Startups may spring from pure academics or from a healthy and heterogenous mixture of science, business experience, and inspired thinking. Rachel King, CEO of GlycoMimetics, and others brought experience to the company. Her cofounder, Dr. John Magnani, brought original science, and an expert team soon joined in response to the inspiration created by the company’s concept. The germ idea was to make a formerly “undruggable” set of disease targets druggable. Deep, careful studies of molecular structures were required, followed by rational drug design to achieve the goal of small molecule therapy mimicking natural carbohydrates critical to the “glycosylation” of cellular proteins.
GlycoMimetics is developing a drug, uproleselan, designed to mimic a glycan in blocking E-selectin, which may have several results when used with chemotherapy, potentially forcing tumor cells out of the marrow and back into the bloodstream, reducing the effective chemo dose, and lowering chemo-caused side effects such as neutropenia and digestive-tract mucositis.
Another lead compound is rivipansel, which inhibits a wide range of selectins to treat some of the worst symptoms of sickle cell disease (SCD). Uproleselan and rivipansel are both in Phase 3 clinical trials. Earlier candidates for oncology indications, some with other new mechanisms of action (MoAs), are also in the pipeline. It is a formidable lineup and a correspondingly large challenge for a small company such as GlycoMimetics.
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