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1. 02 August 2021 Lp(a) HORIZON achieves 50% enrollment in trial to assess the safety and efficacy of pelacarsen in reducing recurrent cardiovascular events

   CARLSBAD, Calif., Aug. 2, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that pelacarsen, formerly known as AKCEA-APO(a)-L_Rx, and licensed by Novartis (NYSE:NVS) for exclusive worldwide development and commercialization,  continues to advance in the clinic as the potential first-in-class treatment for lipoprotein(a) or Lp(a)-driven cardiovascular disease. The Novartis pivotal Phase 3 cardiovascular outcomes study of pelacarsen, Lp(a) HORIZON (NCT04023552), has reached 50% enrollment with a target goal of 7,680 trial participants.

   

   Pelacarsen is an investigational antisense medicine that uses Ionis' proprietary Ligand Conjugated Antisense (LICA) technology platform. It is designed to inhibit the production of apolipoprotein(a) in the liver to target elevated Lp(a) levels, an independent genetic risk factor for cardiovascular diseases (CVD), that are determined at birth and cannot be controlled with diet or exercise. High Lp(a) levels are associated with significant risk of cardiovascular disease, including heart attacks and strokes. There are no approved pharmacological therapies to effectively lower Lp(a).

   "We are pleased by Novartis progress in advancing the Lp(a) HORIZON study and enrolling nearly 4,000 study participants around the world. Pelacarsen represents a potential first-in-class treatment to address a significant unmet need with the potential to become the new standard of care for Lp(a)-driven cardiovascular disease," said Sotirios "Sam" Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis, who specializes in Lp(a). "Ionis' vision is that pelacarsen emerges as an effective therapy to improve cardiovascular outcomes through normalizing Lp(a) levels, as there are an estimated eight million people globally living with elevated Lp(a) and cardiovascular disease."

   Data from a Phase 2 study published in the New England Journal of Medicine showed pelacarsen provided potent dose-dependent reductions of Lp(a) compared to placebo, with a favorable safety and tolerability profile in patients who had elevated Lp(a) levels and established CVD. These data support the potential of antisense-mediated reduction of Lp(a) with pelacarsen.

   Ionis earned a $25 million milestone payment from Novartis for achieving 50% enrollment in the pivotal Phase 3 study. In 2017, Novartis entered a collaboration agreement with Ionis for pelacarsen. In February 2019, Novartis exercised an option to license the rights to develop and commercialize pelacarsen for targeted cardiovascular therapy for $150 million. Under the terms of the agreement, Novartis is exclusively responsible for worldwide development and commercialization of pelacarsen and Ionis is eligible to receive up to $675 million in regulatory and sales milestones. Ionis is also eligible to receive tiered royalties in the mid-teens to low 20% range on net sales of pelacarsen.

   Additional information about Lp(a) HORIZON may be found at www.ClinicalTrials.gov (NCT04023552). 

   About Lp(a) HORIZON

   Lp(a) HORIZON is a pivotal, global multicenter, double-blind, placebo-controlled pivotal Phase 3 study conducted by Novartis. The trial is designed to support an indication for the reduction of cardiovascular risk in patients with established CVD and elevated Lp(a) with 80 mg of pelacarsen administered monthly via subcutaneous administration. The study has a targeted enrollment of 7,680 participants. The estimated study completion date is in 2024.

   The primary objectives of the trial are to demonstrate superiority compared to placebo in reducing the risk of expanded MACE (major adverse cardiac events such as cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and urgent coronary re-vascularization requiring hospitalization) in the overall study population with established CVD and Lp(a) ≥ 70 mg/dL, and in the population with Lp(a) ≥ 90 mg/dL.

