Fennec Pharmaceuticals (NASDAQ:FENC) is a specialty pharmaceutical company built on the mission of addressing one of the most significant and long-standing unmet needs in pediatric oncology: the prevention of chemotherapy-induced hearing loss. With its headquarters in Research Triangle Park, North Carolina, the company has spent years developing therapies aimed at improving the quality of life for children undergoing cancer treatment, focusing particularly on the toxicities associated with platinum-based chemotherapies such as cisplatin. Over time, Fennec has established itself as a pioneer in the supportive care segment of oncology, distinguishing its work from traditional drug development by concentrating on survivorship outcomes rather than tumor-targeting therapies. This focus on reducing treatment-related toxicities reflects a deep commitment to improving long-term functional outcomes for pediatric cancer survivors, many of whom face lifelong cognitive and developmental challenges due to ototoxicity.
The company’s flagship therapy, PEDMARK, represents the culmination of decades of research in understanding cisplatin-induced hearing damage and exploring effective ways to prevent it without compromising the anticancer activity of the drug. PEDMARK is the first and only FDA-approved therapy specifically designed to reduce the risk of ototoxicity in pediatric patients receiving cisplatin for localized, non-metastatic solid tumors. Its development and approval mark a historic milestone in pediatric oncology, as no previous treatment had demonstrated the ability to protect hearing while maintaining full therapeutic efficacy of cisplatin. This achievement has placed Fennec Pharmaceuticals at the forefront of innovation within the niche field of chemotherapy supportive care, giving the company a unique and impactful foothold in a space where clinical needs have remained largely unmet for decades.
Fennec’s background is grounded in extensive clinical partnerships, collaborative research, and rigorous evaluation of pharmacologic approaches to mitigating treatment-related toxicity. Through relationships with pediatric oncology groups, global cancer centers, and leading researchers, the company has built a robust foundation of clinical evidence supporting its work. The pivotal Phase 3 studies ACCL0431 and SIOPEL-6, conducted across multiple continents, provided the scientific basis for PEDMARK’s approval by demonstrating significant reductions in hearing loss while confirming no interference with cisplatin’s antitumor effects. These studies helped position Fennec as a company with the scientific credibility and clinical rigor needed to make a meaningful impact on survivorship outcomes in childhood cancers.
As the company continues to expand its research footprint, Fennec remains focused on bringing PEDMARK to international markets, including Japan and other regions where cisplatin use is prevalent and ototoxicity prevention remains a critical unmet need. The company is also exploring additional therapeutic opportunities that align with its overarching mission of improving the lives of children and young adults undergoing cancer treatment. This commitment to enhancing survivorship quality sets Fennec apart in the biopharmaceutical industry, reflecting a deeper purpose that extends beyond tumor response rates and into long-term functional health.
With a strong foundation built on clinical evidence, scientific innovation, and a clear vision for global expansion, Fennec Pharmaceuticals continues to solidify its role as a leader in pediatric oncology supportive care. Its focus on protecting hearing and preserving quality of life underscores a broader dedication to ensuring that cancer survival does not come at the cost of a child’s future development, communication abilities, and long-term well-being. As the company advances its mission, it remains committed to addressing overlooked yet critically important dimensions of cancer treatment, shaping a future where survivorship and quality of life are inseparable outcomes for pediatric patients worldwide.
Fennec Pharmaceuticals Delivers Breakthrough STS-J01 Trial Results as PEDMARK Shows Powerful Hearing Loss Protection in Children
Fennec Pharmaceuticals Inc. (NASDAQ:FENC) has captured global attention following the release of compelling topline data from the Phase 2/3 STS-J01 clinical trial in Japan, a study that further validates the transformative potential of PEDMARK®, the company’s FDA-approved therapy designed to reduce cisplatin-induced ototoxicity in pediatric cancer patients. As the only approved treatment of its kind, PEDMARK has already reshaped the landscape of pediatric oncology in the United States, and these new results reinforce its importance across global cancer centers striving to protect young patients from life-altering hearing loss.
PEDMARK was developed to address one of the most damaging and irreversible side effects of cisplatin therapy: permanent hearing loss. Cisplatin remains a cornerstone chemotherapeutic agent used to treat a wide range of solid tumors, yet its ototoxicity risk threatens the long-term cognitive, social, and educational outcomes of young survivors. For nearly four decades, there has been no approved pharmaceutical capable of preventing this form of hearing damage without compromising the cancer-fighting activity of cisplatin. PEDMARK’s emergence as the first and only therapy targeting cisplatin-induced ototoxicity represents a watershed moment for pediatric oncology, and the latest clinical evidence provides even stronger justification for its expansion into global markets.
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New Clinical Data Demonstrate Dramatic Reductions in Hearing Loss Compared to Historical Cisplatin-Only Outcomes
The STS-J01 study enrolled 27 patients between ages 3 and 18 in the primary cohort, with an additional 6 patients in exploratory groups, marking the first large-scale evaluation of PEDMARK in Japanese pediatric and adolescent oncology patients. The trial’s results offer compelling evidence of PEDMARK’s protective impact. Hearing loss was observed in only 24 percent of patients using ASHA criteria and 16 percent using Brock grading. These results stand in stark contrast to the historically reported 56 percent to 63 percent hearing loss rates seen in cisplatin-only arms of prior pivotal studies such as ACCL0431 and SIOPEL-6.
