MDA clinical and scientific conference: organized online from March 15 to 18, 2021

19 April 2021

This year, the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference brought together more than 1,200 participants and 100 speakers “online” from March 15 to 18, 2021. Connected to a virtual platform, everyone could follow videoconferences or watch e-posters on research and development, treatment of neuromuscular diseases, the impact of Covid-19 on neuromuscular diseases. Several clinical trial results were presented. 

 

In SMA

  • For Zolgensma® (onasemnogen abeparvovec): adapted motor development (sitting, standing, walking in particular) in infants treated presymptomatically, a real benefit in older children as well as a lasting effect over the long term (more 5 years) after treatment. 
  • For Evrysdi™ (risdiplam): improvement or stabilization of motor function in children and adults with SMA type II and III after two years on Evrysdi (SUNFISH trial).

 

In DMD

  • For vamorolone (Santhera – ReveraGen), a recent anti-inflammatory: confirmation of its ability to slow the decline in motor function after 2.5 years of treatment; 
  • For ataluren (PTC Therapeutics) administered for 4.5 years: a slowdown in loss of walking for 2.5 years and a reduced impairment of breathing capacity.
  • Regarding innovative therapies, results focused on 3 antisense oligonucleotides (Sarepta Therapeutics): eteplirsen (exon 51 skipping) given over the long term would preserve walking while casimersen (exon 45 skipping) is relatively well tolerated. As for SRP-5051 (exon 51 skipping) already tested in a clinical trial, dose-dependent efficacy in mice has been shown, confirming the value of the protocol set up for the clinical trial.
  • Finally, scAAV.U7.ACCA (Audentes Therapeutics), an exon skipping molecule targeting DMD gene exon 2 duplications, in a single administration in 2 patients, is well tolerated after 3 months and allows the production of a modest level of full-length dystrophin and a decrease in CKemia.
  • In gene therapy, the SGT-001 trial  (Solid Biosciences) (AAV9-micro-dystrophin) has resumed (IGNITE-DMD): the first results on the first patient treated with the new formulation of the product show good tolerance and dystrophin production.

 

In LGMD R4 linked to β-sarcoglycan (ex-LGMD2E) 

  • For the SRP-9003 gene therapy product providing the β-sarcoglycan gene: a functional improvement is observed two years after the injection for the three patients who received a low dose and one year after the injection for the three who received (later) a high dose. This improvement is associated with a intensive expression of β-sarcoglycan in the muscles. 

 

New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children and durability 5+ years post-treatment. 

Novartis, Communiqué de presse du 15 mars 2021.

 

Results from the Second Year of Evrysdi™ (risdiplam) Treatment Demonstrated Sustained Improvement of Motor Function in a Broad Range of SMA Patients. 

PTC Therapeutics, Communiqué de presse du 16 mars 2021.

 

New two-year data show Roche’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA).

Roche, Communiqué de presse du 16 mars 2021.

 

Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference

Sarepta Therapeutics, Communiqué de presse du 15 mars 2021.

 

Solid Biosciences Reports Efficacy and Safety Data from the Ongoing IGNITE DMD Clinical Trial and Resumption of Patient Dosing in the 2E14 vg/kg Cohort 

Solid Biosciences, Communiqué de presse du 15 mars 2021. 

 

Sarepta Therapeutics’ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration. 

Sarepta Therapeutics, Communiqué de presse du 18 mars 2021.