MeiraGTx Reports First Quarter 2024 Financial and Operational Results
MeiraGTx reported positive data from the Phase 1 AQUAx study, received a $50 million milestone for the Phase 3 LUMEOS clinical trial, and remains on track with clinical development for various programs. The company highlighted significant progress in gene therapy programs for xerostomia and Parkinson's disease. The riboswitch platform shows promising results in metabolic disease models. Financially, the company had cash and cash equivalents of $120.3 million as of March 31, 2024, and anticipates having sufficient capital until the first quarter of 2026.
Positive data from the Phase 1 AQUAx study in radiation-induced xerostomia presented at AAOM
Received $50 million milestone for Phase 3 LUMEOS clinical trial for XLRP treatment
Clinical development milestones met and remain on track for various programs
Gene therapy programs for xerostomia and Parkinson's disease progressing well
Riboswitch platform shows promising results in metabolic disease models
Financially, the company had cash and cash equivalents of $120.3 million as of March 31, 2024
Decrease in service revenue compared to the previous year
No license revenue for the three months ended March 31, 2024
Increase in research and development expenses
Foreign currency loss of $0.5 million for the three months ended March 31, 2024
Net loss attributable to ordinary shareholders for the quarter ended March 31, 2024
Insights
The first quarter financial results for MeiraGTx Holdings plc present a robust pipeline progression, particularly the achievement of clinical milestones in gene therapy programs. The initiation of a pivotal Phase 2 study and discussions with the FDA are positive indicators that the company is moving closer to potential commercialization. Moreover, the receipt of a
Receipt of additional near-term milestones, alongside the
The clinical advancements for MeiraGTx indicate progress in gene therapy treatments which could meet significant unmet medical needs. Particularly, the Phase 1 AQUAx study for radiation-induced xerostomia (RIX) suggests promising results, with the therapy showing potential life-changing improvements. This could represent a breakthrough for patients with no current treatment options, addressing a market of over 170,000 U.S. patients. Such advancements not only offer therapeutic value but also demonstrate the company's capability in developing gene therapies.
Moreover, the Phase 3 LUMEOS trial for bota-vec in XLRP, in collaboration with Janssen, is progressing towards data revelation by year-end. The success of this trial could influence the company’s valuation significantly. It's worth noting, however, that the reliance on a single product for near-term success can represent a risk to investors. The technology platforms being developed, like the riboswitch gene regulation, could redefine the therapeutic approach to metabolic diseases and the potential for these technologies to be applied across a range of disorders adds strategic value to the company’s long-term prospects.
- 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
- Clinical development milestones met and remain on track for the rest of the year for AAV-hAQP1 for RIX, bota-vec for XLRP, and AAV-GAD for Parkinson’s disease
LONDON and NEW YORK, May 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the first quarter ended March 31, 2024, and provided a corporate update.
“MeiraGTx’s first quarter was highlighted by significant progress in our wholly-owned late stage clinical programs, including our pivotal xerostomia program and Parkinson’s program as well as our riboswitch platform,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “At this year’s AAOM meeting in April, extremely encouraging data was presented from the Phase 1 AQUAx study, showing that treatment with AAV2-hAPQ1 resulted in significant improvements in 3 different patient reported outcomes (PROs) as well as unprecedented improvement in the production of saliva. We continue to hear from investigators that our therapy is ‘life changing’ for their patients fortunate enough to have had access to the treatment. Dosing in our pivotal Phase 2 AQUAx2 trial is ongoing, and we are pleased to have aligned with FDA on requirements for this Phase 2 study to be considered pivotal to support potential BLA filing for this significant unmet need.”
Dr. Forbes continued: “Having completed dosing in our bridging study for AAV-GAD manufactured at MeiraGTx, we remain on track to discuss Phase 3 study design with global regulators for Parkinson’s disease in the second half of 2024. In addition, we continue to anticipate data from the large, multi-center Phase 3 study of bota-vec for XLRP-RPGR in collaboration with Janssen Pharmaceuticals, Inc. (Janssen) towards the end of this year, setting up the potential
“We are particularly excited by the recent data we have seen with our riboswitch platform for in vivo delivery of short acting metabolic peptides,” said Dr. Forbes. “The data in metabolic disease models are striking and the approach highly differentiated. By precisely controlling the timing and levels of production of native forms of short acting peptides in the body, we demonstrate significant increase in efficacy versus the same persistently active peptide combinations. This degree of improvement in efficacy is consistent with the data we have demonstrated with CAR-T, where precise periodic control of CAR levels transforms the CAR-T cell profile to that of naïve T-cells, prevents exhaustion and increases efficacy in vivo by three to four fold over CAR-T cells containing unregulated CAR.”
