β-thalassemia:  Unmet Medical Need

What are β-hemoglobinopathies? 1

  • Hemoglobin diseases are a global health burden
  • β-hemogloinopathies cause:
    • Life-threatening anemias
    • organ damage
    • early mortality
  • No drugs approved for β-thalassemia
  • One drug for Sickle Cell Disease (SCD

New therapeutics are urgently needed for these global, lethal diseases

Market Opportunity2

  • Markets for β-thalassemia & Sickle Cell Disease
  • Orphan conditions in the US and Europe
  • Worldwide – China, India, Africa, South America, SE Asia

Market Size:  

  • 2 M patients worldwide
  • The market will grow as our product extends patient lifespan

Revenue Projections:

5Projecting 5-10% of total patients:

  • $ 1B US/EU   
  • $ 2B ROW

 

Product Profile

PB-04

  • 8A small molecule, excellent oral bioavailability
  • Based on the new discovery of an existing product – a model for repurposing
  • Not an NCE
  • Abbreviated Dose-ranging evaluation should not be extensive
  • Validation: Efficacy in vitro and in animal models, including primate
  • Clinical Development: 40 yrs clinical safety experience (for a different indication) – Safety surprises are not anticipated
  • Approved in EU, Canada, Asia, South America (only in a combination)
  • Potential global impact
  • Already used in many countries where target populations are prevalent

Phoenicia’s Solution

9Our Therapeutic Approach

    • Reactivate silenced fetal (γ) globin to replace defective β-globin
    • Utilizes an endogenous gene, present in all humans
    • PB-04 : Discovered through HTS of FDA or EMEA-approved drugs
    • PB-04 selected for safety and in vivo efficacy
    • Validation: Efficacy in vitro and in animal models, including primate

Technology

  • Small molecule Inducer of endogenous fetal (γ) globin to replace the defective adult (β) 8globin in the β-hemoglobinopathies
  • Mechanism of Action:
  • PB-04 disrupts repression of the silenced γ-globin gene promoter
  • Activates transcription of the γ-globin gene

Preclinical Efficacy:  Induction of Fetal (γ) Globin

Validation

  • Efficacy in vitro  – increases γ-globin in cultures of patients’ blood
  • In vivo Efficacy – Transgenic Mice
  • Increases γ-globin-containing red blood cells
  • In vivo Efficacy – Baboon Model (predictive of human activity
  • Increased γ-globin mRNA & total Hgb, despite ongoing phlebotomy

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Value Proposition / Differentiation

  • Competitive Advantage
    • There are no drugs approved for the definitive correction of beta-thalassemia.  
    • Against current treatments such as transfusion + iron chelation, or stem cell transplantation/gene therapy, PB-04 wins on price and accessibility
    • Against the only drug approved for SCD (hydroxyurea), PB-04 wins on performance.
  • De-risked for safety: 40 years of clinical safety experience
  • Approved in EU, Canada, Asia, South America (only in combination)
  • Other candidates under development in the area
    • iron modulators, receptor antagonists
    • siRNAs, biologics
    • All present difficulties for lifelong use
  • Rapid regulatory path,  Exit in 3 years
    • IND for a repurposed product, initial focus ex-US, where approved in a combination for another condition
    • Repurposing as a single agent in a new condition allows a rapid path to registration. Anticipate sale or license of PB-04 within 3 years of initial investment.

Intellectual Property

  • Utility and formulation patent applications
  • Orphan Drug protection

Development & Milestones

    • Dose optimization (3 mos) → efficacy (6 mos)10
    • Partner / Exit within 3 years
    • Most likely Buyers:
      • Global Pharma companies with orphan drug or hematology focus (iron chelators, PNH) or geographic presence

Management Team

Thomas Dahl, Ph.D.: CEO – 25-year drug development veteran in academia, biotech, and global pharma.

Susan P. Perrine, MD:  CMO – Multiple INDs, from bench to Phase II international trials, CSO & VP Clinical Affairs, biotech

Douglas V. Faller, Ph.D., MD: CSO – Discovery to Ph II international trials, SAB Chair, biotech

Jason Walsh: CFO – Experienced Biotech/Pharma executive, co-founder Provenance Biopharmaceuticals

Curt Scribner: Regulatory, 25 years former FDA division chief Biotechnology R & D expertise from start-up to exit, 70 clinical trials Strategic and operational expertise in global clinical trials

Company Overview

Phoenicia Biosciences, founded in 2011, is a clinical-stage pharmaceutical company engaged in developing therapeutics for serious hematology-oncology conditions through reactivation of silenced genes to replace the function of abnormal genes. Our lead therapy is a repurposing of a commercial product in a new indication, supporting an abbreviated development path.