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HemaQuest Announces Initiation of Phase I Clinical Trial of HQK-1001

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Aym:
Hello everyone...

Does anyone has more information about this clinical trial? how long will it take to get approval from the FDA? thank you...


Jan 17, 2008 09:00 ET
HemaQuest Announces Initiation of Phase I Clinical Trial of HQK-1001 to Treat Hemoglobin Disorders

NEWTON, MA--(Marketwire - January 17, 2008) - HemaQuest Pharmaceuticals, which received its first round of venture financing three months ago, today announced that the U.S. Food and Drug Administration has accepted its application for an investigational new drug (IND) and has started a Phase I clinical trial of its orally administered, patented therapeutic, HQK-1001. The product is being developed to treat the two most common inherited blood diseases, sickle cell anemia and thalassemia.
The Phase I clinical study is being carried out in healthy volunteers, who will receive increasing doses of HQK-1001 to evaluate its safety and pharmacokinetics in anticipation of future clinical trials in patients with these hemoglobin disorders.
HemaQuest President and CEO Ronald Berenson, MD, said, "We are pleased to reach this important milestone within three months of the founding of our company. We are committed to continuing the rapid development path for HQK-1001, which has the potential to be a major advance in treating serious and life-threatening blood disorders."
Susan Perrine, MD, the company's chief scientific officer and vice president of clinical affairs, whose discoveries led to HQK-1001, added, "This clinical trial is the culmination of many years of laboratory and animal studies of HQK-1001. It represents the first step in the clinical development of this exciting new therapeutic, which could make a significant impact on improving the lives of the many patients with sickle cell anemia and thalassemia."
ABOUT HEMAQUEST PHARMACEUTICALS
HemaQuest Pharmaceuticals (www.HemaQuest.com) is a Boston-based biopharmaceutical company focused on developing small molecule therapeutics based on its short chain fatty acid derivative (SCFAD) technologies to treat anemias and other blood disorders. Its initial therapeutic focus is on thalassemia and sickle cell anemia, which afflicts several million patients worldwide and approximately 80,000 patients in the USA. There are currently few therapeutic alternatives for these diseases, which are associated with significant morbidity and shortened patient survival. HemaQuest is also developing other SCFADs that could prove useful in treating other hematological disorders, such as other kinds of anemia and neutropenia.
The company's investors include De Novo Ventures, a Palo Alto, CA life sciences venture capital partnership; Forward Ventures, a life sciences venture capital firm based in San Diego; and Lilly Ventures of Indianapolis, IN, the venture capital arm of Eli Lilly and Company.

 

Andy Battaglia:
Hi Aym,

Nice to see you posting.  :happyyes

Dr Susan Perrine has been studying short-chain fatty acids as potential fetal hemoglobin inducers for many years. Butyrate is one of these drugs that is already in use and has had some limited success in some thal patients. A major deficiency in drugs like butyrate and hydroxyurea has been that while they increase fetal Hb production, they also suppress erythropoiesis (the process of RBC formation or production). The goal has been to find a drug that can induce fetal Hb without also reducing RBC production. The culmination of this research is now about to begin human trials. I do not believe that any details of these trials, including their length, has yet been released.

I have previously mentioned that researchers feel that they do know of fetal hemoglobin inducers that will work well enough to have a significant impact on the lives of thalassemics, but that the money for research was very hard to obtain. In November, Ashish Vazirani showed me an article on his news website that he had been developing with the intention of linking with our site, that talked about HemaQuest having raised $20 million to begin trials. In just a short time these trials have been approved.  http://thalguy.blogspot.com/2007/07/new-biotech-raises-20m-first-vc-round.html
It is believed that hemoglobin levels can be raised to levels that would eliminate the need for transfusions in many.

Since this technology is related to treatment that already is used, it has the potential of moving along quickly. Hopefully, more information will soon be available.

Dr Perrine has been at the forefront of this research for many years, and as one of the founders of HemaQuest, she stands to make a lot of money if this research is successful and I feel that she deserves every penny for her long dedication to finding ways to better the lives of those suffering from blood disorders. I wish her the best in this endeavor which may radically alter the way thalassemia and sickle cell anemia are currently treated. This one could be huge, folks. We will be following this closely.

Kathy11:
THis is fantastic news. we can only hope for a good result after the trial.
Kathy

Sharmin:
Hi Andy,

I hope that these trials are successful and soon.

Do you know what this treatment entails?  In the past fetal hg inducers were chemotherapy drugs with many potential side effects.  Is this the same concept? 

Thanks Andy,

Sharmin

priya:
WOW its really very good new. I wish for the success and good results of these trails. This type of news really gives me great hopes.

Thanks Mr. Andy  & Amy

Regards

Dimple

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