AI Earnings Predictions for MeiraGTx Holdings plc (MGTX)

EX-99.1

2 mgtx-20260813xex99d1.htm

EX-99.1

Exhibit 99.1

MeiraGTx Reports Second Quarter 2026 Financial and Operational Results

-Received FDA Breakthrough Therapy Designation for AAV2-hAQP1 and reported positive three-year data from Phase 1 AQUAx clinical trial evaluating AAV2-hAQP1 for the treatment of moderate to severe grade 2/3 radiation-induced xerostomia

-Completed the acquisition of all interests and rights to botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP) from Johnson & Johnson* (J&J) for $25 million

-Strengthened balance sheet with $100 million equity financing concurrent with the bota-vec acquisition

-Secured up to $400 million strategic investment from Oberland Capital, with up to $375 million in non-dilutive capital to support development and commercialization of AAV2-hAQP1 and bota-vec

-Anticipate submission of global regulatory filings for approval of bota-vec in 2026 and potential BLA filing for AAV2-hAQP1 in mid-2027

LONDON and NEW YORK, August 13, 2026 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the second quarter ended June 30, 2026, and provided a corporate update.

“During the second quarter of 2026, we made tremendous progress towards transforming MeiraGTx into a commercial company,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We acquired bota-vec from J&J and started working on the global filings for this product and establishing the market access and commercial infrastructure ahead of the potential first commercial launch in 2027. In addition, we made meaningful progress with our wholly owned AAV2-hAQP1 program for a significant unmet need, radiation induced xerostomia (RIX). We were awarded Breakthrough Therapy Designation in March 2026 based on very strong 3-year data from our Phase 1 AQUAx clinical study (n=24), indicating strong durable responses in late-stage RIX patients. We completed enrollment in our pivotal Phase 2 AQUAx2 clinical study of AAV2-hAQP1 in RIX in the second quarter and are now working expeditiously to submit regulatory filings for bota-vec this year and preparing for AAV2-hAQP1 filings mid next year.”

Dr. Forbes continued, “We are engaging high quality market access and commercial teams as we build our internal infrastructure to expedite potential launches of these first in class disease modifying therapies. We are particularly excited to be joined by two senior leaders previously with J&J, Penny Fleck as our Chief Development Officer, who has two decades of experience leading research and development at Takeda Pharmaceuticals, Johnson & Johnson Innovative Medicine, and ONL Therapeutics, including running the bota-vec program at J&J, and more recently, John Knighton as Executive Vice President of Global Manufacturing and Supply Chain. John has over 30 years of experience in biologic manufacturing and previously served as the Head of Cell & Gene Therapy API at Janssen Pharmaceuticals, Inc., supporting the successful launch of CARVYKTI®. Together, Penny and John provide extensive experience and expertise in achieving marketing approvals and successful commercial launches of many pharmaceutical products, and will be instrumental as we transition the Company into one that is well prepared for the potential launches of two products over the next two years.”

Dr. Forbes added, “We also remain very excited about our Riboswitch platform. Following discussion with the FDA, we are finalizing the requirements for clinical development and progressing to first in human studies with our Ribo-Leptin program. We anticipate following this first clinical study of the Riboswitch technology with a second program in neuropathic pain which is supported by very strong animal data. In each case, a precise dose of the therapeutic gene product is produced in vivo based on an oral small molecule daily pill. We are eager to progress this powerful novel technology through clinical development to address conditions that cannot be readily addressed using any of the current therapeutic modalities available.”

*Janssen Pharmaceuticals, Inc., a Johnson & Johnson company

​

​

Second Quarter 2026 Highlights

Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-Linked Retinitis Pigmentosa (XLRP):

●In May 2026, MeiraGTx completed the acquisition of full rights and interests in bota-vec from J&J for a one-time $25 million upfront cash payment, with J&J eligible to receive a one-time regulatory and commercial milestone tied to U.S. approval and U.S. sales performance, plus potential mid-teens royalty on global net sales beginning in mid-2029.

●XLRP is a rare inherited retinal disease with early onset and progressive degeneration to complete blindness by the third decade of life, with no currently approved treatment options.

