SRSD107 / Sirius Therap, CRISPR Therap - LARVOL DELTA

A Phase 1, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered SRSD107 in Healthy Subjects (ASH 2024) - P1 | "Conclusions : In healthy volunteers, a single dose of SRSD107 was safe and well tolerated, with PK parameters consistent with a typical siRNA product. Marked, prolonged changes from baseline in FXI activity and aPTT were observed, suggesting that SRSD107 could be a potent anticoagulant with a sustained effect over time."

Clinical • P1 data • PK/PD data • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Ischemic stroke • Myocardial Infarction • Thrombosis

CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in Europe (GlobeNewswire) - "The ongoing Phase 2 clinical trial is a randomized, multicenter, global study evaluating the safety and efficacy of SRSD107 for the prevention of VTE in patients undergoing TKA."

Trial status • Venous Thromboembolism

A Study to Compare the Efficacy and Safety of SRSD107 and Enoxaparin in Adult Subjects Undergoing TKA (clinicaltrials.gov) - P2 | N=450 | Not yet recruiting | Sponsor: Sirius Therapeutics Co., Ltd.

New P2 trial • Cardiovascular • Orthopedics • Pain • Venous Thromboembolism

A phase 1 study of the safety pharmacokinetics and pharmacodynamics of SRSD107 in healthy subjects (ISTH 2025) - "FXI levels remained suppressed for at least 24 weeks. Table or Figure Upload"

Clinical • P1 data • PK/PD data • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Thrombosis

CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies (GlobeNewswire) - "CRISPR Therapeutics...and Sirius Therapeutics...announced a strategic partnership to develop and commercialize siRNA therapies....Collaboration brings together complementary capabilities to co-develop and co-commercialize SRSD107, a next generation, long-acting Factor XI (FXI) small interfering RNA (siRNA) for the treatment of thromboembolic disorders. Under the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics; CRISPR Therapeutics also has rights to exclusively license up to two additional siRNA programs."

Licensing / partnership • Thrombosis • Venous Thromboembolism

SRSD107-101: A Phase 1 Study to Evaluate the Safety, Tolerability, PK/PD of SRSD107 in Healthy Participants (clinicaltrials.gov) - P1 | N=40 | Completed | Sponsor: Sirius Therapeutics Co., Ltd. | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Hematological Disorders • Thrombosis

A PHASE 1, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SRSD107 IN HEALTHY ADULT SUBJECTS - Patrick Yue (ACC 2025) - "In healthy volunteers, a single dose of SRSD107 was safe and well tolerated, with PK consistent with a typical siRNA product. Marked, prolonged changes from baseline in FXI activity, FXI antigen, and aPTT were observed, suggesting that SRSD107 could be a potent anticoagulant with a sustained effect over time."

Clinical • P1 data • PK/PD data • Cardiovascular

SRSD107-101: A Phase 1 Study to Evaluate the Safety, Tolerability, PK/PD of SRSD107 in Healthy Participants (clinicaltrials.gov) - P1 | N=40 | Active, not recruiting | Sponsor: Sirius Therapeutics Co., Ltd. | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2025 ➔ Jun 2025

Enrollment closed • Trial completion date • Cardiovascular • Hematological Disorders • Thrombosis

Sirius Therapeutics Announces Promising Phase I Clinical Data from Its Factor XI siRNA, a Long-Acting Next-Generation Anticoagulant for Thromboembolic Disorders (Businesswire) - P1 | N=40 | NCT06116617 | Sponsor: Sirius Therapeutics Co., Ltd. | "Sirius Therapeutics today announced promising preliminary data from its Phase 1 first-in-human clinical trial of SRSD107, a next generation siRNA therapeutics under clinical development for the prevention and treatment of thromboembolic disorders, such as myocardial infarction, ischemic stroke, and venous thromboembolism...In the trial, SRSD107 was safe and well-tolerated. Significant changes from baseline in PD biomarkers were observed, with maximal reductions in FXI antigen and activity > 90% and an aPTT (thromboplastin time) increase of > 100% (i.e., an aPTT ratio > 2.0) at the highest doses tested. The pharmacodynamic effects were durable, with FXI antigen and activity levels remaining suppressed for more than 16 weeks after dosing."

P1 data • Hematological Disorders • Venous Thromboembolism

A Phase 1 Study to Evaluate the Safety, Tolerability, PK/PD of SRSD107 in Healthy Participants (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Sirius Therapeutics Co., Ltd. | N=64 ➔ 40 | Trial completion date: Aug 2025 ➔ Jan 2025 | Trial primary completion date: Apr 2025 ➔ Jan 2025

Enrollment change • Trial completion date • Trial primary completion date • Cardiovascular • Hematological Disorders • Thrombosis

A Phase 1 Study to Evaluate the Safety, Tolerability, PK/PD of SRSD107 in Healthy Participants (clinicaltrials.gov) - P1 | N=64 | Recruiting | Sponsor: Sirius Therapeutics Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Hematological Disorders • Thrombosis

Sirius Therapeutics Announces Submission to Begin First Clinical Trial of Factor XI siRNA, a Long-Acting Next-Generation Anticoagulant for Thromboembolic Disorders (Businesswire) - "Sirius Therapeutics today announced it has submitted an application in Australia to begin a first-in-human clinical trial of SRSD107, its next generation siRNA therapeutic for the prevention and treatment of thromboembolic disorders. SRSD107, which specifically targeting coagulation factor XI (FXI), is the first Sirius-developed compound to reach clinical stage of development globally."

New trial • Cardiovascular • Thrombosis