censavudine (TPN-101) / Oncolys BioPharma, Transposon Therap, Yale University - LARVOL DELTA

TPN-101 in Aicardi-Goutières Syndrome (AGS) (clinicaltrials.gov) - P2 | N=16 | Active, not recruiting | Sponsor: Transposon Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Phase classification: P2a ➔ P2 | Trial completion date: Apr 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Enrollment closed • Phase classification • Trial completion date • Trial primary completion date • Aicardi Goutieres Syndrome • Genetic Disorders • RNASEH2B • RNASEH2C • TREX1

Transposon to Present Results from Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related ALS/FTD at the 35th International Symposium on ALS/MND (PRNewswire) - "Transposon Therapeutics...today announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD) will be presented at the 35th International Symposium on ALS/MND. The symposium will take place in Montreal, Canada, on December 6-8, 2024."

P2a data • Amyotrophic Lateral Sclerosis • Dementia

A Phase 2A study of TPN-101, a nucleoside reverse transcriptase inhibitor, in patients witH C9ORF72-related ALS/FTD (ALS-MND 2024) - "TPN-101 was well-tolerated in participants with C9ORF72-HRE ALS/FTD. Biomarker and clinical effects provide evidence that TPN-101 may have disease-modifying effects in patients with these disorders and support advancing to larger studies."

Clinical • P2a data • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation • IL6 • NEFH • NEFL • SPP1 • TARDBP

Transposon to Present Results from Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related ALS/FTD at the 7th Annual ALS ONE Research Symposium (PRNewswire) - "Transposon Therapeutics...today announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD) will be presented at the 7th Annual ALS ONE Research Symposium. The symposium will take place virtually on November 13-15, 2024."

P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Dementia

Transposon to Present Results from Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related ALS/FTD at the 2024 Annual NEALS Meeting (PRNewswire) - "Transposon Therapeutics...announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD) have been accepted for both oral and poster presentation at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting."

P2a data • Amyotrophic Lateral Sclerosis • Frontotemporal Lobar Degeneration

Oncolys BioPharma Inc. (4588 Growth) Telomelysin is moving full speed ahead toward filing for approval. Detailed results of OBP-601 for C9-ALS are awaited. [Google translation] (Yahoo Finance) - "Oncolys BioPharma Inc...Telomelysin is moving full speed ahead towards filing OBP-601 detailed results for C9-ALS are awaited...OBP-601...has shown excellent efficacy in Phase 2a. This makes it highly likely that the drug will move to Phase 3 for PSP and ALS as early as 2024. However, judging from its current fundraising plans, Transposon is unlikely to pursue Phase 3 development for PSP and ALS or other neurodegenerative diseases all on its own. Several major pharmaceutical companies interested in this drug have already begun due diligence activities, but their core interest is in ALS, and due diligence activities are expected to begin in earnest after the detailed results of Phase 2 for ALS are announced within the year."

New P3 trial • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Progressive Supranuclear Palsy

Current status of the small molecule anti-HIV drugs in the pipeline or recently approved. (PubMed, Bioorg Med Chem) - "These compounds include analogues of nucleoside reverse transcriptase inhibitors (NRTIs) - islatravir and censavudine; non-nucleoside reverse transcriptase inhibitors (NNRTIs) - Rilpivirine, elsulfavirine and doravirine; integrase inhibitors namely cabotegravir and dolutegravir and chemokine coreceptors 5 and 2 (CC5/CCR2) antagonists for example cenicriviroc. Also, fostemsavir is being developed as an attachment inhibitor while lenacapavir, VH4004280 and VH4011499 are capsid inhibitors. Others are maturation inhibitors such as GSK-254, GSK3532795, GSK3739937, GSK2838232, and other compounds labelled as miscellaneous (do not belong to the classical groups of anti-HIV drugs or to the newer classes) such as obefazimod and BIT225. There is a considerable progress in the development of new anti-HIV drugs and the effort will continue since HIV infections has no cure or vaccine till now. Efforts are needed to reduce the toxicity of available drugs or discover new drugs with new..."

