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Ligand/Retrophin Rare Disease Drug Positive in Phase II

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Ligand Pharmaceuticals Incorporated (LGND - Free Report) said that its partner Retrophin, Inc. announced positive top-line results from a phase II study. The DUET study is evaluating their pipeline candidate sparsentan for the treatment of a rare kidney disorder, focal segmental glomerulosclerosis (FSGS).

While Ligand Pharma’s shares increased almost 6% on Wednesday, Retrophin’s soared almost 28%.

There are no approved pharmacologic treatment options for FSGS at present and it is therefore a leading cause of end-stage renal disease.

The international, randomized, double-blind phase II DUET study achieved statistical significance in the primary efficacy endpoint of reduction of proteinuria as compared to irbesartan.

Irbesartan is part of a class of drugs used to manage FSGS in the absence of an approved pharmacologic treatment. Proteinuria is a condition where protein is found in the urine due to a breakdown of the normal filtration mechanism in the kidney. Therefore, reduction of proteinuria is widely recognized to be beneficial in the treatment of FSGS.

The overall sparsentan treatment group in the study demonstrated a greater than two-fold reduction of proteinuria compared to irbesartan after eight weeks of treatment. Preliminary safety findings showed that sparsentan was generally safe and well-tolerated in the study.

The results suggest that sparsentan could be a significant advancement in the treatment of FSGS. According to information provided by the companies in their press releases, FSGS is estimated to affect up to 40,000 patients in the U.S. We note that sparsentan enjoys orphan drug status from the FDA.

Retrophin plans to present detailed results from the study at an upcoming medical meeting or in a peer-reviewed publication.

While Ligand Pharma carries a Zacks Rank #1 (Strong Buy), Retrophin has a Zacks Rank #3 (Hold). Some other stocks worth considering in the healthcare sector include Anika Therapeutics Inc. (ANIK - Free Report) and Geron Corporation (GERN - Free Report) which enjoy the same rank as Ligand Pharma

You can see the complete list of today’s Zacks #1 Rank  stocks here.

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