Item 8.01 Other Events.
On June 27, 2014, Retrophin, Inc. (the “Company”) provided preliminary single patient data from the first Pantothenate kinase-associated neurodegeneration (“PKAN”) patient dosed with RE-024 under an investigator-initiated research protocol. The patient received the first dose of RE-024 on May 21, 2014. On June 5, 2014, after two weeks of treatment, the patient developed a modest elevation of liver enzymes (AST and ALT) to 1-2 times the upper limit of normal and dosing of RE-024 was temporarily discontinued. At the next follow-up visit on June 11, the patient’s liver enzyme levels had trended downward and RE-024 was restarted at a reduced dose. The patient has maintained normal liver enzyme levels and remains on RE-024 as of June 25, 2014. Results for several clinical outcome measures are listed below. The Company does not believe data on a single patient can be interpreted in a meaningful fashion and is releasing this data to accommodate investor requests. Further, the results are inconclusive at this time with regard to RE-024’s ability to treat patients that suffer from PKAN. Potential investors should not place undue reliance on this single patient data.
| | 5/21/2014 | 5/28/2014 | 6/4/2014 | 6/11/2014 | 6/18/2014 | 6/25/12014 |
UPDRS1 | | | | | | |
| Part 1 | 8/10 | 3/10 | 3/10 | 3/10 | 4/10 | 3/10 |
| Part 2 | 36/52 | 26/52 | 26/52 | 27/52 | 26/52 | 26/52 |
| Part 3 | 30/56 | 27/56 | 27/56 | 33/56 | 28/56 | 27/56 |
| | | | | | | |
| EQ-5D-3L | 12/15 | 12/15 | 10/15 | 10/15 | 9/15 | 9/15 |
| | | | | | | |
| Barry-Albright Dystonia Scale | 14/24 | 15/24 | 14/24 | 16/24 | 13/24 | 14/24 |
| | | | | | | |
| 25 foot walk test | | | | | | |
| # of steps | 25.5 | 19.5 | 17.0 | 16.0 | 16.5 | 15 |
| Time in seconds | 10.54 | 11.31 | 8.59 | 11.08 | 8.86 | 8.21 |
ABOUT PKAN
Pantothenate kinase-associated neurodegeneration or PKAN is the most common form of neurodegeneration with brain iron accumulation. Classic PKAN is a genetic disorder that is typically diagnosed in the first decade of life. Consequences of PKAN include dystonia, dysarthria, rigidity, retinal degeneration, and severe digestive problems. PKAN is estimated to affect 1 to 3 persons per million. PKAN typically manifests in childhood with a profound, progressive dystonia and is usually lethal. There are currently no viable treatment options for patients with PKAN.
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1 Unified Parkinson’s Disease Rating Scale
ABOUT RE-024
RE-024 is a phosphopantothenate prodrug replacement therapy with the goal of restoring the supply of this operative substrate in PKAN patients. The results discussed in this Report on Form 8-K relate to the first patient treated with RE-024 and are not indicative of future responses by this patient or by other patients.
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
