AFT Completes Pascomer Clinical Study and Takes Back Rights

AFT Pharmaceuticals Limited,
Level 1, 129 Hurstmere Road, Takapuna, Auckland 0622, New Zealand
Incorporated in New Zealand ARBN:
ARBN 609 017 969 investor.relations@aftpharm.com





27 July 2022
AFT Completes Pascomer Clinical Study and Takes
Back Rights in North America
AFT Pharmaceuticals (NZX: AFT, ASX: AFP) today announces the analysis of its study
examining its medicine Pascomer as a topical treatment for facial angiofibromas (FA)
associated with Tuberous Sclerosis Complex (TSC).
The study showed Pascomer delivered statistically significant [p<0.05] benefits against
the clinically relevant IGA
1
, FASI
2
and patient-physician improvement scales.
However, the medicine did not reach the threshold on the IGA scale that the US Food
and Drug Administration (FDA) considered necessary for its registration in the United
States (US) as a treatment for FA.
AFT also announces its Pascomer development partner and US licensee Timber
Pharmaceuticals has terminated its agreements with AFT.
AFT will now take over funding the remaining development work for the orphan
3
drug
indication of Pascomer in addition to any non-orphan indications. It will also take over
the commercialisation programme in North America. Germany’s Desitin Arzneimittel
GmbH continues as the licensee for the FA orphan drug indication in Europe,
Switzerland and UK.
Neither the clinical study results, nor the change in the medicine’s licensing,
development and commercialisation arrangements, are expected to diminish the
$12.5 million carrying value of AFT’s Pascomer assets.
AFT Managing Director Dr Hartley Atkinson said: “While the Pascomer trial did not
deliver results that meet the FDA’s thresholds, the medicine continues to show promise
in the treatment of this rare and distressing disease.
“We also continue to believe Pascomer has good potential for non-orphan
indications, which combined may offer markets larger than those for FA in TSC and
we therefore plan to continue to progress the development of the medicine.
“As previously noted, there were always significant uncertainties associated with both
the conclusion of the present Phase II/III clinical trial and the successful registration of

1
The Investigators Global Assessment (IGA)
2
Facial Angiofibroma Severity Index (FASI)
3
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so
rare, attract government assistance

AFT Pharmaceuticals Limited,
Level 1, 129 Hurstmere Road, Takapuna, Auckland 0622, New Zealand
Incorporated in New Zealand ARBN:
ARBN 609 017 969 investor.relations@aftpharm.com

Pascomer with orphan status. It was for these reasons that we adopted a conservative
valuation of the Pascomer assets.”
Dr Atkinson also said the new research, development and commercialisation
arrangements would be well accommodated within the company’s existing overall
research and development budget, signalled in May 2022 at ~ $12 million for the 2023
financial year.
“We see a growing potential to expand our R&D pipeline. In the current environment,
many cashflow negative research healthcare companies are finding it difficult to fund
their R&D programs. AFT’s positive cashflows position the company well to undertake
such projects either alone or in partnership with others.”
He said Timber Pharmaceutical’s termination of the licensing agreement had been a
possibility since a competitor, Japan’s Nobelpharma, secured FDA approval for a
treatment for FA. The FDA’s decision effectively blocked Timber Pharmaceuticals'
entry into the US market for 7 years with Pascomer as an orphan drug for treatment of
FA.
Pascomer study detail
Tuberous Sclerosis Complex (TSC), is a genetic disease, estimated to affect one in
6,000 people
4
. Around 70-80% of those with TSC over the age of two suffer from facial
angiofibromas, which can cause adverse effects on appearance and in serious
cases, impair breathing and vision
5
.

The Pascomer clinical study compared the change in facial angiofibromas in patients
treated with either placebo, or Pascomer low or high strength for 26 weeks. A total of
107 patients were enrolled with 38 in the placebo group and 36 in the Pascomer low
strength and 33 in the Pascomer high strength patient groups.
The US FDA required a comparison of responders amongst groups using a 2-point
improvement in the IGA scale as the criteria for a success. This is a challenging
endpoint to reach and although higher for both treatment groups in comparison with
the placebo it did not reach statistical significance [p>0.05].
On the less rigorous exploratory primary endpoint using a 1-point improvement in the
IGA scale [see Figure 1] both low and high strength Pascomer had a statistically
significant higher responder rate than the placebo [p<0.05].
The secondary endpoints for the IGA scale, FASI, Patient or Physician rated
improvement all reached statistical significance [p<0.05] for both the low and high
strength Pascomer treatments in comparison with the placebo.



4
https://www.tscalliance.org/about-tsc/what-is-tsc/
5
Verheyden, Charles. 1996. “Treatment of the Facial Angiofibromas of Tuberous Sclerosis.” Plastic and Reconstructive Surgery 98 (5): 777–
83.

AFT Pharmaceuticals Limited,
Level 1, 129 Hurstmere Road, Takapuna, Auckland 0622, New Zealand
Incorporated in New Zealand ARBN:
ARBN 609 017 969 investor.relations@aftpharm.com

FIGURE 1: Example of a 1-point change in the IGA scale over the study period












For and on behalf of AFT Pharmaceuticals Limited by Malcolm Tubby, Chief Financial
Officer.
For more information:
Investors Media
Dr Hartley Atkinson Richard Inder
Managing Director The Project
AFT Pharmaceuticals Tel: +64 21 645 643
Tel: +64 9488 0232

About AFT Pharmaceuticals
AFT is a growing multinational pharmaceutical company that develops, markets, and
distributes a broad portfolio of pharmaceutical products across a wide range of
therapeutic categories which are distributed across all major pharmaceutical
distribution channels: over the counter (OTC), prescription and hospital. Our product
portfolio comprises both proprietary and in-licensed products, and includes patented,
branded, and generic drugs. Our business model is to develop and in-license products
for sale by our own dedicated sales teams in our home markets of Australia and New
Zealand and to out-license / distribute our products to local licensees and distributors
to over 125 countries around the world. For more information about the company, visit
our website www.aftpharm.com.

Before treatment
After 26 weeks treatment