SAR4490288 / Sanofi - LARVOL DELTA

Study of BLU-808 in Allergic Rhinoconjunctivitis (clinicaltrials.gov) - P2 | N=54 | Active, not recruiting | Sponsor: Blueprint Medicines Corporation | Recruiting ➔ Active, not recruiting

Enrollment closed • Conjunctivitis • Ocular Infections • Ocular Inflammation • Ophthalmology

BLU-808, a potent and selective inhibitor of wild-type KIT, in patients with chronic inducible urticaria and chronic spontaneous urticaria: trial-in-progress (EADV 2025) - P2 | "Current treatments, such as second-generation H1-antihistamines and the anti-IgE monoclonal antibody omalizumab, do not fully alleviate symptoms for many patients, underscoring the urgent need for therapies with novel mechanisms of action. The BLU-808-1201 study will assess the safety, tolerability, and clinical activity of BLU-808 in patients with CIndU and CSU, potentially offering a new therapeutic option for patients with these conditions."

Clinical • Allergic Rhinitis • Asthma • Cardiovascular • Chronic Spontaneous Urticaria • Dermatology • Immunology • Inflammation • Pruritus • Respiratory Diseases • Urticaria

Press Release: Sanofi completes acquisition of Blueprint Medicines (Sanofi Press Release) - "Sanofi today announces the completion of its acquisition of Blueprint Medicines Corporation (Blueprint), adding to its portfolio a commercialized medicine, a promising pipeline, and the expertise of a company specializing in systemic mastocytosis (SM), a rare immunological disease, and other KIT-driven diseases....The acquisition includes a rare immunology disease medicine, Ayvakit/Ayvakyt (avapritinib), approved in the US and EU....The acquisition also includes elenestinib, a next-generation medicine for SM that is a potent and highly selective KIT D816V inhibitor with limited central nervous system penetration....Sanofi also acquired BLU-808, an investigational oral, highly potent and selective wild-type KIT inhibitor."

M&A • Aggressive Systemic Mastocytosis • Immunology

Study of BLU-808 in Chronic Inducible Urticaria (CIndU) and Chronic Spontaneous Urticaria (CSU) (clinicaltrials.gov) - P2 | N=105 | Recruiting | Sponsor: Blueprint Medicines Corporation | Not yet recruiting ➔ Recruiting

Enrollment open • Chronic Spontaneous Urticaria • Dermatology • Immunology • Urticaria

Study of BLU-808 in Allergic Rhinoconjunctivitis (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: Blueprint Medicines Corporation | Not yet recruiting ➔ Recruiting

Enrollment open • Conjunctivitis • Ocular Infections • Ocular Inflammation • Ophthalmology

Study of BLU-808 in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=95 | Completed | Sponsor: Blueprint Medicines Corporation

New P1 trial

Study of BLU-808 in Allergic Rhinoconjunctivitis (clinicaltrials.gov) - P2 | N=54 | Not yet recruiting | Sponsor: Blueprint Medicines Corporation

New P2 trial • Conjunctivitis • Ocular Infections • Ocular Inflammation • Ophthalmology

Study of BLU-808 in Chronic Inducible Urticaria (CIndU) and Chronic Spontaneous Urticaria (CSU) (clinicaltrials.gov) - P2 | N=105 | Not yet recruiting | Sponsor: Blueprint Medicines Corporation

New P2 trial • Chronic Spontaneous Urticaria • Dermatology • Immunology • Urticaria

BLU-808: Positive Healthy Volunteer Data (PRNewswire) - P1 | N=NA | "Further, we are building on our foundational expertise in ISM with our oral wild-type KIT inhibitor BLU-808, with healthy volunteer data showing a differentiated clinical profile and wide therapeutic index that supports broad development across a range of mast cell diseases. Based on these data, we are initiating multiple proof-of-concept trials, with initial data expected later this year...In the Phase 1 single-ascending dose (SAD) and multiple-ascending dose (MAD, 14-day dosing) trial, BLU-808 was well-tolerated at all doses tested. There were no serious AEs, no discontinuations or dose modifications due to AEs, and no significant changes in laboratory measures. BLU-808 achieved dose-dependent serum tryptase reductions exceeding 80 percent, reflecting evidence of mast cell target engagement. BLU-808 had a half-life supporting once-daily oral dosing and showed sustained target coverage across a range of doses."

