GSK Inks $1B+ siRNA Deal for Metabolic Disease Candidate SA030; Avalo’s IL-1β Antibody Meets Phase 2 Primary Endpoint in Hidradenitis Suppurativa

Over $1 billion: GSK secures rights to develop siRNA therapy

Suzhou Shian Biotechnology Co., Ltd. ("Shian Biotech") announced that it has entered into a global exclusive license agreement with GSK for SA030 (excluding mainland China, Hong Kong, Macao, and Taiwan).SA030 is a long-acting small interfering RNA (siRNA) currently in development for the treatment of metabolic and cardiovascular diseases.SA030 has recently entered Phase 1 clinical trials, targeting activin receptor-like kinase 7 (ALK7).

Cardiometabolic diseases are the leading cause of death in approximately 50% of patients with chronic kidney disease and liver disease. Targeting ALK7 can reduce visceral adipose tissue (VAT) while preserving lean body mass, thereby improving insulin sensitivity, lipid profiles, and alleviating adipocyte-driven inflammation. Preclinical studies indicate that SA030 possesses differentiated long-acting properties, enabling intervention in fundamental inflammation associated with cardiometabolic risks through adipocyte-targeted delivery and a low-frequency dosing regimen.SA030 has a complementary and unique mechanism with GLP-1 receptor agonists and SGLT2 inhibitors, providing support for potential future combination therapy regimens to reduce residual cardiometabolic risks that current therapies have not fully addressed.

Under the agreement, GSK will pay an upfront fee and future milestone payments based on the successful achievement of development, registration, and commercialization milestones, with a total amount of up to $1.005 billion. Shian Bio will lead the clinical development of SA030 until the completion of Phase 1 clinical trials. After that, GSK will assume responsibilities for development, regulatory submissions, and commercialization outside mainland China, Hong Kong, Macao, and Taiwan.

IL-1β Antibody Phase 2 Clinical Trial Meets Primary Endpoint

Avalo Therapeutics Announces Positive Topline Results from Phase 2 LOTUS Trial of Abdakibart for the Treatment of Adults with Moderate to Severe Hidradenitis Suppurativa (HS). The LOTUS trial successfully met its primary endpoint in both dose groups studied. Based on these data, Avalo plans to advance abdakibart into a pivotal Phase 3 clinical trial.

Abdakibart is a humanized monoclonal antibody that binds with high affinity to interleukin-1β (IL-1β) and neutralizes its activity.IL-1β is a pro-inflammatory cytokine that plays a central role in the pathogenesis of various human diseases. It can activate immune cells, prompting them to produce pro-inflammatory cytokines including IL-6, TNF-α, and IL-17. Dysregulation of IL-1β signaling is a key driver of inflammatory responses and can promote the progression of autoimmune diseases. IL-1β inhibition has been shown to be effective in a variety of immune-mediated inflammatory diseases.

Phase 2 LOTUS trial successfully met the primary endpoint in both dose groups studied (150 mg group, p=0.018; 300 mg group, p=0.015; pooled analysis p=0.004).The proportion of subjects achieving a clinical response of HiSCR75 for hidradenitis suppurativa at Week 16 improved by 42.2 and 42.9 percentage points compared to the placebo group (response rate in the placebo group was 25.6%).

Bayer to Acquire Perfuse Therapeutics, Gaining Rights to Small Molecule Endothelin Receptor Antagonist

Bayer and Perfuse Therapeutics Announce Agreement for Bayer to Acquire Perfuse TherapeuticsBayer and Perfuse Therapeutics today announced an agreement under which Bayer will acquire Perfuse Therapeutics. Through this acquisition, Bayer will obtain full rights to PER-001. Under the terms of the agreement, the potential total value of the transaction could reach up to $2.45 billion, including a $300 million upfront payment and additional development, regulatory, and commercial milestone payments based on success criteria.

PER-001 is a potential "first-in-class" small molecule endothelin receptor antagonist.Endothelin is one of the most potent vasoconstrictive factors in the human body and is upregulated in glaucoma, diabetic retinopathy, age-related macular degeneration, and retinal vein occlusion. Endothelin participates in inflammation and cell death processes through receptors expressed on retinal vascular cells and neuroretinal cells. PER-001 is currently in Phase 2 clinical development for the treatment of glaucoma and diabetic retinopathy (DR).