LONDON and NEW YORK,
During the quarter,
“We continued to make significant progress this quarter advancing our clinical programs,” said
Third Quarter and Recent Corporate and Clinical Development Highlights
Botaretigene sparoparvovec for the Treatment of XLRP:
- Twelve-month clinical data from MeiraGTx’s Phase 1/2 dose-escalation phase of study (MGT009) of botaretigene sparoparvovec were presented as an oral presentation at the EURETINA 2021 Virtual Meeting in September.
- The retinal function of ten adult males aged 18-30 years with RPGR-associated XLRP was assessed 12 months post-treatment in MGT009.
- For the intermediate dose-escalation cohort (N=4), intervention with botaretigene sparoparvovec in the poorer-seeing eye altered the course of natural disease progression.
- At 12 months post-intervention, mean retinal sensitivity (MS) and volumetric analysis of the central 30 degrees of the retinal field (V30) in the treated eye were similar to levels observed 24 months pre-intervention, while the untreated eye showed a continued downward trajectory.
Riboswitch Gene Regulation Platform:
- Data from the Company’s riboswitch gene regulation platform was presented at the ESGCT 2021 Annual Congress demonstrating reversible regulation of gene expression to high dynamic range in mammalian cells and precise dose-responsive activation of transgene expression in-vivo.
Pre-clinical Programs for Inherited Retinal Diseases (IRDs):
Data on novel vector development for gene replacement therapies in Inherited Retinal Diseases was presented at the ESGCT 2021 Annual Congress in October.
- AAV-KCNV2 Gene Therapy for Cone Dystrophy:
- Meira’s novel vectors successfully delivered the hKCNV2 gene to photoreceptors in retinal organoids following transduction, restoring protein expression in the correct subcellular location as well as native protein-to-protein interactions.
- Transcriptomic analyses indicated disease correction at a deep transcriptional level, and that AAV-mediated KCNV2 gene supplementation has the potential to benefit patients with cone dystrophies due to KCNV2 mutations.
- AAV-GUCY2D Gene Therapy for Cone Dystrophy:
- Meira’s novel vectors improved quantitative expression levels of GUCY2D and PDE6β relative to non-transduced controls, and total cGMP levels in transduced GUCY2D KO (GUCY2D knockout) human retinal organoids were restored to close to those observed in healthy human retinal organoids, demonstrating vector potency and transgene function.
- These results indicate that gene replacement in GUCY2D-deficient human retinal organoids restored protein expression and cGMP levels in transduced organoids.
- This provides support for the potential use of AAV-mediated GUCY2D gene supplementation as a treatment for patients with inherited retinal dystrophy caused by mutations in GUCY2D.
Anticipated Upcoming Milestones and Corporate Activities
- Provide a clinical update of the Phase 1 AQUAx trial of AAV-AQP1 for the treatment of Grade 2/3 radiation-induced xerostomia in early
December 2021 . - Complete enrollment of the Phase 1 AQUAx trial of AAV-AQP1 for the treatment for Grade 2/3 radiation-induced xerostomia during the fourth quarter of 2021.
- The Company will hold a Research and Development Day in
December 2021 where additional data on its synthetic riboswitch gene regulation platform as well as the Company’s proprietary promoter platforms will be presented. - File an Investigational New Drug (IND) application for AAV-GAD for the treatment of Parkinson’s Disease during the fourth quarter of 2021.
- Initiate, with Janssen, later stage clinical studies for AAV-CNGB3 and AAV-CNGA3 for the treatment of achromatopsia (ACHM) in 2022.
- Initiate a Phase 3 pivotal trial of AAV-RPE65 for patients with RPE65-associated retinal dystrophy in early 2022.
Financial Results
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
As of
The Company believes it will have sufficient capital to fund operating expenses and capital expenditure requirements into the second quarter of 2023.
