Lysogene, a biopharmaceutical company, announced at the end of May that enrollment in SAMOS (Sanfilippo A Multinational Observational Study) was completed.
This is extremely exciting news for Sanfilippo syndrome (MPS IIIA) patients, who currently have no viable treatment nor cure for their rare condition.
Apparently, the multinational enrollment of 23 children for this study was extremely quick, which further reflected the strong concern and overall unmet needs in the MPS III community.
Though Lysogene has already successfully completed a Phase 1/2 trial and five-year follow up study of MPSIIIA patients under gene therapy, the company cannot progress development further without a validated biomarker (measurable substance whose presence is indicative of the disease) that reflects disease progression and response to therapy.
Thus, this study is vital in order to provide Lysogene a “non-concurrent control group,” i.e. a necessary baseline so that clinical change in untreated MPSIIIA patients can be measured.
After SAMOS is completed, Lysogene can conduct the upcoming Phase 2/3 gene therapy trial, which is scheduled to begin in the first quarter of 2018. A healthy copy of the SGSH gene would be inserted in patients, allowing the body to start making the missing enzyme whose absence causes MPS IIIA.
Hopefully, this neurosurgical procedure would permanently reverse the abnormalities at the core of the condition.
Overall, Lysogene is hoping to make one-time, in vivo gene therapy treatment a reality for MPS IIIA patients, and now with the commencement of SAMOS, this reality could be just around the corner.
In the meantime, if you want to familiarize yourself more with MPS IIIA and its causes and symptoms, click here.