New and Improved: Rebranding Liver Disease to Change Outcomes for Patients

This week, Madrigal Therapeutics (Nasdaq: MDGL) won FDA approval for Rezdiffra™, the first and only drug to be approved for metabolic dysfunction-associated steatohepatitis (MASH). The landmark decision has shone a light on MASH, an advanced liver disease, and a leading cause of liver-related mortality, that affects between six and eight million adults in the US. The number of MASH cases is expected to skyrocket to an estimated 27 million cases in the US by 2030, according to the Global Liver Institute.

There are more than 100 types of liver disease. It can be the result of genetics, caused by viruses such as hepatitis, or lifestyle-induced through the excessive use of alcohol or poor diet. The good news for patients is that when diagnosed early, liver disease can be reversed with a well managed treatment plan. Diseases like MASH have been waiting decades for an approved treatment, and experts say that a simplistic approach to naming steatotic liver disease could be partially to blame.

In spite of the complexity of steatotic liver disease, for many years it was reduced to two indications: fatty liver disease and non-alcoholic fatty liver disease, which refers to liver disease in the absence of heavy alcohol use. Patients have felt stigmatized, and their disease trivialized, by the use of the words ‘fatty’ and ‘alcohol’, which implies a negative tie to alcohol for many. Jonathan Stine, director of the Fatty Liver Program at Penn State Health, said in an interview with STAT, “I’ve had patients hear the word ‘fatty’ before, become very emotional, sit with me, cry in the office.”

In 2023, years after doctors and patient advocates first called for a name change and evolved diagnostic criteria, global liver disease groups came together to establish a stigma-free name and diagnosis. The result – metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH) – may be the rebrand that liver disease needed to generate better clinical results for specific patient populations.

The name change is already positively impacting disease awareness among patients and doctors, and has resulted in simplified, but more specific, diagnostic criteria, and less invasive testing. Researchers believe that liver disease’s ‘rebrand’ will accelerate the pace of existing research and result in new treatments coming down the pipeline. Rezdiffra’s approval will also be instrumental in shining a light on a diverse disease in desperate need of new, effective therapeutics.

These are PRISM MarketView’s top picks for delivering the next potential treatments for the liver disease patient population:

Inventiva (Nasdaq: IVA)

 Inventiva is focused on the research and development of oral small molecule therapies for the treatment of MASH, a disease with significant unmet medical need. The company’s lead product candidate, lanifibranor, is currently in a pivotal Phase 3 clinical trial for the treatment of adult patients with MASH. Inventiva expects to announce the results of its LEGEND Phase 2 trial, a study evaluating lanifibranor alone and in combination with the SGLT2 inhibitor empagliflozin in patients with MASH and type 2 diabetes, next week. Inventiva’s market cap is currently $216.91 million.

NeuroBo Pharmaceuticals (Nasdaq: $NRBO)

NeuroBo is a clinical-stage biotechnology company focused on the development of DA-1241 for the treatment of MASH and Type 2 Diabetes Mellitus (T2DM). In January 2024, NeuroBo announced positive preclinical data which showed that DA-1241 demonstrated a positive effect on liver inflammation, lipid metabolism, and reduced hepatic steatosis, hepatic inflammation, and liver fibrosis. This week, NeuroBo received Safety Review Committee (SRC) approval recommending that the company’s two-part Phase 2a trial of DA-1241 continue without modification following a blinded safety review of the first six months of study conduct. The company expects to report the full data from the study in the second half of 2024. NeuroBo’s market cap stands at $22.568 million.

Better Therapeutics (Nasdaq: BTTX)

Prescription digital therapeutics company, Better Therapeutics, is developing a novel form of cognitive behavioral therapy (CBT) to help patients manage underlying factors that contribute to cardiometabolic diseases. The company’s proprietary platform for the development of FDA-regulated, software-based solutions for MASH, T2D, heart disease and other conditions. In February 2024, Better Therapeutics was granted Breakthrough Device Designation by the FDA for its CBT platform intended to treat adults with MASH. Better Therapeutics earned breakthrough status based on the outcomes of its LivVita clinical study, which successfully met its primary endpoint by reducing liver fat within 90 days, while also achieving key secondary endpoints related to improved liver health without any device-related adverse events. The company’s market cap is $2.43 million.

89bio (Nasdaq: ETNB)

89bio is developing best-in-class therapies for liver and cardiometabolic diseases, and is rapidly advancing its lead candidate, pegozafermin, for the treatment of MASH and severe hypertriglyceridemia (SHTG). This week, the company announced it had initiated its Phase 3 ENLIGHTEN Program evaluating the efficacy and safety of pegozafermin in patients with MASH. 89bio is also on track to initiate a second Phase 3 trial in MASH patients with compensated cirrhosis in the second quarter of 2024. 89bio’s market cap stands at $1.26 billion.

Ionis Pharmaceuticals (Nasdaq: IONS)

 Ionis describes its novel approach to treating MASH as a ‘unique precision medicine opportunity’ to address a progressive disease in need of better treatment options. This week, the company announced positive data from its Phase 2 trial of ION224 for the treatment of MASH. ION224 is designed to reduce the production of DGAT2, which decreases the overproduction of triglycerides that contribute to excess liver fat, which can result in liver damage and inflammation. The study found ION224 to be safe and well-tolerated and it met its primary endpoint, achieving liver histologic improvement, its secondary endpoint of MASH resolution. Ionis’ market cap stands at $6.20 billion.

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New and Improved: Rebranding Liver Disease to Change Outcomes for Patients

Catie Corcoran

Biotech Editor