Retrophin buys up Orphan Technologies, spending $90M on rare disease drug

Retrophin has snapped up Orphan Technologies for a $90 million upfront payment, gaining access to the biotech's rare metabolic disorder drug OT-58.

The enzyme replacement therapy, currently in phase 1/2, is being tested against classical homocystinuria (HCU), which can increase the risk of stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delays.

The deal, made up initially of the $90 million upfront, also comes with $427 million in biobucks, plus royalties, should the med gain approval. Retrophin is also tied in to make an extra payout should it nab a pediatric rare disease voucher.

Retrophin has spent years trying to distance itself from the infamy of its former CEO Martin Shkreli, who sued the company from prison last year. They pair eventually settled over claims the company illegally ousted him, and the company now wants to be seen as an R&D-focused biotech.

Its late-stage pipeline currently consists of its work on sparsentan, a small molecule focusing on two diseases: focal segmental glomerulosclerosis, a serious kidney disorder that often leads to end-stage renal disease, and immunoglobulin A nephropathy, or Berger’s disease, which also can lead to end-stage renal disease.

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“Many people with HCU face a continuous risk of developing life-threatening complications because current treatment options are largely ineffective in managing homocysteine levels,” said Eric Dube, Ph.D., president and CEO of Retrophin.

“OT-58 has demonstrated an ability to meaningfully reduce homocysteine levels in preclinical models and has the potential to ultimately become the first disease modifying therapy for HCU. This promising, novel development candidate fits directly with our mission to identify, develop and deliver life-changing therapies to people living with rare disease and brings exciting growth potential to Retrophin.”

As part of the deal, the pair has also penned a spinout pact for Orphan’s preclinical asset, OT-15, which is targeting mitochondrial neurogastrointestinal encephalopathy syndrome, a rare autosomal recessive mitochondrial disease.