Press Release: Positive Phase 1/2 study results of rilzabrutinib in
people with immune thrombocytopenia published in The New England
Journal of Medicine
Positive Phase 1/2 study results of
rilzabrutinib in people with immune thrombocytopenia published in
The New England Journal of Medicine
- Results published today in the New
England Journal of Medicine showed that treatment with
rilzabrutinib resulted in a rapid and durable increase in platelet
count in patients with heavily pretreated immune thrombocytopenia
(ITP)
- Data support an acceptable safety
profile
- Rilzabrutinib is an investigational
oral Bruton tyrosine kinase inhibitor (BTKi) for the treatment of
ITP, a rare acquired autoimmune disorder in which platelets are
destroyed or damaged and for which there are limited treatment
options
PARIS –
April 14, 2022
- Positive results from the Phase 1/2 dose-finding
study evaluating the safety, pharmacokinetics and clinical activity
of rilzabrutinib, an investigational oral Bruton’s tyrosine kinase
(BTK) inhibitor, in adults with heavily pre-treated immune
thrombocytopenia (ITP) were published in the New England Journal of
Medicine. Results demonstrate treatment with rilzabrutinib led to a
rapid and durable increase in platelet count and support an
acceptable safety profile. Sanofi is investigating the safety and
efficacy of twice daily rilzabrutinib (400 mg) for adults and
adolescents with chronic ITP in the ongoing Phase 3 clinical study
LUNA 3, initiated in April 2021.
David
Kuter, M.D.Director of clinical
hematology at Massachusetts General Hospital and professor of
medicine at Harvard Medical School, lead author of the study
“Currently, there are no standard treatment
recommendations for ITP patients with multiple relapses. Despite
advances in treatment options over the years, some patients remain
refractory to existing therapies and durable remission remains
elusive. The Bruton’s tyrosine kinase is a critical signaling
molecule in the immune system that is involved in certain
immune-mediated diseases, and our research suggests that targeting
BTK may represent a promising approach to addressing the underlying
cause of ITP.”
ITP is an acquired autoimmune blood disorder
characterized by low platelet count (thrombocytopenia) resulting
from immune-mediated platelet destruction and impairment of
platelet production. A decrease in platelet counts – whether
temporary or persistent – can predispose a person to a higher risk
of bleeding, hospitalization, fatigue, impaired quality of life,
and even death. The incidence of ITP increases with age and is more
common over the age of 60.
Dietmar
Berger, M.D.,
Ph.D.Global Head of Clinical Development and Chief Medical
Officer, Sanofi
“We are pleased to share these encouraging early
clinical results through this publication. These findings
demonstrate a clinically meaningful response in difficult-to-treat
ITP patients who received a median of four prior ITP
therapies. Moreover, the overall study population, which also
included less refractory patients, showed a numerically higher
response. Rilzabrutinib could become a first-in-class BTK inhibitor
therapy with the potential to increase platelet counts quickly and
durably for people with ITP.”
Rilzabrutinib was granted Fast Track Designation
by the U.S. Food and Drug Administration (FDA) for treatment of ITP
in November of 2020 and was previously granted orphan drug
designation. Rilzabrutinib is being investigated in multiple
clinical trials across a range of diseases including immunological
and inflammatory diseases.
Phase 1/2 Study
Results
The global Phase 1/2 adaptive, open-label,
dose-finding study evaluated rilzabrutinib in 60 people with ITP
with a median age of 50 years (range, 19-74). Patients had received
a median of four different ITP therapies previously. Initial doses
could be 200 mg once daily, 400 mg once daily, 300 mg twice daily
(600 mg/day), or 400 mg twice daily (800 mg/day). The median
platelet counts at the start of the study were 15×109/L, indicating
a very low platelet count and high risk of bleeding. The primary
endpoint measured the number of participants who achieved at least
two consecutive platelet counts of ≥50×109/L and an overall
platelet count increase of ≥20×109/L from the start of treatment
without requiring rescue medication.
Study results showed:
- Overall, 24 of 60 people enrolled
in the study at any dose achieved the primary endpoint. Of the 45
people who initiated rilzabrutinib at 400 mg twice daily, 18 met
the primary endpoint.
- Median time to first platelet count
of at least 50×109/L was rapid at 11.5 days, which was maintained
in patients with primary platelet response for a mean of 65% of
weeks during the 24-week treatment period.
- 52% of participants experienced at
least one treatment related adverse event, all of which were grade
1 or 2; the most common adverse events were diarrhea (32%), nausea
(30%), and fatigue (10%).
- There were no grade 3 or higher
treatment-related adverse events or serious adverse events.
Rilzabrutinib Clinical
ProgramThe safety and efficacy of rilzabrutinib in ITP are
being evaluated in the ongoing randomized, double-blind, Phase 3
LUNA 3 study in adults and adolescents (aged ≥12 years) with
persistent/chronic ITP. In addition, phase 2 studies are ongoing to
evaluate rilzabrutinib as a potential therapy for the autoimmune
condition IgG4 disease and immunological diseases, including
asthma, atopic dermatitis, chronic spontaneous urticaria and warm
autoimmune hemolytic anemia.
About
RilzabrutinibRilzabrutinib is an oral
Bruton’s tyrosine kinase inhibitor incorporating Sanofi’s TAILORED
COVALENCY® technology being investigated for the treatment of
immune-mediated diseases, including ITP. BTK is an intracellular
signaling molecule involved in innate and adaptive immune responses
related to certain immune-mediated diseases. By inhibiting BTK,
rilzabrutinib has the potential to target the underlying disease
pathogenesis.
Rilzabrutinib is currently under clinical
investigation and its safety and efficacy have not been evaluated
by any regulatory authority.
About SanofiWe are an innovative global
healthcare company, driven by one purpose: we chase the miracles of
science to improve people’s lives. Our team, across some 100
countries, is dedicated to transforming the practice of medicine by
working to turn the impossible into the possible. We provide
potentially life-changing treatment options and life-saving vaccine
protection to millions of people globally, while putting
sustainability and social responsibility at the center of our
ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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