Idorsia announces financial results for the first quarter 2024 – advancing the
company with renewed vigor
Ad hoc announcement pursuant to Art. 53 LR
Allschwil, Switzerland – May 21, 2024
Idorsia Ltd (SIX: IDIA) today announced its financial results for the first
quarter of 2024.
Business highlights Viatris collaboration : Global research and development
collaboration, focused on the development and commercialization of two
innovative compounds, selatogrel and cenerimod.
Commercial highlights QUVIVIQ™ (daridorexant): Total net sales of CHF 10
million in Q1 2024. QUVIVIQ in the US: Citizens petition to deschedule the DORA
class progressing. QUVIVIQ in Europe: Further launches, including France,
provide a solid base to increase European sales in 2024.
Pipeline highlights TRYVIO™ (aprocitentan): Approved by the US FDA in March
2024. JERAYGO™ (aprocitentan): Recommended for approval in Europe.
Daridorexant: Phase 3 study conducted by Simcere in Chinese patients fully
recruited.
Financial highlights Net revenue Q1 2024 at CHF 10 million. US GAAP operating
expenses Q1 2024 benefiting from extraordinary income from Viatris deal with
non-GAAP operating expenses Q1 2024 at CHF 96 million. US GAAP operating income
Q1 2024 of CHF 31 million and non-GAAP operating loss of CHF 85 million. The
Viatris deal: The upfront consideration of USD 350 million (CHF 308 million)
was fully paid by Viatris to Idorsia in Q1 2024. Convertible bond 2024:
Bondholders approve an extension of maturity by six months.
Guidance for 2024 QUVIVIQ net sales of around CHF 55 million. US GAAP
operating loss to reach CHF 340 million (which includes a one-off benefit of
CHF 125 million from the Viatris deal), non-GAAP operating loss of around
CHF 420 million (excluding contract revenues and the one-off benefit from the
Viatris deal) – unforeseen events excluded.
Jean-Paul Clozel, MD and Chief Executive Officer, commented:
“We have already reached significant milestones in 2024. The deal with Viatris
to accelerate the development of selatogrel and cenerimod brought cash and
security for these assets, while retaining shareholder value. We came to an
arrangement with holders of the convertible bond 2024 due for repayment in
July, giving us the time we need to secure additional funding and avoid
liquidity constraints. Also, the quality of our research and development engine
was once again confirmed by the FDA’s approval of TRYVIO (aprocitentan) and the
CHMP positive opinion received for JERAYGO (the trade name of aprocitentan in
Europe). This will now unlock value for Idorsia as we evaluate possible launch
strategies – including potential partnership – for the first antihypertensive
working on a new pathway seen in almost 40 years.”
Jean-Paul continued: “Since the first launch of QUVIVIQ, more than 14 million
tablets have been dispensed worldwide, with well over 150,000 patients
benefiting from QUVIVIQ. We continue to believe in the huge potential offered
by this product and – thanks to a long patent life – there is plenty of time
for this potential to be realized. Continued innovation is essential to
securing the company’s future. Despite the reduction of the workforce, we
continue to discover and develop new drugs with great potential in many areas
of medicine. I am very confident that our vision to create an innovative,
profitable, and sustainable science-based company will become a reality in the
coming years.”
Financial results US GAAP results First Quarter in CHF millions, except EPS
(CHF) and number of shares (millions) 2024 2023 Net revenues 10 21 Operating
expenses 20 (219) Operating income (loss) 31 (198) Net income (loss) 30 (212)
Basic EPS 0.17 (1.19) Basic weighted average number of shares 179.1 178.0
Diluted EPS 0.13 (1.19) Diluted weighted average number of shares 233.3 178.0
Net revenue of CHF 10 million in the first quarter of 2024 is the result of
QUVIVIQ product sales. This compares to CHF 21 million in the first quarter of
2023, which included CHF 13.5 million sales of PIVLAZ in Japan (now assigned to
Nxera Pharma as part of a transaction, more details can be found in the
dedicated press release ) and CHF 1 million revenue share from Johnson &
Johnson related to ponesimod sales (revenue-sharing agreement now eliminated as
part of the reacquisition of aprocitentan, more details can be found in the
dedicated press release ).
