SONALA-001/Transcranial Ultrasound Device Gets FTD in Pediatric Brain Cancer

SONALA-001 previously received orphan drug designation for malignant gliomas in 2021.

The FDA has granted fast track designation to SONALA-001 plus INSIGHTEC Exablate 4000 Type-2 Device—a transcranial ultrasound device for sonodynamic therapy—for the treatment of patients with diffuse intrinsic pontine glioma (DIPG), according to a press release from SonALAsense.

SONALA-001 previously received orphan drug designation for malignant gliomas in 2021.

“Receiving fast track designation underscores the immediate and urgent need to bring new therapy options for patients [with DIPG],” Ely Benaim, MD, chief medical officer and executive vice president of Development at SonALAsense, said in the press release. “We are committed to working as efficiently as possible to bring SONALA-001 SDT to patients with this universally fatal disease.”

The agent’s safety and efficacy are currently being assessed as part of the open-label, energy dose ranging and expansion phase 1/2 SDT-201 study (NCT05123534). Preliminary findings from the dose ranging cohort are set to be presented at the upcoming 2023 Society for Neuro-Oncology (SNO) Annual Meeting in Vancouver, Canada.

“We are excited for the opportunity to present promising results generated from our DIPG study to date,” Mark de Souza, SonALAsense chief executive officer at SonALAsense, explained in the press release. “We are fully committed in our efforts to provide hope to this group of patients of the utmost medical need, and to bring this novel treatment to patients as quickly as possible.”

Intravenous SONALA-001 is an aminolevulinic acid HCI formulation (ALA), while the non-invasive sonodynamic therapy utilizes ultrasound to produce light in the brain and activate protoporphyrin IX, which is a known metabolite of ALA.

The SDT-201 study’s primary end point was to determine SONALA-001’s safety, tolerability, and maximum tolerated dose, with secondary end points including pharmacokinetics, progression-free survival, duration of response, and overall survival.

The study includes several patient cohorts, with cohort 1 receiving 5 mg/kg of SONALA-001, cohort 2 receiving 5 mg/kg, and cohort 3 receiving 10 mg/kg; all intervention arms also included use of an MR-guided focused ultrasound device.

To be included on the study, patients were required to have newly diagnosed DIPG and receive previous treatment with at least 54 Gy of standard focal radiotherapy over the course of 42 to 49 days. Patients also needed to be 4 to 24 weeks post-radiotherapy and recover from adverse effects down to grade 1 or baseline prior to day 1. Additionally, patients needed to have adequate organ and bone marrow function, as well as cardiac function.

Those who showed signs of having progressive disease, required increasing steroid dosage prior to day 1, or a diagnosis of porphyria were ineligible for the study. Additional exclusion criteria included having a hypersensitivity to porphyrins; other current or past malignancies, physical or psychiatric illness, or any other medical disorder requiring treatment; or a known history of human immunodeficiency virus. Patients also couldn’t participate following use of potentially phototoxic substance, or receipt of previous or concurrent anticancer or investigational therapies.

Use of fish oil supplements within 24 hours of day 1 is allowed provided that patient’s clotting factor is within normal limits. Use of blood thinners within 7 days of day 1, diagnosis of acute neurologic status deterioration, or an inability to undergo MRI were also grounds for exclusion.

Reference

FDA grants fast track designation to SonALAsense’s SONALA-001 in combination with the INSIGHTEC transcranial ultrasound device for sonodynamic therapy of DIPG, a rare pediatric brain cancer. News release. SonALAsense. November 14, 2023. Accessed November 15, 2023. https://bit.ly/40C29WH