Trial Outcomes & Findings for Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS (NCT NCT05349721)

Last Updated: 2025-08-01

Results Overview

The CAFS is a composite endpoint based on time to earlier occurrence of death and change from baseline in ALSFRS-R score. ALSFRS-R is a rating scale where 12 functions were rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome in a pairwise fashion by time to death and change on ALSFRS-R, assigned a score which was sum of comparisons (+1 \[better\], 0 \[tie\], -1 \[worse\]), and summed scores were ranked, from 1 to 306 (ITT1 Analysis Set) lowest rank corresponds to participant who died first and highest rank to the participant with best ALSFRS-R outcome among those who survived. Multiple imputation was used to impute participants with missing ALSFRS-R score at Week 24. A higher rank was considered a better outcome. Least square (LS) means and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

336 participants

Primary outcome timeframe

Week 24

Results posted on

2025-08-01

Participant Flow

The study included Treatment (Part A), long-term extension (LTE) (Part B), and Continued LTE (Part C) periods.

Participant milestones

Participant milestones
Measure	Utreloxastat Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Part A (24 Weeks) STARTED	222	114
Part A (24 Weeks) Received at Least 1 Dose of Double-blind Study Drug in the Part A	220	114
Part A (24 Weeks) COMPLETED	179	100
Part A (24 Weeks) NOT COMPLETED	43	14
Part B (28 Weeks) STARTED	173	100
Part B (28 Weeks) Received at Least 1 Dose of Open-label Utreloxastat During Part B	173	100
Part B (28 Weeks) COMPLETED	38	27
Part B (28 Weeks) NOT COMPLETED	135	73
Part C (108 Weeks) STARTED	35	27
Part C (108 Weeks) Received at Least 1 Dose of Open-label Utreloxastat During Part C	35	27
Part C (108 Weeks) COMPLETED	0	1
Part C (108 Weeks) NOT COMPLETED	35	26

Reasons for withdrawal

Reasons for withdrawal
Measure	Utreloxastat Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Part A (24 Weeks) Adverse Event	9	2
Part A (24 Weeks) Death	8	3
Part A (24 Weeks) Disease Progression	1	1
Part A (24 Weeks) Screen Failure	2	0
Part A (24 Weeks) Withdrawal by Subject	18	7
Part A (24 Weeks) Other Than Specified	5	1
Part B (28 Weeks) Adverse Event	3	0
Part B (28 Weeks) Death	21	11
Part B (28 Weeks) Disease Progression	2	1
Part B (28 Weeks) Lost to Follow-up	2	1
Part B (28 Weeks) Physician Decision	1	0
Part B (28 Weeks) Study terminated by sponsor	85	53
Part B (28 Weeks) Withdrawal by Subject	17	5
Part B (28 Weeks) Other Than Specified	4	2
Part C (108 Weeks) Death	0	1
Part C (108 Weeks) Lost to Follow-up	0	1
Part C (108 Weeks) Study terminated by sponsor	34	23
Part C (108 Weeks) Withdrawal by Subject	1	1

Baseline Characteristics

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Utreloxastat n=220 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=114 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Total n=334 Participants Total of all reporting groups
Age, Continuous	58.0 years STANDARD_DEVIATION 10.97 • n=5 Participants	57.1 years STANDARD_DEVIATION 9.88 • n=7 Participants	57.7 years STANDARD_DEVIATION 10.61 • n=5 Participants
Sex: Female, Male Female	77 Participants n=5 Participants	28 Participants n=7 Participants	105 Participants n=5 Participants
Sex: Female, Male Male	143 Participants n=5 Participants	86 Participants n=7 Participants	229 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	27 Participants n=5 Participants	19 Participants n=7 Participants	46 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	189 Participants n=5 Participants	93 Participants n=7 Participants	282 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	183 Participants n=5 Participants	96 Participants n=7 Participants	279 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African Descent	5 Participants n=5 Participants	0 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian Descent	10 Participants n=5 Participants	5 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	22 Participants n=5 Participants	13 Participants n=7 Participants	35 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 24

Population: ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population)	172.22 units on a scale Standard Error 13.25	165.36 units on a scale Standard Error 14.65

SECONDARY outcome

Timeframe: Week 24

Population: ITT2 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period.

