Science
Overview
One of our key competitive advantages is that our systemically dosed product candidates, due to the improvements we have made using our protease engineering platform, can be delivered subcutaneously which is less invasive, more convenient and more efficacious than intravenous IV drugs currently on the market. Our SQ product candidates demonstrate prolonged duration of activity enabling them to provide continuous therapeutic levels.
Publications
Click or tap the “Expand” buttons below to see a full list of our scientific publications, presentations and posters.
Publications
Subcutaneous engineered factor VIIa marzeptacog alfa (activated) in hemophilia with inhibitors: Phase 2 trial of pharmacokinetics, pharmacodynamics, efficacy, and safety Johnny Mahlangu MB BCh, MMed, FCPath (HAEM), BSc (Lab Med), Howard Levy MD, PhD, MMM, Marina V. Kosinova MD, Heghine Khachatryan MD, PhD, Bartosz Korczowski MD, PhD, Levani Makhaldiani MD, PhD, Genadi Iosava MD, Martin Lee PhD, Frank Del Greco BA, MBA. Res Pract Thromb Haemost. 2021;5:e12576.
Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia Gruppo RA, Malan D, Kapocsi J, Nemes L, Hay CRM, Boggio L, Chowdary P, Tagariello G, von Drygalski A, Hua F, Scaramozza M, for the Marzeptacog alfa (activated) Study Group Investigators. J Thromb Haemost 2018; 16: 1984–93.
Posters
Dose Selection For Subcutaneous Marzeptacog Alfa (Activated) In Subjects With Factor VII Deficiency Using Population Pharmacometric Clinical Trial Simulations Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) Congress 2021 – July 19, 2021
Subcutaneous Marzeptacog Alfa (Activated) Effectively Treats Bleeding in FVII Deficient Rats Both When Administered Prior To or After Bleeding Has Started Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) Congress 2021 – July 19, 2021
Dose Selection Of Marzeptacog Alfa (Activated) In Children With Hemophilia: A Population Pharmacokinetic Exposure Matching Strategy Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) Congress 2021 – July 19, 2021
Phase 1 study to evaluate the pharmacokinetics, pharmacodynamics, and safety of ascending doses of subcutaneous marzeptacog alfa (activated) in adult subjects with hemophilia International Society for Thrombosis and Haemostasis (ISTH) Virtual Congress 2020 – July 13, 2020
Marzeptacog alfa (activated) population pharmacokinetics (PK): Simulations for dose selection in Phase 3 trials Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) Virtual Congress 2020 – July 13, 2020
Fast Onset of Action of Subcutaneously Administered Marzeptacog Alfa (Activated) Supports On-Demand Treatment in Hemophilia A Mice Catalyst Biosciences at the EAHAD 2020 meeting – February 5, 2020
Phase 1 study to evaluate the pharmacokinetics, pharmacodynamics, and safety of ascending doses of subcutaneous marzeptacog alfa (activated) in adult subjects with hemophilia Catalyst Biosciences at the EAHAD 2020 meeting – February 5, 2020
The Combination of Marzeptacog Alfa (Activated) or Eptacog Alfa (Activated) with Emicizumab Appears Comparable in the Thrombin Generation Test in Hemophilia A Catalyst Biosciences at the ASH 2019 meeting – Dec 8, 2019
ISTH 2019 – Quality of Life of Hemophilia Patients with Inhibitors – Opportunities for Improvement Catalyst Biosciences at the ISTH 2019 meeting – July 6-10, 2019
Phase 2/3 Trial of Subcutaneously Administered Marzeptacog Alfa (Activated) An Engineered FVIIa In Hemophilia With Inhibitors -Pharmacokinetics, Pharmacodynamics, Safety and Efficacy Catalyst Biosciences at the 60th American Society of Hematology Annual Meeting & Exposition, San Diego Convention Center, San Diego, CA – December 01, 2018
Pharmacokinetics and Pharmacodynamics of daily Subcutaneously Administered Marzeptacog Alfa (Activated) in Hemophilia dogs CBIO EAHAD2017 FVIIa SC PK/PD Dogs – February 01, 2017
Presentations
Crimson 1: A Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptocog Alfa (activated) for On-Demand Treatment of Bleed Events in Subjects with Hemophilia A or B, with Inhibitors Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) 2022 Congress – July 11, 2022
Subcutaneous Marzeptacog Alfa (Activated) is Effective for On-Demand Treatment of Spontaneous Bleeding in Hemophilia A Rats Catalyst Biosciences at the EAHAD 2021 meeting – February 3, 2021
Subcutaneous Marzeptacog Alfa (Activated) is Effective for On-Demand Treatment in Dogs with Hemophilia A Catalyst Biosciences at the EAHAD 2021 meeting – February 3, 2021
Crimson 1 Study: Phase 3 study to evaluate the efficacy and safety of subcutaneous marzeptacog alfa (activated) for on-demand treatment and control of bleeding episodes in subjects with hemophilia A or hemophilia B, with inhibitors Catalyst Biosciences at the ASH congress 2020 – December 7, 2020
ISTH 2019 – Phase 2/3 Trial of Subcutaneous Engineered FVIIa Marzeptacog Alfa (Activated) in Hemophilia A or B with Inhibitors: Efficacy, Safety and Pharmacokinetics Catalyst Biosciences at the ISTH 2019 meeting – July 6-10, 2019
Publications
Efficacy and safety of subcutaneous prophylaxis with dalcinonacog alfa in adults with haemophilia B Johnny Mahlangu, Howard Levy, Martin Lee, Frank Del Greco. Haemophilia. 2021;27:574–580 – April 3, 2021
Safety, pharmacokinetics, and pharmacodynamics of a next-generation subcutaneously administered coagulation factor IX variant, dalcinonacog alfa, in previously treated hemophilia B patients Chur Woo You, Seung-Beom Hong, Suyeong Kim, Ho-Jin Shin, Jin Seok Kim, Jung Woo Han, Soo-Jeong Kim, Do Young Kim, Martin Lee, Howard Levy. J Thromb Haemost. 2021;00:1–9.
