MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated,
clinical stage gene therapy company, today announced financial and
operational results for the fourth quarter and full-year ended
December 31, 2022, and provided a corporate update.
“As we begin 2023, we are increasingly confident in all three of
our lead clinical programs, as well as our transformative
riboswitch gene regulation technology,” said Alexandria Forbes,
Ph.D., president and chief executive officer of MeiraGTx. “In the
fourth quarter of last year, we announced positive clinical data
from our Phase 1 trial of AAV-hAQP1 for the treatment of grade 2/3
radiation-induced xerostomia and presented positive data at AAO
demonstrating positive safety and sustained vision improvement in
patients with X-linked retinitis pigmentosa who were treated with
botaretigene sparoparvovec in our Phase 1/2 trial. We also
presented 15 abstracts at the European Society of Gene and Cell
Therapy (ESGCT) Annual Congress highlighting just some of the data
from our gene control platforms, including for the first time our
riboswitch gene regulation applied to cell therapy. In addition, we
began dosing patients with adeno-associated virus (AAV) encoding
glutamic acid decarboxylase (AAV-GAD), an investigational gene
therapy for Parkinson’s disease.”
Dr. Forbes continued, “This year, in XLRP, we intend to complete
enrollment of the pivotal Lumeos Phase 3 study and are on track for
a BLA filing in 2024. In xerostomia, we intend to begin a
randomized, double-blind, placebo-controlled, Phase 2 study in the
second quarter, and in AAV-GAD for Parkinson’s, we anticipate
completing enrollment of our current study in the second half of
2023 as we move forward with discussions with global regulators
regarding a path to BLA. This progress across multiple programs and
platforms is enabled by our broad end-to-end capabilities in
vectorology optimization and manufacturing. In addition, in 2023,
we intend to share more about our plans to use our riboswitch
technology to allow gene therapy to be applied to more prevalent
diseases in a cost-effective way. This has been one of our primary
goals since the inception of MeiraGTx, and we are very excited to
be advancing towards potentially achieving this ambition.”
Recent Development Highlights and Anticipated 2023
Milestones
Botaretigene Sparoparvovec for the Treatment of
XLRP:
- On October 1, 2022, clinical data from a Phase 1/2 MGT009
clinical trial (NCT03252847) were presented in a late-breaking oral
presentation at the Retina Subspecialty Day program of the AAO 2022
Annual Meeting; treatment with botaretigene sparoparvovec was found
to have an acceptable safety profile and efficacy assessments in
this study and demonstrated improvements in retinal sensitivity,
visual function and functional vision.1
- Further sensitivity analysis was conducted on study
participants by applying the Phase 3 LUMEOS (NCT04671433) study
eligibility criteria that corroborated the endpoints selected for
the Phase 3 study.1
- MeiraGTx, in collaboration with Janssen Pharmaceuticals,
Inc. (Janssen), one of the Janssen Pharmaceutical Companies of
Johnson & Johnson, is dosing patients in the pivotal Phase 3
LUMEOS clinical trial of botaretigene sparoparvovec and 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.
- Clinically meaningful improvements in xerostomia symptoms and
disease burden in two validated Patient-Reported Outcome (PRO)
measures in both unilateral and bilateral treated cohorts were
demonstrated.
- 18/24, or 75% achieved clinically meaningful symptom
improvement using the Global Rate of Change (GRCQ) PRO.
- Using the Xerostomia Questionnaire (XQ), 71% (17/24) reported
an improvement of >8 points (clinically meaningful), and 67%
(16/24) had an improvement of ≥10 (considered transformative by
KOLs).
- Meaningful increases in whole saliva flow rates were observed
post-treatment, providing objective evidence of the biological
activity of AAV-hAQP1 treatment.
- Early long-term follow-up data suggest durability of
improvement 2+ years post-treatment.
- AAV-hAQP1 appears safe and well-tolerated at each dose
tested.
- All participants are followed for 1 year post-treatment and
then enter a long-term follow-up study for another 4 years.
