Ionis Pharmaceuticals Stock Price, News & Analysis

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals (NASDAQ: IONS), has received approval from the Scottish Medicines Consortium (SMC) for volanesorsen, enabling access via the NHS in Scotland for treating familial chylomicronaemia syndrome (FCS). This follows NICE's earlier approval in England. Volanesorsen is the only treatment available for FCS in the UK, aimed at reducing triglyceride levels significantly. The SMC's decision is a milestone for FCS patients, allowing access while additional clinical data is collected over three years.

Ionis Pharmaceuticals reported its third-quarter 2020 financial results, showing a net income of $5 million (non-GAAP) but a GAAP net loss of $31 million. The company achieved revenue growth, with commercial revenues of $95 million driven by SPINRAZA royalties of $74 million. The recent acquisition of Akcea strengthens Ionis's pipeline, projecting 10 or more marketing applications by 2025. Cash reserves stand at $2.3 billion post-acquisition, maintaining solid financial health.

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.

Ionis Pharmaceuticals announced the dosing of the first patients with ION541, an investigational therapy for amyotrophic lateral sclerosis (ALS). This milestone is significant as ION541 targets ataxin-2 RNA, potentially reversing TDP-43 toxicity, a common characteristic of ALS. Supported by a $10 million payment from Biogen, this Phase 1/2 trial aims to address both familial and sporadic ALS cases. Ionis continues to advance its ALS pipeline, which includes tofersen and IONIS-C9Rx, enhancing its commitment to developing innovative therapies for this devastating condition.

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) will host a live webcast on November 4, 2020, at 11:30 a.m. ET to discuss its third quarter 2020 financial results and provide updates on its pipeline and business progress. The webcast will be available on their website, www.ionispharma.com, with a replay for a limited time after the event.

Ionis Pharmaceuticals (NASDAQ: IONS) announced that IONIS-PKK-L_Rx is being tested in a Phase 2 clinical study aimed at reducing respiratory complications in COVID-19 patients. Conducted in Brazil, this study will enroll up to 110 patients across 25 hospitals. The primary endpoint is to monitor the number of days patients are alive and free of oxygen support over 15 days. IONIS-PKK-L_Rx targets bradykinin signaling, a key factor in severe COVID-19 cases. Additionally, promising data from a related HAE study has been published.

Ionis Pharmaceuticals (NASDAQ: IONS) announced significant results from a clinical trial of IONIS-ENAC-2.5_Rx, an investigational antisense medicine for cystic fibrosis (CF). The trial demonstrated a mean 55.6% reduction in ENaC mRNA expression at a 75 mg dose (p<0.05), marking a breakthrough in aerosol delivery methods. This study lays the groundwork for future developments in treating CF and other lung diseases. The company plans to present additional data at the 2020 North American Cystic Fibrosis Conference from Oct. 21-23 and initiate a study for COPD later this year.