Supranuclear Palsy, Progressive

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Switch Therapeutics, a biotechnology company developing novel three-stranded RNA that function as conditionally activated siRNA (CASi) therapeutics for central nervous system (CNS) diseases, announced today the appointment of Dr. David M. Holtzman to its Scientific Advisory Board and disclosed pipeline programs targeting APOE (apolipoprotein E) and MAPT (microtubule-associated protein tau) for treatment of neurodegenerative diseases. Dr. Holtzman is a world-renowned physician-scientist and brings more than three decades of experience researching and treating neurological diseases with a specific expertise in Alzheimer’s disease (AD). He is currently scientific director of the Hope Center for Neurological Disorders, director of the Knight Alzheimer’s Disease Research Center and professor of neurology at Washington University in St. Louis. Dr. Holtzman has been elected to the National Academy of Medicine and the National Academy of Inventors and is a Fellow of the American Association for the Advancement of Science. His notable research and accomplishments include showing, in part, how APOE4 contributes to AD, developing a method to measure protein synthesis and clearance in the CNS and developing cerebrospinal fluid and plasma biomarkers for AD. “ Dr. Holtzman brings invaluable experience to our team both as a treating physician and with his extensive neurodegeneration and APOE-specific research background that will support the advancement of CASi-APOE and future neurodegeneration programs. The nomination of CASi-APOE as our lead development candidate is a big step for Alzheimer’s patients. APOE4 carriers have limited treatment options, though 60% of Alzheimer’s disease patients are APOE4 carriers compared to 25% of the general population,” said Dee Datta, Ph.D., co-founder and CEO of Switch Therapeutics. “ This is an exciting time for Switch as we prepare to advance our pipeline towards the clinic.” Switch’s CASi-APOE program is designed to knock down APOE in the CNS without affecting APOE in the liver, where it plays a vital role in systemic lipid homeostasis. “ I am delighted to be joining Switch’s Scientific Advisory Board during a key phase of the company’s growth in the neurodegenerative space,” said Holtzman . I am especially enthusiastic about the unique profile of CASi-APOE which blocks APOE production in the brain, while sparing the liver. APOE4 carriers need treatment options, and CASi-APOE is particularly exciting as genetic knockdown of APOE has been shown in animal models of Alzheimer’s to reduce both amyloid-beta and tau levels, two key hallmarks of Alzheimer’s disease.” Switch has initiated IND-enabling studies and plans to conduct a Phase 1 trial in patients with AD who are APOE4 carriers in 2026. By the end of 2024, Switch anticipates naming its second development candidate, targeting production of tau, which is directly responsible for neurodegenerative diseases called tauopathies, including primary and secondary tauopathies such as progressive supranuclear palsy and Alzheimer’s disease, among others. Switch’s MAPT program knocks down tau at the genetic level, thus preventing intra-cellular tau production. Switch also is advancing two earlier CASi programs with cell-specific activity, one limited to microglia and the second program directed at astrocytes. Targeting these cells individually will enable cell-specific genetic knockdown of disease targets for which broad suppression is unsafe. About Switch Therapeutics Switch Therapeutics is an emerging biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science, with the goal of treating a range of diseases – affecting the central nervous system – with significant unmet need. The CASi (Conditionally Activated siRNA) Platform, the company’s novel gene knockdown approach, is based on technology developed by renowned researchers in the field of RNA and drug development from Caltech, Harvard Medical School and City of Hope. CASi combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for efficient self-delivery and uptake, potency and durable gene knockdown, as well as cell selectivity. Switch is based in South San Francisco, California. For more information, connect with Switch on LinkedIn or visit www.switchthera.com .

