Modalis Therapeutics Reports Operational Highlights andFirst Quarter 2024 Financial Results
08-May-2024 TOKYO & Waltham, Mass.- Modalis Therapeutics Corporation ("Modalis" 4883.T TSE),a pioneer in developing cutting-edge solutions for rare genetic diseases through its proprietary
CRISPR-GNDM® epigenetic gene modification technology, today announced its financial results forthe first quarter ended March 31, 2024, alongside recent operational milestones.
“In 2023, Modalis announced the prioritization of its pipeline programs that support our focus as aneuromuscular-focused epigenome editing company,” said Haru Morita, Chief Executive Officer ofModalis. “Modalis further clarified our focus program and shifted to a development centered on
MDL-101. We continued to advance its pre-clinical pipelines this quarter and our MDL-101 program
has completed process development, which is one of the biggest hurdles for a gene therapy program
before entering clinical trial, and as a result has reached a level of productivity, yield, and quality thatmakes it feasible to proceed to clinical trials in the production of engineered capsid-based AAV
molecules, alongside preparations for the phase 1/2 clinical trial expected to commence next year. In
the current environment, careful choices must be made when deciding how many programs to pursue
at any one time. To that end, we are pleased to announce that we are narrowing our strategic focus
and prioritizing our investments in the most promising programs. To this end, we have made thedifficult but necessary decision to pull back from some of our preclinical assets, downsize some of
our infrastructure, and reallocate our investments to maximize the potential of the programs webelieve have the most value.”
Recent Preclinical and Business Highlightsl

MDL-101 is advancing to INDØØ

l

MDL-202 for DM1ØØl

Disease model mice study and analysis of pilot NHP study ongoing.
In alignment with the announced strategic transformation, Modalis slows down the development of non-coreprograms including the DM1 program and continues to seek a potential collaboration partner or externalinvestment.

Research collaboration
ØØlEstablished manufacturing process. Achieved reasonable productivity, yield, and quality that can reach clinicaltrials. Further development of analytical methods for the product.
At the same time, discussions were held on establishing protocols for conducting clinical trials, networking withpatient groups, and building assay systems.

Began collaboration with Company JCR Pharma in the area of CNS, validating the approach to CNS targets bycombining BBB trans-passing capsid, JBC-AAV, and our CRISPR-GNDM technology.
In April, we entered into a partnership agreement with Ginkgo Bioworks. to combine Ginkgo's synthetic biology
technology and biofoundry capabilities with our epigenome editing technology and to develop crosspromotion.

Conference and presentation
ØØ4th Annual Next Generation Gene Therapy Vectors Summit (June 12-14)
Cell and Gene Therapy Summit 2024 (July 8-10)

First Year 2024 Financial Results:•••••

Cash Position: Cash and deposits as of December 31, 2024, was ¥1,413 million, compared to¥1,883 million as of December 31, 2023, a decrease of ¥470 million. The decrease in Cashand deposits was primarily due to R&D Expenses and G&A Expenses.
Research & Development (R&D) Expenses: R&D expenses were ¥422 million for the three
ended March 31, 2024, compared to ¥443 million for March 31, 2023, a decrease of ¥20
million. Decreases in R&D expenses were primarily due to decreases in development costs
associated with the advancement of the Company’s proprietary GNDM platform andproduct candidates.
General & Administrative (G&A) Expenses: G&A expenses were ¥67 million for the three
ended March 31, 2024, compared to ¥71 million for March 31, 2023, a Decrease of ¥4million. Decreases in G&A expenses were primarily due to Decreased personnel costs.
Net Loss: Net Loss was ¥457 million for the three ended March 31, 2024, compared to ¥532
million for March 31, 2023.
Please refer to 1Q Consolidated Financial Results disclosed according to Japanese accounting
rules in the English version below.

