Vasomera (pemziviptadil) / PhaseBio Pharma - LARVOL DELTA

Augmentation of VIP signaling prevents the development of DMD-Associated Cardiomyopathy through inhibition of NF-κB signaling (AHA 2025) - "Collectively, the study data suggests augmentation of cardiac-specific VIP signaling prevents cardiac dysfunction through inhibition of NF-kB signaling in a murine model of DMD."

Late-breaking abstract • Cardiomyopathy • Cardiovascular • CNS Disorders • Duchenne Muscular Dystrophy • Fibrosis • Immunology

ImmunoForge Receives IND Approval for Phase 2 Clinical Trial of 'Pemziviptadil' for DMD Cardiomyopathy Treatment from FDA... (PRNewswire) - "ImmunoForge announced that it has received approval for the Phase 2 clinical trial IND for 'Pemziviptadil (development code name PF1804),' a treatment for DMD (Duchenne Muscular Dystrophy) cardiomyopathy, from the U.S. Food and Drug Administration (FDA)....Pemziviptadil was selected for a strategic technology-type international joint technology development project by the Korea Institute for Advancement of Technology in 2023 and is receiving a total of KRW 3 billion in support until 2026."

Commercial • IND • New P2 trial • Duchenne Muscular Dystrophy

Study to Assess the Safety, Tolerability and PK/PD After 4 Weekly SC Injections of PB1046 in Subjects With Stable HFrEF (clinicaltrials.gov) - P2 | N=29 | Completed | Sponsor: PhaseBio Pharmaceuticals Inc. | Phase classification: P2a ➔ P2

Phase classification • Cardiovascular • Heart Failure

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC Pemziviptadil (PB1046) in Subjects With Symptomatic PAH (clinicaltrials.gov) - P2 | N=35 | Terminated | Sponsor: PhaseBio Pharmaceuticals Inc. | Suspended ➔ Terminated; Terminated: Study drug resupply delayed (Covid-19)

Trial termination • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

LTE: Long-Term, Open Label Extension Study of Pemziviptadil (PB1046) in PAH Subjects Following Completion of Study PB1046-PT-CL-0004 (clinicaltrials.gov) - P2 | N=25 | Terminated | Sponsor: PhaseBio Pharmaceuticals Inc. | Suspended ➔ Terminated; Terminated: Study drug resupply delayed (Covid-19)

Trial termination • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC Pemziviptadil (PB1046) in Subjects With Symptomatic PAH (clinicaltrials.gov) - P2; N=35; Suspended; Sponsor: PhaseBio Pharmaceuticals Inc.; N=63 ➔ 35; Recruiting ➔ Suspended

Clinical • Enrollment change • Trial suspension • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

LTE: Long-Term, Open Label Extension Study of Pemziviptadil (PB1046) in PAH Subjects Following Completion of Study PB1046-PT-CL-0004 (clinicaltrials.gov) - P2; N=25; Suspended; Sponsor: PhaseBio Pharmaceuticals Inc.; N=63 ➔ 25; Trial completion date: Feb 2024 ➔ Jan 2022; Recruiting ➔ Suspended; Trial primary completion date: Jan 2024 ➔ Jan 2022

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial suspension • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

"$PHAS voluntarily ending Phase 2b trial of pemziviptadil in pulmonary arterial hypertension (PAH)" (@BioStocks)

P2b data • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

VANGARD: Pemziviptadil (PB1046), a Long-acting, Sustained Release Human VIP Analogue, Intended to Provide Clinical Improvement to Hospitalized COVID-19 Patients at High Risk for Rapid Clinical Deterioration and Acute Respiratory Distress Syndrome (ARDS). (clinicaltrials.gov) - P2; N=54; Terminated; Sponsor: PhaseBio Pharmaceuticals Inc.; Active, not recruiting ➔ Terminated; Company no longer pursing indication

Clinical • Trial termination • Acute Respiratory Distress Syndrome • Cardiovascular • Heart Failure • Immunology • Infectious Disease • Inflammation • Myocardial Infarction • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • IL6 • TNFA

