Recent Updates
Recently added Catalysts

ATYR1940

Phase 1

Facioscapulohumeral Muscular Dystrophy | Monoclonal antibody | Neurology |aTyr Pharma, Inc.|Last Updated: Dec 22, 2023

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLEDDMC
Total Trials2
Total Enrollment17

FDA Designations

No designations recorded

Clinical trial landscape

ATYR1940 · 5 trials · 4 indications

Phase 1 5
NCT02836418Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)Facioscapulohumeral Muscular Dystrophy
COMPLETED8 Analytics
NCT02603562Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular DystrophyFacioscapulohumeral Muscular Dystrophy (FSHD)
COMPLETED8 Analytics
NCT02579239Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)Limb-Girdle Muscular Dystrophies
COMPLETED18 Analytics
NCT02531217Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study ExtensionFacioscapulohumeral Muscular Dystrophy
COMPLETED9 Analytics
NCT02239224Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular DystrophyFacioscapulohumeral Muscular Dystrophy (FSHD)
COMPLETED20 Analytics
PHASE1COMPLETED
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular DystrophyUnlock trial analytics
PHASE1COMPLETED
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy (FSHD)Unlock trial analytics
PHASE1COMPLETED
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
Limb-Girdle Muscular DystrophiesUnlock trial analytics
PHASE1COMPLETED
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Facioscapulohumeral Muscular DystrophyUnlock trial analytics
PHASE1COMPLETED
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy (FSHD)Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Up to End of Study (up to approximately Week 39)

TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section.

Number of Participants With Positive Anti-Drug Antibodies (ADA)
Up to End of Study (up to approximately Week 39)

Summarized titers are reported below.

Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL)
Up to End of Study (up to approximately Week 39)

Participants with Jo-1 Ab levels ≥1.5 U/mL were to be discontinued from dosing of the study drug.

Number of Participants With a Clinical Laboratory Abnormality Leading to an AE
Up to End of Study (up to approximately Week 39)

Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count neutrophils, lymphocytes, monocytes, eosinophils, basophils, and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
Up to End of Study (up to approximately Week 39)

Pulmonary evaluations included pulmonary function tests and pulse oximetry. Clinically significant changes were to be reported as adverse events. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE
Up to End of Study (up to approximately Week 39)

ECG parameters that were evaluated included heart rate and PR, QR, and QT intervals. A clinically significant ECG abnormality was based upon the Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With Vital Sign Abnormality Resulting in a TEAE
Up to End of Study (up to approximately Week 39)

The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12
Baseline, Week 12

MMT (muscle strength) will be graded using a modified Medical Research Council scale. Scores were converted to 13-point scale (range from 0 \[on contraction palpable\] to 12 \[normal strength\]). An overall total score was calculated by summing all scores for a total possible score of 336. Decreased muscle strength was indicated by a decreased score. An overall total score was calculated as long as 24 of the 28 individual scores were non-missing.

Number of Participants With an Ocular Abnormality Leading to a TEAE
Up to End of Study (Up to Week 25)

Ocular parameters included vitreous, retina, macula, choroid, optic nerve, and optic nerve pallor. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With an Impact on Hearing From ATYR1940 Treatment
Up to End of Study (Up to Week 25)
Number of Participants With Clinically Significant Physical Examination Abnormality
Up to End of Study (up to Week 36)

Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat, cardiovascular system, respiratory system, gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, reflexes, and sensory testing. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
Up to End of Study (up to Week 36)

The vital sign parameters that were evaluated included blood pressure, pulse and respiration rates, and body temperature. Vital signs abnormalities that were considered by the Investigator to be clinically significant were reported as AEs if the finding represented a change from baseline. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE
Up to End of Study (Up to Week 36)

Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count with differential \[neutrophils, lymphocytes, monocytes, eosinophils, basophils\], and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Up to End of Study (up to Week 25)

TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result
Baseline up to Week 14

Criterion for a positive Jo-1 Ab test result: \>10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as \<7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study.

Number of Participants With a Clinically Significant Laboratory Abnormality
Baseline up to Week 14

Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase \[will be fractionated if elevated\], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol \[non-fasting\]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Physical Examination Abnormality
Up to End of Study (up to Week 25)

Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Number of Participants With a Pulmonary Function Event Resulting in a TEAE
Up to End of Study (Up to Week 25)

Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Secondary Endpoints

Change From Baseline in Creatinine Kinase at Week 12
Baseline, Week 12
Number of Participants With Positive Anti-Drug Antibodies (ADA)
Baseline up to Week 12
Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL)
Baseline up to Week 12
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ATYR1940EXPERIMENTALParticipants will receive ATYR1940 up to 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation (up to 34 weeks).
Group A - FSHDEXPERIMENTALParticipants with FSHD will receive single dose of placebo intravenous (IV) infusion followed by ATYR1940 IV infusion at doses of 0.3 up to 1.0 milligrams/kilograms (mg/kg) once weekly for 8 weeks and then twice weekly for 4 weeks using intraparticipant dose escalation for up to 12 weeks.
Group B - LGMD2B and FSHDEXPERIMENTALParticipants with LGMD2B and FSHD will receive single dose of placebo IV infusion followed by ATYR1940 IV infusion at doses of 0.3 up to 1.0 and 3.0 mg/kg once weekly for 8 weeks and then twice weekly for 4 weeks using intraparticipant dose escalation for up to 12 weeks.
Cohort 1: ATYR1940 0.3 mg/kgEXPERIMENTALParticipants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks.
Cohort 2: ATYR1940 1.0 mg/kgEXPERIMENTALParticipants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.
Cohort 3: ATYR1940 3.0 mg/kgEXPERIMENTALParticipants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.

Interventions

NameTypeDescription
ATYR1940DRUGConcentrate for solution for infusion
PlaceboBIOLOGICALConcentrate for solution for infusion
Unlock Study Design Details

Eligibility Criteria

Age Range16 Years to 25 Years
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Enrolled in and completed the treatment period in the parent study. 2. Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940. 3. In the Investigator's opinion, participant has shown acceptable compliance with ATYR1940 and the study pr...

Countries:United StatesDenmarkItalyFranceNetherlands
Unlock Eligibility Criteria

Frequently asked questions about ATYR1940

What is ATYR1940 used for?

ATYR1940 is an investigational monoclonal antibody being studied for the treatment of muscular dystrophies, including Facioscapulohumeral Muscular Dystrophy (FSHD) and Limb-Girdle Muscular Dystrophies. It is being developed by aTyr Pharma, Inc. (NASDAQ: ATYR) and is currently in Phase 1 clinical development.

Who makes ATYR1940?

ATYR1940 is being developed by aTyr Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol ATYR. The company is conducting clinical trials to evaluate the safety and biological activity of ATYR1940 in patients with certain forms of muscular dystrophy.

What phase is ATYR1940 in?

ATYR1940 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials have been completed to assess its safety, tolerability, pharmacokinetics, and biological activity in adult participants with muscular dystrophy.

What clinical trials has ATYR1940 been in?

ATYR1940 has been studied in four completed Phase 1 clinical trials. These include NCT02239224 and NCT02531217 in patients with Facioscapulohumeral Muscular Dystrophy, and NCT02579239 and NCT02836418 in patients with Limb-Girdle Muscular Dystrophy and FSHD. The trials were conducted in the United States and several European countries.

Is ATYR1940 the same as resolarzumab?

ATYR1940 is also known as resolarzumab. It is a monoclonal antibody being investigated for the treatment of muscular dystrophies. The drug is being developed by aTyr Pharma, Inc. and has undergone Phase 1 clinical trials to evaluate its safety and biological activity.