●There are more than 20,000 XLRP-RPGR

EX-99.1

2 mgtx-20260514xex99d1.htm

EX-99.1

Exhibit 99.1

MeiraGTx Reports First Quarter 2026 Financial and Operational Results

-Received FDA Breakthrough Therapy Designation for AAV2-hAQP1

-Reported positive three-year data from the Phase 1 AQUAx study of AAV2-hAQP1 for the treatment of grade 2/3 late radiation-induced xerostomia

-Entered into an asset purchase agreement with Johnson & Johnson* (J&J) to acquire all interests in botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP)

-Strengthened balance sheet with $100 million financing

LONDON and NEW YORK, May 14, 2026 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the first quarter ended March 31, 2026, and provided a corporate update.

“Our achievements in the first few months of 2026 have materially strengthened MeiraGTx – we are now in a position to file for potential approval and launch two wholly-owned therapies in the next 2 years,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “The compelling three-year durability data from our AAV2-hAQP1 Phase 1 study continue to demonstrate disease-modifying benefit following a simple one-time treatment of patients with moderate to severe persistent radiation-induced xerostomia, an otherwise debilitating life-long condition. The recent receipt of Breakthrough Therapy Designation for this program reinforces the strength of the data and the potential for an expedited development and regulatory pathway.”

Dr. Forbes continued, “We are very excited to have re-acquired bota-vec for the treatment of X-linked retinitis pigmentosa (XLRP). This is a highly strategic addition to our pipeline, given our long-term experience developing this drug while partnered with J&J, our deep expertise in ophthalmology, and our long-standing relationships with the inherited retinal disease patient community and KOL networks globally. Data from the Phase 3 LUMEOS study of bota-vec highlight the potential of this therapy to improve vision and significantly change the lives of those suffering with this otherwise inexorably degenerative disease. We are now working expeditiously to complete regulatory submissions in the U.S., EU, UK and Japan.”

“To that end, I am extremely pleased to announce that Penny Fleck has joined MeiraGTx as Chief Development Officer,” said Dr. Forbes. “Penny brings tremendous experience from her 20+ years at J&J and Takeda leading development of many assets, including multiple global regulatory approvals. Importantly, while at J&J as Global Head of Specialty Ophthalmology, Penny worked closely with MeiraGTx on the development of bota-vec, from the licensing of the drug by J&J through Phase 3. Her extensive experience and broad expertise across drug development will help us achieve the potential approvals of bota-vec and AAV2-hAQP1, as well

​

​

as progress our early stage programs such as Ribo-Leptin into the clinic and through development. Stuart Naylor, Ph.D. will be taking on a new role with the Company as Chief Scientific Officer, Ophthalmology. Stuart is a founder of MeiraGTx and led the incorporation of the UCL ophthalmology assets into the Company, including AAV-RPGR. He has experience with this therapy from pre-clinical through Phase 3 and he will be focusing his efforts on obtaining global regulatory approval of bota-vec as well as advancing our ophthalmology product candidates at all stages of development.”

*Janssen Pharmaceuticals, Inc., a Johnson & Johnson company

First Quarter 2026 Highlights

​

Strategic Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP):

●MeiraGTx entered into an asset purchase agreement with Johnson & Johnson in April 2026 to acquire full rights to bota-vec, a late-stage therapy for the treatment of X-linked retinitis pigmentosa (XLRP).

●MeiraGTx paid J&J a one-time $25 million upfront cash consideration, and J&J is eligible to receive a one-time regulatory and commercial milestone tied to U.S. approval and U.S. sales performance of bota-vec, as well as a mid-teens royalty on global net sales starting in mid-2029.

●The Company plans to rapidly advance the program toward global regulatory filings in the U.S., Europe, UK and Japan, leveraging its prior involvement in the program’s development and its established manufacturing readiness.

Bota-vec for the Treatment of X-linked Retinitis Pigmentosa (XLRP):

●XLRP is a rare inherited retinal disease with early onset and progressive degeneration to complete blindness in the third decade of life. There are currently no treatment options.

●There are >20,000 XLRP-RPGR patients in the U.S. and EU.

●The Phase 3 LUMEOS study was a global randomized study (n=95). All patients were treated bilaterally.

●Data from the Phase 3 LUMEOS trial of bota-vec for the treatment

EX-99.1

2 mgtx-20260325xex99d1.htm

EX-99.1

Exhibit 99.1

MeiraGTx Announces FDA Breakthrough Therapy Designation for AAV2-hAQP1 for the

Treatment of Grade 2 and Grade 3 Radiation-Induced Xerostomia (RIX) and Reports

Fourth Quarter and Full Year 2025 Financial and Operational Results

●   FDA granted Breakthrough Therapy Designation for AAV2-hAQP1 for the treatment of Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract

●   MeiraGTx to hold a program update and present long-term data for AAV2-hAQP1 program for the treatment of Grade 2/3 Radiation-Induced Xerostomia on Thursday, April 16th, 2026

LONDON and NEW YORK, March 26, 2026 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2025, and provided a corporate update.

“We are delighted to have been awarded Breakthrough Designation for our AAV2-hAQP1 treatment for Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This Breakthrough application was supported by 3-year data from the Phase 1 dose escalation study. On April 16th, we will be providing an AAV2-hAQP1 program update with information about the commercial opportunity for this therapy, as well as presenting the 3-year data. We have also had huge enthusiasm about our Phase 2 AQUAx2 study in the RIX community, amongst physicians and patients.”

“In 2025 we executed two important strategic collaborations bringing immediate non-dilutive financing into the company, as well as potential significant near-term financial milestones. We signed a collaboration with Eli Lilly and Company (Lilly) focused on our AAV-AIPL1 program for the treatment of LCA4, one of the most severe forms of inherited retinopathies. In addition to AAV-AIPL1, Lilly gained exclusive rights to two preclinical ocular programs as well as our intravitreal capsids, bespoke promoters and certain rights to our riboswitch platform in the eye. Lilly is working with global regulatory agencies to expeditiously gain approval of AAV-AIPL1 and to provide access to this life changing therapy for LCA4 to children globally.”

Dr. Forbes continued, “Earlier in the year, we entered into a strategic collaboration with Hologen Limited, a world-leader in the development of multi-modal generative AI foundation models which were built specifically to remove noise from clinical data and to allow the real clinical effects of treatment to be clearly seen. The use of Hologen’s AI

​

​

technology applied to MeiraGTx’s statistically significant double-blind Phase 2 data-sets has de-risked the AAV-GAD program and identified disease modifying changes in the physiology of the brain in response to treatment. We are working closely with Hologen to initiate the pivotal Phase 3 double blind sham-controlled study of AAV-GAD in Parkinson’s in the coming months at global centers of excellence in Parkinson’s disease treatment.”

“We have also been successful in the further development of our Riboswitch platform. We are currently in conversation with the FDA preparing our first Riboswitch IND with our Ribo-leptin product to deliver leptin using a daily oral small molecule inducer. We have very strong long-term data in animal models demonstrating the small molecule controlled riboswitch dynamics are durable for the life of the animal – out to 19 months so far. We have also now demonstrated very encouraging data in the second Riboswitch program that we intend to take into the clinic which is in neuropathic pain.”

2025 and Recent Highlights

AAV2-hAQP1 for the Treatment of Radiation-Induced Xerostomia (RIX):

●The FDA has now granted Breakthrough Therapy Designation for AAV2-hAQP1 for the treatment of Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract.

●This is in addition to the Regenerative Medicine Advanced Therapy (RMAT) designation already granted by the FDA for AAV2-hAQP1.

●The Company has aligned with the FDA on the clinical requirements for the Phase 2 AQUAx2 (NCT05926765) study to support a potential BLA with the primary endpoint being the change from baseline in the Xerostomia Questionnaire at 12 months following the one time treatment.

●The final patients are currently enrolling and the Company anticipates data 12 months after the last patient is treated, with a potential BLA filing in the first half of 2027 and potential approval around the end of 2027 with launch in the US targeted in early 2028.

●MeiraGTx will be hosting a program update April 16th to discuss the commercial opportunity as well as presenting the full 3-year data from all cohorts of the Phase 1 study.

AAV-GAD for the Treatment of Parkinson’s Disease:

●In 2025 the FDA granted R