SCIENCE + PIPELINE

BINDING OUR WAY TO THE TOP

THERAPEUTIC

POTENTIAL

Addressing up to 30 diseases

AL Amyloidosis patient from Phase 1

INNOVATIVE

IMAGING

Quantitative monitoring of the peptide radiotracer p5+14 may provide a rapid quantitative method for repeated evaluation and monitoring of response to therapy or disease progression in patients with amyloidosis. PET/CT imaging using this radiotracer may provide a rapid, first-line diagnostic reagent for quantitative monitoring of response to therapy in patients with diverse forms of systemic amyloidosis, including AL, ATTR and ALECT2.

Spleen & Kidneys

Heart

Liver

TECHNOLOGY PLATFORM: Development of a Synthetic Pan-Amyloid Reactive Peptide

Development of a technology that would

  1. tightly bind all types of amyloid

  2. utilize multiple binding sites

  3. remain highly specific

  4. be utilized for imaging, diagnostic, patient selection and endpoint purposes

  5. serve as a backbone for therapeutics

Heparin sulfate on all amyloid fibrils is structurally distinct from ubiquitous heparin sulfate proteoglycans in extracellular matrix and could be specifically targeted.

Screened natural peptides against amyloid heparin sulfate, leading to the creation of a synthetic version, peptide P5+14.

P5+14 met all goals

  1. tightly bind all types of amyloid

  2. utilize multiple binding sites

  3. remain highly specific

  4. be utilized for imaging, diagnostics, patient selection, and endpoint purposes

  5. serve as a backbone for therapeutics

Unique approach that overcomes challenge of development of a different therapeutic for each amyloid disease and challenge of binding to a single epitope.

ATTR patient from Phase 1

The first-in-man, Phase 1, study of p5+14 labeled with iodine-124 for PET/CT imaging of patients with systemic amyloidosis is currently underway (NCT03678259).

 

Initial image data (n=14) show 124I-p5+14 capable of detecting:

AL amyloid in the heart, liver, spleen, kidney, lung, adrenal, and pancreas. 

ATTR amyloid in the heart, nerves and connective tissue ALECT amyloid in the kidneys and liver

Sagittal View

Axial View

PEPTIDE TECHNOLOGY PLATFORM IS BASE TECHNOLOGY FOR IMAGING AND THERAPEUTICS

FC-PEPTIDE MULTI-AMYLOID OPSONIZING AGENT

BI-FUNCTIONAL PEPTOPE OPSONIZING AGENT

CHIMERIC ANTIGEN RECEPTOR (CAR)

CAR macrophage binds amyloid fibrils through receptors

Amyloid is taken up avidly by CAR macrophages

Car macrophages clear and destroy the tissue amyloid

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