Two leading Australian biotech companies working to solve global unmet needs
Updated: Feb 19, 2022
Antisense Therapeutics Limited (ASX: ANP; FSE: AWY)
Antisense is an Australian publicly listed biotechnology company (with German dual-listing), developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases.
The Company is developing ATL1102, an antisense inhibitor of the CD49d receptor, for Duchenne Muscular Dystrophy (DMD) patients and recently reported highly promising Phase II trial results and is moving forward with Phase IIb/III Clinical trials in Europe in 2022.
The company’s drugs are licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS) - a global established leader in antisense drug development.
The Company’s mission and purpose statement may be viewed below.
What are antisense Drugs?
Antisense drugs are small snippets of synthetic genetic material that bind to RNA (short for ribonucleic acid). The main factor driving where the antisense drug binds to the RNA is the nucleic acid sequence used. Nucleic acids consist of a chain of linked units called nucleotides. Antisense drugs are typically 12-21 nucleotides that are chemically modified to bring about the appropriate drug response.
There is increasing evidence that antisense oligonucleotides (ASOs) are showing promise as a therapeutic platform for treating neurological diseases. ASOs are designed to bind to the RNAs encoded by a target gene, thereby suppressing expression by catalysing degradation of those RNAs or to elevate expression by correcting faulty RNA splicing.
Antisense drugs are currently in development for amyotrophic lateral sclerosis, Huntington’s disease, Alzheimer’s disease, Parkinson’s, disease and Angelman syndrome – and Duchenne Muscular Dystrophy (DMD).
Below is a video explaining how antisense drugs work
Why the urgency to treat Duchenne Muscular Dystrophy?
Duchenne Muscular dystrophy is a sever type of muscular dystrophy that primarily affects boys. It is caused by a mutation in the gene for the protein dystrophin which is vital to maintain the muscle’s fibre cell membrane. Muscle weakness usually commences around age of 4 and very quickly deteriorates. Most are unable to walk by age 12 and most do not survive past age 18. There is currently no know cure. Currently about 1 in 3,500 boys are impacted by DMD. This translates to about 48,000 in Europe and the United States.
What is Antisense doing that is so special?
The Company is developing ATL1102, an antisense inhibitor of the CD49d receptor, for Duchenne muscular dystrophy (DMD) patients.
In 2021 the company reported highly promising Phase II trial results. ATL1102 has also successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients with relapsing-remitting multiple sclerosis (RRMS).
During the December 2021 quarter the company received a positive final opinion from the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) for the Paediatric Investigation Plan (PIP) for the development of ATL1102 for Duchenne Muscular Dystrophy (DMD). This positions the company for commencement of Phase IIb/III trials in Europe in 2022. These trials will be a multi-centre, randomised, double-blind, placebo-controlled study to determine the efficacy, safety, and pharmacokinetic profile of ATL1102 over 52 weeks in non-ambulatory participants with DMD.
Professor Thomas Voit (Director of NIHR GOSH UCL Biomedical Research Centre, UK) has been appointed as the coordinating Principal Investigator (CPI) of the trial.
Also during the December 2021 quarter the company received gross proceeds of AU $22.6 million via a capital raising for funds to be deployed towards preparation activities for imitation of the Phase IIb/III trial of ATL1102 for DMD in Europe.
What the research reports are saying
ATL1102 is the only antisense drug currently in Phase IIb/III trials for treatment of Duchenne Muscular Dystrophy (DMD) for which the only currently available treatments are corticosteroids. Research on the company has valued shares at a significant premium to current share price.
Corporate Connect in October 2021 valued the shares at AU $0.48 or €0.304. The report is attached here
The company’s December 2021 investor deck can be viewed here
The company has delivered excellent shareholder value over last 5 years of 300% to mid-February 2022. Antisense Therapeutics is also traded in Germany on Frankfurt Stock Exchange, Munich, Stuttgart, Berlin and Tradegate exchanges.
Recce Pharmaceuticals Limited (ASX: RCE; FSE: R9Q)
Recce Pharmaceuticals Ltd (ASX: RCE, FSE: R9Q) is developing New Classes of Synthetic Anti-Infectives designed to address the urgent global health problems of antibiotic resistant superbugs and emerging viral pathogens.
