Business
Vor Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Company Update
Trem-cel and VCAR33ALLO clinical trial data expected in the second half of 2024VCAR33ALLO granted Fast Track and Orphan Drug Designation by U.S. Food & Drug
About this update from Vor Biopharma Inc.
[{"type":"text","content":"Trem-cel and VCAR33ALLO clinical trial data expected in the second half of 2024VCAR33ALLO granted Fast Track and Orphan Drug Designation by U.S. Food & Drug Administration Cash runway extends into second half of 2025 CAMBRIDGE, Mass., March 20, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2023, and provided a business update. “We are very pleased with the foundational progress we made in 2023. Building on this, we expect 2024 to be an exciting year in which we demonstrate the Vor Bio approach to be instrumental in reducing the disease burden of AML, a devastating cancer where we desperately need effective treatment options,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. Corporate Updates Multiple patients expected to be dosed with VCAR33ALLO in the first half of 2024; initial data anticipated in second half of 2024 VBP301, a Phase 1/2, multicenter, open-label, first-in-human study of VCAR33ALLO, is a transplant donor-derived anti-CD33 CAR-T cell therapy for patients with AML who have relapsed following a standard-of-care or trem-cel transplant. The first patient was dosed in January 2024, and multiple patients are expected to be dosed in the first half of 2024. We anticipate initial data in the second half of 2024.The U.S. Food & Drug Administration (FDA) has granted Fast Track Designation and Orphan Drug Designation to VCAR33ALLO. The FDA Fast Track process aims to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. Orphan Drug Designation entitles companies to development incentives including tax credits for clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity in the event of regulatory approval. VCAR33ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell therapy that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a pat...