Publications

Evaluation of patient‐reported outcome measures for on‐demand treatment of hereditary angioedema attacks and design of KONFIDENT, a phase 3 trial of sebetralstat

Cohn DM, Aygören-Pürsün E, Bernstein JA, Farkas H, Lumry WR, Maurer M, Zanichelli A, Iverson M, Hao J, Smith MD, Yea CM, Audhya PK, Riedl MA

2023 Clinical and Translation Allergy

Sebetralstat, Investigational Oral On-Demand Treatment for HAE: KONFIDENT Trial Design

Aygören-Pürsün E, Zanichelli A, Cohn DM, Farkas H, Bernstein JA, Audhya PK, Smith MD, Yea CM, Lumry WR, Riedl MA, Maurer M

IWAA 2023

Short-Term Prophylaxis With Sebetralstat, an Investigational Oral On-Demand Treatment for Hereditary Angioedema, in KONFIDENT-S

Bernstein JA, Riedl MA, Lumry WR, Audhya PK, Smith MD, Yea CM

IWAA 2023

In Their Own Words - Patient Descriptions of the Earliest Recognition of HAE Attack Onset

Manning ME, Burnette AF, van Kooten S, Heckmann M, Danese S, Goga L, Guilarte M

HAEA National Summit 2023

Recognizing the Importance of Early On-Demand Treatment in the HAE Attack Journey

Jones DH, Ogbogu P, van Kooten S, Heckmann M, Danese S, Goga L, Magerl M

HAEA National Summit 2023

Preferences of People with Hereditary Angioedema for On-Demand Treatment: A US-Based Qualitative Study

Bukstein DA, Vibha Desai V, Goga L, Czado S, Brown M, Myers K, Audhya PK, Settipane RA, Busse PJ

HAEA National Summit 2023

PB0536: Oral Factor XIIa inhibitor KV998086 protects against FeCl3 induced thrombosis in mice.

Clermont A, Pinckney F, Hill M, Hampton SL, Feener EP

ISTH 2023

Understanding the complex decision-making associated with on-demand treatment of hereditary angioedema (HAE) attacks

Grumach A, van Kooten S, Heckmann M, Danese S, Goga L, Garcez T

EAACI 2023

Remaining burden of hereditary angioedema (HAE) attacks despite modern long-term prophylaxis

Betschel S, van Kooten S, Heckmann M, Danese S, Caballero T

EAACI 2023

Short-Term Prophylaxis with Sebetralstat, an Investigational Oral On-Demand Treatment for Hereditary Angioedema, in KONFIDENT-S

Bernstein JA, Riedl MA, Lumry WR, Audhya PK, Smith MD, Yea CM

EAACI 2023

Results From a Randomized, Double-Blind, Placebo-Controlled, Phase 1 Trial Evaluating Sebetralstat Pharmacokinetics, Pharmacodynamics, and Safety/Tolerability in Healthy Japanese, Chinese, and White Adults

Hide M, Iverson M, Jhee S, Hansen E, Duckworth EJ, Hampton SL, Yoon E, Honda D

EAACI 2023

Patients delay treating hereditary angioedema (HAE) attacks with currently available, injectable, on-demand therapies: associated with on-demand treatment of hereditary angioedema (HAE) attacks

Valerieva A, van Kooten S, Heckmann M, Danese S, Goga L, Longhurst H

EAACI 2023

Sebetralstat Effectiveness in the Treatment of Hereditary Angioedema Attacks Rated Mild or Moderate at Baseline in the Phase 2 Trial

Longhurst HJ, Smith MD, Yea CM, Audhya PK

EAC 2023

Reporting of Administration Site Reactions with Parenteral Drugs for the On-Demand Treatment of Hereditary Angioedema Attacks –Analysis of the FAERS Database 2009 to 2022

Tachdjian R, Savic S, Fridman M, Frade J, Audhya PK

EAC 2023

Preferences of People with Hereditary Angioedema for On-Demand Treatment: A US-Based Qualitative Study

Bukstein D, Desai V, Goga L, Brown M, Settipane RA

EAC 2023

Characterizing Attack-Related Health Utility in Hereditary Angioedema

Craig T, Cribbs KA, Czado S

ISPOR 2023

A Systematic Review of Healthcare Resource Utilization and Direct Medical Costs in Hereditary Angioedema

Craig T, Cribbs KA, Czado S

ISPOR 2023

A Systematic Review of Socioeconomic Burden in Hereditary Angioedema

Craig T, Cribbs KA, Czado S

ISPOR 2023

Assessing the Utility of Existing Hereditary Angioedema Disease Models for Health Economic Evaluation of Future Therapies

