Dyne-101
- Generic Name
- Dyne-101
JPM25: Pfizer Focuses on Obesity, Regeneron Anticipates Key Data, BioNTech Expands Cancer Pipeline
• Pfizer is prioritizing its oral obesity drug, danuglipron, with late-stage studies planned for the second half of 2025, aiming for a novel mechanism of action.
• Regeneron anticipates Phase III data readouts for itepekimab in COPD and fianlimab in melanoma, potentially expanding its portfolio alongside Dupixent.
• BioNTech is advancing its mRNA-based cancer immunotherapies, including BNT327/PM8002 for solid tumors and BNT323/DB-1303, an ADC for endometrial cancer.
• Sarepta Therapeutics reported strong initial sales for Elevidys in Duchenne muscular dystrophy and is pursuing a Phase III trial for limb-girdle muscular dystrophy.
Dyne Therapeutics' DYNE-101 Receives FDA Fast Track Designation for Myotonic Dystrophy Type 1
• Dyne Therapeutics' DYNE-101 has been granted Fast Track Designation by the FDA, potentially accelerating its development for myotonic dystrophy type 1 (DM1).
• Clinical data for DYNE-101 demonstrates robust splicing correction and broad functional improvements in patients with DM1, including CNS benefits.
• Oppenheimer maintains an Outperform rating with a $60 price target, citing consistent data correlating splicing correction to functional improvements.
• Dyne plans to initiate registrational cohorts in 2025, aiming for accelerated approval in the first half of 2026, with splicing as the primary endpoint.
Pfizer's Sasanlimab Plus BCG Shows Promise in BCG-Naive, High-Risk NMIBC
• Pfizer's Phase 3 CREST trial showed that sasanlimab combined with BCG significantly improved event-free survival in BCG-naive, high-risk NMIBC patients.
• The combination therapy demonstrated a clinically meaningful and statistically significant improvement compared to BCG alone in the study.
• Sasanlimab's safety profile in combination with BCG was consistent with the known profiles of both agents, according to the trial data.
• Pfizer plans to submit the CREST trial results for presentation at an upcoming medical congress and discuss potential regulatory filings.
Dyne Therapeutics' DYNE-101 Shows Promise in DM1 Trial, FDA Grants Fast Track Designation
• Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1).
• The FDA grants Fast Track designation to DYNE-101, expediting its development and regulatory review process.
• Dyne plans to initiate a global Registrational Expansion Cohort, aiming for U.S. Accelerated Approval submission in H1 2026.
• DYNE-251 for Duchenne muscular dystrophy (DMD) is also advancing, with potential regulatory submissions expected in early 2026.
Myotonic Dystrophy R&D Surges with Over 20 Companies Developing Novel Therapies
• The myotonic dystrophy therapeutic landscape is experiencing a surge in R&D, with over 20 companies actively involved in developing 22+ novel therapies.
• Key players like Avidity Biosciences, Dyne Therapeutics, and Vertex Pharmaceuticals are advancing promising drugs such as AOC 1001, DYNE-101, and VX-670 through clinical trials.
• Regulatory designations, including Breakthrough Therapy and Fast Track, have been granted to several pipeline drugs, expediting their development for myotonic dystrophy.
• Recent clinical trial initiations and data presentations highlight the progress in addressing unmet needs in myotonic dystrophy treatment, fostering hope for improved patient outcomes.
Dyne Therapeutics Reports Third Quarter 2024 Financial Results and Corporate Update
Dyne Therapeutics announced its third quarter 2024 financial results, highlighting progress in its ACHIEVE and DELIVER trials, including FDA clearance for DYNE-101's IND application and enrollment in a registrational cohort for DYNE-251. The company also shared its financial position, with a cash reserve expected to fund operations into the second half of 2026.
Dyne Therapeutics' DYNE-251 Shows Promising Dystrophin Expression and Functional Improvements in Duchenne Muscular Dystrophy Trial
• DYNE-251 demonstrated a mean absolute dystrophin expression of 3.71% of normal, over 10-fold higher than eteplirsen, in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
• Clinical data from the DELIVER trial indicated meaningful improvements in motor function, including the Stride Velocity 95th Centile, meeting clinically important benchmarks defined by the European Medicines Agency.
• The investigational therapy exhibited a favorable safety profile, with most treatment-emergent adverse events being mild or moderate, supporting its potential for long-term use.
• Dyne Therapeutics is initiating registrational cohorts in the DELIVER trial and plans to provide an update on the path to registration for DYNE-251 by the end of 2024.
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