Retrophin, Inc. (NASDAQ:RTRX) announced new data from a physician-initiated study on RE-024 for the treatment of pantothenate kinase-associated neurodegeneration (PKAN).
Data revealed that patients (n=2) treated with RE-024 demonstrated clinically meaningful improvements, followed by stabilization of disease progression over 47 weeks of treatment. The study also showed that patients experienced a gradual improvement in gait and could walk unassisted for short distances within eight weeks of treatment initiation. In addition, RE-024 was found to be safe and well tolerated. Results were presented at the Congress of Parkinson's Disease and Movement Disorders.
Retrophin is planning to initiate an efficacy study on RE-024 in the second half of 2016.
We note that RE-024 enjoys Orphan Drug status in both the U.S. and the EU for the treatment of PKAN. The candidate also has Fast Track status for PKAN in the U.S.
PKAN, a rare and life-threatening genetic condition, is estimated to affect up to 5,000 individuals around the world. With no treatments approved currently for the disease, the company has significant potential in this area.
Meanwhile, Retrophin is progressing with other candidates in its pipeline. Currently, the company is conducting a phase II study (DUET) on sparsentan for the treatment of patients with focal segmental glomerulosclerosis (FSGS). Top-line data from the DUET study are expected in the third quarter of 2016.
Retrophin currently sports a Zacks Rank #1 (Strong Buy). Other favorably placed stocks in the health care sector, sharing an equal rank with Retrophin, include Pfizer Inc. (NYSE:PFE) , ANI Pharmaceuticals, Inc. (NASDAQ:ANIP) and Bio Blast Pharma Ltd. (NASDAQ:ORPN) .
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Retrophin (RTRX) RE-024 Positive In Physician-Initiated Study
Published 06/24/2016, 06:54 AM
Updated 07/09/2023, 06:31 AM
Retrophin (RTRX) RE-024 Positive In Physician-Initiated Study
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