Allschwil,
Business highlights
- Positive results in the Phase 3 program of daridorexant, demonstrating improved overall sleep and daytime functioning of patients with insomnia
- Daridorexant new drug application (NDA) submitted to the
US FDA onJanuary 8, 2021 - Positive results in the Japanese registration program for clazosentan, demonstrating reduction in vasospasm-related morbidity and all-cause mortality
- US commercial operations established, with leadership team in place, and Syneos Health selected as commercialization partner to build the salesforce for the launch of daridorexant in the US
- Neurocrine Biosciences entered into a license agreement for the development and commercialization of Idorsia’s novel T-type calcium channel blocker
- Axovan arbitration claim dismissed by
Arbitral Tribunal
Financial highlights
- Successful capital increases secured over
CHF 865 million of funding to prepare for the launch of daridorexant and to develop our diversified pipeline - US GAAP operating expenses 2020 at
CHF 482 million - Non-GAAP operating expenses 2020 at
CHF 444 million - Guidance for 2021: US GAAP operating expenses of ~
CHF 685 million and non-GAAP operating expenses of ~CHF 640 million (both measures include inventory build of aroundCHF 35 million and exclude unforeseen events).
“I am very proud of all that has been accomplished at
Financial results
US GAAP results | Full Year | Fourth Quarter | ||
in CHF millions, except EPS (CHF) and number of shares (millions) | 2020 | 2019 | 2020 | 2019 |
Revenues | 72 | 24 | 6 | 4 |
Operating expenses | (482) | (506) | (128) | (131) |
Operating income (loss) | (411) | (482) | (123) | (127) |
Net income (loss) | (445) | (494) | (137) | (142) |
Basic EPS | (3.11) | (3.76) | (0.85) | (1.08) |
Basic weighted average number of shares | 142.8 | 131.2 | 160.8 | 131.2 |
Diluted EPS | (3.11) | (3.76) | (0.85) | (1.08) |
Diluted weighted average number of shares | 142.8 | 131.2 | 160.8 | 131.2 |
US GAAP revenue of
US GAAP operating expenses in 2020 amounted to
US GAAP net loss in 2020 amounted to
The US GAAP net loss resulted in a net loss per share of
Non-GAAP* measures | Full Year | Fourth Quarter | ||
in CHF millions, except EPS (CHF) and number of shares (millions) | 2020 | 2019 | 2020 | 2019 |
Revenues | 72 | 24 | 6 | 4 |
Operating expenses | (444) | (470) | (142) | (122) |
Operating income (loss) | (372) | (446) | (136) | (118) |
Net income (loss) | (392) | (448) | (148) | (121) |
Basic EPS | (2.75) | (3.41) | (0.92) | (0.92) |
Basic weighted average number of shares | 142.8 | 131.2 | 160.8 | 131.2 |
Diluted EPS | (2.75) | (3.41) | (0.92) | (0.92) |
Diluted weighted average number of shares | 142.8 | 131.2 | 160.8 | 131.2 |
*
Non-GAAP net loss in 2020 amounted to
The non-GAAP net loss resulted in a net loss per share of
Issuance of new registered shares
In 2020,
As a result of the capital increases, the share capital of
“Our strengthened balance sheet with
Liquidity and indebtedness
At the end of 2020, Idorsia’s liquidity (including cash, cash equivalents, short- and long-term deposits) amounted to
(in CHF millions) | ||||
Liquidity | ||||
Cash and cash equivalents | 141 | 148 | 263 | |
Short-term deposits | 867 | 668 | 476 | |
Long-term deposits | 192 | - | - | |
Total liquidity* | 1,200 | 816 | 739 | |
Indebtedness | ||||
Convertible loan | 388 | 386 | 380 | |
Convertible bond | 199 | 199 | 199 | |
Other financial debt | - | - | - | |
Total indebtedness | 587 | 585 | 579 |
*rounding differences may occur
Clinical Development
In April and July of 2020,
In November of 2020,
In addition to the progress made in 2020, the company is initiating a Phase 2 proof-of-concept study with ACT-539313, a selective orexin 1 receptor antagonist, in binge eating disorder. Preclinical studies have shown that orexins play an important role in compulsive, binge-like feeding behaviors. Furthermore, orexin receptor antagonists have reduced binge-like eating behavior in animal models. Our study will be the first to study orexin 1 receptor antagonism as a new mechanism of action for patients with eating disorders.
The company is also initiating a Phase 1 clinical pharmacology program with a new Immunology compound ACT-777991 resulting from our drug discovery efforts.
Following initial exploratory results with ACT-774312, a CRTH2 receptor antagonist, in nasal polyposis, the company has decided not to pursue this indication further. There were no safety concerns raised, however the company has no further plans for the compound at this time.
