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Building a modern biotech: Novel approaches, partnering and cross-functional agility

Moving away from the traditional biotech model where the end goal of drug development was focused mainly on internal discovery and development of new, safe, and effective medicines, Horizon Therapeutics is embracing multiple scientific and business pathways to create successful treatments for autoimmune, severe inflammatory, and rare disease patients who have limited or no treatment options. The company has evolved into a fast-growing biotech, leveraging an agile three-pronged approach to expand its pipeline, while staying true to its founding principles.

These principles drive Horizon’s approach to innovation and patient impact and underpin the company’s approach to bringing groundbreaking treatments to patients who lack options: 1) digging deeper into the science to find new ways to solve scientific challenges and exploring new uses for existing medicines to allow them reach their fullest potential, 2) partnering with or acquiring companies to bring in external scientific expertise and 3) internal cross-functional collaboration that ensures scientific advancements not only get done fast, but are meaningful for patients.

Science first

Horizon’s R&D organization has expanded four-fold in recent years, bolstering its in-house capabilities across the development spectrum in the process. In March 2021, the acquisition of Viela Bio added four new molecules to the pipeline that represent new approaches to targeting disease biology and pathogenesis. For example, daxdilimab (HZN-7734), targets plasmacytoid dendritic cells (pDCs), an immune cell type that appears to play an important role in autoimmune diseases such as cutaneous lupus. As the only pDC depleter in clinical development, it targets a key driver of the chronic inflammation that underlies many autoimmune diseases.

“Our R&D team is constantly considering new ways to tackle the underlying biology of a disease, because we know this approach has the potential to improve patient outcomes,” says Elizabeth H.Z. Thompson, executive vice president, research and development, Horizon. “At the same time, we are always looking for new ways that our approved therapies can offer better options for patients.”

To that end, Horizon recognizes that innovation can come in many forms. Exploring the full potential of existing medicines often unlocks new benefits for patients.

For example, the company has become a leader in the treatment of uncontrolled gout, a painful and often overlooked condition. Rather than focusing only on developing new medicines for this disease, the company is also investigating approaches to enhance the benefits of its existing medicine approved for uncontrolled gout — combining it with an immunomodulator — to improve clinical outcomes for more patients.

This drive to deliver on the promise of breakthrough medicines has guided Horizon since its start. “We’ve always followed a simple philosophy: make a meaningful difference for patients,” says Tim Walbert, chairman, president and chief executive officer, Horizon.

Maximizing through partnerships

Beyond building internal capabilities to accelerate innovation, Horizon amplifies its scientific expertise by embracing external partnerships. Through its deep knowledge of uncontrolled gout, Horizon recognized that finding novel ways to tackle the condition could be valuable for these patients. In partnership with HemoShear Therapeutics, the companies hope to discover and validate next-generation therapeutic targets for gout using an innovative disease modeling platform. Additionally, in a collaboration with Arrowhead Pharmaceuticals, the companies are testing a new way of targeting a validated therapeutic pathway with short interfering RNA therapy to silence genes of interest and potentially address treatment burden.

“Partnering to solve scientific challenges is essential, because innovation comes from many sources,” says Andy Pasternak, executive vice president, chief strategy officer, Horizon. “As we pursue our science-forward R&D approach, we are excited to deepen and expand relationships across different areas of the drug development ecosystem to not only increase efficiencies in early R&D, but also to ensure our efforts meet the needs of patients.”

Collaborating within

Although bringing vital therapies to patients faster is an imperative in biotech today, the complexity of turning molecules into medicines for patients with rare and other severe diseases often thwarts internal efforts to increase speed and efficiency. Horizon has found ways to expedite development through internal collaboration to meet the needs of smaller patient populations who often have little treatment options available.

In 2020, Horizon launched the first FDA-approved medicine for Thyroid Eye Disease, and the first to address the underlying biology of the disease. The approval resulted in one of the most successful rare disease medicine launches in history, despite taking place amid the Covid-19 pandemic, in large part due to cross-functional collaboration that fuels Horizon’s nimble and patient-centric approach to drug development.

“We’re not a siloed organization, and we work hard to ensure our decision-making process honors that mindset — always bringing the full breadth of perspectives needed to set our programs up for success,” Thompson says. “By bringing all our disciplines together, we make and execute decisions fast — always as a team.”

Forward together

As the biotech landscape becomes more diverse and complex, Horizon will continue to respond by advancing its multi-pathway vision of R&D in its relentless pursuit of improving patients’ lives.

“The goal of our patient-focused and highly collaborative approach is to translate scientific breakthroughs into better outcomes for patients who need them most,” explains Pasternak. “We want our partners to see that we not only develop medicines but have the right expertise in place to ensure they are commercially successful.”

To learn more about how Horizon is charting new pathways to innovation, please visit