Pipeline - AelisFarma

Drug candidates

Indications

Discovery

Preclinical Development

Clinical Phase 1

Clinical Phase 2

Clinical Phase 3

AEF0117

for cannabis-related
disorders

AEF0217

for cognitive
disorders

New<br>CB<sub>1</sub>-SSi

for other cannabinoid-
dependent diseases

AEF0117

for cannabis-related disorders

Stage:

Clinical phase 2

AEF0117

for cannabis-related disorders

Stage:

Clinical phase 2

AEF0117

for cannabis-related disorders

AEF0217

for cognitive disorders

Stage:

Clinical phase ½

AEF0217

for cognitive disorders

Stage:

Clinical phase ½

AEF0217

for cognitive disorders

Stage:

Discovery

New
CB₁-SSi

for other cannabinoid-dependent diseases

Stage:

Discovery

New
CB₁-SSi

for other cannabinoid-dependent diseases

Stage:

Discovery

New
CB₁-SSi

for other cannabinoid-dependent diseases

AEF0117 has an open FDA IND and entered a phase 2b clinical trial in the United States in Q2 2022.

AEF0117 is a new molecular entity (NME) developed by Aelis Farma to treat the harmful effects of excessive cannabis use.

AEF0117's major characteristics:

•

High potency in inhibiting the effects of cannabis’ active ingredient, THC, in several animal species including primates without side effects on normal behavior.

•

Favorable ADMET profile, with good oral absorption, brain access, long half-life and no toxic or adverse effects identified to date (Therapeutic index >13,000)

•

Good pharmacokinetic, safety and efficacy profile in healthy volunteers and cannabis addicts.

The clinical development of AEF0117 is supported by grants from NIDA-NIH (National Institute on Drug Abuse of the National Institute of Health, US).

AEF0217 has successfully completed the Phase 1 clinical program and will enter Phase 1/2 study in subjects with Down syndrome before end of 2022 in Europe.

AEF0217 is the second drug candidate developed by Aelis Farma. It is specifically designed for the treatment of cognitive deficits.

AEF0217's major characteristics:

•

High potency in reversing cognitive deficits in validated translational models of Down syndrome, Fragile X syndrome and aging related cognitive impairments without identifiable behavioral side effects even in fragile trisomic and aged mice.

•

Very favorable ADMET profile, with good oral absorption, brain access, long half-life and a good tolerability (therapeutic index = 640).

•

Good pharmacokinetic, safety and efficacy profile in healthy volunteers

Clinical development of AEF0217, for which its first therapeutic indication is the cognitive deficits in Down syndrome, is supported by a grant from the EU (Horizon Programme H2020; Improving Cognition in Down syndrome, Grant N° 899986).

Aelis Farma’s platform provides new Signaling Specific inhibitors of the CB₁ receptor.

New CB₁-SSi are New Molecular Entities (NME) with an original structure and improved pharmacodynamic properties, which will provide drug candidates for the treatment of other CB₁-dependent diseases, including several orphan diseases.