Scientific literature

The published record we build on

Azalla has not yet published its own preclinical data. This page collects the third-party literature and regulatory sources behind the claims made elsewhere on this site — the disease epidemiology, the receptor biology, the prior clinical evaluation of this mechanism, and the regulatory pathway. Where we cite a number in our copy, its source is here.

Dermatomyositis

Disease burden, the approved landscape, and the prior CB2 trial

Including the full public record of the Phase 3 evaluation of a CB2 agonist in this indication. We think that trial is important and we would rather you read it directly than take our summary of it.

Prevalence and incidence of adult dermatomyositis in the United States
The population estimate underpinning our orphan designation strategy.
Arthritis Care & Research
Octagam 10% approval for adult dermatomyositis
The single approved therapy for adult dermatomyositis, and an intravenous one.
US Food and Drug Administration
Topline results, DETERMINE Phase 3 trial of lenabasum in dermatomyositis
The prior Phase 3 evaluation of a CB2 agonist in this indication, including the prespecified subgroup analyses we discuss on the J-041 page.
Corbus Pharmaceuticals, June 2021
Efficacy and safety of lenabasum in dermatomyositis: results of the Phase 3 DETERMINE study
The peer-reviewed report of the same trial, including the week 40 and week 52 subgroup outcomes.
Annals of the Rheumatic Diseases
Safety and efficacy of lenabasum in patients with dermatomyositis
The earlier study that established tolerability and described the compound’s receptor pharmacology.
Peer-reviewed Phase 2 report
Cutaneous Dermatomyositis Activity and Severity Index (CDASI)
The skin-specific endpoint on which we intend to build our clinical strategy.
Validated outcome measure

Multiple sclerosis

Prevalence and the gap in progressive disease

The epidemiology and the unmet-need evidence behind the second program.

MS prevalence in the United States
The US population estimate we cite for multiple sclerosis.
National Multiple Sclerosis Society
Atlas of MS, global prevalence
The worldwide prevalence figure.
Multiple Sclerosis International Federation
Progressive multiple sclerosis: the treatment gap
Why the therapeutic need in progressive disease remains largely unmet.
Practical Neurology

Mechanism

Receptor biology and disease pathways

Background literature on CB2 distribution, cannabinoid agonist pharmacology, PPARγ activity and the interferon signature in dermatomyositis.

CB2 receptor expression and distribution in immune cells
The basis for describing CB2 as an immune-restricted receptor with limited central nervous system expression.
Peer-reviewed literature
Cannabinoid pharmacology: agonist efficacy and receptor selectivity
Review literature describing how cannabinoid-derived compounds differ in agonist efficacy and CB1/CB2 selectivity.
Molecular Pharmacology
PPARγ activation in neuroinflammation and neuroprotection
Background to the dual mechanism carried by J-019.
Peer-reviewed literature
Type-I interferon signature in dermatomyositis
The disease biology that our terminal cytokine analysis maps onto.
PLOS ONE

Regulatory

Orphan designation and development pathway

The regulatory basis for sequencing the orphan indication first.

Orphan Drug Designation: program overview and benefits
The designation pathway and the exclusivity that follows approval.
US Food and Drug Administration
Development costs and timelines for orphan versus non-orphan programs
The comparative cost and timeline evidence behind our orphan-first sequencing.
Orphanet Journal of Rare Diseases

On our own data. Azalla’s preclinical results are not published. Study designs, dose levels, potency and selectivity values, effect sizes and statistical detail are held as confidential data and are shared with partners and prospective investors under a confidentiality agreement. We intend to publish and to present at scientific congresses once the intellectual property position and the development program make that appropriate.

External links are provided for reference. Azalla is not affiliated with these publishers or organizations, does not control their content, and their inclusion is not an endorsement of Azalla’s compounds.

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