Chinese BET Inhibitor NHWD-870 Receives Breakthrough Therapy Designation for Rare NUT Carcinoma
核心洞察
Wenda Pharma (搜索)'s oral BET (搜索) inhibitor NHWD-870 HCI received Breakthrough Therapy Designation from China's NMPA for advanced thoracic NUT carcinoma (搜索) patients who failed prior chemotherapy.
Phase II clinical data showed a 45% objective response rate in thoracic NUT carcinoma (搜索) patients and median overall survival of 9.33 months, significantly extending survival compared to standard chemotherapy.
The designation addresses a critical unmet need for NUT carcinoma (搜索), a rare cancer with median survival of only 6.5 months and no approved targeted therapies globally.
Zhejiang Wenda Pharmaceutical Technology Co., Ltd. announced that its novel oral BET (搜索) inhibitor NHWD-870 HCI has received Breakthrough Therapy Designation from the China National Medical Products Administration (NMPA) for treating advanced thoracic midline (NUT) carcinoma in patients who have failed prior chemotherapy. This regulatory milestone represents the first targeted therapy breakthrough for a rare cancer that has historically left patients and physicians with limited treatment options.
Addressing Critical Unmet Medical Need
NUT carcinoma (搜索) is a rare, poorly differentiated carcinoma characterized by NUTM1 (搜索) gene rearrangement that affects primarily young patients with a median age of onset of 23.6 years. The disease progresses rapidly with a median survival of only approximately 6.5 months and commonly occurs in the lungs, head, and neck regions. Due to lack of specificity in clinical manifestations and insufficient awareness, misdiagnosis and missed diagnosis rates are extremely high, with most patients diagnosed at advanced stages.
Currently, no effective targeted drug for NUT carcinoma (搜索) has been approved globally, leaving the treatment landscape extremely dire for patients facing this aggressive malignancy.
Promising Phase II Clinical Results
The Breakthrough Therapy Designation was granted based on significant efficacy and survival benefits demonstrated in NHWD-870's Phase II clinical study. As of December 27, 2025, the trial enrolled 40 evaluable subjects with advanced NUT carcinoma (搜索), showing positive anti-tumor activity across different lesion sites.
The data revealed that the objective response rate (ORR) in 20 patients with advanced thoracic NUT carcinoma (搜索) reached 45.00%. More significantly, the median overall survival (mOS) for both advanced thoracic NUT carcinoma patients and all subjects reached 9.33 months, representing a substantial extension compared with standard chemotherapy regimens. Some patients continue receiving ongoing treatment and close follow-up.
Preliminary safety data indicated that NHWD-870 was generally well tolerated, demonstrating high potential for clinical application.
Clinical Impact and Expert Perspectives
Professor YIN Mingzhu, Director of the Midline (NUT) Carcinoma Specialty at the Affiliated Three Gorges Hospital of Chongqing University, emphasized the transformative potential of this development: "NUT carcinoma (搜索) once plunged countless patients and doctors into despair. The Breakthrough Therapy Designation for NHWD-870 is an important achievement integrating medical research and clinical practice. In the future, with the accelerated market entry of this drug, the treatment landscape of NUT carcinoma is expected to be completely transformed, injecting new momentum into the cause of rare disease diagnosis and treatment in our country."
The designation enables accelerated regulatory review, potentially bringing this targeted therapy to patients sooner and allowing them to escape the current dilemma of having no effective treatment options. This development drives the transformation of NUT carcinoma (搜索) treatment from "symptomatic treatment" to "precision targeted therapy."
Company Pipeline and Development Strategy
Founded in 2013 and headquartered in Shaoxing City, China, Wenda Pharma (搜索) focuses its research on oncology, immunology, and neurodegenerative diseases. The NHWD-870 program has successfully completed Phase II clinical trials and holds potential to become a "First-in-Class" oral small-molecule BET (搜索) inhibitor.
Mr. Nenghui Wang, Founder of Wenda Pharma (搜索), stated: "We are delighted that our product has received Breakthrough Therapy Designation. This progress highlights China's growing capabilities in innovative drug development for rare cancers. NHWD-870 not only brings hope to NUT carcinoma (搜索) patients but also offers new avenues for exploring treatments for other rare malignancies."
The company maintains a robust pipeline with WD-890 TYK2 (搜索) (JH2) inhibitor entering Phase III clinical trials for moderate-to-severe plaque psoriasis (搜索), with Phase II trials for systemic lupus erythematosus (搜索) indication set to commence shortly. Additionally, WD-910, targeting neurodegenerative diseases, is poised to enter Phase II clinical trials in China after completing Phase I trials in Australia, featuring 100% blood-brain barrier penetration capability as a potential "Best-in-Class" drug candidate.