MeiraGTx Reports Fourth Quarter and Full Year 2021 Financial and Operational Results
MeiraGTx Holdings plc (MGTX) reported key advancements in its clinical pipeline, including the initiation of Phase 3 trials for botaretigene sparoparvovec in collaboration with Janssen Pharmaceuticals, resulting in a $30 million milestone payment. The company also shared positive preliminary results from the Phase 1 AQUAx trial for AAV-hAQP1, aimed at treating radiation-induced xerostomia, where 6 of 7 patients showed symptom improvement. As of December 31, 2021, MeiraGTx had cash reserves of $137.7 million, ensuring operational funding through mid-2023. However, net losses increased to $79.6 million.
- Received $30 million milestone payment from Janssen Pharmaceuticals.
- Initiated Phase 3 Lumeos trial for botaretigene sparoparvovec.
- Positive preliminary data from AAV-hAQP1 Phase 1 trial with 6 of 7 patients showing symptom improvement.
- Net loss increased to $79.6 million, up from $58 million in 2020.
- Cash and cash equivalents decreased from $209.5 million in 2020 to $137.7 million.
- Phase 3 Lumeos Clinical Trial of botaretigene sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) Enrolling and Dosing Patients
- Received
- Positive Preliminary Clinical Data from AQUAx AAV-hAQP1 Phase 1 Trial for Treatment of Grade 2/3 Radiation-Induced Xerostomia Announced
- First In Vivo Data from Riboswitch Gene Regulation Platform at R&D Day Presented
LONDON and NEW YORK, March 10, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the fourth quarter and full-year ended December 31, 2021, and provided a corporate update.
“MeiraGTx had an exceptional year in 2021, marked by advancements in all aspects of our pipeline and technology platforms,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We initiated our first pivotal study with our partner Janssen for the treatment of XLRP using drug product manufactured in-house at our cGMP facility using our proprietary manufacturing process, we announced positive preliminary clinical data from our wholly-owned grade 2/3 xerostomia program and we presented the first in vivo data from our transformative gene regulation platform.”
“This progress is enabled by the broad end-to-end capabilities we have focused on building at the Company since inception,” continued Dr. Forbes. “We have established leading capabilities in vector engineering and optimization, viral vector production process development, manufacturing and quality, all while developing our synthetic biology platform which for the first time allows precise and specific control of genetic medicines using novel orally dosed small molecules.”
Recent Development Highlights and Anticipated 2022 Milestones
Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of XLRP:
- MeiraGTx, in collaboration with Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, is dosing and enrolling patients in the Phase 3 Lumeos clinical trial of botaretigene sparoparvovec.
- MeiraGTx received a
$30 million payment from Janssen for a clinical milestone in the Phase 3 Lumeos trial of botaretigene sparoparvovec.
AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:
- MeiraGTx reported positive preliminary data from the Phase 1 AQUAx clinical trial in December 2021.
- Clinically meaningful improvements in xerostomia symptoms and disease burden in two validated Patient-Reported Outcome (PRO) measures were demonstrated.
- 6 of the 7 participants through 90-day assessments following treatment achieved clinically meaningful improvement in symptoms using both the McMaster Global Rate of Change PRO and the Xerostomia Questionnaire.
- One participant with the maximum response evaluable at 12 months has now reached 24 months and the same level of response/xerostomia symptom improvement was maintained.
- AAV-hAQP1 appears safe and well-tolerated at each dose tested.
- MeiraGTx completed treatment of patients in all four cohorts of the unilateral dose escalation (n=12) Phase 1 AQUAx trial in the fourth quarter of 2021.
- Four cohorts of dose escalating bilateral treatment (n=12) were added to the protocol to further assess potential efficacy. Treatment of all bilateral patients was completed in the first quarter of 2022.
- Based on the safety and efficacy profile of AAV-hAQP1 in the AQUAx Phase 1 study and regulatory precedent, the Company intends to initiate a randomized, double-blind, placebo-controlled Phase 2 study evaluating two active doses of AAV-hAQP1 by the end of 2022.
