8-K: Fennec Pharmaceuticals' PEDMARQSI Receives Positive NICE Recommendation in England and Wales

Fennec Pharmaceuticals announced that its partner, Norgine Pharmaceuticals, received positive draft guidance from NICE recommending PEDMARQSI for preventing cisplatin-induced hearing loss in young patients.

Summary

• Fennec Pharmaceuticals announced that Norgine Pharmaceuticals received positive draft guidance from the National Institute for Health and Care Excellence (NICE) for PEDMARQSI.
• PEDMARQSI is recommended for preventing cisplatin-induced hearing loss in patients aged 1 month to 17 years with localized, non-metastatic, solid tumors.
• This makes PEDMARQSI the first and only treatment available within NHS England and Wales for this condition.
• Clinical trials, SIOPEL 6 and COG Protocol ACCL0431, showed approximately a 50% reduction in ototoxicity with PEDMARQSI compared to cisplatin alone.
• Fennec received an upfront payment of approximately $43 million from Norgine and is eligible for up to $230 million in additional milestone payments, plus tiered royalties on net sales.
• PEDMARK, the US version of the drug, is already FDA approved and recommended by the National Comprehensive Cancer Network for similar use.

Sentiment

Score: 9

Explanation: The document is highly positive due to the significant regulatory approval, strong clinical data, and substantial financial benefits for Fennec. The positive NICE recommendation is a major milestone.

Positives

• The positive NICE recommendation significantly expands the market access for PEDMARQSI in England and Wales.
• The 50% reduction in ototoxicity shown in clinical trials is a major benefit for young cancer patients.
• The upfront payment of $43 million and potential for $230 million in milestone payments provide significant financial upside for Fennec.
• The tiered royalty structure ensures long-term revenue potential for Fennec.
• PEDMARQSI is the first and only approved therapy in the EU and UK for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localized, non-metastatic solid tumors.

Negatives

• The document highlights that PEDMARK is contraindicated in patients with a history of severe hypersensitivity to sodium thiosulfate or any of its components.
• Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials.
• Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case.
• Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients.
• The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours.

Risks

• Regulatory and guideline developments may change, potentially impacting the approval and use of PEDMARQSI.
• Scientific data and/or manufacturing capabilities may not be sufficient to meet regulatory standards.
• Clinical results may not be replicated in actual patient settings.
• Unforeseen global instability, including political instability, or instability from an outbreak of pandemic or contagious disease, such as the novel coronavirus (COVID-19), could impact the business.
• The company's patents and patent applications may be challenged, invalidated or circumvented by its competitors.
• The available market for the company's products may not be as large as expected.
• The company's products may not be able to penetrate one or more targeted markets.
• Revenues may not be sufficient to fund further development and clinical studies.
• The company may not be able to obtain necessary capital when needed on acceptable terms or at all.
• The company may not meet its future capital requirements in different countries and municipalities.

Future Outlook

The company anticipates continued commercialization of PEDMARK and PEDMARQSI, and expects to receive further milestone payments and royalties from Norgine. The company also mentions potential access to further funding after the date of this release.

Management Comments

• Jeff Hackman, chief executive officer and director of Fennec Pharmaceuticals, stated that securing access to PEDMARQSI is a critical milestone for the cancer community in England and Wales.
• Jeff Hackman congratulated Norgine on their collaboration with NICE to reach this important agreement.

Industry Context

This announcement highlights the growing focus on supportive care in oncology, particularly in mitigating the side effects of chemotherapy. The positive recommendation from NICE underscores the importance of preventing cisplatin-induced hearing loss in pediatric patients, aligning with broader efforts to improve the quality of life for cancer survivors.

Comparison to Industry Standards

• The 50% reduction in ototoxicity demonstrated by PEDMARQSI in clinical trials is a significant improvement compared to the standard of care, which previously had no preventative pharmacological interventions.
• While cochlear implants are available, they are expensive, technically difficult, and do not reverse hearing loss, making PEDMARQSI a more effective and less invasive option.
• The approval and recommendation of PEDMARQSI by NICE is a significant step forward in the treatment of cisplatin-induced ototoxicity, setting a new benchmark for care in this area.
• Other companies developing similar therapies include those focused on otoprotective agents, but PEDMARQSI is the first and only approved therapy in the EU and UK for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localized, non-metastatic solid tumors.

Stakeholder Impact

• Shareholders will benefit from the increased market access and potential revenue from PEDMARQSI.
• Patients and their families will benefit from the availability of a treatment to prevent cisplatin-induced hearing loss.
• Healthcare providers will have a new tool to improve the outcomes for young cancer patients.
• Norgine will benefit from the exclusive rights to commercialize PEDMARQSI in Europe, Australia, and New Zealand.

Next Steps

• Norgine will proceed with commercializing PEDMARQSI in England and Wales.
• Fennec will continue to work with Norgine to achieve further regulatory and commercial milestones.
• Fennec will continue to monitor the market and sales of PEDMARK and PEDMARQSI.

Key Dates

Keywords

PEDMARQSI, PEDMARK, ototoxicity, cisplatin, hearing loss, NICE, Norgine Pharmaceuticals, oncology, chemotherapy, pediatric, sodium thiosulfate