Core Viewpoint - MeiraGTx Holdings plc has announced a strategic collaboration with Hologen Limited, which includes a $200 million upfront cash payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at developing therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2][10]. Financial Aspects - MeiraGTx will receive $200 million in upfront cash at closing [2][10] - Hologen will provide up to $230 million in committed capital to fund the development of AAV-GAD for Parkinson's disease and other CNS programs [2][6] - The collaboration is expected to extend MeiraGTx's cash runway and support its internal manufacturing capabilities [7] Joint Venture Details - The joint venture, Hologen Neuro AI Ltd, will be co-owned, with MeiraGTx retaining 30% ownership and leading clinical development and manufacturing [3][10] - Hologen will contribute its generative AI technology to optimize MeiraGTx's manufacturing processes [3][10] - The joint venture will focus on the development of AAV-GAD and other locally delivered CNS genetic medicines [6][10] Clinical Development - MeiraGTx's AAV-GAD program for Parkinson's disease is Phase 3 ready, with positive data reported from a clinical bridging study showing significant improvements in patient outcomes [4][10] - The collaboration aims to enhance the robustness and efficiency of the AAV-GAD Phase 3 clinical study using Hologen's AI technology [5][7] Company Background - MeiraGTx is a clinical-stage genetic medicines company with a broad pipeline, including four late-stage clinical programs targeting various diseases [9] - The company has developed advanced manufacturing capabilities and innovative technologies for gene therapy, including a novel riboswitch gene regulation technology [9]

Core Viewpoint - MeiraGTx Holdings plc has announced a strategic collaboration with Hologen Limited, which includes a $200 million upfront cash payment and the formation of a joint venture, Hologen Neuro AI Ltd, aimed at developing therapies for neurodegenerative diseases, particularly Parkinson's disease [1][2][10]. Financial Terms - MeiraGTx will receive $200 million in upfront cash at closing [2][10] - Hologen will provide up to $230 million in committed capital to fund the development of AAV-GAD for Parkinson's disease and other CNS programs [2][6][10] - MeiraGTx will retain 30% ownership in the joint venture and will lead all clinical development and manufacturing [3][10] - Hologen will own a minority stake in MeiraGTx's manufacturing subsidiary and contribute to its annual funding [3][10] Joint Venture Details - The joint venture, Hologen Neuro AI Ltd, will utilize Hologen's proprietary multi-modal generative foundation models for drug development [2][10] - The joint venture aims to transform the discovery and development of therapies targeting CNS circuitry in neurodegenerative and neuropsychiatric disorders [2][3] Clinical Development - MeiraGTx's AAV-GAD program for Parkinson's disease is Phase 3 ready, with ongoing commercial manufacturing [4][10] - Positive data from a randomized clinical study showed significant improvement in the Unified Parkinson's Disease Rating Scale (UPDRS) and quality of life measures [4][10] Strategic Importance - The collaboration is expected to enhance the robustness and efficiency of the AAV-GAD Phase 3 clinical study [5][7] - The partnership aims to leverage AI technology to elucidate brain circuitry and improve the probability of success in clinical trials [5][7] Company Overview - MeiraGTx is a clinical-stage genetic medicines company with a broad pipeline, including four late-stage clinical programs targeting various diseases [9] - The company has developed comprehensive manufacturing capabilities and innovative technologies for gene delivery and expression control [9]

Core Insights - MeiraGTx Holdings plc announced positive results from a first-in-human interventional study using rAAV8.hRKp.AIPL1 to treat children with AIPL1-associated severe retinal dystrophy, showing significant improvements in visual acuity and functional vision [1][2][5] Study Details - The study involved 11 children, with 4 treated unilaterally and 7 treated bilaterally, all of whom were legally blind at birth and showed improved visual acuity after treatment [1][5][6] - The treatment was administered via subretinal injection, and the outcomes were measured using various methods including visual acuity tests and retinal imaging [3][4] Results - Prior to treatment, children's visual acuities were limited to light perception, but post-treatment, the mean visual acuity improved to 0.9 LogMAR from an equivalent of 2.7 LogMAR [4] - All treated children demonstrated enhanced visual function and structural preservation of the retina, with no serious adverse effects reported [2][4][6] Regulatory Pathway - Following the positive results, MeiraGTx is working with global regulators for expedited approval of rAAV8.hRKp.AIPL1, intending to submit a Marketing Authorization Application (MAA) based on the current data without the need for further clinical trials [7][8] Company Overview - MeiraGTx is a clinical-stage genetic medicines company with a focus on developing treatments for severe genetic conditions, including AIPL1-associated retinal dystrophy, and has capabilities in viral vector production and gene therapy technologies [10]

