MeiraGTx Enters Into Asset Purchase Agreement With Johnson & Johnson To Buy All Interests In Bota-Vec For Treatment Of XLRP; MeiraGTx To Pay J&J $25M Upfront, Milestone Fees, High Double‑Digit Royalties On Bota‑Vec Sales

• Company 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)
  
   
• MeiraGTx intends to immediately pursue global regulatory filings for approval of bota-vec
   

LONDON and NEW YORK, April 16, 2026 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced that it has entered into an asset purchase agreement with Johnson & Johnson* (J&J) to acquire all interests in bota-vec for the treatment of XLRP.

The IRD community is a concentrated one with 40-50 centers of excellence in the EU, U.S. and Japan caring for approximately 80% of IRD patients. Through the initial formation of MeiraGTx in 2015 in collaboration with University College London (UCL) and the Moorfields Eye Hospital, MeiraGTx has close relationships with most of the KOLs at these leading sites, with 32 of these sites participating in the Phase 3 LUMEOS study of bota-vec.

Since the release of the LUMEOS Phase 3 data, MeiraGTx has heard from numerous investigators about the clinically meaningful benefit that bota-vec has afforded a significant number of patients who participated in the study, with unprecedented improvements demonstrated in each of the 3 domains of vision. Investigators around the world are enthusiastically supporting filing for regulatory approval of bota-vec in order to allow access to treatment for the patients they are seeing in their clinic today who are waiting for this potentially life-changing therapy.

In re-acquiring bota-vec, MeiraGTx intends to expeditiously file for approval in the U.S. and EU with the aim of a potential launch in 2027. With the data from the AQUAx 2 pivotal study of AAV-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia expected in the second quarter of 2027, the Company's intent is to become a commercial stage company with two potential products launching over the next 2 years into concentrated markets, both addressing severe unmet needs and both being disease modifying in areas where patients have no treatment options.

Bota-vec Asset Purchase Terms:

MeiraGTx will pay J&J a $25 million upfront cash payment and a one-time regulatory and commercial milestone tied to U.S. approval and U.S. sales performance of bota-vec for the treatment of XLRP, as well as a high double-digit royalty on global net sales starting in mid-2029.

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 botaretigene sparoparvovec (bota-vec) for the treatment of XLRP was presented at the Foundation Fighting Blindness 2025 Retinal Therapeutics Innovation Summit.
• Following the release of the compelling Phase 3 data at their summit, the Foundation Fighting Blindness issued a public letter to J&J strongly supporting the filing and ultimate approval of this treatment for XLRP and stating that it had a remarkable benefit for many of the patients treated.
   

Phase 3 LUMEOS Study Data:

• The novel primary endpoint to assess the effect of bilaterial treatment with bota-vec on functional vision as measured by a Visual Mobility Assessment (VMA), or maze, did not meet statistical significance. However, it was directionally supportive with treated subjects 2.4x more likely to respond than untreated subjects.
• LLQ PRO showed significant benefit in mobility and dim light function, which is what the VMA tested, indicating the maze was not sensitive enough to capture these benefits.
• The data from the secondary endpoints were very strong, with clinically meaningful and statistically significant improvements demonstrated in each of the three domains of vision.
   

Additional Functional Vision Endpoints:

• Significant change in the LLQ Extreme lighting domain score, LS mean p=0.006; statistically significant improvements in questions relating to mobility (p= 0.001), general dim lighting (p= 0.007) and emotional distress (p= 0.019)
• IVI-A: significant improvement in total score vs control at week 52 p=0.024 with greater significance in the emotional wellbeing questions (p=0.005)

Retinal Function:

• All measures of retinal sensitivity showed highly significant difference between treated and untreated groups
• Pointwise responders (repeated 5-point 7-decibel) in the Central 30 degrees p=0.001
• Pointwise responders (repeated 5-point 7-decibel) in the Full visual field p=0.001
• Change in Mean retinal sensitivity in the central 10 degrees, p=0.001
• Change in Mean retinal sensitivity full field 90 degrees p= 0.004
   

Visual Function:

• Change in Low luminance visual acuity (LLVA, EDTRS letters) LS mean p=0.003
• 45% of treated patients gained >10 letters in LLVA
• 20% of treated patients achieved >15 letters in LLVA
   

Multi-endpoint Responder Analysis:

• 40% (22/55) of treated patients showed improvement in ≥2 endpoints each in different domains of vision compared to 0% in the control group. This was consistent whichever endpoints were tested.

Safety:

• Safety profile of bota-vec was as expected and manageable, no new safety signals in the Phase 3 with improved inflammatory profile compared to the Phase ½.

CMC:

• MeiraGTx is the commercial manufacturer of bota-vec and have successfully completed PPQ. The Company has received a commercial license from the MHRA for its London manufacturing facility, as well as a commercial license for the Company's QC facility that conducts the release and stability assays for the product in Shannon, Ireland. The Company currently has several hundred vials of product in hand that on QP release can be used to treat patients immediately following approval.
   

The FDA has granted Fast Track and Orphan Drug Designations to bota-vec, and the regulatory authorities in the EU have granted Priority Medicines, or PRIME, advanced therapy medicinal product, or ATMP, and Orphan Drug Designations to bota-vec.

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

MeiraGTx has a licensing agreement with Mass Eye and Ear. Dr. Huckfeldt does not have a personal financial interest in bota-vec or MeiraGTx.