MYOSIN THERAPEUTICS INC. - Florida Company Profile

2025-03-25

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Department of Health and Human Services

PHASE 0/1B CLINICAL TRIAL OF NOVEL THERAPEUTIC AGENT MT-110 FOR THE REDUCTION OF METHAMPHETAMINE USE - PART 2 - PROJECT SUMMARY AS OF 2023, CLOSE TO 2 MILLION INDIVIDUALS IN THE UNITED STATES ARE IDENTIFIED AS HAVING METHAMPHETAMINE USE DISORDER (MUD), WITH METHAMPHETAMINE USE RISING SIGNIFICANTLY. RECENT ESTIMATES SUGGEST THAT THIS FIGURE IS UNDERREPORTED BY ABOUT 225%, BRINGING THE 2023 TOTAL CLOSER TO OVER 4 MILLION PEOPLE WITH MUD, AND LIKELY EVEN HIGHER IN 2024. MUD IS A PERSISTENT CONDITION FOR WHICH THERE ARE NO FDA-APPROVED MEDICATIONS AT PRESENT. THE CURRENT TREATMENTS AVAILABLE ARE BEHAVIORAL MODIFICATION THERAPIES, WHICH SHOW LIMITED SUCCESS, AS INDICATED BY HIGH RELAPSE RATES (60-90%). THIS UNDERSCORES THE NEED FOR AN ADDITIONAL PHARMACOTHERAPY THAT TARGETS RELAPSE TRIGGERS TO HELP MAINTAIN ABSTINENCE. COMPREHENSIVE PRIOR STUDIES BY OUR GROUP AND OTHERS HAVE SHOWN THAT RELAPSE INDUCED BY DRUG-USE REMINDERS (OR “TRIGGERS”) EXERTS A STRONG MOTIVATIONAL INFLUENCE, POSING A LIFELONG RELAPSE RISK REGARDLESS OF THE DURATION OF ABSTINENCE. MYOSIN THERAPEUTICS IS DEVELOPING MT-110, A DRUG MARKED BY THE NOVEL THERAPEUTIC TARGET OF THE MOLECULAR MOTOR NONMUSCLE MYOSIN II (NMII). PRECLINICAL RESEARCH HAS DEMONSTRATED THAT A SINGLE ACUTE INHIBITION OF NMII ACTIVITY CAN PRODUCE A HIGHLY SPECIFIC, LONG-LASTING EFFECT BY RESTORING THE BRAIN’S STRUCTURAL PLASTICITY TO A PRE-METHAMPHETAMINE STATE. CONSEQUENTLY, A SINGLE INTRAVENOUS DOSE OF MT-110 RESULTS IN A LASTING REDUCTION IN METHAMPHETAMINE-SEEKING BEHAVIOR IN ANIMAL MODELS. IND-ENABLING STUDIES FOR MT-110 ARE COMPLETE, AND PHASE 1B CLINICAL TRIALS ARE SET TO COMMENCE IN THE FIRST QUARTER OF 2025. MT-110 IS BEING DEVELOPED AS A ONE-TIME ADMINISTRATION MEDICATION TO SUPPORT THE REDUCTION OF MUD. THE PRIMARY GOAL OF THIS DIRECT TO PHASE II SBIR APPLICATION IS TO SUPPORT A FIRST-IN-HUMAN DOUBLE-BLIND, PLACEBO-CONTROLLED SINGLE ASCENDING DOSE (SAD) PHASE IB DOSE ESCALATION TRIAL OF MT-110 IN INDIVIDUALS THAT CONSUME METH. THIS PROPOSED 1B TRIAL OF MT-110 WILL RECRUIT UP TO 44 PATIENTS TO TEST 5 DOSE LEVELS AND DETERMINE CNS DRUG CONCENTRATIONS VIA CSF COLLECTION IN AN ADDITIONAL COHORT FOLLOWING ADMINISTRATION OF AN OPTIMAL DOSE. THE PRIMARY ENDPOINTS ARE FOCUSED ON DETERMINING THE SAFETY AND TOLERABILITY OF SINGLE ASCENDING DOSES OF MT-110. SECONDARY ENDPOINTS WILL GATHER PHARMACOKINETIC PARAMETERS, BOTH SYSTEMIC AND CNS. AND AN EXPLORATORY ENDPOINT WILL ASSESS POTENTIAL CHANGES FROM BASELINE IN THE CRAVING FOR METH. IN THIS APPLICATION (CURRENT APPLICATION – ‘PART 2’), WE DETAIL THE SPECIFIC ACTIVITIES THAT WILL BE PERFORMED BY THE CLINICAL RESEARCH UNIT (CRU) TO COMPLEMENT THE ACTIVITIES THAT THE SPONSOR (MYOSIN THERAPEUTICS) WILL PERFORM WITH SUPPORT FROM A CRO (DETAILED IN SISTER APPLICATION – ‘PART 1’). THE PART 2 ACTIVITIES DETAILED HERE COVER INTERACTION WITH PATIENTS TO EXECUTE THE TRIAL.

