This week on Pipeline Moves, we kick off by looking at success of a Phase III trial of Ironwood Pharmaceuticals’ apraglutide in short bowel syndrome which met its primary endpoints.
Meanwhile, Merck terminated a Phase II metastatic melanoma trial of gebasaxturev while Tianjin Hemay Pharmaceutical’s terminated a Phase I solid tumour trial.
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On a positive note, Kallyope’s small molecule obesity trial completed while Jiangsu Hengrui Medicine’s Phase II trial of esketamine hydrochloride in general anesthetic effect also completed.
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Ironwood Pharmaceuticals’s apraglutide saw its Likelihood of Approval (LoA) rise in short bowel syndrome (SBS) after a Phase III trial in the same indication reached its endpoints.
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The LoA for apraglutide rose by 11 percentage points from 39% to 50% in SBS. 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.
On 29 February, Ironwood Pharmaceuticals announced that the trial met its primary endpoint of relative change from baseline in actual weekly parenteral support (PS) volume at week 24. GlobalData evaluated the asset on 1 March. Ironwood also reported that the candidate was generally well tolerated with the safety profile remaining consistent with previous studies.
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By GlobalDataThe Phase III STARS trial (NCT04627025) investigated the efficacy and safety of weekly subcutaneous injections of apraglutide in adult SBS patients with intestinal failure. The trial enrolled 164 patients, both stoma and colon-in-continuity, who were evaluated over a maximum of 48 weeks. Patients were randomised on a 2:1 ratio to once weekly apraglutide or placebo.
The study was initiated in 2021 by VectivBio AG, which was acquired by Ironwood Pharmaceuticals in a $1bn deal in May 2023.
Apraglutide is an investigational long-acting synthetic GLP-2 analog that is being developed for a range of rare gastrointestinal diseases. It acts by activating the GLP-2 receptor via activation of adenylate cyclase and consequently restores the normal physiological function of the gastrointestinal tract affected by mucositis exhibiting intestine-trophic and intestine-protective activity.
Following the data announcement, Ironwood said it intends to file an NDA and other regulatory filings for apraglutide for use in adult patients with SBS who are dependant on PS.
Merck’s metastatic melanoma trial terminated
Merck’s gebasaxturev saw its Phase Transition Success Rate (PTSR) in metastatic melanoma drop 10 points to 41% after a Phase II trial for advanced metastatic melanoma was terminated. 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 (NCT04152863) status was updated from completed to terminated with business reasons being cited on ClinicalTrials.gov on 1 March and GlobalData evaluating the product on 5 March.
The randomised, parallel assignment study evaluated the safety, tolerability, and efficacy of gebasaxturev in conjunction with Merck’s PD-1 inhibitor Keytruda (pembrolizumab) as a treatment for advanced metastatic melanoma. The therapy was administered intratumorally and intravenously in 85 adult patients. The study employed objective response rate (ORR) as its primary endpoint and assessed the outcome through Blinded Independent Central Review (BICR) per Response Evaluation Criteria in Solid Tumours Version 1.1 (RECIST 1.1).
Gebasaxturev is an oncolytic virus anticancer agent designed to promote the lysis of tumour cells expressing the ICAM-1 protein and induce an immune response to the tumour following cell lysis.
Phase II small molecule obesity trial completes
Kallyope’s K-757 saw its PTSR increase after a Phase II trial with the drug was completed. The drug candidate’s PTSR increased by ten points in obesity, reaching 27%.
The Phase II trial’s (NCT06019559) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 7 March and GlobalData evaluated the asset on the following day.
The purpose of the study was to evaluate the safety and efficacy of K-757 alone and in combination with K-833 in obese patients. The study enrolled 150 patients.
K-757 exhibits therapeutic intervention by targeting the circuits involved in the gut-brain axis. The New York, New York-based company is developing the drug candidate for the treatment of metabolic disorders.
Phase II solid tumour trial completion
Blueprint Medicines’s Gavreto (pralsetinib) saw its PTSR increase after a Phase II trial with the therapy was completed. The drug candidate’s PTSR increased by nine points in solid tumours, reaching 25%.
The Phase II trial’s (NCT04632992) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 7 March and GlobalData evaluated the asset on the following day. Genentech, a subsidiary of Roche, sponsored the trial.
The purpose of the multi-centre, non-randomised, open-label, multi-arm study was to evaluate the safety and efficacy of targeted therapies, including Blueprint Medicines’ Gavreto, as single agents and in rational combinations in participants with advanced solid tumours. The study enrolled 252 patients.
Gavreto is marketed for the treatment of rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), thyroid cancer, and RET-mutant medullary thyroid cancer (MTC). The small molecule is under development for the treatment of solid tumours including MTC and NSCLC.
Phase II general anaesthetic trial completes
Jiangsu Hengrui Medicine’s esketamine hydrochloride saw its PTSR as a drug for inducing a general anaesthetic effect increase after a Phase II trial in that indication was completed. The PTSR jumped by 54 points to 63%.
The Phase II trial’s (NCT04867343) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 1 March with GlobalData evaluating the asset on 5 March.
The study was divided into two stages to evaluate intravenous esketamine hydrochloride in 70 patients aged between two and 18-years-old.
The purpose of the first stage of the trial was to verify the dosage in patients undergoing general anaesthesia using an open, single arm trial design. The second stage was a randomised, parallel assignment trial measuring the success rate of anaesthesia induction of esketamine hydrochloride.
Esketamine hydrochloride, a small molecule glutamate ionotropic NMDA receptor antagonist, is designed for use in combination with sedative anaesthetics in paediatric patients who are going under general anaesthesia.
Phase I solid tumour trial termination
Tianjin Hemay Pharmaceutical’s Hemay-020 saw its PTSR decrease in solid tumours after a Phase I trial was terminated. The PTSR dropped by 21 points to 17%.
The Phase I trial’s (NCT02467569) status was updated from completed to terminated on ClinicalTrials.gov on 1 March, and GlobalData evaluated the asset on 4 March. The reason for termination was not disclosed on the study’s ClinicalTrials.gov listing.
The purpose of the study was to evaluate the safety, tolerability, and pharmacokinetics of Hemay-020 in patients with solid tumours. The study enrolled ten patients before being terminated.
Hemay-020 elicits anti-neoplastic activity by irreversibly inhibiting epidermal growth factor receptor (EGFR). The drug candidate was under development for the treatment of solid tumours and advanced or metastatic non-small cell lung cancer.
Read the last edition:
Pipeline moves: Prospects drop after investigator-led trial of Roche’s Tecentriq terminatedNeed 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.
