BioCentury | Aug 22, 2019
Clinical News

After neurodegeneration miss, Retrophin looks to rebound with renal program

With plans to discontinue fosmetpantotenate after its Phase III miss, Retrophin President and CEO Eric Dube believes the $425 million with which it ended last quarter is enough to bring its remaining clinical candidate to...
BioCentury | Jun 19, 2019
Financial News

Coenzyme A company Comet debuts with $28.5M series A

Led by former Syntimmune CEO David de Graaf, Comet launched Wednesday with $28.5 million in series A funding to develop precursors of coenzyme A to treat metabolic, immune and neurological disorders that involve dysregulated coenzyme...
BioCentury | Dec 3, 2018
Company News

NIH may give ElevateBio exclusive CAR T license

NIH's National Cancer Institute is considering granting ElevateBio LLC (Cambridge, Mass.) an exclusive license to develop an undisclosed CAR T therapy targeting FMS-like tyrosine kinase 3 (FLT3; CD135). In a Federal Registry notice , NIH said...
BioCentury | Nov 6, 2018
Distillery Therapeutics

Neurology

INDICATION: Neurology In vitro , cell culture and mouse studies identified a PANK3 activator that could help treat pantothenate kinase-associated neurodegeneration (PKAN), which is caused by PANK2 deficiency and leads to low Coenzyme A levels in...
BioCentury | Jun 13, 2018
Company News

BridgeBio launches rare genetic disorder subsidiary

BridgeBio Pharma LLC (Palo Alto, Calif.) launched another subsidiary this week, forming CoA Therapeutics Inc. to develop small molecules which increase coenzyme A levels to treat rare genetic disorders. CoA CEO Shafique Virani told BioCentury...
BioCentury | Sep 20, 2017
Distillery Therapeutics

Neurology

INDICATION: Neurology Cell culture and mouse studies suggest the acetyl CoA precursor acetyl-phosphopantetheine could help treat pantothenate kinase-associated neurodegeneration (PKAN), which is caused by PANK2 deficiency. In HEK cells with chemical-induced PANK2 deficiency and a...
BioCentury | Sep 1, 2017
Clinical News

Retrophin starts Phase III of RE-024 to treat PKAN

Retrophin Inc. (NASDAQ:RTRX) began the international Phase III FORT trial of fosmetpantotenate (RE-024) to treat pantothenate kinase-associated neurodegeneration (PKAN). The double-blind, placebo-controlled trial will evaluate the primary endpoint of change from baseline to week 24...
BioCentury | Mar 28, 2016
Clinical News

RE-024: Phase I data

A double-blind, placebo-controlled Phase I trial in 40 healthy volunteers showed that single doses of 75, 225, 450, 900 and 1,800 mg oral RE-024 were well tolerated with no serious adverse events reported. Separately, data...
BioCentury | Jun 15, 2015
Clinical News

RE-024 regulatory update

FDA granted Fast Track designation to RE-024 from Retrophin to treat pantothenate kinase-associated neurodegeneration (PKAN). The product is in Phase I testing and has Orphan Drug designation. The phosphopantothenate molecule restores disruption in coenzyme A...
BioCentury | May 18, 2015
Clinical News

RE-024 regulatory update

FDA granted Orphan Drug designation to RE-024 from Retrophin to treat pantothenate kinase-associated neurodegeneration (PKAN). The product is in Phase I testing. RE-024 is a phosphopantothenate molecule that restores disruption in coenzyme A biosynthesis caused...
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