How does Insmed integrate in the field of rare diseases and what is the general vision for the company's contribution to the industry?
In ten years time, Insmed has grown from about 40 to more than 700 employees, becoming Science magazine’s number one top employer two years in a row—an achievement only previously earned by Regeneron and Genentech. However, this external recognition pales in comparison to the gratification we feel when we see how our medicines impact patients. Today, we have four different programs that we are developing to potentially address unmet needs in serious and rare diseases. When we entered the field, we focused on a rare pulmonary disease that at the time had no approved treatment; we were able to successfully deliver the first approved therapy in the U.S., Europe and Japan. We hope to follow a similar trajectory for diseases like bronchiectasis and pulmonary hypertension, where there is either no approved treatment or the current treatment landscape is inadequate. We are also advancing a collection of platform technologies that includes gene therapy and AI-driven protein engineering.
The industry finds itself at an inflection point in 2023; what are the main reasons behind this evolution?
This inflection is the result of an interesting confluence of several factors. It all started with new technologies that have been brought to fruition and that are looking at diseases in a fundamentally different way. Tangible change is happening and with the abundance of capital that flowed into the industry in the last couple of years, we are going to witness a lot of innovation in the years to come—whether that’s driving forward newer technologies like gene therapy and artificial intelligence or harnessing the full potential of existing modalities to address diseases that still have significant unmet needs. The silver lining of the pandemic has been that it worked as an amazing catalyst to all these advancements, and we are sure that even more exciting times are coming ahead.
The FDA recently started speed tracking rare disease approvals; what is the relationship like from Insmed’s perspective?
Rare disease innovation has been the success story of the last two or three decades since the Orphan Drug Act aligned the FDA with the companies that were trying to develop therapies in this space.
As just one example, Insmed worked with the FDA to design the development program and secure conditional approval of our first therapy, and we continue to work with the agency on ongoing clinical development.
The need for new therapies for rare diseases is still significant; there are more than 8,000 rare diseases, many of which have no treatment or inadequate treatment. In the next 18 months, data from all four of our programs will be read out and, pending results, we hope to have meaningful clinical data that can potentially change the course of life-threatening diseases.
How do you reconcile the power of the industry with the negative perception the general public tends to have regarding pharma companies in the US?
This paradox is actually one of the greatest challenges of our time and it is our responsibility, as industry participants, to shine a light on all the good that is being done in this space. For many of us, the motivation to deliver new drugs that transform patients' lives is deeply personal and driven by a desire to reduce suffering. As an industry, we must redouble our efforts to make a positive contribution to patients and families. When we are developing a drug at Insmed, we have something I call the "holiday test." We imagine we are sitting around the holiday table when one family member tells us they have a serious disease. If Insmed makes a treatment for that disease, can we feel good telling our own family member, “I have something that can make a meaningful difference for you”? If the answer is yes, we know we are on the right path.
What are the main ambitions that Insmed has for the next five years?
As we grow our footprint, the most important thing is to preserve our culture. In order to achieve great things in biotech it is vital to have a cohesive team driven by the same ambitious mission. And in the years to come, we are going to pursue exactly that vision to significantly impact patients' lives.