Core Insights - The article discusses the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1: Service Offerings - Biotech Analysis Central offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - The service is priced at $49 per month, with a discounted annual plan available at $399, representing a 33.50% savings for subscribers [1]. Group 2: Analyst Background - The author of the article is associated with Biotech Analysis Central and emphasizes the importance of informed decision-making for healthcare investors [2]. - The article does not indicate any personal investment positions in the companies discussed, ensuring an unbiased perspective [3].

Core Viewpoint - MeiraGTx has received Rare Pediatric Disease Designation from the FDA for four inherited retinal diseases, highlighting the potential of its proprietary technology platforms in treating severe childhood blinding conditions [1][2][3] Group 1: Rare Pediatric Disease Designation - The FDA granted Rare Pediatric Disease Designation to MeiraGTx's AAV8-RK-RetGC program for treating Leber congenital amaurosis due to GUCY2D mutations, marking the fourth designation received in three months [1] - The other three designations include AAV8-RK-AIPL1 for LCA4 retinal dystrophy, AAV8-RK-BBS10 for Bardet-Biedl syndrome, and AAV5-RDH12 for RDH12 associated retinal dystrophy [1][3] Group 2: Company Strategy and Regulatory Milestones - The receipt of the designation is viewed as a regulatory milestone that underscores the therapeutic potential of the company's technology [2] - The company plans to leverage its manufacturing infrastructure and clinical expertise to expedite the delivery of treatments to affected children [3] Group 3: Overview of Genetic Medicines - AAV8-RK-RetGC targets GUCY2D mutations, which account for 88% of LCA1 cases, leading to early blindness [4] - AAV8-RK-AIPL1 is designed to treat severe forms of LCA through a one-time subretinal injection [5] - AAV8-RK-BBS10 addresses Bardet-Biedl syndrome, affecting approximately 1 in 250,000 people globally, with visual impairment as a primary symptom [6] - AAV5-RDH12 targets RDH12 associated retinal dystrophy, which accounts for 3-10% of LCA cases [7][8] Group 4: Company Profile - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline and end-to-end manufacturing capabilities [9] - The company has developed a proprietary manufacturing platform and focuses on applying genetic medicine to address common diseases with high unmet needs [9]

Group 1 - The biotech forum has seen significant discussions around profitable buy-write or covered call strategies on selected biotech stocks in recent months [1] - MeiraGTx Holdings plc has experienced a stock price increase of over 40% following the release of Parkinson's disease data on October 15, 2024, which is expected to facilitate a pivotal trial [2] - The investing group, The Biotech Forum, offers a model portfolio featuring 12-20 high upside biotech stocks, along with live chat for trade discussions and weekly market commentary [2]

Company Performance - MeiraGTx Holdings PLC reported a quarterly loss of $0.54 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.47, representing an earnings surprise of -14.89% [1] - The company posted revenues of $10.91 million for the quarter ended September 2024, exceeding the Zacks Consensus Estimate by 4,264%, compared to revenues of $5.1 million in the same quarter last year [2] - Over the last four quarters, MeiraGTx has not surpassed consensus EPS estimates and has topped consensus revenue estimates only once [2] Stock Performance - MeiraGTx shares have declined approximately 5.1% since the beginning of the year, while the S&P 500 has gained 25.5% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.48 on $0.25 million in revenues, and -$0.63 on $1.48 million in revenues for the current fiscal year [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which MeiraGTx belongs, is currently ranked in the top 26% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

Financial Performance - The company reported net losses of $39.3 million for Q3 2024, compared to $44.3 million for Q3 2023, and total net losses of $108.4 million for the nine months ended September 30, 2024, compared to $104.2 million for the same period in 2023[176]. - Total operating expenses for Q3 2024 were $51.0 million, up from $37.9 million in Q3 2023, and for the nine months ended September 30, 2024, total operating expenses were $144.6 million, compared to $105.3 million for the same period in 2023[177]. - The net loss for Q3 2024 was $39.3 million, an improvement of $4.97 million compared to a net loss of $44.3 million in Q3 2023[211]. - The company incurred $81.2 million in cash flows from operations for the nine months ended September 30, 2024, with no positive cash flows generated during the period[235]. - The company expects to incur significant expenses and operating losses for the foreseeable future as it advances preclinical and clinical development[235]. Revenue and Expenses - Service revenue for Q3 2024 was $10.9 million, an increase of $10.9 million compared to no revenue in Q3 2023[207]. - License revenue was $0 for Q3 2024, down from $5.1 million in Q3 2023 due to the termination of the Collaboration Agreement[208]. - Cost of service revenue for Q3 2024 was $12.0 million, attributed to progress in PPQ services under the Asset Purchase Agreement[208]. - General and administrative expenses increased to $12.7 million in Q3 2024 from $10.0 million in Q3 2023, primarily due to higher legal and accounting fees[209]. - Research and development expenses totaled $26.2 million in Q3 2024, a decrease of $1.6 million from $27.9 million in Q3 2023[212]. - Research and development expenses for the nine months ended September 30, 2024, were $95.5 million, an increase of $25.4 million from $70.1 million in the same period of 2023[230]. Cash and Funding - The company had cash, cash equivalents, and restricted cash of $125.0 million as of September 30, 2024, along with $3.3 million in receivables from Johnson & Johnson Innovative Medicine[175]. - The company expects to fund its operating expenses and capital expenditures into Q2 2026 based on current cash and anticipated milestone payments, excluding $285.0 million in potential future milestones under the Asset Purchase Agreement[179]. - The company estimates it can fund its operating expenses into the second quarter of 2026 based on current cash and expected milestone payments[237]. - For the nine months ended September 30, 2024, the company reported a net cash used in operating activities of $81.2 million, primarily due to a net loss of $108.4 million[239]. - Net cash provided by financing activities was $50.3 million for the nine months ended September 30, 2024, consisting of $52.6 million from the issuance of ordinary shares[243]. Clinical and Research Developments - AAV-GAD demonstrated a statistically significant 18-point average improvement in the UPDRS Part 3 "off" medication score at Week 26 in the high dose group (p=0.03)[186]. - AAV-AIPL1 has shown meaningful responses in 11 out of 11 children treated, all of whom gained visual acuity following treatment[187]. - The Phase 2 AQUAx2 clinical trial for AAV2-hAQP1 is ongoing, with alignment gained from the FDA for it to be considered a pivotal trial[189]. - The company expects to continue incurring research and development costs for AAV-hAQP1 and AAV-GAD, among other therapeutic areas[199]. - The riboswitch technology platform is progressing with a focus on obesity and metabolic diseases, with first human studies planned for 2025[191]. Legal and Regulatory Matters - The FDA granted Rare Pediatric Disease Designation to three of the company's inherited retinal disease programs, which may provide eligibility for priority review vouchers valued between $100 million to $158 million[188]. - The company is not subject to any material legal proceedings[255]. - There were no changes in internal control over financial reporting that materially affected the company during the quarter ended September 30, 2024[253]. Foreign Currency and Interest - Foreign currency gain was $3.5 million for the three months ended September 30, 2024, compared to a loss of $8.7 million for the same period in 2023, resulting in a change of $12.1 million[216]. - Interest income increased to $1.2 million for the three months ended September 30, 2024, up from $0.5 million in the same period of 2023, reflecting higher interest rates and cash balances[217]. - A 10% unfavorable movement in foreign currency exchange rates could result in an additional foreign currency loss of approximately $7.5 million for the nine months ended September 30, 2024[248]. - The annual interest rate on the company's term loan was 15.35% as of September 30, 2024, with an outstanding balance of $75.0 million[249]. - The company did not hold any foreign currency forward contracts as of September 30, 2024[248].

Exhibit 99.1 C MEIRAGTX MeiraGTx Announces Third Quarter 2024 Financial and Operational Results and Recent Business Updates - Received 3 Rare Pediatric Disease Designations (RPDD) from FDA for each of 3 potential therapies for 3 different rare inherited retinopathies including AAV-AIPL1 - Agreed on pathway with MHRA for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical stud ...

MeiraGTx Holdings' (MGTX) clinical bridging study of its gene therapy candidate, AAV-GAD, for treating Parkinson's disease (PD) met its primary study objective of safety and tolerability. The stock soared 14.9% in response to the news. MGTX's PD Gene Therapy Candidate Betters Sham Treatment The MGT-GAD-025 study of AAV-GAD for PD demonstrated significant and clinically meaningful improvements from baseline for key efficacy endpoints after 26 weeks of treatment. Per the data readout, treatment with the candi ...