Market Opportunities

1. Metabolic Indications

Obesity and Metabolic Dysfunction Associated Fatty Liver Disease (MAFLD) are growing and costly pandemics in need of safe, effective and convenient therapies

A. Obesity

Obesity prevalence has nearly tripled since 1975 and is now a pandemic:

  • Affecting one third of the world population
  • Responsible for more than 10% of US health care costs

The Economic burden of obesity is large:

Current medical treatments for obesity:

  • have poor benefit-to-risk profiles with unpleasant side effects leading to discontinuation and poor compliance
  • fail to achieve long-term therapeutic results
  • do not meet patients’ expectations
  • often are not covered by health insurance plans

The US Weight Loss and Diet Control market was worth $66 Billion in 2017

A safe and effective (>10% weight loss) drug should garner up to $20 Billion in annual revenues


  • Metabolic Dysfunction Associated Fatty Liver Disease (MAFLD) affects 25% of the world adult population with a range from 13.5% in Africa to 31.8% in the Middle East.
    • Lifetime costs of all MAFLD patients in the United States in 2017 were $223 billion, and the cost of the advanced MAFLD population was $95.4 billion.
  • MAFLD prevalence can be inferred from the prevalence of obesity and diabetes
  • Prevalence of liver steatosis is 76% in obese patients
  • There is currently no approved drug for the treatment of MAFLD/NASH
  • The MAFLD market is expected to reach $21 billion globally, by 2025 (

Investment Highlights

  • Target Fat Accumulation and related Metabolic Diseases with high unmet medical needs: The Obesity and Diabetes (Diabesity) and the Metabolic Dysfunction Associated Fatty Liver Disease (MAFLD) pandemics
  • Focus on targeting Oligonucleotide Therapeutics (ONTs) microRNA analogs which increase lipid oxidation, insulin sensitivity, mitochondrial activity and energy expenditure
  • Convenient self-local administration of drug to increase compliance

Target Product Profile

  • Convenient regimen (monthly to twice a year local administrations under the abdomen skin) for targeted delivery to the adipose tissues. Transdermal patches or microneedles are also tested
    • Better patient compliance and persistence with therapy
    • Expected weight loss > 10% meeting patient expectations
    • Improvement of T2DM, MAFLD and circulating cholesterol
    • No requirement for lifestyle changes
    • No expected cardiovascular and intestinal adverse events
    • Unaltered brain functions
    • No expected drug interactions
    • Better quality of life

2. Oncologic Indication

  • Ovarian cancer is one of the most common gynecologic cancers that has the highest mortality rate
  • The prognosis for ovarian cancer remains poor, with an overall 46% 5-year survival rate
  • Worldwide, every year more than 300,000 women are diagnosed with ovarian cancer and more than 200,000 succumb to ovarian cancer
  • 90% of women are diagnosed with High Grade Serous Ovarian Cancer (HGSOC) at an advanced stage. As the most lethal type of ovarian cancer, 80-90% of women diagnosed with HGSOC die of their disease. Unfortunately it is also the most common type of epithelial ovarian cancer, accounting for 70-80% of all cases
  • Due to its clinical, biological and molecular complexity, ovarian cancer is still considered one of the most difficult tumors to manage as it lacks a clear driver mutation
  • Ovarian Cancer is a multi-factorial disease that cannot be easily controlled by classical therapeutic agents whose Mechanism of Action is one drug-one target or one drug-two/three targets
  • Presently, debulking cytoreductive surgery represents the gold standard for the treatment of ovarian cancer along with platinum based chemotherapy regimens
  • For patients who become platinum resistant, few options are available and efficacy is limited for those regimens
  • An effective therapy for High Grade Serous Ovarian Cancer should qualify for FDA Expedited Programs based on the following criteria:
    • Serious Condition
    • Available Therapies
    • Unmet Medical Need
  • The global market for Ovarian Cancer Drugs is expected to reach $10.1 billion by 2027 driven by the introduction of targeted therapies

Probability of Success (POS) of the pre-IND Phase for the targeting generation 2.5 of AptamiR’s ONTs for HGSOC is based on:

  • Target identification and validation in silico, in vitro and in vivo: High POS based on our knowledge of generations 1 and 2 compounds
  • Efficacy demonstration in silico, in cultures of human cells and in animal models of Ovarian Cancer
  • Toxicity in primary cultures of human cells and in 2 species of animal models: POS greatly improved with the chemical structure of generation 2.5 compounds with targeted delivery
  • Chemistry, Manufacturing and Control (CMC): High POS related to Solid Phase Synthesis process
  • Unmet Medical Need: High level for Ovarian Cancer


  • AptamiR successfully completed three rounds of Angels Financing and a Series A, raising a total of $7,000,000
  • AptamiR is now seeking to raise tranches of a $15-20M Series B financing while concurrently evaluating geographic licensing opportunities and strategic partnerships

AptamiR’s Pipeline


Please contact us regarding interest in business development, licensing and collaboration opportunities: