- Grant to support the in vivo development of first-in-class condensate-modifying drugs (c-mods) targeting the TDP-43 condensatopathy in ALS
BOSTON, Aug. 26, 2024 (GLOBE NEWSWIRE) -- Dewpoint Therapeutics Inc., the leading biotechnology company that develops therapeutics by targeting biomolecular condensates, announced today that it was awarded for the second time a Target ALS Foundation grant to validate its leading condensate-modifying compound (c-mod) in a mouse model of slow-progressing amyotrophic lateral sclerosis (ALS). This in-kind grant is a collaboration with Biospective contract research organization.
Today, there are no disease-modifying therapeutic options for the ~ 20,000 patients that suffer from ALS in the US. The lack of efficacious drugs is reflected in the poor life expectancy of patients, at 2-5 years post diagnosis. Dewpoint is approaching ALS drug discovery and development from a new angle, by taking advantage of a pathology-driving aberration shared by over 97% of all patients – the wrongful localization of the protein TDP-43 from the nucleus to membrane-less cytoplasmic bodies, called biomolecular condensates. The TDP-43 mislocalization causes loss of TDP-43 splicing function and gain of toxic function caused by aberrant localization of other proteins into the TDP-43 cytoplasmic condensates. Together, these lead to systemic defects and the complex pathophysiology of ALS. Dewpoint’s lead c-mod was discovered through a high-content, high throughput approach that selected compounds that dissolve the aberrant TDP-43 condensates. Repairing the disease-driving aberrant condensate (condensatopathy), also repairs systemic TDP-43 loss of function and restores neuronal health, as well as clinically relevant ALS biomarkers in animal models.
The slow-progressing ALS NLS8 mouse model offered by Biospective and Target ALS recapitulates the TDP-43 cytoplasmic mislocalization, neurodegeneration and motor deficit phenotypes experienced by patients. Validation of Dewpoint’s c-mod into the NLS8 model will complement the existing data package, and the ongoing work in a C9orf72 mouse model, supported through another grant awarded by Target ALS, to better understand the efficacy, safety and mechanism of action of this pre-clinical candidate.
“Target ALS is thrilled to have the opportunity to help advance Dewpoint’s mission to deliver a novel, effective treatment to ALS patients. Their molecule will be one of the first to test a central hypothesis regarding the role of TDP-43 in motor neuron degeneration and may have the potential to treat both sporadic and familial forms of ALS,” said Dr. Amy Easton, Senior Director of Scientific Programs at Target ALS.
“We are excited to expand our partnership with Target ALS and are appreciative of the foundation’s transformational effort to bring the academic and biotech communities together. This alliance will accelerate the path to the clinic for Dewpoint’s revolutionary TDP-43 condensatopathy-targeted c-mod, which we believe holds great promise for patients,” said Isaac Klein, MD PhD, CSO of Dewpoint Therapeutics.
ABOUT CONDENSATES
Condensates are membraneless organelles that form dynamically throughout the cell via a process called phase separation. These subcellular compartments organize and concentrate molecules within cells to enable a diversity of key biochemical processes. The dysregulation of biomolecular condensates has been observed in many diseases, including cancer, diabetes, and neurological disorders. Condensate-modifying drugs (c-mods) potentially provide novel therapeutic options for complex diseases and historically undruggable targets.
ABOUT DEWPOINT THERAPEUTICS
Dewpoint is the leading biotech company in the application of biomolecular condensate biology towards the development of a new generation of therapeutics to address diseases of high unmet need. The realization that a vast range of conditions are regulated by or arise from the dysfunction of condensates has provided new possibilities for modulating the function of high-value targets previously deemed ‘undruggable,’ opening unexplored avenues to identify hundreds of novel therapeutic targets. Dewpoint’s proprietary AI/ML-powered state-of-the-art platform underlies a drug discovery pipeline that spans multiple therapeutic areas, including oncology, neurodegenerative, cardiopulmonary, and metabolic diseases. Through collaborations with Bayer, Novo Nordisk and Evotec, Dewpoint pushes the boundaries to accelerate the translation of condensate biology into medicine for patients suffering from difficult to treat diseases. Learn more at Dewpointx.com and follow us on X and LinkedIn.
ABOUT TARGET ALS
Target ALS is a 501(c)(3) medical research foundation committed to the search for effective treatments for Amyotrophic Lateral Sclerosis (ALS). Founded in 2013 by former New York City deputy mayor Dan Doctoroff — who lost both his father and uncle to ALS and was himself diagnosed in 2021 – Target ALS has transformed ALS research through their landmark Innovation Ecosystem model. The organization has fostered unprecedented collaborations between academia and the pharma and biotech industry, lowered barriers to access for critical research tools, and become a hub of communication and networking for the worldwide scientific community, resulting in the launch of 7 clinical trials and several drug discovery programs over the last decade. Building upon this momentum, Target ALS launched a groundbreaking Seven Pillar Strategy that will expand the breadth and depth of their ecosystem to accelerate ALS drug discovery with the goal of realizing a world where Everyone Lives. For more information and to get involved, visit www.targetals.org.
Media contact: media@dewpointx.com
Investor contact:
Michael Fenn, Ph.D.
Head of External Innovation & Investor Relations
mfenn@dewpointx.com
