NR5A1

SEOUL, South Korea, May 18, 2023 /PRNewswire/ -- ImmunoForge has announced on the 18th that it has expanded its license rights with Duke University to enhance joint research and drug development opportunities in relation to the elastin-like polypeptide (ELP) platform technology. Through this agreement, ImmunoForge expects to accelerate the development of new drugs based on the ELP technology that was originally developed by Professor Ashutosh Chilkoti at Duke University. Dr. Jim Ballance, Chief Technology Officer (CTO) of ImmunoForge, has pioneered the clinical development of peptide drugs that are genetically fused to ELPs. The powerful research partnership between ImmunoForge and Duke University will revolutionize the delivery of peptide and protein drugs. Duke University will have the right to develop new anticancer drugs with the ELP platform technology, and, if such drugs are commercialized with a partner, the university will share a portion of the profits with ImmunoForge. ImmunoForge retains the right to develop new products in all therapeutic areas other than anticancer drugs, and if it succeeds in global commercialization, a portion of the profits will be distributed to Duke University. ImmunoForge recently received approval from the Korean Ministry of Food and Drug Safety at the end of March 2023 for a Phase 2 clinical trial for the treatment of Idiopathic Inflammatory Myopathy (IIM) with PF1801, an ELP platform pipeline candidate, and is set to enter clinical trials at 10 university hospitals in Korea. Idiopathic inflammatory myopathies are diseases with high unmet medical need with currently no treatments other than steroids. Idiopathic inflammatory myopathy: A group of chronic autoimmune diseases that mainly affect proximal muscle. IIM includes polymyositis (PM), dermatomyositis (DM), and inclusion body myositis (IBM). It is characterized by severe deterioration in quality of life of patients, such as difficulty in standing up, climbing stairs, lifting objects, and swallowing foods. The aim of treatment is to improve the patients' quality of life such as treating inflammation and restoring muscle strength. ImmunoForge is a venture-backed biopharma company that focuses on the development of new drugs for rare diseases and has received a total of four Orphan Drug Designations (ODD) from the US Food and Drug Administration (FDA), including one for Dermatomyositis that was received on March 29 2023. This is the second-highest number of ODD received by a single company from the US FDA in Korea. If a drug receives an ODD from the US FDA, it benefits from market exclusivity for up to seven years, regulatory assistance and guidance from FDA, tax credit on clinical trial expenses and eligibility for research grants. ImmunoForge has also disclosed that it has signed a license agreement with a global pharmaceutical company for development of one or more drugs based on the ELP technology. However, specific contract details, such as the financial terms of the contract and technical matters related to the drugs, remain confidential. "We are pleased to expand our relationship with ImmunoForge to continue development of novel therapeutics using the ELP platform, originally developed by Professor Chilkoti at Duke University", said Duke University Associate VP for Translation & Commercialization, Robin Rasor. ImmunoForge's co-CEOs Sung-min Ahn and Kiho Chang stated "It is very meaningful that our capabilities in new drug development have been reinforced through expanding our cooperation with Duke—a leading global university–and by outlicensing to global companies", and that they "will continue to create open innovation and joint research opportunities with domestic and foreign companies, universities and research institutes for the ELP technology and ImmunoForge's new drug pipeline." SOURCE ImmunoForge

SAN DIEGO--(BUSINESS WIRE)-- Orphagen Pharmaceuticals, Inc., a biotech company pioneering the screening, discovery, and development of small molecule ligands that modulate orphan or unexplored members of the nuclear receptor family, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small molecule antagonist to steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland. Orphagen is developing OR-449 for both the adult and pediatric forms of ACC as well as other cancers known to express a high level of SF-1. “We are gratified that OR-449 has received an RPDD from the FDA. We currently plan to Investigational New Drug (IND) application with the FDA later this year to support initiation of a Phase 1 clinical trial,” said Orphagen CEO, Scott Thacher, Ph.D. “We speak to clinicians regularly who remind us of the urgent need for an improved therapy for patients with ACC, and this drives our commitment to the clinical development of OR-449, our first internal program to reach this stage.” About ACC and SF-1 ACC is a rare and aggressive cancer of the adrenal gland. Surgical removal of an affected adrenal gland is effective treatment if the tumor has not metastasized. Once an ACC tumor becomes metastatic, as it does for most patients, it is difficult to control and five-year survival in both adult and pediatric patients with metastatic disease is low, about 10-20%. The estimated annual incidence of ACC in the U.S. is 600 patients/year. In clinical practice, SF-1 is widely used as a marker for ACC, and it is recognized as a potential therapeutic target for both adult and pediatric ACC. SF-1 is commonly amplified at the gene level in pediatric ACC, and SF-1 is recognized as a cell lineage marker in the FDA’s Pediatric Cancer Target List. Rare Pediatric Disease Designation A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act to include a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. To address the challenges that drug companies face when developing treatments for these unique patient populations, Congress reauthorized the Creating Hope Act. Since first enacted by Congress in 2012, the Creating Hope Act has been extended multiple times, most recently on December 27, 2020. Under this Act, companies are eligible to receive a Priority Review Voucher (PRV) following approval of a product with an RPDD if the marketing application receives FDA approval by September 30, 2026, under current legislation, or at a later date if the Act is extended by Congress. If issued, a sponsor may redeem a PRV for priority review of a subsequent marketing application for a different product candidate, or the PRV can be sold or transferred to another sponsor. About Orphagen Pharmaceuticals Orphagen unlocks the power of orphan nuclear receptors. Our scientists excel in discovery and development of small molecule ligands to these largely unexplored drug targets. Starting with a therapeutic area agnostic approach, we are exploring proprietary lead molecules with potential in autoimmune disease and oncology. Orphagen successfully partnered its first program for ROR-gamma antagonists with a mid-size pharmaceutical company ahead of all competitors in the field. Funding from its partnerships and other non-dilutive sources, including federal grants, has allowed Orphagen to advance its proprietary first-in-class drug discovery programs, including OR-449 for adrenocortical cancer. For more info, visit .

