Sharp Therapeutics to Present Pipeline and Program Updates at World Orphan Drug Congress 2025
Sharp Therapeutics (OTCQB: SHRXF) will present preclinical data for its lead, Phase 1–ready small‑molecule ‘901 program targeting Gaucher disease and GBA Parkinson's disease at the World Orphan Drug Congress 2025 in Amsterdam on Oct 27–29, 2025. The company will present on Oct 27 at 2:45 p.m. CET and will review its clinical targets and pipeline, including programs for Niemann‑Pick disease type C and familial frontotemporal dementia. Sharp describes ‘901 as an oral candidate designed to restore lysosomal enzyme function with potential to treat peripheral and CNS manifestations and notes plans to initiate clinical studies in the near term.
Sharp Therapeutics (OTCQB: SHRXF) presenterà dati preclinici sul suo lead, piccolo-molecola pronto per la fase 1 ‘901 programma mirato alla malattia di Gaucher e alla malattia di Parkinson con GBA al World Orphan Drug Congress 2025 ad Amsterdam dal 27 al 29 ottobre 2025. L'azienda presenterà il 27 ottobre alle 14:45 CET e fornirà una panoramica sui propri obiettivi clinici e sul pipeline, inclusi i programmi per la malattia di Niemann‑Pick di tipo C e la demenza frontotemporale familiare. Sharp descrive ‘901 come un candidato orale progettato per ripristinare la funzione enzimatica lisosomiale con potenziale di trattare manifestazioni periferiche e CNS e segnala piani per avviare studi clinici a breve.
Sharp Therapeutics (OTCQB: SHRXF) presentará datos preclínicos de su programa principal, una molécula pequeña lista para la fase 1 ‘901 que apunta a la enfermedad de Gaucher y a la enfermedad de Parkinson con GBA en el World Orphan Drug Congress 2025 en Ámsterdam del 27 al 29 de octubre de 2025. La empresa presentará el 27 de octubre a las 2:45 p.m. CET y repasará sus objetivos clínicos y su pipeline, incluidos los programas para la enfermedad de Niemann-Pick tipo C y la demencia frontotemporal familiar. Sharp describe ‘901 como un candidato oral diseñado para restaurar la función de la enzima lisosomal con potencial para tratar manifestaciones periféricas y del SNC y señala planes para iniciar estudios clínicos en breve.
Sharp Therapeutics (OTCQB: SHRXF)는 세계 풍부 약물 회의 2025(World Orphan Drug Congress 2025)에서 자사의 리드인 1상 진입 준비 소분자 ‘901 프로그램이 고셔병과 GBA 파킨슨병을 표적으로 하는 데이터를 발표할 예정이며 암스테르담에서 2025년 10월 27–29일에 개최됩니다. 회사는 2025년 10월 27일 오후 2:45 CET에 발표하고 임상 목표와 파이프라인을 검토하며 Niemann‑Pick 질환 유형 C 및 가족성 전두측두엽 치매에 대한 프로그램을 포함합니다. Sharp는 ‘901을 리소좀 효소 기능 회복을 목표로 한 경구 후보로 설명하며 말초 및 중추 신경계 증상을 치료할 가능성이 있고, 가까운 시일 내 임상 연구를 시작할 계획이라고 밝힙니다.
Sharp Therapeutics (OTCQB: SHRXF) présentera des données précliniques sur son programme phare, une petite molécule en phase 1 prête ‘901 visant la maladie de Gaucher et la maladie de Parkinson avec GBA lors du World Orphan Drug Congress 2025 à Amsterdam du 27 au 29 octobre 2025. L'entreprise présentera le 27 octobre à 14h45 CET et fera le point sur ses objectifs cliniques et son pipeline, y compris les programmes pour la maladie Niemann-Pick de type C et la démence frontotemporale familiale. Sharp décrit ‘901 comme un candidat oral conçu pour restaurer la fonction enzymatique lysosomiale avec un potentiel de traitement des manifestations périphériques et du SNC et indique des plans pour lancer des études cliniques prochainement.
Sharp Therapeutics (OTCQB: SHRXF) wird präklinische Daten zu seinem führenden, Phase-1-fertigen Kleinstmolekül ‘901-Programm vorstellen, das auf Morbus Gaucher und GBA-Parkinson-Krankheit abzielt, beim World Orphan Drug Congress 2025 in Amsterdam vom 27. bis 29. Oktober 2025. Das Unternehmen wird am 27. Oktober um 14:45 Uhr MEZ präsentieren und seine klinischen Ziele und die Pipeline überprüfen, einschließlich Programme für Niemann-Pick-Krankheit Typ C und familiale frontotemporale Demenz. Sharp beschreibt ‘901 als einen oralen Kandidaten, der darauf ausgelegt ist, die lysosomale Enzymfunktion wiederherzustellen, mit Potenzial zur Behandlung peripherer und zentrales Nervensystem Manifestationen, und gibt an, in naher Zukunft klinische Studien zu beginnen.
