This week on Pipeline Moves, we start by investigating terminations of a Phase III trial in breast cancer, a Phase II trial in amyotrophic lateral sclerosis and Phase I trial in chronic heart failure. We also review a suspension of a Phase II trial investigating gene therapy in lecithin-cholesterol acyltransferase deficiency. We finish the week by looking at trial completions in oncology and blood disorder.

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Termination of Phase III breast cancer trial

Jiangsu Hengrui Medicine’s Airuika (camrelizumab) saw its Likelihood of Approval (LoA) drop in triple-negative breast cancer (TNBC) after a Phase III trial was terminated. The LoA decreased by nine points to 52% in TNBC.

GlobalData evaluated the asset on 8 May after an update on the study’s (NCT04335006) entry on 6 May. The trial was terminated due to the sponsor R&D strategy adjustment, according to the trial registry.

LoA is identified via GlobalData’s analysis using a combination of machine learning and a proprietary algorithm. LoA can be calculated for a drug by considering characteristics like therapy area, indication and molecule type.

The open-label and randomised trial intended to evaluate Airuika in combination with nab-paclitaxel or with nab-paclitaxel and apatinib, compared to nab-paclitaxel alone. The trial enrolled 80 patients with unresectable locally advanced or metastatic TNBC before termination.

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Airuika blocks the activity of programmed cell death protein (PD-1). In 2019, it was marketed for various cancer indications in China.

Institution-sponsored Phase II ALS trial terminated

Eisai’s Fycompa (perampanel) saw its Phase Transition Success Rate (PTSR) decrease in amyotrophic lateral sclerosis (ALS) after an institution-sponsored Phase II trial was terminated. The drug’s PTSR decreased by 11 points, settling at 48% in ALS. PTSR is the probability, given as a percentage, of a drug progressing successfully from one development stage to the next.

The Phase II trial’s (NCT03020797) status was updated from recruiting to terminated on on 5 May, and GlobalData evaluated the asset on 9 May. According to, the trial was terminated due to enrolment being below the study target and because too many subjects had early termination due to disease progression. The trial was sponsored by Stony Brook University, based in New York, US.

The quadrupled-masked study evaluated the safety and efficacy of Fycompa. The study was initially anticipated to recruit 60 adult participants with a diagnosis of ALS that presented their first clinical weakness within the past three years. However, the trial ended up enrolling only 12 subjects.

Fycompa is approved by the FDA for the treatment of partial, generalised, and tonic-clonic seizures in patients with epilepsy. In this study, Fycompa was used off-label for adults with ALS at an oral dose on the low end of the recommended dose range for epilepsy.

The drug is a glutamate ionotropic receptor AMPA type subunit antagonist, which acts by inhibiting the excess activity of AMPA receptors to reduce the excitatory-inhibitory nerve imbalance in the brain.

Bayer terminates Phase I trial in heart failure

Bayer’s BAY-2413555 saw its PTSR drop in chronic heart failure after a Phase I clinical trial was terminated. The PTSR declined by 27 points to 23% in chronic heart failure.

The Phase I trial (NCT05532046) was terminated due to new preclinical findings in a chronic toxicology study as per Clinical, but no further details were provided. The listing was updated on 8 May and GlobalData evaluated the asset the following day.

The purpose of the double-blind, randomised, placebo-controlled, dose titration study was to evaluate the safety, pharmacokinetics, and tolerability of BAY-2413555 in patients with heart failure and implanted cardiac defibrillators or cardiac resynchronisation devices. The trial enrolled only 22 patients out of the 129 originally anticipated to participate.

BAY-2413555 acts by an undisclosed mechanism of action. The drug candidate is under development for the treatment of chronic heart failure.

Gene therapy trial suspension

CellGenTech’s DYD-701 saw a 13-point drop in its PTSR, settling at 8% in lecithin-cholesterol acyltransferase (LCAT) deficiency after a Phase II trial was suspended.

The trial’s (jRCTa030190230) recruitment status in the Japan Registry of Clinical Trials changed from recruiting to suspended on 25 April. GlobalData evaluated the asset on 1 May. The reason for the suspension was not listed on the trial entry.

The Phase II, single-arm open-label study’s primary outcome investigated the number of adverse events caused by treatment with DYD-701.

DYD-701 is a gene therapy made from genetically modified preadipocytes. A mutation in the LCAT gene causes LCAT deficiency. CellGenTech’s therapy allows the body to produce LCAT to correct impaired high density lipoprotein (HDL) levels associated with the condition.

The Chiba, Japan-based CellGenTech has a joint development and commercialisation agreement with Osaka, Japan-based pharmaceutical company DyDo Pharma for DYD-701 in Japan. 

Harpoon completes Phase I/II oncology trial

Harpoon Therapeutics’s HPN-536 saw its PTSR rise in fallopian tube cancer and epithelial cancer after a Phase I/II trial was completed. The drug’s PTSR grew by six points to 38% in both indications.

The Phase I/II trial’s (NCT03872206) status was updated from active, not recruiting to completed on on 6 May, and GlobalData evaluated the asset on 8 May. The objective of the open-label trial was to assess the safety, tolerability, and pharmacokinetics of HPN-536 in patients with advanced cancers associated with mesothelin expression. The trial enrolled 95 patients.

HPN-536 is a trispecific monoclonal antibody (mAb) that exhibits antineoplastic properties by targeting mesothelin and CD3. The mAb is under development for the treatment of various mesothelin-associated solid tumours.

Phase I trial completion in blood disorder

Sanofi’s efanesoctocog alfa (BIV001) saw its PTSR rise after a Phase I trial completion in Von Willebrand disease (vWD). The PTSR increased by seven points to 83% in vWD.

The Phase I study’s (NCT04770935) status changed from recruiting to completed on 27 April, and GlobalData evaluated the asset on 1 May. The trial was sponsored by Sanofi’s subsidiary Bioverativ.

The open-label study assessed the pharmacokinetics, safety and tolerability of efanesoctocog alfa in adults with type 2N and 3 vWD. The primary objective of the study was to characterise the pharmacokinetics of efanesoctocog alfa after a single intravenous administration.

vWD is a blood disease that causes the blood to lose the ability to clot correctly, leading to heavy bleeding. Von Willebrand factor helps platelets stick together to form a blood clot. Type 2 vWD occurs when the Von Willebrand factor does not work properly, but type 3 patients have very low levels of Von Willebrand factor or none, causing very severe symptoms.

Type 2N specifically includes patients with gene mutations located in the region coding for the binding site of Von Willebrand factor for factor VIII.  Efanesoctocog alfa is a coagulation factor VIII replacement therapy. The introduction of factor VIII allows blood clots to form in the body.

In February 2023, the FDA approved efanesoctocog alfa for routine prophylaxis and as an on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with haemophilia A. The therapy is sold under the brand name Altuviiio.

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.