According to a story from Markets Insider, the biopharmaceutical company Principia Biopharma Inc. has recently issued new updates in regards to the timeline of its phase 3 clinical study. This trial is testing the company’s investigational product candidate PRN1008 as a treatment for pemphigus, a rare autoimmune disease. The company has also announced complete response rates from the phase 2 trial of the drug as well. Principia is focused on developing orally active therapies to fulfill currently unmet medical needs in diseases involving the immune system.
Pemphigus vulgaris is the most common form of pemphigus, a rare disease that causes blisters to form on the skin and mucus membranes. Pemphigus is an autoimmune disease in which the immune system creates autoantibodies. This disease is more common in women and in people of Ashkenazi Jewish descent, revealing that people with certain genetic variants are more likely to get pemphigus. Symptoms include blisters that break and form into lesions and open wounds. They often first appear in the throat and mouth, making eating and swallowing extremely painful. Other surfaces where blister may occur include the eyelids, genitalia, anus, and esophagus. Diagnosis of the disease can take a long time. In the past, this disease was often lethal as the open wounds that form can become easily infected, and the painful nature of mouth lesions can prevent patients from eating normally. Click here to learn more about pemphigus.
Updates on the PRN1008 Program
The company’s chief medical officer, Dr. Dolca Thomas, says that the complete response rate in the phase 2 trial was 40 percent. This was despite the patients receiving a 400 mg dose per day, which was regarded as a sub-therapeutic dosage level. A total of 15 patients were dosed in the trial and nine of them were able to achieve the primary endpoint “control of disease activity” after four weeks. 12 patients obtained this endpoint by week twelve.
In the meantime, the company’s phase 3 trial is currently in the process of enrolling participants. The company ultimately hopes to recruit a total of 120 pemphigus patients for the study. With the current pace of development, Dr. Thomas says that the company expects final data from the trial to be ready in the last half of 2021.