IONIS PHARMACEUTICALS, INC.
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public, Private, Subsidiary
- Established
- 1989-01-01
- Employees
- 927
- Market Cap
- -
- Website
- http://www.ionis.fr
Ionis Reports Positive Phase 3 Results for Olezarsen in Hypertriglyceridemia
• Olezarsen demonstrated significant triglyceride reductions of 61% and 58% at 6 months with 80mg and 50mg monthly doses respectively in patients with moderate hypertriglyceridemia.
• The Phase 3 Essence study met all primary and secondary endpoints, with most participants achieving triglyceride levels within normal range despite already being on standard lipid-lowering therapies.
• Following recent FDA approval of olezarsen (Tryngolza) for familial chylomicronemia syndrome, these results support potential expanded indication for severe hypertriglyceridemia pending upcoming CORE and CORE2 trial data.
Eli Lilly's Lepodisiran Shows 95% Reduction in Lipoprotein(a), Offering New Hope for South Asian Heart Patients
• Eli Lilly's experimental drug lepodisiran demonstrated up to 95% reduction in lipoprotein(a) levels with a single 400mg dose in midstage trials, potentially addressing a significant cardiovascular risk factor prevalent in South Asian populations.
• The once-yearly injectable targets LPA cholesterol particles that are found in elevated levels in approximately 25% of South Asians and are not addressed by current cholesterol-lowering medications like statins.
• With an estimated 64 million Indians suffering from heart conditions and above-average death rates, lepodisiran represents a targeted intervention that could significantly impact cardiovascular disease management in the region.
Merck Licenses Oral Lipoprotein(a) Inhibitor HRS-5346 from Hengrui Pharma in $1.97 Billion Deal
• Merck has secured exclusive global rights (excluding Greater China) to HRS-5346, an investigational oral small molecule Lipoprotein(a) inhibitor currently in Phase 2 trials in China.
• The $1.97 billion deal includes a $200 million upfront payment to Hengrui Pharmaceuticals, with potential milestone payments of up to $1.77 billion plus royalties on net sales.
• Elevated Lipoprotein(a), a genetic condition affecting approximately 1.4 billion people worldwide, is an independent risk factor for cardiovascular disease with limited treatment options currently available.
FDA Advisory Panel Backs Alnylam's Onpattro for ATTR Cardiomyopathy Despite Efficacy Concerns
• An FDA advisory committee voted 9-3 in favor of approving Alnylam's Onpattro (patisiran) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), with a final decision expected by October 8th.
• Despite FDA reviewers questioning the clinical significance of Onpattro's modest efficacy in the APOLLO-B trial, the panel was swayed by the drug's established safety profile and potential to address the underlying disease mechanism.
• If approved, Onpattro would compete with Pfizer's Vyndamax/Vyndaqel (tafamidis), which generated $2.5 billion in sales last year, marking another milestone for RNA interference technology following its historic 2018 approval for ATTR polyneuropathy.
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis (CTX), Offering Hope to Patients with Rare Genetic Disorder
• The FDA has approved chenodeoxycholic acid (Ctexli) as the first official treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder that affects cholesterol metabolism and causes progressive neurological damage.
• OHSU researchers developed critical biomarker tests that were instrumental in the clinical trial leading to FDA approval, with their Sterol Analysis Laboratory being the only facility worldwide capable of performing certain tests to FDA standards.
• Advocates are now pushing for CTX to be added to the Recommended Uniform Screening Panel for newborns, which would enable early diagnosis and treatment before irreversible damage occurs.
Roche and Zealand Pharma Forge $5.3 Billion Partnership to Develop Novel Obesity Treatments
• Roche has entered into a $5.3 billion collaboration with Zealand Pharma to co-develop petrelintide, a promising amylin analog for obesity treatment, both as monotherapy and in combination with Roche's incretin asset CT-388.