US GAAP operating expenses in the first quarter of 2024 benefitted from
extraordinary income of CHF 125 million from the Viatris deal resulting in a
negative expense of CHF 20 million (CHF 219 million in the first quarter of
2023), of which CHF 4 million related to cost of sales (CHF 1 million in the
first quarter of 2023), CHF 33 million to R&D expenses (CHF 93 million in the
first quarter of 2023), and CHF 68 million to SG&A expenses (CHF 125 million in
the first quarter of 2023).
US GAAP net income in the first quarter of 2024 amounted to CHF 30 million
(CHF 212 million net loss in the first quarter of 2023). The decrease of the
net loss is mainly attributable to the one-off income related to the Viatris
Deal but was also driven by lower operating expenses throughout all functions.
The US GAAP net income resulted in a basic net income per share of CHF 0.17
(diluted net income per share of CHF 0.13) in the first quarter of 2024,
compared to a net loss per share of CHF 1.19 (basic and diluted) in the first
quarter of 2023. Non-GAAP* measures First Quarter in CHF millions, except EPS
(CHF) and number of shares (millions) 2024 2023 Net revenues 10 21 Operating
expenses (96) (202) Operating income (loss) (85) (181) Net income (loss) (86)
(189) Basic EPS (0.48) (1.06) Basic weighted average number of shares 179.1
178.0 Diluted EPS (0.48) (1.06) Diluted weighted average number of shares 179.1
178.0
* Idorsia measures, reports, and issues guidance on non-GAAP operating
performance. Idorsia believes that these non-GAAP financial measurements more
accurately reflect the underlying business performance and therefore provide
useful supplementary information to investors. These non-GAAP measures are
reported in addition to, not as a substitute for, US GAAP financial
performance.
Non-GAAP net loss in the first quarter of 2024 amounted to CHF 86 million: the
CHF 116 million difference versus US GAAP net income was mainly due to the
one-off effect of the Viatris Deal (CHF 125 million income), depreciation and
amortization (CHF 4 million), and share-based compensation (CHF 4 million).
The non-GAAP net loss resulted in a net loss per share of CHF 0.48 (basic and
diluted) in the first quarter of 2024, compared to a net loss per share of CHF
1.06 (basic and diluted) in the first quarter of 2023.
Viatris collaboration
In March 2024, Idorsia closed agreements with Viatris Inc. (NASDAQ: VTRS), a
global healthcare company, for collaboration on the global development and
commercialization of two Phase 3 assets – selatogrel and cenerimod – with
Idorsia receiving an upfront payment of USD 350 million, and the right to
potential development and regulatory milestone payments of up to USD 300
million, potential sales milestone payments of up to USD 2.1 billion, and
potential contingent tiered royalties from mid-single- to low-double-digit
percentage on annual net sales.
A joint development committee is overseeing the development of the ongoing
Phase 3 programs for selatogrel and cenerimod up to regulatory approval.
Idorsia will contribute up to USD 200 million in the next 3 years and
transferred the dedicated personnel for both programs to Viatris.
Viatris has worldwide commercialization rights for both selatogrel and
cenerimod (excluding, for cenerimod only, Japan, South Korea, and certain
countries in the Asia-Pacific region). Idorsia has also granted Viatris a right
of first refusal and first negotiation for certain other pipeline assets.
Convertible bonds 2024
In July 2018, the Group issued CHF 200 million of senior unsecured convertible
bonds (ISIN: CH0426820350), which were due to mature on July 17, 2024. On May
6, 2024, a bondholder meeting was held, where 83.5% of the total outstanding
bondholders voted in favor of amendments to the terms of the bonds. The
approved bond terms include an amended conversion price of CHF 6.00, extended
maturity date of January 17, 2025, and the option to call the bonds at par, in
full or in part, at any time upon giving ten trading days' notice. The company
has applied to the higher cantonal composition authority and upon approval the
amendments to the bond terms will become binding and effective. A consent fee
of 8,000,000 Idorsia shares will be delivered through SIX SIS once the
amendment of the bond terms is effective.
