Kv7.2

Saniona (OMX: SANION), a clinical-stage biopharmaceutical company, today announces significant progress in the preclinical development of SAN2355, a new generation Kv7.2/Kv7.3 activator aimed at treating focal onset seizures. The company has identified a stable solid form of the compound and completed synthesis optimization, enabling a robust, scalable manufacturing process. As a result, Saniona has successfully produced 4 kg of SAN2355 for GLP toxicology studies. “We are delighted to have completed the analytical development, synthesis optimization, and production of the GLP toxicology batch. We expect the remaining preclinical work to take about nine months, which means that SAN2355 could enter Phase 1 clinical trials by Q3 2025,” said Janus Schreiber Larsen, Chief Development Officer at Saniona. “SAN2355 is a key asset in our epilepsy portfolio. Kv7 activators have previously been clinically and commercially validated for treating refractory focal onset epilepsy, but no products are currently available on the market. SAN2355 stands out due to its unique selectivity profile and has the potential to become best in class, addressing significant unmet needs in the epilepsy market,” added Thomas Feldthus, CEO of Saniona. The GLP toxicology batch was produced with assistance from the Danish firm Niels Clauson-Kaas, and the project is now ready for preclinical toxicity and safety studies, in compliance with GLP standards. About Kv7 Channels and Epilepsy Epilepsy is a neurological disorder characterized by recurrent seizures, affecting millions globally. Around 30% of patients do not respond to existing treatments, highlighting a critical unmet need. Kv7 channels are crucial for regulating potassium ion transport across neuronal membranes, helping to prevent uncontrolled nerve impulses. Kv7.2 and Kv7.3 subunits, primarily expressed in the brain, play a key role in dampening overactive neurons, thus preventing seizures. While the non-selective Kv7 activator retigabine demonstrated proof-of-concept for treating resistant focal onset seizures, it was withdrawn from the market due to side effects unrelated to its target. About SanionaSaniona (OMX: SANION) is a clinical-stage biopharmaceutical company leading the way in ion channel modulation for the treatment of epilepsy and other neurological disorders. Saniona’s epilepsy pipeline features SAN711, a Phase 2-ready candidate drug targeting absence seizures, SAN2219 for acute repetitive seizures, and SAN2355, addressing refractory focal onset seizures. Beyond epilepsy, Saniona oversees four clinical programs poised for collaboration. Tesofensine, Saniona’s most advanced candidate, is progressing towards regulatory approval for obesity in Mexico through a partnership with Medix. Tesomet™ is ready for Phase 2b, targeting rare eating disorders, while SAN903 is ready for Phase 1 for inflammatory bowel disease and SAN2465 is set for preclinical development for major depressive disorder. Saniona has esteemed partners, including Boehringer Ingelheim GmbH, Productos Medix, S.A de S.V, AstronauTx Limited, and Cephagenix ApS. Saniona is based in Copenhagen and listed on Nasdaq Stockholm Main Market.For more information, please visit www.saniona.com.

