Kringle Pharma,Inc. Q4 FY2025 earnings call

Corporate Mission and Core Pipeline Focus

• University-originated biotech venture focused on developing novel treatments for intractable diseases using HGF, a naturally occurring human protein with tissue regeneration, anti-fibrotic, and neuroprotective properties
• International non-proprietary name for the company's HGF asset is Oremepermin Alfa

Late-stage Pipeline Progress

• Acute Spinal Cord Injury (ASCI): Completed Phase III trial in Japan; after 1 year of consultation with PMDA, the company decided in July 2025 to conduct an additional trial before submitting approval application. The originally planned January 2025 manufacturing approval was returned in August 2025 and will be re-obtained when approval submission is ready. Analysis of completed trial data shows a reproduced improvement trend in severity, but no statistical significance vs. external database; additional trial will exclude cases with extremely severe injury to concentrate responders, increase response rate, enable smaller trial size and higher success probability, design is currently being finalized with PMDA. Post-approval supply chain agreement with Maruishi Pharmaceutical (sales) and Toho Holdings (distribution) remains unchanged.
• Vocal Fold Scar (VFS): Phase III placebo-controlled double-blind trial (62 total patients, 31 per arm) is ongoing at 8 sites in Japan; patient enrollment is on track to reach target by December 2025, with final patient follow-up completed by end of 2026 and trial completion in 2027. If ASCI additional trial proceeds as planned, VFS approval submission is expected to occur earlier than ASCI.
• ALS: Investigator-initiated Phase II trial at Tohoku University completed; no statistical significance on primary/secondary endpoints, but progression inhibition was observed in some patients. Additional biomarker analysis is ongoing: biomarker measurement is complete, and data is now being cross-referenced with clinical outcomes for further analysis.

Three-Pillar Growth Strategy

1. Launch two products in Japan: Obtain approval and launch ASCI and VFS assets, own the product intellectual property, and meet Tokyo Exchange listing maintenance requirements. Development costs will be covered by ongoing fundraising.
2. Expand to global markets: All three core indications are rare diseases with 10x larger patient populations outside Japan; HGF is applicable across all ethnicities. Prioritize US development for ASCI: obtained Orphan Drug Designation from FDA, established 100% owned subsidiary Kringle Pharma USA to accelerate development, pursue partnership for development, prioritize co-development over full out-licensing to retain long-term value and increase development probability.
3. Expand indications through academic collaboration: Added 3 new collaborative research projects this period (Peyronie's disease with Kobe University, peripheral neuropathy with Keio University, sustained-release wound healing treatment with Kyoto University), multiple ongoing projects with leading Japanese universities, and promising early data from preclinical animal models.

Key Corporate Updates

• Obtained Orphan Drug Designation for ASCI in both Japan and the US
• Established 100% owned US subsidiary Kringle Pharma USA to accelerate overseas development
• Relocated headquarters to Nakanoshima Qross and expanded headcount to support pipeline growth

Kringle Pharma is a clinical-stage biotech focused on HGF (Oremepermin Alfa) development for intractable diseases, with only one revenue stream in the 2025 September full year: 72 million yen in technology access fee from Clarus Corp. Total operating loss was 909 million yen, ordinary loss 914 million yen, net loss 916 million yen. Total assets stood at 2,079 million yen, net assets 1,309 million yen, cash and cash equivalents decreased by 700 million yen year-over-year to 991 million yen. R&D expenditure was 681 million yen, flat year-over-year, mostly allocated to preparation for acute spinal cord injury (ASCI) approval and vocal fold scar (VFS) trial costs. SG&A increased by 83 million yen year-over-year due to office relocation and increased hiring for pipeline progress.

• For 2026 September full year, revenue guidance only includes confirmed Clarus Corp technology access fee, with no uncertain milestone revenue included
• Total R&D expenditure guidance is 898 million yen, an increase from 2025 full year; 433 million yen of this is allocated to ASCI (domestic additional trial and US preparation costs), with increased spending for VFS commercial manufacturing trial production for approval submission
• Projected operating loss is 1,172 million yen, projected net loss is 1,173 million yen
• Management expects to provide an update on ASCI additional trial initiation by spring 2026 after trial design is finalized with PMDA

• ASCI approval submission is delayed from original 2025 plan due to PMDA requirement for additional trial, creating extended development timelines and increased R&D costs
• The company is operating with consistent net losses, relies on ongoing fundraising to cover development costs, and will need to complete planned fundraising to support current late-stage trial activities
• Partnership negotiations for US development depend on external counterparties, with no guarantee of successful deal completion on favorable terms
• Clinical trial success is not guaranteed: even with enriched patient selection for the ASCI additional trial, there is no certainty of achieving statistically significant efficacy results required for approval
• The company is subject to Tokyo Stock Exchange increasingly strict listing maintenance requirements for the Growth market, and depends on successful domestic product launch to meet these requirements

Q: When will the additional domestic trial for acute spinal cord injury start? / A: Management is currently negotiating trial design details with PMDA, and expects trial design to be finalized by the end of 2025. The company projects it will be able to announce trial initiation by spring 2026. At this stage, trial duration cannot be confirmed as patient enrollment size is still being finalized, and timelines will be shared once confirmed.

Q: What is the design of the additional ASCI trial? / A: The additional trial will follow the same single-arm open-label design as the completed Phase III trial, comparing outcomes to the national spinal cord injury patient database, which PMDA has not objected to. The key change is that patients with extremely severe complete spinal cord injury will be excluded using specific eligibility criteria to concentrate patients more likely to respond to HGF. This approach allows for a smaller trial size and higher probability of success, and the company is currently negotiating the final exclusion criteria with PMDA.

Q: What is the purpose of establishing the US subsidiary? / A: The main purposes are to increase local presence to improve eligibility for US government and patient organization grant funding (which is rarely approved for foreign companies applying from outside the US), and to accelerate business development and partnership negotiation by enabling in-person outreach and eliminating time zone barriers. The subsidiary will also accelerate IND application preparation for US ASCI development. Management prioritizes co-development over full out-licensing, as full out-licensing cedes most long-term value and carries risk of partner development prioritization changes, and the company's existing domestic trial experience increases development success probability for co-development.

Q: Why hasn't Kringle Pharma disclosed the results of Clarus Corp's ongoing corneal disease trials, which are reported to have missed endpoints on ClinicalTrials.gov? / A: Clarus Corp is a private unlisted company conducting three corneal HGF trials in the US, supplied by Kringle Pharma. Kringle Pharma is bound by confidentiality obligations with Clarus, and has an agreement to only disclose results after Clarus makes an official announcement. Clarus has not made a formal public announcement yet, so Kringle cannot release results unilaterally. Management expects Clarus to release an announcement in the near future, and will promptly share a Japanese summary once that occurs.