Biopharmaceutical company Entrada Therapeutics Inc. (TRDA:NASDAQ), which is working to develop a new category of medicines called Endosomal Escape Vehicles (EEV™) that are designed to treat a wide range of neuromuscular diseases, yesterday announced that it has entered into a US$735 million collaborative development agreement with Vertex Pharmaceuticals Inc. (VRTX:NASDAQ).
The two companies have agreed to establish a global partnership tasked with the discovery and development of intracellular EEV therapeutics for use in the treatment of myotonic dystrophy type 1 (DM1), a disorder affecting skeletal and smooth muscle, the heart, eyes, and endocrine and central nervous systems.
A key part of the joint development efforts is expected to be centered around the advancement of Entrada's ENTR-701, which is now in the late stages of preclinical development. The report explained that ENTR-701 is "a proprietary EEV-conjugated phosphorodiamidate morpholino oligomer designed to address the underlying cause of DM1."
Under the terms of the agreement, Vertex Pharmaceuticals is required to make an upfront payment to Entrada Therapeutics in the amount of US$224 million, along with a direct US$26 million equity investment in the company.
In addition, the agreement provides that Entrada is eligible to receive up to US$485 million if certain research, development, regulatory and commercial milestones are achieved, plus royalties from any future net sales of products brought to market from the collaboration.
Entrada Therapeutics' President and CEO Dipal Doshi commented, "Our collaboration with Vertex represents an important step for Entrada as we work to make intracellular therapeutics a reality through our novel EEV approach . . . DM1 is a progressive disease with no treatment options available. Working with Vertex will enable us to expeditiously move this program forward while focusing the majority of our internal resources on advancing new therapeutic options for patients living with Duchenne and expanding our commitment to non-neuromuscular disease programs."
Entrada Therapeutics believes that ENTR-701 offers the potential to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts and restore normal muscle function.
Vertex Pharmaceuticals' EVP of Global Research and Chief Scientific Officer David Altshuler, M.D., Ph.D. stated, "DM1 has been a disease area of interest to Vertex for some time . . . Entrada's innovative EEV approach, the significant progress in their DM1 program, and the potential for it to reach the clinic in the near-term hold exciting potential for patients."
"Working together, we believe we have the opportunity to develop a transformative treatment for this devastating disease," Dr. Altshuler added.
The agreement is structured to last for a period of four years, during which time Entrada will maintain responsibility for ENTR-701 research activities and further DM1-related research efforts. Vertex's role will be to manage ENTR-701's worldwide development, manufacturing, and commercialization operations as well as any additional programs arising from Entrada's continued DM1-related research work.
Entrada Therapeutics believes that ENTR-701 offers the potential to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts and restore normal muscle function. Entrada claimed that results from preclinical studies indicate that ENTR-701 may be capable of correcting disease-relevant biomarkers in certain muscle groups.
Entrada Therapeutics is a biopharmaceutical firm headquartered in Boston, Mass., that is focused on the development of a new class of medicines called Endosomal Escape Vehicle (EEV™) that are designed to treat a broad range of neuromuscular diseases. These EEVs are engineered to engage intracellular targets that previously were considered inaccessible and undruggable.
Entrada's development pipeline includes several oligonucleotide-, antibody- and enzyme-based programs that are investigating potential treatments for use in neuromuscular and central nervous system diseases, immunology, and oncology. The company's leading programs include ENTR-601-44, which is targeting Duchenne muscular dystrophy (DMD), and ENTR-701, which is being developed to target myotonic dystrophy type 1 (DM1).
Vertex is a large biotechnology firm based in Boston, Mass. The company has a market cap of about US$81.5 billion and owns a portfolio of commercially approved medicines that combined generate annual revenues totaling US$8.55 billion. The firm's asset portfolio includes multiple medicines that have been approved to treat cystic fibrosis (CF), a rare, life-threatening genetic disease. The company's investigational small molecule development pipeline includes several additional research and clinical studies in the areas of CF, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, beta thalassemia, pain, type 1 diabetes, and sickle cell disease.
Entrada Therapeutics began the day yesterday with a market cap of around US$556.51 million, with approximately 31.41 million shares outstanding and a short interest of about 3.66%. TRDA shares opened 9% higher yesterday at US$19.30 (+US$1.58, +8.92%) over the previous day's US$17.72 closing price. The stock traded yesterday between US$18.51 and US$21.64 per share and closed for trading at US$21.00 (+US$3.28, +18.51%).
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