Cure SMA 2023 Conference report

A number of clinical trial results were announced at the Cure SMA Research & Clinical care conference, held in Orlando (USA) from 28 to 30 June 2023:

  • the RESPOND trial revealed additional benefits of Spinraza® at six months after treatment with Zolgensma® in 29 participants;
  • the FIREFISH trial confirmed the good tolerability and efficacy of Evrysdi® over four years in type I SMA;
  • the TOPAZ trial with apitegromab demonstrated an improvement in motor function over three years. Other measures reported by non-ambulant SMA patients on fatigue or activities of daily living are improved.

At the same time, Alcyone Therapeutics announced that it had obtained FDA approval for the start of the one-year PIERRE trial, which will evaluate a subcutaneous implantable device, similar to a port-a-cath with an intrathecal catheter, as an alternative to repeated intrathecal injections.


New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy. Biogen. 30 June 2023 press release.


Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA® (nusinersen). Alcyone Therapeutics. 27 June 2023 press release.


Four-year follow up data for Roche’s Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk. Roche. 30 June 2023 press release.


New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy. Scholar Rock. 30 June 2023 press release.