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Alvelestat

Phase 2

Alpha-1 Antitrypsin Deficiency (AATD) | Small molecule | Rare Disease |Mereo BioPharma Group plc|Last Updated: Aug 20, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment63

FDA Designations

ORPHAN_DRUGFAST_TRACK

Clinical trial landscape

Alvelestat · 2 trials · 4 indications

Phase 2 1Phase 1 1
NCT03679598Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency (AATD)
COMPLETED63 Analytics
PHASE2COMPLETED
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency (AATD)Unlock trial analytics

Study Endpoints

Primary Endpoints

Within-individual % Change in Plasma Desmosine/Isodesmosine
baseline, week 12

To evaluate the effect of alvelestat (MPH966) administered twice daily (bid) for 12 weeks on blood markers of within-individual % change in plasma desmosine/isodesmosine will be measured.

Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event
baseline, week 16

To evaluate the safety and tolerability of alvelestat (MPH966) administered twice daily (bid) for 12 weeks treatment numbers and % of subjects who experience at least 1 treatment-emergent adverse event (TEAE) will be measured.

Secondary Endpoints

Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo
baseline, week 12
Effect of Alvelestat on Blood Pharmacodynamic Biomarkers of NETosis
Randomization through Day 10 or hospital discharge, whichever was shorter.
Effect of Alvelestat on Blood Pharmacodynamic Biomarkers of Inflammation
Randomization through Day 10 or hospital discharge, whichever was shorter.
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Alvelestat (MPH966)ACTIVE_COMPARATORAlvelestat (MPH966) 120mg (4 30mg tablets) twice daily by mouth for 12 weeks
PlaceboPLACEBO_COMPARATOR4 Placebo tablets twice daily by mouth for 12 weeks
Placebo oral tabletPLACEBO_COMPARATORplacebo
Alvelestat oral tablet - dose 1ACTIVE_COMPARATORMPH966
Alvelestat oral tablet - dose 2ACTIVE_COMPARATORMPH966
Alvelestat oral tablet - dose 3ACTIVE_COMPARATORMPH966

Interventions

NameTypeDescription
Alvelestat (MPH966)DRUGAlvelestat was developed as treatment for lung diseases like Chronic Obstructive Pulmonary Disease. Alevelestat works by blocking certain proteins in the body that are responsible for inflammation and damage to the lungs that can lead to COPD symptoms.
PlaceboOTHERPlacebo is a pill or tablet that does not contain any study drug.
AlvelestatDRUGoral tablet
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: Participants are eligible to be included in the study only if ALL of the following criteria apply: 1. Capable of giving signed informed consent as described in Appendix 3, which includes compliance with the requirements and restrictions listed in the informed consent form and i...

Countries:United States
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Frequently asked questions about Alvelestat

What is Alvelestat used for?

Alvelestat is an investigational small molecule being developed for Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic condition, and for Covid-19. It has been studied in clinical trials for AATD-related COPD and emphysema, as well as for Covid-19.

What does Alvelestat target?

Alvelestat is a small molecule that targets neutrophil elastase, an enzyme involved in tissue destruction in Alpha-1 Antitrypsin Deficiency. By inhibiting this enzyme, it aims to reduce lung damage associated with the condition.

Who makes Alvelestat?

Alvelestat is being developed by Mereo BioPharma Group plc, a biopharmaceutical company listed on the stock exchange under the ticker MREO. The company is focused on rare disease therapeutics.

What phase is Alvelestat in?

Alvelestat is in Phase 2 clinical development. It has completed Phase 2 trials for Alpha-1 Antitrypsin Deficiency and a Phase 1 trial for Covid-19. It is not yet approved by the FDA and remains investigational.

What clinical trials is Alvelestat in?

Alvelestat has completed three clinical trials: NCT03636347, a Phase 2 study in AATD-related COPD; NCT03679598, a Phase 2 study in AATD; and NCT04539795, a Phase 1 study in Covid-19. All trials are completed.

Is Alvelestat the same as MPH966?

Yes, Alvelestat is also known as MPH966. Clinical trial records reference both names, with MPH966 appearing in the titles of studies such as NCT03636347.