XOMA Acquires Royalty & Milestone Economics to Phase 3 First-In-Class Orphan Disease Asset for Niemann-Pick Disease Type C (NPC) & Phase 2 Oncology Asset


XOMA Corporation recently announced it has acquired the royalty and milestone rights associated with two assets from LadRx Corporation: arimoclomol, an oral therapeutic for Niemann-Pick disease type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.

Arimoclomol is a first-in-class investigational therapy that has completed a randomized, placebo-controlled, multinational Phase 2/3 program in Niemann-Pick Disease Type C, an ultra-rare, genetic, progressive neurogenerative disorder.  The compound has received Orphan Drug Designation for the treatment of NPC by the Regulatory Authorities in the US and EU. Additionally, the US FDA has granted the compound Fast-Track Designation, Breakthrough Therapy Designation and Rare Pediatric Disease Designation.  Zevra Therapeutics has announced its intention to file a New Drug Application for arimoclomol as early as the third quarter of 2023.

The second asset is aldoxorubicin, a Phase 2 program that is being developed by ImmunityBio as a potential therapy for pancreatic cancer.  The FDA has granted aldoxorubicin Orphan Drug Designation for the treatment of soft tissue sarcoma.

“This is a notable acquisition of the economics associated with a therapy that has the potential to address a significant unmet need in a devastating disease,” said Owen Hughes, Executive Chairman of XOMA.  “This transaction is very much aligned with our strategy of seeking assets that have the potential to establish new standards of care for patients while driving cashflow generation for XOMA shareholders.”

“NPC is an ultra-rare genetic disorder where patients can lose their vision and hearing, their ability to walk and swallow, and it leads to premature death.  In the US, there are no approved treatments for NPC. Zevra is actively preparing to file a New Drug Application, as these patients clearly need access to therapy as evidenced by over 150 patients receiving arimoclomol therapy through expanded access,” added Brad Sitko, Chief Investment Officer at XOMA. “We provided a non-dilutive capital solution to LadRx, which had been exploring strategic alternatives.  LadRx can now advance an internal pipeline of new therapeutics to treat patients with high unmet needs.”

Under the terms of the agreement, XOMA will receive a mid-single digit royalty on arimoclomol’s commercial sales upon approval and up to $52.6 million, net, in potential milestone payments. Should aldoxorubicin be approved for marketing, XOMA will receive a mid-single-digit to mid-teens royalty rate on aldoxorubicin commercial sales depending upon the indication, in addition to potential milestone payments of up to $343 million in development and commercial milestones. LadRx will be entitled to receive up to $6 million in certain pre-specified milestones associated with arimoclomol and aldoxorubicin. XOMA acquired these royalty and milestone interests for $5 million.

XOMA was represented by Gibson Dunn & Crutcher LLP.  LadRx was represented by Roth Capital Partners and Haynes and Boone LLP.

XOMA is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The company has an extensive and growing portfolio with more than 70 assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information, visit www.xoma.com.