This week on Pipeline Moves, we kick off by looking at the completion of a Phase III trial by Corcept Therapeutics of its candidate relacorilant in Cushing’s syndrome.

Meanwhile, Regeneron has completed a Phase II trial of its candidate mibavademab in lipodystrophy and Bayer has completed a Phase II trial of Stivarga in metastatic hepatocellular carcinoma (HCC).

Also, Lyvgen’s monoclonal antibody (mAb) dalnicastobart has seen its transition prospects increase after a Phase I trial completed.

Phase III Cushing’s syndrome trial completes

Corcept Therapeutics’s relacorilant saw its Likelihood of Approval (LoA) rise after a Phase III trial was completed.

The LoA increased by seven points to 73% in Cushing’s syndrome. LoA is determined using a combination of machine learning and GlobalData’s proprietary algorithm. It can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

The Phase III trial’s (NCT03697109) status was updated from ongoing to completed on ClinicalTrials.gov on 2 May with GlobalData evaluating the product on the following day.

The randomised, double-blind, placebo-controlled withdrawal study evaluated the efficacy, safety, and pharmacokinetics of relacorilant in patients with Cushing’s syndrome and concurrent type 2 diabetes. The trial enrolled 152 patients.

Relacorilant exhibits therapeutic intervention by acting as a GR-II antagonist and is under development for the treatment of Cushing’s syndrome and various cancer indications.

Phase II trial of Regeneron‘s mibavademab completes

Regeneron Pharmaceuticals’s mibavademab saw its Phase Transition Success Rate (PTSR) in lipodystrophy increase nine points to 66% after a Phase II study for metabolic abnormalities in patients with familial partial lipodystrophy (FPLD) was completed.

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 (NCT05088460) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 2 May, with GlobalData evaluating the product on 6 May.

The randomised, double-blind, placebo-controlled study evaluated the effects of the treatment in patients with FPLD who were divided into two cohorts based on baseline leptin levels. Cohort A consisted of patients with leptin levels <8.0 ng/mL, while Cohort B enrolled patients with levels of 8.0 to ≤20.0 ng/mL. The coprimary endpoints of percent change in fasting serum triglyceride (TG) and absolute change in haemoglobin A1c (HbA1c) were measured only in patients from Cohort A.

Mibavademab is an investigational human leptin receptor agonist monoclonal antibody designed to target the leptin receptor (LEPR) and modulate feelings of satiety and consequently contribute to the regulation of body weight.

Regeneron completes Phase I advanced malignancy trial

Regeneron Pharmaceuticals has reported another win with its candidate fianlimab which saw its PTSR in advanced malignancies increase by six points to 34% after a Phase I study in the indication was completed.

The Phase I trial’s (NCT03005782) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 29 April, with GlobalData evaluating the product on 2 May.

The open label, dose escalation and cohort expansion study evaluated the safety, tolerability, activity, and pharmacokinetics of the treatment in patients with advanced malignancies. The purpose of the dose escalation phase was to identify safe dose level(s) to take into the dose expansion phase, in which preliminary anti-tumor activity of fianlimab would be assessed.

The drug would be evaluated both as a monotherapy and in combination with Regeneron’s anti-PD-1 monoclonal antibody Libtayo (cemiplimab) by using objective response rate (ORR).

Fianlimab is an intravenously administered monoclonal antibody targeting the immune checkpoint receptor LAG-3 on T cells. The therapy was created using Regeneron’s proprietary Velocimmune platform.

Stivarga a step closer to HCC approval after Phase II completion

Bayer’s Stivarga (regorafenib) saw its PTSR increase after a Phase II trial was completed. The drug candidate’s PTSR increased by ten points in metastatic hepatocellular carcinoma (HCC), reaching 33%.

The Phase II trial’s (NCT04696055) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 1 May, and GlobalData evaluated the asset on the following day.

The purpose of the open-label study was to evaluate Stivarga plus Merck’s Keytruda (pembrolizumab) in patients with HCC who have been previously treated with PD-1/PD-L1 checkpoint inhibitors. The study enrolled 95 patients.

Stivarga is a tumour deactivation agent that potently blocks multiple protein kinases. The drug is approved for the treatment of metastatic colorectal cancer patients who have been previously treated with other therapies and is being evaluated as a treatment for various cancer indications.

Phase II trial of marginal zone B-cell lymphoma completes

InnoCare Pharma’s Inokai (orelabrutinib) saw its PTSR increase after a Phase II trial was completed. The drug candidate’s PTSR increased by five points in marginal zone B-cell lymphoma, reaching 36%.

The Phase II trial’s (NCT03797456) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 19 April, and GlobalData evaluated the asset on 22 April.

The purpose of the multi-centre, open-label study was to evaluate the safety and efficacy of Inokai in patients with relapsed/refractory marginal zone lymphoma. The study enrolled 111 patients.

Inokai is a BTK inhibitor. The drug is marketed and in development for the treatment of various cancer indications.

Phase I trial of Lyvgen’s mAb in advanced malignancies completes

Lyvgen Biopharma’s dalnicastobart saw its PTSR increase after a Phase I trial was completed. The monoclonal antibody’s (mAb) PTSR increased by 11 points in advanced malignancies, reaching 32%.

The Phase I trial’s (NCT05152212) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 19 April, and GlobalData evaluated the asset on 22 April.

The study used a bridging dose escalation design to establish the maximum tolerated dose of dalnicastobart. The study enrolled 33 patients.

Dalnicastobart acts as a CD40 agonist. The Shanghai, China-based company is developing the mAb for the treatment of various solid tumours.

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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.