Analyst Conference Summary

biotechnology

conference date: May 7, 2024 @ 8:30 AM Pacific Time
for quarter ending: March 31, 2024 (Q1, first quarter 2024)

Forward-looking statements

Overview: Declining revenue, but could launch Olezarsen this year.

Basic data (GAAP):

Revenue was $119 million, down 63% sequentially from $325 million, and down 9% from $131 million year-earlier.

Net income was negative $143 million, down sequentially from negative $6 million, and down from negative $124 million year-earlier.

EPS (diluted) was negative $0.98, down sequentially from negative $0.06, and down from negative $0.87 year-earlier.

Guidance:

On track to achieve prior 2024 guidance.

Conference Highlights:

CEO Brett Monia said "Ionis is off to a great start in 2024, as we continue to execute on our vision to bring better futures to people with serious diseases. The Wainua launch for hereditary ATTR polyneuropathy is progressing well with AstraZeneca. And we are one step closer to our first independent launch with our NDA submission for olezarsen, which is supported by robust data positioning olezarsen to make a profound difference for people with FCS. We look forward to presenting positive Phase 3 donidalorsen data, along with data from our open-label extension and 'switch' studies in patients with HAE at EAACI later this month, setting the stage for Ionis' second independent launch. Additionally, we have multiple upcoming data readouts from our mid-stage programs that, if positive, could advance into Phase 3 development."

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 Canada launch later this year. 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.

Olezarsen NDA submitted to FDA; if it receives priority review, could become commercial in late 2024. 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

In Q1 2024 Bepirovirsen was granted Fast Track for chronic hepatitis B.

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.

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).

Neurology pipeline is transitioning from preclinical to clinical trials.

In Q1 2023 Biogen sales of Spinraza were $341 million, down 23% from $443 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $112 million, sequentially down from $20 million, and down from negative $97 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.21 billion, down sequentially from $2.3 billion. Debt was $1.2 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 $269 million, consisting of $2 million for cost of goods sold; $214 million for R&D and $53 million for selling, general and administrative. Operating income was negative $150 million. Other income was $7 million. Income tax $0 million.

Q&A selective summary:

Olezarsen patients that could roll over to commercial drug? Most rolled over. Expanded access program just getting started. Donidalorsen similarly.

Wainua access? Going well, led by Astra-Zeneca. Payer landscape is positive.

Olezarsen timeline? Expect to hear from FDA 60 days after our April submission. Then should get a PDUFA date. Do not expect an Ad Com. They will also decide on a priority review. We have marketing capabilities in place, also data analytics and medical affairs group. We learned from the Wainua launch. FCS is rare, a small number of patients and doctors, so an appropriate sales force.

Donidalorsen is mainly a switch market. About 20,000 patients US + EU. Focus on durability and reduced rates of attacks, plus longer between dosing.

Path to positive cash flow? The late stage pipeline is a multi-billion revenue opportunity. But it will take a few years. We are not that far away from being cash flow positive.

Non-genetic ALS? This first study is mainly a dosing and safety study. With Biogen.

The hereditary angioedema market is a dissatisfied one, with patients often switching among the current therapies.

Cardiomyopathy, competitor data readout? Blinded event rates in our trial are tracking well, both mortality and hospitalizations. No changes to our plans. Our timing for reading out the study could still change, but is currently 2026.

ION582 for Angelman syndrom? Biogen has an option, so they could be making the decisions going forward.

We think our Medicare Part D situation for cardiomyopathy is good and should get better as costs to patients decrease.

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