Latest news with #CNS-targeted
Business Wire
01-07-2025
- Business
- Business Wire
Quiver Bioscience Secures Non-Dilutive Funding to Enhance CNS Genetic Medicine Drug Discovery Platform Capabilities and Advance Powerful AI/ML-Based In Vitro / In Silico Predictor of CNS Drug Safety
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Quiver Bioscience ('Quiver'), a discovery technology and therapeutics company advancing programs for treatment of serious central nervous system (CNS) disorders and chronic pain, today announced receipt of a Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH). The Phase II grant, awarded by the National Institute of Neurological Disease and Stroke (NINDS) is titled ' Safe-OPTION: Optical Physiology To Interrogate Oligonucleotide Neurotoxicity.' It will provide Quiver with $2.15 million over three years to continue development of an integrated platform for improved prediction of safety and tolerability of CNS-targeted antisense oligonucleotide (ASO) therapeutics. ASO therapeutics are increasingly being developed for the treatment of neurological conditions, as they offer precision regulation of disease target activity. Despite demonstrated clinical success of ASOs for neurological disorders, ASO drug development in this area faces the challenge of ensuring that candidates avoid modality-specific neurotoxicity, requiring lengthy preclinical safety studies in several animal species. Incorporating improved earlier predictors of ASO-induced acute and delayed onset neurotoxicity will allow for more efficient ASO drug development. Quiver has built an exceptional drug discovery platform which combines disease relevant human neuronal models, all-optical electrophysiology-based functional readouts, and AI/ML-enabled analytics to provide unique insights into neurological disorders and accelerate drug development. With the new SBIR funding, Quiver will further augment their existing ASO drug development capabilities by applying their platform to better prediction of ASO CNS toxicity using a combination of machine learning (ML)-based in silico ASO design tools and a series of all-optical electrophysiology platform-based in vitro neuronal functional assays, benchmarking their predictive algorithm with empirical data from rodent studies. This enhanced platform will accelerate the ASO drug development process by rapidly identifying lead molecules with better probability of clinical success while also reducing costs and time associated with preclinical animal studies. Quiver's objectives in this newly funded project are aptly aligned with the FDA's push towards replacement of preclinical animal safety studies with New Approach Methodologies (NAMs), including in vitro human cellular systems and artificial intelligence (AI) / machine learning (ML)-based in silico models. While animal models have been historically valuable in understanding disease biology and facilitating drug development, they do have limitations including the fact that animal studies are not scalable, and they can be prohibitively expensive and time consuming. In addition, due to species-specific variations in biology, animal models may not accurately predict performance of a drug compound in humans. Advanced human cellular models have transformed the field of drug discovery in neurological disorders by allowing for the study of biological mechanisms and evaluation of drug targets in live functional neurons carrying the same genetic basis as neurons in human subjects affected by the disease. Quiver's platform uniquely probes neuronal biology at a scale that is not possible with alternative technologies and fills a gap in the ability to directly target fundamental neurophysiological mechanisms for therapeutic discovery. With the added capabilities built partly with this new funding, Quiver now addresses the outstanding challenges associated with predicting and mitigating neurotoxicity related to ASO therapeutics. Quiver has already leveraged their unique ASO design and in vitro screening platform to advance internal precision gene-targeted therapy programs including their lead asset, an ASO candidate targeting the genetically and clinically validated pain target Na v 1.7, a voltage gated sodium channel implicated in several neuropathic pain disorders. In addition to NIH SBIR awards, Quiver has recently received grant support from key patient advocacy and research groups who are focused on advancing precision medicines for neurological disorders. These include grants from the Dup15q Alliance, the FRAXA Research Foundation, and the KCNT1 Epilepsy Foundation in partnership with CURE Epilepsy and are all intended to support Quiver's drug discovery programs related to each of the three CNS disorders (Dup15q syndrome, Fragile X syndrome (FXS), and KCNT1-related epilepsy, respectively). Quiver recently announced a FXS-focused research collaboration with QurAlis, which will be partially supported by the new FRAXA Research Foundation grant. These partnerships are instrumental to Quiver's mission to accelerate transformative therapeutic discovery for both rare and common disorders of the CNS. About Quiver Bioscience Quiver Bioscience is a technology-driven company established to create transformational medicines for the brain while simultaneously uncovering new biology and novel, effective drug targets. Using advanced single-cell imaging and multi-omics, we are building the world's most information-rich neuronal insight map via our "Genomic Positioning System." Our approach integrates cutting-edge scalable human models, state-of-the-art technology and proprietary engineering, and learning and surrogate AI/ML models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients. For more information, including partnerships and publications describing application of Quiver's GPS to drug discovery, visit or follow us on LinkedIn.
