Dewpoint to Collaborate with Pfizer on Muscular Dystrophy

Dewpoint Therapeutics has announced a new research collaboration with Pfizer for the development of potential therapeutics for the treatment of myotonic dystrophy type 1 (DM1), a rare genetic disorder and one of two types of myotonic dystrophy. Under the agreement, Dewpoint will receive an upfront payment and will be eligible to receive research, development, and sales milestones payments, totaling up to $239 million should all milestones be achieved. Dewpoint will also be eligible to receive royalties on any approved products.

DM1 is an inherited genetic disorder linked with the DMPK gene. People diagnosed with DM1 experience muscle loss and weakness, difficulty breathing, cataracts, heart conditions, intellectual disability, and early death. DM1 affects approximately one in 8,000-20,000 people in the general population, with significant geographic and ethnic variation.

“DM1 is a devastating disease for which there is no treatment that addresses its underlying cause, so we are eager to carry out our collaboration with Pfizer to accelerate the development of new treatments into this space,” said Ameet Nathwani, M.D., Chief Executive Officer of Dewpoint. “We are excited about applying our platform to a wide array of diseases where biomolecular condensates may play a role in key disease pathways, as Dewpoint believes they do in DM1.”

Biomolecular condensates, formed through a process called phase separation, are membraneless droplets inside cells that facilitate molecular interactions and help cells perform vital functions. Condensates have been shown to play a critical role in key biological processes and in serious, intractable diseases across areas including neurodegeneration, cancer, inflammation, infectious disease, metabolic disease, and rare genetic disorders. Dewpoint develops drugs that exploit this biology, believing that a vast range of conditions have pathways that are regulated by condensates or arise from the dysfunction of condensates — including cancer, neurodegeneration, infectious disease, and metabolic disease. (Source: Dewpoint Therapeutics Website, 06 January, 2021)