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April 26, 2022

Pipeline Moves: Nektar’s approval shot in renal cell carcinoma, bladder cancer tumbles

We also look at the likelihood of further study of three different assets in mucopolysaccharidosis type III, binge-eating disorder, and mild cognitive impairment.

By Clinical Trials Arena Team

In this week’s edition of Pipeline Moves, we look at the Likelihood of Approval (LoA) of Nektar Therapeutics’ bempegaldesleukin (bempeg) in renal cell carcinoma and bladder cancer, as well as Amgen’ Prolia (denosumab) in osteogenesis imperfecta, also known as brittle bone disease. LoA is identified via GlobalData’s analysis using a combination of machine learning and its proprietary algorithm.

Further, we look at the Phase Transition Success Rate (PTSR) of three separate assets in rare disease mucopolysaccharidosis type III, binge-eating disorder, and mild cognitive impairment. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

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Nektar’s bempeg cancer trials dropped

Nektar’s bempeg had its LoA drop after the termination of the Phase III PIVOT-09 trial in renal cell carcinoma (RCC) and the Phase II PIVOT-10 trial in bladder cancer. The trial terminations led to a 14-point LoA decrease to 2% in bladder cancer and by 30 points to 9% in metastatic RCC.

Nektar is in partnership with Bristol Myers Squibb in both investigations, which studied bempeg in combination with the latter’s Opdivo (nivolumab). Nektar announced the trial terminations in a company press release on 14 April and GlobalData reported its LoA update on 19 April.

In PIVOT-09 (NCT03729245), bempeg with Opdivo did not reach the pre-specified boundary for statistical significance in comparison to Pfizer’s Stutent (sunitinib) or Exelixis’ Cabometyx (cabozantinib), and that overall survival data was also not statistically significant.

Meanwhile, in PIVOT-10 (NCT03785925), the combo did not meet the efficacy threshold needed to support the continuation of the trial. All other ongoing studies evaluating the combination of bempeg plus Opdivo will also be discontinued as both companies have jointly ended their global clinical development.

Abeona terminates rare disease study

Abeona Therapeutics’ gene therapy ABO-102 (rebisufligene etisparvovec) saw its PTSR in mucopolysaccharidosis type III (MPS III) plummet 17 points to 28% following a Phase I/II trial termination. ClinicalTrials.gov updated the trial from recruiting to terminated on 13 April, and the PTSR change took effect the next day.

The Phase I/II trial (NCT04088734) was anticipated to enrol 12 MPS IIIA patients but was terminated after accruing five due to a lack of efficacy. Still, patients will receive an annual follow-up for five years following their dosing, ClinicalTrials.gov stated.

MPS III, also known as Sanfilippo syndrome, is a rare genetic childhood disorder caused by the lack of an enzyme responsible for breaking down the complex sugar heparan sulphate, leading to dementia. ABO-102 delivers the enzyme sulfoglucosamine sulfohydrolase to the central nervous system, which could help break down heparan sulfate and restore function.

Idorsia completes binge-eating disorder trial

Idorsia Pharmaceuticals’ ACT-539313 saw its PTSR in binge-eating disorders jump nine points to 55% after a Phase II trial was completed. ClinicalTrials.gov updated the trial’s status from active but not recruiting to completed on 13 April, and the PTSR change taking effect the next day.

The 136-patient Phase II trial (NCT04753164) tested ACT-539313 in patients who had an average of at least four binge-eating episodes per week over the previous six months. As a primary endpoint, the study measured the change in binge-eating days per week after 12 weeks. ACT-539313 is an oral orexin-1 receptor antagonist, which could regulate neuropeptides in the hypothalamus.

Actinogen makes strides in mild cognitive impairment

Actinogen Medical’s Xanamem (UE-2343) saw its PTSR rise after a Phase I/II mild cognitive impairment trial was completed. The PTSR increased by nine points to 32%. GlobalData evaluated the asset on 11 April after the ClinicalTrials.gov update on 8 April.

The Phase I/II trial (NCT04983368), ran in collaboration with Avance Clinical, enrolled 107 healthy elderly volunteers. The study assessed the dose regimens of Xanamem which will be used in the upcoming clinical trials in patients.

The asset is being developed as a potential drug to reduce cortisol levels in the brain. The reduction of cortisol levels is believed to be a benefit in treatment of mild cognitive impairment in Alzheimer’s disease.

Amgen completes brittle bone disease study

Amgen’s Prolia saw its LoA grow by five points to 30% in osteogenesis imperfecta following the completion of a Phase III study. ClinicalTrials.gov updated the trial’s status to completed on 18 April, with the LoA changing the next day.

The 75-subject study (NCT03638128) evaluated the long-term safety of Prolia in participants with paediatric osteogenesis imperfecta. The open-label trial measured the subject incidence of treatment-emergent adverse events over a 30-month timeframe in patients 5-to-20 years-old as one of its nine primary outcomes. It also tracked the number of subjects with clinically significant changes from baseline in vital signs over the same timeframe as an additional primary outcome.

Osteogenesis imperfecta, also known as brittle bone disease, is a genetic disorder which results in bones breaking easily. Prolia is a human monoclonal antibody (IgG2), which targets and binds to the apoptosis regulator gene RANKL.

Additional writing by Clinical Trials Arena healthcare researcher Irena Maragkou

Need to Know:

GlobalData’s proprietary model uses a combination of machine learning and an algorithm to calculate an individual drug’s PTSR. PTSR indicates the probability of a drug’s advancement to the next stage of clinical development. The model uses data points from the individual drugs, clinical trials, regulatory milestones, company, and financial databases.

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