7:50 am Check In, Coffee & Light Breakfast
8:50 am Chair’s Opening Remarks
NAVIGATING COMMERCIAL BOTTLENECKS TO DETERMINE ACCESS TO TREATMENT FOR PEOPLE LIVING WITH ALS
9:00 am Panel Discussion: Promoting Earlier Commercial Discussions to Accelerate the Post-Approval Path to Market
Synopsis
- What must be considered when deciphering commercially viable targets for ALS?
- How can we ensure access to promising, yet expensive, ALS treatments to patients who really need them?
- Why is it important to assess market viability during the early stages of initial R&D discussions?
9:45 am Session Reserved: Answer ALS
10:15 am Morning Break & Refreshments
REVITALIZING IN VIVO MODELS FOR REPLICATING MOTOR NEURONAL PATHOLOGIES
Synopsis
Chair: Mansuo Lu Shannon, Chief Scientific Officer, Prevail Therapeutics, a Wholly Owned Subsidiary of Eli Lilly
10:45 am Novel Humanized Mouse Models of ALS Recapitulate the Human Molecular & Pathological Hallmarks of ALS But Overcome the Artifacts Associated with TDP-43 Over-Expression
Synopsis
- Overexpression of TDP-43 leads to aggressive phenotypes and potential artifacts that are not related to ALS
- Humanized C9orf72 and TDP-43 mouse models show degeneration, TDP-43 mislocalization, aggregation and mis-splicing of TDP-43 targets
- These models can be used to rapidly screen therapeutics that restore TDP-43 function
REVIEWING THE LATEST PRECLINICAL PROGRESS OF NOVEL ALS TARGETS
11:15 am Session Reserved: bit.bio
11:30 am Showcasing SOL-257, a Chaperone-based Gene Therapy Targeting Misfolded TDP-43 in ALS
Synopsis
- Highlighting in vivo proof-of-concept data demonstrating successful re-folding or degradation of misfolded TDP-43
- Outlining corroborating iPSC-derived evidence
- Demonstrating translational potential of SOL-257 gene therapy to treat TDP-43 dysfunction in ALS patients
KEEPING PACE WITH THE EVOLVING APPROVAL LANDSCAPE FOR ALS THERAPEUTICS
Synopsis
Chair: Sharon Tamir, Digital Healthcare Innovation, Business & Research Strategy, Mitsubishi Tanabe Pharma Americaa
10:45 am Panel Discussion: Striving for Consensus on the Level of Evidence Required for Approval
Synopsis
- How can we best reconcile the variance in acceptable measures of ALSFRS-R decline across geographies to get drugs to patients faster?
- How translatable is success of accelerated approval in familial ALS to sporadic ALS?
- When are expanded access programs deemed acceptable ahead of final study results?
- If there’s going to be a move towards accelerated approvals based on surrogate endpoints, what else needs to be done to get full approval?
EXPLORING EMERGING CLINICAL ALS PIPELINE INNOVATION
11:30 am Characterizing Safety & Proof-Of-Concept for VRG50635, a Novel Small Molecule Inhibitor ALS Candidate Therapeutic Targeting PIKfyve
Synopsis
- Rationale for testing PIKfyve inhibition in ALS
- Learnings of safety, tolerability and PK/PD in healthy volunteers
- Design of a novel proof-concept study for the initial investigation of VRG505635 in ALS
12:00 pm Lunch & Networking
1:10 pm Translating A Non-Viral Anti-Inflammatory Gene Therapy into ALS Mouse Models & Preparing for First In-Human Studies
Synopsis
- Developing a unique therapeutic approach of targeting IL-10 with a non-viral gene therapy
- Showcasing preclinical data of anti-inflammatory effect in TDP-43 mouse models
- Outlining future directions for translation into phase 1 clinical study
1:40 pm Reinventing SOD1 Targeting with CRISPR-Based Gene Editing Technology to Diminish Mutant SOD1 & Restore Motor Neuronal Function
Synopsis
- Demonstrating disruption to the human SOD1 transgene using in vivo gene editing technology
- Revealing functional restoration of motor neurones following intracerebroventricular injection
- Utilizing this CRISPR editing preclinical approach for target evaluation
2:10 pm Demonstrating Success of a One-Dose Gene Therapy to Prevent Motor Neuronal Loss in Both Sporadic & Familial ALS
Synopsis
- Transitioning from monogenetic to sporadic populations: transformative progress to demonstrate efficacy in rodents
- Outlining implications for ‘what’s next’, to drive AAV-UPF1 into clinical sporadic and familial ALS populations
1:10 pm Coya 302: A Dual Mechanism Biologic Immunotherapy in the Treatment of ALS By Treg Activity to Reduce Inflammation
Synopsis
- Treg dysfunction is a core driver of neurodegeneration in ALS
- Coya 302 immunotherapy targets adaptive and innate immune pathways in ALS with strong efficacy signal in an open label exposure trial
- Advancing Coya 302 in a double-blind placebo controlled clinical trial in ALS
1:40 pm Showcasing Clinical Success of a Novel STMN2-Targeting ASO to Restore Functional Expression & Promote Motor Neuronal Repair
Synopsis
- Outlining latest advancements in the QRL-201 program
- Achieving successful restoration of STATHMIN-2 expression in ALS patients
- Highlighting upcoming steps in the advancement of QRL-201 towards later phase studies
2:10 pm Novel Multi-functional Small Molecule TBN Proves Promising in ALS Phase II Trial
Synopsis
- Optimizing recruitment strategy to achieve a significantly large phase 2 cohort of Chinese patients
- Outlining the observed effects on grip strength and other key markers of ALS progression
- Advancing TBN into a phase 3 study in China
2:40 pm Afternoon Break & Refreshments
FUTURE DIRECTIONS & RESEARCH PRIORITIES: WHAT WILL THE ALS LANDSCAPE LOOK LIKE IN YEARS TO COME?
3:10 pm Lessons Learned & Future Implications from the Zilucoplan Regimen in the HEALEY ALS Platform Trial
Synopsis
- Results of the zilucoplan regimen in the Healey ALS Platform Trial
- Considerations for joint placebo group and interim analyses in perpetual trial
- Aspects of academic-industry collaboration
3:40 pm Panel Discussion: Exploring Ongoing Research Efforts Globally in ALS: How Will Future Study Read-Outs Transform the ALS Landscape?
Synopsis
- How are different research consortia implementing the Act for ALS, and what advancements are anticipated as a result?
- From expanded access programs to natural history studies, in what ways might these investigations influence the trajectory of ALS drug development?
- What are the current leading research priorities aimed at uncovering effective interventions for diagnosing, treating, managing, preventing, or ultimately curing ALS?