This week on Pipeline Moves, we kick off by looking at a Phase III trial by Roche in its oesophageal squamous cell carcinoma candidate tiragolumab, which met its endpoints.

Meanwhile, AbbVie has terminated a Phase I/II trial of Venclexta in acute myeloid leukaemia. On a positive note, a Phase I/II atopic dermatitis trial, a Phase II neurology trial and a Phase I/II oncology trial have all completed.

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Approval prospects for Roche’s ESCC candidate rise

Roche’s tiragolumab saw its Likelihood of Approval (LoA) rise in oesophageal squamous cell carcinoma (ESCC) after a Phase III trial reached its endpoints.

The LoA for tiragolumab rose by nine points from 46% to 55% in ESCC. LoA is calculated by GlobalData’s analysis using a combination of machine learning and a proprietary algorithm. LoA can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

According to an abstract released at the 2024 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, the study met both its primary endpoints of independent review facility (IRF)-assessed progression-free survival (PFS) and overall survival (OS). GlobalData evaluated the asset on January 22.

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By GlobalData

The Phase III trial (NCT04540211) evaluated tiragolumab plus Tecentriq (atezolizumab) in combination with paclitaxel and cisplatin for the treatment of participants with unresectable locally advanced, unresectable recurrent, or metastatic ESCC. The study enrolled 461 patients.

Tiragolumab binds to the T cell immunoglobulin and ITIM domain protein. The monoclonal antibody (mAb) candidate is under development for the treatment of various cancers. According to GlobalData, tiragolumab is projected to reach peak global sales of 875 million in 2029.

Abbvie terminates Phase I/II AML trial

Abbvie’s Venclexta (venetoclax) saw its Phase Transition Success Rate (PTSR) decline following a Phase I/II trial termination in relapsed or refractory acute myeloid leukaemia (AML).

The drug’s PTSR decreased by five points in myeloproliferative disorders, settling at 24%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

The study was sponsored by the University Health Network in Toronto.

The trial’s status was updated on its ClinicalTrials.gov from ongoing, not recruiting to terminated on 24 January and GlobalData evaluated the asset on the next day.

The study was terminated due to insufficient funding according to the trial’s ClinicalTrials.gov listing.

The Phase I/II trial (NCT04092179) included a Phase Ib and a Phase II portion. In the Phase Ib portion, the study evaluated the efficacy and safety of increasing Venclexta doses combined with a flat dose of Bristol Myers Squibb’s Idhifa (enadisenib).

In the Phase II portion, the study evaluated the highest dose of Venclexta found in the Phase Ib part, in combination with a flat dose of Idhifa in the treatment of relapsed or refractory AML. The study enrolled 27 subjects with DH2 (R140 or IDH R172) mutated AML disease status as determined by laboratory tests.

The asset acts as an antineoplastic agent that is designed to block the function of the protein Bcl-2, which allows cancer cells to become long-lived. It is a small molecule that regulates apoptosis and impacts tumour growth. Venclexta is indicated for the treatment of patients with chronic lymphocytic leukaemia (CLL), or small lymphocytic lymphoma (SLL).

Phase I/II atopic dermatitis trial completion

Amytrx Therapeutics’ AMTX-100 CF3 saw its PTSR increase after the completion of a Phase I/II study (NCT04313400). The drug’s PTSR rose by nine points to 31% in atopic dermatitis.

The study’s status was updated on its ClinicalTrials.gov from recruiting to completed on 18 January, with GlobalData appraising the asset on 19 January.

The two-part trial investigated the efficacy and safety of topically applied AMTX-100 CF3 in adult patients with mild to moderate atopic dermatitis.

The first Phase I part of the trial was designed to enrol approximately 25 subjects and study the maximum tolerable dose.

The second part of the study was meant to feature roughly 60 participants who were either treated with AMTX-100 CF3 or a placebo. The second part studied the proportion of responder subjects at day 28 as the primary endpoint.

AMTX-100 CF3 is a novel peptide that is developed for inflammatory conditions, based on a July 25, 2023 press release.

Phase II trial of Dravet syndrome and tuberous sclerosis completes

Longboard Pharmaceuticals’s bexicaserin saw its PTSR increase in Dravet Syndrome and tuberous sclerosis and after a Phase II trial was completed. The drug’s PTSR increased by nine points in both indications, reaching 62%.

The Phase II trial’s (NCT05364021) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 19 January and GlobalData evaluated the asset on the following day.

The purpose of the randomised, double-blind, placebo-controlled, parallel-group, dose-escalation study was to investigate safety, tolerability, pharmacodynamics, and pharmacokinetics of bexicaserin in subjects with developmental and epileptic encephalopathies. The study enrolled 52 patients.

Bexicaserin acts as 5-Hydroxytryptamine Receptor 2C (5-HT2C) agonist. The drug candidate is under development for the treatment of developmental and epileptic encephalopathies (DEEs) refractory focal epilepsies, Lennox-Gastaut Syndrome, Dravet syndrome, tuberous sclerosis complex and CDKL5 deficiency disorder.

Phase II lichen sclerosus trial success

Incyte’s Jakafi (ruxolitinib phosphate) saw its PTSR increase in vulvar lichen sclerosus after a Phase II trial was completed. The drug’s PTSR increased by nine points, reaching 21%.

The Phase II trial’s (NCT05593445) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 24 January and GlobalData evaluated the asset on the following day.

The purpose of the randomised, double-blind, vehicle-controlled study was to investigate safety and efficacy of Jakafi in subjects with vulvar lichen sclerosus. The study enrolled 61 patients.

Jakafi is an orally bioavailable Janus-associated kinase (JAK) inhibitor with potential antineoplastic and immunomodulating activities.

Jakafi is indicated for patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythaemia vera myelofibrosis, post-essential thrombocythemia myelofibrosis. It is also indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant to hydroxyurea. The small molecule is under development for the treatment of various cancers and other indications.

Phase transition prospects for oncology candidate rise

Mural Oncology’s nemvaleukin alfa saw its PTSR increase in colorectal cancer and transitional cell carcinoma (urothelial cell carcinoma) after a Phase I/II trial was completed.

The drug’s PTSR increased by ten points in colorectal cancer, reaching 32%, and eight points in transitional cell carcinoma (urothelial cell carcinoma), reaching 30%.

The Phase I/II trial’s (NCT02799095) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 24 January and GlobalData evaluated the asset on the following day.

The purpose of the study was to investigate safety and tolerability of nemvaleukin alfa in humans. The study enrolled 243 patients.

Nemvaleukin alfa is a fusion protein of interleukin-2 (IL-2) and interleukin-2 (IL-2) receptor alpha. The Waltham, Massachusetts-based company is developing the drug candidate for the treatment of various solid tumours.

Read the last edition:

Pipeline Moves: Approval chances for thalassaemia candidate rise after Phase III trial meets endpoints

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.