Latest news with #MeiraGTx
Globe and Mail
5 days ago
- Business
- Globe and Mail
MeiraGTx Sales Jump 1,133 Percent
Key Points Revenue (GAAP) increased sharply year-over-year in Q2 2025, but missed GAAP revenue estimates by 43.1% Earnings per share (GAAP) was $(0.48). Cash reserves (GAAP) were $34.4 million as of June 30, 2025, compared to $101.0 million a year earlier. These 10 stocks could mint the next wave of millionaires › MeiraGTx Plc (NASDAQ:MGTX), a biotechnology company specializing in gene therapies for neurodegenerative, ocular, and glandular disorders, released its second quarter results on August 14, 2025. The key news centered on a sizable year-over-year revenue gain (GAAP)—reflecting progress in manufacturing work for major partners—but results fell significantly short of estimates. The company reported GAAP revenue of $3.7 million against an expected $6.48 million. Earnings per share (GAAP) were $(0.48). Despite progress in its research and regulatory pipeline, Ongoing GAAP net losses and a sharp decline in cash reserves underscored continued financial pressures during the period. Metric Q2 2025 Q2 2025 Estimate Q2 2024 Y/Y Change EPS (GAAP) $(0.48) $(0.47) $(0.76) 37.0 % Revenue (GAAP) $3.7 million $6.48 million $0.3 million 1,133.3 % Net Loss (GAAP) $(38.8) million $(48.6) million -20.2 % R&D Expense $33.5 million $34.9 million -4.0 % Cash, Cash Equivalents & Restricted Cash $34.4 million $101.0 million (65.9 %) Source: Analyst estimates for the quarter provided by FactSet. Business Overview and Key Success Factors MeiraGTx Plc develops gene therapies to address complex diseases impacting the nervous system, eyes, and salivary glands. Its core business focuses on designing, manufacturing, and advancing gene-based medicines through clinical trials into commercial use. The company has invested heavily in specialized production sites, giving it tight control over product quality and the ability to scale both in-house and for third parties. Recently, MeiraGTx has centered its strategy around three pillars: developing its clinical pipeline, expanding its manufacturing capabilities, and deepening partnerships with larger pharmaceutical companies. Success depends on pushing drug candidates through clinical milestones, achieving regulatory approvals, and securing milestone payments from collaboration agreements. Control of manufacturing is particularly important in gene therapy, where product customizability and quality are crucial differentiators. MeiraGTx owns specialized facilities in London and Shannon. Quarter in Review: Operations, Pipeline, and Financial Performance Service revenue (GAAP) grew substantially year-over-year in Q2 2025. However, it sharply missed expectations, with GAAP revenue of $3.691 million falling short of analysts' estimate of $6.48 million. Service revenue growth was almost entirely attributed to progress in process performance qualification (PPQ) services. These are detailed tests required to prove the consistency and safety of manufacturing processes for regulatory filings. The company noted, "Service revenue (GAAP) was $3.7 million, compared to $0.3 million a year earlier." The $3.4 million increase was due to progress in PPQ services under the asset purchase agreement with Johnson & Johnson Innovative Medicine. " Despite the year-over-year jump, the revenue figure (GAAP) still fell short of what analysts expected by $2.79 million. Investment in research and development (R&D) remained high, totaling $33.5 million. Research and development expenses were $33.5 million for the three months ended June 30, 2025, compared to $34.9 million a year earlier. This reduction was mainly due to cost transfers associated with partnered programs now led by Johnson & Johnson. Meanwhile, general and administrative costs, covering legal, payroll, and accounting, increased modestly to $12.3 million (GAAP). A favorable foreign currency gain of $8.6 million (GAAP) was recognized, driven by currency movements between the U.S. dollar and British pound/euro, partially offsetting ongoing expenses. The net loss (GAAP) improved to $(38.8) million compared to $(48.6) million a year earlier. MeiraGTx continued making notable strides in clinical and regulatory advancement. The lead programs include AAV2-hAQP1, a gene therapy for radiation-induced xerostomia (chronic dry mouth after head and neck radiation), which finalized agreement with the U.S. Food and Drug Administration (FDA) on its primary outcome and trial design. The therapy is now in a pivotal phase 2 trial, with completion of high-dose enrollment targeted for the fourth quarter and a potential filing for marketing approval by late 2026. Another notable program, AAV-GAD for Parkinson's disease, secured RMAT (Regenerative Medicine Advanced Therapy) designation from the FDA, which can accelerate the review process. Also advancing rapidly, AAV-AIPL1 for childhood blindness (LCA4) produced data published in The Lancet showing all treated children gained vision, with filings for commercial use planned late in the year. The quarter also marked progress in cementing manufacturing leadership. The company's two main production facilities—the London site and the campus in Shannon, Ireland—each completed major regulatory inspections. The Shannon plant added viral vector manufacturing to its license this February, making it the first such facility in Ireland authorized for use in clinical-trial material. These regulatory achievements are vital, as they support MeiraGTx's ability to deliver clinical and commercial-scale supplies for both new medicines and partners' needs. Management