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02.11.2023 07:00
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Ionis reports third quarter 2023 financial results
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PR Newswire
CARLSBAD, Calif., Nov. 2, 2023
Olezarsen Phase 3 data showed significant triglyceride lowering, substantial reductions in acute pancreatitis attacks and favorable safety and tolerability in patients with FCS; on track for regulatory filings in early 2024
Eplontersen marketing applications accepted for review in the EU and Canada; potential U.S. approval in December 2023
On track to achieve 2023 financial guidance
CARLSBAD, Calif., Nov. 2, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the third quarter of 2023.
"We continue to successfully execute on our strategy to deliver a steady cadence of potentially transformational medicines to patients," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "Eplontersen is on track for its first potential approval in the U.S. and is under regulatory review in the EU and Canada. We believe the positive efficacy and safety data coupled with an attractive self-administration dosing profile positions eplontersen to be the preferred therapy in the largely underserved hereditary ATTR polyneuropathy population. We reported positive data from the olezarsen Phase 3 Balance study in patients with familial chylomicronemia syndrome, showing statistically significant triglyceride lowering, substantial reductions in acute pancreatitis attacks and favorable safety and tolerability, positioning olezarsen to be our first independent commercial launch. We also made additional progress across our wholly owned and partnered pipeline, and further expanded our rich Phase 3 pipeline with the advance of zilganersen for patients with Alexander disease into Phase 3 development. Looking ahead, we expect to continue our positive momentum with the potential approval and launch of eplontersen and the Phase 3 data readout of donidalorsen in hereditary angioedema."
Third Quarter 2023 Summary Financial Results:
| | | Three months ended September 30, | | Nine months ended September 30, | ||||
| | | 2023 | | 2022 | | 2023 | | 2022 |
| | | (amounts in millions) | ||||||
| Total revenue | | $144 | | $160 | | $463 | | $435 |
| Operating expenses | | $287 | | $219 | | $811 | | $637 |
| Operating expenses on a non-GAAP basis | | $261 | | $195 | | $732 | | $562 |
| Loss from operations | | ($143) | | ($59) | | ($348) | | ($202) |
| Loss from operations on a non-GAAP basis | | ($117) | | ($35) | | ($269) | | ($127) |
Financial Highlights
- Revenue continued to be substantial and sustained, with revenues of $144 million and $463 million in the three and nine months ended September 30, 2023, reflecting a 10% decrease and 6% increase compared to the same periods last year, respectively, driven by the timing of certain partner payments
- Operating expenses increased in the three and nine months ended September 30, 2023 compared to the same periods last year, primarily due to strategic investments to bring eplontersen, olezarsen and donidalorsen to patients
- Cash and short-term investments of $2.2 billion as of September 30, 2023 enables continued investments to drive increasing value
- Reaffirmed 2023 financial guidance
Near-Term Commercial Opportunities and Late-Stage Pipeline Highlights
- Achieved multiple milestones with eplontersen:
- Eplontersen is under regulatory review by the European Medicines Agency (EMA) and Health Canada for the treatment of hereditary ATTR polyneuropathy (ATTRv-PN)
- The EMA granted orphan drug designation to eplontersen for the treatment of ATTR in the EU
- Published positive data from the Phase 3 NEURO-TTRansform study in patients with ATTRv-PN in the Journal of the American Medical Association (JAMA) showing eplontersen halted measures of disease progression and continuously improved quality of life at 35-, 66- and 85-weeks
- Presented positive new data showing continued benefit in secondary endpoints from the Phase 3 NEURO-TTRansform study in patients with ATTRv-PN at the European ATTR Amyloidosis (EU-ATTR) meeting
- Presented positive exploratory data from a pre-defined cardiac sub-population of patients in NEURO-TTRansform showing improvement in cardiac function and structure compared to external placebo at the Heart Failure Society of America (HFSA) Annual Scientific Meeting
- Completed enrollment of the Phase 3 CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy (ATTR-CM), the largest study ever conducted in ATTR-CM; on track for data readout as early as H1:2025
- Reported positive data from the Phase 3 Balance study of olezarsen in patients with familial chylomicronemia syndrome (FCS)
- Olezarsen demonstrated robust, dose-dependent reductions in APOCIII, statistically significant reductions in triglycerides, substantial reductions in acute pancreatitis attacks and a favorable safety and tolerability profile
- On track to file for regulatory approval in the U.S. and EU in early 2024
- The FDA granted orphan drug designation to donidalorsen for the treatment of patients with hereditary angioedema (HAE); on track for data readout in the Phase 3 OASIS-HAE study in H1:2024
- Advanced zilganersen (GFAP) into Phase 3 development for the treatment of patients with Alexander disease
- The FDA granted orphan drug designation to ulefnersen (FUS) for the treatment of patients with FUS-ALS
Partnered Program Highlights
- GSK reported positive data from the Phase 2b B-Together study of bepirovirsen followed by pegylated interferon in patients with chronic hepatitis B virus (HBV)
- Reported positive interim data from the ongoing Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN)
- Biogen reported positive data from the Phase 1/2 study of IONIS-MAPTRx (BIIB080) in patients with Alzheimer's disease
- Completed enrollment in the Phase 1/2 HALOS study of ION582 (BIIB121) in patients with Angelman syndrome
- Entered a new agreement with Roche to advance two novel RNA-targeted programs for Alzheimer's disease and Huntington's disease
Third Quarter 2023 Financial Results
"Our year-to-date financial results keep us on track to achieve our 2023 guidance as we execute on a strategy to unlock next-level value," said Elizabeth L. Hougen, chief financial officer of Ionis. "Our strong financial foundation includes more than $2B in cash, significant royalty revenue with SPINRAZA, and substantial and sustained R&D revenue from multiple partners. We are well positioned to continue investing in our key priorities to drive future positive cash flow, including advancing our go-to-market activities, growing our wholly owned pipeline and optimizing new cutting-edge technologies for future medicines. We look forward to the potential U.S. eplontersen ATTRv-PN approval next month followed closely by launch. Together with our partner, AstraZeneca, we believe we are well positioned to identify new patients to further grow the market and become the treatment of choice for this population that remains largely underserved by current therapies."
Revenue
Ionis' revenue was comprised of the following:
| | | Three months ended | | Nine months ended | ||||
| | | September 30, | | September 30, | ||||
| | | 2023 | | 2022 | | 2023 | | 2022 |
| Revenue: | | (amounts in millions) | ||||||
| Commercial revenue: | | | | | | | | |
| SPINRAZA royalties | | $67 | | $62 | | $179 | | $175 |
| Other commercial revenue: | | | | | | | | |
| TEGSEDI and WAYLIVRA revenue, net | | 8 | | 6 | | 25 | | 23 |
| Licensing and royalty revenue | | 9 | | 5 | | 26 | | 25 |
| Total commercial revenue | | 84 | | 73 | | 230 | | 223 |
| Research and development revenue: | | | | | | | | |
| Amortization from upfront payments | | 18 | | 18 | | 47 | | 54 |
| Milestone payments | | 16 | | 15 | | 90 | | 60 |
| License fees | | 5 | | 35 | | 25 | | 37 |
| Other services | | 5 Werbung Mehr Nachrichten zur Ionis Pharmaceuticals Inc Aktie kostenlos abonnieren
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