Idorsia’s clinical development pipeline
Compound | Mechanism of Action | Target Indication | Status |
Daridorexant | Dual orexin receptor antagonist | Insomnia | NDA submitted, MAA in preparation |
Aprocitentan* | Dual endothelin receptor antagonist | Resistant hypertension management | Phase 3 |
Clazosentan | Endothelin receptor antagonist | Cerebral vasospasm assoc. with aneurysmal subarachnoid hemorrhage | Global: Phase 3 |
Lucerastat | Glucosylceramide synthase inhibitor | Fabry disease | Phase 3 |
Selatogrel | P2Y12 receptor antagonist | Suspected acute myocardial infarction | Phase 3 in preparation |
Cenerimod | S1P1 receptor modulator | Systemic lupus erythematosus | Phase 2b |
ACT-539313 | Selective orexin 1 receptor antagonist | Binge eating disorder | Phase 2 |
Sinbaglustat | GBA2/GCS inhibitor | Rare lysosomal storage disorders | Phase 1 complete |
ACT-1004-1239 | CXCR7 antagonist | Immunology | Phase 1 |
ACT-1014-6470 | - | Immunology | Phase 1 |
ACT-541478 | - | CNS | Phase 1 |
ACT-777991 | - | Immunology | Phase 1 |
* In collaboration with
Neurocrine Biosciences has a global license to develop and commercialize ACT-709478, a novel T-type calcium channel blocker. In
Further details including the current status of each project in the pipeline can be found in our clinical development fact sheet.
Human Resources
Annual Report
Full details on the progress made in 2020 are available in
Legal update on the Axovan arbitration
As a result of the demerger of
Axovan sellers and
In 2018, the assignee of 65% of former Axovan shareholders (the “Claimants”) entered into an arbitration against Actelion claiming that the acquisition of Actelion by Johnson & Johnson and/or the Demerger triggers the accelerated payment of all outstanding milestones mainly relating to clazosentan (the “Claim”) plus statutory interest for late payment.
On
In the first half 2020, the company accrued
Note to Shareholders
The Annual General Meeting (AGM) of Shareholders to approve the Annual Report of the year ending
Registered shareholders with voting rights individually or jointly representing at least 5% of the share capital of the company, being entitled to add items to the agenda of the general meeting of shareholders, are invited to send in proposals, if any, to
In order to vote at the Annual General Meeting, shareholders must be registered in the company's shareholder register by
Results Day Center
Investor community: To make your job easier, we provide all relevant documentation via the Results Day Center on our corporate website: www.idorsia.com/results-day-center.
Upcoming Financial Updates
- First Quarter 2021 Financial Results reporting on
April 22, 2021 - Annual General Meeting of Shareholders on
May 12, 2021 - Half-Year 2021 Financial Results reporting on
July 27, 2021 - Nine-months 2021 Financial Results reporting on
October 26, 2021
Notes to the editor
Letter to Shareholders from the Chairman (as published in
Dear Shareholders,
It is my great honor to write to you as Chairman of your company for the first time. I am only sorry that we have not yet had the opportunity to interact personally. Our virtual Annual General Meeting was just one of the many disruptions caused by the COVID 19 pandemic – a crisis which has impacted every area of our lives in 2020, bringing misery and hardship to countless millions. In our industry, COVID-19 has upended supply chains and clinical trial timelines, threatening longer waiting times for much-needed treatments.
Despite the devastation all around us, I am very proud and excited to report that
Thanks to the investments made during Idorsia’s first few years, our technology platforms are state-of-the-art and scalable. As a result, when the call went out for office-based staff to work from home and for those who need to work on-site to be protected, our workforce was able to transition seamlessly. Our laboratory-based staff willingly turned up every day, motivated to move the company forward.
The resilience and determination of our workforce contributed to an incredibly successful year: the company has delivered on every one of the ambitious goals set by the management and Board at the end of 2019. Jean-Paul Clozel’s update on these achievements (see the CEO’s letter below) makes for very enjoyable reading. As you reflect on these successes, please bear in mind that it is very unusual for a clinical pipeline to advance so positively. For a young company to have so many innovative compounds – at a late stage of development, and in a wide variety of therapeutic areas – progressing as planned is, in itself, an impressive feat. For two of those compounds to achieve positive results and move towards regulatory submissions, in parallel, is outstanding. Accordingly, as you will see, the Board has recognized this performance through Idorsia’s employee reward system.
As Chairman, I am well aware that positive results alone are not enough to ensure sustainable success. With several newcomers to the Board, we have undertaken a critical review of the company to make sure that all the appropriate checks and balances are in place. For me, this is another of the beauties of
As a Board, we are committed to transparency on the topics that are important to you. This year, we initiated a dialogue to identify and prioritize what matters most to you, and these lines of communication will remain open. I look forward to developing our reporting on these important non-financial measures, in line with the company’s development.
