BeyondSpring Pharmaceuticals Announces New Patient-Derived Cancer Model Data for Plinabulin Monotherpy at the AACR-NCI-EORTC Virtual International Conference to Further Support the Positive Clinical Data Already Reported for the Treatment of Small Cell Lung Cancer

Rhea-AI Summary

BeyondSpring Pharmaceuticals (BYSI) announced promising preclinical data showcasing the effectiveness of plinabulin against small cell lung cancer (SCLC) at the 2021 AACR-NCI-EORTC conference. Plinabulin demonstrated strong **anti-cancer effects** in 80 patient-derived tumor models, particularly in SCLC with an **IC₇₀ of 35 nM**. The combination of plinabulin with nivolumab and ipilimumab reported a **46% objective response rate** in clinical trials. These findings bolster BeyondSpring’s strategy for developing plinabulin as a key cancer treatment option and highlight its potential in combination therapies.

Positive

• Plinabulin shows a **46% objective response rate** in combination with nivolumab and ipilimumab in SCLC.
• Preclinical data indicates plinabulin's strong efficacy against SCLC with an **IC₇₀ of 35 nM**.
• Ongoing development of plinabulin may expand its indications in treating various cancers.

Negative

• None.

• Plinabulin’s anti-cancer effects were tested against approximately 80 patient-derived tumor models with small cell lung cancer (SCLC) tumor types being the most sensitive to plinabulin monotherapy at IC₇₀ of 35 nM
• The preclinical data is consistent with clinical data in SCLC: plinabulin, in combination with nivolumab and ipilimumab, shows a 46% objective response rate (ORR) in 13 evaluable patients with PD-1/PD-L1 naïve or resistant tumors in 2^nd line and beyond in SCLC
  

NEW YORK, Oct. 20, 2021 (GLOBE NEWSWIRE) -- BeyondSpring Pharmaceuticals (the “Company” or “BeyondSpring”) (NASDAQ: BYSI), a global pharmaceutical company focused on the development of cancer therapeutics, today announced the presentation of preclinical data in patient-derived (PDX) cancer models supporting the use of plinabulin in small cell lung cancer (SCLC) at the 2021 AACR-NCI-EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics, held October 7-10, 2021. Plinabulin is a selective immunomodulating microtubule-binding agent (SIMBA), which induces dendritic cell maturation for durable anti-cancer benefit, and selectively induces certain types of cancer cell apoptosis, both through immune defense protein GEF-H1 release and activation. This study demonstrates plinabulin’s direct single agent anti-cancer tumor effects in PDX models of SCLC and additional cancer types, including glioblastoma multiforme, bladder cancer, gastric cancer, sarcoma and triple-negative breast cancer.

“The positive PDX preclinical data increases our confidence in pursuing SCLC as an indication for plinabulin, which has shown high response rate data in human studies. This is supplemental to the maturation of dendritic cells in the cancer immune system and the induction of downstream mechanisms in specific cancer cell types,” said Dr. Kenneth Lloyd, chief scientific officer of BeyondSpring. “The fact that plinabulin has already been associated with tumor responses in SCLC patients in a Phase 1 study presented at the 2021 ASCO Annual Meeting is a good indication that these PDX models are predictive. The preclinical data presented here supports our intended strategy for developing an option for cancer patients by combining plinabulin and checkpoint inhibitors in SCLC.”

The study was intended to screen cancer cells for susceptibility to plinabulin. For this purpose, PDX models of cancer established from patient tumor biopsies were used in a manner that results in minimal chance for changes in cancer cell genetics, and hence, these models are considered more predictive of clinical response than common cancer models. Eighty PDX three-dimensional culture models were tested. Data showed that the cancer types most sensitive to plinabulin were:

• SCLC (mean absolute IC₇₀ = 35 nM; n = 7)
• Bladder cancer (mean absolute IC₇₀ = 38 nM; n = 9)
• Soft tissue sarcoma (mean absolute IC₇₀ = 57 nM; n = 10)
  

Dr. Lan Huang, BeyondSpring’s co-founder, CEO and chairwoman concluded, “We have been developing plinabulin in multiple cancer indications based on its effects on dendritic cells and macrophages. Plinabulin’s anti-cancer effect is supported by the positive Phase 3 DUBLIN-3 non-small cell lung cancer study with evidence of the extension of overall survival. Plinabulin has also shown efficacy in preventing chemotherapy-induced neutropenia, which has received Breakthrough Designation and Priority Review from the U.S. FDA. Plinabulin’s single agent anti-cancer activity in PDX SCLC models and the relevant positive immuno-oncology combo preliminary clinical data in SCLC could potentially expand its cancer indications. We look forward to continuing to expand these anti-cancer indications in the clinic and to validate plinabulin as a ‘pipeline in a drug.’”

About Plinabulin
Plinabulin, BeyondSpring’s lead asset, is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell (APC) inducer. It is a novel, intravenous infused, patent-protected, NDA stage asset for CIN prevention and a Phase 3 anti-cancer candidate for non-small cell lung cancer (NSCLC). Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anticancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells, and the second is early-onset of action in CIN prevention after chemotherapy by boosting the number of hematopoietic stem/progenitor cells (HSPCs). It is being developed as a “pipeline in a drug” in multiple cancer indications.

About BeyondSpring Pharmaceuticals
Headquartered in New York City, BeyondSpring is a global biopharmaceutical company focused on developing innovative cancer therapies to improve clinical outcomes for patients who have high unmet medical needs. BeyondSpring’s first-in-class lead asset plinabulin, is being developed as a “pipeline in a drug” in various cancer indications as direct anti-cancer agent and to prevent chemotherapy induced neutropenia (CIN). Plinabulin and G-CSF combination has filed for approval and has received breakthrough designation and Priority Review in the U.S. and China for the prevention of CIN with a PDUFA date of November 30, 2021, in the U.S. In the DUBLIN-3 study, a global, randomized, active controlled Phase 3 study, the plinabulin and docetaxel combination has met the primary endpoint of extending overall survival compared to docetaxel alone, in 2^nd/3^rd line NSCLC (EGFR wild type). Additionally, it is being broadly studied in combination with various immuno-oncology regimens that could boost the efficacy of PD-1/PD-L1 antibodies in seven different cancers. In addition to plinabulin, BeyondSpring’s extensive pipeline includes three pre-clinical immuno-oncology assets and a subsidiary, SEED Therapeutics, which is leveraging a proprietary targeted protein degradation drug discovery platform.

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.

Investor Contact:
Ashley R. Robinson
LifeSci Advisors, LLC
+1 617-430-7577
arr@lifesciadvisors.com

Media Contact:
Darren Opland, Ph.D.
LifeSci Communications
+1 646-627-8387
darren@lifescicomms.com

FAQ

What were the results of the plinabulin studies related to SCLC?

Plinabulin demonstrated a **46% objective response rate** in combination with nivolumab and ipilimumab in SCLC.

How effective is plinabulin against small cell lung cancer?

In preclinical testing, plinabulin showed **IC₇₀ of 35 nM**, indicating strong anti-cancer effects.

What are the next steps for BeyondSpring in developing plinabulin?

BeyondSpring plans to pursue further clinical development and combination studies for plinabulin in multiple cancer types.

What types of cancers are being targeted with plinabulin?

Plinabulin is being studied in various cancers, including **SCLC, glioblastoma multiforme, bladder cancer, and triple-negative breast cancer**.

What is the significance of the PDUFA date for plinabulin?

The PDUFA date of **November 30, 2021**, is crucial for potential FDA approval of plinabulin for preventing chemotherapy-induced neutropenia.