Clinical Trials
8
2 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
N/A
Active, not recruiting
2
25.0%
Completed
5
62.5%
Recruiting
1
12.5%
No approval data available
- QurAlis reported first-time evidence of target engagement with selective Kv7.2/7.3 ion channel opener QRL-101 in ALS patients, showing reduced motor-neuron hyperexcitability compared to placebo. - The Phase 1 proof-of-mechanism trial demonstrated consistent safety and tolerability profiles, with no serious adverse events or discontinuations reported in the 12-participant study. - QRL-101 targets hyperexcitability caused by KCNQ2 gene mis-splicing linked to TDP-43 dysfunction, with the company planning Phase 2 trials for both ALS and developmental epileptic encephalopathies. - Preclinical studies show QRL-101 is more potent than ezogabine with potential for fewer adverse events, representing a potentially best-in-class approach to treating hyperexcitability-driven neurological diseases.
- Quiver Bioscience received a $2.15 million Phase II SBIR grant from the National Institute of Neurological Disease and Stroke to develop an integrated platform for predicting safety of CNS-targeted antisense oligonucleotide therapeutics. - The three-year "Safe-OPTION" project will combine machine learning-based in silico ASO design tools with all-optical electrophysiology platform-based in vitro neuronal functional assays to better predict ASO CNS toxicity. - The enhanced platform aims to accelerate ASO drug development by rapidly identifying lead molecules with better probability of clinical success while reducing costs and time associated with preclinical animal studies. - Quiver's drug discovery platform combines disease-relevant human neuronal models, all-optical electrophysiology-based functional readouts, and AI/ML-enabled analytics to provide insights into neurological disorders.
• QRL-101 demonstrated a dose-dependent, statistically significant decrease in SDTC, a biomarker linked to ALS disease progression and mortality, with effects approximately 50% greater than the previous Kv7 opener ezogabine. • The clinical trial showed significant impact on epilepsy-related biomarkers, including TMS-EMG intracortical facilitation and EEG spectral power changes, suggesting potential anti-seizure effects without sedation. • QurAlis plans to advance QRL-101 into proof-of-concept studies for both ALS and epilepsy based on the favorable safety profile and promising biomarker data from this healthy volunteer study.
