Axovant Sciences Ltd. (NASDAQ: AXGT) has produced results from two separate studies, although they both have ties to neurodegenerative diseases, and its shares made a massive gain to start off the week.
Even excluding Monday’s move, Axovant shares had absolutely exploded in 2019, with the stock up nearly 50% year to date. While investors are definitely interested in the stock’s movement, these two neurodegenerative studies are painting the picture for a bright future at Axovant.
The first trial was AAV-GM2 gene therapy in a patient with advanced infantile Tay-Sachs disease, a rare and fatal pediatric neurodegenerative genetic disorder characterized by impaired β-Hexosaminidase A enzyme production.
In this study, AXO-AAV-GM2 was generally well-tolerated and no serious adverse events had been reported as of the three-month visit. At three months, no clinically relevant laboratory abnormalities were observed following AXO-AAV-GM2 administration.
Dr. Gavin Corcoran, executive vice president of research and development at Axovant, commented:
This is the first time a gene therapy has been administered to a child with Tay-Sachs disease, and it is remarkable that we have not only seen good safety and tolerability to date, but also evidence of functional β-Hexosaminidase A enzyme activity. These encouraging early clinical results suggest that AXO-AAV-GM2 may offer a meaningful treatment option for patients who currently have no approved therapies.
Separately, Axovant reported three-month data from its the ongoing Sunrise-PD Phase 2 trial of AXO-Lenti-PD for the treatment of Parkinson’s disease. The cohort consisted of two patients with advanced Parkinson’s disease who received a one-time administration of the lowest dose of AXO-Lenti-PD. AXO-Lenti-PD was generally well-tolerated, and no serious adverse events were reported
Patients in the first cohort experienced an average Unified Parkinson’s Disease Rating Scale (UPDRS) Part III (motor) OFF score improvement of 25 points at three months after administration of AXO-Lenti-PD, representing an average improvement of 42% from baseline.
Overall, improvements were seen across all subparts of the UPDRS scale, with an average UPDRS Total OFF score improvement of 54.5 points at three months after receiving AXO-Lenti-PD, representing an average improvement of 55% from baseline.
Shares of Axovant were last seen up about 47% at $2.18, with a 52-week range of $1.15 to $2.43. The stock has a consensus analyst price target of $5.00.