MeiraGTx (Nasdaq: MGTX) reclaims bota-vec XLRP therapy in $25M deal
Rhea-AI Filing Summary
MeiraGTx Holdings plc has reacquired full rights to botaretigene sparoparvovec (bota-vec), its gene therapy for X-linked retinitis pigmentosa (XLRP), through an asset purchase from Janssen Pharmaceuticals. The company will pay an upfront $25 million in cash, a $50 million contingent milestone tied to U.S. approval and U.S. net sales first exceeding $250 million, and mid-teens royalties on global net sales commencing on or after July 1, 2029.
The deal transfers Janssen’s UCL license and related RPGR program assets and includes a perpetual, worldwide license to key know-how and patents. Johnson & Johnson’s investment arm, a holder of more than 5% of MeiraGTx shares, agreed to a 12-month lockup on its holdings plus advance notice before any later sales. MeiraGTx plans rapid U.S., EU and Japan filings for bota-vec, aiming for a potential launch in 2027, and ultimately expects to become a commercial-stage company with two products, including AAV-hAQP1 for radiation-induced xerostomia, over the next two years.
Positive
- Reacquisition of late-stage XLRP asset with supportive Phase 3 data: MeiraGTx regains full rights to bota-vec, a gene therapy for X-linked retinitis pigmentosa backed by Phase 3 LUMEOS results and multiple regulatory designations, creating a clear path toward its first potential commercial product.
- Defined, back-end–weighted deal economics: Upfront cash of $25 million plus a $50 million U.S. approval and sales milestone and royalties beginning in 2029 defer most obligations into a period when bota-vec could be revenue generating.
- Progress toward becoming a commercial-stage company: Management outlines a goal of launching bota-vec around 2027 and adding AAV-hAQP1 for radiation-induced xerostomia after pivotal data expected in Q2 2027, targeting two products in concentrated, high-need markets.
Negative
- None.
Insights
Reacquiring bota-vec gives MeiraGTx a late-stage lead asset with defined economics.
MeiraGTx is buying back full rights to the RPGR-based XLRP therapy bota-vec for an upfront $25 million, a $50 million U.S. approval and sales milestone, and mid-teens royalties starting after July 1, 2029. This concentrates future commercial upside while keeping near-term cash outlay relatively contained.
The program already has Phase 3 LUMEOS data and multiple regulatory designations, and MeiraGTx states it has completed key CMC work as commercial manufacturer. That supports its plan to file BLA and MAA submissions in major markets and positions bota-vec as a potential first commercial product.
The filing also notes a prior $105 million Upfront Payment from Hologen and amendments that will result in Hologen holding a minority stake in MeiraGTx Manufacturing. Together, these arrangements highlight a capital-efficient strategy: leveraging partners while retaining product and manufacturing control. Overall, this appears strategically positive, though ultimate value depends on regulatory outcomes and realized sales.
8-K Event Classification
Key Figures
Key Terms
Asset Purchase Agreement financial
UCL License Agreement financial
royalties financial
non-exclusive, perpetual, irrevocable license regulatory
Deed of Commitment Agreement financial
Fast Track and Orphan Drug Designations regulatory
UNITED STATES
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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 1.01Entry into a Material Definitive Agreement.
Janssen Asset Purchase Agreement
On April 15, 2026 (the “Closing Date”), MeiraGTx Holdings plc (the “Company”) and its wholly-owned subsidiary MeiraGTx Ocular UK Limited, a company incorporated in England and Wales (“MeiraGTx Ocular” and together with the Company, collectively the “Buyer”), entered into and consummated an Asset Purchase Agreement (the “Asset Purchase Agreement”) with Janssen Pharmaceuticals, Inc., a Pennsylvania corporation (“Seller”), pursuant to which Seller sold and assigned to Buyer, and Buyer purchased and assumed, that certain License Agreement, dated February 5, 2019, by and between UCL Business Plc (now UCL Business Ltd.) and Janssen (the “UCL License Agreement”), relating to the research, development, manufacture and exploitation of Seller’s gene therapy product for the treatment of X-linked retinitis pigmentosa related to mutations in the RPGR gene (the “RPGR Product”), and other related assets as described in the Asset Purchase Agreement.
