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Preliminary results from the ongoing FIGHT-203 study of Pemazyre (pemigatinib) in patients with FGFR1-mutant myeloid and lymphoid neoplasms (MLN) suggest that pemigatinib may offer utility as a bridge for allogeneic hematopoietic stem cell transplant (HSCT), or as a long-term treatment option for patients who are ineligible for transplant. FGFR1-mutant MLN may present with chronic or blast phase (BP) involvement of bone marrow.

About FGFR and Pemazyre (pemigatinib)

Fibroblast growth factor receptors (FGFRs) play an important role in cancer cell growth and survival by enhancing migration and the formation of new blood vessels known as angiogenesis. A variety of genetic mutations in FGFRs are closely correlated with the development of various cancers. Pemigatininb binds to and inhibits FGFRs which prevents the growth and proliferation of FGFR over-expressing cancer cells

FIGHT-203 is a phase 2, multi-center trial with a total of 34 patients enrolled with FGFR1-mutant neoplasms. Among the 31 patients with BM and/or extra-medullary disease complete remission rates were 64%.

Pemigatinib is the first therapy to demonstrate durable and high rates of remission in patients with FGFR1-mutant MLN, most of whom had progressed on prior therapies including intensive chemotherapy or HSCT.

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Reference

Gotlib J, Kiladjian J, Vannucchi A, et al. A Phase 2 Study of Pemigatinib (FIGHT-203; INCB054828) in Patients with Myeloid/Lymphoid Neoplasms (MLNs) with Fibroblast Growth Factor Receptor 1 (FGFR1) Rearrangement (MLNFGFR1). https://ash.confex.com/ash/2021/webprogram/Paper148103.html. Presented at: the 63rd ASH Annual Meeting & Exposition; December 11-14, 2021; Atlanta, GA. Abstract 385.).