Eli Lilly and Company Joins cTAP, Helping to Accelerate Enriched Clinical Trial Design for the Provision of New Treatments for Duchenne Muscular Dystrophy Patients

Lilly to share data from Phase III trial of tadalafil in Duchenne,
increasing knowledge of placebo-arm responses

CAMBRIDGE, Mass.–(BUSINESS WIRE)–#CureDuchenne–Eli Lilly and Company, one of the top 15 global pharmaceutical
companies, has joined the Collaborative
Trajectory Analysis Project (cTAP), a unique partnership of many
stakeholders all committed to accelerating scientific discovery and
bringing new treatments to Duchenne Muscular Dystrophy (DMD) patients
more rapidly.

“Lilly is proud to support the efforts of cTAP, which is making a real
difference in our understanding of the progression of DMD, and how
variations across patients make it particularly difficult to design
effective clinical trials,” said Kraig Kinchen, M.D., senior medical
director of Lilly’s Bio-medicines Core Team. “cTAP is a model that
brings together the collective talent of a multi-stakeholder community.
We all share a common interest in speeding the development of
potentially effective therapies for Duchenne patients and their
families.”

Debra Miller, founder and CEO of CureDuchenne, a leading advocacy group
in the fight against Duchenne, said: “Advancing our scientific knowledge
of Duchenne and the way in which its progression varies across patients
is cTAP’s mission. CureDuchenne was the founding advocacy group
supporting cTAP and we are thrilled by Lilly’s contribution.”

“By sharing trial data, Lilly is showing real leadership,” added Pat
Furlong, founding president and CEO of Parent Project Muscular
Dystrophy. “PPMD has a long history with Lilly and their tadalafil
trial, and we believe the data collected from our community will be
valuable to the mission of cTAP.”

Duchenne Muscular Dystrophy is a progressive, fatal disease of boys
characterized by gradual weakening of muscles. It is the most common
fatal genetic disorder diagnosed in childhood. Most Duchenne patients
die in their 20s.

Though a Phase III human clinical trial of tadalafil, a
phosphodiesterase inhibitor, in Duchenne ultimately failed to meet its
targets for proving efficacy in slowing the decline in patients’ ability
to walk, the data generated by the trial are essential to expanding
scientific understanding of the rate of decline in boys with Duchenne.

cTAP has characterized the variable rates at which Duchenne patients
that exhibit similar symptoms often progress. This variation makes it
difficult to design clinical drug trials that can definitively prove the
efficacy of new treatments. Because Duchenne is a rare disease, with
only about 20,000 new cases worldwide each year, it is virtually
impossible to conduct large-scale trials with many patients, an approach
that can be used to overcome variability in more prevalent disorders.

“Through our collaboration with cTAP we have been able to create new
models that eliminate much of the statistical variation we see in
patients in clinical trials of experimental Duchenne treatments,” said
Professor Nathalie Goemans, head of the Neuromuscular Reference Center
for Children at the University Hospitals in Leuven, Belgium. “With these
new data from Lilly, we will be able to assess how well our predictive
tools – developed using natural history data – perform in the placebo
arm of a clinical trial for a Duchenne treatment. That’s why Lilly’s
participation is so critically important.”

About cTAP

The Collaborative Trajectory Analysis Project, or cTAP, is a novel group
enabling leading clinical experts to solve the most urgent problems in
Duchenne drug development. cTAP is a dynamic alliance of scientists,
drug companies, patient advocacy organizations, and registries and
clinical centers in DMD across Europe and the U.S. The collaboration
also brings leaders in biostatistical outcomes research to the fight
against Duchenne. cTAP is curating and growing what is already the
largest natural history database of DMD. This enables it to develop
near-term solutions for some of the key problems in designing clinical
trials and analyzing their results. To learn more, please visit ctap-duchenne.org.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is a uniformly fatal, progressive,
muscle-wasting disease affecting about one in 3,500-6,000 male live
births. Patients with Duchenne lack the ability to make dystrophin, a
protein crucial to muscle function. As their muscles deteriorate, they
progressively lose the ability to walk, feed themselves, turn over in
bed, and ultimately to breathe. While there is no cure, the past decade
has seen an explosion in research, resulting in more than 15 therapies
entering clinical development, with some receiving limited approval.
Learn more about Duchenne at cureduchenne.org
and parentprojectmd.org.

Contacts

cTAP
Susan J. Ward, Ph.D., +1-617-448-2617
susanjward@ctap-duchenne.org