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Science & Pipeline

Genvara specializes in identifying common genetic variation that can be therapeutically targeted (“pharmacogenetics”) in cardiovascular disorders. Our basic and clinical research programs focus on human cardiac systems in order to provide a direct path from discovery to clinical application, utilizing our decades-long experience in molecular genetics, molecular pharmacology, clinical trials and drug development. Our pharmacogenetic, precision therapeutic approach, on which Genvara was founded, is to target haplotypes that contain multiple polymorphisms in protein drug targets, since it is protein haplotypes that dictate function. This has long been recommended as the preferred approach to pharmacogenetics, but has yet to be realized. We are poised to embark on a single, modest size (N=350) Phase 3 trial with our lead compound (Gencaro™) that targets a combination of β1- and β2-adrenergic receptor haplotypes, which based on extensive interactions with FDA will be the basis for approval in the indication of prevention of atrial fibrillation in patients with heart failure.

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Genvara Late-Stage Pipeline

Treating Serious Medical Disorders with High Unmet Need

Indication
Research/ Preclinical
Phase 1
Phase 2
Phase 3

GENCARO (bucindolol hydrochloride)

AF* in HFmprEF Primary Indication
PRECISION-AF Pivotal Trial - Efficacy Cohort (300 patients)
Genotype-defined population with mildly reduced to preserved LVEF (40 - 54%)
AF* in HFpEF Label Expansion
PRECISION-AF Exploratory Cohort (50 patients) for Label Expansion
Genotype-defined population with preserved LVEF (55 - 64%)
AF* in HFmprEF Label Expansion
PRECISION-AF Adjunctive Cohort (75 patients) for Label Expansion - NIH Grant-Funded
Haplotype-defined population, LVEF 40 - 54%
AF* in HFrEF Label Expansion
Expected Labeling if PRECISION-AF is Positive
Genotype-defined population, LVEF < 40%
Treatment of HFrEF Label Expansion
Expected Labeling if PRECISION-AF is Positive
Genotype-defined population, LVEF < 40%

rNAPC2 (recombinant nematode anticoagulant protein C2)

Autoimmune Diseases (Orphan Indications)
Treatment of Antiphospholipid Antibody Syndrome (APS) and/or
Systemic Lupus Erythematosus (SLE) Flare

* Includes AF prevention and rate control

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