A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)

The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

Start Date14 Jun 2021

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

100 Clinical Results associated with Ulefnersen

100 Translational Medicine associated with Ulefnersen

100 Patents (Medical) associated with Ulefnersen

Literatures (Medical) associated with Ulefnersen

01 Jan 2022·Nature MedicineQ1 · MEDICINE

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Q1 · MEDICINE

Article

Author: Shneider, Neil A ; Jafar-Nejad, Paymaan ; Blanco-Redondo, Beatriz ; Korobeynikov, Vladislav A ; Lyashchenko, Alexander K

AbstractFused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). To explore the mechanisms by which mutant FUS causes neurodegeneration in ALS-FTD, we generated a series of FUS knock-in mouse lines that express the equivalent of ALS-associated mutant FUSP525L and FUSΔEX14 protein. In FUS mutant mice, we show progressive, age-dependent motor neuron loss as a consequence of a dose-dependent gain of toxic function, associated with the insolubility of FUS and related RNA-binding proteins. In this disease-relevant mouse model of ALS-FUS, we show that ION363, a non-allele-specific FUS antisense oligonucleotide, efficiently silences Fus and reduces postnatal levels of FUS protein in the brain and spinal cord, delaying motor neuron degeneration. In a patient with ALS with a FUSP525L mutation, we provide preliminary evidence that repeated intrathecal infusions of ION363 lower wild-type and mutant FUS levels in the central nervous system, resulting in a marked reduction in the burden of FUS aggregates that are a pathological hallmark of disease. In mouse genetic and human clinical studies, we provide evidence in support of FUS silencing as a therapeutic strategy in FUS-dependent ALS and FTD.

News (Medical) associated with Ulefnersen

02 Nov 2023

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Ionis reports third quarter 2023 financial results

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: 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: Commercial revenue for the three and nine months ended September 30, 2023 included $67 million and $179 million from SPINRAZA royalties, respectively, which were essentially flat compared to the same periods last year reflecting SPINRAZA's resilience against emerging competition. Ionis' commercial revenue in the three and nine months ended September 30, 2023 also included royalties from the U.S. launch of QALSODY. R&D revenue decreased for the three months ended September 30, 2023 and increased for the nine months ended September 30, 2023 compared to the same periods last year due to the timing of certain partner payments, including the $35 million license fee for IONIS-FB-LRx that Ionis earned from Roche in the three months ended September 30, 2022. Operating Expenses Ionis' operating expenses increased in the three and nine months ended September 30, 2023 compared to the same periods in 2022, consistent with expectations. As Ionis advanced its robust pipeline, study costs increased compared to the same periods in 2022 as most of the Company's Phase 3 studies are either fully enrolled or approaching full enrollment, resulting in higher R&D expenses year over year. Ionis' SG&A expenses also increased year over year primarily due to launch preparation activities for eplontersen, olezarsen and donidalorsen. Balance Sheet As of September 30, 2023, Ionis' cash, cash equivalents and short-term investments increased to $2.2 billion compared to $2.0 billion at December 31, 2022 primarily due to the $500 million Ionis received from Royalty Pharma in January 2023. Ionis' working capital also increased over the same period primarily due to the Company's higher cash and short-term investments balance. In the first quarter of 2023, the Company recorded a long-term liability for future royalties due to Royalty Pharma. In June 2023, Ionis issued $575 million of senior convertible notes due in June 2028 with an interest rate of 1.75%. The Company used the majority of the proceeds to repurchase $504 million of its 0.125% convertible notes. Webcast Management will host a conference call and webcast to discuss Ionis' third quarter 2023 results at 11:30 a.m. Eastern time on Thursday, November 2, 2023. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company's third quarter 2023 earnings slides click here. About Ionis Pharmaceuticals, Inc. For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies. To learn more about Ionis visit or follow us on Twitter @ionispharma. Ionis' Forward-looking Statement This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of QALSODY (tofersen), SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen, zilganersen, ulefnersen, pelacarsen, bepirovirsen, IONIS-FB-LRx, Ionis' technologies and Ionis' other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2022, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. QALSODYTM is a trademark of Biogen. SPINRAZA® is a registered trademark of Biogen. Reconciliation of GAAP to Non-GAAP Basis As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' non-GAAP results is consistent with how Ionis' management internally evaluates the performance of its operations. Key 2023 Value Driving Events(1) SOURCE Ionis Pharmaceuticals, Inc.