- The Company intends to present the final 12 month data from the
bilateral treated cohorts from the AQUAx Phase 1 study in the
second quarter of 2023.
- 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 using 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 one of two doses of
AAV-GAD or sham control.
- The objective of the AAV-GAD trial (NCT05603312) is to evaluate
the safety and tolerability of AAV-mediated delivery of glutamic
acid decarboxylase (GAD) gene transfer into the subthalamic nuclei
(STN) of participants with Parkinson's disease.
- Completion of enrollment is anticipated by the third quarter of
2023.
Riboswitch Gene Regulation Platform & Vector
Engineering:
- The Company exhibited 15 poster presentations at the ESGCT 2022
Annual Congress, which included data from MeiraGTx’s novel gene
regulation platform, including the first data demonstrating the
potential to regulate cell therapies including CAR-T, as well as
data from the Company’s promoter platforms and several new,
optimized pre-clinical programs addressing severe unmet needs for
indications such as amyotrophic lateral sclerosis (ALS) and
Wilson’s disease. In addition, the Company made several
presentations on its proprietary viral vector manufacturing
technology and potency assay development.
- 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 30 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.
Gene Therapy Manufacturing:
- MeiraGTx’s wholly-owned facilities have now produced GMP
clinical trial material for 6 different indications, using multiple
AAV serotypes, including administration into the eye, salivary
gland and central nervous system.
- The Company believes that its proprietary platform production
process has produced one of the highest yields and full ratios in
the industry.
- The Company believes that bringing all aspects of testing and
vector production in-house reduces regulatory risk, ensures the
highest quality of products, lowers costs and helps avoid
bottlenecks in clinical development.
- In addition to its 30,000-square-foot facility in London,
MeiraGTx now has a 150,000-square-foot plant in Shannon, Ireland
which contains three facilities: one built to be flexible and
scalable for viral vector production, another to manufacture
plasmid DNA – the critical starting material for producing gene
therapy products – and third, a Quality Control (QC) hub performing
advanced biochemical quality control testing appropriate for
commercialization.
For more information related to our clinical trials, please
visit www.clinicaltrials.gov
As of December 31, 2022, MeiraGTx had cash and cash equivalents
of approximately $115 million, as well as approximately $21 million
in receivables due from Janssen from the fourth quarter of 2022.
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
fourth quarter of 2024.
Financial Results
Cash and cash equivalents were $115.5 million as of December 31,
2022, compared to $137.7 million as of December 31, 2021.
License revenue was $15.9 million for the year ended December
31, 2022, compared to $37.7 million for the year ended December 31,
2021. This decrease is a result of MeiraGTx receiving a $30.0
million milestone payment in connection with the Janssen
collaboration during the year ended December 31, 2021.
General and administrative expenses were $46.6 million for the
year ended December 31, 2022, compared to $43.8 million for
the year ended December 31, 2021. The increase of $2.8 million
was primarily due to an increase in share-based compensation, legal
and accounting fees, consulting fees and depreciation, which was
partially offset by decreases in payroll and payroll-related costs,
insurance, rent and facilities costs and other general and
administrative expenses.
Research and development expenses for the years ended December
31, 2022, and 2021 were as follows (in millions):
|
|
2022 |
|
|
2021 |
|
|
Change |
Gross research and development expenses |
|
$ |
165.8 |
|
|
$ |
141.1 |
|
|
$ |
24.7 |
|
Janssen
reimbursements |
|
|
(73.3 |
) |
|
|
(69.0 |
) |
|
|
(4.3 |
) |
Tax incentive
reimbursement |
|
|
(6.8 |
) |
|
|
(5.4 |
) |
|
|
(1.4 |
) |
Research and
development expenses |
|
$ |
85.7 |
|
|
$ |
66.7 |
|
|
$ |
19.0 |
|
|
|
|
|
|
|
|
|
|
|
Gross research and development expenses for the year ended
December 31, 2022, increased $24.7 million as compared to the
prior year primarily due to an increase in costs related to
manufacturing of our clinical trial materials, payroll and
payroll-related costs, costs related to our pre-clinical research
and clinical trials, share-based compensation, rent and facility
costs, depreciation and other research costs, which was partially
offset by a decrease in license fees and acquired research and
development costs.
