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 positive interim data from the Company’s Phase 2 IGNITE
proof-of-concept clinical trial in patients with adult-onset
leukoencephalopathy with axonal spheroids and pigmented glia
(ALSP). The interim data, representing the first six patients
following six months of treatment with 20 mg/kg of iluzanebart
(formerly referred to as VGL101), further support the favorable
safety and tolerability profile as was previously seen in healthy
volunteers. In addition, these data demonstrated clear target
engagement and downstream pharmacological activity at 20 mg/kg
consistent with the Company’s previously reported Phase 1 data in
healthy volunteers. Directionally supportive changes at 6 months on
magnetic resonance imaging (MRI) and neurofilament light (NfL)
biomarkers of disease progression in individual patients with ALSP
were also observed.
The Company also reported findings from its
ongoing natural history study, ILLUMINATE, which continued to
provide critical insights on MRI and NfL biomarkers and supports
soluble colony stimulating factor 1 receptor (sCSF1R) as a
potential key biomarker of ALSP disease pathology.
"The positive interim results from our Phase 2
IGNITE trial represent the first clinical data reported from an
interventional study in patients with ALSP and reaffirm our belief
in the potential of iluzanebart as a novel treatment option.
Importantly, we are the first company to show clinical data on
TREM2 agonism as a potential therapeutic approach in patients with
a neurodegenerative disease,” said Ivana Magovčević-Liebisch,
Ph.D., J.D., President and Chief Executive Officer of Vigil. “In
the 6 patients observed in the interim analysis, iluzanebart
demonstrated a favorable safety and tolerability profile,
compelling pharmacological activity, and positive quantifiable
trends in NfL and MRI biomarkers in individual patients.”
“In addition, our ongoing natural history study
ILLUMINATE continues to provide critical insights into ALSP and
biomarkers that we believe correlate with disease progression,”
continued Dr. Magovčević-Liebisch. “We expect to use the promising
findings from IGNITE and ILLUMINATE to engage with the FDA to
initiate discussions regarding a potential accelerated development
pathway. We look forward to further executing on our precision
medicine strategy in neurodegenerative diseases – because patients
cannot wait."
Key Highlights from Phase 2 IGNITE
Interim Data:
- Favorable safety and tolerability
profile, including no hematologic adverse events.
- Predictable pharmacokinetic and
brain penetration profile consistent with Phase 1 data in healthy
volunteers.
- Clear target engagement, based on
sTREM2 levels, and downstream pharmacological activity, based on
sCSF1R and osteopontin levels, at 20 mg/kg, consistent with Phase 1
data in healthy volunteers.
- Directionally supportive changes in both NfL and MRI
measurements on ventricular volume and gray matter volume in
individual patients.
- We believe the quality and consistency of the interim data
further support the continuation of IGNITE without
modification.
Key Updates from Natural History Study
ILLUMINATE:
- sCSF1R and NfL levels are remarkably altered in ALSP,
supporting our hypothesis that these are key biomarkers of disease
pathology.
- Totality of the data, including longitudinal progression
observed on selected MRI measures and clinical endpoints, support
engagement with regulatory authorities.
- We believe the quality and consistency of data in this interim
analysis support chosen biomarkers for pharmacological
activity.
- MRI measurements on ventricular volume and gray matter volume
are emerging as key indicators of disease progression.
- Interim Montreal Cognitive Assessment (MoCA) and Cortical Basal
Ganglia Functional Scale data support use as clinical endpoints in
ALSP at 12 months.
“ALSP is a rare, devastating and
fast-progressing disease with no approved treatments that target
the underlying cause of the disease or slow its progression," said
Zbigniew Wszolek, M.D., Neurologist at Mayo Clinic. “These interim
results are a great step forward for patients and caregivers
impacted by ALSP and I am encouraged by the progress the Vigil team
has made developing a potential novel treatment option for this
autosomal dominant disease. To date, iluzanebart has been
well-tolerated, and the data seem to suggest that we are seeing
directional changes in CSF1R and positive trends in NfL and MRI
measurements. This is an important milestone for the underserved
ALSP community of patients and caregivers.”
