Ann: Quarterly Activities/Appendix 4C Cash Flow Report, page-3

ASX Announcement
25 January 2023
Quarterly Activities Report & Appendix 4C
Antisense Therapeutics Limited [ASX:ANP | US OTC:ATHJY | FSE:AWY] (Antisense or Company) is pleased
to provide its Appendix 4C and quarterly update for the period ended 31 December 2022.
ATL1102 for DMD: Clinical Trial Application submitted for Phase IIb DMD trial in Europe
During the quarter the Company submitted a Clinical Trial Application (CTA) in three European countries
(UK, Bulgaria and Turkey) for approval to conduct its Phase IIb trial of ATL1102 in non-ambulant boys
with Duchenne muscular dystrophy (DMD)1
. The CTA submission for Ethics Committee (EC) approval to
initiate trial sites in Australia is scheduled for submission to the EC for review in Q1’23.
CTA is undergoing evaluation by relevant regulatory bodies and is at various stages of the approval
process. As previously highlighted1
, trial approvals are expected to come through in a staggered manner
in early 2023 depending on the respective regulatory agencies’ evaluation process. Timeline guidance for
reporting of the results from the blinded phase of the Phase IIb trial remains unchanged and is anticipated
in 1H’24.
ATL1102 tox study to support clinical program in the US
In the period the Company advised that it had initiated the process to conduct a nine-month chronic
monkey toxicology study of ATL1102 at Contract Research Organisation (CRO) Pharmaron to support the
advancement of the ATL1102 program in the US for Duchenne muscular dystrophy (DMD) or any other
clinical application of ATL11022
. Since that time, the study protocol has been agreed and test article
(ATL1102) has been received at the Pharmaron site. Dosing of animals is scheduled to commence next
month with the study outcomes remaining on track for reporting in 1H’24.
Successful completion of the toxicology study is expected to be the final requisite step for the FDA to
allow dosing of ATL1102 for a term longer than 6 months in the US. Successful completion of the ninemonth chronic monkey toxicology study should also allow ANP to apply for expedited program status with
the US Food and Drug Administration (FDA) including Fast Track or potential Breakthrough Therapy
designation. US FDA has already granted ATL1102 an Orphan Drug Designation and a Rare Pediatric
Disease Designation for the treatment of DMD.
The reporting of key study findings in 1H’24 is around the same time as the six-month dosing results from
the ATL1102 in DMD Phase IIb clinical study are expected which could then allow the Company to share
with FDA and other regulatory bodies a compelling data package encompassing the Phase IIb study clinical
results along with the outcomes from the nine-month toxicology study for potential discussions with the
regulators on accelerated regulatory pathways to registration.
DMD combination therapy study
During the quarter, the dosing of animals has been completed in a muscular dystrophy (mdx) mouse
model of DMD to assess the potential clinical utility of ATL1102 in combination with dystrophin restoration
drugs to improve on therapeutic outcomes for patients with DMD on the dystrophin restoration drugs
(study details announced 12 September 2022).
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ANTISENSE THERAPEUTICS LIMITED - ABN 41 095 060 745
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In this blinded and controlled study, run under the collaborative research agreement with the Murdoch
Children’s Research Institute’s (MCRI), the mice were dosed alone with an antisense oligonucleotide to
CD49d (mouse equivalent of ATL1102) or control oligonucleotide or saline treatments, or a dystrophin
restoration drug alone and additionally a combination of the antisense oligonucleotide drug to CD49d with
a dystrophin restoration drug (morpholino oligonucleotide exon skipping drug of the same drug chemistry
as the exon skipping treatments marketed in the US). The samples were then processed under a two part
analysis: functional (effect on muscle) and cellular (RNA and protein levels). Functional analysis results
are due imminently with the cellular analysis to follow next month. Any applicable patent filings would be
made ahead of disclosure of the results.
