For those who don’t know, Regenxbio is a gene therapy leader seeking to improve lives through the curative potential of a successful gene therapy. The firm’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AVrh10. Regenxbio and its NAV Technology Platform Licensee firms are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Healthcare Royalty Management LLC

HCR is a private investment firm that purchases royalties and uses debt-like structures to invest in commercial or near-commercial stage biopharmaceutical assets. HCR has raised $5.7 billion in cumulative capital commitments with offices in Stamford, CT, San Francisco, CA, Boston, MA and London, UK.

From the President & CEO of Regenxbio

Kenneth T. Mills, President and Chief Executive Officer of Regenxbio, explained, “Our rapidly advancing internal pipeline has enabled us to broaden the potential impact that gene therapies can have for patients in both large and orphan indications. This agreement with HCR provides us with significant additional non-dilutive funding to continue our momentum in the clinic focused on RGX-314 and our rare neurodegenerative disease platform, including RGX-121, as well as the opportunity to develop new innovations for patients in other disease areas. The capital will continue to support our pipeline transition into late-stage development and the establishment of internal manufacturing facilities with 2,000 liter scale using our platform suspension cell culture process for emerging commercial requirements, so that we can continue to work towards our mission of improving the lives of patients.”

Under the Terms of the Agreement:

Regenxbio will receive $200 million from HCR as an upfront payment in exchange for the royalty rights from the net sales of Zolgensma, including a portion of the royalties received in the fourth quarter of 2020, up to 1.3 times the purchase price until November 7, 2024 or, if such cap is not met by November 7, 2024, up to 1.5 times the purchase price thereafter. If either cap is met the royalty rights would revert to Regenxbio.

Clarke B. Futch, Managing Partner & Chairman of HCR, said, “We are pleased to partner with Regenxbio in this royalty agreement to recognize the value of this therapy, and to enable further development of their internal pipeline of new gene therapies for patients in need,”

Prohost Observations

You already know that Zolgensma, which currently belongs to Novartis (NVS), is approved for the treatment of Spinal Muscular Atrophy (SMA) in the United States, Japan, Europe, Brazil and Canada. Novartis is also pursuing registration in additional countries.

You know as well that Zolgensma gene therapy is based on Regenxbio’s NAV technology. The company’s revenues are emanating now from the licensees of its NAV technology and its royalty. Both are expected to increase with the approval of new products by the licensees and the increase in the licensees’ numbers. Regenxbio’s pipeline has many gene therapy products in clinical trials which are moving forward.

The agreement with HCR will definitely help advance the lead products in clinical trials. The approval of these products, which are mostly cures, are expected to bring in revenues in the billions of dollars.

The firm is not there yet; however, we believe in the Regenxbio NAV Technology and in the improvement of AAV, which led to the successful creation and development of gene therapy cures.

Happy Holidays

To read more about Regenxbio please click here.