Celiac Disease Drug Therapy - $8 Billion Market by 2019

A new study about the market for Celiac Disease Drug Therapy was recently released by a company called DataMonitor. The UK-based Datamonitor Group is an independent, premium business information and market analysis company that assists clients with operational and strategic decision-making.

Some interesting tidbits from the study...

A drug launch in Celiac Disease is up to 5 years away. Datamonitor estimates that under optimal conditions drug sales could reach $8 billion by 2019 in the seven major markets (US, UK, Germany, France, Japan, Spain & Italy). Two market access strategies are available to companies, which are dictated by the targeted Celiac Disease subpopulation and drug price point.

The pipeline is small, and not advanced past Phase II, but candidates show various approaches to potentially treating Celiac Disease. Small, venture-backed biopharmaceutical companies power R&D, but Shire Pharmaceuticals recent in-licensing of the lead pipeline candidate AT-1001 (Alba Therapeutics) shows the promise of future Big Pharma involvement.

Low disease awareness among primary care physicians is a major factor behind low diagnosis rates (5% in the US to 25% in the EU) in Celiac Disease. Continued efforts to increase awareness, especially once a drug comes to market, will drive Celiac Disease diagnosis rates up to 50%-60% by 2019, which will expand the overall patient population.

Some other interesting points...

• Datamonitor estimates there are between 4 million and 7.5 million Celiac Disease sufferers in the seven major markets in 2009.
• Potential worldwide Celiac Disease population could be upwards of 25 million.
• Over three million Celiac Disease sufferers in the US. Celiac Disease is extremely rare in Japan and few epidemiology studies exist. France lacks population-based studies for Celiac Disease. Lower Celiac Disease prevalence in Germany than in the UK. There is an abundance of robust epidemiology studies for Celiac Disease originating from Italy. Spain has the smallest Celiac Disease population in the seven major markets. UK prevalence of Celiac Disease is the highest of the major EU regions outside the seven major markets.
• Pharmacological treatment will not replace the gluten-free diet.
• Small, privately held, biopharmaceutical companies are the power houses in the field of Celiac Disease research and development.
• Key opinion leaders believe that enzymatic degradation of gluten holds the most promise for Celiac Disease.
• Alba Therapeutics (in Phase II trials) estimates AT-1001 could launch for Celiac Disease in the US by 2011 and would have a value of $500 million.
  
• GlaxoSmithKline could exercise its option to commercialize CCX-282 (Traficet-EN; ChemoCentryx - also in Phase II trials) with proof-of-concept data.
• ALV-003 (Alvine Pharmaceuticals) is an oral, two-enzyme cocktail designed to degrade dietary gluten. Alvine Pharmaceuticals initiated a Phase I proof-of-concept study in human volunteers and Celiac Disease patients.
• Celiac Disease vaccine currently under development by Nexpep in Australia. Celiac Disease is well suited to a therapeutic peptide-based vaccine. Nexpep aims to put a peptide-based vaccine into Phase I trials in H1 2009.
  

The 150 page report can be purchased here for only a mere $3800.

The End Of The Gluten Free Diet for Celiacs?

Some of you may have already seen the news that came out in April about the Celiac Disease vaccine trials that started in Australia. The research being conducted by Bob Anderson of the Walter and Eliza Hall Institute of Medical Research is the first of it's kind in the world. Basically, people would be able to get a vaccine for Celiac Disease which would turn off the immune-system-attack-gluten-response in the body. The research has already shown successful on mice (I didn't know mice could get Celiac Disease) and now 40 brave soles will get doses of the vaccine over the next 11 months. If all goes well, a phase II trail could begin in 2011.

The vaccine therapy involves repeatedly injecting solutions of gluten at increasing concentrations. The goal is to reduce and ultimately eliminate gluten sensitivity slowly, in a manner similar to common allergy desensitization treatments.

So is this the end of the Gluten Free Diet treatment for Celiac Disease (and thus the end of the Gluten Free Raleigh Blog)? If this works, couldn't I just start with a crumb of a donut and then eat larger and larger portions each day? Sorry, that's a joke - even though I really miss donuts. Bob Anderson (a gastroenterologist and PhD scientist) has been working for years to raise funding for his research on this topic. He formed a company called Nexpep in 2007 to conduct this research and find a viable vaccine. Check out Nexpep's Youtube page here. There are hopes that this type of research can work for other immune diseases like Diabetes, RA and MS.

Question - if a vaccine were available for Celiac Disease, would you take it? Is the Gluten Free Diet too expensive and too difficult to follow?

