This week on Pipeline Moves, we kick off by looking at the completion of a Phase III investigator-led trial of Gilead Sciences’s epratuzumab in acute lymphocytic leukaemia.

Meanwhile, Novartis has terminated a Phase III trial of Cosentyx in metabolic dysfunction-associated steatotic liver disease. The team also looks at GSK’s Phase II terminated sarcoma trial.

On a positive note, Sarepta Therapeutics has completed a Phase II trial of its Duchenne muscular dystrophy candidate.

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Phase III investigator led ALL trial completes

Gilead Sciences’s saw its likelihood of approval (LoA) in acute lymphocytic leukaemia (ALL) increase after an investigator-led Phase III trial was completed.

The LoA for epratuzumab increased by six points to 43% in ALL following the completion. LoA is determined using a combination of machine learning and GlobalData’s proprietary algorithm. It can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

The Phase III trial’s (NCT01802814) status was updated from ongoing to completed on on 12 February with GlobalData evaluating the product on the same day.

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The study was sponsored by Charite University, Berlin, Germany.

The randomised, parallel assignment study evaluated intravenous epratuzumab in 700 adolescents with standard risk (SR) first relapsed ALL.

Epratuzumab, a humanised CD22-directed monoclonal antibody, is designed to inhibit the CD22 protein on B cells and reduce B cell signalling and activation, thereby promoting antitumor activity.

Novartis MASLD Phase III trial terminated

Novartis’s Cosentyx (secukinumab) saw LoA in metabolic dysfunction-associated steatotic liver disease (MASLD) decreased after a Phase III trial for psoriasis and coexisting MASLD was terminated due to low enrolment. The LoA for Cosentyx dropped by ten points to 4% in MASLD following the termination.

The Phase III trial’s (NCT04237116) status was updated from completed to terminated on on 8 February with GlobalData evaluating the product on 12 February.

The randomised, double-blind study, dubbed pINPOINt, evaluated the efficacy of intravenous Cosentyx in adults with psoriatic skin and coexisting NAFLD.

The trial was supposed to enrol 90 patients but was terminated after accruing 10 participants.

Cosentyx is a human IgG1 monoclonal antibody designed to selectively bind to interleukin-17A inhibitor and inhibit its interaction with the IL-17 receptor to promote anti-inflammatory responses.

It is marketed for the treatment of moderate to severe plaque psoriasis in patients six years and older who are candidates for systemic therapy or phototherapy and adults with active psoriatic arthritis, active ankylosing spondylitis, or active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation.

GSK terminates Phase II sarcoma trial

GlaxoSmithKline’s Jemperli (dostarlimab) has seen its Phase Transition Success Rate (PTSR) fall after a sponsor-led Phase II trial of the candidate in sarcomas was terminated.

The PTSR for Jemperli in sarcomas has more than halved, dropping by 20 percentage points to 16% following the announcement. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

According to the record, the trial has been terminated due to “difficulty with enrolment”. The listing adds that the goal was to enrol 16 patients, however the trial was terminated after only three patients were enrolled.

The record was updated on 31 January, with GlobalData evaluating the asset on 2 February.

The Phase II, single-arm study (NCT04274023) was exploring the activity of Jemperli in patients with a diagnosis of advanced or metastatic clear cell sarcoma (CCS). The sponsor led trial was being run by the Italian Sarcoma Group with GSK as a collaborator.

Jemperli is an anti-PD-1 monoclonal antibody for cancer immunotherapy. The candidate acts by targeting programmed death-1 (PD-1) which has the potential to activate anti-tumour immunity. The drug candidate is developed based on somatic hypermutation (SHM) technology platform (SHM-XEL platform).

Sarepta’s PTSR increases after Phase II DMD success

Sarepta Therapeutics’s PTSR has increased for its Duchenne Muscular Dystrophy (DMD) exon 51 skipping candidate vesleteplirsen after a Phase II trial met its endpoints.

The PTSR for the candidate rose in DMD by 15 percentage points to 53%.

The company announced positive results from the trial in a press release on 29 January, with GlobalData evaluating the asset on 1 February.

The data showed vesleteplirsen dosed every four weeks recorded substantially higher increases in dystrophin and exon skipping compared to current standard of care (SoC) eteplirsen dosed weekly.

Sarepta Therapeutics’s executive vice president Dr Louise Rodino-Klapac says that the favourable data will help in discussing the candidates next steps with the FDA.

The Phase II trial (NCT04004065) was a global, multi-ascending dose trial which enrolled patients with DMD amenable to exon 51-skipping treatment aged between eight and 21 years.

The FDA halted the Phase II trial in June 2022 due to the serious AE of hypomagnesaemia. In September of the same year, the trial continued after Sarepta changed the protocol and agreed to provide supplemental magnesium.

Vesleteplirsen is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

Phase I/II kidney disease trial completes

Unicocell Biomed’s Elixcyte saw its PTSR increase in chronic kidney disease (chronic renal failure) after a Phase I/II trial was completed. The cell therapy’s PTSR increased by seven points, reaching 38%.

The Phase I/II trial’s (NCT02933827) status was updated from active, not recruiting to completed on on 9 February and GlobalData evaluated the asset on 12 February.

The purpose of the study was to evaluate the safety and efficacy of Elixcyte in moderate to severe chronic kidney disease. The study enrolled 39 patients.

Elixcyte is an immunomodulatory agent. The Taipei, Taiwan-based company is developing the cell therapy candidate for the treatment of chronic kidney disease, chronic wounds, retinal degeneration, and knee osteoarthritis.

Phase I GVHD trial terminated

Genentech’s RG-6287 saw its Phase Transition Success Rate (PTSR) decrease in graft versus host disease (GVHD) after a Phase I trial was terminated. RG-6287’s PTSR dropped by 28 points to 22%. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next. Genentech is a subsidiary of F. Hoffman-La Roche.

The Phase I trial’s (NCT05673876) status was updated from completed to terminated on on 30 January, and GlobalData evaluated the asset the following day.

According to the study’s listing, the study was terminated by the sponsor due to a business decision, not due to safety or efficacy.

The purpose of the randomised, open-label, dose-finding, multi-centre study was to assess the safety, pharmacokinetics, efficacy, of RG-6287 for the treatment of GVHD. The study enrolled 7 patients before being terminated.

Read the last edition:

Pipeline Moves: Approval prospects for Roche’s tiragolumab rise in oesophageal squamous cell carcinoma

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.