TRDA — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-08-05

• ELEVATE-LTE: A Long Term Extension (LTE) platform study protocol (ENTR-DMD-202) was accepted by U.K. and European authorities. This is a phase 2, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerabili…

• ELEVATE-50-201 and ENTR-601-51: We expect to submit additional regulatory applications following a review of data from the ongoing trials of our lead programs. We are evaluating a variety of options to optimize clinical study execution, and we expect to provide more specific timeline guidance as the…

• VX-670: Vertex has completed enrollment and continues dosing in the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1. The study is assessing safety and preliminary efficacy, including change from baseline in the splicing index and other endpoints evaluating mus…

• Improvement in TTR and in TTRV was seen across the majority of treated participants, irrespective of age:

• •Statistically significant TTR improvement versus placebo of 2.40 seconds

Text removed vs the prior filing · source: 10-Q · 2026-05-07

• ELEVATE-50-201: The Company received regulatory authorization from the UK’s Medicines and Healthcare Products Regulatory Agency (“MHRA”) and Research Ethics Committee to initiate a Phase 1/2 MAD clinical study of ENTR-601-50 in ambulatory patients living with DMD who are amenable to exon 50 skipping…

• ENTR-601-51: The Company has completed CTA-enabling studies for people living with DMD who are amenable to exon 51 skipping, which is applicable to the largest sub-population of exon skipping amenable patients. We are reviewing our global regulatory strategy of the program to determine how best to a…

• VX-670: Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical study of VX-670 in people with DM1. The study assesses both safety and efficacy and Vertex is on track to share results during the second half of 2026.

• In ELEVATE-44-201 Cohort 1, a statistically significant change from baseline in TTR velocity was observed:

• The mean change in TTR velocity in the study was 3.5 times higher than the MCID threshold (post hoc analysis) of 0.023 (literature derived), suggesting that ENTR-601-44 has the potential to change the trajectory of the disease at the lowest dose tested in the Phase 1/2 study.

Text added vs the prior filing · source: 10-Q · 2026-08-05

• will increase substantially as we continue our development of, seek regulatory approval for and potentially commercialize any of our therapeutic candidates.

• To become and remain profitable, we must succeed in developing and eventually commercializing products that generate significant revenue. This will require us to be successful in a range of challenging activities, including completing preclinical studies and clinical trials of our therapeutic candid…

• shares of common stock having an aggregate offering price of up to $150.0 million through the Sales Agent. However, there can be no assurance that the Sales Agent will be successful in consummating future sales based on prevailing market conditions or in the quantities or at the prices that we deem …

• time, including our underlying stock price and stock price volatility, the magnitude of the expense that we must recognize may vary significantly.

• We are early in our development efforts and all our development programs, including our lead therapeutic candidates ENTR-601-44, ENTR-601-45, our clinic ready candidates ENTR-601-50 and ENTR-601-51, our partnered

Text removed vs the prior filing · source: 10-Q · 2026-05-07

• To become and remain profitable, we must succeed in developing and eventually commercializing products that generate significant revenue. This will require us to be successful in a range of challenging activities, including completing preclinical studies and clinical trials of our therapeutic candid…

• divert our management from our day-to-day activities, which may adversely affect our ability to develop our therapeutic candidates. Our future capital requirements will depend on many factors, including, but not limited to:

• We are early in our development efforts and all our development programs, including our lead therapeutic candidates ENTR-601-44, ENTR-601-45, ENTR-601-50, and our partnered candidate VX-670, which are in the early clinical stage and ENTR-601-51 and ENTR-801, which are in the preclinical stage. We ha…

• The clinical trial requirements of the FDA and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a therapeutic candidate vary substantially according to the type, complexity, novelty and intended use and market of the therapeutic candidate. No…

• Where appropriate, we plan to seek approval from the FDA, EMA or comparable foreign regulatory authorities through the use of accelerated approval pathways. If we are unable to obtain such approval for any of our candidates, we may be required to conduct additional preclinical studies or clinical tr…

Text added vs the prior filing · source: 10-Q · 2026-08-05

• DateType of Trading ArrangementNature of Trading Arrangement

• (1)For purposes of this disclosure, the shares included in this table reflect the aggregate maximum number of shares that may be sold under the 10b5-1 trading arrangement. This 10b5-1 trading arrangement covers RSUs, shares underlying stock options exercisable and shares held by the officer. The act…

Text removed vs the prior filing · source: 10-Q · 2026-05-07

• Natarajan Sethuraman (President of Research and Development)

• Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c)

• Sale of the Company's common stock pursuant to the terms of the plan

• Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c)

• Sale of the Company's common stock pursuant to the terms of the plan