BeyondSpring Receives Breakthrough Therapy Designations from Both U.S. FDA and China NMPA for Plinabulin in Chemotherapy-Induced Neutropenia Indication
- Positive interim analysis results from the PROTECTIVE-2 Phase 3 study ? The clinically meaningful primary endpoint was the prevention of severe neutropenia, an ?industry first,? building on the previous standard for approval, the Duration of Severe Neutropenia (DSN). In the protocol-specified interim analysis, Plinabulin, in combination with Neulasta, was significantly better than Neulasta alone in achieving the primary endpoint with p<0.01, along with a well-tolerated safety profile and fewer Grade 4 adverse events, compared to Neulasta alone.
- These results were further strengthened by the Company?s other CIN studies that have confirmed Plinabulin?s 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.
About Plinabulin Plinabulin, BeyondSpring?s lead asset, is a differentiated immune and stem cell modulator. Plinabulin is currently in late-stage clinical development to increase overall survival in cancer patients, as well as to alleviate chemotherapy-induced neutropenia (CIN). The durable anticancer benefits of Plinabulin have been associated with its effect as a potent antigen-presenting cell (APC) inducer (through dendritic cell maturation) and T-cell activation (Chem and Cell Reports, 2019). Plinabulin?s CIN data highlights the ability to boost the number of hematopoietic stem / progenitor cells (HSPCs), or lineage-/cKit+/Sca1+ (LSK) cells in mice. Effects on HSPCs could explain the ability of Plinabulin to not only treat CIN but also to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34+ cells in patients.
About Plinabulin in CIN Study The PROTECTIVE-1 (Study 105) and PROTECTIVE-2 (Study 106) trials are both multicenter, double-blind, active controlled Phase 3 trials to support Plinabulin?s broad application in preventing CIN: Plinabulin for concurrent administration with myelosuppressive chemotherapy regimens in patients with non-myeloid malignancies for the presentation of chemotherapy-induced neutropenia (CIN).PROTECTIVE-1 (Study 105) This study was designed to evaluate the safety and efficacy in non-small cell lung cancer (NSCLC), breast cancer and prostate cancer patients with risk factors, treated with docetaxel (Day 1 dose) in a 21-day cycle with a single dose of Plinabulin (40mg, Day 1 dose) versus a single dose of Neulasta (6mg, Day 2). Docetaxel is one example of an intermediate-risk chemotherapy. This is a non-inferiority study in CIN efficacy comparing Plinabulin and Neulasta in high-risk patients (intermediate chemotherapy, plus one or more additional risk factor). Study 105 Phase 3 interim data had achieved statistical significance based on the primary endpoint of the Duration of Severe Neutropenia (DSN) in the first cycle.
PROTECTIVE-2 (Study 106) This study was designed to evaluate the safety and efficacy in breast cancer, treated with docetaxel, doxorubicin and cyclophosphamide (TAC, Day 1 dose) in a 21-day cycle with Plinabulin (40 mg, Day 1 dose) in combination with Neulasta (6 mg, Day 2 dose) versus a single dose of Neulasta (6 mg, Day 2 dose) alone. TAC is an example of high-risk chemotherapy. Plinabulin and G-CSFs have complementary mechanisms in preventing chemotherapy-induced neutropenia (CIN). This is a superiority study in CIN efficacy in the rate of Grade 4 neutropenia prevention (primary endpoint), comparing the combination head-to-head against Neulasta, and is currently enrolling. Literature shows that the Grade 4 neutropenia rate for TAC and Neulasta at 6 mg is 83 to 93 percent, which presents severe unmet medical needs.
Covance is the clinical contract research organization (CRO) for patient recruitment and monitoring of global sites for this study. The CIN studies are conducted in over 60 clinical centers in the U.S., China and Europe. In addition, Absolute Neutrophil Count (ANC) data, which is used to calculate these endpoints, was obtained through central laboratory assessments by Covance Bioanalytical Methods using standardized and validated analytical tests.
About BeyondSpring Headquartered in New York, BeyondSpring is a global, clinical-stage biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients with high unmet medical needs. BeyondSpring?s first-in-class lead immune asset, Plinabulin, is a potent antigen-presenting cell (APC) inducer. It is currently in two Phase 3 clinical trials for two severely unmet medical needs indications: one is for the prevention of chemotherapy-induced neutropenia (CIN), the most frequent cause for a chemotherapy regimen dose?s decrease, delay, downgrade or discontinuation, which can lead to suboptimal clinical outcomes. The other is for non-small cell lung cancer (NSCLC) treatment in EGFR wild-type patients. As a ?pipeline drug,? Plinabulin is in various I/O combination studies to boost PD-1 / PD-L1 antibody anti-cancer effects. In addition to Plinabulin, BeyondSpring?s extensive pipeline includes three pre-clinical immuno-oncology assets and a drug discovery platform dubbed ?molecular glue? that uses the protein degradation pathway.
Cautionary Note Regarding Forward-Looking Statements This press release includes forward-looking statements that are not historical facts. Words such as "will," "expect," "anticipate," "plan," "believe," "design," "may," "future," "estimate," "predict," "objective," "goal," or variations thereof and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are based on BeyondSpring's current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to, difficulties raising the anticipated amount needed to finance the Company's future operations on terms acceptable to the Company, if at all, unexpected results of clinical trials, delays or denial in regulatory approval process, results that do not meet our expectations regarding the potential safety, the ultimate efficacy or clinical utility of our product candidates, increased competition in the market, and other risks described in BeyondSpring?s most recent Form 20-F on file with the U.S. Securities and Exchange Commission. All forward-looking statements made herein speak only as of the date of this release and BeyondSpring undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.
Media Contacts Caitlin Kasunich / Raquel Cona KCSA Strategic Communications ckasunich@kcsa.com?/?rcona@kcsa.com