Emmy Corp

DEVELOPMENT OF NOVEL THERAPEUTICS FOR CANCER CACHEXIA - PROJECT SUMMARY / ABSTRACT CANCER-INDUCED SKELETAL MUSCLE ATROPHY IS A CENTRAL AND DEFINING FEATURE OF THE CANCER CACHEXIA SYNDROME, A HIGHLY PREVALENT CONDITION THAT AFFECTS OVER 50% OF PATIENTS WITH ADVANCED CANCER AND OVER 500,000 PATIENTS PER YEAR IN THE U.S. IN ADDITION TO BEING HIGHLY PREVALENT, CANCER-INDUCED SKELETAL MUSCLE ATROPHY HAS DEVASTATING CONSEQUENCES FOR PATIENTS; IT REDUCES PHYSICAL FUNCTION AND QUALITY OF LIFE, OFTEN COMPLICATES OR PRECLUDES CANCER TREATMENT, AND STRONGLY PREDICTS EARLY MORTALITY FROM CANCER. UNFORTUNATELY, A PHARMACOLOGIC THERAPY FOR CANCER-INDUCED MUSCLE ATROPHY DOES NOT EXIST. THUS, CANCER-INDUCED SKELETAL MUSCLE ATROPHY REPRESENTS A HIGHLY SIGNIFICANT UNMET MEDICAL NEED WITH BROAD RELEVANCE TO CANCER PATIENTS. A MAJOR GOAL OF EMMYON, INC. IS TO DISCOVER AND DEVELOP A PHARMACOLOGIC THERAPY FOR CANCER-INDUCED SKELETAL MUSCLE ATROPHY. TO THAT END, WE RECENTLY DISCOVERED A NATURAL COMPOUND THAT SIGNIFICANTLY REDUCES CANCER-INDUCED MUSCLE ATROPHY IN FIVE WELL-ESTABLISHED AND DISTINCT IN VIVO MOUSE MODELS OF CANCER. WE THEN USED THAT NATURAL COMPOUND AS A LEAD IN MEDICINAL CHEMISTRY PROGRAM AND DISCOVERED AND PATENTED A CONFIDENTIAL AND PROPRIETARY CHEMICAL DERIVATIVE (EMMY1-06) THAT APPEARS TO BE SIGNIFICANTLY MORE POTENT AND MORE EFFICACIOUS THAN THE LEAD COMPOUND IN AT LEAST TWO DISTINCT MOUSE MODELS OF CANCER-INDUCED MUSCLE ATROPHY. IN THIS PHASE II SBIR PROPOSAL, WE SEEK TO CONTINUE THIS EXCITING WORK BY ADVANCING THE DEVELOPMENT OF EMMY1-06 AND RELATED MOLECULES AS PHARMACEUTICALS FOR CANCER-INDUCED SKELETAL MUSCLE ATROPHY. SPECIFICALLY, WE WILL FURTHER INVESTIGATE EMMY1-06'S SAFETY, EFFICACY, AND MECHANISMS OF ACTION IN SEVERAL DISTINCT AND COMPLEMENTARY MOUSE MODELS OF CANCER-INDUCED MUSCLE ATROPHY THAT INVOLVE MULTIPLE TUMOR TYPES, SEXES, AND AGES; THESE STUDIES WILL SIGNIFICANTLY ADVANCE EMMY1-06 TOWARDS CLINICAL DEVELOPMENT AND COMMERCIALIZATION IN SBIR PHASE III. IN PARALLEL TO OUR DETAILED STUDIES OF EMMY1-06, WE WILL ALSO DESIGN, SYNTHESIZE, AND CHARACTERIZE NOVEL COMPOUNDS THAT ARE STRUCTURALLY RELATED TO EMMY1-06, SEEKING TO DISCOVER ADDITIONAL COMPOUNDS WITH PHARMACOLOGIC PROPERTIES THAT ARE SIMILAR TO OR PERHAPS EVEN BETTER THAN THOSE OF EMMY1-06 IN PRECLINICAL MODELS OF CANCER-INDUCED MUSCLE ATROPHY. TOGETHER, THESE STUDIES WILL RIGOROUSLY ADVANCE THE SCIENTIFIC UNDERSTANDING AND COMMERCIAL DEVELOPMENT OF A HIGHLY PROMISING NEW CLASS OF PHARMACEUTICAL AGENTS. THROUGH THIS WORK, WE HOPE TO ULTIMATELY DISCOVER AND DEVELOP A NEW PHARMACOLOGIC THERAPY THAT COULD BROADLY IMPROVE CLINICAL OUTCOMES FOR MILLIONS OF PATIENTS WHO SUFFER FROM CANCER.

DEVELOPMENT OF NOVEL SMALL MOLECULE THERAPIES FOR SKELETAL MUSCLE ATROPHY

DISCOVERY OF NOVEL PHARMACEUTICAL AGENTS FOR SKELETAL MUSCLE ATROPHY

DEVELOPMENT OF NOVEL THERAPEUTICS FOR OBESITY AND TYPE 2 DIABETES - PROJECT SUMMARY / ABSTRACT OBESITY AND TYPE 2 DIABETES AFFECT MILLIONS OF PATIENTS WORLDWIDE AND HAVE ENORMOUS CLINICAL IMPACT BUT ARE NOT FULLY ADDRESSED BY EXISTING PHARMACOTHERAPIES. TWO OF THE MOST IMPORTANT UNMET MEDICAL NEEDS ARE FOR PHARMACOLOGIC AGENTS THAT PROMOTE WEIGHT LOSS WITHOUT MUSCLE LOSS, AND FOR PHARMACOLOGIC AGENTS THAT PROTECT AGAINST DYSFUNCTION OF PANCREATIC BETA CELLS. A MAJOR GOAL OF OUR COMPANY, EMMYON, INC., IS TO DISCOVER AND DEVELOP NEW PHARMACOLOGIC AGENTS FOR OBESITY AND TYPE 2 DIABETES. EMMYON IS LED BY PHYSICIANS AND SCIENTISTS WITH CLINICAL AND SCIENTIFIC EXPERTISE IN OBESITY AND TYPE 2 DIABETES, AND BY AN EXCEPTIONAL MEDICINAL CHEMIST WHO HAS BEEN LEAD INVENTOR OF 8 FDA-APPROVED DRUGS TO DATE. IN PHASE I-EQUIVALENT STUDIES, EMMYON DISCOVERED AND PATENTED A CONFIDENTIAL AND PROPRIETARY SMALL MOLECULE (EMMY1-25) THAT PROMOTES WEIGHT LOSS WITHOUT MUSCLE LOSS AND THAT PROTECTS AGAINST DYSFUNCTION OF PANCREATIC BETA CELLS IN WELL-ESTABLISHED IN VIVO MOUSE MODELS OF OBESITY AND TYPE 2 DIABETES. EMMY1-25 IS ORALLY ADMINISTERED AND POSSESSES EXCELLENT ORAL BIOAVAILABILITY, PHARMACOKINETICS, AND TOLERABILITY IN RODENT MODELS, STRONG EFFICACY TOWARDS OBESITY AND TYPE 2 DIABETES, AND PHARMACOLOGIC EFFECTS (PROMOTION OF WEIGHT LOSS WITHOUT MUSCLE LOSS, AND PROTECTION AGAINST DYSFUNCTION OF PANCREATIC BETA CELLS) THAT COULD FILL IMPORTANT UNMET NEEDS IN CLINICAL MANAGEMENT OF OBESITY AND TYPE 2 DIABETES. IN THIS PHASE II SBIR PROPOSAL, WE SEEK TO CONTINUE THIS EXCITING WORK BY ADVANCING THE DEVELOPMENT OF EMMY1-25 AND RELATED MOLECULES AS PHARMACEUTICALS FOR OBESITY AND TYPE 2 DIABETES. SPECIFICALLY, WE WILL FURTHER INVESTIGATE EMMY1-25'S SAFETY, EFFICACY, DOSE RESPONSE, AND MECHANISMS OF ACTION IN FOUR DISTINCT AND COMPLEMENTARY MOUSE MODELS OF OBESITY AND TYPE 2 DIABETES, AND IN CULTURED PANCREATIC ISLETS FROM HUMANS WITH TYPE 2 DIABETES. IN PARALLEL TO OUR DETAILED STUDIES OF EMMY1-25, WE WILL ALSO DESIGN, SYNTHESIZE, AND CHARACTERIZE NOVEL COMPOUNDS THAT ARE STRUCTURALLY RELATED TO EMMY1-25, SEEKING TO DISCOVER ADDITIONAL COMPOUNDS WITH PHARMACOLOGIC PROPERTIES THAT ARE SIMILAR TO OR PERHAPS EVEN BETTER THAN THOSE OF EMMY1-25 IN PRECLINICAL MODELS OF OBESITY AND TYPE 2 DIABETES. COLLECTIVELY, THESE STUDIES WILL RIGOROUSLY ADVANCE THE SCIENTIFIC UNDERSTANDING AND COMMERCIAL DEVELOPMENT OF A HIGHLY INNOVATIVE AND PROMISING NEW CLASS OF PHARMACEUTICAL AGENTS. IN SBIR PHASE III, THE MOST PROMISING COMPOUND FROM THIS PROJECT WILL BE CARRIED FORWARD INTO AN IND APPLICATION AND CLINICAL STUDIES GEARED TOWARDS FDA APPROVAL FOR THE TREATMENT OF OBESITY AND TYPE 2 DIABETES. THROUGH THIS WORK, WE HOPE TO ULTIMATELY DISCOVER AND DEVELOP A PHARMACOLOGIC AGENT THAT COULD SIGNIFICANTLY IMPROVE CLINICAL OUTCOMES FOR MILLIONS OF PATIENTS WHO SUFFER FROM OBESITY AND TYPE 2 DIABETES.

DISCOVERY OF NOVEL PHARMACEUTICAL AGENTS FOR SKELETAL MUSCLE ATROPHY

DEVELOPMENT OF NOVEL SMALL MOLECULE THERAPIES FOR SKELETAL MUSCLE ATROPHY

DEVELOPMENT OF NOVEL THERAPIES FOR AGING-INDUCED SKELETAL MUSCLE ATROPHY.