• The partnership includes upfront payments of $1.65 billion to Zealand Pharma, with profits and losses to be shared 50/50 in the US and Europe, while Roche gains exclusive commercialization rights for the rest of the world.
• Clinical data suggests petrelintide could deliver weight loss comparable to GLP-1 receptor agonists but with improved tolerability, potentially addressing unmet needs in the obesity market that is projected to affect 4 billion people globally by 2035.
Ionis and Ono Forge $940 Million Deal for Sapablursen in Polycythemia Vera Treatment
• Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront.
• Sapablursen, currently in Phase 2 trials, has received FDA Fast Track and Orphan Drug designations and works by increasing hepcidin production to regulate iron homeostasis in PV patients.
• Ono will gain exclusive global rights for development and commercialization, while Ionis will complete the ongoing IMPRSSION study before transferring responsibilities.
EU Approves WAINZUA (Eplontersen) for Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
• The European Commission has approved WAINZUA (eplontersen), developed by Ionis Pharmaceuticals and AstraZeneca, for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or 2 disease.
• WAINZUA is the only EU-approved treatment for ATTRv-PN that can be self-administered monthly via an auto-injector, offering consistent TTR protein suppression and improvements in neuropathy impairment and quality of life.
• The approval follows positive results from the NEURO-TTRansform Phase 3 trial, which demonstrated sustained benefits through 66 weeks on serum TTR concentration, neuropathy impairment, and quality of life compared to placebo.
Ionis Advances Antisense Therapy for Angelman Syndrome to Phase 3 Following Promising Clinical Results
• Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.
• In the Phase 1/2 HALOS study, 97% of participants receiving medium or high doses of ION582 showed improvements in communication, cognition, and motor function, with favorable safety profiles.
• The race to develop treatments for Angelman syndrome is intensifying, with Ultragenyx's GTX-102 already in Phase 3 trials and Neuren Pharmaceuticals advancing an oral synthetic peptide, highlighting significant progress in addressing this rare genetic disorder.
Alnylam's Vutrisiran Shows Strong Efficacy in ATTR Cardiomyopathy, Setting Stage for New Treatment Standard
• Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis.
• The HELIOS-B trial showed vutrisiran's benefits increased over time, with a 36% reduction in the primary endpoint at 42 months, positioning the quarterly-administered injection as a potential new standard of care.
• Vutrisiran, already approved as Amvuttra for ATTR polyneuropathy, could reach multibillion-dollar sales if approved for cardiomyopathy, though it faces competition from BridgeBio's acoramidis and AstraZeneca/Ionis' eplontersen.
Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments
• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.
• KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks.
• The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.
FDA Expands Evrysdi Approval to Include Infants Under Two Months with Spinal Muscular Atrophy
• Roche and PTC Therapeutics' oral SMA drug Evrysdi (risdiplam) receives FDA approval for use in infants younger than two months, enabling treatment across all age groups from newborns to adults.
• The RAINBOWFISH study demonstrated significant efficacy, with treated pre-symptomatic babies achieving key developmental milestones including sitting, standing, and walking within 12 months.
• The expanded approval positions Evrysdi to compete directly with Biogen's Spinraza and Novartis' Zolgensma, while offering the advantage of at-home oral administration.
Praxis Precision Medicines Announces Strategic Priorities and Pipeline Milestones for 2025
• Praxis Precision Medicines anticipates four pivotal clinical trial readouts in 2025, positioning the company for potential commercial launches between 2026 and 2028.
• An interim analysis of the Essential3 program for ulixacaltamide in essential tremor is expected in Q1 2025, with NDA filing anticipated later in the year.
• Enrollment in the EMBOLD study of relutrigine for developmental and epileptic encephalopathies is progressing, with NDA filing targeted for 2026.
• Vormatrigine's ENERGY program is advancing, with results from the RADIANT study expected in the first half of 2025 and POWER1 study results by year-end.