   About pelacarsen

   Pelacarsen, licensed by Novartis for exclusive worldwide development and commercialization, is an investigational antisense medicine designed to reduce apolipoprotein(a) production in the liver to offer a direct approach for reducing circulating lipoprotein(a), or Lp(a), an atherogenic, pro-inflammatory and thrombogenic lipoprotein that induces additional cardiovascular risk independent of LDL-cholesterol, in patients already treated with LDL-C-lowering therapies (such as statins or PCSK9 inhibitors). Elevated Lp(a) is recognized as an independent, genetic cause of coronary artery disease, heart attack, stroke and peripheral arterial disease. Currently, there is no effective drug therapy to specifically and robustly lower elevated levels of Lp(a). Lp(a) levels are determined at birth and, therefore, lifestyle modification, including diet and exercise, does not impact Lp(a) levels. Even patients with LDL-C lowered to target levels (<70 mg/dL) remain at high-risk of cardiovascular events if they have high levels of Lp(a).

   Pelacarsen is being developed for patients who are at significant risk of CVD because of their elevated Lp(a).

   About Lp(a)

   Lp(a) is a lipoprotein particle assembled in the liver that consists of an LDL-C-like particle and apolipoprotein(a). Lp(a) is considered a key driver for cardiovascular disease due to its association with an increased risk of coronary heart disease. There is evidence that elevated Lp(a) levels may contribute directly to coronary artery disease, heart attacks, strokes and peripheral artery disease. Lp(a) levels in the blood can vary greatly between individuals primarily due to genetic variations and do not correlate with LDL-C levels. Because elevated Lp(a) is a genetically determined condition that is not responsive to lifestyle changes, patients are unable to adequately control their Lp(a) levels through improved diet or increased physical activity. Moreover, current therapies are not able to normalize Lp(a) levels in patients who have high Lp(a). Although Lp(a) can be measured by a variety of reliable and readily available simple tests, the lack of drugs to effectively lower Lp(a) has made treating patients with Lp(a)-driven cardiovascular disease difficult.

   About Ionis Pharmaceuticals, Inc.

   For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

   To learn more about Ionis, visit www.ionispharma.com and follow us on twitter @ionispharma.

   Ionis' Forward-looking Statement

   This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of Ionis' technologies, pelacarsen and 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2020, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and other documents are available from the Company.

    

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   SOURCE Ionis Pharmaceuticals, Inc.

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2. 29 July 2021 Ionis completes enrollment in pivotal NEURO-TTRansform study of eplontersen

   CARLSBAD, Calif., July 29, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in RNA-targeted therapies, today announced it has completed enrollment of NEURO-TTRansform, the company's Phase 3 study of eplontersen, formerly AKCEA-TTR-L_Rx, for people with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). The company has exceeded the enrollment target in NEURO-TTRansform and expects to register more than 160 patients in the study.  

   

   Eplontersen is an investigational antisense medicine designed to inhibit production of transthyretin (TTR), a protein that forms the amyloid deposits that cause debilitating nerve damage throughout the body. Eplontersen utilizes Ionis' advanced LIgand Conjugated Antisense (LICA) technology. In a Phase 1 clinical trial, patients treated with eplontersen experienced reductions in TTR of up to 94 percent.

   "The speed of enrollment in our pivotal NEURO-TTRansform study speaks to the urgent need for new treatment options for people living with hATTR amyloidosis," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "Because eplontersen utilizes Ionis' LICA technology, it offers the advantages of high potency, convenient, infrequent dosing and an attractive safety and tolerability profile. We remain dedicated to advancing eplontersen as rapidly as possible and look forward to announcing results from a formal interim analysis by mid-year 2022, with NDA filing to potentially follow by year-end 2022." 

   Ionis is also studying eplontersen in the CARDIO-TTRansform Phase 3 study in patients with hereditary and wild-type cardiomyopathy (ATTR-CM).

   For more information on the NEURO-TTRansform study, please visit: https://clinicaltrials.gov/ct2/show/NCT04136184.

