Sanofi provides update on Phase 3 study evaluating rilzabrutinib
for the treatment of pemphigus
Sanofi provides update on Phase
3 study evaluating
rilzabrutinib for the treatment of
pemphigus
PARIS –
September 9, 2021 – The Phase 3 PEGASUS trial
evaluating rilzabrutinib to treat pemphigus, a rare autoimmune skin
condition, did not meet its primary or key secondary endpoints.
Rilzabrutinib’s safety profile remained consistent with previous
results and no new safety signals were identified.
The Phase 3 study, which is the first
placebo-controlled trial of a BTK inhibitor in pemphigus, enrolled
adult patients with moderate-to-severe pemphigus vulgaris or
pemphigus foliaceus. The primary endpoint was complete remission
from weeks 29 to 37 with minimal doses of corticosteroids (≤10/mg
day). Complete remission was defined as the absence of new and
established skin lesions. Results show the proportion of patients
meeting the primary endpoint on rilzabrutinib was not significantly
different from placebo.
Sanofi is continuing to evaluate the data and
plans to share detailed findings at a future medical meeting.
“While these results are disappointing, we
believe the rilzabrutinib clinical program holds great potential to
address the unmet treatment needs of people living with
immune-mediated diseases,” said Naimish Patel, Head of Global
Development, Immunology and Inflammation. “Our mission is to
improve outcomes by exploring new scientific approaches and novel
therapies to advance the standard of care. We are committed to
investigating rilzabrutinib further and progressing our clinical
programs forward to deliver new treatment options for
patients.”
Pemphigus is a group of potentially
life-threatening disorders characterized by blisters and ulceration
affecting the skin and mucous membranes. Currently options for the
treatment of pemphigus (including pemphigus vulgaris and pemphigus
foliaceus) are limited and systemic corticosteroid treatment
remains the standard of care.
Rilzabrutinib is a potential first-in-class,
oral Bruton's tyrosine kinase (BTK) inhibitor in development for
immune-mediated diseases. The BTK enzyme plays a key role in a
number of immune processes including B cell expansion, production
of immunoglobulins, and activation of innate cells such as mast
cells, eosinophils, and basophils. Positive clinical trial data
from placebo-controlled studies of BTK inhibitors have revealed the
potential role for BTK in rheumatoid arthritis and in chronic
spontaneous urticaria1,2. Thus the function of BTK is biologically
diverse and supports continued investigation in a range of diseases
with significant unmet need where BTK is implicated.
Rilzabrutinib is being investigated in a Phase 3
trial for the treatment of immune thrombocytopenia, a rare blood
disorder, and in a Phase 2 study for the autoimmune condition
IgG4-related disease. Additional Phase 2 studies in immunological
diseases including asthma, atopic dermatitis, chronic spontaneous
urticaria and warm autoimmune hemolytic anemia are planned to start
in 2021.
About the PEGASUS study
The PEGASUS study is a Phase 3 randomized,
parallel-group, double-blind, placebo-controlled trial which
enrolled 131 patients with newly diagnosed or relapsing moderate-
to-severe pemphigus in 19 countries worldwide. The primary endpoint
was complete remission from weeks 29 to 37 with minimal doses of
corticosteroids (CS) (≤10/mg day). Complete remission is defined as
the absence of new and established skin lesions. Key secondary
endpoints include cumulative CS dose (from Baseline to Week 37),
cumulative duration of complete remission with a CS dose ≤10 mg/day
and time to first complete remission with a CS dose ≤10 mg/day.
(NCT03762265)
About Rilzabrutinib
Rilzabrutinib is an oral Bruton’s tyrosine kinase inhibitor
incorporating Sanofi’s TAILORED COVALENCY®3 technology being
investigated for the treatment of immune-mediated diseases. BTK is
an intracellular signaling molecule involved in innate and adaptive
immune responses involved in certain immune-mediated diseases. By
inhibiting BTK, rilzabrutinib has the potential to target the
underlying disease pathogenesis.
Orphan drug designation was granted by the US
Food and Drug Administration (FDA) for pemphigus vulgaris (and from
the European Commission for the treatment of pemphigus) and for its
investigational use in immune thrombocytopenia (ITP). Rilzabrutinib
was granted FDA Fast Track Designation for ITP in November 2020 and
for pemphigus vulgaris in May 2021.
Rilzabrutinib is currently under clinical
investigation and its safety and efficacy have not been evaluated
by any regulatory authority.__________1 Cohen S, et al,
Fenebrutinib versus Placebo or Adalimumab in Rheumatoid Arthritis:
A Randomized, Double-Blind, Phase II Trial (ANDES Study). Arthritis
Rheumatol. 2020 Apr 9;72(9):1435–46. doi: 10.1002/art.41275. Epub
ahead of print. PMID: 32270926; PMCID: PMC7496340
2. Metz,M, et al Fenebrutinib in Refractory
Chronic Spontaneous Uticaria, 2020, Abstract. Allergy, 75:
100-119. https://doi.org/10.1111/all.14505
3 TAILORED COVALENCY is a registered trademark
of Principia Biopharma Inc., a Sanofi Company.
About Sanofi
Sanofi is dedicated to supporting people through
their health challenges. We are a global biopharmaceutical company
focused on human health. We prevent illness with vaccines, provide
innovative treatments to fight pain and ease suffering. We stand by
the few who suffer from rare diseases and the millions with
long-term chronic conditions.
With more than 100,000 people in 100 countries,
Sanofi is transforming scientific innovation into healthcare
solutions around the globe.
Media Relations Contact Sally BainTel.: +1
(781) 264-1091Sally.Bain@sanofi.com
Investor Relations Contacts Paris Eva
Schaefer-Jansen Arnaud Delepine Nathalie Pham
Investor Relations Contacts North
America Felix Lauscher
Tel.: +33 (0)1 53 77 45 45
investor.relations@sanofi.com
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