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Ionis Pharmaceuticals Company Profile | Drug Discovery & DDS

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Company Profile

Ionis Pharmaceuticals
— A biotech that spent 30 years developing antisense oligonucleotides and moved from licensing its technology to selling its own medicines

Ionis PharmaceuticalsAntis Nucleic Acid (ASO)This is a specialized biotech for nucleic acid drugs. I have long earned in the form of “Find drugs and pass them to major pharmaceuticals and receive loyalty.” TRYNGOLZA was approved in December 2024.Company selling in-houseContact Us The net sales of 2025 is US$9,373 million and the net income is US$38,140 million. In this article,Chemical modification of nucleic acid and GalNAc binding (LICA) to deliver to the liverWe will organize the core of the technology and the affiliation surrounding it with the following information.

Composition: Form 10-K (submitted February 26, 2026), Form 8-K (submitted July 29, 2026), Form 8-K (submitted July 29, 2026), Form 8-K (submitted July 29, 2026), Form 8-K (submitted July 29, 2026)

In this article
  1. Ionis in 30 seconds and standing position in drug discovery and DDS
  2. About logo and company name
  3. Where is this company in the world of drug discovery and DDS?
  4. Company size
  5. Drug discovery and DDS resources
  6. Past Initiatives
  7. Future plans
  8. What is DDS?
  9. Collaboration mapping of drug discovery and DDS
  10. Technologies, Products and Services
  11. The risk of this company
  12. Glossary/Source/Ins ment and Source Response Table

1. Ionis in 30 seconds and standing position in drug discovery and DDS

NameIonis Pharmaceuticals, Inc.Changed company name from I Pharmaceuticals in December 2015
HeadquartersCarlsbad, California, USAEstablished in January 1989 to Delaware Corporation
ListingNasdaq: IONSUS company to submit 10-K to SEC
Categories in this fieldDrug discovery biotech
(Nuclear acid modalities)
Biotech, a commercial stage that began selling in-house
Core modalityAntis Nucleic Acid (ASO)siRNA and gene editing
Sales (FY2025)US$9,437 millionUS$75.15 million
Research/Development/Patent CostUS$956 millionSame as sales
Number of commercial drugs7 items10-Q for FY2026 No ZANVASTRO approved in September
1 point to press

Sales of Ionis"Payment from other companies" is still larger than "Sales of our products"The sales of the company's products is USD 1,530 million, The R&D revenue from Biogen’s SPINRAZA and other royalties was $285.55 million, and the R&D revenue from partners was $57.9 million. FactsHome Product sales stay at about 12.2% overall The calculation of this paperHome In other words,Biotech, a technology-based technology based on the design, chemical modification and delivery of nucleic acid drugs to major pharmaceuticalsHome I started selling medicine using the technology.

2. Logo and company name

Ionis Japan Ltd. is a trademark of the company. In this article, we do not reproduce the generated image, Images obtained from the official websiteHome img/ionis_logo.png Contact Us

Note: Corporate name Ionis Pharmaceuticals, Inc.(Delaware Corporation) December 2015 Isis Pharmaceuticals, Inc. The old papers and patents appear in the old company name. In Japanese, it is written as "Ionis". WAINUA is the name WAINZUA in Europe.

Official Site:https://www.ionis.com/

3. Where is this company in the world of drug discovery and DDS?

3 types of companies involved in drug discovery and DDS In the same "pharmacy company", it is completely different where the drug is made Pharmaceutical Company Drug discovery biotech Research and development From search to approval and sales Create candidates with new modalities and methods Contract development and manufacturing of other drugs Eli Lilly/Novo Nordisk Pfizer Alnylam/Arvinas Moderna/Insilico Lonza/Samsung Biologics AppTec Secondary clinical and sales network funding Specific technical base depth Mass production capability for equipment and regulation Sales can be reduced rapidly due to the lack of patents Rely on partnering with funds for late development Disco・ation of customer development and impact of policy Ionis is a central drug discovery biotech. Technical foundations are chemical modification of antis nucleic acid and delivery to the liver Since December 2024, we have introduced many candidates to major pharmaceutical companies.
Figure 1 3 types of companies involved in drug discovery and DDS and the position of ionis. The classification is based on this article, and the company name is an example from the company handled by this series. The calculation of this paperHome Ionis is a drug discovery company targeting RNA. In 2025, it is based on Form 10-K for the fiscal year ending December 2025.