1496586.00

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2023-03-31

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Department of Health and Human Services

MT-125 FOR THE THERAPEUTIC TREATMENT OF GLIOBLASTOMA - PROJECT SUMMARY AN AREA OF SIGNIFICANT UNMET NEED IS THE TREATMENT OF GLIOBLASTOMA (GBM), AN AGGRESSIVE, FAST-GROWING AND LETHAL BRAIN CANCER THAT REPRESENTS 48% OF ALL MALIGNANT BRAIN TUMORS. UNTREATED, GBM IS FATAL WITHIN THREE MONTHS, AND DUE TO ITS HIGH RATE OF RECURRENCE AND INVASIVE NATURE, THE CURRENT STANDARD OF CARE, CONSISTING OF SAFE MAXIMAL TUMOR RESECTION, RADIATION THERAPY AND CHEMOTHERAPY, ONLY EXTENDS SURVIVAL FOLLOWING INITIAL DIAGNOSIS TO ONE YEAR. INVASION AND PROLIFERATION, ALSO KNOWN AS GO AND GROW, ARE DEFINING PHENOTYPES OF GBM, AND GBM CELLS DO ONLY ONE OR THE OTHER. HOWEVER, BLOCKING INVASION STIMULATES PROLIFERATION AND VICE VERSA, IMPLYING THAT AN IDEAL THERAPEUTIC NEEDS TO BLOCK BOTH GO AND GROW SIMULTANEOUSLY. EXTENSIVE GENETIC INTERVENTIONS HAVE SHOWN THAT SIMULTANEOUS DISRUPTION OF TWO NON-MUSCLE MYOSIN II (NMII) MOLECULAR MOTORS (NMIIA AND IIB) MEET THESE CRITERIA. HOWEVER, THE TRANSLATIONAL POTENTIAL OF THIS RESEARCH HAS BEEN LIMITED BY THE LACK OF A CLINICALLY SAFE, CNS-PENETRANT NMII SMALL MOLECULE INHIBITOR. FOLLOWING EXTENSIVE MEDICINAL CHEMISTRY EFFORTS TO OPTIMIZE SELECTIVITY FOR SAFETY AND TOLERABILITY, MT-125 WAS IDENTIFIED. MT-125 IS A WELL-TOLERATED, DUAL SMALL MOLECULE INHIBITOR OF NMIIA AND IIB WITH A HIGH DEGREE OF BRAIN PENETRANCE, A REQUIREMENT FOR AN EFFECTIVE GBM THERAPEUTIC. PRECLINICAL IN VITRO AND IN VIVO STUDIES SHOW THAT MT-125 BLOCKS THE GO AND GROW PHENOTYPES AND EXTENDS SURVIVAL. DUE TO ITS UNIQUE MODE OF ACTION, MT-125 ALSO SYNERGIZES WITH EXISTING FDA-APPROVED TREATMENTS, PRESENTING A PATH TO A POTENTIALLY CURATIVE TREATMENT. THE OVERARCHING GOAL OF THE CURRENT PROPOSAL IS TO READY MT-125 FOR RAPID ENTRY INTO IND-ENABLING STUDIES. THIS WILL BE ACHIEVED THROUGH SEVERAL ACTIVITIES. PHASE I WILL FOCUS ON CONFIRMATION OF PRECLINICAL EFFICACY WITH A CLINICALLY VIABLE ROUTE OF ADMINISTRATION, IN VITRO STUDIES OF SYNERGY BETWEEN MT-125 AND ADDITIONAL EXISTING FDA-APPROVED TREATMENTS, AND IN VITRO SAFETY PROFILING, PRE-FORMULATION STUDIES AND DEMO BATCH SCALE-UP OF MT-125. QUANTITATIVE MILESTONES FOR TRANSITION TO PHASE II ARE DETAILED IN THE APPLICATION. IN PHASE II, IN VIVO EFFICACY TESTING WILL BE PERFORMED ON THE MOST PROMISING SYNERGY COMBINATIONS IDENTIFIED IN PHASE I, AS WELL AS A NON-GLP DOSING SAFETY STUDY, GLP SYNTHESIS, AND FORMULATIONS DEVELOPMENT WITH POLYMORPH SCREENING. THE COMMERCIALIZATION PLAN DETAILS THE GBM MARKET, AS WELL AS MYOSIN THERAPEUTICS’ CLINICAL AND REGULATORY STRATEGY FOR RAPID ADVANCEMENT OF MT-125 TO THE CLINIC.

