U.S. Food and Drug Administration accepts the submitted Biologic License Application for Coagulation Factor VIIa (Recombinant), activated eptacog beta.
Healthcare professionals, learn more about the science behind HEMA Biologics investigational treatments for the rarest bleeding disorders.
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“We aspire to support patients living with these disorders, partner with the broader community that cares for them, and bring meaningful treatments and services to help better their daily lives.”
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W. Allan Alexander, MD brings 26 years of experience in the care and management of clinical bleeding in rare disorders and acute illness. A Cardiac Anesthesiologist who practiced in elite hospitals, Dr Alexander’s drug and device career includes leadership roles with Thermo CardioSystems, Inc, Micromed Technologies, Inc, CEDRA Clinical Research, ZymoGenetics, Inc (a subsidiary of Novo Nordisk then BMS) and CSL Behring, USA. Dr Alexander was the first employee of HEMA Biologics, in January 2016.
Dr Marsh brings over 20 years of experience in clinical pharmacy, industry medical affairs work, and leadership experience at pharmaceutical companies including GlaxoSmithKline, Meditech Media, Solvay Pharmaceuticals (now Abbvie), MedInfo Results, and UCB, Inc. Dr Marsh joined HEMA Biologics in 2017 to build the Medical Affairs practice team with a focus on helping offer solutions for healthcare providers, caregivers, and patients in the rare bleeding disorders community. Dr Marsh enjoys fitness, family time, and cheering on her daughter in competitive figure skating.
Gary leads the customer interface team that has responsibility for payer and specialty channel relationships and contracting, as well as contributing to the managed markets marketing strategy for HEMA Biologics. Gary has worked in the industry for over 30 years and built the HEMA Biologic’s Market Access team.
Gary is a fan of the Cincinnati Reds and Bengals and the Ohio State Buckeyes. He resides in Ohio with his wife, Cathy, and enjoys traveling, fishing, golf, fine wines, and Kentucky’s great bourbons.
Brandon is focused on commercializing innovative therapy options for the bleeding disorder community through HEMA Biologics. He has over 15 years of experience in pharmaceutical marketing, business development, sales, and brand management. Prior to HEMA Biologics, Brandon was in sales and brand management at Procter and Gamble Pharmaceuticals. In his spare time, Brandon loves spending time with his family and friends and traveling to new places.
Kate has a passion for engaging with the bleeding disorder community and developing resources that positively impact the lives of individuals affected by rare bleeding disorders. Prior to HEMA Biologics, she spent 10 years working for Eli Lilly and Company, with the majority of that time in oncology. In her free time, Kate loves spending time outside with her husband, son, and four-legged child.
As a consumer brand manager at HEMA Biologics, Elyse is responsible for consumer marketing initiatives. Prior to HEMA Biologics, she spent 4 years creating and marketing consumer programs and services for the rare bleeding disorder community at Shire. Elyse earned a B.S. in Life Sciences Communication from the University of Wisconsin. In her spare time Elyse enjoys spending time with family, cooking, and reading.
Darcie attended Florida State University and practiced marketing in New York City before she moved back to her hometown of Louisville, KY. For HEMA Biologics, Darcie manages commercial marketing efforts, including media and advertising, promotional materials, and conference/symposia initiatives and execution. She loves spending time with friends and family, hiking with her dog Bailey, and any excuse to escape to the beach.
As Social Media Coordinator, Blake is always in search of new ways to connect with the community in the digital space. He is a recent graduate of the University of Kentucky where he earned his Bachelor’s Degree in Kinesiology. In his spare time, Blake enjoys playing his guitar, listening to music, and spending time with his friends. For links to all HEMA Biologics Social Media accounts, check the bottom of the page!
Vince is the National Sales Director for HEMA Biologics and has over 20 years’ experience in healthcare in various specialties, including cardiovascular, neurology, hematology, and immunology. He is particularly passionate about the hemophilia community and building lasting and meaningful relationships. He has been with HEMA Biologics since its beginning and is enthusiastic about building a company that has winners (products, people, culture, leadership, and vision).
