- Phase 3 LUMEOS clinical trial of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) enrollment target has now been surpassed
- Announced positive clinical data from the completed AQUAx Phase 1 study of AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia (RIX), showing bilaterally treated participants reaching normal levels of whole saliva flow rate by 2 months post-treatment and persisting through the final Month 12 assessment in the study
- Initiated Phase 2 randomized, double-blind, placebo-controlled study of AAV2-hAQP1 for the treatment of grade 2/3 RIX
- Wholly-owned gene therapy manufacturing facility in Shannon,
Ireland received Commercial MIA Authorization for QC Testing and is the first manufacturing site for gene therapy to receive a commercial license inIreland - Presented data on proprietary RiboCAR platform demonstrating improved CAR-T anti-cancer efficacy and durability at the 2023
American Society of Gene andCell Therapy (ASGCT) Spotlight on Immuno-Oncology
LONDON and NEW YORK,
“Progress across our programs and infrastructure continues to be extremely impressive,” said
Recent Development Highlights and Anticipated Milestones
Bota-vec for the Treatment of XLRP:
- Enrollment target in the pivotal Phase 3 LUMEOS clinical trial of bota-vec has been surpassed, with BLA submission expected in 2024.
- In late April, immune-response data from a Phase 1/2 MGT009 clinical trial (NCT03252847) were presented at the
Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting.
AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:
- Initiated a Phase 2 randomized, double-blind, placebo-controlled study with participants currently being enrolled and dosed.
- Reported clinically meaningful improvements from the AQUAx Phase 1 study in xerostomia symptoms measured by two different xerostomia patient reported outcome questionnaires (PROs) demonstrated across both unilaterally and bilaterally treated cohorts at 12 months:
- Increases in whole saliva flow rates observed post-treatment, providing objective evidence of biological activity, reaching the normal range in bilaterally treated participants by 2 months and persisting through the Month 12 assessment.
- Across assessments, greater improvements were observed in bilaterally treated participants compared to those treated unilaterally.
- Early long-term follow-up data suggest durability of improvement to at least 3 years post-treatment.
- AAV2-hAQP1 appears safe and well tolerated at each dose tested.
- The strong safety and encouraging, clinically meaningful activity data support further clinical development of AAV2-hAQP1.
- The Company anticipates presenting the full data from the AQUAx Phase 1 study at the next appropriate medical meeting.
Wholly-Owned Gene Therapy Manufacturing Facility in Shannon,
- 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.
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 in 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 manufactured at MeiraGTx’s cGMP facility in
London, United Kingdom when delivered to the subthalamic nucleus (STN) of patients with Parkinson's disease. - Completion of enrollment is anticipated in the third quarter of 2023.
Riboswitch Gene Regulation Platform & Vector Engineering:
- Presented data at the 2023 ASGCT Spotlight on Immuno-Oncology
- RiboCAR-T cell activity can be precisely tuned and “remotely” controlled to improve the efficacy, durability, and safety of CAR-T cell therapy.
- Proprietary switch allows the Company to precisely and reversibly control the expression of CAR in a dose-response to novel small molecule inducers to unprecedented levels, from undetectable at baseline to at least as high as levels of constitutively expressed CAR driven by the small molecule dose.
- Presentations at the ASGCT 2023 Annual Meeting
- The Company exhibited nine poster presentations highlighting the depth and novelty of MeiraGTx’s technology platforms for gene and cell therapy.
- The Company exhibited nine poster presentations highlighting the depth and novelty of MeiraGTx’s technology platforms for gene and cell therapy.
