One thing that was different for me at the Singapore conference compared to the Dubai conference, was that at Singapore It wasn't always me who initiated new contacts. The recognition that I have the ear of many patients and parents and work very hard to help them, has grown considerably and our group has become well known as a good way to reach the thalassemia community. One person who introduced himself to me at Singapore was Bo Hedlund, President of Biomedical Frontiers, the company that has been developing starch desferal, S-DFO. This has been in the works for years and stage 1 trials were successfully completed. The document is attached to this post. An announcement was made at the end of November about an agreement between Therapure Biopharma Inc. and Biomedical Frontiers to produce S-DFO for stage two trials. This is an important step as they get closer to stage 2 trials, but funding remains a big issue.
S-DFO offers a big advantage of other chelators because it is taken only once per week, by IV for about an hour. The chelating effect last a full week. Treatment could also coincide with transfusions to reduce the amount of time spent chelating even further. Desferal only chelates while it is being used. L1 (Ferriprox, Kelfer) only works for about 6 hours so it has to be taken 3 times daily. Exjade works for 24 hours. All have side effects in some patients. I am sure that some patients will also have side effects from S-DFO because a percentage of patients will have problems with any drug, but I do believe there would be a good demand for a chelator that lasts a full week. For those who react to the other chelators, it gives a new possibility and to those who have trouble complying, this would provide an option that may be far more manageable than daily chelation.
This is a very promising drug but it ranks right up there with gene therapy and fetal hemoglobin inducing drugs, as therapies that are showing great success in early trials but underfunding has greatly slowed their development. We have to find ways to help them get funded and by raising awareness about thalassemia on the big stage could do wonders in getting attention. As it turns out, one of our new members has an informal connection to this and also has a career in marketing and wants to make sure that the S-DFO trials get funding. I discussed our Oprah idea with this member and there may actually be a way of getting a proposal to Oprah through this member, so let's get to it and come up with ideas on what should be included if Oprah was to do a show focused on thalassemia. We need to get moving if we are going to see these things become options for thalassemics. We need awareness and we need funding and it won't happen unless we can get some attention for thalassemia. Who knows, but with some big time attention we might even get noticed by the Bill Gates Foundation.
Here is the new announcement. The trial one report is attached.
http://www.prweb.com/releases/2008/11/prweb1675504.htmFor immediate release
Therapure Biopharma Inc. announces manufacturing agreement with Biomedical Frontiers Incorporated.
Contract will support Phase II trials for thalassemia treatment
TORONTO, ON: 26 November 2008 – Thomas Wellner, President and CEO of Therapure Biopharma Inc. today announced that the Company has signed a biomanufacturing services agreement with Minnesota-based Biomedical Frontiers Incorporated for the production of an iron chelating agent used in the treatment of thalassemia, an inherited blood disorder.
Therapure Biopharma will apply experience gained previously in the production of Biomedical Frontiers’ deferoxamine conjugates, which are injectable iron-binding drugs used to neutralize iron build-up in the blood of patients with an iron overload disorder. Biomedical Frontiers believes that their product offers advantages over existing treatments for thalassemia that will result in improved patient compliance and therefore better clinical outcomes.
In addition to manufacturing clinical trial supplies, Therapure Biopharma will provide debt financing that will partially support Biomedical Frontiers’ Phase II clinical program for the product. The contract also gives Therapure Biopharma long-term commercial manufacturing rights when the product is approved by regulators.
“Production of this compound for Biomedical Frontiers is a great example of the flexibility of our modern biomanufacturing facility,” said Thomas Wellner. “We are able to produce the necessary supplies of their compound quickly to support the continuing clinical development of this important new product.”
“We previously contracted with Therapure Biopharma in the manufacture of drug material to support our successful development program”, said Bo Erik Hedlund, President and Chief Executive Officer of Biomedical Frontiers. “Because of this earlier experience, we are very confident in Therapure Biopharma’s ability to produce a high-quality product within the timelines we need to start our Phase II trials. Securing both a manufacturer and a financial contribution from Therapure Biopharma will make it much easier for us to acquire the additional funding we need to complete these studies and bring a more effective and user-friendly treatment for thalassemia and other iron overload disorders to markets.”
About Therapure Biopharma:
Therapure Biopharma Inc. is an integrated biopharmaceutical company that develops, manufactures, purifies, and packages therapeutic proteins. Therapure Biopharma acquired its Canadian-built and conceived facility from Hemosol Corporation, a specialist in therapies derived from hemoglobin, a blood protein. Therapure Biopharma applies scientific, manufacturing, and downstream purification expertise with an intimate understanding of advanced biology, complex proteins, and regulatory processes to develop effective and innovative solutions to advance products from discovery to market.
For more information, please visit: www.therapurebio.com
About Biomedical Frontiers, Incorporated:
Biomedical Frontiers Incorporated (“BMF or the Company”) is a privately held biopharmaceutical company with a focus on developing innovative iron-binding drugs for the treatment of iron overload disorders and acute iron poisoning. BMF's proprietary technical platform involves chemical coupling of drugs to biocompatible polymers. The Company is currently preparing for a Phase II clinical trial for its proprietary lead iron-binding drug, starch-conjugated deferoxamine, in patients with transfusion dependent thalassemia.
For more information, please contact: Bo Hedlund, Ph.D. 612-281-4033
- 30 -
Media Contact:
Alison Withey
alison@bluewhalecommunications.com
Here are some excerpts from the stage 1 trials.
It has been suggested that many of the complications of
thalassaemia, including heart disease, are more closely related
to the ‘labile’ or ‘chelatable’ component of non-transferrinbound
iron (NTBI) than to total body iron burden (Hershko
et al, 1978; Porter et al, 1996; Jensen et al, 2003; Cabantchik
et al, 2005). Reversal of cardiac failure occurs rapidly during
continuous administration of DFO (Davis & Porter, 2000;
Anderson et al, 2004), long before significant changes in
cardiac iron. This suggests that the presence of continuous
chelating capacity in the circulation may be beneficial. S-DFO
appears to provide such protection in a dose-dependent
fashion with excess free iron-binding capacity in the circulation
for more than 6 d, on average, in the high-dose group...
In summary, a single infusion of S-DFO provided several
days of iron balance and almost a week of unsaturated iron
binding capacity in the plasma. The latter property may
reduce organ damage caused by toxic free iron. With regard
to iron balance, it is anticipated that multi-dose studies of
S-DFO, infused at the time of transfusion, in combination
with an oral chelator will demonstrate enhanced efficacy.
Finally, the low acute toxicity of S-DFO suggests that it
may be an optimal agent for treatment of acute iron
poisoning.