Analyst Conference Summary

biotechnology

conference date: April 30, 2025 @ 8:30 AM Pacific Time
for quarter ending: March 31, 2024 (Q1, first quarter 2025)

Forward-looking statements

Overview: Improved y/y revenue, strong pipeline.

Basic data (GAAP):

Revenue was $132 million, down 42% sequentially from $227 million, and up 11% from $119 million year-earlier.

Net income was negative $147 million, dpwm sequentially from negative $104 million, and down from negative $143 million year-earlier.

EPS (diluted) was negative $0.93, down sequentially from negative $0.66, but up from negative $0.98 year-earlier due to increased share count.

Guidance:

Increased revenue guidance for 2025 substantially to $725 to $750 million. Non-GAAP operating loss reduced to $375 million. Cash at year end increased to $1.9 billion.

Conference Highlights:

CEO Brett Monia said "With an encouraging start to the Tryngolza launch for familial chylomicronemia syndrome, the first of four independent launches expected over the next two years, Ionis’ new chapter as a fully integrated, commercial-stage biotechnology company is well underway. We look forward to continued momentum this year, including our second independent launch for donidalorsen in hereditary angioedema and Phase 3 results for olezarsen for severe hypertriglyceridemia in the third quarter. We also continue to advance our next wave of wholly owned neurology medicines, including ION582 for Angelman syndrome, which is on track to start Phase 3 development shortly. Our advancing pipeline of transformational medicines, together with strong commercial and financial execution, position Ionis to deliver increasing value for all stakeholders." Commercial product revenue was up 28% y/y. Sees long-term peak annual revenue over $5 billion.

Ionis believes it has a clear path to positive cash flow.

In Q1 2024 Ionis licensed sapablursen global development and commercialization rights to Ono Pharmaceutical, generating $280 million in an upfront payment in q2 2025. Potential to earn up to $660 million in additional payments plus royalties in the mid-teen percentage range

In Q2 2024 IONIS-MAPTRx (BIIB080) received FDA Fast Track designation for the treatment of Alzheimer's disease; licensed to Biogen.

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 EU approval in Q2 2025. [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, with $6 million Q1 2025 sales. Working on reimbursements, most in Q1 were given special access. Already seeing prescriptions and repeats; well received by physicians. But most potential patients are undiagnosed; estimated U.S. total is just 3,000. EU approval likely in H2 2025. Licensed global rights ex U.S., Canada, and China to Sobi. 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. Estimated U.S. sHTG patioants over 1 million.

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.

Non-GAAP numbers: net income negative $118 million, sequentially down from negative $68 million, and down from negative $112 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.1 billion, down sequentially from $2.3 billion. About $1.2 billion in long-term debt (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 13 in clinical development, and 9 in Phase 3 trials. A growing number are wholly-owned.

GAAP Operating expense was $278 million, consisting of $1 million for cost of goods sold; $201 million for R&D and $76 million for selling, general and administrative. Operating income was negative $146 million. Other expense net was $1 million. Income tax $0 million.

Q&A selective summary:

Olezarsen color? Tryngolza launch strong, currently targetting cardiologists, a few thousand. For sHTG the overlap with FCS among physicians is signicant. Many patients in trial have AP (acute pancreatitis). Large unmet need. AP is a secondary endpoint in the studies.

Donidalorsen marketing prep? HAE market has 9 out of 10 patients interested in trying new therapies. Efficacy, tolerability, and convenience should be sales drivers. It is a switch market, hopes peak sales over $500 million. Sales team is being hired and trained.

Tariffs? FDA delays? No changes so far at FDA for us. We see no meaningful impact on our business from tariffs in the near term. We make our own drugs for clinical trials, withs some help from CMOs.

FCS price erosion with sHTG launch? We have moved to payments for FCS very quickly. We will find more patients in the 18 months or so before the sHTG launch. FCS price based on value and population size. Have not set price yet for sHTG, but population still small enough for specialty pricing.

Tryngolza sales reflect demand, no inventory effect in Q1.

Wainua, Part D out of pocket cap effect? AstraZeneca sees strong demand in the U.S. for Wainua. Expects new patient starts and higher volume because more affordable, also improve compliance rates. Also expects growth from ex-US launches.

Cardiomyopathy, competition with Alnylam, pricing? Up the them how that market evolves. The Wainua pricing strategy will be determined by AstraZeneca.

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