FDA Approves Neurocrine's Treatment for Rare Adrenal Condition

Neurocrine Biosciences has concluded a challenging 2024 on a positive note after receiving FDA approval for its new drug aimed at treating congenital adrenal hyperplasia (CAH), a disorder characterized by adrenal gland dysfunction. This approval marks the second product sanctioned for Neurocrine, following its treatment for movement disorders, Ingrezza. The newly approved medication, Crenessity, represents the first novel treatment for classic CAH in seven decades.

This achievement also serves as a significant milestone in honoring the legacy of Wylie Vale, a late founder of Neurocrine and a noted scientist at the Salk Institute. Vale's pioneering work in peptide hormone research and his exploration of the brain-endocrine system relationship led to the identification of the corticotropin-releasing factor. This discovery plays a critical role in the mechanism of the new drug, also known as crinecerfont, which functions by blocking this factor.

Crenessity's approval covers both adult and pediatric patients with CAH and is backed by what Neurocrine describes as the largest ever clinical trial for classic CAH. The trials demonstrated that in children, Crenessity effectively reduced androstenedione hormone levels, subsequently decreasing androgen and associated steroid usage. Similar results were observed in the adult trials, where there was a significant reduction in glucocorticoid doses by the 24th week, which was the primary endpoint of the study.

Following the FDA's decision, Neurocrine's stock ($NBIX) saw a rise of over 5% as markets opened on Monday. Analysts from William Blair have labeled the drug's approval as "nonrestrictive," projecting US sales to reach $150 million in 2025 and worldwide sales to potentially peak at $1.47 billion by 2030. Neurocrine has already initiated an educational campaign targeted at the estimated 20,000 to 25,000 individuals affected by CAH in the US.

Additionally, the approval of Crenessity has brought Neurocrine a priority review voucher, a valuable asset that other biotech firms have previously sold for prices exceeding $150 million.

The FDA's endorsement of Crenessity offers a much-needed boost to Neurocrine following a year marked by volatility. Earlier in August, the company faced setbacks when it released Phase 2 data for its muscarinic 4 agonist schizophrenia drug, which did not meet the expectations set by similar treatments from Bristol Myers Squibb and Karuna Therapeutics. Although Neurocrine's schizophrenia candidate, NBI-’568, showed a statistically significant improvement in symptoms over placebo, the results were less impressive than those reported by Karuna during their late-stage clinical trial, causing Neurocrine’s shares to drop by 20%. Despite this, Neurocrine intends to proceed with Phase 3 testing.

In addition to these challenges, the company discontinued luvadaxistat, a mid-stage treatment for cognitive impairment linked with schizophrenia, due to its failure to replicate previous promising outcomes.

Nevertheless, Neurocrine's collaborative deal with Takeda in April has shown encouraging results. A new depression treatment surpassed placebo in terms of symptom improvement during a Phase 2 study among patients who had not responded to earlier antidepressant treatments. Phase 3 studies for this promising treatment are planned for 2025, indicating potential future successes for Neurocrine.