New data at Cure SMA highlight potential benefit of Spinraza in infants and toddlers with unmet clinical needs after gene therapy for spinal muscular atrophy.- Biogen

Interim Clinical Outcomes from RESPOND : RESPOND is an ongoing two-year, phase IV open-label study to evaluate clinical outcomes and safety following treatment with Spinraza in infants and toddlers with SMA who have unmet clinical needs after treatment with Zolgensma (onasemnogene abeparvovec). Interim efficacy results at six months from 29 study participants treated with Spinraza show: i. Improvements in motor function in most participants as measured by increased mean total Hammersmith Infant Neurological Examination Section 2 (HINE-2) score from baseline. a. Participants with two SMN2 copies (n=24) improved by a mean of over 5 points on HINE-2. b. All participants with three SMN2 copies (n=3) improved; a mean change from baseline was not calculated due to the small number of participants. ii. Most participants (25/27) with investigator-reported suboptimal motor function at baseline improved.

After a median of 230.5 days in the study, serious adverse events (AEs) were reported in 13/38 (34%) participants. No serious AEs were considered related to Spinraza or led to study withdrawal. No new emerging safety concerns have been identified in enrolled participants who received Spinraza after Zolgensma. Additional interim clinical outcomes from the RESPOND study are being presented at the conference.

"We are learning that gene therapy may not be treating all motor neurons leaving the potential for disease progression,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters. “The RESPOND study has begun to characterize remaining unmet need in some SMA patients treated with Zolgensma whose outcomes have not met clinical expectations. These interim results provide the community with the first clinical study data evaluating Spinraza treatment following Zolgensma and suggest there may be potential for additional benefit with Spinraza treatment.”

New Analysis Evaluating Real-World Impact of Spinraza : A systematic literature review and meta-analysis evaluating real-world impact of Spinraza for infantile-onset SMA was presented and highlights the importance of generating real-world evidence to achieve a comprehensive understanding of the treatment benefits of Spinraza. Improvements in motor function and motor milestones observed in real-world studies were greater than or comparable to those observed in clinical trials, and patients continued to improve with longer duration of Spinraza treatment.

Progress on Novel Device Aimed at Enhancing Treatment Experience : Together with Alcyone Therapeutics, Biogen is working to develop the first implantable device designed to enable routine subcutaneous access for delivery of antisense oligonucleotide therapies. This week, Alcyone announced that the FDA has approved an Investigational Device Exemption to initiate a pivotal trial of the ThecaFlex DRx System (ThecaFlex). This summer, Alcyone plans to begin initial enrollment of the PIERRE study (clinicaltrials.gov), which will evaluate the safety and performance of ThecaFlex for the delivery of Spinraza in SMA patients.