June 14, 2024 8:45am

RARE closed down -$1.52 at $43.12 with a -$1.94 or -4.50% to $41.18

The offering is expected to close on or about 6/17/24, subject to satisfaction of customary closing conditions. 

J.P. Morgan, Goldman Sachs & Co. LLC, BofA Securities, and TD Cowen are acting as joint book-running managers for the offering.

 

RARE is offering pre-funded warrants to purchase 1,538,501 shares of its common stock at a purchase price of $38.999 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.

The aggregate gross proceeds to the company from this offering is expected to be $350 million, before deducting underwriting discounts and commissions and other offering expenses, and excluding the exercise of any pre-funded warrants.

In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1,346,153 shares of the company's common stock at the public offering price, less the underwriting discount.

Why the offering …

On June 12, RARE held a successful meeting with the U.S. FDA during which the company reached agreement with the Agency that cerebral spinal fluid (CSF) heparan sulfate (HS) is a reasonable surrogate endpoint that could support submission of a biologics license application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy for the treatment of Sanfilippo syndrome (MPS IIIA). The company will need to finalize details of its BLA with the Agency in a pre-BLA meeting with the intent to file late this year or early next.

However, cost of not settling …

I have an additional question … faces lawsuit over “Alleged Unjust Use of Henrietta Lacks' Cells”

  • The estate of Henrietta Lacks can proceed with a lawsuit against RARE over the use of cells taken from Lacks’ body in the 1950s, as ruled by a Maryland federal court on Monday.
  • U.S. District Judge Deborah Boardman rejected Ultragenyx’s motion to dismiss the case, determining that the estate had plausibly claimed that Ultragenyx wrongly profited from its research using the “immortal” HeLa cell line.
  • Previously, the Lacks estate had filed a lawsuit against Thermo Fisher Scientific Inc. (NYSE: TMO) for the alleged misuse of the HeLa line, which resulted in a settlement last year.
  • The current lawsuit against Ultragenyx was initiated days after the Thermo Fisher settlement. The estate accused Ultragenyx of using HeLa cells “like a dairy farm treats cows” to mass-produce materials for gene therapy.
  • RARE defended its position by emphasizing the significant impact of HeLa cells on medical research but argued that the estate had targeted the wrong defendant under an invalid legal theory in pursuit of non-existent “huge profits.” However, Boardman denied Ultragenyx’s motion to dismiss, stating that the estate’s case was robust enough to continue.