   About the NEURO-TTRansform Study

   NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of eplontersen in patients with polyneuropathy due to hATTR amyloidosis. The study has enrolled adult patients with stage 1 or stage 2 polyneuropathy and will be compared to the historical placebo arm from the TEGSEDI® (inotersen) NEURO-TTR registrational study that Ionis completed in 2017. There will be a formal interim analysis at week 35 analyzing the co-primary efficacy endpoints of percent change from baseline in serum TTR concentration and change from baseline in mNIS+7.

   The final primary endpoint analysis will be completed at week 66 and all patients will be followed until week 85 when they will have the option to transition into the open label extension study. 

   The co-primary efficacy endpoints at week 66 are:

   • Percent change from baseline in serum TTR concentration
   • Change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression
   • Change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN)  

   About Eplontersen

   Eplontersen is an antisense drug that uses Ionis' advanced Ligand Conjugated Antisense, or LICA, technology designed to inhibit the production of the transthyretin (TTR) protein at its source. Eplontersen is in development to treat a broad population of patients with both hereditary and wild-type forms of transthyretin amyloidosis, or ATTR amyloidosis.

   About Hereditary Transthyretin (hATTR) Amyloidosis

   hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract. The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis.

   About Ionis Pharmaceuticals, Inc.

   For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

   To learn more about Ionis, visit www.ionispharma.com and follow us on twitter @ionispharma.

   Ionis' Forward-looking Statement

   This press release includes forward-looking statements regarding Ionis' business, the therapeutic and commercial potential of Ionis' technologies, eplontersen and 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2020, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and other documents are available from the Company.

    

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   SOURCE Ionis Pharmaceuticals, Inc.

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3. 26 July 2021 Biogen and Ionis report positive topline clinical data on investigational Alzheimer's disease treatment at AAIC

   CAMBRIDGE, Mass. and CARLSBAD, Calif., July 26, 2021 /PRNewswire/ -- Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that topline data from a Phase 1b placebo-controlled, multiple ascending dose clinical study showed that BIIB080/IONIS-MAPT_Rx met its primary objective of safety and tolerability in patients with mild Alzheimer's disease. The study demonstrated robust time and dose dependent lowering of tau protein in cerebrospinal fluid (CSF) over the three-month treatment period and sustained reductions during the six-month post-treatment period.

   

   In patients receiving BIIB080, there were dose-dependent decreases in the concentration of total-tau in CSF eight weeks post-last dose (Day 141) with a mean percentage reduction of 30 percent, 40 percent and 49 percent in the low, medium and high dose groups treated every four-weeks, respectively, and 42 percent in the group treated every 12 weeks. Total-tau in the CSF continued to decline 16 weeks post-last dose in patients treated with BIIB080 in the high dose four-week and 12-week dose groups, showing a 55 percent and 49 percent mean reduction from baseline, respectively. CSF was not collected 16 weeks post-last dose in the low and medium four-week dose groups. There were similar dose-dependent decreases in the levels of phosphorylated tau. All participants (n=46) completed the Multiple Ascending Dose (MAD) period and 43 participants completed the Post-Treatment (PT) period (3 participants voluntarily withdrew). These data were presented in a poster session at the 2021 Alzheimer's Association International Conference (AAIC) held virtually and in Denver, Colo., July 26 – 30.

   "There is clearly an urgent need to develop and deliver effective treatments for Alzheimer's disease, a devastating disorder for which there currently are limited therapeutic options. We are encouraged by the topline results from this study of BIIB080, which demonstrate the potential of Ionis' antisense technology to successfully target what we believe is a root cause of Alzheimer's disease," said C. Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs. Dr. Bennett added, "These study results support further investigation of BIIB080 for the treatment of Alzheimer's disease and suggest that antisense-mediated suppression of tau protein may be a feasible therapeutic approach for other tauopathies."

   "Biogen is deeply committed to the development of novel treatments for patients with Alzheimer's disease. This commitment extends across multiple modalities, including antisense oligonucleotides, as with BIIB080," said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. "Biogen is encouraged by the results of this trial, and we look forward to our continued research in future clinical studies with this promising investigational asset."

