Phoenicia BioSciences is developing therapeutics for serious hematology-oncology conditions through reactivation of silenced native genes to replace the function of abnormal genes. Our lead therapeutic has a rapid development path.
Abnormal or deficient hemoglobin, the major protein in red blood cells, causes severe anemia and organ damage in beta-thalassemia and sickle cell anemia (beta-hemoglobinopathies). Another type of hemoglobin, fetal hemoglobin or HbF, a form expressed in all humans before birth, functions well, but is silenced during infancy. When re-activated, HbF can replace the function of abnormal “adult” genes, reducing the pathology in the beta-hemoglobinopathies.
PB-04 was identified in a high-throughput screen as a potent inducer of fetal hemoglobin and validated in multiple systems, including primates. PB-04 has 40 years of commercial use, in a combination with another drug in an unrelated therapeutic area. PB-04 has never been formulated or approved as a single agent drug, however, allowing Phoenicia to develop it as a novel proprietary therapeutic for beta-hemoglobin disorders.
Novel mechanism of action: PB-04 reactivates fetal hemoglobin (HbF) by inhibiting two gene repressors that silence HbF in adults. HbF alleviates the anemia in beta-thalassemia, and prevents sickling and reduces anemia in sickle cell disease.
There are no drugs approved for definitive correction of beta-thalassemia. Against the only drug approved for SCD (hydroxyurea), PB-04 wins on performance. Against current treatments such as transfusion + iron chelation, or stem cell transplantation, PB-04 wins on price and accessibility. Development Schema: Phase 2 Trial
Development Schema: Phase 2 Trial
Lead Indication & Market:
Thalassemia Intermedia: (Non transfusion dependent): Hemolytic anemia
Sickle cell disease
No therapeutic approved for treatment of underlying pathology
2 M patients world wide
Potential market for 5-10% of total patients : $ 360 M US/EU $2 B ROW
Rapid Regulatory Path, Exit in 3 years
IND, initial focus ex-US, where approved in a combination for another condition
Development as a single agent in a new condition allows rapid path to registration. Anticipate sale or license of PB-04 within 3 years of initial investment.
Biotechnology R & D expertise from start-up to exit, 70 clinical trials
Strategic and operational expertise in global clinical trials
Thomas Dahl, PhD:
CEO - Veteran drug developer, start-up to exit
Susan Perrine MD:
Founder & CMO - Strategic and operational experience in global trials
Douglas V. Faller MD, PhD:
Founder & CSO, Cancer Center Director Boston University School of Medicine
Jason Walsh, MBA:
CFO - Co-Founder, Provenance Pharmaceuticals
Phoenicia BioSciences, Inc. Weston, MA
Contact: Thomas Dahl CEO 617 818-2735