emprumapimod (ARRY-797) / Pfizer - LARVOL DELTA

REALM-DCM: A Phase 3, Multinational, Randomized, Placebo-Controlled Trial of ARRY-371797 in Patients With Symptomatic LMNA-Related Dilated Cardiomyopathy. (PubMed, Circ Heart Fail) - P2, P3 | "An unmet treatment need remains among patients with LMNA-related dilated cardiomyopathy. URL: https://classic.clinicaltrials.gov; Unique Identifiers: NCT03439514, NCT02057341, and NCT02351856."

Clinical • Journal • P3 data • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • LMNA

REALM-DCM: a phase 3, multinational, randomized, placebo-controlled study of ARRY-371797 (PF-07265803) in patients with symptomatic LMNA-related dilated cardiomyopathy (HEART FAILURE 2023) - "Findings from the terminated REALM-DCM trial confirm futility without safety concerns. An unmet treatment need remains among patients with LMNA-related DCM."

Clinical • P3 data • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • LMNA

Long-term effectiveness of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene: a plain language summary. (PubMed, Future Cardiol) - P2, P3 | "One such study (called REALM-DCM) was started in 2018 but ended early, as it was unlikely to show a clear treatment benefit of ARRY-371797. Phase 2 long-term extension study (NCT02351856) Phase 2 study (NCT02057341) Phase 3 REALM-DCM study (NCT03439514)."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • LMNA

Plain Language Summary of Publication of the safety and efficacy of ARRY-371797 in people with dilated cardiomyopathy and a faulty LMNA gene. (PubMed, Future Cardiol) - P2, P3 | "Overall, this study showed that people with LMNA-related DCM who took ARRY-371797 had improved functional capacity (exercise tolerance), improved heart function, and improved quality of life. Phase 2 study (NCT02057341) Phase 2 long-term extension study (NCT02351856) Phase 3 REALM-DCM study (NCT03439514)."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • LMNA

Efficacy and Safety of ARRY-371797 in LMNA-Related Dilated Cardiomyopathy: A Phase 2 Study. (PubMed, Circ Genom Precis Med) - P2 | "ARRY-371797 was well tolerated and resulted in increases in functional capacity and lower concentrations of cardiac biomarker NT-proBNP in patients with LMNA-related dilated cardiomyopathy."

Journal • P2 data • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • LMNA

REALM-DCM: A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3 | N=79 | Terminated | Sponsor: Pfizer | Trial completion date: Feb 2025 ➔ Oct 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Apr 2024 ➔ Oct 2022; The Sponsor terminated the study due to futility. The decision to stop the study was not based on safety concerns.

Trial completion date • Trial primary completion date • Trial termination • Cardiomyopathy • Cardiovascular • LMNA • NPPB

REALM-DCM: A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3 | N=78 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting | N=200 ➔ 78

Enrollment change • Enrollment closed • Cardiomyopathy • Cardiovascular • LMNA • NPPB

Long-Term Efficacy and Safety of ARRY-371797 (PF-07265803) in Patients With Lamin A/C-Related Dilated Cardiomyopathy. (PubMed, Am J Cardiol) - P2 | "The ongoing, pivotal, phase 3, randomized, placebo-controlled study REALM-DCM investigates the efficacy and safety of ARRY-371797 (PF-07265803) in LMNA-related DCM. (ClinicalTrials.gov Identifier: NCT02351856)."

Journal • Cardiomyopathy • Cardiovascular • LMNA

A Study To Evaluate The Pharmacokinetics, Excretion, Mass Balance and Metabolism of PF-07265803 (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Pfizer | Recruiting ➔ Completed

Trial completion

A Study To Evaluate The Pharmacokinetics, Excretion, Mass Balance and Metabolism of PF-07265803 (clinicaltrials.gov) - P1 | N=6 | Recruiting | Sponsor: Pfizer | Not yet recruiting ➔ Recruiting

Enrollment open

REALM-DCM: A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3 | N=200 | Recruiting | Sponsor: Pfizer | Trial completion date: Oct 2024 ➔ Feb 2025

Trial completion date • Cardiomyopathy • Cardiovascular • LMNA • NPPB

A Study To Evaluate The Pharmacokinetics, Excretion, Mass Balance and Metabolism of PF-07265803 (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Pfizer

