Analyst Conference Summary

biotechnology

conference date: November 9, 2022 @ 8:30 AM Pacific Time
for quarter ending: September 30, 2022 (Q3, third quarter 2022)

Forward-looking statements

Overview: Good revenue growth, still in the red.

Basic data (GAAP):

Revenue was $160 million, up 19% sequentially from $134 million, and up 20% from $133 million year-earlier.

Net income was negative $47 million, up sequentially from negative $105 million, and i[ from negative $82 million year-earlier.

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

Guidance:

Expects to end 2022 with about $2.0 billion in cash. Full 2022 revenue over $575 million. Net loss non-GAAP under $275 million.

Conference Highlights:

CEO Brett Monia said "We have made significant progress on our key priorities this year, building our commercial pipeline, delivering an abundance of new medicines to the market and expanding and diversifying our technology. We delivered positive data from eight key programs, positioning us to potentially add two new marketed medicines to our portfolio and expand our rich Phase 3 pipeline to eight medicines next year. Additionally, we have recently begun work on a manufacturing facility to support our pipeline growth." 8 positive data readouts so far in 2022. Tofersen is likely to be the next commercial product.

In October 2022 Ionis entered into a long-term lease in to construct a new manufacturing facility supporting continued growth. Also entered into a sale-leaseback transaction in for several real estate assets, generating net proceeds of $240 million plus full funding to expand the R&D campus.

In Q3 2022 presented positive data from the Phase 3 NEURO-TTRansform study of eplontersen in patients with polyneuropathy caused by hereditary TTR amyloidosis; on track to file a New Drug Application this year.

In Q3 2022 the NDA for tofersen was accepted and granted priority review by the FDA; PDUFA date April 25, 2023. Tofersen is licensed by Biogen for ALS.

In Q3 2022 Ionis initiated a confirmatory Phase 3 study of olezarsen in patients with severe hypertriglyceridemia. Also initiated a supporting Phase 3 study of olezarsen in patients with SHTG or hypertriglyceridemia and cardiovascular disease.

In Q3 2022 partner GSK presented positive end of study data from the Phase 2b B-Clear study of bepirovirsen demonstrating potential for functional cures in patients with chronic hepatitis B. GSK plans to advance bepirovirsen into Phase 3 development in the first half of 2023.

In Q3 2022 Ionis presented positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy. Roche plans to advance IONIS-FB-LRx into Phase 3 development in the first half of 2023.

In Q3 2022 Bayer presented positive data from the Phase 2b study of fesomersen in patients with end-stage renal disease. Ionis regained rights to fesomersen from Bayer and is assessing next steps.

In Q3 2022 AstraZeneca announced it is not advancing ION449 for hypercholesterolemia, even though the Phase 2b study met its primary endpoint targeting PCSK9.

In Q3 2022 Biogen sales of Spinraza were $431 million. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $23 million, up sequentially from negative $80 million, and up from negative $48 million year-earlier. No non-GAAP EPS given.

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

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.

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.

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.

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 $219 million, consisting of $2 million for cost of goods sold; $183 million for R&D and $34 million for selling, general and administrative. Operating income was negative $59 million. Other expense was $12 million. Income tax $0 million.

Q&A selective summary:

Eplonerson rational for cardiomyopathy study expansion? More internal and external data has become available, changing the population. Patients are aware earlier, so study was not powered properly, and we had to resize. But we did not extend the duration of the study. Enrollment is going well, we do not need to open new sites.

Tofersen extension? Delay to April 24, FDA did not request substantial new data, they just needed more time to review the data. We still assume there will be an Advisory Committee.

Lpa, view of olpasiran (Amgen/Arrowhead competitor) v. pelacarsen? Does not change our view on pelacarsen. Our Phase 2 dose was effective, our Phase 3 has a very effective dose, we are well ahead of the competition. There also can be problems from going too low on Lpa. Our Phase 2 trial was large, theirs was small, so we believe we have much less risk.

Fesomersen? We were hoping Bayer would take it forward based on the Phase 2 data. Bayer had 3 independent modalities for the indication. Bayer was focused on end-stage renal disease. We believe it can be used to prevent thrombosis, end state renal dialysis, etc. We will be looking for a partner to maximize the opportunity.

We expect to move our first SI into the clinic next year, we see advantages for SI and ASO, we are also looking at other molecule types.

HAE data on Sunday? We are hoping to show sustained efficacy for a year. Zero attack rate is the most important effect to patients. We had a 97% zero attack rate. The current standard of care still results in about 3 attacks per year.

Bepirovirsen for HBV? Combining agent at GSK, clearance rate in Phase 2 is unprecedented. With combinations should get even better results. GSK will take a comprehensive Phase 3 approach.

Cimdelirsen for acromegaly? We will review the data this year, not able to present until next year.

Any anticipated milestone payments soon? The next milestones would be on regulatory approval.

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