Civitas Therapeutics, Inc., a biopharmaceutical company developing
transformative therapeutics using the ARCUS® respiratory
delivery platform, today announced the initiation of a Phase 2b clinical
study of CVT-301, an inhaled formulation of levodopa (L-dopa). CVT-301
is being developed as an adjunct, as needed (PRN) therapy that will
provide rapid and reliable relief from intermittent debilitating motor
fluctuations (OFF episodes) that affect many Parkinson’s disease
patients. This Phase 2 study will evaluate the efficacy and safety of
CVT-301 in treating emergent OFF episodes during one month of continued

The Phase 2b study follows a successful Phase 2a study for CVT-301,
which demonstrated that the administration of CVT-301 to patients in the
OFF state produced a rapid and durable improvement in motor function.
The pharmacokinetic data recapitulated the Phase 1 study results showing
CVT-301 provided immediate L-dopa absorption and consistent increases in
plasma concentrations in marked contrast to the delayed and variable
L-dopa levels seen with Sinemet® (oral L-dopa/carbidopa). All doses of
CVT-301 were safe and well tolerated with no increase in the frequency
or severity of dyskinesias relative to oral.

“We are excited to move to this next stage of development in which
patients will self-administer CVT-301 upon the emergence of their OFF
symptoms as they go about their normal daily activities, exactly aligned
with the intended treatment paradigm,” said Martin Freed, M.D., Chief
Medical Officer of Civitas. “The ease of use of the simple ARCUS®
inhaler allows patients to use CVT-301 wherever and whenever their oral
Parkinson’s medications begin to fail them in between their regularly
scheduled doses.”

This Phase 2b study of CVT-301 is funded in part by a grant from The
Michael J. Fox Foundation for Parkinson’s Research.

About the Phase 2b Study (CVT-301-003)

CVT-301-003 is a randomized, double blind, placebo controlled, parallel
group trial that is being conducted in the United States and Europe.
Patients will use CVT-301 to treat emerging OFF episodes over a 28 day
study period. The study is intended to test the efficacy, safety and
tolerability of CVT-301 in the treatment of intermittent OFF episodes.
Objective motor responses will be evaluated during regularly schedule
clinic visits using the Unified Parkinson’s Disease Rating Scale (UPDRS)
Part 3. In addition, efficacy will also be evaluated during out-patient
use with diary based outcomes measures. Eighty (80) patients will be
enrolled in the study. The company will report preliminary data from
this study in the first half of 2014.

About CVT-301

Civitas’ lead program, CVT-301, is an inhaled formulation of L-dopa
being developed for the rapid and reliable relief from debilitating
motor fluctuations (OFF episodes) associated with Parkinson’s disease.
For chronic symptomatic management, oral L-dopa is administered to
maintain dopamine levels in the brain above the therapeutic threshold;
yet the reliability of oral L-dopa is significantly compromised by
delayed and unpredictable absorption and excessive variability in the
circulating plasma drug concentrations inherent to the oral delivery
route. Oral L-dopa remains widely recognized as the most efficacious
treatment of Parkinson’s disease symptoms in spite of this intrinsic
unreliability and the resulting OFF episodes. CVT-301 is being developed
as an adjunct PRN therapy to standard oral L-dopa therapy to address the
OFF episodes as they emerge and enable patients to reliably manage their
symptoms. CVT-301 is an ARCUS® therapeutic that incorporates
L-dopa and is optimized to deliver a precise dose to the deep lung for
rapid and predictable L-dopa absorption. The ARCUS® platform
is uniquely able to deliver the necessary L-dopa dose with the required
precision. A Phase 1 study in healthy volunteers showed that CVT-301
rapidly achieved target L-dopa plasma levels with a pharmacokinetic (PK)
profile supportive of its therapeutic potential. The Phase 2a double
blind placebo controlled dose finding study (CVT-301-002) recapitulated
the PK profile in patients, produced rapid and durable improvement in
motor function when administered to patients in the OFF state, and was
generally safe and well tolerated at all doses tested.

About Parkinson’s Disease

Over one million people in the US and six million people worldwide
suffer from Parkinson’s disease, a neurodegenerative disorder caused by
the diminished production of dopamine, resulting in progressive
impairment of motor function including tremors, rigidity, and difficulty
in moving. The unreliability of available medications for symptomatic
treatment of Parkinson’s disease remains a significant unmet need. Even
when treated with the current standard of care, the majority of
Parkinson’s patients continue to experience motor fluctuations. These
unpredictable OFF episodes reduce patients’ ability to lead productive,
independent lives and are recognized by patients, care givers, and
healthcare professionals as one of the most troubling and debilitating
issues associated with the disease.

About ARCUS®Platform

The ARCUS(R) platform is a proprietary dry powder and device
combination with a unique ability to deliver a large, precise dose
independent of inspiratory flow rate from a simple, breath actuated
device. The platform is protected by a large intellectual property
estate including over 130 issued patents. The technology has
successfully delivered more than one million doses to patients and the
manufacturing technology has been scaled to accommodate a significant
commercial launch.

About Civitas Therapeutics

Civitas is a privately-held biopharmaceutical company focused on
developing a robust pipeline of inhaled therapeutics with the clinically
proven ARCUS® dry powder pulmonary delivery platform. In
addition to the lead program, CVT-301 for treating Parkinson’s disease,
other programs encompass respiratory disease, central nervous system
disorders, and infectious disease. The company is headquartered in
Chelsea, Mass. in a facility that includes both development and
commercial scale GMP manufacturing capabilities. The Company is financed
by leading investors including Canaan Partners, Fountain Healthcare
Partners, Longitude Capital and Alkermes plc. For further information on
Civitas, please visit

About The Michael J. Fox Foundation for Parkinson’s Research

As the world’s largest private funder of Parkinson’s research, The
Michael J. Fox Foundation is dedicated to accelerating a cure for
Parkinson’s disease and improved therapies for those living with the
condition today. The Foundation pursues its goals through an
aggressively funded, highly targeted research program coupled with
active global engagement of scientists, Parkinson’s patients, business
leaders, clinical trial participants, donors and volunteers. In addition
to funding more than $350 million in research to date, the Foundation
has fundamentally altered the trajectory of progress toward a cure.
Operating at the hub of worldwide Parkinson’s research, the Foundation
forges groundbreaking collaborations with industry leaders, academic
scientists and government research funders; increases the flow of
participants into Parkinson’s disease clinical trials with its online
tool, Fox Trial Finder; promotes Parkinson’s awareness through
high-profile advocacy, events and outreach; and coordinates the
grassroots involvement of thousands of Team Fox members around the world.

Civitas Therapeutics
Stephanie Gillis, 617-660-4121
[email protected]
Suda Communications LLC
Maureen L. Suda (Media), 585-387-9248