Vince has a degree in Finance from Clemson University and lives in Louisville, KY, with his wife, son, and daughter. He enjoys watching his kids compete in athletics and is a Clemson Football Fanatic.
Heather is an experienced accounting manager with an established history of working in the healthcare industry. At HEMA Biologics, she has focused on creating a supportive accounting department for both internal and external customers. Prior to her role with HEMA Biologics, Heather spent 9 years in the accounting and finance department in the physical therapy industry. In her spare time, Heather enjoys spending time with family and friends, exercising, and reading.
Melanie is the Senior Director of Human Resources at HEMA Biologics. She brings 20 years of Human Resources and Employee Relations expertise, and holds her Professional in Human Resources certification. In her free time, Melanie enjoys spending time with her family, traveling, and is an avid sports fan.
Rebecca is passionate about accelerating company growth and sourcing top talent to assist in achieving company goals. Offering over 10 years of expertise in HR, Executive Search, Talent Management and Leadership Development, Rebecca has expertise in a wide variety of areas within Human Resources. Rebecca holds her Bachelor’s degree in Business/ HR Management from Indiana Wesleyan University. Outside of the office, Rebecca loves enjoying life with her husband and 2 sons, being outdoors, playing sports, and cooking.
Phase: 4 | Status: Currently recruiting participants | Intervention: Von Willebrand Factor Concentrate | NCT01949220
Collect information about WILLFACT or WILFACTIN in their real-life clinical use and identify the therapeutic practices in an international environment.
Learn more about this trial at clinicaltrials.gov
Phase: 3 | Status: Active, Not Recruiting | Intervention: Eptacog Beta (LR769) | NCT02548143
Phase: 3 | Status: Active, Not Recruiting | Intervention: Eptacog Beta (LR769) | NCT02448680
To assess the safety, efficacy, and pharmacokinetics of two separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients (birth to <6 years old), and 12 patients (≥6 years old to <12 years)
Wang, et al. 2017
Phase: 3 | Status: Completed | Intervention: Eptacog Beta (LR769) | NCT02020369 | doi: 10.1111/hae.13301
Haemophilia A or B patients with inhibitors have been treated with Factor VIIa (FVIIa)-containing bypassing agents for over 20 years. However, due to uncertainty regarding dose response and thrombotic risk, the use of a gradual, titrated, minimal dosing strategy remains prevalent, potentially hampering early hemostasis.
Evaluate the dose-dependent efficacy, safety, and immunogenicity of activated eptacog beta (rhFVIIa)—a new recombinant inhibitor bypassing agent for the treatment of bleeding episodes (BEs).
A Phase 3, randomized, crossover study of two Initial Dose Regimens (IDRs) in 27 bleeding congenital haemophilia A or B subjects with inhibitors was conducted to evaluate on-demand treatment of mild/moderate BEs. Intravenous 75 μg/kg or 225 μg/kg initial doses with 75 μg/kg subsequent doses by schedule were administered until clinical response.
The primary endpoint was sustained clinical response 12 hours following initiation of treatment, determined by a composite of objective and patient-reported indicators of clinical response. In the 75 μg/kg IDR, 84.9% (95% confidence interval [CI]; 74.0%, 95.7%) of mild/moderate BEs at 12 hours were successfully treated compared to 93.2% (95% CI; 88.1%, 98.3%) treated in the 225 μg/kg IDR. Efficacy between the IDRs was statistically different (P<.020) in mild/moderate bleeding episodes. Both IDRs were well tolerated with no detectable immunogenic or thrombotic responses to rhFVIIa or host cell proteins.
The dose-dependent efficacy seen in this study supports individualizing the initial dose of eptacog beta to optimize clinical response. By reducing uncertainty, the PERSEPT 1 results should increase the adoption of early hemostasis as a treatment goal for clinicians who treat haemorrhage in the inhibitor population.
