Dyne Therapeutics to Host Virtual Investor Event to Review New Clinical Data from the ACHIEVE and DELIVER Trials Tomorrow, May 20 at 8:00 a.m. ET

Rhea-AI Summary

Dyne Therapeutics (Nasdaq: DYN) will host a virtual investor event on May 20, 2024, at 8:00 a.m. ET to discuss new clinical data from its Phase 1/2 ACHIEVE and DELIVER trials. The ACHIEVE trial focuses on DYNE-101 for myotonic dystrophy type 1 (DM1) and has enrolled 56 patients. Efficacy data from various cohorts will be presented. The DELIVER trial evaluates DYNE-251 for Duchenne muscular dystrophy (DMD) and has enrolled 48 patients. Safety, tolerability, and efficacy data, including dystrophin expression, will be discussed.

Both trials aim to provide comprehensive pharmacokinetic and pharmacodynamic data. The event will be webcast live and available for replay for 90 days.

Positive

• Dyne Therapeutics is providing an update to its timeline, now reporting data in the first half of 2024 instead of the second half, which may indicate accelerated progress.
• The ACHIEVE trial enrolled 56 patients and has administered approximately 500 doses, showcasing substantial trial activity.
• Efficacy data from multiple dosing cohorts in both ACHIEVE and DELIVER trials will be presented, offering a comprehensive view of the drug's performance.
• The DELIVER trial enrolled 48 patients and has administered approximately 480 doses, highlighting significant clinical trial activity.
• Data on safety, tolerability, pharmacokinetics, and pharmacodynamics, including dystrophin expression for the DELIVER trial, will be shared, which can provide insights into the drug's effectiveness and safety.

Negative

• The efficacy data being reported is to specific cohorts and time frames, which may not provide a complete picture of the drug's overall performance.
• Potential risks include the safety and tolerability outcomes, which if negative, could impact investor confidence and the company's stock value.
• The clinical update focuses heavily on data from the early phases of trials, which may not fully predict long-term outcomes.
• The announcement of the virtual event might set high expectations; any unfavorable data presented could negatively affect stock performance.

Medical Research Analyst

This announcement is significant due to the upcoming release of clinical data for two of Dyne Therapeutics' key trials: ACHIEVE and DELIVER. Both trials are investigating the efficacy and safety of treatments for serious muscle diseases, specifically myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD).

ACHIEVE Trial: This trial is important because it targets DM1, a progressive muscle disorder with no current cure. By examining multiple dose cohorts at different intervals, the study aims to establish the drug's safety profile and its impact on muscle strength and function, as well as patient-reported outcomes. Notably, the change in splicing suggests the drug's potential effectiveness at the molecular level, which can be a strong indicator of therapeutic benefit.

DELIVER Trial: Similarly, this trial targets DMD, a severe form of muscular dystrophy. The focus on dystrophin expression—a critical protein missing in DMD patients—is particularly important. Dystrophin's presence, measured by Western blot, offers a direct assessment of the treatment's biological impact, which could translate into clinical benefits for patients.

These updates will provide insights into the drugs' performance and potential market readiness. For investors, positive results could imply significant long-term value, but it's essential to consider the inherent uncertainties and risks associated with early-stage clinical trials.

Financial Analyst

From a financial perspective, the upcoming release of clinical data for Dyne Therapeutics' ACHIEVE and DELIVER trials is likely to have a noticeable impact on the company's stock price. Clinical trial updates, especially for diseases with significant unmet medical needs like DM1 and DMD, can drive investor sentiment and stock movement.

Investors should focus on the safety and tolerability data as well as the efficacy outcomes. Positive results could lead to a substantial revaluation of the company's market capitalization, potentially attracting new investment and partnerships. However, it's important to weigh these potential gains against the costs and timelines associated with bringing a new drug to market, which can be substantial.

The company’s strategic decision to host a virtual investor event immediately following the data release indicates confidence in the upcoming results. For retail investors, this could signal a potentially favorable outlook, but caution is advised as outcomes could also be mixed, impacting short-term stock performance.