Buyer agreed to pay an upfront cash purchase price of $25,000,000 to Seller. Additionally, pursuant to and subject to the terms and conditions set forth in the Asset Purchase Agreement, Buyer agreed to pay Seller a one-time, future contingent consideration of $50,000,000 upon both of the following milestones being achieved: (i) Buyer’s or its affiliates’ receipt of regulatory approval for an RPGR Product in the United States and (ii) aggregate net sales by Buyer or its affiliates of all RPGR Products in the United States since the Closing Date first exceeds $250,000,000. Buyer has also agreed to pay Seller royalties, based on future net sales globally of the RPGR Product by Buyer or its Affiliates, in the mid-teens percentage of annual net sales for the RPGR Product commencing on or after July 1, 2029. Additionally, Buyer will pay a portion of upfront and milestone payments to Seller in the event Buyer or any of its affiliates may receive payments from a third party if Buyer or any of its affiliates grant any license or right to develop or commercialize any RPGR Product to such third party, as well as make royalty payments to Seller for a given RPGR Product based on (A) royalty payments Buyer or its affiliates may receive from such third party (after deduction of any royalty payments due under the UCL License Agreement) and (B) net sales of a given RPGR Product by such third party.
Johnson & Johnson Innovation – JJDC, Inc. (“JJDC”), the investment arm of Johnson & Johnson and owner of Seller, owns more than 5% of the Company’s outstanding shares. JJDC and Seller have agreed not to sell or transfer any of the Company’s ordinary shares or securities convertible into, exchangeable for, or exercisable for the Company’s ordinary shares, for twelve months after the Closing Date and following such twelve month period, if they ever intend to sell the Company’s shares after the twelve month period, they will provide written notice to the Company at least five business days prior to taking any action.
The Asset Purchase Agreement contains customary representations, warranties, and covenants from each of Seller and Buyer, including provisions that require Seller to indemnify Buyer and its affiliates and representatives against certain losses related to, among other things, breaches of Sellers’ representations, warranties, covenants, and agreements as well as any excluded liabilities or excluded assets, as described in the Asset Purchase Agreement. Similarly, subject to certain customary limitations, Buyer agreed to indemnify Seller and their respective affiliates and representatives against certain losses related to, among other things, breaches of Buyer’s representations, warranties, covenants, and agreements as well as the assumed liabilities and any use of the Licensed Intellectual Property (as defined below) by or on behalf of Buyer. Buyer will be responsible for any royalty or milestone amounts that become payable on the RPGR Product under the UCL License Agreement.
The Asset Purchase Agreement includes a grant by Seller, on behalf of itself and its affiliates, to Buyer of a non-exclusive, perpetual, irrevocable, non-transferable (subject to the terms of the Asset Purchase Agreement), royalty-free, fully paid-up, worldwide license (with the right to grant sublicenses through multiple tiers, subject to the terms and provisions of the Asset Purchase Agreement) under certain know-how and patents that are owned or otherwise controlled by Seller or its affiliates as of the Closing Date and are necessary or reasonably useful for the exploitation of the RPGR Product (the “Licensed Intellectual Property”), solely to research, develop, manufacture, commercialize and otherwise exploit any RPGR Product throughout the world.
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Pursuant to the Asset Purchase Agreement, each of Seller and Buyer, on behalf of itself and its affiliates, agreed not to solicit or encourage any employee or consultant of the other party or its affiliates to terminate or diminish its relationship with such party or its affiliates for a period of five years after the Closing Date.
The foregoing description of the Asset Purchase Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Asset Purchase Agreement, a copy of which is attached as Exhibit 10.1 hereto and incorporated herein by reference.
Item 1.02Termination of a Material Definitive Agreement.
In connection with the Seller and Buyer entering into the Asset Purchase Agreement described in Item 1.01 of this Current Report on Form 8-K, which description is incorporated by reference into this Item 1.02, Seller and Buyer entered into a Termination Agreement on April 15, 2026 (the “Termination Agreement”) terminating that certain Asset Purchase Agreement, dated as of December 20, 2023 (the “Original Asset Purchase Agreement”), by and among Seller and the Company and its wholly-owned subsidiary MeiraGTx UK II Limited, a company incorporated in England and Wales (“MeiraGTx UK II”), that certain Supply Agreement, dated as of December 20, 2023 by and between MeiraGTx UK II and Seller, and certain other documents related to the Original Asset Purchase Agreement.
The foregoing description of the Termination Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Termination Agreement, a copy of which is attached as Exhibit 10.2 hereto and incorporated herein by reference.