TypeDrug BiotechComment 10-K writes its company as a pioneer of pharmaceutical products targeting RNA. All 7 products are oligonucleotides (sh nucleic acids). FactsHome Center modalitiesAntis Nucleic Acid (ASO)In 10-KsiRNA and gene editingThe ability to add FactsHome The mechanism of nucleic acid drugs is handled by the technical commentary "nucleic acid medicine" of this series, and the difficulty of delivering to organs other than the liver is "DDS and targeting" and "blood brain barrier".

4. Company Scope

Indicators (US Accounting Standards)FY2024FY2025January to June 2026
Sales (USD million)705.1943.7514.0
Sales of products (USD million)―115.374.1
Royalty (Million USD)257.3285.5134.3
Research/Development/Patent Cost (USD million)901.5915.6427.3
Net Income (USD million)△453.9△381.4△207.2
Cash, cash equivalents, short-term investment (USD million)2,297.72,677.4―

TRYNGOLZA・DAWNZERA Cash balance between January and June 2026 is not confirmed in this article.

Our employees1,402 people(As of February 19, 2014), the majority of them are in the United States FactsHome 2025 sales ended 33.8% year on year The calculation of this paperHome sapablursen to Ono Pharmaceutical IndustryUS$28,000 millionIncluded FactsHome The company explains that the sales of January to June 2026 fell below the previous year’s。hronization ($583.73 million) because this temporary money was no longer available.

Contact Us

Sales of IonisYou can move largely every year with temporary money or milestone from partnersHome It is not possible to sell medicines that have increased sales. You need to separate your product sales and loyalty.

5. Drug discovery and DDS resources

Resource Typesation
Person: Research CenterResearch Institute and Office in Karlsbad, California (fee 17,6,300 sq. ft. and 2,800 sq. ft.) Office in Dublin, IrelandFacts
Person:1,402 employees (as of February 19, 2026). No number of people in the production and development departmentFacts
Gold: R&D expensesUS$956 million in research, development and patent costs (2025) US$1,140 million, US$5,870 millionFacts
Gold: Manufacturing EquipmentCarlsbad’s own manufacturing wings (26,000 square feet) and manufacturing support wings (25,000 square feet). We have produced the majority of original drugs for R&D in-house, and we have produced original drugs for approved drugs and commercial products. No new investment in manufacturing equipmentFacts
External: Investment and TechnologyBicycle The)eutics (Institute a technical license in 2021 and invest as part of payment), Metagenomi (2022, genetic editing),ect-Horus (2023, December)Facts
External: AcquisitionAcquisition of company in drug discovery and DDS fieldNot specified
Graduate SchoolPatents for SPINRAZA exclusively from Cold Spring Harbor Laboratory and the University of Massachusetts to pay royalty for the first half of salesFacts
Public FundsGovernment Research GrantsNot specified
Perspective of material ians: “Creating another drug with the same component”

10-K is the drug of oligonucleotidesame nucleotide unit variants and same type of equipmentto make I wrote that the method of efficiency by one drug has been effective in other medicines, and the raw material cost has been greatly reduced by improving yield and process efficiency. FactsHome In the case of antibody medicine, we regenerate the cell stock and culture and purification conditions for each product."Change the array" of solid phaseClose The penetration is transferred to the cost of amidaito and solid-phase carriers, the amount of sulfide and solvent used, and the yield in refining. For this reason, "special chemicals for oligonucleotide manufacturing" is listed in the explanation of the manufacturing and development chemical costs of 2025. Facts。

6. Past Initiatives

TimeHomeThe meaning of drug discovery and DDS
January 1989I PharmaceuticalsANTI as an antis nucleic acid
Year 2010Partner with GSK B hepatitis bepirovirsen in 2019In ious Diseases
Year 2012Joint Development Agreement with Biogen and SPINRAZAASO directly administers the spinal cord to the largest royalty source
Year 2015Partnership with AstraZeneca with cardio。, kidney and metabolic diseases. Mutual license of Alnylam and 4。 rights. Changed to the current company in DecemberExpanding Joint Drugs with Majors
Year 2017Partnership between Nov。s andaacarsen. Buy Carlsbad's Manufacturing BuildingOur own manufacturing base
Year 2018Contract betweenef and sefaxersen. Biogen acquires QALSODY rightsDevelopment of kidneys and nerves
Year 2021AstraZeneca and eplontersen (WAINUA) co-development agreement. Exercise Bicycle technical license (including investment)Liver delivery technology to non-liver (muscle/brain)
2022 yearCollaboration between Metagenomi and gene editingAdd a way to rewrite DNA directly
December 2023Vect-Horus introduces brain migration technology exclusively. Otsuka and DAWNZERA partner in EuropeTechnology to aim at breakthrough of blood brain barrier
January 2024Launched WAINUA in the US with AstraZenecaSelf-injectable drugs for GalNAc binding (LICA)
December 2024TRYNGOLZA(Family hyperchromicemia syndrome)First-time Own Products
March 2025pad sapablursen in Ono Pharmaceutical Industry (T。 amount US$28,000 million). TRYNGOLZACo ation model
August 2025DAWNZERA (donidalorsen) approved by the U.S.2nd In-house Sale
June 2026TRYNGOLZA Approved by the U.S. with Severe TriSelevride Hemo (sHTG)First in-house sales in disease with large number of patients
July 2026CARDIO-TTRansform phase III trial with AstraZeneca (ATTR myocardia) does not achieve key evaluation itemsInverse style to expand adaptation of partner products
September 2026ZANVASTRO (zilganersen) approved for Alexander's disease (September 3). Novartis' pelacarsen Phase III Lp(a)HORIZON did not achieve the main evaluation items (September 4). Alnylam's sefaxersen phase III achieved key evaluation items in an interim analysis (September 23)In-house products are moving forward and affiliated products are divided

Clinical trial example: Clinical trial/HT2(sHTG)

This test was based on the expansion of TRYNGOLZA adaptation in June 2026. FactsHome The number is quoted from the company's official announcement,Does not recommend specific treatment。

Test name(NCT05079919, 617 examples) and 2 (NCT05552326。46 examples). Phase III, randomization, double blindness, placebo contrast
Over 18 years of age receiving standard treatment withTriSelevride 500mg/dL or higher
olezarsen 50mg or 80mg, or subcutaneous injections per 4 weeks, 12 months
Main evaluation itemsChanges inTriSelevrides. Achieve both trials and doses
Result (Company Presentation)6 monthsCompared to placeboTriSelevrides are reduced by up to 72% and maintained until 12 months

7. Future plans

In-house salesTRYNGOLZA・DAWNZERA
ZANVASTRO
Sales in the US
Phase III2 items
Partner 8
June 2026 10-Q
Company ProfileobudanersenAngelmann syndrome. Completion of Phase III
Featured Productsbepirovirsen
sefaxersen
GSK・Roche Announces Major Evaluation Items in Phase III

The company says that the company expects to expand its product sales and loyalty by the end of 2027. Undetermined / FutureHome The items under development are as follows:Not confirmed。

ProductsStage
obudanersenAngelmann syndrome (intrathenal administration)Developed (phase III). CompanyUndetermined / Future
ulefnersenMuscle atrophic side curing by FUS mutationsDeveloped (phase III). Otsuka Pharmaceutical Co., Ltd.Undetermined / Future
bepirovirsen[Type 2] Hepatitis BUnder development GSK Announces Good Major Results for Phase III B-Well 1 & 2 in January 2026Undetermined / Future
sefaxersenIgA renal diseaseDeveloped (Phase III). Results announced good results of an interim analysis in September 2026Undetermined / Future
ION826(AZD4063)The first program to use Bicycle technologyDeveloped (phase I). AstraZenecaUndetermined / Future
Not confirmed