To access the peer-reviewed article, visit http://onlinelibrary.wiley.com/doi/10.1111/hae.13301/epdf
To learn more about this trial visit: clinicaltrials.gov
Ducore, et al. 2017
Phase: 1 | Status: Completed | Intervention: Eptacog Beta (LR769) | NCT01708564 | doi: 10.1111/hae.13357
The availability of different initial doses of activated eptacog beta
(recombinant human FVIIa, rhFVIIa) may provide therapeutic options when treating
bleeding in patients with congenital haemophilia who have developed inhibitory antibodies to
factor VIII (FVIII) or factor IX (FIX). This study evaluated escalated doses of a new
rhFVIIa product as a prelude to selecting the doses for clinical efficacy evaluation in
To assess the safety, pharmacokinetics, and laboratory pharmacodynamics of 3 doses
of rhFVIIa in non-bleeding patients with congenital haemophilia A or B with or without
Adult male patients (18-75 years old) with congenital haemophilia A or B (with
or without inhibitors) received infusions of rhFVIIa at doses of 25, 75, or 225 μg/kg body
weight. Ten patients were treated at each dose level, and each patient received two different
dose levels. Descriptive methods were used to analyse the data.
Administration of rhFVIIa at all doses was well tolerated. Pharmacokinetic
analyses showed that peak FVIIa plasma levels (Cmax) were approximately proportional to
dose and correlated well with peak thrombin generation. Total AUC0-inf also was
approximately dose proportional. Clot formation and duration correlated with FVIIa activity.
Repeat doses did not produce an immunological response.
In the first dose-escalation study of rhFVIIa to support product registration,
eptacog beta at doses of 25, 75, and 225 μg/kg was pharmacodynamically active and well
tolerated in non-bleeding patients with congenital haemophilia A or B.
To access the peer-reviewed article, visit http://onlinelibrary.wiley.com/doi/10.1111/hae.13357/epdf
Borel-Derlon, et al. 2007
Phase: 4 | Status: Completed | Intervention: Von Willebrand Factor Concentrate | doi: 10.1111/j.1538-7836.2007.02562.x
A plasma-derived von Willebrand factor (vWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers.
Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU/dL and 46% had FVIII < 20 IU/dL) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was “excellent” or “good” in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was “excellent” or “good” in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications, and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies.
This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.
To access the peer-reviewed article, visit onlinelibrary.wiley.com
Goudemand, et al. 2005
Phase: 4 | Status: Completed | Intervention: Von Willebrand Factor Concentrate | doi: 10.1111/j.1538-7836.2005.01435.x
In order to correct the primary von Willebrand factor (vWF) defect and avoid supra-physiologic plasma levels of factor VIII, a pure VWF concentrate almost devoid of FVIII was developed and used in France since 1989.
The pharmacokinetic (PK) profile of the most recent version of this concentrate (Wilfactin®; LFB, Les Ulis, France), treated with three virus-inactivation/removal methods (solvent/detergent, 35 nm filtration, dry heat treatment), was investigated in 25 patients. Seventeen patients with various types of clinically severe von Willebrand disease (VWD) were included in a crossover, randomized trial carried out in five European centers and comparing Wilfactin® with concentrates containing both FVIII and VWF (FVIII/VWF). Eight type 3 VWD patients were included in another trial carried out in six French centers comparing Wilfactin® with its previous version (Facteur Willebrand-LFB®; LFB) that adopted one virus-inactivation method only.
For both the measurements evaluated in this study (VWF antigen, VWF:Ag; and VWF ristocetin co-factor activity, VWF:RCo), Wilfactin® had a PK profile similar to that of the FVIII/VWF concentrates and of Facteur Willebrand-LFB®. VWF:RCo and VWF:Ag recoveries were 2.1 ± 0.3 and 1.8 ± 0.3 per IU/kg, respectively, and the half-lives were 12.4 ± 1.8 and 15.9 ± 1.5 h. The FVIII synthesis rate was 5.8 ± 1.0 IU/dL/h, with a half-life of 15.8 ± 2.4 h.
The PK of vWF and FVIII have not been altered by the three virus-inactivation/removal steps during the manufacturing of Wilfactin®.
To access the peer-reviewed article, visit onlinelibrary.wiley.com