Sangamo Therapeutics Announces U.S. FDA Clearance of IND Application for ST-503 for the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain
SGMO 11.19.2024
Drug:ST-503-001 ST-503
Diseases:idiopathic small fiber neuropathy (iSFN)
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Sangamo plans toinitiate enrollment of patients in thePhase 1/2 study for ST-503 in mid-2025
Neuropathic pain can be caused by a broad array of pathologies impacting the central or peripheral nervous systems, such as surgical trauma, spinal cord injury, nerve compression, neurological and infectious diseases, or metabolic and hereditary syndromes. ST-503 is not intended for sporadic or acute pain, but for chronic, intractable pain that completely dominates and often destroys the lives of patients over many years. The Phase 1/2 study will assess the safety and efficacy of ST-503 in addressing iSFN, a peripheral neuropathy that results in highly debilitating symptoms of burning, prickling, stabbing or “lightning-like” pain. iSFN has an estimated prevalence of at least 43,000 patients in the
“The FDA’s clearance of the IND application to evaluate ST-503 in idiopathic small fiber neuropathy represents an important milestone for Sangamo on our journey to becoming a neurology genomic medicine company,” said
A significant body of evidence implicates sodium channels in mediating the pathophysiology of neuropathic pain. ST-503 uses an adeno-associated virus (AAV) vector carrying an engineered zinc finger repressor (ZFR) to specifically target the human gene,SCN9A, that encodes the Nav1.7 sodium channel and is critical for pain signaling. Developing small molecules that specifically target Nav1.7 is challenging due to the high structural similarities between different sodium channels, making it difficult to achieve selectivity and avoid off-target effects. By directly targeting theSCN9Agene, ST-503 was shown to selectively reduce the expression of Nav1.7 sodium channels in sensory neurons in animal models and significantly reduce pain hypersensitivity, following a single intrathecal administration of ST-503. Sangamo’s preclinical research has shown ST-503 to be well tolerated in nonhuman primates, with substantial Nav1.7 reduction observed with no off-target effects, demonstrating the promise of ST-503 as a potential therapy for chronic neuropathic pain, regardless of cause.
Sangamo is preparing for the Phase 1/2 clinical study to assess the safety, tolerability and preliminary efficacy of a one-time dose of ST-503, administered intrathecally to patients with intractable pain due to iSFN, and plans to initiate patient enrollment in mid-2025.
Sangamo believes that if this study is successful, the development of ST-503 could be broadened to patient populations suffering from other types of chronic neuropathic pain.
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Forward-Looking Statements
This press release contains forward-looking statements regarding our future plans and current expectations. These forward-looking statements include, without limitation, statements relating to: expectations concerning the ability of our zinc finger technology to address neurological conditions; the anticipated advancement of ST-503 to the clinic, including the timing related thereto and the potential for a Phase 1/2 study to assess its safety, tolerability and preliminary efficacy in addressing iSFN; the potential for ST-503 to treat chronic neuropathic pain, regardless of cause; the potential to broaden development of ST-503 to other patient populations; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to our lack of capital resources to obtain regulatory approval for and commercialize our product candidates in a timely manner or at all; the uncertain timing and unpredictable nature of clinical trial results; our need for substantial additional funding to execute our operating plan and to continue to operate as a going concern, including the need for adequate additional funding toinitiate patient enrollment inthe Phase 1/2 study of ST-503; the effects of macroeconomic factors or financial challenges on the global business environment, healthcare systems and our business and operations; the research and development process; the potential for technological developments that obviate technologies used by Sangamo; and our ability to achieve expected future financial performance.
All forward-looking statements about our future plans and expectations, are subject to our ability to secure adequate additional funding. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and our collaborators. These risks and uncertainties are described more fully in our
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