China grants lisaf toclax breakthrough therapy designation as combination undergoes Phase 3 trial
Key Facts
Ascentage Pharma said on October 20 that lisaf toclax in combination with acalabrutinib had received breakthrough therapy designation in China under a programme to develop its use in previously untreated patients. The company said in a statement that the Center for Drug Evaluation of China's National Medical Products Administration granted the designation to the combination. The targeted use covers adults with chronic lymphocytic leukaemia or small lymphocytic lymphoma, expanding development into first-line treatment for both diseases. The announcement follows an earlier Chinese approval for lisaf toclax in patients who had previously received systemic therapy. However, the combination with acalabrutinib still lacks marketing approval, with the company confirming that it "has not been approved in any market".
Development of the combination is based on a clinical collaboration between Ascentage Pharma and AstraZeneca, according to the companies' announcement of the registrational study programme. The confirmatory study was designed as a global, multicentre Phase 3 trial to test the combination in previously untreated patients. The trial uses a randomised, open-label design and compares the combination with chemoimmunotherapy. When announcing the collaboration, Ascentage Pharma said earlier results for the combination showed an objective response rate of 100% in previously untreated patients with chronic lymphocytic leukaemia or small lymphocytic lymphoma. That result relates to the use of the two drugs together under the combination development programme addressed by the confirmatory study.
The earlier Chinese approval for lisaf toclax covers adults with either disease who have received at least one systemic therapy, including Bruton's tyrosine kinase inhibitors. That approval was based on a pivotal Phase 2 registrational study, according to a company filing with the U.S. Securities and Exchange Commission. The study tested lisaf toclax alone in patients with relapsed or refractory disease, a use that differs from the combination intended for previously untreated patients. The company says the drug works by inhibiting an anti-apoptotic protein to restore the natural process of programmed cell death in cancer cells. Ascentage Pharma retains global rights to the drug, according to published information on its research and development programme.
In another clinical update, Ascentage Pharma presented an analysis of the pivotal study involving patients whose chronic lymphocytic leukaemia or small lymphocytic lymphoma had become resistant to Bruton's tyrosine kinase inhibitors. The analysis included 72 evaluable patients, according to the company's announcement of its updates at the European Hematology Association congress. Median progression-free survival in this group was 23.9 months. The objective response rate was 62.5%, as assessed by an independent review committee. These results concern the treatment-resistant patient group in the pivotal study testing the drug alone, while the new Chinese designation targets the combination in previously untreated patients.
On the financial front, Ascentage Pharma reported revenue of $44.5 million in the first half of the current year, compared with $32.6 million in the corresponding period a year earlier. The company attributed the increase mainly to product sales, according to its unaudited interim results. Meanwhile, cash and bank balances stood at $279.4 million at the end of June. That was down from $353.2 million at the end of the preceding December, as the global clinical trial programme proceeded. The company said the decline in cash and bank balances was mainly due to the acceleration of those trials and increased research and development spending.
Commercial sales of lisaf toclax began in China on July 25, 2025, following approval for its use in previously treated patients. Revenue from sales of the drug totalled about $10.1 million over the five months from August to December 2025, according to the company's annual results. By the end of June of the current year, the number of direct-to-patient pharmacies and hospitals that had added it to their formularies had reached 415 institutions. That total included 60 hospitals, according to the interim disclosure. Ascentage Pharma plans during 2026 to advance the inclusion of lisaf toclax in China's National Reimbursement Drug List as part of its next commercial steps for the drug.