A New Era in Neuroscience Drug Development

Neuroscience is poised to reach a significant inflection point over the coming decade with the potential to transform the way we treat many neurological diseases. This is perhaps best illustrated by the recent successes achieved for the rare-orphan neuromuscular disease Spinal Muscular Atrophy (SMA), the leading genetic cause of infant mortality. The convergence of the understanding of underlying disease pathophysiology, the right therapeutic hypothesis and ability to achieve the right biodistribution in target tissues helped fuel the regulatory approvals for three different therapies for SMA in the United States.

Building on the learnings from SMA and aiming to harness advances in genetics, precision medicine and innovative modalities from the past decade, Takeda has recently evolved its Neuroscience Therapeutic Area strategy to focus on rare neurological indications where we can directly address targets and underlying mechanisms of disease. This strategy is already playing out across our pipeline as highlighted by the early signals of efficacy from our orexin 2 receptor agonist in narcolepsy type 1 and our Phase 2 ELEKTRA study of soticlestat in children with Dravet Syndrome (DS)/Lennox-Gastaut Syndrome.

Sarah Sheikh, Head, Neuroscience Therapeutic Area Unit at Takeda

Building in Neuromuscular Disease

Within rare neurology, neuromuscular diseases are a key strategic area for Takeda Neuroscience. Building on our foundation today in amyotrophic lateral sclerosis (ALS), we see significant opportunity to expand our efforts to other neuromuscular diseases. Many of these diseases carry devastating burdens for patients and their families, resulting in significant disability and often mortality.

Significant advances in translational and clinical tools are advancing drug development in neuromuscular diseases, and Takeda has invested significantly to build these capabilities. However, there remain key challenges to solve to optimally treat neuromuscular diseases, including the broad biodistribution required to target the key tissues impacted by these diseases. Platform technologies that aim to solve these challenges have the potential to address multiple neuromuscular diseases, thereby unlocking significant opportunity.

Unlocking the Opportunity in Neuromuscular Disease

To help solve these biodistribution challenges, we recently announced a research collaboration with Japanese biotechnology company, PeptiDream, granting Takeda an exclusive license to develop peptide drug conjugates for neuromuscular diseases. Combining Takeda’s long history and strength in neuroscience drug discovery research with PeptiDream’s innovative peptide technology, this collaboration aims to develop superior treatments with enhanced tissue biodistribution creating transformational therapies for rare neuromuscular diseases. 

We embrace innovation wherever it originates whether it’s discovered from our own labs or as part of collaborations with biotech, other pharmaceutical companies and universities across the globe. Partnering with PeptiDream, a Kawasaki, Japan-based company, is another testament to that philosophy as we look to develop highly differentiated, next generation medicines for neuromuscular diseases. Building on this momentum will be critical as we aspire to be a trusted leader and partner in neuroscience and ultimately deliver transformative therapies for patients and their families.