Worth reading
42Recurring
0–100. This measures how worthwhile something is to read, not how good or bad the company is.Direction
Mixed performance
Direction inferred from the company’s description.Scale
129
¥ million (year-on-year reduction in operating loss)Wording
Conditional
How definitive the wording is.Contents of this document
Earnings presentation materialsThird quarter of the fiscal year ending December 20269 items taken from the company’s own explanation
Financial Results Figures (The key points of this material are explained below)
Net Sales¥0 million
Operating profit¥-1,686 million
Ordinary income¥-1,650 million
Net income¥-1,608 million
Selling, general and administrative expenses¥149 million
What the company explained in this material
Self-assessment against the planThe company's self-assessment against its own plan.
Generally on the planned timelineKey points of the Q&AKey questions raised by investors at the briefing and the company's answers
- The remaining tasks for the IND filing are the acquisition and evaluation of 3-month GLP toxicity study data, completion of release testing for the GMP product, and integration into the filing documents.
- In the additional GLP toxicity study, the pretreatment protocol was optimized, and gene transfer and increased LAMA1 expression were confirmed.
- GMP manufacturing has been completed, and a preliminary assessment indicates that the quantity of drug product required for the planned clinical trial has been secured.
- The target for the IND filing remains late 2026 to the first quarter of 2027, but this is not a commitment because it depends on the GLP toxicity study, quality testing and consultations with regulatory authorities.
- The suspension of exercise of the 19th Series of Stock Acquisition Rights was due not to a funding shortfall, but to considerations including funding efficiency, supply and demand for the shares, and the impact of dilution.
Breakdown of Factors Affecting the ChangeReasons cited by the Company for the change in the figures.
Business expenses: ¥1,686 million; R&D expenses: ¥1,536 million; SG&A expenses: ¥149 million. Year-on-year differences: ¥129 million lower, ¥96 million lower and ¥33 million lower, respectively.
Assumptions Underlying the Business OutlookThe assumptions used by the company in preparing its forecast.
- Acquisition and evaluation of 3-month GLP toxicity study data expected in December 2026
- AAV GMP manufacturing has been completed, and release testing is being conducted
New initiatives
- Obtained a license from Exegenesis Bio for a muscle-targeting capsid for MDL-201
- Consideration of collaboration between GNDM and JUST-AAV® and BBB delivery technology through cooperation with JCR Pharmaceuticals
- Exercise of the 19th Series of Stock Acquisition Rights temporarily suspended through 18 December 2026
Key KPIsA metric the company considers important as a management benchmark.
- Number of employees17 people
- Shares outstanding113,634,098 shares
- Exercise progress of the 19th Series of Stock Acquisition RightsMore than 80%
- Consolidated cash and deposits¥23.7 100 million
Financial targets
Equity ratio
Actual results91.9%
Market Size / Share
- LAMA2-RDMore than $500M
- LAMA2-RDNumber of patients2,500 people in the United States
- DMDNumber of patientsApproximately 200,000 people
- DM1Number of patientsApproximately 90,000 people
- FSHDNumber of patientsApproximately 150,000 people
Number of employees
Number of Employees17 people
| Item | Details |
|---|---|
| Document type | Earnings presentation materials |
| Fiscal Period Covered | Third quarter of the fiscal year ending December 2026 |
| Net Sales | ¥0 million |
| Operating profit | ¥-1,686 million |
| Ordinary income | ¥-1,650 million |
| Net income | ¥-1,608 million |
| Selling, general and administrative expenses | ¥149 million |
| Self-assessment against the plan | Generally on the planned timeline |
| Breakdown of Factors Affecting the Change | Business expenses: ¥1,686 million; R&D expenses: ¥1,536 million; SG&A expenses: ¥149 million. Year-on-year differences: ¥129 million lower, ¥96 million lower and ¥33 million lower, respectively. |
| Key KPIs | Indicator name Number of employees / Value 17 people / Year on year against the same period last year Not disclosed, Indicator name Shares outstanding / Value 113,634,098 shares / Year on year against the same period last year Not disclosed, Indicator name Exercise progress of the 19th Series of Stock Acquisition Rights / Value More than 80% / Year on year against the same period last year Not disclosed, Indicator name Consolidated cash and deposits / Value ¥23.7 100 million / Year on year against the same period last year Not disclosed |
| Number of employees | Number of Employees 17 people / Change during the period Not disclosed / Hiring plan Not disclosed |
| Assumptions Underlying the Business Outlook | Acquisition and evaluation of 3-month GLP toxicity study data expected in December 2026, AAV GMP manufacturing has been completed, and release testing is being conducted |
| Financial targets | Equity ratio Actual results 91.9% / Target Not disclosed |
| Market Size / Share | Subject LAMA2-RD / Market Size More than $500M, Subject LAMA2-RD / Number of patients 2,500 people in the United States, Subject DMD / Number of patients Approximately 200,000 people, Subject DM1 / Number of patients Approximately 90,000 people, Subject FSHD / Number of patients Approximately 150,000 people |
| Key points of the Q&A | The remaining tasks for the IND filing are the acquisition and evaluation of 3-month GLP toxicity study data, completion of release testing for the GMP product, and integration into the filing documents., In the additional GLP toxicity study, the pretreatment protocol was optimized, and gene transfer and increased LAMA1 expression were confirmed., GMP manufacturing has been completed, and a preliminary assessment indicates that the quantity of drug product required for the planned clinical trial has been secured., The target for the IND filing remains late 2026 to the first quarter of 2027, but this is not a commitment because it depends on the GLP toxicity study, quality testing and consultations with regulatory authorities., The suspension of exercise of the 19th Series of Stock Acquisition Rights was due not to a funding shortfall, but to considerations including funding efficiency, supply and demand for the shares, and the impact of dilution. |
| New initiatives | Obtained a license from Exegenesis Bio for a muscle-targeting capsid for MDL-201, Consideration of collaboration between GNDM and JUST-AAV® and BBB delivery technology through cooperation with JCR Pharmaceuticals, Exercise of the 19th Series of Stock Acquisition Rights temporarily suspended through 18 December 2026 |
Items this document does not state (15)
Gross Profit MarginOperating profit marginResults for the three months of the current quarterFull-year earnings forecastProgress against full-year forecastSegment resultsUnit price and quantity metricsNumber of stores and locationsRisk amount incorporated into the outlookMedium-term management plan targetsAnnual DividendPayout ratioAcquisition of treasury sharesCapital expendituresOrders received and order backlog
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.Although development activities centered on MDL-101 continued, business expenses, R&D expenses and SG&A expenses decreased from the same period of the previous year. The company explained that the decrease in R&D expenses reflected its pursuit of a financial strategy, including capital policy and the use of external funding, while continuing development activities for MDL-101.
How to read this page
This is not the documentKomori reads the disclosure the company filed and writes this summary. Figures are as stated by the company and are not audited.
Weight is not a verdictIt measures how much this changes what the company had already told the market. It is not a view on the shares.
Corrections happenIf the company reissues this disclosure, the page is rebuilt from the corrected version and the revision number moves up.