SILLAJEN announced on Tuesday that its cancer treatment candidate, BAL0891, has received rare disease drug designation for acute myeloid leukemia (AML) from the U.S. Food and Drug Administration (FDA).
BAL0891 is a novel compound that simultaneously targets TTK and PLK1, two key proteins involved in cancer cell division. The company is currently conducting a global Phase 1 clinical trial for patients with solid tumors and blood cancers.
The FDA’s rare disease drug designation offers various benefits during clinical development. SILLAJEN now qualifies for FDA research funding, tax credits for U.S. clinical trial expenses, guidance on clinical trial design, expedited review processes, and fee waivers for new drug applications.
If BAL0891 receives final approval, SILLAJEN could secure seven years of market exclusivity for the drug in the U.S. for the same indication.
AML is an aggressive blood cancer where white blood cells in the bone marrow transform into cancer cells and multiply rapidly. The disease progresses quickly, and current treatments face significant challenges, including relapse and drug resistance.
SILLAJEN is optimistic that BAL0891, with its unique mechanism of inhibiting two core proteins involved in cancer cell division, could offer new hope for AML patients who have developed resistance to existing therapies.
A spokesperson for SILLAJEN stated that receiving rare disease designation at this early stage of clinical development underscores BAL0891’s medical significance and potential. They’re committed to achieving positive results in the upcoming trials.
In 2022, SILLAJEN acquired BAL0891, a next-generation targeted cancer drug candidate, from Swiss pharmaceutical company Basilea. The company is expanding its clinical trials for solid tumors and blood cancers, focusing on BAL0891 while diversifying its pipeline with its proprietary cancer virus platform, the SJ-600 series.
Recently, SILLAJEN has been concentrating on the clinical development of BAL0891 and exploring combination therapies with immuno-oncology agents, marking a shift towards developing new drugs in the post-Pexavec era.