MGTX News & Events

MeiraGTx announced that it has entered into an agreement with Oberland Capital for an investment of up to $400M in the company, including up to $375M in non-dilutive capital for capped royalty payments on certain products and up to $25M in equity. The investment by Oberland Capital provides for up to $400M in total capital to MeiraGTx, including up to $375M in non-dilutive royalty funding and up to $25M in equity, as follows: the initial $135M funded includes $125M in exchange for low single-digit royalties on the included products, and a $10M equity investment; an additional $50M will be available at the company's option tied to AAV2-hAQP1 positive data readouts from the Phase 2 AQUAx2 study in 2027; an additional $50M will be available at the company's option tied to regulatory approval of bota-vec in 2027; an additional $50M will be available at the company's option tied to regulatory approval of AAV2-hAQP1 in 2028; a further $100M is available upon mutual agreement for new products or business development and Oberland Capital has the right to purchase an additional $15M in equity in MeiraGTx. The agreement with Oberland Capital includes flexible provisions for potential change of control, with the ability for the Company to buy back the entire funded royalty note at any time by paying certain specified amounts. Royalty payments are capped at a multiple of the amounts funded.

Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas pledged to prevent political interference, Endpoint News' Zachary Brennan reports. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells. "Today's action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research. "By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on.Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible." This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

Reports Q1 revenue $293,000 vs. $1.93M last year. "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, 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."