MeiraGTx announced that it has entered into a purchase agreement with Johnson & Johnson (J&J) to acquire all interests in botaretigene sparoparvovec (bota-vec), an investigational treatment for X-linked retinitis pigmentosa (XLRP).

As part of the agreement, MeiraGTx will pay J&J a $25 million upfront cash payment and a one-time regulatory and commercial milestone tied to US approval and US 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. MeiraGTx intends to file for approval in the United States and European Union with the aim of a potential launch in 2027.

Data from the global randomized phase 3 LUMEOS trial (n = 95, all patients treated bilaterally) of bota-vec was presented at the Foundation Fighting Blindness 2025 Retinal Therapeutics Innovation Summit. Although the novel primary endpoint to assess functional vision as measured by a Visual Mobility Assessment (VMA), or maze, did not meet statistical significance, 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. The safety profile of bota-vec was as expected and manageable, with no new safety signals in the phase 3 trial with improved inflammatory profile compared with the phase 1/2 study.

Additional Functional Vision Endpoints:

• Significant change in the LLQ Extreme lighting domain score, LS mean (P = .006); statistically significant improvements in questions relating to mobility (P = .001), general dim lighting (P = .007) and emotional distress (P = .019)
• IVI-A: significant improvement in total score vs control at week 52 (P = .024) with greater significance in the emotional wellbeing questions (P = .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 = .001)
• Pointwise responders (repeated 5-point 7-decibel) in the Full visual field (P = .001)
• Change in Mean retinal sensitivity in the central 10 degrees (P = .001)
• Change in Mean retinal sensitivity full field 90 degrees (P = .004)

Visual Function:

• Change in Low luminance visual acuity (LLVA, EDTRS letters) LS mean (P = .003)
• 45% of treated patients gained > 10 letters in LLVA
• 20% of treated patients achieved > 15 letters in LLVA

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