PIKFYVE

Feb. 15, 2024 -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today that it has received Orphan Drug Designation (ODD) in the European Union (EU) for the treatment of amyotrophic lateral sclerosis (ALS). AIT-101 previously received ODD from the U.S. FDA in June 2023 for the treatment of ALS. “OrphAI Therapeutics now has orphan designation for AIT-101 both in the US and the EU for the treatment of ALS, an important step toward bringing the drug to patients suffering from this neurodegenerative disorder,” said Dr. Brigette Roberts, CEO and Director of OrphAI. “AIT-101 has demonstrated compelling activity in its ability to clear toxic aggregates, which are a hallmark of ALS and other neurodegenerative diseases. We look forward to moving AIT-101 into the next stage of clinical development." AIT-101 is a first-in-class, potent and highly selective inhibitor of the lipid kinase PIKfyve. Inhibition of PIKfyve leads to activation of the transcription factor TFEB which drives the increased clearance of toxic protein aggregates via the lysosomal autophagy pathway. AIT-101 has now been demonstrated to reduce aggregates in human subjects, to improve the survival of motor neurons in in vitro models of familial and sporadic ALS, and to ameliorate functional deficits in a mutant TDP-43 animal model of ALS. AIT-101 is the most clinically advanced PIKfyve inhibitor in development. OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2 for Group 1 and 3 pulmonary hypertension and bronchiolitis obliterans syndrome (BOS); and AIT-102, a proprietary analogue of mithramycin, in preclinical development, for the treatment of SWI/SNF mutated or dysregulated tumors. The content above comes from the network. if any infringement, please contact us to modify.

Seasoned leader in drug discovery and developmentGUILFORD, Conn., Jan. 24, 2024 (GLOBE NEWSWIRE) -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today the appointment of Jay S. Fine, Ph.D. to its Board of Directors. Dr. Fine has a strong track record of drug discovery and development leadership in respiratory diseases, oncology, immunology and fibrotic disease indications. His leadership has contributed to the progression of over 30 new molecular entities into clinical development, leading to the commercial launch of multiple products including Skyrizi™ for Plaque Psoriasis, Psoriatic Arthritis and Crohn’s Disease and Spevigo™ for Generalized Pustular Psoriasis. He has chaired various governance and steering committees at several global pharmaceutical companies. Dr. Fine currently serves as President of Research and Development at EvolveImmune Therapeutics, a development-stage company focused on advancing differentiated immunotherapeutic approaches for the treatment of cancer. Previously, he served as Senior Vice President and Global Therapeutic Area Head for Immunology and Respiratory Diseases Research at Boehringer Ingelheim and as the Discovery Research Site Head at their Ridgefield, CT location. Dr. Fine also served as Head of Translational Biology at Roche Pharmaceuticals, was a Director in the Inflammation department at Schering-Plough and worked in the Biologic Response Modifiers Program at the National Cancer Institute. He holds a B.S. degree from Cornell University and a Ph.D. from the University of Rochester School of Medicine. Dr. Fine has authored over 75 scientific publications and patents and has been the recipient of numerous honors and awards during his career. “Jay Fine represents an extraordinary resource both for the Board as well as for the OrphAI management team, given his years of experience and success in the operational leadership of drug discovery and development. I am personally excited to work with him in this capacity,” said Dr. Brigette Roberts, CEO and Director of OrphAI. “I am very pleased to join the OrphAI Therapeutics’ Board of Directors,” said Dr. Fine. “The company has established an exciting product pipeline which holds tremendous potential to meaningfully improve the lives of patients with rare diseases. I look forward to working closely with the OrphAI team and its board to accelerate the progress of its promising pipeline.” “We are excited to welcome Jay Fine to our Board of Directors,” said David Scheer, Chairman of the Board of Directors at OrphAI. “Jay’s extensive leadership experience and drug development insights will be especially valuable to OrphAI and our Board as we collectively embark on this next stage of the company’s growth.” About OrphAI Therapeutics OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2 for bronchiolitis obliterans syndrome (BOS) and pulmonary arterial hypertension (PAH); and AIT-102, a proprietary analogue of mithramycin, in preclinical development for the treatment of SWI/SNF mutated or dysregulated tumors. To learn more, please visit: OrphAI-Therapeutics.com

