FIRST-in Class Therapy with PAX-001

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Humans:

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The initial target for humans is PAX-001 to optimize repair of hand flexor tendon injuries, with 100,000 Flexor tendon injuries per year in the US alone. PAX has completed a GLP toxicology and dose-range finding study under guidance by the FDA, in preparation for filing an IND application anticipated for 2027.

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Future conditions for which PAX-001 will be applicable include other tendon injuries (e.g. Achilles tendon and extensor tendon injuries) as well as ligament tears (e.g., Anterior Cruciate Ligament- ACL, and medial collateral ligament- MCL). Future applications will include administration via injection through the skin when the rupture is incomplete, (such as for some flexor tendon injurites) thus avoiding surgery.

 

Veterinary applications:

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Horses: 30% of thoroughbreds have career-threatening Achilles tendinosis, and up to 20% of horses with Superficial Digital Flexor Tendon injuries need euthanasia on the racetrack.

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Dogs: Dogs commonly tear the Cranial Cruciate Ligament (the equivalent of the human ACL).  

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Note: in order to attain Proof of Concept as rapidly as possible, PAX Therapeutics if focusing our efforts and financial resources in advancing as rapidly as possible for humans. 

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Goal of PAX-001

 

Delivery of a growth factor via a vector results in the production of the protein for the optimal duration of expression; recombinant protein did not persist long enough for in vivo effect, whereas the vector-mediated expression was optimal. In our animal studies, the protein was no longer detected by 16 weeks, i.e., after healing was complete (around 8 weeks).

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One of the most valuable aspects of our therapy is that it is a one-time local injection and not a systemic drug. The doses required are orders of magnitude less than what are used for other indications.