The FDA granted breakthrough therapy designation to BeyondSprings first-in-class agent plinabulin for the chemotherapy-induced neutropenia (CIN) indication, according to a BeyondSpring press release.
Data for the full PROTECTIVE-2 phase 3 study is expected for the fourth quarter of 2020 and the company will file a new drug application (NDA) with the FDA by the end of 2020.
“Receipt of breakthrough therapy sesignation from the FDA acknowledges both the significant unmet need among patients with CIN and the highly encouraging clinical results generated by Plinabulin, said Douglas Blayney, MD, global principal investigator for plinabulins CIN studies, in a press release.
The designation was granted based on positive interim analysis data from the phase 3 PROTECTIVE-2 study. The data found that plinabulin combined with pegfilgrastim (Neulasta) was significantly better than pegfilgrastim alone in achieving the primary end point (p < 0.01).
More, there was a well-tolerated safety profile and fewer grade 4 adverse events with plinabulin combined with pegfilgrastim compared to pegfilgrastim alone. The primary end point of the research was the prevention of severe neutropenia.
This should expedite plinabulins move into the clinic, which is beneficial for patients, said Blayney. The currently approved CIN prevention agents are all G-CSF-based and not available to all patients. Even with the use of G-CSFs, over 80 percent of cancer patients undergoing chemotherapy may still experience Grade 4 neutropenia, which could lead to severe infection, hospitalization and even death. Thus, CIN still represents an unmet medical need.
The results from the study were further strengthened by other CIN studies from BeyondSpring confirming early onset action in week 1 with protecting neutrophils in various cancer types and various chemotherapies, which is complementary to Week 2 neutrophil protection with G-CSFs.
For patients undergoing treatment involving the destruction of white blood cells, CIN is a common side effect. For patients with grade 4 neutropenia, there exists an abnormally low concentration of neutrophils, which could lead to infection, hospitalization, or death.
Plinabulin is a differentiated immune and stem cell modulator in the late stages of clinical development to increase patient overall survival and alleviate CIN. The benefits of plinabulin are associated with its effect as a potent antigen-presenting cell (APC) inducer and T-cell activation.
“The clinical profile Plinabulin has shown truly represents a breakthrough in the CIN space since G-CSFs,” Ramon Mohanlal, MD, PhD, MBA, chief medical officer and executive vice president of research and development, at BeyondSpring, said in a press release. We look forward to continuing to work with the FDA as we advance the development of Plinabulin to address this urgent medical need.
BeyondSpring Receives Breakthrough Therapy Designations from Both U.S. FDA and China NMPA for Plinabulin in Chemotherapy-Induced Neutropenia Indication [news release]. New York, New York. Published September 8, 2020.https://www.globenewswire.com/news-release/2020/09/08/2089925/0/en/BeyondSpring-Receives-Breakthrough-Therapy-Designations-from-Both-U-S-FDA-and-China-NMPA-for-Plinabulin-in-Chemotherapy-Induced-Neutropenia-Indication.html. Accessed September 8, 2020.