Kringle Pharma, Inc.

Business Details

A single-segment company engaged in the research and development of recombinant human HGF protein (development code: KP-100, international nonproprietary name: Oremepermin Alfa) for intractable diseases. The company holds a domestic clinical pipeline for acute spinal cord injury, vocal fold scarring, and ALS, and its basic policy is to file for manufacturing and marketing approval on its own. At present, revenue consists solely of technology access fees (royalty income) from Claris Biotherapeutics, Inc. in the United States, and the loss-making structure in which R&D expenses substantially exceed revenue continues.

Recent Overview

H1 cumulative net sales of ¥36 million and operating loss of ¥515 million; funds secured through a surge in advance payments and exercise of stock acquisition rights

For the H1 cumulative period of FY2026 (ending September 2026) (October 2025 to March 2026), net sales were ¥36 million (up 2.2% year on year), operating loss was ¥515 million, and net loss for the interim period was ¥517 million. The company received an advance payment of ¥232 million associated with an order for HGF drug substance from Claris Biotherapeutics, resulting in a substantial increase in current liabilities (advance payment balance of ¥298 million). Exercise of the 16th series of stock acquisition rights increased common stock and capital surplus by ¥70 million and ¥80 million, respectively. Cash on hand stood at ¥960 million (of which ¥628 million is restricted as collateral for AMED). As a subsequent event, 6,830 units of stock acquisition rights were exercised between April and May 2026, raising ¥284 million. There is no change to the full-year earnings forecast.

Key Products

Growth Drivers

• Enrollment of the final case in the Phase III trial for vocal fold scarring (VFS) was completed in January 2026, advancing to the next step toward an approval filing
• Planning an additional clinical trial for acute spinal cord injury, aiming for an approval filing after obtaining additional efficacy data
• Obtained Orphan Drug Designation from the FDA for acute spinal cord injury in June 2025, establishing a foundation for development in the United States
• Established U.S. subsidiary Kringle Pharma USA, Inc. in November 2025 to accelerate development in the United States
• Fundraising through exercise of the 16th series of stock acquisition rights (issued August 2025) is underway (¥284 million raised including subsequent events)
• Public funding support (vocal fold scarring project) through AMED's CiCLE program
• Building a global supply framework and improving manufacturing process efficiency through the business alliance with Claris Biotherapeutics
• Full-year net sales forecast of ¥332 million (up 360% year on year) driven by an order for HGF drug substance from Claris Biotherapeutics (advance payment of ¥232 million)
• Expansion of the intellectual property portfolio, including a jointly filed patent application in December 2025 for a novel therapeutic agent for fibrotic diseases
• Expansion of pipeline indications through new joint research agreements concluded with Kyoto University, Kobe University, Keio University, and others

Risks

• Material doubt about the company's ability to continue as a going concern exists (continued operating losses and cash outflows, delays in the timing of approval filings)
• Risk that the additional clinical trial for acute spinal cord injury will further delay the approval filing and monetization
• Risk of deteriorating cash flow if exercise of stock acquisition rights does not proceed as planned (cash on hand of ¥960 million, of which ¥628 million is restricted as collateral for AMED)
• Risk of development discontinuation due to clinical trial failure or difficulties in discussions with regulatory authorities
• Revenue concentration risk, as essentially 100% of net sales depends on technology access fees and drug substance supply to a single counterparty, Claris Biotherapeutics
• No statistically significant difference was obtained in the primary endpoint of the ALS Phase II trial, and the design and funding of the next-phase trial remain uncertain
• Share dilution risk (continued issuance and exercise of stock acquisition rights has increased the number of shares outstanding to 7,395,200, with a further 683,000 shares to be issued as a subsequent event)
• The equity ratio has sharply declined from 61.5% (end of FY2025 (ended September 2025)) to 45.9% (end of Q2), indicating a weakening financial base
• Partnerships with pharmaceutical companies for vocal fold scarring (VFS), ALS, and overseas spinal cord injury remain undetermined, concentrating development and financial risk on the company