Lisata Therapeutics: Urgent Need for Improved CAH Treatments Highlighted at ENDO 2025
- Neurocrine's CAHtalog® reveals 95% of CAH patients face glucocorticoid treatment inconsistencies and fluctuating health statuses.
- Dr. Sanjay Keswani highlights the unpredictable disease control in CAH, necessitating innovative therapies beyond high-dose glucocorticoids.
- The CAHtalog® findings emphasize the urgent need for stable treatment options to better manage congenital adrenal hyperplasia.
New Insights into Congenital Adrenal Hyperplasia Treatment Unveiled at ENDO 2025
Neurocrine Biosciences, Inc. presents significant findings from its CAHtalog®, a comprehensive registry tracking patients with classic congenital adrenal hyperplasia (CAH) in the United States, at the Endocrine Society's Annual Meeting, ENDO 2025. The analysis reveals that around 95% of both adult and pediatric CAH patients have either been administered supraphysiologic doses of glucocorticoids or have experienced elevated androstenedione levels at least once during their treatment. This highlights critical inconsistencies in glucocorticoid treatment regimens and the fluctuating health statuses of patients, underscoring the pressing challenges in managing this complex condition.
Dr. Sanjay Keswani, Neurocrine's Chief Medical Officer, emphasizes the unpredictable nature of disease control among CAH patients. While high-dose glucocorticoid therapy is often necessary for addressing cortisol deficiency, it does not guarantee stable long-term hormonal control. The CAHtalog, developed in partnership with the CARES Foundation, aims to capture the longitudinal treatment patterns of CAH, focusing on glucocorticoid usage and androstenedione levels over time. The findings reflect the ongoing complexities associated with CAH management and reinforce the clinical need for innovative therapies that can effectively mitigate androgen excess without the reliance on high doses of glucocorticoids.
The findings from the CAHtalog® analysis underline a critical gap in current treatment strategies, revealing the necessity for advancements in therapeutic options that provide more stable disease control throughout a patient's life. The unpredictable nature of CAH not only poses challenges for individual patients but also highlights broader implications for healthcare providers managing this condition. With the current reliance on glucocorticoids, the need for alternative therapies becomes increasingly urgent, paving the way for future research and development efforts within the field.
In other relevant news, Waters Corporation and Becton, Dickinson and Company have announced a merger of BD's Biosciences & Diagnostic Solutions business with Waters. This strategic move is set to enhance Waters' presence in high-growth markets, with an estimated addressable market of approximately $40 billion. The merger, valued at around $17.5 billion, aims to drive innovation in biopharmaceutical and diagnostic solutions, thereby expanding operational capabilities and market reach.
Additionally, Senhwa Biosciences has garnered attention as its investigational drug Pidnarulex (CX-5461) receives selection by the U.S. National Cancer Institute for a five-year cancer research program. This initiative includes clinical trials targeting advanced solid tumors, substantially alleviating the financial burden of research and development for Senhwa and showcasing the potential of CX-5461 in the oncology landscape. Such advancements reflect the ongoing commitment within the industry to explore novel therapeutic avenues and improve patient outcomes in both rare diseases and prevalent conditions.