The dystrophin restoration drugs are approved conditionally in the US for the treatment of approximately
a third of ambulant DMD patients with mutations to dystrophin exons 45, 51, and 53. As an indication of
the potential market for dystrophin restoration drugs , Sarepta Therapeutics Ltd recently announced
preliminary net product revenues for the fourth quarter and full-year 2022 are expected to total US$235.5
million and US$843.3 million, respectively for their 3 approved exon restoration drugs in the US alone.3
Long Neuro COVID-19
As previously advised4
, the Company had completed a successful world first Long COVID-19 study that
identified novel blood markers as potential targets for the diagnosis and treatment of the neurological
deficits of Long COVID e.g. brain fog measured using established memory tests. A subset of the study
results were included in a scientific publication pre-print.
(https://www.medrxiv.org/content/10.1101/2021.08.08.21261763v4).
Recent scientific publications report that neurological symptoms remain a major feature of Long COVID
with cognitive impairment identified in approximately a quarter of subjects at 12 months post SARS-CoV2
infection. (https://www.nature.com/articles/s41579-022-00846-2).
The Company has continued discussions with targeted companies to explore interest in
licensing/commercialising our Long Covid- 19 Intellectual Property, these discussions include an ongoing
dialogue with a diagnostic company on a potential development collaboration.
Limb Girdle Muscular Dystrophy R2
There are no approved treatments for LGMDR2. Having successfully demonstrated target drug activity
(reducing target and immune cell RNA in muscle) using an antisense oligonucleotide to CD49d (mouse
equivalent of ATL1102) in a dysferlin mutation animal model5
, during the quarter the Company advanced
its planning for a chronic mouse study to assess key disease progression endpoints. Mice with the dysferlin
mutation and disease characteristics have been sourced via Jain Foundation in the US with the study to
be initiated in January 2023. Mice will be dosed for 4 months with results to follow.
Board and Management changes
During the quarter Antisense has strengthened its leadership team with the appointment of Anthony
Filippis as the Company’s Chief Commercial Officer6
. Anthony’s key focus will be on the negotiation and
execution of partnering transactions, providing commercial advice and leadership on the Company’s
development programs and commercialisation plans, assisting in the process for accessing additional
development capital and supporting the Company’s investor relations activities with a strategic focus on
the US to increase the Company’s profile in that key market.
Non-Executive Director of the Company, Dr Gary Pace, retired from the Board of Directors following
completion of his director term at the 2022 Annual General Meeting7
.
As announced on 15 November 2022, following his significant tenure as the Company's Chief Executive
Officer and Managing Director (CEO), Mark Diamond advised of his retirement as CEO. Mark will continue
his responsibilities as CEO until a successor is appointed. The Board has commenced executive search
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ANTISENSE THERAPEUTICS LIMITED - ABN 41 095 060 745
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activity both externally and internally, for a new Chief Executive Officer that can build on Mark’s legacy
and spearhead the Company’s next phase of growth. Mark will continue as CEO providing leadership and
continuity until the appointment of a successor, to ensure a smooth transition.
Ongoing engagement with DMD community, investors and pharmaceutical companies
The Company continued its communication and active engagement with key opinion leaders, potential
collaborators, investors and commercial partners as a key operational priority. During the quarter the
Company presented and participated at the following events:
• Attendance at the JP Morgan Healthcare Week – San Francisco, USA, 9 - 11 January 2023
• US Institutional virtual roadshow – various dates October, November, December 2022
• Biotech & Medical Devices Webinar – Singapore, 3 November 2022
• Annual General Meeting Presentation – Melbourne, 17 November 2022
• US IR and Media engagements – October - December 2022
Cash Flow
As at 31 December 2022 the Company reported cash of $16.6 million.
The Company is focused on deploying its existing cash reserves in the most effective manner for advancing
the ATL1102 in DMD clinical development program as well as the progressing the new indications for
ATL1102, e.g. Limb Girdle (MDR2), dystrophin restoration combination study and Long Covid-19
collaboration.
During the quarter the Company made payments to related parties of the entity and their associates as
disclosed in Item 6 of the Appendix 4C amounting to $293,112. The payments are related to salaries,
directors’ fees and consulting fees on normal commercial terms.
This announcement has been authorised for release by the Board.
For more information please contact:
Antisense Therapeutics Investment Enquiries
Mark Diamond Gennadi Koutchin
Managing Director XEC Partners
+61 (0)3 9827 8999 [email protected]
www.antisense.com.au 1300 932 037