Vor Biopharma, a cancer cell therapy developer that laid off nearly all of its employees in a wind down of operations last month, has reemerged with new management, an in-licensed lead drug candidate, and $175 million in financing to support its new focus on autoimmune diseases.
Cambridge, Massachusetts-based Vor announced late Wednesday an agreement for exclusive rights to telitacicept, a drug that RemeGen has already steered to regulatory approvals in China for generalized myasthenia gravis (gMG), systemic lupus erythematosus, and rheumatoid arthritis. China-based RemeGen retains rights to the drug in its home country. This drug is currently in a global Phase 3 study in gMG that could support an FDA submission.
In gMG, autoantibodies attack proteins important for the communication between nerves and muscles. The rare disorder leads to problems such as difficulty swallowing and muscle weakness. Progression of gMG is mediated by a type of immune cell called a B cell. Vor’s new drug is a fusion protein designed to selectively inhibit B cell activating factor, or BAFF (also known as BlyS), and APRIL, two signaling proteins key to B cell survival.
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Vor is not the only company going after one or both of those targets, but its focus on gMG could help it stand apart. Novartis’s zigakibart, a monoclonal antibody that blocks APRIL, is in Phase 3 testing for immunoglobulin A nephropathy (IgAN). Vera Therapeutics blocks BAFF and APRIL with a fusion protein called atacicept. In early June, Vera announced this drug met its Phase 3 goals in IgAN; an FDA submission is planned for the fourth quarter of this year. Vertex Pharmaceuticals is going after BAFF and APRIL with povetacicept, a fusion protein that was the centerpiece of its $4.9 billion acquisition of Alpine Immune Sciences last year. Povetacicept has reached Phase 3 testing in IgAN and a Phase 2/3 clinical trial in primary membranous nephropathy.
Treatment of gMG has welcomed new therapies in recent years, but none yet that address BAFF and APRIL. The therapies available include drugs that block complement proteins associated with the disorder (AstraZeneca’s Ultomiris and UCB’s Zilbrysq) and FcRn inhibitors (Argenx’s Vyvgart and Vyvgart Hytrulo; UCB’s Rystiggo). The newest FcRn inhibitor for gMG is Johnson & Johnson’s Imaavy, which landed its FDA approval in May. Vor said the Phase 3 study for telitacicept is enrolling patients in the U.S., Europe, and South America; preliminary results are expected in the first half of 2027.
Vor Biopharma, co-founded by oncologist Siddhartha Mukherjee and startup creator PureTech Health, was developing stem cell-derived cell therapies for blood cancers that could offer alternatives to currently available CAR T-therapies for cancer that are made by harvesting and engineering a patient’s own T cells. The biotech went public in 2021, raising nearly $177 million. Despite making progress with clinical-stage programs, Vor was running low on cash. In May, the company ceased its clinical and manufacturing operations and laid off about 95% of its staff as it explored strategic alternatives for the business.
Vor’s CEO, Robert Ang, resigned Thursday, the company said. The board of directors has appointed Jean-Paul Kress to serve as the new chief executive. Kress is the former CEO of MorphoSys, which was acquired by Novartis last year. He is also the former chief executive of Syntimmune, which was acquired by Alexion Pharmaceuticals (now AstraZeneca’s rare diseases subsidiary).
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“Targeting BAFF/APRIL signaling with telitacicept represents a significant advancement in addressing autoantibody driven diseases, which is highly differentiated from other modalities in this space,” Kress said in a prepared statement. “With a clinically advanced asset, we are uniquely positioned to develop this innovative therapy, with the goal of making a meaningful impact for patients living with autoimmune diseases around the world.”
In its first quarter 2025 financial report, Vor reported its cash position was about $60 million. The company has shored up its finances with a $175 million private placement announced after the telitacicept licensing deal. Participants in the new financing include RA Capital Management, an existing Vor stockholder. The other disclosed investors are Mingxin Capital, Forbion, Venrock Healthcare Capital Partners, Caligan Partners, and NEXTBio. Vor said it would use the proceeds to advance its pipeline and for general corporate purposes.
According to Vor’s agreement with RemeGen, Vor will pay the biotech $125 million, consisting of a $45 million upfront payment along with warrants to purchase about $80 million worth of Vor stock. In a regulatory filing, Vor said RemeGen could receive up to $330 million in regulatory milestone payments and up to $3.7 billion tied to sales milestones. RemeGen would also receive royalties from sales, if the drug reaches the market.
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