Journal • Review • Human Immunodeficiency Virus • Infectious Disease • CCR2

Transposon Announces Final Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia (PRNewswire) - P2a | N=42 | NCT04993755 | Sponsor: Transposon Therapeutics, Inc. | "Transposon Therapeutics...announced final results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD)....NfL is the primary biomarker of neurodegeneration, and the NfL results at both Weeks 24 and 48 are consistent with findings from the company's Phase 2 study of TPN-101 for the treatment of PSP. TPN-101 also had lowering effects on additional biomarkers of neurodegeneration and neuroinflammation, including neurofilament heavy chain (NfH), interleukin 6 (IL-6), neopterin, and osteopontin....Transposon intends to present detailed data from this study at upcoming scientific meetings."

Biomarker • P2a data • Amyotrophic Lateral Sclerosis • CNS Disorders

Transposon Receives US FDA Fast Track Designation for TPN-101 for Progressive Supranuclear Palsy (PRNewswire) - "Transposon Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to TPN-101 for progressive supranuclear palsy (PSP)....The designation of TPN-101 as a Fast Track product for PSP is supported by data from a Phase 2, randomized, double-blind, placebo-controlled study of TPN-101 in patients with PSP."

Fast track • CNS Disorders • Progressive Supranuclear Palsy

A PHASE 2A STUDY OF TPN-101, A NUCLEOSIDE REVERSE TRANSCRIPTASE INHIBITOR, IN PATIENTS WITH PROGRESSIVE SUPRANUCLEAR PALSY (ADPD 2024) - "All doses of TPN-101 were well-tolerated in patients with PSP. Numerical trends toward reductions in NfL and IL-6 support the potential for a positive effect on neuroinflammation and neurodegeneration. Clinical improvements were not expected due to the size and duration of the study."

Clinical • P2a data • CNS Disorders • Inflammation • Movement Disorders • Progressive Supranuclear Palsy • IL6

Transposon Announces Interim Results from a Phase 2 Study of TPN-101 for the Treatment of Progressive Supranuclear Palsy to be Presented at the AD/PD™ 2024 International Conference on Alzheimer's and Parkinson's Diseases (PRNewswire) - P2 | N=40 | NCT04993768 | Sponsor: Transposon Therapeutics, Inc. | "Transposon Therapeutics...today announced its abstract of interim results from its Phase 2 study of TPN-101 for the treatment of progressive supranuclear palsy (PSP) has been accepted for poster presentation at the hybrid AD/PD 2024: 18th International Conference on Alzheimer's and Parkinson's Diseases....In the treatment group receiving 400 mg of TPN-101 once daily for 24 weeks, TPN-101 showed an 18.4% reduction in NfL levels in CSF as compared to placebo. NfL is a biomarker of neurodegeneration that correlates with disease severity and progression in PSP. In the same 400 mg treatment group, TPN-101 also resulted in a 51.6% reduction in IL-6 levels in CSF as compared to placebo."

P2 data • CNS Disorders • Progressive Supranuclear Palsy

A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP) (clinicaltrials.gov) - P2a | N=40 | Active, not recruiting | Sponsor: Transposon Therapeutics, Inc. | Trial completion date: Jul 2023 ➔ Dec 2023 | Trial primary completion date: Jul 2023 ➔ Dec 2023

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

TPN-101 in Aicardi-Goutières Syndrome (AGS) (clinicaltrials.gov) - P2a | N=16 | Recruiting | Sponsor: Transposon Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Aicardi Goutieres Syndrome • Genetic Disorders

TPN-101 in Aicardi-Goutières Syndrome (AGS) (clinicaltrials.gov) - P2a | N=16 | Not yet recruiting | Sponsor: Transposon Therapeutics, Inc. | Initiation date: Dec 2022 ➔ Mar 2023