Schwander B, Lahue BJ, Cribbs KA, Czado S

ISPOR 2023

Assessing the Burden of Injection with Parenteral Hereditary Angioedema Therapies

Soteres DF, Cribbs KA, Czado S

ISPOR 2023

Rationale for the Short-term Prophylaxis Regimen with Sebetralstat in KONFIDENT-S

Iverson M, Duckworth E, Hansen E, Hampton SL, Smith MD, Audhya PK, Yea CM

C1 Inhibitor Workshop 2023

HAE Patients Decision to Carry On-demand Treatment When Away from Home

Betschel S, van Kooten S, Heckmann M, Danese S, Goga L, Guilarte M

C1 Inhibitor Workshop 2023

Route of Administration Preferences of People with Hereditary Angioedema for On-demand Treatment: A US-based Qualitative Study

Desai V, Ledia Goga L, Czado S, Brown M, Myers K, Bukstein D, Audhya PK, Bouillet L

C1 Inhibitor Workshop 2023

Factor XII contributes to VEGF-induced retinal edema and neuroretinal responses in mice

Clermont AC, Murugesan N, Ustunkaya T, Aiello LP, Feener EP

ARVO 2023

Assessing the Potential Benefits of Oral Therapies in Hereditary Angioedema

Soteres DF, Cribbs KA, Guo W, Czado S

AMCP 2023

Treatment with Sebetralstat Reduces the Cumulative Symptom Severity of Hereditary Angioedema Attacks in a Phase 2 Trial

Bernstein JA, Riedl MA, Lumry WR, Audhya PK, Smith MD, Yea CM

AAAAI 2023

A Comparative Bioavailability of Sebetralstat Following Administration of Orally Disintegrating Tablets and Film-Coated Tablets in Healthy Volunteers

Smith MD, Iverson M, Hansen E, Audhya PK, Yea CM

AAAAI 2023

Anxiety Associated With Parenteral On-demand Treatment For Hereditary Angioedema (HAE)

Burnette A, Anderson J, Longhurst H, van Kooten S, Heckmann M, Danese S, Goga L

AAAAI 2023

Patient Perspectives On Early Use Of On-demand Treatment For Hereditary Angioedema (HAE) Attacks to Reduce Severity and Duration

Radojicic C, Manning M, Guilarte M, Heckmann M, van Kooten S, Danese S, Goga L

AAAAI 2023

Reporting of Adverse Drug Reactions with Parenteral Drugs for the On-Demand Treatment of Hereditary Angioedema Attacks – Analysis of the FAERS Database 2009 to 2022

Tachdjian R, Savi S, Fridman M, Frade J, Audhya PK, Fasehun M

AAAAI 2023

Targeted Literature Review to Assess the Studies Supporting Short-term Prophylactic Treatment Options for the Preprocedural Prevention of Attacks in Patients With Hereditary Angioedema

Riedl MA, Bernstein JA, Lumry WR, Audhya PK, Smith MD, Yea CM

WSAAI 2023

Pharmacokinetic, Pharmacodynamic, and Safety Profile of Sebetralstat in Healthy Japanese and White Adults: Results From a Phase 1, Randomized, Double-Blind, Placebo-Controlled Trial

Iverson M, Jhee S, Hansen E, Duckworth EJ , Hampton SL, Yoon E

WSAAI 2023

Efficacy of the Oral Plasma Kallikrein Inhibitor Sebetralstat (KVD900) by Attack Location in a Phase 2 Clinical Trial in Patients With Hereditary Angioedema

Aygören-Pürsün E, Zanichelli A, Cohn DM, Audhya PK, Williams P, Yea CM, Smith MD

WSAAI 2023

Impact of Hereditary Angioedema (HAE) Attacks on Quality of Life and Activities of Daily Living

Busse PJ, Caballero T, van Kooten S, Danese S, Goga L

ACAAI 2022

Real-World Treatment Burden Associated with Parenteral On-Demand Therapies for Hereditary Angioedema

Tachdjian R, Savic S, Fridman M, Frade J, Audhya PK, Fasehun M

ACAAI 2022

Sebetralstat (KVD900): A Potent and Selective Small Molecule Plasma Kallikrein Inhibitor Featuring a Novel P1 Group as a Potential Oral On Demand Treatment for Hereditary Angiodema

Davie R, Edwards H, Evans D, Hodgson S, Stocks M, Smith A, Rushbrooke L, Pethen S, Roe M, Clark D, McEwan P, Hampton S