   Alzheimer's disease is a progressive neurodegenerative disorder characterized by cognitive and functional decline resulting in significant disability. Until recently, treatment was limited to management of symptoms. BIIB080 is an investigational antisense therapy designed to target microtubule-associated protein tau (MAPT) mRNA and prevent production of tau protein. Growing evidence suggests that aggregated, hyperphosphorylated tau may be a key driver of neurodegeneration in Alzheimer's disease as well as other tauopathies including progressive supranuclear palsy and frontotemporal degeneration. In preclinical studies in MAPT transgenic mice, MAPT-targeted antisense treatment demonstrated robust tau-lowering in CNS tissues and prevention and reversal of disease.

   The primary objective of the Phase 1b first-in-human study was to assess safety and tolerability of multiple intrathecal (IT) bolus administrations of BIIB080. The study was divided into two parts: Part 1, a MAD study of 46 patients with mild Alzheimer's disease comprising a three-month Treatment Evaluation Period and a six-month PT period; Part 2, an open label long-term extension study comprising a 12-month Treatment Evaluation Period and a four- or six-month PT period. Four ascending dose cohorts were enrolled sequentially and randomized 3:1 to IT bolus administrations of BIIB080 or placebo. Patients aged 50-74 years with mild Alzheimer's disease and confirmed amyloid positivity (via CSF) at screening were considered eligible. Part 1 is now complete; Part 2 is currently ongoing (EudraCT: 2016-002713-22; NCT03186989).

   The characteristics of patients at baseline were representative of relatively younger, mild Alzheimer's disease patients and were generally similar across trial groups. All adverse events were mild to moderate in severity with no serious adverse events occurring in any patients that received BIIB080. There were no deaths, dose-limiting adverse events or dosing discontinuations.

   About Ionis' Neurology Franchise

   The Ionis neurology franchise addresses all major brain regions and central nervous system cell types and currently has three Phase 3 studies ongoing with 11 medicines in clinical development, three of which are wholly owned. Ionis is leading the way in treating root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's and Parkinson's as well as rare diseases like amyotrophic lateral sclerosis (ALS) and Alexander disease. Ionis' marketed neurological disease medicines include SPINRAZA^®, a global foundation of care for spinal muscular atrophy (SMA), commercialized by Biogen, and TEGSEDI^®, the first and only self-administered, subcutaneous treatment for the polyneuropathy of hereditary ATTR amyloidosis in adults.

   About Biogen 

   At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimer's disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, neuropsychiatry, immunology, acute neurology and neuropathic pain. 

   We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

   Biogen Safe Harbor Statement

   This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to the potential clinical effects of BIIB080; the potential benefits, safety and efficacy of BIIB080; the results of the Phase 1b study of BIIB080; the clinical development program for BIIB080; the identification and treatment of Alzheimer's disease; the potential of Biogen's commercial business and pipeline programs, including BIIB080; the anticipated benefits and potential of Biogen's collaboration arrangements with Ionis; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "possible," "prospect," "will," "would" and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

   These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including unexpected concerns that may arise from additional data, analysis or results obtained during clinical trials; the occurrence of adverse safety events; risks of unexpected costs or delays; the risk of other unexpected hurdles; uncertainty of success in the development and potential commercialization of BIIB080; failure to protect and enforce Biogen's data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; third party collaboration risks; the direct and indirect impacts of the ongoing COVID-19 pandemic on Biogen's business, results of operations and financial condition; and any other risks and uncertainties that are described in other reports Biogen has filed with the U.S. Securities and Exchange Commission. These statements are based on Biogen's current beliefs and expectations and speak only as of the date of this news release. Biogen does not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

   About Ionis Pharmaceuticals, Inc.

   For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

   To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.

   Ionis' Forward-looking Statement

   This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of Ionis' technologies, IONIS-MAPT_Rx/BIIB080 and 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2020, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. 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" refers to Ionis Pharmaceuticals and its subsidiaries.

