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

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.

 

Hi Aym,

Nice to see you posting. 

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.

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

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

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

I believe the drug is similar to butyrate but does not suppress bone marrow production of RBC's. These are natural fatty acids that do not have the side effects of hydroxyurea.

Ever since I have been involved with thal, my intuition has told me that treatment is approaching thal from the wrong direction. Blood from someone else will always be rejected by the body. My feeling has always been that finding a way to get the body to produce its own blood was the direction that made the most sense. We may finally be on the verge of this and I plead with every single patient to comply with treatment fully, because things are happening so fast that treatment will undergo radical changes in the next 20 years. Have faith because this is for real. These doctors who have been working on hemoglobin induction and gene therapy are closer than ever to real success. The great thing about hemoglobin induction is that there is no iron overload caused when your own blood is produced. Fetal Hb may not be quite as efficient as adult Hb but it does the work needed and is an adequate substitute.

Thank you Andy,
Wow, you are a wealth of information - with great intuition as well. 
I hope for the sake of everyone who can be potentially affected - that this oral drug will replace the current transfusion/chelation treatment.  What a lifestyle change that would be! 

Andy, if the trials are a success do you think that this treatment will be available to patients soon?  (within the next couple of years?) 

Thanks again,
Sharmin

Hi

Last year i emailed Dr. Suzane Perrine to ask if i can enroll Ahmad in the butyrate studies as one of the patients here that i know. She answered me saying that the study was closed and there will be a new one in summer 2007 with an oral medicne. I guess that this is the one we are talking about. She asked me to send Ahmad's Blood in a tube  to Boston to try the medicne on his blood in the lab to see the effect. Actually, i searched every possible way to send the sample but couldn't because no shipping company would carry a blood sample without any kind of agreement or consent from the receiving country especially the States. I tried to email her to solve this, but she seems too busy and didn't answer. I will try again and hope she will answer my email

Manal

It's hard to say how long between the beginning of trials and eventual approval of a drug, but the average time is around 8 years. Prior to that, many patients normally get the chance to participate in phase 3 trials. If a drug is put on the fast track after early trials it can speed up the process, as it did with exjade. The potential  importance of this drug may allow for fast track status if early trials are successful and side effects, if any, are manageable.

I've had high hopes for the new fetal hemoglobin drug that Dr Susan Perrine has spent over 20 years developing and with the recently announced completion of stage 1 trials, HQK-1001 has performed as expected. In terms of therapies that have the possibility of helping the largest number of people, fetal hemoglobin inducers, led by HQK-1001 hold the most promise. To me, this is the best approach. If your own body makes your blood, you avoid the myriad of problems transfusions can lead to, basically making thalassemics normal. Such a simple idea but so long in coming.

http://www.medicalnewstoday.com/articles/132542.php

  Medical News Today
HemaQuest Pharmaceuticals Announces Clinical Progress On New Sickle Cell Anemia Drug
10 Dec 2008   

HemaQuest Pharmaceuticals announced today the successful completion of Phase 1 clinical trials of HQK-1001, an orally-administered therapeutic which the company is developing to treat hemoglobin disorders, including sickle cell anemia and beta thalassemia. The company presented the results at the annual meeting of the American Society of Hematology in San Francisco.

HemaQuest performed two different trials. In the first placebo-controlled clinical study, 32 healthy volunteers were given single doses of HQK-1001, ranging from 2 to 20 mg/kg. This trial was followed by a second placebo-controlled study, in which 41 healthy volunteers received two weeks of daily doses of HQK-1001 ranging from 5 to 15 mg/kg.

In both studies, there were no clinically significant adverse effects, and the incidence of mild side effects was similar in subjects receiving placebo or HQK-1001, the company said. Pharmacokinetic studies showed that single doses at, or above, 10 mg/kg reached the targeted plasma drug levels. Significantly, subjects treated with HQK-1001 in the multiple dose Phase 1 clinical trial provided preliminary evidence of its therapeutic potential, as demonstrated by statistically significant increases in young red blood cells, known as reticulocytes.

