Market Overview

Proclara Biosciences Announces Initiation of NPT189 Clinical Development for Systemic Amyloidoses and Provides Pipeline Update


-- First Subject Dosed in Phase 1a Trial of NPT189, Broadening
Clinical Pipeline with Next Generation GAIM-Based Therapy Targeting
Orphan Indications --

-- Enrollment Complete in Phase 1b Trial of NPT088 for Alzheimer's
Disease; Topline Data Expected 1Q19 --

Proclara Biosciences, a biotechnology company developing novel therapies
that utilize the general amyloid interaction motif (GAIM) to treat
disorders of protein misfolding, today announced the initiation of a
Phase 1a clinical trial of NPT189. This next-generation molecule is in
development for the treatment of systemic amyloidoses, which are a
family of rare diseases that include amyloid light chain (AL)
amyloidosis and hereditary transthyretin amyloidosis (ATTR).
The objective of the trial is to evaluate the safety and tolerability of
NPT189 in healthy volunteers.

Proclara also announced that it has completed enrollment in its ongoing
Phase 1b clinical trial of NPT088 for the treatment of Alzheimer's
disease. This randomized, double-blind, placebo-controlled study is
designed to evaluate the safety and tolerability of NPT088 and its
effect on Alzheimer's disease pathology as assessed by amyloid-β and tau
PET imaging in patients with mild-to-moderate Alzheimer's disease.
Topline data from this study is expected in the first quarter of 2019.

"We are pleased to announce the expansion of our clinical development
pipeline into orphan systemic diseases with NPT189, the second asset to
emerge from our GAIM-based platform," said Suzanne Bruhn, Ph.D.,
president and chief executive officer of Proclara. "This milestone,
together with the completion of enrollment in our Phase 1b study of
NPT088, marks an important step towards our goal of leveraging our
unique technology to address a range of diseases caused by protein

"Our team is eager to evaluate NPT189, a potentially important new
therapeutic, in patients with systemic amyloidoses, for whom no approved
disease-modifying treatments currently exist," said David Michelson,
M.D., chief medical officer of Proclara. "We also look forward to
obtaining the preliminary results from our Phase 1b study of NPT088 in
patients with Alzheimer's disease, and to the further progression of
these therapeutic candidates in clinical development."

About Systemic Amyloidoses

Systemic amyloidoses are life threatening diseases caused by the
systemic buildup of amyloid protein fibers in organs and nerves. Over
time, this build-up results in dysfunction that leads to significant
morbidity and increased mortality. Current treatments are limited and
new, more efficacious options are badly needed. Systemic amyloidoses are
classified according to the type of deposited misfolded protein. Amyloid
light chain (AL) amyloidosis is caused by an accumulation of misfolded
immunoglobulin protein resulting in vital organ dysfunction, with up to
4,000 new cases diagnosed in the U.S. each year.1 Hereditary
transthyretin (ATTR) amyloidosis, which is caused by mutant
transthyretin (TTR) amyloid deposits in multiple organs, causes
significant morbidity and mortality within two to 15 years from symptom
onset and affects approximately 50,000 people worldwide.2,3
Proclara is developing NPT189, which targets LC and TTR deposits, for
the treatment of these two orphan amyloidoses.

About Alzheimer's Disease

Alzheimer's disease is the most common neurodegenerative disease and the
leading cause of dementia. A progressive and irreversible brain disorder
that impacts memory and thinking skills, Alzheimer's often affects the
ability to carry out basic daily activities. The disease is
characterized by the buildup of misfolded protein aggregates in the
brain, including plaques, made of aggregated β-amyloid; tangles, made of
aggregated tau; and sometimes α-synuclein aggregates. These toxic
aggregates cause brain dysfunction and eventually, nerve cell death.
Alzheimer's affects approximately 47 million victims worldwide, and that
number is expected to grow to 132 million by 2050.4

About Proclara Biosciences

Proclara Biosciences is a biotechnology company advancing product
candidates developed based on its proprietary GAIM technology, which is
capable of simultaneously targeting multiple toxic misfolded proteins.
The broad applicability of GAIM technology enables the company to target
multiple protein misfolding diseases, including neurodegenerative
diseases and systemic amyloidoses. Proclara has developed a pipeline of
drug candidates that use GAIM to target the common amyloid protein
conformation, dissociating and preventing the formation of misfolded
protein assemblies, and blocking the cell-to-cell transmission of toxic
aggregates. No other single molecule or drug candidate displays such
broad potential therapeutic activity for mitigating pathologic
mechanisms in protein misfolding diseases. The company has two unique
GAIM fusion proteins in clinical development. NPT088 is being evaluated
in a Phase 1b trial for Alzheimer's disease; and NPT189 is currently in
Phase 1a in a clinical development program targeting systemic


1. Amyloidosis Research Consortium, Guidance for Industry: AL
Amyloidosis – Developing Drugs for Treatment, 2016 (

2. Ando et al., Orphanet J Rare Dis, 2013

3. Ruberg et al., Circulation, 2012

4. Alzheimer's Disease International, World Alzheimer Report, 2016, (

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