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OPKO Health Licenses Oligonucleotide Therapeutics Platform to CAMP4 Therapeutics

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MIAMI, July 12, 2021 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) today announced it has entered into an exclusive worldwide agreement with privately held CAMP4 Therapeutics Corporation (CAMP4) for the development, manufacture and commercialization of therapeutics utilizing the AntagoNAT technology, an oligonucleotide platform developed under OPKO CURNA. AntagoNATs are oligonucleotide compounds that target non-coding natural antisense transcripts leading to an upregulation of a desired functional protein. CAMP4 has prioritized OPKO’s lead AntagoNAT compound to progress into clinical trials for the treatment of Dravet syndrome.

Under the terms of the agreement, OPKO will receive an upfront payment and shares of CAMP4. In addition, OPKO will be eligible to receive up to $93.5 million and additional shares upon the achievement of certain development and sales milestones for products developed from this technology and associated intellectual property. CAMP4 will also pay OPKO double-digit royalties on product sales.

“We are delighted to enter into this licensing agreement to accelerate the development and commercialization of potential disease-modifying therapeutics. With a clear focus on therapeutics that restore healthy gene expression, CAMP4 has the expertise to advance our Dravet syndrome compound through the necessary patient trials. This agreement validates our technology and allows OPKO to focus resources on other areas of our business. We continue to seek license opportunities to monetize and leverage our early-stage assets,” said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO.

According to the Epilepsy Foundation, Dravet syndrome is a rare, drug-resistant epilepsy that begins in the first year of life in an otherwise healthy infant. It is estimated that 1-in-20,000 to 1-in-40,000 individuals have Dravet syndrome. Most cases are due to SCN1A gene mutations and most children develop varying degrees of neurodevelopmental disability.

CAMP4 is pioneering a novel approach to programmable therapeutics that combines a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. The company’s RNA Actuating Platform’s proprietary insights enable drug discovery and development that aims to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients.

About OPKO Health

OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit www.opko.com.

Cautionary Statement Regarding Forward Looking Statements

This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding product development efforts as well as other non-historical statements about our expectations, products, beliefs or intentions regarding our business, financial condition, strategies or prospects including statements regarding expectations about the success of the collaboration and licensing agreement with CAMP4, whether CAMP4 will successfully develop, obtain regulatory approval for, launch or commercialize any product using Dravet or non-Dravet technology that is part of the CURNA platform, whether payment milestones and royalty obligations will ever be triggered, and the expected market for the licensed products. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and under the heading “Risk Factors” in our other filings with the Securities and Exchange Commission as well as risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the PSLRA.

Contacts:

LHA Investor Relations
Yvonne Briggs, 310-691-7100
ybriggs@lhai.com
or
Bruce Voss, 310-691-7100
bvoss@lhai.com