• Challenge 79 for World Duchenne Awareness Day
  • About Us
    • Our vision
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Mental Health Awareness Week 2025
    • Science on Tour
    • Support Calendar – What’s On
    • Support for you and your family
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Connect with others
    • Support for 8-14 yrs ‘Turning Point’
    • Support for 14-25 yrs ‘Yes I Can’
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • SAVE THE DATE for the Action Duchenne Annual International Conference 2025
    • Highlights from the Annual Action Duchenne Annual International 2024
    • Annual International Conference 2023 Video Recordings
    • Annual International Conference 2022 Recordings
      • Adults with Duchenne
      • Growing up with Duchenne
      • The Duchenne Journey
      • What is new in Duchenne research?
  • News, Webinars and Blogs
    • News
    • Webinar Series 2025
      • Webinar Series 2025
      • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Blogs
  • Support Us
    • Help Make a Life Beyond Duchenne Possible – Every Month
    • Fundraising Events and Challenges
    • Take on a challenge for Duchenne
    • Shop
  •  0 items - Free
  • Menu
  • Skip to right header navigation
  • Skip to main content
  • Skip to secondary navigation
  • Skip to primary sidebar
  • Skip to footer

Before Header

  • BECOME A MEMBER
  • SHOP
  • My account
  •  0 items - Free

Action Duchenne

Header Right

  • Challenge 79 for World Duchenne Awareness Day
  • About Us
    • Our vision
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Mental Health Awareness Week 2025
    • Science on Tour
    • Support Calendar – What’s On
    • Support for you and your family
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Connect with others
    • Support for 8-14 yrs ‘Turning Point’
    • Support for 14-25 yrs ‘Yes I Can’
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • SAVE THE DATE for the Action Duchenne Annual International Conference 2025
    • Highlights from the Annual Action Duchenne Annual International 2024
    • Annual International Conference 2023 Video Recordings
    • Annual International Conference 2022 Recordings
      • Adults with Duchenne
      • Growing up with Duchenne
      • The Duchenne Journey
      • What is new in Duchenne research?
  • News, Webinars and Blogs
    • News
    • Webinar Series 2025
      • Webinar Series 2025
      • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Blogs
  • Support Us
    • Help Make a Life Beyond Duchenne Possible – Every Month
    • Fundraising Events and Challenges
    • Take on a challenge for Duchenne
    • Shop
You are here: Home / News / Pharmaceutical Companies Working in Duchenne Report Q2 Financial Results and Provide Business Updates

Pharmaceutical Companies Working in Duchenne Report Q2 Financial Results and Provide Business Updates

August 14, 2017 by abzali123

A number of pharmaceutical and biotech companies working in the field of Duchenne muscular dystrophy, have recently reported their 2nd quarter financial results and provided updates on their business activities and programmes.

First to update the community was PTC Therapeutics on 8 August.

Since our founding nearly 20 years ago, it has been our mission to provide treatments to patients living with rare diseases who have limited treatment options, said Stuart W. Peltz, CEO, PTC Therapeutics. I am proud to have introduced Translarna, the first therapy for nonsense mutation Duchenne muscular dystrophy patients and we remain dedicated to working with patients and their families to bring Translarna to the U.S…”

A couple of the key highlights from their report were:

  • Translarna net product sales were $45.8 million for the second quarter of 2017, representing 197% growth over $15.4 million reported in the second quarter of 2016.
  • Total revenues for the second quarter of 2017 were $48.0 million compared to $15.6 million in the same period of 2016. The change in total revenue was a result of the expanded commercial launch of Translarna and the successful U.S. EMFLAZA launch.

PTC Therapeutics’ full press release can be found here.

Wave Life Sciences released their update the following day.

Paul Bolno, President and CEO of Wave Life Sciences: “Our lead DMD Exon 51 programme is on track to enter the clinic later this year… Our collaboration with Pfizer continues to advance with multiple programmes progressing through in-vivo lead optimisation… With the progression of our pipeline and advancement in our platform capabilities, we are closer to potentially bringing new hope and precision medicines to patients living with serious, genetically-defined diseases.”

The key highlight relating to Duchenne was that Wave’s lead programme for Duchenne (called ‘WVE-210201’), is expected to enter the clinic in the second half of 2017 as planned.

Wave presented additional pre-clinical data at the PPMD conference; these data supported previously reported pre-clinical findings, and demonstrated efficient exon 51 skipping, dystrophin protein restoration and high tissue stability.

Wave Life Sciences full update is available here.

Yesterday Capricor Therapeutics posted their Q2 results and a summary of their programmes.