Reimbursements under the Janssen collaboration agreement for the
year ended December 31, 2022, increased $4.3 million as compared to
the prior year primarily due to an increase in activity in the
programs licensed under the Janssen collaboration agreement.
Tax incentive reimbursement for the year ended December 31,
2022, increased $1.4 million as compared to the prior year
primarily due to the increase in allowable research and development
costs.
Foreign currency loss was $9.5 million for the year ended
December 31, 2022, compared to a loss of $6.3 million for the
year ended December 31, 2021. The increase in the loss of
$3.2 million was primarily due to an unrealized loss on the
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 year ended December 31, 2022.
Interest income was $0.8 million for the year ended December 31,
2022, compared to $0.2 million for the year ended December 31,
2021. The increase was due to a higher interest rate during
2022.
Interest expense was $4.9 million for the year ended December
31, 2022, compared to $0.3 million for the year ended December 31,
2021. The increase was primarily due to the interest on the
Company’s outstanding debt.
Net loss attributable to ordinary shareholders for the year
ended December 31, 2022, was $129.6 million, or $2.87 basic and
diluted net loss per ordinary share, compared to a net loss
attributable to ordinary shareholders of $79.6 million, or $1.80
basic and diluted net loss per ordinary share for the year ended
December 31, 2021.
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, 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 Annual Report on Form 10-K for
the year ended December 31, 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.
1 Michaelides, M et al. Ph1/2 AAV5-RPGR (Botaretigene
Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked
Retinitis Pigmentosa (XLRP). Abstract #30071754. Presented at the
2022 American Academy of Ophthalmology Annual Meeting.
Contacts
Investors:MeiraGTxInvestors@meiragtx.com
or
Media:Jason Braco, Ph.D.LifeSci
Communicationsjbraco@lifescicomms.com
MEIRAGTX HOLDINGS PLC AND
SUBSIDIARIESCONSOLIDATED STATEMENTS OF OPERATIONS
AND COMPREHENSIVE LOSS(in thousands, except share
and per share amounts)
|
|
For the Years Ended December 31, |
|
|
2022 |
|
|
2021 |
|
|
|
|
|
|
|
|
License revenue - related
party |
|
$ |
15,920 |
|
|
$ |
37,701 |
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
General and administrative |
|
|
46,550 |
|
|
|
43,765 |
|
Research and development |
|
|
85,725 |
|
|
|
66,694 |
|
Total operating expenses |
|
|
132,275 |
|
|
|
110,459 |
|
Loss from operations |
|
|
(116,355 |
) |
|
|
(72,758 |
) |
Other non-operating income
(expense): |
|
|
|
|
|
|
Foreign currency loss |
|
|
(9,452 |
) |
|
|
(6,293 |
) |
Interest income |
|
|
777 |
|
|
|
212 |
|
Interest expense |
|
|
(4,946 |
) |
|
|
(288 |
) |
Fair value adjustments |
|
|
361 |
|
|
|
(434 |
) |
Net loss |
|
|
(129,615 |
) |
|
|
(79,561 |
) |
Other comprehensive
income: |
|
|
|
|
|
|
