MeiraGTx to Present 12-month Data from All Cohorts of the Completed Phase 1 AQUAx Clinical Study and an Update on the Company’s Recently Initiated Phase 2 Study of AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia on Tuesday, June 27,
23 June 2023 - 6:01AM
MeiraGTx Holdings plc (NASDAQ: MGTX), a vertically
integrated, clinical stage gene therapy company, today announced it
will host a conference call and webcast to present an update on the
Company’s AAV-hAQP1 clinical program for the treatment of grade 2/3
radiation-induced xerostomia (RIX) on Tuesday, June 27, 2023, at
8:00 a.m. ET.
The presentation will include:
1) Data from the completed Phase 1 AQUAx clinical study for the
24 patients treated with AAV-hAQP1 in the unilateral and bilateral
cohorts:
- Safety and tolerability
- 12-month data for PRO assessments of xerostomia symptoms in the
unilateral cohorts (n=12)
- 12-month data for PRO assessments of xerostomia symptoms in the
bilateral cohorts (n=12)
- Change over time in the objective measure of saliva flow out to
12 months for both bilateral and unilateral cohorts
- Long-term follow up data to 2 or 3 years for the participants
who have reached those timepoints
2) Summary of biopsy data from a subset of patients in the Phase
1 National Institutes of Health (NIH) study of AAV-hAQP1 in RIX
demonstrating parotid transduction and durability out to at least
24-months post-treatment.
3) Overview of the recently initiated randomized, double blind,
placebo-controlled Phase 2 study of AAV-hAQP1 for the treatment of
RIX, now enrolling patients.
A question-and-answer session will follow the formal
presentation.
To register and attend the event, please click here.
A live webcast of the call, as well as a replay, will be
available on the Investors page of the Company’s website at
www.investors.meiragtx.com/.
About the Phase 1 AQUAx Clinical Trial
The Phase 1 AQUAx clinical trial is an open-label,
non-randomized, dose escalation trial designed to evaluate the
safety of MeiraGTx’s investigational gene therapy AAV-hAQP1 when
administered via Stensen's duct to one or both parotid glands in
patients who have been diagnosed with grade 2 or 3
radiation-induced xerostomia and who have remained cancer free for
at least five years (or at least two years if HPV+) after receiving
radiation treatment for head and neck cancer. Primary endpoint of
the trial is safety, with efficacy endpoints including patient
reported measures of xerostomia symptoms and the evaluation of the
change in parotid gland salivary output after treatment with
AAV-hAQP1.
About the Phase 1 NIH StudyThe Phase 1 NIH
study is an open-label, dose-escalation study evaluating the safety
of a single administration of an adeno-associated virus vector
encoding human aquaporin-1 to one parotid salivary gland in
individuals with irradiation-induced parotid salivary
hypofunction.
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 which allows tight, 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, including inherited
retinal diseases and 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 the development of AAV-hAQP1
and our clinical data and reporting of such data and the timing of
results of data, 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 most recent quarterly report on
Form 10-Q or annual report on Form 10-K or subsequent 8-K reports,
as filed with the Securities and Exchange Commission. 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.
ContactsInvestors:MeiraGTxInvestors@meiragtx.com
Media:Jason Braco, Ph.D.LifeSci
Communicationsjbraco@lifescicomms.com
MeiraGTx (NASDAQ:MGTX)
Historical Stock Chart
From Jun 2024 to Jul 2024
MeiraGTx (NASDAQ:MGTX)
Historical Stock Chart
From Jul 2023 to Jul 2024