Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients living with serious hematologic diseases, presented five posters spanning several of its hematology programs at the 64th
Disc Medicine continues to make considerable progress towards the development of potentially first-in-class therapeutic candidates for hematologic disorders,' said
A copy of each poster presentation from the ASH Annual Meeting is available on Disc's website.
Bitopertin: Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1, GlyT1, that is designed to inhibit heme biosynthesis, with two Phase 2 trials currently ongoing in erythropoietic protoporphyria (EPP), a disease characterized by extreme phototoxic reactions and liver disease caused by an accumulation of the heme metabolite metal-free PPIX.
Poster 2346: BEACON: A Phase 2, Randomized, Open Label Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX Concentrations in Participants with Erythropoietic Protoporphyria (EPP)
BEACON Phase 2 study is an ongoing randomized, open-label, multiple dose clinical trial being conducted in
Patients will be stratified by average time to prodrome then randomized into two open-label parallel arms of 20mg (30 minutes to prodrome) oral bitopertin once daily for 24 weeks
Primary endpoint is the percent change from baseline in metal-free protoporphyrin IX (PPIX) levels
Key secondary endpoint is total hours of sunlight exposure from
Upon completion of the 24-week treatment period, patients have the option of continuing in an open-label extension of the study
Poster 3661: Bitopertin, a Selective Glycine Transporter 1 Inhibitor, Reduced PPIX Level and Improved Liver Fibrosis in a Mouse Model of Erythropoietic Protoporphyria (EPP)
Data demonstrated that bitopertin, an investigational, orally available selective inhibitor of GlyT1, can reduce PPIX accumulation in blood and reduce cholestasis and fibrosis in the liver of an EPP mouse model
Mice that received 100 ppm and 200 ppm bitopertin exhibited a 31% and 49% reduction in free PPIX in blood, respectively
Bitopertin treatment led to 51% and 45% reductions in PPIX concentration in the livers of EPP mice at 100 ppm and 200 ppm, respectively
Addition of bitopertin to the diet of EPP mice reduced the incidence of ductular reaction and the severity of liver fibrosis, with 60% of the control group exhibiting ductular reaction as compared to 30% of the 100 ppm bitopertin group and 10% of the 200 ppm bitopertin group as evaluated through histopathological analysis
We believe these data suggest that bitopertin may be disease-modifying and may be effective in treating the photosensitivity and modifying the hepatoxicity in EPP patients
DISC-0974: DISC-0974 is an investigational monoclonal antibody (mAb) targeting a bone morphogenetic protein (BMP)-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation.
Poster 2339: DISC-0974, an Anti-Hemojuvelin Antibody, Reduces Hepcidin and Mobilizes Iron in
A double blind, placebo-controlled Phase 1a study in healthy volunteers, evaluated single ascending doses of DISC-0974 at 7mg and 28mg IV and 14mg, 28mg, and 56mg SC
DISC-0974 demonstrated favorable PK profiles, and important aspects of the compound's pharmacokinetic (PK)/pharmacodynamics (PD) relationship were observed
Demonstrated tolerability, with only Grade 1 adverse events observed across all dose levels
DISC-0974 was shown to lower serum hepcidin-25 and increase serum iron in a dose-dependent manner.
56mg SC dose group (N=6) also showed an increase in red blood cells and hemoglobin compared to placebo
A study of once-monthly dosing of DISC-0974 is ongoing in myelofibrosis and anemia patients (NCT05320198) and studies are planned in other diseases with anemia of inflammation, such as chronic kidney disease (CKD)
Poster 3641: DISC-0974, an Anti-Hemojuvelin (HJV) Monoclonal Antibody, Reduced Hepcidin and Improved Anemia in a
Treatment of adenine-induced rat models of CKD with DISC-0974 resulted in a reduction in HAMP gene expression in the liver as measured at the end of the study, consistent with the proposed mechanism of action of blocking the formation of the BMP/BMPR/HJV complex
DISC-0974 led to a reduction in serum hepcidin levels, increased iron availability, and substantially prevented the reduction in hemoglobin that is seen in animals with renal impairment induced by adenine
Study provides preclinical proof of concept for the development of DISC-0974 for the treatment of patients with CKD anemia and Disc is planning a Phase 2 study of DISC-0974 in CKD anemia
DISC-0998: DISC-0998 is an investigational, potent and highly selective HJV mAb engineered with the mutation combination of T250Q/M429L (QL-mutation) in the Fc region, which is designed to alter binding to the FcRn receptor and increase PK half-life.
Poster 3657: Preclinical Pharmacokinetics and Pharmacodynamics of DISC-0998, a Humanized Anti-Hemojuvelin (HJV) Monoclonal Antibody to Suppress the Production of Hepcidin
Study evaluated the PK/PD relationships of DISC-0998 with hepcidin, serum iron, and transferrin saturation (TSAT) in male cynomolgus monkeys
Following single SC or IV dose, DISC-0998 exhibited low clearance (SC CL/F 0.14 - 0.83 mL/hr/kg, IV CL 0.14 mL/hr/kg), small volume of distribution (Vz 50 -104 mL/kg), and nonlinear PK as expected for a mAb
Compared to DISC-0974, DISC-0998 clearance was 33% lower, and half-life (t1/2) was over 2 times longer
Findings support further development of DISC-0998 for disorders related to anemia of inflammation, with the potential for less frequent dosing compared to DISC-0974
About Disc Medicine
Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that are designed to affect fundamental pathways of red blood cell biology. We are committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic diseases.
On
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