New P1 trial

REALM-DCM: A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3 | N=160 | Recruiting | Sponsor: Pfizer | Trial completion date: May 2024 ➔ Oct 2024 | Trial primary completion date: Nov 2023 ➔ Apr 2024

Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular • LMNA • NPPB

[VIRTUAL] Long-Term Efficacy and Safety of ARRY-371797 (PF-0765803) in an Open-Label Rollover Study in Patients With Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (AHA 2021) - "ARRY-371797 was generally well tolerated, with evidence suggesting preserved exercise capacity over >2 years’ follow-up. Efficacy and safety of ARRY-371797 is being investigated in the REALM-DCM trial, an ongoing, pivotal, Phase 3, randomized, placebo-controlled study in LMNA-DCM."

Clinical • Cardiomyopathy • Cardiovascular • LMNA

A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy (clinicaltrials.gov) - P2; N=8; Completed; Sponsor: Pfizer; Trial primary completion date: Sep 2020 ➔ Dec 2020

Clinical • Trial primary completion date • Cardiomyopathy • Cardiovascular • LMNA

Realm-dcm (HFSA 2021) - P3 | "A Phase 3, Multinational, Randomized, Placebo-Controlled Study of ARRY-371797 (PF-07265803) in Patients with Symptomatic Dilated Cardiomyopathy due to a Lamin A/C Gene Mutation.REALM-DCM (NCT03439514) is a multinational, Phase 3 study that will evaluate the efficacy, safety, and pharmacokinetics (PK) of ARRY-371797 (PF-07265803) in at least 120 patients with New York Heart Association (NYHA) functional Class II and III dilated cardiomyopathy (DCM) due to lamin A/C gene (LMNA) mutations (LMNA-related DCM)...REALM-DCM is currently ongoing in over 60 centers in 9 plus countries and anticipated to complete in 2024. This is the first phase 3 study evaluating a treatment in LMNA-DCM patients."

Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Heart Failure • LMNA

REALM-DCM: A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3; N=160; Recruiting; Sponsor: Pfizer; Trial completion date: Dec 2024 ➔ May 2024; Trial primary completion date: May 2024 ➔ Nov 2023

Clinical • Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular • LMNA

A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3; N=160; Recruiting; Sponsor: Array BioPharma; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Cardiomyopathy • LMNA

A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3; N=160; Recruiting; Sponsor: Pfizer; Trial completion date: Jul 2020 ➔ Dec 2024; Trial primary completion date: Apr 2020 ➔ May 2024

Clinical • Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular • LMNA

A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (clinicaltrials.gov) - P3; N=160; Not yet recruiting; Sponsor: Array BioPharma

Clinical • New P3 trial • Cardiomyopathy • LMNA

A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy (clinicaltrials.gov) - P2; N=8; Completed; Sponsor: Pfizer; Active, not recruiting ➔ Completed

Clinical • Trial completion • Cardiomyopathy • Cardiovascular • LMNA

A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy (clinicaltrials.gov) - P2; N=8; Active, not recruiting; Sponsor: Pfizer; Trial completion date: Feb 2022 ➔ Nov 2020; Trial primary completion date: Feb 2022 ➔ Sep 2020

Clinical • Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular

Q4 & FY 2012 Results (Array Biopharma) - Anticipated partnership for further development and for commercialization for pain by Dec 2013

Anticipated licensing / partnership • Pain

Investor Presentation (Array Biopharma) - "ARRY-797 Provides Durable Pain Relief; Phase 2, 12-week study in ankylosing spondylitis patients who have inadequate response to continuous NSAIDs"

P2 data • Pain

Array BioPharma’s ARRY-797 meets primary endpoint in clinical proof of concept trial in osteoarthritis patients whose pain is poorly controlled by NSAIDs (Businesswire) - P2, N=157; Patients on ARRY-797 had a mean reduction in WOMAC pain subscale score vs. placebo at day 28 (2.4 vs. 1.6) and was comparable to oxycodone ER; Patients on active control arm (oxycodone ER) had higher discontinuation rate (34%) due to adverse event vs. ARRY-797 (6%) or placebo (8%); In other ongoing multiple dose ascending trial in healthy volunteers, ARRY-797 was well tolerated till date and greater QTc prolongations reported at higher dose levels warranting further evaluation; Array intended to seek partner for further development of ARRY-797

Anticipated licensing / partnership • P1 data • P2 data • Pain