M6P Therapeutics

- Preclinical data provide proof of concept for co-expression of GBA gene with the truncated S1S3 PTase to produce highly phosphorylated GCase (β-glucocerebrosidase) - - Company’s M012 gene therapy had high M6P content and enhanced binding to CI-MPR as compared to the enzyme produced without S1S3 PTase - - Results suggest that M012 AAV can enable effective cross-correction in the CNS, and M012 AAV is a potential novel gene therapy for Gaucher disease, including neuronopathic phenotypes - ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced a poster presentation titled, “A novel AAV9 gene therapy for producing β-glucocerebrosidase enzyme with high mannose 6-phosphate content to treat Gaucher disease,” by Lin Liu, Ph.D., Director, R&D of M6P Therapeutics, at the 29th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre. “The data from this preclinical study illustrate the potential of our AAV-GBA-S1S3 gene therapy candidate for the treatment of Gaucher disease, including neuronopathic phenotypes,” said Pawel Krysiak, President and CEO of M6P Therapeutics. “M012 is part of our pipeline of candidates leveraging a first-in-class co-expression S1S3 platform technology for the treatment of lysosomal storage disorders. Using our technology platform, we enhance mannose 6-phosphate content on lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved enzyme uptake across target tissues.” Gaucher disease is a rare inherited metabolic disorder of defective lipid catabolism caused by deficient β-glucocerebrosidase (GCase) encoding by the GBA1 gene resulting in accumulation of glycosphingolipids in the periphery and central nervous system (CNS). Multiple recombinant human GCase enzyme replacement therapies have been approved to treat Gaucher disease type 1, but there are no effective treatments to address the neuronopathic manifestations for Gaucher disease types 2 and 3. Gene therapy could be a potential therapeutic approach to treat all three types of Gaucher disease. "In this study, gene copy and transcriptome analysis showed that the dosed novel M012 AAV vector enabled transduction of peripheral tissues and various regions of brain in wild-type mice post-intravenous injection,” said Dr. Liu. “We observed increased GCase enzyme activity with enhanced CI-MPR affinity in peripheral tissues of treated animals and noted strong GCase protein signal with broad distribution detected in M012 AAV vector-treated brain samples. These results strongly suggest that M012 AAV enables effective GCase cross-correction in the CNS. We look forward to sharing additional updates and data as the program progresses.” Presentation highlights from the poster include: Data from previous publications show that co-expression of truncated GlcNAc-1-phosphotransferase (designated as S1S3 PTase) greatly increases the levels of mannose 6-phosphate (M6P) on lysosomal enzymes, which enables efficient cellular uptake via the cation-independent mannose 6-phosphate receptor (CI-MPR) that is the broadly expressed on most cells including neurons and other cell types in the CNS The study demonstrated M6P Therapeutics’ ability to regulate and enhance M6P content on lysosomal enzymes for both enzyme replacement and gene therapies, which improves enzyme uptake across target tissues, restoring the enzymatic activity within this natural pathway The M012’s novel AAV gene therapy approach uses a dual promoter construct design for co-expression of GBA gene with the truncated S1S3 PTase to produce highly phosphorylated GCase as a potential treatment for Gaucher disease The produced hGCase with S1S3 PTase co-expression was shown to have high M6P content and enhanced binding to CI-MPR as compared to the enzyme produced without S1S3 PTase GCase uptake and distribution in the CNS was also evaluated by immunohistochemical staining About M6P Therapeutics M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted enzyme replacement and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics’ proprietary co-expression S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics’ team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of enzyme replacement and gene therapies by harnessing the power of protein phosphorylation using its co-expression S1S3 platform. M6P Therapeutics’ mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: .