“Our technology also allows the in vivo production of novel peptides, for example those that directly regulate muscle mass and fat accumulation. The ability to deliver any combination of peptides in a controlled physiological timeframe with oral small molecules has the potential to address many of the issues that arise with current therapies for obesity and diabetes, including: significantly enhanced efficacy, improved tolerability, improved muscle mass and prevention of the regain of fat. Importantly, our technology also decreases the manufacturing burden for peptides since the body produces the peptides in vivo in response to oral small molecules.”
Dr. Forbes continued, “We are prioritizing moving one or more of these incretin, myokine and adipokine combinations towards the clinic to provide a completely differentiated approach to addressing obesity and metabolic disease.”
Recent Development Highlights and Anticipated Milestones
Phase 1 AQUAx Study of AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:
Grade 2/3 radiation-induced xerostomia (RIX) is a severely debilitating consequence of radiation treatment for head and neck cancer that affects approximately 30
An oral presentation of the Phase 1 AQUAx study, entitled “Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction – The AQUAx Study” was delivered at the Academy of Oral Medicine 2024 annual meeting that took place April 17-20, 2024.
Summary of Findings presented at AAOM:
- No treatment-related serious adverse events or dose-limiting toxicities were reported, and all participants completed the study.
- The 3 different PRO instruments showed statistically significant improvements by Day 30 that were maintained through Month 12:
- At Month 12, the average Total XQ Score improved by 17 points (
39.5% ) from baseline and 16 of 24 participants reported an improvement of ≥8 points - At Month 12, the MDASI-HN-DM score improved by 2.7 points (
42.2% ) from baseline - At Month 12, the average improvement in GRCQ Score was 3.8
- Across the PROs, bilaterally-treated participants reported greater improvement than those treated unilaterally
- At Month 12, the average Total XQ Score improved by 17 points (
- At Month 12, the Unstimulated Whole Saliva Flow Rate increased from baseline by
112.8%
Phase 2 AQUAx2 Study:
- The Company continues to enroll and dose participants at multiple sites in the U.S. and Canada in the Phase 2 AQUAx2 randomized, double-blind, placebo-controlled study.
- The Company aligned with the FDA on requirements for the ongoing Phase 2 AQUAx2 clinical trial for Grade 2/3 radiation-induced xerostomia to be considered pivotal to support potential BLA filing.
- Received Clinical Trial Authorization approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for AQUAx2 study in the UK. Site activations are ongoing.
AAV-GAD for the Treatment of Parkinson’s Disease:
Parkinson’s is the second most common neurodegenerative disease after Alzheimer’s with approximately 90,000 patients diagnosed annually in the U.S. Most Parkinson’s patients respond to dopamine replacement therapy, however, after about 5 years, even higher doses of dopamine no longer manage the motor symptoms, leaving little effective treatment for this large population of patients. MeiraGTx’s gene therapy treatment for Parkinson’s involves the delivery of a very small dose of AAV-GAD encoding the enzyme that converts the activating neurotransmitter glutamate to the calming neurotransmitter GABA, to the specific nucleus of the brain targeted that blocks signaling to the motor cortex. We have demonstrated that localized treatment with AAV-GAD leads to a change in circuitry to the motor cortex, resulting in alleviation of motor symptoms. This is a one-time treatment with a small dose of viral vector that has a low cost of goods addressing a significant need in a large patient population.
- The Company completed dosing patients in the Phase 1 trial of AAV-GAD under a new IND with material manufactured in its GMP facility in London, United Kingdom using MeiraGTx’s proprietary production process.
- The AAV-GAD trial is a three-arm randomized Phase 1 clinical bridging study with subjects randomized to sham control or one of two doses of AAV-GAD.
