In an April 30 research note, analyst Joseph Pantginis reported that H.C. Wainwright & Co. initiated coverage on Sio Gene Therapies Inc. (SIOX:NASDAQ) with a Buy rating and an $8 per share target price. In comparison, its current share price is about $2.70.
Pantginis described the pharmaceutical company and its pipeline of potential therapeutics.
Sio Gene develops lentivirus- and adenoassociated virus-based therapies, the purpose of which is to permanently modify genes involved in various neurologic diseases, including Parkinson's in adults and GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff) in pediatric patients, the analyst relayed.
The company's most advanced gene therapy is AXO-Lenti-PD, designed to "provide a durable solution to neurocognitive decline for those diagnosed with Parkinson's disease," Pantginis indicated. He highlighted that AXO-Lenti-PD could meet the need for a new therapy and that its potential is blockbuster in scope. With 1 million people in the U.S. having Parkinson's disease and another 60,000 people getting diagnosed with it each year, the market size is big.
Also, Pantginis pointed out, AXO-Lenti-PD has three clear advantages over other treatments, because it is a viral vector-based gene therapy. One, only a single dose should be needed to restore gene function. Two, the therapy provides a permanent solution as the resulting gene expression is stable and durable. Three, because lentiviral vectors are not new, the regulatory risk surrounding therapies based on them is greatly reduced. Thus, advancement through clinical trials should be efficient and quick.
The available AXO-Lenti-PD data are positive, Pantginis added. Results from the SUNRISE-PD, Phase 2 dose escalation study in Parkinson's disease patients showed the therapeutic to be safe and well tolerated. Also, patients reported significant benefit from AXO-Lenti-PD, even at suboptimal doses.
"These results are indicative of AXO-Lenti-PD's differentiated capacity to treat and stabilize Parkinson's disease symptoms, especially compared to L-DOPA supplementation," the current standard of care, Pantginis wrote.
He discussed two other candidates in Sio Gene's pipeline: AXO-AAV-GM1 and AXOAAV-GM2. The company intends for these adenoassociated virus-based gene therapies to prevent neurologic decline in pediatric patients, from infants to juveniles. Currently, no such treatments exist.
"This, coupled with the growing incidence of Parkinson's disease in aging populations globally, not only establishes a compelling clinical justification but outlines a clear target market population with substantial commercial opportunity that provides what we think is a meaningful advantage over current and future competitors in the space," wrote Pantginis.
As for clinical progress, AXO-AAV-GM1 is currently being tested, in a two-stage, Phase 1/2 study, on both types 1 and 2 GM1 gangliosidosis. In the second stage, efficacy of the dose determined in Phase 1 will be measured. The next set of results, an update on patients getting the low dose along with results of a biomarker analysis, is anticipated by mid-2021. Full, 12-month topline data are expected by year-end 2021.
Regarding AXO-AAV-GM2, the U.S. Food and Drug Administration granted it two designations, rare pediatric disease and orphan drug, in October 2020. Sio Gene has started an open-label, two-stage, Phase 1/2 trial of the therapy, which it is conducting in a manner similar to that for AXO-AAV-GM1. Already one patient has been treated.
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