NEWPORT BEACH, Calif., June 25, 2021 (GLOBE NEWSWIRE) -- via InvestorWire -- Roth Capital Partners (“Roth”), , is set to virtually present the Roth Virtual Healthcare Private Company Forum on June 28th, 2021. This event will consist of Virtual Panels on Emerging Therapeutics and select Fireside Chats with Roth Research Analysts. This format will provide an opportunity for C-level executives representing established private healthcare companies in the Biotechnology, Pharmaceuticals and Medical Technologies verticals to tell their stories. “We are excited to host our Virtual Healthcare Private Company Forum for private companies. As the event continues to grow, we are bringing together a broader range of private healthcare companies, from across the US and beyond, to tell their stories,” said James Antonopoulos, Managing Director & Head of Healthcare Investment Banking at Roth. “This event is another example of Roth’s continued leadership in supporting the growth of healthcare companies, including biotechnology, specialty pharmaceuticals, medical devices & diagnostics.” Since 2010, Roth has been involved in over 530 transactions for its Healthcare clients with a total transaction value of over $24 billion. (Source: Roth Capital Partners | 06.24.21) For more information and event registration, please see , or contact your Roth event representative at (949) 720-5700 or registration@roth.com. Event Agenda (All times in ET): MONDAY | JUNE 28, 2021 | Full details: 8:00 am       Cullgen Inc. – Fireside Chat  8:20 am       M6P Therapeutics – Fireside Chat  8:40 am       Ryvu Therapeutics – Fireside Chat  9:00 am       Novel Approaches to Tumor Eradication – Panel with Enterome & Invectys10:00 am       Renaissance Bioscience – Fireside Chat10:20 am       Aruna Bio – Fireside Chat10:40 am       Dyve Biosciences – Fireside Chat11:00 am       Next Wave of Psychedelic Drug Developers – Panel with Diamond Therapeutics, Field Trip Health (FTRP.TO), Mydecine (OTC: MYCOF) & Small Pharma (DMT.V)12:00 pm       Natural Killer Cell Approaches to Eradicate Tumors – Panel with Alloplex Biotherapeutics, Alphageneron, Cytovia & NKGen Biotech  1:00 pm       Active Motif - Fireside Chat  1:20 pm       Intensity Therapeutics – Fireside Chat  1:40 pm       Fibralign – Fireside Chat  2:00 pm       Reva Medical – Fireside Chat Participating Company List: Subject to Change | As of 06/22/2021 Event Media Sponsor: About Roth Capital Partners, LLC: Roth Capital Partners, LLC (“Roth”) is a relationship-driven investment bank focused on serving emerging growth companies and their investors. As a full-service investment bank, Roth provides capital raising, M&A advisory, analytical research, trading, market-making services, and corporate access. Headquartered in Newport Beach, CA, Roth is privately-held, employee owned, and maintains offices throughout the U.S. For more information on Roth, please visit . Investor Contact:Roth Capital PartnersIsabel Mattson-PainDirector of Marketing & Corporate AccessImattson-pain@roth.com 949.720.7117 Media Contact:InvestorBrandNetwork (IBN)Los Angeles, California OfficeEditor@InvestorBrandNetwork.com

NEW YORK--(BUSINESS WIRE)-- The International Gaucher Alliance (IGA) and Kantar Health announce today the launch of the disease registry GARDIAN: Gaucher Registry for Development Innovation and Analysis of Neuronopathic Disease. This launch follows the announcement of the IGA and Kantar Health collaboration in 2020. Gaucher Disease (GD) is an autosomal recessive disease and the most common Lysosomal Storage Disorder, with an incidence of about 1 in 100,000 live births. Three types exist: type I, the most common, and types II and III, also called neuronopathic GD (nGD). Type II (GD2) is characterized by rapidly progressive neurological symptoms in babies; whereas type III (GD3) appears later in childhood and is defined by slower progression of neurological symptoms. Today, GD has no cure; treatment options are available to address some aspects, but none are specific to the neurological impairments of nGD. GARDIAN is a global registry focused on patients with nGD. Through GARDIAN, the IGA aims to better understand the natural history of the disease and its impact on both patients and caregivers. As part of GARDIAN, disease specific patient-reported and observer-reported outcome scales will be developed. Registry results will provide a comprehensive understanding of nGD which is essential to accelerate the early diagnosis and care for patients and ultimately improving patient outcomes. Real world evidence generated via GARDIAN will support clinical trial development and inform regulatory decision making. “We’re excited to launch GARDIAN and wish to show our gratitude of our donors that are generously supporting this endeavor,” said Tanya Collin-Histed, CEO, International Gaucher Alliance. “The combined support of M6P Therapeutics; Pfizer; Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company; and Sanofi Genzyme is a testament to the importance of GARDIAN and the difference it can make to our patient community.” “We are delighted to partner with the IGA and our donors in an effort to help better understand neuronopathic Gaucher Disease and bring new therapies quicker to patients,” said Michael Fronstin, Global Head of Innovation, Real World Evidence, Kantar Health. “We welcome other organizations to contact us should they be interested in learning more or getting involved.” Cerner Corporation (NASDAQ: CERN), a global healthcare technology company, acquired Kantar Health on April 1. ABOUT THE INTERNATIONAL GAUCHER ALLIANCE (IGA) The IGA is an international umbrella group representing the interests of Gaucher patients and those of not-for-profit Gaucher patient groups as well as rare disease groups throughout the world. While the IGA has an office in England, the IGA has almost 60 member associations around the globe. The GARDIAN registry will be under the control of International Gardian Limited (IGL), a wholly owned subsidiary of the IGA. ABOUT KANTAR HEALTH Kantar Health provides data, analytics and research to the life sciences industry. As real-world evidence and commercialization partners, Kantar Health enables clients to bring safe and effective treatment to patients around the world. The team has expertise across a myriad of therapy areas, including unique thought leadership in oncology and rare diseases. For more information on how to optimize commercial launch plans, demonstrate real-world effectiveness and safety, and build ongoing value substantiation to unlock your full potential, visit .