IONIS-DGAT2Rx
- Generic Name
- IONIS-DGAT2Rx
OliX and Eli Lilly Forge $630M Deal for Novel RNAi Therapy Targeting MASH and Obesity
• OliX Pharmaceuticals has secured a landmark $630 million licensing agreement with Eli Lilly for OLX75016, an RNAi-based therapeutic targeting MASH and obesity.
• The deal grants Lilly global rights to develop and commercialize OLX75016, with OliX maintaining responsibility for Phase 1 trials before transferring development control to Lilly.
• Preclinical studies showed promising results when OLX75016 was combined with semaglutide, demonstrating enhanced weight loss and reduced rebound weight gain in primates.
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.
Ionis Pharmaceuticals Advances Pipeline with Key Regulatory and Clinical Milestones
• Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and Europe for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, potentially addressing an unmet need.
• Donidalorsen's NDA for HAE has been accepted by the FDA, with a PDUFA date of August 21, 2025, positioning it as a novel prophylactic treatment.
• Phase 3 results for olezarsen in severe hypertriglyceridemia and pelacarsen in Lp(a)-driven cardiovascular disease are expected next year.
Ionis Pharmaceuticals Highlights Pipeline Progress and Financials in Q3 2024
• Ionis Pharmaceuticals reports strong progress with WAINUA launch for ATTRv-PN, generating $5 million in royalties for Q3 2024, and securing approvals in the UK and a positive CHMP opinion in Europe.
• The company is advancing olezarsen for FCS, with a PDUFA date set for December 19, 2024, and donidalorsen for HAE, with a PDUFA date of August 21, 2025, positioning them for potential launches.
• Positive Phase 2/3 DEVOTE study data for higher dose nusinersen in SMA was presented, with global regulatory applications planned, alongside QALSODY's marketing approval in China for SOD1-ALS.
• Ionis reaffirms its 2024 financial guidance, increasing cash guidance to $2.2 billion, reflecting proceeds from an equity offering to support pipeline advancements and commercial preparations.
Ionis Pharmaceuticals Highlights Pipeline Progress and Financial Results in Q3 2024
• Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and EU for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, marking a potential treatment for this rare disease.
• Donidalorsen's Phase 3 data positions it as a potential RNA-targeted prophylactic treatment for hereditary angioedema, with an FDA action date set for August 21, 2025.
• The company reaffirms its 2024 financial guidance and increases cash guidance to $2.2 billion, reflecting proceeds from an equity offering.
Ionis Pharmaceuticals Advances Pipeline with Key Regulatory and Clinical Milestones
• Ionis Pharmaceuticals reports positive progress with WAINUA, achieving commercial success and regulatory approvals for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, potentially addressing an unmet need.
• Donidalorsen's NDA for HAE has been accepted by the FDA, with a PDUFA date of August 21, 2025, positioning it as a novel prophylactic treatment.
• The company is advancing multiple Phase 3 programs, including olezarsen for sHTG and ION582 for Angelman syndrome, with key data expected in the coming years.
Drug Development Updates
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