Takeda, BioXcel Partner to Repurpose Compounds for Rare Diseases

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BioXcel said today it has joined with Takeda Pharmaceutical’s U.S.-based global development group to repurpose compounds into new treatments for rare diseases. The value of the partnership was not disclosed.

BioXcel said the expertise of its Big Data Innovation Lab will be tapped in its new partnership with Takeda Development Center Americas. The “innovation lab” is an integrated product-discovery engine designed to apply recursive mapping and big data algorithms toward drug discovery.

Lab components include the Integrated Center of Xcellence—a hub for medical, clinical, commercial, scientific, IP, and technology experts—and PharmGPS™ Orphan Disease Suite, a live analytics platform that encompasses more than 9,000 rare and ultra-rare diseases, 4,000-5,000 associated genes, 1,500 disease pathways, and distinct target-indication tiles for antibody, protein, RNA, small molecule, and gene therapy modalities.

BioXcel says PharmGPS enables commercialization of orphan drugs either via de novo drug discovery, or repurposing and reformulation of older compounds based on disease severity, gene ontology, disease pathways, proteinopathy, standard of care, emerging innovation, enabling technologies, and current drug pipeline.

“Discovering novel applications for existing molecules is an increasingly attractive strategy to address patient needs, while capitalizing on previous investments and de-risking clinical development. Our Big Data Innovation Lab facilitates the repurposing of the most attractive candidates applicable to a given disease,” BioXcel CEO and Chairman, Vimal Mehta, Ph.D., said in a statement.

BioXcel provides cloud-based big data solutions for biopharmas—both in discovering new products, and benchmarking pipeline innovation and marketed products.

Rare diseases do not show up among Takeda’s core therapeutic areas (cardiovascular and metabolic, CNS, immunology and respiratory, oncology, and “general medicine”).

However, the pharma giant has had a recent success in the specialty: In December, Takeda won FDA’s Breakthrough Therapy designation for its Phase III treatment oral proteasome inhibitor ixazomib (MLN9708) in relapsed or refractory systemic light-chain (AL) amyloidosis. Nearly 3,000 cases of the rare protein misfolding disorder are diagnosed in the U.S. every year. Ixazomib is also under study for multiple myeloma and other malignancies.

Takeda is the second drug developer this year to ink a partnership with BioXcel using Big Data Innovation Lab. In January, Alexion joined BioXcel to launch a big data project intended to characterize and prioritize pertinent sets of rare diseases for potential treatments.








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