Astrazeneca Blockbuster Prospect is a bust in pivoting in the amylosis of the light chain

A drug that Astrazeneca distinguished as one of its main drivers of potential income growth over the next decade has failed a pivotal study in the amylosis of the light chain, a rare disease with few treatment options. Despite the setback, the company highlights the encouragement of signs in a subgroup of patients who could offer a track.

Astrazeneca has provided no specific details on the results of the phase 3 test other than saying that the drug, the handlemimab, has not reached a statistical meaning on the main objective measuring the frequency of hospitalizations and death time after no cause. Astrazeneca said that its stolen, its subsidiary of rare diseases, will share the results with the regulatory authorities and present them to a future medical meeting.

Amylosis is a disorder in which abnormally folded proteins, called amyloid fibrils, accumulate in organs and tissues. In the amyloidosis of the light chain, plasma cells in the bone marrow produce abnormally immunoglobulin light chain proteins. The accumulation of these abnormal proteins is particularly pronounced in the heart and the kidneys. Heart insufficiency is the most common cause of death for patients with this disease.

The standard treatment of light chain amyloidosis is the cyclophosphamide chemotherapy alongside the velcade of cancer drugs and dexamethasone of steroid drugs. A new drug option is Darzalex Faspro, an injectable subcutaneous version of the Johnson & Johnson Multimome Drug Darzalex blockbuster. In 2021, this antibodies extended its approval to include amylosis of the newly diagnosed light chain.

ANSELAMIMAB is a monoclonal antibody designed to bind to the poorly folded amyloid fibrils to reduce or eliminate the deposits of these proteins in tissues and organs. This medication is intended to bind specifically to targets on poorly folded, saving amyloid light chains. The phase 3 program for Anselamimab consists of two phase 3 studies controlled by placebo, each testing the drug at a different stage of amyloidosis of the light chain. These studies evaluate ANSELAMIMAB parallel to the standard treatment of the disease.

In total, studies have recruited 406 patients from 19 countries. The participants were assigned to random to receive the study medication or a placebo administered in the form of an intravenous infusion once a week during the first four weeks, then every two weeks until week 50. Darzalex was authorized but not required as part of the standard diet. Astrazeneca said that around 80% of participants had received the J & J drug as part of their treatment. Participants who finished the study had the possibility of participating in an outdoor extension study evaluating the Anselamimab plus the treatment of the care standard up to 24 months.

In the announcement by Astrazeneca of reading phase 3, Dr. Ashutosh Wechalekar, a consultant hematologist at the University College London Hospitals NHS Foundation Trust, professor of medicine and hematology at the University College of London, and the main investigator of the phase 3 program, highlighted the results of the sub-group-without describing the subgroup.

“Although the study did not meet the main evaluation criterion of the overall patient population, the results of a predefined sub-group suggest that Anselamimab, by targeting and cleaning amyloid deposits, can tackle a main cause of damage to organs and functional alteration in these patients,” said Wechalekar. “The potential of survival and the reduction of cardiovascular hospitalizations would represent progress that changes the practice for this group of patients.”

Astrazeneca said the results show that Anselamimab was well tolerated, the majority of the balanced events between the handle of handlemimab and the placebo arm. No details on these events have been disclosed. The company said that the assessment of the full test results is underway to further characterize the efficiency and safety of Anselamimab.

Amylosis of the light chain has proven difficult for drug developers. In May, Prothena announced that her Birtamimab antibody medication did not oppose the main objective of her phase 3 test. Birtamimab had previously failed a mid-term study, but was revived and advanced towards phase 3 according to the improvement of a subgroup of high-risk patients. After the last failure of the clinical trial, Prothena, based in Dublin, interrupted the program and began a corporate restructuring. The other companies that have failed with light chain amyloidosis drugs include Takeda Pharmaceutical and GSK.

In a presentation of investors in 2024 on the portfolio and the rare diseases of rare ancestor diseases, Astrazeneca scored Anselamimab as one of the three candidates drugs likely to contribute to $ 1 billion to $ 3 billion in potential income from the peak year.

Anselamimab, formerly known as Cael-101, came with the acquisition of pharmaceutical alexion in 2021 from Astrazeneca. Alexion had associated itself with the initial developer of the drug, Caelum BioSciences, in 2019. This alliance gave Alexion a minority participation in biotechnology and the possibility of acquiring the rest of the actions that it did not already have. Astrazeneca exerted this option, paying around $ 150 million. Additional $ 350 million is linked to the realization of milestones.

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