The World Health Organization notes that antimicrobial resistance one of the greatest global health threats. Investors are also looking closely at the sector. The Former U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb is focused on investing in companies developing new antibiotics. “I’m very interested in finding opportunities in the anti-infective space, particularly around multi-drug resistant organisms,” Gottlieb said in an interview. “Anti-infectives have been unloved for a very long time. There’s a huge clinical need.”
Recce’s anti-infective pipeline includes three patented, broad-spectrum, synthetic polymer anti-infectives: RECCE 327 as an intravenous and topical therapy that is being developed for the treatment of serious and potentially life-threatening infections due to Gram-positive and Gram-negative bacteria including their superbug foormations.
Through their multi-layered mechanisms of action, Recce’s anti-infectives have the potential to overcome the hypercellular mutation of bacteria and viruses – the challenge of all existing antibiotics to date.
How do the drugs work?
Here is an excellent video explaining the mechanisms of Recce’s patented drugs.
Current antibiotics are usually typically recurring in certain fungi and soil bacteria. Recce’s anti-infectives are synthetic and based upon a patented polymeric structure and have been specifically engineered to overcome resistance.
With traditional antibiotics they operate on a ‘lock and key’ mechanism and only bind to a few active sites on the bacterial target and if a mutation is introduced to target site, the antibiotic will cease to be effective.
RECCE 327 may be administered for intravenous, topical, nasal, and inhaled use. The patented mechanism of action will continuously kill bacteria without tendency for the emergence of resistance – even through repeated use.
The FDA has awarded RECCE 327 Qualified Infectious Disease product designation under the generating Antibiotic Initiatives Now (GAIN) Act. This provides possible Fast Track Designation and 10 years of post-approval market exclusivity.
Significantly RECCE 327 has been included in Pew Charitable Trusts Global New Antibiotics as the world’s only synthetic polymer and sepsis drug in development.
Why the urgency to treat superbugs and sepsis and dangerous pathogens?
The World Health Organisation (WHO) released a report in 2021 noting the emergence and spread of drug-resistant pathogens continues to threaten our ability to treat common infections.
WHO found especially alarming the rapid global spread of multi- and pan-resistant bacteria (also known as “superbugs”) that cause infections that are not treatable with existing antimicrobial medicines such as antibiotics. The World Health Organisation has declared that anti-microbial resistance (AMR) is one of the top ten global public health threats facing the world.
In 2020 WHO also released a report on sepsis noting sepsis kills eleven million people each year, many of them children and it disables millions more. Sepsis occurs in response to an infection. When sepsis fails to be diagnosed early and managed promptly, it can lead to septic shock, multiple organ failure and death. Patients who are critically ill with severe COVID-19 and other infectious diseases are at higher risk of developing and dying from sepsis.
Clinical trial development
The Company has two clinical trials in progress.
The first is a Phase I/II broad-spectrum efficacy across all patients for R327 to treat burn wound infections. The trial is sponsored by the West Australian Health Department at Fiona Stanley Hospital. During the December 2021 quarter clinicians reported visible signs reductions of broad ranging bacterial infections within the first 24 hours of R327 treatment across all patients treated to date.
The second clinical trial is a Phase I intravenous trial to test efficacy for sepsis. In January 2022 the Company announced 9 subjects in cohort one intravenously dosed with R327 at 50mg met all endpoints, indicating safe and well tolerated. The Independent Safety Committee has cleared the second study cohort dosing at 150mg.
The path ahead
The world is looking for solutions to anti-microbial resistance and the CEO James Graham discuss the company’s vision and passion in the interview below
The Company delivered the opening R&D address at the World Anti-Microbial Resistance (AMR) congress in 2021. With two human clinical trials of R327 underway and expected positive news flow from real world patient data 2022 promises to be an exciting time ahead.
The company’s January 2022 Investor deck can be viewed here
The company has delivered excellent shareholder value over last 5 years of 330% to mid-February 2022. Recce Pharmaceuticals is also traded in Germany on Frankfurt Stock Exchange, Munich, Stuttgart and Tradegate exchanges.