Positive Phase III Interim Analysis Results Investor Webinar
Released
Released
Announcement PDF
Positive Phase III
Interim Analysis Result
Dr Paul Gavin | Chief Executive Officer
www.avecho.com.au | ASX:AVE
244 participants randomised for the interim cohort
3 arms 75mg CBD | 150mg CBD | placebo
8 weeks of nightly dosing per participant
2 endpoints Insomnia Severity Index (ISI) + subjective sleep efficiency (sSE)
Blinded patients, investigators and Avecho all blinded to allocation
Insomnia —
what we're treating
Difficulty falling or staying asleep
Affects ~10-30% of Australians
No approved OTC pharmaceutical
product currently available in Australia
for ongoing use
Australia's unique Schedule 3 OTC
pathway for CBD opens a first-mover
commercial opportunity
2
T H E T R I A L
Phase III design — what we're testing
Largest CBD insomnia trial globally; designed to support a TGA submission on positive completion.
What the interim analysis is
1
What gets tested
The interim analysis examines whether the CBD
TPM® capsule is improving sleep in the 244
patients dosed to date — measured using two
independent primary endpoints (ISI and sSE).
Either endpoint succeeding is sufficient.
2
Who decides
An independent Data Monitoring Board reviews
the unblinded data. The DMB is independent of
Avecho and the trial investigators. Avecho
remains fully blinded throughout the process.
3
What we'll learn
The DMB will recommend one of three outcomes:
1) Positive - Continue the trial with a second
cohort of patients - confirmed sample size
2) Positive - Stop the trial for efficacy
3) Negative - stop the trial for futility
U N D E R S T A N D I N G T H E R E A D O U T
3
Independent Data Monitoring Board (DMB)
• External sleep, safety and biostatistical experts
• Independent of Avecho and trial investigators
The DMB is the sole body with access to unblinded interim data -- standard best practice for pivotal placebo-controlled trials.
Why this trial is positioned to deliver a clinical signal
Three key design features have been built into this trial specifically to maximise the probability of a successful result
01
Two independent primary endpoints
1) Insomnia Severity Index (ISI)
2) Subjective Sleep Efficiency (sSE)
Only one needs to work for trial success.
02
Adaptive design interim analysis
The interim analysis can recalibrate the
trial, adjusting the required patient
numbers during the study to ensure the
result is conclusive.
03
Placebo-effect control mechanisms
The protocol is designed to minimise
damage from the placebo response —
improving the chance that genuine drug
effect is detected cleanly.
Supporting design features
150mg
Maximum allowed OTC
dose tested
519
Largest CBD insomnia
trial globally
8 weeks
Treatment duration per
patient
ISI ≥ 15
Tight inclusion criteria
FDA + EMA
Trial designed to global
standards
T R I A L D E S I G N
4
Three possible outcomes
Stop for efficacy -- positive
Upside scenario — not expected, but
possible
The drug/trial is working so well results
are highly statistically significant after
244 patients. The DMB can recommend
stopping the trial early, declaring Phase
III successful.
Stop for futility -- negative
The drug is not showing the
required effect
Dosing of further patients ceases —
preserving shareholder capital. The CBD
asset retains commercial value for
alternative indications.
The expected positive result was continuing the trial to 519 patients.
W H A T H A P P E N S A F T E R T H E R E A D O U T
Trial continues -- positive
Base case positive outcome — this
is what we are aiming for
The drug/trial is working, with an
observable difference between treatment
and placebo. DMB recommends
continuing the trial with a confirmed
sample size needed to reach statistical
significance at completion.
5
Positive – DMB recommends trial continues to 519 patients
Take Away - So far, product performance appears in line with approved insomnia medications
V E R D I C T
6
What does the recommendation tell us - Efficacy
1. Interim analysis met the pre-specified criteria to continue for efficacy — a treatment effect for the CBD TPM capsule
versus placebo on at least one of the two primary endpoints
2. Observed data variability is in line with the assumptions used to power the study
3. If the second cohort performs consistently with the first, the completed 519-participant trial is well positioned to meet its
primary endpoint
4. By recommending continuation at the originally planned sample size — rather than a larger one — the independent DMB
has signalled that Avecho's original assumptions on effect size* and variability are tracking at, or better than, expectation
*Original assumptions were that the product performs as well as approved insomnia drugs
Positive – DMB recommends trial continues to 519 patients
Take Away - So far, the product safety profile is competitive with approved insomnia drugs
V E R D I C T
7
What does the recommendation tell us - Safety
1) CBD TPM® capsule — no serious adverse events
across 244 participants at two doses in the interim
analysis. Safety profile looks good.
