Peptides
Domagrozumab
A myostatin-blocking antibody whose Duchenne trial failed to show functional benefit, illustrating why muscle-targeting mechanisms need outcome evidence.
Negative randomized DMD trial; development terminated
Also known as: PF-06252616
Practical use
Evidence interpretation and clinical context
- Read the prespecified endpoint
- Use the stair-climb result as the main functional finding.
- Keep negative studies visible
- Do not rely only on animal muscle-growth images or early mechanism descriptions.
- Identify the molecule
- An antibody name, target and disease population are more informative than the broad term myostatin blocker.
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.
Overview
Domagrozumab is a monoclonal antibody developed to inhibit myostatin, a regulator that restrains muscle growth. The principal human program studied Duchenne muscular dystrophy (DMD). It is an engineered antibody, not interchangeable with a short peptide or another myostatin-pathway drug.
Risks and limitations
Most participants in the DMD trial experienced at least one adverse event; most were mild and judged unrelated. A trial with no clear efficacy advantage cannot establish a favorable benefit–risk balance for healthy muscle building.
How to read this
DMD involves progressive disease in muscle, so adding size would not necessarily correct the underlying problem. Function has to be measured directly.
Research explained
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.
Who was studied
120 ambulatory boys aged 6 to under 16 with DMD; 80 received domagrozumab and 40 placebo in the randomized phase.
What was found
At week 49, the between-group difference in four-stair-climb change was 0.27 seconds, with a wide confidence interval crossing zero and p=0.94. Secondary clinical endpoints also did not significantly differ.
What it cannot establish
Nonsignificant muscle-volume changes do not rescue the negative functional result. The trial did not test healthy trainees.
Read the original studyDevelopment status
Pfizer announced termination of the DMD phase 2 and extension studies in 2018. This history should accompany references describing domagrozumab as a promising investigational antibody.
Supporting sourceA pathway is not a class-wide success
Apitegromab, bimagrumab and domagrozumab intervene differently in related signaling. Results from one cannot establish benefit for the others or for a product sold under a generic myostatin-inhibitor label.
Supporting sourceClaims versus findings
Not supported
Blocking myostatin guarantees improved strength or function.
The randomized functional endpoints did not show a significant domagrozumab benefit.
Check the evidenceCommon questions
Does the failed DMD trial prove all myostatin research is ineffective?
No. It limits this molecule in this setting; other interventions and populations require their own trials.
SourceSources
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.
Development status — primary or official source
Sources checked 2026-10-01.
General education; not a personal treatment plan.