Financial outlook 2024
For 2024 – excluding unforeseen events – the company expects QUVIVIQ net sales
of around CHF 55 million; SG&A expenses of around CHF 300 million; R&D expense
of around CHF 165 million for Idorsia-led pipeline assets; non-GAAP operating
expenses of up to CHF 470 million. This performance would result in a non-GAAP
operating loss of around CHF 420 million (excluding contract revenues and the
one-off benefit from the Viatris deal).
The company expects US GAAP operating loss for 2024 to reach CHF 340 million
which includes a one-off benefit of CHF 125 million from the Viatris deal.
André C. Muller, Chief Financial Officer, commented:
“In addition to the funds already raised from our business development
activities, I am confident in our ability to raise additional funding this
year. We will continue to evaluate and prepare possible launch strategies –
including potential partnership – for TRYVIO. The significant progress with
access and availability of QUVIVIQ has started to gain traction, particularly
in Europe, this will translate into higher sales in 2024. At the same time, the
cost reduction initiative that took place in the latter part of 2023 is fully
effective and reflected in our 2024 guidance, with significantly lower
expenses. We must continue to control our costs and explore all avenues to
extend our cash runway, but I see many reasons to be optimistic for the future
of Idorsia.”
Liquidity and indebtedness
At the end of the first quarter of 2024, Idorsia’s liquidity amounted to CHF
335 million. (in CHF millions) March 31, 2024 Dec 31, 2023 Liquidity Cash and
cash equivalents 335 145 Short-term deposits - - Total liquidity* 335 145
Indebtedness Convertible loan 335 335 Convertible bond 797 796 Other financial
debt 162 162 Total indebtedness 1,293 1,293
*rounding differences may occur
Commercial operations
In the first quarter of 2024, QUVIVIQ™ (daridorexant) in the US, Germany,
Italy, Switzerland, Spain, UK, Canada, Austria, and France generated total
product sales of CHF 10 million.
United States Product Mechanism of action Indication Commercially available
since Dual orexin receptor antagonist Treatment of adult patients with
insomnia, characterized by difficulties with sleep onset and/or sleep
maintenance May 2022
In the US, net sales of QUVIVIQ ® (daridorexant) in the first quarter of 2024
reached CHF 6.5 million. This net sales number includes the QUVIVIQ copay
program aimed at driving demand and product uptake, and thus does not reflect
the actual number of prescriptions dispensed.
As of the end of the first quarter of 2024, more than 140,000 patients have
been treated with QUVIVIQ, almost 400,000 prescriptions have been dispensed,
and the product has been prescribed by more than 42,000 healthcare
professionals. To begin with, the company ran a direct-to-consumer (DTC)
television and digital campaign and offered a copay program. The strategy was
to create a recognizable brand, enabling market access discussions. During
2023, the company made significant progress, reaching over 65% reimbursement in
the commercial sector. As access increased, the commercial approach was
adjusted, with the aim being to switch from a consignment model (providing
substantially reduced or free prescriptions) to a payer paid model. In the
first quarter of 2024, paid prescriptions accounted for 68% of the total – an
increase of 36 percentage points from the same period in 2023 and of 7
percentage points from the previous quarter.
The first Medicare Part D coverage – reaching 27% of covered lives – began in
January 2024, opening an entirely new channel which has the potential to
substantially improve product access and paid prescriptions.
In February, there was a cyberattack on Change Healthcare (UnitedHealth
Group), the largest adjudicator/processor of copay cards in the US, causing
major disruption across the pharmaceutical industry, including the QUVIVIQ
copay cards, with a negative impact on prescription dispensing levels. In
March, the Idorsia US Market Access team put a solution in place to remedy the
disruption, though the impact was still appreciable through to the end of
March.
In April 2023, Idorsia filed a citizen petition (CP), urging the Drug
Enforcement Administration (DEA) to deschedule the DORA class of chronic
insomnia medications, based on a review of evidence from available data,
including post-marketing surveillance data. Starting in 2015, the independent
FDA approvals of other DORAs included a recommendation that these drug products
be scheduled based on preclinical data. The CP to deschedule the DORA class
outlines current scientific and medical evidence demonstrating that the DORA
class has a negligible abuse profile and potential for abuse, lacks non-medical
use in the community, lacks physical and psychological dependence, and
therefore, should not be a scheduled class under the Controlled Substances Act.
