iQure Pharma Receives Approval for iQ-007 Phase 1 First in Human Study

Princeton, NJ, March 12, 2025 ─ iQure Pharma Inc. (iQure), a biotechnology company focused on the development of novel therapies for neurological conditions, has received IRB (Institutional Review Board) approval for the first entry-into-human Phase 1 study with its lead asset iQ-007 in Healthy Volunteers. iQ-007, iQure’s flagship compound, is a novel therapeutic candidate under development to treat epilepsy and other neurodegenerative conditions.

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, Alzheimer’s Disease and Parkinson’s Disease or Pain.

In March 2025, iQure secured formal approval from the Human Research Ethics Committee (HREC) in Australia, for the iQ-007 Phase 1 study, IQ-007-CL-001. The study is a randomized, double-blind, placebo-controlled, single and multiple ascending dose study to assess the safety, tolerability and pharmacokinetics of oral iQ-007 in healthy male and female adult participants. Anticipated start for the study is targeted for April, with results expected in Q4 2025.

Pawel Zolnierczyk, iQure Pharma CEO: “We are delighted to secure approval to start clinical development of our lead asset, iQ-007. iQure Pharma is proud to be the first to enter clinical development with a new potential medication with a mechanism of action focused on the uptake of glutamate via EAAT2 PAM. Preclinical efficacy data indicate a highly promising outlook for iQ-007 in neurodegeneration including epilepsy, Alzheimer’s Disease, Parkinson’s Disease, ALS and pain. Data from Phase 1 will help to assess iQ-007’s safety and pharmacokinetic profile, which will allow us to reach the next milestone and optimize further development toward efficacy studies with iQ-007.”

Henk de Wilde, Chief Development Officer, added, “This significant milestone marks the first entry of an EAAT2 modulator into the clinical phase and will give us the opportunity to explore the extraordinary mechanism of action of iQ-007 in humans.”

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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.

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.

iQure Pharma Secures $4M Funding – Advancing Phase 1 Clinical Trial for iQ-007

Princeton, NJ, January 07, 2025 ─ iQure Pharma Inc. (iQure), a biotechnology company focused on the development of novel therapies for neurological conditions, has completed its latest round of funding. The company has raised $4.0 million to progress with a Phase 1 clinical trial for its lead asset iQ-007, a novel therapeutic candidate to treat epilepsy and other neurodegenerative conditions.

The funding round was completed by a syndicate of prominent investors, including Munich-based life sciences fund Ventura Ace and i&i Biotech, a Prague-based life sciences fund, as well as early-stage med-tech anchor investor, OKG Capital. “iQure’s potential to revolutionize epilepsy and neurodegenerative disease care is extraordinary,” said Karim Galzahr of OKG Capital. “This funding round, backed by specialist life science investors, is a pivotal step in advancing breakthrough treatments for serious CNS conditions.”

iQure’s flagship compound, iQ-007, represents a first-in-class EAAT2 Positive Allosteric Modulator (PAM), designed to address toxic levels of glutamate in the synaptic cleft. Excess glutamate is a key driver of neurotoxicity and neurodegeneration in conditions such as epilepsy, including currently non-treatable Drug Resistance Epilepsy, stroke, Alzheimer’s, neuropathic pain, amyotrophic lateral sclerosis (ALS), and multiple sclerosis (MS). 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. iQ-007 originates from Prof. Krzysztof Kaminski laboratory, one of the leading research groups at prestigious Jagiellonian University in Krakow, Poland.

Preclinical studies have demonstrated the efficacy and safety of iQ-007, including its successful completion of the NIH’s prestigious ETSP epilepsy screening program. Pivotal toxicology studies were finalized in Q4 2024, paving the way for Phase 1 trials.

Pawel Zolnierczyk, iQure Pharma CEO: “The funding we have secured will support the transformation of iQure into a clinical-stage biotech company. The Phase 1 clinical trial for iQ-007 is an important step forward to assess iQ-007’s safety profile, tolerability, and pharmacokinetics in healthy volunteers. Our preclinical data indicate that this compound has the potential to transform the treatment of various neurological conditions.”

Henk de Wilde, Chief Development Officer of iQure Pharma added, “Our area of interest is human astroglia and their important role in maintaining brain homeostasis and defense. This role offers the opportunity for a valid 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, pain, and diseases such as Alzheimer’s and Parkinson’s. This innovative approach places iQure at the forefront of neurotherapeutics development.”

For more information about supporting iQure’s initiatives, contact iQure CEO Pawel Zolnierczyk at pawel.zolnierczyk@iqurepharma.com.

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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 Ventura Ace

Ventura Ace invest in early-stage life science companies developing pioneering technologies and innovative therapies. As serial entrepreneurs with many years of experience, we help shape the long-term development of our portfolio companies and provide them access to our broad investor and business network. 

About i&i Biotech

i&i Bio is a Luxemburg-based venture capital firm that invests in innovative European Life Sciences companies focused on drug discoveries, medical devices, diagnostics, and digital health. The Fund was created in cooperation with the biotech incubator i&i Prague and the European Investment Fund (see below). With over €53M under management, i&i Bio plans to invest in about 20 early-stage companies. i&i Bio is led by an experienced team of professionals with backgrounds in private equity, healthcare and venture capital, supporting entrepreneurs on their journey to global success. Thanks to close cooperation with the fund’s main sponsor, the biotech academic incubator i&i Prague, i&i Bio is supporting and advancing transformative Central European technology companies.

About the European Investment Fund (EIF)

The Fund is supported by an investment from the EIF, with the support of:

• lnnovFin Equity, with the financial backing of the European Union under Horizon 2020 Financial Instruments and the European Fund for Strategic Investments (EFSI) set up under the Investment Plan for Europe. The purpose of EFSI is to help support financing and implementing productive investments in the European Union and to ensure increased access to financing; and

• the Pan-European Guarantee Fund (EGF), implemented by the EIF with the financial support of the Participating Member States. The objective of EGF is to respond to the economic impact of the COVID-19 pandemic by ensuring that companies in the Participating Member States have sufficient short-term liquidity available to weather the crisis and are able to continue their growth and development in the medium to long-term.

About OKG Capital

OKG Capital is a specialist med-tech and life science investor based in the US, Delaware with operations in London and Geneva. OKG invests in the management and diagnosis of serious chronic diseases affecting individuals and burdening healthcare systems. OKG aims to bridge the gap between cutting edge science and clinical practice, operating at the intersection between patient need and long-term financial return.