Foreign currency translation
gain |
|
|
8,718 |
|
|
|
2,226 |
|
Comprehensive loss |
|
$ |
(120,897 |
) |
|
$ |
(77,335 |
) |
|
|
|
|
|
|
|
Net loss |
|
$ |
(129,615 |
) |
|
$ |
(79,561 |
) |
Basic and diluted net loss per
ordinary share |
|
$ |
(2.87 |
) |
|
$ |
(1.80 |
) |
Weighted-average number of
ordinary shares outstanding |
|
|
45,177,857 |
|
|
|
44,139,655 |
|
MEIRAGTX HOLDINGS PLC AND
SUBSIDIARIESCONSOLIDATED BALANCE
SHEETS(in thousands, except share and per share
amounts)
|
|
|
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
2022 |
|
2021 |
|
|
|
|
|
|
|
ASSETS |
|
|
|
|
|
|
|
|
|
|
|
|
|
CURRENT ASSETS: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
115,516 |
|
|
$ |
137,703 |
|
Accounts receivable - related
party |
|
|
21,334 |
|
|
|
22,384 |
|
Prepaid expenses |
|
|
8,133 |
|
|
|
8,102 |
|
Tax incentive receivable |
|
|
7,689 |
|
|
|
12,634 |
|
Other current assets |
|
|
1,667 |
|
|
|
2,420 |
|
Total Current Assets |
|
|
154,339 |
|
|
|
183,243 |
|
|
|
|
|
|
|
|
Property, plant and equipment,
net |
|
|
109,266 |
|
|
|
75,860 |
|
Intangible assets, net |
|
|
1,335 |
|
|
|
1,791 |
|
In-process research and
development |
|
|
742 |
|
|
|
783 |
|
Other assets |
|
|
1,402 |
|
|
|
1,404 |
|
Equity method and other
investments |
|
|
6,326 |
|
|
|
6,656 |
|
Right-of-use assets -
operating leases, net |
|
|
20,109 |
|
|
|
22,782 |
|
Right-of-use assets - finance
leases, net |
|
|
24,718 |
|
|
|
27,645 |
|
TOTAL ASSETS |
|
$ |
318,237 |
|
|
$ |
320,164 |
|
|
|
|
|
|
|
|
LIABILITIES AND SHAREHOLDERS' EQUITY |
|
|
|
|
|
|
CURRENT LIABILITIES: |
|
|
|
|
|
|
Accounts payable |
|
$ |
16,616 |
|
|
$ |
15,348 |
|
Accrued expenses |
|
|
39,818 |
|
|
|
27,586 |
|
Lease obligations,
current |
|
|
3,884 |
|
|
|
3,374 |
|
Deferred revenue - related
party, current |
|
|
15,123 |
|
|
|
21,820 |
|
Other current liabilities |
|
|
6,631 |
|
|
|
— |
|
Total Current Liabilities |
|
|
82,072 |
|
|
|
68,128 |
|
|
|
|
|
|
|
|
Deferred revenue - related
party |
|
|
27,436 |
|
|
|
43,046 |
|
Lease obligations |
|
|
17,331 |
|
|
|
20,359 |
|
Asset retirement
obligations |
|
|
2,179 |
|
|
|
2,081 |
|
Deferred income tax
liability |
|
|
186 |
|
|
|
196 |
|
Note payable, net |
|
|
71,033 |
|
|
|
— |
|
Other long-term
liabilities |
|
|
262 |
|
|
|
953 |
|
TOTAL LIABILITIES |
|
|
200,499 |
|
|
|
134,763 |
|
|
|
|
|
|
|
|
COMMITMENTS AND CONTINGENCIES
(Note 15) |
|
|
|
|
|
|
|
|
|
|
|
|
|
SHAREHOLDERS' EQUITY: |
|
|
|
|
|
|
Ordinary Shares, $0.00003881
par value, 1,288,327,750 authorized, 48,477,209 and 44,548,925
shares issued andoutstanding at December 31, 2022 and 2021,
respectively |
|
|
2 |
|
|
|
2 |
|
Capital in excess of par
value |
|
|
581,893 |
|
|
|
528,659 |
|
Accumulated other
comprehensive income (loss) |
|
|
6,047 |
|
|
|
(2,671 |
) |
Accumulated deficit |
|
|
(470,204 |
) |
|
|
(340,589 |
) |
Total Shareholders' Equity |
|
|
117,738 |
|
|
|
185,401 |
|
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY |
|
$ |
318,237 |
|
|
$ |
320,164 |
|
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