mGluR5

Safety of ALX-001 Reinforced by New Data Presented at AD/PD™ 2024 Conference in Lisbon Two Oral Doses Selected for Further Study in Alzheimer’s and Parkinson’s Disease Patients NEW HAVEN, Conn., March 11, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics, a clinical-stage biotechnology company, announced that its lead compound, ALX-001, is ready to proceed to Phase 2 clinical development in Alzheimer’s and Parkinson’s disease. This announcement was made in conjunction with the presentation of positive findings from the company’s Phase 1b multiple ascending dose study at the AD/PD™ 2024 Conference in Lisbon. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The multiple ascending dose study (NCT05804383) examined the safety, tolerability and pharmacokinetics of twice-daily oral doses of ALX-001 in 32 heathy adult participants aged 50-80. Study results show that ALX-001 was safe in cognitively normal older adults at all tested doses. ALX-001 achieved high exposure and successfully characterized a wide safety window at all doses, which ranged from 50mg to 150mg. Two doses were identified for future studies: 50mg and 100mg. All doses were well-tolerated and there were no serious adverse events. “The data show that ALX-001 achieved high target engagement without any adverse events related to mGluR5, which supports our vision to mediate synaptic dysfunction and loss while avoiding the on-target toxicity observed with other treatment modalities,” commented Stephen Strittmatter, M.D., Ph.D., scientific founder of Allyx Therapeutics and professor and chair of Neuroscience at the Yale University School of Medicine. Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in ongoing studies. Allyx Therapeutics has initiated a 28-day safety study of ALX-001 in Alzheimer’s disease patients (NCT05804383) and is initiating a study in Parkinson’s disease patients. “We are excited to begin the next phase of development with the initiation of two 28-day patient studies, the first studies of ALX-001 in people living with Alzheimer’s and Parkinson’s disease,” commented Tim Siegert, Ph.D., chief operating officer and co-founder of Allyx Therapeutics. “We believe that the results from the Phase 1b MAD study directly support larger scale Phase 2 clinical development to more fully understand the potential for ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases.” The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others. In addition to Allyx’s continued clinical development of ALX-001 in Alzheimer’s disease, the company has announced an expansion into Parkinson’s disease clinical research with the opening of an Investigational New Drug Application with the U.S. Food and Drug Administration (FDA). About ALX-001 ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Stephen Strittmatter at Yale. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale University. About Allyx Therapeutics Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. Contact Media: Eliza Schleifstein917.763.8106Eliza@schleifsteinpr.com Investors: Tim Siegert203-691-6543tsiegert@allyxthera.com

March 05, 2024 -- Allyx Therapeutics, a clinical-stage biotechnology company which is developing ALX-001, a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy for neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug Application (IND), enabling the company to proceed with clinical research in Parkinson’s disease. A 28-day safety study of ALX-001 in Parkinson’s disease patients will be initiated in Q1 2024. “We are working with urgency to understand how the unique mechanism of action of ALX-001 at mGluR5, which preserves and protects synapses, can introduce meaningful clinical benefits for people living with Alzheimer’s disease and Parkinson’s disease,” commented Allyx Therapeutics co-founder and CEO Stephen Bloch, M.D. “Our aim is to deliver the first-ever disease-modifying small molecule for these and other neurodegenerative diseases.” The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs and The Michael J. Fox Foundation for Parkinson’s Research, among others. As studies in Parkinson’s disease begin, the company will continue to advance clinical development of ALX-001 in Alzheimer’s disease, which is funded through grants from the National Institutes of Health and other Alzheimer’s disease research nonprofit organizations. “The generous grant from The Michael J. Fox Foundation for Parkinson’s Research contributes to research efforts around the potential of ALX-001, enabling our work to move into clinical research without delay,” commented Tim Siegert, Ph.D., chief operating officer and co-founder of Allyx Therapeutics. “We have the talent and the capital needed to rapidly advance our clinical development program and progress toward transforming the future for people living with serious and debilitating neurodegenerative diseases.” “The Michael J. Fox Foundation is excited to leverage Allyx’s work in developing this approach for Alzheimer’s disease to assess its potential for those living with Parkinson’s disease,” said Brian Fiske, Ph.D., Chief Scientific Officer, The Michael J. Fox Foundation. “We look forward to seeing results from these clinical trials and the additional data they will provide.” ALX-001 continues to demonstrate promise in ongoing studies that build on twelve years of research. A Phase 1b multiple ascending dose study (NCT05804383 Stage 1) in healthy volunteers was recently completed, following presentation of results from a single ascending dose study in October 2023 at the 16th Clinical Trials on Alzheimer’s Disease (CTAD) meeting. The Phase 1a open-label single ascending dose study (NCT04805983) evaluated the safety, tolerability, pharmacokinetics, and brain receptor occupancy of increasing doses of orally-administered ALX-001. All doses were well-tolerated and there were no serious adverse events. Results from the recently-completed Phase 1b multiple ascending dose study will be presented in March at the AD/PD™ 2024 Conference in Lisbon. ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Professor Stephen Strittmatter at Yale. Allyx Therapeutics obtained an exclusive worldwide license for use of ALX-001 from Bristol Myers Squibb and Yale University. Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. The content above comes from the network. if any infringement, please contact us to modify.

Geneva, Switzerland, January 16th, 2024 – Stalicla SA, a clinical-stage biopharmaceutical company advancing precision medicine pipelines for neuropsychiatric and neurological disorders, announces the successful completion of the first closing of its Series B financing round, securing $17.4 million. Led by SPRIM Global Investments Pte, Ltd, with key participation from core investors, this funding includes a $3.8 million credit facility. The proceeds will be utilized for: (i) preparation of Stalicla’s pioneering precision Autism Spectrum Disorder (ASD) Phase 2 trial – STP1; (ii) the establishment of its STP7 mGluR5 Negative Allosteric Modulator (mGluR5 NAM) platform, including the launch a Phase III study in Substance Use Disorders (SUDs) slated for 2025, fully supported by the NIH /National Institute on Drug Abuse; (iii) the ramp up of its ongoing biosampling/ patient identification trial STA-B-001 currently enrolling in sites in the USA, Spain and Australia. Alongside these key developments, Stalicla will further strengthen the package supporting its second precision ASD asset, STP2. Lynn Durham, CEO of Stalicla commented: “This financial backing underscores Stalicla's role as a trailblazer in the neuro precision space with first applications in precision psychiatry. Additionally, our STP7 mGluR5 NAM platform is offering multifaceted advanced clinical development opportunities in the wider neurology space in a timely period of recent clinical progress & increased transaction activity level in the neurology and neuropsychiatry space. There has never been a more promising time to advance transformative treatment options for millions of patients with brain diseases and Stalicla is determined to play a key role in this progress.” To support these advancements, Stalicla will strengthen its Management Team and Board of Directors, reinforcing the foundation for the exciting journey ahead. About STALICLA SASTALICLA is a precision molecular neuroscience clinical stage company, advancing through its DEPI precision neuro platform 2 precision autism assets: STP1 and STP2. Following the in-licensing of mavoglurant from Novartis in 2023, STALICLA has also established an advanced mGLuR5 NAM platform with multi-faceted late-stage clinical development opportunities.For further information, please visit: https://stalicla.com/ Contact:Stalicla SALynn Durham, Founder & CEOLynn.durham@stalicla.com Attachment Press Release 2024-01-SeriesB_final