CAMBRIDGE, Mass. - Clinical-stage cell and genome engineering company Vor Bio (NASDAQ:VOR) has released new clinical data from its Phase 1/2 VBP101 study, showing positive results for patients with relapsed/refractory acute myeloid leukemia (AML). The study observed the effects of trem-cel followed by Mylotarg™, demonstrating reliable engraftment, protection from toxicity, a broadened therapeutic window for Mylotarg, and early indications of patient benefit.
The data, which included 18 patients treated with trem-cel, of whom ten received Mylotarg as of July 19, 2024, indicated a 100% success rate in primary neutrophil engraftment with a median of 9 days and robust platelet recovery within a median of 16.5 days. High CD33 editing efficiency and full myeloid chimerism by Day 28 were also reported, along with maintained neutrophil and platelet counts across multiple Mylotarg doses.
Dr. Eyal Attar, Vor Bio's Chief Medical Officer, expressed optimism about the potential of trem-cel in combination with Mylotarg for patients with AML, a condition known for poor outcomes even after transplant. The company is considering a registrational trial and is planning to discuss trial designs with the U.S. Food & Drug Administration by the end of the year.
Additionally, Vor Bio highlighted progress with VCAR33ALLO, another treatment option following trem-cel, which has shown encouraging biomarker data at the lowest dose in the ongoing VBP301 study. Vor Bio also announced a new preclinical asset, VADC45, targeting CD45, a protein associated with various blood cancers. VADC45 shows potential across multiple applications, including oncology, gene therapy, and autoimmune disorders, and is advancing towards IND-enabling studies for future Phase 1 trials.
Vor Bio aims to improve the standard of care for blood cancer patients by engineering hematopoietic stem cells for targeted therapies post-transplant. The company's approach could significantly alter treatment outcomes for these patients, though the results are subject to further clinical validation.
This report is based on a press release statement from Vor Bio.
"In other recent news, Vor Biopharma Inc. has been notified of noncompliance with Nasdaq's minimum bid price rule, as disclosed in a recent 8-K filing with the Securities and Exchange Commission (SEC). The biotechnology firm has been trading below the required $1.00 per share threshold for 30 consecutive business days, leading to a February 25, 2025 deadline for the company to elevate its share price above $1.00 for at least ten consecutive business days. If this compliance is not met, Vor Biopharma may seek an additional 180-day period by transferring to The Nasdaq Capital Market, given it meets all other initial listing standards, barring the bid price. Failure to meet the requirement or secure a second grace period could potentially result in Vor Biopharma's stock being delisted from Nasdaq. The company, however, plans to actively monitor its share price and evaluate options to regain compliance with Nasdaq's Listing Rule 5450(a)(1). These recent developments underline the challenges biotech companies face in maintaining compliance with stock exchange regulations, particularly amidst volatile market conditions."
InvestingPro Insights
Vor Bio (NASDAQ:VOR), while pushing the boundaries of medical science with its innovative therapies for blood cancers, presents a mixed financial picture according to real-time data from InvestingPro. The company's market capitalization stands at a modest $56.57 million, reflecting the high-risk, high-reward nature of biotech startups. The financial metrics underscore the challenges Vor Bio faces, with an operating income of -$124.8 million over the last twelve months as of Q2 2024, and a concerning return on assets of -60.16% in the same period. These figures are pivotal for investors to consider as they weigh the company's scientific progress against its financial health.
One of the InvestingPro Tips for Vor Bio is its higher cash reserves compared to its debt, which could provide some financial flexibility in the near term. However, analysts have revised their earnings expectations downwards for the upcoming period, signaling potential concerns about the company's profitability. In addition, the stock has significantly underperformed in recent months, with a six-month price total return of -61.42%, and a year-to-date return of -62.44% as of the date of the article, which might reflect market sentiment about the company's near-term prospects.
For investors interested in a deeper dive into Vor Bio's financials and future outlook, there are additional InvestingPro Tips available at https://www.investing.com/pro/VOR. These tips offer further insights that could be crucial for making informed investment decisions in the biotech sector.
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