Analyst Conference Summary

biotechnology

conference date: August 1, 2024 @ 8:30 AM Pacific Time
for quarter ending: June 30, 2024 (Q2, second quarter 2024)

Forward-looking statements

Overview: Revenue improvement mainly due to R&D collaboration revenue.

Basic data (GAAP):

Revenue was $225 million, up 89% sequentially from $119 million, and up 20% from $188 million year-earlier.

Net income was negative $66 million, up sequentially from negative $143 million, and up from negative $85 million year-earlier.

EPS (diluted) was negative $0.45, up sequentially from negative $0.98, and up from negative $0.60 year-earlier.

Guidance:

Maintained for 2024.

Conference Highlights:

CEO Brett Monia said "Over the first half of this year, we continued to deliver on our goal to bring a steady cadence of medicines to people with serious diseases. The Wainua launch for hereditary ATTR polyneuropathy (ATTRv-PN) continues to progress well with AstraZeneca. Qalsody is now approved in the EU, expanding the number of patients who can benefit from the first approved treatment for a genetic form of ALS. We are well positioned for our first independent launch with olezarsen, which was accepted for Priority Review with a December FDA action date for people with familial chylomicronemia syndrome. Additionally, we completed enrollment in our Phase 3 olezarsen program for the much larger severe hypertriglyceridemia (sHTG) patient population, keeping us on track for data in the second half of next year. Based on recent positive Phase 3 results, we believe donidalorsen, our second planned independent U.S. launch, is positioned to be a preferred choice for people with hereditary angioedema. We also advanced our next wave of potentially transformational medicines, including announcing plans to independently advance ION582 into a Phase 3 study next year, based on positive data in Angelman syndrome." Revenues will increase with new medicine introductions, but so will expenses. As revenues ramp will reach positive cash flow.

The Wainua launch is off to a good start. Believes royalties could add meaningfully to revenue in the growing ATTRv-PN market. 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. Expects EU approval in 2024. Expects Canada launch later this year. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until 1H 2025.

Olezarsen NDA submitted to FDA for FCS, with December 19, 2024 PDUFA. 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. Will also apply in EU. In SHTG (severe hypertriglyceridemia) the Phase 3 CORE data, and from 2 other Phase 3 trials, should be available in mid 2025. In Q1 2024 received Breakthrough Therapy and orphan drug status from the FDA.

Qalsody for SOD1-ALS was approved in the EU in Q2 2024.

Donidalorsen for hereditary angioedema was granted orphan drug status in Q3 2023. Phase 3 data positive top line data read out in Q1 2024. Showed statistically significant reduction in HAE attack rates. Sees potential peak sales at $500 million per year. EU rights were licensed to Otsuka. Preparing to submit the NDA in the US.

Zilganersen for Alexander disease Phase 3 study is fully enrolled, data expected in 2025.

Bepirovirsen for chronic hepatitis B Phase 3 study is fully enrolled, data expect in 2026.

In H1 2024 Ionis reported positive Phase 2 data for ION224 for MASH (metabolic dysfunction associated steatohepatitis). Looking to license this program.

In H1 2024 Ionis began a Phase 1/2 Orbit study of ION356 (PLP1) for PMD (Pelizaeus-Merzbacher disease).

But in Q2 2024 discontinued development of IONIS-FB-LRx for geographic atrophy and ION541 for amyotrophic lateral sclerosis ALS.

Neurology pipeline is transitioning from preclinical to clinical trials.

In Q2 2024 Biogen sales of Spinraza were $429 million, down 2% from $437 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $35 million, sequentially down from $112 million, and down from negative $58 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.08 billion, down sequentially from $2.21 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 $291 million, consisting of $4 million for cost of goods sold; $222 million for R&D and $65 million for selling, general and administrative. Operating income was negative $66 million. Other income was $0 million. Income tax $0 million.

Q&A selective summary:

ATTR-CM debate, endpoints? We do not see any evidence of a threshhold effect to achieve TTR lowering for endpoints. In CM the primary endpoint is in mortality and hospitalization.

CardioTransform study is advancing well. We are interested in other data readouts (competitors) on the silencer class.

Wainua launch insights? Pleased with our early insights. Seeing both new to treatment and switching and add-on patients. Believes it is a significant opportunity. Seeing patients from a variety of prescribers. Payers are covering Wainua very quickly. We also get feedback directly from patients, who like the autoinjectors.

Roche AngelMan program? We were pleased to see Roche data. Supports our confidence. We believe we have better efficacy at lower doses.

Monotherapy v. combinations for TTR-CM? Will be data driven. So far data does justify adding to current therapy, but also starting with monotherapy.

Pelacarsen study is on track, readout next year, partnered with Novartis. Follow on moleculte also going well.

FCS launch, opportunity size? Have built out our commercial functions. Hiring the field team. Expanded access will move patients from trials onto commercial drug, and new patients are being recruited. Should be ready to launch on approval.

Neurology after Angelman? It is a rich portfolio. Several studies enrolled. [see pipleine page]. We have a new backbone chemistry for neurology for less frequent doses, and we are working on transversing the blood-brain barier.

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