Interview: Challenges of partnering in the rare disease industry

In Partnering & Investment by Matt TurnerLeave a Comment

rare disease, partnering, endocyte, merck, orphan drugsWith the interest to treat rare diseases growing and the commercial opportunities increasingly abundant for orphan drug developers, both large pharma, innovative biotech and patient groups alike are  tackling the strategic questions of whether to partner, who to partner with and how best to initiate such alliances.

Is partnering with a large pharma the best option for a biotech to commercialise their orphan product? What alternative sources of funding are available? What happens when a rare disease biotech acquires another rare disease biotech?

Endocyte, a biopharmaceutical company developing targeted therapies for the treatment of cancer and inflammatory diseases, recently entered an agreement to develop and commercialise their novel investigational therapeutic candidate vintafolide (EC145) with Merck. See official press release here.

Ron Ellis, CEO of Endocyte stated that “Following a rigorous selection process we believe Merck represents the ideal strategic partner to achieve the full potential of vintafolide, accelerating our development in numerous cancers”. Ron will be leading a session at the World Orphan Drug Congress 2012 and sharing his fresh experiences of how to form a strategic alliance based on your needs and their expertise.

I was privileged  had the opportunity to speak with Ron this week and understand his perspectives on the industry going forward:

What are the major challenges of partnering and seeking external opportunities in the rare disease industry?

"Of course the a major challenge with partnering drugs for rare disease is the future market potential is often too small to be attractive for partners.  It often is not an area where potential partners have much expertise or experience."

Where do you foresee the main opportunities lying to achieve sustainable growth in the rare disease industry?

"I think it greatly depends on the drug's total potential. Developing a drug that only works in a rare disease category is very different than developing a drug that has application in rare disease, but other diseases as well. This is often the case with oncology drugs. The initial approval may be in a very small patient population, but the drug also may be active in larger populations."

What are the foreseeable game changers set to influence the operations of orphan drug development?

"In general the challenges of operations deal with clinical trial recruitment. Often times the regulator requirements for a large study make it impossible to conduct the study over a reasonable amount of time due to the small available patient population."

Regardless of the financial details of any acquisition/co-development deal, arguably the important point for the industry is at which stage are large pharma and investors willing to "go all in" with a promising therapy or pioneering biotech? How far along the clinical development process do candidates need to be in order for wallets to be opened? And what makes one potential deal standout amongst others for a particular pharma giant looking to replenish a portfolio? The complexity lies in the fact that investors are only financially committed to a "sure thing" but orphan product developers need the resources and importantly the experience of established pharma early on in order to commercialise and ensure their innovations reach patients in need.

With a diverse mix of perspectives confirmed to lead the Corporate Development & Partnerships track at the World Orphan Drug Congress, you will hear partnering case studies that have made the industry take notice and discover what can be expected on a practical level when seeking a partner, negotiating an alliance and whether the partnering decision was the right choice in hindsight.

To see who will be covering these burning issues and more, DOWNLOAD THE FREE BROCHURE HERE.

If you have any opinions on the above questions, comments are always welcome and if you see value in joining the live conversation at the World Orphan Drug Congress in Geneva on the 29th-30th November, learn about the registration process here.

Leave a Comment

Current ye@r *