HemaQuest Chief Scientific Officer and Vice President, Clinical Affairs, Susan Perrine, MD, said, "We are pleased to report these results demonstrating biological activity and favorable pharmacology of HQK-1001, and show that the drug was well-tolerated. We are very encouraged that a brief two-week treatment with HQK-1001 generated significant increases in reticulocytes, indicating that it may offer therapeutic benefit in hemoglobin disorders and other anemias."

HemaQuest President and CEO Ronald Berenson, MD, said, "These initial clinical studies provide the foundation for subsequent testing of HQK-1001 in patients with hemoglobin disorders, including sickle cell anemia and thalassemia in early 2009. If results of our Phase 1 clinical trials are confirmed in these upcoming studies, HQK-1001 may also be beneficial for treating other, common types of anemia characterized by reduced production of red blood cells."

Sickle cell anemia and beta thalassemia afflict millions of people, and are the most common genetic diseases. In the United States, there are nearly 80,000 patients suffering from one of these two inherited blood diseases. Few worldwide therapeutic alternatives exist for these serious and life-threatening diseases, which are associated with significant morbidity and reduced survival, creating a strong and pressing need for new treatments.

About Hemaquest Pharmaceuticals

HemaQuest Pharmaceuticals, Inc., established in late 2007, is a biopharmaceutical company focused on developing oral, small molecule therapeutics to treat hematological diseases including hemoglobin disorders. These therapeutics are based on short chain fatty acid derivative (SCFAD) technologies, which were discovered by Susan Perrine, MD, and her colleagues at Boston University. The company's first therapeutic, HQK-1001, has received orphan status drug designation in the U.S. for both sickle cell anemia and beta thalassemia. HemaQuest's investors include De Novo Ventures, based in Palo Alto, Calif.; Forward Ventures, based in San Diego; and Lilly Ventures of Indianapolis, Ind., the venture capital arm of Eli Lilly and Company.

Hemaquest Pharmaceuticals

Article URL: http://www.medicalnewstoday.com/articles/132542.php

This is excellent news and combined with the orphan drug status given this drug by the FDA, we can expect to see progress continue at a fast pace. Getting the body to make its own hemoglobin is the goal whether it's bone marrow transplant, gene therapy, or hemoglobin inducing drugs, but the latter holds the most promise for the most people and will result in much better lives for thalassemics and also make treatment far more simple and far less costly over the lifetime of the patients.This was a very short trial but the results were exactly what they hoped for and my prediction is that stage two trials will create the biggest news in the world of thalassemia since the introduction of desferal.

Andy,

This is so terrific!!!  Is this drug something that is most likely to help thalassemia intermedias only or would it be helpful to thal majors as well?  I ask because during our recent visit the doc told us that my son is definitely a beta zero and unlikely to benefit from drugs such as hydroxy urea. 

If this drug would come in time we can stop worrying about antibodies etc..... and so many people world wide would benefit so much!!!

Thank you for sharing this news Andy,

Sharmin

Sharmin,

I would not discount the possibility that fetal hemoglobin inducers may have benefit for beta zero majors. In her article from 2005, Dr Perrine talks about the potential for using a combination of these drugs to raise the hemoglobin level even more. (http://asheducationbook.hematologylibrary.org/cgi/reprint/2005/1/38.pdf) I think we will have to wait and see what a longer trial brings in terms of overall improvement in hemoglobin. Dr Vichinsky has spoken to you about the possibility of using some of these drugs. Obviously the best way to avoid blood reactions, including antibody reactions, is to not take blood, so this does have to be examined as a possibility for little A, if it becomes possible. Part of the ongoing investigation with these drugs involves what combination of inducers works well, along with what else can be done to help create more red blood cells and extend their lives. I think antioxidants will play a role in this combination.

One ironic point, is that it has been found in intermedias using Hb inducers, is that they often need iron supplementation to fully take advantage of the potential of these drugs, even though they have high iron stores. The body does not easily use stored iron and prefers to extract it from the gut, so it may be necessary to provide the iron to promote the formation of new red blood cells.

This can be the answer to our prayers.

Thanks for the info Andy.

Zaini.

I'm glad for you guys!!!

 

My apologies, but I am not in the correct shape to understand this perfectly. Do you think it can only help patients with pyruvate kinase deficiency? To respons at my previous post: I don't think so.... (Does I use the correct English grammar?)

Dore