Business
Vor Bio Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Company Update
Initial clinical data supports founding vision that engineered hematopoietic stem cells can enable treatment options after AML transplantAdditional trem-cel
About this update from Vor Biopharma Inc.
[{"type":"text","content":"Initial clinical data supports founding vision that engineered hematopoietic stem cells can enable treatment options after AML transplantAdditional trem-cel data expected by year-end 2023; VCAR33ALLO on track for IND submission in first half of 2023$116M financing extends expected cash runway into Q1 2025 CAMBRIDGE, Mass., March 23, 2023 (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, 2022, and provided a business update. “We are encouraged with the initial proof of concept demonstrated in patients treated in our VBP101 study,” said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer. “We remain focused on rapid enrollment and plan to share additional clinical data later this year. Our IND for VCAR33ALLO is on-track for submission in the first half of 2023 which, together with trem-cel, has the potential to transform outcomes for patients with blood cancers.” Corporate Highlights Initial VBP101 clinical data represents an important milestone for Vor Bio’s founding vision and further validates the Company’s novel platform. Clinical data presented at the 2023 TANDEM meetings (Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR®) in February 2023 demonstrated sustained hematopoiesis in the first patient treated with trem-cel five months (147 days) post-transplant and Mylotarg (gemtuzumab ozogamicin) was well-tolerated through three cycles of treatment at the initial dose level of 0.5 mg/m2. Mylotarg first-dose pharmacokinetics revealed 0.5 mg/m2 achieved Cmax and AUC parameters equivalent to Mylotarg doses of 1-2 and 4-5 mg/m2, respectively, potentially due to the decreased CD33 antigen sink. CD33 deletion was observed in donor cells of myeloid and lymphoid origin which were both enriched following Mylotarg, suggesting that CD33 is expressed early in hematopoietic differentiation and that Mylotarg treatment effectively removes CD33-positive cells. Due to detectable measurable residual disease (MRD), the patient was moved to other therapies following administration of the third dose of Mylotarg and subsequently relapsed with CD33+ blasts. A second patient successfully received a trem-cel transplant and showed robust cell recovery with neutrophil engraftment occurring a...