BioCity Biopharma announced that China's National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) to its highly selective endothelin receptor type A (ETA) antagonist SC0062 for treating diabetic kidney disease (DKD) with albuminuria. This represents the second BTD for SC0062, following its initial designation for IgA nephropathy (IgAN) with proteinuria, highlighting the compound's transformative potential across multiple chronic kidney disease indications.
Clinical Trial Results Drive Regulatory Recognition
The BTD designation is supported by compelling data from the DKD cohort of the Phase 2 2-SUCCEED study. SC0062 demonstrated statistically and clinically significant albuminuria reduction at the 20 mg dose versus placebo, accompanied by a favorable safety profile with no increase in fluid retention risk observed in the SC0062 group.
The trial evaluated SC0062's safety both as monotherapy and in combination with standard-of-care therapies. Most participants received SGLT2 (sodium-glucose co-transporter-2) inhibitors and RAAS (renin-angiotensin-aldosterone system) inhibitors as background therapies, with over 70% receiving GLP-1 (Glucagon-like peptide-1) receptor agonists and/or insulin, and approximately one-third on Finerenone (a mineralocorticoid receptor antagonist).
Addressing Significant Unmet Medical Need
"With over 700 million people affected globally, kidney disease represents one of our greatest unmet medical challenges," said Dr. Ivy Wang, BioCity Co-founder and Executive President. "This dual BTD recognition validates SC0062's potential to redefine treatment paradigms across renal diseases. We're accelerating development to deliver this promising therapy to patients worldwide."
DKD affects approximately 20.9 million patients in China as of 2021, driving the nation's CKD epidemic. Without therapeutic innovation, age-standardized incidence rates are projected to rise 25% by 2036. Current therapies provide limited protection against progression to end-stage renal disease (ESRD).
Mechanistic Advantages of ETA Selectivity
As an ETA antagonist optimized for CKD treatment, SC0062 represents a mechanistically distinct approach that modulates renal hemodynamics, reduces proteinuria and albuminuria, and counters inflammation and fibrosis. The compound's high selectivity for ETA compared to endothelin receptor B (ETB) suggests greater potential than non-selective endothelin receptor antagonists for slowing CKD progression while avoiding associated side effects.
Preclinical studies demonstrated that SC0062 significantly improved pathological scores in acute and chronic kidney disease models. Completed Phase 1 studies showed good safety, tolerability, and pharmacokinetics with no signs of fluid retention.
Comprehensive Development Program
The completed Phase 2 2-SUCCEED program met all efficacy and safety endpoints in both IgAN and DKD cohorts at 12- and 24-week timepoints. Full DKD results will be presented at an upcoming international congress. For the IgAN cohort, treatment with SC0062 resulted in clinically meaningful and statistically significant proteinuria reduction with a clear dose-response relationship. Peripheral edema occurred at a much lower rate in SC0062-treated subjects compared to placebo.
BioCity is conducting two Phase 3 trials: SUCCESS-01 for IgAN and SUCCESS-02 for CKD. SUCCESS-01 has launched at Guangdong Provincial People's Hospital, led by Professor Xueqing Yu, President of the hospital and President of the Asia-Pacific Society of Nephrology.
Company Pipeline and Focus
Founded in December 2017, BioCity is a clinical-stage biopharmaceutical company developing novel therapeutics for cancer and autoimmune disorders including CKD. The company has established a pipeline of more than 10 innovative drug candidates, including small molecules, monoclonal and bispecific antibodies, and antibody-drug conjugates (ADC).
Beyond SC0062, BioCity has five core novel oncology assets in clinical development, including first-in-class CDH3- and GPC3-targeting antibody drug conjugates, WEE1 and ATR inhibitors targeting the DNA damage response pathway, and a monoclonal antibody targeting TIM-3.
