Biotechs turn to successful biotechs in rare diseases

March 13, 2014 ctheodoropulos

The panel “Dealmaking Strategies Around Orphan Diseases” at BIO-Europe Spring® in Turin delivered insights to a highly specialized area where quick and and innovative biotechs have found a clear advantage over big pharmaceutical companies.

“We have seen biotechs successful where big pharma has not been,” noted session moderator Mike Ward, who is the Global Director of Content for Datamonitor Healthcare & SCRIP Intelligence at Informa. Are bigger biotechs who have succeeded in the field becoming partners of choice? he asked.

“The good news is there are more people to talk to today,” replied Luc Dochez, Chief Business Officer, Prosensa.

James Weissman at Dicerna Pharmaceuticals finds it great that “small, specialized companies have grown to the scale and size where they are legitimate credible partners for smaller companies to consider.”

But can these new players punch at the same weight as pharma? asked Ward.

Absolutely, said Jeremy Springhorn with Alexion Pharmaceuticals, who gave a sharper point to the strategic shift in partnering, saying, “If a seller just wants cash then they should go to pharma. If they want to develop their therapy, then come to a company that knows how to develop in this area and does it well.”

Dochez agreed, saying that if a company is looking to enter the orphan drug space because development of a product will be cheaper and easier to get approved, then they are pursuing a poorly founded strategy. Patient stories that touch and inspire are what push companies forward, as well as potential sources of financing.

Ultimately Alexion was able to go it alone due to the powerful efficacy of its drug with patients, said Springhorn. In contrast to this, most opportunities in the rare disease space are single enzymes for single defects and single pathways and do not support the kind of capital expenditure needed to build the infrastructure needed to go it alone, he said, suggesting that here companies will need a partner with the experience or capabilities to get it through.

Weissman provided a framework for companies in a sell mode seeking a partner. What is the probability of technical success, can you hit the targets? he asked. Then consider the probability of regulatory success. Hitting the technical targets is not any guarantee that the product will be approved. Next look hard at the probability for a level of commercial success that will reward investors.  Finally, what is the real need for a partner? If financing is sufficient and a company can afford to be choosy, a partner needs to bring something very special to the table, he said, to help hit other objectives for  technical, regulatory or commercial success.

In the final analysis, he said, if the therapy helps patients, entering the rare disease space is the right decision.

Springhorn agreed, saying what will pull a company through the years of development, the hurdles and setbacks, is the drug.  “If it works extremely well, it will cure the mistakes you will make, the burns from landmines you step on,” he said.



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