Santhera Pharmaceuticals has signed an exclusive license and collaboration agreement for vamorolone in North America (NA) with Catalyst Pharmaceuticals, a commercial-stage biopharmaceutical company focused on novel medicines for patients living with rare diseases.
Total consideration to Santhera is up to USD $231 million (including equity investment), plus royalty payments from product sales, with near-term cash proceeds for Santhera of USD 126 million.
Under the terms of the agreement, Santhera will grant Catalyst exclusive commercialisation rights to vamorolone in North America, comprising the US, Canada and Mexico. At closing, Santhera will receive an upfront cash payment of USD $75 million.
In addition, Catalyst will make an equity investment of USD $15 million through the purchase of 14,146,882 treasury shares at a price of CHF 0.9477 per share which corresponds to the ten-day volume-weighted average price ending two days prior to signing of the agreement, subject to a six-month post-closing lock-up and standstill.
Use of proceeds from the equity investment will support Phase 4 studies in DMD and joint development of additional indications. Upon US FDA approval of vamorolone in DMD, a decision expected on October 26, 2023 (PDUFA date), Santhera would receive an additional USD 36 million from Catalyst, of which Santhera would pay contractually agreed third-party regulatory milestone obligations (USD $26 million).
Furthermore, Catalyst may pay Santhera sales-based milestones of up to USD $105 million as well as up to low-teen percentage royalties and will assume corresponding third-party royalty obligations of Santhera on vamorolone sales in all indications in NA.
Santhera CEO Dario Eklund said: “Duchenne muscular dystrophy is a devastating condition. Everyone at Santhera has been working diligently to advance vamorolone in order to make this important treatment option available to patients.”
Eklund continued: “We believe Catalyst is well placed to maximise the value of vamorolone and, subject to regulatory approval, ensure patients in North America receive this transformational therapy as quickly as possible. 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.”
In Europe, Santhera plans to commercialise vamorolone in key geographies (including Germany, France, UK, Italy, Spain, Benelux), and will seek partners for commercialisation in all other countries. Market access, the build-up of a core organisation and stakeholder engagement activities in these priority countries are ongoing.
Already ahead of the European approval decisions expected at year-end, the early access programs submitted in the UK and France could allow treatment of first DMD patients with vamorolone. For certain countries, where Santhera chooses not to market vamorolone directly, including certain European countries and Japan, it has granted Catalyst a right of first negotiation in partnering discussions.
The new drug application for vamorolone in DMD is under review in the US by FDA, which has set October 26, 2023 as the Prescription Drug User Fee Act date for its regulatory decision on approval. In Europe, the review of the marketing authorisation application (MAA) for vamorolone by the European Medicines Agency is ongoing.
An opinion from the Committee for Medicinal Products for Human Use is expected in Q3-2023, followed by an approval decision by the European Commission in late 2023. In the UK, a corresponding MAA is under review by the Medicines and Healthcare products Regulatory Agency. Subject to approvals, vamorolone could be launched in both the first EU countries and the US in late 2023 and early in the first quarter 2024, respectively.
For indications in addition to DMD, Santhera and Catalyst will establish a joint steering committee to undertake the joint clinical development of vamorolone for global indications, in which both parties would participate in the development process and funding.
The closing of the transaction is expected to occur early in the third quarter 2023, subject to customary conditions and regulatory clearances in the United States.
