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]

Regulatory Designations and Applications - MeiraGTx received three Rare Pediatric Disease Designations (RPDD) from the FDA for its AAV8-RK-AIPL1, AAV8-RK-BBS10, and AAV5-RDH12 programs, each targeting inherited retinal diseases[1]. - The company plans to submit a Marketing Authorization Application (MAA) for AAV-AIPL1 in the UK under exceptional circumstances, with discussions ongoing with the FDA for a similar pathway in the U.S.[1][6]. Clinical Study Results - In a randomized, sham-controlled clinical study of AAV-GAD for Parkinson's disease, a statistically significant 18-point average improvement in UPDRS Part 3 "off" score was observed in the high dose group at Week 26 (p=0.03)[4]. - The PDQ-39 quality of life score improved by 8 points in the high dose AAV-GAD group (p=0.02) and by 6 points in the low dose group (p=0.04), with no significant change in the sham group[5]. Financial Performance - As of September 30, 2024, MeiraGTx had cash and cash equivalents of approximately $122.9 million, down from $130.6 million as of December 31, 2023[11][12]. - Service revenue for the three months ended September 30, 2024, was $10.9 million, attributed to process performance qualification services under the asset purchase agreement with Johnson & Johnson[12]. - Research and development expenses for the quarter were $26.2 million, a decrease from $27.9 million in the same period last year, primarily due to reduced manufacturing costs[16]. - The net loss attributable to ordinary shareholders for the quarter was $39.3 million, or $0.55 per share, an improvement from a net loss of $44.3 million, or $0.74 per share, in the prior year[20]. - Foreign currency gain was $3.5 million for the quarter, a significant improvement compared to a loss of $8.7 million in the same period last year, reflecting a change of $12.1 million[17]. - Total revenue for the three-month period ended September 30, 2024, was $10.91 million, compared to $5.10 million for the same period in 2023, representing a 114% increase[27]. - Research and development expenses for the three-month period ended September 30, 2024, were $26.24 million, slightly down from $27.86 million in the same period of 2023, a decrease of 5.8%[27]. - The net loss for the three-month period ended September 30, 2024, was $39.33 million, an improvement from a net loss of $44.30 million in the same period of 2023, reflecting an 11% reduction in losses[27]. - Total current assets decreased to $139.05 million as of September 30, 2024, down from $159.62 million at the end of December 31, 2023, a decline of 12.9%[28]. - Total liabilities increased to $203.75 million as of September 30, 2024, compared to $188.57 million at the end of December 31, 2023, an increase of 8%[28]. - The company reported cash and cash equivalents of $122.87 million as of September 30, 2024, down from $129.57 million at the end of December 31, 2023, a decrease of 5.2%[28]. - The weighted-average number of ordinary shares outstanding increased to 71,633,150 for the three-month period ended September 30, 2024, compared to 59,526,642 for the same period in 2023, an increase of 20.3%[27]. Research and Development Focus - The company has developed a proprietary manufacturing platform aimed at increasing yield and quality, with a focus on gene regulation technologies[24]. - MeiraGTx is focusing on expanding its riboswitch platform for metabolic peptides and cell therapy applications in oncology and autoimmune diseases[24]. - The company anticipates significant growth opportunities in addressing unmet needs in common diseases through its genetic medicine technologies[24].

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]

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

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].