Acceleron is rewarded for failing fast


Acceleron is taking the admirable step of discontinuing its lead wholly owned asset, ACE-083, in facioscapulohumeral muscular dystrophy (FSHD) after mixed results from a phase II trial. Many biotechs might have continued given that the study met its primary efficacy endpoint, showing an increase in mean muscle volume; however, the trial did not hit functional secondary endpoints. Acceleron’s willingness to cut its losses sent its stock down only 1% this morning, and a look at the group’s upcoming catalysts shows that it has plenty more irons in the fire. Still, many of these events involve luspatercept, which is partnered with Celgene, with Acceleron only due minimal milestones, plus royalties. ACE-083 will also soon yield data in another neuromuscular disorder with a different pathophysiology, Charcot-Marie-Tooth disease; however, the sellside does not forecast any sales in this indication, according to EvaluatePharma. ACE-083 inhibits TGF-beta proteins such as myostatin with the aim of increasing muscle mass and strength; its failure could be bad news for similarly acting projects including Scholar Rock’s SRK-015 and Roche/Bristol-Myers Squibb’s RG6206. in April Acceleron discontinued another TGF-beta inhibitor, ACE-2494, which had been in phase I development for neuromuscular disorders.

Acceleron's upcoming catalysts
Project Indication Event Note 2024e indication sales ($m)
ACE-083  Facioscapulohumeral muscular dystrophy Phase II data (NCT02927080) Failed, discontinued 9
Luspatercept  Myelofibrosis Phase II data (NCT03194542) Data due H2 2019 -
Luspatercept  Transfusion-dependent beta-thalassaemia FDA approval decision Dec 4, 2019 340*
ACE-083  Charcot-Marie-Tooth Disease Phase II data (NCT03124459) Data due Q1 2020 -
Sotatercept Pulmonary arterial hypertension  Phase II data (Pulsar, NCT03496207) Data due Q1 2020 46
Luspatercept  Second-line MDS with ring sideroblasts FDA approval decision Apr 4, 2019 615**
Sotatercept Pulmonary arterial hypertension  Phase II data (Spectra, NCT03738150) Data due 2020 46
Luspatercept  Non-transfusion-dependent beta-thalassaemia Phase II data (Beyond, NCT03342404) Data due 2020 340*
Luspatercept  First-line MDS Phase III data (Commands, NCT03682536) Data due H2 2020 615**
*Overall beta-thalassaemia sales; **overall MDS sales. Source: EvaluatePharma,


Select myostatin inhibitors in clinical development
Project Company/ies Indication Trial details Primary completion
Phase II/III
RG6206 Roche/Bristol-Myers Squibb/Chugai Duchenne muscular dystrophy NCT03039686 Dec 2020
Phase II 
SRK-015 Scholar Rock Spinal muscular atrophy Topaz, NCT03921528 Jan 2020
Phase I
BIIB110 Biogen Spinal muscular atrophy ? -
Source: EvaluatePharma &

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