Neurocrine Biosciences, Inc. is on a roll this summer – rolling out positive Phase 3 clinical trial results for a treatment of congenital adrenal hyperplasia and announcing FDA approval of its INGREZZA drug for treating symptoms caused by Huntington’s disease (HD).
VP, Clinical Development
Neurocrine Biosciences, Inc.
The FDA approval of INGREZZA for treating chorea in HD – a disorder that causes sudden jerking movements – is the drug’s second FDA approval. In 2017, it was approved to treat Tardive Dyskinesia – a movement disorder associated with long-term use of antipsychotic drugs that affects around 600,000 adults in the U.S., said Dr. Grace Liang, Neurocrine vice president of clinical development.
The FDA approval for treating chorea in HD adds about 41,000 adult patients that could benefit from INGREZZA.
INGREZZA is a once-a-day capsule that inhibits vesicular monoamine transporter 2 (VMAT2), “a transporter in the brain we know is really important for regulating levels of neurotransmitters such as dopamine,” Liang said. “Dopamine is one of the key neurotransmitters involved in controlling voluntary movement – psychiatric symptoms as well.”
The FDA approval of INGREZZA follows two successful clinical studies that yielded “about a threefold greater improvement compared to placebo,” Liang said. “That’s a really robust efficacy.”
CEO
Neurocrine Biosciences, Inc.
In a statement released after the FDA approval, Neurocrine CEO Kevin Gorman thanked the HD community for helping reach an “important milestone” in treating the disease.
“We remain committed to bringing medicines to patients with unmet medical needs for debilitating neurological disorders,” he added.
Neurocrine is exploring two other potential indications for INGREZZA, both are currently in Phase 3 clinical studies. One is schizophrenia as an adjunctive treatment to compliment other antipsychotic treatments, Liang said.
The other program is to reduce involuntary movement for both pediatric and adult patients with cerebral palsy – the most common motor impairment in childhood, she added.
Promising Pipeline
In addition to exploring other indications for INGREZZA, Neurocrine has other neuropsychiatric programs in its pipeline. The company is currently conducting Phase 2 studies for new drug compounds in major depression and schizophrenia.
Neurocrine released Sept. 12 positive results from a Phase 3 study of crinecerfont – a treatment for classic congenital adrenal hyperplasia – a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal hormones which are essential for life. If left untreated, classic CAH can result in salt wasting, dehydration, and even death.
The results of the study showed statistically significant results compared to the placebo group with only mild side effects and no major adverse effects. The current standard of care for CAH – glucocorticoids – have been associated with significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease, osteoporosis and may also lead to psychological and cognitive issues such as changes in mood and memory.
“CAH patients suffer from a number of debilitating symptoms and have had suboptimal treatment options with existing standard of care for their whole lives,” Gorman stated.
The Phase 3 data for crinecerfont along with data from the open label treatment period, allows Neurocrine to proceed with regulatory submissions to the FDA in 2024 and European Medicines Agency afterwards, he added.
Street Reaction
The positive data from crinecerfont’s Phase 3 was welcome news for Neurocrine (Nasdaq: NBIX) investors. Shares of NBIX had been on a steady climb since the INGREZZA FDA announcement, and jumped more than $17 on Sept. 12, closing the day at $117.64 – an eight-month high for the company.
Neurocrine’s summer of good fortune began in June when the company posted its Q2 2023 earnings with revenue of $452.7 million, up 19.7% year-over-year.
Neurocrine Biosciences, Inc.
Founded: 1992
CEO: Kevin Gorman
Headquarters: San Diego
Business: biopharmaceutical company
Stock: NBIX (NASDAQ)
Revenue: $1.49 billion (FY2022)
Market Cap: $10.73 billion
Employees: 1,200
Website: www.neurocrine.com
Notable: Neurocrine was founded by Wylie Vale, Ph.D., of the Salk Institute, where he contributed to Nobel Prize–winning work in endocrinology, and award-winning Stanford University neurologist Lawrence Steinman, Ph.D.
