Other indications, page-39

"Given the reported ameliorative effects of ATL1102 on inflammatory markers3 together with the positive
functional impact noted in the non-clinical and the clinical settings, we believe it is imperative that
ATL1102 continues clinical development. As the DMD field’s understanding of the degenerative changes
in cellular and supporting muscle architecture evolves, the Company believes that ATL1102’s
mechanism of action is likely equally relevant to potentially slowing disease progression in both
ambulant and non-ambulant boys. Furthermore, the recent positive functional activity results from
Antisense’ s mdx mouse combination study4
raises the possibility of benefit from using both ATL1102
and exon skipping drugs in ambulant boys. Further work is ongoing, with results still to be reported,
to better understand the cellular pathways involved in the observed functional improvements (i.e., due
to dystrophin and/or fibrosis changes).
I mention these different clinical applications to highlight the potential of ATL1102. Despite our initial
focus in non-ambulant boys using ATL1102 as monotherapy, the Phase IIb study has the potential to
deliver an important contribution to the understanding and treatment of DMD. The study is designed
to demonstrate (i) a difference in PUL2.0 against placebo at 6 months; (ii) stabilisation or improvement
of clinical effect after 12 months of treatment; and (iii) the clinical impact of delayed treatment between
the placebo and active treatment groups. Clinically significant results, from a Phase IIb study, would
represent a major advancement in the treatment of DMD. It is our intent to share the study results
with the FDA as precedent exists in the space of rare neuromuscular disease for positive results from
a well-designed and executed study serving as the basis for an accelerated approval. With the FDA
IND 9-month monkey toxicology study underway (12 out of 40 doses successfully administered to
date), we are likely permitted to submit either a DMD clinical study protocol or the Phase IIb study
results to initiate lifting of the partial hold on the IND. This latter activity, which opens the USA market,
is planned for 2H-2024."

The above is from the newsletter released 6th June. The design of the trial, blinded and placebo controlled with an extension phase, with Tox study also underway, maximizes bang for the buck, and will provide enough data, if successful, to allow drug to market (if fast tracked approved), while further trials take place. Rare pediatric disease with no SOC allows this pathway, if the drug can show some benefit. Cortisone steroid is the benchmark against which new drugs in DMD are measured. This steroid only slows disease progression against natural history. It does have several adverse side effects.

Getting ATL1102 into arms is the best thing the company can do now. Getting it into the arms of a rare pediatric disease indication with no SOC ensures the fastest pathway to market. Until we have placebo controlled proof that the drug works we only have a hypothesis. Be it a well-researched and science supported hypothesis.

The next 18 months are the build up to the ANP money shot. If ANP can prove the drug works, and it has supportive proof to suggest it does, the present market cap will be a tiny fraction of its potential then.

As Sam says, for now we wait.