Research
Two New Parkinson’s
Drugs Approaching
Market
(continued from cover)
DONOR-RAISED CAPITAL
DRIVES PATIENT-
RELEVANT RESULTS
MJFF takes no IP position on
the research it funds, evaluating
ROI exclusively on the basis
of potential to better address
patients’ unmet medical needs.
Foundation grants of $1.3 million
and $1.5 million (respectively)
funded early clinical trials of the
inhaled levodopa and dissolvable
apomorphine, and helped
drugmakers secure hundreds of
millions of dollars in additional
investment to push the therapies
through Phase II and Phase III
clinical trials. Both are “rescue
therapies,” designed to rapidly
alleviate movement symptoms
including tremor, stiffness and
slowness during “off” times.
The inhaled levodopa, to be
marketed as “Inbrija,” was
developed by biotech Civitas
Therapeutics, which received
MJFF grants in 2011 and 2013.
After promising early results
helped the company raise
three rounds of capital totaling
$121 million Civitas was
acquired by biopharmaceutical
company Acorda for $525
million in 2014.
In a similar chain of events,
biotech Cynapsus Therapeutics
received MJFF grants for early
trials of its apomorphine strip
in 2012 and 2014. It went on
to raise $99 million in follow-
on funding (including its
2015 IPO) and was acquired
by pharmaceutical company
Sunovion for $624 million
in 2016.
DE-RISKING MOVES THE
FIELD CLOSER TO CURES
It is no exaggeration to say
that since Michael started the
Foundation, MJFF funding
of more than $800 million
in research has transformed
Parkinson’s drug development.
Today, Parkinson’s research
represents tremendous
opportunity to many companies
and investors, and the pipeline is
brimming with novel therapeutic
approaches in development.
MJFF support is propelling
new treatments for Parkinson’s
symptoms, as well as therapies
that may slow, stop or even
prevent the disease. A rich
portfolio of strategies is taking
aim at leading genetic targets:
+ San Francisco biotech Denali
Therapeutics announced
promising results from its
Phase I clinical study of LRRK2
in healthy volunteers, and is
now planning a Phase II trial in
Parkinson’s patients.
MJFF support also enables
research on treatments approved
for other conditions that show
promise in Parkinson’s disease.
After we supported Phase II
trials of isradipine (a high blood
pressure medication) and inosine
(an antioxidant), the National
Institutes of Health is funding
ongoing Phase III trials to test
these drugs’ efficacy in slowing
Parkinson’s disease.
MJFF'S ROLE EVOLVES
WITH ROBUST PIPELINE
While we continue to fund
specific therapies, our non-
financial resources are de-risking
Parkinson’s research as a whole,
attracting scientists and drug
developers with the tools to
pursue new therapies.
Perhaps MJFF’s most valued
contribution beyond funding
is galvanizing a community
of research volunteers. Our
Foundation is gathering critical
information to help researchers
better understand Parkinson’s
+ Seven alpha-synuclein therapies disease and design better drug
are now in clinical trials (three trials. Through large-scale
studies such as the Parkinson’s
received early grants from
Progression Markers Initiative
MJFF). After Foundation
and the online Fox Insight, MJFF
grants totaling $740,000, San
is creating rich troves of data
Diego biotech Neuropore
signed a deal worth up to $460 on patients and their unique
million with pharma firm UCB experience with Parkinson’s.
Companies are using this data —
to continue developing its
all de-identified and available to
therapeutic pipeline.
qualified researchers — to learn
+ Cambridge biotech Lysosomal
more about whom to enroll in
Therapeutics received
their studies and how to measure
MJFF funding of $230,000
success, for example.
to develop a drug against
We are also providing accessible
GBA dysfunction, which is
laboratory tools for Parkinson’s
associated with GBA gene
mutations, the most common research, along with study
recruitment materials and support.
known genetic cause of
These “therapeutic enabling”
Parkinson’s. Allergan paid
resources not only help companies
for the right to acquire
execute their studies, but also
Lysosomal Therapeutics
help tilt the odds in favor of their
after Phase I trials of its drug.
decision to pursue Parkinson’s
The companies are currently
drug development.
analyzing trial data.
2
The Fox Focus