The biologics license application follows months of public speculation—and regulatory reversals—for uniQure’s AMT-130. If accepted by the FDA for priority review, approval could come in the second quarter of 2027.

Appointed CTC Clinical Trial Consultants AB (CTC) as the clinical research organization (CRO) for the Phase 2 study. Appointed Dr Juha Savola as Chief Medical Officer, bringing more than 25 years of global experience in drug development, regulatory affairs and strategic leadership across neurology, gene therapy and rare diseases... Herantis Pharma Plc is a clinical-stage biotechnology company developing disease-modifying therapies for Parkinson’s disease. The Company’s lead product, HER-096, is a first-in-class small peptide that combines the neuroprotective mechanism of cerebral dopamine neurotrophic factor (CDNF), with the convenience of subcutaneous administration. In a Phase 1b clinical trial, HER 096 was shown to be generally safe and well tolerated in Parkinson’s disease patients. Espoo, Finland, August 20, 2026: Herantis Pharma Plc ("Herantis") a clinical-stage company developing disease-modifying therapies to stop the progression of Parkinson’s disease, releases today the Company’s 1H 2026 report. Selected to lead a consortium that was awarded an EUR 8.0 million Horizon Europe 2025 Research and Innovation grant to support the execution of the planned Phase 2 trial of HER-096 in Parkinson’s disease. Presented Phase 1b data for HER-096 at 20th International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD 2026) Announced a collaboration to integrate Indivi’s digital biomarker platform into the upcoming Phase 2 study of HER-096, aiming to detect early treatment-related changes in motor and cognitive function. Factors, including risks and uncertainties that could cause these differences include, but are not limited to risks associated with implementation of Herantis’ strategy, risks and uncertainties associated with the development and/or approval of Herantis’ drug candidates, ongoing and future clinical trials and expected trial results, the ability to commercialize drug candidates, technology changes and new products in Herantis’ potential market and industry, Herantis’ freedom to operate in respect of the products it develops (which freedom may be limited, e.g., by competitors’ patents), the ability to develop new products and enhance existing products, the impact of competition, changes in general economy and industry conditions, and legislative, regulatory and political factors.

Adaptive Clinical Trial Design ... Clinical Trials Digital Health Technologies eClinical Tech News eClinical Technology EMA Executive Interviews FDA Enforcement FDA Guidance FDA Regulation FDA Regulatory Strategy GCP Compliance GLP-1 Receptor Agonists Ketamine Therapy Neurodegenerative Disease Oncology Trials Opinion Parkinson's Disease Pediatric Oncology Platform Trials PSYCH PULSE Real-World Evidence site activation Site Management Site Operations study startup Treatment-Resistant ... Adaptive Clinical Trial Design Adaptive Trial Design Addiction Psychiatry AI in Clinical Trials alcohol use disorder Article Cell and Gene Therapy Clinical Decision Support Clinical Operations Clinical Research Clinical Trial Design Clinical Trial Operations Clinical Trial Ops Brief Clinical Trial Quality Clinical Trials CLINOPS WATCHDOG CNS Drug Development CNS Trials Conference Coverage Decentralized Clinical Trials Digital Health Technologies eClinical Tech News eClinical Technology EMA Executive Interviews FDA Enforcement FDA Guidance FDA Regulation FDA Regulatory Strategy GCP Compliance GLP-1 Receptor Agonists Ketamine Therapy Neurodegenerative Disease Oncology Trials Opinion Parkinson's Disease Pediatric Oncology Platform Trials PSYCH PULSE Real-World Evidence site activation Site Management Site Operations study startup Treatment-Resistant Depression EMA revoked Tavneos (avacopan) in August 2026 over GCP breaches in the pivotal ADVOCATE trial. FDA approved the same data in 2021. Someone should answer for… When a rare-disease approval survives four years of commercial use and then collapses on data integrity rather than new clinical evidence, the monitoring infrastructure that was supposed to catch deviations in real time failed at every level it was designed to operate. Under ICH E6(R2), the sponsor holds primary accountability for trial conduct regardless of how much operational authority has been delegated to a CRO. Rare-disease trials operate under conditions that make GCP compliance harder to enforce and easier to obscure. ADVOCATE enrolled patients with severe ANCA-associated vasculitis, a population that is small, geographically dispersed, and clinically complex.