Riboswitch Gene Regulation Platform:
- MeiraGTx presented data from its proprietary riboswitch gene regulation platform in December 2021 demonstrating regulation of multiple therapeutic genes in multiple tissues in vitro and in vivo, as well as in vivo models showing dose responsive physiological effects indicative of potential therapeutic activity.
- Transformative riboswitch technology platform has unprecedented dynamic range of greater than 5,000-fold.
- Proprietary technology platform allows precise and specific control of gene therapy expression levels via dose-response to orally delivered small molecules.
- MeiraGTx has developed a library of novel small molecules that tightly regulate aptamer driven cassettes with drug properties designed specifically for the regulation of different genes in different tissues.
- This technology is applicable to the control of any gene in the context of any vector, including both gene editing and RNA editing.
AAV-GAD for the Treatment of Parkinson’s Disease:
- MeiraGTx anticipates filing an Investigational New Drug application (IND) in the coming weeks, with material that has been manufactured with our proprietary manufacturing process at the MeiraGTx cGMP manufacturing facility in London.
AAV-CNGB3 and AAV-CNGA3 for the Treatment of Achromatopsia (ACHM):
- With partner Janssen, MeiraGTx expects to initiate later stage clinical studies in 2022 for both AAV-CNGB3 and AAV-CNGA3 for the treatment of ACHM associated with mutations in the CNGB3 and CNGA3 genes.
Manufacturing and Process Development:
- MeiraGTx successfully manufactured cGMP material for six different clinical programs, including three for Janssen-partnered programs, highlighting the breadth and expertise of the Company’s manufacturing and quality infrastructure.
For more information related to our clinical trials, please visit www.clinicaltrials.gov
As of December 31, 2021, MeiraGTx had cash and cash equivalents of approximately
The Company believes it will have sufficient capital to fund operating expenses and capital expenditure requirements through the second quarter of 2023.
Financial Results
Cash and cash equivalents were
License revenue was
General and administrative expenses were
Research and development expenses for the years ended December 31, 2021 and 2020 were as follows (in millions):
| 2021 | 2020 | Change | ||||||||||
| Gross research and development expenses | $ | 141.5 | $ | 96.6 | $ | 44.9 | ||||||
| Janssen reimbursements | (69.0 | ) | (57.4 | ) | (11.6 | ) | ||||||
| Tax incentive reimbursement | (5.4 | ) | (5.3 | ) | (0.1 | ) | ||||||
| Research and development expenses | $ | 67.1 | $ | 33.9 | $ | 33.2 | ||||||
Gross research and development expenses for the year ended December 31, 2021 increased
Reimbursements under the Janssen collaboration agreement for the year ended December 31, 2021 increased
Tax incentive reimbursement for the year ended December 31, 2021 increased
Foreign currency loss was
Interest income was
Net loss attributable to ordinary shareholders for the year ended December 31, 2021 was
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, and a transformative gene regulation platform technology which allows tight, dose responsive control of gene expression by oral small molecules with dynamic range that may exceed 5,000-fold. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring, and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: ocular, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.
