A $230M Deal for New Leukemia Drug Awaits FDA

Rigel Pharmaceuticals and Forma Therapeutics announced an exclusive, worldwide license agreement on 2 Aug which covered the development, manufacture, as well as commercialization of olutasidenib, a novel oral drug for acute myeloid leukemia (AML) that currently awaits FDA approval.

Related article: Ascentage Pharma Announces Clinical Trial Application for Olverembatinib (HQP1351) Approved in Canada

The Potential New Drug

Olutasidenib is an oral, small molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes. This targeted therapy has the potential to reduce 2-HG levels thus restoring normal cellular differentiation. IDH1 is an essential enzyme for the normal metabolism of all cells, while mutated IDH1 is known to promote blood malignancies and the formation of solid tumors. 

In the Phase 2 registrational study of olutasidenib in relapsed or refractory acute myeloid leukemia patients with IDH1 mutation, olutasidenib demonstrated a robust composite complete remission rate and duration of response and was well-tolerated. 

The primary efficacy-evaluable population of the cohort includes 123 relapsed or refractory acute myeloid leukemia patients with IDH1 mutation. 150 mg olutasidenib twice daily were prescribed at least six months prior to the interim analysis cutoff date. The primary endpoint was a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh), with the definition of less than 5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts. 

Results from the interim analysis of the trial 1 demonstrated a 33% CR+CRh in the corresponding cohort. Among those with CR+CRh, the estimated 18-month survival was 87% and the median duration of CR+CRh was not yet reached. More importantly, the adverse events (AEs) are consistent with the late stage of the disease and the heavily pre-treated population. 

A Win-win Situation

Under the terms of the agreement, Forma will receive an upfront payment of $2 million, with an additional $17.5 million upon the achievement of certain near-term regulatory, approval, and first commercial sale milestones, plus a total of up to $215.5 million for future development and commercial milestone and tiered royalties in the low-teens to mid-thirties. Moving forward, Rigel will take over the potential launch and commercialization of olutasidenib in the US, as well as potential collaboration to further develop and commercialize olutasidenib outside the US.

Olutasidenib will not only offer an alternative therapy for those who remain underserved with currently available therapies but will also potentially increase Rigel’s hematology-oncology portfolio. Rigel is looking forward to Olutasidenib becoming its second commercial product after Tavalisse, which aims for adults with low platelet counts due to chronic immune thrombocytopenia.

As for Forma, Rigel’s experience in hematologic diseases and cancers and their powerful commercial infrastructure would definitely push olutasidenib to another level.

Nevertheless, all the subsequent funding would only come true once the drug gets approval. The US Food and Drug Administration (FDA) has accepted Forma’s New Drug Application (NDA) for olutasidenib, and the result will be released by 15 Feb, 2023.

Author

Nai Ye Yeat

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