Heat Exhaustion

- U.S. Food and Drug Administration (FDA) provides a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec using data from ongoing Phase 1/2 STAAR study, avoiding requirement for additional registrational study and accelerating estimated time to potential approval by approximately three years. - FDA confirms that estimated glomerular filtration rate (eGFR) slope data at one year across all Phase 1/2 patients can serve as primary basis for approval under Accelerated Approval pathway. - Data to support Accelerated Approval pathway available in first half of 2025, with a potential Biologics License Application (BLA) submission expected in the second half of 2025. RICHMOND, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the outcome of a recent successful interaction with the U.S. FDA, providing a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease. The FDA has agreed in a Type B interaction that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. The complete dataset to support an Accelerated Approval pathway will be available in the first half of 2025. This approach unlocks a potential BLA submission in the second half of 2025, three years ahead of previous estimates, and avoids the requirement for an additional registrational study to establish clinical efficacy. Sangamo engaged with the FDA on alternative pathways to potential approval following analysis of clinical data from the Phase 1/2 STAAR study showing encouraging safety and efficacy data, including promising preliminary evidence of improved kidney function. Renal manifestations, such as proteinuria or a decreased glomerular filtration rate, occur early in life in almost all male, and in many female, patients with Fabry disease, and can lead to end-stage renal disease and early death. In the 18 male and female patients treated with isaralgagene civaparvovec with more than one year of follow-up data, statistically significant improvements were observed in both mean and median eGFR levels, resulting in a positive annualized eGFR slope. Based on these latest data, the FDA agreed that eGFR slope at 52 weeks can serve as an intermediate clinical endpoint to support a potential Accelerated Approval. The FDA also advised that eGFR slope at 104 weeks may be assessed to verify clinical benefit. “Fabry is a debilitating disease, for which there is a serious unmet medical need,” said Sandy Macrae, Chief Executive Officer of Sangamo. “I strongly believe in the potential for ST-920 to alleviate many manifestations of Fabry disease and am delighted to have a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated”. Dosing was completed in the Phase 1/2 STAAR study in April 2024, with a total of 33 patients dosed. The longest treated patient recently achieved four years of follow-up. The 18th and final patient who started the study on Enzyme Replacement Therapy (ERT) was successfully withdrawn from ERT in September 2024, and all 18 patients remain off ERT as of today. The 52-week eGFR slope data from all enrolled patients in the Phase 1/2 STAAR study will be available in the first half of 2025. A potential BLA submission is anticipated in the second half of 2025. Sangamo has begun to execute BLA readiness activities for isaralgagene civaparvovec, while continuing to advance ongoing business development discussions with potential collaboration partners. 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 male and female 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 and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency. 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 believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand 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 and connect with us on LinkedIn and 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 potential of isaralgagene civaparvovec; the potential for isaralgagene civaparvovec to qualify for the FDA’s Accelerated Approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the availability of additional data to support a potential BLA submission for isaralgagene civaparvovec, and the timing of such submission; the potential to accelerate the expected timeline to approval and bring isaralgagene civaparvovec to patients sooner than previously expected ; the anticipated advancement of isaralgagene civaparvovec to registration, including Sangamo’s plans to seek a potential collaboration partner; 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, including our ability to secure a partnership; 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, including that the 52-week data from the Phase 1/2 STAAR study will not support a BLA submission and/or that the 104-week data from such study will not verify the clinical benefit of isaralgagene civaparvovec or support FDA approval, and that the patients withdrawn from ERT will remain off ERT; 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 on the global business environment, healthcare systems and our business and operations; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; 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, 2023, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, 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. Contacts Investor Relations & Media Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com

