Analyst Conference Summary

biotechnology

conference date: August 9, 2023 @ 8:30 AM Pacific Time
for quarter ending: June 30, 2023 (Q2, second quarter 2023)

Forward-looking statements

Overview: Qalsody (tofersen) approved by FDA in April.

Basic data (GAAP):

Revenue was $188 million, up 44% sequentially from $131 million, and up 40% from $134 million year-earlier.

Net income was negative $91 million, up sequentially from negative $124 million, and down from negative $86 million year-earlier.

EPS (diluted) was negative $0.60, up sequentially from negative $0.87, and up from negative $0.74 year-earlier.

Guidance:

On track to hit prior 2023 guidance.

Conference Highlights:

CEO Brett Monia said "Ionis is creating significant value in 2023 as we successfully execute on our strategy to bring a steady cadence of transformational medicines to the market. Our growing late-stage pipeline now includes eight medicines for 10 indications, highlighted by eplontersen. Based on the strong and consistent data generated to date and an attractive self-administration profile, we expect eplontersen to be an important new medicine for people with ATTRv-polyneuropathy. We also added Qalsody to our commercial portfolio, a breakthrough treatment for people with SOD1-ALS further strengthening Ionis' leadership in RNA-based therapies for neurological diseases. In the second half, we expect continued positive momentum including the Phase 3 readout for olezarsen in familial chylomicronemia syndrome and the potential approval of eplontersen for ATTRv-PN in December." Building a commercial organization for launches. Expects many data readouts in 2023. Expects olezarsen to be a substantial driver of future growth. Q2 revenue growth was driven by partner payments, not sales. Expenses are increasing as planned about 20% y/y to advance the pipeline and go-to-market activity. Near-term commercial opportunities have a combined multi-billion dollar sales peak potentia.

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 Q2, earned $16 million in a milestone payment from Biogen for Qalsody approval in the U.S.

In Q2 Biogen presented data demonstrating IONIS-MAPTRx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer's disease.

In Q2 2023 Ionis completed enrollment in the Phase 3 donidalorsen study for HAE (hereditory angiodema). Believes differentiated from competition.

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. In Q2 2023 licenses South American rights to Astra-Zeneca. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until 2025.

In 2023 expects the olezarsen for familial chylomicronemia syndrome Phase 3 readout. Would be the first Ionis independent commercial drug launch. In SHTG the Phase 3 data should be available in 2024.

In Q2 2023 interim Spinraza Phase 4 data for SMA showed it met unmet medical needs for patients who had gene therapy.

In Q2 2023 Roche advanced IONIS-FB-LRx into Phase 3 development in patients with IgA, or immunoglobulin A nephropathy. Phase 2 data should be available in 2024.

In Q2 2023 GSK reported more positive bepirovirsenfor HBV Phase 2 data.

In Q2 2023 Phase 2 enrollment was completed for IONIS-FB-LRx for geographic atrophy.

In Q2 2023 Ionis agreed to collaborate with Novartis on a next-generation Lp(a) program.

R&D revenue for Q2 2023 included $20 million from AstraZeneca for its share of the global Phase 3 development costs for eplontersen. Also $15 million from Biogen for Qalsody approval.

In Q2 2023 Biogen sales of Spinraza were $437 million, up from $431 million year-earlier. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $58 million, sequentially from negative $na million, and up from negative $80 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.4 billion, up sequentially from $2.3 billion. In June 2023 issued $575 million senior converibles due 2028, mostly used to repurchase $434 million of 2024 convertibles. Debt was $1.3 billion in convertible senior notes. $115 million is earmarked to repay remaining 2024 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. A growing number are wholly-owned.

GAAP Operating expense was $279 million, consisting of $3 million for cost of goods sold; $230 million for R&D and $46 million for selling, general and administrative. Operating income was negative $91 million. Other income was $14 million. Income tax $8 million.

Q&A selective summary:

Olezarsen outcomes study? Absolutely not. We are focussed on severe metabolic diseases like FCS from severely elevated triglicerides. We are preparing for launch. Unmet need is great. 3.5 million potential patients (with label expansion), currently poor standard of care.

siRNA options in ATTR-PN market, competition with? We are excited with our week 85 data. Halting disease progression, even seeing improvement in some patients. Also easier administration. Believes those edges will allow entry into market where only 20% of patients have been treated so far.

Eplontersen in other markets? Working with AstraZeneca we plan to be first to market in several international markets, including in Europe, eastern Europe, Russia, China and Japan.

Lpa new molecule timeline? Novartis proposed to us follow-on pelacarsen, which is doing well, on track for 2025 filing, then first in market. Follow up program is to reach more patients. We have expanded our chemistry capabilities over the last few years. But tough to beat pelacarsen efficacy. Focus would be on less frequent dosing, maybe annual. [no timeline yet, still in research phase]

[More eplontersen questions. Basically, because Tegsedi and Waylivra lost out to the Alnylam equivalents, the question is can it compete against Alnylams's upgrades for ATTR therapies.]

Angelman syndrome program? It is going well, with Biogen, enrolling Phase 1/2 patients, should have data mid 2024.

Donidalorsen preparation? We have a switch study to give physicians confident in switching from the standard of care.

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