We are a stem cell company headquartered in South San Francisco, California and focused on drug rescue and regenerative medicine. We believe better cells lead to better medicines™ and that the key to making better cells is precisely controlling the differentiation of human pluripotent stem cells, which are the building blocks of all cells of the human body. For over 15 years, our stem cell research and development teams and collaborators have focused on controlling the differentiation of pluripotent stem cells to produce multiple types of mature, functional, adult human cells, with emphasis on human heart and liver cells for drug rescue applications.
Our stem cell technology platform, which we refer to as Human Clinical Trials in a Test Tube, is based on a combination of proprietary and exclusively licensed technologies for controlling the differentiation of human pluripotent stem cells into multiple types of mature, functional, adult human cells that we use, or plan to develop, to reproduce complex human biology and disease. We are currently producing heart cells and liver cells for our drug rescue applications. Upon completion of this offering, we intend to focus on the drug rescue applications utilizing human heart and liver cells, and further advance, through collaborative research projects, pharmaceutical applications of stem cell-derived blood, bone, cartilage, heart, liver and pancreatic beta-islet cells, including exploring opportunities to leverage our stem cell technology platform for regenerative medicine purposes. Our emphasis in the regenerative medicine arena will be on developing novel human disease models for discovery of small molecule drugs and biologics that activate the endogenous growth and healing processes enabling the body to repair tissue damage caused by certain degenerative diseases.
We believe drug rescue is the highest-value near term commercial application of the human cells we produce and our novel in vitro bioassay systems, CardioSafe 3D and LiverSafe 3D, the foundation of our Human Clinical Trials in a Test Tube platform. Detailed information is often available in the public domain regarding the efficacy, pharmacology, formulation and toxicity of promising drug candidates which already have been tested extensively in in vitro studies, live animal models and even humans, but subsequently have failed due to unexpected toxicity. “Drug rescue” refers to research and development using small molecule drug candidates previously discovered and validated in efficacy studies by pharmaceutical companies, but terminated during development prior to FDA approval due to unexpected heart or liver safety concerns. We refer to these drug candidates as Drug Rescue Candidates™. We have designed our drug rescue model to leverage publicly available information and substantial prior investment by pharmaceutical companies and others in Drug Rescue Candidates. The key commercial objective of our drug rescue model is to generate revenue from license, development and commercialization arrangements involving Drug Rescue Variants™ that we develop having improved safety characteristics. These are proprietary, new, safer small molecule variants of Drug Rescue Candidates we are focused on producing with our medicinal chemistry collaborator and validating internally in our bioassay systems prior to license. We anticipate that each validated lead Drug Rescue Variant will be a proprietary, new molecular entity (NME), suitable as a promising drug development program, either internally or in collaboration with a strategic partner. Through stem cell technology-based drug rescue, we intend to become a leading source of proprietary, small molecule drug candidates to the global pharmaceutical industry.
Using mature cardiomyocytes (heart cells) differentiated from human pluripotent stem cells, we have developed CardioSafe 3D, as a novel, in vitro bioassay system used to assess new drug candidates for potential cardiac toxicity before they are tested in animals or humans. We believe CardioSafe 3D is capable of predicting the in vivo cardiac effects, both toxic and non-toxic, of small molecule drug candidates with greater speed and precision than the long-established, surrogate safety models most often used in drug development, including models using animal cells or live animals, and cellular assays using cadaver, immortalized or transformed cells. Our pluripotent stem cell derived cardiomyocytes (heart cells) and CardioSafe 3D are key components of our Human Clinical Trials in a Test Tube platform and drug rescue programs.
Using mature, hepatocytes (liver cells) derived from human pluripotent stem cells, with adult functional properties, we are currently validating LiverSafe 3D, our second novel stem cell technology-based bioassay system. We believe LiverSafe 3D will enable us to assess new drug candidates for potential liver toxicity and metabolism-based safety issues resulting in adverse drug-drug interactions, early in development, long before animal or human testing. Drug-related liver toxicity and adverse drug metabolism, as a group, represent one of the top-two reasons for safety-related drug failure during clinical development. We plan to use LiverSafe 3D, and the clinically predictive liver biology insight we believe it provides, to expand the scope of our commercial opportunities related to drug rescue.
Regenerative medicine and drug discovery
Although we believe the best and most valuable near term commercial application of our stem cell technology platform, Human Clinical Trials in a Test Tube, is for small molecule drug rescue, we also believe stem cell technology-based regenerative medicine has the potential to transform healthcare in the U.S. over the next decade by altering the fundamental mechanisms of disease and help slow rapidly rising healthcare costs in the U.S. Upon completion of this offering, we intend to explore opportunities to leverage our stem cell technology platform for regenerative medicine purposes, with emphasis on developing novel human disease models for discovery of small molecule drugs and biologics with regenerative and therapeutic potential. Our regenerative medicine focus will be based on our expertise in human biology and differentiation of human pluripotent stem cells to develop functional adult human cells and tissues involved in human disease, including blood, bone, cartilage, heart, liver and insulin-producing pancreatic beta islet cells. Our objective will be to explore regenerative medicine opportunities through pilot nonclinical proof-of-concept studies, after which we intend to assess any potential opportunities for further development and commercialization of therapeutically and commercially promising regenerative medicine programs, either on our own or with strategic partners.
AV-101 for neuropathic pain, epilepsy and depression
With $8.8 million of grant funding awarded from the U.S. National Institutes of Health, we have successfully completed Phase 1 development of AV-101. AV-101, also known as “L-4-chlorokynurenine” and “4-Cl-KYN”, is an orally available small molecule prodrug candidate aimed at the multi-billion dollar neurological disease and disorders market, including neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, epilepsy and depression. Our AV-101 IND application on file at the FDA covers clinical development for neuropathic pain. However, we believe the Phase 1 AV-101 safety studies completed to date will support development of AV-101 for multiple indications, including epilepsy and depression. Upon completion of this offering, we intend to pursue potential opportunities for further development and commercialization of AV-101 for neuropathic pain, epilepsy and depression, either on our own or with a strategic partner. In the event that we successfully complete a strategic partnering arrangement for AV-101, we plan to use the net proceeds from such an arrangement to expand our drug rescue and regenerative medicine programs.