FDA’s review of Emcitate® NDA progressing according to plan towards September 28, 2026, PDUFA action date
- Egetis granted patent for MCT8 Deficiency Composition in the U.S.
- Emcitate sales in Europe during Q2 2026 was MSEK 17.4, a 23% increase at constant exchange rates (CER) compared with Q2 2025
- Egetis successfully carried out an oversubscribed directed share issue amounting to MSEK 350 or approximately MUSD 38 (gross)
Financial overview April-June
· Quarterly revenue MSEK 17.4 (14.5), +23% at CER
· Quarterly result MSEK -108.9 (-77.6)
· Cash at the end of the quarter amounted to MSEK 378.4 (202.6)
· Cash flow for the quarter MSEK 230.2 (-69.2)
· Earnings per share before/after dilution SEK -0.2 (-0.2)
Financial overview January-June
· Revenue for the period MSEK 30.7 (27.1), +13% at CER
· Quarterly result for the period MSEK -203.4 (-140.5)
· Cash at the end of the period amounted to MSEK 378.4 (202.6)
· Cash flow for the period MSEK 157.5 (-143.4)
· Earningspersharebefore/afterdilution SEK-0.5 (-0.4)
Significant events during the quarter
· FDA’s review of Emcitate NDA progressing towards September 28, 2026, PDUFA action date
· Egetis successfully carried out an oversubscribed directed share issue amounting to MSEK 350 or approximately MUSD 38 (gross)
· Egetis granted patent for MCT8 Deficiency Composition in the U.S.
· Egetis received conditional FDA acceptance of proprietary name Emcitate®
· Tiago Nunes appointed Chief Medical Officer
· Birgitte Volck and Jay Donovan Wu elected as new members to the Board of directors at the Annual General Meeting
· The Emcitate price negotiations within the German reimbursement process (AMNOG) concluded in Q2 2026 and are valid from August 1
· Egetis announced U.S. national re-airing of Behind the Mystery Episode on Lifetime Spotlighting MCT8 Deficiency
Significant events after the quarter
· Egetis signed collaboration and supply agreement with Orspec Pharma for Australia and New Zealand
Comments from the CEO
Progress on the Emcitate® NDA review in the U.S.
The most significant event during the first half of 2026 was the acceptance, and the grant of priority review, of our New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. The application was assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026. Thus far, the review has been constructive, and the mid-cycle as well as the late-cycle meetings have been held with the agency, which has confirmed that FDA expects to finish its review by the PDUFA date of September 28, 2026 and does not plan to hold an advisory committee meeting.
Preparatory launch activities in the U.S.
We have accelerated our preparatory launch activities in the U.S. and the build out of our commercial and medical affairs infrastructure. During the period we have contracted a specialty pharmacy and a distributor to assist us in providing Emcitate to patients with MCT8 deficiency after approval. As previously communicated, our updated healthcare provider (HCP) and caregiver disease education websites, at www.mct8deficiency.com and www.lifewithmct8deficiency.com respectively, are designed to deliver clear and comprehensive, and easier‑to‑navigate disease information tailored to each audience.
We also announced national re-airing of Behind the Mystery Episode on Lifetime Spotlighting MCT8 Deficiency. The television series featured on The Balancing Act®, which is broadcast nationally in the U.S. on the Lifetime Network. The episode highlights MCT8 deficiency (Allan-Herndon-Dudley syndrome), a rare, devastating, and life-shortening genetic disorder caused by mutations in the gene coding for MCT8. A replay of the episode can be found here: www.lifewithmct8deficiency.com
Expanded Access Program (EAP) in the U.S.
At FDA’s request, Egetis has implemented an Expanded Access Program (EAP) in the U.S. Currently, 17 hospitals are included and approximately 60 patients are receiving tiratricol in the EAP. The EAP allows physicians to access tiratricol for patients not eligible for clinical trials prior to marketing approval, as well as for continued treatment of patients who completed the ReTRIACt and Triac Trial II studies. For more information about the EAP, please see: https://clinicaltrials.gov/study/NCT05911399
The value of Priority Review Vouchers remains high
As Emcitate has been granted Rare Pediatric Disease Designation by the FDA, Egetis is eligible to receive a Priority Review Voucher (PRV), upon potential approval. Through the program, a company that obtains FDA approval for an eligible therapy is awarded a voucher that can be used to secure a priority review for a future NDA. Priority review shortens the FDA’s standard review period from ten months to approximately six months. PRVs are transferable and can be sold to other companies, creating an active secondary market for these vouchers. PRVs sold in 2026 have fetched USD 180–220 million each. The highest price of USD 220 million was announced by the buyer, BMS, on July 30, 2026. Egetis is entitled to 50 percent of net proceeds from any Emcitate-related PRV sale, with the rest paid as earnout to Rare Thyroid Therapeutics International AB sellers following acquisition of Emcitate in 2020.