Sharp Therapeutics (OTCQB: SHRXF) ستعرض بيانات ما قبل السريرية لبرنامجها الرائد، مركب جزيئي صغير جاهز للمرحلة 1 ‘901 الذي يستهدف مرض غيشير ومرض باركنسون المرتبط بـ GBA في World Orphan Drug Congress 2025 في أمستردام من 27 إلى 29 أكتوبر 2025. ستقدم الشركة في 27 أكتوبر الساعة 2:45 مساءً بتوقيت وسط أوروبا وستراجع أهدافها السريرية وخط أنابيبها، بما في ذلك برامج لمرض Niemann-Pick من النوع C والخَرَف الجبهي الصدغي العائلي. تصف Sharp ‘901 كمرشح فموي مصمم لاستعادة وظيفة الإنزيم في الليزوزوم ويمتلك إمكانية علاج المظاهر الطرفية والجهاز العصبي المركزي، وتشير إلى خطط لبدء الدراسات السريرية في القريب العاجل.
Sharp Therapeutics (OTCQB: SHRXF) 将在2025年阿姆斯特丹举行的世界罕见病药物大会(World Orphan Drug Congress 2025)上发布其领先药物、准备进入1/阶段的小分子程序‘901，该程序针对戈谢病和GBA帕金森病。在2025年10月27–29日举行。公司将在2025年10月27日欧洲中部时间下午2:45发表演讲，并将回顾其临床目标和管线，包括用于尼曼-皮克病C型和家族性额颞叶痴呆的计划。Sharp将< b>‘901描述为一个口服候选药物，旨在恢复溶酶体酶功能，并有潜力治疗周围和中枢神经系统表现，并指出在不久的将来启动临床研究的计划。
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PITTSBURGH, Pa. and TORONTO, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Sharp Therapeutics Corp. ("Sharp" or the "Company") (TSX-V: SHRX) (OTCQB: SHRXF), a biotechnology company developing small-molecule therapeutics for genetic diseases, today announced that it will present preclinical data from its lead Phase 1 ready ‘901 program being developed to treat Gaucher disease and Glucocerebrosidase (GBA) Parkinson's disease at the World Orphan Drug Congress 2025. The meeting will take place from October 27 to 29, 2025, in Amsterdam, the Netherlands, and the Company will present at 2:45 p.m. (CET).
At the Congress, Sharp will highlight the status of its clinical targets and program pipeline, centered around Gaucher disease, Niemann-Pick disease type C, and familial frontotemporal dementia. The Company’s most advanced program, ‘901, is a differentiated small-molecule candidate designed to address a lysosomal storage disorder caused by a deficiency in the GBA product enzyme. Mutations in GBA are known to result in the development of both Gaucher disease and GBA Parkinson's disease.
“We are pleased to share Sharp’s progress with the rare-disease community at the World Orphan Drug Congress,” said Scott Sneddon, Ph.D., J.D., Chief Executive Officer of Sharp Therapeutics. “Our mission is to develop pill-based medicines that restore function in defective proteins and meaningfully improve the lives of patients with genetic diseases. With ‘901 for Gaucher disease and a growing pipeline of programs in other high-need genetically defined indications, we aim to deliver transformative therapies for patients. Based on our promising preclinical data, we believe ‘901 has the potential to treat both the peripheral and central nervous system manifestations of Gaucher disease, while offering a favorable safety profile and the convenience of an oral medicine. We look forward to initiating clinical studies in the near term that are designed to efficiently establish proof of biology and lay the foundation for future registrational trials.”
About Sharp Therapeutics Corp.
First-Choice Therapies for Genetic Diseases
Sharp Therapeutics is a preclinical-stage company developing first-choice small-molecule therapeutics for genetic diseases. The Company’s discovery platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines.
Caution Regarding Forward-Looking Information
Certain statements contained in this press release constitute "forward-looking information" as such term is defined in applicable Canadian securities legislation. The words "may", "would", "could", "should", "potential", "will", "seek", "intend", "plan", "anticipate", "believe", "estimate", "expect" and similar expressions are intended to identify forward-looking information. All statements other than statements of historical fact may be forward-looking information. Such statements reflect Sharp's current views and intentions with respect to future events, and current information available to Sharp, and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements that may be expressed or implied by such forward-looking information to vary from those described herein should one or more of these risks or uncertainties materialize. Should any factor affect Sharp in an unexpected manner, or should assumptions underlying the forward-looking information prove incorrect, the actual results or events may differ materially from the results or events predicted. Any such forward-looking information is expressly qualified in its entirety by this cautionary statement. Moreover, Sharp does not assume responsibility for the accuracy or completeness of such forward-looking information. The forward-looking information included in this press release is made as of the date of this press release and Sharp undertakes no obligation to publicly update or revise any forward-looking information, other than as required by applicable law.
Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.
For additional information on Sharp, please visit: www.sharptx.com.
Sharp Therapeutics Corp.
Scott Sneddon, PhD, JD
CEO/CSO
Email: scott@sharptx.com
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