Success Story

Rho Guidance Leads to Expanding Sponsor’s Proprietary Formulation to Unmet Needs in Rare Disease

Rho supported a small pharma company that was developing a product for males with low testosterone beginning with their initial Phase 1 study. The sponsor received results on a Phase 2 study showing that their product was very safe but only modestly effective in the target population, forcing them to go back to the drawing board on their formulation and program. Following consultation with Rho, the sponsor began exploring the possibility of using their proprietary formulation to treat other unmet needs, including cancer and a rare disease that causes delayed growth in adolescents.

Rho successfully authored and submitted an orphan drug designation application to FDA for the adolescent rare disease, and the product was granted orphan designation. In parallel, Rho led an IND submission for the sponsor’s cancer program on a very tight timeline. Because of these efforts, the sponsor was able to solicit investment funding, establish strategic and long-term business partnerships, and file patent applications for these products.

This sponsor has continued to award new work to Rho, including another IND submission for a new product and a second orphan drug designation application. The Chairman & CEO had the following to say about this success:

Congratulations to the entire team for a tremendous effort! Thank you [Rho] for an excellent job executed very efficiently!

Chairman & CEO, sponsor company