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MeiraGTx Reports Second Quarter 2022 Financial and Operational Results

MeiraGTx
MeiraGTx

-- Recent Positive Topline Data from the Phase 1/2 Trial of Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP)

-- Financing Secured by Manufacturing Facilities Extends Cash Runway to Fourth Quarter 2024

LONDON and NEW YORK, Aug. 11, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gene therapy company, today announced financial results for the second quarter ended June 30, 2022, and provided an update on recent progress.

“Over the past quarter we have continued to advance our gene therapy programs and platforms, including the recently announced positive topline data from the Phase 1/2 clinical study demonstrating that our investigational gene therapy, botaretigene sparoparvovec, has the potential to improve vision in patients with retinitis pigmentosa,” said Alexandria Forbes, Ph.D., president, and chief executive officer of MeiraGTx. “We look forward to presenting additional data from this study at the American Academy of Ophthalmology (AAO) annual meeting this year as we continue to enroll the pivotal Phase 3 Lumeos clinical trial of botaretigene sparoparvovec with our partner Janssen.”

Dr. Forbes continued, “We also recently announced a financing of up to $100 million with one of our largest shareholders, extending our cash runway to the fourth quarter of 2024. Our wholly-owned manufacturing facilities collateralized this transaction, enabling us to access minimally dilutive capital while retaining the significant and growing value of our proprietary programs and transformative genetic medicine platforms.”
  
Recent Highlights and Anticipated Milestones

Botaretigene Sparoparvovec for the Treatment of XLRP:

  • In June 2022, MeiraGTx announced positive topline data from the MGT009 Phase 1/2 clinical trial of botaretigene sparoparvovec (formerly referred to as AAV-RPGR) for the treatment of patients with XLRP with disease-causing variants in the RPGR gene.

  • Treatment with botaretigene sparoparvovec was found to be generally safe and well-tolerated, with no dose-limiting events.

  • Significant improvements were demonstrated in multiple endpoints across each of the three domains of vision -- retinal function, visual function, and functional vision -- in patients treated with botaretigene sparoparvovec when compared to the randomized untreated control arm of the study at 6 months post-treatment.

  • Full MGT009 data will be presented at the AAO annual meeting, being held September 30 – October 3, 2022, in Chicago, IL.

  • The Company is currently enrolling patients in the Phase 3 Lumeos clinical trial of botaretigene sparoparvovec and targeting a Biologics License Application (BLA) filing in 2024.

  • MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing botaretigene sparoparvovec as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

AAV-CNGB3 and AAV-CNGA3 for the Treatment of Achromatopsia (ACHM):

  • Development partner Janssen plans to initiate a Phase 2b clinical trial for the treatment of ACHM associated with mutations in the CNGB3 gene in 2022 followed by a Phase 2b clinical trial for the treatment of ACHM associated with mutations in the CNGA3 gene in early 2023.

  • Both ACHM clinical trials to be initiated by Janssen will use material manufactured at MeiraGTx’s cGMP facility in London, United Kingdom.

AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:

  • MeiraGTx plans to present data from all four cohorts (n=12) in the unilateral dose escalation Phase 1 AQUAx trial as well as data from the bilateral cohorts (n=12) in the fourth quarter of 2022.

  • The Company plans to initiate a randomized, double-blind, placebo-controlled Phase 2 study by the end of 2022 with material manufactured in its cGMP facility in London, United Kingdom.

AAV-GAD for the Treatment of Parkinson’s Disease:

  • The Company expects to initiate enrollment in its study of AAV-GAD during the second half of 2022 with material manufactured in its cGMP facility in London, United Kingdom.

Gene Control Platforms:

  • The Company will present new data from its gene regulation platforms at medical meetings in the second half of 2022.

  • MeiraGTx is advancing several small molecule candidates from its gene regulation platform with the aim of initiating first-in-human safety and tolerability studies this year.

  • Novel regulation platform can be used to precisely control gene expression in cell therapy, gene editing, with any gene and any vector with unprecedented dynamic range using an oral small molecule.

Financing Agreement with Perceptive for Up to $100 Million:

  • In August 2022, MeiraGTx and Perceptive announced a senior secured financing arrangement for up to $100 million secured by MeiraGTx’s wholly-owned manufacturing facilities in London, United Kingdom and Shannon, Ireland.

