Kyverna Therapeutics Releases Strong Longer-Term Phase 2 Results for Miv-cel in Generalized Myasthenia Gravis

Key Highlights

• Deep, Durable Responses Through 52 Weeks: 100% of patients treated with mivocabtagene autoleucel (miv-cel, KYV-101) in the Phase 2 portion of the KYSA-6 trial achieved rapid, robust, and sustained clinical improvements in both MG-ADL and QMG scores at 24 weeks, with responses sustained up to one year in those with extended follow-up.
• Drug-Free, Disease-Free Remission: All patients were able to discontinue chronic immunosuppressants, high-dose steroids, and other biologics, remaining drug-free through at least 24 weeks.
• Favorable Safety Profile: Miv-cel was well-tolerated. No high-grade cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. Only two cases of transient grade 3/4 neutropenia were reported, which resolved fully.
• Potential Best-in-Class Profile: The robust and durable responses, combined with the safety data, support miv-cel’s potential as a transformative, single-dose therapy for generalized myasthenia gravis (gMG) and possibly other B-cell-driven autoimmune diseases.
• Phase 3 Trial Ongoing: The Phase 3 portion of the registrational KYSA-6 trial is currently enrolling at 14 clinical sites across three geographies, reinforcing the company’s leadership in neuroimmunology CAR T-cell therapy.
• Upcoming Investor Call and Scientific Presentations: Key results and updates will be discussed in a conference call on April 22, 2026, and presented at the American Academy of Neurology (AAN) Annual Meeting.

Detailed Clinical Results

Efficacy Data

• Seven patients with moderate-to-severe gMG (mean MG-ADL: 10.6, QMG: 16.9, MGC: 21.4) were treated with a single dose of 1×10⁸ miv-cel CAR T-cells after failing prior therapies, including FcRn and complement inhibitors.
• Median follow-up was 10.2 months (range 3.3–16.0 months).
• All patients achieved clinically meaningful, rapid, and robust reductions in MG-ADL and QMG scores:
  • Mean changes at 24 weeks: MG-ADL -8.5 points, QMG -11.3 points.
  • Deep responses seen as early as two weeks post-infusion.
  • Responses were sustained out to 52 weeks in the three patients with available long-term data.
  • 100% of patients responded by achieving ≥3-point reductions in both MG-ADL and QMG.
  • 57% of patients reached minimal symptom expression (MSE, MG-ADL 0 or 1) at last follow-up.
  • All patients achieved a clinically meaningful response by MGC (mean reduction: -16 points at 24 weeks).
• Drug-free remission: All patients were free of nonsteroidal immunosuppressants, high-dose steroids (>10mg), and FcRn or complement inhibitors through 24 weeks.

Biomarker and Mechanistic Data

• Robust CAR T-cell expansion led to deep B-cell depletion and evidence of immune reset.
• Reduced pathogenic autoantibody levels with preservation of overall humoral immunity observed at week 12.

Safety Data

• No high-grade CRS or ICANS observed.
• Two transient cases of grade 3/4 neutropenia, consistent with lymphodepletion, fully resolved.
• Overall, safety profile supports the potential for outpatient administration.

Potential Market and Shareholder Impact

• Potential Paradigm Shift: Miv-cel’s single-dose, curative potential for gMG (and by extension, other B-cell-driven autoimmune diseases) positions Kyverna to disrupt the existing chronic therapy market and set a new standard of care. This could significantly expand the addressable market and drive long-term shareholder value.
• Strong Unmet Need: Up to 80,000 patients in the US have generalized myasthenia gravis, with many experiencing inadequate symptom control and requiring chronic immunosuppression. Miv-cel could address a substantial portion of this market.
• Pipeline Expansion: Kyverna is advancing miv-cel in additional indications, including stiff person syndrome (SPS), multiple sclerosis, and rheumatoid arthritis, further increasing its market potential.
• Regulatory Progress: The KYSA-6 trial is FDA-aligned and registrational, with the Phase 3 portion actively enrolling. Success in this trial could support a future BLA submission, accelerating time to market and revenue realization.
• Near-Term Catalysts: Investors should monitor the upcoming investor call and AAN presentations for additional data, which could impact sentiment and share price.

Management Commentary

CEO Warner Biddle: “Our data reinforces confidence in miv-cel’s differentiated profile, strengthens our conviction in the ongoing Phase 3 trial, and more broadly solidifies our leadership in neuroimmunology CAR T.”

KYSA-6 Lead Investigator, Prof. Srikanth Muppidi, MD: “The ability of miv-cel to achieve minimal symptom expression while eliminating the need for chronic immunotherapies following a single dose represents a meaningful clinical advancement with the potential to significantly improve daily function and quality of life for patients.”

CMO Naji Gehchan, MD: “These profound and unprecedented results are driven by miv-cel’s unique ability to target the disease at the source, deeply depleting B-cells to drive an immune reset and achieve durable drug-free, disease-free remission for patients with generalized myasthenia gravis.”

Upcoming Events

• Investor Conference Call: April 22, 2026, at 7:00 am ET (details and webcast on Kyverna’s investor relations site).
• AAN Oral Presentation: April 20, 2026 (Phase 2 update).
• AAN Poster Presentation: April 21, 2026 (Phase 3 trial design).

About Kyverna Therapeutics

Kyverna Therapeutics (Nasdaq: KYTX) is a late-stage clinical biopharma company focused on developing cell therapies for autoimmune diseases. Its lead program, miv-cel, is an autologous, fully human, CD19-targeting CAR T-cell therapy aimed at achieving durable, drug-free remission in B-cell-driven autoimmune indications.

Disclaimer

Forward-looking statements in this article are based on current expectations and subject to risks and uncertainties, including but not limited to clinical development outcomes, regulatory requirements, and market conditions. Investors should review Kyverna’s most recent filings with the SEC and consult with their financial advisors before making investment decisions. This article is for informational purposes only and does not constitute investment advice.

View Kyverna Therapeutics, Inc. Historical chart here