A Potential Cure for Sickle Cell Anemia from GlycoMimetics (NASDAQ:GLYC) and Pfizer (NYSE:PFE)

May 3, 2017

Rachel K. King is Co-Founder and Chief Executive Officer of GlycoMimetics Inc (NASDAQ:GLYC).  In a recent interview with the Wall Street Transcript,  Mrs. King outlined the prospects for a drug therapy her company has created and is currently in Stage III testing.  

From the interview:

TWST: How many people have sickle cell disease in the United States?

Mrs. King: About 100,000, which makes it actually the most common genetic disease in the United States. If you think about diseases like hemophilia, cystic fibrosis or muscular dystrophy, you add them all together and there are still more people with sickle-cell disease. In the U.S., it mostly affects people of African descent, but it also affects people who have come from other parts of the world where malaria is prevalent. The disease grew up along with malaria because people with one copy of the gene are actually protected from malaria while people with two copies have sickle cell.

Mrs. King: People with sickle cell disease have, as one of the most problematic manifestations of the disease, these events called sickle cell crisis or vaso-occlusive crisis. They are intensely painful events whereby the blood flow becomes occluded in the micro vessels throughout the body. Right now, the only thing that the patients can be given when they are having a vaso-occlusive crisis to get through the crisis is pain relief and supportive care like hydration. Because the events are so intensely painful, they have to get intravenous narcotics and to be hospitalized while they are getting those powerful drugs.

On average, people in the U.S. are hospitalized for about six days, and there are 90,000 to 100,000 of these hospitalizations every year in the United States for people who have vaso-occlusive crisis. Virtually every patient with sickle cell disease will have a crisis. But there is quite a lot of variability in terms of how often an individual person might have a crisis, and that might change over their lifetime.

So a patient might have one in a certain year, and then have none in the next and in another year have three. Or some people might regularly have five crises a year. There is heterogeneity in the population in terms of how many crises a person will have, but these crises are the most visible, common and serious manifestations of sickle-cell disease. Over the lifetime of a patient, the added effects of the crises and of the consequences of impeded blood flow all add up to people dying young. Most sickle-cell patients in the U.S. die in their 40s.

A description of the joint testing with Pfizer Inc. (NYSE:PFE):

“…We signed that deal in 2011 to basically give Pfizer Inc. the right, once we completed Phase II, to then take it forward if they chose to do so into a Phase III and commercialize it. In fact, the Phase II data was very positive. So Pfizer decided to take the drug into Phase III, which is currently what they are doing in treatment of sickle-cell crisis.”

For more drug therapies currently being developed by GlycoMimetics, and many other interviews regarding Medical Research and Genomic Diagnostic companies, read the full report at the Wall Street Transcript.