- Received strategic investment from Sanofi purchasing
- Announced a right of first negotiation (ROFN) with Sanofi for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Central Nervous System (CNS) and Immunology and Inflammation (I&I) targets, including IL-4 and IL-13, as well as for GLP-1 and other gut peptides for metabolic disease, and for MeiraGTx’s Phase 2 Xerostomia program
- Enrollment in the Phase 2 randomized, double-blind, placebo-controlled study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced Xerostomia (RIX) is ongoing
- Presented eight posters at the
- Received a 2nd Commercial Manufacturer’s/Importer’s Authorization (MIA) for Quality Control (QC) testing at GMP manufacturing facility in Shannon,
LONDON and NEW YORK,
“We are very happy to now have Sanofi as one of our top shareholders,” said
Recent Development Highlights and Anticipated Milestones
- On
October 30, 2023 , Sanofi purchased$30 million of ordinary shares of the Company at a price of$7.50 per share. - Sanofi received a right of first negotiation (ROFN) for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Central Nervous System (CNS) and Immunology and Inflammation (I&I) targets, including IL-4 and IL-13, as well as for GLP-1 and other gut peptides for obesity, and for MeiraGTx’s Phase 2 Xerostomia program.
Bota-vec for the Treatment of XLRP:
- Enrollment completed in second quarter 2023 in the pivotal Phase 3 LUMEOS clinical trial in collaboration with
Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company. - In
September 2023 , the IND for bota-vec for the treatment of XLRP was transferred fromMeiraGTx to Janssen.
AAV-hAQP1 for the Treatment of Grade 2/3 RIX:
- Initiated a Phase 2 randomized, double-blind, placebo-controlled study in
June 2023 with participants currently being enrolled and dosed across multiple sites in theU.S. andCanada . - Results from AQUAx Phase 1 open-label, dose-escalation study of gene therapy with AAV2-hAQP1 as a treatment for RIX and parotid gland hypofunction presented at ESGCT 2023 Annual Congress on
October 26, 2023 .
AAV-GAD for the Treatment of Parkinson’s Disease:
- The Company is dosing patients in the AAV-GAD clinical trial under a new IND with material manufactured from its cGMP 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.
- Completion of enrollment is anticipated in the fourth quarter of 2023.
ESGCT 2023 Annual Congress:
ALS and Frontotemporal Dementia (FTD) Program Oral Presentation:
- Presented preclinical efficacy of AAV-hUPF1 with an optimized vector genome and novel CNS capsid: Gene Therapy for ALS and FTD.
Poster Presentations:
- Presented eight posters, including Riboswitch Gene Regulation Platform:
- RiboCAR-T cell activity can be precisely tuned and “remotely” controlled to improve the efficacy, durability, and safety of CAR-T cell therapy.
- T cells with RiboCAR showed delayed exhaustion during expansion in the absence of small molecule inducer and enhanced target cell-stimulated T cell activation and anti-cancer cytotoxicity in the presence of small molecule inducer when compared with T cells constitutively expressing CAR.
Wholly-Owned Gene Therapy Manufacturing Facility in Shannon, Ireland Received 2nd Commercial MIA Authorization for QC Testing:
- The QC facility in Shannon,
Ireland performs advanced biochemical quality control testing for release and stability testing for MeiraGTx’s and its partner’s programs. - Unique in its scale and integrated capabilities and stretching over 150,000 square feet, the GMP Shannon facility is Ireland’s first commercial-scale gene therapy manufacturing site and contains facilities for flexible and scalable viral vector production for clinical and commercial supply as well as a facility for plasmid DNA production in addition to the GMP licensed QC facility.
As of
For more information related to our clinical trials, please visit www.clinicaltrials.gov
Financial Results
Cash, cash equivalents and restricted cash were
License revenue was
General and administrative expenses were
Research and development expenses were
Foreign currency loss was
Net loss attributable to ordinary shareholders for the quarter ended
About
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 our pre-clinical data and reporting of such data and the timing of results of data, Evercore’s work with management and our Board of Directors, the review or pursuit of any potential strategic transactions, the nature, timing or likelihood of any strategic transactions or announcements of any strategic transactions, the anticipated benefits of any strategic transactions and their expected impact on the Company’s outlook, operations, opportunities, financial condition, business plan and overall strategy, 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 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 Quarterly Report on Form 10-Q for the quarter ended
There can be no assurance that the review and evaluation of potential strategic transactions will result in any particular transaction or transactions or other strategic changes or outcomes and the timing of any such event is similarly uncertain.
