Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals (NASDAQ: IONS) has published promising results from a compassionate-use study of its investigational drugs, IONIS-PKK_Rx and IONIS-PKK-L_Rx, for treating severe hereditary angioedema (HAE). The study shows these antisense medicines effectively reduced plasma prekallikrein activity and cut breakthrough attack rates in patients. Notably, one patient achieved complete resolution of symptoms. Ionis is advancing the development of IONIS-PKK-L_Rx, which offers a potential monthly treatment option, aiming to improve patient outcomes.

Akcea Therapeutics and Ionis Pharmaceuticals announced positive results from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-L_Rx) 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-L_Rx, 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.

Ionis Pharmaceuticals will host a live webcast on September 2 at 10:00 a.m. ET to discuss its cardio-renal franchise. The 90-minute session will feature presentations from its executives, including Brett P. Monia and Sam Tsimikas. Key topics will include advances in RNA-targeted drug discovery and specific medicines aimed at treating triglyceride-driven diseases, thrombosis, and treatment-resistant hypertension. The webcast can be accessed at www.ionispharma.com.

On August 24, 2020, Akcea Therapeutics and Ionis Pharmaceuticals announced that data from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-L_Rx) 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-L_Rx 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.

Ionis Pharmaceuticals reported Q2 2020 results, achieving a non-GAAP net income of $8 million despite a GAAP net loss of $32 million. Revenue rose to $146 million, driven by $72 million in SPINRAZA royalties and increased product sales. Ionis maintains its 2020 financial guidance, targeting profitability. The company has over $2.3 billion in cash, supporting future initiatives. Progress includes advancements in neurological and cardio-renal programs, with expectations for multiple marketing applications through 2025. Key studies for new treatments are set to begin, enhancing growth potential.

Ionis Pharmaceuticals (NASDAQ: IONS) will host a live webcast on August 5, 2020 at 11:30 a.m. ET to discuss its Q2 2020 financial results and provide updates on its pipeline and business progress. This event will focus on the company's advancements in RNA-targeted drug discovery, highlighting their innovative antisense technology that addresses unmet medical needs.