– Corporate Presentation April 2026

WE CARE

FOR THE RARE

Corporate presentation

April 2026

Emcitate® (tiratricol) launched in Germany in May 2025 FDA PDUFA date September 28, 2026

For investors and analysts only

©2026 Egetis Therapeutics. All rights reserved. 1

WE CARE

FOR THE RARE

1

Focus on Emcitate® (tiratricol) for the treatment of MCT8 deficiency

Strong regulatory status

BTD

Breakthrough Therapy Designation - FDA

ODD Orphan Drug Designation - EMA & FDA

Market exclusivity 10y (EU) & 7y (US)

Fast

track

Fast track designation - FDA

Rare pediatric disease designation - FDA

PRV Priority Review Voucher upon approval

Supplied to over 230 patients in over 25 countries including US, EU

2

The first and only approved drug for the treatment of MCT8 deficiency Approved in EU in Feb 2025. Launched in Germany May 2025

3

FDA accepted NDA and granted Priority Review on March 27, 2026 PDUFA date September 28, 2026

A significant market opportunity & potential for expansion into RTH-beta

4

Launch through focused in-house commercial organization in EU and US with

partnership for RoW

(Japan: Fujimoto; Türkiye, Central-, Eastern-, Southeastern Europe: Er-Kim; Gulf region: taiba rare)

5

A strong team with late-stage orphan clinical development, registration and commercialization experience

Listed on NASDAQ Stockholm (EGTX)

HQ in Stockholm, Sweden

~50 FTEs

4

© 2026 Egetis Therapeutics. All rights reserved.

Positive Phase 3 ReTRIACt withdrawal study topline readout

Feb 2025

EC approves Emcitate

as first and only treatment for MCT8 deficiency in EU

Rolling NDA initiated for MCT8 deficiency

Emcitate granted a rolling NDA review by FDA

• PDUFA date September 28

• Priority Review Voucher (upon approval)

EU Launch in first country Germany

• Japan - Development plan alignment with PMDA

• Completion of NDA submission

• Acceptance of NDA

• Priority Review

July 2025

FDA Breakthrough Therapy Designation

Expected launch of

Emcitate in the U.S.

Q4 2026

MCT8 deficiency key features

Estimated incidence: 1 per 70k male births Median onset of symptoms: 4 months Median age of diagnosis: 10 months Median life expectancy: 35 years Patients dying in childhood: ~30%

Main cause of mortality: Sudden cardiac death

Severe underweight:

Cardiac arrhythmias (PAC): Hypotonia, hypertonia

75%

76%

& persistence of primitive reflexes: 90%

Severe intellectual disability:

Ability to sit independently:

Life long 24-hour care:

100%

8%

100%

Median life expectancy of 35 years with 30% of patients dying in childhood

6

© 2026 Egetis Therapeutics. All rights reserved.

Tiratricol in action

Tiratricol

TR

T3

Without a

functioning MCT8, T3 is unable to be transported across the cell

membrane to enter the target cell

MCT8

Tiratricol

Tiratricol

with clear scientific and mechanistic rationale

• Tiratricol is a small molecule, thyroid hormone agonist

• Unlike T3, tiratricol can cross cellular membranes without a functional MCT8 transporter

• Tiratricol can bypass the problem in patients with MCT8 deficiency, enter MCT8 deficient cells and restore thyroid hormone signalling

Cellular membrane

7

© 2026 Egetis Therapeutics. All rights reserved.

Tiratricol has been shown to be associated with a 3x lower risk of mortality in patients with MCT8 deficiency

Retrospective real-world cohort study in >300 patients - Abstracts Aug. 2024 & May 2025

In the Triac Trial I, tiratricol reached target level serum T3 & improvements in clinically relevant outcome measures

T0 (Baseline)

T12 (12 month)

p < 0.0001

10

Key demonstrated clinical results

• Significant and durable reduction of T3 levels within the normal range

• Normalization of thyrotoxicosis in patients of all ages

• Statistically & clinically significant effects on key disease parameters such as cardiovascular health and bodyweight

• Beneficial effects are maintained or continue to improve over time, up to six years

• Benign safety profile

8

Serum T3 (nmol/l)

6

4

2 Normal

range

0

Source: Groeneweg et al; Lancet D&E 2019 8

© 2026 Egetis Therapeutics. All rights reserved.