QRL-101 is the only Kv7.2/7.3 ion channel opener being actively studied for the treatment of hyperexcitability-induced disease progression in ALS Kv7.2/7.3 is a clinically validated target to regulate the hyperexcitable state in epilepsy Company initiates exploratory Phase 1 proof-of-mechanism study in healthy volunteers to characterize the potential anti-seizure effects of QRL-101 CAMBRIDGE, Mass., September 19, 2024 – 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 company is expanding its Kv7 development program to include epilepsy. The Company’s lead investigational candidate, QRL-101 is the only Kv7.2/7.3 ion channel opener being actively studied for the treatment of hyperexcitability-induced disease progression in ALS, which is present in approximately 50 percent of ALS patients. Kv7 is a clinically validated target to regulate the hyperexcitable state in epilepsy. QurAlis also announced the initiation of an exploratory Phase 1 proof-of-mechanism electroencephalogram biomarker study in healthy volunteers of QRL-101 to characterize the potential anti-seizure effects of QRL-101. “Epilepsy, one of the most common and most disabling neurological seizure disorders, is characterized by spontaneous recurrent seizures, which disrupt normal brain functions, lead to neuronal loss, and result in cognitive and emotional deficits. In about one-third of people living with epilepsy, the seizures are resistant to current treatments; so more effective treatments are urgently needed,” said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. “QRL-101, is a highly selective Kv7.2/7.3 ion channel opener, which in preclinical models shows a strong potential to control motor neuron hyperexcitability-induced neurodegeneration with an attractive side effect profile. Since Kv7 is a clinically validated target in controlling hyperexcitability in epilepsy, we are excited to expand our scope of QRL-101 into a new therapeutic area and explore the potential of QRL-101 in epilepsy so that QurAlis can continue the goal of making a real difference in patients’ lives.” 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. The activation of this channel shows the potential to decrease spinal and cortical motor neuron excitability and to positively affect several electrophysiological biomarkers. This suggests that this may be an effective therapeutic approach in several neurodegenerative and neurological diseases including ALS and epilepsy. About Epilepsy Epilepsy is one of the most common chronic neurological diseases and, according to the Centers for Disease Control, affects more than 65 million people around the world of which 3.4 million are in the U.S. Epilepsy is characterized by unpredictable, recurrent seizures, which are brief episodes of involuntary movement that may involve a part of the body (partial) or the entire body (generalized). Seizure episodes are a result of excessive electrical discharges in a group of brain cells. According to the World Health Organization, recurrent seizures disrupt normal brain functions, lead to neuronal loss, and result in cognitive and emotional deficits. Patients suffer from stigmatization, social isolation, combined with disability, educational underachievement, and poor employment outcomes. The Epilepsy Foundation estimates that one-third of people with epilepsy live with uncontrollable seizures because no available treatments are effective. 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.

QRL-101 aims to reduce hyperexcitability-induced neurodegeneration, which is present in approximately 50 percent of all ALS patients Completed Phase 1 single-ascending dose (SAD) clinical trial of QRL-101 enrolled 88 participants; no reported significant safety concerns or serious adverse events MAD data expected to be reported in first half of 2025; results will support larger global studies in people living with ALS CAMBRIDGE, Mass., Sept. 10, 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 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 is a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS. Kv7.2 is a mis-spliced protein in sporadic ALS patients. 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. The dose range of QRL-101 for this MAD study was determined by results from QurAlis' Phase 1 single-ascending dose (SAD) clinical trial (QRL-101-01; NCT05667779). Of the 88 healthy participants in the SAD clinical trial, no significant safety concerns or serious adverse events have been reported for QRL-101. "We are excited to complete dosing of our first participant cohort in our Phase 1 MAD clinical trial of QRL-101. In the SAD study, QRL-101 was shown to be well tolerated, with no significant safety concerns or serious adverse events," said Doug Williamson, M.D., chief medical officer of QurAlis. "ALS is a devastating, fatal neurodegenerative disease and there are currently no therapies that can significantly extend patients' lives. QRL-101 has the potential to be a first-in-class effective therapy for ALS patients suffering from hyperexcitability-induced motor neuron degeneration. We look forward to advancing the clinical program for QRL-101 so QurAlis can bring much-needed therapies to people living with ALS." "Motor system hyperexcitability occurs in approximately 50 percent of all ALS patients and is linked to potassium channel dysfunction," said Leonard H. van den Berg, M.D., Ph.D., professor of neurology and chair, TRICALS. "QRL-101, is a highly selective Kv7.2/7.3 ion channel opener, which in preclinical models shows a strong potential to control motor neuron hyperexcitability-induced neurodegeneration with an attractive side effect profile. We are encouraged by the findings from the SAD study of QRL-101 and look forward to results from the MAD study." QurAlis anticipates reporting topline data from the Phase 1 MAD clinical trial of QRL-101 in the first half of 2025. More information about the QRL-101 Phase 1 clinical trials can be found at . 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 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). 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 or follow us on X @QurAlisCo or LinkedIn. SOURCE QurAlis