Worth reading
45Recurring
Scored from 5 of 6 sectionsDirection
Mixed performance
Direction inferred from the company’s description.Scale
1000
million yenWording
Avoiding a definitive answer
How definitive the wording is.Contents of this document
Earnings presentationSecond quarter of the fiscal year ending December 20268 items taken from the company’s own explanation
Financial Results Figures (The key points of this material are explained below)
Net Sales0 million yen
Operating profit-1,000 million yen
Ordinary income-965 million yen
Net income-920 million yen
Selling, general and administrative expenses88 million yen
What the company explained in this material
Self-assessment against the planThe company's self-assessment against its own plan.
Progress in line with planKey points of the Q&AKey questions raised by investors at the briefing and the company's answers
- Issues Basis for the clarified timing of the IND filing / Summary of response The timing was clarified through the completion of the mouse IND-enabling studies, confirmation of delivery to muscle tissue and increased LAMA1 expression in additional NHP validation, and the development of a concrete execution plan for the IND filing.
- Issues Additional GLP toxicology studies / Summary of response The CRO has been selected and the contract has been signed, with dosing scheduled to begin during the third quarter of 2026.
- Issues Progress on GMP manufacturing / Summary of response Plasmid GMP manufacturing is underway, and AAV GMP manufacturing is scheduled to begin in the third quarter of 2026.
- Issues First-in-human dosing / Summary of response The company plans to file first in the United States and aims to begin as soon as practicable once preparations are complete following FDA review and clearance.
- Issues Other programs / Summary of response For MDL-201, MDL-202 and MDL-103, the company will continue expanding nonclinical data and establishing product candidates by leveraging external funding, collaborative research and partnering.
Breakdown of Factors Affecting the ChangeReasons cited by the Company for the change in the figures.
- Operating Costs¥1,000 million
- Research and Development Expenses¥911 million
- Selling, general and administrative expenses¥88 million
New initiatives
- Establishment of 240 thousand Series 19 stock acquisition rights (24 million shares)
- Issuance of ¥300 million in straight bonds
- Collaboration on CRISPR-GNDM® and JUST-AAV® through a strategic partnership with JCRファーマ
- Additional GLP toxicology studies for MDL-101, AAV GMP manufacturing and preparation for the IND filing
- SolveFSHD grant program and collaborative research with UMASS
Key KPIsA metric the company considers important as a management benchmark.
- Number of employees21 people
- Shares outstanding97,334,098 Shares
- MDL-101 IND filing targetEnd of 2026 to first quarter of 2027
- Planned number of patients in MDL-101 clinical trialsLow dose N=3, high dose N=3
- LAMA2-CMD market sizeMore than $500M
Financial targets
Equity ratio: 76.9% (actual as of the end of June 2026); target: Not disclosedMarket Size / Share
- LAMA2-CMDMore than $500M
- Example: gene therapy drug market$130 million〜$11B
Number of employees
Number of Employees21 people
| Item | Details |
|---|---|
| Document type | Earnings presentation |
| Fiscal Period Covered | Second quarter of the fiscal year ending December 2026 |
| Net Sales | 0 million yen |
| Operating profit | -1,000 million yen |
| Ordinary income | -965 million yen |
| Net income | -920 million yen |
| Selling, general and administrative expenses | 88 million yen |
| Self-assessment against the plan | Progress in line with plan |
| Breakdown of Factors Affecting the Change | Factors Operating Costs / Amount ¥1,000 million, Factors Research and Development Expenses / Amount ¥911 million, Factors Selling, general and administrative expenses / Amount ¥88 million |
| Key KPIs | Indicator name Number of employees / Value 21 people / Year on year against the same period last year Not disclosed, Indicator name Shares outstanding / Value 97,334,098 Shares / Year on year against the same period last year Not disclosed, Indicator name MDL-101 IND filing target / Value End of 2026 to first quarter of 2027 / Year on year against the same period last year Not disclosed, Indicator name Planned number of patients in MDL-101 clinical trials / Value Low dose N=3, high dose N=3 / Year on year against the same period last year Not disclosed, Indicator name LAMA2-CMD market size / Value More than $500M / Year on year against the same period last year Not disclosed |
| Number of employees | Number of Employees 21 people / Changes during the period and hiring plan Not disclosed |
| Financial targets | Equity ratio: 76.9% (actual as of the end of June 2026); target: Not disclosed |
| Market Size / Share | Subject LAMA2-CMD / Market Size More than $500M, Subject Example: gene therapy drug market / Market Size $130 million〜$11B |
| Key points of the Q&A | Issues Basis for the clarified timing of the IND filing / Summary of response The timing was clarified through the completion of the mouse IND-enabling studies, confirmation of delivery to muscle tissue and increased LAMA1 expression in additional NHP validation, and the development of a concrete execution plan for the IND filing., Issues Additional GLP toxicology studies / Summary of response The CRO has been selected and the contract has been signed, with dosing scheduled to begin during the third quarter of 2026., Issues Progress on GMP manufacturing / Summary of response Plasmid GMP manufacturing is underway, and AAV GMP manufacturing is scheduled to begin in the third quarter of 2026., Issues First-in-human dosing / Summary of response The company plans to file first in the United States and aims to begin as soon as practicable once preparations are complete following FDA review and clearance., Issues Other programs / Summary of response For MDL-201, MDL-202 and MDL-103, the company will continue expanding nonclinical data and establishing product candidates by leveraging external funding, collaborative research and partnering. |
| New initiatives | Establishment of 240 thousand Series 19 stock acquisition rights (24 million shares), Issuance of ¥300 million in straight bonds, Collaboration on CRISPR-GNDM® and JUST-AAV® through a strategic partnership with JCRファーマ, Additional GLP toxicology studies for MDL-101, AAV GMP manufacturing and preparation for the IND filing, SolveFSHD grant program and collaborative research with UMASS |
Items this document does not state (16)
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 locationsAssumptions 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.Expenses related to activities toward clinical trials for MDL-101 were the primary component, resulting in ¥1,000 million in business expenses. While continuing research and development investment as development activities advance, the company is pursuing a financial strategy that includes capital policy and the use of external funding. The company states that the completion of the mouse studies, data obtained from additional NHP validation, and the development of an execution plan for the IND filing, including GLP toxicology studies and GMP manufacturing, have clarified the outlook for the timing of the IND filing.
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.