VRG50635, a small molecule PIKfyve inhibitor, is one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform Verge’s proof-of-concept study uses first-of-its-kind, cutting-edge design and technology to deliver more precise and comprehensive insights into how VRG50635 may benefit individuals with ALS “I applaud Verge for creating a bold proof-of-concept design that incorporates cutting-edge technology and scientific innovation in their efforts to create groundbreaking therapies for ALS.” - Dr. Angela Genge, Director, ALS Centre of Excellence for Research and Patient Care, Montreal Neurological Institute-Hospital SOUTH SAN FRANCISCO, Calif., Jan. 09, 2024 (GLOBE NEWSWIRE) -- Verge Genomics, a clinical-stage biotechnology company transforming drug discovery and development using artificial intelligence (AI) and human data, today announced the initiation of its Phase 1b proof-of-concept study of VRG50635 for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS). The study will assess the safety and tolerability of escalating doses of VRG50635, a potential best-in-class, small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered in diseased human tissues using CONVERGE®, the company’s all-in-human, AI-powered platform. VRG50635 is one of the first drugs to enter the clinic that was entirely discovered and developed from an AI-enabled platform. In traditional ALS clinical trials, a patient's progress is measured in a clinical setting using poorly responsive physician rating scales, which typically provide sparse data to establish proof of concept early in clinical development. In contrast, Verge's study harnesses the power of digital devices to collect thousands of objective, quantitative, relevant measurements directly from the patient, including mobility, breathing, and sleeping all from the comfort of their home. This level of patient data greatly increases the ability to detect disease progression and treatment-related changes, allowing to personalize the assessment of change for each study participant as he/she moves from no treatment to increasingly higher doses of VRG50635, and for longer treatment periods. “Incorporating cutting-edge technology into our ALS proof-of-concept study is a necessity born from the urgent unmet need to efficiently study promising therapeutics for people battling this devastating disease. Our approach and the technology we are incorporating into the VRG50635 proof-of-concept study will make it possible to collect dense amounts of unbiased, objective disease-relevant data to properly assess the safety, tolerability, and potential efficacy, including dose-response, early in clinical development,” said Diego Cadavid, M.D., FAAN, FANA, Chief Medical Officer at Verge Genomics. “Initiating our proof-of-concept study marks another important milestone for Verge and further demonstrates that by leveraging our AI-enabled platform in combination with patient data using a novel study design we are able to more efficiently discover and develop potential drugs for deadly diseases like ALS,” said Alice Zhang, CEO and co-founder of Verge Genomics. “We are tremendously excited by the potentially transformative power of our CONVERGE® platform in redefining the landscape and expediting the journey from lab bench to patients and improving lives worldwide." “I applaud Verge for creating a bold proof-of-concept design that incorporates cutting-edge technology and scientific innovation in their efforts to create groundbreaking therapies for ALS,” said Dr. Angela Genge, Director, ALS Centre of Excellence for Research and Patient Care, Montreal Neurological Institute-Hospital. About VRG50635 VRG50635 is a potent, orally bioavailable PIKfyve inhibitor that improves survival in amyotrophic lateral sclerosis (ALS) patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration. VRG50635 is the only PIKfyve inhibitor in clinical development that has been specifically optimized for the treatment of central nervous system disorders like ALS, with the potential to become a best-in-class therapy. About Amyotrophic Lateral Sclerosis (ALS) ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects motor neuron cells in the brain and spinal cord causing progressive paralysis of voluntary muscles. ALS often strikes people between the ages of 40 and 70, and it is estimated that 100,000 people worldwide may have the disease at any given time. ALS is fatal and there is currently no cure, and available therapies have only modest benefits for the majority of affected patients, who often succumb to the disease within 2 to 5 years after diagnosis. About Verge Genomics Verge is focused on developing therapeutics for complex diseases with high unmet need, using human genomics from patient disease tissues and machine learning. Verge has created a proprietary all-in-human CONVERGE® platform, featuring one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is led by experienced computational biologists and drug developers who are successfully advancing clinical and preclinical therapeutic programs in various diseases, including ALS and Parkinson’s disease. For additional information, please visit and follow us on LinkedIn and Twitter/X. Media Contact: Adam Silverstein Scient PR adam@scientpr.com