Tildacerfont: New Drug Provides Hope, Treatments After 50-Year-Long Drought
Photo by Pablo Heimplatz on Unsplash
The light at the end of the tunnel just got a little brighter for those suffering from the life-threatening disease congenital adrenal hyperplasia (CAH). Thanks to a recent study by Spruce Biosciences Inc. (NASDAQ: SPRB), patients could have a new treatment option on the horizon: the non-steroidal, oral, and once-daily investigational therapy, tildacerfont.
To understand the importance of this study, it’s imperative to learn about CAH, a disease that affects the adrenal glands. People with CAH are born without a gene that produces the enzyme responsible for making cortisol, an essential hormone for controlling the body’s stress response. The result can be complications such as adrenal crisis, where low levels of cortisol can cause low blood sugar, low blood sodium levels, cardiovascular problems, and even shock. CAH also causes increased adrenal hormone levels, which leads to complications such as short stature, infertility, and early puberty in children.
CAH affects about 20,000 to 30,000 people in the United States. The current standard of care is steroid replacement therapy, which helps to replenish cortisol and reduce adrenal hormone levels. Very high doses of steroids, however, are required to effectively manage this disease, presenting long-term and serious health complications such as shortened lifespan, obesity, osteoporosis, and various metabolic diseases including insulin resistance, diabetes and high blood pressure. People living with CAH have not benefited from new treatment options in over 50 years. However, that’s precisely where Spruce Biosciences comes into play. As a late-stage biopharmaceutical company focused solely on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, CAH is center stage for its work.
Tildacerfont binds to CRF1 receptors on the pituitary gland, inhibiting excessive production of adrenocorticotropic hormone (ACTH). In the end, this ultimately reduces adrenal hormone levels without increasing the daily total of steroids. As many people with CAH have experienced, living with this disease is a difficult balance between managing excessive adrenal hormone levels and high doses of steroids. In many cases, people living with CAH are unable to tolerate these steroids and live with the consequences of excessive adrenal hormones.
Spruce Biosciences’s 12-week study focused on the ability of tildacerfont to reduce adrenal hormones in adults with classic CAH, the severe form of the disease. The study assessed the ability of a daily dose of 400mg of tildacerfont to lower ACTH, 17-hydroxyprogesterone (17-OHP) and androstenedione (A4). The results of the studies showed that tildacerfont reduced key hormone biomarkers towards normal levels in patients with poor disease control, including normalization of ACTH and A4 in 60% and 40% of patients, respectively. Tildacerfont was generally safe and well-tolerated.
According to Dr. Rosh Dias, the company’s chief medical officer, normalizing these highly elevated hormones in classic CAH patients without increases to daily steroid doses has not been reported with any other potential treatment. Not to mention, tildacerfont is the first CRF1 receptor antagonist studied beyond 2 weeks in CAH.
Spruce Biosciences’s most recent study supports the ongoing global late-stage studies of tildacerfont in adults with classic CAH (CAHmelia-203 and CAHmelia-204). The company also plans to initiate a Phase 2 proof of concept study in women with polycystic ovary syndrome (PCOS) suffering from excess adrenal hormones due to adrenal dysfunction later this year.
It is clear that through its novel treatment and dedication to addressing underserved patients, Spruce Biosciences has the potential to change the treatment paradigm for people suffering from CAH, PCOS, and other rare endocrine diseases.
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