  • The Company received $75 million upon closing and may request an additional $25 million during the first two years of the term under the same terms and collateral, subject to the lender’s approval.

In addition to the $75.0 million gross proceeds from this recent financing, the Company had cash and cash equivalents of approximately $72.1 million as of June 30, 2022, as well as approximately $15.9 million in receivables due from Janssen from the second quarter of 2022. The Company believes it will have sufficient capital to fund operating expenses and capital expenditure requirements into the fourth quarter of 2024.

Financial Results

License revenue was $10.8 million for the three months ended June 30, 2022, compared to $5.1 million for the three months ended June 30, 2021. This increase represents increased amortization of the $100.0 million upfront payment as well as amortization of the $30.0 million milestone payment received in connection with the Collaboration Agreement.

General and administrative expenses were $10.5 million for the three months ended June 30, 2022, compared to $10.4 million for the three months ended June 30, 2021. The increase of $0.1 million was primarily due to an increase of $1.0 million in share-based compensation, $0.4 million in legal and accounting fees, $0.1 million in depreciation and $0.2 million in other office related costs. These increases were partially offset by decreases of rent and facilities costs of $0.9 million due to additional allocations to research and development, $0.4 million in insurance costs and $0.3 million in payroll and payroll-related costs.

Research and development expenses for the three months ended June 30, 2022 were $24.0 million, compared to $15.2 million for the three months ended June 30, 2021. The increase of $8.8 million was primarily due to an increase of $2.2 million in payroll and payroll-related costs, $1.9 million in costs related to the manufacture of material for the Company’s clinical trials, $1.1 million in share-based compensation, $1.0 million in rent and facilities costs, $0.4 million in costs related to the Company’s pre-clinical research and clinical trials, $0.2 million in other research and development costs and a decrease of $2.3 million in research funding provided under the Collaboration Agreement with Janssen. These increases were partially offset by a decrease of $0.2 million in license fees and $0.1 million in depreciation.

Foreign currency loss was $10.4 million for the three months ended June 30, 2022 compared to a gain of $0.4 million for the three months ended June 30, 2021. The change in the amount of $10.8 million was primarily due to an unrealized loss on the quarterly valuation of the Company’s intercompany payables and receivables due to the strengthening of the U.S. dollar against the pound sterling and euro during the three months ended June 30, 2022.

Net loss attributable to ordinary shareholders for the quarter ended June 30, 2022 was $34.0 million, or $0.76 basic and diluted net loss per ordinary share, compared to a net loss attributable to ordinary shareholders of $20.1 million, or $0.46 basic and diluted net loss per ordinary share for the quarter ended June 30, 2021.

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 precise, dose responsive control of gene expression by oral small molecules with dynamic range that can exceed 5000-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 diseases, including both inherited retinal diseases as well as 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,” “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 June 30, 2022, 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

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 Period Ended June 30,

 

For the Six-Month Period Ended June 30,

 

 

2022

 

 

2021

 

 

2022

 

 

2021

 

License revenue - related party

 

$

10,759

 

 

$

5,116

 

 

$

16,392

 

 

$

9,711

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

General and administrative

 

 

10,518

 

 

 

10,409

 

 

 

21,786

 

 

 

20,327

 

Research and development

 

 

23,999

 

 

 

15,190

 

 

 

47,098

 

 

 

31,900

 

Total operating expenses

 

 

34,517

 

 

 

25,599

 

 

 

68,884

 

 

 

52,227

 

Loss from operations

 

 

(23,758

)

 

 

(20,483

)

 

 

(52,492

)

 

 

(42,516

)

Other non-operating income (expense):

 

 

 

 

 

 

 

 

 

 

 

 

Foreign currency (loss) gain

 

 

(10,426

)

 

 

381

 

 

 

(13,073

)

 

 

(1,234

)

Interest income

 

 

41

 

 

 

67

 

 

 

57

 

 

 

156

 

Interest expense

 

 

(82

)

 

 

(51

)

 

 

(159

)

 

 

(110

)

Fair value adjustment

 

 

252

 

 

 

 

 

 

649

 

 

 

 

Net loss

 

 

(33,973

)

 

 