For more information related to our clinical trials, please visit www.clinicaltrials.gov
As of
Financial Results
Cash and cash equivalents were
License revenue was
General and administrative expenses were
Research and development expenses for the three months ended
Foreign currency gain 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 anticipated milestones regarding 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 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
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) | ||||||||||||||||
Forthe Three-Month Period Ended June 30, | Forthe Six-Month Period Ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
License revenue - related party | $ | 3,540 | $ | 10,759 | $ | 6,874 | $ | 16,392 | ||||||||
Operating expenses: | ||||||||||||||||
General and administrative | 12,388 | 10,518 | 25,160 | 21,786 | ||||||||||||
Research and development | 19,937 | 23,999 | 42,259 | 47,098 | ||||||||||||
Total operating expenses | 32,325 | 34,517 | 67,419 | 68,884 | ||||||||||||
Loss from operations | (28,785 | ) | (23,758 | ) | (60,545 | ) | (52,492 | ) | ||||||||
Other non-operating income (expense): | ||||||||||||||||
Foreign currency gain (loss) | 1,905 | (10,426 | ) | 5,762 | (13,073 | ) | ||||||||||
Interest income | 655 | 41 | 1,200 | 57 | ||||||||||||
Interest expense | (3,355 | ) | (82 | ) | (6,415 | ) | (159 | ) | ||||||||
Fair value adjustment | (1 | ) | 252 | 53 | 649 | |||||||||||
Net loss | (29,581 | ) | (33,973 | ) | (59,945 | ) | (65,018 | ) | ||||||||
Other comprehensive (loss) income: | ||||||||||||||||
Foreign currency translation (loss) gain | (2,541 | ) | 7,357 | (4,894 | ) | 9,290 | ||||||||||
Comprehensive loss | $ | (32,122 | ) | $ | (26,616 | ) | $ | (64,839 | ) | $ | (55,728 | ) | ||||
Net loss | $ | (29,581 | ) | $ | (33,973 | ) | $ | (59,945 | ) | $ | (65,018 | ) | ||||
Basic and diluted net loss per ordinary share | $ | (0.53 | ) | $ | (0.76 | ) | $ | (1.15 | ) | $ | (1.46 | ) | ||||
Weighted-average number of ordinary shares outstanding | 55,349,534 | 44,668,240 | 52,012,382 | 44,585,239 | ||||||||||||
CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
(unaudited) | ||||||||
(in thousands, except share and per share amounts) | ||||||||
June30, | December31, | |||||||
2023 | 2022 | |||||||
ASSETS | ||||||||
CURRENT ASSETS: | ||||||||
Cash and cash equivalents | $ | 92,773 | $ | 115,516 | ||||
Accounts receivable - related party | 32,690 | 21,334 | ||||||
Prepaid expenses | 8,108 | 8,133 | ||||||
Tax incentive receivable | 8,026 | 7,689 | ||||||
Other current assets | 1,547 | 1,667 | ||||||
Total Current Assets | 143,144 | 154,339 | ||||||
Property, plant and equipment, net | 114,004 | 109,266 | ||||||
Intangible assets, net | 1,252 | 1,335 | ||||||
In-process research and development | 753 | 742 | ||||||
Other assets | 1,465 | 1,402 | ||||||
Equity method and other investments | 6,326 | 6,326 | ||||||
Right-of-use assets - operating leases, net | 18,693 | 20,109 | ||||||
Right-of-use assets - finance leases, net | 24,626 | 24,718 | ||||||
TOTAL ASSETS | $ | 310,263 | $ | 318,237 | ||||
LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
CURRENT LIABILITIES: | ||||||||
Accounts payable | $ | 28,497 | $ | 16,616 | ||||
Accrued expenses | 24,889 | 39,818 | ||||||
Lease obligations, current | 4,126 | 3,884 | ||||||
Deferred revenue - related party, current | 12,058 | 15,123 | ||||||
Other current liabilities | 2,233 | 6,631 | ||||||
Total Current Liabilities | 71,803 | 82,072 | ||||||
Deferred revenue - related party | 25,364 | 27,436 | ||||||
Lease obligations | 15,508 | 17,331 | ||||||
Asset retirement obligations | 2,301 | 2,179 | ||||||
Deferred income tax liability | 189 | 186 | ||||||
Note payable, net | 71,571 | 71,033 | ||||||
Other long-term liabilities | — | 262 | ||||||
TOTAL LIABILITIES | 186,736 | 200,499 | ||||||
COMMITMENTS AND CONTINGENCIES (Note 10) | ||||||||
SHAREHOLDERS' EQUITY: | ||||||||
Ordinary Shares, authorized, 59,535,314 and 48,477,209 shares issued and outstanding at | 2 | 2 | ||||||
Capital in excess of par value | 652,521 | 581,893 | ||||||
Accumulated other comprehensive income | 1,153 | 6,047 | ||||||
Accumulated deficit | (530,149 | ) | (470,204 | ) | ||||
Total Shareholders' Equity | 123,527 | 117,738 | ||||||
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 310,263 | $ | 318,237 | ||||
Source:
2023 GlobeNewswire, Inc., source