Slide 1: CLEAR MIND Phase 2a Proof of Concept Topline Results
Cell - Based Therapies 05 October 2023 CLEAR MIND Phase 2a Proof Of Concept Topline Results
Slide 2: Forward-looking statements
• Certain statements in this presentation that are not historical facts are forward - looking statements made pursuant to the safe h arbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estim ate s of future opera
Slide 3: Study Schema
Study Schema 3 Screening Baseline Randomization 1:1:1:1 Infusion 1 - 4: Placebo Group 1 (n=12) Infusion 1: 25M Lomecel - B Infusions 2 - 4: Placebo Group 2 (n=12) Infusions 1 - 4: 25M Lomecel - B Group 3 (n=12) Infusions 1 - 4: 100M Lomecel - B Group 4 (n=12) Infusions: Day 0, Week 4, Week 8 & Week
Slide 4: Summary of Baseline Demographics
Summary of Baseline Demographics 4 All Patients (N=49) G4: 100M x 4 (N=11) G3: 25M x 4 (N=13) G2: 25M x 1 (N=13) G1: Placebo (N=12) Statistic ITT Population 74.1 (6.65) 74.5 (6.07) 70.5 (7.14) 74.9 (7.51) 76.7 (4.40) Mean (SD) Age (Years) 22 (44.9) 27 (55.1) 5 (45.5) 6 (54.5) 7 (53.8) 6 (46.2) 5 (38
Slide 5: Primary endpoint met: SAEs 4 weeks after any infusion
Primary endpoint met based on statistical and medical evaluation Primary Endpoint – SAEs 4 Weeks After Any Infusion 5 Pooled 3 and 4 (N=24) Pooled 2, 3 and 4 (N=37) G4: 100M x 4 (N=11) G3: 25M x 4 (N=13) G2: 25M x 1 (N=13) G1: Placebo (N=12) Statistic 1 Adverse Events ITT population 2 (8.3) (1.0, 27
Slide 6: Most Frequent TEAEs
Most Frequent TEAEs 6 All Patients N=(49) G4: 100M x 4 (N=11) G3: 25M x 4 (N=13) G2: 25M x 1 (N=13) G1: Placebo (N=12) Adverse Events ITT Population, n (%) 5 (10.2) 4 (8.2) 2 (18.2) 2 (18.2) 1 (7.7) 0 2 (15.4) 2 (15.4) 0 0 TEAEs COVID - 19 Urinary tract infection
Slide 7: Change in Composite Alzheimer's Disease Score (CADS)
Secondary Endpoint - CADS ( Composite Alzheimer’s Disease Score) 7 -0.45 -0.35 -0.25 -0.15 -0.05 0.05 0.15 0.25 0.35 C h a n g e i n C o m p o s i t e A l z h e i m e r ' s D i s e a s e S c o r e 0 16 26 39 Weeks (relative to first infusion) Pooled Lomecel-B (25M x 4, 100M x 4)Pooled Lomecel-B (25M
Slide 8: Left Hippocampal Volume
Secondary Endpoint – Left Hippocampal Volume* 8 • Statistically significant slowing of disease progression in left hippocampal volume at Visit 9 (Week 39) was observed in • Lomecel - B (25Mx1) treatment group relative to Placebo (p=0.015) • Pooled Lomecel - B treatment group (25Mx1, 25Mx4, 100Mx4) r
Slide 9: ADCS-ADL
Secondary Endpoint – ADCS - ADL 9 • Statistically significant improvement at Visit 9 (Week 39) in ADCS - ADL was observed in the • Lomecel - B (100Mx4) treatment group relative to Placebo (p=0.040) • Pooled Lomecel - B treatment group (25Mx4, 100Mx4) relative to Placebo (p=0.034) • Pooled Lomecel -
Slide 10: CDR-SB and ADAS-cog-13
Secondary Endpoint – CDR - SB and ADAS - cog - 13 10 • CDR - SB - all Lomecel - B treatment groups experienced greater benefit than placebo (not statistically significant) At Visit 9 (Week 39) • ADAS - cog - 13 - No statistically significant differences were observed between any Lomecel - B dose and
Slide 11: Conclusions
• Primary endpoint met based on statistical and medical evaluation ▪ Lomecel - B is safe, well tolerated and comparable to Placebo ▪ Lomecel - B demonstrated benefit over Placebo by preventing deterioration in cognitive and atrophy signals ▪ We are encouraged by the finding from the CLEAR MIND study