QRL-101 / QurAlis - LARVOL DELTA

QRL-101-06: A Study Evaluating the Pharmacokinetics of Three QRL-101 Formulations in Healthy Participants (clinicaltrials.gov) - P1 | N=25 | Active, not recruiting | Sponsor: QurAlis Corporation | Recruiting ➔ Active, not recruiting

Enrollment closed

QRL-101-04: A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: QurAlis Corporation | Trial primary completion date: Mar 2025 ➔ Nov 2025

Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

QurAlis Announces Topline Data From its Phase 1 Clinical Trial of QRL-101 Evaluating Biomarkers of ALS and Epilepsy in Healthy Volunteers (PRNewswire) - P1 | N=24 | NCT06681441 | Sponsor: QurAlis Corporation | "QurAlis Corporation...today announced positive topline data from QurAlis' Phase 1 proof-of-mechanism (PoM) clinical trial of QRL-101 in healthy volunteers evaluating biomarkers related to amyotrophic lateral sclerosis (ALS) and epilepsy. Topline results demonstrated a dose-dependent, statistically significant decrease in motor nerve excitability threshold tracking (mNETT) strength-duration time constant (SDTC), the co-primary endpoint of the trial related to ALS...The decrease in SDTC observed with QRL-101 in this study was approximately 50% greater than previously reported for the Kv7 potassium channel opener ezogabine in a single-dose study in people with ALS...The study did not reach statistical significance on the co-primary endpoint of TMS-EMG motor evoked potential (MEP) amplitude, another measure of corticospinal excitability."

P1 data • Amyotrophic Lateral Sclerosis • Epilepsy

A Study Evaluating the Pharmacokinetics of Three QRL-101 Formulations in Healthy Participants (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: QurAlis Corporation

New P1 trial

QRL-101-03: A Study Evaluating Multiple Ascending Doses of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=60 | Completed | Sponsor: QurAlis Corporation | Recruiting ➔ Completed

Trial completion

A 3-way Cross-over Study on the Effects of QRL-101 on Transcranial Magnetic Stimulation and Nerve Excitability in Healthy Participants (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: QurAlis Corporation | Recruiting ➔ Completed

Trial completion

QurAlis Doses First Patient With ALS in Phase 1 Clinical Trial Evaluating QRL-101, a First-in-Class Kv7 Precision Therapy for ALS (PRNewswire) - "QurAlis Corporation...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-101-04 (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 anticipates reporting topline data from the QRL-101-04 Phase 1 clinical trial in the first half of 2025."

P1 data • Trial status • Amyotrophic Lateral Sclerosis

A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: QurAlis Corporation

New P1 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

QRL-101-01 – a single, ascending dose study evaluating safety of a KCNQ2/3 modulator in healthy volunteers and next steps for development in ALS (ALS-MND 2024) - "QRL-101-01 included single doses of QRL-101, from 1 mg to 96 mg, administered via two different methods of administration. QRL-101 was found to be generally safe and well tolerated. Based on preliminary data, the most common reported adverse events were headache and nausea."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Pain

Development of an exploratory electrophysiology endpoint for use in clinical development in amyotrophic lateral sclerosis (ALS-MND 2024) - "The electrophysiology endpoint is ready for piloting in a clinical trial where altering hyperexcitability is a component of the therapeutic strategy, such as in QurAlis' Kv7.2/7.3 opener, QRL-101 program. ALS is in need of quantitative, clinical outcomes, and this proposed endpoint is scalable to other, and larger, multi-centre, global clinical trials. Use in early phase trials will contribute data for additional phase appropriate pivotal validation studies."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

A 3-way Cross-over Study on the Effects of QRL-101 on Transcranial Magnetic Stimulation and Nerve Excitability in Healthy Participants (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: QurAlis Corporation

New P1 trial

QurAlis Doses First Participant Cohort in Phase 1 Multiple-Ascending Dose (MAD) Clinical Trial Evaluating QRL-101, a First-in-Class Kv7 Precision Therapy for ALS (PRNewswire) - "QurAlis Corporation...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-101-03 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....QurAlis anticipates reporting topline data from the Phase 1 MAD clinical trial of QRL-101 in the first half of 2025."

