Mr. Rosty Raykov reports

FENNEC PHARMACEUTICALS ANNOUNCES EUROPEAN COMMISSION MARKETING AUTHORIZATION FOR PEDMARQSI (SODIUM THIOSULFATE) TO REDUCE THE RISK OF HEARING LOSS IN PEDIATRIC ONCOLOGY PATIENTS

Fennec Pharmaceuticals Inc. has noted that Pedmarqsi -- known as Pedmark in the United States -- was granted marketing authorization by the European Commission. Pedmarqsi is the first and only approved therapy in the EU (European Union) for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients one month to less than 18 years of age with localized, non-metastatic, solid tumours. Further, Pedmarqsi was granted the marketing authorization under the pediatric-use marketing authorization (PUMA) which includes eight years plus two years of data and market protection.

"Today's approval by the European Commission for Pedmarqsi to help reduce the risk of hearing loss is critical for the approximately 4,000 children treated with cisplatin for solid tumours that have not spread," said Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. "Fennec is proud to bring this safe and effective therapy to the marketplace to mitigate the risk of permanent and irreversible bilateral hearing loss, which occurs in approximately 60 per cent of children treated with cisplatin, and can be as high as 90 per cent. We look forward to making this important treatment available to the pediatric oncology community in the EU as soon as possible."

The European Commission granted marketing authorization approval to Pedmarqsi following the adoption of a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) earlier this year. The CHMP's decision was based on safety and efficacy data from two pivotal open-label, randomized phase 3 trials (SIOPEL 6 and clinical oncology group [COG] protocol ACCL0431), which compared Pedmarqsi plus cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients.

The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein. Pedmark was approved by the U.S. Food and Drug Administration (FDA) in September 2022.

About cisplatin-induced ototoxicity

Cisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.

The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time. Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational achievement.

Pedmark (sodium thiosulphate)

Pedmark is the first and only United States Food and Drug Administration (FDA)-approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients with localized, non-metastatic, solid tumours. It is a unique formulation of sodium thiosulphate in single-dose, ready-to-use vials for intravenous use in pediatric patients. Pedmark is also the only therapeutic agent with proven efficacy and safety data with an established dosing paradigm, across two open-label, randomized phase 3 clinical studies, the clinical oncology group (COG) protocol ACCL0431 and SIOPEL 6.

In the U.S. and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss, and only expensive, technically difficult and suboptimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

Pedmark has been studied by co-operative groups in two phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumour, osteosarcoma, neuroblastoma, medulloblastoma and other solid tumours. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumours.

Indications and usage

Pedmark (sodium thiosulphate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients one month of age and older with localized, non-metastatic solid tumours.

Limitations of use

The safety and efficacy of Pedmark have not been established when administered following cisplatin infusions longer than six hours. Pedmark may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

About Fennec Pharmaceuticals Inc.

Fennec Pharmaceuticals is a specialty pharmaceutical company focused on the development and commercialization of Pedmark and Pedmarqsi to reduce the risk of platinum-induced ototoxicity in pediatric patients. Further, Pedmark received FDA approval in September, 2022, and European Commission marketing authorization in June, 2023, for Pedmarqsi. Pedmark has received orphan drug exclusivity in the U.S. for seven years of market protection, and Pedmarqsi has received pediatric use marketing authorization in Europe which includes eight years plus two years of data and market protection. Fennec has a licence agreement with Oregon Health and Science University (OHSU) for exclusive worldwide licence rights to intellectual property directed to sodium thiosulphate and its use for chemoprotection, including the reduction of risk of ototoxicity induced by platinum chemotherapy, in humans.

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