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 ...

Company Overview - MeiraGTx Holdings PLC (MGTX) shares increased by 14.9% to $5.33, with a trading volume higher than average, following a 10.5% gain over the past four weeks [1][2] - The company reported positive topline data from its clinical bridging study of AAV-GAD for treating Parkinson's disease, showing superior efficacy over sham treatment at week 26, with the treatment being safe and well-tolerated [2] Financial Performance - The company is expected to report a quarterly loss of $0.47 per share, reflecting a year-over-year change of +36.5%, with revenues projected at $0.25 million, down 95.1% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not continue without trends in earnings estimate revisions [4] Industry Context - MeiraGTx is part of the Zacks Medical - Biomedical and Genetics industry, where Merus N.V. (MRUS) closed 1.6% lower at $51.35, with a return of 3.9% in the past month [4] - Merus has a consensus EPS estimate of -$0.90 for the upcoming report, representing a change of -109.3% from the previous year, and also holds a Zacks Rank of 3 (Hold) [5]

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LONDON and NEW YORK, Oct. 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicine company, today announced a poster presentation at the 2024 Society for Neuroscience Conference (SfN), which is being held from October 5-9, 2024, in Chicago, IL. "We are pleased to share data at this year's Society for Neuroscience Conference on the remarkable efficacy of our AAV-BDNF treatment in diet-induced obesity animal models," said Alexandria Forbes, Ph ...

MeiraGTx Holdings PLC (MGTX) came out with a quarterly loss of $0.76 per share versus the Zacks Consensus Estimate of a loss of $0.31. This compares to loss of $0.53 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -145.16%. A quarter ago, it was expected that this company would post a loss of $0.32 per share when it actually produced a loss of $0.77, delivering a surprise of -140.63%. Over the last four quarters, the company ...

Financial Performance - The net loss attributable to ordinary shareholders for Q2 2024 was $48.6 million, or $0.76 per share, compared to a net loss of $29.6 million, or $0.53 per share in Q2 2023[16]. - Total revenue for the three-month period ended June 30, 2024, was $282,000, a decrease from $3,540,000 in the same period of 2023[21]. - Net loss for the three-month period ended June 30, 2024, was $48,620,000, compared to a net loss of $29,581,000 in the same period of 2023[21]. - Basic and diluted net loss per ordinary share for the three-month period ended June 30, 2024, was $0.76, compared to $0.53 in the same period of 2023[21]. - Total operating expenses for the three-month period ended June 30, 2024, were $46,191,000, an increase from $32,325,000 in the same period of 2023[21]. Cash and Assets - As of June 30, 2024, MeiraGTx reported cash and cash equivalents of approximately $101.0 million, down from $130.6 million as of December 31, 2023[11][12]. - Cash and cash equivalents as of June 30, 2024, were $99,974,000, down from $129,566,000 as of December 31, 2023[22]. - Total current assets decreased to $110,774,000 as of June 30, 2024, from $159,622,000 as of December 31, 2023[22]. - Total liabilities as of June 30, 2024, were $186,612,000, slightly down from $188,567,000 as of December 31, 2023[22]. - The accumulated deficit increased to $623,293,000 as of June 30, 2024, from $554,231,000 as of December 31, 2023[22]. Research and Development - Research and development expenses increased to $34.9 million for Q2 2024, compared to $19.9 million for Q2 2023, primarily due to decreased reimbursements from Johnson & Johnson and increased preclinical program costs[14]. - Research and development expenses for the three-month period ended June 30, 2024, were $34,934,000, up 75% from $19,937,000 in the same period of 2023[21]. - MeiraGTx is progressing its pivotal Phase 2 AQUAx2 clinical trial for radiation-induced xerostomia, with potential BLA filing anticipated in 2026[2][4]. - The company anticipates results from the AAV-GAD trial for Parkinson's disease in Q4 2024, which will inform discussions with global regulatory agencies regarding the Phase 3 program[5][11]. - AAV8-RK-AIPL1 received Innovation Passport Designation from the U.K. for the treatment of LCA4, with all 11 treated children showing significant visual acuity improvements[3][7]. - The riboswitch in vivo delivery platform is showing promising data in obesity and metabolic disease, with plans to present updates later this year[9][10]. - MeiraGTx's Phase 1 AQUAx study demonstrated significant improvements in patient-reported outcomes and saliva production with AAV2-hAQP1 treatment, with no serious adverse events reported[1][3]. Funding and Investments - MeiraGTx announced a $50 million offering of 12.5 million ordinary shares at a price of $4.00 per share, led by Sanofi's $30 million investment[1][2]. - The company expects to receive up to $285 million upon the first commercial sales of bota-vec in the U.S. and EU, along with an additional $15 million in milestone payments later in 2024[6][11]. Shareholder Information - The weighted-average number of ordinary shares outstanding increased to 64,376,396 for the three-month period ended June 30, 2024, from 55,349,534 in the same period of 2023[21].

- Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented at the American Academy of Oral Medicine 2024 annual meeting (AAOM) showed meaningful improvements in patient-reported outcomes and saliva production with AAV2-hAQP1 treatment - Company awarded Innovation Passport Designation by the U.K. Innovative Licensing and Access Pathway Steering Group for AAV8-RK-AIPL1 for the treatment of AIPL1-Leber congenital amaurosis 4 (LCA4) LONDON and NEW YORK, Aug. 12, 2024 (GLOBE NEW ...

Core Viewpoint - MeiraGTx Holdings plc has announced an underwritten offering of 12,500,000 ordinary shares at a price of $4.00 per share, aiming to raise gross proceeds of $50 million before expenses [1][2]. Group 1: Offering Details - The offering is led by Sanofi, which invested $30 million in MeiraGTx through this offering [2]. - Other participants include Perceptive Advisors and various institutional healthcare funds [2]. - BofA Securities is acting as the sole underwriter for the offering, which is expected to close around August 13, 2024, pending customary closing conditions [2]. Group 2: Regulatory Information - The ordinary shares are being offered under an effective shelf registration statement on Form S-3, filed with the SEC on December 21, 2023, and declared effective on December 29, 2023 [3]. - The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC [3].

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company presents its unaudited condensed consolidated financial statements for the period ending March 31, 2024 [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet (in thousands) | Account | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $119,206 | $129,566 | | Total Current Assets | $149,300 | $159,622 | | TOTAL ASSETS | $309,235 | $326,744 | | **Liabilities & Shareholders' Equity** | | | | Total Current Liabilities | $47,543 | $67,078 | | Deferred revenue - related party | $53,331 | $34,017 | | Note payable, net | $72,391 | $72,119 | | TOTAL LIABILITIES | $187,501 | $188,567 | | Total Shareholders' Equity | $121,734 | $138,177 | | TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $309,235 | $326,744 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations (in thousands, except per share data) | Account | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Total revenue | $697 | $3,334 | | General and administrative | $13,147 | $12,772 | | Research and development | $34,322 | $22,322 | | Total operating expenses | $47,469 | $35,094 | | Loss from operations | $(46,772) | $(31,760) | | Gain on sale of nonfinancial assets | $29,018 | $— | | Net loss | $(20,442) | $(30,364) | | Basic and diluted net loss per ordinary share | $(0.32) | $(0.62) | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Statement of Cash Flows (in thousands) | Activity | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(36,741) | $(37,392) | | Net cash provided by (used in) investing activities | $27,340 | $(8,605) | | Net cash used in financing activities | $(1,270) | $(1,231) | | Net decrease in cash, cash equivalents and restricted cash | $(10,671) | $(47,228) | - Cash provided by investing activities in Q1 2024 was primarily driven by **$29.0 million in proceeds from the sale of nonfinancial assets** to Janssen, a significant change from the **$8.6 million used in Q1 2023** for property and equipment purchases[29](index=29&type=chunk)[233](index=233&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Key disclosures cover the Janssen asset sale, liquidity position, share-based compensation, and debt financing agreements - On December 20, 2023, the Company entered into an Asset Purchase Agreement with Janssen, selling assets related to its XLRP-RPGR product candidate (bota-vec) for **$65.0 million upfront** and up to **$350.0 million in future contingent payments**[32](index=32&type=chunk)[33](index=33&type=chunk) - A **$50.0 million milestone payment** from Janssen was achieved during the first quarter of 2024 related to the initiation of the extension study for the Phase 3 LUMEOS clinical trial[34](index=34&type=chunk)[130](index=130&type=chunk) - As of March 31, 2024, the Company had **$120.3 million in cash, cash equivalents, and restricted cash**, which management estimates is sufficient to cover expenses for at least the next twelve months[39](index=39&type=chunk) - The