Dive Brief:

Fortress Biotech is hard to characterize. It is a hybrid of a traditional biopharma developing its own clinical candidate and a venture capital company securing financing and providing hands-on support for the companies it launches.

Describing itself as therapeutic area-agnostic, Fortress and its ten subsidiaries are developing drugs and devices across a wide range of fields, including dermatology, cancer, trauma, transplantation and neurology.

Most of the candidates under development by its subsidiaries are in preclinical or Phase 1 testing, but several from Avenue Therapeutics and Cyprium Therapeutics have advanced into mid- and late-stage testing. Another Fortress company, Journey Medical Corp, markets four drugs for skin conditions and wounds.

Aevitas will take aim at aHUS and PNH, two rare diseases with still unmet medical needs. While gene therapy offers an attractive approach to addressing each, other companies are already established in the space. Alexion Pharmaceuticals, Inc. has had success marketing Soliris (eculizumab) for both conditions.

Elsewhere in clinical testing, Ra Pharmaceuticals, Inc. is developing its Phase 2 agent RA101495 while Alnylam Pharmaceuticals, Inc. has ALN-CC5 in Phase 1/2.

Aevitas isn't Fortress' first step into the growing field of gene therapy. Cyprium, which was launched in March, is developing a gene therapy for a rare inherited copper metabolism disorder called Menkes disease.

If this Menkes disease is not treated before or soon after birth, it can cause developmental delay, epilepsy and death before the age of three. AAV-ATP7A, as Cyprium's gene therapy is known, is currently in preclinical development.

Under a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child health and Human Development, Cyprium gained access to a Phase 3 candidate for Menke's disease that it hopes to pair with its gene therapy.

As for Fortress Biotech itself, its business was boosted considerably through the September 2016 acquisition of National Holdings Corporation, which added $46.3 million to net revenues from the second quarter of this year.

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Fortress adds 10th subsidiary to biotech network - BioPharma Dive