The standard care for the treatment of potentially life-threatening chemotherapy complications is a 30-year-old drug that is not used until the second week of treatment. BeyondSpring Pharmaceuticals’ attempts to provide a new treatment that is effective in the first week must wait a period of time after the FDA Refuse Biotechnology applications seek regulatory approval.
BeyondSpring said on Wednesday that the agency’s full response letter stated that the company’s individual clinical trial results for its drug prinabulin were “not enough to prove its benefits” and a second well-controlled trial was needed to provide evidence to support the new drug application. New York-based BeyondSpring said it plans to request a meeting with the FDA to discuss the matter.
The dangerous complication that BeyondSpring aims to solve is called chemotherapy-induced neutropenia (CIN). Neutropenia is a dangerously low level of white blood cells called neutrophils, and patients with CIN are susceptible to infection. The standard treatment for CIN is Granulocyte Colony Stimulating Factor (G-CSF)The drug is sold under the names Neuopgen, Zarxio, and Nivestym, and includes a bone marrow stimulant called filgrastim and polyethylene glycol filgrastim, which is a long-acting form of filgrastim. The period after chemotherapy starts but before G-CSF starts to work is sometimes referred to as the “neutropenia vulnerability gap.”
Pranabrin is derived from a compound found in marine microorganisms. According to BeyondSpring, this small molecule triggers the release of immune defense proteins that provide anti-cancer benefits by activating antigen-specific T cells that target cancer cells. This protein also provides immune system benefits by increasing the number of hematopoietic stem and progenitor cells. BeyondSpring says this effect is aimed at preventing CIN.
BeyondSpring has obtained global rights to prenabulin from Nereus Pharmaceuticals and continues to test its new assets in two phase 2/3 studies of CIN. These clinical trials recruited approximately 500 patients who were randomly assigned to receive prinabulin or polyethylene glycol filgrastim. The main goal of the first phase 3 study was to measure the non-inferiority compared with polyethylene glycol filgrastim by the duration of severe neutropenia in the first chemotherapy cycle. BeyondSpring reported that the drug achieved this goal.
In the second phase 3 clinical trial—the single study that serves as the basis for the new drug application for prenabulin—BeyondSpring reported that its drug combined with pegfilgrastim can prevent CIN better than pegfilgrastim alone. The drug achieved its main goal of showing a statistically significant improvement in the prevention rate of grade 4 neutropenia. The company also reported that the drug reduced clinical complications, including febrile neutropenia and the length of stay in patients with febrile neutropenia.
BeyondSpring’s drug pipeline relies heavily on punablin. The company is evaluating the drug’s anti-cancer properties in several cancer studies. The most advanced of these studies is the phase 3 trial of non-small cell lung cancer. The clinical trial is evaluating Pranabrin combined with chemotherapy docetaxel as a second-line or third-line treatment for cancer.
Adding another phase 3 study to punablin in CIN will become expensive. In its second-quarter financial report, BeyondSpring reported that its cash position as of June 30 was US$76.3 million. The company said at the time that this was sufficient to support its ongoing clinical projects to 2022, and to launch pranablin. prepare for.
After the FDA announced its refusal to use punabrine in CIN, BeyondSpring’s stock price plummeted on Wednesday. The company’s stock price closed at $4.93 on the trading day, down more than 61% from Tuesday’s closing price.
Photos of Flickr users Magdalena Wicklund Through knowledge sharing license
