Kringle Pharma,Inc. Q1 FY2025 earnings call

Company Overview & Core Strategy

• Kringle Pharma is a university-born biotech venture with a mission to develop breakthrough treatments for intractable rare diseases, focused exclusively on commercializing HGF (Hepatocyte Growth Factor), a naturally occurring human protein with multiple regenerative, anti-fibrotic, and neuroprotective biological functions.
• The company is the only firm globally that has established mass production capabilities for pharmaceutical-grade HGF, having completed process validation for the lead candidate to support regulatory approval.
• Core business model: Prioritize in-house development through regulatory approval for domestic indications (rather than early out-licensing) to maximize long-term revenue, with sales partnership with a domestic Japanese pharma company post-approval. Target rare diseases largely undeveloped by large pharma, with business viability supported by high biologic drug pricing and benefits from Japan's orphan drug designation.

Pipeline Development Progress

• Lead Pipeline: Acute Spinal Cord Injury (aSCI): Phase III trial completed, with 56% of treated complete cervical spinal cord injury patients achieving at least 1-stage improvement in severity (vs 19.8% in historical control, statistically significant), which management calls a clinically meaningful result that reduces mortality risk and enables further recovery from future treatments. All preparation for approval is complete, including manufacturing validation and acquisition of a Class 1 pharmaceutical manufacturing and marketing business license in January 2025. The company is currently in pre-application consultation with Japan's PMDA.
• Second Pipeline: Vocal Fold Scarring: Phase III randomized placebo-controlled trial is ongoing, with 2 additional trial sites added in January 2025 to accelerate patient enrollment, for a total of 8 active sites. Enrollment is targeted for completion by June 2025, with final follow-up and summary report completion expected in first half of 2026.
• Third Pipeline: ALS (Amyotrophic Lateral Sclerosis): Phase II trial completed, with no statistically significant difference in the primary endpoint, but observed slowed progression in some treated patients. Additional biomarker analysis in collaboration with Tohoku University is ongoing, on track to complete in March 2025, to support future partner seeking.
• Fourth Pipeline: Acute Kidney Injury: Phase I trial completed, the company is currently seeking a partner to conduct Phase II development.

Corporate Growth Strategy

• Short-term: Submit aSCI for approval in 2025, gain approval and launch in FY2026 (ending September 2026), achieve early profitability from product sales. Sufficient funding is already secured for aSCI and vocal fold scarring domestic development via existing cash and AMED CiCLE grant funding.
• Mid-long term: Expand aSCI development to the US/ Europe, with trial preparation ongoing using proceeds from prior year fundraising, targeting a collaborative development partnership with a global pharma firm to access the much larger global market (10x larger than Japan's market for the company's target indications). Pursue indication expansion: expand aSCI to chronic spinal cord injury, expand vocal fold scarring to other fibrotic diseases, and add new pipeline candidates via academic collaborations with Japanese universities.

Kringle Pharma is a clinical-stage biotech focused exclusively on developing Oremepermin Alfa (recombinant HGF protein) for rare intractable diseases, with no commercial product revenue in the quarter. Total revenue for the quarter was 18 million yen (1.8 million yen converted per rules), consisting entirely of a technology access fee from Clarus Corporation. Selling, general and administrative expenses totaled 230 million yen, of which 161 million yen was research and development expense. The company reported an operating loss, ordinary loss, and net loss of 212 million yen for the quarter. No separate product segments are reported, as all operations are centered on HGF-based regenerative drug development.

• The approval submission timeline for acute spinal cord injury (aSCI) has been revised from the prior guidance of March 2025 to April 2025 or later; no specific new submission date is provided, as the timeline is dependent on the outcome of ongoing pre-application consultation with PMDA.
• The company maintains its prior guidance of gaining aSCI regulatory approval and launching the product in FY2026 (ending September 2026).
• Vocal fold scarring Phase III enrollment is still guided to complete by June 2025, with trial completion and final report expected in first half of 2026, unchanged from prior plans.
• ALS additional biomarker analysis is still on track to complete in March 2025 as planned.

• The timing of the aSCI approval submission is uncertain due to ongoing consultation with PMDA, and the original March 2025 target is no longer achievable. There is no guarantee that the Phase III trial data will meet PMDA's requirements for regulatory approval, even though the trial met the secondary endpoint with statistical significance.
• Clinical development of all pipeline candidates carries inherent risk of failure to demonstrate sufficient safety or efficacy to gain regulatory approval.
• The company is currently operating at a net loss, and will remain unprofitable until the lead product is launched and generates sustained commercial revenue, which depends on successful approval and commercial partnership execution.
• Partner seeking for ALS and acute kidney injury may not result in a favorable partnership on acceptable terms.

No substantive question and answer section is included in the provided transcript.