Gold prices slip after rallying on Powell rate cut talk
BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA) received authorization from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial for its Duchenne muscular dystrophy (DMD) drug candidate, ENTR-601-44. The company announced Monday that the trial, named ELEVATE-44-201, will assess the safety, tolerability, and efficacy of the investigational drug in patients with a specific mutation amenable to exon 44 skipping. According to InvestingPro data, Entrada, currently valued at $476 million, appears undervalued based on Fair Value analysis, with a strong financial health score of 3.59 out of 5.
The authorization marks a significant step for Entrada, with CEO Dipal Doshi expressing the company’s commitment to addressing the urgent needs of families affected by DMD. The upcoming trial will involve multiple ascending doses (MAD) to determine the optimal therapeutic dose range. The company’s solid financial position is evident through its impressive current ratio of 6.59 and more cash than debt on its balance sheet, providing ample runway for clinical development. (InvestingPro subscribers can access 8 additional key financial insights about Entrada’s market position.)
ENTR-601-44 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO) designed to restore the mRNA reading frame and allow for the production of a functional, albeit slightly shortened, dystrophin protein. Dystrophin is crucial for maintaining muscle cell integrity, and its deficiency leads to the progressive muscle weakening characteristic of DMD.
The Phase 1/2 study, set to commence in the second quarter of 2025, will be randomized, double-blind, and placebo-controlled. It aims to enroll approximately 24 ambulatory patients with DMD, with dosing every six weeks across three cohorts ranging from 6 mg/kg to 18 mg/kg. Part B of the study will focus on further assessing the established optimal dose for safety and efficacy, including patient-reported outcomes and quality of life measures.
Entrada’s ENTR-601-44 has shown promise in a completed Phase 1 clinical study, demonstrating tolerability in healthy volunteers without serious adverse events and significant plasma concentration, muscle concentration, and exon skipping. Wall Street appears optimistic about the company’s prospects, with analysts maintaining a strong buy consensus and three analysts recently revising their earnings estimates upward, according to InvestingPro data.
Duchenne muscular dystrophy, a rare disease affecting about 41,000 individuals in the U.S. and Europe, has limited therapeutic options. Entrada’s EEV-therapeutics platform aims to address this unmet medical need by delivering therapeutics into cells to engage previously inaccessible intracellular targets.
The company’s broader pipeline includes RNA-, antibody-, and enzyme-based programs targeting neuromuscular, ocular, metabolic, and immunological diseases. Entrada has also partnered to develop VX-670, a clinical-stage program for myotonic dystrophy type 1.
The information for this article is based on a press release statement from Entrada Therapeutics. The company’s forward-looking statements involve risks and uncertainties, and actual results may differ materially from those projected.
In other recent news, Entrada Therapeutics has seen substantial progress in its therapeutic programs. The company reported a net income of $55 million and a robust cash balance of $470 million in the third quarter. Roth/MKM initiated a Buy rating on Entrada, with a price target of $29.00, based on positive results from the Phase 2a study of its drug neflamapimod. Additionally, Oppenheimer maintained an Outperform rating and increased the price target to $28.00, while H.C. Wainwright also maintained a Buy rating and lifted its price target to $20.00.
Entrada’s preliminary data from its Phase 1 ENTR-601-44-101 study for Duchenne muscular dystrophy (DMD) has shown promising results. The company is also making strides in its collaboration with Vertex Pharmaceuticals (NASDAQ:VRTX) on the DM1 program, having completed a Single Ascending Dose study. Regulatory filings for DMD are expected in the fourth quarter of 2024.
In line with these developments, Natarajan Sethuraman, PhD, has been promoted to President of Research and Development at Entrada. In another major move, Dipal Doshi, CEO of Entrada, has been elected to the Board of Directors of Azenta, Inc., a global provider of life sciences solutions. This recent news underscores Entrada’s continuous progress and potential in its therapeutic programs.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.