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Ionis Pharmaceuticals and Akcea Therapeutics have won the Prix Galien USA Award for the Best Biotechnology Product for 2020, recognizing TEGSEDI, the first self-administered treatment for the polyneuropathy of hereditary ATTR amyloidosis. Presented in a virtual ceremony on October 29, 2020, this prestigious award highlights significant advancements in biomedicine. Brett P. Monia emphasized TEGSEDI's role in improving the lives of patients affected by this severe disease. TEGSEDI is FDA-approved and offers hope for individuals suffering from hATTR amyloidosis.
Akcea Therapeutics and Ionis Pharmaceuticals announced positive results from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-LRx) at the ESC Congress 2020. The trial showed significant reductions in triglyceride levels, achieving a maximum reduction of 53% with the 80 mg dose. The therapy demonstrated a favorable safety profile, with injection site reactions being the most common adverse events. This investigational antisense therapy targets ANGPTL3 to reduce cardiovascular risks. Pfizer plans further Phase 2b studies to optimize dosing.
Akcea Therapeutics (NASDAQ: AKCA) and Ionis Pharmaceuticals (NASDAQ: IONS) announced positive results from the Phase 2 study of AKCEA-APOCIII-LRx, presented at the ESC Congress 2020. The study demonstrated significant reductions in triglyceride levels (TG) and apoC-III, with a notable 62% reduction in TG at the highest dose administered. Furthermore, 91% of patients achieved TG levels below 150 mg/dL at six months. The drug exhibited a favorable safety profile, with plans for a Phase 3 trial targeting familial chylomicronemia syndrome (FCS) later this year.
On August 24, 2020, Akcea Therapeutics and Ionis Pharmaceuticals announced that data from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-LRx) will be presented at the ESC Congress 2020, scheduled for August 29-September 1, 2020. Vupanorsen is an investigational therapy targeting cardiovascular diseases by reducing ANGPTL3 protein levels in patients with conditions such as hypertriglyceridemia and type 2 diabetes. The companies previously secured a $250 million licensing deal with Pfizer, which will assume further development responsibilities.
Akcea Therapeutics and Ionis Pharmaceuticals announced that data from the Phase 2 study of AKCEA-APOCIII-LRx will be presented at the ESC Congress 2020 from August 29 to September 1, 2020. The drug aims to reduce apolipoprotein C-III levels, a protein linked to triglyceride regulation and cardiovascular risk. Previous studies showed promising results, with dose-dependent reductions in triglycerides and increases in HDL-C. This presentation may influence the drug's market potential and investor outlook.
Akcea Therapeutics has announced new pricing and reimbursement terms for WAYLIVRA® (volanesorsen) in Germany, effective August 15, 2020. This renegotiation, required 12 months post-commercialization, highlights the drug's value for patients with familial chylomicronemia syndrome (FCS) at high risk for pancreatitis. WAYLIVRA is now the only therapy approved for FCS in the EU, following its launch in August 2019. Akcea aims to expand WAYLIVRA's availability to more European countries, supporting the needs of patients suffering from this ultra-rare disease.
Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, will host a live webcast on August 4, 2020, at 4:30 p.m. ET to discuss its Q2 2020 financial results and pipeline progress. The company's medicines, including TEGSEDI and WAYLIVRA, target serious and rare diseases. With a robust pipeline, Akcea is poised for future growth in the biopharmaceutical sector.
Investors can access the webcast at this link.
Akcea Therapeutics (NASDAQ: AKCA) announces the appointment of Christophe Hotermans, M.D., Ph.D., as senior vice president of global medical affairs. With over 20 years of experience, including a decade at Biogen, Dr. Hotermans will lead the company's medical strategies and operations worldwide. His expertise in specialty care and rare diseases aligns with Akcea's goals, focusing on the commercial launch of TEGSEDI and WAYLIVRA. Akcea, a majority-owned affiliate of Ionis Pharmaceuticals (NASDAQ: IONS), continues to develop medicines for serious and rare diseases, supported by a strong pipeline.
Akcea Therapeutics (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals (NASDAQ:IONS), will participate in a fireside chat at the BMO 2020 Prescriptions for Success Healthcare Conference on June 23, 2020, at 3:30 p.m. ET. CEO Damien McDevitt and CCO Kyle Jenne will represent the company in this virtual event. Participants can access the live webcast through Akcea's website.
Akcea focuses on developing drugs for serious and rare diseases, including TEGSEDI and WAYLIVRA, leveraging Ionis' proprietary technology.