Company has a senior secured financing agreement with Perceptive for an initial **$75.0 million**, with an option for an additional **$25.0 million**, at an annual interest rate of **15.36%** as of March 31, 2024[153](index=153&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=52&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial performance, clinical developments, the Janssen asset sale, and the company's liquidity outlook [Recent Development Highlights and Anticipated Milestones](index=56&type=section&id=Recent%20Development%20Highlights%20and%20Anticipated%20Milestones) - **AAV-hAQP1 (Xerostomia):** The Phase 1 AQUAx study showed **statistically significant improvements** in patient-reported outcomes, and the pivotal Phase 2 study is ongoing with FDA alignment to support a potential BLA filing[175](index=175&type=chunk)[181](index=181&type=chunk) - **AAV-GAD (Parkinson's Disease):** Dosing is complete in the Phase 1 bridging study, with plans to initiate **Phase 3 study design discussions** with regulators in the second half of 2024[182](index=182&type=chunk) - **Riboswitch Platform:** The company is advancing its gene regulation platform for obesity and CAR-T therapies, with data to be presented at an **R&D Day in the second half of 2024**[182](index=182&type=chunk) - **Bota-vec (XLRP):** Received a **$50 million milestone payment** from Janssen in Q1 2024, with an additional **$15 million in near-term milestones** anticipated in 2024[180](index=180&type=chunk)[183](index=183&type=chunk)[189](index=189&type=chunk) [Results of Operations](index=66&type=section&id=Results%20of%20Operations) Comparison of Three Months Ended March 31, 2024 and 2023 (in thousands) | Account | 2024 | 2023 | Change | | :--- | :--- | :--- | :--- | | Total revenue | $697 | $3,334 | $(2,637) | | General and administrative | $13,147 | $12,772 | $375 | | Research and development | $34,322 | $22,322 | $12,000 | | Loss from operations | $(46,772) | $(31,760) | $(15,012) | | Gain on sale of nonfinancial assets | $29,018 | $— | $29,018 | | Net loss | $(20,442) | $(30,364) | $9,922 | - License revenue **decreased by $3.3 million** due to the termination of the Janssen Collaboration Agreement in December 2023[211](index=211&type=chunk) - Research and development expenses **increased by $12.0 million**, primarily because Janssen reimbursements decreased by **$21.9 million** following the collaboration's termination[217](index=217&type=chunk)[219](index=219&type=chunk) - A **gain on sale of nonfinancial assets of $29.0 million** was recognized in Q1 2024 from the allocation of the **$50.0 million** Janssen milestone payment[223](index=223&type=chunk) [Liquidity and Capital Resources](index=70&type=section&id=Liquidity%20and%20Capital%20Resources) - As of March 31, 2024, the company had **$120.3 million in cash, cash equivalents, and restricted cash**[228](index=228&type=chunk) - Management estimates current cash and expected milestones will fund operations and capital expenditures **into the first quarter of 2026**[227](index=227&type=chunk) - Net cash used in operating activities was **$36.7 million** for the three months ended March 31, 2024, compared to **$37.4 million** for the same period in 2023[228](index=228&type=chunk)[229](index=229&type=chunk)[230](index=230&type=chunk) - The company raised gross proceeds of **$1.6 million** in Q1 2024 through its **$100.0 million "at-the-market" (ATM) equity offering program**[122](index=122&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=74&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company details its primary market risk exposures from foreign currency fluctuations and variable-rate debt - **Foreign Currency Risk:** A hypothetical **10% unfavorable movement** in foreign currency exchange rates would have resulted in an additional loss of approximately **$10.0 million** for Q1 2024[239](index=239&type=chunk) - **Interest Rate Risk:** A hypothetical **1% increase in SOFR** would increase annual interest expense by approximately **$0.8 million** due to its variable-rate debt[240](index=240&type=chunk) [Item 4. Controls and Procedures](index=76&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective with no material changes in internal controls - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** as of March 31, 