London: GSK plc and Empirico Inc., a clinical-stage biotechnology company with capabilities in human genetics-driven target discovery and siRNA medicines, have announced that they have entered into a worldwide exclusive license agreement for EMP-012, a highly selective first- and potentially best-in-class siRNA, a type of oligonucleotide. EMP-012 addresses a novel therapeutic target and is currently in a phase I trial for the treatment ofchronic obstructive pulmonary disease(COPD), with the potential for expansion into other inflammatory respiratory diseases. Medical need in COPD remains high despite ongoing innovation. By 2050, COPD prevalence is expected to increase to about 600 million people and become the leading cause of all hospital admissions, costing healthcare systems approximately $4 trillion. Given the heterogeneous nature of COPD, novel therapeutic approaches are needed to treat the full spectrum of patients, especially those who do not qualify for biologics and face continued symptoms, exacerbations and hospitalisations. EMP-012’s novel mechanism of action, discovered by Empirico, targets a distinct inflammatory pathway introducing the potential for a therapeutic approach that is agnostic of baseline type 2 inflammation, smoking or co-morbid disease. The target is backed by extensive genetic data and translational insight. Based on this mechanism, EMP-012 could offer clinical benefit in patients with non-type 2 inflammation, a key patient sub-group where treatment options are limited. EMP-012’s enhanced potency and longer dosing interval, resulting from Empirico’s proprietary siRNA chemistry, strategically complementsGSK’s ongoing programmes in COPD, including long-acting biologics. In addition to its potential as a monotherapy, EMP-012 provides GSK further optionality for combinations across its COPD portfolio and pipeline. Kaivan Khavandi, SVP & Global Head, Respiratory, Immunology & Inflammation R&D, GSK said: “This agreement reflects our ambition to transform care in COPD by advancing novel targets, backed by data, to address underlying drivers of disease. With its expected long-acting characteristics and ability to target distinct inflammatory pathways, EMP-012 complements our pipeline of diverse modalities in COPD and builds on the current landscape of inhaled and biologic therapeutics in this area of substantial unmet need.” Oligonucleotides, such as siRNA therapeutics, have a distinct ability to modulate gene expression, making them an attractive modality to address a significant proportion of therapeutic targets that are not amenable to traditional small molecules or biologics. EMP-012 bolsters GSK’s already extensive oligonucleotide pipeline across GSK’s respiratory, immunology and inflammation and infectious diseases therapy areas. Omri Gottesman, CEO and President, Empirico said: “This collaboration further validates the enormous potential of Empirico’s proprietary target discovery and siRNA platforms to rapidly generate differentiated clinical programs of significant value. GSK’s global reach and deep expertise in COPD will help to accelerate the development of EMP-012, and we look forward to working closely with GSK on advancing a potentially transformative precision medicine for patients with COPD and other inflammatory respiratory diseases.” The agreement grants GSK full worldwide development and commercial rights to EMP-012. Empirico will continue to lead the clinical development of EMP-012 through the completion of the ongoing phase I clinical trial, following which GSK will assume responsibility for worldwide development, regulatory filings, and commercialisation. GSK will pay an $85 million upfront payment and up to $660 million in success-based development, regulatory and commercial milestones, as well as tiered royalties on net sales worldwide.