MeiraGTx Announces AAV-CNGB3 Granted Fast Track Designation by U.S. FDA for Treatment of Achromatopsia
August 20 2018 - 8:00AM
MeiraGTx Holdings Plc (NASDAQ:MGTX), a vertically integrated,
clinical stage gene therapy company, today announced that the U.S.
Food and Drug Administration (FDA) has granted Fast Track
designation for its AAV-CNGB3 gene therapy product candidate for
the treatment of achromatopsia (ACHM) caused by mutations in the
CNGB3 gene.
ACHM is an inherited retinal disease that severely limits a
person’s sight by preventing cone photoreceptors in the eye from
functioning. Individuals with ACHM are often legally blind from
birth, have extreme sensitivity to light, and experience
involuntary eye movements. AAV-CNGB3 is an investigational gene
therapy treatment designed to restore cone function, delivered to
the cone receptors at the back of the eye via subretinal
injection.
“We are thrilled by this designation and the continued
recognition by the FDA of AAV-CNGB3’s potential to become a
much-needed treatment option for ACHM patients suffering from this
debilitating disease,” said Alexandria Forbes, Ph.D.,
president and chief executive officer of MeiraGTx. “Fast Track
designation is not just a milestone for the company, but also for
the ACHM community. With the ability to communicate closely and
often with the FDA, we are hopeful that we can expedite our
commitment to bring a safe and effective therapy to patients in
need.”
AAV-CNGB3 has been granted orphan drug designation (ODD) by the
FDA and orphan medicinal product designation by the European
Medicines Agency (EMA), as well as rare pediatric disease
designation by the FDA and PRIME designation by the EMA, for the
treatment of ACHM caused by mutations in the CNGB3 gene. MeiraGTx
is currently conducting a Phase 1/2 clinical trial of AAV-CNGB3 in
both adult and pediatric patients, with 14 patients treated to
date. Eleven adult patients have been treated in three dose
escalation cohorts and three pediatric patients have now been
treated in the extension phase of the study. The company
anticipates completing dosing of up to eight pediatric patients in
the second half of 2018.
The FDA’s Fast Track process is designed to expedite the
development and review of drugs used to treat serious conditions
and fill an unmet medical need. Fast Track designation enables the
company to have early and frequent communication with the FDA
throughout the drug development and review process, with the
potential for faster drug approval and patient access. Today’s
announcement marks the second Fast Track designation granted to
MeiraGTx in 2018. In April 2018, the company was also granted Fast
Track designation for AAV-RPGR for the treatment of another
debilitating ocular disorder, X-linked retinitis pigmentosa (XLRP)
due to defects in the RPGR gene.
About Achromatopsia Achromatopsia is an
inherited retinal disorder that specifically prevents cone
photoreceptors from functioning. ACHM is characterized by severely
reduced visual acuity of 20/200 or worse, disabling light
sensitivity (photoaversion) and involuntary back and forth eye
movements (nystagmus). ACHM occurs in approximately one in 30,000
people in the United States, with 92 percent of cases caused by
mutations
in CNGB3 and CNGA3 genes. Currently, there
are no effective treatments for this disease.
About MeiraGTx MeiraGTx (NASDAQ:MGTX)
is a vertically integrated, clinical stage gene therapy company
with four ongoing clinical programs and a broad pipeline of
preclinical and research programs. MeiraGTx has core
capabilities in viral vector design and optimization and gene
therapy manufacturing, as well as a potentially transformative gene
regulation technology. 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: inherited
retinal diseases, severe forms of xerostomia and neurodegenerative
diseases. Though initially focusing on the eye, salivary gland and
central nervous system, MeiraGTx intends to expand its
focus in the future 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 StatementsThis 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 product
pipeline, anticipated product benefits, goals and strategic
priorities, product candidate development, growth expectations or
targets and pre-clinical and clinical data, as well as statements
that include the words “expect,” “intend,” “plan,” “believe,”
“project,” “forecast,” “estimate,” “may,” “should,” “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, acquire additional capital, identify
additional and develop existing product candidates, continue
operating as a going concern, successfully execute strategic
priorities, bring product candidates to market, build-out the
manufacturing facility 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; 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; 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; litigation risks; and the other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarterly period ended June
30, 2018 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
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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
or
Media: W2O pure Kelly Boothe (415)
946-1076kboothe@w2ogroup.com
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