Trial Outcomes & Findings for A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis (NCT NCT05176093)

Last Updated: 2025-10-06

Results Overview

To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Day 0 to Day 365

Results posted on

2025-10-06

Participant Flow

Participant milestones

Participant milestones
Measure	Engensis Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
Overall Study STARTED	4	4
Overall Study COMPLETED	4	3
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	39.0 years STANDARD_DEVIATION 16.99 • n=5 Participants	52.3 years STANDARD_DEVIATION 10.63 • n=7 Participants	45.6 years STANDARD_DEVIATION 14.91 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment South Korea	3 participants n=5 Participants	2 participants n=7 Participants	5 participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 0 to Day 365

Population: Percentages were based on the number of subjects in the Safety Analysis Population by treatment group and overall. Subjects were counted once within for each unique Preferred Term.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Injection site bruising	3 Participants	3 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Asthenia	0 Participants	2 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Pyrexia	2 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Injection site pain	1 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Cough	3 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Rhinorrhoea	1 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Dysphagia	3 Participants	2 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Arthralgia	1 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Myalgia	1 Participants	1 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term COVID-19	3 Participants	2 Participants
Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term Headache	1 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 and Day 365

Population: Revised Amyotrophic Lateral Sclerosis Function Rating Total Score and Change from Baseline to Day 365 (Safety Analysis Population)

Changes from Baseline (Study VMALS 002-2, Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating TOTAL scores at Day 365 for Engensis compared to Placebo There are 12 separate assessments in this neuromuscular assessment covering Bulbar, Fine Motor, Gross Motor and Breathing assessments. Each individual assessment is scored from 0 (worst case) to 4 (best case). Total score is calculated as the sum of all 12 assessment subscores. Note that only the Total Score will be reported in the clinical study report due to the small number of subjects. The Total Score range is from a minimum of 0 (worst case) to a maximum of 48 (best case).

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Baseline - actual value	39.0 score on a scale Standard Deviation 1.41	30.8 score on a scale Standard Deviation 5.62
To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Day 365 / Early Termination - change from baseline	-10.5 score on a scale Standard Deviation 8.23	-8.7 score on a scale Standard Deviation 9.61

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 and Day 365

Population: ITT Population

As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities. Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 365 by Muscle Group (ITT Population)

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Elbow Flexion- Right - Baseline - Actual	30.53 Pounds Standard Deviation 26.522	13.73 Pounds Standard Deviation 11.667
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Shoulder Flexion-Left - Baseline - Actual	24.10 Pounds Standard Deviation 12.572	20.08 Pounds Standard Deviation 11.538
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Shoulder Flexion-Left - Day 365 - Change from Baseline	-16.13 Pounds Standard Deviation 12.419	-8.40 Pounds Standard Deviation 5.173
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Shoulder Flexion- Right - Baseline - Actual	24.645 Pounds Standard Deviation 11.5448	17.850 Pounds Standard Deviation 12.0423
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Shoulder Flexion-Right - Day 365 - Change from Baseline	-16.395 Pounds Standard Deviation 12.8508	-5.900 Pounds Standard Deviation 5.1798
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Elbow Flexion- Left - Baseline - Actual	29.440 Pounds Standard Deviation 25.1779	14.525 Pounds Standard Deviation 10.1923
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Elbow Flexion-Left - Day 365 - Change from Baseline	-20.190 Pounds Standard Deviation 15.8662	-5.433 Pounds Standard Deviation 3.6501
To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Elbow Flexion-Right - Day 365 - Change from Baseline	-20.45 Pounds Standard Deviation 16.083	-4.1 Pounds Standard Deviation 1.082

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0, Day 240, Day 300 and Day 365

Population: Slow Vital Capacity Percent Change from Baseline by Visit (ITT Population)

Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation. It is measured in Liters of air and the percentage of change from baseline is the Percent Predicted Value and Change from Baseline. Change from Baseline (Study VMALS 002-2, Day 0) in Slow Vital Capacity on Day 240, Day 300 and Day 365 for Participants with intramuscular administration of Engensis compared to Placebo. Increasing negative Change from Baseline results indicates worsening of respiratory capacity.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants Day 240 - Change from Baseline	0.0 percentage of Change from Baseline Standard Deviation 1.41	-16.5 percentage of Change from Baseline Standard Deviation 7.78
To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants Day 300 - Change from Baseline	-9.3 percentage of Change from Baseline Standard Deviation 11.90	-7.3 percentage of Change from Baseline Standard Deviation 19.33
To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants Day 365/Early Term - Change from Baseline	-17.3 percentage of Change from Baseline Standard Deviation 19.39	-15.0 percentage of Change from Baseline Standard Deviation 17.52

