Rigel Pharmaceuticals and Forma Therapeutics Announce Licensing Agreement for Olutasidenib, a Novel Mutant IDH1 Inhibitor for the Potential Treatment of Relapsed or Refractory Acute Myeloid Leukemia
- Registrational Phase 2 data demonstrate olutasidenib's potential as a market-leading, oral, mutant isocitrate dehydrogenase 1 (mIDH1) inhibitor for the treatment of relapsed or refractory acute myeloid leukemia
- FDA has accepted Forma's NDA for olutasidenib, with a PDUFA target action date of
February 15, 2023
- Forma to receive an upfront payment of
$2.0 millionand is eligible to receive an additional $17.5 millionupon the achievement of certain near-term regulatory, approval, and first commercial sale milestones, as well as potential future development and commercial milestone payments of $215.5 millionand tiered royalties in the low-teens to mid-thirties
- If approved, olutasidenib would be Rigel's second commercial product in hematology-oncology and highly synergistic with Rigel's existing commercial and medical affairs infrastructure
- Rigel to host conference call today to discuss transaction details at
4:30 p.m. Eastern Timeand will be joined by Key Opinion Leader and olutasidenib Phase 2 trial investigator, Jorge E. Cortes, M.D.
In a Phase 2 registrational study of olutasidenib in patients with mIDH1 R/R AML, olutasidenib demonstrated a robust composite complete remission rate and duration of response and was well-tolerated. The
"Olutasidenib is a potential market-leading treatment that we believe, based on the registrational Phase 2 data, can improve outcomes in patients with mIDH1+ relapsed or refractory acute myeloid leukemia, and is a strategic fit for our business," said
"The compelling efficacy and safety data generated to date highlight the potential for olutasidenib to transform the treatment of mIDH1+ R/R AML. The development and approval of olutasidenib, pending a favorable FDA decision, would represent an important milestone for Forma that highlights our R&D capabilities," said
The registrational cohort of the open-label Phase 2 study evaluated olutasidenib as monotherapy in 153 mIDH1+ R/R AML patients. The primary efficacy-evaluable population of the cohort was comprised of 123 R/R AML patients, who received olutasidenib 150 mg twice daily at least six months prior to the interim analysis cutoff date of
Results from the interim analysis of the trial1 demonstrated a 33% CR+CRh in mIDH1+ R/R AML patients. Among those with CR+CRh, the estimated 18-month survival was 87% and the median duration of CR+CRh was not yet reached, with a more conservative sensitivity analysis indicating a median duration of 13.8 months. Importantly, these data provide compelling evidence of clinical efficacy with a durable response and a favorable tolerability profile, both of which we believe differentiates olutasidenib from other currently available treatment options for mIDH1+ R/R AML patients.
Olutasidenib was well-tolerated, with adverse events (AEs) being consistent with the late stage of disease and the heavily pre-treated population. A safety analysis for all 153 patients enrolled in the registrational Phase 2 study found the most common grade 3/4 (≥ 10%) treatment-emergent adverse events (TEAEs) were febrile neutropenia (20%), anemia (19%), thrombocytopenia (16%), and neutropenia (13%).
Updated data from the registrational study will be presented at an upcoming medical congress.
"The data from the Phase 2 registrational trial of olutasidenib demonstrated encouraging results, particularly on durability and survival, with median duration of response that appears to be longer than currently available treatment options and an 18-month survival rate among those with CR+CRh of 87%," said
Under the terms of the agreement, Forma will receive an upfront payment of
Conference Call and Webcast Today at
Rigel will host a live conference call and webcast today at
Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company's website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.
About Olutasidenib and AML
Olutasidenib is an oral, small molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes. This targeted treatment has the potential to provide therapeutic benefit by reducing 2-HG levels and restoring normal cellular differentiation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells. When mutated, IDH1 activity can promote blood malignancies and solid tumors. IDH1 mutations are present in 6 to 9 percent of patients with AML2. AML is a rapidly progressing cancer of the bone marrow and blood3. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that about 20,940 new cases, most in adults, arose in 2021 in the United States alone.4 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.
Fostamatinib is currently being studied in a Phase 3 clinical trial (NCT03764618) for the treatment of warm autoimmune hemolytic anemia (wAIHA)5; a Phase 3 clinical trial (NCT04629703) for the treatment of hospitalized high-risk patients with COVID-195 and an
Rigel's other clinical programs include its interleukin receptor-associated kinase (IRAK) inhibitor program, and a receptor-interacting serine/threonine-protein kinase (RIPK) inhibitor program in clinical development with partner Eli Lilly and Company. In addition, Rigel has product candidates in development with partners BerGenBio ASA and Daiichi Sankyo.
Please see www.TAVALISSE.com for full Prescribing Information.
- De Botton, S., et al.
Journal of Clinical Oncology39, no. 15_suppl ( May 20, 2021) 7006-7006.
- NCCN Clinical Practice Guidelines in Oncology, Acute Myeloid Leukemia. Version 2.2022 –
June 14, 2022. https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1411 Leukemia & Lymphoma Society. Accessed July 25, 2022. https://www.lls.org/leukemia/acute-myeloid-leukemia
American Cancer Society. Key statistics for acute myeloid leukemia (AML). Revised January 12, 2021. Accessed Dec. 2, 2021 at https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html.
- The product for this use or indication is investigational and has not been proven safe or effective by any regulatory authority.
Rigel Forward Looking Statements
This press release contains forward-looking statements relating to, among other things, that olutasidenib may provide a meaningful benefit to people with relapsed or/ refractory acute myeloid leukemia, our ability to commercialize olutasidenib in the
Forma Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding the company's beliefs and expectations regarding: therapeutic potential, clinical benefits, mechanisms of action, efficacy, and safety of olutasidenib; the potential commercial and collaboration opportunities, including potential future collaborators, as well as the potential value and market for olutasidenib; potential milestone payments; and presentation of additional data at upcoming scientific conferences. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties associated with the following: positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; adverse regulatory decisions relating to olutasidenib; Rigel's ability to successfully develop and commercialize olutasidenib and achieve milestones, including identifying successful collaboration opportunities as well as those risks and uncertainties set forth more fully under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended
View original content to download multimedia:https://www.prnewswire.com/news-releases/rigel-pharmaceuticals-and-forma-therapeutics-announce-licensing-agreement-for-olutasidenib-a-novel-mutant-idh1-inhibitor-for-the-potential-treatment-of-relapsed-or-refractory-acute-myeloid-leukemia-301598297.html