Boehringer / Zealand Phase 2 results

March 1, 2024

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Phase 2 results

The Phase II trial for Survodutide, a novel treatment targeting metabolic dysfunction-associated steatohepatitis (MASH), has yielded promising results, as announced by Boehringer Ingelheim. This trial has demonstrated significant efficacy in improving the condition of adults suffering from MASH, a liver disease characterized by inflammation and fat accumulation in the liver, which can progress to fibrosis (scarring of liver tissue).

Key findings include:

• Efficacy of Survodutide: The trial showed that 83% of participants treated with Survodutide achieved statistically significant improvements in MASH compared to only 18.2% in the placebo group. This response difference was 64.8% with a confidence interval of 51.1% to 78.6%, and a p-value of less than 0.0001, indicating a very high level of statistical significance.
• Primary and Secondary Endpoints Met: The primary endpoint was a biopsy-proven improvement in MASH after 48 weeks of treatment without worsening fibrosis in stages F1 to F3. All secondary endpoints were also met, including statistically significant improvements in liver fibrosis.
• Mechanism of Action: Survodutide acts as a glucagon/GLP-1 receptor dual agonist. The glucagon agonist component potentially increases energy expenditure and directly impacts the liver to improve fibrosis. The GLP-1 agonist component works by decreasing appetite and increasing satiety, contributing to the treatment's overall effectiveness.
• Safety and Tolerability: The treatment was well-tolerated without unexpected safety issues, even at the higher dose of 6.0mg.
• Regulatory Progress: Survodutide has received Fast Track Designation from the U.S. FDA and has been accepted into the EMA PRIME scheme, which are both efforts to expedite the development and review of drugs addressing unmet medical needs.
• Future Developments: Full data from the trial will be presented in the upcoming months, and the drug is also being evaluated in Phase III studies for overweight and obesity in various populations, including those with cardiovascular disease and type 2 diabetes.

This trial represents a significant advancement in the treatment of MASH, a condition associated with high morbidity and increasing prevalence worldwide. The efficacy of Survodutide in improving both MASH and fibrosis without significant safety concerns suggests its potential as a best-in-class treatment option for patients with this condition. The comprehensive approach of targeting both the glucagon and GLP-1 receptors highlights a novel mechanism that could offer benefits across the cardiovascular, renal, and metabolic spectrum, addressing the interconnected nature of these diseases with MASH. Further details on the secondary endpoints and long-term outcomes will be crucial in fully understanding the impact of Survodutide and its role in treating MASH and related conditions.

Market Opportunity

Description of MASH:

Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), is a severe stage of non-alcoholic fatty liver disease (NAFLD). It is characterized by the accumulation of fat in the liver, inflammation, and liver cell damage, leading to fibrosis or scarring of the liver tissue. MASH can progress to cirrhosis, liver failure, or liver cancer if not treated effectively. The disease is closely associated with metabolic conditions such as obesity, type 2 diabetes, and insulin resistance.

Standard of Care and Unmet Need:

The current standard of care for MASH primarily involves lifestyle interventions aimed at weight loss, dietary changes, and physical activity. Pharmacological treatments focus on managing associated conditions, such as diabetes and hyperlipidemia. However, there are no FDA-approved drugs specifically for MASH, representing a significant unmet need in the treatment of this condition. The lack of targeted therapies underscores the importance of developing new pharmacological interventions to halt or reverse disease progression.

Patient Population and Market Size:

The patient population for MASH is growing, driven by the global epidemic of obesity and type 2 diabetes. It is estimated that more than 115 million people worldwide suffer from MASH, with the number expected to rise. This growing patient population represents a substantial market for effective treatments. MASH is predicted to become the leading cause of liver transplantation by 2030, highlighting the urgent need for therapeutic interventions.

Despite the presence of these competitors, the lack of approved therapies leaves room for new entrants like Survodutide. The efficacy demonstrated in phase II trials, particularly in improving liver fibrosis without worsening of MASH, positions Survodutide as a potential leading candidate in the MASH treatment landscape.

Conclusion:

The market opportunity for Survodutide in MASH is significant, given the large and growing patient population, the critical unmet medical need for effective treatments, and the potential for first-mover advantage in a market without approved pharmacological therapies. Survodutide’s novel mechanism of action, efficacy in improving MASH and fibrosis, and favorable safety profile underscore its potential to become a best-in-class therapy. The ongoing phase III trials for overweight and obesity further expand its therapeutic potential across related metabolic conditions, enhancing its marketability and addressing the interconnected nature of these diseases with MASH.