EDITED BY EMMETT T. CUNNINGHAM JR., MD, PHD, MPH, AND PRAVIN U. DUGEL, MD

“Very early in life, I knew that I wanted to be part of a scientific breakthrough that helped people. I was committed to do at least one drug that we could develop and bring to the world.”

So says Elisabet de los Pinos, PhD, the charismatic young founder and CEO of Aura Biosciences in Cambridge, Massachusetts, a businessperson and scientist who has already been named by Time Magazine, Goldman Sachs, and the World Economic Forum in Davos, Switzerland, as an entrepreneur to watch.

As a biotech entrepreneur, Dr. de los Pinos has chosen a tough disease as an initial target — ocular melanoma — for which there is no FDA-approved medical treatment. Her company has initiated a first human clinical trial in ocular melanoma utilizing a science so disruptive that it is designed to bind and destroy cancer cells while leaving healthy cells alone.

Based on the discoveries of John T. Schiller, PhD, at the National Cancer Institute (NCI), the 12-patient, phase 1 safety study of AU-011 will employ intravitreally delivered viral nanoparticles modeled on the human papilloma virus (HPV). These nanoparticles are a vehicle for potent cytotoxic therapies that can selectively eliminate cancer cells following laser activation. Dr. Schiller of NCI was awarded the National Medal of Technology and Innovation in 2014 for his contributions to medicine and public health. In addition, AU-011 was granted orphan drug status for ocular melanoma by the FDA in 2015.

Dr. de los Pinos, a native of Barcelona, Spain, compiled a superb academic record in molecular biology in Spain, the United Kingdom, and the United States. She followed this achievement with 5 years of high-level business experience launching and marketing key oncology drugs such as Alimta and Gemzar for Eli Lilly. Having acquired the business and scientific skills critical for a budding biotech entrepreneur, she was quick to decide in 2009 that Dr. Schiller’s research could be the foundation of an important new drug. She founded Aura Biosciences, attracted some “angel” financing, and purchased the exclusive drug-development rights to the Schiller patents from the NCI.

“I started the company with 1 employee,” she says. “It’s difficult to be an entrepreneur. We’ve done some things well and other things we could have done better. If you knew at the beginning the difficulties you were going to face, maybe you would never even start.”

OCULAR MELANOMA WAS FIRST CHOICE

Though the science indicated that drugs based on the Schiller patents could be effective for several types of tumors, including bladder and head/neck cancers, Dr. de los Pinos chose ocular melanoma (also called uveal melanoma) as Aura’s initial target.

“We saw a major unmet medical need in ocular melanoma and we also recognized that much of the pioneering work in virus-delivered therapies has been in ophthalmology, particularly gene therapy for retinal diseases,” she says.

Depending on how early the disease is diagnosed, people afflicted with ocular melanoma face either radiation therapy or surgery that often includes enucleation. If not caught at an early stage, ocular melanoma usually metastasizes to the liver, where it is generally fatal.

“Diagnosis can be through a routine eye exam, or the person experiences symptoms like vision loss or flashes and floaters,” notes Dr. de los Pinos. “Our drug is intended for people with early- to midstage ocular melanoma, so early diagnosis and referral to an ophthalmologist who specializes in ophthalmic oncology is important.”

Dr. de los Pinos estimates that there are about 2,000 patients with ocular melanoma treated with radioactive therapies or enucleation each year in the United States, but 3,000 new cases are diagnosed yearly at an early stage with the potential to be treated earlier if there was a vision-sparing targeted therapy. The highest incidence of ocular melanoma is in white males age 50 and over, especially those with blue eyes and pale complexions.

Dr. de los Pinos says AU-011 is designed to shrink and then eliminate the cancer with a single course of treatment, though retreatment may be needed for some larger tumors.

“Our preclinical studies have been very encouraging, with animal models that come close to replicating the human eye,” she says.

In vivo research data have demonstrated how synthetic viral-like therapeutic particles modeled on HPV bind uniquely to cancer cells while leaving healthy surrounding tissue unharmed. Researchers determined that tumor cells differ from healthy cells in the overexpression and modifications of heparan sulfate proteoglycans on their membrane as well as on the extracellular matrix, which provides a unique binding site for engineered viral-like therapeutic particles.

VALUES AND VISION

In building her team at Aura, Dr. de los Pinos looks for “people who want to change the world.” She says the qualities she most prizes in her colleagues are passion and purpose. She has been able to attract venture capital to fund the company’s activities until now. She is not certain what future path Aura may take but that “every company has a life of its own. Any partner must share our values and vision.”

Dr. de los Pinos says she has had many mentors along the way who inspired and encouraged her, but particularly points to three: Henri Termeer, co-founder and longtime CEO of the pioneering biotech giant Genzyme; the German scientist Harald Zur Hausen, whose seminal work on papilloma viruses won him the Nobel Prize; and California retina specialist Emmett Cunningham Jr., MD, PhD, MPH, who is also a managing director of the ophthalmic venture capital company Clarus, and an adviser to the Innovation in Retina series.

She also credits Aura’s scientific advisory board, which includes such experts in ocular cancer as Miguel N. Burnier, Jr., MD, PhD, FRCSC, Carol Shields, MD, Brian Marr, MD, Jay Duker, MD, Arun Singh, MD, and Evangelos Gragoudas, MD, as providing ongoing advice and encouragement on behalf of Aura’s efforts.

Asked where she thinks Aura Biosciences would be 5 years from now, Dr. de los Pinos is quick to answer.

“I think we will be launching our first drug globally, but maybe even sooner than 5 years.” RP