BioAxone Therapeutic Inc.

SCIENTIFIC PLATFORM

Since its inception, BioAxone has been advancing its expertise in the biology of Rho GTPase signaling. There is broad diversity within the proteins of this signaling pathway, affording multiple opportunities for therapeutic intervention in a wide variety of clinical indications. BioAxone began building its repertoire of Rho pathway modulators with a series of C3 exoenzyme-based Rho inhibitors (e.g. BA-210). When these bacterially derived enzymes gain access to the cytoplasm of mammalian cells, they specifically target and inactivate the host cell’s Rho GTPase molecules. In collaboration with Dr. Lisa McKerracher’s laboratory at the University of Montreal, BioAxone scientists re-engineered the native form of C3 exoenzyme, reducing its size and adding a transport motif that dramatically enhances its ability to penetrate mammalian cells. The Company moved early to patent these inventions, thereby gaining strong composition-of-matter protection and “freedom to operate”.

While C3 exoenzyme variants were under development, BioAxone also initiated, again in collaboration with the University of Montreal, a program to synthesize and screen small-molecule inhibitors of Rho kinase (or ROCK). This kinase, whose activity is directly regulated through interactions with Rho GTPases, is a key modulator of the mammalian cytoskeleton. Company scientists have already produced and characterized more than fifty such inhibitors based on three independent structural scaffolds. Again, BioAxone has filed to secure patent protection for these inventions.

As mentioned above, BioAxone is leveraging its technical prowess in the fields of molecular biology, protein chemistry and formulation, and preclinical animal models to expand further into the Rho signaling field. For example, based on its success in the treatment of spinal cord injury, BioAxone has begun investigating the use of its compounds to potentially treat neurological pathologies related to eye diseases. One of the Company’s current goals is to transfer the successful neuro-protection and neuro-regeneration seen with spinal cord injury to the rescue of retinal neurons that become damaged by eye diseases such as age-related macular degeneration (AMD), glaucoma, and retinal vascular occlusion (a.k.a. retinal stroke). Preliminary results are very promising, leading BioAxone toward further INDs in these, and eventually, other indications.
BioAxone will also continue to advance its expertise and IP position within the Rho signaling domain by initiating collaborative ventures to screen compound libraries in search of other molecules that modulate key Rho signaling pathway players. The Company also has an eye to evaluate the in-licensing of promising compounds and technologies that would strengthen its scientific platform.