On February 1, 2023 Beactica Therapeutics AB, the Swedish precision oncology company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BEA-17 for the treatment of glioblastoma (GBM) (Press release, Beactica, FEB 1, 2023, View Source [SID1234626741]).
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BEA-17 is a first-in-class small molecule targeted degrader of the epigenetic enzyme LSD1 and its co-factor CoREST. Overexpression of LSD1 has been implicated in the pathogenesis of a variety of cancers, including GBM. There are currently no approved therapies for GBM that target this enzyme.
"The FDA’s decision to grant orphan drug status to BEA-17 marks an important milestone for the programme and highlights the significant need for novel therapies to treat these devastating brain tumours." said Dr Per Källblad, Co-Founder and CEO of Beactica Therapeutics. "The designation will facilitate our development of this agent which we believe has the potential to serve as a much-needed therapeutic option for patients affected by glioblastoma."
The FDA’s Orphan Drug Designation program supports the development of drugs that address rare diseases which affect fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon FDA approval.
About BEA-17
BEA-17 is a first-in-class small molecule targeted degrader (non-PROTAC) of the epigenetic enzyme LSD1 and its co-factor CoREST. The compound has shown promising preclinical in vivo potentiation of immune-modulating treatments in several cancer forms, including standard of care (temozolomide and radiation) in state-of-the-art models of GBM. Pharmacokinetic studies of BEA-17 show good blood-brain-barrier penetration and oral availability. BEA-17 is investigational and not approved anywhere globally. Its efficacy and safety in humans have not been established.
About Glioblastoma (GBM)
GBM is the most common and most aggressive brain tumour. Approximately 35,000 people in the U.S. and Europe are diagnosed with GBM each year. The median overall survival is 15 months, and the five-year overall survival is only 5%.