Worth reading
55In focus
Scored from 5 of 6 sectionsDirection
Mixed performance
Direction inferred from the company’s description.Scale
290
¥ millions (improvement in operating loss)Wording
Avoiding a definitive answer
How definitive the wording is.Contents of this document
Presentation materials for financial resultsFirst quarter of the fiscal year ending December 20266 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¥-342 million
Ordinary income¥-322 million
Net income¥-323 million
Selling, general and administrative expenses¥48 million
Results for the three months of the current quarter
Business profitability
¥0 million
Operating Costs
¥342 million
Research and development costs
¥293 million
Selling, general and administrative expenses
¥48 million
Operating profit
¥-342 million
Ordinary income
¥-322 million
Net income for the current fiscal year
¥-323 million
What the company explained in this material
Key points of the Q&AKey questions raised by investors at the briefing and the company's answers
- Questions Background to the review of the MDL-101 IND timeline / Answer The company has not identified any significant new safety signals, but is reassessing its development plan, including CMC, additional assessments, analytical platform development, and clinical trial preparations, to optimize these activities and increase the probability of success after entering clinical development.
- Questions Key priorities for advancing into clinical development / Answer Analytical characterization, manufacturing and quality control, long-term expression profiles, translational assessments, and optimization of dose and dosing strategy.
- Questions Ability to continue development at the current cash level / Answer As of the end of March 2026, the company had secured more than ¥2.8 billion in cash and cash equivalents and maintained the financial foundation required for its core development activities.
- Questions MDL-202's competitive advantages / Answer MDL-202 demonstrated greater DMPK suppression than benchmark drugs in disease models, and high suppression efficacy was also confirmed in cardiac muscle and the diaphragm.
- Questions Differentiating factors amid intensifying competition / Answer Knowledge of CRISPR-GNDM centered on gene activation, muscle-targeted delivery, and the development of systemically administered AAVs.
Breakdown of Factors Affecting the ChangeReasons cited by the Company for the change in the figures.
Operating Costs
¥-290 million
Research and development costs
¥-278 million
Selling, general and administrative expenses
¥-13 million
Improvement in operating income
¥290 million
Improvement in ordinary income
¥329 million
Improvement in net income for the period
¥329 million
New initiatives
- Optimization of MDL-101's manufacturing process and quality assessment systems
- Detailed assessment of long-term expression profiles
- Optimization of dosing conditions and immunosuppression protocols
- Preparations to advance into clinical development, including the selection of clinical trial sites and principal investigators
- Consideration of diversifying funding sources, including grants, collaborative research, and partnering
Key KPIsA metric the company considers important as a management benchmark.
- DMPK mRNA knockdown rate (GNDM2-MyoAAV, heart)87 %
- DMPK mRNA knockdown rate (GNDM2-MyoAAV, diaphragm)52 %
- DMPK mRNA knockdown rate (GNDM2-MyoAAV, quadriceps)60 %
- DMPK mRNA knockdown rate (GNDM2-MyoAAV, tibialis anterior)63 %
- DMPK mRNA knockdown rate (GNDM2-MyoAAV, gastrocnemius)49 %
- Suppression rate of DUX4 downstream gene expression70〜80
Financial targets
Equity ratio
Actual results93.8%
Market Size / Share
- LAMA2-CMDMore than $500M
- DM1$2.2B (2032 forecast)
- FSHDMore than $500M (2022)
| Item | Details |
|---|---|
| Document type | Presentation materials for financial results |
| Fiscal Period Covered | First quarter of the fiscal year ending December 2026 |
| Net Sales | ¥0 million |
| Operating profit | ¥-342 million |
| Ordinary income | ¥-322 million |
| Net income | ¥-323 million |
| Selling, general and administrative expenses | ¥48 million |
| Results for the three months of the current quarter | Business profitability ¥0 million / Operating Costs ¥342 million / Research and development costs ¥293 million / Selling, general and administrative expenses ¥48 million / Operating profit ¥-342 million / Ordinary income ¥-322 million / Net income for the current fiscal year ¥-323 million |
| Breakdown of Factors Affecting the Change | Operating Costs ¥-290 million / Research and development costs ¥-278 million / Selling, general and administrative expenses ¥-13 million / Improvement in operating income ¥290 million / Improvement in ordinary income ¥329 million / Improvement in net income for the period ¥329 million |
| Key KPIs | Indicator name DMPK mRNA knockdown rate (GNDM2-MyoAAV, heart) / Value 87 % / Year on year against the same period last year Not disclosed, Indicator name DMPK mRNA knockdown rate (GNDM2-MyoAAV, diaphragm) / Value 52 % / Year on year against the same period last year Not disclosed, Indicator name DMPK mRNA knockdown rate (GNDM2-MyoAAV, quadriceps) / Value 60 % / Year on year against the same period last year Not disclosed, Indicator name DMPK mRNA knockdown rate (GNDM2-MyoAAV, tibialis anterior) / Value 63 % / Year on year against the same period last year Not disclosed, Indicator name DMPK mRNA knockdown rate (GNDM2-MyoAAV, gastrocnemius) / Value 49 % / Year on year against the same period last year Not disclosed, Indicator name Suppression rate of DUX4 downstream gene expression / Value 70〜80 / Year on year against the same period last year Not disclosed |
| Financial targets | Equity ratio Actual results 93.8% / Target Not disclosed |
| Market Size / Share | Subject LAMA2-CMD / Market Size More than $500M, Subject DM1 / Market Size $2.2B (2032 forecast), Subject FSHD / Market Size More than $500M (2022) |
| Key points of the Q&A | Questions Background to the review of the MDL-101 IND timeline / Answer The company has not identified any significant new safety signals, but is reassessing its development plan, including CMC, additional assessments, analytical platform development, and clinical trial preparations, to optimize these activities and increase the probability of success after entering clinical development., Questions Key priorities for advancing into clinical development / Answer Analytical characterization, manufacturing and quality control, long-term expression profiles, translational assessments, and optimization of dose and dosing strategy., Questions Ability to continue development at the current cash level / Answer As of the end of March 2026, the company had secured more than ¥2.8 billion in cash and cash equivalents and maintained the financial foundation required for its core development activities., Questions MDL-202's competitive advantages / Answer MDL-202 demonstrated greater DMPK suppression than benchmark drugs in disease models, and high suppression efficacy was also confirmed in cardiac muscle and the diaphragm., Questions Differentiating factors amid intensifying competition / Answer Knowledge of CRISPR-GNDM centered on gene activation, muscle-targeted delivery, and the development of systemically administered AAVs. |
| New initiatives | Optimization of MDL-101's manufacturing process and quality assessment systems, Detailed assessment of long-term expression profiles, Optimization of dosing conditions and immunosuppression protocols, Preparations to advance into clinical development, including the selection of clinical trial sites and principal investigators, Consideration of diversifying funding sources, including grants, collaborative research, and partnering |
Items this document does not state (17)
Gross Profit MarginOperating profit marginFull-year earnings forecastProgress against full-year forecastSelf-assessment against the planSegment resultsUnit price and quantity metricsNumber of stores and locationsNumber of employeesAssumptions Underlying the Business OutlookRisk 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.While continuing development activities centered on MDL-101, R&D expenses decreased from ¥571 million in the same period of the previous year to ¥293 million, and SG&A expenses decreased from ¥61 million to ¥48 million. For MDL-101, the company is optimizing its development plan, including CMC, additional assessments, analytical platform development, and clinical trial preparations, with the aim of maximizing the probability of success after entering clinical development.
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.