MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated,
clinical stage gene therapy company, today announced financial and
operational results for the first quarter ended March 31, 2023, and
provided a corporate update.
“We are off to a strong start this year with progress across
multiple clinical-stage programs and a growing body of data
supporting our proprietary gene regulation technology,” said
Alexandria Forbes, Ph.D., president and chief executive officer of
MeiraGTx. “Enrollment of the pivotal Lumeos Phase 3 study is going
well and we remain on track for a BLA filing in 2024. We are also
excited to be progressing towards later stage studies in our
wholly-owned programs, including a randomized, double-blind,
placebo-controlled, Phase 2 study for the treatment of Grade 2/3
radiation-induced xerostomia, and our randomized, sham-controlled
bridging study for our Parkinson’s disease program.”
Dr. Forbes continued, “At this year’s ARVO Annual Meeting, we
presented data from our AI-driven promoter discovery platform
illustrating our ability to precisely target specific cells and
expression levels with synthetic promoters. In addition we
presented immune-response data from the Phase 1/2 trial for the
investigational gene therapy bota-vec in patients with XLRP
associated with mutations in the RPGR gene. We are also pleased
that nine poster presentations showcasing our novel gene and cell
therapy platforms will be presented at the upcoming ASGCT Annual
Meeting, including the application of our riboswitch technology to
precisely regulate CAR-Ts. We are also presenting data
demonstrating efficacy in animal models of vector delivered human
growth hormone and anti-HER2 antibody in tight dose response to an
orally dosed small molecule inducer. This provides support for the
broad applicability of our riboswitch gene regulation platform for
therapeutic delivery of a range of biologic drugs and cell
therapies with enhanced safety and efficacy.”
Recent Development Highlights and Anticipated
Milestones
Bota-vec for the Treatment of XLRP:
- 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.
- Dosing in the pivotal Phase 3 LUMEOS clinical trial of bota-vec
continues and the program remains on track for a BLA submission in
2024.
AAV-hAQP1 for the Treatment of Grade 2/3
Radiation-Induced Xerostomia:
- MeiraGTx reported positive clinical data from the AQUAx Phase 1
clinical trial in December 2022, and remains on track to present
the full data from the AQUAx Phase 1 study in the second quarter of
2023, including the 12 month data from bilaterally treated
subjects.
- Based on the favorable safety and efficacy profile of AAV-hAQP1
in the AQUAx Phase 1 study, the Company intends to initiate a
randomized, double-blind, placebo-controlled, Phase 2 study
evaluating the bilateral administration of two active doses of
AAV-hAQP1 in the second quarter of 2023.
AAV-GAD for the Treatment of Parkinson’s
Disease:
- The Company is now 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:
- The Company will exhibit nine poster presentations at the ASGCT
2023 Annual Meeting, including data demonstrating the ability to
regulate CAR-T driving increased efficacy and reduced exhaustion in
vitro.
- ASGCT posters will also show data demonstrating rescue of
B.little mouse model via hGH activated using an orally delivered
small molecule, as well as AAV-mediated riboswitch-controlled
delivery of anti-HER2 antibody suppressing HER2-positive
tumorigenesis in a dose response to an orally delivered small
molecule.
- The Company’s next-generation riboswitch-based gene regulation
platform can be used to precisely control the expression of any
gene delivered in any context with an unprecedented dynamic range
using novel, synthetic, orally delivered small molecules.
- The Company now has over 40 novel orally available small
molecules with high specificity and potency to its riboswitch
aptamers moving through PK, biodistribution, and toxicology
studies, with the first GMP material for IND currently being
manufactured. Two of these small molecules show good brain
penetrance to enable activation of genes within the blood brain
barrier.
Recent Corporate Developments In May 2023,
MeiraGTx closed the previously announced private investment in
public equity (PIPE) financing, raising approximately $60 million
in aggregate gross proceeds. The financing was entirely led by
several of the Company’s top holders Perceptive Advisors, Adage
Capital, Prosight Management, and 683 Capital Management.