The DEA and FDA acknowledged the CP, and the process to analyze and examine
the request is moving forward. Notably, a report accompanying the FDA
appropriations bill that was finalized in March 2024 informed the FDA that the
process for descheduling the DORA class is a priority for Congress.
Tausif ‘Tosh’ Butt, President, and General Manager of Idorsia US, commented:
“I believe one of the biggest barriers to prescribing QUVIVIQ is the fact it
is currently a scheduled drug. Apart from the obstacles to prescribing
scheduled drugs, some payers require patients to be treated with low-cost drugs
not indicated for insomnia, and others that carry black box warnings before
covering QUVIVIQ. The US Congress has long supported the efforts of the FDA to
address the opioid and addiction crisis, and this year it encouraged the FDA to
also consider the impact of treatments for insomnia as a part of that larger
public health mission. I am very hopeful for our citizen petition requesting a
review of the evidence can lead to the descheduling of the DORA class of
chronic insomnia medications.”
For more information about QUVIVIQ in the US, see the Full Prescribing
Information (PI and Medication Guide). Product Mechanism of action Indication
Commercially available since Dual endothelin receptor antagonist Treatment of
hypertension in combination with other antihypertensive drugs, to lower blood
pressure in adult patients who are not adequately controlled on other drugs
Approved Mar. 2024
Planned availability: H2 2024
On March 19, 2024, the US Food and Drug Administration (FDA) approved TRYVIO™
(aprocitentan) for the treatment of hypertension in combination with other
antihypertensive drugs, to lower blood pressure in adult patients who are not
adequately controlled on other drugs. Lowering blood pressure reduces the risk
of fatal and non-fatal cardiovascular events, primarily strokes and myocardial
infarctions. The recommended dosage of TRYVIO is 12.5 mg orally once daily,
with or without food.
Idorsia plans to make TRYVIO available in the second half of 2024 to the
millions ofpatients in the US whose high blood pressure is not adequately
controlled by other drugs.
Further details on the approval, together with commentary from company
management can be found in the dedicated press release and investor webcast
available from the company corporate website.
For more information see the Full Prescribing Information including BOXED
Warning ( PI and Medication Guide ).
Europe and Canada Product Mechanism of action Indication Commercially
available Dual orexin receptor antagonist Treatment of adult patients with
insomnia characterised by symptoms present for at least three months and
considerable impact on daytime functioning France: Mar. 2024
Austria: Feb. 2024
UK: Oct. 2023
Spain: Sep. 2023
Switzerland: Jun. 2023
Germany: Nov. 2022
Italy: Nov. 2022 Management of adult patients with insomnia, characterized by
difficulties with sleep onset and/or sleep maintenance Canada: Nov. 2023
QUVIVIQ (daridorexant) net sales in the first quarter of 2024 reached CHF 3.5
million in the EUCAN region.
In November 2023, treatment with daridorexant was added to the insomnia
treatment guidelines for Europe. In “The European Insomnia Guideline: An update
on the diagnosis and treatment of insomnia 2023”, published in the Journal of
Sleep Research , the authors note that “The introduction of DORAs has probably
been the most significant recent development in the pharmacological treatment
of insomnia.”
In Germany, QUVIVIQ was launched in November 2022. By law, sleep medications
were then subject to a 4-week prescribing limitation (Anlage III BtMG).
Following a review by the Federal Joint Committee (G-BA) – the highest
decision-making body of the joint self-government of physicians, dentists,
hospitals, and health insurance funds in Germany – this limitation was lifted
for QUVIVIQ in November 2023. This makes it the only sleep medication in
Germany that can be prescribed for long-term treatment of chronic insomnia. In
December 2023, the price negotiated for QUVIVIQ under the AMNOG process became
effective. Following the lifting of the prescribing limitation, the company
submitted a second AMNOG dossier for the long-term treatment of chronic
insomnia disorder (beyond 4 weeks), reflecting the indication approved by the
EMA in 2022. The progress made in Germany is reflected by the performance of
QUVIVIQ on the market, with a 63% increase in demand seen in Q4 2023 (compared
to Q3 2023), followed by a strong start to 2024 (February +41% compared to
December 2023).
