SLC6A8

NEW YORK--( BUSINESS WIRE )-- RTW Charitable Foundation (“RTWCF”) announced today that it has awarded $900,000 in research grants to support six research studies. These studies aim to drive transformational progress in genetic therapies and align with RTWCF’s mission to power community initiatives and scientific research to improve the health of underserved communities. Grant Recipients The following studies and researchers each received $150,000 research grants from RTWCF: “ Establishing gene replacement therapy for NEDAMSS, an ultra-rare neurological disorder caused by mutations in the IRF2BPL gene” (Kathrin Meyer, Nationwide Children’s Hospital, Columbus, OH). “ Optimizing AAV-SPL gene therapy for sphingosine phosphate lyase insufficiency syndrome” (Julie Saba, University of California, San Francisco, San Francisco, CA). “Gene therapy for SURF1-related Leigh Syndrome” (Steven Gray, The University of Texas Southwestern, Dallas, TX). “ Safety and toxicity assessment of AAV-based gene therapy for creatine transporter deficiency” (Jagdeep Walia, Queen’s University, Kingston, Ontario, Canada). “Advanced therapeutics for SELENON-related myopathy” (Alan Beggs, Boston Children’s Hospital, Boston, MA). “ Preclinical studies of ASO therapy for SPTLC1-related hereditary sensory neuropathy” (Haiyan Zhou, University College London, London, UK). “ Despite considerable advancements in gene and cell therapies that deliver more targeted and effective treatments for rare diseases, a significant amount of additional research is still required to move these advances out of the lab and safely to patients,” said Juan Carlos López, PhD, Managing Director, Research Grants, RTW Charitable Foundation. “ RTW Charitable Foundation is proud to provide financial and scientific support to these six incredible researchers and their respective studies. We believe that each of these studies will help expedite the development of gene therapies for rare and ultrarare diseases, and greatly improve the lives of individuals with unmet medical needs.” Grant Recipient Commentary Julie Saba, MD, PhD, commented : “ I am honored and grateful to receive this award from RTWCF, which will support studies to optimize gene therapy for sphingosine phosphate lyase insufficiency syndrome. This newly recognized inborn error of metabolism leads to kidney failure in most of the affected children, causing more than half of them to die in their first years of life. While kidney transplantation can be lifesaving, it is invasive, costly, unavailable to many, and does not address the many other serious consequences of the disorder. Gene therapy offers a potentially lifesaving alternative treatment. The studies undertaken with RTWCF funding should improve our gene therapy potency, allowing us to advance to toxicology studies and clinical trials. I appreciate RTWCF’s dedication to advancing research in ultrarare diseases, which are overwhelmingly underfunded. Every child deserves personalized therapy!” Alan Beggs, PhD, commented: “ Our group has studied a neuromuscular condition called Multiminicore Disease or Rigid Spine Muscular Dystrophy for many years to understand how mutations of a gene called SELENON lead to weakness. We are now on the cusp of translating our laboratory experiments into a treatment that can be tested in patients. I’m thrilled that support from the RTWCF is helping us to work towards developing a next-generation gene therapy candidate designed for eventual testing in a clinical trial.” Haiyan Zhou, MD, PhD, commented: “ We are extremely grateful for the generous award from RTWCF. This grant serves as a vital catalyst in advancing our groundbreaking work on a novel nucleic acid therapy designed for an ultrarare neurodegenerative condition, currently devoid of effective treatment options. The crucial support from RTWCF will propel the preclinical development and hasten the clinical translation of this experimental therapy. Moreover, this award will play a pivotal role in elevating the impact of our translational genetic therapy program at the Great Ormond Street Institute of Child Health and NIHR GOSH Biomedical Research Centre. It also contributes to the growth of the UK platform for nucleic acid therapy in rare diseases.” To date, RTWCF has awarded over 20 research grants, totaling over $3 million in funding. In addition to delivering financial resources, RTWCF also provides grant recipients with hands-on scientific support, helping them improve or further develop their research and research tools and identify new therapeutic targets. About RTW Charitable Foundation Founded in 2018 as the charitable foundation arm of RTW Investments, LP, RTW Charitable Foundation works to power community initiatives and scientific research to improve the health of underserved communities. The Foundation is able to provide capital, manpower, and logistical support to help scientists push such projects forward. In addition, the Foundation aims to assist in advocacy, disease awareness and directly support organizations and communities in New York City. To learn more, visit www.rtwcf.org .

Merck KGaA has said it will be paying $45m upfront for licensing rights to Inspirna’s mid-stage colorectal cancer drug. The candidate, ompenaclid (RGX-202), is an oral inhibitor of the creatine transport channel SLC6A8 and is currently being evaluated in a phase 2 trial for the second-line treatment of RAS-mutated advanced or metastatic colorectal cancer (mCRC). The agreement gives Merck an exclusive licence to ompenaclid outside of the US, with an option to co-develop and co-promote the drug in the US, as well as global co-development and co-commercialisation rights for follow-on SLC6a8 candidates. Recent data from a phase 1b/2 of ompenaclid in combination with FOLFIRI chemotherapy and bevacizumab showed encouraging efficacy and safety data, the companies said. The results, which were presented by Inspirna at the European Society for Medical Oncology Congress in October last year, demonstrated a median progression-free survival of 10.2 months and a median overall survival of 19.1 months across all 41 patients with RAS-mutated mCRC. Victoria Zazulina, head of cancer drug development at Merck, said: “Over the past decade, the treatment paradigm for patients with RAS-mutated colorectal cancer, accounting for approximately 45% of the second-line population, has not seen major innovation. “With our expertise in the treatment of CRC and based on the encouraging early data for ompenaclid, this agreement with Inspirna offers the opportunity to advance a potential new first-in-class therapy that may improve outcomes for patients.” Dr Usman Azam, chief executive officer of Inspirna, said the company was "excited to partner with Merck KGaA… to help bring [its] novel therapies to more patients in need”. “The data to date validates our belief in ompenaclid as a potential first-in-class therapy for advanced colorectal cancer and underscores the power of our proprietary target discovery platform, RNA-DRIVEr,” he said. The agreement comes just one month after Merck announced a licensing agreement with Abbisko Therapeutics for pimicotinib (ABSK021), which is currently being evaluated in a late-stage study to treat tenosynovial giant cell tumour. The company also announced two new drug discovery collaborations with BenevolentAI and Exscientia in September last year to harness artificial intelligence-driven design and discovery capabilities to accelerate drug discovery.

NEW YORK--( BUSINESS WIRE )--Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class cancer therapeutics, announced today that Chief Executive Officer Dr. Usman “Oz” Azam, M.D., will participate in a corporate presentation at the 42 nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11, 2024 at 8:30am PT / 11:30am ET. About Inspirna Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates. Inspirna’s lead drug candidate, ompenaclid (RGX-202), is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. Ompenaclid is currently being tested in multiple mid stage clinical trials in patients with advanced or metastatic RAS mutant colorectal cancer. Inspirna’s second clinical stage drug candidate, abequolixron (RGX-104), is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. Abequolixron is currently being tested in a Phase 1b/2 clinical trial in patients with advanced or metastatic lung cancer in combination with docetaxel and endometrial cancer in combination with ipilimumab through a clinical collaboration with Bristol Myers Squibb. Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, Silicon Valley Bank, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit https://inspirna.com/ .