BioMarin Pharma (BMRN) has announced a preclinical collaboration and license agreement with DiNAQOR AGto develop novel gene therapies to treat rare genetic heart diseases.
“With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology,” said Jean-Jacques Bienaimé, CEO of BioMarin. “This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies.”
DiNAQOR, a gene therapy platform company, will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones. The company did not disclose financial terms.
BioMarin management also reiterated its 2020 GAAP net income guidance of $20 to $80 million, including the collaboration payments.
The license initially covers DiNAQOR’s lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). This a disease in which the heart muscle becomes abnormally thick, impairing the heart’s ability to pump blood effectively.
Additionally, the companies will collaborate on several of DiNAQOR’s other pipeline programs, and BioMarin can extend the license to include these additional programs on similar terms.
“Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR” the press release also reveals.
BioMarin stock is currently trading up 4% on a year-to-date basis, and the Street sees potential for prices to climb a further 30%. Indeed the stock boasts a Strong Buy analyst consensus, with 14 recent buy ratings vs 3 hold ratings. (See BioMarin stock analysis on TipRanks).
“We like BMRN as an established leader in rare diseases with a well-appreciated sustainable base business and promising long-term growth prospects in hemophilia A (BMN-270)” commented RBC Capital analyst Kennen MacKay on April 30. He has a buy rating on the stock and $113 price target.
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