ATL1102 / Antisense Therap, Ionis - LARVOL DELTA

Antisense to CD49d Reduced Seizure Frequency over a 15-Day Period in a Pilocarpine Induced Model of Epilepsy (AAN 2025) - "This is the first study with ASO to CD49d in mouse pilocarpine AE models and support potential use of ATL1102 in RE and AE patients resistant to current treatments."

CNS Disorders • Epilepsy • Immunology • Inflammation • Multiple Sclerosis • ITGA4

A Study of ATL1102 or Placebo in Participants with Non-ambulatory Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Terminated | Sponsor: Percheron Therapeutics | Active, not recruiting ➔ Terminated; The study failed to meet its primary or secondary efficacy endpoints. In consultation with investigators, the sponsor determined that it is not in the best interests of patients for the study to continue.

Trial termination • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • ITGA4

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: Percheron Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • ITGA4

Mdx mice dosed with antisense to CD49d & dystrophin exon skip morpholino; improved muscle force & affected pathways support ATL1102 combination in DMD (MDA 2024) - "The affected targets were involved in immune response (BTLA*, PPML1, TNFSM13), lipolysis (FAPBP4*, G0S2), fibrosis (IGFBP-7, Calu), muscle cell (ASB15) and muscle stem cell function (ADAM10, Mt-Tp, MyoM1). These genes can play roles in EDL muscle function improvement in mdx mice treated with ASO+PMO and pathways support the rationale for ATL1102 combination therapy in DMD patients."

Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Immunology • ADAM10 • BTLA • G0S2 • IGFBP7 • ITGA4

ATL1102 treatment of non-ambulant boys with DMD stabilizes function modifying plasma proteins with roles in immune, fibrosis, bone & growth physiology (MDA 2024) - "ATL1102 increases of IGF-I suggest a potential for increasing linear growth. These proteins involved in muscle and bone physiology and linear growth can be important in osteopenia and growth retardation presenting in DMD patients, exacerbated by corticosteroids, and treatment with ATL1102 may improve patient outcomes."

Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Muscular Dystrophy • Osteoporosis • Rheumatology • BMP6 • IGF1 • ITGA4 • TGFB1 • THBS1

Design of a Phase 2b study evaluating the efficacy and safety of ATL1102 in non-ambulant DMD (MDA 2024) - P2 | "Additional endpoints for assessment of muscle function, strength, respiratory function and Quality of Life will also be evaluated. The study is ongoing at multiple sites across the UK, Australia, Turkey and Bulgaria."

Clinical • P2b data • Duchenne Muscular Dystrophy • ITGA4

A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice. (PubMed, PLoS One) - "ATL1102, a novel antisense drug being developed for the treatment of inflammation that exacerbates muscle fibre damage in DMD, appears to be safe and well tolerated in non-ambulant boys with DMD. The apparent stabilisation observed on multiple muscle disease progression parameters assessed over the study duration support the continued development of ATL1102 for the treatment of DMD."

Journal • P2 data • Preclinical • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Inflammation • Muscular Dystrophy • ITGA4

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=45 | Recruiting | Sponsor: Antisense Therapeutics Limited

New P2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy. (PubMed, Pharmaceutics) - "These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown."

Journal • Review • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Predictors of Outcomes Measured after Loss of Ambulation in Duchenne Muscular Dystrophy: A Literature Review and Grading of the Evidence (ISPOR 2022) - "Predictors of cardiac health and function included body mass index, cardiac medication (i.e., angiotensin-converting enzyme inhibitors and β-blockers), carvedilol, DMD genetic modifiers, DMD mutation type, glucocorticoid exposure, idebenone, and ventilation support. Predictors of respiratory health and function included ataluren, DMD genetic modifiers, DMD mutation type, eteplirsen, glucocorticoid exposure, idebenone, ventilation support, and weight...Predictors of upper extremity function included glucocorticoid exposure and ATL1102 (an antisense oligonucleotide)... Our review delineates predictors of outcomes measured after loss of ambulation in DMD and helps raise awareness of relationships between complications, milestones, and clinical interventions across stages of the disease."

Biomarker • Review • Duchenne Muscular Dystrophy • Gastrointestinal Disorder • Genetic Disorders • Muscular Dystrophy

Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis. (PubMed, PLoS One) - "This study provides a current summary of prognostic indicators of disease progression in DMD, which will help inform the design of comparative analyses and future data collection initiatives in this patient population."

Biomarker • Journal • Review • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Antisense Therapeutics (ASX:ANP) submits plan for muscular dystrophy drug (The Market Herald) - "Antisense Therapeutics (ANP) has submitted a paediatric investigation plan (PIP) to the European Medicines Agency (EMA) Paediatric Committee (PDCO)....The PIP submission includes a planned phase 2b clinical trial of ATL1102 that will include non-ambulant DMD patients and will be conducted in Europe....Once patients are recruited, they'll be dosed with the muscular dystrophy drug for 12 months at two levels....Antisense is now manufacturing the ATL1102 active ingredient for the trial and is planning to have this formulated into an injectable product in the June quarter."

