- Shares of Axovant Sciences rocketed up Monday morning after the biotech reported interim data that the company pitched as promising for two of its investigational gene therapies.
- Three months after receiving the lowest dose of Axovant's AXO-Lenti-PD gene therapy, two advanced Parkinson's disease patients showed an average improvement of 42% from baseline on a physician-rated scale assessing motor function. Those patients, enrolled in the mid-stage SUNRISE-PD study, also have yet to report any serious adverse events, according to Axovant.
- A separate study is evaluating the ability of another Axovant therapy to treat advanced infantile Tay-Sachs disease, a rare and fatal neurodegenerative disorder where patients don't produce enough of a key enzyme involved in brain and spinal cord function. The one toddler treated with AXO-AAV-GM2 has experienced increased enzyme activity in the cerebrospinal fluid — from 0.46% of normal at baseline to 1.44% of normal three months following administration.
Despite immense investments and rigorous R&D programs, the world's most powerful drug companies haven't been able to successfully treat the underlying causes of neurodegenerative diseases like Alzheimer's, Huntington's and Parkinson's.
In the wake of numerous clinical failures, some developers have turned to gene therapy as an approach for the most elusive targets in neuroscience. In the last few months alone, Voyager Therapeutics inked deals with AbbVie and Neurocrine Biosciences focused on creating a gene therapy for Parkinson's disease.
Axovant, which was forced to rebuild following a damaging clinical failure in Alzherimer's, has bet its future on gene therapy. Its programs in Parkinson's and Tay-Sachs are among its most advanced.
The biotech snagged rights to its lead candidate, AXO-Lenti-PD, from Oxford BioMedica in mid-2018. With early data from SUNRISE-PD now in hand, Axovant intends to move onto a planned second cohort that will test a larger dose of the therapy.
AXO-Lenti-PD is a successor to Oxford's Prosavin, a lentiviral gene therapy that is also under investigation as a Parkinson's disease treatment. Long-term data from a Phase 1/2 study found moderate improvements in motor behavior in the majority of patients who received Prosavin and could still be evaluated for up to five years of follow-up. Nearly 100 drug-related adverse events were reported in the trial, including at least three deemed severe.
So far, there haven't been any serious adverse events reported in SUNRISE-PD. Additionally, investigators saw improvements across all subparts of the Unified Parkinson’s Disease Rating Scale, with an average OFF score improvement of 54.5 points three months after receiving AXO-Lenti-PD.
"Taken together, these results suggest that the lowest dose of AXO-Lenti-PD at 3 months may have greater efficacy compared to the highest dose of ProSavin previously tested," Axovant said in a March 11 statement.
Jefferies analyst Michael Yee noted the data from SUNRISE-PD beat expectations and "looks at least as good already or better than other programs" in the Parkinson's disease gene therapy space.
AXO-AAV-GM2, meanwhile, appears to have a modest effect on beta-hexosaminidase A (HexA) enzyme activity. Still, Axovant argues that may be enough to provide health benefits to patients.
"As disease severity correlates with HexA activity, even restoration to 0.5% of normal could result in a clinically meaningful improvement and/or save the life of the baby," Yee wrote in a March 11 investor note.
Shares of Axovant traded at $2.12 apiece at market's open Monday, up more than 43% from Friday.