Fennec Announces Positive Results From Phase 3 SIOPEL 6 Study on PEDMARK™ (sodium thiosulfate) Presented at the 49th Congress of the International Society of Pediatric Oncology (SIOP) 2017 Meeting
- Study met primary endpoint (p=0.0033)
- Significant reduction in cisplatin induced hearing loss without any evidence of tumour protection in patients with Standard Risk Hepatoblastoma (SR-HB)
- Company plans to pursue regulatory approvals with
Top Line Efficacy Data
The SIOPEL 6 study met its primary endpoint. The study demonstrated that the addition of STS significantly reduces the incidence of cisplatin-induced hearing loss without any evidence of tumour protection. Among the 99 evaluable patients, hearing loss occurred in 30/45=67% treated with Cisplatin (Cis) alone and in 20/54=37.0% treated with Cis+STS, corresponding to a relative risk of 0.56(P=0.0033).
Fennec plans to pursue regulatory approval for PEDMARKTM based on the data from the SIOPEL 6 study along with the proof of principle data from COG ACCL0431. STS has received Orphan Drug Designation in the US in this setting and plans to pursue European Market Exclusivity for Pediatric Use upon approval.
“I am absolutely delighted that after 30 years of research we have found a safe way to reduce ototoxicity in children receiving platinum containing chemotherapy,” said Penelope Brock, M.D., PhD, International Chair of SIOPEL. “This means that children who are cured from cancer after receiving platinum treatment can look forward to a normal healthy life, fully integrated into society. I believe this marks a new standard of care in pediatric oncology."
The Company also reported top-line data for secondary endpoints Event Free Survival (EFS) and Overall Survival (OS). The combination of Cis+STS was generally well tolerated. With a follow up of 52 months, 3yr EFS is Cis 78.8% and Cis+STS 82.1%; 3yr OS is Cis 92.3% and Cis+STS 98.2%.
"We are very pleased with the results of this study," stated
Safety and Tolerability
In the study, the results presented showed that treatment was well tolerated and acute toxicity similar and expected between arms. The table below presents the toxicities of the two arms:
SIOP 2017 Presentation
Fennec will provide access to the recording of SIOP 2017 late breaker presentation on the Company's website.
To access the archived recording, visit the Fennec website at www.fennecpharma.com.
SIOPEL 6 is a multi-centre open label randomized phase 3 study evaluating the efficacy of STS in reducing ototoxicity in patients receiving cisplatin monotherapy for standard risk hepatoblastoma. From the beginning of 2007 to the end 2014, 52 sites from 11 countries enrolled 113 evaluable patients. The study is closed to recruitment and all protocol pre-specified IDMC safety reviews are now complete. The primary efficacy hearing endpoint analysis can be performed once patients have reached 3.5 years of age and an audiometry test can be carried out. The SIOPEL 6 study trial was designed with 80% power and a 5% significance level to detect an absolute 25% reduction in the rate of Brock grade ≥1 hearing loss with a chi-square test, from a 60% hearing loss in Cis alone arm to a 35% hearing loss in Cis+STS arm. The primary endpoint is the rate of Brock grade ≥ 1 hearing loss determined after the end of treatment at the age of ≥3.5 years by pure tone audiometry.
About PEDMARKTM (sodium thiosulfate/STS)
Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity in many patients, and are particularly harmful to the survivors of pediatric cancer.
In the U.S. and
STS has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies are closed to recruitment. The COG ACCL0431 protocol enrolled one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. COG ACCL0431 final results were published in the Lancet Oncology.
Forward looking statements
Except for historical information described in this press release, all other statements are forward-looking. Forward-looking statements are subject to certain risks and uncertainties inherent in the Company’s business that could cause actual results to vary, including such risks that regulatory and guideline developments may change, scientific data may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results may not be replicated in actual patient settings, protection offered by the Company’s patents and patent applications may be challenged, invalidated or circumvented by its competitors, the available market for the Company’s products will not be as large as expected, the Company’s products will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical studies, the Company may not meet its future capital requirements in different countries and municipalities, and other risks detailed from time to time in the Company’s filings with the
The scientific information discussed in this news release related to PEDMARKTM is preliminary and investigative. Such product candidates are not approved by the
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Chief Executive Officer
T: (919) 636-5144
Source: Fennec Pharmaceuticals