Dyne Therapeutics, Inc. · NASDAQ:DYN
Dyne Therapeutics — AAN Annual Meeting 2023
8 slides from Dyne Therapeutics's conference presentation, Apr 25, 2023
Slide 1: ACHIEVE Trial: Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1
ACHIEVE Trial, a Randomized, Placebo- Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1 (DM1) Daniel Wolf, PhD AAN Annual Meeting | 25 April 2023 Data previously presented at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding Dyne’s strategy, future operations, prospects a
Slide 3: DM1 Is a Progressive Neuromuscular Disease with No Disease-Modifying Treatment Approved
DM1 Is a Progressive Neuromuscular Disease with Multisystem Involvement with no Disease-Modifying Treatment Approved Overview Clinical Presentation Population • Mutation in the DMPK gene • Myotonia • >40,000 (US) • Onset at any point, depending • Muscle weakness • >74,000 (Europe) on DM1 phenotype •
Slide 4: FORCE Targets the Genetic Basis of DM1 to Correct Splicing
FORCE Targets the Genetic Basis of DM1 to Correct Splicing DNA Triplet Toxic RNA Forms RNA Binds Clinical Repeats Foci in Nucleus Splicing Proteins Presentation Cytoplasm 449bp 479bp DMWD DMPK SIX5 Toxic DMPK mRNA MBNL Sequestered • Myotonia proteins Sequestered MBNL Toxic RNA is unable • Muscle wea
Slide 5: Robust Preclinical Data Support the Potential of DYNE-101 to Drive Disease Modification in the Clinic
Robust Preclinical Data Support the Potential of DYNE-101 to Drive Disease Modification in the Clinic PK PD Functional PK/Muscle Delivery Mutant DMPK Splicing Myotonia DYNE-101 Validated Cynomolgus NHP ✓ hTfR1/DMSXL hemizygous ✓ hTfR1/DMSXL homozygous ✓ HSALR homozygous ✓ DM1 patient-derived cells ✓
Slide 6: Phase 1/2 Clinical Trial to Evaluate DYNE-101 in Adults with DM1
Phase 1/2 Clinical Trial to Evaluate DYNE-101 in Adults with DM1 Population Primary Endpoints Key Secondary Endpoints Stages of ACHIEVE • Adult patients living with • Safety and tolerability • Pharmacokinetics • Multiple Ascending Dose DM1 • Change from baseline of: (MAD): 24 weeks • Ages 18 to 49 y
Slide 7: Randomization Stage of ACHIEVE
Multiple Ascending Dose Stage of ACHIEVE Global, Randomized, Placebo-Controlled Stage Evaluating Once Monthly or Less Frequent Administration of DYNE-101 in ~64 Adult Patients Living with DM1 10.2 mg/kg MAD Study Details N=8 (3:1) Q8W with Booster, Placebo 10.2 mg/kg • IV administration of DYNE- N=8
Slide 8: Acknowledgements
Acknowledgements ❖ ACHIEVE participants and their families ❖ ACHIEVE investigators and site staff ❖ Dyne co-authors Chris Mix Baoguang Han Ashish Dugar Wildon Farwell Visit us at poster 009 8