None of the following are listed in the primary information Undetermined / Future。

ProductsSchedule of application and approval(Until the end of 2027)/ Investment plan for manufacturing equipment/MsPA skeletal use clinical psychologyName and target disease/ Our productsSales forecastHome This article does not adopt sales forecasts for media and s.

8. What is this company aiming for drug discovery and DDS?

This includes parts that the company does not directly state.Calculation in the range that can be read from primary informationand write with the basis.

ReadPrimary Information
From "Draw out"Move axis foot to "sold by yourself"10-K has been transferred to a fully integrated commercial phase biotech. In 2026, the company was sold to three items.Facts
Next technical battlefieldDelivery outside the liverBicycle two delivery technologies of Bicycle (to muscle and brain) andect-Horus (to the brain)
Not sticking to ASOIncrease the means of nucleic acid and insurance10-K has added siRNA and genetic editing capabilities and partnered with Metagenomi
In the generation of chemical modificationDifferent of dose interval and safetySearchMsPA skeleton enhances metabolic stability and reduces non-specific protein binding and 10-K explains
Large-scale in-house salesTwo areas of nerve and cardio andClose10-K indicates the priority area as "neural, cardio」 and」, and other high-needs"Facts
Only one basis for guessing: "Outside the liver"

TRYNGOLZA, WAINUA, DAWNZERAASO that combines ligand with three GalNAc (N-acetyl galactosamine) residuesHome It is written in the attached document when it is designed to be delivered to liver cells FactsHome On the other hand, the drug of the nerve (SPINRAZA, QALSODY, ZANVASTRO) is directly injected into the myeloid FactsHome In other words, current products"Injection of subcutaneous injection to the liver, direct injection to the brain and spinal cord"There are only two ways. 10-K expands LICA to skeletal muscles and cardiac muscles,Possibility to cross the blood brain barrierI'm writing FactsHome The following technical wins of IonisDelivering to muscles and brains through systemic administrationread Undetermined / FutureHome However, ION826 using Bicycle technology is still phase I and does not know the clinical condition.

9. Collaboration mapping of drug discovery and DDS

Partnership with Ionis-based drug discovery and DDS Form 10-K・10-Q・8-K No media-based relationships Ionis Nucleic Acid Biotech Biogen SPINRAZA・QALSODY Roche sefaxersen GSK Bepirovirsen Ono Pharmaceutical Industry sapablursen (March 2025) AstraZeneca Joint development and joint sales of WAINUA Novartis lacarsen Otsuka Pharmaceutical Sales of DAWNZERA Europe and Asia Sobi(TRYNGOLZA) Sales outside U.S., Canada and China Bicycle Therapeutics Technology for delivery to muscle and brain + investment Metagenomi・Vect-Horus technological editing and brain migration Joint Development and out Sales and Distribution Technology introduction with investment Technology
Figure 2 Collaboration between drug discovery and DDS centered on ionis. Source: Form 10-K (Collaborative Arrangements), Form 10-Q, June 2026. AstraZeneca・Biogen・GSK・Roche・Nov。s The relationship between Alnylam and Alnylam is handled in the body.