Privosegtor has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) as well as Orphan Drug designation from both the FDA and the EMA for ON. ZUG, Switzerland and BARCELONA, Spain, August 10, 2026 Oculis Holding AG (Nasdaq: OCS / XICE: OCS) (“Oculis” or the “Company”), a global biopharmaceutical company focused on breakthrough innovations to address significant unmet medical needs in neuro-ophthalmology and ophthalmology, and Accure Therapeutics, S.L. Building on the ACUITY Phase 2 dataset in optic neuritis and constructive FDA pre-IND feedback, Oculis is also planning an IND submission for Privosegtor for the treatment of acute MS relapses. Privosegtor is an investigational drug and has not received regulatory approval for commercial use in any country. ... Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) focused on breakthrough innovations to address significant unmet medical needs in neuro-ophthalmology and ophthalmology. Oculis also recently announced positive FDA pre-IND feedback supporting a regulatory pathway for Privosegtor in acute MS relapses, ahead of a planned IND submission in the fourth quarter of 2026. ... About Privosegtor Privosegtor, a novel peptoid small-molecule candidate that crosses the blood-brain and retinal barriers, has the potential to become the first neuroprotective therapy for optic neuritis (ON) and other neuro-ophthalmic and neuro-axonal diseases. It has a unique portfolio of three first-in-class new chemical entities programs, pursuing innovative targets and potential game changers in the treatment of serious diseases of the central nervous system: ACT-01 (initially sourced from IDIBAPS-Hospital Clinic, Barcelona, Spain, and the Spanish National Council CSIC and then licensed to Oculis – NASDAQ: OCS) at positive phase II clinical trial completed stage in acute optic neuritis, ACT-02 (initially sourced from the Institute for Research in Biomedicine IRB, Barcelona, Spain, and the Universitat de Barcelona UB) at IND-enabling stage in Parkinson’s disease and ACT-03 at CCS-stage in epilepsy. With an experienced business and scientific team, Accure Therapeutics is one of the few companies that operate in an agnostic fashion on initial science to deliver cutting-edge drugs in CNS.

Forward-Looking Statements This ... gene therapy product candidates under the Novartis licenses and collaboration and Neurocrine collaboration, including the anticipated initiation of clinical trials by Neurocrine for NBIB-‘223 in FA, pending successful IND clearance; the role of tau in the treatment of AD, including as a critical disease-modifying ... Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including, without limitation, implied and express statements about Voyager’s belief and expectations regarding the development of its product candidates and advancement of its preclinical and clinical development programs, including Voyager’s advancement of the tau silencing gene therapy program in AD, VY1706, including the timing of clinical development milestones such as Voyager’s intentions to i VY7523 (anti-tau antibody): Voyager expects tau positron emission tomography (PET) imaging efficacy data in Q4 2026 from the ongoing multiple ascending dose (MAD) clinical trial in participants with Alzheimer’s disease (AD). VY1706 (tau silencing gene therapy): The U.S. “We continue to advance our two tau-targeted programs toward potential inflection points in clinical trials for Alzheimer’s disease before the end of the year, with the tau-targeted antibody VY7523 expected to generate tau PET imaging data and the tau-targeted gene therapy VY1706 expected to begin dosing.” In July, Health Canada cleared Voyager’s Clinical Trial Application (CTA), enabling the inclusion of Canadian clinical trial sites in the study. NBIB-‘223 (Friedreich’s ataxia gene therapy): Voyager’s partner Neurocrine Biosciences has stated that it intends to initiate a clinical trial with NBIB-‘223 in H2 2026, pending successful FDA IND clearance.