P1 data • Trial status • Amyotrophic Lateral Sclerosis

A Study Evaluating Multiple Ascending Doses of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: QurAlis Corporation

New P1 trial

A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=88 | Completed | Sponsor: QurAlis Corporation | Recruiting ➔ Completed | N=128 ➔ 88

Enrollment change • Trial completion

Evaluating the Safety, Tolerability, and Pharmacokinetics of QRL-101 in Two Phase 1 Studies: QRL-101-01 in Healthy Adults and QRL-101-02 in Adults with ALS (AAN 2024) - "The findings from these studies will be used to advance the development of QRL-101, and other next-generation precision medicines for people living with ALS and other neurodegenerative diseases."

Clinical • P1 data • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders

Evaluating the Safety Tolerability and Pharmacokinetics of QRL-101 in a Single Ascending Dose Study in Healthy Adults and a Multiple Ascending Dose Study with Exploratory Electrophysiological Markers in adults living with ALS (ALS-MND 2023) - "The findings from these studies will be used to advance the development of QRL-101 as well as provide electrophysiological information that may be used in the development of other next-generation precision medicines for people living with ALS. angela.genge@mcgill.ca"

Clinical • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders • Frontotemporal Lobar Degeneration

A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=128 | Recruiting | Sponsor: QurAlis Corporation | N=72 ➔ 128 | Trial completion date: Sep 2023 ➔ Jan 2024 | Trial primary completion date: Sep 2023 ➔ Jan 2024

Enrollment change • Trial completion date • Trial primary completion date

QurAlis and Unlearn Announce Collaboration to Accelerate and Optimize ALS Clinical Trials With Generative Artificial Intelligence Technologies (PRNewswire) - "QurAlis Corporation...today announced they have entered into a collaboration to accelerate and optimize QurAlis' clinical program in ALS with Unlearn's advanced generative artificial intelligence (AI) technology....The collaboration aims to reduce variability and increase the study power in QurAlis' clinical trials for QRL-201 and QRL-101, the Company's lead product candidates in ALS."

Licensing / partnership • Amyotrophic Lateral Sclerosis • CNS Disorders

A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=72 | Recruiting | Sponsor: QurAlis Corporation | N=40 ➔ 72 | Trial completion date: May 2023 ➔ Sep 2023 | Trial primary completion date: May 2023 ➔ Sep 2023

Enrollment change • Trial completion date • Trial primary completion date

QurAlis secures $88M to advance ALS therapies (ALS News Today) - “QurAlis has secured $88 million to advance the clinical development of QRL-101 and QRL-201, its lead candidates for amyotrophic lateral sclerosis (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 QurAlis’ pipeline targeting components of ALS and the related condition of frontotemporal dementia (FTD).”

Financing • Amyotrophic Lateral Sclerosis • CNS Disorders • Frontotemporal Lobar Degeneration

QurAlis Announces First-in-Human Dose in Phase 1 Clinical Trial of QRL-101, a First-in-Class Kv7 Precision Therapy for ALS (PRNewswire) - “QurAlis Corporation…today announced that the company recently initiated dosing in a Phase 1 clinical trial of QRL-101 (QRA-244), a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS….This first-in-human Phase 1 study (NCT05667779) is a randomized, double-blind, placebo-controlled, single-ascending-dose, single-site clinical trial designed to evaluate the safety, tolerability, and pharmacokinetics of QRL-101 in adult healthy volunteers.”

Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: QurAlis Corporation | Not yet recruiting ➔ Recruiting

Enrollment open

A Study Evaluating Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants (clinicaltrials.gov) - P1 | N=40 | Not yet recruiting | Sponsor: QurAlis Corporation

New P1 trial