Dive Brief:

  • Dewpoint Therapeutics, a young drug company focused on an emerging field of research known as biomolecular condensates, said Wednesday that it is teaming up with Pfizer to develop new treatments for a rare kind of muscular dystrophy.
  • Dewpoint will receive an initial payment and, provided certain goals are hit, could take home up to $239 million more. The biotech is also eligible to receive royalties on any approved products that stem from the collaboration.
  • The companies' work will target myotonic dystrophy type 1, a genetic disease characterized by muscle wasting, problems in the heart, lungs and brain, and early death. The National Organization for Rare Disorders estimates that DM1 affects around one in 8,000 to 20,000 people. The disease is also of interest to Faze Medicines, another biotech exploring biomolecular condensates.

Dive Insight:

As the name suggests, biomolecular condensates are tiny concentrations of molecules found inside cells. Scientists have observed these clusters, which take the form of liquid-like droplets, for decades, but only recently determined that they help regulate cellular reactions and activities.

Like other cell-managing structures, researchers suspect that biomolecular condensates can give rise to a variety of diseases if they malfunction. This thinking has led to the formation of several new drug companies in the last couple years, with Dewpoint being the first to come out of stealth mode.

Dewpoint arrived in early 2019, backed by the venture capital firm Polaris Partners and a string of other investors. Company leadership said the initial focus would be cancer and neurodegenerative diseases, though other areas like immunology and infectious disease also appear to be on Dewpoint's radar. The Boston-based biotech quickly drew interest from pharmaceutical giants, too, with Bayer and Merck & Co. inking separate deals potentially worth $100 million or more.

With the Pfizer deal, Dewpoint joins a handful of companies targeting DM1. Audentes Therapeutics, a gene therapy developer now owned by Astellas, has been exploring two approaches to treat the disease. Vertex and CRISPR Therapeutics also recently expanded a gene editing partnership to include DM1 and Duchenne muscular dystrophy.

Faze Medicines has its sights set on DM1 as well. The Cambridge, Massachusetts-based biotech debuted in December with $81 million in Series A funding, which was supplied, in part, by some big pharmaceutical firms, including the Novartis Venture Fund and AbbVie Ventures.

Faze is also trying to find and develop treatments for ALS. Interim CEO Cary Pfeffer recently told BioPharma Dive that Faze may take aim at other neurological and neurodegenerative disorders, as well as cancer, immune diseases and viral illnesses.

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