SP-420 / AbFero - LARVOL DELTA

Safety of SP-420 in the Treatment of Transfusional Iron Overload (clinicaltrials.gov) - P1 | N=2 | Terminated | Sponsor: The University of Texas Health Science Center at San Antonio | N=28 ➔ 2 | Trial completion date: Aug 2025 ➔ Jan 2024 | Suspended ➔ Terminated | Trial primary completion date: Dec 2024 ➔ Jan 2024; Study is on hold by FDA in the US.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Hematological Disorders

Experimental iron chelator therapy reduced signature spinal cord injury (SCI) motor disabilities in a rodent model: neurobiology of improvements (Neuroscience 2024) - "The current set of studies was performed to test the preclinical evaluation of the safety and efficacy of a new iron chelator, SP420, in a rodent model of contusion C-SCI...These data may provide innovative, non-invasive, and patient-centered technologies and treatments that will significantly facilitate the treatment of patients with SCI. Translation of this new, user-friendly therapeutic can significantly benefit veterans and civilians."

Preclinical • CNS Disorders

Safety of SP-420 in the Treatment of Transfusional Iron Overload (clinicaltrials.gov) - P1 | N=28 | Suspended | Sponsor: The University of Texas Health Science Center at San Antonio | Trial completion date: Aug 2024 ➔ Aug 2025 | Recruiting ➔ Suspended | Trial primary completion date: Aug 2023 ➔ Dec 2024

Trial completion date • Trial primary completion date • Trial suspension • Hematological Disorders

Iron chelator therapy improved hallmark spinal cord injury (SCI) disabilities by reducing hemorrhagic iron toxicity, inflammation, and pyroptosis in a rodent model of cervical SCI (Neuroscience 2023) - "To amplify the robustness necessary to significantly improve function in a chronic setting of SCI, future studies will apply the combination of locomotor exercise and SP420 as complementary therapies. Achievement of these goals will provide innovative, non-invasive, and patient-centered technologies and treatments that will greatly facilitate the treatment of patients with SCI."

Preclinical • CNS Disorders

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia (clinicaltrials.gov) - P2 | N=90 | Recruiting | Sponsor: Pharmacosmos A/S | Not yet recruiting ➔ Recruiting | Initiation date: Apr 2023 ➔ Sep 2023

Enrollment open • Trial initiation date • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders

A study of open administration of a drug, which removes iron from the body of patients with transfusion-dependent β-thalassemia, aiming to find the most appropriate dose through use of different dose levels in different patients (clinicaltrialsregister.eu) - P2 | N=90 | Ongoing | Sponsor: Pharmacosmos A/S

New P2 trial • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Rare Diseases

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia (clinicaltrials.gov) - P2 | N=90 | Not yet recruiting | Sponsor: Pharmacosmos A/S

New P2 trial • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders

Safety of SP-420 in the Treatment of Transfusional Iron Overload (clinicaltrials.gov) - P1 | N=28 | Recruiting | Sponsor: The University of Texas Health Science Center at San Antonio | Trial completion date: Mar 2024 ➔ Aug 2024 | Trial primary completion date: Mar 2023 ➔ Aug 2023

Trial completion date • Trial primary completion date • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Oncology

How does a small molecule bind at a cryptic binding site? (PubMed, PLoS Comput Biol) - "To this end, we have conducted unbiased, all-atom MD simulations of the binding of four small-molecule inhibitors (SP4206 and three SP4206 analogs) to interleukin 2 (IL2)-which performs its function by forming a PPI with its receptor-without incorporating any prior structural information about the ligands' binding...Binding of the small molecule stabilized the IL2 binding groove, which when the small molecule was not bound emerged only transiently and incompletely. Moreover, free energy perturbation (FEP) calculations successfully distinguished between the native and non-native IL2-small-molecule binding poses found in the simulations, suggesting that binding simulations in combination with FEP may provide an effective tool for identifying cryptic binding sites and determining the binding poses of small molecules designed to disrupt PPI interfaces by binding to such sites."

Journal • IL2

Safety of SP-420 in the Treatment of Transfusional Iron Overload (clinicaltrials.gov) - P1; N=28; Recruiting; Sponsor: The University of Texas Health Science Center at San Antonio; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Oncology

AbFero Pharmaceuticals Announces Funding From Cure Parkinson's For Lead Compound SP-420 (PRNewswire) - "AbFero...today announced the award of a grant from Cure Parkinson's for the development of their lead, brain-penetrant iron chelating agent, SP-420, as a disease modifying therapy for Parkinson's disease (PD)...This is the second grant AbFero has received to support development of SP-420 for the condition, following recent funding from Eureka EUROSTARS."

Financing • CNS Disorders • Parkinson's Disease

Safety of SP-420 in the Treatment of Transfusional Iron Overload (clinicaltrials.gov) - P1; N=28; Not yet recruiting; Sponsor: The University of Texas Health Science Center at San Antonio

Clinical • New P1 trial • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myelofibrosis • Oncology

AbFero to Receive € 2M Eurostars Funding for Parkinson's Disease Therapy (PRNewswire) - "AbFero Ltd....today announced the funding of approximately € 2M from EUREKA Eurostars for the development of iron chelating compounds including their lead agent, SP-420, as a disease modifying therapy for Parkinson's disease....'With three Phase 1 clinical trials complete, this funding will accelerate our drive to a Phase 2 trial of SP-420 in Parkinson's patients, for whom disease modifying agents remain much needed.'"

Financing • CNS Disorders • Parkinson's Disease

AbFero Announces PK Study Results For SP-420 In Transfusional Iron Overload -- Proceeds With Phase 1-2 Trial Of Iron Chelator (PipelineReview) - "Additionally, AbFero anticipates conducting a Phase 3 pivotal trial in patients with MDS, MFS, β-thalassemia and sickle cell disease using the FDA-approvable endpoint of LIC as measured by MRI."

New P3 trial • Beta-Thalassemia

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias (clinicaltrials.gov) - P2; N=0; Withdrawn; Sponsor: Abfero Pharmaceuticals, Inc; N=74 ➔ 0; Not yet recruiting ➔ Withdrawn

Clinical • Enrollment change • Trial withdrawal • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias (clinicaltrials.gov) - P2; N=74; Not yet recruiting; Sponsor: Abfero Pharmaceuticals, Inc; Trial completion date: Jan 2022 ➔ Jan 2023; Trial primary completion date: Sep 2021 ➔ Sep 2022

Clinical • Trial completion date • Trial primary completion date • MRI

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias (clinicaltrials.gov) - P2; N=74; Not yet recruiting; Sponsor: Abfero Pharmaceuticals, Inc

Clinical • New P2 trial