GSK Secures $745M License Deal for Novel COPD siRNA Therapy, Receives Orphan Drug Status for Cancer ADC
核心洞察
GSK entered a worldwide exclusive license agreement with Empirico (搜索) for EMP-012, a first-in-class siRNA targeting a novel therapeutic pathway for COPD treatment, with potential expansion to other inflammatory respiratory diseases.
The deal involves $85 million upfront and up to $660 million in milestones plus royalties, addressing the growing COPD burden expected to reach 600 million patients by 2050.
GSK's B7-H3 (搜索)-targeted antibody-drug conjugate GSK576422 (搜索) received orphan drug designation from the EMA for pulmonary neuroendocrine carcinoma, marking its fourth regulatory designation.
GSK has secured worldwide exclusive rights to a promising siRNA therapy for chronic obstructive pulmonary disease through a license agreement worth up to $745 million with San Diego-based Empirico (搜索) Inc. The deal centers on EMP-012, a highly selective first- and potentially best-in-class siRNA currently in phase I trials for COPD treatment.
Major Investment in Novel COPD Approach
The pharmaceutical giant will pay $85 million upfront and up to $660 million in success-based development, regulatory and commercial milestones, plus tiered royalties on worldwide net sales. EMP-012 addresses a novel therapeutic target discovered by Empirico (搜索) and represents a significant addition to GSK's respiratory portfolio.
"This agreement reflects our ambition to transform care in COPD by advancing novel targets, backed by data, to address underlying drivers of disease," said Kaivan Khavandi, SVP & Global Head, Respiratory, Immunology & Inflammation R&D at GSK. "With its expected long-acting characteristics and ability to target distinct inflammatory pathways, EMP-012 complements our pipeline of diverse modalities in COPD."
Addressing Growing COPD Burden
The medical need for new COPD treatments remains substantial. By 2050, COPD prevalence is expected to increase to approximately 600 million people and become the leading cause of all hospital admissions, costing healthcare systems around $4 trillion. Given COPD's heterogeneous nature, novel therapeutic approaches are needed to treat the full spectrum of patients, particularly those who do not qualify for biologics and continue to experience symptoms, exacerbations and hospitalizations.
EMP-012's novel mechanism of action targets a distinct inflammatory pathway, introducing potential for a therapeutic approach that is agnostic of baseline type 2 inflammation, smoking status, or co-morbid disease. The target is supported 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 subgroup where treatment options are currently limited.
Enhanced siRNA Technology
The therapy's enhanced potency and longer dosing interval result from Empirico (搜索)'s proprietary siRNA chemistry, strategically complementing GSK's ongoing COPD programs, including long-acting biologics. Beyond its potential as monotherapy, EMP-012 provides GSK additional optionality for combinations across its COPD portfolio and pipeline.
Empirico (搜索) will continue leading clinical development of EMP-012 through completion of the ongoing phase I trial, after which GSK will assume responsibility for worldwide development, regulatory filings, and commercialization.
"This collaboration further validates the enormous potential of Empirico (搜索)'s proprietary target discovery and siRNA platforms to rapidly generate differentiated clinical programs of significant value," said Omri Gottesman, CEO and President of Empirico. "GSK's global reach and deep expertise in COPD will help accelerate the development of EMP-012."
Cancer ADC Receives Regulatory Recognition
Separately, GSK announced that its B7-H3 (搜索)-targeted antibody-drug conjugate GSK576422 (搜索) received orphan drug designation from the European Medicines Agency for treating pulmonary neuroendocrine carcinoma, a form of small-cell lung cancer. The designation was supported by preliminary clinical data showing durable responses in patients with extensive stage small-cell lung cancer treated with GSK576422 in the phase I Artemis-001 clinical trial.
This marks the fourth regulatory designation for GSK576422 (搜索), which is being developed across multiple solid tumor types including lung, prostate and colorectal cancers. The compound previously received Priority Medicines designation from the EMA for relapsed or refractory extensive stage small-cell lung cancer and Breakthrough Therapy Designations from the FDA for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma.
Strategic Portfolio Expansion
EMP-012 strengthens GSK's extensive oligonucleotide pipeline across respiratory, immunology and inflammation, and infectious diseases therapy areas. Oligonucleotides like siRNA therapeutics possess distinct abilities to modulate gene expression, making them attractive for addressing therapeutic targets not amenable to traditional small molecules or biologics.