Clinical Trials
The process from lab to patient
We believe in our investigational medicines and their 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.
Neuromuscular and Cardiometabolic Disorders
Spinal Muscular Atrophy
TOPAZ
The TOPAZ trial was a proof-of-concept, open-label Phase 2 trial evaluating the safety and efficacy of apitegromab in patients with Type 2 and Type 3 SMA. In the main treatment period, participants were dosed intravenously every four weeks as monotherapy or with nusinersen, an approved SMN-targeted therapy. The trial enrolled 58 participants in the U.S. and Europe. The primary efficacy endpoints were mean change from baseline in Revised Hammersmith Scale (RHS) score at 12 months for the ambulatory population (Cohort 1), and mean change from baseline in HFMSE score at 12 months for the non-ambulatory population (Cohorts 2 and 3). The trial also included multiple 12-month extension periods designed to evaluate longer-term participant outcomes.
Spinal Muscular Atrophy
SAPPHIRE
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 are receiving current standard of care (either nusinersen or risdiplam). SAPPHIRE enrolled 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that enrolled 32 patients aged 13-21 years old was also evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo.
Spinal Muscular Atrophy
ONYX
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.
Cardiometabolic Disorders
EMBRAZE
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 participants aged 18-65 who are overweight or obese without diabetes. As part of the 24-week-long study design, all participants will receive a GLP-1 RA. In addition, all participants will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. Results from the EMBRAZE trial will inform the development of SRK-439, a novel, preclinical muscle-targeted therapy designed to support healthier weight management and lean muscle preservation during and after treatment with obesity therapies.
Primary data from EMBRAZE are expected in mid-2025.
Immunology and Oncology
Cancer (locally advanced or metastatic solid tumors)
DRAGON
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.
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Patients seeking more information on potential clinical trials should contact their physician.
