Pasatru (garetosmab-grts) / Regeneron, Sanofi - LARVOL DELTA

Garetosmab-grts. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Lipid Effects of Trevogrumab and Garetosmab in Addition to Semaglutide Treatment in People Living with Obesity: Results from the COURAGE Study (AHA 2026) - "Abstract is embargoed at this time."

Genetic Disorders • Obesity

Cardiac safety of myostatin and activin A inhibition with trevogrumab and garetosmab in adults living with obesity: echocardiographic assessment from COURAGE phase 2 study (EASD 2026) - P2 | "Myostatin inhibition with TREVO ± activin A inhibition with GAR, on top of SEMA, revealed no adverse cardiac safety signals with respect to LVM or systolic function at 26 weeks, supporting the cardiac safety of these agents in adults living with obesity."

Clinical • P2 data • Diabetes • Metabolic Disorders • Obesity • Type 2 Diabetes Mellitus

Trevogrumab (an anti-myostatin) mitigates semaglutide‑associated lean body mass loss in adults with obesity and fully restores lean mass after semaglutide is discontinued (EASD 2026) - P2 | "Materials and People with obesity (BMI ≥30 kg/m 2 ), without type 2 diabetes (N = 599), were randomized 1:1:1:1 to receive semaglutide titrated up to 2.4 mg (SC QW) alone or in combination with trevogrumab 200 mg (SC QW), or trevogrumab 400 mg (SC QW) ± garetosmab (anti-activin A, 10 mg/kg IV Q4W), or placebos for 26 weeks (treatment period 1, TP1). Trevogrumab effectively minimized lean mass loss during GLP1-RA treatment and fully restored lean mass after GLP1-RA discontinuation."

Clinical • Diabetes • Metabolic Disorders • Obesity • Type 2 Diabetes Mellitus

OPTIMA-2: A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (clinicaltrials.gov) - P3 | N=18 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals | Initiation date: Jul 2026 ➔ Feb 2027

Trial initiation date

The Long-Term Safety and Efficacy of Garetosmab in Adults with Fibrodysplasia Ossificans Progressiva: Week 84 Results From the Phase 3 OPTIMA Study (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data

The U.S. FDA on Wednesday approved Regeneron's drug for a rare genetic disorder after it significantly reduced abnormal bone formation in certain soft tissues. (The Economic Times) - "The drug, garetosmab, branded ‌as Pasatru, ⁠was approved ⁠for treating adults with fibrodysplasia ossificans progressiva - a condition where muscle, tendon and ligament tissue gradually turn into bone, leading to a 'second skeleton' that causes progressive loss of mobility and reduced life expectancy."

FDA approval • Musculoskeletal Diseases • Rare Diseases

Inhibition of Activin A by the Neutralizing Antibody Garetosmab for Resolution of Nascent Heterotopic Bone Lesions in FOP Mice and People with FOP (ASBMR 2026) - No abstract available

Preclinical • Orthopedics

Effect of Garetosmab on Participant-reported Flare-ups in Fibrodysplasia Ossificans Progressiva: A Post Hoc Analysis of the Phase 3 OPTIMA Study (ASBMR 2026) - No abstract available

P3 data • Retrospective data

Clinical Pharmacology of Garetosmab in Patients with Fibrodysplasia Ossificans Progressiva (ASBMR 2026) - No abstract available

Clinical

Clinical Pharmacology of Garetosmab in Patients with Fibrodysplasia Ossificans Progressiva (ASBMR 2026) - No abstract available

Clinical

Inhibition of Activin A Stops the Regrowth of Surgically Resected Heterotopic Bone in a Mouse Model of Fibrodysplasia Ossificans Progressiva (ENDO 2026) - "Using FOP mice, we demonstrate that whereas surgical excision of heterotopic bone lesions (in the absence of garetosmab) induces explosive growth of the resected lesions as well as nearby lesions, this effect is completely inhibited by garetosmab. Our results not only demonstrate that Activin A remains a key HO-inducing factor even after initiation of HO but also indicate that inhibition of Activin A may enable surgical excision of pre-existing HO lesions while minimizing the chances of recurrence."

Preclinical • Genetic Disorders • ACVR1

Assessing the Participant Experience with Fibrodysplasia Ossificans Progressiva: Qualitative Interviews from the OPTIMA Trial (ENDO 2026) - "The efficacy and safety of garetosmab (GAR), a fully human monoclonal anti-activin A antibody, was investigated in adults with FOP in the phase 2 LUMINA-1 study and the phase 3 OPTIMA study; GAR demonstrated an acceptable safety profile and reduced new heterotopic bone lesions... The data show a significant pretreatment burden of disease in people with FOP and provide insights into the benefits to pts on GAR in the OPTIMA study."

Interview • Genetic Disorders • ACVR1

Safety and Efficacy of Garetosmab in Adults with Fibrodysplasia Ossificans Progressiva: Week 56 Results from the Phase 3 OPTIMA Study (ENDO 2026) - P3 | "GAR demonstrated an acceptable safety profile and showed a substantial reduction in the number and volume of new heterotopic bone lesions providing robust evidence of its efficacy in adults with FOP."

Clinical • P3 data • Acne Vulgaris • Genetic Disorders • Infectious Disease • ACVR1

Inhibition of Activin A Stops the Regrowth of Surgically Resected Heterotopic Bone in a Mouse Model of Fibrodysplasia Ossificans Progressiva (ENDO 2026) - "Using FOP mice, we demonstrate that whereas surgical excision of heterotopic bone lesions (in the absence of garetosmab) induces explosive growth of the resected lesions as well as nearby lesions, this effect is completely inhibited by garetosmab. Our results not only demonstrate that Activin A remains a key HO-inducing factor even after initiation of HO but also indicate that inhibition of Activin A may enable surgical excision of pre-existing HO lesions while minimizing the chances of recurrence."

