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The technology company <a href="https://www.unlearn.ai/">Unlearn</a> has joined with <a href="https://quralis.com/">QurAlis</a> to use artificial intelligence (AI) to speed up clinincal trials for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The aim of the collaboration is to minimize variability and increase the statistical strength of QurAlis&#8217; clinical trials that are evaluating their primary ALS therapeutic candidates, QRL-201 and QRL-101.
&#8220;We are excited to partner with Unlearn to help advance our clinical program with AI and other innovative technologies to generate evidence suitable for supporting regulatory decisions and help speed new, lifesaving precision medicines to patients with ALS and other neurodegenerative diseases,&#8221; Kasper Roet, PhD, QurAlis&#8217; founder and CEO, said in a <a href="https://www.prnewswire.com/news-releases/quralis-and-unlearn-announce-collaboration-to-accelerate-and-optimize-als-clinical-trials-with-generative-artificial-intelligence-technologies-301863473.html">company press release</a>.
Unlearn uses an AI technique called machine learning to create &#8220;digital twins&#8221; of patients enrolled in clinical trials. Its patented machine learning models are trained on large volumes of completed clinical data and can predict, at the individual level, the outcomes of these digital twins if they were given the control treatment.
Data collected from each patient is fed to the AI model to generate a digital twin, which will be utilized in randomized controlled trials (RCTs) called <a href="https://www.unlearn.ai/twinrcts">TwinRCTs</a>.
TwinRCTs are designed to provide more statistical power with smaller control groups than traditional trial designs. Many patients are wary of enrolling in clinical trials because they might receive a placebo, but a smaller control group boosts the chance of getting the investigational treatment.
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<h3>Benefits of using AI in ALS clinical trials</h3>
The technology will also speed up a trial&#8217;s enrollment process, accelerating a therapy&#8217;s time to market.
&#8220;By using machine learning to leverage the wealth of existing patient data from completed clinical trials, our technology significantly shortens typical timelines by months while generating evidence suitable for supporting regulatory decisions,&#8221; said Charles Fisher, PhD, Unlearn&#8217;s founder and CEO.
At the end of the trial, outcome scores derived from the digital twins will be integrated into the primary trial analysis. This will allow for an accurate estimate of treatment effects and minimize false positives (Type-1 errors), a result that indicates a treatment&#8217;s effectiveness.
&#8220;Advances in machine learning and AI make it possible to enhance trial power to detect a positive result when one truly exists while controlling for Type-1 error and significantly shorten timelines without introducing bias into the study,&#8221; Roet said.
The technology will be used to accelerate the development of QRL-201, a treatment designed to raise stathmin-2 levels and lower the toxic TDP-43 clumps that build up and damage nerve cells in ALS.
It will also boost clinical trials of QRL-101, which is designed to reduce the excessive firing of electrical signals, known as hyperexcitability, seen in about half of ALS cases.
QRL-201 is being investigated in a Phase 1 clinical trial called <a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459">ANQUR (NCT05633459)</a>, which recently <a href="https://alsnewstoday.com/news/dosing-phase-1-clinical-trial-als-qrl-201-protect-neurons/">dosed its first patient</a>. The study is testing the tolerability and safety of ascending doses against a placebo in up to 64 adults with ALS without SOD1 or FUS gene mutations.
The trial is ongoing in Canada and U.S. sites are expected to open soon. QurAlis anticipates <a href="https://alsnewstoday.com/news/phase-1-trial-experimental-als-treatment-qrl-201-opening-eu/">sites in the European Union</a> by the end of the year.
QRL-101 is also undergoing clinical testing in a <a href="https://clinicaltrials.gov/ct2/show/NCT05667779?term=NCT05667779&amp;draw=2&amp;rank=1#contacts">Phase 1 trial (NCT05667779)</a>. The study, which <a href="https://alsnewstoday.com/news/first-participants-dosed-phase-1-trial-als-therapy-qrl-101/">began dosing this year</a>, will assess the therapy&#8217;s safety, tolerability, and pharmacological properties in up to 40 healthy adults at a single center in the Netherlands.
&#8220;The AI technology we&#8217;re developing today will revolutionize the future of clinical research,&#8221; Fisher said.

