I’ve found another recent orphan drug deal which may be of...

I’ve found another recent orphan drug deal which may be of interest. While this licensing deal struck last month doesn’t concern a Phase 3 ready drug (ezutromid is still at early-stage Proof of Concept Phase 2), it does have some financial details provided. Interestingly, as in the case of this month's Grunenthal/Thar deal, this is a deal in which a larger biopharma has paid to gain control of a rival orphan disease asset at an earlier stage of development.

The licensee in last month's deal is US-based Sarepta Therapeutics, which has been previously mentioned on this forum with respect to its battle for FDA approval for eteplirsen in the treatment of Duchenne Muscular Dystrophy (DMD). The licensor is Summit Therapeutics which is headquartered in the UK and has been listed on the NASDAQ since mid-last year.

This deal provides Sarepta with exclusive licensing rights in Europe, as well as in Turkey and the CIS states (Azerbaijan, Armenia, Belarus, Georgia, Kazakhstan, Kyrgyzstan, Moldova, Russia, Tajikistan, Turkmenistan, Uzbekistan and Ukraine). The rights are to Summit’s pipeline of utrophin modulators, including its lead clinical candidate, ezutromid, which has commenced a Phase 2 POC clinical study. As part of the agreement, Sarepta has also obtained an option to license Latin American rights to Summit’s utrophin modulator pipeline. Summit Therapeutics has retained commercialisation rights in all other countries.

The headline value of the deal is US$522 million (AU$711 million). Summit will receive an upfront fee of $40 million and a further $22 million milestone upon the first dosing of the last patient of its current Phase 2 trial. In addition, Summit will be eligible for future ezutromid related development, regulatory and sales milestone payments and escalating royalties ranging from a low to high teens percentage of net sales in the licensed territory.

Summit will also be eligible to receive development and regulatory milestones related to its other nextgeneration utrophin modulators. Sarepta and Summit will share specified utrophin modulator-related research and development costs at a 45%/55% split, respectively, beginning in 2018. If Sarepta elects to exercise its option for Latin American rights, Summit would be entitled to additional fees, milestones and royalties.

Summit’s current Phase 2 study of ezutromid in 40 boys with DMD includes a 48-week open label treatment phase with a 30-day safety follow up. The trial is scheduled for completion in December 2017.
NASDAQ-listed shares in Summit Therapeutics jumped 66% (to a MC US$177 m/AU$240m) on the news of the licensing deal. It has since retreated to MC US$130m/AU$177m).


http://g923gvc6go14zuzz1vaotb19.wpe...tromid-License-and-Collaboration-FINAL-UK.pdf

https://clinicaltrials.gov/ct2/show/NCT02858362