Biotech Companies are Increasingly Focused on Deadly Diseases With Significant Unmet Need

According to a story from, biotechnology companies are starting to set their sights on treating deadly diseases that are lacking effective treatment options, including some diseases that are considered rare. While advancements are constantly being made in the biotech field, that fact remains that the number of patients that are being impacted is rising as well. Here is an overview of the latest progress that certain biotechnology companies have made recently in addressing these life-threatening diseases.

Idiopathic Pulmonary Fibrosis

An increasingly hot topic in the biotech field is idiopathic pulmonary fibrosis. While this disease is considered rare, the annual death toll speaks for itself: around 40,000 people in the US die because of this illness every year. The five year survival rate is poor as well at just 40 percent. One company that is stepping in to development a new treatment for this disease is Algernon Pharmaceuticals Inc. Early data testing the company’s experimental anti-fibrosis drug NP-251 has encouraged the company to continue testing their investigational product in this indication. NP-251 has also show potential in treating other diseases where fibrosis (scarring) is a major problem, such as nonalcoholic steatohepatitis (NASH) and chronic kidney disease (CKD). With no cure available and the currently limited effectiveness of therapies for idiopathic pulmonary fibrosis, more powerful treatments are urgently needed.

Chronic Kidney Disease

Chronic kidney disease is another target that is drawing attention from biotech firms. One company that is focused in this area is Akebia Pharmaceuticals. This company is close to completing the development of its Phase 3 experimental product candidate vadadustat. This drug is designed to stimulate the production of red blood cells and hemoglobin in order to address anemia that is associated with chronic kidney disease. Rockwell Medical, Inc., is also working on getting an intravenous formulation of its drug Triferic approved by the FDA. Triferic is the only approved therapy for maintaining hemoglobin and replacing lost iron in chronic kidney disease patients that are using dialysis.

Other Updates

The company Kadmon Holdings is in the midst of a Phase 2 trial of its experimental drug KD025 as a treatment for chronic graft-versus-host disease, a potentially lethal complication that can appear in stem cell transplant patients. Data for this therapy is looking promising so far.

Conatus Pharmaceuticals is in working on an experimental therapy for nonalcoholic steatohepatitis (NASH), a form of liver disease that is more common than previously thought. The company is in the midst of registering participants for its Phase 2b clinical trial which will test the company’s drug emricasan as a therapy in this indication. Top-line results for this trial are expected by mid-2019.

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