Contacts
Investors:
Investors@meiragtx.com
or
Media:
jbraco@lifescicomms.com
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except share and per share amounts) | |||||||||||||||
For the Three-Month Period Ended September 30, | For the Nine-Month Period Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
License revenue - related party | $ | 5,103 | $ | 4,816 | $ | 11,977 | $ | 21,208 | |||||||
Operating expenses: | |||||||||||||||
General and administrative | 10,009 | 10,762 | 35,169 | 32,548 | |||||||||||
Research and development | 27,856 | 16,862 | 70,115 | 63,960 | |||||||||||
Total operating expenses | 37,865 | 27,624 | 105,284 | 96,508 | |||||||||||
Loss from operations | (32,762 | ) | (22,808 | ) | (93,307 | ) | (75,300 | ) | |||||||
Other non-operating income (expense): | |||||||||||||||
Foreign currency loss | (8,677 | ) | (12,838 | ) | (2,915 | ) | (25,911 | ) | |||||||
Interest income | 523 | 288 | 1,723 | 345 | |||||||||||
Interest expense | (3,381 | ) | (1,892 | ) | (9,796 | ) | (2,051 | ) | |||||||
Fair value adjustment | — | (34 | ) | 53 | 615 | ||||||||||
Net loss | (44,297 | ) | (37,284 | ) | (104,242 | ) | (102,302 | ) | |||||||
Other comprehensive (loss) income: | |||||||||||||||
Foreign currency translation gain | 6,007 | 8,772 | 1,113 | 18,062 | |||||||||||
Comprehensive loss | $ | (38,290 | ) | $ | (28,512 | ) | $ | (103,129 | ) | $ | (84,240 | ) | |||
Net loss | $ | (44,297 | ) | $ | (37,284 | ) | $ | (104,242 | ) | $ | (102,302 | ) | |||
Basic and diluted net loss per ordinary share | $ | (0.74 | ) | $ | (0.83 | ) | $ | (1.91 | ) | $ | (2.29 | ) | |||
Weighted-average number of ordinary shares outstanding | 59,526,642 | 44,687,635 | 54,544,660 | 44,620,900 | |||||||||||
CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands, except share and per share amounts) | |||||||
2023 | 2022 | ||||||
ASSETS | |||||||
CURRENT ASSETS: | |||||||
Cash and cash equivalents | $ | 63,365 | $ | 115,516 | |||
Accounts receivable - related party | 22,398 | 21,334 | |||||
Prepaid expenses | 6,997 | 8,133 | |||||
Tax incentive receivable | 10,013 | 7,689 | |||||
Other current assets | 758 | 1,667 | |||||
Total Current Assets | 103,531 | 154,339 | |||||
Property, plant and equipment, net | 111,880 | 109,266 | |||||
Intangible assets, net | 1,140 | 1,335 | |||||
In-process research and development | 732 | 742 | |||||
Restricted cash | 1,038 | — | |||||
Other assets | 1,421 | 1,402 | |||||
Equity method and other investments | 6,326 | 6,326 | |||||
Right-of-use assets - operating leases, net | 17,446 | 20,109 | |||||
Right-of-use assets - finance leases, net | 23,680 | 24,718 | |||||
TOTAL ASSETS | $ | 267,194 | $ | 318,237 | |||
LIABILITIES AND SHAREHOLDERS' EQUITY | |||||||
CURRENT LIABILITIES: | |||||||
Accounts payable | $ | 20,773 | $ | 16,616 | |||
Accrued expenses | 28,251 | 39,818 | |||||
Lease obligations, current | 4,092 | 3,884 | |||||
Deferred revenue - related party, current | 7,922 | 15,123 | |||||
Other current liabilities | 2,476 | 6,631 | |||||
Total Current Liabilities | 63,514 | 82,072 | |||||
Deferred revenue - related party | 23,191 | 27,436 | |||||
Lease obligations | 14,256 | 17,331 | |||||
Asset retirement obligations | 2,319 | 2,179 | |||||
Deferred income tax liability | 184 | 186 | |||||
Note payable, net | 71,844 | 71,033 | |||||
Other long-term liabilities | — | 262 | |||||
TOTAL LIABILITIES | 175,308 | 200,499 | |||||
COMMITMENTS AND CONTINGENCIES (Note 10) | |||||||
SHAREHOLDERS' EQUITY: | |||||||
Ordinary Shares, authorized, 59,597,151 and 48,477,209 shares issued and outstanding at | 2 | 2 | |||||
Capital in excess of par value | 659,170 | 581,893 | |||||
Accumulated other comprehensive income | 7,160 | 6,047 | |||||
Accumulated deficit | (574,446 | ) | (470,204 | ) | |||
Total Shareholders' Equity | 91,886 | 117,738 | |||||
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 267,194 | $ | 318,237 | |||
Source:
2023 GlobeNewswire, Inc., source