One result of the company’s willingness to engage is the growth of our analyst base during 2020: 14 analysts are now covering
Of course, if
Sincerely,
Chairman of the Board
Letter to Shareholders from the CEO (as published in
Dear Shareholders,
I want to begin by recognizing that, while
This year, the public has gained a new appreciation of the efforts of all frontline healthcare workers, and also of the mission that drives everyone in the pharma world – finding medicines to prevent or treat illness. Let us hope that the vaccines developed in record time can help the world return to some semblance of normality. I myself am very proud to be a member of this industry, and to see how our efforts in research are helping patients.
In spite of the pandemic, the company has made great strides this year. Our employees achieved every one of the ambitious key goals set for 2020 – and much else besides. Their performance was simply phenomenal. In the development of new medicines, hard work does not always guarantee good results, which makes it all the more rewarding when our efforts are crowned with success.
Revolutionizing the field of insomnia
Our review of the progress made in 2020 must start with the outstanding results achieved with daridorexant. The Phase 3 registration program demonstrated statistically significant and clinically meaningful improvements in sleep maintenance, sleep onset, total sleep time and daytime functioning, which were sustained over time. Daridorexant was well tolerated, with a favorable safety profile in adult and elderly patients.
Particularly important, as well as efficacy during the night, are the effects seen during the day. While a negative impact on daytime functioning is part of the definition of insomnia, not one of the treatments currently available have rigorously assessed their effect on this key aspect of the condition. Here, in fact, most therapies have a negative impact – especially first thing in the morning, when patients may feel hangover effects of their medication. For the millions of people suffering from insomnia, daridorexant is an absolute gamechanger, whose excellent benefit and safety profile will encourage patients to seek treatment.
It is important to recognize that these gratifying results were no accident. They are a direct result of our efforts to design a dual orexin receptor antagonist (DORA) with the properties required to deliver all the benefits of a good night’s sleep. For more details, I invite you to read the interview with
The team responsible worked tirelessly to evaluate the huge amount of data generated and to present the results appropriately for regulatory agencies. A new drug application was submitted to the FDA in
Perseverance finally paying off
Our efforts to design the perfect DORA began in our labs as long ago as 1998, but our work with clazosentan goes back even further. This year, the Japanese studies of clazosentan for patients suffering cerebral vasospasm following aneurysmal subarachnoid hemorrhage also demonstrated excellent efficacy results, without any unexpected safety findings (for more information on clazosentan, see pages 52 to 57 of the Business Report). I am truly delighted to finally have the evidence that clazosentan can improve outcomes for these patients – often young adults with so much life left to live. These results give the commercial team even more to get their teeth into. The impressive data has renewed the enthusiasm of the whole REACT team to complete the global study of clazosentan as soon as possible, so that patients around the world can benefit.
More to come from the pipeline
Thanks to the development group’s speedy response to the COVID-19 pandemic, our ongoing late-stage studies were able to continue, and risk mitigation procedures were agreed with global health authorities.
Recruitment for both the
In addition, again despite the impact of the pandemic, recruitment for the PRECISION (aprocitentan) study accelerated, with randomization now expected to be completed by mid 2021.
As well as the conclusion of studies, 2021 will also see the initiation of a very exciting study. Under a Special Protocol Assessment (SPA) agreed with the FDA in 2020, a 14,000 patient Phase 3 study will be commenced with selatogrel – a drug-device product for patients with suspected heart attack. The investigation of selatogrel has been designated as a “fast-track” development program, indicating the FDA’s interest in this innovative approach. Patients themselves will play a crucial role in the preparations for this study, as they need to be trained to identify symptoms and self-administer treatment. The potential implications for future heart attack patients are enormous, and I look forward to sharing more details in due course.
Taking our innovation to the patient
Work on building our commercial capabilities began in 2019, and we continued to fill key strategic roles on the global team in 2020. We also established our US commercial organization, securing premises and – most importantly – a leadership team. This is a very exciting development for
Following the positive results achieved with daridorexant, a tremendous amount of work has been done to prepare for the US launch of this product in 2022 – not least, engaging Syneos Health as the ideal partner to reach the large US primary care market. In addition, after the excellent results with clazosentan, the Japanese team is also gearing up for our first product launch in
With two product launches in preparation, we have also been building our global supply chain function, to ensure consistent supplies of our innovative medicines to patients. More information on our preparations can be found in the interview with Chief Commercial Officer
Ongoing innovation
We have always had a long-term focus for
Financing our future
Through a series of financing activities in 2020, we have secured additional funding of more than
I am very proud of all that has been accomplished at
Warmest regards,
Chief Executive Officer
About
Headquartered near
For further information, please contact
Senior Vice President, Head of Investor Relations & Corporate Communications
+41 58 844 10 10
www.idorsia.com
The above information contains certain "forward-looking statements", relating to the company's business, which can be identified by the use of forward-looking terminology such as "estimates", "believes", "expects", "may", "are expected to", "will", "will continue", "should", "would be", "seeks", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.
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