10. Technologies, Products and Services

Ionis TechnologyArray design of short guanine nucleic acid (oligonucleotide)、 Chemical modification to prevent decomposition、technology to deliver to organsmade of three layers Facts。

ProductStructure and Delivery2025 sales and revenueSales
SPINRAZA(nusinersen)2′-O-Methoxyethyl(2′-MOE) modifier and ASO of phosphatioate binding. Intramyal administrationRoyalty US$21,230Biogen
WAINUA(eplontersen)ASO (LICA) combined with ligands with three GalNAc residues. Self injection once a monthRoyalty4,910 million USDCollaboration with AstraZeneca (USA)
TRYNGOLZA(olezarsen)GalNAc binding ASO. targets mRNA of apoC-III. Self injection once a monthUS$17.5 millionCompany (USA)
DAWNZERA(donidalorsen)GalNAc binding ASO. Targeting precali crane. Self injection per week or 8 weeksProduct sales: $78 millionIn-house (USA), Otsuka Pharmaceutical (Europe, Asia)
ZANVASTRO(zilganersen)RNA-targeted drugs that reduce production of GFAP. Intrathecal administration per 3 monthsApproved in September 2026Company (USA)

The technology generation follows from the patent list of 10-K. 2′-MOE, binding ethyl (cEt), etc.ification of sugar contentHome Join GalNAcLICA Technology(main U.S. and European patent expiration 2034), and newsilylphosphoramide (MsPA) boneHome FactsHome LICA patents not only ASOBinding to bi-strand siRNA10-K is written if included.

Perspective of Material Engineers: Directions to Reduce the Number of Sulfurs in the Skeleton

In addition to the molecular formula of the U.S. attachment, GalNAc binding ASO with the same 20Lipinrus s, 19 olezarsen , 15 donidalorsen13, 13 eplontersenHome FactsHome Since ASO、 is mainlyPS from phosphatioate (PS) bonds, New drug"Mixed skeleton" which returns part of PS bond to normal phosphate diester bondRead if Undetermined / FutureHome PS binding makes it easy to stay in the body by binding to decomposition proteins, but also causes side effects from non-specific bindings. 10-K writes that “increases metabolic stability and reduces non-specific protein bindings that cause inflammatory reactions,” as MsPA skeleton benefits.Resolve the merits of PS bonds by the modifier itselfPositioned as an attempt Undetermined / FutureHome In terms of production, it is necessary to switch the sulfide process and the PO (oxidation) process to each position of the array, and it should be pressed that the difficulty of process management increases.

11. Risks that this company has

Risks
Red letterNet income is US$4,390 million in 2024, US$3,814 million in 2025, US$2,720 million in 2026Facts
Concentration of RoyaltySPINRAZA’s royalty of US$23,30 million (approx. 48.7%) out of US$43,580 million in commercial revenue in 2025The calculation of this paper
Depends on the test result of the partnerCARDIO-TTRansform with AstraZeneca in 2026,Novacarsen Lp(a)HORIZON in Nov)s does not achieve key evaluation itemsFacts
Sales swingIn 2025, the company swelled in US$28,000 million, and in January to June 2026, it was less than the previous year.Facts
Launch of own salesSales and general administrative expenses (segment disclosure outside of stock compensation) increased to US$3,052 million in 2025. Priority of sales system costsFacts
Manufacturing external dependenciesWe also use contract manufacturers (CMO) for the manufacture of phase III products and commercial products.Facts
Technical patent deadlineThe patent of major patents of LICA technology was 2034. The transition to the next generation of MsPA skeleton is questioned

12. Glossary

Antis Nucleic Acid (ASO)
A short single-stranded nucleic acid that complements the target RNA. It can either complement RNA or alter how it is read.
siRNA
Short RNA of two chains. Decompose target mRNA using the mechanism in the cell (RNA ).
GalNAc
N-acetyl galactosamine. It is a sugar that binds to the receptor of the hepatocellular system, and the nucleic acid is easily delivered to the liver when three are attached.
LICA
LIgand-Conjugated Antis . The name of the technology that Ionis joins ligands such as GalNAc to ASO.
2′-MOE
Qualification to replace 2′ of nucleic acid sugar with metoxyethyl base. It is difficult to decompose and strong binding to targets.
Phospho (PS) binding
Oxygen in the phosphate connecting nucleic acid with . It prevents decomposition, but also non-specific protein binding.
MsPA Skeleton
Mesilhos Holami Dating. The new skeletal chemistry developed by Ionis to improve metabolic stability.
Intramyal administration
How to directly inject the liquid that is the spinal cord from the spine. Used in nucleic acid drugs of brain and spinal cord disease.
Royalty
The price to receive according to the sales of the partner who reflects the rights of technology and medicine.
Conceptual diagram: The nucleic acid of a short single chain is delivered by a small syrup of sugar, and the appearance of the heading to the cells of the liver and delivering it to the brain and spinal cord in another route.Concept diagram of AI generation
Figure 3 Concept diagram of two paths that deliver antis nucleic acid to organs (a concept diagram of AI generation). Actual structure and products are not copied).