Alba Therapeutics & Larazotide Acetate Update

I have written previously about the Celiac Disease drug therapies that are currently under development around the world. Alba Therapeutics is one company that is doing research and development with a drug called larazotide acetate. Here is an update from Alba posted yesterday on the PRNewsWire.

Alba Therapeutics Corporation announced today it will initiate further clinical studies for patients diagnosed with celiac disease with their lead compound, larazotide acetate. "Our most recent Phase IIb study, the largest trial with an investigational medication conducted in patients with celiac disease, was a randomized, double-blind, placebo controlled, dose ranging, multicenter study to determine the safety, tolerance and efficacy of larazotide acetate in 184 adults with controlled celiac disease during a gluten challenge. The data are promising and will be publicly presented at upcoming scientific meetings this spring," said Bruce Peacock, CEO of Alba Therapeutics. "Recently, Alba restructured to concentrate its efforts on advancing the clinical program for larazotide acetate, and we are now moving forward with initiating our next clinical trial in patients with celiac disease."

I had written earlier about the "restructuring" (layoffs) at Alba and have since been wondering what the status was at the company. It appears as if the company is moving forward, solely focused on using larazotide acetate as a treatment for Celiac Disease. Just as a side note - this drug and all the others I have written about are designed to be used in conjunction with the lifelong Gluten Free Diet. Their goal is to eliminate the damage done by unintentional Gluten exposure - not to allow the person to eat as much Gluten as they would like and then just pop a pill afterward.

Alba Contact Information
Contact: Wendy Perrow, MBA
Phone: 410-878-9850
E-mail: info@albatherapeutics.com
Web site: http://www.albatherapeutics.com

Alba Therapeutics Layoffs

I wrote about Alba Therapeutics last year in my series on Celiac Disease Drug Therapies currently under development. They are currently developing a drug for Celiac Disease called Larazotide acetate (formerly referred to as AT1001). There are 2 clinical trials underway for the drug to test it's effectiveness in treating Celiac Disease.

On Jan 23rd, 2009, the Baltimore Business Journal reported that Alba has announced layoffs due to the current economical crisis in the US. Twenty-two employees (nearly half of it's workforce) were let go on Jan 7th.

This can't be good news for the company that had high hopes to release the first Celiac Disease drug treatment.

A Good Read: Celiac Vaccine?

Check out this article about a potential Celiac Vaccine being studied in the UK.

A quote from the article...

We have developed a peptide-based therapeutic vaccine based on the dominant problem T-cell epitopes of gluten. The "vaccine" has the potential to treat at about 80% of people with the disease and having the appropriate genetic background. In common with traditional desensitization therapy for allergy, peptide-based therapeutic vaccines administered in many small doses over a course of injections could induce immune tolerance not only to the selected gluten fragments but also suppress the toxicity of other toxic gluten components," explained Dr Anderson. Dr Anderson is joint founder and CEO of Nexpep Pty Ltd, the company developing the coeliac vaccine in Australia. Dr Anderson said, "Nexpep is currently raising capital for a clinical trial program for a peptide-based therapeutic vaccine and intends to commence a Phase 1 clinical trial in the first half of 2009.

Interesting...

Celiac Disease Drug Therapy: ChemoCentryx, Inc.

I wrote last week about 2 potential Celiac Disease drug therapies that are currently under development by Alvine Pharmaceuticals (ALV003) and Alba Therapeutics (Larazotide acetate). Then I saw another press release from ChemoCentryx which is currently studying Traficet-EN for use in Celiac patients. The drug is currently in Phase II Clinical Trials.

The primary objective of this study is to evaluate the effect of Traficet-EN compared to placebo on the small intestinal mucosal morphology of biopsy specimens taken from subjects with celiac disease on a long-standing gluten-free diet, before and after gluten exposure. Secondary objectives of this study include evaluation of Traficet-EN compared to placebo on small intestinal mucosal inflammation, gluten-induced celiac-type serology, symptom scores, malabsorption parameters and safety and tolerability profiles. If our Phase II clinical trial in celiac disease is successful, we plan to launch further trials to obtain an indication for Traficet-EN in treatment of patients with this disease.

This drug was originally created for use in people with Crohn's Disease but is now being studied for Celiac Disease. It focuses on Chemokines and other chemoattractants which direct inflammatory responses by serving to precisely coordinate the movement of cells of the immune system. Inappropriate activity of the chemokine network is at the core of numerous autoimmune and inflammatory conditions like Celiac Disease. By selectively blocking a given chemokine and chemokine receptor combination, and largely leaving other chemokine-chemokine receptors interactions unimpeded, even aggressive forms of chronic inflammation and autoimmunity can potentially be brought under control in a safe and effective manner.

This page has good information on Chemokines.

Click here for the ChemoCentryx Web site.

