Pivoting toward neuromuscular diseases and Duchenne, Entrada locks down $116M crossover round

01 Apr 2021
endpts.com
OligonucleotideAccelerated Approval
A little over two years since its last $59 million round, Entrada Therapeutics is making some more noise Wednesday with a nine-figure crossover. And they’ve got a new focus on neuromuscular diseases, led by Duchenne muscular dystrophy, as their research has broadened over the years. Entrada pulled in a $116 million Series B, with the funds primarily slated for getting its first Duchenne program into the clinic. The Boston-based biotech hopes to get past the IND acceptance phase for the lead candidate and then expand into other neuromuscular areas. The platform here, originally based on Dehua Pei’s lab research from Ohio State University, centers around what Entrada calls endosomal escape vehicles, or EEVs. At their core, EEVs are cyclic peptides that trigger a process enabling cell membranes to take in biologic conjugates, CEO Dipal Doshi told Endpoints News. Back in 2018 during the Series A fundraise, Entrada’s main efforts dealt with applying the EEVs to enzyme replacement therapies, with a primary target of the mitochondrial disease known as MNGIE. But last year, Doshi said, the company saw a “remarkable opportunity” in its oligonucleotide research and decided to pivot toward this area in earnest. Doshi now describes Entrada as being broken into three parts: enzyme replacement, oligonucleotides and protein degradation. As their sights have turned toward neuromuscular diseases with the oligonucleotides research, Entrada is now looking to partner out the MNGIE program. “We’ve been very explicit to say, look, we’re not going to be cute here, we want to find a home for this program,” Doshi told Endpoints. “We want to put it into a company that has the ability to recruit an ultra-rare clinical study in the immediate time frame.” Entrada doesn’t necessarily have a specific partnership model in mind, Doshi added, but they’re hoping to find a rare disease-focused pharma that can run the trial while teaming up on manufacturing. When it comes to applying the EEVs to oligonucleotides for Duchenne and its other neuromuscular programs, Entrada does so by targeting the RNA. In such diseases, patients have mutations that prevent the translation of RNA into proteins — in Duchenne’s case, the protein in question is dystrophin. The oligonucleotides connected to the EEVs force the cells to skip these mutations in their genetic code, Doshi says, allowing for the creation of dystrophin. And through this method, the dystrophin can then get into the appropriate muscles. In early research, Entrada believes it can differentiate itself from other Duchenne players due to the dystrophin uptake they’ve seen in animal models. Specifically, researchers have recorded significant uptake in the heart, which could validate the EEV technology for use in other neuromuscular diseases with similar pathologies, Doshi said. Entrada isn’t disclosing yet which exon it’s skipping in the lead program, but Doshi noted that they see a “basket opportunity” in going after exons 44, 45, 51 and 53 at around the same time. That would comprise about 40% of all DMD patients. With Wednesday’s financing now in hand, Entrada is looking to get the lead Duchenne candidate into the clinic sometime next year. Duchenne is an area that’s seen heavy investment but few successes in recent years. Last August, the FDA approved the third Duchenne drug on the market, but regulators continued to express concerns about the efficacy of the drug from NS Pharma. The other two drugs come from Sarepta, where in one case the FDA infamously flip-flopped from CRL to accelerated approval after just four months. The round was led by Wellington Management Company and joined by Redmile Group, TCG Crossover, Greenspring Associates, Point72, Qatar Investment Authority (“QIA”), Moore Strategic Ventures, Goldman Sachs, CureDuchenne Ventures and one undisclosed global investment firm. Existing investors 5AM Ventures, MPM Capital, Roche Venture Fund, MRL Ventures Fund and Agent Capital also participated in the Series B.
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