Analyst Conference Summary

biotechnology

conference date: November 2, 2023 @ 8:30 AM Pacific Time
for quarter ending: September 30, 2023 (Q3, third quarter 2023)

Forward-looking statements

Overview: Revenue declined y/y due to R&D revenue decline; waiting for new drug approvals.

Basic data (GAAP):

Revenue was $144 million, down 23% sequentially from $188 million, and down 10% from $160 million year-earlier.

Net income was negative $147 million, down sequentially from negative $91 million, and down from negative $47 million year-earlier.

EPS (diluted) was negative $1.03, up sequentially from negative $0.60, and down from negative $0.33 year-earlier.

Guidance:

Reaffirmed.

Conference Highlights:

CEO Brett Monia said "Eplontersen is on track for its first potential approval in the U.S. and is under regulatory review in the EU and Canada. We believe the positive efficacy and safety data coupled with an attractive self-administration dosing profile positions eplontersen to be the preferred therapy in the largely underserved hereditary ATTR polyneuropathy population. We reported positive data from the olezarsen Phase 3 Balance study in patients with familial chylomicronemia syndrome, showing statistically significant triglyceride lowering, substantial reductions in acute pancreatitis attacks and favorable safety and tolerability, positioning olezarsen to be our first independent commercial launch. We also made additional progress across our wholly owned and partnered pipeline, and further expanded our rich Phase 3 pipeline with the advance of zilganersen for patients with Alexander disease into Phase 3 development. Looking ahead, we expect to continue our positive momentum with the potential approval and launch of eplontersen and the Phase 3 data readout of donidalorsen in hereditary angioedema."

In Q4 2022 submitted the NDA for FDA approval of eplontersen. PDUFA is December 22, 2023. In Q2 2023 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 this year. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until 1H 2025. The EMA granted orphan drug status. Already has launched disease awareness campaigns. Hopes eplontersen will become the choice for first line ATTR polyneuropathy.

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. Preparing to submit NDA to FDA; if it received priority review, could become commercial in late 2024. In SHTG the Phase 3 data should be available in 2024.

In Q3 2023 zilganersen (GFAP) started Phase 3 development for the treatment of patients with Alexander disease.

Donidalorsen for hereditary angioedema was granted orphan drug status in Q3 2023. Phase 3 data should read out in H1 2024. Sees potential peak sales at $500 million per year.

In Q3 2023 the fDA granted orphan drug status to ulefnersen for FUS-ALS.

In Q3 2023 GSK reported positive data from the Phase 2b B-Together study of bepirovirsen followed by pegylated interferon in patients with chronic hepatitis B virus (HBV).

In Q3 2023 Biogen reported positive data from the Phase 1/2 study of IONIS-MAPTRx (BIIB080) in patients with Alzheimer's disease. Also Biogen Completed enrollment in the Phase 1/2 HALOS study of ION582 (BIIB121) in patients with Angelman syndrome.

In Q3 2023 Roche agreed to advance two novel Ionis programs for Alzheimer's and Huntington's.

Expenses increased modestly y/y on accelerated research plus preparations for commercial launches. In 2024 expects sustained Spinraza royalties, eplontersen milestone, and modest but ramping emplontersen royalties.

In April 2023 the FDA approved Qalsody (tofersen) for SOD1 ALS. The EU has accepted an MAA for review with decision expected in Q4 2023. Tofersen is licensed to Biogen.

In Q3 2023 Biogen sales of Spinraza were $448 million, up 4% from $431 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $117 million, sequentially down from negative $58 million, and down from negative $47 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.2 billion, down sequentially from $2.4 billion. Debt was $575 billion in convertible senior 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 $287 million, consisting of $2 million for cost of goods sold; $215 million for R&D and $70 million for selling, general and administrative. Operating income was negative $143 million. Other income was $2 million. Income tax benefit $6 million.

Q&A selective summary:

Eplontersen interactions with FDA, payers? Pleased with interactions with FDA. Filings have been accepted in Canada and Europe.

Eplontersen launch prep? AstraZeneca hired the sales team and they are in the field. Ionis nurse case managers have been hired. Payer expectations are good, does not believe prior authorization should be a significant problem.

Hep B situation? Good relationship with GSK, were aware of extra therapy licensing announcement. Tripling down on Bepirovirsen. The possible plan is a sequential multi-drug treatment to increase the % of cures.

Olezarsen pricing? We have been working with payers. The approval would be for a rare disease. With the more prevelant indication price would line up more in line with other products. FCS sales team will be calling on a small group of physicians that cover the FCS patients. With a broader label we will extend the size of the sales team.

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