RARE Daily

Ultragenyx Raises $300 Million in Public Offering

October 19, 2023

Rare Daily Staff

Days after reporting positive results of a mid-stage study in a rare bone disease, Ultragenyx Pharmaceutical tapped the public markets to raise new capital.

The rare disease focused biotech priced 8.3 million shares of its common stock at $30.00 per share. In addition, in lieu of issuing common stock to certain investors, the company offered pre-funded warrants to purchase 1.7 million shares of its common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant.

Ultragenyx expects to raise $300 million in the underwritten public offering, before deducting underwriting discounts and commissions and other offering expenses and excluding the exercise of any pre-funded warrants. The company also granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1.5 million shares of the company’s common stock at the public offering price, less the underwriting discount.

On Tuesday, Ultragenyx and Mereo BioPharma reported interim data from the phase 2 portion of the phase 2/3 ORBIT study demonstrating that treatment with setrusumab significantly reduced incidence of fractures in patients with the rare bone metabolism disorder osteogenesis imperfecta with at least six months of follow-up and continues to demonstrate ongoing and meaningful improvements in lumbar spine bone mineral density.

Osteogenesis imperfecta (OI) includes a group of genetic disorders impacting bone metabolism. Approximately 85 percent to 90 percent of OI cases are caused by mutations in the COL1A1 or COL1A2 genes, leading to either reduced or abnormal collagen and changes in bone metabolism. The collagen mutations in OI can result in increased bone brittleness, which contributes to a high rate of fractures. Patients with OI also exhibit inadequate production of new bone, which leads to decreased bone mass, bone fragility and weakness. OI can also lead to bone deformities, abnormal spine curvature, pain, decreased mobility, and short stature. No treatments are approved for OI.

Setrusumab (UX143) is a fully human monoclonal antibody that inhibits sclerostin, a negative regulator of bone formation. Ultragenyx and Mereo are collaborating on the development of setrusumab globally based on the collaboration and license agreement between the parties. The companies have developed a comprehensive late-stage program to continue development of setrusumab in pediatric and young adult patients across OI sub-types 1, 3 and 4.

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