Contact:
Corporate Headquarters
ChemoCentryx, Inc.
850 Maude Avenue
Mountain View, CA 94043
voice +1 (650) 210-2900

Celiac Disease Drug Therapy: Alvine Pharmaceuticals

As I wrote yesterday, this is the second part of the drug therapies that are currently under development for CD. The first therapy, Larazotide acetate, being developed by Alba Therapeutics is a pill that is being studying to reduce the "leaky gut" condition of CD. Any ingested Gluten will not be allowed to permeate in the intestines and thus the auto-immune reaction will be suppressed. That pill and the one I am writing about today are to be taken in conjunction with a Gluten Free diet and NOT meant to replace being GF.

Alvine Pharmaceuticals is based in San Carlos, California and is currently in Phase I trial of a drug called ALV003. The drug is meant to be taken with your meals (like lactose intolerant medications) to degrade the gluten to a point where the auto-immune response would be non-existant. This would be extremely helpful for those of us who get accidentially "Glutened" while eating out. Read more about their press release announced 10-29-08 below...

Alvine Pharmaceuticals Inc., of San Carlos, Calif., reported proof-of-concept in a Phase I trial of ALV003, an oral protease therapy in development to detoxify gluten, for use by patients with celiac disease. The trial results confirmed that single doses of up to 1,800 mg of ALV003 were safe and tolerable in healthy volunteers. Doses at the 300 mg level achieved up to a 96 percent reduction of gluten in a meal in the stomach at 30 minutes, and were well tolerated by patients with celiac disease. In addition, doses as low as 100 mg were shown to be biologically active in degrading gluten in the stomach. Based on these results, Alvine plans to initiate Phase 1/2a multidose trials in early 2009.

The trial for this drug began in February 2008 and is currently recruiting participants.

About ALV003
ALV003 is an orally administered combination of two proteases engineered to degrade gluten into non-immunotoxic fragments, by targeting the glutamine and proline residues that are common in gluten. ALV003 consists of a glutamine specific cysteine protease (EP-B2) and a proline specific prolyl endopeptidase (PEP).

Click here for the Alvine Pharmaceuticals Web site.
Contact:
Kirk Essenmacher M.D., M.B.A.
Phone: (650) 596-2434
Vice President of Marketing, Corporate and Strategic Development
or
Revati Shreeniwas M.D.
Phone (650) 596-2433 (Clinical Contact)
Vice President, Clinical Research and Development

Celiac Disease Drug Therapy: Alba Therapeutics

I have been doing some reading on recent research announced about Drug Therapy for Celiac Disease sufferers. There are currently 2 drugs under research and clinical trials at the moment. The first is called Larazotide acetate (formerly referred to as AT1001) and is being developed and studied by Alba Therapeutics in Baltimore, Maryland. I will write about the other in a separate post.

This drug would be used in conjunction with a Gluten Free diet to reduce Celiac Disease's "leaky gut" condition. "Leaky Gut" can also be described as intestinal permeability and is not only the cause of Celiac Disease issues but is also made worse by Celiac Disease. Two Clinical Trials have been conducted on this drug with promising results.

Trial 1 began in June 2007 and is still currently ongoing although not recruiting participants.
This study will look at 3 doses of AT1001 for efficacy and safety for the treatment of Celiac Disease. The primary efficacy outcome is to evaluate the efficacy of multiple dose levels of AT-1001 in preventing intestinal permeability changes induced by a 6- week gluten challenge.

Trial 2 began in February 2008 and is currently still recruiting participants.
The purpose of this research study is to look at how effective and safe larazotide acetate (AT-1001) is when it is given to subjects who have active Celiac Disease. A "leaky gut" is often found in Celiac Disease patients, and it is thought that the leakiness contributes to the disease, possibly by letting more gluten enter the body. Larazotide acetate (AT-100l) is an experimental drug that may possibly reduce gut leakiness and is being investigated to see if it can help people with Celiac disease along with a gluten free diet.

In an October 8th, 2008 press release, Alba presented new data from the two clinical trials listed above at the 2008 American College of Gastroenterology Annual Scientific Meeting. They confirmed that their studies have shown that Larazotide Acetate Prevented Immunologic Changes Induced by Gluten in Patients with Celiac Disease. Larazotide acetate has the potential to become the first approved medicine to treat Celiac Disease and has been granted “Fast Track” designation from the U.S. Food and Drug Administration (FDA) for this indication. Both trials are in Phase II and expected to move to Phase III soon.

For more information about Alba Therapeutics and Larazotide acetate, see the following...
Click here for Alba's Web site
Phone: 410-878-9850
E-mail: info@albatherapeutics.com