For over three decades, Neurocrine Biosciences, Inc. has been working to support individuals with neurological, neuroendocrine and neuropsychiatric disorders through the development of breakthrough treatment options.
One of the company’s key focuses is developing a novel therapy for individuals living with congenital adrenal hyperplasia (CAH). A rare genetic disorder, CAH often presents significant challenges for those affected, their families, and even healthcare providers. Despite advances in medical knowledge and practice, the CAH landscape has remained relatively stagnant. We still see a notable gap in comprehensive education and support for this community.
In April 2024, Neurocrine Biosciences launched a fantastic educational initiative to provide resources, offer support, and open discussions around life with congenital adrenal hyperplasia. The WHAT THE C@H?! initiative unites the CAH community and brings CAH awareness to an entirely new level.Recently, Patient Worthy sat down with Neurocrine Biosciences’ Eric Benevich to understand this initiative, its impact, and why it’s so important to pursue heightened research and development in the CAH sphere.
Interview: Eric Benevich, Neurocrine Biosciences
JL: Hey Eric! Thanks so much for speaking with Patient Worthy about WHAT THE C@H?! Before we jump in, can you tell us a little about yourself?
EB: I’m Eric Benevich and I have served as Neurocrine Biosciences’ Chief Commercial Officer since 2015. I am responsible for all aspects of commercial development, marketing, and sales for Neurocrine’s products.
My role is sometimes challenging. It can sometimes be difficult keeping up with all the laws, regulations, and standards for this industry. Biopharmaceuticals is a heavily regulated healthcare sector. It’s critical that we comply with all federal and state healthcare laws and maintain high ethical standards. But my role is also incredibly rewarding. Seeing how our medicines can dramatically improve the lives of people living with serious neurological health conditions drives and inspires me.
How did you end up at Neurocrine Biosciences? What about this company made you believe in its mission?
I’ve been working in the biopharmaceutical industry for over 30 years. Prior to Neurocrine, I worked at other pharmaceutical companies including AstraZeneca, Peninsula Pharmaceuticals, Amgen, and Avanir Pharmaceuticals.
I was initially drawn to Neurocrine Biosciences because of its mission and commitment to patients, first and foremost. Neurocrine is dedicated to discovering and developing life-changing treatments for people with debilitating neuroendocrine, neurological and neuropsychiatric disorders with few good therapeutic options.
I’m so proud of the work we do here tackling intractable diseases with brave science. I’m also proud that we have stayed true as a company to our roots in neuroscience. It gives me great satisfaction to work with the caliber of people at Neurocrine and the dedicated health professionals we strive to serve.
Neurocrine’s WHAT THE C@H?! Initiative focuses on providing educational information for congenital adrenal hyperplasia (CAH). In your words, what is congenital adrenal hyperplasia?
Congenital adrenal hyperplasia is a rare, lifelong genetic condition involving the adrenal glands that is estimated to affect up to 30,000 people in the United States. People living with CAH have very little, if any, of a particular enzyme called 21-hydroxylase that the body needs to change cholesterol into two essential hormones (cortisol and aldosterone) that regulate important body processes. Without 21-hydroxylase, the building blocks for cortisol and aldosterone pile up. Instead of doing what they should be doing, they make too many androgens. Androgens, which are also hormones, contribute to growth, puberty, and reproduction.
Depending on whether individuals have the classic or nonclassic form of CAH, they may show signs or symptoms of severe acne, early appearance and growth of excess facial and body hair, irregular or absent menstruation in females, rapid growth or bone age during childhood with a shorter-than-average height, blood sugar and blood pressure issues, and atypical genital development.
Can you provide an overview of the WHAT THE C@H?! initiative and its primary objectives? What inspired Neurocrine to launch this educational initiative for the CAH community?
Neurocrine Biosciences launched WHAT THE C@H?!, an educational initiative, to help close the gap in the need for helpful information about CAH. WHAT THE C@H?! acknowledges the frustrations and challenges experienced by the community in managing the condition. It was created for adults and adolescents living with CAH, parents of children with CAH, and healthcare providers who treat the condition.
The effort includes a dedicated website for the community, WhatTheCAH.com, which offers educational information to help individuals living with CAH and their families navigate their journeys. There is also a healthcare provider-specific website which features interactive educational resources on current CAH monitoring and management practices, in addition to an overview of the potential of the corticotropin-releasing factor (CRF) pathway in CAH management. For physicians that are not as confident or as well-versed, there is a wealth of resources there for them to learn more about CAH.
