QRL-101 aims to reduce
hyperexcitability-induced neurodegeneration, which is present in
approximately 50 percent of all ALS patients
Completed Phase 1 single-ascending dose (SAD)
clinical trial of QRL-101 enrolled 88 participants; no reported
significant safety concerns or serious adverse events
MAD data expected to be reported in
first half of 2025; results will support larger global studies in
people living with ALS
CAMBRIDGE, Mass., Sept. 10,
2024 /PRNewswire/ -- QurAlis Corporation ("QurAlis"),
a clinical-stage biotechnology company driving scientific
breakthroughs into powerful precision medicines that have the
potential to alter the trajectory of amyotrophic lateral sclerosis
(ALS), frontotemporal dementia (FTD), and other neurodegenerative
and neurological diseases, today announced that the company
recently completed dosing of the first participant cohort in the
Phase 1 multiple-ascending dose (MAD) clinical trial evaluating
QRL-101 (QRL-101-03; NCT06532396). QRL-101 is a first-in-class
selective Kv7.2/7.3 ion channel opener for the treatment of
hyperexcitability-induced disease progression in ALS. Kv7.2 is a
mis-spliced protein in sporadic ALS patients.
QRL-101-03 is a randomized, double-blind, placebo-controlled,
single-site Phase 1 clinical trial designed to evaluate the safety,
tolerability, and pharmacokinetics of multiple-ascending doses of
QRL-101 in adult healthy volunteers. The study is expected to
enroll approximately 60 participants, who will be randomized in a
9:3 ratio of QRL-101 to placebo into five planned cohorts. The dose
range of QRL-101 for this MAD study was determined by results from
QurAlis' Phase 1 single-ascending dose (SAD) clinical trial
(QRL-101-01; NCT05667779). Of the 88 healthy participants in the
SAD clinical trial, no significant safety concerns or serious
adverse events have been reported for QRL-101.
"We are excited to complete dosing of our first participant
cohort in our Phase 1 MAD clinical trial of QRL-101. In the SAD
study, QRL-101 was shown to be well tolerated, with no significant
safety concerns or serious adverse events," said Doug Williamson, M.D., chief medical officer of
QurAlis. "ALS is a devastating, fatal neurodegenerative disease and
there are currently no therapies that can significantly extend
patients' lives. QRL-101 has the potential to be a first-in-class
effective therapy for ALS patients suffering from
hyperexcitability-induced motor neuron degeneration. We look
forward to advancing the clinical program for QRL-101 so QurAlis
can bring much-needed therapies to people living with ALS."
"Motor system hyperexcitability occurs in approximately 50
percent of all ALS patients and is linked to potassium channel
dysfunction," said Leonard H. van den
Berg, M.D., Ph.D., professor of neurology and chair,
TRICALS. "QRL-101, is a highly selective Kv7.2/7.3 ion channel
opener, which in preclinical models shows a strong potential
to control motor neuron hyperexcitability-induced neurodegeneration
with an attractive side effect profile. We are encouraged by the
findings from the SAD study of QRL-101 and look forward to results
from the MAD study."
QurAlis anticipates reporting topline data from the Phase 1 MAD
clinical trial of QRL-101 in the first half of 2025.
More information about the QRL-101 Phase 1 clinical trials can
be found at www.clinicaltrials.gov.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic
lateral sclerosis (ALS), also known as
Lou Gehrig's disease, is a progressive
neurodegenerative disease impacting nerve cells in the brain
and spinal cord, reducing muscle function and control. ALS
can be traced to mutations in more than 25 different genes
and is often caused by a combination of multiple sub-forms of the
condition. Cases usually cannot be predicted, although a small
percentage are inherited. ALS has a devastating impact
on patients and families. ALS patients' average life
expectancy is three years; after diagnosis patients only have two
years to live. There is currently no cure for the
disease.
About Kv7
Kv7.2/7.3 is a voltage-gated potassium
channel whose role is crucial for the regulation of neuronal
excitability and membrane potential. Kv7.2 is mis-spliced
in sporadic ALS leading to loss of function and
abnormal electrical activity in the spinal cord and brain. The
activation of this channel shows the potential to decrease spinal
and cortical/motor neuron excitability and to positively
affect CMAP (compound muscle action potential). This
suggests that this may be an effective therapeutic approach
for ALS patients suffering from
hyperexcitability-induced motor neuron
degeneration.
About QurAlis Corporation
At
QurAlis, we are neuro pioneers on a quest to
cure. We work with a relentless pursuit of knowledge, a precise
attention to craft, and an optimistic mindset to
discover and develop effective precision medicines that have the
potential to alter the trajectory of amyotrophic
lateral sclerosis (ALS), frontotemporal
dementia (FTD), and other
neurodegenerative and neurological diseases. Founded by an
internationally recognized team of neurodegenerative
biologists from Harvard Medical
School and Harvard
University, QurAlis is advancing a robust
precision medicine pipeline with therapeutic candidates aimed at
modifying severe disease pathology in defined patient populations
based on both disease-causing genetic mutation(s) and
clinical biomarkers. For more information, please visit
www.quralis.com or follow us on X @QurAlisCo
or LinkedIn.
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