MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated,
clinical stage gene therapy company, today announced financial and
operational results for the third quarter ended September 30, 2023,
and provided a corporate update.
“We are very happy to now have Sanofi as one of our top
shareholders,” said Alexandria Forbes, Ph.D., president and chief
executive officer of MeiraGTx. “Sanofi’s interest in large market
indications validates what MeiraGTx has been pioneering since
inception of the Company – the use of genetic medicines and gene
regulation to address large chronic diseases, not just rare
inherited disorders. With our end-to-end manufacturing
infrastructure and novel platform technologies, we are confident we
can use our genetic medicines toolkit to treat much larger
populations of patients without prohibitively high costs.”
Dr. Forbes continued, “With our Phase 3 LUMEOS trial for
X-linked retinitis pigmentosa (XLRP) fully enrolled and progressing
towards BLA filing, we continue to focus on expediting the clinical
development of our wholly-owned Xerostomia and Parkinson’s programs
as well as advancing our Riboswitch platform.” In addition Dr.
Forbes said, “As stated last month, in parallel with Sanofi’s
investment, due to the breadth of interest over the past several
months from multiple parties around strategic transactions
involving certain assets of the Company, we have engaged Evercore
and Wachtell Lipton to work with management and our Board of
Directors to execute one or more of these potential strategic
transactions to maximize value for our shareholders.”
Recent Development Highlights and Anticipated
Milestones
Strategic Investment from Sanofi:
- 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 from MeiraGTx 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 the U.S. and
Canada.
- 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 September 30, 2023, MeiraGTx had cash and cash equivalents
of approximately $64 million, as well as approximately $22 million
in receivables due from Janssen. Together with the $30 million
investment from Sanofi on October 30, 2023, the Company believes
that it will have sufficient capital to fund operating expenses and
capital expenditure requirements into mid-2025.
For more information related to our clinical trials, please
visit www.clinicaltrials.gov
Financial Results
Cash, cash equivalents and restricted cash were $64.4 million as
of September 30, 2023, compared to $115.5 million as of December
31, 2022.
License revenue was $5.1 million for the quarter ended September
30, 2023, compared to $4.8 million for the quarter ended September
30, 2022. This increase represents increased amortization of the
$100.0 million upfront payment as well as increased amortization of
the $30.0 million milestone payment received in connection with the
Janssen collaboration.
General and administrative expenses were $10.0 million for the
three months ended September 30, 2023, compared to $10.8 million
for the three months ended September 30, 2022. The decrease of $0.8
million was primarily due to a decrease in consulting fees,
insurance costs, legal and accounting fees and an employee
retention tax credit. These decreases were partially offset by
increases in payroll and payroll-related costs, share-based
compensation and other office related costs.
Research and development expenses were $27.9 million for the
three months ended September 30, 2023, compared to $16.9 million
for the three months ended September 30, 2022. The increase of
$11.0 million was primarily due to an increase in manufacturing
costs primarily due to a decrease in the number of batches of
clinical trial material produced during the three months ended
September 30, 2023 compared to the three months ended September 30,
2022 which were charged to the clinical programs and a decrease in
research funding provided under our Janssen collaboration. These
increases were partially offset by decreases in expenses related to
our preclinical programs primarily related to preclinical ocular
diseases, clinical trial expenses primarily due to a decrease in
the number of batches of clinical trial material produced in the
three months ended September 30, 2023 compared to the three months
ended September 30, 2022 and other research and development costs
primarily related to an increase in the estimated research and
development tax credit refund in the three months ended September
30, 2023 compared to a reduction in the three months ended
September 30, 2022.
Foreign currency loss was $8.7 million for the three months
ended September 30, 2023, compared to a loss of $12.8 million for
the three months ended September 30, 2022. The unrealized losses
were a result of a strengthening of the U.S. dollar against the
pound sterling and euro as it relates to the quarterly valuation of
our intercompany payables and receivables. The decrease in the loss
of $4.2 million was primarily due to a smaller fluctuation in
exchange rates during the three months ended September 30, 2023
compared to the three months ended September 30, 2022.
Net loss attributable to ordinary shareholders for the quarter
ended September 30, 2023 was $44.3 million, or $0.74 basic and
diluted net loss per ordinary share, compared to a net loss
attributable to ordinary shareholders of $37.3 million, or $0.83
basic and diluted net loss per ordinary share for the quarter ended
September 30, 2022.
About MeiraGTxMeiraGTx (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 that 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 StatementThis 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 September 30, 2023, 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.
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. MeiraGTx
does not intend to disclose or comment on developments related to
the foregoing unless or until it determines that further disclosure
is appropriate or required.
Contacts
Investors:MeiraGTxInvestors@meiragtx.com
or
Media:Jason Braco, Ph.D.LifeSci
Communicationsjbraco@lifescicomms.com
MEIRAGTX HOLDINGS PLC AND
SUBSIDIARIESCONDENSED 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 September 30, |
|
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 |
|
|
MEIRAGTX HOLDINGS PLC AND
SUBSIDIARIESCONDENSED CONSOLIDATED BALANCE
SHEETS(unaudited)(in thousands,
except share and per share amounts) |
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
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, $0.00003881
par value, 1,288,327,750 authorized, 59,597,151 and 48,477,209
shares issued andoutstanding at September 30, 2023 and December 31,
2022, respectively |
|
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 |
|
|
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