Approval prospects drop after Hengrui Medicine completes Phase III breast cancer treatment

This week Move pipelineWe begin by examining the completion of a phase III trial in breast cancer, a phase II trial in amyotrophic lateral sclerosis, and a phase I trial in chronic heart failure. We also review the termination of a phase II trial investigating gene therapy in lecithin-cholesterol acyltransferase deficiency. We finish this week with a look at the completion of clinical trials in oncology and hematology.

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Completion of breast cancer phase III clinical trial

Jiangsu Hengrui Medicine’s Airuika (camrelizumab) has a decreased likelihood of approval (LoA) in triple-negative breast cancer (TNBC) after completing a Phase 3 trial. On TNBC he dropped 9 points in LoA to 52%.

GlobalData evaluated the property on May 8 after updating the May 6 study (NCT04335006) entry. The trial was terminated due to adjustments in the sponsor’s research and development strategy, according to the trial registry.

LoA is identified through GlobalData’s analysis that combines machine learning and proprietary algorithms. LoA can be calculated for drugs considering characteristics such as therapeutic area, indication, and molecule type.

This open-label, randomized trial aimed to evaluate the combination of dolphin plus nab-paclitaxel or nab-paclitaxel plus apatinib compared with nab-paclitaxel alone. The trial enrolled 80 patients with unresectable, locally advanced or metastatic TNBC before completion.

Dolphin blocks the activity of programmed cell death protein (PD-1). It was launched in China in 2019 for various cancer indications.

Institution-Sponsored Phase II ALS Clinical Trial Completed

Eisai’s Fycompa (perampanel) reduced phase transition success rate (PTSR) in amyotrophic lateral sclerosis (ALS) after completion of a site-sponsored Phase II trial. His PTSR on the drug decreased by 11 points and settled at 48% on ALS. PTSR is the percentage probability that a drug will successfully progress from one stage of his development to the next.

The Phase II study (NCT03020797) status was updated May 5 on from open to closed, and GlobalData evaluated the assets on May 9. According to, the trial was closed because enrollment was below study goals and because too many subjects were terminated early due to disease progression. The trial was sponsored by Stony Brook University, based in New York, USA.

A quadruple mask trial evaluated the safety and efficacy of Fycompa. The study was originally expected to recruit 60 adult participants diagnosed with ALS with first clinical debilitating disease within the past three years. However, the trial ultimately enrolled only 12 subjects.

Fycompa is approved by the FDA for the treatment of partial-onset, generalized, and tonic-clonic seizures in people with epilepsy. In this study, Fycompa was used off-label for adult ALS at an oral dose corresponding to the lower end of the recommended dose range for epilepsy.

This drug is a glutamate ion channel receptor AMPA-type subunit antagonist that inhibits excessive AMPA receptor activity to reduce the imbalance between excitatory and inhibitory neurons in the brain.

Bayer halts phase I trial in heart failure

Bayer’s BAY-2413555 reduced PTSR in chronic heart failure after completing a Phase I clinical trial. Chronic heart failure lowered his PTSR by 27 points to 23%.

According to, a Phase I trial (NCT05532046) was closed due to new preclinical findings in a chronic toxicity study, but no further details were provided.’s listing he updated on May 8, with GlobalData evaluating the property the next day.

The objective of a double-blind, randomized, placebo-controlled, dose escalation study was to determine the safety, pharmacokinetics, and tolerability of BAY-2413555 in patients with heart failure and with an implanted cardioverter-defibrillator or cardiac resynchronization device. to assess gender. The trial enrolled only 22 of the 129 patients originally scheduled to participate.

BAY-2413555 works by an undisclosed mechanism of action. This drug candidate is being developed for the treatment of chronic heart failure.

Discontinuation of gene therapy trial

CellGenTech’s DYD-701 reduced the PTSR in lecithin-cholesterol acyltransferase (LCAT) deficiency by 13 points, settling at 8% after the phase II trial was discontinued.

The recruitment status of this study (jRCTa030190230) in the Japan Clinical Trials Registry was changed from recruitment to suspension on April 25. GlobalData evaluated his property on May 1st. The experience entry did not state the reason for the cancellation.

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

DYD-701 is a gene therapy drug made from genetically engineered preadipocytes. Mutations in the LCAT gene cause LCAT deficiency. CellGenTech’s treatments allow the body to generate her LCAT to correct disordered high-density lipoprotein (HDL) levels associated with symptoms.

Chiba, Japan-based CellGenTech has an agreement with Osaka, Japan-based pharmaceutical company DyDo Pharma to co-develop and commercialize DYD-701 in Japan.

Harpoon Completes Phase I/II Oncology Clinical Trials

Harpoon Therapeutics’ HPN-536 increased PTSR in fallopian tube and epithelial carcinomas after completion of Phase I/II trials. His PTSR for the drug increased by 6 points to 38% in both indications.

The Phase I/II study (NCT03872206) status was updated from Ongoing, Unrecruited to Completed on May 6 on, and GlobalData evaluated the asset on May 8. The aim of the open-label study was to evaluate the safety, tolerability, and pharmacokinetics of HPN-536 in patients with advanced cancer associated with mesothelin expression. Ninety-five patients were enrolled in this trial.

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

Phase I study completed in hematologic disease

Sanofi’s efanesoctocog alfa (BIV001) increased PTSR following completion of a Phase 1 study in von Willebrand disease (vWD). vWD increased PTSR by 7 points to 83%.

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

This open-label study evaluated the pharmacokinetics, safety and tolerability of efanesoctocog alfa in adults with vWD types 2N and 3. The primary aim of the study was to characterize the pharmacokinetics of efanesoctocog alfa after a single intravenous dose.

vWD is a blood disorder in which the blood loses its ability to clot properly, causing heavy bleeding. Von Willebrand factor helps platelets stick together to form a clot. Type 2 vWD occurs when the von Willebrand factor does not work properly, whereas type 3 patients have very low or no von Willebrand factor levels and have very severe symptoms. cause.

Type 2N specifically includes patients with genetic mutations located in the region encoding the binding site of von Willebrand factor for factor VIII. Ephanes Soctocog Alfa is a clotting factor VIII replacement therapy. The introduction of factor VIII causes the formation of blood clots in the body.

In February 2023, the FDA approved efanesoctocog alfa for routine prophylaxis and on-demand treatment to control bleeding episodes and for perioperative management (surgery) in adults and children with hemophilia A. . This treatment is marketed under the brand name Altuviiio.

Need to know:

GlobalData’s proprietary model uses a combination of machine learning and algorithms to calculate PTSR and LoA for individual drugs. LoA indicates the probability that a drug will ultimately receive market approval, while PTSR indicates the probability that the drug will advance to the next stage of clinical development. The model uses data points from individual drugs, clinical trials, regulatory milestones, companies and financial databases.

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