Aytu Licenses Late-Stage Pediatric Onset Rare Disease Asset from Rumpus Therapeutics

Rare Daily Staff

Specialty pharmaceutical Aytu BioPharma said it acquired a global license to Rumpus Therapeutics’ AR101 for rare disease indications, with plans for a pivotal study for the treatment of vascular Ehlers-Danlos syndrome.

Vascular Ehlers-Danlos syndrome (vEDS), the severe subtype of EDS, is a rare genetic connective tissue disorder typically diagnosed in childhood and characterized by arterial aneurysm, dissection and rupture, bowel rupture, and rupture of the gravid uterus. It affects 1 in 50,000 people worldwide and results from pathogenic variants in the COL3A1 gene, which encodes the chains of type III procollagen, a major protein in vessel walls and hollow organs. Twenty-five percent of vEDS patients have a first complication by the age of 20 years, and more than eighty percent have at least one complication by the age of 40. vEDS is a devastating condition, and vEDS patients have a median lifespan of 51 years. There are currently no U.S. Food and Drug Administration-approved treatments for vEDS.

Under the terms of the transaction, Aytu has acquired the vEDS enzastaurin program of Rumpus and all associated intellectual property for $1.5 million upfront in cash. In addition, Aytu paid fees associated with the transfers of third-party licenses from Rumpus to Aytu and will take over any royalty obligations under these licenses. Aytu has also agreed to make performance-based milestone payments to Rumpus upon the achievement of regulatory, clinical and commercial milestones.

The company will pay up to $22.5 million in milestone payments if certain regulatory milestones are met, including $15.0 million in milestone payments if AR101 receives approval by the FDA and another major market regulatory authority. Commercial milestones of up to an additional $45.0 million may be paid if a series of global commercial milestones are met over the life of the product. All milestone consideration may be paid in any combination of cash or stock at the company’s option, with the provision that shares will not be issued in excess of 19.9 percent of the now-outstanding shares of Aytu unless subsequently approved by Aytu’s shareholders.

“This acquisition positions us to greatly accelerate our growth plans both through the addition of this novel rare disease asset to our development pipeline, as well as the expansion of our management team with Rumpus co-founders Topher Brooke and Nate Massari joining the team,” said Josh Disbrow, CEO of Aytu BioPharma. “The AR101 program is expected to progress directly to a single pivotal study, and the Rumpus team has employed numerous strategies in the development plan to minimize clinical risk associated with the trial. We are greatly encouraged by the potential speed to FDA approval, especially as we believe it can provide much needed hope for vEDS patients and their families.”

AR101 (enzastaurin) is an orally available investigational first-in-class small molecule, serine/threonine kinase inhibitor of the PKC beta, PI3K and AKT pathways that has been studied in more than 3,300 patients across a range of solid and hematological tumor types. Pivotal phase 3 studies for both newly diagnosed diffuse large B-cell lymphoma (DLBCL) and glioblastoma multiforme (GBM) are currently being conducted by Denovo Biopharma, enzastaurin’s owner. Enzastaurin received orphan drug designation in DLBCL and GBM from the FDA and EMA, and Fast Track designation from the FDA. Aytu has secured exclusive global rights to enzastaurin/AR101 from Denovo Biopharma in the fields of rare genetic pediatric onset or congenital disorders outside of oncology.

Photo: Josh Disbrow, CEO of Aytu BioPharma