Patent granted for MCT8 deficiency composition patent
On May 5, 2026, the United States Patent and Trademark Office (USPTO) granted Patent No. US 12611383B1 for the Company’s patent application No. 19/261,360 entitled “Pharmaceutical Compositions for Treating MCT8 Deficiency”. This is the first patent granted for Emcitate and provides protection for a novel composition, which contains tiratricol as the active ingredient, designed to correct the disrupted thyroid hormone signaling characteristic of MCT8 deficiency. The claims cover, among other things, a method of treating MCT8 deficiency with the claimed pharmaceutical composition that encompasses tiratricol, dosing regimens, and tiratricol compositions with specific excipients. This patent represents a significant milestone in strengthening the Company’s intellectual property portfolio. Egetis expects the granted patent will be Orange Book-listable, with a patent expiration date in 2045.
Commercialization of Emcitate in the EU
The revenue for Emcitate sales in Europe during the second quarter of 2026 was MSEK 17.4, a 23% increase at constant exchange rates (CER) compared with 2025. We initiated the launch of Emcitate in the first country, Germany, in May 2025, and in October 2025 we started price negotiations with the German national statutory health insurers, GKV-Spitzenverband. The process was successfully concluded in the second quarter of 2026, and we are pleased that the German authorities recognize the value of our orphan drug.
As described previously, our main commercial strategy is to continue to develop the market through meetings with physicians, congress presence, educational initiatives and disease awareness activities. We are continuing our engagements primarily with pediatric endocrinologists and pediatric neurologists.
In Spain we have recently submitted a national pricing and reimbursement dossier for Emcitate. In Italy and France, we plan to strengthen our value dossiers with survival data for Emcitate, once it has been published in a peer-reviewed journal by Erasmus Medical Center.
Our partners are making progress
Our Japanese partner Fujimoto Pharmaceuticals, who has an exclusive license for the development and commercialization of Emcitate for MCT8 deficiency in Japan, continues to prepare the Japanese NDA submission dossier, utilizing existing data generated from the global clinical development program. The NDA submission in Japan for Emcitate is expected to be submitted in the beginning of 2027.
Our distribution partner companies Er-Kim (Turkey, Central, Eastern, and Southeastern Europe) and Taiba rare (Gulf region) are actively identifying patients in their respective territories and have initiated funded treatment to more patients. In Australia we have recently signed a supply agreement with Orspec Pharma to enable us to reach more patients with MCT8 deficiency in Australia and New Zealand.
What’s next: Emcitate in RTH-beta
Work is ongoing to finalize the development plan for Emcitate in RTH-beta. We are convening a scientific advisory board and have started to prepare for regulatory interactions to discuss our proposed clinical development plan.
Cash
We report cash of approximately MSEK 378 (MSEK 203) as of June 30, 2026. During the period, on April 21, 2026, we successfully carried out an oversubscribed directed share issue amounting to MSEK 350 (approximately MUSD 38) (gross) at SEK 5.25 per share, corresponding to the closing price on Nasdaq Stockholm on April 21, 2026. We were particularly pleased to see strong participation from both existing shareholders and several new international specialist healthcare investors, further broadening our shareholder base.
Upon the potential approval of Emcitate in the U.S. on September 28, 2026, we aim to monetize the accompanying PRV, primarily to support our launch activities in the U.S.
Outlook
2026 is a year marked by several important milestones for Egetis. Our team focuses on delivering four key priorities:
· Successfully engage with FDA during the review of the Emcitate NDA to gain FDA approval by the PDUFA date of September 28, 2026.
· Preparatory launch activities in the USA
· Optimize pricing- and reimbursement and continue launch of Emcitate in Europe
· Finalize the development plan for Emcitate in RTH-beta
Stockholm, Sweden, August 21, 2026
Nicklas Westerholm
CEO
For further information, please contact
Nicklas Westerholm, CEO
nicklas.westerholm@egetis.com
+46 (0) 733 542 062
Yilmaz Mahshid, CFO
yilmaz.mahshid@egetis.com
+46 (0) 722 316 800
Karl Hård, Head of Investor Relations & Business Development
karl.hard@egetis.com
+46 (0) 733 011 944
This information is information that Egetis Therapeutics is obliged to make public pursuant to the EU Market Abuse Regulation and the Securities Markets Act. The information was submitted for publication, through the agency of the contact persons set out above, at 2026-08-21 07:00 CEST.
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 www.egetis.com