Axovant still has a lot to prove despite positive data for PD gene therapy, says GlobalData

Following the news that Axovant announced positive six-month follow-up data from its Phase II trial of AXO-Lenti-PD for the treatment of Parkinson’s Disease (PD);

Philippa Salter, Neurology Analyst at GlobalData, a leading data and analytics company, offers her view:

“Showcasing an improvement in the Unified PD Rating Scale (UPDRS) Part III ‘OFF’ score from baseline is an important demonstration for AXO-Lenti-PD with its aims to impact the PD market because dyskinesia and OFF episodes remain an ongoing challenge in PD. In fact, according to key opinion leaders (KOLs) interviewed by GlobalData*, over 60% of PD patients will eventually have OFF periods. However, while AXO-Lenti-PD’s positive results show that there is huge potential for this gene therapy, there is a need for caution as these results have only come from two patients.

“Perhaps more notable than AXO-Lenti-PD’s positive results is the omission of reported results from another two patients in the cohort as a result of COVID-19 and a patient refusal. This raises concerns over what else might have been omitted, reflected in Axovant’s decrease in shares as a result of this news. In its EXPLORE-PD trial next year, Axovant will need to show that AXO-Lenti-PD can demonstrate positive results in multiple patients if it is to strengthen its hand for approval and favor with physicians.

“Axovant will have to compete with the other strategies being employed to improve the treatment for dyskinesia and OFF episodes in PD – for example, Sunovion Pharmaceuticals’ novel sublingual film Kynmobi, which was approved in May 2020. Any gene therapy will need to demonstrate significant efficacy in order to compete with an easy-to-use sublingual therapy – largely to show cost-effectiveness. AXO-Lenti-PD effectively needs to prove it is worth what is likely to be a hefty price tag.

“The lack of disease-modifying drugs is one of the highest unmet needs in PD treatment. As gene therapies offer the opportunity for a one-time treatment, if one were to be approved it could transform treatment for patients. As such, it is not surprising that there are other companies also pursuing this strategy, and, according to GlobalData’s proprietary drugs database, there are 25 gene therapies currently in development for PD. None of these drugs are beyond Phase II development, so there is still a long way to go before the possibility of a transformative gene therapy being available for PD patients.”

* KOLs interviewed as part of GlobalData’s report: GlobalData (2018) Parkinson’s disease: Global Drug Forecast and Market Analysis to 2026, May 2018, GDHC167PIDR

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