William Blair & Company initiated research coverage of Prevail Therapeutics Inc. (PRVL $11.90), a clinical-stage company developing adeno-associated virus (AAV) gene therapies for neurodegenerative disorders of the central nervous system.
Analyst Myles Minter estimated peak sales could reach $1.1 billion in the United States alone for lead asset PR001 as the first potential disease-modifying therapy for Parkinson's disease patients who harbor a GBA1 mutation (PD-GBA). Prevail is also developing PR001 for neuronopathic Gaucher disease (nGD), and a second asset, PR006, in frontotemporal dementia linked to a GRN mutation (FTD-GRN).
"Prevail's gene therapy portfolio uses the AAV9 capsid to deliver transgenes to CNS tissue via intra-cisterna magna injection," Minter said. "This allows it to access deep brain structures without an invasive intraparenchymal injection. PR001 is being evaluated in the Phase I/II PROPEL trial in PD-GBA patients and the Phase I/II PROVIDE trial in type 2 nGD patients. In August, Prevail announced initial clinical data from a patient treated with the low PR001 dose in PROPEL. At three months post-administration, GCase enzyme activity went from undetectable levels at baseline to normal levels, which we view as exceeding the goal of a 20%-30% increase in activity. However, three months after PR001 administration, this patient experienced serious adverse events presumed to be related to adaptive immunity against the AAV9 vector. In response, Prevail is amending the treatment regimen to include sirolimus and reduce glucocorticoid exposure to increase tolerability and compliance. Additional patient enrollment in PROPEL and enrollment of type 2 nGD patients in PROVIDE will begin in the second half of 2020 under the amended regimen."
Minter continued, "Early-stage clinical development programs in gene therapy are not without significant risks and Prevail is not immune to these; however, we believe the rationale for clinical development of PR001 and PR006 is strong for its respective genetically defined CNS disorders and despite an early safety concern, preliminary clinical data looks impressive, in our view."
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