The non-alcoholic fatty liver disease/non-alcoholic steatohepatitis (NAFLD/NASH) epidemic is roughly 2.5 times greater than diabetes in the U.S. Despite the significant size and unmet need of the potential market, pharmaceutical companies have yet to capitalize on the opportunity.
History is littered with failed NAFLD/NASH clinical trials, with last Friday’s Gastrointestinal Disease Advisory Committee (GIDAC) potentially serving up another, as it denied Intercept Pharmaceutical’s accelerated approval for Obeticholic Acid (OCA) to treat NASH. While OCA may still have a chance to find its way to patients, the advisory committee voted 12 to 2 against the risk benefit profile offered by the potential therapy.
Hope for NASH patients is on the horizon; however, with Madrigal Pharmaceuticals resmetirom having recently received a thumbs up from ICER, along with breakthrough status granted by the FDA. Madrigal noted in its first quarter financial results it is on track to file resmetirom’s NDA in the first half of 2023, potentially making it the first drug indicated to treat NASH fibrosis.
Both resmetirom and OCA are being evaluated in NASH patients with confirmed liver fibrosis. Insights from Spherix’s Market Dynamix™ NAFLD/NASH service highlight reducing liver fibrosis is currently the highest priority for gastroenterologists.
“Fibrosis is directly linked to clinical outcomes. That’s why we prioritize fibrosis in general…We do have treatments for fibrosis, it’s just that they’re too extreme. There are multiple studies on bariatric surgery patients where it’s shown fibrosis in the liver disappears. Unfortunately, we can’t do bariatric surgery on everybody”.
– US Gastroenterologist
NASH is generally stratified by the level of liver fibrosis present from F0 (no fibrosis) to F4 (fibrotic scarring and cirrhosis). Gastroenterologists described urgent need for the most severe fibrotic liver patients (NASH F3 & F4) followed by those with early and progressing fibrosis (NASH F2 & F1). Resmetirom’s MAESTO-NASH trial includes patients with F1-F3 fibrosis.
Controlling and reducing fibrosis is only the tip of the NAFLD/NASH iceberg confronting healthcare workers worldwide. In a series of interviews with Spherix, gastroenterologists stressed NAFLD is a liver disease correlated with metabolic syndrome, obesity, type 2 diabetes, hyperlipidemia, hypertension, cardiovascular disease, and other complications. Earlier treatment of comorbidities leading to NAFLD is vitally needed to slow and prevent the incidence of progression to NASH.
Spherix is exploring mechanisms under development to treat the constellation of NAFLD/NASH issues above, including Novo Nordisk’s GLP1 receptor agonist (weight loss), Akero Therapeutics and 89bio’s FGF21 analogues (weight & lipid management), Inventiva Pharma’s pan-PPAR agonist (metabolism, inflammation and fibrogenesis), Viking Therapeutics and Madrigal Pharmaceutical’s THR-β agonists (fibrosis, liver inflammation and fat reduction), Northsea Therapeutics’ structurally engineered fatty acid – SEFA – (insulin resistance, inflammation, lipotoxicity, fibrosis), Galmed Pharmaceutical’s SCD1 inhibitor (lipotoxicity and liver fibrosis), FXR agonists, including Intercept Pharmaceutical’s OCA, and several others.
Gastroenterologists stressed the volume of NAFLD patients is beyond the capacity of specialists to treat. The majority interviewed suggest a reasonable referral point occurs when a patient crosses the F2 fibrosis barrier. This would place the burden of managing many medical issues mentioned previously – most notably weight loss and diabetes – on primary care physicians.
It seems reasonable that pharmaceutical companies delivering both new therapies and helping shape NAFLD/NASH management across primary and specialty care can achieve a strategic advantage, while helping the greatest number of patients afflicted with this condition. The road to achieving life changing treatment for NAFLD/NASH is long and fraught with difficulty, but for the companies committed to helping these patients, opportunities abound.
About Market Dynamix™
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.
Spherix Global Insights is a leading provider of market research, business intelligence and advisory services to the global life sciences industry. The company’s unique service offerings are powered by deep therapeutic knowledge, the Spherix Network specialty physician panel, and commercially relevant analyses to enable strategic decision-making by our valued customers.
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Spherix Global Insights Contacts
Geoff Red, Franchise Leader, Gastroenterology
Scott Upham, Corporate Communications
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SOURCE Spherix Global Insights