Longeveron Issues Letter to Shareholders Highlighting Corporate Strategy, Clinical Pipeline and 2024 Key Priorities and Goals - Seite 2

At present, we have completed approximately 60% of the enrollment of our randomized Phase 2 clinical trial that builds upon our successful Phase 1 trial. Our Phase 2 trial will compare outcomes of babies treated with the standard of care (SOC) plus Lomecel-B^TM vs. the SOC alone. We expect this trial to finish enrollment in 2024 with data read-out at the end of 2025. We are laser focused on the effective execution of this program and we will be communicating with the FDA regarding the potential to use this trial as a pivotal trial for accelerated or final approval of Lomecel-B^TM for HLHS.

Alzheimer’s Disease:

In our Alzheimer’s Disease (AD) program, we recently completed our Phase 2a CLEAR-MIND trial with 49 patients that builds upon our successful Phase 1 trial. The results of our CLEAR MIND trial provided encouraging signals of efficacy that clearly warrant further investigation.

As such, we are aggressively pursuing partnerships and funding opportunities to move this exciting program forward. Given the massive unmet need presented by AD, large resources are potentially available from federal and private funding sources. We will be heavily focused on seeking partnership opportunities and/or non-dilutive funding for this program.

Our Core Business Objectives:

1. Focus on HLHS as the Main Value Driver:
   • We endeavored to strategically position our Phase 2 study for HLHS as a pivotal head-to-head study, with potential for accelerated or full FDA approval, reflecting our commitment to expedite treatment access.
   • We plan to leverage existing relationships with several treating surgeons in order to maximize our commercialization planning and potential future launch in HLHS.
   • Recognizing substantial revenue potential from worldwide commercialization, we anticipate capitalizing on recent rare disease pricing trends and the unique aspects of our treatment.
   • With in-house manufacturing capacity adequate to meet anticipated global supply demands, we expect that scale-up expenses for commercialization of this indication will be substantially reduced.
   • Possession of a Rare Pediatric Disease priority review voucher associated with HLHS upon successful FDA approval could have significant monetary value and further enhance our product’s value proposition upon approval.
2. HLHS Commercialization and BLA Readiness:
   • In 2024, there will be increased efforts and focus on organizational readiness for potential BLA filing for HLHS in 2026. These efforts are expected to include strengthening processes, conducting mock audits, and enhancing regulatory interactions which are paramount to expedite BLA readiness and ensure compliance.
3. Strategic Collaborations:
   • While Alzheimer’s Disease programs hold immense potential, their larger-scale studies and commercialization requirements due to the large size of the affected population necessitate significant investment, prompting our focus on potential strategic partnerships with biotech/pharmaceutical companies or pursuit of grants and other non-dilutive funding sources.
   • Our openness to collaborations with public and private organizations aligns with our mission of advancing therapies across various therapeutic areas.
4. Leveraging Science and Manufacturing Capabilities:
   • With our significant manufacturing capabilities and expertise, we intend to capitalize on these assets to provide greater strategic diversification and generate additional revenue by further soliciting and engaging in contract development and manufacturing services (CDMO) for third parties.
   • We plan to continue investment in new products and technologies supportive of innovation and potency assay development and fostering long-term competitiveness and product differentiation.
5. Efficient Resource Management:
   • We plan to continue pursuing strategic advancement as much as possible without increasing headcount in order to maximize efficiency and alignment with strategic priorities.
   • Our termination of the Japan Aging-related Frailty study reallocates resources to our priority programs in order to optimize resource utilization and ensure strategic focus.