Dewpoint Therapeutics Announces TDP-43 Condensate Modulator Development Candidate for Treatment of ALS and Related Neurodegenerative Diseases

• First-in-class condensate-modulating small molecule Development Candidate targeting pathogenic TDP-43 condensates.
• Restores global TDP-43 splicing function in disease-relevant systems.
• Demonstrates in vivo activity in multiple animal models with biomarker readouts supportive of disease modification.
• Potential applicability across TDP-43 proteinopathies including Traumatic Brain Injury and Frontotemporal Dementia.

BOSTON, Jan. 08, 2026 (GLOBE NEWSWIRE) -- Dewpoint Therapeutics, Inc., a clinical-stage biotechnology company pioneering condensate-modulating therapeutics, today announced the selection of a Development Candidate (DC) for its TDP-43 program. The DC is a first-in-class small molecule designed to correct disease-associated TDP-43 condensates, restoring normal TDP-43 function and addressing the core molecular pathology that drives neurodegeneration in ALS and related diseases.

Pathogenic condensation and dysfunction of TDP-43 is observed in the vast majority (>97%) of ALS cases and across multiple related neurodegenerative diseases. Despite decades of effort, TDP-43 has remained largely undruggable using traditional therapeutic approaches. Dewpoint’s condensate biology platform enabled a fundamentally new strategy to directly modulate TDP-43 by targeting the aberrant condensate state that underlies its loss of function in disease.

The TDP-43 DC was selected based on a comprehensive preclinical data package demonstrating restoration of TDP-43 function in relevant cellular systems, robust activity in TDP-43 driven in vivo models of neurodegeneration including neurofilament light chain (NfL) reductions, clear evidence of on-mechanism activity, and pharmacologic properties supportive of advancement into IND-enabling studies. The molecule also shows applicability across a broader spectrum of TDP-43–associated neurodegenerative disorders, including frontotemporal dementia and traumatic brain injury.

“ALS remains one of the most devastating diseases, and pathologic TDP-43 condensates are the central driver for the vast majority of patients,” said Isaac Klein, MD, PhD, Chief Scientific Officer of Dewpoint Therapeutics. “This development candidate represents the first time anyone has achieved full restoration of TDP-43 function in preclinical models by directly correcting the pathologic condensates that underlie ALS. We believe this approach has the potential to deliver disease-modifying outcomes for patients.”

“This announcement reflects more than a single program milestone, it validates an emerging therapeutic approach and modality we are pursuing at Dewpoint,” said Ameet Nathwani, MD, Chief Executive Officer of Dewpoint Therapeutics. “Our condensate biology platform has enabled what has not been possible before: direct, mechanistic restoration of TDP-43 function, one of the most important and historically inaccessible targets in neurodegeneration. Advancing this program underscores our conviction that condensate-modulating therapeutics can redefine what is achievable in neurology.”

Dewpoint plans to initiate IND-enabling studies while continuing to engage closely with the ALS community to inform clinical development strategy and trial design.

About Dewpoint Therapeutics
Dewpoint is a clinical-stage biotechnology company applying condensate biology to the discovery and development of novel therapeutics. The recognition that many diseases are driven by dysfunction of biomolecular condensates creates new opportunities to modulate high-value and historically undruggable targets. Dewpoint’s proprietary AI-powered integrated platform supports a pipeline spanning oncology, neurology, and cardiometabolic disease, with strategic collaborations including Bayer and Novo Nordisk. Learn more at Dewpointx.com and follow us on LinkedIn.

About Condensates
Condensates are membraneless organelles that form dynamically within cells through a process known as phase separation. These compartments organize and concentrate biomolecules to regulate diverse cellular functions. Dysregulation of biomolecular condensates has been implicated in many diseases, including cancer, diabetes, cardiopulmonary disease, and neurodegenerative disorders. Condensate-modulating drugs, or c-mods, represent a new therapeutic modality with the potential to address complex diseases and historically undruggable targets. Learn more at Condensates.com.

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