Preclinical evaluation of a next-generation, subcutaneously administered, coagulation factor IX variant, dalcinonacog alfa Timothy C. Nichols, Howard Levy*, Elizabeth P. Merricks, Robin A. Raymer, Martin L. Lee. PLOS ONE – October 28, 2020
Activity measurements of dalcinonacog alfa Williams SC & Gray EM. Haemophilia. 2020; 26: 346-353.
Posters
Mitigation of Injection Site Reactions after Subcutaneous Administration of Dalcinonacog Alfa (DalcA) in Hemophilia B Using Preclinical Models Catalyst Biosciences at the International Society for Thrombosis and Haemostasis (ISTH) Congress 2021 – July 19, 2021
Phase 2B Trial to Evaluate the Safety and Factor IX Levels of a Daily Subcutaneous Prophylaxis Treatment Regimen of Dalcinonacog alfa in Hemophilia B Catalyst Biosciences at the WFH 2020 Virtual Meeting – June 15, 2020
ISTH 2019 – A Comprehensive In Silico And In Vitro Immunogenicity Risk Assessment of Dalcinonacog Alfa Shows No Increased Risk Compared With Wild-type FIX Catalyst Biosciences at the ISTH 2019 meeting – July 6-10, 2019
Phase 1/2 Trial of Single and Multiple Dose Subcutaneously Administered Factor IX Variant Cb2679d/ISU304: Pharmacokinetics and Safety Catalyst Biosciences at the 64th Annual Scientific and Standardization Committee Meeting of the International Society on Thrombosis and Haemostasis – July 18, 2018
Pharmacokinetics and Pharmacodynamics of daily Subcutaneously Administered CB2679D/ISU304 in Hemophilia B Dogs CBIO EAHAD2017 FIX SC PK/PD Dogs – February 01, 2017
Pharmacokinetics of Subcutaneously Administered CB 2679D/ISU304 in minipig compared with BeneFIX CBIO EAHAD2017 FIX SC PK/PD Minipigs – February 01, 2017
Pharmacokinetics of Subcutaneously Administered CB2679D/ISU304 in wild-type and Hemophilia B mice Catalyst Biosciences, Inc.: Coagulation Factor IX at ASH Annual Meeting – December 03, 2016
Presentations
Phase 2b Trial to Evaluate the Safety & Factor IX Levels Resulting from a Daily Subcutaneous Prophylaxis Treatment Regimen of Dalcinonacog Alfa in Haemophilia B Catalyst Biosciences at the EAHAD 2020 meeting – February 7, 2020
A Comprehensive In Silico and In Vitro Immunogenicity Risk Assessment of Dalcinonacog Alfa Shows No Increased Risk Compared with Wild-Type FIX Catalyst Biosciences at the HDD 2019 meeting – August 21, 2019
Publications
Gene therapy for hemophilia B using
Posters
Using recombinant adeno-associated viral vectors for long-term expression of a hyperactive human Factor IX mutant in hemophilic mice and comparison of AAV-LK03 and AAV-KP1 in nonhuman primates American Society of Gene & Cell Therapy meeting – May 17, 2022
CB 2679d-GT – A Novel Human Factor IX Variant Shows Enhanced Activity After Delivery Into Hemophilic Mice Using an AAV Capsid With High Liver Transduction American Society of Gene & Cell Therapy meeting – May 12, 2020
AAV Based Hemophilia B Gene Therapy in Mice Using FIX-CB2679d-GT Catalyst Biosciences at the 12th Annual Congress of the European Association for Haemophilia and Allied Disorders – February 6-8, 2019
Presentations
Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B Gene Therapy Catalyst Biosciences at the World Federation of Hemophilia Virtual Summit 2020 – June 19, 2020
Hemophila B Gene Therapy in Mice using a Novel Chimeric AAV Capsid Combined with a Potency Enhanced FIX Variant Catalyst Biosciences at the Gene Therapy for Blood Disorders meeting – March 4, 2020
