This week on Pipeline Moves, we start off on a good note by looking at positive topline results from a Phase II trial investigating an epilepsy drug. We continue by reviewing trial terminations in several oncology indications, cholestasis, glomerulonephritis, and malaria.
Interested in seeing these updates in your inbox? Sign-up for our Pipeline Moves newsletter. You can read the last edition here.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataEpilepsy drug sees positive Phase II topline data
Équilibre Biopharmaceuticals’s EQU-001 saw its Phase Transition Success Rate (PTSR) in epilepsy jump 16 points to 53% after the company reported Phase II topline results. New York City-based Équilibre reported positive topline results on 30 January, and the PTSR change took effect on 1 February. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The placebo-controlled Phase II trial (NCT05063877) tested adjunctive EQU-001 for 43 patients with epilepsy taking one to four antiepileptic drugs. The primary endpoint was the incidence of Grade 2 or higher adverse events (AEs).
No treatment-related serious AEs were reported, as per the company press release. The study also demonstrated dose-dependent decreases along the secondary endpoint of 28-day focal seizure frequency, though the company said the trial was not powered for efficacy.
Although Équilibre has not publicly disclosed EQU-001’s exact mechanism, the company has said the oral capsule is designed to have anti-inflammatory effects in epilepsy. Additional exploratory biomarker data from the completed Phase II study, including reduced interleukin-17 (IL-17) and interleukin 1 beta (IL-1b) levels, support the drug’s anti-inflammatory properties, as per the press release.
Équilibre is currently recruiting a 300-patient Phase II trial of EQU-001 in uncontrolled focal onset seizures (NCT05473442). The trial has a primary endpoint of percentage change in the number of monthly seizures and an estimated primary completion date of January 2025, as per the trial listing.
Termination of Phase II in cholestasis of pregnancy
Mirum Pharmaceuticals’s volixibat saw a drop in its PTSR in cholestasis after a Phase II trial was terminated after the company’s assessment of enrolment feasibility. The PTSR dropped by 25 points to 24%. GlobalData evaluated the asset on 9 February after a ClinicalTrials.gov update the previous day.
The purpose of the double-blind, placebo-controlled trial (NCT04718961) was to evaluate the efficacy and safety of volixibat in adult women with intrahepatic cholestasis of pregnancy. Only 4 subjects had been enrolled.
Volixibat is a selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT). The therapy is under development for the treatment of primary sclerosing cholangitis and its associated pruritis and primary biliary cholangitis.
Phase II of oncology tablet terminated
CSPC Pharmaceutical Group’s SYHA-12128 saw a drop in its PTSR in four oncology indications after a Phase II trial was terminated because the primary endpoint did not meet the expectation.
The PTSR declined by 13 points to 12% and by 13 points to 14% in bile duct cancer and gallbladder cancer, respectively. The PTSR also dropped by seven points to 5% and by 15 points to 17% in extrahepatic bile duct cancer and metastatic biliary tract cancer, respectively. GlobalData evaluated the asset on 7 February after a ClinicalTrials.gov update the day before.
The open-label, single-arm trial (NCT04784520) enrolled 31 patients with advanced biliary tract cancer (BTC). The study aimed to evaluate the efficacy, safety, and pharmacokinetics characteristics of oral SYHA-12128. The primary endpoint measured progression-free survival after 3.5 years.
SYHA-12128 is a multi-tyrosine kinase inhibitor that inhibits EGFR, FGFR, VEGFR and RET, and elicits antitumor activity.
BMS terminates Phase II in lung cancer
Bristol Myers Squibb’s BMS-986207 saw its PTSR drop after a Phase II trial was terminated. The drug’s PTSR dropped by 18 points to 11% in non-small cell lung cancer (NSCLC) and 19 points to 14% in squamous NSCLC.
The trial’s status was updated from ongoing, recruiting to terminated on ClinicalTrials.gov on 1 February, and GlobalData evaluated the asset the following day. The trial was terminated because of safety reasons.
The purpose of the Phase II trial (NCT05005273) was to assess the safety and efficacy of BMS-986207 in combination with BMS’s Opdivo (nivolumab) and Yervoy (ipilimumab) as first-line treatment of stage IV NSCLC.
BMS-986207 is a monoclonal antibody under development for several cancers. The drug candidate exhibits therapeutic intervention by binding to the T-cell immunoglobin and ITIM domain protein.
Termination of Phase II in kidney disorder
Novartis’s iptacopan saw its PTSR drop after a Phase II trial was terminated. The PTSR decreased by 13 points to 15% in membranous glomerulonephritis.
The trial’s status was updated to terminated on ClinicalTrials.gov on 6 February, and GlobalData evaluated the asset the following day. The trial was terminated because of the sponsor’s decision.
The purpose of the randomised, open-label, two-arm trial (NCT04154787) was to evaluate the safety and efficacy of iptacopan versus rituximab in subjects with membranous nephropathy who are at high risk of disease progression defined on the basis of anti-PLA2R antibody titters and proteinuria. The trial only enrolled 37 patients while 72 patients were originally anticipated to participate. Iptacopan acts as an inhibitor of complement factor b (CFB).
Institution-led Phase I/II prostate cancer trial terminated
Leap Therapeutics’s sirexatamab saw its PTSR decrease in metastatic castration-resistant prostate cancer (mCRPC) after an institution-led Phase I/II trial was terminated. The PTSR dropped by 13 points to 10% in mCRPC. The trial was sponsored by NYU Langone Health.
The trial’s status was updated from ongoing, recruiting to terminated on ClinicalTrials.gov on 1 February, and GlobalData evaluated the asset the following day. The trial was terminated because of slow accrual in the context of changing practice patterns.
The Phase I/II trial (NCT03837353) was a non-randomised, dose escalation and expansion study testing sirexatamab alone or in combination with docetaxel in mCRPC. Sirexatamab is a monoclonal antibody under development for several cancers. The drug candidate acts by inhibiting dickkopf-1 (Dkk-1).
Phase I malaria trial terminated
Novartis’s INE-963 saw its PTSR drop after a Phase I trial was terminated. The drug’s PTSR dropped by 23 points to 28% in malaria.
The trial’s status was updated to terminated on ClinicalTrials.gov on 8 February, and GlobalData evaluated the asset the following day. The trial was terminated because of the sponsor’s decision.
The purpose of the randomised, placebo-controlled, single ascending dose and multiple dose trial (NCT04896632) was to assess the safety, tolerability, and pharmacokinetics of INE-963. The trial enrolled 64 healthy participants. INE-963 is a fast-acting, long-lasting anti-malarial drug.
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.
