Kyverna Therapeutics Receives U.S. FDA RMAT Designation for KYV-101 in the Treatment of Patients With Progressive Myasthenia Gravis
Kyverna Therapeutics (KYTX) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for KYV-101, its autologous CD19 CAR T-cell therapy for progressive myasthenia gravis. This designation was based on positive clinical outcomes from patients treated in Germany under a named-patient program. The RMAT status allows Kyverna to benefit from expedited meetings and senior FDA leadership involvement throughout KYV-101's development cycle.
Dr. Srikanth Muppidi, a principal investigator in the KYSA-6 trial, emphasized that this designation highlights the FDA's interest in potentially transformative therapies for severe autoimmune diseases. Kyverna's CEO, Peter Maag, expressed satisfaction with the company's scientific rapport with the FDA and hopes this rigorous approach will benefit patients most in need.
Kyverna Therapeutics (KYTX) ha ricevuto la designazione di Terapia Avanzata di Medicina Rigenerativa (RMAT) dalla FDA per KYV-101, la sua terapia CAR T-cell autologa CD19 per la miastenia grave progressiva. Questa designazione si basa su risultati clinici positivi ottenuti da pazienti trattati in Germania nell'ambito di un programma per pazienti nominati. Lo status RMAT consente a Kyverna di beneficiare di incontri accelerati e del coinvolgimento di alti dirigenti della FDA durante il ciclo di sviluppo di KYV-101.
Il Dr. Srikanth Muppidi, un investigatore principale dello studio KYSA-6, ha sottolineato che questa designazione mette in evidenza l'interesse della FDA per terapie potenzialmente trasformative per gravi malattie autoimmuni. Il CEO di Kyverna, Peter Maag, ha espresso soddisfazione per il rapporto scientifico dell'azienda con la FDA e spera che questo approccio rigoroso possa beneficiare maggiormente i pazienti più bisognosi.
Kyverna Therapeutics (KYTX) ha recibido la designación de Terapia Avanzada de Medicina Regenerativa (RMAT) de la FDA para KYV-101, su terapia autóloga CAR T-cell CD19 para la miastenia gravis progresiva. Esta designación se basó en resultados clínicos positivos de pacientes tratados en Alemania bajo un programa para pacientes nominados. El estatus RMAT permite a Kyverna beneficiarse de reuniones aceleradas y la participación de altos líderes de la FDA durante el ciclo de desarrollo de KYV-101.
El Dr. Srikanth Muppidi, investigador principal del ensayo KYSA-6, enfatizó que esta designación resalta el interés de la FDA en terapias potencialmente transformadoras para enfermedades autoinmunes severas. El CEO de Kyverna, Peter Maag, expresó su satisfacción con la relación científica de la empresa con la FDA y espera que este enfoque riguroso beneficie a los pacientes que más lo necesitan.
Kyverna Therapeutics (KYTX)는 FDA로부터 재생 의학 고급 치료(RMAT) 지정을 받았으며, 이는 진행성 중증 근무력증에 대한 자가 면역 CD19 CAR T-세포 치료제 KYV-101에 해당합니다. 이 지정은 독일에서 이름이 지정된 환자 프로그램에 따라 치료받은 환자들의 긍정적인 임상 결과를 기반으로 하였습니다. RMAT 상태는 Kyverna가 KYV-101의 개발 주기 동안 신속한 회의 및 FDA 고위 관리의 참여를 통해 이점을 누릴 수 있도록 합니다.
KYSA-6 시험의 주요 연구자인 Dr. Srikanth Muppidi는 이 지정이 중증 자가 면역 질환을 위한 잠재적으로 혁신적인 치료법에 대한 FDA의 관심을 강조한다고 말했습니다. Kyverna의 CEO인 Peter Maag는 FDA와의 과학적 관계에 대한 만족을 표하며, 이 엄격한 접근 방식이 가장 필요로 하는 환자에게 도움이 될 것이라고 기대하고 있습니다.
Kyverna Therapeutics (KYTX) a reçu la dénomination de thérapie avancée de médecine régénérative (RMAT) de la FDA pour KYV-101, sa thérapie par cellules T CAR CD19 autologue pour la myasthénie grave progressive. Cette désignation repose sur des résultats cliniques positifs chez des patients traités en Allemagne dans le cadre d'un programme de patients nommés. Le statut RMAT permet à Kyverna de bénéficier de réunions accélérées et d'une implication de la direction supérieure de la FDA tout au long du cycle de développement de KYV-101.
Le Dr. Srikanth Muppidi, investigateur principal de l'essai KYSA-6, a souligné que cette désignation met en avant l'intérêt de la FDA pour des thérapies potentiellement transformantes pour les maladies auto-immunes sévères. Peter Maag, PDG de Kyverna, a exprimé sa satisfaction quant aux relations scientifiques de l'entreprise avec la FDA et espère que cette approche rigoureuse bénéficiera surtout aux patients les plus dans le besoin.