WALTHAM, Mass., May 19, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced it now plans to report new efficacy and safety data from its Phase 1/2 ACHIEVE and DELIVER clinical trials on May 20, 2024, and to host a virtual event at 8:00 a.m. ET. This represents an update to Dyne’s prior guidance for the second half of 2024. The company intends to issue a press release prior to the start of the event. 

Phase 1/2 ACHIEVE Trial of DYNE-101 in DM1

• ACHIEVE is a Phase 1/2 global clinical trial evaluating DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1) who are 18 to 49 years of age. ACHIEVE, which is designed to be a registrational trial, consists of a 24-week multiple ascending dose (MAD), randomized, placebo-controlled period, a 24-week open-label extension (OLE) and a 96-week long-term extension (LTE). Enrollment is complete through the 6.8 mg/kg (approximate ASO dose) cohort evaluating once every eight-week dosing. A total of 56 patients have been enrolled in the study with approximately 500 doses administered to date.
  
  
• In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts. Additionally, pharmacokinetic and pharmacodynamic data, including change from baseline in splicing, measures of muscle strength and function, and patient reported outcomes will be shared. Efficacy data will be shared from the 1.8 mg/kg Q4W cohort (n=16) at 12 months, 3.4 mg/kg Q4W cohort (n=16) at 6 months, and 5.4 mg/kg Q8W cohort (n=8) at 3 months.
  

Phase 1/2 DELIVER Trial of DYNE-251 in DMD

• DELIVER is a Phase 1/2 global clinical trial evaluating DYNE-251 in ambulant and non-ambulant males with Duchenne muscular dystrophy (DMD) who are ages 4 to 16 and have mutations amenable to exon 51 skipping. DELIVER, which is designed to be a registrational trial, consists of a 24-week MAD, randomized, placebo-controlled period, a 24-week OLE and a 96-week LTE. Enrollment is complete through the 40 mg/kg (approximate PMO dose) cohort evaluating once every eight-week dosing. A total of 48 patients have been enrolled in the study with approximately 480 doses administered to date.
  
  
• In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts. Additionally, pharmacokinetic and pharmacodynamic data, including dystrophin expression measured by Western blot, will be shared from the 10 mg/kg Q4W cohort (n=8) at 6 months.
  

Virtual Investor Event

The live event webcast will be available on the Events & Presentations page of the Investors & Media section of Dyne’s website and a replay will be accessible for 90 days following the presentation. An accompanying slide presentation will also be available. To register for the live webcast and replay, please visit https://investors.dyne-tx.com/news-and-events/events-and-presentations.

About Dyne Therapeutics

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com/, and follow us on X, LinkedIn and Facebook.

Contacts:

Investors
Amy Reilly
areilly@dyne-tx.com
857-341-1203

Media
Stacy Nartker
snartker@dyne-tx.com
781-317-1938

FAQ

When is Dyne Therapeutics hosting the virtual investor event?

Dyne Therapeutics will host the virtual investor event on May 20, 2024, at 8:00 a.m. ET.

What trials will be discussed in Dyne Therapeutics' virtual event?

The event will cover new clinical data from the Phase 1/2 ACHIEVE and DELIVER trials.

What is the focus of the ACHIEVE trial?

The ACHIEVE trial evaluates DYNE-101 for myotonic dystrophy type 1 (DM1) in adults aged 18 to 49.

How many patients are enrolled in the ACHIEVE trial?

The ACHIEVE trial has enrolled 56 patients.

What is the DELIVER trial investigating?

The DELIVER trial evaluates DYNE-251 for Duchenne muscular dystrophy (DMD) in males aged 4 to 16 with specific mutations.

How many patients are enrolled in the DELIVER trial?

The DELIVER trial has enrolled 48 patients.

What types of data will Dyne Therapeutics report from the ACHIEVE trial?

Dyne will report safety, tolerability, pharmacokinetic, and pharmacodynamic data, including muscle strength and function, and patient-reported outcomes.

What efficacy data will be shared for DYNE-251 in the DELIVER trial?

Efficacy data including dystrophin expression measured by Western blot will be shared from the 10 mg/kg Q4W cohort at 6 months.

How can one access the live webcast of Dyne Therapeutics' virtual event?

The live webcast will be available on Dyne's website under the Events & Presentations section, with a replay accessible for 90 days.