In Italy, QUVIVIQ was launched in November 2022. Currently, QUVIVIQ can only
be prescribed by neurologists, psychiatrists, and specialists from sleep
centers, and no sleep therapy is reimbursed. The company submitted a
reimbursement dossier in June 2023 and requested the expansion of the
prescriber base. The submission – detailing the efficacy and safety profile of
QUVIVIQ and its estimated budget impact and cost-effectiveness in Italy – is
under review, with the final outcome expected in the second half of 2024.
In Switzerland, QUVIVIQ was launched to the self-pay market in June 2023.
Following the launch of QUVIVIQ, awareness has increased among all specialties,
and demand has increased solidly (+32% in Q4 2023 compared to Q3 2023) ahead of
reimbursement, which is expected in the summer of 2024.
In Spain, QUVIVIQ was launched to the self-pay market in September 2023. Spain
represents the largest insomnia market in Europe, as was apparent in the first
months of this product’s availability, despite it only being launched to the
self-pay market. The company is assessing the opportunity to submit a
reimbursement dossier to the Spanish authorities, in order to allow equal
access for all patients with chronic insomnia.
In the UK, QUVIVIQ was launched in October 2023. At the same time, technology
appraisal guidance was published by the National Institute for Health and Care
Excellence (NICE), allowing the transition to local access discussions and
listing by healthcare boards for England, Wales, and Northern Ireland. In April
2024, the Scottish Medicines Consortium (SMC) also accepted QUVIVIQ for use
within NHS Scotland. This means that the company has achieved full
reimbursement throughout the UK, where QUVIVIQ is now recommended as first-line
pharmaceutical treatment for patients with chronic insomnia, after, or as an
alternative to, cognitive behavioral therapy for insomnia (CBT-I). The priority
in the UK now, is to secure regional access.
In France, QUVIVIQ was launched in March 2024 as the first and only
pharmacotherapy recommended for the treatment of chronic insomnia disorder. In
January 2024, the inclusion of QUVIVIQ in both the hospital and the retail
formulary list of reimbursed pharmaceutical specialties was announced in the
French Official Gazette, together with the French public price. This official
publication means that, with a prescription from their doctor, patients with
chronic insomnia in France have access to the treatment if they meet the
requirements of the EU prescribing label for QUVIVIQ. The publication follows
the positive recommendation by the Transparency Committee in May 2023,
recognizing QUVIVIQ as providing clinical added value.
In Canada, after being approved in April 2023, QUVIVIQ was launched in
November 2023 to the private market, representing 55% of the Canadian insomnia
market. The reimbursement dossier was submitted to private market payers in the
third quarter of 2023, and just a few months after the submission the team had
secured reimbursement for more than 60% of private market patients. The focus
is now on public payers with the submission to INESSS (Institut national
d’excellence en santé et en services sociaux) finalized in March 2024 and the
submission to CADTH (Canada’s Drug and Health Technology Agency) expected in
the second quarter of 2024.
Jean-Yves Chatelan, President of Europe and Canada region, commented:
“The launch of Europe’s first and only dual orexin receptor antagonist is
progressing well across all markets where we have made QUVIVIQ available.
Including Canada, we have expanded availability into more markets and improved
the reimbursement environment beyond many expectations. With continued positive
feedback from physicians and patients on the differentiated profile of QUVIVIQ,
I am very optimistic that the progress we have made will now translate into
many more patients benefiting from QUVIVIQ and increasing volumes advancing the
region towards profitability.”
For more information about QUVIVIQ in the EU, see the Summary of Product
Characteristics . For more information about QUVIVIQ in Switzerland, see the
Patient Information and Information for Healthcare Professionals . For more
information on the marketing authorization of QUVIVIQ in Canada, see the
Product Monograph .
Research & Development
Idorsia has a diversified and balanced portfolio, comprising assets developed
and/or marketed by Idorsia and assets that are partner-led to maximize the
value we have created. Our drug discovery engine has produced innovative drugs
with the potential to transform the treatment paradigm in multiple therapeutic
areas, including CNS, cardiovascular, and immunological disorders, as well as
orphan diseases.
The company also has a vaccine platform for the discovery and development of
glycoconjugate vaccines containing synthetic antigenic glycan molecules, with
or without a carrier protein, to prevent infection.
