2023-07-03 16:38:07
American Biogen has presented new data on nusinersen to address the growing needs of the community of people with spinal muscular atrophy (SMA). The findings were released at the SMA Research & Clinical Care Congress, hosted by Cure SMA this week in Orlando, Florida. The event “presents a unique opportunity to connect and hear the voices of healthcare professionals, patients and caregivers, sharing research findings that address the unmet needs of the Sma community,” said Maha Radhakrishnan, Chief Biogen Medical Officer – We are pleased to present new data, including preliminary results from our Respond study evaluating the clinical benefit and safety of nusinersen treatment after gene therapy.”
Respond is a two-year, open-label, Phase IV study to evaluate the clinical outcomes and safety of nusinersen treatment in infants and young children with SMA who have an unmet clinical need following Biogen in a statement. of treatment with gene therapy. From the intermediate efficacy results at 6 months on 29 participants treated with nusinersen, it emerges that in the majority of participants there are improvements in motor function measured by the increase in the mean total score on the Hammersmith Infant Neurological Examination Section 2 (Hine-2) scale compared at baseline; patients with two copies of the Smn2 gene (24) improved by more than 5 points (mean) on the Hine-2 scale, while patients with three copies of the Smn2 gene (3) had improvements, but no change was calculated mean from baseline considering small sample size; the majority of patients (25 of 27) with suboptimal motor function (assessed by the investigator at baseline) improved.
After a median of 230.5 days in the study serious adverse events were reported in 13 of 38 participants (34%). No correlation was found between serious adverse events and nusinersen, nor did these events lead to withdrawal from the study. No new safety concerns were identified in participants enrolled and treated with nusinersen after onasemnogene abeparvovec.
“What we are realizing is that gene therapy may fail to treat all motor neurons, making disease progression possible,” said Crystal Proud, a pediatric neurologist at Children’s Hospital of the King’s Daughters. Remaining unmet clinical needs in some SMA patients treated with onasemnogene abeparvovec, whose clinical effects have not met expectations.These preliminary results provide the community with the first data from clinical trials evaluating nusinersen treatment after onasemnogene abeparvovec and suggest the possibility of obtaining additional benefits from nusinersen therapy”.
Nusinersen – recalls the note – is approved in more than 60 countries for the treatment of infants, children and adults suffering from spinal muscular atrophy. It is an antisense oligonucleotide (antisense oligonucleotide or Aso) that acts on the main cause of Sma by continuously increasing the amount of Smn (survival motor neuron) protein produced in the body. The drug is delivered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease originates.
A systematic review of the literature was presented – highlights Biogen – with a meta-analysis evaluating the real-world impact of nusinersen in infantile-onset Sma, from which emerges the importance of generating real-world evidence to arrive at a complete evaluation of the benefits of treatment with nusinersen. The improvements in motor function and motor milestone acquisition observed in real-world studies were greater than or comparable to the improvements observed in clinical studies; patients continued to improve with continued nusinersen therapy. Biogen is collaborating with Alcyone Therapeutics to develop the first implantable device designed to provide subcutaneous access for the delivery of antisense oligonucleotide-based therapies. This week Alcyone announced that the US regulatory body FDA has granted the Investigational Device Exemption (IDE) to start a pivotal study on the ThecaFlex system. During the summer, Alcyone expects to begin enrollment procedures for the Pierre study (Clinicaltrials.gov), which will evaluate the safety and performance of ThecaFlex in the administration of nusinersen in patients with Sma.
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