[VIRTUAL] Augmentation of Vasoactive Intestinal Peptide Signaling Prevents the Development of Duchenne Muscular Dystrophy-Associated Cardiomyopathy Through Inhibition of NF-κB Signaling (AHA 2020) - "Utilizing a genetically modified form of VIP (PB1046) targeting cardiomyocytes, we hypothesized that augmentation of VIP signaling prevents the development of DMD-associated cardiomyopathy through inhibition of NF-κB...The molecular mechanism underlying the benefits of VIP signaling suggests an upregulation of cAMP-CREB signaling with downregulation of NF-kB signaling leading to inhibition of inflammation and fibrosis within drug-treated DMD hearts. VIP signaling in DMD may serve as a new therapeutic target for the treatment of DMD-associated cardiomyopathy."

Cardiomyopathy • Cardiovascular • CNS Disorders • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy

VANGARD: Pemziviptadil (PB1046), a Long-acting, Sustained Release Human VIP Analogue, Intended to Provide Clinical Improvement to Hospitalized COVID-19 Patients at High Risk for Rapid Clinical Deterioration and Acute Respiratory Distress Syndrome (ARDS). (clinicaltrials.gov) - P2; N=54; Active, not recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Recruiting ➔ Active, not recruiting; N=210 ➔ 54

Clinical • Enrollment change • Enrollment closed • Acute Respiratory Distress Syndrome • Cardiovascular • Heart Failure • Immunology • Infectious Disease • Inflammation • Myocardial Infarction • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • IL6 • TNFA

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC Pemziviptadil (PB1046) in Subjects With Symptomatic PAH (clinicaltrials.gov) - P2; N=63; Recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Active, not recruiting ➔ Recruiting

Clinical • Enrollment open • Hypertension • Pulmonary Arterial Hypertension

PhaseBio Provides Pemziviptadil (PB1046) Program Update (Businesswire) - P2, N=210; VANGARD (NCT04433546); Sponsor: PhaseBio Pharmaceuticals Inc.; "...after a strategic review of the VANGARD clinical trial...feedback from the U.S. Food and Drug Administration (FDA) and an interim analysis of the VANGARD study data, it has elected to discontinue the trial....FDA highlighted the likely need for additional clinical trials with sufficient sample size to adequately assess mortality risk....PhaseBio did not observe any data trends in the 40 mg or 100 mg pemziviptadil-treated arms of the trial that suggested a reasonable probability of achieving the primary efficacy endpoint....Pemziviptadil was generally well tolerated, and an independent data safety monitoring board did not identify any safety concerns related to pemziviptadil."

Discontinued • P2 data • Trial termination • Infectious Disease • Novel Coronavirus Disease

[VIRTUAL] LONG-ACTING VIP ANALOGUE PB1046 REDUCES DISEASE PROGRESSION AND CORRECTS F508DEL-CFTR FUNCTION IN C57BL/6 MICE LUNG AND DUODENUM (NACFC 2020) - "Support by PhaseBio Pharmaceuticals Inc. and Cystic Fibrosis Foundation."

Preclinical • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Inflammation • Respiratory Diseases

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC PB1046 in Subjects With Symptomatic PAH (clinicaltrials.gov) - P2; N=63; Active, not recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Hypertension • Pulmonary Arterial Hypertension

PhaseBio Doses First Patient in VANGARD Phase 2 Clinical Trial to Evaluate PB1046 for Hospitalized COVID-19 Patients (Businesswire) - "PhaseBio Pharmaceuticals...announced that it has dosed the first patient in VANGARD, a potentially pivotal Phase 2 clinical trial to evaluate PB1046....PhaseBio received FDA clearance in May to initiate the VANGARD trial and, subject to the pace of enrollment and any further impacts from the COVID-19 pandemic itself, the Company is targeting to report results late in the fourth quarter of 2020....Based on feedback from the FDA, PhaseBio believes that positive, clearly interpretable and clinically meaningful results from this trial may enable PhaseBio to submit a Biologics License Application."