Trial initiation date • Aicardi Goutieres Syndrome • Genetic Disorders

Oncolys BioPharma Inc. Announces Completion of Enrollment in Phase IIa Clinical Trial of OBP-601 (censavudine, TPN-101) in Patients with Amyotrophic Lateral Sclerosis [Google translation] (Market Screener) - "Oncolys BioPharma Inc. announced that it has received a message from Transposon Therapeutics Inc. ('Transposon'), licensee of OBP-601 (censavudine, TPN-101), that has completed enrollment in the Phase IIa, double-blind, placebo-controlled trial of OBP-601 in patients with amyotrophic lateral sclerosis (ALS) and/ or Frontotemporal Degeneration (FTD) who have the C9ORF72 repeat expansion mutation. In addition, Transposon has completed enrollment in a Phase IIa, double-blind, placebo-controlled study of TPN-101 in patients with Progressive Supranuclear Palsy (PSP) and has opened enrollment in a Phase IIa, Open Label study of TPN-101 in patients with Aicardi-Goutieres Syndrome (AGS)."

Enrollment closed • Enrollment open • Aicardi Goutieres Syndrome • Amyotrophic Lateral Sclerosis • CNS Disorders • Frontotemporal Lobar Degeneration • Progressive Supranuclear Palsy

A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD (clinicaltrials.gov) - P2a | N=42 | Active, not recruiting | Sponsor: Transposon Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

TPN-101 in Aicardi-Goutières Syndrome (AGS) (clinicaltrials.gov) - P2a | N=16 | Not yet recruiting | Sponsor: Transposon Therapeutics, Inc.

New P2a trial • Aicardi Goutieres Syndrome • Genetic Disorders

A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP) (clinicaltrials.gov) - P2a | N=40 | Active, not recruiting | Sponsor: Transposon Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Synthesis of Carbon-14 and Stable Isotope Labeled Censavudine. (PubMed, J Labelled Comp Radiopharm) - "Deuterium labeled Censavudine was synthesized in two steps in a 68% overall yield from [D ]-thymine. A total of 237 mg were prepared with a UV purity of 99%."

Journal • Human Immunodeficiency Virus • Immunology • Infectious Disease

4'-Modified Nucleosides for Antiviral Drug Discovery: Achievements and Perspectives. (PubMed, Acc Chem Res) - P=N/A, P3 | "Currently, seven 4'-modified nucleoside drug candidates such as azvudine (also known as FNC), islatravir, censavudine, balapiravir, lumicitabine, AL-335, and 4-azidothymidine have progressed into clinical stages for treating viral infections. FNC cured the COVID-19 disease in almost all patients and showed better therapeutic efficacy than remdesivir. In this Account, we provide an overview of 4'-modified nucleoside analogs in clinical stages for antiviral therapies, highlighting the drug discovery strategies, structure-activity relationship studies, and preclinical/clinical studies and also give our perspectives on nucleoside-based antiviral drug discovery."

Journal • Human Immunodeficiency Virus • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • CD4

Oncolys BioPharma : Announcement of Enrollment of First Patient in Phase IIa study for OBP-601 (Censavudine, TPN-101) in patients with ALS and/or FTD (Market Screener) - “Oncolys BioPharma Inc…announced that it has received notice from Transposon Therapeutics, Inc. (‘Transposon’), licensee of OBP-601 (Censavudine, TPN-101), that the first patient has been enrolled in the Phase IIa, double-blind, placebo-controlled study of patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Degeneration (FTD). The study will be conducted in multiple centers in the United States and Europe. In addition, Transposon continues to enroll patients with Progressive Supranuclear Palsy (PSP) in a Phase IIa, double-blind,placebo-controlled study conducted in the US.”

Enrollment open • Enrollment status • Amyotrophic Lateral Sclerosis • CNS Disorders • Frontotemporal Lobar Degeneration • Progressive Supranuclear Palsy

A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD (clinicaltrials.gov) - P2a; N=40; Recruiting; Sponsor: Transposon Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Immunology

Oncolys --- Significantly repulsive, milestone achieved with nucleic acid reverse transcriptase inhibitor OBP-601 [Google translation] (Reuters) - "It announced that it has achieved the first milestone based on the exclusive license agreement for...OBP-601 signed with Transposon in June. Details of milestone income are not disclosed. The lump-sum contract payment is expected to be received from December 2008 to February 2009, and will be disclosed promptly as soon as it is received. The total license agreement is said to be over $ 300 million (about 33 billion yen)."

Commercial • Human Immunodeficiency Virus