The application was evaluated based on the
positive clinical outcomes in patients treated in Germany under the named-patient
program
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
EMERYVILLE, Calif., Aug. 12,
2024 /PRNewswire/ -- Kyverna Therapeutics, Inc.
(Kyverna), a patient-centered, clinical-stage biopharmaceutical
company focused on developing cell therapies for patients suffering
from autoimmune diseases, announced today the designation as
Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and
Drug Administration (FDA) for its autologous, fully human CD19
chimeric antigen receptor (CAR) T-cell product candidate, KYV-101,
to be used for the treatment of patients suffering from progressive
myasthenia gravis.
"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
Palo Alto, CA, and a principal
investigator in the KYSA-6 trial. "We are witnessing an era of
profound changes in the approach to autoimmune conditions and
ultimately, we hope this leads to a symptom-free state for
patients."
"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 80% of patients. Up to 20% of MG
patients experience respiratory crisis at least once in their
lives1.
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 the United States and Germany across two broad areas of autoimmune
disease: rheumatology and neurology.
With 50 patients treated so far with the CAR in KYV-101 in both
oncological and autoimmune conditions at more than 15 locations in
Europe and the U.S., we believe
that the differentiated properties of KYV-101 are critical for the
potential success of CAR T cells as autoimmune disease
therapies.
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 the United States and
Germany for patients with lupus
nephritis.
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 U.S. Securities and Exchange
Commission. Any forward-looking statements contained in this press
release are based on the current expectations of Kyverna's
management team and speak only as of the date hereof, and Kyverna
specifically disclaims any obligation to update any forward-looking
statement, whether as a result of new information, future events or
otherwise.
For more information, please
visit https://kyvernatx.com.
Investor Contact:
George Thampy
Kyverna Therapeutics
InvestorRelations@kyvernatx.com
Kyverna Media Contact:
Consort Partners for Kyverna
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.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/kyverna-therapeutics-receives-us-fda-rmat-designation-for-kyv-101-in-the-treatment-of-patients-with-progressive-myasthenia-gravis-302220288.html
SOURCE Kyverna Therapeutics