Item 2.01Completion of Acquisition or Disposition of Assets.
The information described in Item 1.01 regarding the Asset Purchase Agreement is incorporated by reference into this Item 2.01.
Item 7.01Regulation FD Disclosure.
On April 16, 2026, the Company issued a press release in connection with entering into the Asset Purchase Agreement, a copy of which is filed as Exhibit 99.1 and incorporated by reference into this Item 7.01.
The information in this Item 7.01 of this Current Report on Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01Other Events.
The Company is providing the following updates:
Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP):
· | Based on data provided by Johnson & Johnson, while the novel primary endpoint to assess the effect of bilateral treatment with botaretigene sparoparvovec (bota-vec) on functional vision as measured by a Visual Mobility Assessment (VMA), or maze, did not meet statistical significance, a positive trend was observed, with the treated group 2.4x more likely to respond than the untreated group. In addition, statistically significant improvements were observed in retinal function (static perimetry and microperimetry), functional vision under low luminance and visual function. 40% (22/55) of treated patients showed improvement in ≥ 2 endpoints, compared to 0% in control group. Consistent 25%-40% treatment benefit observed across all endpoint combinations, with most showing 0% in the control arm. |
| ● | The safety results from the trial were as expected and manageable, with no new safety signals. |
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· | We estimate there are more than 20,000 patients in the United States and the European Union with inherited retinitis pigmentosa and, if approved, a potential peak market of approximately $1.7 billion in the United States, European Union and Japan and cumulative net revenues globally over 10 years expected to be approximately $7.7 billion. |
Hologen Transactions
On April 2, 2026, the Company, MeiraGTx Manufacturing Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Manufacturing”), MeiraGTx Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Limited”), MeiraGTx Neuro UK Limited, a private company limited by shares incorporated in England and a wholly-owned subsidiary of the Company (“MeiraGTx Neuro UK”), and MeiraGTx Neuro I, LLC, a Delaware limited liability company and a wholly-owned subsidiary of the Company (“MeiraGTx Neuro US”), on the one hand, and Hologen Limited, a non-cellular company limited by shares incorporated in Guernsey (“Hologen”), Hologen Neuro AI Limited, a non-cellular company limited by shares incorporated in Guernsey and an affiliate of Hologen (“HNAI”), and Hologen Neuro AI UK Limited, a private company limited by shares incorporated in England and an affiliate of Hologen (“HNAI UK”), on the other hand, entered into Amendment No. 1 to Deed of Commitment Agreement (the “Amendment”). Under the Amendment, the parties agreed to, among other things, the following:
(i) | Hologen agreed to issue to the Company 250,000 Class A shares in Hologen concurrently with entering into the Amendment; |
(ii) | to amend the Framework Agreement, dated March 9, 2025 by and among the Company, MeiraGTx Neuro UK, HNAI and Hologen (the “Neuro Framework Agreement”), to provide (A) for additional conditions that must be met prior to Completion (as defined under the Neuro Framework Agreement), including MeiraGTx Neuro UK subscribing for Class A shares in HNAI in consideration for the provision of services to HNAI and HNAI UK as specified in the Collaboration and License Agreement to be entered into upon Completion of the Neuro Framework Agreement, and Hologen subscribing for Class B shares in HNAI in consideration for a portion of the $105 million in payments Hologen previously made to the Company as part of its commitment toward the upfront cash payment of $200 million (the “Upfront Payment”) provided for under the Framework Agreements (as defined below), and (B) that following Completion, Hologen shall fund the remaining portion of the Upfront Payment provided for under the Neuro Framework Agreement by purchasing a portion of the Class A shares held by MeiraGTx Neuro UK, such that following the purchase, such Class A shares purchased by Hologen shall be converted to Class B shares and Hologen shall own 70% of the issued share capital of HNAI and MeiraGTx Neuro UK shall own 30% of the issued share capital of HNAI; |
(iii) to amend the Framework Agreement, dated March 9, 2025, by and among MeiraGTx Manufacturing, MeiraGTx Limited and Hologen (the “Manufacturing Framework Agreement, and together with the Neuro Framework Agreement, the “Framework Agreements”) to provide (A) for additional conditions that must be met prior to Completion (as defined in the Manufacturing Collaboration Agreement), including Hologen purchasing shares in MeiraGTx Manufacturing from MeiraGTx Limited in consideration for a portion of the $105 million in payments Hologen previously made to the Company as part of the Upfront Payment, and (B) that following Completion, Hologen shall fund the remaining portion of the Upfront Payment provided for under the Manufacturing Framework Agreement by purchasing additional shares in MeiraGTx Manufacturing from MeiraGTx Limited such that following the purchase, Hologen will own a minority interest in MeiraGTx Manufacturing; and
(iv) | Hologen committed to deploying the funds it raises to pay the remaining portion of the Upfront Payment as required by the Framework Agreements. |
The foregoing description of the Amendment does not purport to be complete and is qualified in its entirety by reference to the full text of the Amendment, a copy of which is attached as Exhibit 99.2 hereto, and incorporated herein by reference.
