Duchenne Muscular Dystrophy: KOL Insight [2018]

How are gene therapies expected to revolutionise the treatment of Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is a devastating and fatal neuromuscular genetic disease with no known cure and few treatment options. Since 2014, two antisense oligonucleotide therapies [Exondys 51 (Sarepta Therapeutics) and Translarna (PTC Therapeutics)] have been approved but how are they faring on the market? Find out how key opinion leaders (KOLs) perceive these therapies amongst other conventional treatment approaches. The pipeline for DMD encompasses many novel therapies with diverse mechanisms of action. Find out how the KOLs weigh up key therapy classes including novel exon-skipping compounds, adenoviral-based microdystrophin/minidystrophin gene therapies, myostatin inhibitors, cell therapies amongst others. Which therapeutic strategy do the KOLs think will offer the most promise for this life-threatening condition and how will they impact future treatment pathways?

Learn how KOLs see the market evolving, and how developers could differentiate their marketed and pipeline therapies in KOL Insight: Duchenne Muscular Dystrophy [2018]. Ten of the world’s foremost KOLs from the US and Europe provide their candid insights on three marketed products and 16 late- and early-stage pipeline programmes.

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• The table of contents >
• The key business questions answered >
• The key KOL quotes >
• See the therapies covered >
• Find out who the 5 EU & 5 US KOLs are >
• Review an extract from the report - 1 drug profile >

• What improvements are needed for the conventional symptomatic treatment of DMD? How do KOLs currently manage DMD and can novel compounds, such as ReveraGen’s vamorolone or Catabasis’ edasalonexent, offer an improved side effect profile or quality of life for patients?
• How are Exondys 51 and Translarna being used in the treatment of DMD? Since their approval in specific patient subtypes,KOLs weigh up each drug based on mechanism of action, clinical efficacy, safety, administration and discuss their future role in DMD.
• Is there future opportunity for novel exon-skipping strategies? Find out how the KOLs perceive other pipeline exon-skipping drugs such as golodirsen/casimersen (Sarepta) NS-065 (Nippon Shinyaku) and next-generation platforms by Sarepta and Wave Life Sciences.
• Can microdystrophin/minidystrophin gene therapies revolutionise future treatment of DMD? How do KOLs view the initial data with Sarepta/Nationwide’s GALGT2 gene therapy and what are their expectations for other gene therapies from Solid BioSciences and Pfizer.
• What opportunities and challenges face adenoviral-based gene therapies going forward? KOLs provide their candid insights on the hurdles that face adenoviral gene therapies but also discuss their future potential as a broad therapeutic approach.
• How do KOLs view myostatin inhibition as a therapeutic strategy in DMD? Bristol-Myers Squibb (BMS)/Roche and Pfizer are developing anti-myostatin compounds for DMD.Find out how the experts view this approach and where they would be best incorporated as a treatment option.
• Do cell-based therapies have any future role in DMD? Many stem cell therapies have led to disappointments in the past but what are KOL viewpoints on Capricor’s CAP-1002?
• How will future treatment pathways evolve for the treatment of DMD? Find out if exon-skipping drugs, microdystrophin/minidystrophin gene therapies, myostatin inhibitors, or combination strategies are expected to play an important role and whether they can help address the pathogenesis of this fatal disease.

“The way the DMD field is moving is in terms of precision medicine and gene-based approaches. I think gene-based therapy targets are going to really play a big and a critical role.”
US Key Opinion Leader

“It's likely that we will be moving from the Duchenne we know now to a modified and hopefully, a significantly milder phenotype but not a complete cure. I would put gene therapy as the one at the top of the list at least in the short-medium term.”
US Key Opinion Leader

Marketed Therapies

• Emflaza (deflazacort; PTC Therapeutics)
• Translarna (ataluren/PTC124; PTC Therapeutics)
• Exondys 51 (eteplirsen; Sarepta Therapeutics)

Pipeline Therapies

• vamorolone (VBP-15; ReveraGen BioPharma)
• edasalonexent (CAT-1004; Catabasis Pharmaceuticals/Sarepta Therapeutics)
• idebenone (Raxone; Santhera Pharmaceuticals)
• golodirsen (SRP-4053; Sarepta Therapeutics)
• casimersen (SRP-4045; Sarepta Therapeutics)
• SRP-5051 (Sarepta Therapeutics)
• NS-065/NCNP-01 (NS Pharma/Nippon Shinyaku)
• WVE-210201 (Wave Life Sciences)
• GALGT2 gene therapy/rAAVrh74.MCK.GALGT2 (Sarepta Therapeutics/Nationwide Children’s Hospital)
• SGT-001 (Solid Biosciences)
• PF-06939926 (Pfizer)
• AAV1-FS344 (Milo Biotechnology/Nationwide Children’s Hospital)
• talditercept alfa (RG6206/BMS 986089; BMS/Roche)
• domagrozumab (PF-6252616; Pfizer)
• ezutromid (Summit Therapeutics/Sarepta Therapeutics)
• CAP-1002 (Capricor Therapeutics)

KOLs from North America

• Dr. Richard Barohn MD BA; Professor and Chairman of Neurology, Department of Neurology at the University of Kansas Medical Center, Kansas City, Kansas
• Dr. Basil Darras MD; Associate Neurologist-in-Chief; Chief, Division of Clinical Neurology; Director, Neuromuscular Center and Spinal Muscular Atrophy Program, Department of Neurology. Boston Children’s Hospital, Boston, Massachusetts
• Dr. Edward Clinton Smith MD; Associate Professor of Pediatrics and Associate Professor of Neurology at Duke Department of Pediatrics at Duke University School of Medicine, Durham, North Carolina
• Dr. Mathula Thangarajh MD PhD; Neuromuscular Neurologist/Neurophysiologist, Department of Neurology, Children's National Health System, George Washington School of Medicine, Washington DC
• Dr. Leo Wang MD PhD; Physician specializing in neurology and an Assistant Professor in the Department of Neurology at the University of Washington Medical Center, Seattle

KOLs from Europe

• Dr. Michela Guglieri MD; Honorary Consultant in Human Genetics and Neurologist/Senior Research Associate at the Institute of Genetic Medicine at the Newcastle upon Tyne Hospital, Newcastle, UK
• Professor Francesco Muntoni MD FRCPCH FMed Sci; Paediatric Neurologist and Director of the Dubowitz Neuromuscular Centre at the Great Ormond Street Hospital for Children, London, UK
• Professor Thomas Sejersen MD PhD; Professor/Senior Physician of Neuropediatrics in the Department of Women's and Children's Health (KBH), Division of Pediatric Neurology, Karolinska Universitetssjukhuset, Stockholm, Sweden
• Professor Jon Andoni Urtizberea MD MSc; Head of the internationally renowned Paris Summer School of Myology and Consultant at the Neuromuscular Reference Centre at Hendaye Hospital, (part of the large Assistance publique - Hôpitaux de Paris (AP-HP) hospitals group), France
• Anonymous German KOL MD PhD; Professor at the Department of Paediatric Neurology at a leading University Hospital, Germany

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