En route to its first human trial, Vor Biopharma raised $177 million in its Nasdaq debut. The IPO comes seven months after a $110 million venture round and will bankroll the development of its lead program, a cell therapy for acute myeloid leukemia (AML), as well as its earlier-stage pipeline.
The company tagged about $40 million of the proceeds for VOR33, an engineered stem cell treatment it’s developing for blood cancers, according to a securities filing. The capital will bankroll the phase 1/2a clinical trial for VOR33 in AML—which is set to begin in the first half of the year—and preclinical development of VOR33 for the treatment of two groups of rare blood cancers called myelodysplastic syndromes and myeloproliferative neoplasms.
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VOR33 is made from hematopoietic, or blood-forming, stem cells from healthy donors. The company uses gene editing to get rid of cancer drug targets in those cells that are “biologically redundant,” which means deleting them doesn’t cause any harm. In the case of VOR33, that target is CD33.
CAR-T and other immunotherapies that target the cell surface generally home in on healthy blood cells as well as cancer cells, causing nasty side effects that limit their use. The idea behind treatments like VOR33 is to give patients stem cells that produce healthy blood cells that are protected from cancer treatments. Vor believes its treatments could boost the reach of targeted therapies by improving their efficacy and increasing the amount of time patients can undergo those treatments.
In its phase 1/2a study, Vor will test VOR33 in patients with CD33-positive AML who are at high risk of relapsing. After receiving VOR33, patients will be eligible to receive Pfizer’s Mylotarg, a CD33-targeting antibody-drug conjugate.
In the future, Vor hopes to offer VOR33 alongside VCAR33, its anti-CD33 CAR-T therapy. Of the IPO proceeds, it will use about $50 million to get VCAR33 into a phase 1/2 study in adults with AML. Any funds left over will help the company expand its platform and pipeline, the company said in the filing.
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Vor Biopharma started out in 2016 when it licensed a CAR-T platform from oncologist and Pulitzer Prize winner Siddhartha Mukherjee, M.D., Ph.D. It raised a $42 million series A in early 2019, followed by a $110 million B round in July 2020. Along the way, Robert Ang, formerly Neon Therapeutics’ chief business officer, joined as CEO.