Conference Day One
November 20, 2024

8:00 am Check In, Morning Coffee & Light Breakfast

8:45 am Chair’s Opening Remarks

CAPITALIZING ON OUR UNDERSTANDING OF MITOCHONDRIAL BIOLOGY

9:00 am Deciphering the Mitochondrial Genome to Model Mitochondrial DNA Mutations

Synopsis

  • Unraveling the intricacies of mitochondrial DNA and associated mutations to uncover disease mechanisms
  • Presenting methods related to de novo mutagenesis in mitochondrial DNA to implement correction
  • Characterizing molecular phenotypes in mitochondrial DNA-edited mouse models

9:30 am Cracking the Code With Mitochondrial Transplant: Reversing the Age of the Immune System

Synopsis

  • Unpicking cellular energetics to understand disease indications and drug translation in older patients
  • Examining how transplanted mitochondria enhance cellular function and survival for evolutionary advantages
  • Discovering Mitrix Bio’s approach to demonstrating age-reversal effects in the immune system of older mice to highlight the potential benefits for elderly human patients

10:00 am Morning Refreshments & Speed Networking

NARROWING THE PRECLINICAL & CLINICAL GAP TO TACKLE MITOCHONDRIAL DYSFUNCTION

11:00 am ALS, Secondary Progressive MS & Alzheimer’s Disease: Commonly Rooted in Mitochondrial Dysfunction in a 14-Day MP-101 Clinical CSF Biomarker Basket Study

  • John Geisler Chief Scientific Officer, Mitochon Pharmaceuticals

Synopsis

  • MP-101: Lowers damage while promoting repair
  • MP-101: Mitochondrial-specific, biophysics MOA, non-genomic pharmacology cascading into cellular remodeling
  • MP-101: Oral brain penetrating once-per-day treatment waking up compensatory mechanism Pro-Survival

11:30 am Micro-Dose MP-101 Preserves Motor & Muscle Function in ALS Mice With Potential Functional Recovery After Disease Onset

Synopsis

  • Micro-dose MP-101 delays ALS disease onset and progression in hSOD1G93A mice
  • ALS mice treated with MP-101 exhibit improved motor function, muscle innervation and muscle contractile function
  • MP-101 treatment in hSOD1G93A from disease onset promotes recovery in mouse running ability

11:50 am Mild Mitochondrial Uncoupling Attenuates Cerebral Mitochondrial Dysfunction After Traumatic Brain Injury

Synopsis

  • Mild mitochondrial uncoupling with MP-201 mitigates oxidative stress in both contusive and concussive traumatic brain injury (TBI)
  • Mitochondrial bioenergetic improvements are observed in a dose-dependent manner after TBI
  • TBI-induced behavioral impairments are mitigated with MP-201 treatment

12:10 pm Lunch & Networking

1:10 pm MP-201 Decreases Neuropathic Pain

  • Karin Westlund Distinguished Professor & Vice Chair for Research, University of New Mexico Health Sciences Center

Synopsis

  • MP-201 attenuates neuropathic pain determined in a chronic trigeminal craniofacial pain model
  • Molecular analysis of trigeminal sensory neurons from mice treated in vivo with MP-201 finds 98 unregulated and 33 downregulated genes

EXPEDITING THE DEVELOPMENT OF MITOCHONDRIAL THERAPEUTICS

1:30 pm Panel Discussion: Translating Modality Into Value for Patients to Establish Clearly Defined Clinical Objectives

Synopsis

  • Building on the industry’s failures to re-evaluate how to select and measure the right clinical outcome
  • Steering the direction of research to acquire robust data that can illustrate clinical translatability and expedite discovery towards Phase I of clinical trials
  • Positioning patients at the heart of the modality to demonstrate its viability, thereby eliminating the need for a cost-risk analysis

2:15 pm Afternoon Refreshments & Networking

2:45 pm Modulating Novel Mitochondrial Targets to Treat Mitochondrial Disease, Diseases of Aging & Metabolism

Synopsis

  • Unveiling small molecule activators of the mitochondrial DNA polymerase, POLG, to treat mitochondrial DNA depletion syndromes and beyond
  • Exploring inhibitors of the mitochondrial RNA polymerase, POLRMT, to treat obesity and metabolic disorders
  • Leveraging a unique mitochondrial biology discovery platform to develop a pipeline of novel therapeutics

PROTECTING THE POWERHOUSE OF THE CELL

3:15 pm Preserving Sight in Patients With Neurodegenerative Diseases Through a First-in-Class Target

Synopsis

  • Highlighting the implications of mitochondrial dysfunction underlying the pathological basis for tissue damage in retinal diseases
  • Building a library of mitochondrial protective compounds to design a new class of small molecules that can treat neurodegeneration through the protection of mitochondrial homeostasis

3:45 pm Chair’s Closing Remarks

4:00 pm End of Day One