7 Lessons from the World Orphan Drug Congress


Gene therapy is here, VCs want to invest in orphan drugs, Rettsyndrome.org foundation is doing a great work, the line between patients and drug developers is blurring, we should start talking about industry (not patient) engagement, networking is not always easy, and Fulcrum Therapeutics should be in your list of companies to watch.  

That’s my 17 seconds version of the main lessons I got from attending the World Orphan Drug Congress in DC April 20-21. The WODC one of the largest meetings dedicated to the development of new medicines for rare diseases and takes place once in the US and once in Europe every year.

In a bit more than 17 seconds, here is the expanded list of what I would like to share with you from the conference:

1- Gene therapy is not a thing of the future, it is already here. I particularly enjoyed the presentations from Bluebird Bio and Spark Therapeutics. Nick Leschly from Bluebird Bio gave us excellent advice for building a rare disease company. My favorite: (1) if you have seen one company do it, you have seen one company do it (=every case is unique); (2) the power of n=1; (3) if you see great improvement for one patient you have to fight for that drug; (4) getting pricing reimbursement and adoption are the real end goal, regulatory approval is just the start; and (5) keep constructive paranoia, don’t get in the comfort zone, do something that has a lasting effect in a human being. Bonus lesson: videos of patients before and after gene therapy are always amazing. No graph with 200 patients has the power of showing a video of a child that was previously blind navigate a circuit with obstacles one year later.

2- Developing drugs is an expensive business and investors need to be part of the game…and the conferences. Listening to Venture Capital partners at the Pitch and Partner track was extremely interesting. A panel shared their views on what they look for in the orphan space, the concept of orphan vs ultra-orphan, how they work with academic founders and partner with patients, and best advice when pitching to them. It is a great decision to give a voice to VCs in orphan drug conferences. Also, Philip Ross from JP Morgan should be the moderator for all conference panels, or at least be in charge of the questions.

3- Anavex’s CEO Christopher Missling talked about partnering with patient organizations and explained that a year ago they didn’t even know what Rett syndrome was. He credits Rettsyndrome.org CSO Steven Kaminsky for making it very easy for them to test their most advanced drug in a Rett mouse model and for mobilizing resources to help them start a clinical trial when those results where very positive. They plan to start a Phase 2 trial this year. Rettsyndrome.org is a great example of how patient organizations can attract companies to their disease by proactively engaging the industry and understanding how to remove drug development bottlenecks, such as offering access to good animal models. 

4- Times are changing. I met several parents of children with rare diseases that attended the WODC that have created their own companies or that are considering starting one. The line between patients and drug developers is blurring. There are more and more patients and parents that have turned drug developers, from the more senior John Crowley from Amicus to companies that are just starting. This trend is likely to make the biggest impact for ultra rare diseases.

5- I’m thinking we should start the hashtag #IndustryEngagement. I spent the last 5 years working with patient organizations trying to get the industry interested in our diseases. Our research strategy was to make it so easy for anyone to work on Dravet syndrome that we would have 300 labs and companies working on it tomorrow. That’s why Dr Kaminsky from Rettsyndrome.org is one of my heroes. And when I sit down with other patient organizations, as I did during the WODC, our conversation is always about best practices to engage the industry. We hear so much about patient engagement from industry speakers that I am not sure they know that the reverse conversation is also taking place under the same roof. I pretty much wrote an entire book about it. I’m thinking we might need to come up with a proper name for that reverse patient engagement. Let me know if you have other ideas about how to call it.

6- The WODC is multiple conferences in one. The conference places a big emphasis in networking, but there are so many vendors in attendance (CROs and similar) that the speed networking sessions and most networking opportunities where not useful for somebody like me. I’m not useful for them either, since I don’t use their services. All of my useful contacts but one happen by going through the attendees list and pre-programing meetings ahead of the conference. So here is my recommendation: maybe we could use different color badges for the different sectors. That way I will go straight to drug developers and patient groups and not struggle to find them in a sea of service providers.

7- Somehow I hadn’t noticed Fulcrum Therapeutics before. Or at least I hadn’t noticed how cool what they are doing is. Co-founder Walter Kowtoniuk gave an amazing presentation at the WODC that not only got me to fall in love with their approach but also with his passion. Fulcrum is doing small molecule regulation of protein levels through gene regulation. So many diseases are caused by having one defective gene copy and therefore another one to upregulate (or a copy to reduce, in case of gain of function or toxic function) that the sky is the limit. And they clearly know it, seeing their aggressive expansion and approach. I need to get them to talk with me about rare epilepsy syndromes!

 And these are my main lessons from the WODC. I hope you also found them interesting.

This is the first time I attended the USA congress after having attended some of the European ones, and Terrapin always does a great work at putting together such a large and varied congress (see lesson #7). I look forward to the one in Barcelona in November.

If you attended the WODC and have more lessons to share leave a comment or reach out in Twitter.

Ana Mingorance PhD