It is estimated that over 300 million people in the world are impacted by rare diseases.
In a recent study it was estimated that rare diseases currently affect about 3.5% – 5.9% of the worldwide population. This is equivalent to a conservative estimate of 300 million people worldwide (4% of an estimated world population of 7.5 billion).
One of the most debilitating diseases is Duchenne Muscular Dystrophy (DMD). This is a devastating genetic muscular disease caused by loss of dystrophin with progressive muscle wasting and associated muscle injury leading to inflammation and fibrosis with 100% mortality.
DMD impacts boys with an incidence rate of 1 in every 3,500 newborns with about 18,000 boys impacted in the United States and about 26,000 in Europe.
The key challenge in management of DMD patients is to reduce the inflammation and muscle fibre damage.
There is currently no cure for DMD.
There are treatments available to lessen the pain and reduce some inflammation such as Corticosteroids. These also have significant side effects such as weight gain, reduced bone density and growth retardation.
One of the key challenges in treating DMD is managing the inflammation-mediated muscle damage that contributes to the progression of the disease.
Antisense Therapeutics are passionate about finding a therapy to reduce the inflammation and stop the progression of this terrible disease and making life easier for the thousands of young boys inflicted.
Addressable market
The DMD market is expected to be worth US $4.1 billion by 2023 and US $10 billion by 2030.
DMD treatments command high prices. Prices in the USA for 3 DMD
treatments approved in the past 4 years range from US $63,000 – US $300,000 per patient per year.
A drug called Tysabri which has been approved to treat relapsing forms of multiple sclerosis (MS) and moderate to severe Crohn’s disease generates sales of over US$1.9 billion a year.
The clinical trails being conducted may show efficacy for a wider range of indications for anti-inflammatory inflictions. The market for anti-inflammatory drugs is immense and is expected to reach US $191 billion by 2027.
It is expected the company will seek to commence negotiations on out licencing the drug to pharmaceutical companies once the clinical trails in Europe have commenced.
How Antisense solves the problem
Antisense Therapeutics lead drug ATL1102 blocks a key inflammatory signal which facilitates survival and multiplication of the white blood cells from the bloodstream too the site of the inflammation.
ATL1102 is an antisense oligonucleotide drug that triggers the breakdown of the messenger RNA (mRNA) carrying the instructions for the inflammatory signal from the nucleus of the site to the site of protein synthesis – thus inhibiting the expression of the inflammatory signal on the cell surface.
ATL1102 was developed originally by Ionis Pharmaceuticals and is licenced to Antisense Therapeutics. Ionis is a global leader in RNA targeted therapeutics and is a sustainably profitable, multi-product company delivering transformative global medicines.
In 2020 Antisense Therapeutics successfully completed a Phase 2 clinical study involving 9 boys with DMD who were confined to a wheelchair (non-ambulant). After 24 weeks of treatment with ATL1102 the boys showed improvement or slower-than-expected decline in measures of upper limb strength and function.
There were also improvements in several biomarkers including reductions in targeted populations of inflammatory cells, preservation of muscle mass and reductions in the fat percentage in forearm muscles.
These excellent results resulted in the FDA granting ATL1102 a Rare Paediatric Disease Designation for DMD in September 2020 which means the company is now eligible for a lucrative Priority Review Designation in the US.
In early 2022 Antisense Therapeutics presented new plasma protein data from the Phase II trial of ATL1102 in DMD at the Muscular Dystrophy Association Clinical & Scientific Conference showing a statistically mean increase in plasma basic metabolic panel. This is supportive of ATL1102’s potential to promote bone regeneration and improve bone density.
The company is in advanced stages of planning for Phase IIb/III clinical trials in Europe in 2022 leading to commercialisation and revenue generation with significant value inflection for the company.
Why an investment in Antisense Therapeutics should be considered
Antisense Therapeutics is in sound financial position and well placed to commence early clinical testing for Phase IIb/III approvable studies. In addition, the company is exploring new indications for its lead drug, ATL1102, which could significantly increase the addressable market and market valuation of the company. Further the Company’s board and management are exceptionally well qualified to lead to company through its next stage of clinical trials and commercialization.
In November 2021 the company received positive news from the Paediatric Committee (PDCO) of the European Medicines Agency (EMA). PDCO adopted a positive final opinion on the Company’s Paediatric investigation Plan (PIP) paving the way for commencement of Phase IIb/III clinical trials in Europe.
The company is working with a tier 1 contract research association (CRO) in Europe to conduct clinical trials. The pivotal trial is being managed by Parexel International – a large globally renowned CRO that has managed more than 1,000 rare disease programs in the last 5 years.
In 2021 the Company completed a capital raise with significant commitments from institutional and exciting shareholders. As of March 2022, the Company’s cash reserves stood at Au $21.7 million and is well-placed to commence initial approvable clinical trial work.
The Company is also exploring new indications for ATL1102 and in 2022 commenced an inflammatory muscle disease animal model with the Murdoch Children’s Hospital to investigate the therapeutic potential of ATL1102 in a new muscle disease, with no current effective treatments.
Early in 2022 the Company commenced a collaboration to study the neurological aspects of Long COVID-19 with US based researchers at the Northwestern Medicine Neuro-COVID clinic in Chicago. Of the first 80 million people in the US diagnosed as infected and surviving COVID-19, about 30% of hospitalised patients and 45% of non-hospitalised patients have developed some manifestation of Long COVID-19 syndrome which suggests more than 24 million people may be impacted by the condition and a massive addressable market.
The Board and management of Antisense is exceptionally well qualified for a small-cap biotech. In late 2021, Dr. Gil Price was appointed to the board. Dr. Price was a director of Sarepta Therapeutics for 9 years – the first company to obtain DMD drug approval. Dr. Charmaine Gittleson was appointed Chairperson of Antisense Therapeutics in 2021 and brings extensive biotech and big-pharma experience to board, including 15 years global ASX-listed speciality biotechnology giant CSL Limited. The CEO, Mark Diamond, has over 30 years’ experience in the pharmaceutical and biotechnology industry.
The Company's May 2022 Investor Deck can be downloaded here.