SONALA-001

Tumor Regression and Improved Patient Performance are Among the Early Findings OAKLAND, Calif.--(BUSINESS WIRE)-- SonALAsense, a pioneer in the development of non-invasive Sonodynamic Therapy (SDT) using SONALA-001 (aminolevulinic acid HCl) in combination with Insightec’s Exablate 4000 Type-2 ultrasound device today announced the presentation of positive preliminary data from the ongoing Phase 1/2 SDT-201 study in Diffuse Intrinsic Pontine Glioma (DIPG). These results were presented at the November 2023 Society for Neuro-Oncology (SNO) Annual Meeting in Vancouver, Canada. The preliminary data from Study SDT-201 shows signals of clinical activity and no safety concerns. Study SDT-201 is a dose escalation and expansion study in patients with DIPG. Six (6) patients have been treated to date and dose escalation is ongoing. No dose limiting toxicities (DLTs) have been observed and there have been no serious adverse events. There were no treatment related deaths observed. One international patient discontinued for personal reasons and there was one patient death due to metastasis outside of the treated area. “The preliminary safety, tumor response and clinical performance data we are seeing has the potential to translate into meaningful benefit for these children and their families,” said Ely Benaim, M.D., SonALAsense’s Chief Medical Officer and Executive Vice President of Development. “Currently, radiotherapy is the primary standard of care and many patients are also prescribed steroids. However, these current interventions have the potential for severe side effects.” Initial clinical activity observed in the first 5 patients showed partial responses of at least 25% reduction in tumor size in 2 (40%) patients through eight weeks post treatment and stable disease in 2 (40%) patients. Four patients treated have exceeded the median survival of 9-11 months post diagnosis. Patients treated have also maintained or improved their patient performance status. “We are pleased with the early results and the safety pro SONALA-001 SDT,” said Hasan R. Syed, M.D., pediatric neurosurgeon at Children’s National Hospital and co-lead in Principal Investigator Roger Packer’s trial. “Sonodynamic therapy is part of a larger trend towards less invasive treatment paradigms in neurosurgery. For families of children with DIPG, this trial offers hope and a chance to potentially alter the course of this aggressive disease.” “As we continue to learn more about our non-invasive therapy through this study, we are extremely encouraged with this preliminary safety and clinical activity data,” said Mark de Souza, CEO, SonALAsense. “Our team remains committed to the DIPG community by continuing to advance this study which shows promise in enhancing quality of life for these patients.” DIPGs are rare, aggressive, and deadly brain tumors affecting primarily the pediatric population. Approximately 300 children are diagnosed with DIPGs each year in the US, usually between the ages of 5 and 9. There are no effective treatments available beyond radiation, which provides minimal short-term clinical improvement, at best. DIPGs are not amenable to surgical resection. Lisa Ward, DIPG/DMG community advocate who lost her son to DIPG, is hopeful that this study will lead to a treatment with a better quality of life. “What these children and families go through is absolutely tragic,” said Ward. “The possibility of providing an incisionless, non-toxic approach with therapeutic benefits for these children, that’s everything.” 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 X (formerly Twitter) and LinkedIn and visit our website at sonalasense.com. Acknowledgements: Research reported in this press release was supported by the National Cancer Institute of the National Institutes of Health under Award Number R44CA261516. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. This research is also supported by the Food and Drug Administration (FDA) Office of Orphan Products Development under award R01FD007538. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA, or the U.S. Government. The research is also supported by the following patient advocacy organizations: DIPG/DMG Research Funding Alliance (DDRFA) which is administered by Tough2gether Against DIPG and is an operating fund of Arms Wide Open Childhood Cancer Foundation. Additional DDRFA members who supported the grant are Rally! Foundation, Tough2gether Against DIPG, Storm the Heavens Fund, Team Landon Clay Foundation, Mighty Madison DIPG Research and Awareness Fund, Arms Wide Open Childhood Cancer Foundation, Gold Rush Cure Foundation and Kinley Sexton Foundation. DIPG/DMG Collaborative, which is supported by The Cure Starts Now Foundation, Brooke Healey Foundation, Jeffrey Thomas Hayden Foundation, Melina Michelle Edenfield Foundation, Yuvaan Tiwari Foundation, The Cure Starts Now Australia, The Cure Starts Now Canada, Aubreigh’s Army Foundation 328, Reflections Of Grace Foundation, Run DIPG, Aidan's Avengers, Cure Brain Cancer, Musella Foundation, Whitley’s Wishes, Laurie’s Love Foundation, Love4Lucas Foundation, Love, Chloe Foundation, Lauren's Fight for Cure, The Isabella and Marcus Foundation, Ryan's Hope, The Gold Hope Project, Robert Connor Dawes Foundation, The DIPG/DMG Collaborative and Snapgrant.com, Rally Foundation for Childhood Cancer Research and Kids Join The Fight.

Encouraging preliminary data from the ongoing DIPG clinical study to be presented at the 2023 Society for Neuro-Oncology Annual Meeting in Vancouver OAKLAND, Calif.--(BUSINESS WIRE)-- 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.