John Crowley established a start-up biotechnology firm, Novazyme Pharmaceuticals, to
develop drugs that treat a lysosomal storage disorder from which his children suffered.
Novazyme’s overall strategy was to quickly develop the Pompe enzyme for clinical trials and
launch a drug to the market after getting FDA approval. It raised a total of $8 million from
Series A round (venture capitalists include Perseus-Soros and HealthCare Ventures).
Novazyme would attempt to get Accelerated Approval and Priority Review since they
have positive preclinical data, and there is a huge market demand. Although the trail phases
have great uncertainties, Novazyme was confident with their trail design, and FDA allowed
them to proceed with the trails.
However, creating an enzyme at a macro level need huge manufacturing facility, which
cost a lot as well as adhere to GMP guidelines. Therefore, Novazyme built a small and
internal GMP plant in Oklahoma City so it can manufacture HP-GAA on its own. This
manufacturing facility had the capacity to produce the material needed for the Phase I and II
clinical trials.
Currently, the company had a strong management team and R&D group and continued to
realize positive preclinical results with HP-GAA and other LSD enzymes. They still need a
significant amount of capital to successfully commercialize HP-GAA. Two companies show
their interest, and Novazyme must make choice:
