Updated: Jul 21, 2021
European investors have shown strong interest in Antisense Therapeutics Limited (ASX: ANP: FWB: AWY) since the company was dual listed in Europe in late November 2020. Volumes on German exchanges in March 2021 recorded over 100% increase from the month of December 2020. Since listing there have been some significant milestones achieved for Antisense, that have contributed towards a 110% increase in share price to 10 April 2021. We discuss these below.
Key points and milestones achieved.
Paediatric Investigation Plan (PIP) for ATL1102 in DMD submitted to the European Medicines Authority (EMA) in February 2021. The PIP is the vital first step ahead of the submission of Phase IIb trial application to the EMA expected in second half 2021. The PIP is the development plan aimed at ensuring all necessary data are obtained through studies in children to support the authorisation of a medicine for children. The PIP addresses the entire paediatric development program for ATL1102 in DMD.
The Company has commenced manufacture of ATL1102 active ingredient for the Phase IIB trial.
Assistance from Dr. Gil Price (Consultant Medical Director) in discussions with US Food and Drug Administration (FDA) and representation in March Rare Disease Day. Dr. Price brings to Antisense a deep experience in the DMD Drug development space. He was non-executive of Sarepta Therapeutics from 2007 to 2016 where he assisted guide the company from a US $80 million market capitalisation to a multi-billion-dollar company. The FDA organised Rare Disease day was a chance for the company to rain additional insights from the rare disease community and the FDA staff. The feedback from Dr. Price was that FDA was very pro-active in working and partnering with companies to assist “get this ball across the touchline in the most efficient and effective way” where the data supported this approach.
Appointment of Dr. Charmaine Gittleson as a non-executive director of the company in March. Dr. Gittleson is a senior executive with extensive international experience as a pharmaceutical physician and enterprise leader in drug pharmaceutical drug development, governance and risk management gained from her 15-year tenure at global pharmaceutical giant CSL Limited (ASX: CSL).
September 2020 the FDA granted the company rare paediatric disease (RPD) designation for ATL1102 for the treatment of DMD. This means if ATL1102 is approved by FDA it may gain priority review voucher (PRV). A PRV can be freely traded, and recent sales have been up to € 80 million.
The perceived failure in January of Sarepta’s exon skipping gene therapy to pass the phase 2 motor function test, has brought global biotech interest to Antisense and the anti-inflammatory actions of ATL1102. It is now widely acknowledged that treatment of DMD into the future will be a multi-faceted approach utilising a combination of intervention points and drugs – as shown in the diagram below, with ATL1102 working in the inflammation and fibrosis intervention points.
Overview of the company
Antisense Therapeutics is an Australian based biotechnology company - developing and commercializing antisense pharmaceuticals for large global unmet markets in rare diseases.
The products are licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS), an established leader in antisense drug development. The effect of this is to significantly de-risk the business and investment proposition as Ionis has already invested billions of dollars of the development and commercialisation of Ionis platform technology where drugs have already been through significant clinical trials and many have already been approved by US and EU regulatory authorities. Mark Diamond, CEO, of Antisense highlights this benefit to the company “there is a well-established development path and a data base of clinical experience for us to draw upon and leverage in the development of our own drugs”.
The Company’s main product is ATL1102 an antisense inhibitor of the CD49d receptor, for Duchenne Muscular Dystrophy (DMD).
Duchenne’s Muscular Dystrophy (DMD) is a devastating generic muscular wasting disorder with a 100% fatality rate. At present, the only treatment for the muscle inflammation that leads to the loss of muscle function and strength is corticosteroids which reduce inflammation and thus slow the progression of the disease. However,long-term use of these drugs can have major side effects such as weight gain, reduced bone density and stunted growth. The disease currently effects about 16 in 100,000 male births in the USA and 21 in 100,000 in Europe.
The global addressable market for DMD treatment is expected to be around USD $4.1 billion in 2023. Drug prices for rare diseases are expensive. In the USA prices for the last 3 DMD treatments approved in the last 4-year years range from €51,000 to €246,000 per patient per year.
The company reported very encouraging signs of efficacy from a small Phase II study last year. Patients treated with ATL1102 for 6 months showed improvement in upper limb function and preservation of muscle mass. Phase IIb will be a larger study of non-ambulant (wheelchair bound) DMD patients in Europe in the latter half of 2021.The trial is expected to be an approvable study, so if the results are successful the company can move quickly towards commercialistion and revenue generation.
A recent research report valued Antisense at AUD $0.57 or €0.36 a share
May 2021 CEO presentation can be viewed below
A June 2021 research report suggests a 12-month value of around AUD $0.48 or € 0.30 a share. This can be downloaded below.
In July 2021 the company produced an excellent video explaining the value proposition and significant value drivers.