The WHAT THE C@H?! website offers a space to share and read stories regarding CAH experiences. Those living with CAH, including parents of children living with CAH, can submit their own WHAT THE C@H?! moment, which is a challenging experience of living with or managing the condition, for potential inclusion on the site. We hope that this website provides the community with a platform to share their experiences and to help others navigate the complexities of managing this disease. We also encourage the community to talk to their doctors about treatment options and management, in addition to turning to advocacy groups for support.
WHAT THE C@H?! recognizes the frustrations associated with living with CAH, a condition in which the symptoms and side effects of current treatment make management especially challenging. We hope this educational initiative, which was informed by insights from the community, is helpful to those navigating and managing this difficult condition.
How was the initiative informed by insights from the CAH community specifically?
We conducted interviews with 63 people with CAH and 124 healthcare providers. All community members expressed that living with CAH can be frustrating, including the challenges of experiencing symptoms of low cortisol or excess androgens, as well as high side effects from high doses of glucocorticoids.
A main takeaway was that members of the CAH community were missing a way to air out their frustrations and challenges in managing the symptoms and treatment. Our goal with this initiative is to provide a space for the community to share their own experiences with the disease — and target newly diagnosed families who are hungry for information.
When it comes to CAH, what are the limitations or challenges with the current standards-of-care? What are common side effects that patients and HCPs should be aware of?
Treatment options for CAH are limited and have not changed in over 70 years. Glucocorticoids (GCs) remain the only treatment and standard of care for both low levels of cortisol and to help manage the overproduction of adrenal androgens. Mineralocorticoids are also used to treat aldosterone insufficiency.
As I said above, long-term exposure to high adrenal androgen levels can cause shorter adult height, excessive hair growth and menstrual irregularity in females, infertility in females and males, and may have a negative mental and emotional impact. GCs are effective at replacing the missing cortisol, but higher doses are needed to reduce the production of adrenal androgens. However, higher doses of GCs come with drawbacks, including serious short-term and long-term side effects such as weight gain, osteoporosis, and changes in mood and memory.
Can you explain the significance of corticotropin-releasing factor (CRF1) receptors in the context of CAH and treatment? Is this something the Neurocrine Biosciences team will use to shape future research and treatment development?
Neurocrine’s co-founder, the late Wylie W. Vale, Ph.D., discovered and studied corticotropin-releasing factor, the stress hormone that regulates the release of adrenocorticotropic hormone (ACTH) from the pituitary gland. This Nobel Prize-winning work led to the understanding of key biological mechanisms that are currently being targeted in the treatment of CAH.
Antagonism of CRF1 receptors in the pituitary has been shown to decrease adrenocorticotropic hormone levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH.
What recent advancements in CAH treatment and/or management have been most promising? Ideally, what research would you like to see done in the future?
We are excited to see so much focus and positive data regarding bringing new medicines to adults and pediatric patients living with CAH. Patients with CAH have so few options, so additional research in this area is always a good thing.
What feedback have you received from the CAH community since launching WHAT THE C@H?!
Since the initiative launched in April, we have received positive feedback and enthusiasm from the CAH community. It’s a bit of a tongue-in-cheek play on words that people can relate to and it’s been well received by the CAH community. We are excited to continue to build out the resources provided to further support individuals living with the condition and those who care for them and treat them.
What role do advocacy groups play in shaping the development of this initiative?
Neurocrine is proud to support the CAH community through a variety of partnerships with the patient advocacy organizations who serve as key resources to those impacted by CAH. We are committed to ongoing partnerships with the CARES Foundation and other advocacy organizations that represent the CAH community to learn about their experiences and unmet needs. We intend that WHAT THE C@H?! will evolve over time to meet those needs.
What are the next steps for this initiative?
Looking ahead, we are committed to fulfilling unmet needs in CAH and will continue to expand the WHAT THE C@H?! website to include stories and experiences, in addition to more educational information and resources.
We are so proud to support the community and are hopeful that WHAT THE C@H?! will provide valuable and empowering information to patients, families and healthcare providers managing CAH.
About CARES Foundation
CARES Foundation is the only organization in the United States solely dedicated to the congenital adrenal hyperplasia (CAH) community. Founded in 2000, CARES represents approximately 8,000 affected individuals, families and healthcare professionals in the U.S. and more than 70 countries. To learn more, visit www.caresfoundation.org.
About Neurocrine Biosciences, Inc.
Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company’s diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington’s disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X (formerly Twitter) and Facebook. (*in collaboration with AbbVie)
