Company: SNY
Filing Date: 2025-02-13
Form Type: 20-F
Source: 0001121404-25-000010
Chunk: 88

Company: Sanofi
Filing Date: 2025-02-13
Form: 20-F
Chunk 88
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 3, in which venglustat is currently being evaluated in Phase 3 studies. Results in these two indications are expected in the second half of 2025. Orphan drug designation has been granted in the US, Europe, and Japan for FD and Gaucher disease type 3, and the FDA has granted venglustat fast- track designation for FD. SAR447537 (formerly INBRX-101): in May 2024, the acquisition of Inhibrx, Inc. closed, adding SAR447537 to Sanofi’s rare disease Phase 2 pipeline. SAR447537 is a human recombinant protein that holds the promise of allowing alpha-1 antitrypsin deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent dosing. AATD is an inherited rare disease characterized by low levels of AAT protein, predominantly affecting the lung with progressive deterioration of the tissue. Results from the ongoing Phase 2 study (ELEVAATE) are expected in the second half of 2025. The FDA has granted SAR447537 fast- track designation for AATD. In 2024, the decision was taken to discontinue the development of riliprubart (see details in “— a) Immunology & Inflammation” above) in cold agglutinin disease, a rare autoimmune disorder characterized by the premature destruction of red blood cells (hemolysis), due to prioritization of other projects. As of now, the data confirmed pharmacological activity and a well-tolerated safety profile, as in other indications. c) Neurology tolebrutinib (SAR442168) is an oral investigational brain-penetrant and bioactive Bruton’s tyrosine kinase (BTK) inhibitor, which achieves cerebrospinal fluid concentrations that are predicted to modulate B lymphocytes and microglial cells. Positive results from the HERCULES Phase 3 study showed that tolebrutinib met the primary endpoint of improvement over placebo in delay ing time to onset of confirmed disability progression in subjects with non-relapsing secondary progressive multiple sclerosis (nrSPMS). Preliminary analysis of liver safety was consistent with previous tolebrutinib studies. In December, the FDA granted breakthr ough therapy designation to tolebrutinib for the treatment of adults with nrSPMS. Sanofi expects to receive regulatory submission acceptance in the US during the first half of 202