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FDA Grants Breakthrough Therapy Designation to AlphaMedixTM for Gastroenteropancreatic Neuroendocrine Tumors, Developed by RadioMedix and Orano Med

Tuesday, February 13, 2024

RadioMedix, Inc. and Orano Med, two companies specializing in radiopharmaceuticals, jointly announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to AlphaMedixTM (212Pb-DOTAMTATE) for the treatment of adult patients with unresectable or metastatic, progressive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) expressing somatostatin receptors, who have not undergone peptide receptor radionuclide therapy (PRRT) previously. AlphaMedixTM is a Targeted Alpha Therapy currently in Phase 2 clinical development, utilizing an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) to generate alpha particles in vivo. Alpha particles have high energy and a short path length, enabling precise targeting and destruction of cancer cells while minimizing damage to surrounding healthy tissue. This designation marks AlphaMedixTM as the first Targeted Alpha Therapy to receive Breakthrough Therapy Designation.

RadioMedix, expressed optimism regarding the potential of AlphaMedixTM to revolutionize the treatment of neuroendocrine tumors. He highlighted the therapy's potential to outperform current PRRT treatments using beta-particle emitters, offering significant benefits to patients with metastatic or inoperable SSTR-expressing GEP-NETs. The FDA's decision is seen as a positive development for patients with this condition, representing a crucial milestone in expediting the therapy's development.

The Breakthrough Therapy Designation is based on findings from both phase 1 and ongoing phase 2 clinical trials evaluating the safety and efficacy of AlphaMedixTM. In the phase 1 trial, treatment was well-tolerated, with a notable response rate (according to RECIST 1.1) of 62.5% among GEP-NET patients who had not previously undergone PRRT with LutatheraTM, which utilizes the beta-particle emitter Lutetium-177. In the phase 2 trial, the therapy has already achieved the target response rate, surpassing expectations ahead of the anticipated top-line data release in mid-2024.

Orano Med, emphasized the significance of securing FDA Breakthrough Therapy Designation for AlphaMedixTM, underscoring the medical community's keen interest in Targeted Alpha Therapies utilizing lead-212. Dodet affirmed the potential of Targeted Alpha Therapies like AlphaMedixTM to usher in the next generation of radioligand therapies, delivering enhanced cytotoxicity against cancer cells while sparing adjacent healthy cells from toxicity. The FDA's recognition further reinforces Orano Med's commitment to making innovative lead-212-based therapies accessible to patients globally.

Breakthrough Therapy Designation expedites the development and review process for drugs aimed at treating serious conditions, where preliminary clinical evidence suggests substantial improvement over existing therapies on clinically significant endpoints.

Neuroendocrine tumors (NETs) encompass a diverse range of rare neoplasms originating from neuroendocrine cells, predominantly occurring in the gastrointestinal tract and pancreas (GEP-NETs). Most NETs exhibit strong expression of somatostatin receptors (SSTRs). In the United States, approximately 12,000 patients are diagnosed with neuroendocrine tumors annually, with a metastatic stage 5-year survival rate averaging 60%.

Targeted Alpha Therapy (TAT) leverages the ability of biological molecules to target cancer cells combined with the potent cell-killing capabilities of alpha-emitting radioisotopes. Alpha decay involves the emission of a helium nucleus (alpha particle) with very high linear energy transfer and a range emission spanning only a few cell layers, inducing irreparable DNA damage in nearby cancer cells while sparing healthy tissue. Consequently, alpha emitters represent powerful payloads for targeted therapies.



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