MeiraGTx (NASDAQ:MGTX) is advancing a portfolio of gene therapy programs across ophthalmology and other large disease areas, President and CEO Alexandria “Zandy” Forbes said during an RBC Capital Markets ophthalmology conference discussion with biotech analyst Lisa Walter.
Forbes described the company as having “three late-stage programs,” including two in inherited retinal diseases (IRDs) and one outside ophthalmology. She added that two additional late-stage programs are in development outside the eye: one for radiation-induced xerostomia and one for Parkinson’s disease. Across its portfolio, Forbes emphasized MeiraGTx’s approach of using small, locally delivered doses of AAV vectors rather than large intravenous doses, which she said impacts safety and cost of goods.
Ophthalmology focus: IRDs and why gene therapy fits
She also characterized the IRD clinical ecosystem as concentrated, saying there are roughly “30 to 50 leading sites” globally where key opinion leaders treat the majority of known patients. MeiraGTx’s presence in the space, she said, was built on a partnership with University College London and Moorfields Eye Hospital, which she described as having one of the largest IRD patient catchments. Forbes said these relationships help with identifying patients, genotyping, and selecting endpoints that show whether therapies are working.
XLRP (RPGR) partnered with Johnson & Johnson
Discussing the company’s X-linked retinitis pigmentosa (XLRP) program partnered with Johnson & Johnson, Forbes said the program is owned by J&J and that MeiraGTx serves as the commercial manufacturer. She referenced the pivotal dataset presented last year, calling it “extremely promising,” and said that investigators and key opinion leaders involved in the study viewed the therapy as having a positive impact on patients’ lives.
Forbes said MeiraGTx is awaiting Johnson & Johnson’s strategy “for getting that to patients.”
She also outlined operational learnings from the development work leading into the phase III trial, including advances in surgical technique, steroid regimens that reduced inflammation, and surgeon training. Forbes said the phase III effort spanned 30 sites in Europe and the U.S. and resulted in more than 60 surgeons being trained, with “virtually no surgical issues” and no major safety issues highlighted in her remarks. She said the experience is broadly applicable to future IRD programs.
LCA4 (AIPL1) partnered with Eli Lilly
Forbes described MeiraGTx’s LCA4 program as targeting a mutation in AIPL1, which she characterized as an “ultra-rare” cause of Leber congenital amaurosis in which infants are born blind. She said that under a U.K. “specials” mechanism, 11 children under the age of four were treated with an AAV vector intended to replace the AIPL1 gene and that “all 11 gained sight.”
According to Forbes, Eli Lilly licensed the program to commercialize it globally. She said the deal included $75 million upfront plus additional milestones totaling $135 million, tied to global approvals and “largely U.S. approval and a PRV.” She added that the priority review voucher (PRV) timeline has been extended, which she framed as lowering risk tied to one milestone, and said MeiraGTx is working closely with Lilly as it prepares regulatory filings worldwide.
BBS10 and the U.K. “specials” pathway
Forbes said MeiraGTx’s U.K.-based manufacturing operations include a “specials license,” which she described as allowing the company to manufacture and release product for specific patients at a physician’s request when no other options exist, even if the therapy has not been used in humans before. She said the BBS10 program originated through a patient advocacy effort in which parents supported the company in designing, manufacturing, and supplying a BBS10 viral vector for physician use in the U.K.
She noted there is not an FDA equivalent to the U.K. specials license, though she said mechanisms exist in the U.S. to treat patients who are not eligible for clinical studies. Forbes also said that in the AIPL1 experience, treated children came from seven different countries, explaining that patients may travel to the U.K. for treatment as physicians request material for use there. On whether specials-generated data can support regulatory filings, Forbes said it can be used if collected and verified in a way acceptable to regulators.
Broader pipeline, technology platform, and non-ophthalmology programs
Beyond the partnered IRD programs, Forbes said MeiraGTx has additional inherited retinal disease candidates moving toward IND, and that the Lilly collaboration also included two other IRD gene therapies—one “ready for IND” with documents being transferred, and another in pre-IND. She said Lilly also obtained rights to use MeiraGTx intravitreal capsids and promoters for five undisclosed Lilly targets, enabled by a large promoter library and work using retinal organoids and AI to develop cell-specific promoters.
On intravitreal delivery, Forbes said MeiraGTx evaluated capsids in non-human primates and human organoids and compared them head-to-head against AAV.7m8, which she called a “gold standard.” She said the company selected capsids that appeared superior in those assays and could be used in larger ophthalmic indications such as wet AMD, dry AMD, and glaucoma, noting separate work on glaucoma-focused targeting.
In non-ophthalmology, Forbes highlighted MeiraGTx’s xerostomia program for severe, long-term radiation-induced dry mouth persisting two years post-treatment. She said the company’s market research suggests a “multi-billion-dollar” opportunity with strong coverage and pricing. Forbes said the ongoing study is a pivotal phase II that the FDA has agreed in writing could support a BLA as a single study, that enrollment is nearing completion, and that the company is targeting a filing “in this quarter next year,” aiming for approval around the end of next year and a 2028 launch.
Forbes also discussed the company’s riboswitch platform, describing it as a way to deliver proteins or peptides through a one-time delivery of a DNA template combined with daily oral small-molecule inducers. She said the company is communicating with the FDA to prepare for an IND filing later this year for a leptin program and noted it has three GMP-manufactured small molecules ready for clinical use, with one intended for leptin. She also pointed to “increasingly strong” preclinical data in neuropathic pain and suggested the platform could support dosing of peptides, proteins, and hormones that are otherwise difficult to deliver effectively.
About MeiraGTx (NASDAQ:MGTX)
MeiraGTx Holdings plc is a clinical-stage biotechnology company dedicated to developing gene therapies for the treatment of rare diseases. Founded in 2014 as an outgrowth of research at University College London, the company focuses on leveraging adeno-associated virus (AAV) vectors to deliver functional genes to target tissues. MeiraGTx’s pipeline spans ocular, central nervous system and systemic indications, addressing conditions such as inherited retinal dystrophies and neurodegenerative disorders that currently lack effective therapies.
The company’s lead programs include AAV-based candidates designed to restore or replace defective genes underlying rare retinal diseases and to modulate cellular pathways in neurological disorders.
