Kyverna Therapeutics completes miv‑cel BLA for stiff person syndrome
The 26-patient trial supports a priority review request, but the Nov. 12 release gives no FDA acceptance date or decision deadline.
Adrian Kessler
A U.S. approval request for miv-cel in stiff person syndrome is fully submitted after Kyverna Therapeutics, Inc. NASDAQKYTX reported on Thursday that it had completed its rolling biologics licence application. The company is seeking priority review ahead of a potential commercial launch in 2027.
Kyverna's Nov. 12 announcement identifies the therapy as mivocabtagene autoleucel, formerly KYV-101, and bases the application on its KYSA-8 trial. Completion of submission is distinct from FDA acceptance for review or approval, and the release gives no acceptance date or decision deadline.
Kyverna is a small, late-stage immunology developer based in Emeryville, Calif., focused on cell therapies for neurologic autoimmune diseases. Its lead treatment uses a patient's own genetically modified T cells to target CD19-bearing B cells.
Shares closed at US$5.12 on Nov. 11 on Nasdaq NASDAQKYTX, according to Google Finance's 4:00 p.m. EST timestamp. That close preceded Thursday's announcement and provides no measure of the market's response to the completed submission.
Walking Improvements Persisted Through One Year
The supporting Phase 2 trial enrolled 26 adults whose stiff person syndrome had responded inadequately to at least one immunotherapy. Each received a single dose of miv-cel in a single-arm study, with no placebo or active-treatment control.
The primary endpoints were change from baseline in the timed 25-foot walk at 16 weeks and the incidence and severity of adverse events. Kyverna reported a median walking-time improvement of 46 per cent at that assessment.
At one year, the median improvement was 49 per cent, with p below 0.0001, according to the Oct. 21 report. Twenty of the 26 patients achieved the trial's threshold for clinically meaningful walking improvement at that follow-up.
The comparison was with patients' own pretreatment performance, limiting conclusions about benefit relative to another treatment. Twenty-four patients remained free of chronic immunotherapies for stiff person syndrome at one year.
No high-grade cytokine release syndrome or immune effector cell-associated neurotoxicity was reported; five patients had grade 3 or 4 neutropenia and three had serious treatment-related adverse events, which all resolved.
Kyverna describes KYSA-8 as registrational and says miv-cel holds the FDA's Regenerative Medicine Advanced Therapy designation. Thursday's release provides no new FDA assessment of the trial design, and the request for priority review does not establish a grant of priority review.
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Runway Includes Available Loan Funding
Kyverna reported US$170.6 million in cash, cash equivalents and marketable securities at Sept. 30. Its forecast of operating runway into 2028 also includes amounts available to draw under an amended loan facility with privately held Oxford Finance, rather than cash already on hand alone.
The company is recruiting field sales and medical specialists while working with treatment centres, neurologists and payers on launch preparations. Those activities anticipate a possible approval; commercial availability remains conditional on the FDA's decision.
"We believe miv-cel's clinical profile, differentiated construct design and established manufacturing process can support a strong launch in SPS, if approved," Kyverna chief executive Warner Biddle said in Thursday's release.
Kyverna says there are no FDA-approved therapies for stiff person syndrome and describes miv-cel as a potential first approved CAR T-cell therapy for autoimmune disease.
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Kyverna expects to share its progressive multiple sclerosis development strategy by early 2027. That update is set to outline the company's development plans for another miv-cel indication.
