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.

Capricor edit

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.