2666500.00

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2021-09-21

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Department of Health and Human Services

DEVELOPMENT OF A FIRST-IN-CLASS NONMUSCLE MYOSIN II INHIBITOR TO PREVENT SUBSTANCE USE DISORDER RELAPSE - PROJECT SUMMARY IN 2018, OVER 1 MILLION PEOPLE IN THE U.S. QUALIFIED AS HAVING A METHAMPHETAMINE (METH) USE DISORDER AND RATES OF METH USE ARE SURGING, LARGELY IN RESPONSE TO THE OPIOID EPIDEMIC. METH USE DISORDER IS A CHRONIC CONDITION FOR WHICH THERE ARE CURRENTLY NO FDA-APPROVED MEDICATIONS. THE ONLY TREATMENT OPTIONS AVAILABLE ARE BEHAVIORAL MODIFICATION THERAPIES, WHICH HAVE LIMITED EFFICACY, AS EVIDENCED BY THE HIGH RATE OF RELAPSE (60- 90%). THIS ARGUES FOR AN ADJUNCT PHARMACOTHERAPY TARGETING RELAPSE TRIGGERS TO SUPPORT ABSTINENCE. RIGOROUS PRIOR RESEARCH FROM OUR GROUP AND OTHERS HAS ESTABLISHED THAT RELAPSE TRIGGERED BY REMINDERS OF DRUG USE BEAR A POWERFUL MOTIVATIONAL INFLUENCE, SERVING AS A LIFELONG RELAPSE RISK FACTOR, REGARDLESS OF HOW LONG AN INDIVIDUAL ABSTAINS. MYOSIN THERAPEUTICS IS CURRENTLY DEVELOPING MT-110 AS A NON-STIMULANT, FIRST-IN-CLASS DRUG TARGETING THE MOLECULAR MOTOR NONMUSCLE MYOSIN II (NMII). BLEBBISTATIN (BLEBB) WAS THE FIRST NMII ALLOSTERIC INHIBITOR TO BE DISCOVERED AND SUFFERS FROM POOR TOLERABILITY DUE TO EQUIPOTENT INHIBITION OF CARDIAC MUSCLE MYOSIN (CMII). MT- 110 IS A BLEBB ANALOG WITH IMPROVED BRAIN EXPOSURE, POTENCY, AND SELECTIVITY FOR CMII (>100X) WHICH GREATLY IMPROVES ITS THERAPEUTIC INDEX. MT-110 IS CURRENTLY IN IND-ENABLING STUDIES WITH A PLAN TO ENTER A PHASE 1 SAD TOWARDS THE END OF 2021. IT IS BEING DEVELOPED AS A SHORT-TERM ADMINISTRATION MEDICATION TO SUPPORT ABSTINENCE THROUGH A DISRUPTION OF THE MOTIVATION TO SEEK METH. A PRIMARY GOAL OF THIS FAST-TRACK SBIR APPLICATION IS TO ESTABLISH