MeiraGTx reports Q3 EPS (62c) vs. (55c) last year

Reports Q3 revenue $410,000 vs. $10.9M last year. “Following the close of the third quarter, we are very pleased to have signed a strategic collaboration in ophthalmology with Eli Lilly, led by our AAV-AIPL1 program for the treatment of LCA4, one of the most severe forms of inherited retinopathies,” said Alexandria Forbes, CEO. “The unprecedented data from the 11 LCA4 children born blind who all gained vision after treatment with AAV-AIPL1 illustrates the power of gene therapy in the eye to cure even the most severe genetic defects, particularly when treatment takes place at a very young age. In addition to AAV-AIPL1, Lilly has gained rights to two preclinical ocular programs as well as our intravitreal capsids, bespoke promoters generated from our AI driven promoter platform, and certain rights to our riboswitch platform in the eye. We are excited to be entering into this collaboration with Lilly and view this as a testament to the power of our broad toolkit of proprietary gene therapy technologies for both rare and prevalent ocular disease. We are particularly pleased that Lilly, a global leader in development and commercialization of innovative medicines, has chosen to partner with us in this area of high unmet need, and shares our dedication to bringing truly life changing therapies to patients with otherwise intractable conditions. During the quarter we continued to make progress in our late-stage clinical programs. Our pivotal Phase 2 study of AAV-hAQP1 for the treatment of grade 2 or 3 radiation-induced xerostomia remains on track to achieve target enrollment at the end of this year, and we expect to have data which if positive may lead to a BLA for AAV-hAQP1 in early 2027, with potential approval later in 2027. We also anticipate initiating a Phase 3 study evaluating AAV-GAD for the treatment of Parkinson’s disease in the coming months and are currently in discussion with sites globally for inclusion in the Phase 3 study. This will be our third double-blind, sham-controlled clinical study of AAV-GAD where we aim to demonstrate significant improvement in UPDRS as well as the potential disease-modifying effect of AAV-GAD treatment on the physiological circuitry controlling movement as well as a potentially protective effect on the substantia nigra. In addition,we have completed optimization of our lead riboswitch program for entry into the clinic, for the delivery of native human leptin controlled by a daily oral small molecule to treat inherited and acquired leptin deficiency. This is an important unmet need due to the immunogenicity of the only available current treatment, metreleptin, a synthetic injectable leptin analog which can result in neutralizing antibody production with catastrophic or even lethal metabolic consequences. We have now demonstrated complete durability of both leptin production dynamics and efficacy of our riboswitch-controlled leptin over more than a year of oral dosing in the mouse model. We have manufactured GMP small molecule inducer for the clinic and we are engaging the FDA in IND enabling discussions. And finally, we have successfully developed, manufactured and provided material for our second Specials program in the UK for the treatment of BBS10-associated retinal dystrophy. Like AAV-AIPL1, this program is in collaboration with a BBS10 philanthropic organization. One child has been treated so far and others may be treated if they are found to be eligible by the treating physician. The BBS10 program has been awarded Rare Pediatric Disease Designation by the FDA, making it potentially eligible for a Priority Review Voucher on approval.”