Slide 1: PRAX-944 Essential Tremor Phase 2a Part B Topline Results
PRAX-944 ESSENTIAL TREMOR Phase 2a Part B Topline Results May 9, 2022
Slide 2: Forward-looking statements
2 Forward-looking statements This presentation may contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to express or implied statements regarding the
Slide 3: PRAX-944 Phase 2a Part B open-label and randomized withdrawal study design
3 PRAX-944 Phase 2a Part B open-label and randomized withdrawal study design DAYS 29-42 1:1 RANDOMIZATION Open-Label Titration of PRAX-944 up to 120 mg Stable Period at High Dose PRAX-944 PLACEBO RANDOMIZED WITHDRAWAL (DAYS 43-56) DAYS 57-70DAYS 1 -28 Screening/ Baseline Safety Follow- up Topline Re
Slide 4: Patient disposition
4 PRAX-944 Phase 2a Part B patient disposition 1 Site suspended due to protocol violations, no evaluable data for three patients after Day 1; See Slide 10 for safety data 2 Three patients discontinued at the 40mg (escalation), 60mg (escalation), and 40mg (down-titration) dose level 3 Three patients
Slide 5: Baseline demographics
5 BASELINE DEMOGRAPHICS EVALUABLE PARTICIPANTS1 (N=14) COMPLETED (N=11) Age, mean (range) 59 (26-76) 62 (43-76) Gender (Male/Female) (n, %) 11/3 (79%/21%) 8/3 (73%/27%) # previously on ET medication (n, %) 9 (64%) 6 (55%) # currently on ET medication (n, %) 3 (21%) 2 (18%) Family History – First-deg
Slide 6: Modified ADLs in open label and randomized withdrawal
6 MODIFIED ADLs IN RANDOMIZED WITHDRAWAL MEAN % CHANGE FROM DAY 42 Marked functional benefit observed on treatment, with withdrawal resulting in regression to baseline severity MODIFIED ADLs IN OPEN LABEL MEAN % CHANGE FROM BASELINE *Nominal p-value based on ANCOVA Source: Praxis Data on file *p<0.0
Slide 7: Kinesia ONE in open label and randomized withdrawal
7 KINESIA ONE IN OPEN LABEL MEAN % CHANGE FROM BASELINE KINESIA ONE IN RANDOMIZED WITHDRAWAL MEAN % CHANGE FROM DAY 42 Functional benefit with PRAX-944 supported by tremor analysis -24% 6% Day 42 (N=11) Day 70 (N=11) PRAX-944 TREATED OFF DRUG 29% 3% Placebo PRAX-944 n/s Day 42 (N=11) Day 56 (N=6 PRA
Slide 8: Example of spiral task from Part B patient
8 EXAMPLE OF SPIRAL TASK FROM PART B PATIENT Impact of PRAX-944 on ability to draw Day 42 On PRAX-944 Day 56 Blinded Off Drug Day 1 Baseline
Slide 9: Safety summary
9 SAFETY SUMMARY PRAX-944 was generally well tolerated with no new safety findings 1 Participant had a pre-existing condition which was unrelated to study drug and required a medical procedure 2One severe AE of essential tremor reported while on placebo following withdrawal of PRAX-944; all other AE
Slide 10: Treatment-emergent adverse events in >1 participant
10 TREATMENT-EMERGENT ADVERSE EVENTS (TEAEs) IN >1 PARTICIPANT PRAX-944 Phase 2a Part B - TEAE summary 1 Excludes the three non-evaluable patients from site suspended due to protocol violations; One severe AE of essential tremor reported while on placebo following withdrawal of PRAX-944; all other A
Slide 11: TEAEs leading to dose down-titration
11 TEAEs LEADING TO DOSE DOWN-TITRATION* TEAEs leading to dose down-titration or discontinuation were mild to moderate *Protocol permitted patients to dose titrate down once during Part B 1 Participant also down-titrated prior to discontinuation 2 Participant had a pre-existing condition which was u
Slide 12: Six phase or registrational topline readouts in 2022
12 Six phase 2 or registrational topline readouts in 2022 PRAX-114 Phase 2/3 Aria Study topline results expected in June 2022; PRAX-944 Phase 2a Part B topline results disclosed in May 2022 FOCUS AREA PROGRAM INDICATION Q2 2022 Q3 2022 Q4 2022 EPILEPSY MOVEMENT DISORDERS PSYCHIATRY MDD PTSD PRAX-114