PMID: 36251573

Absorption, metabolism, and excretion of [14C]-sebetralstat (KVD900 following a single oral does in healthy male participants

Mutch P, Bashir M, Jung B, Yi P, Iverson M

PMID: 36200371

The Global and Regional Impact of Hereditary Angioedema (HAE) Attacks on Mental Health, Activities of Daily Living and Quality of Life

Busse PJ, Caballero T, van Kooten S, Danese S, Goga L

2022 HAEi Global Leadership Workshop

Patient Perspectives on an Optimal Outcome Measure to Assess Efficacy in the Acute Treatment of Hereditary Angioedema Attacks

Riedl MA, Cohn DM, Aygören-Pürsün E, Zanichelli A, Farkas H, Bernstein JA, Lumry WR, Smith MD, Yea CM, Audhya PK

2022 HAEi Global Leadership Workshop

Pharmacology of KVD824, an Investigational Drug for Prophylaxis of HAE

Grivcheva-Panovska V, Smith MD, Duckworth EJ, Hampton SL, Yea CM, Mutch P, Iverson M, Hansen E, Feener EP

BRADYKININ SYMPOSIUM

Sebetralstat Effectiveness in the Treatment of Hereditary Angioedema Attacks Rated Mild or Moderate at Baseline in the Phase 2 Trial

Longhurst HJ, Smith MD, Yea CM, Audhya PK

ASCIA 2022

Efficacy and Safety of the Oral Plasma Kallikrein Inhibitor Sebetralstat (KVD900) in Adolescent and Adult Patients With Hereditary Angioedema: Phase 3 Trial Design

Aygören-Pürsün E, Zanichelli A, Cohn DM, Farkas H, Bernstein JA , Audhya PK, Smith MD, Yea CM, Lumry WR, Riedl MA, Maurer M

EAACI 2022

Novel Oral Small Molecule FXIIa Inhibitor Blocks Contact System Activation In Vivo

Clermont A

EAACI 2022

Population Pharmacokinetic Analysis of Sebetralstat (KVD900) in Healthy Adult Volunteers and Patients With Hereditary Angioedema Predicts Similar Exposure in Adolescent Patients

Smith MD, Rodgers T, Sharman J, Mutch P, Tachdjian R, Savic S,  Farrell C

EAACI 2022

Efficacy of the Oral Plasma Kallikrein Inhibitor Sebetralstat (KVD900) by Attack Location in a Phase 2 Clinical Trial in Patients With Hereditary Angioedema

Aygören-Pürsün E, Zanichelli A, Cohn DM, Audhya PK, Williams P, Yea CM, Smith MD

EAACI 2022

Oral factor XIIa inhibition blocks angiotensin converting enzyme inhibitor induced angioedema in mouse

Clermont AC, Murugesan N, Feener EP

KININ 2022

Oral FXIIa inhibitor KV998086 suppresses FXII zymogen mediated kallikrein kinin system activation

Murugesan N, Lee DK, Duckworth EJ, Smith SA, Clermont AC, Hampton SL, Feener EP

KININ 2022

Oral sebetralstat (KVD900) provides rapid inhibition of the kallikrein kinin system in patients with hereditary angioedema

Duckworth EJ, Lee DK, Murugesan N, Rushbrooke L, Hampton SL, Smith M, Yea C, Audhya P, Feener EP

KININ 2022

KONFIDENT Phase 3 Trial Design for Sebetralstat (KVD900), a Novel Investigational Oral Plasma Kallikrein Inhibitor for the On-Demand Treatment of Hereditary Angloedema Attacks

Lumry WR, Aygören-Pürsün E, Zanichelli A, Cohn DM, Farkas H, Bernstein JA, Audhya PK, Smith MD, Yea CM, Riedl MA, Maurer M

EAC 2022

Agreement Between Improvements in Patient Global Impression of Change and Other Measures of Improvement and Attack Resolution Observed in a Phase 2 Trial With Sebetralstat (KVD900) in Patients With Hereditary Angioedema

Audhya P, Smith MD, Williams P, Yea CM, Cohn DM

EAC 2022

Oral Sebetralstat (KVD900) Provides Rapid Inhibition of Plasma Kallikrein and Fast Improvement in Attack Symptoms in Patients With Hereditary Angioedema

Smith MD, Duckworth EJ, Hampton SL, Yea CM, Audhya P, Feener EP

 EAC 2022

Agreement of Patient Global Impression of Change With Attack Resolution or Use of Rescue Medication in Patients With Hereditary Angioedema
Audhya P, Williams P, Yea C, Cohn D

