Analyst Conference Summary

biotechnology

conference date: November 6, 2024 @ 8:30 AM Pacific Time
for quarter ending: September 30, 2024 (Q3, third quarter 2024)

Forward-looking statements

Overview: Weak revenue, but getting ready for possible new approvals.

Basic data (GAAP):

Revenue was $134 million, down 40% sequentially from $225 million, and down 7% from $144 million year-earlier.

Net income was negative $140 million, down sequentially from negative $66 million, and up from negative $147 million year-earlier.

EPS (diluted) was negative $0.95, down sequentially from negative $0.45, and up from negative $1.03 year-earlier.

Guidance:

Expects to end 2024 with $2.2 billion in cash and equivalents. 2024 revenue of $575 million. Non-GAAP operating loss $475 million.

Conference Highlights:

CEO Brett Monia said "Today, we stand on the cusp of a new era for Ionis, with our first co-commercialization launch proceeding well with Wainua, our first planned independent launch fast approaching and continued strong progress across our rich pipeline. With an upcoming December FDA action date, we are ready to independently bring olezarsen to people with familial chylomicronemia syndrome, a serious rare disease with no approved treatments in the U.S. We are similarly well positioned for our second independent launch for donidalorsen, which we believe could become a preferred treatment choice for people with hereditary angioedema, with an FDA action date of August 21, 2025. In parallel, we are making great progress across the rest of our rich Phase 3 pipeline. We expect Phase 3 results supporting olezarsen's second indication in severe hypertriglyceridemia and pelacarsen in Lp(a)-driven cardiovascular disease next year, as well as Phase 3 results supporting eplontersen's second indication in ATTR cardiomyopathy in the second half of 2026. We are also advancing our next wave of potentially transformational wholly owned medicines, including ION582 for Angelman syndrome, which we expect to enter Phase 3 development in the first half of next year"

Investing both in the current and near-term launches and in the long-term pipeline. [Probably code for expenses will go up faster than revenue for a while]

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. Preparing of first independent commercial launch.

ION582 for Angelman syndrome should start Phase 2 in the first half of 2025. Data from the Phase 1/2 trial were positive.

Donidalorsen for hereditary angioedema submitted an NDA in the US. PDUFA date is August 21, 2025. Otsuka is planning to submit the European MAA. HAE has over 20,000 patients in the US and EU. Preparing for commercial launch.

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).

IONIS-FB-LRx reported positive Phase 2 data in immunoglobulin A nephropathy (IgAN). Partner Roche continues the Phase 3 study.

Neurology pipeline is transitioning from preclinical to clinical trials.

In Q3 2024 Biogen sales of Spinraza were $381 million, down 15% from $429 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $108 million, sequentially down from negative $35 million, and up from negative $121 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.5 billion, up sequentially from $2.08 billion. Debt was $ billion in convertible senior notes. About $1.2 billion in long-term debt. In Q3 2024 raised $500 million with a stock offering.

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 13 in clinical development, and 9 in Phase 3 trials. A growing number are wholly-owned.

GAAP Operating expense was $282 million, consisting of $1 million for cost of goods sold; $220 million for R&D and $61 million for selling, general and administrative. Operating income was negative $148 million. Other income was $4 million. Income tax benefit $4 million.

Q&A selective summary:

Launch timeline, labelling, pricing of olezarsen? In labelling discussions with the FDA. Commercial and sales team are ready to go on December 19, should have product in channel by end of year. Will launch with ultra-rare pricing, which we will announce upon approval.

Angelman Phase 3 details? No plans for an interim data look. Robust design. FDA supportive in end of Phase 2 meeting.

Cardiotransform study? Pleased with Wainua study. We were considering an early readout, but given the new competitor, we and Astra-Zeneca decided to go the full 140 week timeline. So 2H 2026.

FCS patient identification, potential competitor? Sales team is helping physicians identify patients. We have an open label extension ongoing. We expect patient identification to go up once olizarsen is on the market. We believe the 9 month lead over the competition will allow us to succeed.

FCS payer dynamics? We have been working with payers, with a 4,000 patient maximum, they have been very understanding of the likely price point. We believe we can get prior authorizations for patients very quickly.

Ultragenyz competition in Angelman? Declined to comment on their trial design, reiterated own trial design.

The donidalorsen market is very competitive but still underserved.

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