Analyst Conference Summary

biotechnology

conference date: February 19, 2025 @ 8:30 AM Pacific Time
for quarter ending: December 31, 2024 (Q3, fourth quarter 2024)

Forward-looking statements

Overview: Revenue down y/y on lower R&D revenue. Product sales revenue grew. Getting ready for new launches.

Basic data (GAAP):

Revenue was $227 million, up 69% sequentially from $134 million, and down 30% from $325 million year-earlier.

Net income was negative $104 million, up sequentially from negative $140 million, and down from negative $9 million year-earlier.

EPS (diluted) was negative $0.66, up sequentially from negative $0.95, and down from negative $0.06 year-earlier.

Guidance:

For full year 2025 expects revenue over $600 million, non-GAAP operating loss under $495 million, ending cash and equivalents around $1.7 billion.

Conference Highlights:

CEO Brett Monia said "With the recent launch of our first independent medicine, Tryngolza for familial chylomicronemia syndrome, Ionis has begun a new chapter as a fully integrated commercial-stage biotechnology company,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “Over the next three years, we expect three more independent launches, including donidalorsen later this year for hereditary angioedema and olezarsen for severe hypertriglyceridemia in 2026, pending Phase 3 results in the second half of this year. Additionally, our partners are on track to launch four Ionis-discovered medicines over the same time period, including several that address broad patient populations. Ionis continues to advance our next wave of potentially transformational wholly owned medicines, including ION582 for Angelman syndrome, which is on track to start Phase 3 development in the first half of this year. Our recent achievements, combined with our strong commercial execution and advancing pipeline, position Ionis to deliver increasing value for all our stakeholders." Expects revenue to shift more towards commercial sales and royalties and away from R&D reimbursement. Sees possible peak annual sales at the end of this decade at around $5 billion.

Revenue for 2024 was substantially higher than guidance. Ionis believes it has a clear path to positive cash flow.

Wainua revenue continues to grow about 1 year after approval. Believes royalties could add meaningfully to revenue in the growing ATTRv-PN market. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until later in 1H 2025. Launches are underway in several nations. Received a positive CHMO opinion in the EU. [But competing drug for ATTR-CM now on market from Alnylam]

Tryngolza (Olezarsen) for familial chylomicronemia syndrome (FCS) approved by FDA on December 19, 2024. Is the first Ionis independent commercial drug launch. Working on reimbursements. Already seeing prescriptions and repeats; well received by physicians. But most potential patients are undiagnosed. EU approval likely in 2025. In SHTG (severe hypertriglyceridemia) the Phase 3 CORE data, and from 2 other Phase 3 trials, should be available in H2 2025. If positive would submit the sNDA by end of 2025.

ION582 for Angelman syndrome should start Phase 3 in the first half of 2025. Data from the Phase 1/2 trial were positive.

Donidalorsen for hereditary angioedema (HAE) 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.

Ulefnersen for FUS-ALS global rights sold to Otsuka; in Phase 3.

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 $68 million, sequentially up from negative $108 million, and down from $17 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.3 billion, down sequentially from $2.5 billion. About $1.2 billion in long-term debt.

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 $337 million, consisting of $4 million for cost of goods sold; $245 million for R&D and $88 million for selling, general and administrative. Operating income was negative $110 million. Other expense net was $3 million. Income tax benefit $3 million.

Q&A selective summary:

Olizarsen SHTG Essense study implications? This patient population is elevated but not severe HTG. Mainly for safety, but also looking for triglyceride reduction.

TTR polyneuropathy market? Q/q was 82%, so demand is strong and growing. New to brand share is about 40% for Wainua. We expect continued growth in 2025.

SHTG trial design, acute pancreatitis, abdominal pain? The 2 Phase 3 studies are powered for triglyceride reduction. Pancreatitis not studied before, this population has a lower triglyceride level, for assume lower pancreatitis, but study is 10 times as large. Not intended to be powered for pancreatitis, currently no known clinical threshhold for this.

Polyneuropathy growth? Less than 20% of potential patients are on a treatment. Physicians are positive about Wainua. Most patients have no out of pocket expense and like self-administration. So some physicians are choosing Wainue over other treatments.

Tryngolza treatment metrics? Label is broad. First mover advantage. Strong positive feedback from all groups. We had product in channel end of 2024. Several hundred patients are currently identitied, but they need to come in and be formally diagnosed. Number of prescribing physicians is good.

Patients for FCS can be confirmed genetically or clinically. We don't have numbers on this. Genetic diagnosis has been taking 1 to 2 weeks.

SHTG patients need to get to below 500 mg/dL to be out of the severe range. There is still risk then, but it is a goal for many patients, currently trying to manage by diet, etc.

SHTG, FCS outside the U.S.? Looking for an international partner to do both outside the U.S.

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