Analyst Conference Summary

biotechnology

conference date: February 21, 2024 @ 8:30 AM Pacific Time
for quarter ending: December 31, 2023 (Q4, fourth quarter 2023)

Forward-looking statements

Overview: Good quarter, boosted by $50 million milestone payment for Wainua.

Basic data (GAAP):

Revenue was $325 million, up 126% sequentially from $144 million, and up 114% from $152 million year-earlier.

Net income was negative $6 million, up sequentially from negative $147 million, and up from negative $208 million year-earlier.

EPS (diluted) was negative $0.06, up sequentially from negative $1.03, and up from negative $0.37 year-earlier.

Guidance:

224 revenue above $575 million. Non-GAAP operating loss less than $475 million. Ending cash and equivalents $1.7 billion. Continues to invest in growth opportunities.

Conference Highlights:

CEO Brett Monia said "This past year included many remarkable achievements as we continued to advance our vision to bring better futures to people with serious diseases. Ionis achieved two FDA approvals, delivered three positive Phase 3 data readouts, expanded our rich Phase 3 pipeline to nine medicines and advanced our next wave of wholly owned medicines as well as our technology. In 2024, we anticipate building on our success with important catalysts and continued value creation. The Wainua U.S. launch is underway for patients with hereditary ATTR polyneuropathy, and we expect additional approvals in other countries this year. We plan to present positive Phase 3 data for olezarsen in familial chylomicronemia syndrome and donidalorsen in hereditary angioedema, positioning Ionis to independently launch these two medicines. We also anticipate additional readouts from multiple mid-stage programs that, if positive, would advance into Phase 3 development, further strengthening our ability to deliver a steady cadence of potentially transformational medicines for years to come"

The Wainua launch is off to a good start. Preparing for possible independent launch of Olezarsen later this year.

In Q4 2023 the FDA approved Wainua (eplontersen) on December 21. In Q2 2023 had presented additional 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 with partner Astra-Zeneca outside the US. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until 1H 2025. In Q4 2023 eplontersen was granted Fast Track status by the FDA for ATTR-CM.

In Q3 2023 reported positive data Olezarsen for familial chylomicronemia syndrome from the Phase 3 trial. This included remarkable reductions in pancreatitis attacks. Would be the first Ionis independent commercial drug launch. Preparing to submit NDA to FDA; if it received priority review, could become commercial in late 2024. In SHTG the Phase 3 data should be available in 2024. In Q1 2024 received Breakthrough Therapy and orphan drug status from the FDA

In Q3 2023 zilganersen (GFAP) started Phase 3 development for the treatment of patients with Alexander disease.

Donidalorsen for hereditary angioedema was granted orphan drug status in Q3 2023. Phase 3 data positive top line data read out in Q1 2024. Sees potential peak sales at $500 million per year. EU rights were licensed to Otsuka.

In Q3 2023 the fDA granted orphan drug status to ulefnersen for FUS-ALS.

In Q3 2023 GSK reported positive data from the Phase 2b B-Together study of bepirovirsen followed by pegylated interferon in patients with chronic hepatitis B virus (HBV). In Q4 2024 the FDA granted Fast Track.

In Q3 2023 Biogen reported positive data from the Phase 1/2 study of IONIS-MAPTRx (BIIB080) in patients with Alzheimer's disease. Also Biogen Completed enrollment in the Phase 1/2 HALOS study of ION582 (BIIB121) in patients with Angelman syndrome.

In Q3 2023 Roche agreed to advance two novel Ionis programs for Alzheimer's and Huntington's.

ION582 (BIIB121) in Q4 2023 completed enrollment in hte Phase 2 study for Angelman Syndrome. Positive clinical data presented.

In Q4 2023 ION826 for heart failure was licensed to AstraZeneca, resulting in a $36 million milestone payment.

In Q4 2023 Biogen sales of Spinraza were $413 million, down 10% from $459 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income $20 million, sequentially up from negative $117 million, and up from negative $183 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.3 billion, up sequentially from $2.2 billion. Debt was $1.19 billion in convertible senior notes. Plans to pay off 2024 convertible notes.

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, with 12 in clinical development. A growing number are wholly-owned.

GAAP Operating expense was $331 million, consisting of $3 million for cost of goods sold; $257 million for R&D and $71 million for selling, general and administrative. Operating income was negative $6 million. Other income was $3 million. Income tax $6 million.

Q&A selective summary:

CardioTransform to continue as planned, unblinding? Versus Alnylam product? No comment on other companies. No changes in plan to read out early. Key driving factor (with AstraZeneca) is blinded event rate. Expect that in 2025. But still could let study play out to completion. Believe we were right to lengthen and expand study.

Tafamadis future? Certainly a dynamic market. Cardiomyopathy has a 3 to 6 year survival rate. So we want strong survival data. We believe Tafamadis will be first line, before silencers. But believes physicians will want to use 2 classes of medication once Tafamadis goes generic.

Overall (combination) population v. monotherapy? Overall population primary endpoint is totality of data. We expect that to be positive. We are very confident.

Angelman? Is an open label study. Goal was to enable progression and dose determination. Readout midyear. Biogen has an option to license. Will release the data readout and Biogen decision at the same time.

There was no requirement for disease progression, whether on Tafamadis or not, to enroll in the eplontersen cardiomyopahty study. Most Tafamadis patients do progress, and many get little benefit from that treatment.

Wainua launch details? Joint teams are executing well. Well positioned. Going well.

Geographic Atrophy study? Data end of this year. Two doses tested. Should have advantages over injections into the eye. Roche is excited.

Payers for combo cardiomyopathy criteria? You need to convince the clinicians. If you can show clinically meaningful results, they will make a case to the payers. We believe because they are different classes and mechanisms, payment will be allowed.

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