iQure Pharma’s iQ-007 Receives FDA Orphan Drug Designation for Dravet’s Syndrome

Princeton, NJ, January 23, 2025 ─ iQure Pharma Inc. (iQure), a biotechnology company focused on the development of novel therapies for neurological conditions, has received Orphan Drug Designation (ODD) from the Federal Drug Administration (FDA) for its lead asset iQ-007, a novel therapeutic candidate under development to treat epilepsy and other neurodegenerative conditions.

iQure’s flagship compound, iQ-007, represents a first-in-class EAAT2 Positive Allosteric Modulator (PAM), designed to clear toxic levels of glutamate from the synaptic cleft. Excess glutamate is a key driver of neurotoxicity and neurodegeneration in conditions such as epilepsy, including rare types of severe epilepsy like Dravet’s Syndrome (DS). By enhancing the function of Excitatory Amino Acid Transporter 2 (EAAT2), which is responsible for up to 90% of glutamate uptake, iQ-007 aims to prevent neuronal damage and degeneration.

Dravet’s Syndrome is a rare type of genetic epileptic encephalopathy, characterized by prolonged seizures that don’t respond well to current seizure medications. These seizures generally begin within the first year of life in an otherwise healthy infant and can seriously impede the overall development of the infant. Patients with Dravet syndrome face a 15-20% mortality rate, as a result of the disease and comorbidities. Reports suggest 1 in 20,000 to 1 in 40,000 people have Dravet’s Syndrome. Three out of eight children who experience a seizure within their first 12 months, may have Dravet’s Syndrome. (Epilepsy Foundation)

Pawel Zolnierczyk, iQure Pharma CEO: “FDA granting Orphan Drug Designation in Dravet’s Syndrome for iQ-007 is an important development and critical external validation of the science underlining the iQ-007 program. As a company, iQure Pharma helping patients with rare types of diseases is a vital part of our wider mission. In conjunction with data we aim to receive from our upcoming Phase 1 study, we will be considering optimum next steps, including further clinical development with Dravet’s Syndrome patients.”

Henk de Wilde, Chief Development Officer of iQure Pharma added, “This significant validation supports our therapeutic approach to the treatment of neurodegeneration. iQ-007 improves the ability of astrocytes to reuptake excess glutamate, addressing neurodegeneration caused by excessive glutamate levels in epilepsy, in diseases such as Davet’s Syndrome and places iQure at the forefront of neurotherapeutics development.”

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About iQure Pharma

iQure Pharma, a global biotech firm headquartered in the US, is focused on the development of new therapeutics for CNS, including neuropathic pain, epilepsy and other neurodegenerative diseases. iQure researchers, clinicians, and pharmaceutical professionals work closely with academic partners, industry experts, and patient organizations to build medically and socially beneficial therapies.

About the FDA ODD Program

The FDA’s Orphan Drug Designation program supports the development and evaluation of new treatments for rare diseases and is a key priority for the FDA. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing.