Can Ionis' (IONS) Wholly-Owned Drugs Cut Dependency on Collabs?
California-based Ionis Pharmaceuticals IONS is focused on the discovery and development of RNA-targeted therapeutics using its proprietary antisense oligonucleotide technology.
The company leverages its proprietary technology to generate a broad pipeline of medicines, targeting neurological, cardiovascular and specialty rare diseases, for itself as well as its partners.
Devoid of any marketed products in its portfolio, Ionis generates the entirety of its revenues in the form of royalty payments and R&D revenues received from partnered programs. The company earns commercial revenues primarily in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. Ionis licensed this drug to Biogen BIIB, which is responsible for commercializing it.
Ionis receives royalties from Biogen on Spinraza’s sales. Ionis and Biogen also market Qalsody (tofersen), which was approved by the FDA in April 2023 for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.
Last December, the FDA approved Wainua (eplontersen) for the treatment of patients with hereditary transthyretin-mediated amyloid polyneuropathy, commonly called hATTR-PN or ATTRv-PN. The drug has been developed in partnership with AstraZeneca AZN. While Ionis and AstraZeneca will jointly market Wainua for ATTRv-PN in the United States, AZN has exclusive rights to commercialize Wainua outside U.S. markets. The U.S. launch of the drug is currently underway.
Regulatory filings seeking approval for eplontersen in ATTRv-PN are under review in the EU and some other countries. AstraZeneca and Ionis are also developing eplontersen as a treatment for cardiomyopathy caused by hATTR amyloidosis (ATTR-CM) in the phase III CARDIO-TTRansform study, which is on track for a data readout in first-half 2025.
Currently, Ionis has phase III studies ongoing for nine medicines (internal as well as partnered) across 11 indications. Some of these candidates are pelacarsen for cardiovascular disease due to elevated Lp(a) levels, olezarsen for familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia, ulefnersen for ALS, with mutations in the fused in sarcoma gene, or FUS (FUS-ALS), donidalorsen for the prophylactic treatment of hereditary angioedema (HAE), bepirovirsen for chronic hepatitis B (CHB), IONIS-FB-LRx for IgA Nephropathy and zilganersen for Alexander’s disease.
Novartis and GSK GSK are its partners for pelacarsen and bepirovirsen, respectively. GSK and Ionis’ bepirovirsen was granted Fast Track designation by the FDA for CHB in fourth-quarter 2023.
Some of its other wholly-owned pipeline candidates include ION224, ulefnersen and donidalorsen. Earlier this month, Ionis reported that a mid-stage study on ION224 in MASH indication achieved the primary endpoint of statistically significant liver histologic improvement. The study also achieved a key secondary endpoint of statistically significant MASH resolution without worsening of fibrosis. Management is currently in the process of determining the next steps to advance the development of the candidate.
Last month, the FDA granted Orphan Drug designation to its wholly-owned pipeline candidate, olezarsen, for treating FCS, a rare genetic disease. Management intends to file for marketing approval in the United States and EU this year based on results from the BALANCE study. If approved, olezarsen will be Ionis’ first medicine that it will launch independently.
In January, Ionis reported top-line results from the phase III OASIS-HAE study on donidalorsen in HAE patients. The study achieved its primary endpoint of a statistically significant reduction in the rate of HAE attacks in patients treated with the drug once every four weeks and eight weeks. Based on these results, Ionis is preparing to file a new drug application (NDA) for donidalorsen with the FDA. Ionis’ partner, Otsuka, is preparing to submit for marketing approval in Europe.
Ionis is advancing and expanding its wholly-owned pipeline to drive future revenue growth. While the company does have a broad pipeline of partnered programs with leading drugmakers/biotech companies like AstraZeneca, Biogen and GSK, its recent decision to increase investments in its internal pipeline is encouraging, given the recent positive data readouts. The expansion of its internal pipeline will help Ionis further diversify its revenue stream and narrow down its dependence on collaboration partners.
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