"Today’s results are a reflection of the
dedication and expertise of our incredible team who brings an
unwavering commitment to patients to their work,” concluded Dr.
Magovčević-Liebisch. “We extend a sincere thank you to all who have
contributed to this progress, including our trial participants,
their caregivers, and the clinical investigators. We look forward
to sharing additional data from the IGNITE trial, including results
from all patients in both the 20 mg/kg and 40 mg/kg cohorts at 6
months, in the third quarter of 2024."
The Company also announced that it will host a
virtual webinar to discuss the Phase 2 IGNITE interim data today,
Thursday, November 16, 2023, from 4:30 p.m. to 5:30 p.m. ET.
The event will include prepared remarks by the
Vigil management team who will be joined by David S. Lynch, M.D.,
Ph.D., Consultant Neurologist National Hospital for Neurology &
Neurosurgery, Queen Square & UCL Institute of Neurology, London
& Clinical Lead, Adult Inherited White Matter Disorders Highly
Specialist Service, NHS England.
To access the live webinar, please register here
or visit “Events & Presentations” in the “Investors” section of
the Vigil website at www.vigilneuro.com. An archived replay of the
webinar will be available for approximately 90 days following the
event.
About Phase 2 IGNITE Clinical
Trial
IGNITE is a global Phase 2, open-label
proof-of-concept trial evaluating iluzanebart in approximately 15
patients with symptomatic ALSP who have a confirmed CSF1R gene
mutation. The primary objective of the IGNITE trial is to evaluate
the safety and tolerability of iluzanebart. Secondary outcome
measures include evaluating the effects of iluzanebart on target
engagement and on MRI and NfL biomarkers of disease progression.
Exploratory outcome assessments include the evaluation of clinical
efficacy measures using standard cognitive, motor and functional
assessments of iluzanebart in patients with ALSP. Patients enrolled
in the trial will receive an intravenous (IV) infusion of
iluzanebart at 20 mg/kg or 40 mg/kg approximately every four weeks
for a treatment duration of one year.
About Iluzanebart (VGL101)
Iluzanebart, Vigil’s lead clinical 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.
Iluzanebart 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 ALSP
ALSP 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 United States, 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,
underscoring the high unmet need in this rare indication.
About Vigil Neuroscience
Vigil 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.
Vigil is utilizing the tools of modern neuroscience drug
development across multiple therapeutic modalities in its efforts
to develop precision-based therapies to improve the lives of
patients and their families. Iluzanebart, Vigil’s 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. Vigil is 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 Statements
This press release includes certain disclosures
that contain “forward-looking statements” of Vigil Neuroscience
(“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
potential of iluzanebart as a novel treatment option for patients
with ALSP; Vigil’s beliefs about TREM2 agonism’s importance in
Alzheimer’s disease and ALSP; the progress and timing of the
clinical development of Vigil’s programs, including the
availability of, and expected timing for reporting, interim and
final data from both the IGNITE Phase 2 clinical trial; and the
success and timing of the Company’s interactions with regulatory
authorities, including with the FDA regarding an accelerated
development pathway. 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 related to uncertainties
inherent in the identification and development of product
candidates, including the conduct of research activities and the
conduct of clinical trials; uncertainties as to the availability
and timing of results and data from clinical trials; whether
results from preclinical studies and interim data from clinical
trials will be predictive of the results of final data from
clinical trials; the timing and content of additional regulatory
information from the FDA; whether Vigil’s cash resources will be
sufficient to fund its foreseeable and unforeseeable operating
expenses and capital expenditure requirements; 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 September 30,
2023 and in any subsequent filings Vigil makes 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.
Internet Posting of
Information
Vigil Neuroscience routinely posts information
that may be important to investors in the “Investors” section of
its website at https://www.vigilneuro.com. The company encourages
investors and potential investors to consult our website regularly
for important information about Vigil Neuroscience.
Investor Contact:Leah GibsonVice President,
Investor Relations & Corporate CommunicationsVigil
Neuroscience, Inc.lgibson@vigilneuro.com
Media Contact:Megan McGrathMacDougall
Advisorsmmcgrath@macdougall.bio
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