FDA Accepts BeyondSpring IND Application for Plinabulin for the Prevention of Chemotherapy-Induced N

BeyondSpring Pharmaceuticals, a global clinical stage biopharmaceutical company focused on the development of innovative immuno-oncology cancer therapies, has announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's Investigational New Drug (IND) application to initiate Phase 2 / 3 trials for its innovative lead asset, Plinabulin, for the prevention of chemotherapy-induced Neutropenia.

"The primary endpoint for this trial is the duration of severe Neutropenia in Docetaxel-treated patients in the first chemotherapy cycle, after randomization to either Plinabulin or Pegfilgrastim (Neulasta®)," said Dr. Douglas Blayney, M.D., FACP, Professor of Medicine (Oncology) at Stanford University and Principal Investigator for BeyondSpring's Neutropenia registrational trial. "With approximately 200 chemotherapy patients who are slated to complete this trial, the study was designed based on the highly statistically significant data that we derived from the Phase 2 data in a lung cancer study. Plinabulin has great potential to improve the care of cancer patients worldwide."

Plinabulin is a novel small molecule with immune-enhancing effects and anti-cancer activity that is given by IV infusion – one hour after chemotherapy – and has the potential to be a safe, cost-effective and convenient alternative to G-CSF, the current standard of care for chemotherapy-induced Neutropenia, with much less bone pain and a more favorable safety profile. Plinabulin offers a same-day dosing advantage over G-CSF, which, per product label, should be administered 24 hours after chemotherapy (next-day dosing), by which time a significant insult to Neutrophils has occurred. Recent data presented at ASH also showed that Plinabulin reduced the clinical sequelae associated with Docetaxel-induced Neutropenia – i.e., infections, sepsis, hospitalizations and the need for Docetaxel dose reductions.

"Going from concept to a FDA-accepted IND within six months could only be achieved through having strong Phase 2 clinical data," added Dr. Ramon Mohanlal, M.D., Ph.D., BeyondSpring Chief Medical Officer. "The acceptance of BeyondSpring's IND application reflects the U.S. FDA's commitment to working with BeyondSpring on this novel approach to address a serious and life-threatening condition, like chemotherapy-induced Neutropenia. This also illustrates BeyondSpring's ability to successfully plan and execute in an expedited and efficient manner."

Neutropenia, a common side effect of chemotherapy in cancer patients, is the destruction of a type of white blood cell (Neutrophil) that is a key component of the innate immune system. Neutrophils are a patient's first line of defense against infections, and patients with severe (grade 4) Neutropenia (an abnormally low concentration of Neutrophils in the blood) are more susceptible to severe bacterial, viral and fungal infections and sepsis, which require hospitalization.

More than 60,000 patients are hospitalized each year for chemotherapy-induced severe Neutropenia, which is associated with fever, infections and death in up to 18 percent of these cases. When severe Neutropenia occurs, the chemotherapy dose has to be reduced or interrupted until the Neutropenia subsides. This reduction or interruption causes patients to receive suboptimal chemotherapy cancer treatment.

"The FDA's acceptance of BeyondSpring's IND application for Plinabulin for the prevention of chemotherapy-induced Neutropenia marks an important regulatory milestone for the Company," concluded Dr. Lan Huang, BeyondSpring CEO. "With this opportunity, the Company is well-positioned to deliver near-term returns to our investors. It also highlights the dedication and hard work of a team that has already built a strong foundation of data for Plinabulin over the years, entered the Phase 3 trial for non-small cell lung cancer and is committed to developing innovative cancer therapies globally to address unmet medical needs. Cancer patients are in need of effective, affordable, safe and convenient treatment options, and Plinabulin has the potential to be a breakthrough solution to these challenges."