New P2 trial • Duchenne Muscular Dystrophy

Antisense Therapeutics (ASX:ANP) locks in U.S. FDA discussions for ATL1102 (The Market Herald) - "The Australian pharmaceutical player has been granted a Type C guidance meeting with the federal agency, scheduled for April 19, 2021....Antisense will gear the discussions towards developing its ATL1102 treatment in Duchenne muscular dystrophy (DMD) in the U.S....The ASX-lister is also requesting the FDA consider allowing data from previously conducted monkey toxicity studies and recently obtained data from a six-month clinical safety trial...Antisense expects to provide an update in late May 2021 upon receipt of formal minutes of the meeting."

FDA event • Duchenne Muscular Dystrophy

Antisense Therapeutics (ASX:ANP) receives Orphan Drug Designation for ATL1102 in DMD (The Market Herald) - "Antisense Therapeutics (ANP) has received Orphan Drug Designation for ATL1102 in Duchenne muscular dystrophy (DMD). The orphan drug designation was granted by the European Commission following a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products."

Orphan drug • Duchenne Muscular Dystrophy

".@US_FDA Orphan Status For @antisense_bio ATL1102 For DMD - subscribe to Biotech Daily https://t.co/4E9331THtC" (@biotech_daily)

[VIRTUAL] ATL1102 treatment improves PUL2.0 in non-ambulant boys with Duchenne muscular dystrophy compared to a natural history control (WMS 2020) - No abstract available

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Antisense ATL1102 final phase II DMD results meet primary endpoint and exceed expectations on secondary endpoints (Businesswire) - P2, N=9; Sponsor: Antisense Therapeutics Limited; "Antisense Therapeutics...is pleased to advise that the Phase II clinical trial of ANP’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) has met its primary endpoint confirming the safety and tolerability of ATL1102...Importantly, the final trial results have also confirmed the drug’s positive effects on the secondary trial endpoints that assessed the drug’s activity and efficacy...move ATL1102 into a Phase IIb trial...next milestone in preparation for submission of clinical trial application for a Phase IIb trial in Europe and UK. The Company is also in the process of preparing submissions for Orphan Drug Designation for ATL1102’s use in DMD in the US and the EU."

European regulatory • Orphan drug • P2b data • Duchenne Muscular Dystrophy

"Antisense ATL1102 Final Phase II DMD Results Meet Primary Endpoint and Exceed Expectations on Secondary Endpoints https://t.co/j2Er61FllC" (@NewsFromBW)

ATL1102 for MS – FDA response to phase IIb study plans (Antisense Press Release) - "Antisense Therapeutics...is pleased to announce that the US Food and Drug Administration (FDA) has responded affirmatively to the Company’s plan to submit a U.S. Investigational New Drug (IND) application for initiation of longer term Phase IIb human trials of ATL1102 for the treatment of Multiple Sclerosis (MS). Supportive guidance was obtained from the agency’s Pre-IND assessment of the development strategy for ATL1102, including plans for a Phase IIb study in MS patients."

Anticipated new P2b trial • FDA event • Multiple Sclerosis

Antisense Therapeutics announces additional preliminary data from the ATL1102 phase II DMD trial presented at the 2019 Action Duchenne International Conference (PRNewswire) - P2, N=7; "Antisense Therapeutics...is pleased to advise that additional preliminary data analyses from the seven patients who have completed their 24 weeks of dosing in the ATL1102 Phase II DMD clinical trial was presented by Dr Ian Woodcock...at the 2019 Action Duchenne International Conference, Hinkley, UK on 15 November 2019...These results continue to appear highly supportive of the Company's clinical development program, with plans for a Phase IIb clinical trial of ATL1102 in DMD presently being reviewed with European regulatory authorities at Scientific Advice (SA) meetings."

European regulatory • P2 data

Melb firm trials muscular dystrophy drug (News.com.au) - "Antisense Therapeutics is in talks with regulators in Europe to begin what could be a pivotal trial of...ATL1102, a possible treatment for boys...with Duchenne muscular dystrophy...Antisense is hoping to begin a Phase IIb trial in Europe in the second half of the year, comparing treatment with ATL1102 to steroids....Given the limited number of options for treating the disease, European regulators have indicated ATL1102 could be approved straight away if the results are positive, bypassing the customary additional studies."

New P2b trial

Antisense Therapeutics share price soars 89% with positive trial results (Money Morning Australia) - "Biopharmaceutical drug discovery and development small-cap Antisense Therapeutics Ltd...have seen a substantial spike in their share price today, up 89.36% at time of writing. The surge appears to be subsequent to the positive results announced for the Phase II clinical trial for their immunomodulatory therapy drug, which treats Duchenne muscular dystrophy (DMD)."

P2 data • Stock price

Positive preliminary results from ATL1102 for DMD phase II trial (PRNewswire) - "Antisense Therapeutics...is pleased to advise that a review of the preliminary data from the 6 patients who have completed their 24 weeks of dosing in the Phase II clinical trial of ANP's immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) therapy, is indicative of a positive drug effect...The results appear highly supportive of the Company's plans for a Phase IIb clinical trial of ATL1102 in DMD with these plans to be reviewed with three European regulatory authorities at Scientific Advice meetings with the first meeting commencing next month....The preliminary data including any further analyzed data is planned to be presented by Dr Woodcock at the Action Duchenne Conference in the UK on 15 - 16 November 2019."

European regulatory • P2 data

"$IONS ATL1102 ANP.ASX announce https://t.co/dvh2QOVGDA" (@LivingOTHedge)