Alberto Gimona, MD and Head of Global Clinical Development of Idorsia,
commented:
“Despite a difficult period for our organization, the team has shown
extraordinary commitment and made great progress with our portfolio. This is
particularly evident in the successful registration of aprocitentan in the US
and the positive opinion from the European Union’s CHMP, with labels that
reflect the value of the compound. I was also very pleased to have found a way
for both selatogrel and cenerimod programs to be fully supported through the
collaboration with Viatris, while maintaining our involvement in their
development. I look forward to advancing the portfolio and bringing benefits to
patients in many areas of medical need.”
Idorsia-led portfolio Compound
Mechanism of action
Target indication Status QUVIVIQ™ (daridorexant)
Dual orexin receptor antagonist
Insomnia Commercially available as QUVIVIQ in the US, Germany, Italy,
Switzerland, Spain, the UK, Canada, Austria, and France; approved throughout
the EU TRYVIO™ (aprocitentan)
Dual endothelin receptor antagonist
Systemic hypertension in combination with other antihypertensives Approved as
TRYVIO in the US, launch planned for H2 2024 JERAYGO™ (aprocitentan)
Dual endothelin receptor antagonist
Resistant hypertension in combination with other antihypertensives Positive
opinion from the European Committee for Medicinal Products for Human Use (CHMP)
received in April 2024 – European Commission decision expected in approx. 2
months Lucerastat
Glucosylceramide synthase inhibitor
Fabry disease Phase 3 primary endpoint not met; open-label extension study
ongoing
Phase 3 focused on renal function in preparation Daridorexant
Dual orexin receptor antagonist
Pediatric insomnia Phase 2 in pediatric insomnia ongoing ACT-1004-1239
ACKR3/CXCR7 antagonist
Demyelinating diseases including multiple sclerosis Phase 2 in preparation
Sinbaglustat
GBA2/GCS inhibitor
Rare lysosomal storage disorders Phase 1 complete ACT-777991
CXCR3 antagonist
Recent-onset Type 1 diabetes Phase 1 complete IDOR-1117-2520
Undisclosed
Immune-mediated disorders Phase 1 ongoing IDOR-1134-2831
Synthetic glycan vaccine
Clostridium difficile infection Phase 1 initiating
Daridorexant
Daridorexant is a dual orexin receptor antagonist (DORA) which blocks the
binding of the wake-promoting orexin neuropeptides. Rather than inducing sleep
through broad inhibition of brain activity, daridorexant only blocks the
activation of orexin receptors. Daridorexant is commercially available as
QUVIVIQ in the US, Germany, Italy, Switzerland, Spain, the UK, Canada, Austria,
and France, and is approved throughout the EU (see “Commercial operations”
above).
A post-approval study to investigate the efficacy of daridorexant in patients
with insomnia and comorbid nocturia has completed recruitment and is expected
to report results in mid-2024 (NCT05597020).
Idorsia has initiated a Phase 2 dose-finding study to assess the efficacy,
safety, and pharmacokinetics of multiple-dose oral administration of
daridorexant in pediatric patients aged 10 to < 18 years with insomnia disorder
(NCT05423717). The primary objective of the study is to characterize the
dose-response relationship of daridorexant on objective total sleep time (TST),
using polysomnography. The study is expected to enroll around 150 patients, who
will be randomized in a 1:1:1:1 ratio to 10 mg, 25 mg, or 50 mg daridorexant,
or placebo. The study is part of a US FDA-approved Pediatric Study Plan and an
EU PDCO-approved Paediatric Investigation Plan.
Aprocitentan
Aprocitentan is a once-daily, orally active, dual endothelin receptor
antagonist, which inhibits the binding of ET-1 to ET A and ET B receptors.
Aprocitentan has a low potential for drug-drug interaction and a mechanism of
action suited for lowering blood pressure in adult patients whose hypertension
is not adequately controlled by other drugs. On March 19, 2024, aprocitentan
was approved as TRYVIO in the US, with availability planned for H2 2024. On
April 25, 2024, Idorsia received a positive opinion for aprocitentan (as
JERAYGO™) from the Committee for Medicinal Products for Human Use (CHMP) as a
treatment of resistant hypertension. A CHMP positive opinion is one of the
final steps before marketing authorization can be granted by the European
Commission; a final decision is expected approximately two months after
publication of the CHMP opinion.