The CAFS is a composite endpoint based on time to earlier occurrence of death and change from baseline in ALSFRS-R score. ALSFRS-R is a rating scale where 12 functions were rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome in a pairwise fashion by time to death and change on ALSFRS-R, assigned a score which was sum of comparisons (+1 \[better\], 0 \[tie\], -1 \[worse\]), and summed scores were ranked, from 1 to 334 (ITT2 Analysis Set) lowest rank corresponds to participant who died first and highest rank to the participant with best ALSFRS-R outcome among those who survived. A higher rank was considered a better outcome. LS mean and SE were calculated using ANCOVA model.

Outcome measures

Outcome measures
Measure	Utreloxastat n=220 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=114 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Combined Assessment of Function (ALSFRS-R) and Survival (CAFS) Rank After 24 Weeks of Treatment (ITT2 Analysis Population)	188.58 units on a scale Standard Error 14.81	181.89 units on a scale Standard Error 15.96

SECONDARY outcome

Timeframe: Baseline, Week 24

The ALSFRS-R is a quickly administered (5-minute) ordinal rating scale that assesses the participant's capability and independence in 12 functional activities across 4 subdomains of bodily function (bulbar, gross motor, fine motor, and respiratory) relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0 (total loss of function) to 4 (no loss of function), with the total score ranging from 0 to 48 and higher scores indicating less functional impairment. LS mean and SE were calculated using mixed model repeated measures (MMRM).

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population)	29.4 units on a scale Standard Error 0.73	31.4 units on a scale Standard Error 0.93

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: ITT2 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period.

Outcome measures

Outcome measures
Measure	Utreloxastat n=220 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=114 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population)	27.7 units on a scale Standard Error 0.71	29.1 units on a scale Standard Error 0.88

SECONDARY outcome

Timeframe: Day 1 through Week 24

Population: Safety set included all randomized participants who received at least 1 dose of study drug.

An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that began after the first administration of study drug or any existing AEs that worsened after the first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

Outcome measures

Outcome measures
Measure	Utreloxastat n=220 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=114 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	174 Participants	90 Participants

SECONDARY outcome

Timeframe: Baseline, Week 24

The SVC is a measure of breathing function. SVC measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. The percent of predicted SVC is reported. LS mean and SE were calculated using MMRM.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in Percent Predicted Slow Vital Capacity (SVC) at Week 24	-14.62 percent predicted SVC Standard Error 2.106	-15.92 percent predicted SVC Standard Error 2.518

SECONDARY outcome

Timeframe: Baseline, Week 24

Modified Norris scale is used to assess the motor/limb and bulbar function. The Modified Norris Scale is a rating scale for ALS that consists of 2 parts: the Limb Norris Scale and the Norris Bulbar Scale. The Limb Norris Scale has 21 items to evaluate extremity function, and the Norris Bulbar Scale has 13 items to evaluate bulbar function. Each item was rated in 4 ordinal categories, corresponding to the following values and ratings or functional scores: normal (3 points), somewhat impaired (2 points), inadequate (1 point), and "cannot do at all" (0 points). The total score was calculated by summing all the scales, ranging from 0 (worst) to 102 (best) where higher scores indicated better functional abilities. LS mean and SE were calculated using MMRM.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in Modified Norris Scale Total Score at Week 24	-18.2 units on a scale Standard Error 1.57	-18.0 units on a scale Standard Error 1.82

SECONDARY outcome

Timeframe: Baseline to Week 24

Overall survival rate was defined as percentage of participants who were alive at given timepoint. Overall survival was defined as the time in months from the date of first dose to the date of death from any cause or date last known alive for those who did not die.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Overall Survival Rate	95.8 percentage of participants Interval 92.9 to 98.6	97.9 percentage of participants Interval 95.1 to 100.0

SECONDARY outcome

Timeframe: Baseline to Week 24

Overall survival was defined as the time in months from the date of first dose to the date of death from any cause or date last known alive for those who did not die.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Overall Survival	NA months Due to smaller number of participants with an event, median and 95% confidence interval (CI) could not be calculated.	NA months Due to smaller number of participants with an event, median and 95% CI could not be calculated.