(20,086

)

 

 

(65,018

)

 

 

(43,704

)

Other comprehensive income (loss):

 

 

 

 

 

 

 

 

 

 

 

 

Foreign currency translation gain (loss)

 

 

7,357

 

 

 

(407

)

 

 

9,290

 

 

 

(678

)

Total comprehensive loss

 

$

(26,616

)

 

$

(20,493

)

 

$

(55,728

)

 

$

(44,382

)

Net loss

 

$

(33,973

)

 

$

(20,086

)

 

$

(65,018

)

 

$

(43,704

)

Basic and diluted net loss per ordinary share

 

$

(0.76

)

 

$

(0.46

)

 

$

(1.46

)

 

$

(0.99

)

Weighted-average number of ordinary shares outstanding

 

 

44,668,240

 

 

 

44,137,773

 

 

 

44,585,239

 

 

 

44,056,535

 


MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES
CONDENSED CONSOLIDATED BALANCE SHEETS
(unaudited)
(in thousands, except share and per share amounts)

 

 

 

 

 

 

 

 

 

June 30,

 

December 31,

 

    

2022

 

 

2021

 

 

 

 

 

 

 

 

ASSETS

 

 

 

 

 

 

CURRENT ASSETS:

 

 

 

 

 

 

Cash and cash equivalents

 

$

72,108

 

 

$

137,703

 

Accounts receivable - related party

 

 

15,942

 

 

 

22,384

 

Prepaid expenses

 

 

6,682

 

 

 

8,102

 

Tax incentive receivable

 

 

6,350

 

 

 

12,634

 

Other current assets

 

 

3,330

 

 

 

2,420

 

Total Current Assets

 

 

104,412

 

 

 

183,243

 

 

 

 

 

 

 

 

Property, plant and equipment, net

 

 

91,388

 

 

 

75,860

 

Intangible assets, net

 

 

1,474

 

 

 

1,791

 

In-process research and development

 

 

723

 

 

 

783

 

Other assets

 

 

1,505

 

 

 

1,404

 

Equity method and other investments

 

 

6,656

 

 

 

6,656

 

Right-of-use assets - operating leases, net

 

 

21,538

 

 

 

22,782

 

Right-of-use assets - finance leases, net

 

 

24,765

 

 

 

27,645

 

TOTAL ASSETS

 

$

252,461

 

 

$

320,164

 

 

 

 

 

 

 

 

LIABILITIES AND SHAREHOLDERS' EQUITY

 

 

 

 

 

 

CURRENT LIABILITIES:

 

 

 

 

 

 

Accounts payable

 

$

18,111

 

 

$

15,348

 

Accrued expenses

 

 

23,572

 

 

 

27,586

 

Lease obligations, current

 

 

3,634

 

 

 

3,374

 

Deferred revenue - related party, current

 

 

21,205

 

 

 

21,820

 

Other current liabilities

 

 

983

 

 

 

 

Total Current Liabilities

 

 

67,505

 

 

 

68,128

 

 

 

 

 

 

 

 

Deferred revenue - related party

 

 

21,337

 

 

 

43,046

 

Lease obligations

 

 

19,063

 

 

 

20,359

 

Asset retirement obligations

 

 

2,099

 

 

 

2,081

 

Deferred income tax liability

 

 

181

 

 

 

196

 

Other long-term liabilities

 

 

304

 

 

 

953

 

TOTAL LIABILITIES

 

 

110,489

 

 

 

134,763

 

COMMITMENTS (Note 9)

 

 

 

 

 

 

SHAREHOLDERS' EQUITY:

 

 

 

 

 

 

Ordinary Shares, $0.00003881 par value, 1,288,327,750
authorized, 44,710,678 and 44,548,925 shares issued and
outstanding at June 30, 2022 and December 31, 2021, respectively

 

 

2

 

 

 

2

 

Capital in excess of par value

 

 

540,958

 

 

 

528,659

 

Accumulated other comprehensive income (loss)

 

 

6,619

 

 

 

(2,671

)

Accumulated deficit

 

 

(405,607

)

 

 

(340,589

)

Total Shareholders' Equity

 

 

141,972

 

 

 

185,401

 

TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY

 

$

252,461

 

 

$

320,164