Applied Genetic Technologies Corp. (NASDAQ: AGTC) shares jumped by more than 50% on Thursday after the firm announced positive interim data from its midstage trial in X-linked retinitis pigmentosa (XLRP).
The study demonstrated that patients treated centrally with its product candidate demonstrated durable improvement in visual function six months after dosing.
The results reinforced the promising efficacy and safety results reported in September 2019 and will help to design the XLRP pivotal trial planned to be initiated by the end of 2020. The company also remains on track to report interim six-month data from the dose escalation cohorts of both of its ongoing trials in achromatopsia later this month.
At the six-month mark for the same nine centrally dosed patients:
- Measurable improvements were observed in visual sensitivity for four of the evaluable eight patients. These are the same four patients discussed in September and indicate encouraging signs of a durable biologic effect.
- All nine patients treated centrally also had stable or improving visual acuity, 78% saw a 5 letter or more improvement, a result not reported in other XLRP trials.
- Patients who improved in either visual sensitivity or BCVA also anecdotally report noticeable improvement in visual function including greater clarity and reduced night blindness.
Sue Washer, president and CEO of AGTC, commented:
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. The positive results observed to date give us confidence that the data as a whole will support advancement of our XLRP clinical program to a pivotal trial in 2020.
Shares were last seen up about 51% at $6.30, in a 52-week range of $2.52 to $7.12. The consensus price target is $12.42.
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