Kyverna Therapeutics (KYTX) hat von der FDA die Bezeichnung für Fortgeschrittene Therapie in der Regenerativen Medizin (RMAT) für KYV-101, seine autologe CD19 CAR-T-Zelltherapie gegen progressive Myasthenia gravis, erhalten. Diese Bezeichnung basiert auf positiven klinischen Ergebnissen von Patienten, die in Deutschland im Rahmen eines benannten Patientenprogramms behandelt wurden. Der RMAT-Status ermöglicht es Kyverna, von beschleunigten Treffen und der Einbeziehung hochrangiger FDA-Führungskräfte während des Entwicklungszyklus von KYV-101 zu profitieren.
Dr. Srikanth Muppidi, ein leitender Ermittler der KYSA-6-Studie, betonte, dass diese Bezeichnung das Interesse der FDA an potenziell transformierenden Therapien für schwere Autoimmunerkrankungen unterstreicht. Peter Maag, CEO von Kyverna, äußerte Zufriedenheit mit dem wissenschaftlichen Verhältnis des Unternehmens zur FDA und hofft, dass dieser rigorose Ansatz den dringendsten Patienten zugutekommen wird.
- RMAT designation received for KYV-101, potentially expediting development and approval process
- Positive clinical outcomes observed in patients treated under named-patient program in Germany
- Increased FDA engagement and support throughout the development cycle
- None.
Insights
The FDA's RMAT designation for KYV-101 in progressive myasthenia gravis (MG) is a significant milestone for Kyverna Therapeutics. This designation, based on positive clinical outcomes from the German named-patient program, could accelerate the development and review processes. The use of CAR T-cell therapy, typically associated with cancer treatment, in autoimmune diseases represents a paradigm shift in MG management.
KYV-101, targeting CD19-positive B cells, aims to address the root cause of MG by eliminating antibody-producing cells. This approach could potentially lead to long-lasting remission, unlike current treatments that often require continuous immunosuppression. However, it's important to note that while promising, CAR T-cell therapy carries risks such as cytokine release syndrome and neurotoxicity, which will need careful monitoring in MG patients.
The RMAT designation highlights the FDA's recognition of KYV-101's potential to address unmet needs in progressive MG treatment. This could significantly impact the
Kyverna Therapeutics' RMAT designation for KYV-101 is a positive development for the company and potentially for investors in the autoimmune disease therapeutics sector. This designation could significantly accelerate the path to market for KYV-101, potentially reducing development costs and time to revenue generation.
The RMAT status provides several benefits, including:
- More frequent interactions with the FDA
- Eligibility for priority review and accelerated approval
- Increased flexibility in clinical trial design
These advantages could translate to faster market entry and potential first-mover advantage in CAR T-cell therapy for autoimmune diseases. If successful, Kyverna could capture a significant portion of the myasthenia gravis market, estimated to reach
However, investors should note that Kyverna is still a clinical-stage company and the path to commercialization involves significant risks and capital requirements. The potential reward must be weighed against these factors and the broader competitive landscape in autoimmune disease treatments.
The application was evaluated based on the positive clinical outcomes in patients treated in
The Regenerative Medicine Advanced Therapies designation allows Kyverna to leverage on more expedited meetings and more senior FDA leadership involvement throughout the development cycle for KYV-101
"The RMAT designation underscores the attention and interest by the FDA in the development of potentially transforming therapies targeting a severe autoimmune disease such as myasthenia gravis," said Srikanth Muppidi, M.D., a neuromuscular disorder specialist at Stanford Medicine in
"We are very happy with the constructive scientific rapport established between Kyverna and the FDA," said Peter Maag, Ph.D., chief executive officer at Kyverna. "We believe the RMAT designation may ultimately add to our rigorous approach to KYV-101 development in the hope of benefitting the most deserving patients."
About Myasthenia Gravis (MG)
Myasthenia gravis is an autoimmune disorder associated with muscle weakness in tissues throughout the body, potentially manifesting in partial paralysis of eye movements, problems in chewing and swallowing, respiratory problems, speech difficulties and weakness in skeletal muscles. MG patients develop antibodies that lead to an immunological attack on critical signaling proteins at the junction between nerve and muscle cells, thereby inhibiting the ability of nerves to communicate properly with muscles. The symptoms of the disease can be transient and in the early stages of the disease can remit spontaneously. However, as the disease progresses, symptom-free periods become less frequent and disease exacerbations can last for months. Disease symptoms reach their maximum levels within two to three years in approximately
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 Medicine2.
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 stiff person syndrome, 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 impact of the clinical outcomes from the named-patient activities; the potential that the results of the KYSA-6 trial could drastically change the treatment landscape for myasthenia gravis; Kyverna's goals to develop certain paradigm-shifting treatment options; the potential for KYV-101 to provide durable, immunosuppressant-free remission for myasthenia gravis patients; Kyverna's beliefs about the differentiated properties of KYV-101; and Kyverna's clinical trials and named-patient activities. 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.
Investor Contact:
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Kyverna Therapeutics
InvestorRelations@kyvernatx.com
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kyvernatx@consortpartners.com
1.Payus et al., Am J Case Rep. 2021; 22: e928419-1–e928419-4.
2 Brudno et al., Nature Medicine 2020; 26:270-280.
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FAQ
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