Lucerastat
Lucerastat is an oral inhibitor of glucosylceramide synthase, offering a
potential new treatment approach for all patients living with Fabry disease,
irrespective of the mutation type of the GLA gene. In October 2021, the company
reported that lucerastat 1000 mg b.i.d. did not meet the primary endpoint of
reducing neuropathic pain during 6 months of treatment versus placebo. However,
Lucerastat demonstrated a substantial reduction in levels of the Fabry disease
biomarker plasma Gb3 during the treatment period, with a decrease of
approximately 50% observed in plasma Gb3 in the lucerastat treatment group
compared to an increase of 12% in the placebo group. Furthermore, results
suggested a treatment effect on kidney function. Lucerastat was well tolerated.
Analysis of the ongoing open-label extension (OLE) of the Phase 3 study
corroborated the long-term effect on plasma Gb3 levels and a potential positive
long-term effect on kidney function. The analysis also showed a safety and
tolerability profile consistent with that observed during the 6-month
randomized treatment period. The company is conducting a kidney biopsy substudy
within a subset of patients currently participating in the OLE study in order
to steer further development in Fabry disease.
Further details including the current status of each project in our portfolio
can be found in our innovation fact sheet .
Idorsia partner-led portfolio
For Idorsia, sophisticated partnerships are a way of gaining strategic access
to technologies or products and fully exploiting our discovery engine and
clinical pipeline. We seek suitable external project partners to maximize the
value of internal innovation. Compound
Mechanism of action
Target indication Partner/ status Daridorexant
Dual orexin receptor antagonist
Insomnia Nxera Pharma: license to develop and commercialize for Asia-Pacific
region (excluding China)
NDA submitted in Japan Daridorexant
Dual orexin receptor antagonist
Insomnia Simcere: license to develop and commercialize for Greater China region
Phase 3 ongoing Selatogrel
P2Y 12 inhibitor
Acute myocardial infarction Viatris: worldwide development and
commercialization rights
Phase 3 “SOS-AMI” program ongoing Cenerimod
S1P 1 receptor modulator
Systemic lupus erythematosus Viatris: worldwide development and
commercialization rights (excluding Japan, South Korea, and certain countries
in the Asia-Pacific region)
Phase 3 “OPUS” program ongoing Daridorexant
Dual orexin receptor antagonist
Posttraumatic stress disorder (PTSD) US Department of Defense (DOD ): Idorsia
is supporting a clinical study sponsored by the US DOD to develop new therapies
to treat PTSD ACT-709478 (NBI-827104)
T-type calcium channel blocker
Epileptic encephalopathy with continuous spike-and-wave during sleep (CSCW)
Neurocrine Biosciences: global license to develop and commercialize
Phase 2 OLE study ongoing ACT-1002-4391
EP 2 /EP 4 receptor antagonist
Immuno-oncology Owkin: global license to develop and commercialize
Phase 1 in preparation
Daridorexant (Nxera Pharma)
Daridorexant is licensed to Nxera Pharma (previously known as Sosei Heptares)
in the Asia-Pacific region (excluding China), and a New Drug Application (NDA)
is under review with the Japanese Ministry of Health, Labor, and Welfare
(MHLW).
In Japan, Idorsia has a license agreement with Mochida Pharmaceutical for the
supply, co-development and co-marketing of daridorexant. All potential
milestones have been assigned to Nxera.
Asia-Pacific region (excluding China): Australia, Brunei, Cambodia, Indonesia,
Japan, Laos, Malaysia, Myanmar, New Zealand, Philippines, Singapore, South
Korea, Thailand, Taiwan, and Vietnam.
Daridorexant (Simcere)
Daridorexant is licensed to Simcere in the Greater China region (Mainland
China, Hong Kong, and Macau), and a Phase 3 study with daridorexant in Chinese
patients hascompleted recruitment. Results are expected in June 2024 and, if
the study is successful, an NDA in Mainland China is planned for the second
half of 2024. An NDA is already under review with the Hong Kong Department of
Health.
