BTD Granted for BioCity's SC0062 by NMPA for IgAN with Proteinuria

SHANGHAI, Sept. 26, 2024 – BioCity Biopharma (BioCity) has announced that its selective endothelin receptor type A (ETA) antagonist, SC0062, has received the Breakthrough Therapy Designation (BTD) from the National Medical Products Administration (NMPA) for the treatment of IgA nephropathy (IgAN) characterized by proteinuria.

The BTD was awarded due to the promising outcomes from the Phase 2 trial, 2-SUCCEED, which focused on patients with IgAN and proteinuria. This trial was a randomized, double-blind, placebo-controlled study aiming to assess the efficacy and safety of SC0062 in patients suffering from two types of chronic kidney disease (CKD): IgAN and diabetic kidney disease (DKD).

Participants treated with SC0062 showed a significant reduction in proteinuria, demonstrating a clear dose-response relationship and a favorable safety profile. Notably, peripheral edema was less common in those treated with SC0062 compared to the placebo group. Moreover, when combined with SGLT2 inhibitors, SC0062 maintained a good safety profile. These findings will be presented at an upcoming scientific conference. The trial continues for the DKD cohort, with results expected by the end of the year.

IgAN is one of the most prevalent primary glomerular diseases globally, with many affected individuals progressing to end-stage renal disease (ESRD) within 10-20 years, necessitating dialysis or kidney transplantation. There is a high demand for more effective and well-tolerated treatments that can delay the progression to ESRD.

Recent studies have indicated that endothelin receptor antagonists can improve renal blood flow, reduce proteinuria, and mitigate inflammatory and fibrotic responses, positioning them as promising treatments for IgAN and other CKD forms. SC0062, as a novel and highly selective ETA antagonist, has shown potential to be a leading treatment option for IgAN and CKD based on Phase 1 study findings in healthy volunteers and Phase 2 results in IgAN patients from the 2-SUCCEED trial.

BioCity is planning global Phase 3 clinical trials for SC0062, targeting registration and market approval for treating IgAN, DKD, and other CKD types.

SC0062 distinguishes itself by its high selectivity for ETA over endothelin receptor B (ETB), suggesting a greater capacity to slow CKD progression while avoiding the safety risks associated with nonselective ET antagonists. Preclinical research has shown that SC0062 improves pathological markers in acute kidney injury and CKD models. The Phase 1 study confirmed its favorable safety profile, good tolerability, and predictable pharmacokinetics. Importantly, fluid retention, a common adverse effect with non-selective ET antagonists, was not observed in SC0062 trials.

The ongoing Phase 2 study is a multi-center, randomized, double-blind, placebo-controlled trial with two parallel cohorts (IgAN and DKD), led by Professor Jianghua Chen from the First Affiliated Hospital of Zhejiang University School of Medicine. The study involves over 40 sites nationwide. The IgAN cohort met its primary endpoint, leading to the BTD from NMPA, while results for the DKD cohort are anticipated later this year.

BioCity, established in December 2017, is dedicated to developing innovative therapeutics for cancer and autoimmune disorders, including CKD. It boasts a pipeline of over 10 drug candidates, including small molecules, monoclonal and bispecific antibodies, and antibody-drug conjugates (ADCs). Among its core assets in Phase 1/2 clinical development are CDH3-targeting and GPC3-targeting ADCs, WEE1 and ATR inhibitors targeting the DNA damage response pathway, and a TIM-3 monoclonal antibody in collaboration with AstraZeneca.

BioCity's SC0062 is in Phase 2 clinical development, with a global Phase 3 trial in the planning stages. The company is also advancing five core oncology assets, demonstrating its commitment to addressing unmet medical needs in oncology and autoimmune diseases.