Analyst Conference Summary

biotechnology

conference date: August 9, 2022 @ 8:30 AM Pacific Time
for quarter ending: June 30, 2022 (Q2, second quarter 2022)

Forward-looking statements

Overview: Product revenue off, waiting for pipeline to mature.

Basic data (GAAP):

Revenue was $134 million, down 6% sequentially from $142 million, but up 6% from $126 million year-earlier.

Net income was negative $105 million, down sequentially from negative $65 million, and down from negative $81 million year-earlier.

EPS (diluted) was negative $0.74, down sequentially from negative $0.46, and down from negative $0.57 year-earlier.

Guidance:

Expects to meet 2022 guidance.

Conference Highlights:

CEO Brett Monia said "Over the first half of this year, we moved significantly closer to delivering an abundance of new medicines to the market. We reported positive Phase 3 data from the NEURO-TTRansform study of eplontersen in patients with hereditary ATTR polyneuropathy and we are on track to file an NDA in the second half of this year. We were also pleased that the FDA accepted the NDA for tofersen and granted priority review, enabling tofersen to potentially be the first disease modifying treatment approved for a genetic form of ALS. These achievements mean eplontersen and tofersen could be our next marketed products as early as next year. We also significantly advanced our late- and mid-stage pipeline. The pelacarsen Lp(a) Horizon and olezarsen Balance Phase 3 studies recently completed enrollment. Additionally, we reported positive data from six mid-stage programs, positioning us to grow our rich Phase 3 pipeline to at least eight medicines across 10 indications. We are looking forward to continuing our positive momentum in the second half of this year by presenting Phase 3 eplontersen data at the International Symposium on Amyloidosis in September, filing our eplontersen NDA, and reporting data from several important programs." Believes Spinraza revenue can return to growth based on newer studies. Investing in future commercial launches. Phase 3 pipeline is poised to expand to 8 medicines for 10 indications.

In Q2 2022 Ionis reported that eplontersen met its endpoints in the Phase 3 in ATTRv-PN neuropathy. Will file with FDA in 2022. Global launch is parnered with AstraZeneca.

Biogen reported that toferson for SOD1-ALS (amyotrophic lateral sclerosis) has a PDUFA date of January 25, 2023.

In Q2 2022 Novartis announced completion of enrollment in pelacarsen Phase 3 Lp(a) cardiovascular outcomes study. Believes pelacarsen is a multi-billion dollar opportunity.

The Phase 3 olezarsen for FCS (familial chylomicronemia syndrome) study completed enrollment in Q2 2022 with data expected in 2023.

Non-GAAP numbers: net income negative $80 million, down sequentially from negative $39 million, and up from negative $36 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.0 billion, down sequentially from $2.05 billion. Debt was $1.16 billion in convertible senior notes.

Waylivra (volanesorsen) is approved in the EU for the treatment of adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis. Commercially available in Germany, Austria, Greece, and France. Approved in Brazil in Q3 2021. Now distributed in Europe by Sobi. Not approved in U.S. Waylivra is under review in Brazil for the treatment of familial partial lipodystrophy (FPL). If approved, it will be the first approved treatment for patients with FPL in Brazil.

Tegsedi (Inotersen) is approved in the EU for polyneuropathy in adults with hATTR (hereditary transthyretin amyloidosis). Now commercial in 15 countries, including Portugal, with additional EU launches planned. Now distributed in Europe by Sobi. Expanding into Canada and Latin America.

In Q2 2022 GSK presented Phase 2b data for bepirovirsen for hepatitis B. Plans to advance to Phase 3.

In Q2 2022 Roche reported positive Phase 2 data for IONIS-FB-Lrx for immunoglobulin A nephropathy. Plans to advance it to a Phase 3 study.

The Phase 2b study of IONIS-AGT-LRx in patients with treatment-resistant hypertension was fully enrolled in Q2 2022, with data expected in 2H 2022.

Bayer reported foesomersen met the primary endpoint in the Phase 2b study of fesomersen in end-stage renal disease. Reduced Factor XI activity levels.

Results from the eplontersen Phase 3 study in patients with TTR polyneuropathy are due mid-year 2022. Eplontersen is being jointly develped with AstraZeneca. In Q1 2022 increased the study size and duration in the Phase 3 CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy to generate even more robust data and ensure a highly positive study outcomet. Data from this study are expected in the first half of 2025. Granted orphan drug status in ATTR.

In Q3 2021 reached full enrollment in the Bayer Phase 2b RE-THINc ESRD study of fesomersen (IONIS-FXI-LRx); data expected in the first half of 2022.

Ionis continues to develop technologies that allow RNA therapies to almost any part of the body, including inhaled agents.

Ionis has a pipeline of about 45 potential drugs. A growing number are wholly-owned.

GAAP Operating expense was $220 million, consisting of $5 million for cost of goods sold; $181 million for R&D and $34 million for selling, general and administrative. Operating income was negative $86 million. Other expense was $17 million. Income tax $2 million.

Q&A selective summary:

Competing TTR silencer data v. eplontersen? The overall package for eplontersen, we could not hope for more. TTR amyloidosis is a big indication. The cardiomyopathy data will be incredibly important and will be a key differeniator (when available in 2025). In cardiomyopathy the outcome data is critical.

Tofersen as template for other therapies? Functional endpoints do not change as rapidly as biomarkers. Showing an actual impact on neurodegeneration. Generally filing on biomarkers alone is not a good play.

More eplontersen trials? No specific comments, talking with partners. Believe profile will be highly differentiated from competitors.

HBV with GSK program? Data was presented by GSK showing remarkable efficacy. Will go to Phase 3 in the near future. Also an open-label extension.

Working on getting a muscle LICA program into preclinical development in multiple therapeutic areas.

Hypertension endpoints? Systolic blood pressure decrease v. placebo.

IGA nephthropathy data is the best disclosed to date, which is why Roche moved quickly to Phase 3.

We do not expect data from the dry AMD study this year. It is still enrolling, it is a big study.

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