Preclinical • Genetic Disorders • ACVR1

Population-Normed Z-scores for Body Composition Enhance Sensitivity and Effect Size: Analysis of a Phase 2 RCT Testing Anti-GDF8 and Anti-Activin A on Top of Semaglutide (COURAGE) (ADA 2026) - P2 | "Z-scores were assessed in the COURAGE trial (NCT06299098), which tested the efficacy of semaglutide vs semaglutide with trevogrumab (anti-GDF8) with or without garetosmab (anti-activin A) for weight, TFM loss, and TLM preservation. The NHANES dataset (n = 26,125) was used to model the distributions of TLM and TFM as functions of covariates. Z-scores account for differences in weight loss across arms, yielding a larger effect size and greater sensitivity to group differences than percent CFB."

Clinical • P2 data • Metabolic Disorders

Lean Mass Effects of Anti-GDF8 (Trevogrumab) ± Anti-Activin A (Garetosmab) in People Living with Obesity Treated with Semaglutide with or without Low Lean Mass at Baseline (ADA 2026) - "Anti-GDF8 with or without anti-activin A on-top of sema preserved LM when compared to sema alone in individuals with obesity, regardless of pre-existing LLM at baseline. Additional research is needed to determine the potential benefit of reduced LM loss."

Diabetes • Metabolic Disorders • Obesity • Type 2 Diabetes Mellitus

OPTIMA-2: A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (clinicaltrials.gov) - P3 | N=18 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals

New P3 trial

Content Validation of the Flares Diary: A Qualitative Analysis of the Flares Experience Within the Fibrodysplasia Ossificans Progressiva (FOP) Population. (PubMed, Adv Ther) - P2 | "The results confirm the adequacy of the Flares Diary and that the symptoms assessed are important to people with FOP. Graphical abstract available for this article."

Journal • Inflammation • Pain

Garetosmab Biologics License Application Accepted for FDA Priority Review for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) (Regeneron Pharmaceuticals Press Release) - "The target action date for the FDA decision is August 2026...The BLA is supported by efficacy and safety data from the positive Phase 3 OPTIMA trial evaluating garetosmab in adults with FOP. Both garetosmab doses (3 mg/kg and 10 mg/kg) evaluated in the trial were highly efficacious in reducing the total number and volume of new HO lesions at 56 weeks, compared to placebo."

FDA filing • PDUFA • Priority review • Genetic Disorders • Rare Diseases

Opportunities to Reduce Patient Burden in Clinical Trials for People Living With Obesity in the US (OBESITY WEEK 2025) - "Therapies, including bimagrumab (anti-activin type II), trevogrumab (anti-myostatin) and garetosmab (anti-activin A), aim to improve the quality of weight loss by preserving lean mass and augmenting the amount of weight loss attributable to fat mass. Integrating the perspective of people living with obesity will ensure that clinical trials are reflective of real-world experiences, reducing patient burden, improving recruitment and retention and ultimately generating high quality data."

Clinical • Genetic Disorders • Obesity

Pharmacological intervention: Challenges and promising outcomes for fat loss and preservation of lean body mass in the treatment of overweight and type 2 diabetes. (PubMed, Diabetes Obes Metab) - "Activin II receptor inhibition with bimagrumab demonstrated significant preservation and increases in LBM, along with FM reduction, in both preclinical and phase 2 studies in individuals with overweight and T2D. Similar effects were observed for myostatin and activin A inhibitors (trevogrumab, garetosmab), latent myostatin inhibitors (apitegromab, SRK-439), and Selective Androgen Receptor Modulators (enobosarm)...Pharmacological adjunct therapies show potential for improving body composition and physical function during GLP-1 RA-induced weight loss. Preliminary findings are promising, but larger, controlled trials are necessary to confirm efficacy and safety before clinical implementation can be considered."

Journal • Review • Diabetes • Genetic Disorders • Metabolic Disorders • Obesity • Type 2 Diabetes Mellitus

A Narrative Review of Phase II and III Clinical Trials for the Pharmacological Treatment of Fibrodysplasia Ossificans Progressiva (FOP): Efficacy, Safety, and Challenges in Treatment Strategies. (PubMed, Drug Des Devel Ther) - "Promising pharmacological approaches include retinoic acid receptor γ (RARγ) agonists, such as palovarotene, and monoclonal antibodies targeting activin A, like garetosmab. Nevertheless, safety concerns and the complexities of clinical trial implementation highlight the need for innovative approaches to ensure effective and equitable treatments. Future research should prioritise long-term safety, efficacy, and patient-centred outcomes to improve FOP management."

Journal • P2 data • P3 data • Review • Genetic Disorders • Rare Diseases

Study of Safety and Effects of Garetosmab in Healthy Obese Men and Post-Menopausal Women (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Regeneron Pharmaceuticals | N=48 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Genetic Disorders • Obesity • IGF1

Results from Phase 2 COURAGE Trial Demonstrating Potential to Improve Quality of GLP-1 receptor agonist-induced Weight Loss by Preserving Lean Mass, Presented at EASD (GlobeNewswire) - "Complete 26-week results further demonstrate that combining semaglutide with trevogrumab (anti-GDF8/anti-myostatin) helped prevent about half of semaglutide-induced loss of lean mass, while increasing fat mass loss. Numeric improvements in metabolic and lipid parameters including waist circumference, blood pressure, cholesterol, triglycerides and A1C, were observed across all treatment groups."

P2 data • Obesity