Business Wire
23-05-2025
- Business
- Business Wire
Autobahn Therapeutics Announces Presentation of ABX-002 Phase 1 Clinical Results at the 2025 ASCP Annual Meeting
SAN DIEGO--(BUSINESS WIRE)--Autobahn Therapeutics, a biotechnology company developing restorative treatments for people affected by neuropsychiatric and neuroimmunologic disorders, today announced the company will present the clinical results from its completed Phase 1 trial of ABX-002 at the 2025 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting, taking place May 27-30, 2025, in Scottsdale, AZ. ABX-002 is a highly potent, oral, thyroid hormone beta receptor (TRβ) selective agonist designed to enhance the CNS benefits of thyroid hormone biology for patients suffering from major depressive disorder (MDD), bipolar disorder depression, and other affective illnesses. 'We are excited to share the Phase 1 findings at ASCP, which demonstrated a favorable safety and tolerability profile and enhanced CNS target engagement with ABX-002, and support its evaluation in the ongoing Phase 2 trials in major depressive disorder and bipolar depression,' said Gudarz Davar, M.D., Executive Vice President, Head of Research and Development for Autobahn. 'Despite availability of existing therapies, many individuals with MDD and bipolar depression struggle to achieve adequate relief. Our team remains deeply committed to developing novel, CNS-targeted treatments that we believe have the potential to meaningfully improve the lives of those affected by these debilitating conditions.' Results from the completed Phase 1 trial of ABX-002 in healthy volunteers demonstrated ABX-002 was safe and well tolerated, with no serious adverse events observed. Additionally, ABX-002 demonstrated dose proportional PK and clinical evidence of CNS target engagement consistent with brain-activating thyroid effects, helping inform Phase 2 dose selection. The company is currently evaluating ABX-002 as a potential adjunctive treatment for people with major depressive disorder in the ongoing AMPLIFY Phase 2 trial and as a potential adjunctive treatment for bipolar depression in a separate ongoing Phase 2 trial. Details for the poster presentation can be found below: Title: A Phase 1 Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose Study of the Safety, Pharmacokinetics, and Pharmacodynamics of the Novel Thyromimetic ABX-002 in Healthy Adult Participants Date: Thursday, May 29, 2025 Time: 11:30 a.m. – 1:15 p.m. MT Presenter: Bridgette Franey, M.D., Senior Medical Director, Global Medical Lead - Clinical Development, Autobahn Therapeutics Location: Fairmont Scottsdale Princess, Scottsdale, AZ The abstract and additional details can be found on the 2025 ASCP annual meeting website. About Autobahn Therapeutics Autobahn Therapeutics is a biotechnology company developing a portfolio of neuropsychiatric and neuroimmunologic clinical candidates leveraging its brain-targeting chemistry platform. Autobahn aims to unlock new therapeutic opportunities through precision tuning of CNS exposure, pursuing validated clinical and biologic targets, and guiding development with biomarkers. The company's pipeline is led by ABX-002, a thyroid hormone receptor beta (TRβ) agonist being developed as a potential adjunctive treatment for people with major depressive disorder and bipolar disorder depression. Autobahn Therapeutics is based in San Diego. For more information, visit About ABX-002 ABX-002 is an orally administered, potent and selective thyroid hormone beta receptor (TRβ) agonist designed to enhance the CNS benefits of thyroid hormone biology while also reducing the peripheral liabilities of synthetic thyroid hormone (e.g., triiodothyronine, T3), a treatment which has shown efficacy in numerous placebo-controlled human studies across MDD and bipolar disorder depression. Thyroid hormone agonism has demonstrated activity on cellular energy metabolism pathways, which play an important role on the regulation of brain bioenergetics and may be uniquely suited to address symptoms of atypical depression, a highly prevalent and underserved sub-population of MDD. In nonclinical and clinical studies, ABX-002 has demonstrated optimized PK properties, target engagement in brain regions associated with depression, and an attractive safety and tolerability profile.
