Calmangafodipir

STOCKHOLM, SE / ACCESS Newswire / April 30, 2026 / Egetis Therapeutics (STO:EGTX) - Egetis Therapeutics AB (publ) ("Egetis" or the "Company") (NASDAQ Stockholm:EGTX), today announced its participation in the Pediatric Endocrine Society (PES) Annual Meeting, taking place April 30 to May 3, 2026, in San Francisco, CA, USA. Egetis will engage with the pediatric endocrinology community through multiple scientific and educational activities during the meeting. The Company will host a disease awareness booth (#321) , providing information on MCT8 deficiency (Allan-Herndon-Dudley syndrome) and ongoing efforts to increase disease awareness, enhance clinician education, and support earlier diagnosis. Anny Bedard, President North America at Egetis, said: "The Pediatric Endocrine Society meeting in the U.S. represents an important opportunity for Egetis to connect with a highly relevant clinical audience. Pediatric endocrinologists play a critical role in the identification, diagnosis, and management of patients with rare metabolic and endocrine disorders, and we look forward to sharing our data and increasing awareness of MCT8 deficiency." In addition, Egetis will present a scientific poster titled " Real-world data-driven insights uncovering clinical and biochemical patterns that inform the identification of undiagnosed patients with MCT8 deficiency (Allan-Herndon-Dudley syndrome) ", underscoring opportunities to improve early recognition and reduce time to diagnosis. Session: Poster Session 2 Poster number: 25 Date: Friday May 1 Time: 7:15 - 8:00 am PDT The Company will also deliver an oral presentation titled "Effects of tiratricol withdrawal in monocarboxylate transporter 8 (MCT8) deficiency: The ReTRIACt trial," adding to the growing body of clinical evidence supporting the safety and efficacy pro tiratricol. Session: Oral Abstracts #3: Growth, Thyroid, and Obesity Date: Saturday May 2 Time: 5:25 - 5:35 pm PDT For further information, please contact: Nicklas Westerholm, CEO +46 (0) 733 542 062 nicklas.westerholm@egetis.com Karl Hård, Head of Investor Relations & Business Development +46 (0) 733 011 944 karl.hard@egetis.com About Egetis Therapeutics Egetis Therapeutics is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment. The Company's lead drug candidate Emcitate® (tiratricol) is developed for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In February 2025 the European Commission approved Emcitate® as the first and only treatment for MCT8 deficiency in EU. Egetis initiated the launch of Emcitate® in Germany on May 1, 2025. Emcitate® (tiratricol) is not approved in the USA. On March 27, 2026, Egetis announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026. The NDA for Emcitate® (tiratricol) for treatment of MCT8 deficiency is based on clinical data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program. Tiratricol holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Tiratricol has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation (RPDD) by the FDA, which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval. The drug candidate Aladote® (calmangafodipir) is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed. The design of a pivotal Phase IIb/III study (Albatross), with the purpose of applying for market approval in the US and Europe, has been finalized following interactions with the FDA, EMA and MHRA. The development program for Aladote® has been parked. Aladote® has been granted ODD in the US and in the EU. Egetis Therapeutics is listed on the Nasdaq Stockholm main market (Nasdaq Stockholm:EGTX). For more information, see Attachments Egetis Therapeutics to Participate in Pediatric Endocrine Society Annual Meeting 2026 SOURCE: Egetis Therapeutics View the original press release on ACCESS Newswire

STOCKHOLM, SE / ACCESS Newswire / April 9, 2026 / Egetis Therapeutics AB (publ) ("Egetis" or the "Company") (STO:EGTX), today announced the appointment of Tiago Nunes, MD, PhD, as Chief Medical Officer (CMO), effective May 1, 2026. Tiago Nunes is an experienced clinical development leader, with significant expertise advancing rare disease therapeutics through regulatory approval and launch, having most recently served as Senior Vice President, Clinical Development at Mirum Pharmaceuticals (NASDAQ: MIRM). Nicklas Westerholm, CEO of Egetis, commented : "I am thrilled to welcome Tiago to Egetis at this exciting time. Tiago is an experienced pharmaceutical executive and medical leader with extensive achievements in rare disease, including successful product approvals and launches driven by his clinical development leadership, in close collaboration with medical affairs, regulatory, and commercial functions. Tiago will add considerable momentum as we continue to build our medical and clinical capabilities toward the planned launch of Emcitate ® (tiratricol) in the U.S. and continue to explore potential label expansion opportunities, such as RTH-beta. "I would also like to thank our outgoing CMO Kristina Sjöblom Nygren, who has decided to retire, for hersubstantial contributions and leadership efforts over the past five years where she amongst other things was instrumental in getting Emcitate ® approved as the first and only treatment for MCT8 deficiency in EU in February 2025. I wish her well in her next phase in life." Tiago Nunes, incoming CMO at Egetis, commented : "I am honored to join Egetis at this pivotal time in the Company's growth trajectory as it plans to launch the first treatment for MCT8 deficiency in the U.S. I look forward to supporting Egetis to achieve its goals to develop and deliver safe and effective rare disease treatments to underserved patient populations." Tiago Nunes previously served as Senior Vice President, Clinical Development at Mirum Pharmaceuticals, where he led the clinical development of Livmarli ® and played a pivotal role in its successful approval and launch. Under his leadership, the organization advanced a rare disease portfolio through multiple clinical milestones, including regulatory approvals, label expansions, and the progression of innovative clinical programs such as volixibat in cholestatic liver diseases. Prior to Mirum, Tiago Nunes served as Clinical Development Lead at Nestlé Health Science, where he provided medical leadership in gastrointestinal and rare disease drug development. Nunes earned his medical degree from the Federal University of Rio de Janeiro, Brazil, where he also completed his residency in gastroenterology. He holds a Master's degree in Biomedicine and a PhD in Medicine from the University of Barcelona, Spain. Egetis outgoing CMO, Kristina Sjöblom Nygren, MD, will remain at Egetis until Emcitate ® (tiratricol) is approved in the U.S. to ensure a smooth transition of medical responsibilities to Tiago Nunes. For further information, please contact: Nicklas Westerholm, CEO +46 (0) 733 542 062 nicklas.westerholm@egetis.com Karl Hård, Head of Investor Relations & Business Development +46 (0) 733 011 944 karl.hard@egetis.com About Egetis Therapeutics Egetis Therapeutics is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment. The Company's lead drug candidate Emcitate ® (tiratricol) is developed for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In February 2025 the European Commission approved Emcitate ® as the first and only treatment for MCT8 deficiency in EU. Egetis initiated the launch of Emcitate ® in Germany on May 1, 2025. Emcitate ® (tiratricol) is not approved in the USA. On March 27, 2026, Egetis announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate ® (tiratricol) for the treatment of MCT8 deficiency. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026. The NDA for Emcitate ® (tiratricol) for treatment of MCT8 deficiency is based on clinical data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program. Tiratricol holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Tiratricol has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation (RPDD) by the FDA, which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval. The drug candidate Aladote ® (calmangafodipir) is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed. The design of a pivotal Phase IIb/III study (Albatross), with the purpose of applying for market approval in the US and Europe, has been finalized following interactions with the FDA, EMA and MHRA. The development program for Aladote ® has been parked. Aladote ® has been granted ODD in the US and in the EU. Egetis Therapeutics is listed on the Nasdaq Stockholm main market (Nasdaq Stockholm:EGTX). For more information, see Attachments Egetis Appoints Tiago Nunes as Chief Medical Officer SOURCE: Egetis Therapeutics View the original press release on ACCESS Newswire