AEF0217 / Aelis Farma - LARVOL DELTA

AELIS…announces today that the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has emitted a favorable opinion on the Paediatric Investigation Plan (PIP) for AEF0217 (EMA/PE/0000243385; IRIS: 0075800000532) in the treatment of Down syndrome. (Yahoo Finance) - "The PIP is fully in line with Aelis Farma’s extensive clinical development plan assessing the efficacy and safety of AEF0217 in people with Down syndrome, structured in two parts: The first part includes a Phase 2B study in older adolescents and young adults with Down syndrome aged 16 to 32 years. The second part, covered by the PIP, foresees subsequent studies progressively including children aged 6 to <16 years, then 2 to <6 years, and potentially from birth to <2 years."

European regulatory • Genetic Disorders

AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P2 | N=188 | Not yet recruiting | Sponsor: Aelis Farma

New P2 trial • Developmental Disorders • Genetic Disorders

MDDS: Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P1/2 | N=40 | Completed | Sponsor: Aelis Farma | Active, not recruiting ➔ Completed

Trial completion • Developmental Disorders • Genetic Disorders • APOE

MDDS: Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P1/2 | N=40 | Active, not recruiting | Sponsor: Aelis Farma | Recruiting ➔ Active, not recruiting | Trial primary completion date: May 2024 ➔ Aug 2024

Enrollment closed • Trial primary completion date • Developmental Disorders • Genetic Disorders • APOE

MDDS: Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P1/2 | N=45 | Recruiting | Sponsor: Aelis Farma | Trial completion date: Dec 2023 ➔ Jun 2024 | Trial primary completion date: Nov 2023 ➔ May 2024

Trial completion date • Trial primary completion date • Developmental Disorders • Genetic Disorders • APOE

MDDS: Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P1/2 | N=45 | Recruiting | Sponsor: Aelis Farma | Trial completion date: Jun 2023 ➔ Dec 2023 | Trial primary completion date: May 2023 ➔ Nov 2023

Trial completion date • Trial primary completion date • Developmental Disorders • Genetic Disorders

AEF0217-101: AEF0217 First-in-Human Phase I Study in 3 Parts (Single and Multiple Ascending Doses, and Food Effect) in Healthy Subjects. (clinicaltrials.gov) - P1 | N=68 | Completed | Sponsor: Aelis Farma | Active, not recruiting ➔ Completed | Trial completion date: Mar 2023 ➔ Jun 2022

Trial completion • Trial completion date

MDDS: Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome (clinicaltrials.gov) - P1/2 | N=45 | Recruiting | Sponsor: Aelis Farma

New P1/2 trial • Developmental Disorders • Genetic Disorders