- The objective of the AAV-GAD trial (NCT05603312) is to evaluate the safety and tolerability of AAV-GAD when delivered to the subthalamic nucleus (STN) of patients with Parkinson’s disease.
- The Company intends to initiate Phase 3 study design discussions with global regulatory agencies in the second half of 2024.
Riboswitch Gene Regulation Technology Platform:
MeiraGTx’s riboswitch technology allows for repeatable, long-term delivery of any messenger RNA (mRNA) from the DNA template encoding any peptide or protein on activation by bespoke orally delivered small molecules.
- For obesity and metabolic disease, the Company has successfully delivered multiple combinations of gut peptides in vivo including GLP-1, GIP, PYY, Glucagon, and Oxyntomodulin as well as novel myokine and adipokine peptides that drive muscle metabolism and fat storage, via the riboswitch platform. The technology allows daily dosing with a small molecule to drive production of peptides within the body in physiologically relevant combinations and timing. This provides a platform for addressing not just weight loss via reduced appetite, but also muscle strength, fat metabolism, and cardiovascular health and neurodegenerative disorders in metabolic disease, with daily oral small molecules.
- In CAR-T for both oncology and autoimmune disease, precise control of levels and timing of the CAR has demonstrated a significant impact on CAR-T efficacy, with a 3-4 fold improvement in in vivo potency of T-cells with regulated CAR compared to the currently approved CAR-T with unregulated constitutively active CAR. In addition, MeiraGTx’s regulated CAR-T display a normal naïve T-cell profile, lacking exhaustion markers and retaining proliferation and killing ability in contrast to CAR-T with unregulated constitutive CAR expression.
- The Company intends to present data from its riboswitch gene regulation technology platform at an R&D Day in the second half of 2024.
Bota-vec for the Treatment of XLRP:
- In March, the Company received a
$50 million milestone after initiation of the extension study for the Phase 3 LUMEOS clinical trial for bota-vec for the treatment of XLRP. - MeiraGTx anticipates receiving an additional
$15 million in near-term milestone payments later in 2024. - The Company will receive up to a further
$285 million upon first commercial sales of bota-vec in the U.S. and EU and for manufacturing technology transfer. - MeiraGTx also entered into a commercial supply agreement with Janssen for bota-vec manufacturing, which the Company anticipates will generate additional revenue during the product launch.
AAV-AIPL1 Specials License in the UK:
- Meaningful responses have been observed in 8 out of 8 LCA4 children treated to date with AAV-AIPL1. All children were treated between 1 and 3 years old, all were blind on treatment, and all gained visual acuity 4 or more weeks following treatment.
- Given the positive results under the Specials License, MeiraGTx has engaged with regulatory agencies in the UK to enable the Company to make this intervention more widely available to the LCA4 patient population globally.
- The Company’s AAV-AIPL1 for the treatment of inherited retinal dystrophy due to defects in the AIPL1 gene has been granted orphan drug designation by the FDA and orphan designation by the European Commission and the Company anticipates receiving a Rare Pediatric Disease Designation (RPDD).
As of March 31, 2024, MeiraGTx had cash and cash equivalents of approximately
Financial Results
Cash, cash equivalents and restricted cash were
Service revenue was
There was no license revenue for the three months ended March 31, 2024, compared to
General and administrative expenses were
Research and development expenses for the three months ended March 31, 2024, were
Foreign currency loss was
Interest income was
Interest expense was
Gain on sale of nonfinancial assets was
Net loss attributable to ordinary shareholders for the quarter ended March 31, 2024, was
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has an internally developed manufacturing platform process, internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. MeiraGTx has core capabilities in viral vector design and optimization and a potentially transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on delivery of metabolic peptides including GLP-1, GIP, Glucagon and PYY using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. Although initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains great.
