On August 25, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported the dosing of the first participant at the highest dose level of 375MBq/kg body weight in the final dose escalation cohort of the Phase I/IIa theranostic trial, CL04 (Press release, Clarity Pharmaceuticals, AUG 25, 2023, View Source [SID1234634679]). No issues were observed during the administration of 67Cu SARTATE and the participant continues to be followed for further safety and efficacy assessments as per protocol. The product administered to the participant was the first patient dose manufactured using copper-67 produced by NorthStar using their commercial scale manufacturing process.
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The CL04 trial (NCT04023331)1 is a theranostic trial evaluating 64Cu/67Cu SARTATE for diagnosis and treatment of high-risk neuroblastoma, an aggressive childhood cancer. It is a multi-centre, dose-escalation, open label, non-randomised, Phase I/IIa clinical trial with up to 34 participants conducted across eight clinical sites in the US.
Cohort 4 is the highest dose level cohort in the dose escalation phase of the CL04 trial. The aim of the dose escalation phase is to determine the optimal dose of 67Cu SARTATE that can be safely administered. Once the optimal dose is established, the trial will progress to the dose expansion phase where an additional 10 participants will receive at least 2 administrations of 67Cu SARTATE. Participants who demonstrate therapeutic benefit may be offered up to 4 therapy cycles of 67Cu SARTATE in total.
Some participants in the earlier cohorts have received additional therapy cycles of 67Cu SARTATE in addition to the single therapy cycle administered under the dose escalation phase of the CL04 trial. These subsequent therapy cycles are strictly contingent on the investigators’ assessment that the participant is demonstrating therapeutic benefit after the first dose.
Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are very excited to have dosed the first participant in this highest dose level cohort of our theranostic neuroblastoma trial in the US, having successfully completed cohort 3 very recently. We hope that the increase in administered activity between cohorts 3 and cohort 4 will have a significant impact on radiation-sensitive disease, such as neuroblastoma, and we look forward to generating the data from the 375MBq/kg body weight dose.
"High-risk neuroblastoma is a devastating cancer that predominantly affects children and has incredibly poor prognosis as there are few treatment options available to these patients with late-stage disease. At Clarity, we are dedicated to actively progressing the CL04 trial in the hope that our SARTATE product will deliver significant improvements to the diagnosis and treatment of children with this insidious disease in need of highly effective treatments."
The first 67Cu SARTATE dose, administered in cohort 4 of the CL04 trial, was manufactured using copper-67 produced by NorthStar, the first commercial-scale supplier of this therapeutic radioisotope. NorthStar’s copper-67 is manufactured using a scalable, efficient and environmentally preferable process based on electron accelerator technology. It has unique advantages compared to current-generation radiopharmaceuticals as they rely on aging nuclear reactors mainly outside the US.
NorthStar Medical Radioisotopes’ President and Chief Executive Officer, Frank Scholz, Ph.D., commented, "We are very pleased and proud to be part of this important milestone, producing and supplying our first batch of Cu-67 for human use for the treatment of some of the most vulnerable patients, children with high-risk neuroblastoma. We are excited to be supporting Clarity’s clinical development programs with copper-67 based therapeutic radiopharmaceuticals across their pipeline of Targeted Copper Theranostic products, with the mutual goal of improving treatment outcomes for children and adults with cancer."
Dr Taylor said, "We are pleased to be working with NorthStar as we develop next-generation theranostics with efficacious, scalable, sustainable and cost-effective supply chain that will enable the expansion of radiopharmaceuticals into the large, global oncology market."
About SARTATE
SARTATE is a next generation, highly targeted theranostic radiopharmaceutical. It is being developed for diagnosing, staging and subsequently treating cancers that express somatostatin receptor 2 (SSTR2), including neuroblastoma and neuroendocrine tumours (NETs). Like all Clarity products, the SARTATE product can be used with copper-64 (64Cu) for imaging (64Cu SARTATE) or copper-67 (67Cu) for therapy (67Cu SARTATE).
In 2020, the US Food and Drug Administration (FDA) awarded Clarity two Orphan Drug Designations (ODDs), one for 64Cu SARTATE as a diagnostic agent for the clinical management of neuroblastoma and one for 67Cu SARTATE as a therapy of neuroblastoma, as well as two Rare Paediatric Disease Designations (RPDDs) for these products. Should Clarity be successful in achieving US FDA New Drug Applications for these two products, RPDDs may potentially allow the Company to access a total of two tradeable Priority Review Vouchers (PRVs).
64Cu SARTATE and 67Cu SARTATE are unregistered products. Individual results may not represent the overall safety and efficacy of the products. The data outlined in this announcement has not been assessed by health authorities such as the US Food and Drug Administration (FDA). A clinical development program is currently underway to assess the efficacy and safety of these products. There is no guarantee that these products will become commercially available.
About Neuroblastoma
Neuroblastoma most often occurs in children younger than 5 years of age and presents when the tumour grows and causes symptoms. It is the most common type of cancer to be diagnosed in the first year of life and accounts for around 15% of paediatric cancer mortality2. High-risk neuroblastoma accounts for approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rates at 40%-50%.