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Expansion Therapeutics raises USD80m Series B financing

Biotechnology company Expansion Therapeutics has closed an USD80 million Series B financing led by Cormorant Asset Management with participation from new investors Westlake Village BioPartners, Surveyor Capital (a Citadel company) and Logos Capital as well as Series A investors RA Capital Management, 5AM Ventures, Kleiner Perkins, Sanofi Ventures and Novartis Venture Fund. 

Proceeds from the financing will be used to advance the company’s small molecule RNA platform (SMiRNA) to identify clinical candidates in myotonic dystrophy type 1 (DM1), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and various tauopathies.

“Drugging RNA with small molecules has the potential to dramatically transform the lives of patients and we are excited to be supported by world-class investors who share our commitment to patients and our vision to develop innovative therapies to treat severe RNA-mediated diseases, including neurodegenerative and neurological disorders,” says Renato Skerlj, PhD, President and Chief Executive Officer of Expansion Therapeutics. “This financing milestone demonstrates significant investor confidence in the leadership team, the science and our mission to accelerate the preclinical and clinical development of our novel medicines utilising our proprietary approach.”

Expansion focuses on structured RNA targets that are evolutionarily conserved or genetically demonstrated to cause disease. Disease-driving RNA structures amenable to ligand binding are identified by combining patient genetics with deep informatic analysis and then screened against Expansion’s proprietary RNA-focused libraries for small molecules that modulate the RNA target in a functionally relevant manner. Expansion’s novel structural biology SMiRNA™ platform is then utilised for the design and advancement of lead-like chemical matter that ultimately delivers an oral medicine to treat disease. Expansion is focused on neurological diseases where few treatment options are available for patients such as DM1 which is a system wide disorder affecting muscle, cardiac and brain function. A small molecule medicine that treats all aspects of the disease offers patients a significant benefit.

“Recent advances in understanding RNA structure and the science of targeting RNA with small molecules offer tremendous opportunities to transform the way RNA-mediated diseases are treated. We believe Expansion’s established focus on structured RNA targets position them as leaders in this emerging space,” says Andy Phillips, PhD, Managing Director at Cormorant. “We’re extremely enthusiastic about the promise that the SMiRNA™ platform holds to deliver disease-modifying therapies for a wide range of devastating neurological disorders, particularly neurodegenerative diseases,” adds Raymond J Kelleher, MD, PhD, also Managing Director at Cormorant.

“The progression of Expansion’s lead programs, broad therapeutic potential of the platform, and the leadership team’s demonstrated track record in the field of neuroscience compelled us to partner with this exceptionally strong group of drug developers, as well as Cormorant and the rest of the investor syndicate,” says Andrew Levin, MD, PhD, Managing Director at RA Capital Management. “We are confident in Expansion’s ability to leverage its differentiated platform to positively impact the lives of patients suffering from severe and debilitating neurological diseases who currently have limited or no treatment options,” adds Laura Tadvalkar PhD, Principal at RA Capital Management.

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