FRIDAY, September 27, 2024 -- In a sign that climate change may be fueling heat illnesses in kids, a new study reveals that such visits to two Texas children's emergency rooms spiked 170% between 2012 and 2023. Study leader Dr. Taylor Merritt , a resident pediatrician at UT Southwestern Medical Center in Dallas, is to present the findings Sunday at the American Academy of Pediatrics (AAP) annual meeting in Orlando, Fla. While research not yet published in a peer-reviewed journal is considered preliminary, study co-author Dr. Andrew Yu , a pediatrician at UT Southwestern, said the findings likely reflect trends in other areas of the country where extreme heat is becoming more common. Children treated for heat-related emergencies at the two large hospitals in Dallas were seen for a variety of conditions, from heat cramps and heat stroke to life-threatening complications such as rhabdomyolosis, according to the study. "As extreme summer temperatures become more frequent due to a changing climate, we're seeing a rise in heat-related illnesses, particularly in vulnerable groups like children," Merritt said in an AAP news release. For the study, researchers looked at 542 heat-related visits to the ER. Nearly 8 in 10 patients (418) had heat-specific diagnoses and a quarter (131) were diagnosed with rhabdomyoloysis. Often called "rhabdo," it is a rare and potentially deadly condition in which muscles break down, often after excessive exercise or heat stress. Most of the kids (63%) with rhabdo had to be admitted to the hospital, the study found. Most children (96%) with diagnoses like heat exhaustion or heat stroke were treated and released. Children presenting with heat-specific health issues tended to be younger, Hispanic and living in disadvantaged neighborhoods, the study found. "As pediatricians, we see firsthand how excessive heat affects children's health," Merritt said. "The significant rise in heat-related illnesses over the past decade underscores a growing public health concern that warrants further attention and action." Yu said the findings underscore the importance for parents and pediatricians alike to understand how the changing climate can affect children's health and safety. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Excessive weather events are becoming more common and severe. More than 2,300 heat-related deaths, which are preventable, occurred in 2023. CVS Health has launched an environmental health impact program to help vulnerable Americans during extreme weather events.The program, already live for a few weeks, initially focuses on extreme heat. Using advanced environmental data analytics and patients' medical and pharmacy data, CVS Health offers timely excessive heat alerts and outreach to at-risk patients up to a week before the event. The initiative will expand to encompass air quality events this fall. It is initially available to members of Aetna with the goal of expanding to pharmacies and MinuteClinics.Excessive weather events are becoming more common and severe. More than 2,300 heat-related deaths occurred in 2023. They are preventable. Meanwhile, air pollution puts patients at risk of reduced lung function, asthma and cardiac problems.“Heat waves and our changing environment is just such a pressing public health threat. I don't think it’s appreciated nearly as much as it should be,” Dan Knecht, M.D., chief clinical innovation officer for CVS Caremark, told Fierce Healthcare.Knecht is also a hospitalist at Mount Sinai West and is seeing some patients coming in for heat stroke, but not all. Many come for worsening chronic conditions, which might be attributed to the natural progression of their disease but is really triggered by extreme weather, he said. That is why official figures on heat-related deaths in the U.S. are likely an undercount, he noted.“It’s really hard to untangle the morbidity associated with heat waves,” Knecht said. “We really don’t have a tremendous appreciation to how detrimental heat waves are to whole health.”Consulting with experts in climate change and public health, CVS Health determined a wet bulb temperature threshold in the mid-80s Fahrenheit. Ingesting third-party weather data, it determines regions that surpass the limit and uses its own algorithm to stratify Aetna members by their vulnerability to extreme heat. Care managers who are registered nurses then reach out by phone, using an evidence-based framework for navigating the conversations. They provide information on the symptoms of heat stroke, how to minimize heat exhaustion and local resources such as cooling centers. Many Oak Street Health centers, now owned by CVS Health, can be used as a safe space to gather during extreme temperatures, Knecht said.  There is also some room for CVS Health to address social needs. It offers home delivery of prescriptions during extreme heat events as needed. If a member needs transportation to a cooling center, they may be eligible through one of two programs for Aetna members: Access2Care and SafeRide Health. However, the company can’t guarantee transportation for all members. It can also loop in its Resources for Living team to address mental health conditions, which, like other health conditions, are exacerbated by extreme weather.During the first two weeks of the program, CVS Health care managers have connected with hundreds of patients across more than 20 states. In addition to improved health, anticipated outcomes include reduced medical expenses and minimized inpatient hospital stays and ED visits.So far, the company has been receiving positive feedback from those with whom care managers have connected. Many of these populations are high-risk and already familiar with Aetna care managers for things like discharge planning.Existing weather alerts are a “patchwork,” per Knecht, varying by state and lacking individualized health recommendations for at-risk individuals. “We feel like there's a big opportunity to go upstream in terms of time and be more proactive and individualized,” Knecht said. “Having that conversation with a clinician at an impactable moment is really powerful.”CVS Health hopes that down the line its pharmacists can also deliver extreme weather event messages to patients, either at the pharmacy or by phone.