Sylvia Dobo

First‐in‐Human Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Oral Complement Factor D Inhibitor BCX9930 in Healthy Participants

Matthew C. Davidson, Xilin Chen, Amanda Mathis, Sylvia Dobo et autres

Abstract BCX9930 is a potent, selective oral inhibitor of complement Factor D that inhibits the activation of the complement alternative pathway (AP) and has the potential for treatment of complement‐mediated diseases. This was a first‐in‐human, randomized, double‐blind, placebo‐controlled study that evaluated safety, …

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P826: FACTOR D INHIBITION WITH ORAL BCX9930 LEADS TO SUSTAINED CONTROL OF HEMOLYSIS AND SYMPTOMS OVER 48 WEEKS IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA INADEQUATELY CONTROLLED ON C5 INHIBITORS

Austin Kulasekararaj, Wolfgang Füreder, Andrew McDonald, Jacques Le Roux Malherbe et autres

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by hemolysis due to uncontrolled activity of the alternative pathway (AP) of complement. BCX9930 is an oral Factor D inhibitor that targets the AP rate-limiting enzyme with the potential to prevent both intravascular hemolysis and …

gb, at, Afrique du Sud, us, it (code pays fourni par la source)

Pharmacokinetics and Safety of the Nucleoside Analog Antiviral Drug Galidesivir Administered to Healthy Adult Subjects

Amanda Mathis, David Collins, Sylvia Dobo, Dennis M. Walling et autres

Galidesivir (BCX4430) is an adenosine nucleoside analog broadly active in cell culture against multiple RNA virus families, and active in animal models of viral diseases associated with Ebola, Marburg, yellow fever, Zika, and Rift Valley fever. Current studies demonstrated the pharmacokinetics and …

us (code pays fourni par la source)

Protocole d’utilisation thérapeutique (PUT) et de recueil d’informations du Berotralstat dans le cadre de l’autorisation temporaire d’utilisation de cohorte (ATUc) de l’Agence Nationale de Sécurité du Médicament (ANSM)

Laurence Bouillet, Isabelle Boccon‐Gibod, Delphine Gobert, O. Fain et autres

fr, us, be (code pays fourni par la source)

Long‐term safety and effectiveness of berotralstat for hereditary angioedema: The open‐label APeX‐S study

Henriette Farkas, Marcin Stobiecki, Jonny Peter, Tamar Kinaciyan et autres

BACKGROUND: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein recently approved for prevention of angioedema attacks in adults and adolescents with hereditary angioedema (HAE). The objective of this report is to summarize results from an interim analysis of an ongoing …

hu, pl, Afrique du Sud, at, de, gb, us, il, dk, fr, sk, nz, mk, ch, au (code pays fourni par la source)

Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial

Isao Ohsawa, Daisuke Honda, Yusuke Suzuki, Tomoo Fukuda et autres

BACKGROUND: With no approved treatments in Japan for the prevention of hereditary angioedema (HAE) attacks, there is a significant unmet need for long-term prophylactic therapies for Japanese patients with HAE. Berotralstat (BCX7353) is an oral, once-daily, highly selective inhibitor of plasma kallikrein …

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Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial

Bruce L. Zuraw, William R. Lumry, Douglas Johnston, Emel Aygören‐Pürsün et autres

BACKGROUND: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein in development for the prophylaxis of hereditary angioedema (HAE) attacks. OBJECTIVE: Our aim was to determine the efficacy, safety, and tolerability of berotralstat in patients with HAE over a 24-week treatment …

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Oral Prophylaxis with Berotralstat (BCX7353) Reduces Use of Standard of Care (SOC) On-demand Medication in Patients with Hereditary Angioedema (HAE): APeX-2 Study Results

Jonathan A. Bernstein, Bruce L. Zuraw, Emel Aygören‐Pürsün, Huamin Li et autres

us, de, fr (code pays fourni par la source)

Gastrointestinal (GI) Adverse Events (AEs) Observed With Berotralstat (BCX7353) Treatment for Hereditary Angioedema (HAE) are Primarily Mild, Self-limited, and Diminish with Time on Treatment

Douglas Johnston, William R. Lumry, Aleena Banerji, Emel Aygören‐Pürsün et autres

us, de, it (code pays fourni par la source)

Patterns of Treatment and Retreatment of Acute Attacks of Hereditary Angioedema (HAE) with Standard of Care (SOC) On-Demand Medication: Results from the APeX-2 Study

Emel Aygören‐Pürsün, Douglas Johnston, William R. Lumry, Huamin Li et autres

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P150 ORAL PROPHYLAXIS WITH BCX7353 REDUCES HAE ATTACK RATES AND IS WELL-TOLERATED: APEX-2 STUDY RESULTS

Bruce L. Zuraw, William R. Lumry, A. K. Banerji, Emel Aygören‐Pürsün et autres

us, it (code pays fourni par la source)

P154 SAFETY AND TOLERABILITY OF ONCE-DAILY ORAL KALLIKREIN INHIBITOR BCX7353 IN PHASE 3 APEX-2 HAE STUDY

Marc A. Riedl, William R. Lumry, Aleena Banerji, Emel Aygören‐Pürsün et autres

us, it (code pays fourni par la source)