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 and anticipated 2022 milestones regarding our pre-clinical and clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words “expect,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “should,” “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, identify additional and develop existing product candidates, successfully execute strategic 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 the COVID-19 pandemic 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 Annual Report on Form 10-K for the year ended December 31, 2021, 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
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(in thousands, except share and per share amounts)
| For the Years Ended December 31, | ||||||||
| 2021 | 2020 | |||||||
| License revenue - related party | $ | 37,701 | $ | 15,563 | ||||
| Operating expenses: | ||||||||
| General and administrative | 43,765 | 44,207 | ||||||
| Research and development | 67,128 | 33,910 | ||||||
| Total operating expenses | 110,893 | 78,117 | ||||||
| Loss from operations | (73,192 | ) | (62,554 | ) | ||||
| Other non-operating income (expense): | ||||||||
| Foreign currency (loss) gain | (6,293 | ) | 3,426 | |||||
| Interest income | 212 | 1,275 | ||||||
| Interest expense | (288 | ) | (139 | ) | ||||
| Net loss | (79,561 | ) | (57,992 | ) | ||||
| Other comprehensive income (loss): | ||||||||
| Foreign currency translation gain (loss) | 2,226 | (3,103 | ) | |||||
| Total comprehensive loss | $ | (77,335 | ) | $ | (61,095 | ) | ||
| Net loss | $ | (79,561 | ) | $ | (57,992 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (1.80 | ) | $ | (1.54 | ) | ||
| Weighted-average number of ordinary shares outstanding | 44,139,655 | 37,724,189 | ||||||
MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES
CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
| December31, | December31, | |||||||
| 2021 | 2020 | |||||||
| ASSETS | ||||||||
| CURRENT ASSETS: | ||||||||
| Cash and cash equivalents | $ | 137,703 | $ | 209,520 | ||||
| Accounts receivable - related party | 22,384 | 38,479 | ||||||
| Prepaid expenses | 8,102 | 7,082 | ||||||
| Tax incentive receivable | 12,634 | 12,930 | ||||||
| Other current assets | 2,420 | 4,565 | ||||||
| Total Current Assets | 183,243 | 272,576 | ||||||
| Property, plant and equipment, net | 75,860 | 44,042 | ||||||
| Intangible assets, net | 1,791 | 2,119 | ||||||
| In-process research and development | 783 | 852 | ||||||
| Other assets | 1,404 | 1,026 | ||||||
| Equity method and other investments | 6,656 | — | ||||||
| Right-of-use assets - operating leases, net | 22,782 | 21,486 | ||||||
| Right-of-use assets - finance leases, net | 27,645 | 21,596 | ||||||
| TOTAL ASSETS | $ | 320,164 | $ | 363,697 | ||||
| LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
| CURRENT LIABILITIES: | ||||||||
| Accounts payable | $ | 15,348 | $ | 7,134 | ||||
| Accrued expenses | 27,586 | 20,861 | ||||||
| Lease obligations, current | 3,374 | 2,583 | ||||||
| Deferred revenue - related party, current | 21,820 | 23,545 | ||||||
| Other current liabilities | — | 24 | ||||||
| Total Current Liabilities | 68,128 | 54,147 | ||||||
| Deferred revenue - related party | 43,046 | 49,297 | ||||||
| Lease obligations | 20,359 | 19,666 | ||||||
| Asset retirement obligations | 2,081 | 1,814 | ||||||
| Deferred income tax liability | 196 | 214 | ||||||
| Other long-term liabilities | 953 | — | ||||||
| TOTAL LIABILITIES | 134,763 | 125,138 | ||||||
| COMMITMENTS | ||||||||
| SHAREHOLDERS' EQUITY: | ||||||||
| Ordinary Shares, authorized, 44,548,925 and 44,189,150 shares issued and outstanding at December 31, 2021 and 2020, respectively | 2 | 2 | ||||||
| Capital in excess of par value | 528,659 | 504,482 | ||||||
| Accumulated other comprehensive loss | (2,671 | ) | (4,897 | ) | ||||
| Accumulated deficit | (340,589 | ) | (261,028 | ) | ||||
| Total Shareholders' Equity | 185,401 | 238,559 | ||||||
| TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 320,164 | $ | 363,697 | ||||
FAQ
What is the status of the Phase 3 Lumeos trial for MGTX's botaretigene sparoparvovec?
What milestone payment did MeiraGTx receive from Janssen?
How did MeiraGTx perform financially in 2021?
What preliminary clinical data did MGTX announce for AAV-hAQP1?
How much cash does MeiraGTx have as of December 31, 2021?