For more information related to our clinical trials, please
visit www.clinicaltrials.gov
In addition to the proceeds from the PIPE financing, MeiraGTx
had cash and cash equivalents of approximately $68.8 million, as
well as approximately $36.3 million in receivables due from
Janssen. The Company believes that with such funds, as well as
anticipated milestones from Janssen, it will have sufficient
capital to fund operating expenses and capital expenditure
requirements into the second quarter of 2025.
Financial Results
Cash and cash equivalents were $68.8 million as of March 31,
2023, compared to $115.5 million as of December 31, 2022.
License revenue was $3.3 million for the quarter ended March 31,
2023, compared to $5.6 million for the quarter ended March 31,
2022. This decrease represents decreased amortization of the $100.0
million upfront payment as well as decreased amortization of the
$30.0 million milestone payment received in connection with the
Janssen collaboration.
General and administrative expenses were $12.8 million for the
three months ended March 31, 2023, compared to $11.3 million for
the three months ended March 31, 2022. The increase of $1.5 million
was primarily due to an increase in legal and accounting fees,
consulting fees and other office related costs. These increases
were partially offset by decreases in share-based compensation,
insurance costs and payroll and payroll-related costs.
Research and development expenses for the three months ended
March 31, 2023 were $22.3 million, compared to $23.1 million for
the three months ended March 31, 2022. The decrease of $0.8 million
was primarily due to a decrease in expenses related to our
preclinical programs primarily due to the timing of expenses in our
gene regulation program and a decrease in other research and
development expenses primarily due to a decrease in share-based
compensation, as well as an increase in research funding provided
under our Janssen collaboration primarily due to the increase in
expenses incurred related to our program for bota-vec for the
treatment of XLRP. These decreases were partially offset by an
increase in clinical trial expenses primarily due to an increase in
expenses related to our Phase 3 Lumeos clinical trial of bota-vec
and our expanded Phase 1 clinical trial and Phase 2 clinical trial
for AAV-hAQP1, as well as an increase in manufacturing expenses
primarily due to the commencement of operations at our Shannon,
Ireland manufacturing facility in 2022.
Foreign currency gain was $3.9 million for the three months
ended March 31, 2023, compared to a loss of $2.6 million for the
three months ended March 31, 2022. The change of $6.5 million
was primarily due to an unrealized gain on the quarterly valuation
of intercompany payables and receivables due to the weakening of
the U.S. dollar against the pound sterling and euro during the
three months ended March 31, 2023.
Net loss attributable to ordinary shareholders for the quarter
ended March 31, 2023 was $30.4 million, or $0.62 basic and diluted
net loss per ordinary share, compared to a net loss attributable to
ordinary shareholders of $31.0 million, or $0.70 basic and diluted
net loss per ordinary share for the quarter ended March 31,
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 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 March 31, 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.
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
March 31, |
|
|
2023 |
|
2022 |
License revenue - related party |
|
$ |
3,334 |
|
|
$ |
5,633 |
|
Operating expenses: |
|
|
|
|
|
|
