QRL-101 is the only Kv7 ion channel
opener being actively studied for the treatment of
hyperexcitability-induced disease progression in ALS, which occurs
in nearly 50 percent of patients
Kv7 is a clinically validated target to
regulate hyperexcitable state in epilepsy
QurAlis' Phase 1 study in healthy volunteers
of QRL-101 to evaluate biomarkers of both ALS and epilepsy also
underway
CAMBRIDGE, Mass., Dec. 4, 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 first patient with ALS has been
dosed in a Phase 1 clinical trial evaluating QRL-101 in people
living with ALS (QRL-101-04). 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, which is
present in approximately 50 percent of ALS patients.
Kv7 hyperexcitability occurs in both sporadic and genetic forms
of ALS, with the majority caused by the mis-splicing of the KCNQ2
gene pre-mRNA. Kv7 is also a clinically validated target to
regulate the hyperexcitable state in epilepsy. QurAlis recently
announced the company has expanded the development program for
QRL-101 to include epilepsy.
"QRL-101 is the only Kv7 ion channel opener being actively
studied for the treatment of hyperexcitability-induced disease
progression in ALS. The dosing of the first patient with ALS in the
clinical development program of QRL-101 is a significant
milestone," said Kasper Roet, Ph.D.,
CEO and co-founder of QurAlis. "Kv7 is implicated in ALS as well as
epilepsy. We believe that the data from this study, along with the
data from our Phase 1 study evaluating biomarkers of ALS and
epilepsy in healthy volunteers, will be valuable as we advance the
clinical program for QRL-101 in ALS so that we can bring a
much-needed therapeutic option to patients rapidly."
"Preclinical models of QRL-101 show its strong potential to
control motor neuron hyperexcitability-induced neurodegeneration
with an attractive side effect profile," said Leonard H. van den Berg, M.D., Ph.D., professor
of neurology and chair, TRICALS. "ALS is a devastating, fatal
neurodegenerative disease and there are currently no therapies that
can significantly extend patients' lives. We look forward to
results from this Phase 1 study in ALS patients."
QRL-101-04 (NCT06714396) is a Phase 1 proof-of-mechanism (PoM)
single-dose, placebo-controlled clinical trial designed to evaluate
the safety and tolerability of QRL-101 in people living with ALS.
The study is expected to enroll approximately 12 participants with
ALS and will evaluate the impact of QRL-101 on excitability
biomarkers including on the strength-duration time constant (SDTC),
a known predictor of survival in ALS patients. QurAlis is also
conducting a Phase 1 PoM biomarker clinical trial (QRL-101-05;
NCT06681441) to evaluate biomarkers of ALS and epilepsy of QRL-101
in healthy volunteers. These PoM studies together will inform
QurAlis' future development program, including dose levels for
proof-of-concept studies.
QurAlis anticipates reporting topline data from the QRL-101-04
Phase 1 clinical trial 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 amyotrophic lateral sclerosis
(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), a disease
progression biomarker for ALS. 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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SOURCE QurAlis