When did Oryzon Genomics, S.A. announce the EMA authorisation for the HOPE-2 study?

Oryzon Genomics, S.A. announced the EMA authorisation on 28 September 2026. The announcement covers the planned HOPE-2 Phase IIa study in adults with Phelan-McDermid Syndrome.

What type of study is Oryzon Genomics, S.A. planning for vafidemstat in PMS?

The HOPE-2 study is a single-centre, single-arm, open-label Phase IIa study. It will be conducted in Spain and will enrol 12 adult patients with Phelan-McDermid Syndrome.

What is the primary objective of Oryzon Genomics, S.A.'s HOPE-2 study?

The primary objective is to evaluate the safety and tolerability of vafidemstat. Oryzon Genomics, S.A. also plans to assess disease-related outcomes and the effect on anger and aggression as secondary objectives.

How long will patients receive vafidemstat in Oryzon Genomics, S.A.'s HOPE-2 study?

Vafidemstat will be administered for 12 weeks in the HOPE-2 study. After the first 12 weeks, the investigator will assess whether a participant may continue treatment through week 24 based on clinical benefit.

How will Oryzon Genomics, S.A. measure anger and aggression in the HOPE-2 study?

The study will assess anger and aggression using the Aberrant Behavior Checklist Irritability Subscale and the Clinical Global Impression of Severity - Anger and Aggression. These are listed as secondary objectives of the study.

Which other measures will Oryzon Genomics, S.A. use to assess PMS in the HOPE-2 study?

The study will assess overall disease using the Repetitive Behavior Scale-Revised, the Phelan-McDermid Syndrome Assessment of Severity and the Aberrant Behavior Checklist subscales for Stereotypic Behaviour, Hyperactivity/Noncompliance, Inappropriate Speech and Social Withdrawal.

What is Phelan-McDermid Syndrome in Oryzon Genomics, S.A.'s announcement?

Phelan-McDermid Syndrome is described as a highly disabling neurodevelopmental disorder caused by deletions or pathogenic mutations in the SHANK3 gene. It is characterised by varying degrees of developmental delay, intellectual disability, delayed or absent speech, and autism spectrum disorder or symptoms of autism.

What treatment gap does Oryzon Genomics, S.A. identify in Phelan-McDermid Syndrome?

Oryzon Genomics, S.A. says there are no approved pharmacologic treatments for PMS or specifically for aggression in PMS. The company says aggression is often managed off-label with medicines that have limited efficacy and significant safety concerns.

Why does Oryzon Genomics, S.A. believe vafidemstat could be relevant to PMS?

Oryzon Genomics, S.A.'s Chief Medical Officer for CNS, Rolando Gutierrez-Esteinou, said vafidemstat's mechanism modulates transcriptional programmes involved in neural plasticity, neuroinflammation and neuronal excitability. He also cited preclinical findings and clinical results from the REIMAGINE Phase IIa trial in autism spectrum disorder patients as the basis for the company's view that vafidemstat could provide a treatment option for PMS.

Who will support participant identification for Oryzon Genomics, S.A.'s HOPE-2 study?

Oryzon Genomics, S.A. will collaborate with the Spanish Phelan-McDermid Syndrome Association to support the identification of potential participants for the HOPE-2 study.

How is Oryzon Genomics, S.A.'s HOPE-2 study being funded?

The HOPE-2 study is being conducted in Spain as part of Oryzon Genomics, S.A.'s VANDAM project. VANDAM has received funding from the Spanish Ministry of Science, Innovation and Universities and the Centre for the Development of Industrial Technology and Innovation under the Recovery, Transformation and Resilience Plan, funded by the European Union – NextGenerationEU.

What are Oryzon Genomics, S.A.'s shares identified by in the announcement?

The announcement identifies Oryzon Genomics, S.A. by ISIN Code ES0167733015 and ticker symbol ORY.

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Oryzon receives EMA authorisation to initiate HOPE-2 Phase IIa study in PMS

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Oryzon Genomics S.A.

BME:ORY
OTC PINK:ORYZF

Oryzon receives EMA authorisation to initiate HOPE-2 Phase IIa study in PMS

Published Monday, 28 September 2026

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Plain English summaries when Oryzon Genomics S.A. announces news.

At a glance

Company: Oryzon Genomics S.A.
Listings: BME:ORY, OTC PINK:ORYZF
Announcement type: Regulatory approval
Published: Monday, 28 September 2026
Source announcement: oryzon.com

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Measure	Figure
Adult patients with Phelan-McDermid Syndrome	12 adult patients

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Announcement Summary

Oryzon Genomics, S.A. (ORY) announced a Phase IIa study of vafidemstat for Phelan-McDermid Syndrome.

The European Medicines Agency authorized Oryzon’s Clinical Trial Application to initiate HOPE-2, a Phase IIa study.
HOPE-2 is a single-center, single-arm, open-label study that will enroll 12 adult patients with Phelan-McDermid Syndrome.
Vafidemstat will be administered for 12 weeks; continuation through week 24 depends on clinical benefit assessed by the Investigator.
The primary objective is safety and tolerability; secondary measures assess anger, aggression, and overall disease using ABC, CGI-S A/A, RBS-R, and PMSA-S.
Oryzon will collaborate with the Spanish Phelan-McDermid Syndrome Association to identify potential participants; HOPE-2 will be conducted in Spain.

The study is part of Oryzon’s VANDAM project, which has received funding under the Recovery, Transformation and Resilience Plan.

Investor FAQs

Oryzon Genomics, S.A. announced that the European Medicines Agency (EMA) authorised its Clinical Trial Application to initiate the HOPE-2 Phase II study of vafidemstat in Phelan-McDermid Syndrome (PMS). The study will evaluate vafidemstat in adults with PMS.

Source announcement

Company: Oryzon Genomics S.A.
Title: ORYZON receives European Medicines Agency approval to initiate the HOPE-2 Phase II study of vafidemstat for the treatment of Phelan-McDermid Syndrome (PMS)
Published: Monday, 28 September 2026
Source site: oryzon.com

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