8.30 Virtual Coffee Networking

9:00 am Chairs Opening Remarks

9:10 am Panel Discussion – Biggest Learnings From Failed or Abandoned Clinical Trials

  • Maria Judit Molnarm Professor of Neurology, Psychiatry & Clinical Genetics, Director of the Institute of Genomic Medicine & Rare Disorders, , Semmelweis University
  • Debra Miller CEO, CureDuchenne


• Scrutinizing the disconnect between preclinical to clinical translation, why were there such promising results in preclinical yet poor translation into positive clinical trials?

• Why did they fail? What was the endpoint? What could have been done differently?

• What were the biggest lessons learned?

• What was surprising in terms of the outcome measures?

• What good can come out of a failed trial?


Discovering Innovative Industry Approaches to Neuromuscular Drug Development

9:50 am Developing an Antisense Inhibitor of the CD49d Receptor for DMD Patients

  • Mark Diamond Managing Director & CEO, Antisense Therapeutics


• Overview of ATL1102, designed to inhibit CD49d expression on lymphocytes and reduce their survival, activation and migration from the blood into sites of inflammation

• Why ATL1102 for DMD?

• Evaluating primary and secondary endpoints from the Phase II clinical trial of immunomodulatory therapy, ATL1102 for DMD

• Exploring MRI data showing how the outcomes on disease progression parameters exceeded expectations with stabilisation in the percentage of fat in muscles and preservation of functional muscle mass

• Progress towards late stage clinical development

10:15 am Pioneering a New Class of Oligonucleotide Therapies: Developing an Oligonucleotide Antibody Conjugate for the Treatment of DM1


• Exploring our AOC platform and how we can deploy various types of oligonucleotides whose specific mechanisms of action modify RNA function in different ways

• Outlining the advantages of our AOC’s and their activity

• AOC 1001, the lead program for DM1 and plans to submit an IND and initiate a Phase 1/2 clinical trial

10:40 am Live Q&A

11:00 Virtual Speed Networking

11:30 am Clinical Development of Fast Skeletal Muscle Troponin Activators for the Potential Treatment of Neuromuscular Diseases

  • Andrew A Wolff Senior Fellow, Clinical Research & Development, Cytokinetics


• Evaluating Cytokinetics’ selective fast skeletal muscle troponin activators – impacting the performance of skeletal muscle with investigational medicines

• Experience with the first selective fast skeletal muscle troponin activator, tirasemtiv


• Myasthenia gravis

• Calf claudication due to peripheral artery disease (maybe)

• Exploring our clinical pipeline and experience: Assessing reldesemtiv as a potential treatment for people living with ALS and SMA

• Plans for phase 3 study in ALS, patient enrolment and data readout

11:55 am Leveraging RNA Targeting CRISPR Therapeutics to Target Myotonic Dystrophy

  • Ranjan Batra Vice President of Research and Development, Locana


• Outlining the principles of Locana’s RNA Targeting approach and outlining its use in myotonic dystrophy (DM1) preclinical models and outlining behavioral and molecular biomarkers

• Global alternative splicing defects in DM1 to explore a battery of molecular endpoints, and how these correlate with regression of symptoms

• Evaluating data across different modalities including Cas9 and Cas13d

• Showing the breadth and opportunity as to how these systems are effective in the treatment of DM1 in animal studies, testing the safety and efficacy

12:20 pm Exploring the Elusive Promise of Myostatin Inhibition for Muscular Dystrophies

  • Kathryn Wagner Vice President, Global Head of Neuromuscular Disorders, F. Hoffmann-La Roche Ltd,


• Preclinical work in mouse models of muscular dystrophy demonstrated increased regeneration and decreased fibrosis from myostatin inhibition

• Clinical trials of selective and non-selective myostatin inhibitors failed to demonstrate increase in muscle function

• Various differences between animal models and human subjects may account for lack of translation of myostatin inhbitors for muscular dystrophy

12:45 pm Live Q&A

  • Andrew A Wolff Senior Fellow, Clinical Research & Development, Cytokinetics
  • Kathryn Wagner Vice President, Global Head of Neuromuscular Disorders, F. Hoffmann-La Roche Ltd,
  • Ranjan Batra Vice President of Research and Development, Locana

1:00 pm Lunch Break

1:40 pm Spearheading a Collaborative Effort to Drive Therapeutic Success for Patients in Need


• Exploring the key strategic goals for Project Hercules

• Building towards a global collaboration bringing together the patients, the regulators, the payers, pharmaceutical executives, HTA, clinicians and academics

• Exploring overall budget impact and how NICE dissects the impact of a new treatment on quality of life • What are other companies and regulators demanding?

• Pushing for faster, more evidence-based decisions

2:05 pm The Operational Challenges of Conducting an Interim Analysis During COVID-19

  • Sabine Pilot Head Clinical Operations, Santhera Pharmaceuticals

2:30 pm Data Collection at Point of Care – MDA MOVR Database Yields First Insights


-Non-profit infrastructure can be used for rigorous data collection
-Challenges in EHR integration and data use agreements
-Preliminary analysis leads to insights in uptake of new therapies and variability of care in DMD, SMA and ALS


2:55 pm Live Q&A

3:20 pm Chair’s Closing Remarks