highlighted these as key accomplishments, "MeiraGTx has built the most comprehensive manufacturing capabilities in the industry, with 5 facilities globally, including two that are licensed for GMP viral vector production" Strategic partnerships remain central to MeiraGTx's business model. The collaboration with Johnson & Johnson is of particular financial importance, tying future revenue to key manufacturing and regulatory milestones. MeiraGTx will become eligible for up to $285 million in payments upon the first commercial sales of bota-vec in the US and EU, as well as manufacturing tech transfer. The company also entered a major joint venture with Hologen during the quarter. This collaboration, focused on the AAV-GAD program and central nervous system drug development, is backed by $200 million in upfront funding and further potential investments. MeiraGTx will hold a 30 % stake, lead clinical and manufacturing work, and possibly benefit from Hologen's generative artificial intelligence technology to optimize new drug development and manufacturing workflows. Operational metrics show both the progress and ongoing financial risks MeiraGTx faces. Cash, cash equivalents, and restricted cash (GAAP) were $34.4 million as of June 30, 2025, compared to $101.0 million a year earlier. An additional $17 million arrived after quarter-close, and the company expects the rest of the Hologen partnership proceeds later in the year. While management states it has "sufficient capital to fund operating expenses and capital expenditure requirements into 2027" this outlook is dependent on incoming partnership cash and successful achievement of key milestones. On the balance sheet, shareholders' equity declined sharply due to continued operating losses. Outstanding debt stands at $75.0 million, with repayment due in August 2026. There is also an increased share count, from an average of 64.38 million shares a year earlier to 80.59 million, signaling some recent dilution as the company sought new capital. Product Developments, Partnerships, and Regulatory Milestones In its pipeline, MeiraGTx's therapies employ viral vectors, engineered viruses that deliver the therapeutic gene to targeted cells. Its AAV2-hAQP1 program, using a vector to restore salivary gland function, advanced its pivotal Phase 2 trial after aligning with the FDA on trial endpoints. The company expects trial completion in high-dose cohorts by the end of the year, and targets a license application in late 2026. Manufacturing for this program will occur entirely in-house, from viral vector production to clinical supply, leveraging the company's core manufacturing strength. For neurodegenerative diseases, the AAV-GAD therapy for Parkinson's disease received RMAT designation. This status from the FDA can speed both development and review of therapies addressing serious conditions with unmet needs. Backed by positive data across three trials, the company aims to launch a Phase 3 study in the second half of 2025, with AI-enabled imaging support through its partnership with Hologen. The joint venture, Hologen Neuro AI Ltd, will channel $200 million in upfront cash, up to $230 million more for future central nervous system programs, and generative AI capabilities for accelerated drug and manufacturing development. In inherited retinal diseases, MeiraGTx's AAV-AIPL1 candidate for LCA4 (Leber congenital amaurosis type 4) demonstrated substantial success, with all eleven treated children showing improved vision—including gains in children previously blind at birth—based on data published in February 2025. Marketing approval applications in the U.K. and the U.S. are planned for the end of the year. This program also benefits from orphan drug and rare pediatric disease designations, which offer incentives like extended market exclusivity and possibly faster review processes. The company's manufacturing achievements play a critical operational role. Regulatory agencies in the U.K. and Ireland renewed licenses and expanded manufacturing permissions—London for ongoing investigational drug production, and Shannon for viral vector manufacture and release testing. These developments, together with the commercial supply agreement for bota-vec under the Johnson & Johnson collaboration, lay a foundation for future revenue as products move through late-stage trials, regulatory review, and potential launch. Over 90% of inflammation-related adverse events in late-stage trials were mild. Outlook and What to Watch MeiraGTx did not provide formal forward revenue or earnings guidance for the coming quarter or year. Management stated it expects current cash combined with anticipated partnership receipts, particularly from Hologen, will be sufficient to fund operations into 2027. This projection depends critically on the receipt of new partnership cash and successful achievement of major clinical and regulatory milestones. The most financially significant potential inflows are the $285 million in milestone payments from Johnson & Johnson and staged funding from Hologen for development-stage programs and manufacturing advances. Looking ahead, several milestones will determine both scientific and financial progress. Investors should focus on progress in regulatory filings—particularly for AAV-AIPL1 in LCA4 and bota-vec commercialization with Johnson & Johnson. Enrollment and data readouts from the pivotal RIX trial are expected next year, and the AAV-GAD Phase 3 trial is on the calendar for launch in the second half. Any delay or underperformance in these programs could directly impact cash timing and future funding needs. As of the current period, MGTX does not currently pay a dividend. Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted. Where to invest $1,000 right now When our analyst team has a stock tip, it can pay to listen. After all, Stock Advisor's total average return is 1,062%* — a market-crushing outperformance compared to 185% for the S&P 500. They just revealed what they believe are the 10 best stocks for investors to buy right now, available when you join Stock Advisor. See the stocks » *Stock Advisor returns as of August 13, 2025
