Funding is expected to carry the candidate through IND-enabling studies.
Charley’s Fund will provide an additional $3 million with the amendment of its existing sponsored research agreement with AVI BioPharma related to Duchenne muscular dystrophy (DMD). This brings the total up to $5 million for the development of AVI-5038 through to an IND application.
The drug candidate is based on the company’s antisense chemistry called phosphorodiamidate morpholino oligomers (PMO) and is a peptide-conjugated PMO. It has the potential to skip dystrophin exon 50 in certain patients with DMD.
This therapeutic approach is similar to that of AVI-4658, which is in Phase I development, according to the firm. AVI-4658 is designed to treat DMD patients with mutations that could benefit from skipping exon 51 of the dystrophin gene.
AVI’s first contract with Charley’s Fund was initiated in October 2007 with $2.45 million and partly supported the research that identified AVI-5038.