AAAAI 2022

Rapid Plasma Kallikrein Inhibition Following Oral KVD900 Is Associated With Early Symptom Relief in Patients With Hereditary Angioedema
Duckworth EJ, Feener EP, Yea CM, Audhya P, Hampton SL, Smith MD

AAAAI 2022

KVD900, an oral on-demand treatment for hereditary angioedema: Phase 1 study results
Maetzel A, Smith MD, Duckworth EJ, Hampton SL, De Donatis GM, Murugesan N, Rushbrooke LJ, Li L, Francombe D, Feener EP, Yea CM

PMID: 35086692

On-demand Oral Treatment With KVD900 for HAE Attacks Achieves Rapid Exposure and Improves Patient Outcomes
Bernstein JA, Huamin LH, Yea CM, Maetzel A, Audhya P, Williams P, Banerji A, Riedl MA

ACAAI 2021

Relationship Between PGI-C Scale and Other PROs in KVD900 Trial in Hereditary Angioedema
Audhya P, Williams P, Yea CM

ACAAI 2021

A single on-demand treatment with orally administered KVD900 significantly slows progression and accelerates resolution of attacks in patients with hereditary angioedema (HAE): Results of a phase 2, placebo-controlled, double-blind cross-over trial
Aygören-Pürsün E, Zanichelli A, Cohn DM, Farkas H, Cancian M, Hakl R, Kinaciyan T, Magerl M, Martinez-Saguer I, Stobiecki M, Grivcheva-Panovska V, Kiani-Alikhan S, Krcmova I, Bernstein J, Manning M, Stroud C, Hanzlikova J, Li HH, Longhurst HJ, Melamed IR, Young P, Feener E, Iverson M, Maetzel A, Morten RM, Smith MD, Watissée MI, Williams P, Yea CM, Maurer M, Banerji A, and Riedl MA

EAACI 2021

Prevalence of hereditary angioedema with normal C1-inhibitor (nC1-HAE) in the United States: Results from a nationwide survey of HAE-treating physicians
Riedl MA, Danese M, Danese S, Ulloa J, and Maetzel A

EAACI 2021

Current management of hereditary angioedema with normal C1-inhibitor (nC1-HAE) in the United States: Results from a nationwide survey of HAE-treating physicians
Riedl MA, Danese M, Danese S, Ulloa J, and Maetzel A

EAACI 2021

Oral plasma kallikrein inhibitor KV998052 improves arterial blood oxygenation in a murine model of acute respiratory distress syndrome (ARDS)
Murugesan N, Clermont AC, and Feener EP

EAACI 2021

Selective Factor XIIa inhibitor KV998083 protects mice against captopril induced vascular leakage and cleavage of high molecular weight kininogen
Clermont AC, Murugesan N, Edwards HJ, Whitney SA, Pethen SJ, Duckworth EJ, Hampton SL, Feener EP

EAACI 2021

Fast improvement of hereditary angioedema (HAE) attacks with the oral on-demand plasma kallikrein inhibitor KVD900: an analysis of the pharmacokinetic and pharmacodynamic profile of KVD900 and attack symptom severity during a double-blind, randomized phase 2 cross-over trial in patients with HAE type I and II
Zanichelli, Andrea

C1-INHIBITOR WORKSHOP 2021

One-Time Intravitreal Injection of KVD001, a Plasma Kallikrein Inhibitor, in Patients with Central Involved Diabetic Macular Edema and Reduced Vision: An Open-Label Phase 1B Study
Sun JK, Maturi RK, Boyer DS, Wells JA, Gonzalez VH, Tansley R, Hernandez H, Maetzel A, Feener EP, Aiello LP.

OPHTHALMOLOGY RETINA

Rapid and nearly complete suppression of plasma kallikrein activity with the oral inhibitor KVD900: results of a phase 1 study evaluating KVD900's potential as a treatment for acute attacks of HAE
Maetzel A, Smith MD, Morten RM, Hampton SL, De Donatis GM, Murugesan NI, Rushbrooke LJ, Li L, Duckworth EJ, Feener EP, Yea C.

EAACI 2019

High plasma exposures of KVD900 achieved in First in Human study markedly inhibit plasma prekallikrein activation; early blockade of plasma kallikrein (PKa) may halt attacks in Hereditary Angioedema (HAE) by reducing contact system activation.
Duckworth EJ, Hampton SL, Li L, Murugesan N, Rushbrooke L, Smith M, Morten R, Maetzel A, Yea C, and Feener EP.

