This week on Pipeline Moves, we kick off by looking at the completion of a Phase III trial for cerebrotendinous xanthomatosis. Meanwhile, an investigator-led Phase Ib/II trial in oropharyngeal cancer with AstraZeneca’s Imjudo has been terminated. On a positive note, we investigate completions with a Phase II trial chronic myelocytic leukaemia or chronic myeloid leukaemia, and Phase II results for a trial in gout.

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Approval prospects rise for Mirum Pharmaceuticals after Phase III completion

Mirum Pharmaceuticals’ chenodiol saw its Likelihood of Approval (LoA) increase after the completion of a Phase III trial in cerebrotendinous xanthomatosis (CTX). The LoA grew by seven points to 25%.

LoA is identified via 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.

The trial’s status (NCT04270682) was updated from recruiting to completed on ClinicalTrials.gov on 22 November, and GlobalData evaluated the asset on the following day.

The purpose of the study was to evaluate the effects of chenodiol in patients with CTX. The trial enrolled 18 patients.

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By GlobalData

AbbVie sees transition prospects rise after Phase II completion

AbbVie’s Venclexta (venetoclax) saw its Phase Transition Success Rate (PTSR) increase in chronic myelocytic leukaemia or chronic myeloid leukaemia (CML) after a Phase II trial was completed.

The drug’s PTSR increased by ten points, reaching 38%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

The Phase II trial’s (NCT03455517) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 24 November, and GlobalData evaluated the asset on 27 November. The trial was sponsored by Gruppo Italiano Malattie Ematologiche dell’Adulto.

The purpose of the study was to investigate the activity and safety of Venclexta in combination with rituximab in patients with CML. The study enrolled 77 patients.

Imjudo’s PTSR drops after investigator-led trial is terminated

AstraZeneca’s Imjudo (tremelimumab) saw its PTSR decrease by 11 points in oropharyngeal cancer to 51% after the termination of an investigator-led Phase Ib/II trial.

The Phase Ib/II trial (NCT03618134) was sponsored by UCLA’s Jonsson Comprehensive Cancer Center. The trial’s status was updated from active, not recruiting to terminated [slow accrual] on ClinicalTrials.gov on 18 November while GlobalData appraised the asset on 20 November.

The trial studied the side effects and efficacy of stereotactic body radiation therapy and Imfinzi (durvalumab) with or without Imjudo prior to surgery work in patients with human papillomavirus positive oropharyngeal squamous cell cancer.

Obesity drug transition prospects rise after Phase II completion

Altimmune’s pemvidutide saw its PTSR increase in obesity after a Phase II trial was completed. The drug’s PTSR increased by 15 points, reaching 44%.

The Phase II trial’s (NCT05295875) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 29 November and GlobalData evaluated the asset on 1 December.

The purpose of the randomised, double-blind, placebo-controlled study was to evaluate the efficacy and safety of pemvidutide, a GLP-1 and glucagon receptor agonist, in patients with obesity. The study enrolled 391 patients.

Phase II completion in COPD

EpiEndo Pharmaceuticals’ EP-395 saw its PTSR increase in chronic obstructive pulmonary disease (COPD) after a Phase II trial was completed. The drug’s PTSR increased by ten points, reaching 31%.

The Phase II trial’s (NCT05572333) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 29 November and GlobalData evaluated the asset on 1 December.

The purpose of the multicentre, randomised, double-blind, placebo-controlled study was to investigate the tolerability and safety of EP-385 in patients with COPD. The study enrolled 61 patients.

EP-395 is an anti-inflammatory drug that enhances the epithelial barrier by targeting the epidermal differentiation complex such that basal cells transition from an unattached state to attached one.

Arthrosi’s Phase II results improve phase transition prospects

Arthrosi Therapeutics’ AR-882 saw a 19-point increase in its PTSR, settling at 58% in gouty arthritis (gout) following the release of Phase II results.

The California, US-based company released results from the Phase II trial (NCT05253833) in a 7 November press release and GlobalData evaluated the asset on November 20.

The trial investigated the safety and efficacy of the therapy as a monotherapy and as a combination therapy with allopurinol compared to placebo. The primary endpoint of the open-label, multicentre study was the serum uric acid (sUA) level below 5 mg/dL at month three.

Arthrosi presented a positive Phase II data readout at the American College of Rheumatology (ACR) Convergence 2023 meeting. In the intent-to-treat population, AR882 showed a significant reduction in sUA at three months.

The results showed that in the 75mg AR-882 group, 86% and 64% of patients reached sUA levels under 6 and 5 mg/dL, respectively, vs 77% and 69% in the 50 mg AR882 + allopurinol group and 46% and 23% in the allopurinol group.

AR-882 is an orally administered drug that acts as a URAT1 transporter protein. The drug inhibits the protein, restoring renal reabsorption of uric acid.

MSD subsidiary terminates Phase I trial

Acceleron Pharma’s ACE-1334 saw a drop in its PTSR after a Phase Ib trial was terminated. The PTSR decreased by 31 points to 39% in interstitial lung disease and 35 points to 35% in systemic sclerosis.

Acceleron, a key subsidiary of Merck & Co (MSD), terminated the trial due to business reasons, according to the ClinicalTrials.gov registry. The trial’s status was updated on 24 November, with GlobalData evaluating the asset on 27 November.

PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

The Phase Ib trial (NCT04948554) aimed to evaluate the safety, pharmacokinetics, and pharmacodynamics of ACE-1334 in combination with standard of care. The trial enrolled five out of the planned 48 patients with systemic sclerosis with and without interstitial lung disease.

ACE-1334 is a recombinant protein administered via a subcutaneous route. It acts by blocking and suppressing the activity of transforming growth factor beta 1 and 3.

Read the last edition:

Pipeline Moves: Approval prospects drop for Bayer’s cardiovascular drug after Phase III termination

Need to know:

GlobalData’s proprietary model uses a combination of machine learning and an algorithm to calculate an individual