GSK's Bepirovirsen Receives FDA Fast Track for Chronic Hepatitis B Treatment
• The FDA has granted fast-track designation to GSK's bepirovirsen, an antisense drug, for chronic hepatitis B (CHB) treatment, expediting its review process.
• Bepirovirsen is the only single agent in phase 3 trials showing potential for a functional cure when combined with standard nucleoside/nucleotide analogue (NA) therapies.
• Phase 2b trial data indicated bepirovirsen reduced hepatitis B surface antigen (HBsAg) levels, suggesting it may benefit patients with low baseline HBsAg levels.
• GSK has initiated the phase 3 B-Well program and a long-term extension study (B-Sure) to further evaluate bepirovirsen's efficacy and long-term effects.
Donidalorsen Shows Sustained Efficacy in Hereditary Angioedema Prophylaxis
• Donidalorsen demonstrates a sustained reduction in HAE attack rates and improved quality of life in patients, according to Phase 3 and Phase 2 clinical program analyses.
• The investigational RNA-targeted medicine is under FDA review, with a PDUFA date set for August 21, 2025, positioning it for potential commercial launch.
• Data from the OASISplus study show that donidalorsen effectively reduces HAE attack rates and improves quality of life in patients switching from other prophylactic treatments.
• Long-term analysis of the Phase 2 open-label extension study reveals that donidalorsen maintains a 96% reduction in HAE attack rates from baseline up to three years.
Roche Partners with Dicerna for Hepatitis B Gene-Silencing Drug
Roche has entered into a $200 million upfront deal with Dicerna Pharmaceuticals for a gene-silencing drug aimed at treating hepatitis B virus (HBV) infections, with potential payments reaching up to $1.47 billion based on the drug's success. The drug, DCR-HBVS, currently in phase 1 testing, targets genes used by the virus to enter liver cells, offering hope for a functional cure by suppressing the virus below detectable levels.
Ionis' Tryngolza Approved for FCS as Arrowhead's Plozasiran Awaits FDA Review
• The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS).
• Tryngolza, an RNA-targeted therapy, significantly reduces triglyceride levels and the risk of acute pancreatitis in FCS patients when used with a low-fat diet.
• Arrowhead Pharmaceuticals' plozasiran, another RNA interference therapeutic for FCS, has been accepted for FDA review, with a decision expected by November 2025.
• Clinical trials showed Tryngolza achieved a 57% placebo-adjusted mean reduction in triglycerides at 12 months, while plozasiran demonstrated an 80% median reduction.
FDA Approves BridgeBio's Attruby, Setting Up Competition with Pfizer's Vyndamax in ATTR-CM Market
• The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), a heart condition leading to heart failure and death.
• Attruby demonstrated a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalizations compared to placebo in the ATTRibute-CM trial.
• BridgeBio is launching Attruby at a list price of $18,759 for a 28-day supply, positioning it to compete with Pfizer's Vyndamax, which dominates the ATTR-CM market.
• Several other companies, including Alnylam and AstraZeneca/Ionis, are also developing therapies for ATTR, potentially disrupting the current treatment landscape.
Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data
• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone.
• The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results.
• Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients.
• Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.
RNAi Therapies Olpasiran and Zerlasiran Show Promise in Lowering Lipoprotein(a) in Cardiovascular Disease
• Olpasiran significantly reduces lipoprotein(a) (Lp(a)) levels by over 95% in patients with atherosclerotic cardiovascular disease, marking a potential breakthrough in managing this potent risk factor.
• Zerlasiran, another siRNA therapeutic, demonstrates an average reduction of over 80% in Lp(a) levels over 36 weeks, with sustained effects observed at 60 weeks, offering a new approach to preventing premature heart disease.
• Both therapies target Lp(a) production in the liver using RNA interference, showing minimal side effects and suggesting a promising strategy for managing elevated Lp(a) levels, which affect millions worldwide.
• Further research is underway to assess the impact of these therapies on clinical outcomes and to address limitations such as the need for more diverse patient representation in trials.
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