Calliditas Therapeutics is focused on developing innovative treatments for rare diseases, particularly in the areas of renal and hepatic conditions. One of their key products is TARPEYO® (budesonide) delayed release capsules, which is specifically designed for the treatment of IgA nephropathy, a chronic autoimmune disease. The company is also advancing a pipeline of novel medications, including Setanaxib, which is being investigated for its potential in treating fibrotic rare diseases and solid tumors. Setanaxib has shown promise in addressing conditions such as Primary Biliary Cholangitis (PBC) and Idiopathic Pulmonary Fibrosis (IPF), with ongoing clinical trials aimed at evaluating its efficacy and safety.

Setanaxib operates as a NOX enzyme inhibitor, targeting the underlying mechanisms of fibrosis in diseases like PBC. This innovative approach aims to disrupt the cycle of inflammation and fibrosis that characterizes these conditions, potentially improving patient quality of life by alleviating symptoms such as chronic fatigue. The drug has received orphan drug designation (ODD) from both the FDA and EMA, highlighting its significance in treating rare diseases with limited existing therapies. Calliditas is committed to expanding access to their treatments through initiatives like the Named Patient Program for TARPEYO, ensuring that patients who have exhausted other options can access these novel therapeutics.

United States

Calliditas is a clinical-stage biopharmaceutical company focused on identifying, developing and commercializing novel treatments in orphan indications, with an initial focus on renal and hepatic diseases with significant unmet medical needs.

Cancer

Immune System Diseases

Kidney Diseases

Liver Diseases

Lung Diseases

Rare diseases

Calliditas Therapeutics 

IgA nephropathy

Nefecon is a patented oral formulation of a potent and well-known active substance –budesonide – for targeted release. The formulation is designed to deliver the drug to the Peyer’s patch region of the lower small intestine, where the disease originates, as per the predominant pathogenesis models. Nefecon &nbsp;is derived from the TARGIT technology, which allows for the substance to pass through the stomach and intestine without being absorbed, and to be released in a pulse like fashion only when it reaches the lower small intestine.
The combination of dose and optimized release profile is required to be effective in patients with IgA nephropathy, as shown in a large Phase 2b study, completed by the company. In addition to its potent local effect, another advantage of using this active substance is that it has very low bioavailability, i.e. around 90% of it is inactivated in the liver before it reaches the systemic circulation. This means that a high concentration can be applied locally where needed but with only very limited systemic exposure and side effects. Another example where this approach has proven to be successful is in asthma treatment where budesonide is successfully used to locally treat the airways and lung tissue.

TARPEYO (Nefecon) (budesonide)

Head and neck squamous cell carcinoma

Solid tumor/s

Setanaxib downregulates the activation of multiple clinically validated fibrogenic and apoptotic pathways.

Calliditas Therapeutics

Company Details