IONIS Pharma (Nasdaq: IONS) Portfolio, Stock and Financial Highlights

Ionis Pharmaceuticals Highlights 

- Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics
- Three approved commercial RNA targeted therapies: SPINRAZA, TEGSEDI, WAYLIVRA
- Numerous in house and out liscened collaborative program
- Net income of $5 million on a non-GAAP basis 
- Cash equivalents, and short-term investments of more than $2.3 billion,
Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. Ionis is a leader in discovering and developing RNA-targeted therapeutics. The efficient, broadly applicable drug discovery platform, called antisense technology, can treat illnesses where no other therapeutic approaches have proven effective or ever existed. The commercial products include SPINRAZA (  for spinal muscular atrophy) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy.

Stock Symbol: IONS


Antisense therapies, also known as antisense oligonucleotides, or ASOs, are designed to bind precisely with RNA, halting the process of creating a disease-causing protein.

IONIS Portfolio & Pipeline

Commercial Medicine Highlights

SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages. More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the third quarter, including patients across commercial, expanded access, and clinical trial settings. (Prescribing Information)

TEGSEDI: the only approved at-home subcutaneous therapy for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients. It is commercially available in 15 countries. (Prescribing Information)

WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis. It is commercially available in 4 countries. (Prescribing Information)

Ionis Pharma Clinical & Research Programs

Vupanorsen for the treatment of hypertriglyceridemia and cardiovascular disease
Vupanorsen (AKCEA-ANGPTL3-LRx), is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3. Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain cardiovascular diseases. Vupanorsen advanced into Phase 2b development with the initiation of the TRANSLATE-TIMI 70 dose-ranging study in statin-treated patients with dyslipidemia, resulting in a $75 million payment from Pfizer

IONIS-FXI-LRx utilizes Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform and is designed to reduce the production of Factor XI (FXI), a clotting factor produced in the liver. High levels of FXI increase the risk of thrombosis and can be responsible for heart attacks and strokes. Alternatively, individuals deficient in FXI have a lower incidence of thrombosis-related events and little to no increase in bleeding risk.

IONIS-HBVRx for the treatment of hepatitis B virus infection

IONIS-ENAC-2.5Rx for the treatment of patients with cystic fibrosis

IONIS-PKK-LRx for the treatment of Hereditary angioedema

ION541 for the treatment of patients with nearly all forms of ALS

ION464 for the treatment of patients with multiple system atrophy

IONIS-MAPTRx for the treatment of patients with Alzheimer's disease 

Financial Highlights 

  • On track to achieve financial guidance of being meaningfully profitable in 2020 
  • Net income of $5 million on a non-GAAP basis and a net loss of $31 million on a GAAP basis for the third quarter
  • Ionis ended September 2020 with cash, cash equivalents, and short-term investments of more than $2.3 billion,
  • Net loss attributable to Ionis' common stockholders for the third quarter of 2020 compared to net income in the same period in 2019 primarily due to higher revenue in 2019, and increased expense

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