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Stealth BioTherapeutics Announces SBT-272 Data Updates and Orphan Drug Designation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
SBT-272 Phase 1 safety and tolerability data support further clinical development
SBT-272 was neuroprotective and reduced neuroinflammation in ALS preclinical…
SBT-272 Phase 1 safety and tolerability data support further clinical development
SBT-272 was neuroprotective and reduced neuroinflammation in ALS preclinical model
SBT-272 Granted Orphan Drug Designation for Treatment of ALS
BOSTON, Nov. 1, 2022 /PRNewswire/ — Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced encouraging Phase 1 data on the safety and tolerability of SBT-272, a next-generation small molecule designed to reach therapeutic concentrations in the brain and to restore mitochondrial structure and function. The Company also announced that positive preclinical data demonstrating that treatment with SBT-272 provided neuroprotection of upper motor neurons harboring ALS pathology will be presented today at the Northeast Amyotrophic Lateral Sclerosis (NEALS) conference in Clearwater, FL. The Company recently received Orphan Drug Designation from the US Food and Drug Administration (FDA) Office of Orphan Products Development for SBT-272 for the treatment of patients with ALS.
“The preclinical data demonstrate that SBT-272 improves the stability and function of mitochondria in upper motor neurons that are diseased with TDP-43 pathology. This also provides neuroprotection and reduces neuroinflammation in the motor cortex of a TDP-43 model of ALS”, said Hande Ozdinler, PhD, Associate Professor of Neurology, Feinberg School of Medicine, Northwestern University. “There appears to be compelling support for the therapeutic potential of targeting mitochondria in ALS and the ongoing clinical development of SBT-272.”
Interim results from the Phase 1 study for SBT-272 demonstrated that selected doses are anticipated to result in therapeutic concentrations in the brain based on observed drug levels achieved in preclinical studies. While final safety analyses …
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