Clinical data • P1 data • Aggressive Systemic Mastocytosis • Immunology

Blueprint Medicines Reports Fourth Quarter and Full Year 2024 Results (PRNewswire) - "For the full year, achieved AYVAKIT net product revenues of $479.0 million, including $421.8 million in the US and $57.1 million ex-US...In the fourth quarter of 2024, AYVAKIT achieved $144.1 million in net product revenues, including $124.1 million in the US and $20.0 million ex-US. Blueprint estimates the peak revenue opportunity for the company's SM franchise is $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030...The company plans to achieve the following milestones in the first half of 2025: i) Present 14 abstracts at the 2025 American Academy of Allergy, Asthma and Immunology (AAAAI)...including: Three-year safety and efficacy data from the PIONEER trial of AYVAKIT. Bone density improvements shown by AYVAKIT...Phase 1 healthy volunteer data for BLU-808...Initiate two proof-of-concept studies of BLU-808 in patients with chronic spontaneous urticaria/chronic inducible urticaria, and allergic rhinitis/allergic conjunctivitis."

Commercial • New trial • P1 data • P2 data • Aggressive Systemic Mastocytosis • Allergic Rhinitis • Chronic Spontaneous Urticaria • Dermatology • Gastrointestinal Cancer • Gastrointestinal Stromal Tumor • Soft Tissue Sarcoma • Urticaria

BLU-808: A Potent and Selective Oral Small Molecule Wild-Type KIT Tyrosine Kinase Inhibitor for Allergic Conditions (AAAAI-WAO 2025) - "Conclusions BLU-808 is efficacious in a preclinical rodent model of asthma. Mast cell inhibition may be a promising therapeutic for other allergic diseases."

Asthma • Atopic Dermatitis • Dermatitis • Dermatology • Food Hypersensitivity • Immunology • Inflammation • Prurigo Nodularis • Respiratory Diseases • Urticaria

Safety and Pharmacokinetics (PK) of BLU-808 Following Oral Dosing in Healthy Volunteers (AAAAI-WAO 2025) - "To date, BLU-808 has been generally well tolerated. Conclusions Plasma PK, PD biomarkers, PK/PD relationship, and safety of BLU-808 will be presented."

Clinical • PK/PD data • Dermatology • Immunology • Inflammation • Urticaria

Blueprint Medicines Provides 2025 Outlook and Highlights Strategy for Continued Growth (PRNewswire) - P1 | N=39 | "Blueprint today announced results from the Phase 1 single-ascending dose (SAD; n=56) and multiple-ascending dose (MAD; n=31, 14-day dosing) trial of BLU-808...Safety: BLU-808 was well-tolerated at all doses tested...Pharmacokinetics: BLU-808 showed a half-life of approximately 40 hours, enabling once-daily dosing, and consistent, dose-dependent increases in drug exposure...Pharmacodynamics: BLU-808 showed dose-dependent serum tryptase responses, reflecting evidence of mast cell target engagement across multiple dose levels....Present topline results from the Phase 1 healthy volunteer trial at the J.P. Morgan conference on January 13, 2025. Initiate proof-of-concept trials in chronic spontaneous urticaria, chronic inducible urticaria, allergic rhinitis and allergic conjunctivitis in the first half of 2025. Initiate proof-of-concept trials in allergic asthma and mast cell activation syndrome in the second half of 2025."

New trial • P1 data • Allergic Rhinitis • Asthma • Chronic Spontaneous Urticaria • Conjunctivitis • Immunology

Blueprint Medicines Reports Second Quarter 2024 Results and Raises AYVAKIT/AYVAKYT (avapritinib) Full Year Revenue Guidance (PRNewswire) - "Achieved AYVAKIT net product revenues of $114.1 million for second quarter of 2024, representing more than 185 percent growth year-over-year. Achieved clearance of an Investigational New Drug application for BLU-808 by the U.S. Food and Drug Administration and initiated the healthy volunteer study. BLU-808 is a highly selective and potent investigational oral wild-type KIT inhibitor with best-in-class potential, for chronic urticaria and other mast cell disorders."

IND • New trial • Sales • Chronic Spontaneous Urticaria • Dermatology • Gastrointestinal Stromal Tumor • Immunology • Oncology • Solid Tumor • Urticaria

BLU-808, a Potent and Selective Small Molecule Inhibitor of Wild-type c-KIT for Mast Cell Disorders (AAAAI 2024) - "BLU-808, a potent and selective wt c-KIT inhibitor, demonstrated an ability to decrease mast cell degranulation in vitro and in vivo . This inhibitor is kinome-sparing, orally available, and may provide therapeutic benefit to patients suffering from wt c-KIT mediated mast cell disorders."

Dermatology • Immunology • Inflammation • Urticaria • CSF1R • FLT3 • PDGFRA