Safety burdens of currently approved insomnia
medicines
• Dependence, tolerance and withdrawal
• Next-day impairment
• Overdose toxicity
• Complex sleep behaviours
• Risk in older adults
Global insomnia: a large, growing market underserved by current therapies
Market context for Avecho’s TPM®-enhanced CBD capsule for insomnia
~850M
adults live with chronic insomnia
worldwide — 16.2% of the adult
population
~1 in 3
adults experience insomnia symptoms at
some point
~US$9.5bn
forecast global insomnia treatment market
by 2033
~6% CAGR
projected annual market growth, 2025–
2033
Insomnia treatment market — global revenue (US$bn)
$6.1
$6.8
$7.6
$8.6
$9.6
2025 2027 2029 2031 2033
Why this is Avecho’s opportunity
• A safety-driven gap. Benzodiazepines, Z-drugs and sedating antihistamines
carry dependence, next-day impairment and overdose risk — shifting demand
to safer, non-scheduled options. The newest class, orexin antagonists, is the
fastest-growing at ~12% CAGR.
• An open OTC pathway in Australia. Since Feb 2021 the TGA permits low-
dose CBD (≤150 mg/day) to be sold pharmacist-only, over the counter — but
no product has yet met the efficacy bar required for ARTG registration.
• First-mover potential. A successful Phase III is the registration-enabling
dataset — positioning Avecho’s CBD TPM capsule to be first to that OTC
market, with Sandoz as Australian commercial partner.
Sources: Market size — consensus of MarketDataForecast, DataBridge & Straits Research (2025–26), figures vary by provider; prevalence — systematic analysis, Sleep Medicine Reviews 2025 (852M adults, 16.2%); OTC pathway — TGA, low-dose CBD down-scheduled to
Schedule 3, Feb 2021.
8
Licensing
Opportunities
› Commercial validation from multinational
pharmaceutical company
› New phase of commercial growth following many
years of hard work advancing the CBD capsule
› Australia the first territory in a global strategy
› Licensing deal marks the first of potentially
several agreements globally
USA 30x
Europe 20x
China 10x
LATAM 4x
Canada 1.5x
MENA 2x
Japan 5x
Partnered
Sandoz
Australia is small compared to international pharmaceutical
markets. Huge opportunity for overseas territories
US $19M*
*For upfront and potential milestone
payments prior to commercial sales
9
Continuing the trial — how we fund it, how we execute
Trial completion requires ~300 additional patients. The two questions shareholders will ask: how much, and how long?
How we fund it
● Priority: pharmaceutical licensing
deal for an ex-Australian territory —
upfront fees from licensing would
fund Phase III completion.
● Pro-forma $6.25M in the bank
already
How we accelerate
● ~300 additional patients required
to complete the trial
● Plan to open ~10 additional clinical
sites — already identified
● With additional sites engaged,
recruitment expected to take ~12
months
● Lessons from interim recruitment
fully embedded in completion plan
What runs in parallel
● TGA dossier construction (with
Sandoz)
● Manufacturing scale-up continues
— funded from earlier placement
● Further ex-Australia licensing
conversations progress to closure
● Engagement with international
regulators (e.g. FDA) for global
pathway optionality
B A S E C A S E I N D E T A I L
10
Next 12-18 months
H2 2026
Trial Continuation
● Pursue multiple licensing deals
● Open additional trial sites and recommence dosing
● Engagement with overseas regulators (e.g. FDA)
● Scale up and manufacturing
2027
Trial completion and submission readiness
● Pursue further licensing deals
● Complete Phase III dosing to final patient
● Complete manufacturing work required for TGA
submission
● Compile and finalise TGA submission dossier
● Develop product with partners in international territories
(specific territories may have their own requirements)
M I L E S T O N E S A N D T R I G G E R S
11
Every part of our thesis has been independently validated
For years we have claimed big things for this product. At each step, an external body has validated those predictions. One piece of validation remained.
Preclinical
validated that
TPM Increased
CBD absorption
US Patent Office
validated that
Product is unique
TGA
validated that
Trial design &
submission
strategy are
sound
Sandoz
validated that
Data, trial
design,
commercial case
are compelling
Market
validated that
Story is good,
value will follow
Interim analysis
validated that
Evidence the
product works
for the indication
June 2026
12
C L O S I N G A R G U M E N T
Positive Interim Analysis in Phase III insomnia trial
Positive signal for efficacy and safety after 244
patients — de-risks remaining Phase III trial to completion.
Significant Commercial Partner for Australia
Sandoz, world's largest generics pharma — US$3M upfront,
US$16M in milestones, 14–19% tiered royalties.
Global licensing potential
Large commercial opportunity in numerous jurisdictions
allows for potentially multiple licensing transactions
Stop thinking if this will work – start thinking what this could be
I N V E S T M E N T C A S E D E - R I S K E D
Clear registration pathway with the TGA
Unique Australian OTC pathway for CBD; trial designed
and conducted for TGA submission.
Significant commercial potential in Australia
9.5M Australians experience insomnia; addressable market
potential >US$125M per annum. Global opportunity larger
Money in the bank
Proforma cash position of $6.25M , provides strong position
to execute deals following a positive interim readout
13
Questions Welcome
Dr Paul Gavin
Chief Executive Officer
T. +61 3 9002 5000
E. pgavin@avecho.com.au
Melbourne Office
Unit A8, 2A Westall Road
Hallmarc Business Park
Clayton VIC 3168
Australia
T: +61 3 9002 5000
E: info@avecho.com.au
Share your feedback
Discover