8-K: Fennec Pharmaceuticals Expands PEDMARK Use to Adult Testicular Cancer

Fennec Pharmaceuticals announced an investigator-sponsored clinical study to evaluate PEDMARK for preventing cisplatin-induced hearing loss in adult men with metastatic testicular germ cell tumors.

Summary

• Fennec Pharmaceuticals Inc. announced the planned initiation of an investigator-sponsored clinical study (IST) by City of Hope.
• The study will evaluate PEDMARK (sodium thiosulfate injection) for preventing cisplatin-induced ototoxicity (CIO) in adult men with stage II-III metastatic testicular germ cell tumors.
• The Phase I study (ClinicalTrials.gov Identifier: NCT07218913) is titled "Testing the Addition of PEDMARK to Cisplatin Chemotherapy for Reducing Drug-Induced Ear Damage in Men With Stage II-III Metastatic Testicular Germ Cell Tumors."
• PEDMARK is currently approved in the U.S. for pediatric patients one month of age and older with localized, non-metastatic solid tumors.
• PEDMARK has received a Category 2A recommendation from the National Comprehensive Cancer Network (NCCN) for use in adolescent and young adult patients.
• Multiple other investigator-initiated studies have been submitted and are currently under internal review, with others in advanced contracting or evaluation stages.

Sentiment

Score: 7

Explanation: The announcement of a new investigator-sponsored study for PEDMARK in an adult population is a positive development, indicating expanding clinical interest and potential market expansion. While it's an early-stage study, it addresses a significant unmet need and leverages an already approved drug. The risks are standard for a pharmaceutical company.

Positives

• Initiation of a new investigator-sponsored study expands the potential market for PEDMARK into adult metastatic testicular germ cell tumors.
• The study addresses a significant unmet medical need, as cisplatin causes permanent hearing loss in 60-90% of patients, impacting quality of life.
• Growing clinical interest in addressing hearing loss among patients receiving cisplatin-based chemotherapy.
• Potential to change the standard of care for adult patients and expand the evidence base for PEDMARK into metastatic disease.
• Additional investigator-initiated studies are in progress, indicating broader interest and potential future growth.

Risks

• Regulatory and guideline developments may change.
• Scientific data and/or manufacturing capabilities may not be sufficient to meet regulatory standards or receive required regulatory clearances or approvals.
• 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), or surrounding the duration and severity of an outbreak.
• Protection offered by patents and patent applications may be challenged, invalidated or circumvented by competitors.
• The available market for products will not be as large as expected.
• Products will not be able to penetrate one or more targeted markets.
• Revenues will not be sufficient to fund further development and clinical studies.
• Inability to obtain necessary capital when needed on acceptable terms or at all.
• Failure to meet future capital requirements in different countries and municipalities.

Future Outlook

The company anticipates that the investigator-initiated study will strengthen its clinical and commercial foundation and potentially expand the evidence base for PEDMARK into metastatic disease. They expect to share additional details as other investigator-initiated programs progress. The company also mentions potential future commercial and regulatory milestone and royalty payments from Norgine, and potential access to further funding.

Management Comments

• Alexander Chehrazi-Raffle, M.D., City of Hope: "Through this investigator-initiated study, City of Hope aims to better understand how protecting hearing can become an integral part of comprehensive cancer care."
• Pierre S. Sayad, PhD, M.S., Chief Medical Officer of Fennec Pharmaceuticals: "Initiation of this study by City of Hope reflects the growing clinical interest in addressing the burden of hearing loss among patients receiving cisplatin-based chemotherapy, an indispensable treatment with high survival rates but significant long-term quality-of-life consequences."
• Pierre S. Sayad, PhD, M.S.: "With cancer rates rising, especially among younger populations, the future of oncological care needs to focus on both survival and survivorship."
• Pierre S. Sayad, PhD, M.S.: "Fennec is proud to see independent investigators driving important research that not only has the potential to change the standard of care for their patients today but also expand the evidence base for PEDMARK into metastatic disease, where cisplatin is widely used but hearing protection options remain limited."

Industry Context

Cisplatin-based chemotherapy is a highly effective treatment for solid tumors, including germ cell tumors, but causes significant, often permanent, hearing loss (ototoxicity) in 60-90% of patients. This impacts long-term quality of life, academic performance, social development, and career potential. The study reflects a growing industry focus on "survivorship" in oncology, addressing long-term side effects of life-saving treatments, especially as cancer rates rise among younger populations. PEDMARK is currently the only FDA-approved therapy for this indication in pediatric patients, and this study aims to extend its use to adults, where options are limited.

Comparison to Industry Standards

• Cisplatin has transformed outcomes for patients with germ cell tumors, turning a highly fatal disease into one of oncology's true success stories.
• As many as four out of five survivors are left with permanent hearing loss that impacts their quality of life long after treatment ends.
• Studies show that between 60-90% of patients treated with cisplatin may develop hearing loss, depending upon the dose and duration of chemotherapy.
• Many of those treated with cisplatin will require lifelong hearing aids or cochlear implants.
• 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.
• PEDMARK is recommended for the adolescent and young adult (AYA) population by the National Comprehensive Cancer Network (NCCN) with a 2A endorsement.
• Approximately 500,000 patients in the U.S. are diagnosed annually with cancers that could be treated with a platinum-based chemotherapy.
• Until the FDA approval of PEDMARK, there were no preventative agents for this hearing loss.
• Patients with hearing loss resulting from cancer treatment have a statistically significant worse quality of life compared with peers who have no hearing loss.

Stakeholder Impact

• Shareholders: Potential for increased market opportunity and future revenue streams if the study is successful and leads to expanded indications.
• Patients (Adult Men with Testicular Germ Cell Tumors): Potential for a new treatment option to prevent debilitating hearing loss associated with life-saving cisplatin chemotherapy, significantly improving quality of life.
• Healthcare Providers/Oncologists: Provides a potential new tool to manage a common and severe side effect of cisplatin, aligning with a focus on comprehensive cancer care and survivorship.

Next Steps

• Conduct the Phase I investigator-sponsored study at City of Hope.
• Progress other investigator-initiated studies currently under review or in advanced contracting/evaluation stages.
• Share additional details as these programs progress.

Key Dates

Recommendation

The initiation of an investigator-sponsored study for PEDMARK in adult metastatic testicular germ cell tumors is a positive long-term development, expanding the drug's potential market. However, it is a Phase I study, meaning results are far off and success is not guaranteed. While it addresses a significant unmet need and leverages an existing approved drug, the immediate impact on financials is limited. The company has existing commercialization efforts for pediatric use and international markets. Given the early stage of this new indication, a "hold" recommendation is appropriate, acknowledging the positive long-term potential while awaiting further clinical data and commercial progress.

Keywords

Fennec Pharmaceuticals, PEDMARK, sodium thiosulfate, cisplatin, ototoxicity, hearing loss, testicular cancer, germ cell tumors, City of Hope, clinical study, oncology, pediatric, adult, metastatic, cancer treatment, specialty pharmaceutical