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NIA Small Business Showcase: Artery Therapeutics, Inc.

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About 5.8 million people in the United States have Alzheimer’s disease (AD). Apolipoprotein E (APOE) is responsible for cholesterol transport in the brain. APOE4, as opposed to APOE3 or APOE2, is a genetic risk factor for developing AD and is found in 65% of all AD patients. Inheriting APOE4 from one parent increases risk of AD threefold, and inheriting it from both parents increases risk 12-fold.

Artery Therapeutics logoUnderstanding causality of APOE4-associated dementia presents a huge opportunity to address the enormous unmet medical need in AD. Based on a lipid transport discovery platform, Artery Therapeutics, Inc., has identified a lead molecule, CS6253, principally ready for human testing.

Artery Therapeutics, Inc., has developed a novel chemical entity CS6253 for the treatment of APOE4-driven dementias, including AD. As demonstrated in multiple mouse models, CS6253 improves lipid loading from cells to APOE4 and improves lipid transport between cells in the brain, a process that is fundamental to nerve growth, amyloid clearance, neuroimmune modulation, and cerebrovascular integrity and functioning. Following pre–Investigational New Drug (IND) discussions with the U.S. Food and Drug Administration (FDA), CS6253 has undergone IND-enabling studies in rats and monkeys showing favorable safety and druggability features.

Interestingly, in monkeys that have APOE, with amyloid and lipid metabolism almost identical to that of humans, CS6253 treatment over nine days showed dose-response reductions of AD risk markers in cerebrospinal fluid (CSF). The primate CSF data support efficacy results in mice models and are predictive of anti-AD effects in humans.

AD affects 1 out of 9 people over 65 years old in the United States, and more than 5.8 million Americans currently suffer from AD. The cost of caring for AD patients and people with dementia is estimated at more than $300 billion in 2020 and is projected to rise to more than $1.1 trillion by 2050 in the United States alone, according to the Alzheimer’s Association. Addressing APOE4 AD would encompass two-thirds of the AD population.

APOE4 is also a risk factor for several other chronic and nonchronic conditions, providing multiple clinical opportunities, some qualifying as orphan indications and breakthrough indications, pending FDA agreement.

CS6253, if successful in clinic, stands to be a powerful new therapeutic for APOE4-associated dementia, including AD. The IND for CS6253 will be compiled in March 2021, and the first-in-human clinical trial would commence soon thereafter.

Although current unsuccessful treatment approaches have focused on removing amyloid and phosphorylated tau in brain, which Artery Therapeutics, Inc., thinks are secondary to lipid transport, the company addresses AD in a precision medicine way by targeting and improving APOE4-mediated cholesterol transport.

Artery Therapeutics, Inc., has discovered and patented a unique class of ABCA1 ligand small molecule peptides for injection that are selective, potent, safe, and druggable. The APOE4-targeted treatment confers specific plasma lipid changes, which will be assessed as biomarkers in the clinic to monitor treatment efficacy. The company projects that the accelerated cognition decline in APOE4 carriers, paired with the pronounced treatment effect size by CS6253, translates to relatively smaller and shorter clinical trials while retaining the same statistical power to meet endpoints. Drug manufacturing is straightforward and consistent and provides a pure and stable drug product. Artery Therapeutics, Inc., has compiled a world-leading translational team with accumulated experience in the areas of basic science, pharmacology, clinical development, and entrepreneurship.

Company Milestones

Scientific

  • 2004-2007: ABCA1 peptide discovery to lead optimization—first campaign
  • 2006-2009: Pharmacology studies for metabolic cardiovascular disease (CVD)
  • 2010-2013: ABCA1 peptide discovery to lead optimization—second campaign and discovery of a unique peptide family including CS6253
  • 2013-2019: Lead selection including pharmacology studies in central nervous system mouse models
  • 2019-2021: IND enabling studies using Good Manufacturing Practice in rats and monkeys and manufacturing of CS6253 for IND compilation in H1 2021

Business

  • 2004: Artery Therapeutics, Inc., was incorporated as a Delaware C-Corp 2004 for purpose of licensing peptide technologies from Lawrence Berkeley National Laboratory at the University of California, Berkeley.
  • 2009-2012: Collaboration development deal with F. Hoffmann-La Roche AG for the treatment of CVD
  • 2013-2016: Submitted now-issued composition-of-matter patents that cover CS6253 and backup compounds
  • 2019-2021: Received NIA Small Business Innovation Research grant to conduct IND-enabling studies

Financial Overview

Artery Therapeutics, Inc., is looking for $12 million in financing to perform human safety, pharmacokinetics, and proof-of-concept studies:

  • Human testing in healthy volunteers (Phase 1 clinical trial)
  • Testing in APOE4 carriers with mild cognitive impairment (Phase 1B clinical trial)

The company anticipates the need for $12 million to reach Phase 2 and an additional $30 million to complete a Phase 2 cognition trial comprising approximately 240 APOE4 carriers with mild cognitive impairment treated for 6 to 12 months.

Intellectual Property

Artery Therapeutics, Inc., has licensed several patents and patent applications from the Lawrence Berkeley National Laboratory, the University of California, and the U.S. Department of Energy, comprising five patent cases: four issued and one pending prosecution. All patents are including composition-of-matter, which have branched out to 37 national, international, and divisional patents. Artery Therapeutics, Inc., takes an active role in patent writing and patent prosecution.

Product Development and Regulatory Strategy

Artery Therapeutics, Inc.’s pre-IND meeting with the Division of Neurology in the FDA Office of Neuroscience in August 2020 provided consensus for key aspects of the development program. Due to the lengthy regulatory path for AD indication, Artery Therapeutics, Inc., flagged for Breakthrough Designation and orphan indications as nonchronic APOE4-associated dementia indications may be a shorter, cheaper, and easier route to registration than the AD indication.

Commercialization Strategy

The management and network of experts have the knowledge and experience to build the company and develop medical therapeutics from concept to proof-of-concept and, if required, to registration. The exceptional medical advisors are experts in the area of neurology, AD, and vascular-metabolic disease and have successful track records performing AD trials worldwide.

Artery Therapeutics, Inc., does not currently intend to become a sales and marketing entity. After proof-of-concept in humans, the technology would be further financed by initial public offering, venture capital, or pharmaceutical companies to garner necessary resources to perform large Phase 2 or Phase 3 trials, to shepherd the New Drug Application/registration, and to commercialize the product.

Company Details

Artery Therapeutics, Inc. website

San Ramon, CA

Industry: Therapeutics

Management Team:

  • Founder and Chief Executive Officer: Jan O. Johansson, M.D., Ph.D.
  • Co-Founder and Chief Operating Officer: Johannes Johansson
  • Co-Founder and Business Officer: Jonas Johansson

Point of Contact:

Jan O. Johansson
Email Jan O. Johansson
(415) 669-4821

Showcase selected for: Longevity Innovations and Neurodegeneration Company Showcase (LINCS), February 2021

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