Mr. Robert Andrade reports

FENNEC PHARMACEUTICALS ANNOUNCES FDA APPROVAL OF PEDMARK(TM) (SODIUM THIOSULFATE INJECTION)

Fennec Pharmaceuticals Inc. has received approval from the U.S. Food and Drug Administration (FDA) for Pedmark (sodium thiosulphate injection) to reduce the risk of ototoxicity associated with cisplatin in pediatric patients one month of age and older with localized, non-metastatic solid tumours. This approval makes Pedmark the first and only treatment approved by the FDA in this area of significant unmet medical need.

"The FDA approval of Pedmark represents an important breakthrough for pediatric patients with localized, non-metastatic solid tumours at risk for cisplatin-induced hearing loss. Cisplatin is a critical, standard-of-care agent, used in the treatment of pediatric cancers; however, even though effective, it could be harmful to children, frequently causing permanent and irreversible bilateral hearing loss. With Pedmark, physicians now have an approved treatment option to reduce the risk of cisplatin-induced hearing loss in pediatric patients," said Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. "We would like to thank the patients, their families, physicians, investigators, employees, consultants and the entire research team at Oregon Health and Science University, who have contributed to the development of Pedmark."

The FDA approval of Pedmark was based upon safety and efficacy data from two pivotal open-label, randomized phase 3 trials (SIOPEL 6 and COG ACCL0431), which compared Pedmark 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 Pedmark plus cisplatin arm compared with the cisplatin alone arm (21.4 per cent versus 73.3 per cent (p equals 0.005) and 32.7 per cent versus 63 per cent (p equals 0.002) 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 more than 5 per cent compared with cisplatin alone) in SIOPEL6 are 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 more than 5 per cent compared with cisplatin alone) in COG ACCL0431 is hypokalemia.

"Historically, there have been no approved treatments for preventing cisplatin-induced hearing loss. As a physician focused in pediatric cancer for many years and a primary investigator in the pivotal Pedmark phase 3 clinical oncology group (COG) trial, the FDA approval of Pedmark addresses an enormous unmet need, and for many children and young adults, has the potential to greatly improve everyday activities for these patients," said David R. Freyer, DO, MS, primary investigator, COG ACCL0431, and director of the Survivorship & Supportive Care program, Cancer and Blood Disease Institute, Children's Hospital Los Angeles.

Advances in chemotherapy-based treatment approaches for pediatric patients with localized, solid tumours have improved, resulting in an 85-per-cent-or-higher five-year survival rate for these patients. However, use of platinum-based chemotherapy, still the treatment of choice in many cases, can be toxic to the ears and cisplatin treatment frequently causes permanent and irreversible bilateral (affecting both ears) hearing loss. Permanent hearing loss can be seen in approximately 60 per cent of children treated with cisplatin and can be as high as 90 per cent. Until now, interventions with management strategies such as cochlear implants and hearing aids only occurred after hearing loss had been detected and these interventions do not return normal hearing.

"Hearing loss can have a profound impact on a person's life, especially in children who are critically dependent upon normal hearing for cognitive, psychosocial and speech development," said Penelope R. Brock, MD, PhD, of Great Ormond Street Hospital in London and international chair of the SIOPEL 6 trial. "Incorporating Pedmark into current treatment strategies with the goal to preserve hearing in children and young adults without reducing the effectiveness of their cisplatin treatment -- is a welcome step towards helping to improve long-term outcomes for these patients."

For more information about product availability and patient support, please contact the Fennec Hears program at 1-833-7Pedmark (1-833-773-3627).

The FDA granted this application priority review designation. Pedmark also received orphan drug designation by the FDA in 2004.

The marketing authorization application (MAA) for sodium thiosulphate (tradename Pedmarqsi) is currently under evaluation by the European Medicines Agency (EMA).

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.

About Pedmark (sodium thiosulphate injection)

Pedmark is the first and only U.S. 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.

Find full prescribing information for Pedmark at the FDA website.

About Fennec Pharmaceuticals Inc.

Fennec Pharmaceuticals is a specialty pharmaceutical company focused on the development and commercialization of Pedmark for the prevention of platinum-induced ototoxicity in pediatric patients. Further, Pedmark received FDA approval in September, 2022, and has received orphan drug designation in the U.S. 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 prevention of ototoxicity induced by platinum chemotherapy, in humans.

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