212Pb-AR-RMX

HOUSTON & PARIS--( BUSINESS WIRE )--RadioMedix, Inc. and Orano Med, two clinical stage radiopharmaceutical companies, today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to AlphaMedix TM ( 212 Pb-DOTAMTATE) for the treatment of adult patients with unresectable or metastatic, progressive somatostatin receptor expressing gastroenteropancreatic neuroendocrine tumors (GEP-NETs) who are naïve to peptide receptor radionuclide therapy (PRRT). AlphaMedix TM is a Targeted Alpha Therapy currently in Phase 2 clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 ( 212 Pb) that serves as an in vivo generator of alpha particles. Due to their high energy and short path length, alpha emitters enable specific targeting and killing of individual cancer cells, while minimizing toxicity to surrounding healthy tissue. AlphaMedix TM is the first Targeted Alpha Therapy to receive Breakthrough Therapy Designation. “The FDA's Breakthrough Therapy Designation underscores AlphaMedix TM potential as an innovative treatment that could redefine how patients with neurendocrine tumors are treated. We believe that AlphaMedix™ has potential to demonstrate substantial benefit over currently FDA approved PRRT with beta-particle emitters for patients with metastatic or inoperable SSTR-expressing GEP-NETs. The FDA's decision is a great news for patients suffering from this illness, and an important milestone to expedite the development of this new therapy,” said Ebrahim Delpassand, MD, Chairman and Chief Executive Officer of RadioMedix. The Breakthrough Therapy Designation is based on the results from phase 1 and the ongoing phase 2 clinical trials that assessed the safety and efficacy of AlphaMedix TM . In the phase 1 study, treatment was well-tolerated, with a response rate (ORR according to RECIST 1.1) of 62.5% for the GEP-NET patients who had never received PRRT with Lutathera TM , which is based on the beta-particle emitter Lutetium-177. In the phase 2 trial, the target response rate has already been achieved ahead of top-line data, expected in mid-2024. “Receiving FDA Breakthrough Therapy Designation for AlphaMedix TM is a great achievement for everyone involved and confirms the strong interest of the medical community for Targeted Alpha Therapies with lead-212. Based on positive results from our clinical studies to date, we are convinced that Targeted Alpha Therapies, such as AlphaMedix TM , will lead the next generation of radioligand therapies, providing increased cytotoxicity against cancer cells but limited toxicity to adjacent healthy cells. This recognition from the FDA reinforces Orano Med’s commitment to make innovative lead-212-based therapies available to the medical community and patients worldwide,” said Julien Dodet, President and Chief Executive Officer of Orano Med. About Breakthrough Therapy Designation Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). About neuroendocrine tumors Neuroendocrine tumors (NETs) are a heterogeneous group of rare neoplasms that originate from neuroendocrine cells. These neoplasms occur mostly in the gastrointestinal tract and pancreas (GEP-NETs) but can also occur in other tissues including the thymus, lung, and other uncommon sites such as ovaries, heart, and prostate. Most NETs strongly express somatostatin receptors (SSTRs). In the United States, around 12,000 patients annually are expected to be diagnosed with neuroendocrine tumors, with an average 5-year survival rate of 60% at a metastatic stage. About Targeted Alpha Therapy Targeted alpha therapy (TAT) relies on a simple concept: combining the ability of biological molecules to target cancer cells with the short-range and highly energetic cell-killing capabilities of alpha-emitting radioisotopes. Alpha decay consists of the emission of a helium nucleus (alpha particle) together with very high linear energy transfer and a range emission of only few cell layers, resulting in irreparable double strand DNA breaks in cells adjacent only to area of alpha emission. This approach results in an increased cytotoxic potential toward cancer cells while limiting toxicity to nearby healthy cells. As a result, alpha emitters are considered as the most powerful payloads to be found for targeted therapies. About RadioMedix RadioMedix, Inc. is a clinical-stage biotechnology company, and sponsor of the AlphaMedix™ trials, based in Houston and Humble, Texas. The company is focused on innovative targeted radiopharmaceuticals for diagnosis, monitoring, and therapy of cancer. RadioMedix is developing radiopharmaceuticals for PET imaging and therapy (alpha- and beta-labeled agents). The company established contract service facilities for academic and industrial partners including a drug discovery center for the early probe development, a pre-clinical core facility for in vitro and in vivo evaluation of radiopharmaceuticals, and cGMP and analytical suite for Phase I-III clinical trials, and the large-scale post-approval commercial manufacturing facility, the Spica Center. More information about RadioMedix, visit: www.radiomedix.com About Orano Med Orano Med is a clinical-stage biotechnology company that develops a new generation of targeted therapies against cancer using the unique properties of lead-212 ( 212 Pb), a rare alpha-emitting radioisotope and one of the more potent therapeutic payloads against cancer cells known as Targeted Alpha-Emitter Therapy (TAT). The company is developing several treatments using 212 Pb combined with various targeting agents. Orano Med has 212 Pb manufacturing facilities, laboratories, and R&D centers in France and in the US and is currently investing to further expand its GMP-manufacturing capacities for 212 Pb radiolabeled pharmaceuticals in North America and Europe. More information about Orano Med, visit: www.oranomed.com .

By coupling the alpha particle emitting radioisotopes to tumour-selective carrier molecules, cancer cells can be targeted while leaving healthy cells alone. Image credit: Shutterstock/Jurik Peter. 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. See Also: Innovate UK grants funds for breast cancer therapy project FDA grants breakthrough status to J&J’s nipocalimab for HDFN 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. 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.”