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
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
Cardiometabolic Disorders
EMBRAZE
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
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.
