First-in-Disease Use of Kyverna Therapeutics' KYV-101 in Patient With Severe Stiff-Person Syndrome Published in Proceedings of the National Academy of Sciences (PNAS)
Kyverna Therapeutics has announced the first use of its anti-CD19 CAR T-cell product, KYV-101, in a patient with severe stiff-person syndrome (SPS), published in the Proceedings of the National Academy of Sciences (PNAS). The 69-year-old patient saw significant improvements, including an increase in walking distance and a 40% reduction in GABAergic medications, following failure to respond to conventional therapies. The treatment was well-tolerated, showing low-grade cytokine release syndrome (CRS) and no immune effector cell-associated neurotoxicity syndrome (ICANS). These promising results support further exploration of KYV-101 for neuroimmunological diseases.
- Significant improvement in walking distance for the patient.
- 40% reduction in GABAergic medications.
- Treatment was well-tolerated with low-grade CRS and no ICANS.
- Publication in a prestigious journal (PNAS), enhancing credibility.
- Potential paradigm shift in treating debilitating autoimmune diseases.
- Positive outcomes in previous case reports for multiple sclerosis and myasthenia gravis.
- Results are based on a single patient case, requiring further studies for validation.
- Patient already had treatment-refractory SPS, indicating severe disease stage.
- Potential risks and side effects of CAR T-cell therapies in broader populations are unknown.
Insights
The publication of the first-in-disease use of KYV-101 represents a significant milestone for Kyverna Therapeutics. This innovative treatment shows potential in addressing a critical gap in autoimmune disease therapy for patients who are refractory to conventional treatments. The reported 40% reduction in GABAergic medications and the dramatic improvement in walking distance highlight the efficacy of KYV-101. The fact that the treatment was well-tolerated, with only low-grade cytokine release syndrome (CRS) and no immune effector cell-associated neurotoxicity syndrome (ICANS), is promising for its safety profile.
From an industry perspective, the lack of neurotoxicity is particularly intriguing as it suggests that KYV-101 may offer a safer alternative to existing therapies. However, the sample size is extremely limited and broad conclusions should not be drawn from a single case. Future trials must confirm these results to establish the treatment's efficacy and safety across a more comprehensive patient population.
For retail investors, understanding the potential of CAR T-cell therapies in the treatment of refractory autoimmune diseases can offer a glimpse into the future growth prospects of Kyverna. However, caution is advised due to the early stage of this research and the complexities involved in translating initial promising results into widely available treatments.
Kyverna Therapeutics' announcement regarding KYV-101 provides significant insights into the company's future growth potential. The positive results published in PNAS underline the therapeutic promise of KYV-101 and can potentially lead to an upward revision in the company's value, contingent on further successful trials. The initial successful application of KYV-101 could lead to an expanded market for Kyverna, particularly if the treatment is validated for broader use in other neuroimmunological diseases such as multiple sclerosis and myasthenia gravis.
From a financial perspective, the successful development and commercialization of KYV-101 could result in substantial revenue streams. However, investors should remain cautious as the company is still in the clinical stage. The actual financial impact will depend on the treatment advancing through clinical trials and obtaining regulatory approvals.
For retail investors, the news signals potential future gains if Kyverna can navigate the remaining clinical and regulatory hurdles. The initial results are promising, but due diligence and a clear understanding of the associated risks are crucial.
Patient received KYV-101, a fully human anti-CD19 CAR T-cell product candidate, as part of a named-patient treatment option after failure to respond to conventional therapies
Significant improvement in walking distance and
Well-tolerated treatment with low-grade CRS and no ICANS supports continued exploration of KYV-101 in neuroimmunological disease
"It is extremely encouraging to see this patient improving the self-reported, uninterrupted walking distance from less than 50 meters to several kilometers within three months after treatment," said Simon Faissner, M.D., professor for translational neuroimmunology at the Department of Neurology, Ruhr University Bochum, St. Josef Hospital, in
"It is remarkable to observe the transformational effects in a patient deemed refractory to available standard treatments. With the disease progressing over several years despite the best medical treatment, I recommended the CAR T-cell therapy approach," said Ralf Gold, M.D., professor of Medicine, chair of Neurology at Ruhr University Bochum, St. Josef Hospital, in
"On the heels of recent case reports of the use of KYV-101 in multiple sclerosis and myasthenia gravis, we are excited to see positive outcomes of KYV-101 in a patient suffering from SPS," said Peter Maag, Ph.D., chief executive officer of Kyverna. "These data underscore the dedication of the Kyverna village to patient care and scientific advancement."
About Stiff Person Syndrome (SPS)
SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms, and legs, impacting the ability to walk or move. Patients typically present with muscle spasms and stiffness, resulting in difficulty turning and bending. When stiffness is severe, the patient's walking resembles a statue. Muscle spasms and stiffness can be precipitated by unexpected stimuli, including sounds, like a phone ring or a siren, sudden touches or conditions triggering anxiety and emotional upset which, when severe, are misdiagnosed as a primary anxiety disorder2. There is no cure for SPS, but only treatments focused on the symptoms.
About KYV-101
KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health (NIH) to improve tolerability and tested in a 20-patient Phase 1 trial in oncology. Results were published by the NIH in Nature Medicine3.
KYV-101 is currently being evaluated in sponsored, open-label, Phase 1/2 and Phase 2 trials of KYV-101 in
With 50 patients treated so far with the CAR in KYV-101 in both oncological and autoimmune conditions at more than 15 locations in
KYV-101 is also being evaluated in investigator-initiated trials for multiple indications in multiple geographies.
About Kyverna Therapeutics
Kyverna Therapeutics, Inc. (Nasdaq: KYTX) is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases.
Our lead CAR T-cell therapy candidate, KYV-101 is advancing through clinical development with sponsored clinical trials across two broad areas of autoimmune disease: rheumatology and neurology, including Phase 2 trials for multiple sclerosis and myasthenia gravis, a Phase 1/2 trial for systemic sclerosis, and two ongoing multi-center, open-label Phase 1/2 trials in
Kyverna's pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats with properties intended to be well suited for use in B cell-driven autoimmune diseases.
Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements." The words, without limitation, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release include, without limitation, those related to: the potential efficacy of KYV-101 in patients with SPS, or that the results of any named patient activities may be repeated in any clinical trials; the potential impact of named-patient case reports; Kyverna's goals to develop certain paradigm-shifting treatment options; Kyverna's beliefs about the differentiated properties of KYV-101; and Kyverna's clinical trials. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties related to market conditions, and other factors discussed in the "Risk Factors" section of Kyverna's most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q that Kyverna has filed or may subsequently file with the
For more information, please visit https://kyvernatx.com.
Kyverna Media Contact:
Consort Partners for Kyverna
kyvernatx@consortpartners.com
1 Faissner S, et al. PNAS. 2024;121: e2403227121. doi.org/10.1073/pnas.2403227121
2 Dalakas, M.C., Neurotherapeutics 2022; 19, 832–847.
3 Brudno et al., Nature Medicine 2020; 26:270-280.
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