SECONDARY outcome

Timeframe: Baseline, Week 24

The ALSAQ-40 is a disease-specific measure of health-related quality of life (QOL) for ALS. The ALSAQ-40 is comprised of 40 questions measuring 5 discrete dimensions of health status that are affected by the disease: physical mobility (10 items); activities of daily living and independence (10 items); eating and drinking (3 items); communication (7 items); emotional reactions (10 items). Participants were asked to indicate the frequency of each event by selecting one of 5 options (Likert scale: 0-4): never/rarely/sometimes/often/ always or cannot do at all. The total score ranging from 0 (no impairment) to 160 (severe impairment) was calculated by adding the 5 domain scores. A lower score indicates a better health state. LS mean and SE were calculated using MMRM.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in ALS Assessment Questionnaire (ALSAQ-40) Total Score at Week 24	27.9 units on a scale Standard Error 3.05	23.9 units on a scale Standard Error 3.48

SECONDARY outcome

Timeframe: Baseline, Week 24

The NfL is a marker of axonal degeneration and is robustly elevated in the blood of many neurological and neurodegenerative conditions.

Outcome measures

Outcome measures
Measure	Utreloxastat n=202 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo n=104 Participants Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Change From Baseline in Neurofilament Light Chain (NfL) Activity at Week 24	69.98 picograms (pg)/milliliter (mL) Standard Error 6.69	64.75 picograms (pg)/milliliter (mL) Standard Error 7.08

SECONDARY outcome

Timeframe: Day 1 and Day 29

Population: Pharmacokinetic (PK) analysis set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and had at least 1 measurable post-baseline plasma or CSF utreloxastat concentration. 'Number analyzed' = participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Utreloxastat n=13 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Area Under the Concentration-time Curve From 0 to Measurable Timepoint (AUC0-t) of Utreloxastat in Plasma Day 1	2670 hours*nanograms (ng)/mL Standard Deviation 1330	—
Area Under the Concentration-time Curve From 0 to Measurable Timepoint (AUC0-t) of Utreloxastat in Plasma Day 29	4220 hours*nanograms (ng)/mL Standard Deviation 1110	—

SECONDARY outcome

Timeframe: Day 1 and Day 29

Population: PK analysis set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and had at least 1 measurable post-baseline plasma or CSF utreloxastat concentration. 'Number analyzed' = participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Utreloxastat n=13 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Maximum Observed Concentration (Cmax) of Utreloxastat in Plasma Day 1	796 ng/mL Standard Deviation 492	—
Maximum Observed Concentration (Cmax) of Utreloxastat in Plasma Day 29	881 ng/mL Standard Deviation 397	—

SECONDARY outcome

Timeframe: Day 1 and Day 29

Population: PK analysis set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and had at least 1 measurable post-baseline plasma or CSF utreloxastat concentration. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Utreloxastat n=9 Participants Participants received utreloxastat during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.	Placebo Participants received matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who entered the LTE Period received open-label utreloxastat for 28 weeks. Following completion of the LTE period, participants who entered the Continued LTE Period received open-label utreloxastat for an additional 108 weeks.
Mean Concentration of Utreloxastat in Cerebrospinal Fluid (CSF) Day 1	NA ng/mL Standard Deviation NA Data not estimable due to below limit of quantification (BLQ).	—
Mean Concentration of Utreloxastat in Cerebrospinal Fluid (CSF) Day 29	NA ng/mL Standard Deviation NA Data not estimable due to BLQ.	—

Adverse Events

Part A: Utreloxastat

Serious events: 36 serious events

Other events: 167 other events

Deaths: 9 deaths

Part A: Placebo

Serious events: 11 serious events

Other events: 88 other events

Deaths: 3 deaths

Parts B and C: Utreloxastat/Utreloxastat

Serious events: 48 serious events

Other events: 115 other events

Deaths: 23 deaths

Parts B and C: Placebo/ Utreloxastat

Serious events: 25 serious events

Other events: 53 other events

Deaths: 15 deaths

Serious adverse events

Other adverse events

Additional Information

Patient Advocacy

PTC Therapeutics, Inc.

Phone: 1-866-562-4620

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
Publication restrictions are in place

Restriction type: OTHER