Shares of micro-cap gene therapy company Applied Genetic Technologies Corp (NASDAQ: AGTC) were advancing to their highest level in more than a year Thursday after the company released a positive midstage readout for its gene therapy to treat an inherited eye disorder.
The Applied Genetic Study Results
The Cambridge, Massachusetts-based company said six-month data from an ongoing Phase 1/2 study that is evaluating its AAV-based gene therapy in X-linked retinitis pigmentosa (XLRP) showed the product candidate produced durable improvement in visual function six months after dosing.
XLRP is an inherited condition causing progressive vision loss in boys and young men that manifests as night blindness in early childhood and progressive constriction of the visual field. It is caused by the mutation of the RPGR gene.
At the six-month mark, eight patients were evaluable out of the nine who were centrally dosed, and four of the eight showed measurable improvements in visual activity.
All nine patients who were treated centrally had stable or improving visual acuity, with 78% seeing a five letter or more improvement, the COMPANY said.
Applied Genetic also noted that patients who improved in either visual sensitivity or best corrected visual acuity, also reported noticeable improvement in visual function, including greater clarity and reduced night blindness.
The data, along with the promising safety and efficacy data reported in September, will help design the pivotal trial that Applied Genetic plans to initiate by the end of this year, the company said.
Applied Genetic CEO: Drug Candidate Has 'Tremendous Potential'
XLRP has no treatment options. Applied Genetic has been accorded an orphan drug designation for the experimental therapy by the FDA and given an orphan medicinal product designation by the European Commission.
"These promising results further demonstrate that our XLRP candidate has tremendous potential to provide meaningful benefit to XLRP patients who today have no treatment options," Applied Genetic CEO Sue Washer said in a statement.
The positive results reported so far will support the advancement of the product candidate in the pivotal trial by the end of 2020, the company said. Applied Genetic intends to enroll additional patients during the first quarter.
Applied Genetic Price Action
The stock was trading 54.33% higher at $6.42 at the time of publication Thursday.