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Forward-Looking Statements
This Current Report on Form 8-K contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this Current Report that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding anticipated payments under the Asset Purchase Agreement and pursuant to the Hologen transactions, execution of the obligations under the Asset Purchase Agreement and pursuant to the Hologen transactions, estimates regarding the market size for bota-vec, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate,” “eligible” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic transactions or priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug or rare pediatric disease designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of pandemics, epidemics or outbreaks of infectious diseases on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this Current Report. Any such forward-looking statements represent management’s estimates as of the date of this Current Report. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this Current Report
Item 9.01Financial Statements and Exhibits
(d)Exhibits
Exhibit No. | | Description |
10.1 | Asset Purchase Agreement, dated April 15, 2026, by and among Janssen Pharmaceuticals, Inc., MeiraGTx Ocular UK Limited and MeiraGTx Holdings plc.* | |
10.2 | Second Termination Agreement, dated April 15, 2026, by and among Janssen Pharmaceuticals, Inc., MeiraGTx UK II Limited, MeiraGTx Holdings plc and MeiraGTx Ocular UK Limited.* | |
99.1 | Press Release of MeiraGTx Holdings plc, dated as of April 16, 2026. | |
99.2 | Amendment No. 1 to Deed of Commitment Agreement, dated April 2, 2026, by and among MeiraGTx Holdings plc, MeiraGTx Manufacturing Limited, MeiraGTx Limited, MeiraGTx Neuro UK Limited, MeiraGTx Neuro I, LLC, Hologen Limited, Hologen Neuro AI Limited and Hologen Neuro AI UK Limited.* |
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104 | Cover Page Interactive Data File (the cover page XBRL tags are embedded in the Inline XBRL document). |
* Portions of this exhibit (indicated by asterisks) have been omitted pursuant to Item 601(b)(10)(iv) of Regulation S-K
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
Dated: April 16, 2026 | MEIRAGTX HOLDINGS PLC | |
By: | /s/ Richard Giroux | |
Name: | Richard Giroux | |
Title: | Chief Financial Officer and Chief Operating Officer | |
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Exhibit 99.1
MeiraGTx Announces the Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP)
-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.
“We are extremely pleased to have reacquired bota-vec for the treatment of XLRP,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This is a unique opportunity to gain an asset at this stage in development with data supporting a meaningful benefit in patients with no alternative treatment, many of whom are waiting for this life changing therapy and hoping for expeditious approval.”
Dr. Forbes continued, “We are intimately familiar with AAV-RPGR, having collaborated with J&J during the development of the program from Phase 1 onward. Importantly, from a regulatory CMC perspective, as the commercial manufacturer of the product, we have completed PPQ with the CMC datasets for filing with global regulators. We intend to start filing BLA and MAA in the U.S., EU and Japan as soon as possible.”
Jason Menzo, CEO of Foundation Fighting Blindness, stated, “For patients living with X-linked retinitis pigmentosa, the need for treatment options is clear and urgent. The data from the LUMEOS Phase 3 study of bota-vec, reflected in both objective measures and patient-reported outcomes, point to real improvements in vision. Our focus is on advancing safe and effective therapies that matter to patients, and we are excited to be working with MeiraGTx and regulators to bring this potential treatment to the global XLRP community.”
Rachel Huckfeldt, M.D., Ph.D., director of Inherited Retinal Disorders Clinical Trials at Mass Eye and Ear and a site principal investigator who has led multiple botaretigene sparoparvovec clinical trials at the hospital, added, “There is clear unmet need for individuals with X-linked retinitis pigmentosa. The Phase 3 trial demonstrated meaningful improvements across multiple outcome measures with 10- and 15-letter gains in low luminance visual acuity as one example. Many participants were able to provide examples from their daily lives of the real-world impact of these gains. These results provide hope for individuals with XLRP, and they warrant further consideration by regulatory agencies.”
