efmitermant alfa (ACE-083) / Merck (MSD) - LARVOL DELTA

Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease. (PubMed, Neurology) - "Despite significantly increased TMV and CMV, patients with CMT receiving ACE-083 in tibialis anterior muscles did not demonstrate greater functional improvement compared with those receiving placebo."

Journal • P2 data • Genetic Disorders

Randomized Phase 2 Study of ACE-083, a Muscle-Promoting Agent, in Facioscapulohumeral Muscular Dystrophy. (PubMed, Muscle Nerve) - "Significant increases in TMV with ACE-083 versus placebo did not result in consistent functional or PRO improvements with up to 12 months' treatment."

Clinical • Journal • P2 data • Muscular Dystrophy

Follistatin-based ligand trap ACE-083 induces localized hypertrophy of skeletal muscle with functional improvement in models of neuromuscular disease. (PubMed, Sci Rep) - "Importantly, ACE-083 also increased the force of isometric contraction in situ by the injected tibialis anterior muscle in wild-type mice and disease models and increased ankle dorsiflexion torque in CMT mice. Our results demonstrate the potential of ACE-083 as a therapeutic agent for patients with CMT, muscular dystrophy and other disorders with focal or asymmetric muscle atrophy or weakness."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy • Myositis

Research advance of underlying pathogenesis and target therapies in Charcot-Marie-Tooth disease type 1A (PubMed, Zhonghua Yi Xue Yi Chuan Xue Za Zhi) - "Other targeting drugs include ascorbic acid, progesterone antagonists, IFB-088, ADX71441, and ACE-083. This review is to sum up the pathogenesis of CMT1A and promising targeting drug therapies for further research."

Journal • Gene Therapies • Genetic Disorders • Pain

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P2; N=76; Not yet recruiting; Sponsor: Acceleron Pharma, Inc.

New P2 trial • Biosimilar

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (clinicaltrials.gov) - P2; N=62; Terminated; Sponsor: Acceleron Pharma, Inc.; Trial completion date: Jul 2020 ➔ Mar 2020; Active, not recruiting ➔ Terminated; (Investigation of ACE-083 for use in patients with CMT is being discontinued as it did not achieve functional secondary endpoints in the A083-03 trial.

Clinical • Trial completion date • Trial termination • MRI

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) (clinicaltrials.gov) - P2; N=62; Terminated; Sponsor: Acceleron Pharma, Inc.; N=150 ➔ 62; Trial completion date: Aug 2022 ➔ Mar 2020; Enrolling by invitation ➔ Terminated; Trial primary completion date: Jun 2022 ➔ Mar 2020; Investigation of ACE-083 for use in patients with CMT is being discontinued as it did not achieve functional secondary endpoints in the A083-03 trial.

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • MRI

Results of a Phase 2 Double-Blind Placebo-Controlled Study of a Local Muscle Therapeutic, ACE-083, in Subjects with Charcot-Marie-Tooth (CMT) Disease (AAN 2020) - "Locally administered ACE-083 was well tolerated in subjects with CMT treated up to three months. Results from the 6-month placebo-controlled portion of the study will inform the effects of ACE-083 on muscle volume, fat fraction, functional endpoints, and patient-reported outcomes."

Clinical • P2 data • MRI

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (clinicaltrials.gov) - P2; N=42; Active, not recruiting; Sponsor: Acceleron Pharma, Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P2; N=95; Terminated; Sponsor: Acceleron Pharma, Inc.; N=58 ➔ 95; Trial completion date: Jun 2020 ➔ Oct 2019; Active, not recruiting ➔ Terminated; Trial primary completion date: Mar 2020 ➔ Sep 2019; Investigation of ACE-083 for use in patients with FSHD is being discontinued as it did not achieve functional secondary endpoints in the A083-02 trial.

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (clinicaltrials.gov) - P2; N=63; Active, not recruiting; Sponsor: Acceleron Pharma, Inc.; N=42 ➔ 63; Trial completion date: Feb 2020 ➔ Jul 2020

Clinical • Enrollment change • Trial completion date

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) (clinicaltrials.gov) - P2; N=150; Enrolling by invitation; Sponsor: Acceleron Pharma, Inc.; Recruiting ➔ Enrolling by invitation

Clinical • Enrollment status

"Acceleron to Discontinue P-II Study of ACE-083 for Facioscapulohumeral Muscular Dystrophy @AcceleronPharma https://t.co/Z7ygazsLfP" (@Pharmashot)

"$XLRN @AcceleronPharma has a pipeline drug ACE-083 targeting CMT https://t.co/K0tB2HNPMi" (@Biotech2050)

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) (clinicaltrials.gov) - P2; N=150; Recruiting; Sponsor: Acceleron Pharma, Inc.

Clinical • New P2 trial

"$XLRN Receives FDA Orphan Drug Designation for ACE-083 in Charcot-Marie-Tooth Disease" (@BioStocks)

Orphan drug