Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease
- FDA agrees to single study with up to 25 patients as basis for BLA approval for isaralgagene civaparvovec, reducing complexity and time to potential approval.
- EMA grants PRIME eligibility to isaralgagene civaparvovec, enhancing regulatory support and expediting review and approval processes.
- ST-920 gene therapy product candidate for Fabry disease receives Orphan Medicinal Product designation from EMA and Orphan Drug, Fast Track, and RMAT designations from FDA.
- Updated Phase 1/2 STAAR study data presented at WORLDSymposium shows sustained clinical benefit and differentiated safety profile across 24 patients.
- All 13 patients withdrawn from ERT in the study remain off ERT as of February 12, 2024.
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The FDA's indication that a single study may suffice for a BLA submission for isaralgagene civaparvovec represents a significant shift in the regulatory landscape for gene therapies targeting rare diseases. This streamlined approach could lead to a faster and more cost-effective route to market, which is particularly relevant for Sangamo Therapeutics. The company's focus on genomic medicines, such as gene therapy for Fabry disease, is part of a strategic area in biotechnology with high growth potential. The reduced complexity of the trial process could enhance Sangamo's ability to allocate resources efficiently and potentially improve its financial performance by shortening the time to market and reducing development costs.
Furthermore, the EMA's PRIME eligibility suggests a similar willingness to expedite the development and review process in Europe. This bodes well for the international commercial potential of the therapy. The orphan drug status and other designations by the FDA and EMA underscore the unmet need in Fabry disease treatment and the potential for isaralgagene civaparvovec to address this gap. These regulatory milestones could positively influence investor sentiment and potentially lead to an increase in Sangamo's market valuation, reflecting the anticipated future cash flows from the new therapy, should it be approved.
The regulatory updates from both the FDA and EMA for Sangamo's gene therapy candidate isaralgagene civaparvovec could have substantial implications for the healthcare sector, specifically within the treatment paradigm for Fabry disease. The current standard of care involves lifelong enzyme replacement therapy, which is costly and may not halt disease progression. A gene therapy that offers a one-time treatment could disrupt the market, shifting revenues from chronic therapies to potentially curative treatments. The market dynamics for Fabry disease therapies would thus undergo a significant transformation, with Sangamo positioning itself as a potential leader in this niche market.
The industry should take note of the regulatory agencies' willingness to consider alternative pathways to approval for treatments targeting rare diseases. This could encourage innovation and investment in gene therapy and other advanced therapy medicinal products (ATMPs). Analysts will be closely monitoring Sangamo's ability to secure a collaboration partner, as this will be critical to the therapy's successful development and commercialization. The partnership strategy could also serve as a model for other biotech firms with promising but resource-intensive therapies.
The financial implications for Sangamo Therapeutics following the FDA's and EMA's regulatory updates are multifaceted. The potential for a more efficient pathway to BLA submission could lead to significant cost savings in clinical development, an aspect that is often one of the largest expenses for biotech companies. Investors will likely assess the reduced risk profile associated with a single trial for approval, which could lead to a reevaluation of Sangamo's risk-adjusted discount rates and consequently, its intrinsic valuation.
However, it's important to note that Sangamo's decision to defer further investment in a registrational trial until a collaboration partner is found introduces a variable that could impact the timeline and financial projections. The ability to secure a partnership will be critical to advancing the therapy without overextending Sangamo's financial resources. A successful partnership could bring not only capital but also expertise in commercialization, which is essential for market penetration and revenue generation. The market will be watching for partnership announcements, as these could serve as a catalyst for stock price movement.
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- European Medicines Agency (EMA) granted priority medicines (PRIME) eligibility to isaralgagene civaparvovec, which includes enhanced regulatory support and scientific guidance.
- Sangamo is actively seeking a collaboration partner to advance isaralgagene civaparvovec through potential registration and commercialization.
The FDA has agreed in a Type D meeting that data from a single, adequate, and well-controlled study may form the primary basis of approval of a BLA for isaralgagene civaparvovec. The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm. A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA. This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.
Additionally, the EMA has granted PRIME eligibility to isaralgagene civaparvovec. PRIME is a program designed to enhance support for the development of medicines that target an unmet medical need and is intended to optimize development plans and expedite review and approval processes so that these medicines may reach patients as early as possible. Isaralgagene civaparvovec has already received Orphan Medicinal Product designation from the EMA as well as Orphan Drug, Fast Track and RMAT designations from the FDA.
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Updated Phase 1/2 STAAR study data showing sustained clinical benefit and a differentiated safety profile across 24 patients were shared at the 20th Annual WORLDSymposiumTM in
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate the safety and tolerability of isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. The STAAR study enrolled patients who are on ERT, are ERT pseudo-naïve (defined as having been off ERT for six or more months), or who are ERT-naïve. The FDA has granted Orphan Drug, Fast Track and RMAT designations to isaralgagene civaparvovec, which has also received Orphan Medicinal Product designation and PRIME eligibility from the EMA.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo’s zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and Sangamo’s capsid discovery platform is making progress toward potentially expanding delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter/X.
Forward-Looking Statements
This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic and commercial potential of isaralgagene civaparvovec, the anticipated plans and timelines for conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, expectations regarding the conclusion of dosing in our Phase 1/2 STAAR study, the anticipated advancement of isaralgagene civaparvovec to late-stage development, including Sangamo’s plans to seek a potential partner to proceed with potential future registrational studies of isaralgagene civaparvovec, the design of any potential future studies of isaralgagene civaparvovec, the potential impact of FDA and EMA feedback on the regulatory pathway for isaralgagene civaparvovec , 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 fully develop, obtain regulatory approval for and commercialize our product candidates, including our ability to secure a partnership required to initiate a potential registrational study of isaralgagene civaparvovec in a timely manner or at all; our need for substantial additional funding to execute our operating plan and to continue to operate as a going concern; the effects of macroeconomic factors or financial challenges, including as a result of the ongoing overseas conflict, current or potential future bank failures, inflation and elevated interest rates, on the global business environment, healthcare systems and business and operations of Sangamo and our collaborators, including the operation of clinical trials; the research and development process, including the operation and results of clinical trials and the presentation of clinical data; the impacts of clinical trial delays, pauses and holds on clinical trial timelines and commercialization of product candidates; the uncertain timing and unpredictable nature of clinical trial results, including the risk that the therapeutic effects observed in the latest preliminary clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, and that the patients withdrawn from ERT will remain off ERT; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results, including the results of any registrational studies of our product candidates; the potential for technological developments that obviate technologies used by Sangamo; our reliance on collaborators and our potential inability to secure additional collaborations, and our ability to achieve expected future financial performance.
There can be no assurance that we and our current or potential future collaborators will be able to develop commercially viable products. 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 Securities and Exchange Commission, or SEC, filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2022, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and we undertake no duty to update such information except as required under applicable law.
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Investor Relations & Media Inquiries
Louise Wilkie
ir@sangamo.com
media@sangamo.com
Source: Sangamo Therapeutics, Inc.
FAQ
What regulatory updates did Sangamo Therapeutics (SGMO) receive for isaralgagene civaparvovec gene therapy?
What is the significance of the FDA agreeing to a single study with up to 25 patients for BLA approval?
What is the purpose of EMA granting PRIME eligibility to isaralgagene civaparvovec?
What designations has ST-920 gene therapy product candidate received from regulatory agencies?
What Phase 1/2 STAAR study data was presented at WORLDSymposium?
What is the status of patients withdrawn from ERT in the study?
