Vor Bio(VOR.US) Gene-edited Stem Cell Therapy Advances, Shares Surge Over 70% After-Hours

Since its listing, Vor Bio(VOR.US) has seen its stock price plunge. However, the company seems to have turned the corner with its stock price surging more than 70% in after-hours trading on Thursday after reporting positive data from its gene-edited stem cell therapy trem-cel in treating acute myeloid leukemia (AML). The gene-edited stem cell therapy trem-cel showed positive results in a phase 1/2 trial when combined with Mylotarg in treating AML, demonstrating potential for combined efficacy and drug safety.

It is understood that Vor Bio's phase 1/2 study, named VBP101, is accelerating the evaluation of trem-cel in combination with Mylotarg in treating relapsed/refractory AML patients.

Vor(VOR) said in a statement that the study data "demonstrated reliable engraftment, abrogated Mylotarg's targeted toxicity, and significantly expanded the therapeutic window for Mylotarg, as well as early evidence of broad patient benefit."

The company added that it plans to discuss the core pivotal trial design for trem-cel plus Mylotarg with the U.S. Food and Drug Administration (FDA) before the end of the year.

Core pivotal trials are usually the last stage for approval of new drugs, meaning that once these trials are successful, trem-cel could quickly enter the market.

Vor also announced that it is conducting a study that will bring another candidate therapy (an antibody-drug conjugate named VADC45) into a phase 1 trial. This diversified product pipeline progress demonstrates the company's research strength and long-term development potential in the cancer field.

The latest positive trial results from Vor Bio's combination with Pfizer's Mylotarg suggest trem-cel may have significant potential in future AML treatments, which has boosted investor confidence in the company.

Gene-editing therapies are considered one of the major breakthrough therapies in the future cancer treatment field. Any slight clinical success indication of Vor Bio, a development-stage company focused on developing innovative gene-edited stem cell therapies, would quickly attract the attention of investors and some retail speculators. Based on this, some investors began to expect a positive outlook for the trem-cel and Mylotarg combination, which was one of the reasons driving Vor Bio's stock price to surge in after-hours trading.