Arimoclomol formulation

An extended-release pharmaceutical formulation in an oral dosage form comprising:

• an active pharmaceutical ingredient selected from the group consisting of:
• N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride, a stereoisomer of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride, an acid addition salt of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride and an addition salt of a stereoisomer of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride;
• and
• a release-controlling excipient that provides for extended release of said active pharmaceutical ingredient,
• wherein said formulation reduces inhibition of Organic Cation Transporter 2 (OCT2), as compared to an equivalent amount of the active pharmaceutical ingredient administered by an immediate release oral dosage form and/or by bolus intravenous (IV) injection, and wherein the active pharmaceutical ingredient is present in one dosage form or formulation unit in a total amount of 5-1000 mg per dosage,
• wherein the extended-release pharmaceutical formulation provides for a Cmax of the active pharmaceutical ingredient of less than 15 μM, thereby reducing inhibition of OCT2 in the individual, and
• wherein 85% of the active pharmaceutical ingredient is released from the formulation after at least 6 hours.