Secretory Component Deficiency

The Age for Porosome Therapeutics is Here BOSTON--(BUSINESS WIRE)-- Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF). The preprint highlights the ground-breaking therapeutic approach that overcomes CF nearly four times more effectively than currently available medications to treat the inherited disorder. “We’re excited about the potential the porosome platform holds for developing new and novel drugs and therapies to address a wide range of secretory disorders,” said Professor Bhanu P. Jena, PhD, Founder and Chairman, Porosome Therapeutics. “Echoing the International Patent Search Agency, our central claims are ‘novel’ and ‘inventive,’” said Guillermo Marmol, President and CEO, Porosome Therapeutics. “We look forward to bringing such new, novel, safe, and curative treatments to market for people living with cystic fibrosis, diabetes, and other secretory disorders.” On December 9, 2023, the preprint entitled “Reprogramming the Cells Secretory Machinery: A Cystic Fibrosis Rescue” was published on bioRxiv. The study identified an innovative therapeutic solution for CF patients by first determining the relationship between the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene and porosome-mediated secretion in human airways. Results demonstrate that mutation in porosome-associated CFTR also affects other proteins within the porosome secretory machine, thereby reducing mucus secretion. Researchers then reconstituted functional porosomes into the plasma membrane of CFTR mutant cells to demonstrate that porosome reconstitution normalizes mucus secretion approximately four times more effectively than currently available CF drugs. “Our company research has aided in the ability to identify the critical role CFTR plays within the porosome secretory machinery. By harnessing this technology, we can advance innovative treatments for currently incurable diseases,” said Marmol. “Our patient-centered approach in drug design and development at Porosome Therapeutics will transform therapeutic options for patients diagnosed with cystic fibrosis, diabetes, cancer, Alzheimer’s, and other neurological, digestive, and immune disorders.” CF is a genetic condition that adversely affects cells in the body that secrete mucus, such as the airways, digestive system, and reproductive organs. Normally, these fluids are slick and thin. However, mutations in the CFTR gene in people with CF make the secretions thick and sticky. The secretions clog tubes, ducts, and passages rather than lubricating them, preventing normal function. While more than 2,000 mutations in the CFTR gene have been identified, the ΔF508 CFTR is the most common, accounting for approximately 70% of all CFTR mutations. It is estimated that more than 160,000 people worldwide are living with CF, with a consequent reduction in life expectancy. With this study, Porosome Therapeutics has demonstrated positive pre-clinical results of porosome-reconstitution therapy in human bronchial air-liquid interface 3D cultures that mimic normal lung physiology, responding to CFTR inhibitors and the CF corrector and modulator drugs tezacaftor and ivacaftor. The company is currently conducting in vivo animal studies. About Porosome Therapeutics, Inc. Porosome Therapeutics, Inc. is a Boston-based biopharmaceutical company utilizing the breakthrough discovery of the porosome, the cell’s secretory machinery, and novel technologies to identify and create proprietary high-value therapeutics for currently undruggable porosome proteins relating to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer. Porosome Therapeutics is the first company with a technology platform that develops novel therapies targeting the porosome, the universal secretory machinery in cells. This platform is based on decades of research supported by more than 200 publications and is effective against many serious diseases involving secretory defects. The platform also specializes in highly specific nanobody-mediated targeted therapies that greatly reduce drug side effects. View source version on businesswire.com: Contacts Laramie Langdon laramie@perrycom.com (916) 658-0144 Source: Porosome Therapeutics, Inc. View this news release online at:

Porosome Therapeutics, Inc. is the first company with a technology platform that develops novel therapies targeting the porosome, the universal cellular structure controlling secretion. Two ground-breaking therapeutic pathways in development: non-invasive cell replacement therapies and the restoration of secretory function to affected porosome proteins. Pre-clinical studies show restoration of normal mucin secretion and function in organs impacted by cystic fibrosis. BOSTON--(BUSINESS WIRE)-- Porosome Therapeutics, Inc. (Porosome Therapeutics) has announced the development of a radically new and distinctive therapeutic offering to treat cystic fibrosis, filing a comprehensive patent addressing the disorder and other health conditions. Porosome Therapeutics’ paradigm-shifting approach is the first therapeutic application of the porosome, the cell’s secretory apparatus. The approach addresses the cell's secretory machinery and water transport regulation to treat secretion and hydration disorders, like cystic fibrosis. More than two decades ago, Porosome Therapeutics’ Founder and Chairman, Professor Bhanu P. Jena, identified a new cellular structure – the porosome. After its discovery within the cell, the porosome was isolated and its structure and composition determined before new porosomes were successfully inserted into cells to enhance secretion. This groundbreaking discovery provides a platform for cystic fibrosis treatments, as well as other secretory and hydration disorders such as certain cancers, Alzheimer’s, and diabetes. The treatment comprises two different therapeutic pathways. Porosome Therapeutics’ first pathway for the treatment of cystic fibrosis calls for the non-invasive replacement of the mutated protein to restore the normal mucin secretion and function in organs by inserting a healthy porosome into a diseased cell. The second pathway uses small molecules to restore secretory function to affected porosome proteins. Porosome Therapeutics’ studies on human lung epithelial cell lines expressing cystic fibrosis have shown significant – and unprecedented – enhancement of mucin secretion. “By incorporating normally functioning CFTR-associated porosomes into diseased cells or by specifically targeting affected porosome proteins, these proposed therapies will ameliorate cystic fibrosis symptoms at a fundamental level, restoring function and thereby treating the disorder,” said Professor Jena. “The porosome platform provides a promising entry into a new era of drug development and therapy.” Porosome Therapeutics has demonstrated positive pre-clinical results and is currently conducting in vivo studies with a leading cystic fibrosis research center. “While treatments exist to lessen symptoms and consequences of cystic fibrosis, until Porosome Therapeutics’ ground-breaking development, none of the existing treatments have addressed the CFTR-mutated protein in terms of its secretory ecosystem,” said Guillermo Marmol, CEO, Porosome Therapeutics. “As the discoverer of the porosome and its potential, and following decades of research, Porosome Therapeutics’ founder has inspired its incredible team to unleash the body’s existing yet untapped capabilities of self-generation to effectively address the core cause of cystic fibrosis symptoms.” About Porosome Therapeutics, Inc. Porosome Therapeutics Inc. is a Boston-based biopharmaceutical company utilizing breakthrough discovery and novel technologies to identify and create patent-pending high-value therapeutics for currently undruggable porosome protein and lipid targets relating to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer. Porosome Therapeutics is the first company with a technology platform that develops novel therapies targeting the porosome, the universal cellular structure controlling secretion. This platform is based on decades of research supported by more than 200 publications and is effective against many serious diseases involving secretory defects. The platform also specializes in highly specific disease-targeting that yields superior therapies and a reduction or elimination of side effects. Porosome Therapeutics Leadership Bhanu P. Jena, PhD, Founder and Chairman Distinguished Professor Bhanu P. Jena is a leading cell biologist and chemist who has made seminal contributions to the understanding of cell secretion, membrane fusion, and transport. He is currently the Founder and Chairman of Porosome Therapeutics, Inc. A full biography can be found here. Guillermo (Gil) Marmol, President and CEO As President and Chief Executive Officer of Porosome Therapeutics, Inc., Gil Marmol applies his broad and unique wealth of strategic planning, development, and operations experience to bring groundbreaking science and technology to market to treat previously intractable and undruggable conditions. A full biography can be found here. Joachim Frank, PhD, Scientific Advisory Board Chairman, 2017 Nobel Laureate in Chemistry Chairman of the Porosome Therapeutics, Inc.’s Scientific Advisory Board, Professor Joachim Frank is a Professor of Biochemistry and Molecular Biophysics and of Biological Sciences at Columbia University and a Distinguished Professor at the State University of New York at Albany. A full biography can be found here. Additional Team Members About Cystic Fibrosis Cystic fibrosis is a genetic condition that adversely affects the cells that secrete mucus, perspiration, and digestive fluids. Normally, these fluids are slick and thin. However, a faulty gene in people with cystic fibrosis makes the secretions in the lungs, digestive system, and other body organs thick and sticky. The secretions clog tubes, ducts, and passages rather than lubricating them, particularly in the pancreas and lungs. It is estimated that more than 160,000 people worldwide are living with cystic fibrosis, with current life expectancy at just 44 years old.

WASHINGTON, Oct. 21, 2022 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VPO-227 for the treatment of cholera. While the incidence of cholera in the U.S. is low, cholera is a widespread infectious diarrheal disease with estimated 1.4 to 4 million cases worldwide, resulting in 21,000 to 143,000 deaths annually.1 Many of these deaths occur in children, as diarrheal diseases are the second leading cause of death globally among children under 5 years of age.2 Cholera remains a major public health challenge and recognized unmet medical need despite decades-long public health efforts. "This designation is an important milestone in the development of VPO-227 and highlights the need for potential new treatment options for patients who suffer from cholera and other conditions of water hyper-excretion," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. VPO-227 is a small-molecule with a novel mechanism of action which blocks the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) ion channel. CFTR plays a pivotal role in cholera-induced intestinal fluid loss as it is affected by the cholera toxin. VPO-227, therefore, has the potential to be an orally administered treatment for cholera. In a recent publication in the journal Toxins, the authors showed that oral VPO-227 protects animals from a lethal cholera challenge using two separate cholera strains.3 Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and the potential for seven years of post-approval marketing exclusivity. Additionally, in order to incentivize development in tropical diseases, including cholera, the FDA has established the Tropical Disease Priority Review Voucher Program, which grants a transferable Priority Review Voucher to sponsors of eligible new drug applications for products treating tropical diseases. Vanda expects to submit an Investigational New Drug (IND) application to the FDA for VPO-227 in 2023. About Vanda Pharmaceuticals Inc. Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on Twitter @vandapharma. About VPO-227 VPO-227 (formerly BPO-27) is a small molecule CFTR inhibitor in development for the treatment of secretory disorders, including cholera. Vanda entered into a license agreement with University of California, San Francisco (UCSF) in 2017, under which Vanda acquired an exclusive worldwide license from UCSF to develop and commercialize a portfolio of CFTR activators and inhibitors, including the activator VPO-227. CFTR activators and inhibitors may have broad applicability in addressing a number of disorders, including chronic dry eye, constipation, polycystic kidney disease, cholestasis, and secretory diarrheas.