FDA selects RayzeBio to Participate in the Inaugural CDRP Program to Expedite Commercial Manufacturing Readiness for RYZ101 in GEP-NETs

SAN DIEGO, Oct. 24, 2023 (GLOBE NEWSWIRE) -- RayzeBio, Inc. (NASDAQ:RYZB), a targeted radiopharmaceutical company developing an innovative pipeline against validated solid tumor targets, today announced the FDA has selected RYZ101 (Actinium-225 DOTATATE) into the inaugural Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The CDRP program was created by the FDA to facilitate CMC readiness for selected therapies with accelerated clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. Under the program, the FDA will work closely with RayzeBio to facilitate CMC development of RYZ101 for the treatment of somatostatin receptor positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that have progressed following prior Lutetium-177 labelled somatostatin analogue therapy.

"We are thrilled to collaborate with the FDA on this important initiative," said Eric Bischoff, SVP of Development and Operations at RayzeBio. "Radiopharmaceuticals is an emerging modality and requires a highly specialized manufacturing process. Our lead program, RYZ101, has the potential to be the first approved Actinium-225 based therapy. We look forward to working with the FDA on ensuring the commercial manufacturing readiness for RYZ101."  

"We applaud the FDA for undertaking this initiative to partner with pioneers in emerging modalities. We are making significant investments to ensure commercial readiness for RYZ101, including building our own manufacturing facility in Indianapolis. RYZ101 has already demonstrated encouraging preliminary response data and is now enrolling in a registrational Phase 3 clinical trial. We are eager to collaborate with the FDA as we aim to develop and bring this important therapy to market," said Ken Song, M.D., President and CEO of RayzeBio.

About gastroenteropancreatic neuroendocrine tumors

Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are rare tumors with an incidence of approximately 18,000 patients annually in the United States. As many GEP-NETs follow an indolent disease course, the prevalence of GEP-NETs in the United States is approximately 200,000 patients. SSTR2 is expressed in 80-90% of GEP-NET tumors. Tumors can be aggressive and resistant to therapy, with metastatic disease present at diagnosis in 40-76% of cases. Lutathera is a targeted radiopharmaceutical therapy comprised of a somatostatin analog peptide labeled with the beta-emitting radioisotope Lutetium-177 (Lu177), which received regulatory approval for treatment of adult patients with SSTR+ GEP-NETs in Europe and the United States in 2017 and 2018, respectively. However, most patients who receive Lu177-based somatostatin therapies eventually experience tumor progression and have limited subsequent treatment options.

About RYZ101

RYZ101 is an investigational targeted radiopharmaceutical therapy, designed to deliver a highly potent radioisotope, Actinium-225 (Ac225), to tumors expressing SSTR2. RYZ101 is being evaluated in clinical studies for patients with SSTR+ GEP-NETs who have previously been treated with Lu177-based somatostatin therapies and also in patients with newly diagnosed extensive stage small cell lung cancer. Details of the studies can be found at https://clinicaltrials.gov/ct2/show/NCT05477576 and https://clinicaltrials.gov/ct2/show/NCT05595460.

Ac225 for the study was provided by multiple sources including the U.S. Department of Energy Isotope Program.

About RayzeBio

RayzeBio is building a vertically integrated radiopharmaceutical therapeutics (RPT) company to treat various cancers, with its lead program in a Phase 3 clinical trial. RayzeBio has created a pipeline of multiple drug candidates in therapeutic areas with significant market opportunities. Much like antibody drug conjugates emerged as a new and transformative treatment modality in certain cancers, the company sees an opportunity for innovative radiopharmaceutical therapeutics to follow a similar path. RayzeBio believes its strategic investments in building a robust product pipeline, development capabilities, and manufacturing infrastructure position the company to be an industry-leading pioneer in the broad application of RPT for cancer.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Such forward-looking statements include, without limitation, statements regarding the potential benefits of RYZ101 based on preliminary response data, the anticipated development timeline of RYZ101, the expected benefits of the CDRP program, and the ability of RayzeBio to expedite commercial manufacturing readiness for RYZ101. Words such as "believe," "anticipate," "plan," "expect," "intend," "will," "may," "goal," "potential" and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements necessarily contain these identifying words. These forward-looking statements are based on the beliefs of the management of the company as well as assumptions made by and information currently available to the company. Such statements reflect the current views of the company with respect to future events and are subject to known and unknown risks and uncertainties, including business, regulatory, economic and competitive risks and uncertainties about the company, including, without limitation, risks inherent in developing drug candidates, future results from the company's ongoing and planned clinical trials, the company's ability to obtain adequate financing to fund its planned clinical trials and other expenses, risks that future clinical trial results may not be consistent with interim, initial or preliminary results or results from prior preclinical studies or clinical trials, trends in the industry, the legal and regulatory framework for the industry and future expenditures. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. The actual results may vary from the anticipated results and the variations may be material. Other factors that may cause the company's actual results to differ from current expectations are discussed in the company's filings with the Securities and Exchange Commission (the "SEC"), including the section titled "Risk Factors" in the company's Registration Statement on Form S-1 filed with the SEC on September 11, 2023. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this press release is given. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact: Arvind Kush

Email: [email protected]