Rilzabrutinib LUNA 3 phase 3 study met primary
endpoint in immune thrombocytopenia
- Pivotal data from the first phase 3
study of a BTKi in immune thrombocytopenia (ITP) underscore the
potential of rilzabrutinib to provide a clinically meaningful
benefit to patients living with ITP
- Regulatory submissions in the US and
EU anticipated by year-end
- Rilzabrutinib is one of 12 potential
medicines and vaccines in Sanofi’s robust immunology pipeline and a
testament to Sanofi’s ability to successfully accelerate and build
a portfolio of next-generation transformative treatments for immune
diseases
- In addition to ITP, rilzabrutinib is
being studied across a variety of immune-mediated diseases
including asthma, chronic spontaneous urticaria, prurigo nodularis,
IgG4-related disease and warm autoimmune hemolytic anemia
Paris, April 23, 2024. Positive
results from the LUNA 3 phase 3 study demonstrated that
rilzabrutinib 400 mg twice daily orally achieved the primary
endpoint of durable platelet response in adult patients with
persistent or chronic immune thrombocytopenia (ITP). The safety
profile of rilzabrutinib was consistent with that reported in
previous studies.LUNA 3 study met its primary endpoint
demonstrating a significantly higher proportion of patients
receiving rilzabrutinib achieved the primary endpoint of durable
platelet response versus placebo. This clinically and statistically
significant result was achieved in a population of patients with
primary ITP that had been refractory to prior therapy. Overall,
study participants had a median of four prior ITP therapies and a
median baseline platelet count of 15,000/μL (normal platelet count
levels typically range from 150,000-450,000/μL). Positive results
on key secondary endpoints also underscore the potential for
rilzabrutinib to deliver clinically meaningful benefits for
patients living with persistent and chronic ITP.
Rilzabrutinib was granted Fast Track Designation
by the US Food and Drug Administration (FDA) for the treatment of
ITP in November 2020 and was previously granted Orphan Drug
Designation.
Houman AshrafianExecutive Vice
President, Head of Research and Development, Sanofi“The results of
this study reinforce rilzabrutinib’s potential to be a
first-in-class oral, reversible BTK inhibitor that can provide
clinically meaningful improvements for people living with severe
immune-mediated diseases like ITP. These pivotal results are a
testament to our commitment and expertise in rare blood diseases
and ability to build a portfolio of next-generation small-molecule
inhibitors that are both more selective and optimized to deliver
robust efficacy and safety outcomes as compared to existing
therapies.”
ITP is a serious, acquired autoimmune blood
disorder characterized by autoantibody-mediated platelet
destruction and impaired platelet production, leading to
thrombocytopenia (low platelet counts of less than 100,000/μL) and
an increased risk of life-threatening bleeding episodes (like
intracranial hemorrhage). In addition, patients with ITP often
experience significant quality-of-life impairments such as fatigue
and cognitive dysfunction. With its dual mechanisms of action that
reduce production of pathogenic autoantibodies and decrease
macrophage mediated platelet destruction, rilzabrutinib could
address the underlying mechanisms responsible for a wide range of
ITP complications.
About LUNA 3LUNA 3 (NCT04562766) is a
randomized, multicenter, phase 3 study evaluating the efficacy and
safety of rilzabrutinib vs placebo in adult and adolescent patients
with persistent or chronic ITP. Patients received either oral
rilzabrutinib 400 mg twice a day or placebo through a 12- to
24-week double-blind treatment period, followed by a 28-week
open-label treatment, and then a 4-week safety follow-up or
long-term extension period. The adolescent part of the study is
ongoing and still recruiting.
The primary endpoint is durable platelet
response defined as the proportion of participants able to achieve
platelet counts at or above 50,000/μL for for at least 8 out of the
last 12 weeks of the 24-week blinded treatment period in the
absence of rescue therapy. Secondary endpoints include the number
of weeks with and time to platelet responses, rescue therapy use,
and physical fatigue and bleeding score.
Detailed results of the LUNA 3 phase 3 study
will be presented at a medical congress later this year.
Rilzabrutinib is currently under clinical
investigation, and its safety and efficacy have not been evaluated
by any regulatory authority.
About Rilzabrutinib Rilzabrutinib is an oral,
reversible, covalent BTK inhibitor that has the potential to be a
first- or best-in-class treatment of several immune-mediated
diseases. BTK, expressed in B cells, mast cells and other cells
from the innate immune system, plays a critical role in
inflammatory pathways and multiple immune-mediated disease
processes. With the application of Sanofi’s TAILORED
COVALENCY® technology, rilzabrutinib can selectively inhibit
the BTK target.
Rilzabrutinib is being studied across a variety
of immune-mediated diseases, including immune thrombocytopenia
(regulatory submission in H2 2024), asthma (phase 2), chronic
spontaneous urticaria (phase 3 start in 2024), prurigo nodularis
(phase 3 start in 2024), IgG4-related disease (phase 2b results in
H2 2024), and warm autoimmune hemolytic anemia (phase 2b results in
H2 2024).
About Sanofi We are an innovative
global healthcare company, driven by one purpose: we chase the
miracles of science to improve people’s lives. Our team, across the
world, 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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| victor.rouault@sanofi.com Timothy
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Investor RelationsThomas Kudsk
Larsen |+ 44 7545 513 693 |
thomas.larsen@sanofi.comAlizé
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