This week on Pipeline Moves, we kick off by looking at Novo Nordisk’s acquired candidate belcesiran which the company has now discontinued.
Meanwhile, Telios Pharma and Cornerstone Pharmaceuticals have each reported Phase II completions in trials in allergic conjunctivitis and metastatic biliary tract cancer, respectively.
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On a positive note, a Phase IIb trial of Cartesian Therapeutics’s Descartes-08 met the primary endpoint, seeing its progression prospects grow.
Interested in more news in your inbox? Sign-up for our daily newsletter.Novo Nordisk discontinues belcesiran development
Novo Nordisk’s belcesiran saw a 20-point decrease in its Phase Transition Success Rate (PTSR) in alpha-1 antitrypsin deficiency (A1AD) following the termination of a Phase II study. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The Phase II ESTRELLA study’s (NCT04764448) ClinicalTrials.gov status was changed from active, not recruiting to terminated on July 11. The PTSR update took place on July 15. The ClinicalTrials.gov listing stated that the trial was terminated due to the development project being discontinued.
The Phase II trial assessed the incidence and nature of treatment emergent adverse events.
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By GlobalDataThe randomised, double-blind, placebo-controlled study recruited 16 participants rather than its initial estimate of 46.
Belcesiran is an alpha 1 antitrypsin (A1AT) inhibitor that acts by allowing the liver to synthesise the A1AT protein and pass it into the bloodstream.
Novo Nordisk was initially investigating the drug in another trial (NCT05146882) for the treatment of A1AD-related liver diseases, but withdrew the trial, as per a ClinicalTrials.gov status update, in June 2022.
Phase II MG trial meets primary endpoint
Cartesian Therapeutics’s PTSR for Descartes-08 rose after the company announced a Phase IIb trial in myasthenia gravis (MG) achieved its primary endpoint. The PTSR for Descartes-08 went up by 11 percentage points to 63% in MG.
The company published a press release on July 2 announcing positive topline results from the Phase IIb trial. GlobalData evaluated the asset on July 4, 2024.
The study (NCT04146051) enrolled 36 MG patients who were heavily pretreated and highly symptomatic, randomized 1:1 to receive Descartes-08 or placebo. The primary endpoint of reduction in MG Composite (MGC) score was met with statistical significance and the drug remained safe with no cases of cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome.
The data showed that 71% of MG patients treated with Descartes-08 observed had a clinically meaningful improvement in MGC score after three months of treatment compared to 25% in the placebo arm.
Descartes-08 is an autologous mRNA-engineered chimeric antigen receptor-T cell therapy (mRNA CAR-T) product targeting B-cell maturation antigen (BCMA).
The pipeline drug was previously granted Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation by the FDA.
Amgen’s Phase Ib/II ALL trial completes
Amgen’s Kyprolis (carfilzomib) saw its PTSR increase following a Phase Ib/II trial completion in acute lymphocytic leukaemia (ALL). The drug’s PTSR rose by seven points to 49% in ALL.
The trial’s status was updated on its ClinicalTrials.gov from active, not recruiting to completed on July 16 and GlobalData evaluated the asset on the next day.
The Phase Ib portion of the Phase Ib/II trial (NCT02303821) evaluated the safety, tolerability and activity of Kyprolis, alone and in combination with induction chemotherapy, in children with relapsed or refractory ALL.
This portion of the study also aimed to determine the maximum tolerated dose (MTD) and recommend a Phase II dosage of the asset when combined with induction chemotherapy. The trial’s co-primary endpoint measured the incidence of adverse events, serious adverse events, toxicity and the MTD in a period of up to 36 months.
The Phase II’s remaining co-primary endpoints evaluated the rate of complete remission (CR) of Kyprolis administered with a combination of chemotherapies such as vincristine, dexamethasone, PEG-asparaginase, or daunorubicin, compared to an appropriate external control at different time period.
Kyprolis is an antineoplastic agent that is already marketed for multiple myeloma (Kahler Disease), refractory multiple myeloma, and relapsed multiple myeloma. Kyprolis targets the chymotrypsin-like activity of the 20S proteasome, which leads to cell cycle arrest, induction of apoptosis, and inhibition of tumour growth.
Phase II metastatic biliary tract cancer trial completes
Cornerstone Pharmaceuticals’s devimistat saw its PTSR in metastatic biliary tract cancer increase by ten points to 41% after an investigator-led Phase II study was completed.
The Phase II trial’s (NCT04203160) status was updated from active, not recruiting to completed on ClinicalTrials.gov on July 11, with GlobalData evaluating the product on July 12.
The study was sponsored by the University of Michigan Rogel Cancer Center.
The randomised, multi-centre study investigated the effects of gemcitabine and cisplatin with or without devimistat as first-line therapy for patients with advanced unresectable biliary tract cancer.
Devimistat induces cancer-specific inhibition of pyruvate dehydrogenase (PDH) and alpha-ketoglutarate dehydrogenase which are key mitochondrial enzymes involved in cancer cell metabolism. Tumour cells are starved of energy and biosynthetic intermediates, culminating in cell death.
Phase II allergic conjunctivitis trial completes
Telios Pharma’s TL-925 saw its PTSR increase after a Phase II trial was completed. The drug candidate’s PTSR increased by nine points in allergic conjunctivitis reaching 33%.
The Phase II trial’s (NCT06293820) status was updated from active, not recruiting to completed on ClinicalTrials.gov on July 3, and GlobalData evaluated the asset on the following day.
The purpose of the single-centre, randomised, double-masked, trial was to evaluate the safety and efficacy of TL-925 ophthalmic solution compared to a vehicle for the treatment of allergic conjunctivitis in the conjunctival allergen challenge model. The study enrolled 80 patients.
TL-925 acts as a Bruton’s tyrosine kinase (BTK) inhibitor. The Redwood City, California-based company is under development for the treatment of keratoconjunctivitis sicca (dry eye disease) and allergic conjunctivitis.
Recruitment woes see Phase Ib Hepatitis B trial terminated
Yangshengtang’sbiological-162 has seen its Phase Transition Success Rate (PTSR) fall in hepatitis B after a Phase Ib trial was terminated.
The PTSR for the hepatitis B virus surface antigen (HBsAg) inhibitor, named biological-162, dropped in hepatitis B by nine points to 52%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The ClinicalTrials.gov listing shows the trial went from not yet recruiting to terminated on July 14. The listing states the termination was due to enrolment difficulty. GlobalData evaluated the asset on July 17.
The Phase Ib trial (NCT06159062) investigated the candidate in patients with chronic hepatitis B infection. The trial was conducted at one site in Melbourne, Australia. Investigators had estimated an enrolment of 10 patients but the trial was terminated after just one patient had been enrolled.
Hangzhou, China-based Yangshengtang is developing biologic-162, which HBsAg secretion and terminates the replication of HBV RNA to exhibit therapeutic intervention.
Read the last edition:
Pipeline Moves: Alnylam’s Amvuttra approval prospects up after Phase III winNeed 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.
