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Promising results from Lysogene MPS IIIA trial; novel PROVide study collecting observations

May 25, 2022

Lysogene announced May 18 that its AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA was showing “promising efficacy data in young patients with persistent increase or stabilization.”

The clinical trial update was shared this month during 25th Annual Meeting of the ASGCT (American Society for Gene & Cell Therapy) in Washington, DC.

Lysogene shared that:

“Patients with Sanfilippo syndrome type A (MPS IIIA) [in the AAVance clinical trial evaluating LYS-SAF302] who have been followed for at least two years demonstrated improvement, stabilization, or slowing down of decline in cognitive developmental age (DA) in about half of the patients. Notably, in all 6 patients enrolled under the age of 30 months, a persistent increase or stabilization of the cognitive, language and motor domains of the BSID-III was observed in the 24 months period post-treatment.”

Also promising, the update stated that:

“Two (2) of these 6 patients, including 1 patient over 4 years of age, have already exceeded by several months the highest cognitive DA (35 months) observed in the natural history cohort. The 2 patients reached 41 and 42 months of cognitive DA in the 24 months period post-treatment with an increase of 25 and 17 months compared to their baseline, respectively.”

“These results, which suggest that younger patients are more responsive to the gene therapy treatment, need to be corroborated and confirmed by the final statistical analyses,” noted Lysogene in a May 18 press release.

The final statistical analysis will also include secondary behavioral and imaging endpoints, as well as data from the observational study using video and parent interviewing, also called the Patient Reported Outcome Videos (PROVide) study.

Cure Sanfilippo Foundation worked with Sarepta Therapeutics, Lysogene, Aparito, and Casimir Trials to create the PROVide study as an innovative approach to recording and measuring disease post gene therapy.

The study uses a home-based method for capturing outcomes for MPS IIIA, as a means to monitor the onset and evolution of disease hallmarks remotely and in a non-invasive way that is suitable for children. Defined video captures were chosen based on identified gaps in clinic-based measures and personalized features identified by caregivers.

Caregivers of patients enrolled in the AAVance trial collect video data of patients doing specific daily-life activities at baseline and follow-up timepoints throughout the clinical trial, post-treatment, through a secure smart phone mobile application.

Video assessments focus on several hallmarks of MPS IIIA progression, including inability to self-care, lack of engagement with environment, progressively abnormal gait, social withdrawal, lack or loss of communication or speech, hyperactivity, disrupted sleep, and evidence of distress without apparent cause.

In addition to standardized activities, caregivers record videos of patients’ newly-acquired abilities or abilities which have significantly changed in quality throughout the study. Caregivers participate in qualitative interviews to provide context for videos and discuss any changes observed during specified intervals.

Activity videos are reviewed and scored by expert clinicians using both Clinical Global Impression of Severity (CGI-S) and Clinical Global Impression of Change (CGI-C) scales at the completion of the study.

“It has been an incredible experience breaking new ground and working with the team to develop the PROVide companion video study,” said Cure Sanfilippo Foundation Chief Science Officer Cara O’Neill, MD, FAAP. “Huge thanks to Karen Aiach, Founder and CEO of Lysogene, for making patient-centered outcome measures a priority.”

In May 2020, Aparito presented a poster on the PROVide study at the 10th European Conference on Rare Diseases.

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Full results of the Lysogene trial are expected to be available in third quarter 2022.

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