Santhera shores up funding ahead of vamorolone verdict

Swiss biotech Santhera has raised CHF 22 million ($23.5 million) in bridge financing that it says will allow it to fund its operations through to October, when the FDA is due to deliver a decision on its review of Duchenne muscular dystrophy drug vamorolone.

Existing investor Highbridge Capital is providing the new funding, which includes a purchase of 3 million shares in Santhera for CHF 22 million.

Santhera completed its rolling license application for vamorolone in December, which was accepted by the FDA in January, kicking off a review that could lead to the launch of the drug in the US in the fourth quarter.

Vamorolone is also under review in the EU after being filed with the EMA last October, so approval decisions are expected towards the end of the year in both the US and Europe, and the company has said it wants to launch immediately after approval.

Santhera had sought a priority review of vamorolone, but in the end the FDA opted for a standard, 10-month review, although it will not require an advisory committee meeting on the drug.

At the same time, the Swiss biotech has launched a strategic review of its business to gauge the potential of vamorolone in additional indications – such as asthma, inflammatory bowel disease, rheumatoid arthritis, and multiple sclerosis – and possibly licensing out the drug to a commercial partner.

Also potentially up for partnering deals are Santhera’s two other therapies, lonodelestat for cystic fibrosis and Leber’s hereditary optic neuropathy (LHON) therapy Raxone (idebenone).

Dario Eklund, Santhera’s chief executive, said the new funding should provide “liquidity” through to the FDA decision.

“Discussions are ongoing with various parties on potential opportunities to access the resources required to ensure an effective roll-out of vamorolone once approval has been secured,” he added. “We will look at these opportunities with our board of directors in the coming weeks and months.”

Vamorolone was licensed from US biotech ReveraGen BioPharma in 2020, after Santhera abandoned development of idebenone for DMD. If it does get approved, it will mark a turnaround in fortunes for Santhera, which was forced to slash staff after idebenone failed a phase 3 trial in DMD in 2020.

In the phase 2b VISION-DMD study, vamorolone had positive effects on mobility measures like the six-minute walk (6MWT) test and time to stand (TTSTAND) velocity compared to placebo at 24 weeks, with the benefits maintained out to 48 weeks, in DMD patients aged four to seven.

Trials of the drug are ongoing in younger DMD patients aged two to four, as well as the seven to 18 age bracket, and also in patients with Becker muscular dystrophy (BMD), which is less common than DMD and tends to cause milder symptoms.