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 to Day 365

Population: Total Tracheostomy procedures for the Safety Analysis Population - Number of Participants - Day 0 to Day 365

Total participants requiring Tracheostomy procedures that received intramuscular administration of Engensis, compared to total Placebo participants requiring Tracheostomy procedures.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 to Day 365

Population: Total all-cause Mortality for participants

Total all-cause Mortality for participants that received intramuscular injections of Engensis, compared to total all-cause Mortality for Placebo participants. Total participant deaths that received intramuscular administration of Engensis, compared to total Placebo participant deaths.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0, Day 240 and Day 365

Population: Percent Change from Baseline (ITT Population) for Engensis and Placebo.

The Participant completes the 40 questions in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case). Note for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms. The Total Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population. The Change from Baseline was calculated and displayed in the Table. ALSAQ-40 was completed at the pre-dose baseline visit (Day 0), and at the Day 240 and Day 365 visits.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Activities of Daily Living/Independence	14.17 percentage of Change from Baseline Standard Deviation 11.815	-.83 percentage of Change from Baseline Standard Deviation 3.819
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Activities of Daily Living/Independence	21.88 percentage of Change from Baseline Standard Deviation 21.250	10.00 percentage of Change from Baseline Standard Deviation 8.660
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Eating and Drinking	0 percentage of Change from Baseline Standard Deviation 0	11.13 percentage of Change from Baseline Standard Deviation 25.411
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Eating and Drinking	20.83 percentage of Change from Baseline Standard Deviation 24.994	33.33 percentage of Change from Baseline Standard Deviation 28.868
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Communication	16.67 percentage of Change from Baseline Standard Deviation 32.036	28.57 percentage of Change from Baseline Standard Deviation 6.207
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Communication	26.80 percentage of Change from Baseline Standard Deviation 33.350	32.17 percentage of Change from Baseline Standard Deviation 35.700
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Emotional Functioning	3.33 percentage of Change from Baseline Standard Deviation 13.769	7.50 percentage of Change from Baseline Standard Deviation 18.875
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Emotional Functioning	10.00 percentage of Change from Baseline Standard Deviation 24.833	12.50 percentage of Change from Baseline Standard Deviation 4.330
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Physical mobility	12.5 percentage of Change from Baseline Standard Deviation 2.500	3.33 percentage of Change from Baseline Standard Deviation 20.817
To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Physical mobility	13.75 percentage of Change from Baseline Standard Deviation 9.465	21.67 percentage of Change from Baseline Standard Deviation 24.664

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 240 and Day 365

Population: Patient Global Impression of Change - ITT Population.

The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life. Each descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that as made all the difference. The test was self-administered on Days 240 and 365.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Impression change - or worse	2 Participants	2 Participants
To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Impression change - Almost the same, hardly any change	1 Participants	1 Participants
To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early termination - Impression change - or worse	4 Participants	2 Participants
To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365/Early Termination - Impression change - Almost the same, hardly any change	0 Participants	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 240 and Day 365

Population: Clinical Global Impression of Change - ITT Population

The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging rom Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 240 and 365/ Early Termination.

Outcome measures

Outcome measures
Measure	Engensis n=4 Participants Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 Participants Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - No change	2 Participants	1 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Minimally worse	1 Participants	1 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 240 - Much worse	0 Participants	1 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365 / Early Termination - Minimally improved	0 Participants	1 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365 / Early Termination - No change	2 Participants	0 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365 / Early Termination Minimally worse	0 Participants	1 Participants
To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo Day 365 / Early Termination Much worse	2 Participants	1 Participants

Adverse Events

Engensis

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Engensis n=4 participants at risk Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis	Placebo n=4 participants at risk Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid
Respiratory, thoracic and mediastinal disorders Dyspnoea	25.0% 1/4 • Number of events 1 • Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365. All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.	0.00% 0/4 • Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365. All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.
Vascular disorders Peripheral arterialocclusive disease	0.00% 0/4 • Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365. All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.	25.0% 1/4 • Number of events 1 • Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365. All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.

Other adverse events

Additional Information

Jinsub Lee, PhD.

Helixmith Co., Ltd.

Phone: +82-10-8256-0439

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60