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 1/2. |
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.
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline with four late-stage clinical programs. Each of these programs uses local delivery of small doses, resulting in disease-modifying effects in both inherited and more common diseases, in the eye, Parkinson’s disease, and radiation-induced xerostomia. MeiraGTx uses its innovative technology in optimization of capsids, promoters, and novel translational control elements to develop best-in-class, potent, safe viral vectors. MeiraGTx’s broad pipeline is supported by end-to-end in-house manufacturing. MeiraGTx has built the most comprehensive manufacturing capabilities in the industry, including two that are licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure. In addition, MeiraGTx has developed a proprietary manufacturing platform process over 9 years based on more than 20 different viral vectors with leading yield and quality aspects and commercial readiness. Uniquely, MeiraGTx has developed a novel technology for in vivo delivery of any biologic therapeutic using oral small molecules. This transformative riboswitch gene regulation technology allows precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the regulated in vivo delivery of metabolic peptides, including GLP-1, GIP, Glucagon, Amylin, PYY, and Leptin, as well as cell therapy, CAR-T for liquid and solid tumors and autoimmune diseases, and additionally, PNS targets addressing long-term intractable pain. MeiraGTx has developed the technology to apply genetic medicine to common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
For more information, please visit www.meiragtx.com.
Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding anticipated payments under the Asset Purchase Agreement, execution of the obligations under the Asset Purchase Agreement and estimates regarding the market size for bota-vec, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate,” “eligible” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or
achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic transactions or priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug or rare pediatric disease designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of pandemics, epidemics or outbreaks of infectious diseases on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.
Contacts
Investors:
MeiraGTx
Investors@meiragtx.com
or
Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
Exhibit 99.2
Certain information marked as [***] has been excluded from this exhibit because it is both not material and is the type that the registrant treats as private or confidential.
AMENDMENT NO. 1 TO DEED OF COMMITMENT AGREEMENT |
This DEED is dated 2nd April 2026 (“Effective Date”) and is made by and among:
THE PARTIES
1) | Hologen Limited, a non-cellular company limited by shares incorporated in Guernsey with company number 74905 whose registered office is at 1st Floor, Royal Chambers, St. Julian’s Avenue, St. Peter Port, GY1 3JX, Guernsey (“Hologen Limited”), together with its affiliates (i) Hologen Neuro AI Limited (company number 74942) (“HNAI”) and (ii) Hologen Neuro AI UK Limited (company number 16283812) (“HNAIUK”) (collectively “Hologen”); and |
2) | MeiraGTx Holdings plc, a Cayman Islands exempted company having a place of business at 655 Third Avenue, Suite 1115, New York, NY 10017, together with its affiliates, (i) MeiraGTx Manufacturing Limited (company number 16128294), (ii) MeiraGTx Limited (company number 0950199), (iii) MeiraGTx Neuro UK Limited (company number 16108121) and (iv) MeiraGTx Neuro I, LLC (collectively “MeiraGTx”), |
each a “Party” and together, the “Parties”.