C1-INHIBITOR WORKSHOP 2019

Novel oral plasma kallikrein (PKa) inhibitors KV998052 and KV998054 ameliorate VEGF-induced retinal thickening in a murine model of retinal edema.
Murugesan N, Clermont AC, Pethen SJ, Li L, Duckworth EJ, Hampton SL, and Feener EP.

ARVO 2019

KVD900 as a Single Dose, Rapid, Oral Plasma Kallikrein Inhibitor for the On-Demand Treatment of Hereditary Angioedema Attacks: Pharmacokinetic and Pharmacodynamic results from a Phase 1 Single Ascending Dose Study.
Hampton SL, De Donatis GM, Murugesan N, Rushbrooke LJ, Li L, Duckworth E, Smith MD, Morten RM, Maetzel A, Yea CM and Feener EP.
AAAAI 2019

Efficacy of plasma kallikrein inhibitors in plasma is dependent on the rate of formation of the enzyme-inhibitor complex.
De Donatis GM, Murugesan N, Duckworth E, Robson PA, Li L, Rushbrooke LJ, Feener EP and Hampton SL.
KININ 2018

KVD900 protects high molecular weight kininogen (HK) from ex vivo plasma kallikrein-mediated cleavage in plasma from patients with hereditary angioedema (HAE): Results from a semi-automated capillary-based immunoassay.
Feener EP, Murugesan N, Robson PA, Li L, Rushbrooke LJ, Thoonen R, De Donatis GM, Riedl M, Zuraw B, and Hampton SL.
EAACI 2018

A novel oral plasma kallikrein (PKal) inhibitor KV123833 blocks VEGF-mediated retinal vascular hyperpermeability in a murine model of retinal edema (#3464).
Murugesan N, Clermont AC, Rushbrooke LJ, Robson PA, Thoonen R, Pethen SJ, Hampton SL, and Feener EP. 
ARVO 2018

Plasma kallikrein mediates brain hemorrhage and edema caused by tissue plasminogen activator therapy in mice after stroke.
Simao F, Ustunkaya T, Clermont AC, and Feener EP. 
Blood. 2017.
PMID:28130211

Plasma Kallikrein Mediates Vascular Endothelial Growth Factor-Induced Retinal Dysfunction and Thickening.
Clermont A, Murugesan N, Zhou Q, Kita T, Robson PA, Rushbrooke LJ, Evans DM, Aiello LP, and Feener EP.
Invest Ophthalmol Vis Sci. 2016; 57:2390-9.
PMID:27138737

Plasma Kallikrein-Kinin System as a VEGF-Independent Mediator of Diabetic Macular Edema.
Kita T, Clermont AC, Murugesan N, Zhou Q, Fujisawa K, Ishibashi T, Aiello LP, Feener EP.
Diabetes. 2015; 64:3588-99.
PMID:25979073

Intraocular hemorrhage causes retinal vascular dysfunction via plasma kallikrein.
Liu J, Clermont AC, Gao BB, Feener EP.
Invest Ophthalmol Vis Sci. 2013;54:1086-1094.
PMID:23299478

Plasma kallikrein mediates retinal vascular dysfunction and induces retinal thickening in diabetic rats.
Clermont A, Chilcote TJ, Kita T, Liu J, Riva P, Sinha S and Feener EP.
Diabetes. 2011;60(5):1590-8.
PMID:21444925

Hyperglycemia induced cerebral hematoma expansion is mediated by plasma kallikrein.
Liu J, Gao B-B, Clermont AC, Blair P, Chilcote TJ, Sinha S, Flaumenhaft R and Feener EP.
Nature Medicine. 2011;17:206-10.
PMID:21258336

Plasma kallikrein and diabetic macular edema.
Feener EP.
Curr. Diab. Rep. 2010 Aug; 10(4):270-5.
PMID:20535647

Plasma kallikrein mediates angiotensin AT1 receptor stimulated retinal vascular permeability.
Phipps JA, Clermont AC, Sinha S, Chilcote TJ, Bursell S-E, and Feener EP.
Hypertension 2009; 53:175-81.
PMID:19124682

The kallikrein-kinin system in diabetic retinopathy: Lessons for the kidney.
Phipps JA and Feener EP.
Kidney Int., 2008;73(10):1114-9.
PMID:18272958

Extracellular carbonic anhydrase mediates hemorrhagic retinal and cerebral vascular permeability through prekallikrein activation.
Gao BB, Clermont A, Rook S, Fonda SJ, Srinivasan VJ, Wojtkowski M, Fujimoto JG, Avery RL, Arrigg PG, Bursell SE, Aiello LP and Feener EP.
Nature Medicine. 2007;13:181-8.
PMID:17259996