The data were even more impressive in the 7-18 age subgroup, where hearing loss dropped to 19 percent (ASHA) and 14.3 percent (Brock). This remarkable reduction confirms the consistency of PEDMARK’s otoprotective effect across varying populations, geographical regions, and clinical protocols. It further underscores the significant unmet need the therapy fulfills, especially in cancer centers where audiologic monitoring remains limited or inconsistent.
Pediatric oncologists have long emphasized the devastating impact of hearing impairment on survivors’ language development, social functioning, and academic performance. For this reason, the Japanese results are not merely statistically significant—they represent a transformative shift in survivorship quality and offer compelling support for regulatory approval outside the United States.
PEDMARK Shows No Reduction in Antitumor Activity, Reinforcing Its Clinical and Regulatory Strength
One of the central concerns oncologists have historically held is whether protective agents might diminish the therapeutic effect of cisplatin. PEDMARK’s mechanism avoids this by introducing sodium thiosulfate after cisplatin has already bound and exerted its antitumor action. Pharmacokinetic results from the STS-J01 study validate this concept, showing no reduction in cisplatin exposure and no negative impact on treatment efficacy.
The overall tumor response rate approached 95 percent, reaffirming that PEDMARK allows clinicians to provide hearing protection without compromising survival outcomes. This distinction is essential for regulatory agencies evaluating new pediatric oncology therapies, and it positions PEDMARK as a uniquely safe, reliable, and clinically advantageous intervention.
The absence of treatment-related serious adverse events further strengthens PEDMARK’s profile. Across more than 200 adverse events recorded, none were attributed to PEDMARK, underscoring its tolerability and compatibility with intensive pediatric oncology regimens.
Global Expansion Opportunities Signal New Growth Potential for Fennec Pharmaceuticals
The compelling STS-J01 results have prompted Fennec to initiate plans for regulatory registration in Japan. Given the high prevalence of cisplatin use in Japanese pediatric oncology and the lack of approved otoprotective agents, the commercial opportunity is significant. Pediatric oncologists have described cisplatin-induced ototoxicity as one of the most urgent unmet needs in Japan, where thousands of children face lifelong auditory impairments each year due to their cancer treatment.
Fennec is also exploring licensing or partnership opportunities that may accelerate commercial launch in Japan and other Asian markets. With over 500,000 annual U.S. cancer diagnoses that could involve platinum-based chemotherapy, and similarly high usage rates globally, the potential expansion of PEDMARK into international markets represents a major growth catalyst for long-term revenue generation.
As a specialty pharmaceutical company focused on pediatric oncology innovations, Fennec’s strategy centers on addressing treatment-related toxicities that directly affect survivorship quality. PEDMARK embodies this mission, distinguishing the company as a developer of supportive care therapies that dramatically improve life outcomes for young patients.
Why PEDMARK Represents a New Standard of Care for Pediatric Cisplatin Therapy
Cisplatin’s effectiveness is undisputed, but its ototoxicity risk has imposed a lifelong burden on survivors who often require hearing aids or cochlear implants that do not fully restore natural hearing. Studies estimate that 60 to 90 percent of cisplatin-treated children experience some level of permanent hearing loss, an alarming statistic that underscores the importance of integrating PEDMARK into standard oncology protocols.
Before PEDMARK’s FDA approval, no therapeutic agent was proven to prevent cisplatin-induced hearing loss. Supportive interventions such as audiologic monitoring, dose adjustments, and communication accommodations were only reactive measures that did nothing to prevent damage. PEDMARK has changed that landscape entirely, offering oncologists a proven regimen supported by multiple randomized trials and real-world clinical evidence.
Its recommendation by the National Comprehensive Cancer Network (NCCN) with a 2A endorsement further solidifies its credibility. As ongoing studies continue to reinforce its benefits across diverse populations—including Japanese pediatric patients—the argument for global standard-of-care adoption becomes increasingly compelling.
Conclusion: PEDMARK’s STS-J01 Success Strengthens Fennec Pharmaceuticals’ Global Expansion Strategy
The successful Phase 2/3 STS-J01 results mark a defining milestone for Fennec Pharmaceuticals. PEDMARK has once again demonstrated superior protection against hearing loss, maintained cisplatin’s antitumor activity, exhibited strong tolerability, and delivered clinical outcomes that align with the needs of pediatric oncology centers worldwide. These achievements not only establish PEDMARK as a clinically essential therapy but also position Fennec as a rising leader in the field of oncology supportive care.
As Fennec Pharmaceuticals moves toward regulatory filings in Japan and evaluates licensing opportunities, the company is preparing for the next phase of PEDMARK’s global impact. With consistent trial results across continents and a validated safety and efficacy profile, PEDMARK is on track to become a transformative therapy in pediatric cancer care and a significant growth engine for FENC stock.
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