Catalyst acquires North American rights to DMD therapy vamorolone

Developer Santhera Pharmaceuticals to net $75M, plus investment, in deal

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review in the U.S. and Europe for Duchenne muscular dystrophy (DMD).

Under the deal, Catalyst will give vamorolone’s developer Santhera Pharmaceuticals an upfront cash payment of $75 million, plus a $15 million investment in the company. Santhera also is entitled to further payments if vamorolone is approved and sold.

“We are extremely pleased to be entering into a partnership with Santhera on the vamorolone program and the prospect of delivering a potential therapy with a desirable profile for DMD patients,” Patrick McEnany, Catalyst’s chairman and CEO, said in a company press release.

“Everyone at Santhera has been working diligently to advance vamorolone in order to make this important treatment option available to patients. We believe Catalyst is well placed to maximize the value of vamorolone and, subject to regulatory approval, ensure patients in North America receive this transformational therapy as quickly as possible,” Dario Eklund, CEO of Santhera, said in a separate press release .

Corticosteroids are a class of medications that exert powerful anti-inflammatory effects by mimicking the activity of the hormone cortisol. Corticosteroid treatment is a part of standard care for DMD as it can help reduce inflammation around muscle tissue and slow muscle damage.

Corticosteroids can cause a range of side effects, however, including high blood pressure, mood changes, fluid retention, and weight gain. These can be difficult for patients to manage, especially in conditions like DMD where they’re given long-term.

Vamorolone is a so-called dissociative corticosteroid, meaning it remains effective, but doesn’t have the side effects other corticosteroids do.

“Vamorolone has the potential to be a differentiated treatment for DMD with a desirable profile in comparison to the current standard of care options, addressing an important unmet medical need for Duchenne patients starting at an early age,” McEnany said.

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A Phase 2b clinical trial dubbed VISION-DMD (NCT03439670) tested vamorolone against a conventional corticosteroid (prednisone) and a placebo in 121 boys with DMD. Both vamorolone and prednisone outperformed a placebo on measures of motor function. The corticosteroids’ effectiveness was comparable, but fewer side effects were reported with vamorolone.

The findings formed the basis of Santhera’s applications for vamorolone as a DMD treatment in the U.S., the U.K., and in Europe. A decision in the U.S. is expected by late October. In Europe, it’s expected before the end of the year.

With the new agreement in place, and assuming vamorolone is approved, Catalyst says it’s planning a commercial launch in the U.S. in early 2024. McEnany said the company, “will be well-positioned to capitalize on the synergies of our expanded and exceptional capabilities in preparation for the launch of vamorolone.”

“Catalyst has a track record of success in the rare disease and neuromuscular space and is our commercial partner of choice for North America with an established infrastructure and commitment to patients,” Eklund said.

Funds from the new agreement will help support vamorolone’s clinical development in other indications as well as the commercial rollout in Europe, assuming it’s approved, according to Santhera. The company says early access programs planned for the U.K. and France could allow for treatment with vamorolone.

Santhera is planning to commercialize the therapy in certain European markets, including Germany, France, the U.K., Italy, Spain, and Benelux (Belgium, the Netherlands and Luxembourg). It will partner with other companies to market it elsewhere. Santhera has granted Catalyst the right to first negotiation for several markets where the company isn’t planning to market vamorolone itself, including in Europe and Japan.

“With our new partner, we also look forward to jointly address the benefits of vamorolone in additional indications,” Eklund said. “As part of this agreement, Santhera benefits from upfront, milestone, and royalty payments, which allows us to focus on the commercial rollout of vamorolone in DMD and future other indications in Europe.”