HemaQuest raises millions, and moves headquarters from Seattle to Carmel Valley

CARMEL VALLEY ---- With $13 million  recently added to its coffers,   HemaQuest Pharmaceuticals Inc.  is proceeding with testing a new approach to treating sickle cell disease.

Also referred to as sickle cell anemia, the disease produces red blood cells with defective hemoglobin, the oxygen-carrying protein. The diseased cells take on a characteristic crescent shape. The cells are both rigid and fragile and die early, reducing the supply of oxygen to the body. They also clump up in capillaries, obstructing blood flow, events known as "crises." These events cause pain, cut off oxygen to the affected area, and sometimes result in tissue death or damage. Life expectancy is shortened.

HemaQuest's experimental drug causes patients to produce a form of hemoglobin normally found found in fetuses. Adults who naturally make the fetal hemoglobin due to a mutation live healthy lives, so there's reason to think boosting fetal hemoglobin will reduce symptoms. The company's drug, called  HQK-1001 , is now progressing through Phase 2 clinical trials, when effectiveness is tested. The company has also completed a Phase 1 trial of the drug for  beta thalassemia , another form of anemia.

In March, the privately held company said it had raised $13 million from venture capital investors to fund its work.

To make its money go farther in drug development, HemaQuest functions as what's called a "virtual" biomedical company, hiring only essential employees and contracting out everything else. The company has just 7 employees, said Tamara A. Seymour, the company's chief financial officer. That's even fewer than when the company was headquartered in Seattle, when it had nine there and one in Boston, where the technology was developed.

HemaQuest moved its headquarters to San Diego about a year ago, said Seymour, who joined the company in late 2010. At the same time, the company hired John P. Longenecker as president and CEO; and Dr. Richard Ghalie as chief medical officer. The hirings and the move were related, Seymour said: All three live in San Diego, the first two living in Carmel Valley itself. So they figured it made sense to locate the company there. The company maintains an office in Seattle.

If the company's drug works, it would be a true breakthrough. Treatments include blood transfusions and the cancer drug hydroxyurea , which reduces the severity of the clumping crises. Neither provide continual relief, and hydroxyurea produces unpleasant side effects such as bone marrow damage.

The drug holds promise as an alternative to hydroxyurea for both sickle cell disease and beta thalassemia, said sickle cell researcher Betty Pace of  Georgia Health Sciences University.

"Inducing fetal hemoglobin and increasing the red cell count are very beneficial in both patient populations," said Pace, a molecular and cell biologist who served on the advisory panel for the Phase 2 trial in sickle cell disease.

The genetic defect that causes sickle cell disease was discovered 60 years ago. It's the smallest possible mutation, a change of just one letter of the genetic alphabet in the gene that codes for hemoglobin. But that tiny change destroys most of the protein's function.

In the United States, the disease is most common in blacks. It's also prevalent in the regions of sub-Saharan Africa where their ancestors were taken as slaves. But the disease is not just a "black" affliction; it's also found in Mediterranean countries such as Italy and Greece.

What these areas have in common is that malaria is or has been prevalent. One copy of the gene offers some protection against malaria without otherwise harming the carriers, so the trait has been preserved by natural selection. But those with two copies, inherited from both the father and the mother, get the sickle cell disease.

"There are approximately 100,000 sickle cell disease patients in the U.S., 40,000 in Europe and more than 250,000 in the Middle East," Seymour said. "It is also estimated that 12 million people in Sub-Saharan Africa have sickle cell disease. ... It is estimated that it cost an average of $230,000 annually for each patient, or $9 million lifetime for each patient."

To help spur development, the U.S. Food and Drug Administration and European regulators have given HemaQuest orphan drug status. The designation, meant for relatively rare diseases, gives the drug more generous protection against competitors.

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