Antisense Therapeutics is an Australian biotechnology company developing antisense oligonucleotide (ASO) therapies for the treatment of rare diseases. Its lead asset, ATL1102, is being investigated as a targeted anti-inflammatory therapy to treat Duchenne muscular dystrophy (DMD). In a positive strategic pivot, trial design was amended to a smaller Phase IIb study (n=42 vs 108) that should provide a nearer-term catalyst, if positive readouts are achieved. As a result of the reduced trial costs, the company’s cash runway is anticipated to be extended (net cash at end-June 2022 of A$19.2m) into Q4 CY23. Management envisages a need to raise funds in the mid-single digit (A$m) region, which it anticipates will fund operations to trial readouts in Q124.
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Antisense Therapeutics |
Breaking the mould in DMD treatment
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Pharma and biotech |
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11 October 2022 |
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Antisense Therapeutics is an Australian biotechnology company developing antisense oligonucleotide (ASO) therapies for the treatment of rare diseases. Its lead asset, ATL1102, is being investigated as a targeted anti-inflammatory therapy to treat Duchenne muscular dystrophy (DMD). In a positive strategic pivot, trial design was amended to a smaller Phase IIb study (n=42 vs 108) that should provide a nearer-term catalyst, if positive readouts are achieved. As a result of the reduced trial costs, the company’s cash runway is anticipated to be extended (net cash at end-June 2022 of A$19.2m) into Q4 CY23. Management envisages a need to raise funds in the mid-single digit (A$m) region, which it anticipates will fund operations to trial readouts in Q124.
ASO’s silencing genes to treat disease
To date, the FDA has granted approval to four ASO therapies to treat DMD; however, their clinical uptake has been limited due to a perceived lack of efficacy. Unlike existing therapies, ATL1102’s unique mechanism of action (inhibition of CD49d) aims to reduce inflammation in DMD. We believe this offers ATL1102 differentiation to marketed ASO therapies and potential combinational synergies with existing DMD treatments which Antisense is investigating in preclinical studies.
ATL1102 proof-of-concept demonstrated
In an Australian Phase IIa trial in non-ambulant DMD patients, ATL1102 demonstrated significant improvements in muscle strength (pinch and grip), increased muscle area and improved performance of upper limb function (PUL 2.0). PUL 2.0 scores are a critical efficacy endpoint when seeking new drug approval in non-ambulant DMD patients. This sub-population represents up to 56% of DMD patients and, with limited treatment options, a significant market opportunity, in our view.
Pivotal Phase IIb on the horizon
Management anticipates study sites to be initiated in Q422 for the Phase IIb multicentre, randomised, double-blind, placebo-controlled, open-label extension study to assess the efficacy and safety of ATL1102. Patients will be treated with either 25mg or 50mg per week of ATL1102, with advantages over existing corticosteroids that lack efficacy and often associated with unwanted side effects. In addition, Antisense is progressing the development of ATL1102 in limb girdle muscular dystrophy R2 and looking to exploit new IP it has generated using SomaScan, a bio-diagnostic platform, for the diagnosis and prognosis of neurological disorders in long COVID-19 patients.
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Consensus estimates
Source: Antisense Therapeutics, Refinitiv |
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