After a phase 3 setback last year in a rare eye disease program, Johnson & Johnson has opted to return full rights to the gene therapy botaretigene sparoparvovec (bota-vec) to MeiraGTx in exchange for a $25 million upfront payment.
Under the agreement, Johnson & Johnson remains eligible for a one-time milestone tied to potential regulatory approval and commercial success, along with future royalties on sales, according to an April 16 announcement. MeiraGTx said it intends to move quickly toward regulatory filings in both the U.S. and Europe, with a potential launch targeted for 2027 if approvals are secured.
However, the therapy faces uncertain approval prospects. Johnson & Johnson originally obtained full rights to bota-vec in a 2023 deal worth up to $415 million, following its earlier development through the company’s Janssen division. The program suffered a major setback in May last year when it failed to meet its primary endpoint (improving vision-guided mobility in patients with X-linked retinitis pigmentosa) prompting J&J to divest the asset.
Regulatory hurdles may further complicate the path forward. Gene therapies have recently encountered challenges at the U.S. Food and Drug Administration, particularly under the oversight of outgoing biologics regulator Vinay Prasad. With no successor yet announced, the agency’s future approach to such therapies remains unclear.
Bota-vec is designed to deliver a functional version of the gene responsible for retinitis pigmentosa GTPase regulation directly to retinal cells. In patients lacking this gene – most of whom are male – vision progressively deteriorates over time, often leading to legal blindness by around age 40.
Despite the clinical setback, MeiraGTx remains encouraged, citing feedback from investigators who observed meaningful improvements in some trial participants across multiple aspects of vision.
Investor sentiment, however, has been less favorable, with the company’s shares dropping 16% in mid-morning trading following the announcement.
Looking ahead, MeiraGTx aims to transition into a fully commercial-stage company, with plans to potentially bring both bota-vec and its gene therapy candidate AAV-hAQP1 for radiation-induced dry mouth to market within the next two years.
As for J&J, the firm has begun executing its plan to reach $100 billion in annual revenue by 2026, according to CEO Joaquin Duato, who indicated that the company’s long-term growth ambitions extend even further. He noted that the pharma giant is relying on a portfolio of 28 potential blockbuster products, with additional candidates expected to contribute if development progresses as planned.
Duato highlighted that 10 of these key brands delivered double-digit sales growth in the first quarter, helping to offset declining revenue from the immunology drug Stelara, which has been impacted by biosimilar competition.
Oncology has emerged as a major growth driver for Johnson & Johnson, fueled by multiple myeloma therapies such as Darzalex and Talvey. In immunology, the psoriasis treatment Tremfya recorded a 64% increase in sales and is projected to achieve peak annual revenue of $10 billion.
Background of the MeiraGTx–J&J Partnership
The collaboration between MeiraGTx and J&J began with high expectations, focusing on developing gene therapies for inherited retinal diseases. J&J had previously acquired full rights to the therapy, known as botaretigene sparoparvovec (bota-vec), in a deal worth hundreds of millions. However, clinical setbacks ultimately led J&J to step away and return the program to MeiraGTx.
Why J&J Returned the Therapy to MeiraGTx
The primary reason behind the return was the therapy’s failure to meet its main goal in a Phase 3 trial targeting X-linked retinitis pigmentosa (XLRP), a rare genetic eye disease. Despite this setback, MeiraGTx believes the therapy still demonstrated meaningful benefits in secondary outcomes and patient-reported improvements.
Under the new agreement, MeiraGTx will pay approximately $25 million upfront to reacquire the therapy. The deal also includes potential milestone payments and future royalties to J&J if the therapy achieves regulatory approval and commercial success.
This arrangement allows MeiraGTx to regain full strategic control while still maintaining a financial link with J&J.