Clinical ResultPhase 3Orphan DrugDrug ApprovalFinancial Statement

18 Oct 2023

·www.prnewswire.com

Ben Stiller, Christine Taylor, and Julianna Margulies Commemorate 25 Years of Project ALS Research Progress

Entertainment Community Leaders Pay Tribute to Their Project ALS Family NEW YORK, Oct. 18, 2023 /PRNewswire/ -- Project ALS –non-profit 501(c)3 leader in the pursuit of treatments and a cure for the neurodegenerative disease ALS– in partnership with hosts Ben Stiller and Christine Taylor, celebrates 25 years of dedicated research and interindustry support at "Tomorrow is Tonight Gala." On Oct. 26th, the 25th annual fundraising event will take place at Jazz at Lincoln Center, part of New York City institution Lincoln Center, where the actor-actress couple, who have been committed to Project ALS since its founding in 1998, will welcome 500 attendees for an evening of research progress reports, dinner, music, and star studded appearances. Founding Project ALS family member Julianna Margulies will also participate. Ben Stiller and Christine Taylor, a member of the Project ALS board of directors, are the faces of the entertainment industry's unwavering support of the Project ALS mission. When Project ALS founder and up-and-coming theater producer, Jenifer Estess, received an ALS diagnosis in 1997, her dear friends, Ben Stiller, Julianna Margulies, and an extended troupe throughout the entertainment community bonded to bolster fundraising efforts. To date, Project ALS has raised nearly $120 million. Approximately 90% of every dollar raised is directed to Project ALS research programs. "I'm looking forward to celebrating 25 years of progress with the Estess sisters and the extended Project ALS family. We've really moved the needle together, and I'm proud to be in this fight for a cure," said Ben Stiller. "When Jenifer called me after she was diagnosed, nobody was talking about ALS. Christine and I continue to honor her legacy and work for those who are in need of treatments and cures." "Ben Stiller, Christine Taylor, and Julianna Margulies anchor the Project ALS family. Their contributions to Project ALS research have broken ground. Today there are more promising ALS drugs in clinical trial and more in the pipeline. Ben, Christine and Julianna put us on the map," said Project ALS president, Meredith Estess. "My sister Jenifer had the power to bring people together, and Ben, Christine, and Julianna ensure that the mission stays at the forefront." They do not work alone. The Project ALS family has extended to include people with ALS, researchers and scientists, who identify and develop new ALS drugs–like prosetin and jacifusen–and thousands of committed donors worldwide. Actors Ebon Moss-Bachrach, Molly Ringwald, Project ALS board member Rob Morrow, are expected to attend. The Tomorrow is Tonight Gala is estimated to raise over $1.5 million. Money raised will go directly to leading scientists and research cohorts that specialize in the innovation of ALS and related disease therapies. Project ALS is a not-for-profit, 501(c)3 whose mission is to identify and fund ALS and related research toward effective treatments and a cure. Project ALS was founded in 1998, by Jenifer Estess, who was diagnosed seemingly out of nowhere with ALS. A close relative of Alzheimer's, Parkinson's, and Huntington's, ALS is a devastating neurodegenerative disease that targets motor neurons, the brain cells that control nearly all human movement. To date, Project ALS has raised over $120 million, and is the first non-profit that has devoted 100 percent to ALS and related research. SOURCE Project ALS

