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Rafael Holdings, Inc. reports developments as a biotechnology company focused on pharmaceutical development and interests in clinical-stage and early-stage pharmaceutical and medical device companies. Its lead program is Trappsol® Cyclo™, a hydroxypropyl-beta-cyclodextrin product candidate evaluated for Niemann-Pick Disease Type C1, with additional cyclodextrin-related work in neurodegenerative disease.
Recurring company news covers clinical development of Cyclo Therapeutics programs, scientific presentations, patent and licensing activity, quarterly and annual financial results, and governance changes. Updates also address the company’s consolidated expenses following the Cyclo Therapeutics acquisition and its portfolio interests in healthcare and medical-device development.
Rafael Pharmaceuticals has initiated a research collaboration to explore the effectiveness of CPI-613® (devimistat) alongside chemotherapy agents azacitidine and venetoclax for treating acute myeloid leukemia (AML). With a current five-year survival rate for AML at just 28%, this partnership aims to address significant treatment gaps.
The company is also conducting a Phase 3 clinical trial involving devimistat and chemotherapy agents cytarabine and mitoxantrone across nearly 50 sites worldwide. Devimistat has orphan drug designation from the FDA for several cancers, enhancing its potential in ongoing trials.
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