This week on Pipeline Moves, we start off by looking at the termination of a Phase I/II trial in GM1 gangliosidosis. We also look at the suspension of a Phase I trial in dementia. On a good note, we review trial completions in diabetic nephropathy, osteoarthritis, and non-Hodgkin lymphoma.Interested in more news in your inbox? Sign-up for our daily newsletter.
Termination of Phase I/II trial in GM1
Lysogene’s lixmabegene relduparvovec saw its Phase Transition Success Rate (PTSR) fall by 16 points to 10% in GM1 gangliosidosis after a Phase I/II trial was terminated. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The Phase I/II trial’s (NCT04273269) status was updated from active, not recruiting to terminated on ClinicalTrials.gov on 9 June, and GlobalData evaluated the gene therapy on 12 June. The trial listing described the reason for the termination as closed due to Lysogene’s cessation of activities, not due to safety reasons.
The purpose of this study was to assess the safety and efficacy of the gene therapy treatment in subjects with infantile GM1 gangliosidosis. GM1 gangliosidosis, also known as beta-galactosidase-1 deficiency, is an inherited genetic disorder that progressively destroys nerve cells in the brain and spinal cord. The trial enrolled only five patients out of the 18 originally anticipated to participate.
Lixmabegagene relduparvovec acts by delivering the GLB1 gene, which encodes an enzyme called beta-galactosidase necessary for recycling the GM1-ganglioside molecule in neurons.
Phase I dementia trial suspended
Enterin’s ENT-01 saw its PTSR fall by 24 points to 28% in dementia following a Phase I trial suspension. The study’s ClinicalTrials.gov status changed from active, not recruiting to suspended, with a possibility to resume in 2024 on 8 June, with GlobalData updating the PTSR on 13 June. The company did not disclose a reason for the trial suspension.
The Phase I trial (NCT03938922) evaluated the safety and efficacy of repeated oral doses of ENT-01 in improving cognition in patients with Parkinson’s disease dementia. ENT-01 could prevent dementia by inhibiting alpha-synuclein.
In dementia, Alpha-synuclein aggregates into clumps in the brain called Lewy bodies which cause the diseased condition. The Philadelphia, Pennsylvania-based company is also developing ENT-01 for constipation, psychosis, cognitive impairment, and autism spectrum disorder (ASD).
AstraZeneca completes diabetic nephropathy trial
AstraZeneca’s tozorakimab saw its PTSR rise in diabetic nephropathy after a Phase II trial was completed. The drug’s PTSR grew by nine points to 33%.
The Phase II trial’s (NCT04170543) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 18 June, and GlobalData evaluated the asset on the following day.
The purpose of this randomized, double-blind, placebo-controlled, study was to evaluate the safety, pharmacokinetics, immunogenicity, and efficacy of tozorakimab in subjects with diabetic kidney disease. The trial enrolled 609 patients.
Tozorakimab exhibits therapeutic intervention by inhibiting interleukin-33. The monoclonal antibody (mAb) is under development for the treatment of diabetic kidney disease, Coronavirus disease (Covid-19), chronic bronchitis, asthma, acute respiratory failure, and chronic obstructive pulmonary disease (COPD).
Completion of Phase I/II osteoarthritis trial
Gwoxi Stem Cell Applied Technology’s GXCPC-1 saw its PTSR rise in osteoarthritis after a Phase I/II trial was completed. The drug’s PTSR grew by nine points to 32%.
The Phase I/II trial’s (NCT03943576) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 6 June, and GlobalData evaluated the asset on the following day.
The aim of this study was to investigate the safety and efficacy of GXCPC-1 for the treatment of osteoarthritis. The trial enrolled 25 patients.
GXCPC-1 elicits cell regenerative and repair activity. The investigational cell therapy is under development for the treatment of osteoarthritis and consists of allogeneic adipose tissue-derived mesenchymal stem cells.
Phase I oncology trial completed
Nektar Therapeutics’ NKTR-255 saw its PTSR rise in non-Hodgkin lymphoma after a Phase I trial was completed. The drug’s PTSR grew by six points to 40%.
The Phase I trial’s (NCT04136756) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 15 June, and GlobalData evaluated the asset on the following day.
The purpose of the open-label, dose escalation and dose expansion study was to evaluate the safety and tolerability of NKTR-255 alone and in combination with Janssen’s anticancer monoclonal antibody (mAb) Darzalex (daratumumab). The trial enrolled 30 patients.
NKTR-255 acts as interleukin 15 receptor subunit alpha agonist. The recombinant protein is under development for the treatment of various cancers.
Read the last edition:Pipeline Moves: Advancement prospects drop for Alzheimer’s drug after trial termination
Need to know:
GlobalData’s proprietary model uses a combination of machine learning and an algorithm to calculate an individual drug’s PTSR and LoA. While LoA provides the probability of a drug ultimately receiving market authorization, PTSR indicates the probability of a drug’s advancement to the next stage of clinical development. The model uses datapoints from individual drugs, clinical trials, regulatory milestones, company, and financial databases.