Selatogrel and cenerimod (Viatris)
A joint development committee from Idorsia and Viatris is overseeing the
development of two ongoing Phase 3 programs up to regulatory approval.
Selatogrel is a potent, fast-acting, reversible, and highly selective P2Y 12
inhibitor being developed in a Phase 3 study ( NCT04957719 ) for the treatment
of acute myocardial infarction (“SOS-AMI”) in patients with a recent history of
AMI. It is intended to be self-administered subcutaneously via a drug delivery
system (autoinjector).
Cenerimod is a highly selective S1P 1 receptor modulator, given as an oral
once-daily tablet, which is being developed in a Phase 3 program known as
“OPUS” ( NCT05648500 , NCT05672576 ) for the treatment of systemic lupus
erythematosus (SLE).
Viatris has worldwide commercialization rights for both selatogrel and
cenerimod (excluding, for cenerimod only, Japan, South Korea, and certain
countries in the Asia-Pacific region).
Daridorexant (US Department of Defense)
Idorsia is supporting a clinical study sponsored by the US Department of
Defense (DOD) to develop new therapies for posttraumatic stress disorder
(PTSD). The Phase 2 study will evaluate the safety, tolerability, and efficacy
of potential therapeutic interventions, including daridorexant, in active-duty
US service members and veterans with PTSD ( NCT05422612 ).
ACT-709478
Neurocrine Biosciences has a global license to develop and commercialize
ACT-709478 (NBI-827104), Idorsia’s novel T-type calcium channel blocker.
ACT-709478 is being investigated in a Phase 2 open-label extension (OLE) study
for the treatment of pediatric patients with epileptic encephalopathy with
continuous spike-and-wave during sleep (CSCW), a rare form of pediatric
epilepsy. While the blinded study did not meet the primary endpoint, ACT-709478
was generally well tolerated and Neurocrine continues to analyze the totality
of data coming from the OLE study to determine the next steps.
ACT-1002-4391
Owkin has a global license to develop and commercialize ACT-1002-4391,
Idorsia’s novel, potent EP 2 /EP 4 receptor antagonist with antitumor efficacy,
to be used both as monotherapy and in combination with other oncology agents.
The compound is in preparation for Phase 1 clinical pharmacology studies. Owkin
will use its proprietary AI-based data-mining platform to generate clinical
trial designs and to identify patients who may benefit from, and potential
targets for, the compound.
Martine Clozel, MD and Chief Scientific Officer, commented:
“The way we work in research is focused on and built around innovation and our
core competencies. While we had to cut back on the number of people conducting
research in 2023, we have maintained this fundamental approach to our drug
discovery efforts. We have taken the restructuring as an opportunity to focus
on fewer key areas of research and will advance our discoveries either through
our own clinical development expertise, or with the right partner, aiming to
maximize the benefit for patients and Idorsia.”
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 Annual General Meeting of Shareholders on June 13,
2024 Half-Year 2024 Financial Results reporting on July 25, 2024 Nine-Months
2024 Financial Results reporting on October 29, 2024
Notes to the editor
About Idorsia
Idorsia Ltd is reaching out for more – We have more ideas, we see more
opportunities and we want to help more patients. In order to achieve this, we
will develop Idorsia into a leading biopharmaceutical company, with a strong
scientific core.
Headquartered near Basel, Switzerland – a European biotech-hub – Idorsia is
specialized in the discovery, development, and commercialization of small
molecules to transform the horizon of therapeutic options. Idorsia has a
25-year heritage of drug discovery, a broad portfolio of innovative drugs in
the pipeline, an experienced team of professionals covering all disciplines
from bench to bedside, and commercial operations in Europe and North America –
the ideal constellation for bringing innovative medicines to patients.
Idorsia was listed on the SIX Swiss Exchange (ticker symbol: IDIA) in June
2017 and has over 750 highly qualified specialists dedicated to realizing our
ambitious targets.
For further information, please contact
Andrew C. Weiss
Senior Vice President, Head of Investor Relations & Corporate Communications
Idorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil
+41 58 844 10 10
investor.relations@idorsia.com
media.relations@idorsia.com
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.
Anhang Medienmitteilung PDF