This week on Pipeline Moves, we kick off by looking at a Phase II termination of an investigator-led trial of Iovance Biotherapeutics’s cellular immunotherapy MDA-TIL.
Meanwhile, a Phase II trial of Karyopharm Therapeutics’s Xpovio (selinexor) was terminated in metastatic melanoma while Worg Pharmaceuticals Hangzhou’s suspended a Phase II trial of WP-1302 in Grave’s disease.
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On a positive note, Sention Therapeutics completed a Phase II trial of its pipeline candidate ST-1891 in hypothyroidism and a Phase II trial of Armata Pharmaceuticals’s APPA-02 was completed in bronchiectasis.
Interested in more news in your inbox? Sign-up for our daily newsletter.Lack of efficacy leads to termination of investigator-led oncology trial
Iovance Biotherapeutics’s cellular immunotherapy MDA-TIL for oncology saw a 24-point drop in its Phase Transition Success Rate (PTSR), falling to 7% in ovarian cancer following the termination of a Phase II investigator-led trial.
The PTSR dropped by 16 points in colorectal cancer to 7% and 21 points in pancreatic ductal adenocarcinoma to 12%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.
The Phase I/II trial’s (NCT03610490) status was updated from active, not recruiting to terminated on ClinicalTrials.gov on 22 August, and GlobalData evaluated the asset on 27 August. The listing states the termination is due to lack of efficacy.
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By GlobalDataThe Phase II study, led by the M.D. Anderson Cancer Center, investigated the efficacy and safety of Iovance’s autologous expanded tumour infiltrating lymphocytes immunotherapy for cancer. The study investigated the five-year objective response rate (ORR) of patients as its primary endpoint.
Iovance is evaluating its cellular immunotherapy in partnership with the National Cancer Institute and Bristol Myers Squibb. The immunotherapy targets immunodominant minor histocompatibility antigens to mitigate T cell responses and reduce the risk of cancer recurrence.
Phase II hyperthyroidism trial completion
Sention Therapeutics’s ST-1891 saw its PTSR rise following a Phase II trial completion in hypothyroidism. The drug’s PTSR rose by nine points, reaching 22% in this indication.
Sention Therapeutics is a private independent company headquartered in San Diego, California, the US.
The trial’s status was updated on its ClinicalTrials.gov listing from “Ongoing, not recruiting” to “Completed” on 26 August and GlobalData evaluated the asset on 27 August.
The multicentre, randomised, double-blind Phase II study (NCT05412979) evaluated the safety and efficacy of ST-1891 compared to Merck’s Euthyrox (levothyroxine sodium) in 490 participants with primary hypothyroidism.
The study’s primary endpoints evaluated Euthyrox in comparison to ST-1891’s dosing conversion factor within a time period of 52 weeks and the percentage of patients with thyroid-stimulating hormone levels within the standard reference range in a time frame of 26 weeks.
ST-1891 is a hormone replacement therapy administered orally. The asset acts by replacing decreased T3/T4 hormone levels of hypothyroidism patients.
Phase II metastatic melanoma trial termination
Karyopharm Therapeutics’s Xpovio (selinexor) saw its PTSR decrease in metastatic melanoma after a Phase II trial was terminated. The PTSR dropped by 14 points to 11%.
The Phase II trial’s (NCT04768881) status was updated from completed to terminated on ClinicalTrials.gov on 23 August, and GlobalData evaluated the asset on 27 August. According to the study’s ClinicalTrials.gov listing, the sponsor terminated the study “due to a lack of sufficient anti-melanoma tumour signal for the combination of selinexor and pembrolizumab”.
The purpose of the open-label, multi-centre study was to evaluate the safety and efficacy of selinexor in combination with Merck’s Keytruda (pembrolizumab) in recurrent advanced melanoma. The study enrolled 15 patients before being terminated.
Selinexor is a selective inhibitor of nuclear export and is both marketed and in development for the treatment of various cancer indications.
Graves’ disease Phase II trial termination
Worg Pharmaceuticals Hangzhou’s WP-1302 saw its PTSR in Graves’ disease decrease by 16 points to 4% after a Phase II study was suspended.
The Phase II trial’s (NCT06240455) status was updated from recruiting to suspended on the ClinicalTrials.gov on 26 August, with GlobalData evaluating the product on the following day.
The purpose of the double-blind, placebo-controlled study was to assess the efficacy and safety of WP-1302 in preventing disease relapse following methimazole withdrawal in subjects with Graves’ disease. According to ClinicalTrials.gov, the trial was suspended by the sponsor because it was not feasible to recruit suitable subjects.
WP-1302 functions as a tolerogen, which is meant to induce an immunological tolerance. The peptide vaccine is under development by the Shanghai, China-based company for the treatment of Graves’ disease.
Phase II bronchiectasis trial completion
Armata Pharmaceuticals’s APPA-02 saw its PTSR increase after a Phase II trial was completed. The drug candidate’s PTSR increased by seven points in bronchiectasis to 43%.
The Phase II trial’s (NCT05616221) status was updated from active, not recruiting to completed on ClinicalTrials.gov on 14 August, and GlobalData evaluated the asset on 16 August.
The purpose of the multi-centre, double-blind, placebo-controlled, trial was to evaluate the safety, phage kinetics, and efficacy of APPA-02 in treating subjects with non-cystic fibrosis bronchiectasis. The study enrolled 48 patients.
APPA-02 is a synthetic phage that can kill specific bacteria through the expression of biofilm-degrading enzymes and antimicrobial peptides and is under development for the treatment of multidrug-resistant chronic pulmonary Pseudomonas aeruginosa infections in cystic fibrosis patients and non-CF bronchiectasis (NCFB).
Friedreich’s ataxia Phase II investigator-led trial completion
Stealth BioTherapeutics’s Bendavia (elamipretide hydrochloride) saw a nine-point increase in its PTSR, settling at 38% in Friedreich’s ataxia, following the completion of a Phase II investigator-led trial.
The study’s (NCT05168774) status on ClinicalTrials.gov changed from active, not recruiting to completed on 14 August, and GlobalData evaluated the asset on 16 August. The Children’s Hospital of Philadelphia led the study investigating the safety, tolerability and efficacy of Bendavia in the treatment of advanced symptoms of Friedreich ataxia. The trial measured the change in high-contrast visual acuity as its primary outcome.
Bendavia is a cardiolipin binder that treats Friedrich’s ataxia by enhancing ATP synthesis in multiple organs including the heart, kidney, neurons and skeletal muscle. The therapy prevents the opening of the mitochondrial membrane permeability transition pore (mPTP) during the reperfusion period and potentially mitigates tissue necrosis and apoptosis. Stealth has been granted an orphan drug designation for the therapy for the treatment of Friedrich’s ataxia. The Massachusetts, US-headquartered company is also investigating the therapy for Barth syndrome, Duchenne muscular dystrophy and various mitochondrial diseases.
Read the last edition:
Pipeline Moves: Moderna’s pandemic influenza vaccine trial completesNeed 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.
