Sanofi Announces Positive Phase 3 Results for Rilzabrutinib in Immune Thrombocytopenia (ITP) at ASH 2024

Nov 06, 2024 11:16 CST Updated 11:16

Sanofi

Pharmaceutical R&D Developer

Today, Sanofi announced positive results from its potential "first-in-class" oral BTK inhibitor rilzabrutinib in a Phase 3 ITP trial, as published in the abstracts of this year's American Society of Hematology (ASH) Annual Meeting:23% of adult patients with immune thrombocytopenia (ITP) reached the primary endpoint, while no patients in the placebo group met the criteria. Sanofi is preparing to submit a regulatory application to the FDA for the use of this therapy in ITP treatment.

Rilzabrutinib is an orally administered, reversible covalent BTK inhibitor., it is expected to become a potential "first-in-class" and "best-in-class" therapy for various immune-mediated diseases.Rilzabrutinib was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in November 2020 for the treatment of ITP.

LUNA 3 is a sub-period, multicenter, randomized, double-blind, placebo-controlled, parallel-group study with an open-label extension phase, evaluating the efficacy and safety of oral Rilzabrutinib in adult and adolescent patients with persistent or chronic ITP.In the LUNA 3 trial, a total of 133 adults were randomly assigned to receive rilzabrutinib treatment,Data show:

• In the Phase 3 trial for the treatment of rare autoimmune blood disorder ITPRilzabrutinib helped 23% of adult patients reach the target platelet count, while no patients in the placebo group achieved this effect.。

• This is calledThe LUNA 3 trial met its primary endpoint of durable platelet response., which showed clinically and statistically significant results in previously treated patients.

• In addition to the primary endpoint, rilzabrutinib also demonstrated positive results on key secondary endpoints, including a 53% reduction in the need for urgent rescue therapy compared to placebo, as well as improvements in physical fatigue. Its safety profile was consistent with previous studies.

Rilzabrutinib Shows Positive Results in Phase 3 Trial for ITP, and the Phase 3 Trial for nrSPMS Also Yields Positive Results in September; Sanofi Is Optimistic About Its Market Prospects.RilzabrutinibOther Studies and IndicationsIncluding asthma, chronic spontaneous urticaria, prurigo nodularis, IgG4-related disease, and warm autoimmune hemolytic anemia.

In August 2020, Sanofi acquired Principia Biopharma for $3.7 billion, obtaining multiple products including this drug.Strengthens Sanofi's drug portfolio in the field of autoimmune disease treatment.

Sanofi expects rilzabrutinib to have the potential to become aBlockbuster with Multiple Indications, potentially bringing peak sales of up to $2.2 billion to $5.5 billion for the company。Sanofi Plans to Submit Regulatory Applications to the U.S. FDA and EU by the End of 2024If successfully approved, Rilzabrutinib will become the first BTK inhibitor approved for ITP indication.

Summary

The BTK target has been proven to be highly effective in treating B-cell tumors, but its development process has encountered setbacks multiple times.In September 2021, the Phase 3 clinical trial PEGASUS (NCT03762265) of Rilzabrutinib for the treatment of pemphigus failed to meet its primary and key secondary endpoints. In 2022, due to several cases of drug-related liver injury, the FDA partially placed a hold on the Phase III studies of Tolebrutinib for MS and myasthenia gravis, and Sanofi announced the discontinuation of the Phase III clinical study of Tolebrutinib for myasthenia gravis.In mid-2023, Sanofi announced the termination of the development of Atuzabrutinib. In October 2023, the Phase II study of Rilzabrutinib for the treatment of atopic dermatitis did not meet its primary endpoint....With the positive results of Rilzabrutinib in the Phase 3 LUNA 3 clinical trial for ITP, Sanofi is finally poised to achieve success in the BTK field.

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