Catalyst Pharmaceuticals has completed the acquisition of an exclusive license for North America for vamorolone, a potential treatment for Duchenne Muscular Dystrophy (DMD), from Santhera Pharmaceuticals. Vamorolone is a promising anti-inflammatory steroid treatment for DMD, showing efficacy with reduced side effects and benefits for patients. It has received FDA Orphan Drug and Fast Track designations. Catalyst plans to launch vamorolone in early 2024 and aims to incorporate it into its Catalyst Pathways® specialty pharmacy program to provide patient access. The transaction includes an all-cash purchase payment and a strategic equity investment in Santhera.
Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) (Nasdaq: CPRX), today announced the completion of its acquisition from Santhera Pharmaceuticals Holdings (“Santhera”) of an exclusive license for North America for vamorolone, a potential treatment for patients suffering with Duchenne Muscular Dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico, as well as the right of first negotiation in Europe and Japan should Santhera pursue partnership opportunities. Additionally, Catalyst will hold North American rights for any future approved indications of vamorolone.
Vamorolone is a promising best-in-class dissociative anti-inflammatory steroid treatment for Duchenne Muscular Dystrophy (“DMD”). In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behavior, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA action date of October 26, 2023.
“With the addition of vamorolone, we have accomplished another important milestone in the execution of our portfolio expansion strategy,” stated Patrick J. McEnany, Chairman and CEO of Catalyst. “The license for vamorolone reflects our strategic focus on opportunities where we can not only apply novel technology to address critical unmet patient needs, but where we can leverage Catalyst’s existing integrated capabilities and infrastructure to commercialize the drug efficiently. We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients.”
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Mr. McEnany continued, “Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions.”
Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a rare, fatal neuromuscular disorder characterized by progressive muscular dysfunction, leading to loss of ambulation, respiratory failure, and death. Corticosteroids are the current standard of care for treating DMD; however, this treatment is associated with significant side effect burdens. The U.S. prevalence for DMD is estimated to be between 11,000 and 13,000 patients. Of patients currently being treated for DMD, approximately 75% receive concomitant steroid treatment.
Transaction Details
As previously disclosed, Catalyst has made an all-cash purchase payment of $75 million to acquire the license. Simultaneously, Catalyst has made a strategic equity investment into Santhera by acquiring 1,414,688 of Santhera’s post reverse-split ordinary shares (representing approximately 11.26% of Santhera’s outstanding ordinary shares following the transaction) at an investment price of CHF 9.477 (corresponding to a mutually agreed volume-weighted average price prior to signing), with the approximately $15 million USD in equity investment proceeds to be used by Santhera for Phase IV studies in DMD and further development of additional indications for vamorolone. Catalyst will also be obligated under certain circumstances to make milestone payments and to pay royalties to Santhera.
Vamorolone Commercial Operational Plan
- Catalyst expects to launch vamorolone early in the first quarter of 2024, assuming regulatory approval on the PDUFA date of October 26, 2023.
- Catalyst anticipates minimal sales and marketing personnel expansion with fewer than 10 additional team members required, resulting from the exceptional synergy within its existing neuromuscular franchise.
- Catalyst plans to incorporate vamorolone for DMD into its Catalyst Pathways® specialty pharmacy program to ensure that patients have access to full patient benefits.
Further details on the vamorolone commercial operational plan will be discussed during the Company’s second-quarter 2023 financial results conference call, which is expected to be held in the second week of August. Catalyst also expects to discuss on a preliminary basis the anticipated accounting treatment for the transaction on the conference call.
Source: BioSpace
