Has anyone thought about Spinal Muscular Atrophy (SMA) lately?
--> (SMA is a genetic condition. It affects the nerves that control muscle movement (the motor neurons). In someone with SMA, the motor neurons in the spinal cord do not work properly. The messages that the brain tries to send along these motor neurons do not get through to the muscles.) --> Similarity, both DMD and SMA are rare neurodegenerative diseases, which cause progressive, proximal-to-distal muscular weakness leading to loss of ambulation and motor function. --> SMA : Symptoms typically start between 6 and 18 months of age. Depending on the severity of symptoms, children with type 2 may have a normal life span. Type 3 is a milder form of SMA. It's also known as Kugelberg-Welander disease and resembles muscular dystrophy.
I came across this news on Herald Sun yesterday (04-04-2021) - Push to get $3.5m drug approved to save kids' live.
"Parents are fighting to make breakthrough gene therapy — which costs $3.5m for a one-off dose — affordable, and to have all Aussie kids screened...It is one of the most expensive drugs to ever hit the market, but at $3.5 million per one-off dose, it can mean the difference between life and death for a baby born with spinal muscular atrophy (SMA)....Zolgensma, a gene therapy, is approved by the Therapeutic Good Administration (TGA) but is currently facing a Pharmaceutical Benefits Advisory Board (PBAC) decision on whether to list it for subsidy on the Pharmaceutical Benefits Scheme."
The FDA by far has approved 3 medications to treat SMA: 1. Nusinersen (Spinraza) - marketed by Biogen 2. Onasemnogene abeparvovec-xioi (Zolgensma) - marketed by Novartis Gene Therapies and 3. Risdiplam (Evrysdi). - marketed by Genentech/Roche
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4240428/pdf/NEUROLOGY2013568121.pdf " Conclusions: In patients with RRMS, ATL1102 significantly reduced disease activity after 8 weeks of treatment and was generally well-tolerated. This trial provides evidence for the first time that antisense oligonucleotides may be used as a therapeutic approach in neuroimmunologic disorders." -- Note: Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis (ALS) are also part of the neuroimmunologic disorders.
From Morgan desk note - ANP- Sensing Big Things Ahead - April 2020 "Mechanism of action ANP’s primary drug ATL1102 is a second-generation antisense inhibitor of CD49d, a subunit of the VLA-4 (Very Late Antigen-4) receptor found on the surface of lymphocytes (a type of white blood cell). Antisense drugs (designed to bind to complementary messenger RNA, or mRNA, a molecule central to translating DNA into protein) have been used over the years to treat such diseases as cancer, Amyotrophic lateral sclerosis (ALS), diabetes, and those with an inflammatory component like asthma and arthritis."
https://florey.edu.au/science-research/research-teams/antisense-therapeutic-laboratory "Our current aims are the development of gene-specific antisense therapy for spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).These are diseases of motor neuron degeneration in the brain and spinal cord leading to paralysis of voluntary muscles and death by respiratory failure. While SMA primarily affects infants and children, the average age of onset for ALS is around 55. "
If you could still remember our Principal Investigator (Dr. Ian Woodcock MD) and Co-Investigator (Professor Monique Ryan MD) for (ATL1102 for DMD Phase II Clinical Trial) See their Research Interest below: 1) Dr Ian Woodcock has extensive research interests within the field of paediatric neurology and in particular in Neuromuscular disorders. Ian is currently working through a PhD at the University of Melbourne and is the primary investigator in two clinical trials: one for children with Duchenne Muscular Dystrophy and the other is the world’s first clinical trial for children and young people with fasioscapulohumeral muscular dystrophy(FSHD).Ian has extensive experience as a sub-investigator in multiple large-scale international industry-sponsored drug trials, including breakthrough drug trials in Spinal Muscular Atrophy. Ian is due to be an investigator for and run a clinical trial in Friedrich’s Ataxia starting in early 2019.
2) Associate Professor Ryan is head of the multidisciplinary Neuromuscular Clinic and Neuromuscular Research Unit at The Royal Children's Hospital (RCH) which includes a growing team of clinicians and researchers aimed at improving diagnosis and management of children affected by muscular dystrophies, myopathies and neuropathies. Projects: Clinical trials into antisense oligonucleotide and nonsense–mediated read-through of Duchenne muscular dystrophy, bisphosphonate therapy and Nutriceuticals for DMD, and antisense oligonucleotide therapy forspinal muscular atrophy type 1.
Additional New Indications (from ANP Investor Presentation August 2020) − Several orphan indications where ATL1102 has an attractive profile − Once new IP protection is in place, ANP could move directly into clinical studies based on existing safety data − Potential for scientific collaboration/partnering with pharma and non-dilutive grant funding
Partnering/Corporate engagement ( from AGM Presentation December 2020) "As previously noted, we have received inbound interest from pharmaceutical companies and potential strategic investors in the Company and its programs. To date our approach has been to engage with these groups to explore such interest. It is important to note that such parties can often seek to undertake comprehensive due diligence ahead of any capital injection and/or collaborative commitment. "
Again, we just can't simply rule out ANP might involve in Spinal Muscular Atrophy (SMA) or other rare diseases (either pre-clinical trial/clinical trial) at background.
Will we work with Dr Ian Woodcock/Associate Professor Ryan again? OR Will we collaborate with Florey Institute for clinical development for both spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) in the near future?
Never say never!
ANP Price at posting:
22.0¢ Sentiment: Buy Disclosure: Held
