Santhera Completes Divestment of Raxone Business to Chiesi Group

Rare Daily Staff

Santhera Pharmaceuticals said it sold its Raxone business worldwide to Chiesi Farmaceutici, a transaction that replaces the existing 2019 license agreement between the two companies.

Under the terms of the agreement, Chiesi acquired all assets and certain liabilities related to Raxone in all indications worldwide, including Raxone in Leber’s hereditary optic neuropathy (LHON), for which Chiesi already held exclusive license rights globally since 2019, except for North America and France.

“This agreement allows Chiesi to accelerate the regulatory and clinical development of Raxone at a global level,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases.

The deal simplifies the Raxone business significantly for both companies with Chiesi becoming the global brand owner while enabling Santhera to focus on the launch of its experimental Duchenne muscular dystrophy therapy vamorolone in Europe, subject to approval. In June, Santhera sold North American rights to vamorolone for up to $231 million including an equity investment to Catalyst Pharmaceuticals.

Under the terms of the agreement, Chiesi Group will assume the responsibility for the settlement agreed between Santhera and the French reimbursement authorities relating to Raxone in LHON amounting to $27.7 million (€25.3 million), reducing near-term financial obligations and strengthening Santhera’s balance sheet. Santhera said ending Raxone-related activities allows it to streamline business processes, thereby reducing operating costs and freeing up resources to be deployed for the European vamorolone launch and strategic projects.

Santhera is eligible to participate in a potential marketing approval of Raxone in LHON in the United States. through single-digit variable payments on net sales or milestone payments of up to $10 million. In the event that Chiesi chooses to pursue Raxone in non-ophthalmological indications, Santhera would be eligible for additional milestone payments in a similar order of magnitude and high single-digit variable payments on net sales.

LHON is a heritable genetic disease-causing vision loss and blindness. The disease presents predominantly in young, otherwise healthy adult males as rapid, painless loss of central vision, usually leading to permanent bilateral blindness within a few months of the onset of symptoms. About 95 percent of patients harbor one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in the complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy production, increased reactive oxygen species production and retinal ganglion cell dysfunction, which cause progressive loss of visual acuity and blindness.

Raxone, a synthetic short-chain benzoquinone and a cofactor for the enzyme NQO1, circumvents the complex I defect, reduces and scavenges reactive oxygen species production, restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity. Current data demonstrate that up to 50 percent of patients benefit from treatment and are protected from progression of visual acuity loss or experience a clinically relevant recovery of visual acuity.

“Securing this agreement is an important step for the business, as we sharpen our focus on the commercialization of vamorolone in Europe, subject to approval,” said Dario Eklund, CEO of Santhera.”

Photo: Giacomo Chiesi, head of Chiesi Global Rare Diseases