8AM - 5PM EST | 5PM - 2PM PST

8:00 am Online Registration

8:45 am Chair’s Opening Remarks

Examine the Oxidative Phosphorylation (OXPHOS) System/ Lipid (per) Oxidation of the Mitochondria

9:00 am Mitochondria as a Target for Ophthalmic Disease

Synopsis

  • Examining mitochondrial dysfunction as a key pathology in dry age-related macular degeneration, glaucoma, diabetic retinopathy and Fuch’s corneal endothelial dystrophy
  • Presenting preclinical data demonstrating elamipretide’s effect in models of ophthalmic disease
  • Showcasing clinical data showing that degree of baseline mitochondrial function may predict therapeutic response in patients with dry AMD
  • Demonstrating clinical data which shows how mitochondrial targeted therapeutics may not only slow disease progression, but also potentially improve visual function and/or visual field

9:30 am Using Precision Medicine in Clinical Trials to Advancing Therapeutics for both Degenerative & Rare Mitochondrial Diseases

Synopsis

  • Assessing clinical data with biomarker-correlated outcomes of ANAVEX2-73 (blarcamesine) in Alzheimer’s, Parkinson’s, and Rett Syndrome
  • Reviewing mechanistic analysis of involvement of mitochondrial function

10:00 am Designing Clinical Trials for Mitochondrial Disease Therapies

Synopsis

  • Understanding how drug development for mitochondrial disease is fraught with challenges due to disease heterogeneity, lack of comprehensive natural history and lack of precedent of approved therapies to guide development
  • Examining how careful consideration of inclusion criteria to minimize heterogeneity and enrich study participants will be most likely to respond to a therapeutic intervention which is essential
  • Learning how trade-offs exist when choosing to include a limited number vs. a larger number of study sites
  • Exploring how ehe COVID-19 pandemic has led to an evolution in study design and conduct

10:30 am Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office and jump straight into the speed networking session. This is your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

11:00 am Targeting Mitochondrial Oxidative Stress in Dry Eye & Glaucoma

Synopsis

  • Examining the role of mitochondrial oxidative stress and cardiolipin peroxidation in Dry Eye Disease and Open Angle Glaucoma
  • Presenting late-stage clinical data from VISTA-1 and VISTA-2 clinical studies of SkQ1 ophthalmic solution in Dry Eye disease
  • Showcasing early-stage clinical data for SkQ1 ophthalmic solution in Glaucoma and therapeutic potential of a mitochondria-targeted IOP lowering drug

11:30 am Mitochondrial Protective Therapeutics for Neurodegenerative & Metabolic Diseases

Synopsis

  • Presenting mitochondrial dysfunction as a hallmark mechanism across rare and challenging diseases
  • Examining the importance of mitochondrial homeostasis in high energy systems – CNS, retina, heart, skeletal muscle, kidney
  • Reviewing a novel small molecule approach to treating mitochondrial dysfunction
  • Analyzing preclinical data in ophthalmic indications of retinitis pigmentosa and dry age-related macular degeneration

12:00 pm Inhibition of Neuroinflammation & Neurodegeneration by a Selective Inhibitor of Mitochondrial Pathological Fission

  • Daria Mochly-Rosen The George D Professor, Translational Medicine, Professor, Dept Chemical & Systems Biology/Founder & Co-director, Stanford University, School of Medicine/SPARK, Stanford

Synopsis

  • Examining mitochondrial dysfunction as a key pathology in ALS
  • Evaluating how excessive mitochondrial fragmentation, seen in patient-derived cells and in a mouse model of the disease, can be blocked by inhibiting the interaction of the mitochondrial fission enzyme, dynamin related protein 1 (Drp1), and one of its adaptors on the mitochondrial surface, Fis1
  • Observing how P110, a heptapeptide inhibitor of this Drp1/Fis1 interaction that we have rationally designed, selectively inhibits mitochondrial fragmentation and dysfunction in the above models, inhibits microglial and astrocytes activation and neuroinflammation and improves the clinical symptoms in ALS mice, without causing any toxicity in healthy mice

12:30 pm Lunch Break

1:30 pm Aging Mitochondria, Aging Cells, & Human Disease

Synopsis

  • Understanding how cell aging underlies age-related human disease
  • Exploring how mitochondrial dysfunction is a prominent – although not the sole – feature of cell aging
  • Examining the key question that still remains the cause of – the “upstream” events behind – mitochondrial dysfunction
  • Understanding the underlying processes presents an optimal point of clinical intervention

Mitochondrial Gene Therapy & Editing: A Promising Therapeutic Technology

2:00 pm Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON):

Synopsis

  • Exploring the main clinical aspects of LHON, a rare and acute mitochondrial disease
  • Evaluating late-stage development of Lumevoq gene therapy
  • Discussing on regulatory pathways
  • Highlighting key learnings

Precision/Patient-Focused Medicine – Standardizing & Improving Patient Outcomes

2:30 pm Development of a New Medication for the Ultra-Rare Mitochondrial Disease Thymidine Kinase 2 Deficiency Disorder (TK2d)

  • David Schaaf Vice President, US Medical Affairs, Senior Medical Officer, Zogenix

Synopsis

  • Reviewing TK2d disease whilst navigating natural history
  • Evaluating the creation of NDA-supporting dataset
  • Examining top-line results supporting NDA-submission

3:00 pm Afternoon Coffee Break

3:30 pm Patient-Focused Drug Development for Mitochondrial Disease

Synopsis

  • Highlighting the impact of patient input on research and development at Abliva
  • Providing an overview of the multiple therapeutic options for mitochondrial disease being pursued at Abliva
  • Presenting placebo controlled PMD patient data from our lead asset, KL1333

4:00 pm Panel Discussion: Emerging Treatments for Chronic Diseases when Targeting Mitochondria– Therapeutic Avenues, Clinical Trials & Future Direction

  • Keshav Singh Joy & Bill Harbert Endowed Chair & Professor/Scientific Founder & Chief Scientific Officer, University of Alabama, Birmingham/Yuva Biosciences

Synopsis

  • Examining the current treatment options and emerging targeted therapies
  • How can the industry design novel therapeutic strategies for the treatment of chronic diseases when targeting mitochondria?
  • Exploring clinical trial development and the continued impact of the pandemic on on-going clinical trials

4:45 pm Chair’s Closing Remarks

5:00 pm End of Summit