Kringle Pharma,Inc. Q2 FY2025 earnings call

• Core Pipeline and Company Mission

  • Company mission: Develop breakthrough treatments for refractory diseases to benefit patients and contribute to society. The company is focused on developing pharmaceutical products based on recombinant hepatocyte growth factor (HGF, international non-proprietary name: Oremepermin Alfa), a naturally occurring human protein with multiple regenerative functions that inhibits cell death, reduces fibrosis, and promotes angiogenesis.
  • The company's strategy is to pursue regulatory approval for domestic pipelines in-house rather than out-licensing, with the long-term goal of transitioning from a biotech venture to a fully-fledged bio-pharmaceutical company.

• Pipeline Development Progress

  • Acute spinal cord injury (ASCI): Phase III trial in Japan is complete. The trial showed 56% of patients achieved at least 1-grade severity improvement (vs 19.8% in the historical control database), which is statistically and clinically meaningful, though the 2-grade improvement endpoint did not reach statistical significance. The company is currently in pre-application consultation with PMDA, following completion of the pre-application meeting. A full supply chain for commercial launch is already in place: Maruishi Pharmaceutical will handle distribution and Toho Holdings will manage wholesale logistics to hospitals.
  • Vocal fold scarring: Phase III double-blind placebo-controlled trial is ongoing. 2 additional trial sites were added in January 2025, and ~80% of patient enrollment is complete. Enrollment completion is delayed from June 2025 to December 2025 due to slower than expected enrollment at new sites, with final study report expected in the first half of 2027. The company will pursue in-house regulatory approval for this indication.
  • ALS: Phase II trial is complete. Additional biomarker analysis in collaboration with Tohoku University has been extended from March 2025 to September 2025, with measurement complete and analysis ongoing. The company plans to secure a development partner after analysis results are available to advance to Phase III.
  • Acute kidney injury: Phase I trial in the US is complete, and the company is currently searching for a partner to advance to Phase II.

• Corporate Infrastructure Updates

  • Obtained Class 1 Pharmaceutical Manufacturing and Marketing Business license in January 2025, which is required for in-house approval applications.
  • Relocated corporate headquarters to the new Nakanoshima Qross facility in central Osaka in April 2025, which provides expanded space for future headcount growth and proximity to other regenerative medicine companies, the PMDA Kansai branch, and an on-site affiliated hospital for better collaboration.

Kringle Pharma is a clinical-stage biotech venture with all revenue derived from out-licensing and partnership activities. In the 2025 September Fiscal Year second quarter, total revenue was 35 million yen (0.035 billion yen), which comes entirely from a fixed annual technology access fee from Clarus Biotherapeutics, accounting for 100% of total revenue. Selling, general and administrative expenses totaled 547 million yen (0.547 billion yen), of which 404 million yen (0.404 billion yen) was research and development expense. Operating loss and ordinary loss were 511 million yen (0.511 billion yen), and net loss for the quarter was 512 million yen (0.512 billion yen). Total assets as of quarter-end were 2.29 billion yen, total liabilities were 0.681 billion yen, total net assets were 1.608 billion yen, and cash and cash equivalents balance was 1.258 billion yen.

• The company maintains its target to submit the NDA for acute spinal cord injury (Japan) within the current 2025 September fiscal year, with approval expected in the 2026 September fiscal year, followed by first product sales and achievement of profitability.
• Vocal fold scarring Phase III patient enrollment is now expected to complete by December 2025, a 6-month downward revision from the original target of June 2025, with the final study report expected in the first half of 2027.
• The additional biomarker analysis for ALS Phase II is now expected to complete by September 2025, a 6-month downward revision from the original March 2025 target.
• The company's long-term guidance targets simultaneous pursuit of global expansion (to access a ~10x larger patient market than Japan alone) and indication expansion via academic collaboration to build new HGF-based pipelines.

• Delays in regulatory consultation with PMDA for the acute spinal cord injury NDA have pushed the expected submission window from April 2025 to May 2025, with no definitive final submission timeline publicly confirmed, creating uncertainty around the approval and launch timeline.
• Patient enrollment for the vocal fold scarring Phase III trial is slower than initially expected, resulting in a 6-month delay to enrollment completion.
• The additional analysis for the ALS program has taken longer than planned, resulting in a 6-month delay to the completion of analysis.
• Cash reserves declined by 558 million yen (0.558 billion yen) quarter-over-quarter due to ongoing R&D spending, creating potential need for additional financing if development timelines extend longer than expected.

Q: The question asks whether Kringle Pharma is conducting genomic analysis to identify how ALS genetic mutations correlate with HGF treatment effect, given that different ALS driver mutations have varying treatment responses. / A: Management notes that most ALS cases are sporadic with unknown causes, and while multiple genetic mutations are linked to hereditary ALS, existing mutation-targeted ALS drugs only benefit a small subset of patients. HGF acts by inhibiting motor neuron cell death regardless of the underlying ALS cause, so it can potentially be effective for all ALS patients by targeting the common downstream disease process rather than individual genetic mutations.