Hemaquest Initiates Clinical Trials in Sickle Cell Disease and Beta Thalassemia

Some Good News:

http://enthrallmedia.wordpress.com/2009/05/26/hemaquest-initiates-clinical-trials-in-sickle-cell-disease-and-beta-thalassemia/

Enthrall Communications & Media Portal
ENTHRALL – Social. Digital. Visual.
HemaQuest Initiates Clinical Trials in Sickle Cell Disease and Beta Thalassemia
SEATTLE – May 27, 2009 - HemaQuest Pharmaceuticals announced today that it has initiated clinical trials of HQK-1001 in the treatment of patients with sickle cell disease and beta thalassemia. These two clinical trials are intended to evaluate safety and provide proof of concept clinical data in patients with these serious and life-threatening chronic illnesses.

“Our team is excited to have the opportunity to work with some of the leading clinical investigators to test HQK-1001 in sickle cell disease and beta thalassemia,” said Ron Berenson, M.D., President and Chief Executive Officer of HemaQuest. “There is a pressing need for new drugs to treat these disorders, which cause significant morbidity and early mortality. The goal of our clinical trials is to have sufficient data to move HQK-1001 into advanced clinical studies.”

Each of these blinded, placebo-controlled studies will assess the safety of HQK-1001 and evaluate indicators of therapeutic activity, including several measures of fetal globin, one of the drug’s primary targets. Increases in fetal globin correlate with improved clinical outcomes in patients with these hemoglobin disorders. The trial in sickle cell disease is being conducted at approximately 10 centers in the U.S. The trial in beta thalassemia is being conducted in Thailand, where there is a high incidence of this disease.

To date, HQK-1001 has been evaluated in 55 healthy human subjects in 2 clinical studies. In the first study, 24 subjects were treated with single doses of HQK-1001 at 4 dose levels. The second study was conducted in 41 healthy human subjects, who were treated with 14 consecutive days of HQK-1001 at 3 dose levels. HQK-1001 was well-tolerated at all dose levels and there were no serious adverse effects in the two studies. Plasma drug levels associated with in vitro biological activity were achieved in both studies.

About HQK-1001

HQK-1001 belongs to a class of compounds originally discovered at Boston University and licensed to the company. These compounds, designated as Short Chain Fatty Acid Derivatives (SCFADs), have been shown to stimulate fetal globin expression in the laboratory and in small clinical trials in patients with hemoglobin disorders, including sickle cell disease and beta thalassemia. HQK-1001 is an orally administered SCFAD, which has shown potent effects on fetal globin induction and red blood cell production in the laboratory and relevant animal models. Additionally, the company has received orphan drug designation for HQK-1001 in the United States and Europe for both sickle cell disease and beta thalassemia.

About Sickle Cell Disease and Beta Thalassemia

Sickle cell disease is a genetic disorder affecting the beta globin chain of adult hemoglobin, resulting in distorted, rigid sickle red blood cells, which block blood vessels. The resulting lack of oxygen causes acute episodes of pain (pain crises), lung injury (acute chest syndrome) and is associated with strokes. Chronic damage occurs in many organs. The only drug available to treat the disease is a cancer chemotherapy drug, hydroxyurea, which has potential risks for patients. The lifespan of sickle cell patients is reduced in the U.S, where there are approximately 75-80,000 patients.

Beta thalassemia is a prevalent blood disease worldwide in which patients are unable to produce normal amounts of beta globin, which results in severe anemia. The primary treatment, red blood cell transfusions, leads to iron overload that damages many organs and requires treatment with iron chelating drugs. There is early mortality in patients with this disease is in the U.S. today.

About HemaQuest Pharmaceuticals

HemaQuest Pharmaceuticals, established in late 2007, is a biopharmaceutical company focused on developing small molecule therapeutics based on its proprietary SCFAD technologies to treat hemoglobin diseases. HemaQuest is also developing other SCFADs that could prove useful in treating other hematologic disorders. The company’s investors include De Novo Ventures, Forward Ventures, and Lilly Ventures.

For More Information
Jerome Lyons
T: 206.826.9900
jlyons (at) hemaquest.com

Hoping that this will yield great results,

Sharmin

Hi Sharmin,

Thanks for the update.

Just to give some background that HQK1001 phase 1 trials were completed in December 2008. These trials were conducted on healthy individuals to demonstrate that the drug is well-tolerated. These trials provided the foundation for subsequent testing of HQK-1001 in patients with hemoglobin disorders (sickle cell and beta thalassemia), so it has began as Sharmin reported.

For background info, please visit

http://www.hemaquest.com/HTML/press_releases.html

Thanks.

Thanks Canadian Family:) 
I hope that considerable benefit is noted and that it will be reported at the upcoming conference in New York.  I hope to see you there:) 

Sharmin

It was great to hear Dr. Perrine talk.  Her discussion of this drug sounded very promising.  I hope that her current trials go very well.  This could change the management of thalassemia greatly. 

Sharmin

When we spoke to Dr Perrine privately, she shared with us that the trials are looking promising, but until the trial ends and data is collected, they won't know for certain, but she said an Hb increase of 3 was being seen.

Anyone in the US interested in taking part in the Hemaquest trial, please message me for the contact info to take part.

Thanks Sharmin and Andy for this update, so eager to know the results and to try this drug

manal

Dr. Perrine was one of a few doctors who really stood out and had something tanglible to offer at the conference.  There were many studies that are essential to the understanding and treatment of thalassemia - but the studies were at such early stages involving mirco biochemical concepts that we could not relate to at this point. 

Dr. Perrine's study can lead to an actual solution for hemoglobinopathies.  Dr. Saidelain's work was also very impressive and relevant in the near future.  I think that Genetics Pharmaceuticals trials will allow themselves and others to learn - and the patient they treated has been transfusion free for quite some time - their work has some details that may need to be ironed out.  Going head to head, these two groups may offer a cure for thalassemia in the next few years. 

Bone marrow transplants have also come a long way, but I think that there is a way to go before they can be considered a safe cure - especially if a matched sibling donor is not available. 

Oakland's work on antioxidant therapies was also very impressive.

Sharmin

that's a good news, sad i am living in Singapore can't take part.


peace...

While this particular trail is only for those in US and Mona, West Indies - please do NOT exclude yourself only thinking trails are for only US residents.

In one of the session, they did talk about the importance of clinical trails and that these trials are worldwide. For those interested in clinical trials related to thalassemia go to http://clinicaltrials.gov and search for Thalassemia.

You can search for the trials in your area if you are interested.