Mr. Rosty Raykov reports

FENNEC PHARMACEUTICALS RECEIVES POSITIVE CHMP OPINION FOR PEDMARQSI (SODIUM THIOSULFATE) TO REDUCE THE RISK OF HEARING LOSS IN PEDIATRIC ONCOLOGY PATIENTS

The committee for medicinal products for human use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion and recommended granting a marketing authorization for Fennec Pharmaceuticals Inc.'s Pedmarqsi (sodium thiosulphate) -- known as Pedmark in the United States -- 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. When formally approved by the European Commission, Pedmarqsi will be the first and only treatment approved in the European Union (EU) to address this area of significant unmet medical need.

"Children treated with cisplatin for solid tumours carry a very high risk of losing their hearing permanently," said Dr. Penelope (Peppy) R. Brock, MD, PhD, of Great Ormond Street Hospital in London and international chair of the SIOPEL6 trial. "As cure rates increase into the high nineties for several cancers, the need to resolve these permanently disabling side effects becomes more and more pressing. I am delighted that finally we have something to offer to counter this life-impacting side effect and can give children the opportunity to live healthy, happy and fully integrated lives after overcoming cancer."

"There are currently no approved treatments in Europe 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. The CHMP positive opinion brings European patients and their families closer to having a preventive treatment option to prevent the devastating consequences of hearing loss following the use of cisplatin chemotherapy, an indispensable treatment of choice in many pediatric cancer cases," said Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. "With approximately 5,000 children eligible for treatment with a platinum-based chemotherapy each year in Europe, we are excited by the potential this therapy can offer to the pediatric oncology community."

The CHMP adopted its positive opinion on safety and efficacy data from two pivotal open-label, randomized phase 3 trials (SIOPEL6 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. In both studies, the incidence of hearing loss was consistently and significantly lower in the Pedmarqsi-plus-cisplatin arm compared with the cisplatin-alone arm 28.6 per cent versus 56.4 per cent (p equals 0.004) and 35.1 per cent versus 67.3 per cent (p equals 0.001) with hearing loss in COG ACCLO431 and SIOPEL6, respectively. The most common adverse reactions (greater than or equal to 25 per cent with difference between arms of greater than 5 per cent compared with cisplatin alone) in SIOPEL6 were vomiting, infection, nausea, decreased hemoglobin and hypernatremia. The most common adverse reaction (greater than or equal to 25 per cent with difference between arms of greater than 5 per cent compared with cisplatin alone) in COG ACCL0431 was hypokalemia.

The CHMP's recommendation will now be reviewed by the European Commission; ratification of the CHMP recommendation is expected by early June, 2023. 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 who 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 injection)

Pedmark is the first and only 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 United States 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 who suffer ototoxicity at critical stages of development lack speech and 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 tumours, osteosarcoma, neuroblastoma, medulloblastoma and other solid tumours. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumours.

About Fennec Pharmaceuticals Inc.

Fennec Pharmaceuticals is a specialty pharmaceutical company focused on the development and commercialization of Pedmark to reduce the risk of platinum-induced ototoxicity in pediatric patients. Further, Pedmark received FDA approval in September, 2022, and has received orphan drug designation in the United States. 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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