QurAlis teaming with Unlearn to use AI to improve ALS clinical trials

QurAlis has secured $88 million to advance the clinical development of QRL-101 and QRL-201, its lead candidates for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The new series B financing, a second round of funding after the company met certain research milestones, brings the total investment to $143.5 million.
The proceeds will also support research for candidates in <a href="https://quralis.com/pipeline/">QurAlis&#8217; pipeline</a> targeting components of ALS and the related condition of frontotemporal dementia (FTD).
&#8220;This financing round recognizes our scientific track record and will help us advance the clinical development of our two lead programs in ALS and robust pipeline through near-term value-creating milestones,&#8221; Kasper Roet, PhD, QurAlis&#8217; founder and CEO, said in a <a href="https://www.prnewswire.com/news-releases/quralis-closes-88-million-series-b-financing-to-advance-precision-medicines-for-neurodegenerative-diseases-301765435.html">company press release</a>. &#8220;We are breaking through the barriers of science in our quest to bring much-needed precision therapies to patients.&#8221;
In ALS, progressive muscle weakness is <a href="https://alsnewstoday.com/causes-of-als/">caused</a> by the loss of motor neurons, the nerve cells that transmit impulses from the brain to muscles to control voluntary movements. In about half of cases, motor neuron damage is due to electrical signals firing excessively, referred to as hyperexcitability.
QRL-101, also named QRA-244, is an oral medication designed to selectively target and activate a protein channel called Kv7.2/7.3 to lower motor neurons&#8217; hyperexcitability and improve their survival.
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<h3>Studies underway of Quralis&#8217; candidate therapies</h3>
A first-in-human <a href="https://clinicaltrials.gov/ct2/show/NCT05667779">Phase 1 trial (NCT05667779)</a> is <a href="https://alsnewstoday.com/news/first-participants-dosed-phase-1-trial-als-therapy-qrl-101/">evaluating</a> QRL-101&#8217;s safety, tolerability, and pharmacokinetics — how the drug moves into, through, and out of the body. The study, which is enrolling up to 40 healthy volunteers, ages 18-70, at a single center in the Netherlands, will assess increasing doses of QRL-101 against a placebo.
QRL-201, the other lead compound, is a first-in-class experimental therapy designed to restore the production of STMN2, a protein found 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 almost all ALS patients. Under normal circumstances, STMN2 is produced at high levels in motor neurons for <a href="https://alsnewstoday.com/news/als-progression-linked-stmn2-protein-loss-study/">nerve cell growth and repair</a>.
Regulators in Canada recently <a href="https://alsnewstoday.com/news/trial-qrl-201-aiming-slow-als-progression-opening-canada/">approved QurAlis&#8217; request</a> to launch a Phase 1 clinical trial called <a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459">ANQUR (NCT05633459)</a> to investigate QRL-201&#8217;s safety, tolerability, and pharmacokinetics in up to 64 ALS patients, ages 18-80. Participants will be randomly assigned different doses of the therapy or a placebo, given by injection into the spinal canal.
<a href="https://www.clinicaltrials.gov/ct2/show/NCT05633459#contacts">Recruitment has begun</a> in Montréal, with sites in the U.S. and several European countries expected to open soon.
&#8220;This financing reflects significant investor confidence in the science behind QurAlis&#8217; next-generation precision medicines, world-class team, and commitment to bringing new therapies to patients suffering from ALS and other neurodegenerative diseases,&#8221; said Anne Whitaker, chair of QurAlis&#8217; board of directors.
The financing effort was led by EQT Life Sciences, with investments from Droia Ventures, LSP Dementia Fund, and Sanofi Ventures, and participation from the ALS Investment Fund.
Existing investors include the Dementia Discovery Fund, the LS Polaris Innovation Fund, Mission BioCapital, INKEF Capital, MP Healthcare Venture Management, Amgen Ventures, Dolby Family Ventures, Mitsui Global Investment, Mission Bay Capital, and Sanford Biosciences.
&#8220;QurAlis stands out as a leader in the field of neurodegenerative diseases with its next-generation precision medicines and genetically validated targets,&#8221; said Philip Scheltens, MD, PhD, head of EQT Life Sciences&#8217; LSP Dementia Fund. &#8220;We are extremely excited to join this distinguished group of investors supporting this world-class team to advance what we believe could become life-changing treatments for patients and their families.&#8221;
Cillian King, PhD, managing director at EQT Life Sciences, and Laia Crespo, PhD, partner at Sanofi Ventures, will join the QurAlis board of directors as part of the series B financing agreement.
&#8220;We are fortunate to be funded by this outstanding group of investors who share our commitment to patients with neurodegenerative diseases and our vision to halt disease progression and significantly improve outcomes,&#8221; Roet said.

Tag: QurAlis

QurAlis teaming with Unlearn to use AI to improve ALS clinical trials

QurAlis secures $88M to advance ALS therapies