A SUPER-powered WP Engine Atlas Test Site

Joe&#039;s Atlas Experimentation Site

An ongoing Phase 1 clinical trial of <a href="https://quralis.com/">QurAlis</a>’ experimental therapy QRL-201 in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is expected to open in the U.K. by the end of the year.
The announcement follows the trial’s clearance by the country’s Medicines and Healthcare Products Regulatory Agency.
“We look forward to advancing ANQUR in the U.K. as part of our global strategy focused on bringing breakthrough precision medicines to patients with ALS and other neurodegenerative diseases,” Kasper Roet, PhD, CEO and co-founder of QurAlis, said in a company <a href="https://www.prnewswire.com/news-releases/quralis-receives-clinical-trial-authorisation-cta-in-the-uk-for-qrl-201-a-first-in-class-stathmin-2-precision-therapy-for-als-301873138.html">press release</a>.
The placebo-controlled <a href="https://classic.clinicaltrials.gov/ct2/show/NCT05633459">Phase 1 ANQUR study (NCT05633459)</a> is recruiting patients at three sites in Canada, but is expected to start enrollment in Germany, Belgium, the Netherlands, and Ireland by the end of the year following its <a href="https://alsnewstoday.com/news/phase-1-trial-experimental-als-treatment-qrl-201-opening-eu/">regulatory clearance</a> in those countries. The company also plans to open sites in the U.S.
According to QurAlis, the trial, which will test several QRL-201 doses, has completed enrollment of the first group in Canada and has already dosed the first patient.
ANQUR’s design was recently presented at the <a href="https://www.encals.eu/meetings/barcelona/">European Network to Cure ALS (ENCALS) meeting</a>, July 12-14 in Barcelona, Spain.
[recommended-reading id=82704]
<h3>QRL-201 works to restore STMN2 levels</h3>
ALS is marked by the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movements. With neuronal loss, the brain can no longer control movement, leading to muscle weakness and atrophy.
In most patients, toxic aggregates of the TDP-43 protein build up inside nerve cells, including motor neurons, contributing to their degeneration. This protein is found at high levels in the brain and spinal cord and normally regulates the first step of protein production from a gene.
Research by Kevin Eggan, PhD, QurAlis’ co-founder and former Harvard professor, showed <a href="https://alsnewstoday.com/news/rescuing-levels-stmn2-potential-new-therapy-als-harvard-study/">TDP-43 regulates Stathmin-2</a> (STMN2), a protein produced in motor neurons and involved in neuronal repair. Toxic TDP-43 clumps affect the protein’s function, resulting in low STMN2 levels being produced.
QRL-201 is a first-in-class investigational therapy designed to increase STMN2 levels. It was shown to effectively increase STMN2 levels in <a href="https://alsnewstoday.com/news/antisense-oligonucleotide-stathmin-2-levels-lab-grown-motor-neurons-qrl-201-als-quralis/">lab-grown motor neurons</a> and <a href="https://alsnewstoday.com/news/mda-2023-designer-dna-drug-als-shows-promise-early-studies/">mouse models</a>, reversing several neurodegenerative processes. Because of this, the therapy is expected to slow ALS progression.
The first in-human ANQUR trial will test the safety, tolerability, and pharmacokinetics of multiple ascending doses of QRL-201 in up to 64 adults,  ages 18-80, who developed ALS symptoms within the past two years. Pharmacokinetics refers to a therapy’s movement into, through, and out of the body.
Participants will be randomly assigned to receive either QRL-201 at one of eight doses or a placebo via an injection into the spinal canal. The study is expected to be completed in May 2025.

