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

SonALAsense, a pioneer in the development of non-invasive Sonodynamic Therapy (SDT) using SONALA-001, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to the development program of SONALA-001 in combination with the INSIGHTEC Exablate 4000 Type-2 Device for the treatment of patients with diffuse intrinsic pontine glioma (DIPG). This builds upon SONALA-001 also having received orphan drug designation in 2021 for malignant gliomas.

The FDA's Fast Track is a process designed to facilitate the development, and expedited review, of drugs to treat serious conditions and fill an unmet medical need. The program allows eligibility for more frequent meetings with the FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval, more frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers, and accelerated approval.

"Receiving Fast Track Designation underscores the immediate and urgent need to bring new therapy options for DIPG patients," said Ely Benaim, M.D., SonALAsense's Chief Medical Officer and Executive Vice President of Development. "We are committed to working as efficiently as possible to bring SONALA-001 SDT to patients with this universally fatal disease."

DIPG is a rare, aggressive, and deadly pediatric brain tumor that strikes the pons, which links the brain to the spinal cord and helps control breathing, blood pressure, heart rate and other vital functions. It diffuses through the pons in a way that makes surgery impossible. Approximately 300 children are diagnosed with DIPG each year in the US, usually between the ages of 5 and 9. The median survival is 7-11 months after diagnosis with no effective treatments available beyond radiation. The current standard of care with fractionated radiotherapy and supportive care show significant side effects and limited efficacy.

SONALA-001 is SonALAsense's proprietary intravenous aminolevulinic acid HCl (ALA) formulation. SonALAsense's SDT uses the energy of ultrasound to produce light within the brain to activate protoporphyrin IX, a metabolite of ALA. SONALA-001 SDT is currently being evaluated for safety and preliminary efficacy in clinical study SDT-201, an open label drug and energy dose ranging and expansion study (NCT 05123534). Preliminary data generated from the dose ranging cohort will be presented at a SonALAsense-hosted symposium at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting in Vancouver, Canada, November 16-19.

"We are excited for the opportunity to present promising results generated from our DIPG study to date," said Mark de Souza, SonALAsense CEO. 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."

About SonALAsense

SonALAsense is a clinical-stage company, developing a proprietary form of sonodynamic therapy (SDT) as a first-in-class, noninvasive drug-device combination to treat recurrent glioblastoma, diffuse intrinsic pontine glioma, and other life-threatening cancers. SDT uses SONALA-001 in combination with MR-guided focused ultrasound to selectively target and kill tumor cells. SONALA-001 has received Orphan Drug Designation for Malignant Gliomas. SONALA-001 in combination with the INSIGHTEC Exablate 4000 Type-2 ultrasound device has received Fast Track Designation for the treatment of patients with DIPG. Follow SonALAsense on Twitter and LinkedIn and visit our website at sonalasense.com.