General and administrative |
|
|
12,772 |
|
|
|
11,268 |
|
Research and development |
|
|
22,322 |
|
|
|
23,099 |
|
Total operating expenses |
|
|
35,094 |
|
|
|
34,367 |
|
Loss from operations |
|
|
(31,760 |
) |
|
|
(28,734 |
) |
Other non-operating income
(expense): |
|
|
|
|
|
|
Foreign currency gain (loss) |
|
|
3,857 |
|
|
|
(2,647 |
) |
Interest income |
|
|
545 |
|
|
|
16 |
|
Interest expense |
|
|
(3,060 |
) |
|
|
(77 |
) |
Fair value adjustment |
|
|
54 |
|
|
|
397 |
|
Net loss |
|
|
(30,364 |
) |
|
|
(31,045 |
) |
Other comprehensive (loss)
income: |
|
|
|
|
|
|
Foreign currency translation
(loss) gain |
|
|
(2,353 |
) |
|
|
1,932 |
|
Comprehensive loss |
|
$ |
(32,717 |
) |
|
$ |
(29,113 |
) |
|
|
|
|
|
|
|
Net loss |
|
$ |
(30,364 |
) |
|
$ |
(31,045 |
) |
Basic and diluted net loss per
ordinary share |
|
$ |
(0.62 |
) |
|
$ |
(0.70 |
) |
Weighted-average number of
ordinary shares outstanding |
|
|
48,638,151 |
|
|
|
44,501,314 |
|
MEIRAGTX HOLDINGS PLC AND
SUBSIDIARIESCONDENSED CONSOLIDATED BALANCE
SHEETS(unaudited)(in thousands,
except share and per share amounts) |
|
|
|
|
|
|
|
March31, |
|
December31, |
|
|
2023 |
|
2022 |
ASSETS |
|
|
|
|
|
|
CURRENT ASSETS: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
68,784 |
|
|
$ |
115,516 |
|
Accounts receivable - related
party |
|
|
36,298 |
|
|
|
21,334 |
|
Prepaid expenses |
|
|
6,981 |
|
|
|
8,133 |
|
Tax incentive receivable |
|
|
7,857 |
|
|
|
7,689 |
|
Other current assets |
|
|
1,561 |
|
|
|
1,667 |
|
Total Current Assets |
|
|
121,481 |
|
|
|
154,339 |
|
Property, plant and equipment,
net |
|
|
112,580 |
|
|
|
109,266 |
|
Intangible assets, net |
|
|
1,295 |
|
|
|
1,335 |
|
In-process research and
development |
|
|
752 |
|
|
|
742 |
|
Other assets |
|
|
1,428 |
|
|
|
1,402 |
|
Equity method and other
investments |
|
|
6,326 |
|
|
|
6,326 |
|
Right-of-use assets -
operating leases, net |
|
|
19,427 |
|
|
|
20,109 |
|
Right-of-use assets - finance
leases, net |
|
|
24,851 |
|
|
|
24,718 |
|
TOTAL ASSETS |
|
$ |
288,140 |
|
|
$ |
318,237 |
|
|
|
|
|
|
|
|
LIABILITIES AND SHAREHOLDERS' EQUITY |
|
|
|
|
|
|
CURRENT LIABILITIES: |
|
|
|
|
|
|
Accounts payable |
|
$ |
29,755 |
|
|
$ |
16,616 |
|
Accrued expenses |
|
|
31,067 |
|
|
|
39,818 |
|
Lease obligations,
current |
|
|
4,018 |
|
|
|
3,884 |
|
Deferred revenue - related
party, current |
|
|
13,693 |
|
|
|
15,123 |
|
Other current liabilities |
|
|
2,571 |
|
|
|
6,631 |
|
Total Current Liabilities |
|
|
81,104 |
|
|
|
82,072 |
|
Deferred revenue - related
party |
|
|
26,425 |
|
|
|
27,436 |
|
Lease obligations |
|
|
16,453 |
|
|
|
17,331 |
|
Asset retirement
obligations |
|
|
2,238 |
|
|
|
2,179 |
|
Deferred income tax
liability |
|
|
189 |
|
|
|
186 |
|
Note payable, net |
|
|
71,301 |
|
|
|
71,033 |
|
Other long-term
liabilities |
|
|
208 |
|
|
|
262 |
|
TOTAL LIABILITIES |
|
|
197,918 |
|
|
|
200,499 |
|
COMMITMENTS AND CONTINGENCIES
(Note 10) |
|
|
|
|
|
|
SHAREHOLDERS' EQUITY: |
|
|
|
|
|
|
Ordinary Shares, $0.00003881
par value, 1,288,327,750 authorized, 48,686,263 and 48,477,209
shares issued and outstanding at March 31, 2023 and December
31, 2022, respectively |
|
|
2 |
|
|
|
2 |
|
Capital in excess of par
value |
|
|
587,094 |
|
|
|
581,893 |
|
Accumulated other
comprehensive income |
|
|
3,694 |
|
|
|
6,047 |
|
Accumulated deficit |
|
|
(500,568 |
) |
|
|
(470,204 |
) |
Total Shareholders' Equity |
|
|
90,222 |
|
|
|
117,738 |
|
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY |
|
$ |
288,140 |
|
|
$ |
318,237 |
|
MeiraGTx (NASDAQ:MGTX)
過去 株価チャート
から 4 2024 まで 5 2024
MeiraGTx (NASDAQ:MGTX)
過去 株価チャート
から 5 2023 まで 5 2024