BACKGROUND
(A) | The Parties entered into a deed of commitment agreement dated March 23, 2026 (“Deed of Commitment”). |
(B) | The Parties now wish to amend the Deed of Commitment as set out in the Schedule to this amendment to the Deed of Commitment (“Amendment Deed”). |
AGREED TERMS
1. | Definitions and Interpretation |
1.1 | In this Amendment Deed: |
(a) | “Amended and Restated Deed” means the Deed of Commitment in the form set out in the Schedule to this Amendment Deed; and |
(b) | “Effective Date” means the date of this Amendment Deed. |
1.2 | Unless otherwise defined in this deed, words and expressions defined in the Deed of Commitment shall have the same meanings when used in this deed. |
1.3 | The rules of interpretation set out in the Deed of Commitment shall apply to this deed as if set out in full in this deed. |
2. | Amendment and restatement |
2.1 | With effect from the Effective Date, the Deed of Commitment is amended and restated so that it reads in its entirety as set out in the Schedule to this Amendment Deed. For the avoidance of doubt, the amendments reflected in the Schedule are indicative only and the text which is shown to be deleted will have no force or effect in respect of the of the Amended and Restated Deed. |
2.2 | The Parties agree that, with effect from the Effective Date, all references in any document to the Deed of Commitment shall, unless the context otherwise requires, be read and construed as references to the Amended and Restated Deed. |
3. | Continuing effect |
3.1 | Save as expressly amended by this Amendment Deed, all provisions of the Deed of Commitment shall remain in full force and effect. |
3.2 | Nothing in this Amendment Deed shall affect any right or obligation that has accrued or arisen under the Deed of Commitment prior to the Effective Date. |
4. | Miscellaneous |
4.1 | This Amendment Deed is subject to the laws of England and Wales, and the courts of England and Wales will have exclusive jurisdiction over any disputes. |
4.2 | If any term of this Amendment Deed is invalid, illegal or incapable of being enforced, all other terms and provisions of this Amendment Deed shall nevertheless remain in full force and effect. |
4.3 | This Amendment Deed may not be amended without the prior written consent of each of the parties hereto. |
4.4 | This Amendment Deed may be executed in counterparts each of which shall be deemed to be an original hereof and all of which together evidence the same Amendment Deed. This Amendment Deed may be executed via email (or transmission of a PDF file) of a counterpart of this Amendment Deed. In addition, PDF or electronic signatures of authorised signatories of any party shall be valid and binding and delivery of an email or PDF signature by any party shall constitute due execution and delivery of this Amendment Deed. |
IN WITNESS WHEREOF, the parties intending to be bound have caused this deed to be executed and delivered as a DEED on the date specified at the beginning of this deed by their duly authorised representatives.
Executed as a Deed by HOLOGEN LIMITED | /s/ Henry Smith |
| Alternate Director for Andrew Henton |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Elliot Dean Worrall |
| |
Name: | Elliot Dean Worrall |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by HOLOGEN NEURO AI LIMITED | /s/ Henry Smith |
| Alternate Director for Andrew Henton |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Elliot Dean Worrall |
| |
Name: | Elliott Dean Worrall |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by HOLOGEN NEURO AI UK LIMITED | /s/ Parashkev Natchev |
| Director |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Elliot Dean Worrall |
| |
Name: | Elliot Dean Worrall |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by MEIRAGTX HOLDINGS PLC | /s/ Alexandria Forbes |
| Director |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Robert Wollin |
| |
Name: | Robert Wollin |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by MEIRAGTX MANUFACTURING LIMITED | /s/ Rich Giroux |
| Director |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Robert Wollin |
| |
Name: | Robert Wollin |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by MEIRAGTX LIMITED | /s/ Rich Giroux |
| Director |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Robert Wollin |
| |
Name: | Robert Wollin |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by MEIRAGTX NEURO UK LIMITED | /s/ Rich Giroux Director |
| |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Robert Wollin |
| |
Name: | Robert Wollin |
| |
Occupation | [***] |
| |
Address | [***] |
Executed as a Deed by MEIRAGTX NEURO I, LLC | /s/ Rich Giroux |
| Director |
acting by a director in the presence of: | |
| |
Witness’ signature | /s/ Robert Wollin |
| |
Name: | Robert Wollin |
| |
Occupation | [***] |
| |
Address | [***] |
SCHEDULE
DEED OF COMMITMENT AGREEMENT |
This DEED is dated ______________________ (“Effective Date”) and is made by and among:
THE PARTIES
1) | Hologen Limited a non-cellular company limited by shares incorporated in Guernsey with company number 74905 whose registered office is at 1st Floor, Royal Chambers, St. Julian’s Avenue, St. Peter Port, GY1 3JX, Guernsey (“Hologen Limited”), together with its affiliates (i) Hologen Neuro AI Limited (company number 74942) (“HNAI”) and (ii) Hologen Neuro AI UK Limited (company number 16283812) (“HNAIUK”) (collectively “Hologen”); and |
2) | MeiraGTx Holdings plc, a Cayman Islands exempted company having a place of business at 655 Third Avenue, Suite 1115, New York, NY 10017, together with its affiliates, (i) MeiraGTx Manufacturing Limited (company number 16128294), (ii) MeiraGTx Limited (company number 0950199), (iii) MeiraGTx Neuro UK Limited (company number 16108121) and (iv) MeiraGTx Neuro I, LLC (collectively “MeiraGTx”), |
each a “Party” and together, the “Parties”.