Newsweek
16-05-2025
- Business
- Newsweek
The New Era of Life Sciences: The Future is Now
When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal dystrophy—had only hope that one day they might see the world. Today, thanks to an investigational gene therapy, we know that the 11 children who participated in the trials can, for the first time in their lives, discern a toy, recognize a human face and even spot a grain of sand. The biotech behind this achievement is MeiraGTx, which has now filed its therapy for approval under exceptional circumstances in the U.K., which would expedite the process. Stories like that of MeiraGTx illustrate the transformative power of gene therapies on patients. More broadly, they highlight the potential for rare diseases R&D to emerge as the principal driver of therapeutic innovation. "Rare diseases have become a critical area for exploring new treatment modalities because, in many cases, there is no existing therapy, or even a model. For patients who have no options, innovative approaches are important," shares Marc Dunoyer, the CEO of Alexion, the rare diseases subsidiary of AstraZeneca. Ironically, the very severity and uncommonness of these illnesses can also be a blessing, as it allows for bolder approaches by both researchers and regulators. Moreover, rare disease drugs have historically had higher approval success rates compared to other drugs. The Tufts Center for the Study of Drug Development found that orphan-designated drugs had a Phase-1-to-approval success rate of 17 percent, compared to 7.9 percent for non-orphan drugs. Benefiting from faster approval pathways and enhanced regulatory incentives, entrepreneurs in the field face better odds of innovating successfully. And when they do, the implications of their therapies go far beyond their original target. "Many of the advances seen today, such as gene editing and RNA-based therapies, originated in rare disease research before moving into larger indications," says Dunoyer. Arcturus Therapeutics, for instance, is addressing cystic fibrosis via a new delivery mechanism. "Our cystic fibrosis program is a prime example, where the ability to inhale mRNA safely could have a huge impact on the pharmaceutical industry. The rare disease field offers a shorter regulatory pathway to approval and a higher likelihood of success. This makes it an attractive space for evaluating next-generation technologies like mRNA therapeutics," shares Arcturus' CEO, Joseph Payne, as the company is expecting results from their Phase 2 trials in 2025. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. We can perform comprehensive genomic profiling to uncover the molecular basis of a tumor. This allows doctors to provide tailored treatments. Over time, this will enable us to move to a future where we understand the molecular drivers for cancer and can deliver truly personalized healthcare. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Whether it is providing clarity for families dealing with rare genetic disorders or enabling rapid and precise cancer diagnoses, our goal is to ensure patients receive the answers they need when they need them. This report has been paid for by a third party. The views and opinions expressed are not those of Newsweek and are not an endorsement of the products, services or persons mentioned. Click here to download the full report
Yahoo
14-03-2025
- Business
- Yahoo
MeiraGTx and Hologen launch AI-backed gene therapy venture
MeiraGTx has struck a deal with Hologen Limited, an artificial intelligence (AI) company specialising in multimodal generative AI models, to advance its Parkinson's disease gene therapy programme. Hologen has committed up to $430m in funding to support the development and commercialisation of AAV-GAD, MeiraGTx's investigational gene therapy for Parkinson's disease. The AI company will provide $200m upfront and allocate up to $230m in additional capital to fully finance the programme. As part of the deal, the companies will set up a newly-formed joint company, Hologen Neuro AI Limited. Hologen Neuro AI will also oversee early-stage clinical programmes targeting the central nervous system, including an investigational gene therapy for genetic obesity dubbed AAV-BDNF. MeiraGTx will retain a 30% ownership stake in Hologen Neuro AI while leading all clinical development and manufacturing activities, as per the 13 March announcement. In addition, MeiraGTx and Hologen will enter into clinical and commercial manufacturing supply agreements, with Hologen also acquiring a minority stake in MeiraGTx's manufacturing subsidiary. MeiraGTx's AAV-GAD programme is currently preparing for a Phase III trial, which is expected to kick off in mid-2025. The gene therapy works by delivering a gene encoding the enzyme responsible for producing the neurotransmitter GABA, which plays a role in motor dysfunction associated with Parkinson's disease. In a Phase II trial (NCT05603312), AAV-GAD demonstrated statistically significant improvements in motor function and quality of life (QoL). The study showed an 18-point improvement on the Unified Parkinson's Disease Rating Scale (UPDRS) Part 3 for patients receiving the high-dose treatment at 26 weeks, along with positive results on the Parkinson's disease Questionnaire (PDQ-39), a quality-of-life assessment. According to MeiraGTx's CEO Alexandria Forbes, Hologen's AI models have already been used to analyse data from the Phase II trial, identifying disease-modifying changes in brain physiology. 