Portfolio of nucleoside chemotherapeutics for the targeted treatment of pancreatic cancer, certain other solid tumors and hematologic cancers Builds upon leadership position in developing best-in-class, late-stage nucleoside reverse transcriptase inhibitors for neurodegenerative and autoimmune diseases SAN DIEGO, Nov. 21, 2024 /PRNewswire/ -- Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced the acquisition of a portfolio of novel nucleoside analogs from PrimeFour Therapeutics, Inc., a biotechnology company focused on cancer chemotherapeutic agents. The portfolio of novel nucleosides consists of a chemically diverse library of unique analogs. The most advanced lead compounds are nucleosides that cause "synthetic lethality" in cancers characterized by distinct tumor-defined genetic signatures, allowing them to be precisely targeted to the most susceptible cancers with reduced toxicity. These compounds can be targeted towards cancers with newly discovered genetic biomarkers that are present in pancreatic and certain other solid tumors as well as in hematologic cancers, allowing for personalized therapy in those patients most likely to respond to treatment. "This transaction allows us to build upon our leadership in developing first-in-class and best-in-class nucleoside reverse transcriptase inhibitors for neurodegenerative and autoimmune diseases, including ALS, PSP and Alzheimer's disease. We are excited to expand our pipeline to include this portfolio of potent nucleoside chemotherapeutics with significant therapeutic potential against a variety of cancers," said Eckard Weber, M.D., founder and Chief Innovation Officer of Transposon. "We look forward to advancing the lead compounds into preclinical development to target DNA-damage repair deficient cancers, such as pancreatic and colorectal cancers." About Transposon Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel nucleoside reverse transcriptase inhibitors for the treatment of neurodegenerative and aging-related diseases, including Alzheimer's disease. The company's lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. Transposon is advancing TPN-101 into Phase 3 registration studies for the treatment amyotrophic lateral sclerosis (ALS) and progressive supranuclear palsy (PSP), and a Phase 2 proof-of-concept study in Alzheimer's disease. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications. About PrimeFour PrimeFour discovered and developed a class of novel nucleoside analogs that are selectively incorporated into cancer cells with biomarker-identifiable DNA damage repair deficiencies and that, once incorporated into some cancer cells, cause irreversible DNA damage and cancer cell death. Contact: Rick Orr Transposon Therapeutics, Inc. (858) 535-4821 [email protected] SOURCE Transposon Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The World Health Organisation estimated that over 8.5 million people had PD in 2019. Credit: LE Photo / Shutterstock. Pipeline treatments across the seven major markets (7MM: France, Germany, Italy, Spain, the UK, the US and Japan) reflect a growing research trend prioritising disease-modifying therapies (DMTs) and non-motor symptom therapies in the treatment of Parkinson’s disease, according to new research. The current therapeutic landscape for Parkinson’s is heavily genericised, exhibiting a notable lack of available neuroprotective treatments. However, Lorraine Palmer, pharmaceuticals analyst at GlobalData, says this is changing. “There are a total of 93 products in Phase I-III development within the 7MM for the treatment of Parkinson’s,” Palmer said. “66% of these are prospective neuroprotective agents or DMTs. These investigational agents target the key mechanisms that have been implicated in the pathophysiology of Parkinson’s, such as alpha-synuclein aggregation and neuroinflammation, with the goal of slowing disease progression.” GlobalData is Pharmaceutical Technology ’s parent company. DMTs look to be a particularly significant piece of the Parkinson’s puzzle, and key opinion leaders interviewed by GlobalData agreed on the need for further research focus, despite complications presented by the differing pathogenesis of Parkinson’s across patients. One of the neuroprotective and DMT agents in late-stage development is Annovis Bio’s Posiphen (buntanetap tartrate) . The drug is aimed at inhibiting alpha-synuclein, and it is currently in Phase III development in the US and five major European markets. Elsewhere, a Phase III trial in the US has been planned for BioVie’s Triolex (bezisterim) , which is an inhibitor of inflammatory mediators and is thought to offer neuroprotective potential. Palmer added: “26% of the pipeline DMTs in the pipeline target alpha-synuclein aggregation. This is a divisive mechanism of action, with some key opinion leaders holding high hopes and others expressing scepticism, highlighting the failure of Prothena and Roche’s prasinezumab to meet its primary endpoint in the Phase II PASADENA trial (NCT03100149) and safety concerns due to the role of alpha-synuclein in healthy functioning.” Alongside DMTs, treating non-motor symptoms is also becoming a key research focus, highlighted by analysis which shows that 3% of the pipeline is specifically targeting Parkinson’s disease dementia (experienced by up to 80% of people with Parkinson’s ) and Parkinson’s disease psychosis (experienced to some degree by between 20% and 40% of people with Parkinson’s ). Research development appears to be addressing a broader spectrum of Parkinson’s challenges, and there is interest in antiparkinsonian agents, dopamine agonists and treatments for Parkinson’s disease related dementia. It suggests that the industry is looking to improve both motor and non-motor symptoms, addressing the broader spectrum of challenges faced by Parkinson’s patients. Palmer concluded: “The distribution of the 93 pipeline agents in the 7MM underscores a strong emphasis on neuroprotective and disease-modifying treatments, demonstrating an industry-wide focus toward therapies that could potentially slow the progression of Parkinson’s.”