Which technology do you think will have the biggest impact on healthcare over the next 10 years?
We don’t believe that only one technology will emerge but rather that many new technologies will facilitate improved personalized and more targeted treatments of severe diseases. Those technologies include targeted therapies with monoclonal antibodies, therapeutic proteins and targeted nanomedicine, the latter the approach Squal Pharma is developing. Cellular therapies (CAR T cells) and stem cells for tissue reconstruction and the microbiome are other technologies with great potential.
The cost of drug discovery is enormous — what should the industry focus on most in dealing with the cost hurdle?
We believe that the Squal Pharma model is a very cost-efficient approach to new drug development, but it is not discovering completely new molecular approaches. It is likely that artificial intelligence will help reduce the cost of drug preclinical and clinical development.
Tell us a bit about Squal Pharma, your latest endeavor. How did you discover the science behind Squal Pharma, and what led you to start the company?
Squal Pharma is a development-stage company that conjugates squalene, a biocompatible lipid found in human skin, to high-potential drugs to form new chemical entities to treat pain, cancer, burns, spinal cord injury, among other uses. These squalene-drug conjugates spontaneously self-assemble as highly coiled, compact nanoparticles in water without surfactant and polymer addition, exhibiting efficient manufacturability and yielding drug products that display extended blood circulation times, improved bioactivity and reduced toxicity that we have demonstrated in multiple preclinical models.
The “squalenoylation” technique was discovered and patented by my colleague and Co-founder Professor Patrick Couvreur. We started the company because we believe that our bioconjugates can potentially provide treatments for unmet medical needs such as pancreatic cancer, potent nonaddictive pain relief, efficient treatment for burns and wound healing, intractable spinal cord injury and others.
What is your current fundraising goal, and which milestones do you envision the company achieving at this time next year?
We are seeking $8 million initial funding, which, at the end of two and a half years, would allow us to take one of our new products through Phase 1 studies and complete investigational new drug (IND) enabling studies for a second product. At the end of the first 15 months, we expect to have completed IND enabling studies for our first product.
Do you intend to build out the Squal Pharma team, and, if so, how will you go about that?
At present we will utilize a capital-efficient virtual organization with a small, experienced management team. Prof. Couvreur as Chief Scientific Officer, me as CEO and Cofounder, Dr. Anita Melikian as COO. Dr. Melikian has 20 years of experience working in pharmaceutical companies, including one of my previously founded companies, and more than five years’ experience in patent law. Initially the three founders will manage outsourcing of the IND enabling and Phase 1 clinical studies.
Is there any person’s or team’s work at UCSF that you find particularly innovative right now?
There are many scientists at UCSF working on exciting innovations. I could imagine that some of these colleagues will be joining the ranks of the four existing Nobel Prize winners at UCSF.
You have received so many well-deserved industry accolades and produced an impressive body of work. Which achievement are you most proud of?
It is hard to say that my nine honorary doctoral degrees, five from European universities and four from U.S. universities, are not my proudest achievement. However, selection for the Remington Honor Medal, the highest honor in American pharmacy, is also very high on the list, especially since I am one of only three scientists (non-administrators) to be selected in the 100 years of the medal. At UCSF I am very proud to have been the first and, for many years, the only Ph.D. recipient of Distinguished Clinical Research Lectureship, as well as twice selected for the Outstanding Faculty Mentorship Award by the UCSF Graduate Division Alumni Association.
Photo credit for Dr. Benet’s headshot: Majed Abolfazli.
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