Phoenicia BioSciences is a virtual company developing therapeutics for serious hematology-oncology conditions. A product ready to enter clinical trials in Q3 2018 acts through reactivation of normal but silenced genes to replace the function of abnormal genes. Our lead therapy is a repurposing of a commercial product in this new indication, with a greatly reduced development path. Other products target established mechanisms.
Abnormal or deficient hemoglobin, the major protein in red blood cells, causes severe anemia and organ damage in β-thalassemia and sickle cell anemia (β-hemoglobinopathies). Another type of hemoglobin, HbF, a form expressed in all humans before birth, functions well to carry O2, but is suppressed during infancy. When re-activated, HbF can replace the function of abnormal “adult” genes, reversing the pathology in the β-hemoglobinopathy blood diseases.
PB-04 was identified as a potent inducer of HbF in a high-throughput screen of approved drugs. Activity is validated in multiple in vitro and in vivo systems, including primates, which are predictive of human clinical responses. PB-04 has 40 years of human use, in combination with another drug in a completely unrelated therapeutic area. PB-04 has never been formulated or approved as a single agent drug, however, allowing Phoenicia to repurpose it as a novel proprietary therapeutic for β-hemoglobin disorders.
Mechanism of action: PB-04 reactivates fetal hemoglobin (HbF) by inhibiting 4 gene repressors that silence HbF. HbF reduces the anemia in β-thalassemia, and reduces sickling and anemia in sickle cell disease.
Preclinical development studies are being conducted by TRND, an NIH program for Rare and Neglected Diseases.
Hemoglobinopathies are the single most-common genetic diseases worldwide, and are classified as a rapidly-growing global health burden and unmet medical need. There are no drugs approved for correction of β-thalassemia, and only one for the underlying pathology in sickle cell disease. While β-thalassemia is an orphan condition in the US and Europe, and PB-04 qualifies for Orphan Drug Status, global prevalence of the diseases is in the millions of patients. Annual revenue projections for PB-04 for β-hemoglobinopathies are $360 M US or EU, and $1B ROW, based on revenue for supportive products.
There are no drugs approved for definitive correction of β-thalassemia. Compared to the drug approved for enhancing fetal globin in SCD (hydroxyurea), PB-04 wins on performance. Against current treatments such as transfusion + iron chelation, gene therapy or stem cell transplantation, PB-04 wins on price and accessibility.
Phoenicia has issued patents on use of PB-04 in these diseases. There are no competing patents for the use of PB-04 in other conditions; the Company has freedom to operate. Phoenicia has received Orphan Drug Designation in the EU and will file shortly in the US.
Phoenicia is seeking partnership or $8.0 M investment to carry PB-04 through dose optimization and proof-of-concept trials, and to prepare for registration trials.
Re-purposing PB-04 as a single agent, from a therapeutic with a known safety profile, offers a relatively lower risk path to registration, particularly in countries where the drug is already approved in a combination. Initiation of a trial of PB-04 in patients is targeted to begin in Q1 2019.
Management and Project Team
Susan P. Perrine MD, President & CMO, has founded 3 biotech companies, and has strategic and operational experience in directing global trials in target diseases.
Douglas V. Faller, MD, PhD, CSO, is expert in gene regulation, inventor of multiple drugs targeting novel pathways, founder of 4 biotech companies, and directs global trials in oncology in a large pharma.