Abdera Therapeutics' ABD-147 Gets FDA Orphan Drug Status for Neuroendocrine Carcinoma

Abdera Therapeutics Inc., a biopharmaceutical company based in South San Francisco, has announced that its drug ABD-147 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for treating neuroendocrine carcinoma. ABD-147 is designed to deliver Actinium-225 (225Ac), a radioactive isotope, to solid tumors that express DLL3, a protein often found in neuroendocrine tumors but rarely in normal cells. Abdera plans to begin a Phase 1 clinical trial in 2024, targeting patients with small cell lung cancer (SCLC) or large cell neuroendocrine carcinoma (LCNEC) who have already been treated with platinum-based chemotherapy.

Philippe Bishop, M.D., the chief medical officer, emphasized the aggressive and difficult nature of treating neuroendocrine carcinomas like SCLC and LCNEC. He believes that ABD-147, which delivers a potent radioisotope to tumors expressing DLL3, could become a leading treatment option for these aggressive cancers. The FDA had also recently granted Fast Track designation to ABD-147 for treating extensive-stage small cell lung cancer (ES-SCLC) in patients who had progressed after platinum-based chemotherapy.

The FDA’s Orphan Drug Designation program assists in developing drugs for rare diseases, providing incentives such as tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity upon approval.

Neuroendocrine carcinoma originates from cells that have both nerve and hormone-producing characteristics. These cancers can appear in various body parts, including the lungs, digestive tract, pancreas, and prostate. They are known for high mitotic rates, extensive necrosis, and a high mutational burden. Small cell lung cancer (SCLC) and large cell neuroendocrine carcinoma (LCNEC) are particularly aggressive forms, representing around 15% of all lung cancer cases. These cancers often metastasize to the brain, liver, and bone, with a median survival of only two to four months without treatment. Even with treatment, the five-year survival rate is low, ranging from 5% to 25%. Consequently, there is an urgent need for new treatment options.

ABD-147 is a biologic therapy that targets DLL3, a protein involved in the differentiation of neuroendocrine and epithelial cells in the lungs. DLL3 is highly expressed in more than 80% of high-grade neuroendocrine carcinomas, including SCLC, but is rarely found in nonmalignant cells. This makes DLL3 a promising target for radiotherapy, as ABD-147's mechanism allows it to selectively deliver the radioactive isotope Actinium-225 to cancer cells, minimizing damage to healthy tissue.

Abdera's ROVEr™ platform (Radio Optimized Vector Engineering) plays a crucial role in this targeted approach. The platform allows for the custom engineering of radiopharmaceuticals with optimized pharmacokinetic properties, ensuring high tumor uptake while reducing exposure to the kidneys and mitigating other systemic radiotoxicities like myelosuppression. This enables the delivery of powerful alpha or beta particles to selectively destroy tumor cells, sparing healthy cells, and offering potentially transformative cancer treatments.

Abdera Therapeutics specializes in designing precision radiopharmaceuticals for cancer using advanced antibody engineering. Their ROVEr platform allows them to develop therapies against both validated and novel targets, delivering potent radioisotopes to selectively kill cancer cells. ABD-147 is their leading program, aimed at treating SCLC and LCNEC by targeting DLL3-expressing solid tumors. Abdera is headquartered in South San Francisco, CA, with additional offices in Vancouver, British Columbia, Canada.

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