This week on Pipeline Moves, we look into a resumption of a Phase II trial in myelofibrosis which was previously suspended by Novartis. We also review the completions of Phase II trials in non-alcoholic fatty liver disease (NAFLD), chronic obstructive pulmonary disease (COPD), and idiopathic pulmonary fibrosis (IPF). We finish off with the completions of Phase I/II paediatric oncology and Phase Ib/IIa colorectal cancer studies.
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Novartis resumes Phase II myelofibrosis trial
Novartis’s NIS-793 saw its Phase Transition Success Rate (PTSR) jump in three indications after a Phase II trial resumed activity after previously being suspended. The PTSR increased by 21 points to 37% in myelofibrosis, by 18 points to 38% in post-polycythaemia vera myelofibrosis (PPV-MF), and by 23 points to 48% in post-essential thrombocythemia myelofibrosis (Post-ET MF).
ClinicalTrials.gov updated the trial listing from suspended to active, not recruiting on 15 November, and the PTSR change took effect the next day. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
Previously, the trial was suspended due to challenges in identifying the patient population as specified by the protocol, and not due to safety concerns, as per the trial listing.
The Phase I/II ADORE trial (NCT04097821) tested Novartis’s Jakafi (ruxolitinib) in a platform trial design with NIS-793, siremadlin, crizanlizumab, sabatolimab, and rineterkib. The primary endpoint of the Phase I portion was dose-limiting toxicities, and the primary endpoint of the Phase II portion was response rate.
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By GlobalDataNIS-793 inhibits transforming growth factor (TGF) beta to alter the tumour microenvironment. Jakafi, a Janus kinase inhibitor (JAK) inhibitor, has FDA approval in multiple indications, including graft versus host disease and myelofibrosis. Myelofibrosis is a bone marrow cancer causing disrupted blood cell production and severe anaemia.
Completion of Merck’s NAFLD Phase II study
Merck’s efinopegdutide saw its PTSR in NAFLD rise nine points to 36% after a Phase II trial completion. ClinicalTrials.gov updated the trial’s status on 2 November and the PTSR changed the next day.
The Phase II trial (NCT04944992) enrolled 145 patients with the primary objective of determining the mean relative reduction in liver fat content from baseline. This was measured by magnetic resonance imaging-estimated proton density fat fraction. The trial was initially supposed to enrol 130 participants.
Efinopegdutide is a long-acting glucagon-like peptide (GLP) acting as a dual agonist of the GLP-1 receptor and glucagon receptor and is subcutaneously administered. Merck is developing the candidate for the treatment of non-alcoholic steatohepatitis, obesity, NAFLD, and type 2 diabetes.
Phase II COPD trial completed
Novartis’ ecleralimab saw its PTSR in COPD rise nine points to 39% after a Phase II trial completion. ClinicalTrials.gov updated the trial’s status on 3 November and the PTSR changed on 5 November.
The Phase II trial (NCT04882124) was a randomised, participant-and investigator-blinded, placebo-controlled, parallel-group study designed to explore the efficacy, pharmacodynamics, safety, and pharmacokinetics of two doses of inhaled ecleralimab in adults with COPD.
The primary outcome was measured by the change in baseline of the Evaluating Respiratory Symptom Scale (E-RS). The trial enrolled 38 subjects. This was a drastic reduction in participants compared to the 300 that were anticipated to participate. Ecleralimab acts as a thymic stromal lymphopoietin (TSLP) inhibitor.
Completion of Phase II study in IPF
Bristol Myers Squibb’s BMS-986263 saw its PTSR jump nine points to 34% in IPF, following the completion of a Phase II trial. ClinicalTrials.gov updated the trial listing from active, not recruiting to completed on 16 November, and the PTSR change took effect the next day.
Bristol Myers Squibb acquired the exclusive worldwide rights to develop and commercialize BMS-986263, also known as ND-L02-s0201, from Nitto Denko, as per a November 2016 press release.
The 123-patient Phase II JUNIPER trial’s (NCT03538301) primary endpoint was the incidence of treatment-related adverse events with secondary endpoints focused on the drug’s pharmacokinetics and biological activity.
BMS-986263 is a siRNA oligonucleotide that inhibits heat shock protein 47 (HSP47), a collagen implicated in causing fibrosis. IPF is a severe lung condition characterized by lung scarring and difficulty breathing.
Eisai completes Phase I/II paediatric oncology trial
Eisai’s Lenvima (lenvatinib mesylate) saw its PTSR improve in four oncology indications after a Phase I/II trial was completed in recurrent and refractory paediatric solid tumours. The PTSR increased by nine points in Ewing sarcoma, primitive neuroectodermal tumour (PNET) and rhabdomyosarcoma reaching 31%, 34% and 22% respectively in each indication. The PTSR also rose by ten points to 28% in high-grade glioma (HGG).
The trial’s status was updated on ClinicalTrials.gov from active, not recruiting to completed on 9 November, with GlobalData evaluating the asset the following day.
The open-label Phase I/II trial (NCT03245151) evaluated the efficacy and safety of Lenvima administered in combination with Novartis’ Afinitor (everolimus). The Phase I part of the study determined a maximum tolerated dose (MTD) and recommended Phase II dose (RP2D). It also evaluated the toxicity of Lenvima combined with Afinitor in patients with recurrent/refractory solid tumours.
The Phase II part assessed the anti-tumour activity of Lenvima and Afinitor in participants with selected recurrent/refractory solid tumours including Ewing sarcoma, peripheral PNET, rhabdomyosarcoma and HGG. Enrolled participants were patients with a histologically or cytologically confirmed diagnosis of recurrent or refractory solid tumours between the ages of two to 21 years of age. The trial was anticipated to enrol 120 participants but eventually recruited only 64 subjects.
The trial’s co-primary endpoints measured the MTD, RP2D, the number of participants with treatment-emergent adverse or serious adverse events up to almost two years after treatment and overall response rate within a timeframe of 16 weeks post-treatment.
Completion of Phase Ib/IIa colorectal cancer trial
YooYoung Pharmaceutical’s YYB-101 saw its PTSR rise by ten points settling at 31% in colorectal cancer, following a Phase Ib/IIa trial completion. The PTSR update took place on 15 November after the study’s Clinicaltrials.gov listing was updated from recruiting to completed on 8 November.
The open-label trial (NCT04368507) evaluated the safety, tolerability, pharmacokinetics and antitumour activity of YYB-101 with irinotecan. Irinotecan is a type of chemotherapy commonly used for the treatment of colorectal cancer.
The study enrolled 35 patients with metastatic or recurrent colorectal cancer. The primary endpoints of the study were the safety and tolerability of YYB-101, assessed by dose-limiting toxicities (DLTs), maximum tolerable dose (MTD) and objective response rate (ORR).
YYB-101 is a monoclonal antibody (mAb) therapy developed by Seoul, South Korea-based YooYoung Pharmaceutical. YYB-101 targets the hepatocyte growth factor (HGF) that acts by inducing cancer cell growth.
