Lilly Trial Finishes With Disappointing Results

Lilly has now completed one of the largest clinical trials in Duchenne (330 patients) with disappointing results. Today the company reports that their Phase 3 study of tadalafil failed to provide evidence for efficacy in slowing decline in walking ability in boys with Duchenne. Subsequently, the company has made the decision to discontinue the open-label extension. The full update to the community is below.


Sadly, this is one more blow for this community. We are deeply grateful to all of the families who participated – to the young men who committed to the study and their parents that did all that was necessary so that they could participate. This is indeed heartbreaking, especially on the heels of the drisapersen decision, the eteplirsen postponement, and the halt to the Akashi trial. But we will learn a great deal from the data collected in this trial, as the analysis continues – information that will inform future trials and future treatments.


Duchenne is extremely complex and difficult in so many ways. But we are grateful to companies like Lilly who continue to spend a tremendous amount of time and resources rigorously investigating therapies with the potential to help the people we love so much. The path is tumultuous but this community is strong and steadfast. And while this chemistry may not be part of the combination of therapies that ends up treating this disease, there are so many great minds and great companies working on a variety of approaches and chemistries to tackle Duchenne.


We are more confident than ever that we will end Duchenne. 

Update on Phase 3 Tadalafil Trial

We are writing to share disappointing news about our Phase 3 study of tadalafil in approximately 330 patients with Duchenne muscular dystrophy (DMD). We recently completed analysis of the initial data from the placebo controlled, double-blind period of the trial, and unfortunately did not see any evidence of efficacy for tadalafil to slow the decline in 6 Minute Walk Distance (6MWD) compared with placebo through 48 weeks. The study also included other secondary assessments of motor function, including the North Star Ambulatory Assessment and timed function tests (10 meter walk/run, rise from floor, and 4-stair climb), and there was no evidence of efficacy observed in these endpoints either. There also was no evidence of efficacy in subgroup analyses of the primary 6MWD endpoint in boys with differing disease severities at baseline. The complete efficacy and safety data from the trial will be reviewed over the coming weeks.

Because the trial provided no evidence that once-daily tadalafil treatment has a meaningful effect to slow disease progression compared with placebo, the open-label extension (OLE) phase of the study will be stopped. This decision was made in consultation with experts in DMD research on the trial Steering Committee, who agreed that stopping the OLE is in the best interest of all the patients in the study and their families. All investigators were informed of this decision late last week and provided with guidance on discontinuing the patients from the study.

Based on initial results, the overall adverse events were consistent with the known tadalafil safety profile and the DMD disease state. In a sub study of 27 boys who had a cardiac MRI, the average increase in left heart ventricle volumes was larger in the tadalafil groups relative to placebo. Other markers of heart function such as ejection fraction on echocardiogram were similar to placebo. Further analyses are ongoing with cardiology experts in the DMD community to understand the clinical interpretation and relevance of these findings.

We will be working closely with the patient advocacy community in the coming months to arrange a webinar to communicate the full results and to provide an opportunity for families to ask questions. We also will be submitting the study results for presentation at scientific and patient advocacy group meetings, and will submit a paper for publication of the full study results in a scientific peer-review journal.

We would like to sincerely thank all the families who participated in the study for their time and commitment. Obviously these are not the outcomes we had hoped for; however, they provide a definitive answer to an important and urgent question that was prompted by compelling pre-clinical and clinical data. We hope that the new data from this trial, which includes functional measures, quality of life, laboratory findings, measures of cardiac function, and muscle imaging data, can also be used to further advance the understanding of DMD and inform the design of future clinical trials.

Views: 3464


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Comment by Jason Darienzo on February 4, 2016 at 2:33pm

We need to encourage drug companies to continue extension studies beyond 48 weeks.  I believe the DMD community must reexamine short 48 week clinical trials. We are starting to see a pattern of failures here.  

It must be known if we are seeing a failure of the trial or the drug or we learn nothing.   

Need help using this community site? Visit Ning's Help Page.



© 2021   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service