Report Highlights Innovative And Diverse Pipeline Of Products For Treating Duchenne And Becker Muscular Dystrophies

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by Charles Moore |

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A new 77-page GBI Research report entitled “Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy – Identifying and Commercializing First-in-Class Innovation” identifies and highlights a highly innovative and diverse pipeline of products for treating Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD)

Currently in the pipeline are 84 products across all stages of development, and GBI Research’s analysis reveals a high degree of innovation and diversity, with 70 percent of the pipeline made up of first-in-class products, acting on 13 first-in-class targets. This exceptional level of innovation is largely due to the high number of first-in-class products solely targeting the dystrophin gene, which is the primary genetic factor causing both DMD and BMD.

Duchenne Muscular Dystrophy is a hereditary disorder, mainly affecting boys (DMD is very rare in girls). As they grow older, their muscles become weaker because the body is unable to produce a muscle protein called dystrophin resulting in the muscle cells weakening and gradually breaking down. Signs of weakness usually first manifest when the boys are between three and five years of age (sometimes earlier) mostly in the legs and hips, with affected children falling frequently, having trouble running as fast as their peers, climbing stairs, or getting up from chairs. Physiologically they tend to develop big calves, frequently walk on their toes and lean backwards to keep their balance. Eventually weakness associated with DMD makes walking more difficult and a wheelchair will be needed. Gradually, all the muscles become very weak, including the muscles used for breathing and the heart.

Becker muscular dystrophy (BMD) is also an inherited degenerative muscle disorder that occurs almost exclusively in males, and similarly to DMD is caused by a mutation in the dystrophin gene on the X chromosome, an important protein building block that helps give muscles structure and strength.

Both DMD and BMD primarily affect skeletal muscles, which are used for movement, and heart (cardiac) muscle. However, BMD is less common — affecting just 1 in 35,000 males worldwide and with a later onset and slower progression than DMD.

Symptoms of BMD typically appear between the ages of five and 15 and include difficulty with walking, rising from the floor, running hopping and jumping. Due to heart muscle weakening, people with BMD also have a high risk of developing heart disease (cardiomyopathy), and their heart health should be monitored closely.

The high population of first-in-class products in the pipeline consequently constitutes a significant alteration of the landscape for a market, reliant on symptomatic treatment glucocorticoids. Although PTC Therapeutics’ drug candidate Translarna (ataluren) is developed to correct the genetic defects and has been granted orphan drug designation by the European Medicines Agency, or EMA and the U.S. Food and Drug Administration (FDA for the treatment of both nmDMD and nmCF, and conditional marketing authorization in the European Union for the treatment of nmDMD in ambulatory patients aged five years and older, becoming the first treatment approved for the underlying cause of DMD, significant unmet needs remain in the market, as Translarna is applicable to only 10-15 percent of all DMD cases caused by nonsense mutations, which are implicated in a variety of genetic disorders and create a premature stop signal in the translation of the genetic code contained in mRNA, which prevents the production of full-length, functional proteins.

Despite a strong focus on personalized treatments in the DMD/BMD pipeline that treat the genetic cause of these diseases, innovation is also concentrated on novel molecular targets that alleviate the dystrophic pathology regardless of gene mutations, thereby allowing widespread use in contrast to mutation-specific treatments. These new therapies are expected to be used alongside primary treatment to repair the mutated gene, halt muscle degeneration, and improve patients’ life expectancies going forward.

Strong Alignment of Innovation to Genetics and Disease Processes in Early Pipeline

DMD, and the disease’s less severe form BMD are neuromuscular disorders caused by heritable mutations in the single dystrophin gene, which ultimately result in progressive muscle weakness and gradual degeneration due to destabilization of the sarcolemma (muscle cell membrane) and resultant loss of muscle integrity. However, the GBI Research report finds increasing evidence suggesting that multiple secondary pathological mechanisms, rather than dystrophin deficiency alone, cause or contribute to the pathological features of DMD/BMD, and drive disease progression. This finding further substantiates the need for better understanding of the downstream events of dystrophin deficiency in order to enable identification of more potential molecular targets that in turn could be translated into disease-modifying treatments.

GBI Research’s proprietary analyses show that the 13 first-in-class targets differ substantially in terms of clinical and commercial potential based on how well their functional roles align to the disease pathophysiology and the strength of evidence in Preclinical studies. Some molecular targets are therefore considered more promising than others due to a stronger potential to be translated into novel treatments, with the most promising targets providing a strong scientific rationale to support their therapeutic development, as indicated by substantial improvement in both muscle histopathology and function in vivo across different animal model systems in clinical trials.

GBI Research’s analysis also identifies opportunities for some of the first-in-class DMD/BMD targets to be repositioned to other Muscular Dystrophies, although this is expected to pose challenges given the currently limited understanding of the common molecular processes defected across multiple types of MD.

Numerous Investment Opportunities In Deals Landscape

The report observes that strategic consolidation is relatively uncommon in the DMD/BMD market, with 15 licensing agreements and 18 co-development deals between 2006 and April 2015. The authors say that supported by findings from the industry-wide analysis, there is a tendency for first-in-class DMD programs to attract higher deal values than non-first-in-class programs, thus highlighting their commercial attractiveness, and despite high-risk profiles of first-in-class products, they have greater potential to revolutionize or improve therapeutic options, meaning that identifying promising first-in-class compounds early in development offers the greatest potential commercial benefit to pharmaceutical companies.

The GBI Research analysts conclude that with 36 first-in-class products currently in development and having not yet been involved in a licensing or co-development deal, there are numerous opportunities for in-licensing or co-development in this indication


The report analyzes innovation in DMD/BMD in the context of the overall pipeline and current market landscape. In addition, it analyzes the deals landscape surrounding first-in-class products in DMD/BMD and pinpoints opportunities for in-licensing.

The report covers and includes:
– A brief introduction to DMD/BMD, including symptoms, pathophysiology, and an overview of pharmacotherapy and treatment algorithms
– The changing molecular target landscape between market and pipeline and particular focal points of innovation in the pipeline
– A comprehensive review of the pipeline for first-in-class therapies, analyzed on the basis of stage of development, molecule type, and molecular target
– Identification and assessment of first-in-class molecular targets, with a particular focus on early-stage programs for which clinical utility has yet to be evaluated, as well as literature reviews of novel molecular targets
– Assessment of the licensing and co-development deal landscape for DMD/BMD therapies and benchmarking of deals involving first-in-class versus non-first-in-class-products

Reasons To Buy The Report

The GBI Research report is designed to assist business development and enable marketing executives to strategize their product launches, by allowing them to:
– Understand the focal shifts in molecular targets in the DMD/BMD pipeline
– Understand the distribution of pipeline programs by phase of development, molecule type, and molecular target
– Access scientific and clinical analysis of first-in-class developmental programs for DMD/BMD, benchmarked against non-first-in-class targets
– Access a list of the first-in-class therapies potentially open to deal-making opportunities

The report table of contents can be found here:

The full report can be purchased at:

GBI Research
PTC Therapeutics
Muscular Dystrophy Canada