May 2026
【 HGF Gene Therapy Product 】：Advancing BLA Preparation in the US
US：Clinical trials have been completed, and preparation for the Biologics License Application (BLA) is underway in consultation with the FDA.
Type B Clinical Meeting with the FDA was conducted.→Agreement was reached on the regulatory strategy related to clinical matters.
JP：Future actions are under consideration based on progress in the US
【NF-κB Decoy Oligonucleotide DNA】：P2Clinical Trial Ongoing in Japan Results from the P1 clinical trial in the US were published in The SPINE Journal, the official journal of the North American Spine Society.
In Japan, patient enrollment for the Phase II clinical trial is progressing.
【Tie2 Receptor Agonist】：P2 Clinical Trial for ARDS Ongoing in the US
In the P2 clinical trial in the United States, planned patient enrollment has been completed.
→ Additional enrollment is ongoing to compensate for drop-out cases.
The first patient dosing has been completed in an investigator-initiated trial evaluating improvement of cognitive impairment in hemodialysis patients.
【Zokinvy】：Sales Progressing Largely in Line with Plan
2
【ACRL】：Order Intake for Expanded Newborn Screening Remains Stable
・Expanded Newborn Screening
The number of target diseases has expanded from 9 to 14, and order intake remains stable, operating at near full capacity.
・Establishment of a Comprehensive Testing Framework
A comprehensive testing system has been developed, covering processes from diagnosis to treatment of rare genetic diseases.
The scope of genetic testing target diseases has been expanded
→In addition to HGPS and PDPL, seven diseases included in expanded newborn screening have been newly added.
Research on the use of biomarker testing as secondary screening has been published in
a scientific journal.
【EmendoBio】：Expansion of Research Outcomes on OMNI Nuclease to the US
Efforts are underway to establish an operational framework to promote research and development activities and academic publications in the US.
Joint research with Stanford University is being advanced on novel cancer genome therapies. Consideration is being given to establishing new research and development sites in the US.
3
Business revenue increased, while operating expenses rose due to HGF application preparation costs
・Expanded newborn screening testing at ACRL totaled 124 millionYen (YoY+9..7%)
・Zokinvy sales amounted to 78 millionYen
（YoY+42.4%）
Revenue（YoY+20.4％）
・Cost of Sales: 121 millionYen (YoY +8 millionYen) Increase mainly due to higher cost of sales for Zokinvy
・R&D Expenses: 1,007 millionYen (YoY+217 millionY) Increase primarily attributable to higher costs related to HGF application preparation
・SGA Expenses: 571 millionYen (YoY+49millionYen) Increase mainly due to higher taxes and dues at EmendoBio
Operating Expenses（YoY+19.3%）
（Unit：Million Yen）
FY2025-1Q | FY2026-1Q | Change | |
Revenue | 169 | 203 | 34 |
Operating Expenses | 1,424 | 1,700 | 275 |
Operating Profit(Loss) | -1,255 | -1,496 | -241 |
Non Operating Income(Expenses) | 17 | 386 | 368 |
Ordinary Profit(Loss) | -1,237 | -1,110 | 127 |
Extraordinary Income(Expenses) | 0 | 0 | 0 |
Net Profit(Loss) | -1,247 | -1,082 | 165 |
・Foreign exchange gain of 385 millionYen from the revaluation of a loan to EmendoBio.
Non Operating Income（YoY368millionYen）
4
（Unit：Million Yen）
Dec-2025 | March-2026 | Change | Curent Assets | ||
・414 millionYen was raised through the issuance of the 46th series of stock acquisition rights. ・Current assets decreased by 1,018 millionYen | |||||
Current Assets | 4,386 | 3,367 | -1,018 | ||
due to expenditures such as operating expenses. | |||||
Cash and Deposits | 1,882 | 835 | -1,046 | ||
Non-Current Assets | 1,019 | 1,104 | 84 | ||
Total Assets | 5,405 | 4,472 | -933 | ||
Liabilities | 2,329 | 2,448 | 118 | ||
Net Assets | 3,076 | 2,023 | -1,052 | ||
Net Assets
・As a result of the exercise of the 46th series of stock acquisition rights, capital stock and capital surplus each increased by 211 millionYen.
・Net assets decreased by 1,052 millionYen due to the quarterly net loss.
5