Kyverna Therapeutics Inc. (KYTX:NASDAQ), developer of cell therapies, disclosed that all of the three compassionate use myasthenia gravis patients treated with KYV-101 remain in a drug-free, disease-free remission at or beyond 15 months, reported Mitchell Kapoor, analyst at H.C. Wainwright & Co., in an Aug. 29 research note. On the news, H.C. Wainwright raised its target price on the cell therapy developer to $10 per share from $5 previously.
"This data strengthens our conviction in KYV-101's long-term prospects, leading us to raise our probability of approval in myasthenia gravis to 25% (from the prior 20%)," Kapoor wrote.
KYV-101 is Kyverna's investigational, autologous CD19 chimeric antigen receptor (CAR) T-cell therapy, currently being evaluated in Phase 2 of the KYSA-6 clinical trial as a treatment for myasthenia gravis.
170% Return Potential
Compared to H.C. Wainwright's new $10 per share target price, the California-based biopharma was trading at about $3.70 per share at the time of Kapoor's report, the analyst noted. From this price, the return to target is 170%.
Kyverna is still a Buy.
The company has 43.2 million shares outstanding. Its market cap is $160 million. Its 52-week range is $1.78–8.28 per share.
Remission Duration Significant
Kyverna announced the remission results for the three compassionate use myasthenia gravis patients at its recent Neuroimmunology key opinion leader (KOL) event, noted Kapoor. Results show that for Patients 1, 2 and 3 treated with KYV-101, they remain in remission at 24 months, 22 months and 15 months, respectively, reported Kapoor. What is new in Kyverna's announcement is the remission duration for Patients 2 and 3 beyond about six months.
This is the first time all three patients were in remission past the one-year target. This is a significant milestone and particularly meaningful for patients and caregivers, the KOLs at Kyverna's event said.
"Durability is now extended across multiple patients, reinforcing the credibility of KYV-101's clinical profile and the case for CAR-T usage in autoimmune disease," noted Kapoor.
The data highlight that durability is what primarily differentiates KYV-101 from FcRn inhibitors and from complement inhibitors. With these other therapies, patients remain symptomatic despite expensive, continuous treatment and background immunosuppressants. Further, the just announced durability outcomes of KYV-101 meet the threshold for commercialization, according to H.C. Wainwright, also important, especially since FcRn therapy costs $400,000–500,000 per year.
Interim results from KYSA-6, follow-up of six patients out to nine months, are expected in Q4/25. In those, H.C. Wainwright will be looking specifically at three key outcomes, the analyst pointed out. One is early trajectories, to see if they are trending toward durability of one year or longer. Another is background therapies, to see if they could be eliminated. A third is safety, to see if the profile continues to be favorable.
"Even without full 12-month data, interim results paired with compassionate use follow-up [data] should provide strong validation," Kapoor noted.
How KYV-101 Differs
Slides shown at the KOL event highlight the shortfalls of noncurative treatment modalities in terms of the disease remaining symptomatic and thus continuing to impair patients' ability to perform activities of daily living (ADLs). This is quantified by the Myasthenia Gravis ADL (MG-ADL) scale, a patient-reported questionnaire. The lower patients score on it, the better.
FcRn inhibitors efgartigimod, rozanolixizumab and nipocalimab produce mean reductions on the MG-ADL scale of about 4–5 at their primary endpoints but require repeat treatment cycles. Complement inhibitors eculizumab and ravulizumab yield 3–4-point improvements in MG-ADL but chronic dosing is required to achieve this. CD19 monoclonal antibody inebilizumab improves MG-ADL but leaves residual disease. Investigational mRNA CAR-T lowers MG-ADL by about 4 points, but patients relapse and need re-dosing.
In contrast, with KYV-101 therapy, as shown in the compassionate-use cases, MG-ADL scores were 0, background treatment could be eliminated and durability was 24, 22 and 15 months. These outcomes suggest a level of remission with KYV-101 not seen before with approved drugs or other investigational agents. KYV-101 appears to differ from all of these therapies with respect to depth of response, freedom from chronic dosing and durability.
"KOLs noted that if safety is maintained, CAR-T could move up in the myasthenia gravis treatment continuum, with applicability beyond the late-line refractory segment," reported Kapoor.
More Clinical Trials
At the KOL event, Kyverna shared the design of the upcoming KYSA-6 Phase 2/3 trial, approved by the U.S. Food and Drug Administration (FDA). The study calls for about 60 myasthenia gravis patients to be randomized on a 1:1 basis versus standard of care. Primary endpoints will be MG-ADL and Quantitative Myasthenia Gravis scores at 24 weeks. Crossover is allowed, but FcRn and anti-CD19/20 background therapies are excluded. The trial is more than 90% powered to show superiority at week 24.
"The design provides a rapid path to a biologics license application submission to the FDA, though its 24-week primary window requires clean execution, with longer-term durability captured in 18-month follow-up," noted Kapoor.
Kyverna also is assessing KYV-101 in stiff person syndrome. That trial, KYSA-8 Phase 2, is fully enrolled. Topline data are expected in H1/26.
At this point, Kapoor noted, stiff person syndrome remains a call option to H.C. Wainwright's valuation of Kyverna.
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