BACKGROUND
(A) | The Parties entered into the Framework Agreements (as defined in the Schedule) on 9 March 2025. |
(B) | Pursuant to clause 22.1 of the HNAI Framework Agreement, the HNAI Framework Agreement may be modified or amended by written agreement of the Company and each of Hologen Limited and MeiraGTx Neuro UK Limited (company number 16108121) (being the Major Members (as defined therein) as at the date hereof) signed by or on behalf of each of the Company and the Major Members (together the “Requisite Parties”). |
(C) | The Requisite Parties wish to amend the HNAI Framework Agreement on the terms of this Deed. |
(D) | The Parties now wish to agree as follows in respect of certain declarations in respect of the Transaction Documents by entering into this commitment agreement (“Deed”). |
ISSUANCE OF ADDITIONAL HOLOGEN SHARES
1. | Concurrently with entering into this Deed, on the Effective Date, Hologen Limited will issue 250,000 Class A Shares in Hologen Limited at a share price of $0.0001 per share to MeiraGTx Holdings plc pursuant to the standard Hologen Class A subscription agreement. This, together with the 250,000 Class A Shares in Hologen Limited that MeiraGTx Holdings plc already owns, corresponds to [***] of all shares issued at the date of this Deed (calculated including the Hologen Limited employee share option scheme). |
COMPLETION OF THE FRAMEWORK AGREEMENTS
2. | The Parties agree that Completion shall occur in accordance with the relevant Framework Agreement upon (a) the satisfaction of the HNAI Completion Conditions (as defined below), and (b) the satisfaction of the Manufacturing Completion Condition (as defined below). |
3. | With effect from the date of this agreement: |
a) | [***]; |
b) | the Requisite Parties will procure the amendment of the Articles of Incorporation of HNAI, in each case: (i) to ensure that each Party has the right to purchase such amount of shares in any future equity offering by HNAI, on the terms of that offering, that preserves the Party’s percentage share of equity in HNAI, and (ii) (A) to ensure that MeiraGTx has the right to sell a number shares it holds in HNAI [***], on the terms of that offering, provided that [***] and (B) in the event MeiraGTx elects to sell shares pursuant to clause (A), HNAI shall have a right of first refusal to purchase all or any portion of such shares that MeiraGTx is proposing to sell, and Hologen Limited shall have a secondary right of first refusal to purchase all or any portion of such shares that MeiraGTx is proposing to sell that are not purchased by HNAI. |
HNAI COMPLETION CONDITIONS AND PURCHASE RIGHTS
4. | In addition to the Completion Conditions (as defined in the HNAI Framework Agreement), which all parties agree as being satisfied as at the date hereof, the following conditions must all be met for Completion to happen in accordance with clause 2 (the “HNAI Completion Conditions”): |
a) | Hologen Limited provides committed working capital of [***] to HNAI in order for these funds to be used to commence the implementation of the Phase III Parkinson’s Disease trial in accordance with the Transaction Documents. |
b) | Hologen Limited subscribes to [***] Class B Shares in HNAI at a price of [***] per share pursuant to a subscription agreement in consideration for a total payment of [***], to be satisfied in accordance with clause 10. |
c) | Following Hologen Limited’s subscription for [***] Class B Shares in HNAI per clause 4b), HNAI shall make a payment of [***] to MeiraGTx Neuro I, LLC to be credited towards the upfront payment due under section 8.1 of the Collaboration and License Agreement, to be satisfied in accordance with clause 10. |
d) | HNAI issues [***] Class A Shares in HNAI to MeiraGTx Neuro UK Limited at a share price of [***] per share pursuant to a subscription agreement, so that MeiraGTx Neuro UK Limited holds [***] of the entire issued share capital (Class A and Class B total of [***] shares) in HNAI at the time of Completion. |
5. | Following Completion in accordance with clause 2, and in accordance with the Commitment of clause 11, the Parties agree that Hologen Limited shall purchase from MeiraGTx Neuro UK Limited a total of [***] Class A Shares at a price of [***] per share pursuant to a share purchase agreement; |
and MeiraGTx Neuro UK Limited agrees for such purchased shares to be reclassified from Class A to Class B by HNAI.
