MeiraGTx reports Q2 2026 results, advances bota-vec and AAV2-hAQP1 toward filings

MeiraGTx Holdings plc reported financial and operational results for the second quarter ended June 30, 2026, in an August 13, 2026 release. Chief executive Alexandria Forbes said the quarter marked progress toward transforming the company into a commercial entity, highlighting the acquisition of bota-vec from J&J and the start of work on global regulatory filings ahead of a potential first commercial launch in 2027.¹

In May 2026, Janssen Pharmaceuticals, Inc., a Johnson & Johnson company,¹ transferred full rights to botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa to MeiraGTx for a one-time $25 million upfront cash payment, with J&J eligible for a regulatory and commercial milestone tied to U.S. approval and sales, plus a mid-teens royalty on global net sales beginning in mid-2029.¹ Bota-vec holds FDA Fast Track and Orphan Drug designations, and EU PRIME, ATMP, and Orphan Drug designations.¹ The company said global regulatory filings for bota-vec are expected in 2026.

For AAV2-hAQP1 in radiation-induced xerostomia, the FDA granted Breakthrough Therapy Designation in March 2026, supported by three-year data from the 24-patient Phase 1 AQUAx study.¹ The pivotal Phase 2 AQUAx2 study, a randomized, double-blind, placebo-controlled trial at 30 sites in the U.S., Canada, and the U.K., completed enrollment in the second quarter of 2026 and remains on track for a 12-month pivotal data readout in the second quarter of 2027, which, if positive, would support a BLA filing and potential approval by the end of 2027, with U.S. launch targeted for 2028.¹

In June 2026, MeiraGTx entered an agreement with Oberland Capital Management LLC for an investment of up to $400 million, including up to $375 million in non-dilutive capital in exchange for low single-digit capped royalties on certain products, plus up to $25 million in equity investment.¹ The initial $135 million funded comprised $125 million in cash and a $10 million equity investment, with additional tranches available at the company's option: $50 million tied to positive AAV2-hAQP1 Phase 2 AQUAx2 data in 2027, $50 million tied to bota-vec regulatory approval in 2027, $50 million tied to AAV2-hAQP1 regulatory approval in 2028, a further $100 million available upon mutual agreement for new products or business development, and Oberland's right to purchase an additional $15 million in equity.¹

As of June 30, 2026, the company had cash and cash equivalents of approximately $143.2 million, and together with additional royalty note and equity proceeds from Oberland and an upfront payment from Hologen, believes it will have sufficient capital to fund operations into the second half of 2028.¹