Move Over Statins, Sartans, Tinibs; It's Time for Cholic Chemistry

Move over Statins, Sartans, Tinibs; it’s time for “Cholic chemistry”

Recently, FDA approved therapies to reduce double chin (yes it’s possible!) and treat patients with bile acid synthesis disorders through compounds derived from bile acids.

Bile, that bitter, alkaline liquid secreted by livers, has formed the base for producing Cholic Acid and Deoxycholic Acid, which led to the recent approvals:

Kythera Pharamceutical’s ATX-101 (Deoxycholic Acid, DCA) injection for submental fat (double chin) and

AskLepion’s CHOLBAM (Cholic Acid, CA) capsules for the treatment of bile acid synthesis due to single enzyme defects.

Kythera modified the age old French practice of mesotherapy (non-surgical cosmetic medicine treatment), where the combination of deoxycholic acid and phosphatidylcholine is used to reduce fat by demonstrating that dexoxycholic acid alone can get the job done. While the combination is used in mesotherapy, studies have demonstrated that phosphatidylcholine has a limited role in fat reduction, so Kythera’s science doesn’t exactly qualify as revolutionary.

Kythera’s ability to navigate the complex regulatory landscape (they won a unanimous approval 17-0 from the FDA Advisory Committee) seems well worth it as ATX-101’s sales potential of $300 million makes Kythera a potential takeover target for larger pharmaceutical companies.

The two approvals is a resurgence for bile acid salts since Cholic Acid has been used for decades to produce Ursodiol (Ursodeoxycholic Acid), a treatment for gallstones with pharma majors like Sanofi and Roche having manufactured the active ingredient in the past.

There is more to come in the story of bile acid salts as they form the base for breakthrough treatments for Nonalcoholic Steatohepatitis (NASH) - a serious chronic liver disease which can lead to cirrhosis, eventual liver failure and death.

There are currently no drugs approved for the treatment of NASH and recent epidemiological studies estimate that more than 10% of the U.S. adult population has NASH, with more than 60% of patients (potentially more than 14 million in total) have liver fibrosis, or cirrhosis, due to progression of the disease.

Intercept Pharmaceuticals, with its modified bile acid Obeticholic Acid (OCA), has been assigned Breakthrough Therapy status by the FDA. The effectiveness of Obeticholic Acid and size of the patient population explains why Intercept has been a darling of the stock market through 2014, especially as the product is expected to gain approval later this year. Expected sales estimates of the potential blockbuster range between $2-4 billion and explain the $6 billion stock market valuation.

Further upstream, Israel based, Galmed Pharmaceuticals has a compound in Phase IIb clinical trials which couples Cholic Acid with the commonly available fatty acid, Arachidic Acid for the same indication as Obeticholic Acid. After Gilead’s recent purchase of Phenex Pharmaceuticals for $470 million to get access to their NASH compound, the potential upside of NASH compounds is HUGE!

We already have established molecule classes like Statins, Sartans & Tinibs, maybe it’s time to get work started on "Cholic chemistry”!

Search our database for Cholic Acid and Deoxycholic Acid to find suppliers, pricing information and a lot more…

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