Niemann-Pick Disease, Type A

- AL00804, a highly brain penetrant GCS inhibitor, efficiently reduced GL1 accumulation in the brain of preclinical models - - In a head-to-head comparison in a neuronopathic model for Gaucher disease, AL00804 exhibited higher potency and greater brain penetration compared to other GCS inhibitors currently in development - - AL00804 is IND-ready and aims to be the best-in-class treatment of neuronopathic Gaucher disease, GM2 and GM1 gangliosidosis - NEWARK, Calif.--(BUSINESS WIRE)-- AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today presented data on the development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid (GSL) storage diseases. AL00804 is a novel, oral, highly brain penetrant glucosylceramide synthase inhibitor (GCSi). The presented work was done in collaboration with Dr. Ying Sun’s team at Cincinnati Children's Hospital Medical Center and will be a poster presentation during the 2023 WORLD Symposium, being held in Orlando, Florida, from February 22- 26, 2023. “We are pleased with the efficacy of AL00804 in reducing symptoms of CNS disease in several preclinical models,” said Jerry Shen, PhD, Chief Executive Officer and Founder of AceLink. “AL00804 was selected based on its unique properties, including high potency and ability to cross the blood-brain barrier, to more effectively treat patients with neuronopathic diseases. We have completed IND-enabling work in support of AL00804 and are excited for its further development in clinical settings.” Data Highlights: Pharmacokinetics studies support once daily, oral administration of AL00804. AL00804 is highly brain penetrant that efficiently reduced brain GL1 and lyso-GL1 accumulation in preclinical disease models. AL00804 reduced CNS disease symptoms, significantly delayed motor function decline and extended survival. When tested head-to-head in a neuronopathic model of Gaucher disease, AL000804 performed better than Venglustat. The increased potency and superior brain penetration compared to Venglustat make AL00804 a potentially safer and more efficacious molecule for treating neuronopathic GSL storage disorders. Details on the AceLink WORLD Symposium Presentation Abstract Title: Development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid storage diseases Authors: Michael Babcock, Ying Feng, Benjamin Liou, Venette Fannin, Yi Lin, Jerry Shen, Ying Sun Session Title: Basic Science – Poster Session III Session Date & Time: Thursday February 23 from 3:00 - 4:00 PM ET Abstract Number: LB-07 Location: Hilton Orlando, Kiosk 26-B About AL00804 AL00804 is a proprietary, oral, highly brain penetrant GCS inhibitor being developed for the treatment of neuronopathic glycosphingolipid storage diseases including Gaucher disease, GM2 gangliosidosis (Tay-Sach and Sandhoff disease), GM1 gangliosidosis, and possibly certain genetic forms of Parkinson’s disease. About GCS inhibitor GCS (glucosylceramide synthase) catalyzes the first step in the synthesis of glycosphingolipids, a group of bioactive molecules that play important roles in various cellular processes and diseases. GCS inhibitors reduce the production of glycosphingolipids, thereby exerting beneficial effects to diseases such as Fabry disease and Gaucher disease, which are caused by the accumulation of these lipids. About AceLink Therapeutics, Inc. Founded in 2018, AceLink Therapeutics is an innovative biopharma startup focusing on developing safe and effective medicines to address genetic diseases with high unmet needs. The company’s initial focus is to develop novel therapeutics for Fabry disease. For more information, please visit .

SAN FRANCISCO & SCOTTSDALE, Ariz.--(BUSINESS WIRE)-- PicnicHealth, the venture-backed startup giving patients unprecedented access to their medical records and the ability to contribute to scientific research, and Wylder Nation Foundation, an organization committed to advancing research for Acid Sphingomyelinase Deficiency (ASMD), are announcing a partnership to generate a real-world ASMD dataset to contribute to medical research. Together, PicnicHealth and Wylder Nation Foundation are forming “ASMD Accelerate,” to understand patient journeys documented in medical record data and translate them into an actionable, de-identified dataset to advance ASMD research. ASMD Accelerate is initially focused on chronic neurovisceral forms of ASMD, commonly referred to as Niemann-Pick Disease Type A (NPA) and Type A/B (NPA/B). ASMD Accelerate centralizes medical records for those living with ASMD or those who have passed away from ASMD. Data from these records will be de-identified and analyzed by Wylder Nation and other researchers to better understand how ASMD progresses over time and how it is managed. Ultimately, the goal is that this data will lead to better care, a better understanding of ASMD, and new treatments. PicnicHealth has helped tens of thousands of patients with chronic or complex illnesses navigate the healthcare system via their patient product. Through the PicnicHealth Research Platform, the company is working with top life sciences, academic institutions, and advocacy organizations to structure anonymized and aggregated medical records, giving researchers access to longitudinal real-world datasets. “Unlocking patient control of medical data is especially important for rare diseases where the data can be incredibly powerful for both clinical care and powering research when too often little is known about the disease,” said Noga Leviner, CEO PicnicHealth. “We couldn't be more excited to partner with Wylder Nation Foundation, patients, and their families.” “Adequately caring for a child with ASMD typically requires the ongoing involvement of multiple physicians associated with different hospitals and academic institutions, creating challenges for families to access and organize their complete medical history,” said Steven Laffoon, Co-founder and President of Wylder Nation Foundation. “This partnership with PicnicHealth will not only help families overcome this barrier but will also lay the groundwork for real-world evidence-based data to help us better understand the true burden of this complex disease, and ultimately help accelerate future treatment options.” Wylder Nation Partnership Leverages Data to Benefit Patients, Their Families, and ASMD Researchers This partnership centralizes medical records for patients and their families. Additionally, it allows Wylder Nation and ASMD researchers to access valuable, anonymized information from medical records that was previously inaccessible. PicnicHealth uses a patient-centered approach and believes keeping patients and their families at the center of research is critical to ensuring patients control their data. In the future, ASMD researchers will be able to apply to access the anonymized dataset for various research projects. Advancing Medical Research with Machine Learning PicnicHealth uses advanced human-in-the-loop machine learning (HITL ML) to digitize and structure complete medical records, including unique details like notes, imaging, and lab records directly from patient providers. PicnicHealth can get records from any facility in the US, in any format, regardless of hospital system or what electronic medical record system they use. Their proprietary algorithm “reads” the records and a nurse validates each abstraction. This approach allows the company to process records at scale while ensuring regulatory-grade accuracy and improving the algorithm with each record. About PicnicHealth PicnicHealth is a healthcare technology company that partners with patients, porting their complete medical records into an easy-to-use online application. The platform gives patients unprecedented access to and control over their medical records and, with their consent, the opportunity to contribute this valuable data to further scientific research. Founded in 2014 by Noga Leviner and Troy Astorino, the company partners with several of the world’s largest biopharma companies and academic research institutions. Learn more at PicnicHealth.com. About Wylder Nation Foundation Wylder Nation Foundation is committed to improving the lives of children and families living with Acid Sphingomyelinase Deficiency (ASMD). Founded in 2013, the organization is focused on fostering collaboration among academic researchers, industry, and other stakeholders to accelerate the discovery and development of treatment options for ASMD and other similar Lysosomal Storage Diseases.