6 Questions With Rocket Pharma's CEO On World Health Day: How The Biotech Is 'Changing The Narrative'

Rocket Pharma RCKT CEO Gaurav Shah is on a mission to "change the narrative" when it comes to rare diseases.

"We are enthusiastic about the potential of [our] strategy to hopefully bring much-needed therapies to patients that wouldn’t otherwise have them," he tells Benzinga.

Read on for the full interview in which Shah discusses Rocket's stock movement, the lessons he learned from his previous roles at Eli Lilly And Co LLY and Novartis AG NVS, whether future M&A plans are in the works and why World Health Day, which started in 1950 and is commemorated on April 7, is still a noteworthy event.

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BZ: Morgan Stanley predicts a target share price of $45, implying Rocket's stock will rally 150% (it closed April 6 at $16.91 per share). Does that create unneeded pressure in an otherwise slow-moving field that takes time and intense R&D?

Shah: All of our clinical programs have demonstrated early and sustained safety and efficacy — an achievement that is unprecedented for a gene therapy company. Despite this progress, there is a general consensus that Rocket is currently undervalued by the market.

It’s true that drug development is a relatively slow-moving and capital-intensive endeavor. Yet by having a deep understanding of the underlying disease biology of our target indications at a fundamental level, we can optimize both our time and our capital more effectively. Ultimately, our focus remains squarely on the patients who we hope will one day benefit from our therapies. They are our North Star.

After earning an M.D. from Columbia, you held leadership roles at Eli Lilly and Novartis. What lessons did you take with you to Rocket?

One of my biggest takeaways from years working in big pharma is recognizing the importance of listening to the U.S. Food and Drug Administration (FDA) and never taking the breadth and depth of their experience for granted. During each step of developing our programs, we are truly collaborators for the sake of patients who don’t have the luxury of time.

In terms of mindset, a philosophy I maintain is to be agile, nimble and humble. These were foundational to us back when we started Rocket in the back conference room at a hedge fund and remain so to this day as a company of more than 250 associates.

Our team understands the critical importance of CMC (chemistry, manufacturing and controls) in drug development. It is the most important thing to get right before starting a clinical trial and we are constantly striving to ensure that our CMC processes are of the highest quality.

Lastly, building and advancing a workplace culture of creativity and innovation that empowers our employees is one of our most important priorities. We invest in people — not in the job. Diverse perspectives and instincts across all levels at our company allows us to move nimbly and smartly, and we are constantly seeking to build and maintain a team of exceptional individuals.

Why is "World Health Day" so important?

For us at Rocket, World Health Day and especially this year’s theme of “Health For All” is an important reminder that more than 300 million people across the globe live with rare disease, but only 5% of these conditions have a FDA approved treatment option. Our sole mission is to close this gap by advancing a pipeline of gene therapy assets applied to a host of diseases with genetic causes.

This week's job report indicates a cooling labor market. Is Rocket hiring?

From our view, we have never been more excited about leading the future of gene therapy based on our progress to-date across our programs and expect to add more than 100 employees to our exceptionally talented team this year of more than 250 experts who have joined since our founding — all passionate about seeking gene therapy cures for patients with unmet needs. So, in short, yes, we are hiring and building on our positive results across all four clinical programs.

What's next for Rocket Pharma?

We have several key objectives on the horizon, including the submission of two product filings, the initiation of a pivotal study for Danon Disease — which causes an enlarged heart — and the submission of an IND (Investigational New Drug Application) for the first of our "Wave 2" programs. Our strategy remains centered on building value and positioning ourselves to navigate a frequently challenging market. We are always on the lookout for opportunities to bring in non-dilutive forms of capital and as any good steward of a publicly traded company, we would be opportunistic to bring in non-diluted forms of capital.

Any acquisition plans?

With regards to M&A, last year we purchased Renovacor, a gene therapy company with an early stage therapeutic for dilated cardiomyopathy. We remain open to exploring additional strategic opportunities that may arise in the future.

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Image courtesy of Rocket Pharma.