About Modalis:Modalis Therapeutics is developing precision genetic medicines through epigenome editing. Modalis
is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology
which enables the locus-specific modulation of gene expression or histone modification without theneed for double-stranded DNA cleavage, gene editing, or base editing. Modalis focuses primarily ongenetic disorders caused by loss of gene regulation – resulting in excess or insufficient protein
production – which includes more than 660 genes that are currently estimated to cause humandisease due to haploinsufficiency. Headquartered in Tokyo with laboratories and facilities inWaltham, Massachusetts. For additional information, visit www.modalistx.com.

Forward-Looking Statements:
This document has been prepared by Modalis Therapeutics corporation and Modalis Therapeutics
Inc. (the “Companies”) solely for information purpose only. This document does not constitute orform part of and should not be construed as, an offer to sell or issue or the solicitation of an offer tobuy or acquire securities of the Companies in Japan, the United States or any other jurisdictions. The
information contained herein is based on current economic, regulatory, market trends and otherconditions. The Companies make no representation or guarantee with respect to the credibility,accuracy or completeness of the information herein. The information contained herein may change
without prior notice. You may not publish or use this document and the contents thereof for any
other purpose without a prior written consent of the Companies. Furthermore, the information onfuture business results are forward-looking statements. Forward-looking statements include but notlimited to expressions such as "believe", “expect", "plan", "strategic", "expect", "anticipate","predict" and "possibility", as well as other similar expressions to explain future business activities,achievements, events and future conditions. Forward-looking statements are predictions about thefuture that reflect management's judgment based on currently available information. As such, theseforward-looking statements are subject to various risks and uncertainties that could cause actual
results to differ materially from those expressed in or suggested by the forward-looking statements.
Therefore, you may not rely entirely on forward-looking statements. The Companies do not assume
any obligation to change or correct any forward-looking statements in light of new information,
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Contacts

Modalis Therapeutics
Sawako Nakamura
media@modalistx.com

Consolidated Financial Results
for the Three Months Ended March 31, 2024
[Japanese GAAP]May 8, 2024
Company name: Modalis Therapeutics Corporation
Stock exchange listing: Tokyo Stock Exchange
Code number: 4883
URL: https://www.modalistx.com/en/
Representative: Haruhiko Morita, CEO and Representative Director
Contact: Naoki Kobayashi, CFO and Executive Officer
Phone: +81-3-6822-4584
Scheduled date of filing quarterly securities report: May 14, 2024
Scheduled date of commencing dividend payments: Availability of supplementary briefing material on quarterly financial results: Available
Schedule of quarterly financial results briefing session: Scheduled (for individual investors)(Amounts of less than one million yen are rounded down.)
1. Consolidated Financial Results for the Three Months Ended March 31, 2024 (January 1, 2024
to March 31, 2024)(1) Consolidated Operating Results (% indicates changes from the previous corresponding period.)
attributable toOperating revenue Operating income Ordinary income Profit
owners of parent
Three months
Million yen%
Million yen%
Million yen%
Million yen%ended
March 31, 2024
(490)
(457)
(457)
March 31, 2023
- (100)
(514)
(509)
(532)
(Note)Comprehensive income:Three months ended March 31, 2024: ¥ (458) million [- %]Three months ended March 31, 2023: ¥ (531) million [- %]Basic earnings
per share

Diluted earnings
per share

Three months
YenYenended
March 31, 2024
(13.57)
March 31, 2023
(17.86)(Notes)
The amount shown as “business revenue” in the consolidated statements of income has been shownas ”Operating revenue” in this document.
Diluted net income per share is not shown in the above table, because net income per share was negative
although there are residual shares.
(2) Consolidated Financial Position
Total assets
Million yen

Net assets
Million yen

As of March 31, 2024
1,559
1,011
As of March 31, 2023
2,025
1,380
(Reference)
Equity:As of March 31, 2024: ¥981 million
As of March 31, 2023: ¥1,353 million

Capital adequacy
ratio%

62.9
66.8