Robust dataset in an ultra-rare genetic disease; all FDA-required studies completed

Data included in NDA

©2026 Egetis Therapeutics. All rights reserved. G

Tiratricol's Effect on T3 Lowering May Explain Mortality Benefit

EMC Survival Study

Source for evidence of efficacy

system

Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort

Study

Triac Trial I, EMC Cohort

Study

RPP Triac Trial I, ReTRIACt

©2026 Egetis Therapeutics. All rights reserved. 10

Executing the US & European market preparations and launches through the Egetis team

To optimize the launch, we will focus our own resources on US and Europe

Japan license deal with Fujimoto

Optimizing additional

countries through partners

Türkiye, Central-, Eastern- &

Southeastern Europe with Er-Kim Gulf region with taiba rare

11

©2026 Egetis Therapeutics. All rights reserved.

ETA recommends the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with RTHβ

Inaugural 2024 Guidelines were commissioned by the Executive Committee of the ETA and developed by an independent team of experts. Authors include well-known

U.S. KOL

Potential to expand to U.S. guidelines post-FDA approval

©2026 Egetis Therapeutics. All rights reserved. 12

Launch execution in 2 waves, starting with EU4

Wave 1

Germany, France, Italy, Spain & countries with

alternative funding pathways

Wave 2

Phased on a country-by-country approach

Rest of Europe

Pricing & Reimbursement processes

*European Thyroid Association guidelines published 2024

13

© 2026 Egetis Therapeutics. All rights reserved.

Building on positive 2025 momentum to optimize access and sales opportunities further

• Strong close to 2025 - Full-year sales MSEK 62.3, +40% vs. 2024 (CER)

• European pricing & reimbursement (P&R) advancement

  ‒ Germany: Progressing well; final reimbursed price in Q2 2026

  ‒ France: Preparing strengthened dossier for resubmission

  ‒ Spain: Local data generation progressing

  ‒ Italy: Regional launch; national P&R process later in the year

  ‒ Rest of Europe: Move towards funded access routes in more

  countries

• Distribution partnerships - Q4

  ‒ taiba rare: Named patient access in the Gulf region

  ‒ Er-Kim: Expanded from Türkiye to Central, Eastern &

  Southeastern Europe

  ‒ First order received for Türkiye

  
  
  

  ©2026 Egetis Therapeutics. All rights reserved.

  United States: Egetis' Priority Market

  From Regulatory Milestone to Commercial Momentum

  
  
  
  
  

  Driving Patient Identification Growth

  
  
  

• Deep engagement in key referral centers

• Strong KOL and advocacy alignment

• Growing identified patient pool (>140)

  Expanded Access Strengthening Launch Readiness

• 17 active Expanded Access specialist sites

• Real-world physician experience established

• Active transition planning from EAP to commercial supply

  Solidifying Market Access Pathway

• Payer expectations validated

• Pricing and value strategy refined

• Integrated specialty pharmacy & patient services implementation underway

  Purpose-Built Rare Disease Organization

• Experienced rare disease leadership in place

• Focused, expert field model deployed

• Built for scalability beyond MCT8 deficiency

  ©2026 Egetis Therapeutics. All rights reserved.

  US: Annual Treatment Costs and Strength of Evidence

  Representative analogues

  Product Disease Estimated avg. annual

  treatment cost (WAC)

  Impact of strength of evidence on price

  Oxlumo®

  Biologic

  Strensiq®

  Biologic

  Brineura®

  Biologic

  Miplyffa®

  Small molecule

  Zokinvy®

  Small molecule

  Primary hyperoxaluria type 1

  Hypophosphatasia

  Ceroid lipofuscinosis type 1

  Niemann-Pick type C

  HGPS

  ~$623K

  ~$683K

  ~$917K

  ~$967K

  ~$1,120K

  • Morbidity-driven disease burden, supported by survival data and/or surrogate endpoints based on objective, quantifiable measurements

  • Mortality-driven disease burden, but surrogate endpoints and/or perceptions of modest efficacy improvements

  • Mortality-driven disease burden based on reductions in mortality specified within the labeled indication

HGPS: Hutchinson-Gilford Progeria Syndrome

16

©2026 Egetis Therapeutics. All rights reserved.

