This week on Pipeline Moves, we start off by reviewing the termination of a Phase IIa trial in Alzheimer’s disease. We continue by looking at trial completions in homocystinuria, kidney disease, traumatic brain injury, breast cancer and sarcomas.
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Cyclerion Therapeutics’ zagociguat (CY6463) saw its Phase Transition Success Rate (PTSR) drop in Alzheimer’s disease by 23 points to 14% after the termination of a Phase IIa trial. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
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The Phase IIa study’s (NCT04798989) status on ClinicalTrials.gov was changed from recruiting to terminated on 2 June, with the asset being evaluated by GlobalData on 6 June. The trial was terminated due to enrolment challenges, according to the trial registry.
GlobalData is the parent company of Clinical Trials Arena.
The purpose of the randomised, placebo-controlled, double-blind, Phase IIa study was to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of zagociguat in participants 65 years or older with Alzheimer’s disease and vascular pathology.
A total of 12 participants were enrolled in the study before the study’s termination. Subjects were randomised to receive approximately three months of once-daily zagociguat or placebo.
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By GlobalDataZagociguat is an orally administered soluble guanylate cyclase (sGC) stimulator. The drug upregulates cGMP, which in turn modulates vascular tone and blood pressure through nitrous oxide and suppresses pathologic conditions.
Phase II metabolic trial completed
Travere Therapeutics’ pegtibatinase saw PTSR rise by 16 points to 50% after a Phase II homocystinuria trial was completed.
The San Diego, California-based company announced the Phase II trial (NCT03406611) completion on 31 May, and GlobalData evaluated the asset on the following day.
The primary objectives of the double-blind, randomised, placebo-controlled trial were the incidence of adverse events and the presence and levels of anti-pegtibatinase and anti-PEG antibodies (neutralising antibodies) in the blood plasma.
Results showed that there were no treatment-related serious adverse events and no evidence of neutralising antibody activity. Data also demonstrated that treatment with pegtibatinase led to a 67.1% mean relative reduction in homocysteine from baseline.
Homocystinuria is an inherited condition that prevents the body from metabolising amino acids, causing a build-up of homocysteine and its precursor methionine in the blood. This can lead to intellectual disability, slower growth, and anaemia in more severe cases.
Pegtibatinase is an enzyme replacement therapy that acts as a recombinant cystathionine beta-synthase (CBS) enzyme, correcting the individual’s ability to process amino acids. Travere is currently discussing Phase III plans with regulators and aims to initiate a Phase III study before the end of 2023.
Novartis completes Phase II kidney disease trial
Novartis’s Adakveo (crizanlizumab) saw its PTSR rise in chronic kidney disease (CKD) after a Phase II trial was completed. The drug’s PTSR grew by eight points to 45% in CKD.
The Phase II trial’s (NCT04053764) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 23 May, and GlobalData evaluated the asset on the following day.
The purpose of the randomized, open-label, two-arm study was to evaluate the effect of Adakveo plus standard of care or standard of care alone on renal function in sickle cell disease patients with CKD. The trial enrolled 58 patients out of the 170 originally anticipated to participate.
Adakveo is a humanized IgG2 kappa monoclonal antibody (mAb) marketed for the treatment of sickle cell disease. The mAb is under development for the treatment of CKD due to sickle cell nephropathy and primary myelofibrosis including post-essential thrombocythemia (PET-MF), post-polycythemia vera (PV) myelofibrosis (PPV-MF) and Covid-19 vasculopathy.
Completion of investigator-led CNS trial
Oxeia Biopharmaceuticals’ OXE-103 saw its PTSR rise in traumatic brain injury after an investigator-led Phase II trial was completed. The drug’s PTSR grew by nine points to 32% in traumatic brain injury.
The Phase II trial (NCT04558346) was sponsored by Dr. Michael Rippee, associate professor in the Department of Neurology at the University of Kansas Medical Center. The trial’s status was updated from active, not recruiting to completed on ClinicalTrials.gov on 31 May, and GlobalData evaluated the asset on the following day.
The purpose of this study was to examine if OXE-103 taken every day for two weeks would help the brain heal faster and help improve or resolve symptoms after a concussion. The trial enrolled 20 out of the 40 patients originally anticipated to participate.
OXE-103 acts as a growth hormone secretagogue receptor GHS-R1a (GHSR) agonist. The synthetic drug candidate is under development for the treatment of traumatic brain injury and long Covid.
Phase I/II breast cancer trial completed
BeiGene’s pamiparib saw its PTSR rise in triple-negative breast cancer (TNBC) after a Phase I/II trial was completed. The drug’s PTSR grew by 15 points to 50% in TNBC.
The Phase I/II trial’s (NCT03150810) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 25 May, and GlobalData evaluated the asset on the following day.
The primary objective of this study was to determine the safety and tolerability of pamiparib, the maximum tolerated dose or maximum administered dose for pamiparib combined with temozolomide, and to determine antitumour activity of pamiparib in combination with temozolomide. The trial enrolled 139 patients.
Pamiparib works by inhibiting the enzymes poly ADP-ribose polymerase (PARP) 1 and 2. The small molecule drug candidate is under development for the treatment of various cancers.
Completion of Phase I/II sarcoma trial
Cornerstone Pharmaceuticals’ devimistat (CPI-613) saw its PTSR rise in sarcomas after the completion of a Phase I/II trial. The PTSR grew by 18 points to 33% in Ewing Sarcoma and 23 points to 43% in soft tissue sarcoma.
The study’s status on its Clinicaltrials.gov page changed from active, not recruiting to completed on 24 May, with GlobalData updating the PTSR on the following day.
The Phase I/II trial (NCT04593758) evaluated the efficacy, safety and maximally tolerated dose of devimistat plus hydroxychloroquine in patients with relapsed or refractory clear cell sarcoma of soft tissue. The trial enrolled 16 patients out of the 29 originally expected to participate.
Devimistat induces cancer-specific inhibition of pyruvate dehydrogenase (PDH) and alpha-ketoglutarate dehydrogenase which are key enzymes involved in cancer cell metabolism. The small molecule drug candidate in under development for the treatment of various cancers.
Read the last edition:
Pipeline Moves: Novo Nordisk Phase III semaglutide completion bumps approval prospectsNeed 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.
