BeyondSpring Inc. Q4 FY2021 earnings call

• Ongoing discussions with China NMPA on the review of Plinabulin NDA in combination with G-CSF for prevention of chemotherapy-induced neutropenia (CIN); G-CSF market in China is significant with $1.2 billion in sales in 2020 and ~30% annual growth since 2017.
• Positive top line data from Phase 3 DUBLIN-3 study in NSCLC (second and third line with EGFR Wild Type) showing significant improvement in overall survival, targeting NDA filing in China by year-end.
• Strategic partnership with Hengrui Pharmaceuticals for development and commercialization of Plinabulin in Greater China; received RMB 200 million upfront and eligible for up to RMB 1.1 billion in milestones.
• Progress with Seed Therapeutics focusing on differentiated molecular glue technology in targeted protein degradation; signed R&D collaboration agreement with Eli Lilly in November 2020.
• CIN program: Plinabulin increases neutrophil count rapidly, positive data in over 1,200 patients, but more data needed for US FDA approval; ongoing discussions with FDA on design of second Phase 3 CIN study.
• NSCLC program: Plinabulin has dual mechanism of action (immune enhancing and direct anti-cancer), positive Phase 3 DUBLIN-3 and Phase 1 trial data in small cell lung cancer, NDA filing in China by year-end as near-term priority with ongoing US FDA discussions.

In the fourth quarter of 2021, R&D expenses were $5.8 million compared to $8.4 million in the same period last year, a decrease of $2.6 million primarily due to lower clinical development expenses and personnel costs (partially offset by higher preclinical and professional expenses). G&A expenses were $5.0 million in the fourth quarter of 2021 (including a non-cash credit of $2.0 million related to the reversal of share-based compensation expense) compared to $10.4 million for the prior year. The net loss attributable to the company in the fourth quarter of 2021 was $9.5 million compared to $17.6 million for the same period last year. For the full year 2021, R&D expenses were $36.9 million compared to $41.8 million for the prior year, a decrease of $4.9 million primarily due to lower clinical development expense and non-cash share-based compensation expense (partially offset by higher personnel costs, pre-clinical and professional services expenses, and a $2.9 million NDA application fee paid to FDA expected to be refunded in Q2 2022). G&A expenses for the full year 2021 were $30.7 million compared to $22.6 million for the prior year, with the majority of the $8.1 million increase due to higher pre-commercialization expenses for Plinabulin. The net loss attributable to the company for the full year was $64.2 million compared to $61.0 million for the prior year. Cash balance at December 31, 2021 was $41.6 million with short-term investments of $30.7 million, totaling $72.4 million, which is sufficient to support ongoing operations and clinical programs over the next year.

• Targeting NDA filing in China for NSCLC by year-end.
• Ongoing discussions with FDA regarding clinical and regulatory pathway for Plinabulin in CIN and NSCLC in the US.
• Cash balance and short-term investments of $72.4 million sufficient to support ongoing operations and clinical programs over the next year.

• Uncertainties in regulatory processes, including potential delays in approvals for Plinabulin in CIN and NSCLC in both China and the US.
• Relevance of data from Chinese patient populations to US patients being a factor in regulatory discussions for NSCLC in the US.
• Inherent uncertainties in the regulatory approval process which can impact timelines and outcomes.

Q: What kind of feedback on a potential approval decision for CIN in China have you received so far and what's the timeframe for approval?

A: The CIN NDA application is under independent review with China NMPA; had multiple positive meetings with CDE, remain hopeful but regulatory process has uncertainties, optimism based on strong data from Asian patients in Phase 3 study.

Q: Can you elaborate on conversations with FDA on the additional study for CIN in the US and when it could start?

A: Active discussions ongoing with US FDA on design of the study, will disclose more when clarity is available.

Q: Is there still a path forward in the US for non-small cell lung cancer and when will we learn more?

A: Active discussions with US FDA ongoing, data is positive, most data derived from Chinese population is an important topic in discussions.

Q: What's the role of Hengrui in the regulatory filing discussion in China for CIN and key factors still to be addressed?

A: Hengrui is an ideal partner, working together to prepare answers for MPA review questions; still answering some review questions from MPA before final review.

Q: What are the chances BeyondSpring will conduct the second study for CIN on its own versus in partnership?

A: Plan to do it ourselves initially, but open to partnership if design is done and partner comes along