Vertex pays Entrada $224M for preclinical rare disease drug, opening new front in assault on dystrophy

Vertex pays Entrada $224M for preclinical rare disease drug, opening new front in assault on dystrophy

Vertex has opened another front in its attempt to treat a rare cause of muscle weakness, paying Entrada Therapeutics $224 million upfront for the preclinical prospect ENTR-701.

Boston-based Entrada made its name as a new challenger for the Duchenne muscular dystrophy (DMD) market, raising $116 million in a private round and pulling off a $181.5 million IPO last year to fund a candidate that is now on the cusp of an investigational new drug filing. The DMD candidate is based on technology for getting biologics across cell membranes to hit intracellular targets.

Entrada has also applied its cyclic cell-penetrating peptides to the treatment of myotonic dystrophy type 1 (DM1), a rare disease that affects about 40,000 people in the U.S. and causes symptoms including muscle weakness and cardiac complications. There are no approved therapies for DM1.

Vertex has identified Entrada as a company with the potential to deliver a DM1 drug, leading it to pay $224 million upfront for the biotech’s late-stage preclinical prospect and make a $26 million investment. Last month, Entrada said it planned to file an IND for the candidate, ENTR-701, in the second half of next year.

The deal gives Vertex responsibility for funding preclinical development of ENTR-701 plus additional DM1 research activities under the terms of a four-year collaboration. Vertex will handle development, manufacturing and commercialization of ENTR-701 and any additional DM1 programs globally and will hand Entrada up to $485 million in milestones as the candidates advance.

Vertex established a toehold in the DM1 space in 2019 when it paid CRISPR Therapeutics $175 million to work on gene editing treatments for the disease and DMD. Other companies with riders in the DM1 race include AMO Pharma, which has a candidate in phase 3, Avidity Biosciences and Dyne Therapeutics.

Entrada is trailing the front-runners but has put together a preclinical data package that convinced Vertex ENTR-701 is a real contender for the market. In mice, a single dose of the drug candidate achieved sustained correction of the aberrant splicing seen in DM1 through at least eight weeks

The Vertex deal gives Entrada more money and narrows the focus of its spending. The biotech ended September with $215.6 million, a sum it said would keep it going into the second half of 2024. Shares in Entrada rose 20% to $21.30 on Thursday morning.

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