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 our product candidate development, our ability to manufacture product candidates, potential milestone payments and the achievement of such milestones, including the receipt of such milestone payments and the impact on our cash runway, and our pre-clinical and clinical data, reporting of such data and the timing of results of data and regulatory matters, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate” 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 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, 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
| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except share and per share amounts) | ||||||||
| For the Three-Month Periods Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Revenues: | ||||||||
| Service revenue - related party | $ | 697 | $ | — | ||||
| License revenue - related party | — | 3,334 | ||||||
| Total revenue | 697 | 3,334 | ||||||
| Operating expenses: | ||||||||
| 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 | ) | ||||
| Other non-operating income (expense): | ||||||||
| Foreign currency (loss) gain | (535 | ) | 3,857 | |||||
| Interest income | 1,097 | 545 | ||||||
| Interest expense | (3,250 | ) | (3,060 | ) | ||||
| Gain on sale of nonfinancial assets | 29,018 | — | ||||||
| Fair value adjustment | — | 54 | ||||||
| Net loss | (20,442 | ) | (30,364 | ) | ||||
| Other comprehensive loss: | ||||||||
| Foreign currency translation loss | (1,691 | ) | (2,353 | ) | ||||
| Comprehensive loss | $ | (22,133 | ) | $ | (32,717 | ) | ||
| Net loss | $ | (20,442 | ) | $ | (30,364 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (0.32 | ) | $ | (0.62 | ) | ||
| Weighted-average number of ordinary shares outstanding | 64,065,895 | 48,638,151 | ||||||
| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands, except share and per share amounts) | ||||||||
| March 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| ASSETS | ||||||||
| CURRENT ASSETS: | ||||||||
| Cash and cash equivalents | $ | 119,206 | $ | 129,566 | ||||
| Accounts receivable - related party | 10,915 | 10,138 | ||||||
| Prepaid expenses | 5,076 | 5,625 | ||||||
| Tax incentive receivable | 13,171 | 13,277 | ||||||
| Other current assets | 932 | 1,016 | ||||||
| Total Current Assets | 149,300 | 159,622 | ||||||
| Property, plant and equipment, net | 111,412 | 115,896 | ||||||
| Intangible assets, net | 1,038 | 1,118 | ||||||
| Restricted cash | 1,059 | 1,083 | ||||||
| Other assets | 1,138 | 1,917 | ||||||
| Equity method and other investments | 6,766 | 6,766 | ||||||
| Right-of-use assets - operating leases, net | 14,835 | 15,910 | ||||||
| Right-of-use assets - finance leases, net | 23,687 | 24,432 | ||||||
| TOTAL ASSETS | $ | 309,235 | $ | 326,744 | ||||
| LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
| CURRENT LIABILITIES: | ||||||||
| Accounts payable | $ | 21,223 | $ | 16,042 | ||||
| Accrued expenses | 17,353 | 42,639 | ||||||
| Lease obligations, current | 4,188 | 4,193 | ||||||
| Deferred revenue - related party, current | 3,772 | 2,926 | ||||||
| Other current liabilities | 1,007 | 1,278 | ||||||
| Total Current Liabilities | 47,543 | 67,078 | ||||||
| Deferred revenue - related party | 53,331 | 34,017 | ||||||
| Lease obligations | 11,796 | 12,952 | ||||||
| Asset retirement obligations | 2,440 | 2,401 | ||||||
| Note payable, net | 72,391 | 72,119 | ||||||
| TOTAL LIABILITIES | 187,501 | 188,567 | ||||||
| COMMITMENTS AND CONTINGENCIES (Note 11) | ||||||||
| SHAREHOLDERS' EQUITY: | ||||||||
| Ordinary Shares, authorized, 64,298,691 and 63,601,015 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | 2 | 2 | ||||||
| Capital in excess of par value | 699,531 | 693,841 | ||||||
| Accumulated other comprehensive loss | (3,126 | ) | (1,435 | ) | ||||
| Accumulated deficit | (574,673 | ) | (554,231 | ) | ||||
| Total Shareholders' Equity | 121,734 | 138,177 | ||||||
| TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 309,235 | $ | 326,744 | ||||
FAQ
What positive data was reported from the Phase 1 AQUAx study?
What milestone did MeiraGTx receive for the Phase 3 LUMEOS clinical trial?
What financial results did MeiraGTx report as of March 31, 2024?
What programs is MeiraGTx focusing on in terms of gene therapy?
What are the highlights of MeiraGTx's riboswitch platform?