Potential indication expansion for Emcitate into larger non-overlapping patient population

Characteristics of RTH-β

• Caused by mutations in thyroid hormone receptor beta

  (TRβ)1

• Reduced target tissue response to thyroid hormone in TRβ

  dependent tissues

• Incidence 1:20,000 to 1:40,000 (both genders)

  
  
  

  Overview of tissues affected in RTH-β

  Emcitate as potential treatment for RTH-β

• Emcitate efficacious in restoring signaling in majority of

  TRβ mutations in vitro

• Initial clinical experience demonstrates positive effects on key clinical symptoms in RTH-β patients, including cardiovascular, thyrotoxic and neuropsychiatric symptoms2

• Emcitate received orphan drug designation for RTH-β from

  FDA and EMA in 2022

• Use of Emcitate recommended by European Thyroid Association for certain patients with resistance to RTH-β in 20243

References:

1. Pappa C Refetoff (2021) Front. Endocrinol. 12, 656551

2. Moran et al. (2025) The Journal of Clinical Endocrinology C Metabolism, 2025, 00, 1-8

3. Persani et al. (2024) European Thyroid Journal 13, 4

©2026 Egetis Therapeutics. All rights reserved. 17

Solid cash position

• Cash position December 31, 2025: SEK 216 million

• Number of outstanding shares: 395,161,938

• Market Cap: ~SEK 1.9 billion* (~USD 205 million)

• Listing venue: Nasdaq Stockholm, Main Market; Ticker: EGTX

Largest shareholders

Note: * March 3, 2026

Directed share issue Oct. 2025 of SEK 183m (USD 19m)

• Swedish investors: Fjärde AP-fonden, Cidro Förvaltning (Peter Lindell), Linc & others

US biotech investors: Frazier Life Sciences, Invus, Petrichor &

Woodline

Oversubscribed with participation from new & existing investors

•

•

18

© 2026 Egetis Therapeutics. All rights reserved.

• Total revenue

  ‒ FY-2025 of 62.4 MSEK vs. 46.1 MSEK for 2024, +40% YoY CER

  ‒ Q4-2025 of 17.9 MSEK vs. 10.8 MSEK for Q4-2024, +74% YoY CER

  
  
  

  MSEK	2025 Oct-Dec	2024 Oct-Dec	2025 Jan-Dec	2024 Jan-Dec
  Revenue Gross Profit Operating result Results after tax	17.9 4.2 -120.3 -119.4	10.8 8.5 -104.7 -110.5	62.4 12.4 -339.9 -342.5	46.1 34.5 -329.4 -343.6
  Cash flow from operations Cash position	-92.9 215.8	-53.6 351.0	-267.0 215.8	-227.9 351.0

• Cost of goods sold impacted by non-recurring milestone and the initiation of intangible R&D depreciation.

  ‒ During the 12-months, non-recurring milestone payment of 3.5 MSEK to Erasmus Medical Center and R&D depreciation of 33.7 MSEK have impacted cost of goods

  ‒ Excluding these items Gross profit would have been 49.6 MSEK vs 34.5 MSEK for the 12-months period 2024, corresponding to an adj. gross margin of 79.5% vs. 74.8% 2024.

• Results after tax in FY-2025 amounted to -343.5 MSEK vs. -343.6 MSEK for FY-2024.

• The cash position per end of December 2025 was 216 MSEK vs. 351 MSEK per end of December 2024.

• October 2nd, Egetis Therapeutics successfully carried out an oversubscribed directed share issue amounting to 183 MSEK.

19

© 2026 Egetis Therapeutics. All rights reserved.

Positive Phase 3 ReTRIACt withdrawal study topline readout

Feb 2025

EC approves Emcitate

as first and only treatment for MCT8 deficiency in EU

Rolling NDA initiated for MCT8 deficiency

Emcitate granted a rolling NDA review by FDA

• PDUFA date September 28

• Priority Review Voucher (upon approval)

EU Launch in first country Germany

• Japan - Development plan alignment with PMDA

• Completion of NDA submission

• Acceptance of NDA

• Priority Review

July 2025

FDA Breakthrough Therapy Designation

Expected launch of

Emcitate in the U.S.

Q4 2026