Key Themes from the 2nd Annual Cell Therapy for Autoimmune Disease Summit

Lumanity Strategy Consulting recently participated in the 2^nd Annual Cell Therapy for Autoimmune Disease Summit in early December. Here’s our interpretation of some of the key themes coming out of the meeting.

Key Themes

• Growing Momentum: The conference expanded greatly from the previous year as there continues to be growing momentum of companies making a switch/entry into autoimmune diseases with cell therapy technologies. This has prompted investigation into an expanded set of target diseases beyond the few initial focus indications as well as an increase in research and clinical trials focusing on a variety of cell therapy types/moieties beyond CAR-T, namely NK- or Treg-cell therapies or T cell engagers which offer potential to overcome some limitations of autologous CAR-T (e.g., scalability/off-the-shelf potential, decreased potential for CRS, etc.).

• Technological Advancements: Companies/innovators are getting increasingly savvy with the refinement of cell engineering techniques to improve specificity and safety of cell therapy approaches. This is especially important in autoimmune diseases where physicians and patients have a lower tolerance for side effects compared to oncology. For example, the use of mRNA-based CAR T-cell therapies has the potential to expand the reach of cell therapy products as it requires no lymphodepletion, can be administered in the outpatient setting, can be delivered at a therapeutic level, and does not carry risk of genomic integration.

• Clinical Promise: Preliminary efficacy and safety has been demonstrated across clinical trials from multiple companies developing cell therapies for autoimmune diseases. These early trials reveal potential for durable disease remission in select patient populations with safety profiles that could support outpatient administration. Indeed, one of the most frequent and exciting discussions around safety at the conference was regarding BMS’ Phase 1 trial of a CD19-directed CAR T-cell therapy in which a 30-year-old female patient with SLE became pregnant shortly after CAR-T infusion, successfully remained off all lupus-directed medications, and had an uncomplicated delivery of a healthy baby boy.

• Facing the Facts: Broadly, there was a concern within the audience that large industry-sponsored clinical trials may not achieve the same level of efficacy and safety that was observed by Dr. Schett, whose data from a controlled environment at a single site in Germany catalyzed this space. Conference attendees from executives to scientists emphasized a need for level-setting expectations for various stakeholders including investors, patients, caregivers, physicians, payers, etc., on understanding “real world” evidence of autologous CAR T-cell therapies vs. Dr. Schett’s data. Despite this, there still seems to be resounding enthusiasm for the clinical results that have been achieved.

Conclusion

It’s obvious that the field of cell therapy for autoimmune diseases is rapidly evolving, bringing hope and excitement to this space.  However, an important observation from both the conference attendance and closely following/working with companies in this space is that there are several key challenges that are not always being discussed.  For example, presentations and investor calls have focused primarily on scientific or clinical evidence, but have largely avoided bringing attention to or addressing what we believe will be potentially meaningful obstacles to commercializing cell therapies, particularly autologous CAR-T.   While we don’t have a crystal ball on who the winners will be in this competitive and innovative space, we are confident that companies who succeed are likely prepared and equipped to manage the logistical, access, and commercial challenges that lie ahead. 

Check out our recent whitepaper “The Cellution to Autoimmunity? The promise and perils of CAR-T”, where we dive deep into these market challenges.  Interested in learning more? Reach out to contact@lumanity.com to chat with us about cell therapies for autoimmune disease, conference coverage capabilities, and more!