RADNOR, Pa.--(BUSINESS WIRE)-- Aclipse Therapeutics (“Aclipse” or “the Company”), a private biopharmaceutical company, today announced that the Company and its collaborator, The Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield in the United Kingdom (UK), were awarded a drug development research grant of £1.6 million (approximately US $2.2 million) from the UK’s Medical Research Council (MRC), one of the largest funders of medical research worldwide, to support the translational development of M102. M102 is Aclipse’s drug candidate for the treatment of amyotrophic lateral sclerosis (ALS), also referred to as motor neuron disease (MND) or Lou Gehrig’s disease. M102 is a potentially disease-modifying drug candidate that has shown promise to impede ALS disease progression in a wide array of preclinical models. Currently, there is no cure for ALS and there are no effective treatments to halt or slow the progression of the disease. “This development funding from MRC is wonderful news for ALS/MND patients who are in dire need of an effective therapy to address this life-threatening neurodegenerative disease,” stated Professor Dame Pamela Shaw, M.D., Director of SITraN and a primary contributor to M102’s development program. “Along with my SITraN colleagues, Dr. Richard Mead and Dr. Laura Ferraiuolo, we spearheaded the ALS/MND biology research that led to the development of M102, including the discovery of a potential precision medicine approach for M102 in ALS/MND, so we are very appreciative of MRC’s funding support.” Aclipse is taking a multiple biological pathway, multiple disease mechanism approach to ALS. M102 activates the NRF2 (nuclear factor erythroid 2-related factor 2) and HSF1 (Heat shock factor 1) signaling pathways, which are recently understood to impact ALS pathophysiology. M102 is expected to be mechanistically superior to currently available drugs and may lead to significant slowing of disease progression in both familial and sporadic ALS. The MRC grant will also support the development of patient stratification biomarkers that will be applied in the M102 clinical studies, potentially enabling a personalized medicine approach in ALS. The goal of the patient stratification biomarkers is to identify M102 drug responders versus non-responders in order to target M102 to those ALS patients most likely to benefit from the drug. “We greatly appreciate the support from MRC for our novel and broad multi-disease patho-mechanism approach to treating ALS patients,” said Raymond K. Houck, CEO of Aclipse Therapeutics. “The MRC award, coupled with our recent FightMND grant award, accelerates M102’s development into its first-in-human clinical studies and validates M102’s biology and potential for a precision medicine approach for the treatment of ALS.” “The research funding from these programs will be key as they will support the completion of our investigational new drug (IND)-enabling work and the regulatory filings for first-in-human studies. Importantly, M102 may have applications in a wide array of conditions associated with impaired neuronal function such as Friedreich’s ataxia, Huntington’s disease and Parkinson’s disease,” added Mr. Houck. About ALS/MND Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a progressive neurodegenerative disease that affects motor neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and limited life-prolonging treatments for the disease. Based on U.S. population studies, approximately 5,600 people in the U.S. are diagnosed with ALS each year and as many as 25,000 Americans have the disease at any given time. About Medical Research Counsel The United Kingdom’s Medical Research Counsel’s mission is to improve human health through world-class medical research. To achieve this, MRC supports research across the biomedical spectrum, from fundamental lab-based science to clinical trials, and in all major disease areas. MRC works closely with the UK’s National Health Service and the UK Health Departments to deliver its mission and give a high priority to research that is likely to make a real difference to clinical practice and the health of the population. About the Sheffield Institute for Translational Neuroscience The Sheffield Institute for Translational Neuroscience (SITraN) is an international center of excellence recognized for its ground-breaking work in the fight against motor neurone disease and other common neurodegenerative disorders. SITraN brings together 300 staff and research students in multi-disciplinary teams with state-of-the-art laboratories and equipment to study neurological illness. The center is unique in its design to unite clinicians and multidisciplinary teams of scientists to translate discoveries in basic neuroscience into benefits for patients. The SITraN teams have developed a robust portfolio of in vitro and in vivo models to facilitate our understanding of disease mechanisms and identify new targets for therapeutic intervention which can be tested in our BRC experimental medicine programs. The work of SITraN is a major pillar of the University of Sheffield’s cross-faculty Neuroscience Institute, one of four flagship research institutes launched in 2019 to tackle the biggest global challenges through pioneering real-world solutions and involving >120 principal investigators in the Faculties of Medicine, Science and Engineering. About Aclipse Therapeutics Aclipse Therapeutics develops novel and differentiated drugs to treat orphan diseases with significant unmet medical needs. Our lead drug candidate, M102, is in development for the treatment of ALS with potential use in other neurodegenerative diseases such as Friedreich’s ataxia, Huntington's disease and Parkinson's disease. M102 targets multiple disease pathomechanisms and enables a precision medicine approach for the identification of patients who are most likely to benefit from the drug. Aclipse has a very experienced orphan drug management team and a clinical advisory board of the top ALS physicians in the world. For more information about Aclipse, visit the website at or email info@aclipsetherapeutics.com. View source version on businesswire.com: