Date: 2015-01-12
Type of information: Product acquisition
Compound: Cholbam® (cholic acid) capsules
Company: Retrophin (USA - CA) Asklepion Pharmaceuticals (USA - MD)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Type agreement: product acquisition
Action mechanism:
Disease: patients with bile acid synthesis disorders due to single enzyme defects patients with peroxisomal disorders (including Zellweger spectrum disorders)
Details: * On January 12, 2015, Retrophin and Asklepion Pharmaceuticals, a privately held rare disease pharmaceutical development company, announced the signing of a definitive agreement under which Retrophin has acquired the exclusive right to purchase from Asklepion, all worldwide rights, titles, and ownership of cholic acid for the treatment of bile acid synthesis defects, if approved by the FDA. Cholic acid is being considered for approval in a late-stage review by the FDA and is currently approved in Europe for the treatment of inborn errors in primary bile acid synthesis. It restores endogenous bile acid pool levels while inhibiting abnormal bile acid synthesis.
Financial terms: Under the terms of the agreement, Retrophin will pay Asklepion an upfront payment of $5 million and up to $73 million in milestones based on approval and net product sales, plus tiered royalties on future net sales of cholic acid. Retrophin has secured a line of credit from current lenders to cover necessary payments.
Latest news: * On March 18, 2015, As a result of the FDA approval, Retrophin will exercise its right to purchase from Asklepion Pharmaceuticals, all worldwide rights, titles, and ownership of Cholbam® and related assets. Under the terms of the agreement announced on January 12, 2015, Retrophin will pay Asklepion a one-time cash payment of $27 million, in addition to approximately 661,278 shares of Retrophin common stock (initially valued at $9 million at the time of the agreement), which assumes Cholbam received an approval for a CTX indication. Asklepion will also be eligible to receive up to $37 million in cumulative sales milestones, as well as tiered royalties based on future net sales of Cholbam. The FDA also granted Asklepion a Rare Pediatric Disease Priority Review Voucher (\"Pediatric PRV\"), a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. The Pediatric PRV will be transferred to Retrophin under the original terms of the agreement with Asklepion. Cholbam will have seven years market exclusivity in the United States conferred by its designation as an orphan drug. Retrophin expects to close the acquisition and be able to begin distributing therapy in as few as two to four weeks, subject to the satisfaction of customary closing conditions.
