Research & Technology

Currently, ChemRegen has two major programs including developing small molecules for heart disease and cancer.

Heart failure is underserved by current therapies. Optimal treatment currently is aimed primarily at improving physiological function of the failing heart rather than regenerating heart muscle cells (cardiomyocytes) that are lost after injury such as myocardial infarct. The heart normally undergoes a regenerative response, but this is inadequate to overcome the progression to failure. Enhancing regeneration is an obvious therapeutic target. However, no pharmaceutical therapy has been described to date. The ChemRegen research program is therefore the first known attempt to commercialize small molecules to regenerate muscle heart endogenously. The lead compounds stimulate cardiomyocyte differentiation and heart cell regeneration. ChemRegen will carry out extensive proof of concept studies for safety and efficacy of its two advanced leads as well as its backup compounds. Because no other US company has a small molecule therapy based on cardiac stem cell differentiation in development at this time, the valuation of ChemRegen has no precedent.

600,000 people die of heart disease annually in the U.S. Heart failure alone, which would be ChemRegen’s primary indication, affects around 5 million people in the United States. About 550,000 new cases are diagnosed each year. More than 287,000 people in the United States die each year from heart failure. Regenerative medicine is a revolutionary approach that focuses on curing conditions as opposed to treating them. One of the greatest needs for regenerative therapy is in the field of organ patches and whole organ replacement. Despite broad public education about organ donation, there remains a large and growing gap in the number of organ donors versus the demand for organs. Each year hundreds of thousands of adults could benefit from a heart transplant but just over 2,000 hearts are available for transplant in the United States. Regenerative medicine will dramatically alter the U.S. healthcare industry and have potential benefits in improved health care and economic savings but again, there is no precedent to place a value on these savings.

The ChemRegen research program is the first known attempt to develop a regenerative medicine small molecule therapy for cardiac disease that is based on the action of cardiomyocyte stem cell differentiation. Currently, to our knowledge, there are no oral medications specifically designed for use as regenerative medicine agents for cardiac cell regeneration in the US market or in the world, and to our knowledge no other US company has a small molecule therapy in development at this time as a small molecule cardiac regenerative medicine.

The drug development program at ChemRegen is in part,` a collaboration of, HBRI’s and the Sanford Burnham Institute’s discovery work in the regenerative medicine area. The company’s objective is to move its discovery program in regenerative medicine to the drug development stage. ChemRegen has identified four distinct compounds that cause cardiomyocyte differentiation from progenitors using in vitro assays. The assays are designed to recapitulate regeneration from progenitors thought to reside in the adult heart after injury, and as such our compounds are expected to stimulate endogenous regeneration in the diseased heart.

An animal model has already been identified and put in place. Because the leads of interest are very drug-like, the barrier to commercialization is different from that of a non-drug-like chemical entity. Drug-like properties of the current lead compounds and the mature nature of their developmental state suggests considerable value (with the composition of matter of new materials and associated utility and attendant technology).

Due to the unique blend of expertise and capabilities, ChemRegen has already entered into preliminary discussions with other stem cell biotechnology companies to collaborate and enter into strategic partnerships. ChemRegen already has an extensive array of collaborators and partners both in the US and abroad. These partnerships acknowledge the value of the technology, the scientific approach and the value of the compounds.