P2 data • Trial status • Infectious Disease • Novel Coronavirus Disease

VANGARD: PB1046, a Long-acting, Sustained Release Human VIP Analogue, Intended to Provide Clinical Improvement to Hospitalized COVID-19 Patients at High Risk for Rapid Clinical Deterioration and Acute Respiratory Distress Syndrome (ARDS). (clinicaltrials.gov) - P2; N=210; Recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Acute Respiratory Distress Syndrome • Cardiovascular • Heart Failure • Immunology • Infectious Disease • Myocardial Infarction • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • IL6 • TNFA

VANGARD: PB1046, a Long-acting, Sustained Release Human VIP Analogue, Intended to Provide Clinical Improvement to Hospitalized COVID-19 Patients at High Risk for Rapid Clinical Deterioration and Acute Respiratory Distress Syndrome (ARDS). (clinicaltrials.gov) - P2; N=210; Not yet recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.

Clinical • New P2 trial • Acute Respiratory Distress Syndrome • Cardiovascular • Heart Failure • Immunology • Infectious Disease • Myocardial Infarction • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases

PhaseBio launches clinical trial to evaluate PB1046 as a treatment for hospitalized COVID-19 patients (Businesswire) - "PhaseBio Pharmaceuticals....announced FDA authorization to proceed with VANGARD, a potentially pivotal clinical trial to evaluate PB1046 as a treatment for hospitalized COVID-19 patients who are at high risk for rapid clinical deterioration and acute respiratory distress syndrome (ARDS)....Having received FDA clearance to initiate the VANGARD trial, PhaseBio expects to begin dosing patients by the end of June. Subject to the pace of enrollment and any further impacts from the COVID-19 pandemic, PhaseBio is targeting to report trial results late in the fourth quarter of 2020. Based on feedback from the FDA, PhaseBio believes that positive, clearly interpretable and clinically meaningful results from this trial may enable PhaseBio to submit a Biologics License Application."

Clinical data • IND • Trial initiation date • Infectious Disease • Novel Coronavirus Disease

PRE-CLINICAL EVALUATION OF VIP-ELASTINLIKE POLYPEPTIDES PB1046 AND PB1120 FOR THE TREATMENT OF CYSTIC FIBROSIS (NACFC 2018) - "Moreover, the molecules enhanced the effect of ivacaftor + lumacaftor. In vitro models indicate that combining PB1120 or PB1046 with ivacaftor and lumacaftor produces an additive positive effect on F508del-CFTR function and in vivo studies further demonstrate the benefit of a VIP-ELP treatment at the tissue level. Acknowledgment: Support by PhaseBio Pharmaceuticals Inc. "

Biosimilar • Cardiovascular • Cystic Fibrosis • Fibrosis • Heart Failure • Hypertension • Immunology

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC PB1046 in Subjects With Symptomatic PAH (clinicaltrials.gov) - P2; N=63; Recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Trial completion date: Jan 2020 ➔ Dec 2021; Trial primary completion date: Jan 2020 ➔ Dec 2021

Clinical • Trial completion date • Trial primary completion date

Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly Subcutaneous Injections of PB1046 (study drug) in Subjects With Symptomatic Pulmonary Arterial Hypertension (clinicaltrialsregister.eu) - P2; N=63; Ongoing; Sponsor: PhaseBio Pharmaceuticals, Inc.

Clinical • New P2 trial

A Study to Assess the Safety, Tolerability, and Hemodynamic Response of PB1046 in Subjects With PAH (clinicaltrials.gov) - P1; N=3; Completed; Sponsor: PhaseBio Pharmaceuticals Inc.; Active, not recruiting ➔ Completed; N=10 ➔ 3

Enrollment change • Trial completion

LTE: Long-Term Extension Study of PB1046 in PAH Subjects Having Completed Studies PB1046-PT-CL-0004 or PB1046-PT-CL-0005 (clinicaltrials.gov) - P2; N=64; Recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

A Study to Assess the Safety, Tolerability, and Hemodynamic Response of PB1046 in Subjects With PAH (clinicaltrials.gov) - P1; N=10; Active, not recruiting; Sponsor: PhaseBio Pharmaceuticals Inc.; Trial completion date: Jan 2019 ➔ Jun 2019; Trial primary completion date: Jan 2019 ➔ Jun 2019

Clinical • Trial completion date • Trial primary completion date