Here's some genuinely good news in pediatric cancer care: Fennec Pharmaceuticals Inc. (FENC) announced Tuesday that its drug Pedmark successfully protected young cancer patients from hearing loss without weakening their chemotherapy. That's the dream scenario when you're adding a protective agent to cancer treatment—you want the shield without sacrificing the sword.
The results come from the STS-J01 trial, an investigator-initiated Phase 2/3 study conducted in Japan. The trial enrolled 27 patients in the primary cohort (ages 3-18) plus six more in exploratory groups, all receiving Pedmark six hours after their cisplatin chemotherapy.
Pedmark holds the distinction of being the first and only FDA-approved therapy designed to reduce ototoxicity—that's medical speak for hearing loss—caused by cisplatin in pediatric patients one month and older with localized, non-metastatic solid tumors. Cisplatin and similar platinum-based chemotherapies are workhorses in cancer treatment, but they come with a painful tradeoff: they can permanently damage hearing, especially in kids.
The Numbers Tell a Compelling Story
The trial hit its primary endpoint with results that should turn heads. Among evaluable patients treated with Pedmark, only 24% experienced hearing loss using the American Speech-Language-Hearing Association criteria, and just 16% showed impairment on the Brock grade scale.
Compare that to Pedmark's earlier pivotal Phase 3 trials where kids got cisplatin alone: 56% developed clinically significant hearing loss, and 63% developed hearing impairment. That's a dramatic difference.
The protection was even more pronounced in the 7-18 age group, where hearing loss dropped to 19% by ASHA standards and 14.3% by Brock grading. For families facing childhood cancer, preserving quality of life matters enormously, and hearing loss can affect everything from education to social development.
No Compromise on Cancer Fighting
The critical question with any protective agent: does it accidentally protect the tumor too? The answer here is a clear no. Pharmacokinetic analyses showed no reduction in cisplatin exposure, meaning the chemotherapy reached its targets just as intended. The overall tumor response rate came in around 95%, confirming that Pedmark doesn't interfere with cisplatin's ability to fight cancer.
On the safety front, Pedmark was well-tolerated. Across more than 200 treatment-emergent adverse events reported during the study, not a single one was attributed to Pedmark itself.
What Comes Next
Fennec plans to pursue registration in Japan based on these results and is exploring partnering or licensing opportunities for Pedmark. Full study results will be presented at a future scientific conference and submitted for peer-reviewed publication.
Fennec Pharmaceuticals shares traded down 0.78% to $7.85 at the time of publication Tuesday.