Apellis Program to Control Host Response to AAV Vector for Gene Therapies

Apellis, (Nasdaq:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the control of the complement system, today announced its plans to develop APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus (AAV) vector administration for gene therapies. APL-9, an investigational drug, is a pegylated synthetic cyclic peptide designed to modulate the complement cascade centrally at C3.

“Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints,” said Cedric Francois, CEO and co-founder of Apellis. “We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles, yielding three important potential advantages. First, controlling C3 may significantly increase the efficiency of gene therapy delivery by protecting the AAV particles on their journey from the site of administration to the target tissue. Second, control of C3 may minimize the formation of anti-AAV neutralizing antibodies that can complicate subsequent retreatment with the same AAV. Finally, by countering the rapid activation of C3 upon administration of AAV particles, control of C3 may prevent inflammatory reactions that can lead to significant organ damage, for example in the kidneys. Apellis is committed to the development of APL-9 to target host responses to AAV vector administration and thus potentially improve patients’ well-being while undergoing AAV administered gene therapies.”

In Phase I testing, Apellis treated 20 healthy volunteers with single doses of APL-9 ranging from 30 mg to 600 mg, administered as an intravenous infusion. APL-9 demonstrated control of complement through modulation of C3 within 1 hour of administration, that lasted up to 12 hours after the end of the infusion. Multiple doses tested achieved complete suppression of the AH50 hemolytic activity. In this Phase I study, APL-9 was well tolerated with no serious adverse events reported.

https://www.marketscreener.com/APELLIS-PHARMACEUTICALS-I-38607086/news/Apellis-Pharmaceuticals-Will-Commence-APL-9-Program-to-Control-the-Complement-System-in-Host-Respons-28922017/