03 May 2023

·www.prnewswire.com

Ionis reports first quarter 2023 financial results

QALSODY approved for SOD1-ALS; MAA under review in EU Reported positive Phase 3 eplontersen ATTRv-PN data; December 22, 2023 PDUFA date On track to achieve 2023 financial guidance CARLSBAD, Calif., May 3, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the first quarter of 2023. Financial results are summarized below: Financial Highlights Revenue for the first quarter of 2023 was in line with expectations and included revenue from numerous diverse sources Operating expenses increased in the first quarter of 2023 compared to the prior year as planned, reflecting investments in advancing Ionis' pipeline, technology and go-to-market activities for eplontersen, olezarsen and donidalorsen Cash and short-term investments of $2.3 billion at March 31, 2023 enables continued investment in creating future growth opportunities Reaffirmed 2023 financial guidance "2023 is off to a strong start. With QALSODY's approval, it joins SPINRAZA as a new groundbreaking medicine to treat a devastating neurological disease, further validating our RNA-targeting therapeutic platform. We also achieved another important milestone with our recent positive eplontersen Phase 3 data. We believe the positive efficacy and safety data, and the attractive self-administered dosing profile, position eplontersen to be an important treatment for ATTRv-PN patients, who today are underserved. We look forward to the first potential approval of eplontersen in the U.S. in December," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In addition, we further expanded our industry-leading late-stage pipeline to seven programs across nine indications following the start of GSK's bepirovirsen hepatitis B program. In the second half, we plan to report results from our olezarsen Phase 3 FCS study, which if positive, positions us for our first independent commercial launch. These recent achievements, together with our upcoming milestones, continue to build value for Ionis stakeholders." Recent Late-Stage Pipeline Highlights FDA granted Biogen accelerated approval of QALSODY (tofersen) for patients with SOD1-ALS FDA accepted eplontersen NDA for patients with polyneuropathy caused by hereditary TTR amyloidosis (ATTRv-PN) with a PDUFA date of December 22, 2023 Presented positive week-35 and week-66 data from the Phase 3 NEURO-TTRansform study of eplontersen in patients with ATTRv-PN GSK advanced bepirovirsen into Phase 3 development in patients with chronic hepatitis B Recent Additional Pipeline Updates Biogen presented data demonstrating IONIS-MAPTRx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer's disease Continued to focus R&D efforts by discontinuing two programs that did not meet Ionis' target product profile, cimdelirsen for acromegaly and sapablursen for beta-thalassemia. Ionis continues to advance the Phase 2 sapablursen study for polycythemia vera First Quarter 2023 Financial Results "Our first quarter results were in line with our expectations. We generated meaningful revenue while continuing to invest in key growth opportunities across our business. These results keep us on track to achieve our 2023 financial guidance," said Elizabeth L. Hougen, chief financial officer of Ionis. "We plan to continue investing in areas with the greatest potential to drive growth. As such, we expect our investments to grow modestly as we advance and expand our late-stage pipeline and move our near-term commercial opportunities toward the market. Additionally, as we keep more programs for ourselves, we expect a greater proportion of commercial revenues compared to R&D revenues, and our commercial revenues to be the primary driver of future revenue growth." Revenue Ionis' revenue was comprised of the following: Ionis continued to derive its revenue for the first quarter of 2023 from diverse sources, with approximately half coming from commercial products and half from numerous partnered programs. Commercial revenue for the first quarter of 2023 included $50 million from SPINRAZA royalties. Global SPINRAZA product sales of $443 million decreased six percent in the first quarter of 2023, compared to the same period last year primarily due to the impact from foreign currency, fewer new patient starts in the U.S. and channel dynamics. R&D revenue for the first quarter of 2023 included $24 million from AstraZeneca for its share of the global Phase 3 development costs for eplontersen, $20 million from Biogen for advancing several neurology disease programs and $15 million from GSK for advancing bepirovirsen into Phase 3 development. Already in the second quarter, the Company earned $16 million in a milestone payment from Biogen when QALSODY was approved in the U.S. Operating Expenses Ionis' operating expenses increased in the first quarter of 2023 compared to the same period in 2022, consistent with expectations. As Ionis advanced its robust pipeline, study costs increased as most of the Company's Phase 3 studies were either fully enrolled or approaching full enrollment resulting in higher R&D expenses year over year. Additionally, as Ionis prepares to launch eplontersen, olezarsen and donidalorsen, the Company's SG&A expenses also increased year over year. Balance Sheet As of March 31, 2023, Ionis' cash, cash equivalents and short-term investments increased to $2.3 billion compared to $2.0 billion at December 31, 2022 primarily due to the $500 million Ionis received from Royalty Pharma in January 2023. Ionis' working capital also increased over the same period primarily due to the Company's higher cash and short-term investments balance. Additionally, the Company recorded a long-term liability for future royalties due to Royalty Pharma in the first quarter of 2023. Webcast Management will host a conference call and webcast to discuss Ionis' first quarter 2023 results at 11:30 a.m. Eastern time on Wednesday, May 3, 2023. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company's first quarter 2023 earnings slides click here. About Ionis Pharmaceuticals, Inc. For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies. To learn more about Ionis visit or follow us on Twitter @ionispharma. Ionis' Forward-looking Statement This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of QALSODY (tofersen), SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen, ION363, pelacarsen, bepirovirsen, Ionis' technologies and Ionis' other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2022, which is on file with the Securities and Exchange Commission. Copies of this and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. QALSODY™ is a trademark of Biogen. SPINRAZA® is a registered trademark of Biogen. Reconciliation of GAAP to Non-GAAP Basis As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' non-GAAP results is consistent with how Ionis' management internally evaluates the performance of its operations. 2023 Key Value Driving Events(1) SOURCE Ionis Pharmaceuticals, Inc.

Clinical ResultPhase 3Drug ApprovalFinancial Statement

100 Deals associated with Ulefnersen

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Phase 3	IT	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	NL	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	TW	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	CH	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	GB	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	BE	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	CA	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	US	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	PL	Ionis Pharmaceuticals, Inc.	14 Jun 2021
Amyotrophic Lateral Sclerosis	Phase 3	ES	Ionis Pharmaceuticals, Inc.	14 Jun 2021

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