I recently had the pleasure to conduct a podcast conversation with Bradley Campbell, who is the President and Chief Operating Officer of Amicus Therapeutics (listen to the podcast here). Bradley recounted the interesting history of Amicus’ development of Galafold™ (migalastat), which is an important and unique therapy – an oral precision medicine for patients with Fabry disease, a rare genetic lysosomal storage disease that causes a wide range of systemic, debilitating symptoms. Galafold was recently approved and launched in the EU for Fabry patients who have amenable mutations, where it is benefitting many Fabry patients, and is in late-stage development to support a regulatory approval submission to the U.S. FDA. The Company estimates that 35% to 50% of Fabry patients globally have amenable mutations that are suitable for this precision medicine approach. In describing the business and clinical development history of migalastat, Bradley talked about the perseverance and persistence that Amicus demonstrated by not giving up when faced with setbacks and by keeping its focus on the mission of maximizing the number of patients with the right genotype who will ultimately benefit from the drug. As a source of perspective and inspiration, Bradley shared a quote that inspired him from a manager he had early in his career, who, when faced with adversity and when reflecting on the challenges of the life science industry, often said, “life is long, don’t give up.”
Amicus has been exemplary in not giving up. Galafold is the only biotech product I am aware of that has been partnered twice, returned twice, and then brought to commercial approval by the originator. In 2007, Amicus licensed the ex-US rights of Galafold to Shire for $50M up-front, $390M in potential milestones and a joint development agreement where the development costs for Galafold and two additional compounds were shared 50/50 1. Then, in October of 2009, Shire and Amicus mutually agreed to terminate the collaboration and Amicus reacquired all global development and commercialization rights. This occurred after Amicus and Shire mutually concluded that Plicera™, an additional compound included in the licensing agreement, would not move forward to Phase 3 trials, making global rights to Galafold “of critical importance to Amicus”2 .
Despite the first partnership being terminated, Amicus persevered with Galafold because the company believed in the drug’s potential to benefit the lives of Fabry patients. As such, Amicus continued the clinical development, and in October 2010, one year after Shire had terminated, Amicus partnered Galafold for a second time; this time with GSK for exclusive worldwide rights in exchange for $30M up-front payment, a $31M equity investment, $170M in potential milestones and jointly funded development costs3. In July 2012, the Amicus / GSK collaboration was amended and expanded. Despite re-structuring the original agreement, in November 2013 it was terminated altogether and the worldwide US rights to Amicus were returned 4 following the initial data readout from a Galafold Phase 3 trial.
Yet again, despite having a partnership agreement terminated, Amicus did not abandon its focus on developing Galafold and providing it to as many suitable patients as possible. Amicus pressed forward with the clinical and regulatory activities that led to Galafold’s registration for full approval in the European Union in May 2016. The company continues to pursue the necessary steps to get the drug approved in the US.
One important take-away from the Galafold experience for life science executives and business development professionals is that when a partnership gets terminated, if your company still believes in the drug, don’t give up. While a partnership termination can be a disappointment and have negative consequences on perception, stock price, financing, etc., we are aware of several development stage “re-partnering” deals in which a second partnering deal has been done after the initial deal was terminated, as shown in Table 1:
Table 1: Development Stage Re-Partnering Deals
Sources: AdisInsight Drug Profiles, GlobalData Deals Database, Company Press Releases
As shown in Table 1, there are numerous examples of development stage re-partnering deals. It is worthwhile to note that of the 18 re-partnering deals we identified, in 13 of those deals, the partnership has enabled a drug to advance in clinical development and 9 of those re-partnered 18 drugs have gone on to get approved. We applaud those companies who did not give up on their drugs when faced with a terminated partnership. As Bradley Campbell reminded us, “life is long…”
If you would like to discuss the transactional dynamics of re-partnering or any other aspects of life science business development and financing, please do not hesitate to reach out to me at email@example.com.
1 Shire Press Release, November 8, 2007 2 Amicus Press Release, October 29, 2009 3 GSK Press Release, October 29, 2010 4 Amicus Press Release, November 20, 2013