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HEMA Biologics

Our Investigational Treatments

Eptacogum Beta, activated (LR769)*: Recombinant Coagulation Factor VIIa

Eptacog Beta (LR769) is a recombinant form of human factor VIIa under FDA review for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors. Currently, two additional phase 3 trials are investigating the use of eptacog beta in pediatric and surgical patients with hemophilia A or B with inhibitors.

* Eptacogum beta, activated is the International Nomenclature Name for the molecule

High-purity von Willebrand Concentrate

High-purity von Willebrand Concentrate, a human plasma-derived von Willebrand factor concentrate, is approved in the European Union for the prevention and treatment of hemorrhage or surgical bleeding in von Willebrand disease when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. LFB and Hema Biologics will file with the FDA in the near future, in hopes of expanding this treatment to patients in the United States.

Our Clinical Trials

Eptacog Beta Studies*

Investigating Eptacog Beta for the on-demand treatment of patients with hemophilia A or B with inhibitors**
*Disclosure: Eptacog beta has not received approval from the Food and Drug Administration (FDA). This information is not intended to draw any conclusions regarding the efficacy and safety of eptacog beta for any indication or dosage.
**PERSEPT: Program for the Evaluation of Recombinant factor Seven Efficacy by Prospective clinical Trials

Phase
1 2 3 4
Dose-Ranging Study (N=15)
Phase 1b
Study Design
Dose escalation, pharmacokinetics, safety and in vivo pharmacodynamics
Ducore, et al. 2017
NCT01708564
doi: 10.1111/hae.13357

Status
Completed

Population
Adults
Hemophilia A or B patients with or without inhibitors, non-bleeding
(20-61 years)
View Study Details
PERSEPT 1 (N=27)
Phase 3a
Study Design
Efficacy, safety, and immunogenicity
Wang, et al. 2017
NCT02020369
doi: 10.1111/hae.13301

Status
Completed

Population
Adolescents & Adults
(12-75 years)
View Study Details
Phase
1 2 3 4
PERSEPT 2 (N=24)
Phase 3b
Study Design
Efficacy, safety, and immunogenicity
NCT02448680

Status
Active, Not Recruiting

Population
Children
(6 months-11 years)
View Study Details
PERSEPT 3
Phase 3b
Study Design
Efficacy and safety in the prevention of excess surgical hemorrhage
NCT02548143

Status
Active, Not Recruiting

Population
Children & Adults
(6 months-75 years)
View Study Details

High-purity von Willebrand Factor Concentrate Studies*

*Disclosure: High-purity von Willebrand concentrate has not received approval from the Food and Drug Administration (FDA). This information is not intended to draw any conclusions regarding the efficacy and safety of high-purity von Willebrand factor concentrate for any indication or dosage.

Phase
1 2 3 4
Pharmacokinetic studies on Wilfactin®, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods (N=25)
Study Design
Pharmacokinetics
Goudemand, et al. 2005
doi: 10.1111/j.1538-7836.2005.01435.x

Status
Completed

Population
Adults
(21-66 years)
View Study Details
Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin®): a prospective study of 50 patients (N=50)
Study Design
Efficacy and Safety
Borel-Derlon, et al. 2007
doi: 10.1111/j.1538-7836.2007.02562.x

Status
Completed

Population
Children, Adolescents & Adults
(5-81 years)
View Study Details
Phase
1 2 3 4
Willebrand International Non-interventional Global Surveillance (N=~80)
Study Design
Post-Marketing Surveillance study
NCT01949220

Status
Currently Recruiting Particpants

Population
Child, Adult, Senior
View Study Details

Disclosure: Eptacog beta and high-purity von Willebrand concentrate have not received approval from the Food and Drug Administration (FDA). This information is not intended to draw any conclusions regarding the efficacy and safety of eptacog beta or high-purity von Willebrand factor concentrate for any indication or dosage.