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Idorsia : Transforming the Horizons of Therapeutic Options

Idorsia Pharmaceuticals Ltd | Jean Paul Clozel
Idorsia Pharmaceuticals Ltd | Jean Paul Clozel

Idorsia is a high-potential biopharmaceutical company focused on the development of small molecules. Without delving into the details of molecular weight and how it works biologically, the most important thing to know from a drug maker’s point of view about small molecules is that small molecules can be taken orally, whereas “large molecule” biologics usually require injection or another form of administration.

“I love small molecules because their field of use is infinite. You can use them in acute diseases or chronic diseases. I don’t know one disease where you can say we cannot treat with a small molecule.” Jean-Paul Clozel, MD, and CEO of Idorsia.

The pharmaceutical industry is full of well-known companies that people can rattle off the top of their heads. For now, Idorsia may not be a household name. But it is a company that you should get to know.

A biopharmaceutical start-up like no other

Idorsia may be young, but the company has a 20+ year heritage of drug discovery, a broad portfolio of innovative drugs in the pipeline, an experienced team of over 1,200 professionals covering all disciplines from bench to bedside, and commercial operations in Europe, Japan, and the US – the ideal constellation for bringing innovative medicines to patients.

Idorsia began its operations on June 15, 2017, following the demerger from Actelion, with registered shares of Idorsia Ltd being listed on the SIX Swiss Exchange the next day. Idorsia, while a start-up at heart and less than five years old on paper, hit the ground running in 2017 with 11 clinical assets, over 500 state-of-the-art laboratories, a team of over 600 professionals, and a strong balance sheet.

Most importantly, the company quickly moved four assets into Phase 3 development within a year due to the 20+ year heritage of discovering and developing innovative therapies for patients in need.

Mission and the Strategy

Idorsia’s mission is to become a leading mid-sized biopharmaceutical company. Since the start, the company has added the marketing and commercialization functions to support the rollout of the first products. Idorsia’s strategy is to harness all of the company’s key strengths and use them to fulfil its mission. The team at Idorsia has formulated five-mid-term key strategic priorities to achieve long-term success:

  • Deliver at least three products to market
  • Build a world-class commercial organization
  • Bring Idorsia to sustainable profitability
  • Fuel the pipelines with new discoveries
  • Utilize state of the art technologies to drive innovation

“We are confident that, once we reach break-even, our rich pipeline can continue to sustain and fuel growth well into the future,” said André C. Muller, CFO.

Inspiring Leadership

 “I love science,” Dr Clozel can be heard to say often.

Since the company’s inception, Dr Clozel has been the CEO and major shareholder alongside his wife, Dr Martine Clozel, the Chief Science Officer. Unlike some pharmaceutical CEOs, Dr Clozel began his career as a practicing cardiologist after achieving his medical degree in France. In addition, he has completed training in pharmacology and physiology at the University of Montreal, Canada, and at the University of California, San Francisco, USA.

Early on, Dr Clozel recognized that as a cardiologist, he could follow, at most, 30-40 patients a week, but as a researcher, he could potentially discover drugs that can treat millions of people. “I thought it was what I can do best to help people,” said Dr Clozel, as he switched his career from a practicing physician to a researcher, eventually becoming the Head of the Drug Discovery Group in the Cardiovascular Department of F. Hoffmann-La Roche.

When he and three others founded Actelion, the focus was to bring new therapeutic options to a specific rare disease – pulmonary arterial hypertension. Dr Clozel served as the CEO of Actelion from 2000 to 2017 and created a company that offered hope to patients living with this rare disease and value creation to the company’s investors.

“I don’t rest on past accomplishments,” said Dr Clozel. Therefore, alongside the Johnson & Johnson acquisition of Actelion, he negotiated a demerger, taking the early-stage pipeline, people, and facilities with him and started Idorsia.

 Maintaining drive

Although the company was well-positioned in 2017 as a start-up like no other, the past five years have been focused on advancing the pipeline and rebuilding missing elements to become a fully-fledged biopharmaceutical company. While the legacy of 20 years of searching for innovative drugs gave the company a rich pipeline, maintaining the drive to bring those assets to the market and filling the pipeline with potential new assets are the main tasks ahead for Idorsia.

As mentioned, four molecules quickly advanced into the next phase of clinical development. Out of the four, daridorexant was first with clinically relevant results for helping patients living with insomnia. Daridorexant, with the market name of QUVIVIQ™, was recently approved by the US FDA and Europe’s EMA. It has been available to patients in the US since May 2022, and other countries are expected to follow thereafter.

On insomnia, Dr Clozel said, “We want to wake up the market by introducing a new therapeutic option that finally deserves the name “sleeping pill”. It helps patients get a restful night of sleep, ultimately leading to a better day. We intend to modernize this market not only with medical innovation but also by how we approach medical professionals and patient education. We will use all tools of modern communication to reach our communities impactfully.”

A second molecule, clazosentan, also showed clinically relevant improvement for patients at risk of developing cerebral vasospasm following an aneurysmal subarachnoid haemorrhage in a Phase 3 study in Japan and is now available for patients in Japan under the market name of PIVLAZ™.

Culture of Innovation and Collaboration

Everyone talks about innovation, but at Idorsia, employees embrace this as part of their DNA.

“You really have to have the culture within the company where we stay focused on the goal, we do what we do best – to innovate and create drugs that will help patients,” remarked Dr Clozel. Furthermore, added Martine Clozel, “We are trying to achieve something which is really best in class.”

Idorsia has chosen to excel in organic chemistry, the first step in identifying small molecules. The company focuses on efforts to develop drugs in this area of expertise. It implements state-of-the-art technologies and artificial intelligence to stay at the forefront of what can be achieved using organic chemistry.

Dr Clozel said, “You cannot imagine giving antibodies for insomnia; giving a pill in the evening to the patient who wants to sleep is still the best solution. And this can only be done with organic chemistry, which is our sweet spot, and where all our innovations are born.”

Imparting the Keys to Success

Dr Clozel advised the enthusiastic entrepreneurs of tomorrow, “Always look for innovation, follow the science, and certainly don’t take shortcuts. This is a business where you need to go step by step. And unfortunately, there is no way you can skip the steps because it usually bites you back.”

 Living up to our promise – becoming a fully-fledged bio-pharmaceutical company

Idorsia has celebrated its fifth birthday this year. Looking ahead, Dr Clozel says of the company, “I would say that we have an incredible chance at Idorsia. We have a group of over 1,200 employees in a company which is five years old but with a research heritage of over 20 years. This is absolutely unique, and it will really allow us not only to build the pipeline but bring many drugs to the patient. And there is nowhere else in the world where you can find this opportunity.”

The company reached a major milestone with the launch of its first two products in two of the largest pharmaceutical markets in 2022. And more will follow. From bench to bedside, the Idorsia slogan reads, and now, with the commercial functions in place, the company is poised to realize this ambition. If you did not already know about the company, now you have some insights. Idorsia is a company to keep an eye on!



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