Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis

Strategic partnering and new product development will enable better biologics for personalized and targeted treatment

Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs). The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies. NMDs present significant clinical challenges because of disease heterogeneity and rarity of occurrence. The lack of measurable early disease markers and clinical outcomes further complicates the management of such disorders.

Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class. An overview of the global clinical trial landscape for emerging Phase 1, 2, and 3 treatments revealed that Amyotrophic Lateral Sclerosis (ALS), Myasthenia Gravis (MG), Duchenne Muscular Dystrophy (DMD), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), Diabetic Neuropathy (DN), and Spinal Muscular Atrophy (SMA) are the key hotspots for clinical studies. These indications are likely to witness new drug developments. Frost & Sullivan's research, "Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis" explores emerging biologics across these six NMDs.

Key biologics emerging for these rare NMDs include: stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments. There were 3 recent US FDA approvals for NMD RNA therapies: Nusinersen for SMA and Eteplirsen for DMD in 2016, and Golodirsen for DMD in 2019. Furthermore, Sarepta's Casimersen for DMD and argenx's efgartigimod for MG were approved as recently as 2021. Biologics have opened doors for ground-breaking disease-modifying treatments and will transform the quality of life for patients across the world.

While biologics may appear to cost more than small molecule treatments, they provide a personalized and, in some cases, the only treatment option. They can also be less expensive than lifetime treatment costs for certain rare NMDs. For example, Zolgensma's $2.1 million price tag (approximate) is still lower than the standard medical costs incurred for SMA patients in a lifetime. Furthermore, the evolving payer landscape is likely to get more standardized in the coming years to offer greater access to biologicals. Therefore, biologics are suitably poised to transform the rare NMD landscape with personalized and targeted treatment strategies and will provide life-saving treatment options for fatal neuromuscular conditions.

Key Points Discussed:

• What are the key emerging biologics for NMDs with high clinical activity?

• What are the key drivers or challenges for biologics development across NMDs?

• How do venture funding and patent landscapes look for NMD biologics?

• Who are the key industry participants developing biologics for NMDs?

• What are the clinical trends emerging across each biologic category for NMDs?

• Which biologic categories provide promising growth opportunities for NMD management?