FDA authorizes Entrada’s DMD drug study

BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA), a biotechnology company with a market capitalization of $476 million and strong financial health according to InvestingPro metrics, has received authorization from the U.S. Food and Drug Administration (FDA) to proceed with a Phase 1b clinical trial for its investigational Duchenne muscular dystrophy (DMD) treatment, ENTR-601-44. This development follows a similar approval by the UK’s Medicines and Healthcare Products Regulatory Agency for a related study.

The ELEVATE-44-102 study is designed to assess the safety, tolerability, and pharmacokinetics of ENTR-601-44 in adult patients with DMD. Specifically, the trial will focus on those with a mutation in the DMD gene amenable to exon 44 skipping, a method that could potentially restore the production of a functional, albeit shortened, dystrophin protein.

ENTR-601-44, which utilizes Entrada’s proprietary Endosomal Escape Vehicle (EEV™) technology, aims to address the root cause of DMD by enabling the delivery of therapeutics into cells to restore dystrophin protein production. DMD is a genetic disorder characterized by progressive muscle degeneration, leading to severe physical disability and life-threatening heart and lung complications.

The randomized, double-blind, placebo-controlled Phase 1b study will enroll approximately 32 non-ambulatory and ambulatory adult patients. The trial will involve multiple ascending doses administered every six weeks, with dosages ranging from 0.16 mg/kg to 1.28 mg/kg. Participants may have the opportunity to join an open-label extension study for further evaluation of the drug’s long-term safety and efficacy. With a healthy balance sheet showing more cash than debt and a current ratio of 6.59, InvestingPro data suggests Entrada is well-positioned to fund its clinical development programs.

Entrada Therapeutics anticipates starting enrollment for the trial in the first half of 2026. The company’s CEO, Dipal Doshi, expressed optimism about the potential of ENTR-601-44 to serve a significant portion of the adult DMD population that is often excluded from clinical studies due to the advanced stage of their disease. Analysts share this optimism, with price targets ranging from $20 to $29 per share, and three analysts recently revising their earnings estimates upward. The company is scheduled to report its next earnings on March 12, 2025, which InvestingPro subscribers can analyze using comprehensive financial metrics and expert insights.

The ELEVATE-44 clinical program represents a global effort to evaluate the treatment in a broad patient population, encompassing both early and advanced stages of DMD. This latest authorization is a critical step in expanding the program and advancing the potential of Entrada’s EEV-therapeutics pipeline.

This news is based on a press release statement from Entrada Therapeutics.

In other recent news, Entrada Therapeutics has made significant strides in its clinical trial efforts and financial outlook. The company 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. This 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. H.C. Wainwright maintained a Buy rating and a $20 price target for Entrada Therapeutics, highlighting the company’s progress in addressing a significant unmet medical need. Additionally, Roth/MKM initiated coverage of Entrada Therapeutics with a Buy rating and a price target of $29, expressing confidence in the company’s DLB treatment growth trajectory. This optimism is based on the positive results from a Phase 2a study of neflamapimod, with further results expected in the fourth quarter of 2024. Furthermore, Dipal Doshi, CEO of Entrada Therapeutics, was elected to the Board of Directors of Azenta, Inc., bringing his extensive experience in biotechnology and pharmaceuticals to the table. These developments reflect Entrada Therapeutics’ ongoing efforts to advance its therapeutic pipeline and strengthen its leadership presence in the life sciences sector.

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