proceeds are expected to be $15.1 million. Curis intends to funnel the money into R&D, working capital and other general corporate purposes.

As of Dec. 31, 2022, the biotech had $71.8 million in cash, cash equivalents and investments, according to Securities and Exchange Commission filings from this May. At the time, Curis expected the balance to stretch into 2025.