It’s not often that a biotech incubator becomes its own commercial-stage pharmaceutical company, but that’s the story of Israel-based BioLineRx, which received FDA approval for its first treatment in 2023.
Founded 20 years ago in 2003, BioLineRx was one of the early examples of regional biotech incubators established to help further diversify the country’s economy. BioLineRx’s mission, initially supported by the Israeli government, global pharmaceutical companies, and several life sciences-focused venture capital funds, was to turn university discoveries into potential treatments.
For its first six years, this young incubator did just that. BioLineRx has advanced multiple programs based on academic research across a variety of therapeutic areas and has successfully licensed out some of its risk-free programs.
Similar to other regional incubators, BioLineRx’s operating model is to license assets and use its development expertise to establish therapeutic clinical proof of concept, at which point it partners with pharmaceutical companies. Conduct large-scale, pivotal clinical trials and ultimately commercialize treatments after approval. As part of this strategy, BioLineRx will earn revenue from development, regulatory and commercial milestones, as well as royalties on future sales.
However, the inherent unpredictability of drug development and more stringent partnering requirements will ultimately put pressure on BioLineRx’s incubator operating model. The timing of out-licensing events is difficult to predict, and pharmaceutical companies have established stricter requirements for bringing in assets that include randomized data, necessitating more expensive Phase 2b trials. So in 2010, BioLineRx overhauled its strategy.
Many of the challenges faced by BioLineRx are familiar to biopharmaceutical leaders. If you invest too many resources in too many products, you may run out of money before you can bring a single product to market. If we fail to invest sufficient resources in promising treatments, we may never discover their potential.
Here’s how BioLineRx has effectively focused on creating a promising pipeline, and how you can too.
consider input carefully
The process of bringing a new drug to market always involves relationships with scientists, manufacturers, regulators, and investors. Use the feedback you receive from these collaborators to direct your efforts and resources.
“If you looked at our pipeline in 2008, you would have found 15 to 20 assets, almost all of them preclinical and across multiple disease states,” said BioLineRx’s chief financial officer in 2009. said Philip Sahlin, who has been appointed as Executive Director. In 2016 he became CEO.
When company leaders considered moving from an incubator model to a publicly traded biotech company, they learned that investors were not interested in such a diverse pipeline of early-stage assets.
“Investors felt it was very important to focus,” Sahlin said. “They preferred companies with specialized therapeutic area expertise and assets in more advanced stages of development.”
Review your goals and strategy
In response, BioLineRx hired a consulting firm to help the company “review the principles on which BioLineRx was founded,” Sahlin said. “Every few years, it’s a good idea to check in to see if your vision and strategy still makes sense.”
BioLineRx had intentionally cultivated a broad pipeline to reduce risk, but company leaders decided to change direction. The business sold all preclinical assets and focused solely on hematology and oncology. Ultimately, BioLineRx selected its lead asset, a next-generation CXCR4 inhibitor with promise in both hematopoietic stem cell mobilization and cancer immunotherapy.
“We were looking for a clinical-stage asset, and this one was ready for Phase 2. We already had Phase 1 safety data,” Sahlin says. BioLineRx also wanted assets that could be managed for as long as possible, and the company’s lead candidates fit that requirement.
The company moved ahead with clinical development, first investigating the drug’s impact on stem cell mobilization during autologous stem cell transplantation in multiple myeloma. The goal was to mature the assets and ultimately seek a commercial partner in this indication.
The company began considering commercialization opportunities as regulatory filings approached. The company spent one year conducting a complete market analysis and then hired an experienced commercial leader to complete the evaluation.
“This study makes it clear that for this particular indication, it makes more sense to commercialize it independently in the United States,” Sahlin said.
“There are only about 200 bone marrow transplant centers in the United States, but the top 80 perform more than 85 percent of all bone marrow transplants,” Sahlin says. “We realized that this was a bespoke sign for small businesses to set up on their own and decided to do so.”
“While this meant a change in strategy, we ultimately found it economically advantageous to commercialize independently in the U.S., with a high level of commitment from an appropriately sized and dedicated sales force. We now know we can secure it,” Sahlin said. “When you become a partner, you can lose that control and run the risk of a partner’s priorities, strategy and commitments changing.”
Stick to where there is a promise
Twelve years after being listed on NASDAQ, BioLineRx has received FDA approval as a leading asset for hematopoietic stem cell mobilization for autologous stem cell transplantation in multiple myeloma patients. The company remains committed to exploring the full potential of this drug, which shows promise. In pancreatic cancer, sickle cell disease, and other solid tumors.
“We feel that continuing to invest in lead molecules is the best path forward,” Sahlin said. “There are still signs that make sense for us.”
The company and its collaborators are conducting two randomized Phase 2b trials evaluating the company’s lead drug in combination with PD-1 inhibitors in patients with pancreatic cancer. The potential for combinations in pancreatic cancer and other solid tumors could have significantly greater market potential.
BioLineRx is partnering with researchers to evaluate lead agents as stem cell mobilization agents for sickle cell disease patients seeking gene therapy.
“Of the thousands of molecules developed around the world each year, only a few dozen brand-name drugs are approved each year,” Sahlin says. “We are proud of the progress we have made and are very pleased to see the continued development of biotech companies in the region. But more importantly, we are proud of our patient and I’m excited to be able to support families.”
It is about directing persistence in action. By carefully focusing efforts, rethinking goals and strategies, letting go of less promising initiatives, and persevering in promising areas, biopharmaceutical companies can succeed.
