With Celiac Deal, Celimmune Aims to Give New Life to Old Amgen Drug

Xconomy New York — 

Sometimes a drug gathering dust at a big company finds new life at a startup. And sometimes the two companies cut a deal, in case that startup is on to something. That’s exactly what biotech giant Amgen is doing with brand-new startup Celimmune, in a deal announced today.

Celimmune, based in Lebanon, NJ, and Bethesda, MD, and formed just last week, has grabbed rights to an old drug from Thousand Oaks, CA-based Amgen (NASDAQ: AMGN), known as AMG-714. It was shelved after tests in rheumatoid arthritis and psoriasis fell short of expectations back in 2008.

Celimmune seeks to resurrect it as a treatment for certain forms of celiac disease. If it’s on to something, Amgen can buy it back.

Terms of the deal aren’t being disclosed, but Celimmune executive chairman Ashleigh Palmer says Amgen is funding the development work in return for the exclusive right to reacquire the drug later.

This is Celimmune’s modus operandi. The company was formed by CEO and chief medical officer Francisco Leon (pictured above), a celiac expert and former Janssen Pharmaceuticals immunology executive; and Palmer, the president of biopharma advisory firm Creative BioVentures.

Celimmune isn’t disclosing the identity of its investors (although Amgen isn’t one), or how much money it has raised.

But the company’s goal is to be a lean, external development shop for celiac disease and potentially other autoimmune disorders.

It will in-license deprioritized drugs from other companies and run small, quick studies to generate proof-of-concept. If the original owners don’t want to buy the drugs at that point, Celimmune will seek other buyers.

Palmer hopes the Amgen deal will prove the value of its model and enable Celimmune to pitch its services to other companies.

Other startups have succeeded with similar strategies. Atlas Ventures, for instance, formed Arteaus Therapeutics in 2011 to take a molecule from Eli Lilly for migraines, generate proof of concept, and sell it back. Stromedix similarly took a shelved fibrosis drug from Biogen Idec (NASDAQ: BIIB), advanced it, and then flipped it back to its old owner. The difference with Celimmune is it aims to stay independent and do these types of deals more than once.

Celimmune’s first stab at this strategy is AMG-714. It’s a monoclonal antibody that targets IL-15, a cytokine thought to play a role in autoimmune disorders. An earlier version of it, HuMax-IL15, was originally developed by Denmark-based Genmab then licensed to Amgen in 2003. Amgen reformulated the drug into AMG-714 in 2006 and tested it in rheumatoid arthritis.

Leon says Amgen’s results showed a 20 percent improvement of symptoms in 60 percent of the patients in a 280-patient study. Those data would be competitive with the current standard-of-care drugs, known as TNF inhibitors, that crowd the rheumatoid arthritis landscape.

But back then, Amgen knew those numbers wouldn’t stand up against the next generation of drugs in development. So after a small “Hail Mary” trial in psoriasis that didn’t work out, Amgen stopped developing AMG-714 in 2008. It’s been shelved ever since.

Celimmune believes it can give AMG-714 new life as a treatment for celiac disease. The condition occurs when the body misidentifies fragments of gluten—a protein found in barley, wheat, and rye—as a foreign invader and mounts an attack. These attacks damage the gut, impair the ability to absorb nutrients, and cause abdominal pain, vomiting, and diarrhea. If left unchecked, celiac disease can cause more serious complications like anemia or osteoporosis. It affects about 1 percent of the U.S. population.

There are no FDA-approved treatments. The only remedy is to avoid gluten. Some companies, like San Carlos, CA-based Alvine Pharmaceuticals and Alba Therapeutics, of Baltimore, MD, are developing treatments to be taken in combination with a gluten-free diet. Cambridge, MA-based ImmusanT is advancing a vaccine that, if proven effective in clinical studies, would enable patients to eat gluten without getting sick.

Celimmune isn’t aiming to treat the entire celiac population with AMG-714; just the roughly 50 percent of diagnosed celiac patients, Leon says, who aren’t responding to gluten free diets. Within that group is a much smaller group with a rare condition called “refractory” celiac disease. This is when symptoms persist despite several months of gluten-free eating due to permanently damaged villi—the tiny protrusions sticking out of the intestinal walls that help the body absorb nutrients and move food through the gut.

Refractory celiac affects about 1 percent of celiac sufferers. It’s a form of lymphoma; a type of white blood cell (intraepithelial lymphocytes) keeps multiplying and invading the gut, even without being triggered by gluten.

Leon says AMG-714 might help by blocking IL-15, which is thought to play a key role in the development of the invading white blood cells that damage the intestinal lining. Cutting off IL-15 could destroy those cells and give refractory patients relief. That’s the theory, anyway.

“It’s far from proven,” Palmer cautions.

Palmer and Leon will test that hypothesis with Amgen’s drug. They won’t say when those trials will begin, but to run them Celimmune will form partnerships with some of the world’s major celiac disease centers in New York, Paris, and Amsterdam. Patients will take what’s known as a ‘gluten challenge’ to their system: eating gluten to see what the effects are.

If the patients ingest gluten for a few weeks, take AMG-714, and don’t show celiac symptoms, Celimmune believes that will be a signal the drug is having a positive effect. And with that signal, perhaps Amgen will once again take a shot on a program it shelved many years ago.