The question at the heart of Vincent Njar’s 2021 David J. Ramsay Entrepreneur of the Year presentation: “Drug Discovery: Ingenuity and Serendipity, or Is It the Other Way Around?”

Vincent Njar, PhD, professor, Department of Pharmacology, University of Maryland School of Medicine (UMSOM), tackled this concept Oct. 26 in a hybrid presentation before an in-person audience that included University of Maryland, Baltimore (UMB) President Bruce E. Jarrell, MD, FACS, and E. Albert Reece, MD, PhD, MBA, executive vice president for medical affairs, UMB, and dean, UMSOM, as well as Njar’s colleagues and family.

Jarrell and Reece introduced Njar, an internationally respected medicinal chemist and oncopharmacologist who is head of the medicinal chemistry section in the Center for Biomolecular Therapeutics at the Institute for Bioscience and Biotechnology Research, UMSOM. Njar is also co-founder of the company Isoprene Pharmaceuticals, Inc. (IPI), an early-stage small molecule oncology company developing oral therapeutics for triple negative breast cancer and other cancers. IPI is part of UMB’s New Ventures Initiative program, a unique model for select UMB startups with significant commercial promise.

“Dr. Njar is an entrepreneur par excellence. First and foremost, he is an outstanding scientist. He has more than $2½ million of extramural research funding active at the present time,” Reece said. “For his profound and creative research, and his collaborative and congenial style of finding solutions, Dr. Njar is what I would describe as the epitome of what a biomedical entrepreneur should be.”

Njar, who is a Distinguished University Professor, focuses his research and inventions on the discovery and development of small molecules as anti-cancer agents. He divided his presentation, titled “Drug Discovery and Development from Academia: The Quest for Novel and Efficacious Small-Molecule Anti-Cancer Drugs,” into two parts: a discussion about Galeterone, his invention that is being researched as a treatment for prostate and pancreatic cancers, and retinoic acid metabolism blocking agents (RAMBAs), or novel retinamides (NRs), that inhibit the growth of breast cancer cells and tumors.

But first he pointed out the differences between “academic” and “entrepreneur,” discussing what those words mean to him.

“In drug development, I could be called academic, but some drug discovery efforts are anything but academic,” he said. “Academic’s synonym is pedantic. Academic means it doesn’t matter. President Jarrell, I want to do things that matter. I certainly don’t want to be associated with the term pedantic.

“In Dean Reece’s letter congratulating me for this Founders Week award, he said, ‘We need more researcher-educator-entrepreneurs like you,’ ” Njar said. “Entrepreneur’s synonyms are creative, creativity, and all the good things. So if I ask you a question, ‘Can a person be an academic and an entrepreneur?’ I would say my answer for all of us, and I believe you would agree with me, is yes, yes, and yes indeed. When somebody is called an academic, it should mean that it does matter.”

Njar, who has a UMB-approved conflict of interest management plan in place, went on to discuss Galeterone, a CYP17 inhibitor he invented as an alternative to castration for prostate cancer treatment. Galeterone is also an androgen receptor (AR) antagonist, AR/AR-Vs degrader, and an Mnk-eIF4E signaling inhibitor.

“We believe that if you inhibit the synthesis of the CYP17, you can block the production from all sides and not just from the testes,” he said in discussing why he thinks it is a viable prostate cancer treatment. “Critical to this Galeterone discovery was that we invented a new reaction.

“We were able to show that our molecules are quite effective. So in essence, we have shown from all our studies over the years, that Galeterone is more efficacious than castration and drugs such as Casodex, Zytiga, or Xtandi,” he said.

Njar also discussed an earlier Phase 3 trial of Galeterone, citing the small trial size and the design of the trial as the reasons it failed, and how he rescued Galeterone to continue to study it. It has been licensed to Educational & Scientific LLC, which formed LTN Pharmaceuticals, Inc. to focus on Galeterone development. Njar’s research studies in pancreatic cancer in vitro and in vivo models have led to a Phase 2 clinical trial in men and women with pancreatic cancer at the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center.

Njar also discussed the development of the next-generation Galeterone analogs, which are supported by a five-year, $2.4 million National Institutes of Health R01 grant. Because of the substantial progress of the project, the National Cancer Institute (NCI) has awarded him a stepping-stones in-kind assistance and a two-year, $421,419 administrative supplement grant to conduct prostate cancer disparity collaborative research.

In talking about RAMBAs, Njar pointed out how his company, IPI, recently was awarded a direct-to-Phase 2, $2 million Small Business Innovation Research (SBIR) grant from NCI for a translational project to develop a novel therapeutic for triple negative breast cancer.

He said an SBIR program director recommended applying for the grant.

“In writing a Phase 2 grant for SBIR, one of the critical things in addition to the supporting proof-of-concept data you have, you have to have a plan on how to move forward with commercialization,” he said.

The grant will support advanced preclinical studies intended to lead to the filing of an investigational new drug application with the Food and Drug Administration as a prelude to Phase I clinical trials. IPI is partnering with DavosPharma on the studies, which Njar said he hopes to finish within two years.

Njar thanked Jarrell and Reece; Angela Brodie, PhD, the late UMSOM cancer research pioneer whom he worked with earlier in his career; as well as colleagues, students, postdoctoral fellows, and his family.

The presentation concluded with Phil Robilotto, DO, MBA, associate vice president, Office of Technology Transfer, announcing the University’s faculty-awarded patents, including 39 from UMSOM and four each from the School of Dentistry and School of Pharmacy.