   Ionis Pharmaceuticals^® is a trademark of Ionis Pharmaceuticals, Inc.

   BIIB080 is licensed to Biogen.

    

   

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   SOURCE Ionis Pharmaceuticals, Inc.; Biogen Inc.

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4. 21 July 2021 Ionis to hold second quarter 2021 financial results webcast

   CARLSBAD, Calif., July 21, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Wednesday, August 4^th at 11:30 a.m. Eastern Time to discuss its second quarter 2021 financial results and report on pipeline and business progress.

   

   Interested parties may access the webcast at https://ir.ionispharma.com/events-and-presentations/upcoming-events. A webcast replay will be available for a limited time at the same address.

   About Ionis Pharmaceuticals, Inc.
   
   For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies. 

   To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.

    

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   SOURCE Ionis Pharmaceuticals, Inc.

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5. 13 July 2021 Ionis enters exclusive licensing agreement with Bicycle Therapeutics aimed at further advancing LICA technology

   CARLSBAD, Calif., July 13, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that it has entered into an exclusive licensing agreement with Bicycle Therapeutics plc (NASDAQ:BCYC) to increase the delivery capabilities of Ionis' advanced LIgand Conjugated Antisense (LICA) medicines. The agreement provides Ionis exclusive access to Bicycle's proprietary macrocyclic peptides, referred to as Bicycles, to design LICAs that target transferrin receptor 1 for use with oligonucleotides. This LICA strategy has demonstrated both the improved delivery of antisense medicines to muscle tissue, including cardiac muscle, as well as the potential to cross the blood brain barrier.

   …

   CARLSBAD, Calif., July 13, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that it has entered into an exclusive licensing agreement with Bicycle Therapeutics plc (NASDAQ:BCYC) to increase the delivery capabilities of Ionis' advanced LIgand Conjugated Antisense (LICA) medicines. The agreement provides Ionis exclusive access to Bicycle's proprietary macrocyclic peptides, referred to as Bicycles, to design LICAs that target transferrin receptor 1 for use with oligonucleotides. This LICA strategy has demonstrated both the improved delivery of antisense medicines to muscle tissue, including cardiac muscle, as well as the potential to cross the blood brain barrier.

   

   Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development.

   "One of our key strategic objectives is to expand our drug discovery capabilities. The agreement with Bicycle Therapeutics represents a strategic addition to our expanding LICA platform, giving us the potential to meaningfully expand our drug discovery capabilities and deliver many more transformational medicines to the market," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "We believe that investing in technologies like Bicycle's cutting-edge transferrin receptor 1-targeting peptides will enhance our ability to design medicines to tackle devastating diseases in need of better treatment options."

   "Our collaboration with Bicycle will accelerate Ionis' progress toward creating best-in-class, low molecular weight ligands for muscle-specific targeted delivery," said Eric E. Swayze, Ph.D., executive vice president of research at Ionis. "This approach offers several potential advantages over related strategies in development, including increased potency, more convenient administration, less complex manufacturing processes and reduced cost of goods." Dr. Swayze added, "We are pleased with the data we have generated to date and look forward to incorporating optimized Bicycle peptides into our LICA delivery platform."

   Under terms of the agreement, Ionis obtained an exclusive license to Bicycle's technology covering the entire class of transferrin receptor 1 Bicycles for use in targeted delivery of oligonucleotide drugs. Ionis made a $45 million upfront payment to Bicycle which included a license fee, an option fee, and an $11 million equity investment in Bicycle. Bicycle will be eligible to earn development and regulatory milestone payments on a program-by-program basis and royalties on product sales.

   About Ionis Pharmaceuticals, Inc.

   For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

   To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.

   Ionis' Forward-looking Statement

   This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of Ionis' technologies and 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2020, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. 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" refers to Ionis Pharmaceuticals and its subsidiaries.

   Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

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   SOURCE Ionis Pharmaceuticals, Inc.

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