The US Food and Drug Administration (FDA) has granted breakthrough device designation to RadioMedix and Orano Med’s AlphaMedix (lead-212-Dotamtate), a targeted alpha therapy currently in Phase II trials for a rare type of neuroendocrine tumour.

The two US-based companies, which are partnered in AlphaMedix’s development, stated that this is the first FDA breakthrough designation for a targeted alpha therapy, according to a 12 February press release.

Targeted alpha therapy involves the emission of an alpha particle – a helium nucleus – combined with high linear energy transfer to kill tumour cells by DNA double-strand breaks. By coupling the alpha particle emitting radioisotopes to tumour-selective carrier molecules, cancer cells can be targeted while leaving healthy cells alone.

RadioMedix and Orano Med’s AlphaMedix uses a somatostatin receptor (SSTR)-targeting peptide complex radiolabelled with lead-212. The FDA designation was based on results from a Phase I trial (NCT03466216) and an ongoing Phase II trial (NCT05153772), investigating the therapy in patients with unresectable or metastatic, progressive SSTR-expressing gastro-enteropancreatic neuroendocrine tumours (GEP-NETs).

Around 12,000 patients in the US are expected to be diagnosed with neuroendocrine tumours annually, with an average survival rate of 60% after five years at a metastatic stage, according to RadioMedix and Orano Med.

A response rate of 62.5% was recorded in the Phase I trial for GEP-NET patients who had never received Novartis’ peptide receptor radionuclide therapy (PRRT) Lutathera. According to the two companies, the target response rate has already been achieved in the Phase II trial. Patient enrolment in the trial was completed in May 2023, with topline data expected in mid-2024.

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By GlobalData

RadioMedix’s CEO Ebrahim Delpassand believes the company’s candidate could demonstrate substantial benefit over current PRRT therapies. It will have to catch up to Novartis however – beta-emitter Lutathera saw sales of $458m in the first nine months of 2023, a 34% increase from 2022. The therapy, approved by the FDA in 2018, was amongst the top 20 best-selling products in Novartis’ portfolio in 2023.

The Swiss company recently reported results from a trial investigating the therapy in combo with long-acting release (LAR) octreotide – data indicated the risk of disease progression or death was significantly cut by 72%.

Exelixis and partner Ipsen are also rising quickly in the neuroendocrine tumour space with their tyrosine kinase inhibitor candidate Cabometyx (cabozantinib). The companies reported positive results from a Phase III trial in October 2023, with an FDA application expected in 2024.

GlobalData forecasts the neuroendocrine tumour market to grow to $3.42bn in 2030 across eight major markets, with Novartis expected to control half of the market with its portfolio that includes its older therapy octreotide, which is marketed as Sandostatin.

GlobalData is the parent company of Pharmaceutical Technology.

Orano Med’s CEO Julien Dodet said: “We are convinced that targeted alpha therapies, such as AlphaMedix, will lead the next generation of radioligand therapies.”