Clinical Trials
The process from lab to patient
We believe in our science and its potential to change patients’ lives. Clinical trials are a critical step in bringing a new therapy from our in-house laboratory to the world.
Read below for important background information and the most recent updates on our clinical trials. Patients seeking more information on potential clinical trials should contact their physician.
Neuromuscular
OPAL
Spinal Muscular Atrophy
Initiated, recruiting
OPAL is a Phase 2 double-blind study designed to evaluate apitegromab in patients under two years of age who have been or are continuing to be treated with any currently approved SMN therapy, including onasemnogene abeparvovec.
ONYX
Spinal Muscular Atrophy
Active, not recruiting
ONYX is an open-label Phase 3 study designed to assess the long-term safety and efficacy of apitegromab in participants with Type 2 and Type 3 SMA who have completed the TOPAZ or SAPPHIRE trial.
SAPPHIRE
Spinal Muscular Atrophy
Completed
SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in non-ambulatory patients with Types 2 and 3 SMA who were receiving either nusinersen or risdiplam.
View the Phase 3 study publication here.
Cardiometabolic Disorders
EMBRAZE
Cardiometabolic Disorders
EMBRAZE was a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab at 10mg/kg in adults with a body mass index (BMI) of ≥27.0 kg/m2 (overweight) with at least one weight-related comorbid condition or a BMI of ≥30.0 kg/m2 (obese) while receiving tirzepatide. The enrollment of EMBRAZE included 100 subjects aged 18-65 who were overweight or living with obesity without diabetes. As part of the study design, the treatment period was 24 weeks, and all subjects received tirzepatide. In addition, all subjects were randomized 1:1 and received either apitegromab 10mg/kg or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint was change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry. Secondary endpoints included additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 included cardiometabolic parameters (e.g. HbA1c), body composition, and physical function.
Immunology and Oncology
DRAGON
Cancer (locally advanced or metastatic solid tumors)
The DRAGON trial is investigating SRK-181, a selective inhibitor of TGFβ1 activation, in participants with locally advanced or metastatic solid tumors that have shown primary resistance to checkpoint inhibitor therapies. Part A was designed to assess safety and tolerability of SRK-181. With some results of Part A available, the Part B dose expansion portion evaluated SRK-181 in combination with an approved anti-PD-(L)1 therapy across multiple solid tumor trial groups to test proof of concept. The results from the combination therapy trial showed encouraging responses in heavily pretreated and anti-PD-(L)1 resistant participants across multiple tumor types. Additional results from the Part B dose expansion can be found here.
Expanded Access Policy
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Patients seeking more information on potential clinical trials should contact their physician.
