NASDAQ Framework: Ionis Pharmaceuticals Inc.

RNA-targeted therapeutics developer with antisense technology 1 2.

Ionis Pharmaceuticals Inc., founded in 1989 and headquartered in Carlsbad, California, is a pioneering developer of RNA-targeted therapeutics using antisense technology 3 4. The company has a broad clinical and preclinical pipeline targeting a variety of diseases, including cardiovascular, metabolic, neurological, and rare diseases 5 6. Ionis has brought several drugs to market, including Spinraza for spinal muscular atrophy and Tegsedi for ATTR amyloidosis, through partnerships and its cardiovascular-focused subsidiary Akcea 7 8. The firm continues to innovate in RNA therapies and gene editing, driven by a deep understanding of disease biology and a commitment to delivering life-changing advances for patients 9.

3: Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core and Akcea Therapeutics segments. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The Akcea Therapeutics segment develops and commercializes drugs for cardiometabolic diseases. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli and Brett P. Monia in 1989 and is headquartered in Carlsbad, CA. View Source4: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source5: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source6: Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). View Source7: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source8: Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). View Source9: About Ionis Pharmaceuticals, Inc.For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn. View Source

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Ionis Pharmaceuticals Inc. was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli, and Brett P. Monia in 1989. While Brett P. Monia currently serves as the Chief Executive Officer, there is no indication that the other founders are still heavily involved in the company's leadership 10.

10: Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core and Akcea Therapeutics segments. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The Akcea Therapeutics segment develops and commercializes drugs for cardiometabolic diseases. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli and Brett P. Monia in 1989 and is headquartered in Carlsbad, CA. View Source

MEDIUM RISK

Ionis Pharmaceuticals Inc. has a promising pipeline and recent positive clinical trial results, which position it well for future growth 11 12 13 14. However, the company has historically been unprofitable, and RNA-based therapies have faced long paths to market 15. Additionally, the competitive landscape for RNA-based therapies is intense, with strong competition from other firms 16. While Ionis has secured significant collaborations and funding, such as the deal with AstraZeneca 17 18, the inherent risks in drug development and commercialization, combined with past financial performance, suggest a medium risk level.

11: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source12: Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome PR Newswire Jul 22, 2024 11:00am Ionis to hold second quarter 2024 financial results webcast PR Newswire Jul 19, 2024 12:24pm Ionis completes enrollment in pivotal trial evaluating zilganersen in people living with Alexander disease PR Newswire Jul 18, 2024 11:00am Ionis announces webcast to report HALOS study results for ION582 in Angelman syndrome PR Newswire Jul 8, 2024 11:05am Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed PR Newswire Jun 25, 2024 10:55am Alnylam's stock surges as heart drug vies with a Pfizer blockbuster MarketWatch Jun 24, 2024 1:12pm Ionis announces expanded licensing agreement with Otsuka in Asia Pacific for investigational medicine donidalorsen in hereditary angioedema PR Newswire Jun 18, 2024 11:00am Ionis to present at TD Cowen Genetic Medicines & RNA Summit PR Newswire Jun 17, 2024 8:09pm View Source13: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source14: About Ionis' Neurology FranchiseIonis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUATM (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 11 therapies, of which five are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including amyotrophic lateral sclerosis (ALS) and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. View Source15: Beyond recent strong collaboration revenue, Ionis has historically been unprofitable, and RNA-based therapies have had a long path to market. View Source16: Ionis is a leader in RNA-based therapies, and its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat. View Source17: AstraZeneca has entered into a new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). View Source18: AstraZeneca and Ionis sign deal to develop and commercialise eplontersen View Source

YES

Ionis Pharmaceuticals Inc. is launching new product offerings. They are advancing ION582 for Angelman syndrome, with plans to initiate Phase 3 development in the first half of 2025 19. They also have several other medicines in development, including eplontersen, olezarsen, and donidalorsen, which are in various stages of clinical trials and regulatory review 20 21 22.

19: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source20: AstraZeneca has entered into a new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). View Source21: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source22: FDA Accepts Ionis' (IONS) NDA for Rare Disease Drug Olezarsen View Source

Ionis Pharmaceuticals Inc. has a robust product roadmap and expansion strategy for 2024 through 2026, focusing on advancing its pipeline of RNA-targeted therapeutics. Key initiatives include the planned Phase 3 development of ION582 for Angelman syndrome in the first half of 2025 23 24, and the continued progression of other promising candidates such as eplontersen, olezarsen, and donidalorsen, which are in various stages of clinical trials and regulatory review 25 26. The company is also leveraging strategic collaborations, such as its partnership with AstraZeneca for the development and commercialization of eplontersen 27 28. These efforts are aimed at expanding Ionis' market presence and delivering innovative treatments for a range of serious diseases.