“In April, we reported positive interim results from our ongoing HOPE-Duchenne clinical trial… These results are particularly notable given the patients’ advanced disease as approximately 70% of trial participants were wheelchair-dependent at entry,” said Linda Marban, Ph.D., president and CEO of Capricor.

“A key finding in HOPE-Duchenne was that, as compared to usual care controls, patients treated with CAP-1002 demonstrated improvement in skeletal muscle function according to the Performance of the Upper Limb (PUL) test, a validated instrument… we are preparing to conduct our next clinical trial in DMD, which will evaluate the ability of intravenous CAP-1002 to improve skeletal muscle function as measured by the PUL. We expect this to commence in the fourth quarter of this year, subject to regulatory approval.”

Among other information Capricor highlighted:

  • the FDA’s willingness to accept the Performance of the Upper Limb (PUL) as the basis for the primary efficacy endpoint for clinical studies intended to support a Biologics License Application
  • (BLA) for CAP-1002 in the DMD indication.
    that CAP-1002 had been granted Rare Pediatric Disease Designation by the FDA.

You can find out more via Capricor’s press release here.

Also on 10 August, Catabasis Pharmaceuticals announced their Q2 results and business activities.

Jill Milne, CEO of Catabasis:

“In the second quarter, we presented an important prespecified crossover analysis of data from boys with Duchenne in our Phase 2 edasalonexent trial. We are very excited to see improvements in the rate of decline of muscle function across multiple assessments in boys treated with edasalonexent for 12 weeks.

The results are very consistent with and support our earlier analysis of functional assessments in boys treated with edasalonexent for 12 weeks as compared to placebo and further strengthen our confidence in the potential of edasalonexent as a novel treatment for DMD. We continue to advance the open-label extension and expect to share 24-week edasalonexent results in the third quarter as well as announce our Phase 3 clinical trial plan for edasalonexent in the second half of 2017…”

The Catabasis update highlights the following regards their work in Duchenne:

  • The open-label extension of the MoveDMD trial is progressing as planned and results from 24 weeks of edasalonexent treatment are expected to be announced in the third quarter of 2017. All boys participating in the open-label extension have now moved to the higher 100 mg/kg/day edasalonexent treatment group. Pending IRB approval, the open-label extension will be extended for an additional 52 weeks so that participating boys can continue to receive edasalonexent.
  • Catabasis expects to announce the Phase 3 clinical trial plan for edasalonexent in DMD in the second half of 2017.

You can find out more by reading the full press release here. In addition Catabasis have released the latest version of their MoveDMD trial newsletter. This provides a helpful summary of their research in Duchenne so far.

As always the best place in the UK to get updates on all aspects of Duchenne is the annual Action Duchenne International Conference. This year the event is 10-12 November at the Hilton Birmingham Metropole.

Share this:

Category: NewsTag: untagged

Previous Post: « Worldwide licence for new drug SOMO266 as SOM Biotech expands to the United States
Next Post: Action Duchenne invites the participation of the Becker muscular dystrophy community in November! »

Primary Sidebar

From our community

The Heart of Care

The Heart of Care We have had some key reminders of what we are working towards as a charity over the last few weeks. Volunteer’s Week (2nd – 8th June) was a chance to thank all of the people who give up their time and expertise for Action Duchenne. From the team of trustees, those …

Mental Health Awareness Week: Alex’s Journal

Written By Alex Berbank 15 Minutes a Day I’ve really enjoyed getting into the frame of mind to look at my mental health. Before this week I wasn’t quite sure what to expect. My ideas of what looking at my own mental health would look like weren’t accurate. I thought there would be more softly …

Mental Health Awareness Week: Alex’s Journal

Mental Health Awareness Week Journal, Written by Alex Berbank Watch Alex’s Vlog for Mental Health Awareness Week Thursday’s Update: Meditation and Mindfulness So, to this point this week has been great. I’ve actually taken to the focus on my mental health and self improvement thing quite well and I hope I’ll keep these new ideas …

Footer

Action Duchenne
Wellesley House
Duke of Wellington Avenue Royal Arsenal
London
SE18 6SS

07535 498 506
info@actionduchenne.org 

 

 

 

 

 

 

 

 

Subscribe to our mailing list

Do you consent to receiving regular email updates? *
Email Format
  • Accessibility
  • Privacy Policy
  • Terms & Conditions

© Action Duchenne - Registered Charity No 1101971 - Scottish Charity No SC043852

Like most websites we use cookies to deliver a personalised service. To use the website as intended please accept cookies.
Privacy & Cookies Policy

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Non-necessary
Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. It is mandatory to procure user consent prior to running these cookies on your website.
SAVE & ACCEPT