Vertex Pursues Myotonic Dystrophy with $250M Entrada Deal

Vertex_David L. Ryan/The Boston Globe via Getty

Courtesy of David L. Ryan/The Boston Globe via Getty

Vertex Pharmaceuticals agreed to a collaboration deal with the pre-clinical biotech Entrada Therapeutics, Thursday. The two companies announced a global partnership to develop intracellular Endosomal Escape Vehicle (EEV) therapeutics. 

As part of the in-licensing agreement, Entrada will receive an upfront cash payment of $224 million and an equity investment of $26 million.

The preclinical therapeutic company will also be eligible to receive up to $485 million upon the successful completion of specific developmental and regulatory milestones. 

The centerpiece of the collaboration will be Entrada’s late-stage pre-clinical myotonic dystrophy type 1 (DM1) candidate, ENTR-701. ENTR-701 is a novel EEV therapeutic designed to address the underlying genetic cause of DM1. 

DM1 is a rare, multisystemic type of muscular dystrophy caused by excessive repeats of the CTG nucleotide sequence within the dystrophia myotonia protein kinase gene.

There are no treatments for this devastating form of muscular dystrophy, according to Entrada. 

Vertex Hits the Gas on Business Development

With cash from its mega-blockbuster cystic fibrosis treatment, Trikafta, Vertex has invested heavily in its internal and external pipeline in 2022. In the second quarter of the year, the company spent $60 million to acquire Catalyst Biosciences for its portfolio of protease medicines targeting the complement system. 

Vertex also acquired the privately held biotech, ViaCyte, in the third quarter for $315 million. The acquisition could lead to the development of ground-breaking therapeutics for type 1 diabetes, an indication that might be worth billions in future revenue.

This latest collaboration and equity deal with Entrada dovetails well with Vertex’s ongoing pipeline expansion efforts. Entrada’s EEV therapeutic pipeline has the potential to lead to the development of functional cures for many rare diseases.

ENTR-701, while still in pre-clinical development, is targeting an unmet medical need. As such, this unique therapeutic could be a significant revenue contributor for the rare disease titan in the latter half of the decade.    

Vertex, a leader in innovation for rare diseases, is adding another key piece to the puzzle with this licensing agreement. 

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