Sichuan Kelun-Biotech's A400/EP0031 NDA Acceptance: A Catalyst for Growth in the Ophthalmology Biotech Sector

The biotech sector has long been driven by the interplay of scientific innovation and regulatory milestones. For Sichuan Kelun-Biotech, the recent acceptance of its New Drug Application (NDA) for A400/EP0031 by China's National Medical Products Administration (NMPA) marks a pivotal moment in its strategic evolution. This next-generation selective RET inhibitor, developed in collaboration with Ellipses Pharma, has demonstrated robust clinical potential in treating RET fusion-positive non-small cell lung cancer (NSCLC), positioning the company to capitalize on a high-growth therapeutic niche.

Strategic Positioning: Leveraging Collaborative Innovation

Sichuan Kelun-Biotech's partnership with Ellipses Pharma underscores its strategic focus on addressing unmet medical needs in oncology. A400/EP0031, a second-generation RET inhibitor, was granted Fast Track Designation by the U.S. FDA in March 2024 for RET fusion-positive NSCLC, a designation that accelerates development timelines and regulatory reviewFDA Grants Fast Track Designation For EP0031/A400 to Treat RET-Altered NSCLC^[3]. This collaboration not only shares the financial and operational burden of drug development but also aligns with global standards, enhancing the drug's market readiness.

The drug's clinical profile further strengthens its strategic appeal. The KL400-I/II-01 trial, which evaluated A400/EP0031 in both first-line (1L) and second-line and above (2L+) settings, reported favorable efficacy and safety outcomes, including durable responses in patients with brain metastases and those previously treated with immunotherapySichuan Kelun Pharmaceutical (002422.SZ): Application for the NDA^[2]. These results position A400/EP0031 as a potential first-line therapy, a critical differentiator in a competitive market.

Regulatory Milestones: A Pathway to Market Access

Regulatory acceptance is a cornerstone of biotech valuation, and Sichuan Kelun-Biotech's NDA acceptance by the NMPA in March 2025Sichuan Kelun Pharmaceutical (002422.SZ): Application for the NDA^[2] represents a significant de-risking event. This milestone follows the successful completion of the KL400-I/II-01 study, which demonstrated the drug's ability to address a patient population with limited treatment options. The NMPA's decision signals confidence in the drug's safety and efficacy, paving the way for commercialization in China—a market with rising demand for targeted cancer therapies.

While the FDA NDA status remains pending as of September 2025, the drug's Fast Track and Orphan Drug designationsFDA Grants Fast Track Designation For EP0031/A400 to Treat RET-Altered NSCLC^[3] provide a regulatory tailwind. These designations, coupled with positive Phase 2 data presented at the 2024 and 2025 ASCO Annual MeetingsSix Abstracts of Kelun-Biotech's Clinical Studies Published at the 2025 ASCO Annual Meeting^[1], suggest a high probability of expedited approval in the U.S., should the company pursue it. Such regulatory clarity reduces uncertainty for investors and enhances the drug's commercial potential.

Market Implications: Redefining the RET Inhibitor Landscape

The global RET inhibitor market is projected to grow as resistance to first-generation therapies and the prevalence of brain metastases drive demand for next-generation options. A400/EP0031's demonstrated efficacy in these challenging patient subgroups positions it to capture market share from existing therapies like LOXO-292 (Selpercatinib) and BLU-945 (Pralsetinib).

Moreover, the drug's dual development by Kelun-Biotech and Ellipses Pharma under a global trial framework (NCT05443126 and NCT05265091)Next-Gen RET Inhibitor EP0031/A400 Advances to Phase 2^[4] ensures a robust commercialization strategy. This approach not only optimizes cost efficiency but also accelerates market penetration in key geographies. For investors, this translates to a scalable revenue model with the potential for cross-border licensing deals or partnerships.

Valuation Potential: Catalysts for Shareholder Value

The NDA acceptance and positive clinical data create a strong foundation for valuation growth. Fast Track and Orphan Drug designationsFDA Grants Fast Track Designation For EP0031/A400 to Treat RET-Altered NSCLC^[3] reduce time-to-market risks, while the KL400-I/II-01 trial's success in diverse patient populations enhances the drug's commercial upside. Analysts estimate that A400/EP0031 could achieve peak sales of $500 million annually in China alone, with additional revenue streams from the U.S. and other marketsSichuan Kelun-Biotech Biopharmaceutical Platform^[5].

From a financial perspective, the drug's regulatory progress aligns with Kelun-Biotech's broader pipeline strategy. The company's expectation to file an NDA for A400 in the second half of 2025Sichuan Kelun-Biotech Biopharmaceutical Platform^[5] reinforces its commitment to a pipeline-driven growth model, which is critical for sustaining long-term shareholder value.

Conclusion

Sichuan Kelun-Biotech's A400/EP0031 NDA acceptance is more than a regulatory checkpoint—it is a strategic catalyst that redefines the company's position in the oncology biotech sector. By combining collaborative innovation, regulatory momentum, and a compelling clinical profile, Kelun-Biotech is poised to deliver transformative value to stakeholders. For investors, this represents a rare opportunity to capitalize on a drug candidate with the potential to reshape treatment paradigms in RET-altered malignancies.