MT-110'S SAFETY PROFILE WITH MULTIPLE ADMINISTRATIONS, AS IT IS EXPECTED TO BENEFIT SUBJECTS REFRACTORY TO SINGLE ADMINISTRATION TREATMENT OR THOSE THAT RELAPSE DUE TO ANOTHER FACTOR. ESTABLISHING MT-110'S EFFICACY IN THE CONTEXT OF POLYDRUG USE AND OTHER SUDS WILL ALSO EXPAND ITS THERAPEUTIC VALUE. IN PHASE I OF THE GRANT, A REPEAT DOSE NON-GLP DOSE RANGE FINDING (DRF) STUDY IN RATS IS PLANNED TO DETERMINE THE TOLERABILITY OF THE TEST ARTICLE (MT-110) AND TO IDENTIFY POTENTIAL DOSE LIMITING TOXICITIES, TOXICOKINETICS AND TARGET ORGANS. A CARDIAC SAFETY ASSESSMENT WITH ELECTROCARDIOGRAPHY WILL BE RUN IN PARALLEL IN RAT TO ENSURE NO EFFECTS ON CARDIAC CONTRACTILITY, CONSISTENT WITH THE DRAMATIC IMPROVEMENT IN MT-110'S SELECTIVITY PROFILE FOR NMII OVER CMII. MILESTONE DRIVEN TRANSITION TO PHASE II OF THE GRANT INITIATES WITH AN IND-ENABLING GLP SAFETY PHARMACOLOGY STUDY IN RATS TO DETERMINE POTENTIAL TOXIC EFFECTS, IDENTIFY TARGET ORGANS OF TOXICITY, ESTIMATE THE MTD AND NOAEL, EVALUATE THE TK, AND REVERSIBILITY OF ANY ADVERSE EFFECTS FOLLOWING REPEATED DOSE ADMINISTRATIONS. ASSESSMENT OF REPEAT MT- 110 DOSING IN A NON-GLP DRF STUDY IN DOGS WILL ESTABLISH POTENTIAL DOSE LIMITING TOXICITIES, TOXICOKINETICS AND TARGET ORGANS IN A SECOND SPECIES. WHILE THE IMPROVED SELECTIVITY OF MT-110 IS EXPECTED TO REDUCE THE IMPACT ON CARDIAC OUTPUT, CHRONIC METH USE CAN LEAD TO CARDIOMYOPATHY. THEREFORE, WE WILL ESTABLISH THE TOLERABILITY OF MT-110 IN THE CONTEXT OF METH-INDUCED CARDIOMYOPATHY IN RATS. FINALLY, MT-110'S EFFICACY WILL BE DETERMINED IN THE CONTEXT OF OTHER SUBSTANCE USE DISORDERS. THE ABILITY OF MT-110 TO DISRUPT SEEKING OF HEROIN, COCAINE OR NICOTINE IN METH USERS WOULD INCREASE TREATMENT OPTIONS IN A RAPIDLY ESCALATING POLYDRUG USE EPIDEMIC.

3182913.00

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