Beactica Secures €2.5M for Orphan Drug

Swedish precision medicine company Beactica Therapeutics, along with researchers at KU Leuven, has been awarded a €2.5 million grant from the European Innovation Council. The funding is intended to advance the company's orphan drug candidate, BEA-17, toward clinical readiness for glioblastoma. The grant reflects continued European investment in novel therapeutics for rare diseases.

- BEA-17 is a first-in-class small molecule that acts as a targeted degrader of the epigenetic enzyme Lysine-specific demethylase 1 (LSD1/KDM1A) and its partner protein CoREST. This mechanism is distinct from other LSD1 inhibitors in clinical trials that only target the enzyme's catalytic site. Preclinical studies show BEA-17 potentiates the efficacy of standard-of-care for glioblastoma (radiation and temozolomide) and has favorable oral availability and blood-brain barrier penetration. - The U.S. FDA granted BEA-17 Orphan Drug Designation for glioblastoma in February 2023. This status is for drugs treating rare diseases affecting fewer than 200,000 people in the U.S. and provides incentives like tax credits, user fee exemptions, and seven years of market exclusivity upon approval. - The current standard of care for glioblastoma involves maximal surgical resection followed by radiation and the chemotherapy drug temozolomide. Despite this aggressive regimen, the prognosis is poor, with a median survival of less than 15 months and a five-year survival rate of only around 6.9%. - The grant is from the EIC Transition funding program, which is designed to mature and validate novel technologies developed in previous EU-funded projects, moving them from lab-based proof-of-concept (TRL 3/4) toward readiness for market application (TRL 5/6). The funding supports activities like demonstrating the technology in relevant environments and developing a business plan. - The collaboration partner, KU Leuven's Centre for Drug Design and Discovery (CD3), is a drug discovery center and investment fund established to translate academic research into new medicines. It partners with universities and biotechs to advance drug discovery projects to a stage where they are attractive for licensing or spin-off creation. - The target, LSD1 (KDM1A), is an epigenetic enzyme that is frequently overexpressed in various cancers, including glioblastoma, where it contributes to tumor growth and the maintenance of glioma stem cells. Inhibiting LSD1 can reduce the "stemness" of these cancer cells, inducing differentiation and apoptosis. - Beactica Therapeutics is a private Swedish precision oncology company utilizing its Eclipsor™ platform to develop a pipeline focused on targeted protein degraders and allosteric modulators for genetically defined cancers. Beyond BEA-17, the company has a program developing TEAD degraders targeting the Hippo signaling pathway. - Preclinical studies have suggested that inhibiting KDM1A can make glioblastoma more sensitive to temozolomide by weakening DNA double-strand break repair pathways. Pharmacokinetic studies have also shown that KDM1A inhibitors can successfully cross the blood-brain barrier, a critical challenge in developing drugs for brain tumors.

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