Phase 1 clinical trial of QRL-201 for ALS approved in the UK

<a href="https://quralis.com/" rel="nofollow">QurAlis</a> soon expects to open its ongoing Phase 1 clinical trial testing QRL-201 in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) at sites in various European Union countries, including Germany, Belgium, the Netherlands, and Ireland.
The company&#8217;s announcement comes after these countries reviewed and accepted an application for the study. The <a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459">Phase 1 ANQUR trial (NCT05633459)</a>, which is currently recruiting in Canada, could start enrolling patients at European sites by year&#8217;s end.
&#8220;This EU Clinical Trial Authorisation marks the second regulatory clearance for QRL-201 as we execute our global strategy focused on bringing breakthrough precision medicines to patients with ALS and other neurodegenerative diseases,&#8221; Kasper Roet, PhD, CEO and co-founder of QurAlis, said in a <a href="https://www.prnewswire.com/news-releases/quralis-receives-clinical-trial-authorisation-cta-in-european-union-eu-for-qrl-201-a-first-in-class-stathmin-2-precision-therapy-for-als-301839151.html">company press release</a>.
[recommended-reading id=84924]
<h3>Trial into safety of varying doses of experimental ALS treatment</h3>
ALS is a progressive neurodegenerative disorder that damages motor neurons, the nerve cells that control voluntary movements, leading to muscle weakness and muscle atrophy (shrinkage).
In most ALS cases, toxic aggregates of the TDP-43 protein accumulate in nerve cells, and they are believed to directly contribute to neurodegeneration. This protein normally works to process messenger RNA, an intermediated molecule made when genes are &#8220;read&#8221; to produce proteins.
<a href="https://alsnewstoday.com/news/rescuing-levels-stmn2-potential-new-therapy-als-harvard-study/">Stathmin-2</a> (STMN2) is the protein most significantly regulated by TDP-43 in people. This protein is important for neural repair and stability, and it is abundant in motor neurons. But its levels drop significantly when TDP-43 is faulty, and stathmin-2 is known to be at <a href="https://alsnewstoday.com/news/antisense-oligonucleotide-stathmin-2-levels-lab-grown-motor-neurons-qrl-201-als-quralis/">lower-than-normal levels</a> in nearly all ALS patients.
QRL-201 is a first-in-class experimental therapy designed to raise STMN2 levels in patients. This has been shown to <a href="https://alsnewstoday.com/news/mda-2023-designer-dna-drug-als-shows-promise-early-studies/">lessen the damage caused by TDP-43 aggregates</a>, and to restore nerve cells&#8217; ability to repair damage. As such, QRL-201 could work to slow or prevent ALS progression.
ANQUR is the first trial evaluating a therapy designed to rescue STMN2 production. It aims to test the safety, tolerability, and pharmacological properties of QRL-201 in up to 64 people with ALS.
Participants will be randomly assigned to one of eight increasing doses of the therapy or to a placebo, both given by an injection into the spinal canal (intrathecal injection). Each dosing group will include eight patients, six being treated with QRL-201 and two being given a placebo. A first group in Canada is fully enrolled and dosing is underway.
The company plans to open additional sites in the U.S. and U.K.
&#8220;We believe QRL-201 has the potential to halt disease progression, which could transform the care and treatment of ALS. We are dedicated to ensuring efficient advancement of our ANQUR clinical trial so that we can fulfill our mission to make a meaningful difference in patients&#8217; lives,&#8221; Roet said.

Phase 1 trial of QRL-201 to open for patients at sites across Europe

A first patient has been dosed in a Phase 1 clinical trial evaluating QRL-201, an experimental antisense oligonucleotide (ASO) molecule in treating <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), the therapy&#8217;s developer, <a href="https://quralis.com/" rel="nofollow">QurAlis</a>, announced.
Dubbed <a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459">ANQUR (NCT05633459)</a>, the study is reported to be the first into a potential therapy that works to increase production of stathmin-2 (STMN2), a <a href="https://alsnewstoday.com/news/als-progression-linked-stmn2-protein-loss-study/">protein needed for neuronal growth</a> and repair. In nearly all ALS patients, <a href="https://alsnewstoday.com/news/antisense-oligonucleotide-stathmin-2-levels-lab-grown-motor-neurons-qrl-201-als-quralis/">STMN2 </a><a href="https://alsnewstoday.com/news/antisense-oligonucleotide-stathmin-2-levels-lab-grown-motor-neurons-qrl-201-als-quralis/">levels</a><a href="https://alsnewstoday.com/news/antisense-oligonucleotide-stathmin-2-levels-lab-grown-motor-neurons-qrl-201-als-quralis/"> are low</a>.
ANQUR is <a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459#contactlocation">expected to enroll</a> up to 64 adults with ALS, ages 18 to 80, and no SOD1 or FUS gene mutations at sites in Canada, Europe, and the U.S. It is testing the safety and tolerability of multiple ascending doses of QRL-201 against a placebo in groups of patients.
The trial currently is ongoing in Canada, and the first dose was given at the University of Montréal Hospital Centre.
&#8220;QRL-201 recently entered the clinic in Canada and we are pleased to dose our first patient. We look forward to advancing the ANQUR clinical trial of QRL-201 for the treatment of ALS so that we can make a meaningful difference in patients&#8217; lives,&#8221; Angela Genge, MD, chief medical officer at QurAlis, said in a <a href="https://www.prnewswire.com/news-releases/quralis-announces-first-patient-dosed-with-qrl-201-a-first-in-class-stathmin-2-precision-therapy-for-als-301790444.html">company press release</a>.
[recommended-reading id=83378]
<h3>Raising stathmin-2 levels may protect neurons, slow ALS progression</h3>
ALS is a progressive condition that affects motor neurons, the nerve cells that control voluntary movements. The loss of these neurons weakens a person&#8217;s muscles. While a handful of <a href="https://alsnewstoday.com/als-treatment/">ALS therapies are approved</a>, their ability to slow the disease&#8217;s advance is limited.
&#8220;There is great need for therapies that could slow disease progression and improve outcomes,” said Merit Cudkowicz, MD, director of the Sean M. Healey &amp; AMG Center for ALS at Massachusetts General Hospital and a member of QurAlis&#8217; clinical advisory board.
Most ALS cases are characterized by abnormalities in the <a href="https://alsnewstoday.com/news/structure-tdp-43-protein-aggregates-characterize-als-identified-first-time/">TDP-43 protein</a>, which forms toxic clumps that damage cells. This protein is normally needed to process messenger RNA, an intermediate molecule that results from genes, which provides the instructions to make proteins.
When TDP-43 is faulty, the mRNA molecule carrying <a href="https://alsnewstoday.com/news/rescuing-levels-stmn2-potential-new-therapy-als-harvard-study/">instructions for stathmin-2 production</a> has an abnormal sequence, resulting in low levels of the STMN2 protein, previous research reported.
Raising stathmin-2 levels also has shown a potential to <a href="https://alsnewstoday.com/news/mda-2023-designer-dna-drug-als-shows-promise-early-studies/">reduce the defects caused by TDP-43 clumping</a> and restore a cell&#8217;s ability to repair neurons. These findings indicate QRL-201 could help to slow disease progression in ALS patients.
&#8220;Stathmin-2 is a well-validated protein important for neural repair and axonal stability and is the most significantly regulated gene by TDP-43 exclusively in humans,&#8221; Genge said. &#8220;Its expression is significantly decreased in nearly all ALS patients and it is the most consistently decreased gene over all sporadic ALS patient data sets.&#8221;
The ongoing trial is testing QRL-201’s safety, tolerability, and pharmacokinetics — how a drug moves into, through, and out of the body — in ALS patients. Participants, in groups of eight people each, will be randomly assigned to one of eight increasing doses of the therapy or to a placebo, both given by intrathecal injection (an injection into the spinal canal).
“This study has the potential to show QRL-201 … could potentially benefit ALS patients who have a loss of STMN2 due to TDP-43 pathology,&#8221; said Cudkowicz, who is also the director and a professor of neurology at Harvard Medical School.

Tag: QRL-201

Phase 1 clinical trial of QRL-201 for ALS approved in the UK

Phase 1 trial of QRL-201 to open for patients at sites across Europe

Dosing begins in Phase 1 trial of QRL-201 in promoting neuronal repair