About MeiraGTx
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, including in light of the COVID-19 pandemic, 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, 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
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 Periods Ended September 30, | For the Nine-Month Periods Ended | |||||||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||||||
License revenue - related party | $ | 6,947 | $ | 5,092 | $ | 16,658 | $ | 11,775 | ||||||||
Operating expenses: | ||||||||||||||||
General and administrative | 7,887 | 8,896 | 28,214 | 32,200 | ||||||||||||
Research and development | 21,612 | 4,626 | 53,512 | 28,911 | ||||||||||||
Total operating expenses | 29,499 | 13,522 | 81,726 | 61,111 | ||||||||||||
Loss from operations | (22,552 | ) | (8,430 | ) | (65,068 | ) | (49,336 | ) | ||||||||
Other non-operating income (expense): | ||||||||||||||||
Foreign currency (loss) gain | (3,366 | ) | 1,875 | (4,600 | ) | 767 | ||||||||||
Interest income | 32 | 158 | 188 | 1,141 | ||||||||||||
Interest expense | (59 | ) | (35 | ) | (169 | ) | (103 | ) | ||||||||
Net loss | (25,945 | ) | (6,432 | ) | (69,649 | ) | (47,531 | ) | ||||||||
Other comprehensive income (loss): | ||||||||||||||||
Foreign currency translation gain (loss) | 2,669 | (4,121 | ) | 1,991 | 342 | |||||||||||
Total comprehensive loss | $ | (23,276 | ) | $ | (10,553 | ) | $ | (67,658 | ) | $ | (47,189 | ) | ||||
Net loss | $ | (25,945 | ) | $ | (6,432 | ) | $ | (69,649 | ) | $ | (47,531 | ) | ||||
Basic and diluted net loss per ordinary share | $ | (0.59 | ) | $ | (0.17 | ) | $ | (1.58 | ) | $ | (1.29 | ) | ||||
Weighted-average number of ordinary shares outstanding | 44,170,299 | 37,223,375 | 44,094,873 | 36,940,372 | ||||||||||||
CONDENSED CONSOLIDATED BALANCE SHEETS
(unaudited)
(in thousands, except share and per share amounts)
2021 | 2020 | |||||||
ASSETS | ||||||||
CURRENT ASSETS: | ||||||||
Cash and cash equivalents | $ | 143,634 | $ | 209,520 | ||||
Accounts receivable - related party | 16,775 | 38,479 | ||||||
Prepaid expenses | 8,840 | 7,082 | ||||||
Tax incentive receivable | 5,383 | 12,930 | ||||||
Other current assets | 2,299 | 4,565 | ||||||
Total Current Assets | 176,931 | 272,576 | ||||||
Property and equipment, net | 63,699 | 44,042 | ||||||
Intangible assets, net | 1,861 | 2,119 | ||||||
In-process research and development | 803 | 852 | ||||||
Security deposits | 1,166 | 812 | ||||||
Other assets | 201 | 214 | ||||||
Equity method and other investments | 6,665 | — | ||||||
Right-of-use assets | 52,010 | 43,082 | ||||||
TOTAL ASSETS | $ | 303,336 | $ | 363,697 | ||||
LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
CURRENT LIABILITIES: | ||||||||
Accounts payable | $ | 11,421 | $ | 7,134 | ||||
Accrued expenses | 21,926 | 20,861 | ||||||
Lease obligations, current | 3,280 | 2,583 | ||||||
Deferred revenue - related party, current | 23,003 | 23,545 | ||||||
Other current liabilities | — | 24 | ||||||
Total Current Liabilities | 59,630 | 54,147 | ||||||
Deferred revenue - related party | 32,488 | 49,297 | ||||||
Lease obligations | 21,262 | 19,666 | ||||||
Asset retirement obligations | 1,916 | 1,814 | ||||||
Deferred income tax liability | 201 | 214 | ||||||
TOTAL LIABILITIES | 115,497 | 125,138 | ||||||
COMMITMENTS (Note 10) | ||||||||
SHAREHOLDERS' EQUITY: | ||||||||
Ordinary Shares, authorized, 44,316,385 and 44,189,150 shares issued and outstanding at | 2 | 2 | ||||||
Capital in excess of par value | 521,420 | 504,482 | ||||||
Accumulated other comprehensive loss | (2,906 | ) | (4,897 | ) | ||||
Accumulated deficit | (330,677 | ) | (261,028 | ) | ||||
Total Shareholders' Equity | 187,839 | 238,559 | ||||||
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 303,336 | $ | 363,697 |
Source:
2021 GlobeNewswire, Inc., source