6. | In case of any conflicts between this Deed and any provisions or clauses of the HNAI Framework Agreement or the Collaboration and License Agreement, the terms of this Deed prevail. |
MANUFACTURING COMPLETION CONDITIONS AND PURCHASE RIGHTS
7. | For Completion to happen in accordance with clause 2, Hologen Limited shall purchase [***] of the ordinary shares in MeiraGTx Manufacturing Limited from MeiraGTx Limited in consideration for [***] pursuant to a share purchase agreement, payable by Hologen Limited to MeiraGTx Limited (the “Manufacturing Completion Condition”) and to be satisfied in accordance with clause 10. |
8. | Following Completion in accordance with clause 2, and in accordance with the Commitment of clause 11, the Parties agree that Hologen Limited shall purchase from MeiraGTx Limited an additional [***] of the ordinary shares in MeiraGTx Manufacturing Limited from MeiraGTx Limited for a total of [***] pursuant to a share purchase agreement. This purchase is in addition to Hologen’s option to purchase additional ordinary shares in MeiraGTx Manufacturing Limited in the amount of [***], as set out in the Manufacturing Framework Agreement. |
9. | In case of any conflicts between this Deed and any provisions or clauses of the Manufacturing Framework Agreement, the terms of this Deed prevail. |
OFFSETTING AGAINST PREVIOUS PREPAYMENTS TO MEIRAGTX, LLC
10. | The Parties acknowledge and agree that Hologen Limited has already paid an aggregate of $105,000,000 to MeiraGTx, LLC in connection with transactions contemplated by the Framework Agreements and the Collaboration and License Agreement, so that: |
a) | the amount payable by Hologen Limited to HNAI under clause 4b) and then payable by HNAI to MeiraGTx Neuro I, LLC under clause 4c) will be satisfied as follows: HNAI hereby directs Hologen Limited, who hereby directs MeiraGTx Holdings plc to direct, and MeiraGTx Holdings plc hereby agrees to direct, MeiraGTx, LLC to pay [***] to MeiraGTx Neuro I, LLC; and |
b) | the amount payable by Hologen Limited to MeiraGTx Limited under clause 7 will be satisfied as follows: Hologen Limited hereby directs MeiraGTx Holdings plc to direct, and MeiraGTx Holdings plc hereby agrees to direct, MeiraGTx, LLC to pay [***] to MeiraGTx Limited, in satisfaction of Hologen Limited’s obligation to pay such amount to MeiraGTx Limited under clause 7. |
COMMITMENT
11. | Hologen Limited commits to making the purchase payments defined in clauses 5 and 8 by deploying first any funds it raises in excess of the [***] and for no other purpose, provided that, [***]. |
[***] – FURTHER ASSURANCE
12. | [***] |
13. | [***] |
14. | [***] |
15. | [***] |
MISCELLANEOUS
16. | This Deed is subject to the laws of England and Wales, and the courts of England and Wales will have exclusive jurisdiction over any disputes. |
17. | If any term of this Deed is invalid, illegal or incapable of being enforced, all other terms and provisions of this Deed shall nevertheless remain in full force and effect. |
18. | This Deed may not be amended without the prior written consent of each of the parties hereto. |
19. | This Deed may be executed in counterparts each of which shall be deemed to be an original hereof and all of which together evidence the same Deed. This Deed may be executed via email (or transmission of a PDF file) of a counterpart of this Deed. In addition, PDF or electronic signatures of authorised signatories of any party shall be valid and binding and delivery of an email or PDF signature by any party shall constitute due execution and delivery of this Deed. |
SCHEDULE: Contract Definitions
(A) | The Hologen Neuro AI Limited Framework Agreement between (i) Hologen Limited; (ii) Hologen Neuro AI Limited; (iii) MeiraGTx Neuro UK Limited; and (iv) MeiraGTx Holdings plc dated 9 March 2025, as amended (the “HNAI Framework Agreement”); |
(B) | The MeiraGTx Manufacturing Limited Framework Agreement between (i) MeiraGTx Manufacturing Limited; (ii) MeiraGTx Limited; and (iii) Hologen Limited dated 9 March 2025, as amended (the “Manufacturing Framework Agreement” and together with the HNAI Framework Agreement, the “Framework Agreements”); and |
(C) | Collaboration and License Agreement to be entered into at Completion of the HNAI Framework Agreement in the form attached to the HNAI Framework Agreement between (i) Hologen Neuro AI UK Limited; (ii) Hologen Neuro AI Limited; (iii) Hologen Limited; (iv) MeiraGTx Neuro I, LLC; (v) MeiraGTx Neuro UK Limited; and (vi) MeiraGTx Holdings plc (the “Collaboration and License Agreement” and together with the Framework Agreements, the “Transaction Documents”). |