23: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source24: Ionis plans to meet with regulators to review and confirm their Phase 3 study design later this year, which puts the company on track for a pivotal study initiation in H1 2025. View Source25: FDA Accepts Ionis' (IONS) NDA for Rare Disease Drug Olezarsen View Source26: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source27: AstraZeneca and Ionis sign deal to develop and commercialise eplontersen View Source28: Financial considerations AstraZeneca will pay Ionis an upfront payment of $200m and additional conditional payments of up to $485m following regulatory approvals. It will also pay up to $2.9bn of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region. The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions. View Source

Sources

1: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source2: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source3: Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core and Akcea Therapeutics segments. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The Akcea Therapeutics segment develops and commercializes drugs for cardiometabolic diseases. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli and Brett P. Monia in 1989 and is headquartered in Carlsbad, CA. View Source4: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source5: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source6: Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). View Source7: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source8: Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). View Source9: About Ionis Pharmaceuticals, Inc.For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn. View Source10: Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core and Akcea Therapeutics segments. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The Akcea Therapeutics segment develops and commercializes drugs for cardiometabolic diseases. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli and Brett P. Monia in 1989 and is headquartered in Carlsbad, CA. View Source11: Ionis Earnings: Maintaining Our $62 Fair Value Estimate as Series of New Launches Begins Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 2 months ago Daily – Vickers Top Buyers & Sellers for 04/18/2024 The Vickers Top Buyers & Sellers is a daily report that identifies the five companies the largest insider purchase transactions based on the dollar value of the transactions as well as the five companies the largest insider sales transactions based on the dollar value of the transactions. Argus• 3 months ago Ionis Earnings: Maintaining $62 Fair Value Estimate; Wainua a Precursor to Two Independent Launches Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 4 months ago Ionis Earnings: Third-Quarter Results Show Growing Late-Stage Pipeline; Shares Remain Undervalued Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Rating Price Target Morningstar• 8 months ago View Source12: Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome PR Newswire Jul 22, 2024 11:00am Ionis to hold second quarter 2024 financial results webcast PR Newswire Jul 19, 2024 12:24pm Ionis completes enrollment in pivotal trial evaluating zilganersen in people living with Alexander disease PR Newswire Jul 18, 2024 11:00am Ionis announces webcast to report HALOS study results for ION582 in Angelman syndrome PR Newswire Jul 8, 2024 11:05am Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed PR Newswire Jun 25, 2024 10:55am Alnylam's stock surges as heart drug vies with a Pfizer blockbuster MarketWatch Jun 24, 2024 1:12pm Ionis announces expanded licensing agreement with Otsuka in Asia Pacific for investigational medicine donidalorsen in hereditary angioedema PR Newswire Jun 18, 2024 11:00am Ionis to present at TD Cowen Genetic Medicines & RNA Summit PR Newswire Jun 17, 2024 8:09pm View Source13: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source14: About Ionis' Neurology FranchiseIonis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUATM (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 11 therapies, of which five are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including amyotrophic lateral sclerosis (ALS) and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. View Source15: Beyond recent strong collaboration revenue, Ionis has historically been unprofitable, and RNA-based therapies have had a long path to market. View Source16: Ionis is a leader in RNA-based therapies, and its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat. View Source17: AstraZeneca has entered into a new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). View Source18: AstraZeneca and Ionis sign deal to develop and commercialise eplontersen View Source19: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source20: AstraZeneca has entered into a new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). View Source21: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source22: FDA Accepts Ionis' (IONS) NDA for Rare Disease Drug Olezarsen View Source23: "Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs." AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain. View Source24: Ionis plans to meet with regulators to review and confirm their Phase 3 study design later this year, which puts the company on track for a pivotal study initiation in H1 2025. View Source25: FDA Accepts Ionis' (IONS) NDA for Rare Disease Drug Olezarsen View Source26: Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California. View Source27: AstraZeneca and Ionis sign deal to develop and commercialise eplontersen View Source28: Financial considerations AstraZeneca will pay Ionis an upfront payment of $200m and additional conditional payments of up to $485m following regulatory approvals. It will also pay up to $2.9bn of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region. The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions. View Source