Vor Biopharma Inc. (VOR:NASDAQ) has announced updated results from its Phase 1/2 VBP101 study of trem-cel + Mylotarg in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), along with initial pharmacokinetic (PK) data for VCAR33ALLO and the development of a new CD45 antibody-drug conjugate (ADC). Wedbush analysts have maintained an Outperform rating on Vor Biopharma, with a 12-month price target of US$11.00, citing significant upside potential. JonesTrading also reiterated a Buy rating on Vor Biopharma with a price target of $18.00.
VBP101 Study: Promising Relapse-Free Survival Data
The updated results from the VBP101 study, evaluating trem-cel combined with Mylotarg, demonstrated encouraging relapse-free survival (RFS) data. According to a Wedbush report published on September 6, 2024, the median RFS has not yet been reached in patients at a median follow-up of 169 days (approximately 6 months). This is notable, as historical data for similar high-risk patients suggest a median RFS of three to six months. The analysts highlighted that this data suggests a potential improvement over existing treatments for AML and MDS.
VCAR33ALLO: Initial PK Results Show In Vivo Expansion
Vor Biopharma also presented initial PK results for VCAR33ALLO, an allogeneic CAR-T cell therapy. The data demonstrated in vivo expansion at the lowest dose of 1 x 106 cells/kg, with plans for dose escalation up to 1 x 107 cells/kg. This early evidence of expansion is a positive sign for the potential efficacy of VCAR33ALLO in treating hematological malignancies.
New CD45 ADC Program (VADC45)
A significant development is the introduction of a new CD45 ADC program (VADC45). Wedbush analysts view the use of VADC45 in conditioning regimens as a promising strategy with a fast path toward commercial development. They also noted limited competition in this area and considerable partnership potential. The company's strategic focus includes advancing trem-cel towards a pivotal trial, with plans to discuss this with the FDA by the end of 2024. The addition of VADC45 expands Vor Biopharma's pipeline and diversifies its therapeutic approaches.
Analyst Outlook
Wedbush analysts expressed a positive outlook on Vor Biopharma's prospects, stating, "With alignment on a pivotal study possible in the next six months and significant upside potential from CD45-based depletion approaches, we view VOR as an increasingly compelling value." Their valuation methodology is based on a sum-of-parts approach, considering the potential sales of VOR33 and VCAR33 programs. With an Outperform rating and a price target of US$11.00, representing a potential return of approximately 1,241% from the price at the time of the report, Wedbush sees substantial upside potential for Vor Biopharma.
JonesTrading analyst Justin Walsh also gave his Buy rating due to a combination of factors related to Vor Biopharma’s clinical trials and strategic developments. The promising data from Vor Biopharma’s VBP101 trial treating acute myeloid leukemia (AML) with trem-cel followed by MYLOTARG has demonstrated high engraftment and indications of clinical benefit.
Strategic Focus and Future Plans
Vor Biopharma is strategically focused on advancing trem-cel towards a pivotal trial and plans to engage with the FDA to discuss trial designs by year-end 2024. The company is also progressing with its VBP301 trial, which focuses on relapsed/refractory AML post-transplant, showing early evidence of in vivo CAR-T expansion. These developments, combined with the new VADC45 program, position Vor Biopharma as a key player in the development of novel therapies for hematological malignancies.