13. Source

  1. Ionis Pharmaceuticals Form 10K for the) year ended December 31, 2025 Business Overview, Technology (MsPA・Bicycle・Vect-Horus・Metagenomi), Partnership Conditions, Manufacturing, Patents, Number of , Facilities, Sales and Costs https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
  2. Ionis Pharmaceuticals Form 10-Q for the quarterly period ended June 30, 2026 Sales, expenses, net profit and loss, market conditions, number of items of phase III, examination deadline of zilganersen from January to June 2026. https://www.sec.gov/Archives/edgar/data/874015/000087401526000251/form10q.htm
  3. Ionis Pharmaceuticals TRYNGOLZA (olezarsen) approved by the FDA ... in patients with hypertri eridemia (June 24, 2026). Design and results of / 2 trials. https://www.sec.gov/Archives/edgar/data/874015/000114036126026232/ef20076807_ex99-1.htm
  4. Ionis Pharmaceuticals・AstraZeneca "Update on CARDIO-TTRansform Phase 3 trial of eplontersen" https://www.sec.gov/Archives/edgar/data/874015/000114036126028092/ef20077664_ex99-1.htm
  5. Ionis Pharmaceuticals Form 8-K Exhibit 99.1 “ZANVASTRO (zilganersen) approved by the FDA ... for Alexander disease” (September 3, 2026). https://www.sec.gov/Archives/edgar/data/874015/000114036126035657/ef20081649_ex99-1.htm
  6. Ionis Pharmaceuticals Form 8-K Exhibit 99.1 “Ionis partner Nov)s Announcements Lp(a)HORIZON Phase 3 topline results for resultslacarsen” (September 4, 2026). https://www.sec.gov/Archives/edgar/data/874015/000114036126035802/ef20081690_ex99-1.htm
  7. Ionis Pharmaceuticals・Roche Form 8-K Exhibit 99.1 “Ionis announces positive interim data from partner Phase’s Phase 3 IMAgINATION study in IgA nephro)” (September 23, 2026). https://www.sec.gov/Archives/edgar/data/874015/000114036126037371/ef20082626_ex99-1.htm
  8. National Library of Medicine DailyMed TRYNGOLZA(olezarsen) Structure and 。 formula of GalNAc binding ASO. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0f51aa8e-8475-8cf9-e063-6394a90a6848
  9. National Library of Medicine DailyMed WAINUA(eplontersen) Structure and 。 formula of GalNAc binding ASO. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096
  10. National Library of Medicine DailyMed DAWNZERA (donidalorsen) Structure and 。 formula of GalNAc binding ASO. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3ff501e0-f75f-07da-e063-6294a90a0cb7
  11. National Library of Medicine DailyMed SPINRAZA (nusinersen) US attachment. 2′-MOE modifier and phosphatioate binding, intrathecal administration. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94
  12. Ionis Pharmaceuticals Official website (for confirmation of company profile and logo) https://www.ionis.com/

