Pasadena Biotech Announces New Drug That Lowers a Key Immune Protein Shows Promise for Treating Rare Diseases

Pasadena biotechnology company Arrowhead Pharmaceuticals has announced promising results from an early trial of a new drug targeting a protein in the immune system. The drug, called ARO-C3, is designed to reduce the production of complement component 3 (C3), which helps fight infections but can also cause damage to healthy tissues in some conditions.

The trial involved healthy volunteers who received different doses of ARO-C3 by injection under the skin. The researchers found that ARO-C3 lowered the levels of C3 and another marker of immune activity called AH50 by up to 91%.

The drug was also apparently safe and well tolerated, with no severe side effects reported.

The company seems ready with plans to test ARO-C3 in patients with various rare diseases that are caused by excessive or abnormal activation of the complement system, such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and C3 glomerulopathy (C3G). These diseases can lead to serious complications such as anemia, kidney failure, and blood clots.

The company said in a statement hopes that ARO-C3 will offer a new treatment option for patients with these conditions, who currently have limited choices and may need frequent infusions of expensive drugs. ARO-C3 has the potential to be administered less often and more conveniently by injection.

The company expects to start dosing patients in the second part of the trial in the first half of 2023. It also plans to present more details about the trial at an upcoming medical meeting focused on complement disorders.