Vigil Neuroscience Receives Positive Opinion from European Medicines Agency on Orphan Drug Designation for VGL101 for the Treatment of ALSP
26 Setembro 2023 - 8:05AM
Vigil Neuroscience, Inc. (Nasdaq: VIGL), a clinical-stage
biotechnology company committed to harnessing the power of
microglia for the treatment of neurodegenerative diseases, today
announced that the Committee for Orphan Medicinal Products (COMP)
of the European Medicines Agency (EMA) has issued a positive
opinion on the Company’s application for orphan drug designation
for VGL101 for the treatment of CSF1R-related leukoencephalopathy,
which includes adult-onset leukoencephalopathy with axonal
spheroids and pigmented glia (ALSP). The Company is currently
evaluating VGL101 in a Phase 2 proof-of-concept trial in patients
with ALSP. VGL101 was previously granted orphan drug designation by
the U.S. Food and Drug Administration for ALSP in July 2022.
“Receiving a positive opinion for orphan drug
designation for VGL101 in the EU is a tremendous achievement in our
efforts to develop a therapy for individuals living with ALSP,
which affects approximately 13,000 people in the EU,” said Ivana
Magovčević-Liebisch, Ph.D., J.D., President and Chief Executive
Officer of Vigil. “ALSP is a rare and rapidly progressive
neurodegenerative disease with no approved treatment. We are
committed to advancing our program through clinical development and
bringing this potentially transformative therapy to these patients
as quickly as possible.”
The EMA may grant orphan drug designation based
on a positive opinion issued by the COMP. The EMA’s orphan drug
designation is available to sponsors developing therapies that aim
to treat or prevent rare, life-threatening or chronically
debilitating conditions that affect no more than five in 10,000
people in the EU, and for which no treatment is approved. Medicines
that meet the EMA’s orphan drug designation criteria qualify for
certain benefits, such as reduced regulatory fees, protocol
assistance, research grants, and, subject to obtaining and
maintaining orphan medicine status, up to ten years of market
exclusivity in the EU upon approval.
About the Phase 2 IGNITE
TrialThe Company’s ongoing Phase 2 IGNITE trial is a
global, open-label clinical trial evaluating VGL101 in
approximately 15 patients with symptomatic ALSP who have a
confirmed CSF1R gene mutation. As part of the protocol, patients
will receive an intravenous (IV) infusion of VGL101 at 20 mg/kg or
40 mg/kg approximately every four weeks, for a treatment duration
of one year. The primary objective of the IGNITE trial is to
evaluate the safety and tolerability of VGL101. Secondary
objectives include evaluating the impact of VGL101 on magnetic
resonance imaging (MRI) and its pharmacodynamic effect on fluid
biomarkers in patients with symptomatic ALSP. Clinical efficacy
outcome measures are also being collected as exploratory endpoints.
In the fourth quarter of 2023, the Company expects to report
interim 6-month data from the IGNITE trial in the first 6 patients
who have received 20 mg/kg of VGL101.
About VGL101VGL101, Vigil’s
lead product candidate, is a fully human monoclonal antibody
targeting human triggering receptor expressed on myeloid cells 2
(TREM2), which is responsible for maintaining microglial cell
function. TREM2 deficiency is believed to be a driver of certain
neurodegenerative diseases. VGL101 is in development for rare
microgliopathies, such as ALSP, as well as other neurodegenerative
diseases for which TREM2 and/or microglia deficiency is believed to
be a key driver of disease pathway.
About ALSPALSP is a rare,
inherited, autosomal dominant neurological disease with high
penetrance. It is caused by a mutation to the CSF1R gene and
affects an estimated 10,000 people in the US, with similar
prevalence in Europe and Japan. The disease generally presents in
adults in their forties, is diagnosed through genetic testing and
established clinical/radiologic criteria and is characterized by
cognitive dysfunction, neuropsychiatric symptoms, and motor
impairment. These symptoms typically exhibit rapid progression with
a life expectancy of approximately six to seven years on average
after diagnosis, causing significant patient and caregiver burden.
There are currently no approved therapies for the treatment of
ALSP, underlining the high unmet need in this rare indication.
About Vigil NeuroscienceVigil
Neuroscience is a clinical-stage biotechnology company focused on
developing treatments for both rare and common neurodegenerative
diseases by restoring the vigilance of microglia, the sentinel
immune cells of the brain. We are utilizing the tools of modern
neuroscience drug development across multiple therapeutic
modalities in our efforts to develop precision-based therapies to
improve the lives of patients and their families. VGL101, our lead
clinical candidate, is a fully human monoclonal antibody agonist
targeting human triggering receptor expressed on myeloid cells 2
(TREM2) in people with adult-onset leukoencephalopathy with axonal
spheroids and pigmented glia (ALSP), a rare and fatal
neurodegenerative disease. We are also developing VG-3927, a novel
small molecule TREM2 agonist, to treat common neurodegenerative
diseases associated with microglial dysfunction, with an initial
focus on Alzheimer’s disease (AD) in genetically defined
subpopulations.
Forward-Looking StatementsThis press release
includes certain disclosures that contain “forward-looking
statements” of Vigil Neuroscience, Inc.’s (“Vigil” or the
“Company”) that are made pursuant to the safe harbor provisions of
the federal securities laws, including, without limitation, express
or implied statements regarding: the progress and timing of the
clinical development of Vigil’s programs, including VGL101 and the
potential impact of the positive opinion of the Committee for
Orphan Medicinal Products on such programs. Forward-looking
statements are based on Vigil’s current expectations and are
subject to inherent uncertainties, risks and assumptions that are
difficult to predict. Factors that could cause actual results to
differ include, but are not limited to, risks and uncertainties
that are inherent in clinical stage therapeutic development; risks
and uncertainties as to whether the Company will obtain and
maintain orphan medicinal product designation in the E.U., the
receipt of such designation may not speed the development or any
potential marketing authorization of Vigil’s therapies, or
determine if Vigil ultimately is able to obtain and maintain a
period of E.U. orphan medicinal product market exclusivity; as well
as the risks and uncertainties identified in the Company’s filings
with the Securities and Exchange Commission (SEC), including
Vigil’s Quarterly Report on Form 10-Q for the quarter ended June
30, 2023 and in any subsequent filings it may make with the SEC.
Forward-looking statements contained in this announcement are made
as of this date, and Vigil undertakes no duty to update such
information except as required under applicable law. Readers should
not rely upon the information on this page as current or accurate
after its publication date.
Investor Contact:Leah Gibson Vice President,
Investor Relations & Corporate CommunicationsVigil
Neuroscience, Inc.lgibson@vigilneuro.com
Media Contact:Megan McGrathMacDougall
Advisorsmmcgrath@macdougall.bio
Vigil Neuroscience (NASDAQ:VIGL)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Vigil Neuroscience (NASDAQ:VIGL)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024