U.S. Food and Drug Administration and European Medicines Agency Reach Landmark Decisions on Critical Path Institute's Clinical

  U.S. Food and Drug Administration and European Medicines Agency Reach
  Landmark Decisions on Critical Path Institute's Clinical Trial Simulation
  Tool for Alzheimer’s Disease

Business Wire

TUCSON, Ariz. -- July 10, 2013

In a big step forward for Alzheimer’s disease (AD) therapy development, both
the U.S. Food and Drug Administration (FDA) and the European Medicines Agency
(EMA) have independently reached favorable decisions on the value of Critical
Path Institute’s new disease simulation tool for improving trial design in
mild and moderate Alzheimer’s disease. The first such instrument to ever
receive this regulatory designation, the tool represents an enabling advance
to improve the design of future clinical trials in AD. The new tool applies
computerized models to simulate “what-if” scenarios for clinical trials. The
goal of this virtual tool is to serve as a public resource for sponsors
designing trials of new therapies for AD.

On June 12, 2013, the FDA issued a regulatory letter to Critical Path
Institute’s (C-Path) consortium, the Coalition Against Major Diseases (CAMD).
It stated the Agency’s decision to deem CAMD’s quantitative clinical trial
simulation tool a “fit-for-purpose” drug development tool for AD. “Model-based
drug development was one of the goals defined in FDA’s 2004 Critical Path
Initiative report, and this new tool sets the stage for applying new
technologies to accelerating medical product development,” said Janet
Woodcock, MD, director of the Center for Drug Evaluation and Research (CDER)
at FDA.

On June 27, 2013, EMA's Committee on Human Medicinal Products (CHMP) gave the
opinion that “the proposed Disease Progression and Trial Evaluation Model is
suitable for use in drug development as a longitudinal model for describing
changes in cognition in patients with mild and moderate AD, and for use in
trial designs in mild and moderate AD.” The CHMP is the EMA committee
responsible for preparing opinions on questions concerning medicines for human
use.

This new tool will make it possible to simulate clinical trials by integrating
all relevant data so future studies will be more efficient and more likely to
be successful. A basic explanation of clinical trial simulation tools can be
found at http://vimeo.com/64332443.

Richard Lalonde, PharmD, Vice President and Global Head of Clinical
Pharmacology at Pfizer, stated, “This model was made possible because major
pharmaceutical companies participating in CAMD were willing to share
de-identified patient-level data for over 6,000 patients who previously
participated in AD trials. The data from these trials were the basis for this
model, and the FDA’s decision on this tool will allow sponsors to apply
modeling and simulation and launch AD trials with a higher degree of
confidence. This is a great example of a rising tide lifting all boats.”

This effort is testament to the power of stakeholders working together in a
non-competitive fashion to pool data and develop methods that will benefit the
public health.

“Alzheimer’s disease clinical development tools that are derived from
real-world findings, and take into account a variety of factors, help to
increase our confidence that a clinical study is accurately designed to detect
a treatment effect,” said Richard Mohs, PhD, Vice President, Early-Phase
Neuroscience Clinical Research Eli Lilly and Company and member of the CAMD
coordinating committee. “This model provides valuable insight into the
relatively slow rate of disease progression in a mild patient population – a
critical area of focus for drug development – and guidance for designing an
effective study in multiple populations, while emphasizing the need to refine
the model as we work to treat patients earlier in the disease process and as
new data emerges.”

Martha Brumfield, PhD, President and CEO of C-Path, stated, “The regulatory
decisions on this tool exemplify how C-Path's efforts result in alignment
between global regulatory agencies when based on consensus science and
supporting data; an alignment thatcan result ingreater efficiencyindrug
development.This could not have been accomplished by any one entity working
in isolation.”

The patient community, as active CAMD members, have played a role in the
consortium since its inception. Eric Sokol, Director of Alzheimer’s Foundation
of America stated that “patients and caregivers should be reassured by the
positive decision on this new simulation tool that they can now participate in
more efficiently designed clinical trials.”

ABOUT CRITICAL PATH INSTITUTE (C‐PATH): An independent, non-profit
organization established in 2005 with public and private philanthropic support
from the Arizona community, Science Foundation Arizona (SFAz), and the U.S.
Food and Drug Administration (FDA), C-Path is committed to improving health
and saving lives by accelerating the development of safe, effective medicines.
An international leader in forming collaborations around this mission, C-Path
has established global, public-private partnerships that currently include
over 1,000 scientists from government regulatory agencies, academia, patient
advocacy organizations, and thirty-five major pharmaceutical companies. C-Path
is headquartered in Tucson, Arizona. For more information, visit
www.c-path.org.

ABOUT THE COALITION AGAINST MAJOR DISEASES (CAMD): The mission of CAMD
(http://www.c-path.org/camd.cfm) is to accelerate the development of therapies
for neurodegenerative diseases by advancing drug development tools for
regulatory approval. The consortium currently consisting of over 150
scientists from pharmaceutical companies, contract research organizations,
regulatory agencies, patient advocacy organizations, academic institutions and
several government agencies.

Contact:

Critical Path Institute
Allison Schott, 520-795-4500
pr@cpath.org