# US 2014/0194466 A1 · Perhexiline for use in the treatment of hypertrophic cardiomyopathy (hcm) > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 2014/0194466 A1 - Document ID: US20140194466A1 - Status: Published application - Kind: A1 - Application number: 14/186,554 - Filed: 2014-02-21 - Published: 2014-07-10 - Earliest claimed: 2009-05-13 - Assignees: The University of Birmingham - Applicants: The University of Birmingham - Inventors: Houman ASHRAFIAN; Michael Paul FRENNEAUX - Web page: https://patentgrep.com/patent/US20140194466A1 ## CPC - A61K 31/4458: only substituted in position 2, e.g. methylphenidate - A61K 31/366: having six-membered rings, e.g. delta-lactones - A61K 31/445: Non condensed piperidines, e.g. piperocaine - A61P 9/00: Drugs for disorders of the cardiovascular system - A61P 9/02: Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives - A61P 9/04: Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure - A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS ## Abstract The invention relates to perhexiline, or a pharmaceutically acceptable salt or enantiomer thereof, for use in the treatment of hypertrophic cardiomyopathy, as well as to a method of treating HCM, which comprises administering to an animal in need thereof an effective amount of perhexiline, or a pharmaceutically acceptable salt or enantiomer thereof, to treat said HCM. The invention further relates to a treatment programme for treating HCM, which involves the co-use or co-administration of perhexiline with one or more other compounds that are advantageous in treating HCM or the symptoms thereof. ## Claims ### Claim 1 A method for treating hypertrophic cardiomyopathy in a mammal in need thereof, comprising: diagnosing the mammal as having hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof; and administering to said mammal a therapeutically-effective amount of (−)-perhexiline. ### Claim 2 (depends on 1) The method of claim 1, wherein the therapeutically-effective amount of (−)-perhexiline is sufficient to reduce or ameliorate the hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof in the mammal. ### Claim 3 (depends on 1) The method of claim 1, wherein the (−)-perhexiline is in the form of a pharmaceutically acceptable salt. ### Claim 4 (depends on 2) The method of claim 2, wherein the (−)-perhexiline is in the form of a maleate salt. ### Claim 5 (depends on 1) The method of claim 1, wherein the mammal is a human. ### Claim 6 (depends on 1) The method of claim 1, further comprising co-administering to said mammal at least one therapeutic compound. ### Claim 7 (depends on 1) The method of claim 1, further comprising co-administering to said mammal at least one therapeutic compound advantageous in treating hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof. ### Claim 8 (depends on 6) The method of claim 6, wherein the therapeutic compound is selected from a member of the group consisting of Alpha Blockers, Beta Blockers, Calcium Channel Blockers, Diuretics, Ace (Angiotensin-Converting Enzyme) Inhibitors, Arb (Angiotensin II Receptor Blockers), Spironolactone, Nitrate, Warfarin, Verapamil, Insulin, Amiodarone, Lisinopril, Ramipril, Perindopril, Enalapril, Trandolapril, At2 Receptor Blockers, Losartan, Valsartan, Irbersartan, Carvedilol, Bisoprolol, Metoprolol, Atenolol, Aspirin, Clopidogrel, Oral Hypoglycaemics, Disopyramide, and Statins. ### Claim 9 A method for treating a mammal having at least one of a symptomatic component, feature or condition associated with hypertrophic cardiomyopathy, comprising: diagnosing the mammal as having at least one of a symptomatic component, feature or condition associated with hypertrophic cardiomyopathy; and administering to said mammal a therapeutically-effective amount of (−)-perhexiline. ### Claim 10 (depends on 9) The method of claim 9, wherein the symptomatic component, feature or condition is member of the group consisting of dyspnoea (shortness of breath), chest pain, fatigue, palpitation and syncope. ### Claim 11 (depends on 9) The method of claim 9, wherein the mammal is further diagnosed as having a member of the group consisting of reduced left ventricular ejection fraction (LVEF), reduced E:EA ratio, abnormally rapid skeletal muscle phosphocreatine depletion with delayed recovery, reduced systolic velocity (PSV), diminished exercise capacity or tolerance, diminished peak oxygen consumption (VO2max) during exercise, diastolic dysfunction at rest and during exercise as measured by Time to Peak LV Filling (nTTPF), no significant LVOT obstruction at rest (gradient<30 mmHg), and impaired myocardial energetic state (PCr/γATP ratio). ### Claim 12 (depends on 9) The method of claim 9, wherein the extent of hypertrophic cardiomyopathy in the mammal is diagnosed in accordance with a New York Heart Association (NYHA) classification. ### Claim 13 (depends on 9) The method of claim 9, further comprising: determining a NYHA classification score (breathlessness) of the mammal before and after administration of perhexline, wherein a decreased NYHA score after administration of perhexline indicates a reduction in the extent of hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof in the mammal. ### Claim 14 (depends on 13) The method of claim 13, wherein the NYHA classification score of the mammal after administration of (−)-perhexiline decreases from Class III to Class II. ### Claim 15 (depends on 9) The method of claim 9, wherein the extent of hypertrophic cardiomyopathy in the mammal is diagnosed in accordance with a Minnesota Living with Heart Failure Questionnaire (MLHFQ) score. ### Claim 16 (depends on 15) The method of claim 15, further comprising determining a MLHFQ (quality of life) score of the mammal before and after administration of (−)-perhexiline, wherein a decreased MLHFQ score after administration of (−)-perhexiline indicates a reduction in the extent of hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof in the mammal. ### Claim 17 (depends on 9) The method of claim 9, further comprising determining peak oxygen consumption (VO2max) in the mammal during exercise wherein an increase in peak oxygen consumption (VO2max) in the mammal after administration of (−)-perhexiline indicates a reduction in the extent of hypertrophic cardiomyopathy or at least one of a symptomatic component, feature or condition thereof in the animal. ### Claim 18 (depends on 9) The method of claim 9, wherein (−)-perhexiline is administered in an amount of 300 mg or less per day. ### Claim 19 (depends on 9) The method of claim 9, wherein (−)-perhexiline is administered in an amount of 100 mg or less per day. ### Claim 20 (depends on 9) The method of claim 9, wherein (−)-perhexiline is administered in an amount of 100 mg to 300 mg per day.