Smaller gene therapy biotechs are taking the spotlight given the M&A interest surrounding them. One such gene therapy company saw its shares rallying by more than 30 percent Monday following the release of clinical trial results for two of its gene therapies.
Axovant Sciences Ltd (NASDAQ: AXGT) announced three-month data from an investigator-initiated study of its investigational gene therapy AXO-AAV-GM2 in a patient with advanced infantile Tay-Sachs disease, which is a fatal pediatric neurodegenerative genetic disorder characterized by impaired β-Hexosaminidase A enzyme production.
The gene therapy acts by restoring the beta-hexosaminidase A enzyme activity in the central nervous system.
The study that evaluated a dose of 1.0x 1014 vg of AXO-AAV-GM2 in a 30-month-old child with advanced infantile Tay-Sachs disease showed that at three months, there was an apparent increase in enzyme activity in the cerebrospinal fluid to 1.44 percent of normal from 0.46 percent of normal at the baseline.
The increase surpassed the 0.5-percent threshold that could represent a clinically important effect, the company said.
AXO-AAV-GM2 was generally well-tolerated, with no serious adverse events reported as of the three-month visit.
Separately, the company released positive interim results from the first cohort of the dose-escalation portion of an ongoing Phase 2 study dubbed SUNRISE-PD of its AXO-Lenti-PD gene therapy that is being evaluated in Parkinson's disease patients.
The cohort, consisting of two patients who received a one-time administration of the lowest dose of AXO-Lenti-PD, showed it was well-tolerated. On the efficacy front, the company said the two patients showed an average improvement of 25 points at three months in the Physician-rated Unified Parkinson's Disease Rating Scale Part III score, which ranges from zero to 108, with lower scores indicating improvement.
The results suggest that the lowest dose of AXO-Lenti-PD at three months may have greater efficacy compared to the highest dose of ProSavin, a predecessor to AXO-Lenti-PD, according to Axovant.
Axovant shares were trading up 32.33 percent at $1.96 at the time of publication Monday.
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