14. Response table of claims and sources

Content
The company name, the former company name and change time, established (January 1989), head office location, Nasdaq listedFactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
Sales and breakdown of 2024 and 2025, research, development, patent and breakdown, net income, cash, etc.FactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
Explanation of net sales and breakdown, research, development, patent costs, net income, and repayment due to the absence of temporary moneyFactsSource 2 https://www.sec.gov/Archives/edgar/data/874015/000087401526000251/form10q.htm
1,402 employees, facilities such as Karlsbad, manufacturing wings (purchase 2017)FactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
Explanation of the range of our own manufacturing, the production record of clinical trials and products to partner companies, the use of CMO, the efficiency of manufacturing and cost reductionFactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
Agreement with Biogen, AstraZeneca, GSK, Nov・s,che, Ono Pharmaceutical Co., Ltd., Otsuka Pharmaceutical Co., Ltd., Sobi, PTC, Cycle, Metagenomi,ect-Horus, AlnylamFactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
Introduction of SPINRAZA related patents from Cold Spring Harbor LaboratoryFactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
MsPA skeleton description, LICA patent (expired 2034, combined with siRNA), 2′-MOE/cEt patentFactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
TRYNGOLZA(December 2024)・DAWNZERA(August 2025)FactsSource 1 https://www.sec.gov/Archives/edgar/data/874015/000087401526000115/form10k.htm
TRYNGOLZA's sHTG approval (June 2026), the design and results of the 2nd examFactsSource 3 https://www.sec.gov/Archives/edgar/data/874015/000114036126026232/ef20076807_ex99-1.htm
CARDIO-TTRansform Phase III trial did not achieve key evaluation itemsFactsSource 4 https://www.sec.gov/Archives/edgar/data/874015/000114036126028092/ef20077664_ex99-1.htm
ZANVASTRO (zilganersen) US approval (September 3, 2026)FactsSource 5 https://www.sec.gov/Archives/edgar/data/874015/000114036126035657/ef20081649_ex99-1.htm
placarsen's Lp(a)HORIZON trial did not achieve the main evaluation itemFactsSource 6 https://www.sec.gov/Archives/edgar/data/874015/000114036126035802/ef20081690_ex99-1.htm
sefaxersen's Phase III IMAgINATION major evaluation itemsFactsSource 7 https://www.sec.gov/Archives/edgar/data/874015/000114036126037371/ef20082626_ex99-1.htm
TRYNGOLZA・WAINUA・DAWNZERA is an ASO that combines GalNAc 3 residue ligands, numbersFactsSource 8・9・10 https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0f51aa8e-8475-8cf9-e063-6394a90a6848
Chemical Qualification of SPINRAZA (2′-MOE, Phosoatiation) and intrathecal administrationFactsSource11 https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94
SPINRAZA royalty boosts sales by approximately 12.2% of sales, 33.8% of sales, about 48.7% of commercial revenueThe calculation of this paperThis document is calculated from the amount of source 1
Classification of type 3 (pharmaceu) company / drug discovery biotech / contract development manufacturing / research)The calculation of this paperThis article does not specify the company name. The company name is an example from the series of companies in this article.
Application and approval time of each product, investment plan of manufacturing equipment, MsPA clinical program name, sales forecast of original productsUndetermined / FutureNot listed in Source 1 and 2. Not in this article
Sales expansion by product launch until the end of 2027Undetermined / FutureSource 2 company and not proven https://www.sec.gov/Archives/edgar/data/874015/000087401526000251/form10q.htm
The next main battlefield reads that the delivery to the outside of the liver is increasing the means of nucleic acid and insurance.Interpre of this paper from the description of the technology and alliance of Source 1
Reading to differentiate between dose intervals and safety in generations of chemical modification, positioning mixed skeleton and MsPAExplanation of this article from the description of source 1, 8, 9, 10
Risks between LICA patent expiration and MsPA transitionInterpre of this paper from the list of patents of Source 1

Last Updated:September 26, 2026/Troy Technical
The number of this document is based on the statutory disclosure document (Form 10-K, 10-Q, 8-K) submitted by Ionis to the US Securities and Exchange Commission (SEC) and the US attachment (DailyMed). We do not use estimates and media-based numbers of survey companies. The "、ulation" section is an interpretation that the article reads from the primary information and is not an announcement of the company. This article is not intended for medical advice, but does not recommend the use of certain drugs.

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