Rilzabrutinib

Sanofi’s BTK inhibitor rilzabrutinib helped 23% of adults with a rare autoimmune blood disorder achieve target platelet counts in a Phase 3 trial, compared to zero patients in the placebo arm, the company said Tuesday. The data, unveiled ahead of the American Society of Hematology meeting in December, bolster rilzabrutinib’s case as Sanofi prepares a regulatory filing in the US for immune thrombocytopenia, or ITP. The drug was part of Sanofi’s $3.7 billion Principia Biopharma buyout in 2020, and the company now touts it as a potential blockbuster that could generate €2 billion ($2.2 billion) to €5 billion ($5.5 billion) in peak sales, according to a December R&D presentation . Of 133 adults randomized to receive rilzabrutinib in the LUNA 3 trial, 31 achieved the primary endpoint of durable platelet response, meeting platelet counts at or above 50,000/μL for at least eight of the last 12 weeks of the 24-week blinded treatment period, with no rescue therapy. Zero of 69 placebo patients achieved the primary endpoint. ITP is a rare autoimmune disorder that leads to low platelet counts and an increased risk of bleeding and bruising. Patients in the trial had failed on or were intolerant to prior therapies. Sanofi said 46% of participating adults have received five or more treatments, and some had been dealing with ITP for years. While the trial enrolled both adults and adolescents, Sanofi said this is the first report of efficacy and safety in the adult population. The company also reported “significant improvements” in key secondary endpoints, including a reduction in the need for rescue therapy by 53% compared to placebo, and improvements in physical fatigue. While Sanofi said a similar percentage of patients in the rilzabrutinib and placebo arms experienced adverse events, it noted that one patient “with multiple risk-factors” had a treatment-related Grade 3 peripheral embolism, also known as a blockage in an artery. Sanofi said on its second-quarter earnings call in July that it wants to file a regulatory submission this year, according to an AlphaSense transcript. Rilzabrutinib’s Phase 3 win is welcome news following the drug’s Phase 2 fail in atopic dermatitis last year and a disappointing Phase 3 readout in pemphigus in 2021. But Sanofi continues to pursue a handful of other mid-stage programs, including asthma, chronic spontaneous urticaria, IgG4-related disease and warm autoimmune hemolytic anemia, according to the company’s pipeline . Stay tuned for more coverage of ASH from Endpoints , and sign up for our virtual ASH event here .

Share EADV: New data reinforce Sanofi’s innovative approach and leadership across immune-mediated skin diseases 24 abstracts for Dupixent, including 4 oral presentations, highlight impact of targeting interleukin-4 (IL4) and interleukin-13 (IL13) across atopic dermatitis (AD), prurigo nodularis (PN) and chronic spontaneous urticaria (CSU) New data show impact of Dupixent on potential for clinical remission in children as young as six years of age with moderate-to-severe AD 15 abstracts for immunology pipeline molecules, including phase 2 presentations in AD for amlitelimab, an OX40-ligand monoclonal antibody Paris, September 25, 2024. Sanofi will present 39 abstracts across approved and pipeline medicines at the 2024 European Academy of Dermatology and Venereology (EADV) medical meeting from September 25-28 in Amsterdam, the Netherlands. Presentations will include 21 abstracts for Dupixent (dupilumab) in partnership with Regeneron, highlighting the impact of targeting IL4 and IL13 across three chronic skin conditions, including disease remission data and long-term data in children with moderate-to-severe atopic dermatitis (AD), and rapid results in adults with prurigo nodularis (PN). In addition, data presentations from Sanofi’s extensive immunology pipeline include oral and poster presentations for amlitelimab, an OX40-ligand monoclonal antibody, demonstrating safety and efficacy results in moderate-to-severe AD, as well as in poster presentations for rilzabrutinib, a novel oral BTK inhibitor, showing its impact on symptoms of AD and moderate-to-severe chronic spontaneous urticaria (CSU). Dietmar Berger, M.D., Ph.D. Chief Medical Officer, Global Head of Development at Sanofi “Our data at EADV highlight the breadth of our diverse and novel approach across inflammatory dermatologic conditions, including AD, PN and CSU. Dupixent data demonstrates the potential benefit of important treatment goals such as clinical remission and the speed and durability of efficacy. We continue to evaluate Dupixent’s real-world safety profile and consistency of patient impact and satisfaction in children, adolescents, and adults with atopic dermatitis across the globe. Additionally, we are excited to share new data from our pipeline, including results for amlitelimab showing the potential to provide durable efficacy that may enable a quarterly dosing interval, with favorable safety profile.” Notable presentations include: Dupixent New results and analyses for Dupixent in children and adults with moderate-to-severe AD and adults with prurigo nodularis. Atopic dermatitis multiple poster presentations will show results in children with moderate-to-severe AD, including those from an open-label extension study on clinical remission in children aged six to 17 years of age, including after stopping treatment with Dupixent, as well as long-term efficacy and safety data for up to two years on children aged six months to five years. Additionally, real-world data in children six months to 11 years of age will show patient-reported disease severity and health-related quality of life outcomes throughout one year. an oral presentation will show real-world safety data from around the world for Dupixent in adults and adolescents with moderate-to-severe AD. Prurigo nodularis PRIME and PRIME2: an oral presentation will show new analyses on disease activity, itch, and skin clearance as early as two weeks from two pivotal clinical studies. The safety results of these studies were generally consistent with the known safety profile of Dupixent in its approved dermatological indications. Dermatology pipeline Data include new results and analyses for amlitelimab, an OX40-ligand monoclonal antibody, in AD, and rilzabrutinib, a novel oral BTK inhibitor, in AD and CSU. Atopic dermatitis amlitelimab: six presentations will share data for amlitelimab in moderate-to-severe AD, including an oral presentation of 68-week safety results. Additional presentations highlight a PK model that support the potential use of a quarterly dosing regimen, a post-hoc analysis showing the impact of amlitelimab on AD of the head and neck, and results from an in vitro analysis demonstrating that the anti-OX40L mechanism of amlitelimab does not deplete T cells. rilzabrutinib: a poster presentation will show the impact of rilzabrutinib on itch in adults with moderate-to-severe AD. Chronic spontaneous urticaria rilzabrutinib: three poster presentations will show the impact of rilzabrutinib on itch, hives and urticaria in adults with moderate-to-severe CSU. Hidradenitis suppurativa three poster presentations will show the impact of disease symptoms on patients with hidradenitis suppurativa and highlight the need for increased disease awareness and effective treatment options. Alopecia areata a poster presentation will show conceptual model and clinical outcome measures in alopecia areata based on the patient experience. Amlitelimab and rilzabrutinib are investigational medicines for which safety and efficacy have not been evaluated by any regulatory authority. Complete list of EADV 2024 presentations: 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 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 Sanofi Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | + 1 215 432 0234 | evan.berland@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.comTimothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com Sanofi Investor RelationsThomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.comAlizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.comArnaud Delépine | + 33 6 73 69 36 93 |arnaud.delepine@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comKeita Browne | + 1 781 249 1766 | keita.browne@sanofi.comNathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.comThibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Sanofi Forward-Looking StatementsThis media update contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. 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