Drug Repurposing Seminar Elicits Lively Discussion, Demonstrates Interest in Strategy

Packed Byers Auditorium, UCSF Mission Bay

UCSF-CTSI Catalyst Awards Event Attracts Large Crowd of Academics and Industry professionals

What do Viagra, Botox and Morphine have in common? More than you think. All three drugs started off addressing a specific indication, but were eventually repurposed for other needs as new scientific findings surfaced. Viagra, initially studied for heart disease, is now marketed for erectile dysfunction; Botox, once a treatment for crossed-eyes and uncontrollable eye blinking, is widely known for its cosmetic uses as well as alleviating migraines. Finally, pharmaceutical company Daiichi Sankyo and Charleston is examining new indications for the age-old opioid Morphine, known for its high intensity pain relief. These are just three cases of repurposed drugs among countless others.

Drug repurposing, or the application of known drugs and compounds to new indications, saw a spike in popularity in the 2000s and is currently  reemerging as a big opportunity highlighted by renewed interest from the NIH National Center for Advancing Translation Sciences (NCATS), patient foundations, industry and investors. Since it is a challenging business model to pursue, the Clinical and Translational Science Institute (CTSI) at UC San Francisco, which runs the Catalyst Awards program, along with campus partners QB3, UCSF Innovation, Technology & Alliances (ITA), Entrepreneurship Center and event sponsor law firm Sheppard Mullin, organized leaders in therapeutics to discuss the issues and potential ways to repurpose drugs to successful clinical outcomes.

From left: Ken Horne, Cyril De Colle, Elin Hartrum, William P. Quinn, Brian G. Atwood. Moderator: Lorna Tanner

More than 120 UCSF faculty, postdocs, residents, students, industry professionals and Catalyst Awards program advisors filled up Byers Auditorium at UCSF Mission Bay on September 16th for a lively seminar and discussion on the opportunities and challenges in repurposing drugs for new indications. The panel, which included CEOs who have moved drug repurposing projects through to development, a venture capital investor, as well as regulatory and legal experts, offered diverse perspectives on the topic.

“Repurposing is seen by many, academic researchers in particular, as a ’short-cut’ to discovering novel drugs to new indications. This approach can offer many opportunities to rapidly progress from lab to patients. However, unless the drug is licensed and available for the precise required population, and dose range, there will be challenges,” said Cathy Tralau-Stewart, PhD, senior program manager in therapeutics for CTSI’s Catalyst program, which administered the seminar. 

The panelists kicked off the seminar by sharing the various challenges they have observed or experienced in this field. Some of the challenges mentioned, like going after multiple indications and pricing strategy, actually apply to any drug development program and fall under the ‘life cycle management’ of a drug, clarified William P. Quinn, MA, MBA, Founder & CEO, Bullet Biotechnology, formerly with Jazz Pharmaceuticals. The challenge strictly unique to drug repurposing, however, is that “you find an opportunity that someone else developed like an opportunity that stalled or was approved in another country for a narrow indication 30 years ago, and try to make sense of it for something else.”

Your best opportunity is to sell the innovator who holds the patent on the science – a good science story that really makes sense.

Elin Hartrum, JD, LLM

Gilead Sciences

Also on the topic of challenges, panelist Brian G. Atwood, MS, MBA, Managing Director, Versant Ventures mentioned the difficulties of the systemization of this process, “It’s like saying how do you make serendipity happen? The interesting part about drug repurposing is thinking about how to use everything at our disposal to create insight. To know that a particular drug could be used for another indication instead of where it’s been used, or failing, for the past 5 or 10 years.”

The discussion then highlighted key intellectual property, regulatory, funding and investment issues, which need to be addressed if researchers are considering this approach. “Despite the challenges I am told that nearly 30% of New Drug Applications (NDAs) currently under consideration by the FDA involve repurposing – the scale of this surprised me and it proves that despite the challenges it can be a successful approach,” said Tralau-Stewart.

Is drug repurposing less challenging for an academic group than taking on the full discovery and development of a new chemical entity (NCE)? Most panelists agreed that no simple answer exists, and it depends on ‘the whole package.’ However, all drug discovery is challenging with high risk and cost but potentially high rewards, and repurposing approaches do offer potential gains in terms of time to the clinic and regulatory hurdles but also strategic challenges in terms of intellectual property.

“I am currently aware of at least ten UCSF projects covering a wide range of indications considering or already progressing repurposing strategies,” said Tralau-Stewart. “After seeing the high turnout and interest at this seminar, I suspect that there are a lot more and would be keen to discuss the issues and options with interested researchers.”

WATCH the entire seminar on Youtube

Video Highlights & Key Themes:

-Welcome & Topic Introduction
-Panelist Introductions [14:00]
-Challenges [21:20]
-Patent Strategies, License Issues, Partnering with the innovator that holds the patent [28:40]
- Data Exclusivity, Intellectual Property, Orphan disease [31:40]
-How do you fundraise for smaller projects, smaller market? [39:25]
-Use Patents [44:30]
-What is the next step after scientist makes an observation? [52:00]
-Investor view on repurposing projects [1:16:00]
-Closing remarks/advice from each panelist [1:22:50]
-CTSI Closing Summary – Intellectual Property, off patent options, “The Whole Package,” Open Innovation [1:30:00]

Event Details

  • Lorna Tanner, JD,Partner, Intellectual Property Practice Group, Sheppard Mullin (sponsor)
  • J. Elin Hartrum, J.D., LL.M., Assoc. General Counsel, IP, Gilead Sciences, Inc.
  • Cyril De Colle, MS, PhD,Pharmaceutical Professional, Entrepreneur, Founder & President, Neurogastrx
  • Ken Horne, MS, CEO, Symic Biomedical, Ruphena, LLC
  • William P. Quinn, MA, MBA, Founder & CEO, Bullet Biotechnolog
  • Brian G. Atwood, MS, MBA, Managing Director, Versant Ventures

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UCSF's CTSI is a member of the Clinical and Translational Science Awards network funded through the National Center for Advancing Translational Sciences (grant Number UL1 TR000004) at the National Institutes of Health. Under the banner of "Accelerating Research to Improve Health," CTSI provides a wide range of resources and services for researchers, and promotes online collaboration and networking tools such as UCSF Profiles.