'Our collaboration with Hologen has broad significance for MeiraGTx and for drug development for neurological disorders in general. Deploying Hologen's LLMs [large language models] on MeiraGTx's clinical data allows the characterisation of disease modification in the CNS with unprecedented fidelity,' Forbes added. Beyond its Parkinson's programme, MeiraGTx is advancing AAV-AIPL1, an investigational gene therapy for congenital blindness, toward potential regulatory approval in the UK. The New York-headquartered biotech hit the headlines in February 2025 after 11 children who were born blind from birth gained significant vision improvements following treatment with AAV-AIPL1. The company said it is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. In October 2023, Sanofi invested $30m into MeiraGTx through a pricing offering of ordinary shares. MeiraGTx sold 4 million ordinary shares for $7.50 each, granting Sanofi access to data from some of its programmes, including in immunology and inflammation, central nervous disorders and glucagon-like peptide-1 (GLP-1) and other gut peptides for metabolic diseases. "MeiraGTx and Hologen launch AI-backed gene therapy venture" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
24-02-2025
- Health
- Yahoo
MeiraGTx seeks UK approval after gene therapy restores sight in children born blind
MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born blind from birth gained significant vision improvements following treatment. The AAV-AIPL1 therapy is designed to address severe sight impairment caused by mutations in the AIPL1 gene. This condition is a form of retinal dystrophy that leads to severe visual impairment from birth, with affected individuals typically only able to distinguish between light and dark. The gene therapy involves injecting functional copies of the AIPL1 gene into the retina using an adeno-associated viral (AAV) vector, aiming to restore retinal cell function and prevent degeneration. Results from four of the 11 children were published in The Lancet on 21 February. The first four children treated were aged one to three years with severe retinal dystrophy linked to mutations in the AIPL1 gene. Each child received the therapy in one eye through subretinal injection. Outcome measures included visual acuity assessments, functional vision evaluations, visual evoked potentials, and retinal structure imaging. Before treatment, the children's visual acuity was limited to light perception. At an average follow-up of three and a half years post-treatment, the treated eyes showed significant improvement, with visual acuity improving. In contrast, the untreated eyes' visual acuity deteriorated to unmeasurable levels. Additionally, objective tests confirmed enhanced visual function and electrophysiological assessments indicated increased visual cortex activity specific to the treated eyes. Imaging revealed better preservation of retinal structure in treated eyes compared to untreated ones. MeiraGTx's CEO Alexandria Forbes said: 'These improvements extended outside the meaningful effects on vision and result in life-changing benefits in all areas of development including communication, behaviour, schooling, mood, psychological benefits and social integration.' Following these positive outcomes, a second cohort of seven children received treatment in both eyes. All 11 children treated with AAV-AIPL1 have so far exhibited meaningful visual improvements. One child experienced cystoid macular oedema in the treated eye, which partially improved over time and did not hinder the overall visual gains. No other significant safety concerns have been reported. In light of these findings, MeiraGTx has engaged in discussions with the UK's Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit AAV-AIPL1 for expedited approval. The company is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. If approved, AAV-AIPL1 would be eligible for a priority review voucher (PRV). While gene therapies hold promise for rare diseases, commercialisation remains a challenge. High development costs, complex manufacturing processes, and limited patient populations often deter investment. Earlier this month, Italian charity Telethon Foundation submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for a gene therapy targeting Wiskott-Aldrich syndrome after the withdrawal of its commercial partner. This marks the second time the foundation has stepped in to support an abandoned gene therapy, following its efforts with simoladagene autotemcel for ADA-SCID. The foundation claims to be the first charity worldwide to take responsibility for drug production and distribution, highlighting the financial and logistical hurdles in bringing gene therapies to market. The incidence of AIPL1-associated severe retinal dystrophy is estimated to be around one in every million live births. During a call with investors, Forbes stated that the company is exploring all options to ensure global access to AAV-AIPL1, noting 'strategic interest' in the therapy. "MeiraGTx seeks UK approval after gene therapy restores sight in children born blind" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
