AnHorn Medicines is using its proprietary drug discovery platform, AIMCADD®, to develop novel therapeutics for the greater good of patients in the areas of cancer, neurodegeneration, and age-related diseases.
The second leading cause of death globally
The WHO estimated 18.1 million new cancer cases and 9.6 million deaths in 2018. During their lifetime, one in five men and one in six women develop cancer. While one in eight men and one in eleven women die from the disease. At present, it is the second leading cause of death in the world.
With a collection of more than 200 types, cancer is a large group of diseases that can affect many parts of the body. During a lifetime, every part of the body continues to grow and replenish itself. With great accuracy in the replication process and error-checking, mistakes like gene mutation increasingly occur throughout a lifespan.
The accumulation of gene mutations over a long period of time, years or a lifetime, resulted in the misfunction of proteins. In the cases of cancer, misfunction results in uncontrolled cell growth.
To begin with, AnHorn is focusing on KRAS and its pathways, and the Androgen Receptor as anticancer targets.
AnHorn turns undruggable cancer target to druggable
RAS proteins are small GTPases function as binary molecular switches, cycling between inactive GDP-bound and active GTP-bound states. They connect signals from upstream membrane receptors to a variety of downstream pathways that are required for cell growth and survival. Aberrant activation of RAS activity can lead to uncontrolled cell proliferation and cancer.
Approximately 19% of cancer patients, equivalent to about 3.4 million new cases per year worldwide, harbor RAS mutations. They are the most commonly observed gene alterations in human cancers. From statistics 88% in pancreatic cancer, 55% in colorectal cancer, and 33% in lung adenocarcinoma were due to oncogenic RAS mutations.
AnHorn Medicines is committed to developing effective treatments against RAS proteins and related regulators/ effectors with direct and indirect means. We are utilizing AIMCADD® to design and test BIGPRO® molecules that redirect target proteins for ubiquitination and proteasomal degradation. Degradation of multiple targets will simultaneously produce sustainable benefits to patients in urgent demand.
AR, the Androgen Receptor
BIGPRO® reduces the most common cancer in men
AR is the responding receptor to the stimulation of the male hormones (testosterone and dihydrotestosterone). It is critical for the development and maintenance of the male sexual phenotype and the master controller of prostate cancer development. Early diagnosis in younger ages and androgen-deprivation therapy have achieved satisfactory 5-year and 10-year survival rates. However, the development of metastatic castrate-resistant prostate cancer with the androgen receptor not responding to therapy over time reduces the survival rate significantly. This trend has been continuing on over the past decades. Prostate cancer is the second most common cancer in men worldwide with 1.3 million new cases and over 350,000 deaths in 2018; a satisfactory treatment option is needed.
New advances in targeted-protein degradation in recent years, with which malfunctioned proteins are removed from the body, shed light on prostate cancer treatment. AnHorn is creating novel BIGPRO® molecules to remove androgen receptor and suppress prostate cancer growth.
A major public health concerns with no disease-modifying treatment to date
Neurodegenerative disorders are illnesses that involve the death of neurons in the brain and spinal cord. They are, by far, some of the toughest diseases to cure with debilitating outcomes. Initially the disorders were presented with dementia, loss of body coordination, followed by loss of the ability to walk independently, think clearly, or function in general. Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), and Huntington’s disease (HD) are the most severe and common neurodegenerative disorders.
More than 60 million people worldwide are affected. The abnormal accumulation of specific proteins, including amyloid beta (Aβ), tau, α-synuclein (α-syn), TAR DNA binding protein 43 (TDP43), and repeat-associated non-ATG (RAN) peptides are the hallmarks of age-related neurodegenerative diseases. These disorders have been recognized as major public health concerns with no disease-modifying treatment to date.
A primary risk factors for major human diseases
Age-related diseases are characterized by a progressive loss of physiological integrity which leads to impaired function and increased vulnerability of death. They are the primary risk factors for major human diseases, including cancer, diabetes, cardiovascular disorders, and neurodegenerative diseases. By 2050, the world’s population under the age of over 60 years are expected to reach 2 billion, about 22% of the whole population. Age-related diseases are largely due to the body’s inability to clear faulty proteins and hamper all cell’s regeneration processes.
BIGPRO® molecules are designed to reverse neurodegenerative and age-related diseases.