2024[242](index=242&type=chunk) - There were **no changes in internal control over financial reporting** during the quarter that have materially affected, or are reasonably likely to materially affect, internal controls[243](index=243&type=chunk) [PART II. OTHER INFORMATION](index=77&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=77&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not subject to any material legal proceedings - The company is **not subject to any material legal proceedings**[245](index=245&type=chunk) [Item 1A. Risk Factors](index=77&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant risks related to its financial position, development, commercialization, and intellectual property [Risks Related to Financial Position and Need for Additional Capital](index=77&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) - The company has a history of significant losses, with an **accumulated deficit of $574.7 million** as of March 31, 2024, and expects continued losses[247](index=247&type=chunk) - The company **will require additional capital** to fund operations, and failure to raise it could force delays or reductions in R&D programs[258](index=258&type=chunk) - There is **no guarantee of receiving future milestone payments** from the Janssen Asset Purchase Agreement, which could adversely affect the company's financial position[253](index=253&type=chunk) [Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory Approval](index=89&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%2C%20Clinical%20Testing%2C%20Manufacturing%20and%20Regulatory%20Approval) - Gene therapy is a novel field with an **uncertain and evolving regulatory landscape**, making it difficult to predict the time and cost of obtaining approval[280](index=280&type=chunk) - Clinical trials are **expensive, time-consuming, and have uncertain outcomes**, with potential for substantial delays[286](index=286&type=chunk)[289](index=289&type=chunk) - The company is subject to **significant manufacturing regulations (GMP)**, and any failure to meet requirements could delay development and commercialization[323](index=323&type=chunk)[329](index=329&type=chunk) [Risks Related to Commercialization](index=136&type=section&id=Risks%20Related%20to%20Commercialization) - The company faces **significant competition** from large pharmaceutical and biotechnology companies[401](index=401&type=chunk)[403](index=403&type=chunk) - Successful commercialization depends on obtaining **adequate coverage and reimbursement** from government and private insurers, which is uncertain[404](index=404&type=chunk) - The company **lacks sales, marketing, and distribution infrastructure** and may not be successful in establishing these capabilities[416](index=416&type=chunk) [Risks Related to Intellectual Property](index=152&type=section&id=Risks%20Related%20to%20Intellectual%20Property) - The company **depends on technology licensed from others** and could lose its rights if it fails to comply with license obligations[450](index=450&type=chunk) - The ability to compete depends on **obtaining and maintaining patent protection**, but the patent process is uncertain and may be challenged[452](index=452&type=chunk) - Third parties may assert **patent infringement claims**, which could result in substantial costs and block commercialization[460](index=460&type=chunk) [Item 5. Other Information](index=189&type=section&id=Item%205.%20Other%20Information) The company discloses the adoption of a Rule 10b5-1 trading plan by its President and CEO - On March 21, 2024, President and CEO Alexandria Forbes, Ph.D., adopted a **Rule 10b5-1 trading plan** for the sale of up to **190,000 ordinary shares**[552](index=552&type=chunk) [Item 6. Exhibits](index=190&type=section&id=Item%206.%20Exhibits) The section lists all exhibits filed with the report, including officer certifications and XBRL data

Exhibit 99.1 MeiraGTx Reports First Quarter 2024 Financial and Operational Results - Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia (RIX) presented in an oral session at the American Academy of Oral Medicine 2024 annual meeting (AAOM) April 17-20, 2024 - Received $50 million milestone following initiation of the extension study for the Phase 3 LUMEOS clinical trial for botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLR ...