Analyst Conference Summary

biotechnology

conference date: August 4, 2021 @ 8:30 AM Pacific Time
for quarter ending: June 30, 2021 (Q2, second quarter 2021)

Forward-looking statements

Overview: tofersen likely to become next Ionis commercial therapy.

Basic data (GAAP):

Revenue was $126 million, up 13% sequentially from $112 million, and down 14% from $146 million year-earlier.

Net income was negative $81 million, up sequentially from negative $90 million, and down from negative $26 million year-earlier.

EPS (diluted) was negative $0.57, up sequentially from negative $0.64, and down from negative $0.18 year-earlier.

Guidance:

2021 revenue guidance unchanged at over $600 million. But op ex upped to $710 to $750 million, resulting in net loss estimate of $110 million.

Conference Highlights:

Tofersen Phase 3 study completed dosing. Ionis hopes to have 12 or more marketed products in 2026.

CEO Brett Monia said "Biogen completed dosing in the tofersen Phase 3 VALOR study and began offering tofersen to SOD1-ALS patients on an individual compassionate use basis. We achieved full enrollment in the eplontersen Phase 3 NEURO-TTRansform study and 50 percent enrollment in the pelacarsen Phase 3 Lp(a) HORIZON study. Additionally, we licensed Bicycle Therapeutics' technology to expand the capabilities of our LICA technology. Looking ahead, we expect data from multiple pipeline programs, including additional data supporting the potential for our IONIS-PKK-LRx program to change the standard of care for patients with hereditary angioedema. And by this fall, we expect data from the Phase 3 VALOR study of tofersen in patients with SOD1-ALS. If results from the VALOR study are positive, we expect tofersen to be our next commercial medicine. These key recent achievements and upcoming catalysts keep us on track for a regular cadence of Phase 3 data and new drug applications, leading to 12 or more products on the market in 2026."

Already in the third quarter, earned $25 million from Novartis for the pelacarsen enrollment milestone. Revising 2021 operating expense and net loss guidance because of the license of Bicycle's technology.

Spinraza sales by Biogen were $500 million. Over 11,000 patients being treated. Spinraza royalty rate resets each year. Reached the highest royalty tier in Q2 2021. New data supports greater use.

Waylivra (volanesorsen) is approved in the EU for the treatment of adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis. Commercially available in Germany, Austria, Greece, and France. Filed in Brazil. Now distributed in Europe by Sobi. Not approved in U.S.

Tegsedi (Inotersen) is licensed and sold by Akcea. Tegsedi was approved in the EU for polyneuropathy in adults with hATTR (hereditary transthyretin amyloidosis). Now commercial in 15 countries, including Portugal, with additional EU launches planned. Now distributed in Europe by Sobi. Expanding into Canada and Latin America.

Non-GAAP numbers: net income negative $36 million, up sequentially from negative $90 million, and down from $15 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.06 billion, up sequentially from $1.82 billion. Debt was $1.2 billion in convertible senior notes. Ionis repurchased no shares of common stock for a total purchase price of $0 million.

In Q2 2021 Roche reported tominersen data related to the halting of the Phase 3 program.

Eplontersen for TTR polyneuropathy Phase 3 full enrolled in Q2 2021.

Pelacarsen for Lp(a) driven cardiovascular disease achieved 50% enrollment in Q2 2021, resulting in a $25 million milestone payment from Novartis.

ALS therapies are in Phase 3 studies partnered with Biogen. ION541 advanced into Phase 1/2 development in patients with nearly all forms of ALS. Has multiple preclinical programs in development for ALS. ION464 advanced into Phase 1/2 development in patients with multiple system atrophy.

Tofersen (IONIS-SOD1Rx) Phase 3 study completed dosing for ALS patients with SOD-1 mutation with data expected in 2H 2021. Also launched Phase 3 Atlas trial for presymptomatic SOD1-ALS.

Vupanorsen positive Phase 2 results were presented at ESC 2020. In Q4 2020 Vupanorsen advanced into Phase 2b development with the initiation of the dose-ranging study in statin-treated patients with dyslipidemia, resulting in a $75 million payment from Pfizer

IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema reported positive topline data in Q1 2021. Advancing into a Phase 3 study. IONIS-PKK-LRx advanced into an investigator-initiated study in hospitalized COVID-19 patients in Brazil in 2020.

The Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS began in Q4 2020. Positive Phase 2 results were presented at ESC 2020. Granted Fast Track designation by FDA.

The IONIS-ENAC-2.5Rx program has been discontinued as of May 2021 because of toxicology issues.

In Q2 2021 advanced ION363 into a Phase 3 trial for patients with FUS-ALS.

In Q1 2021 advanced ION373 into Phase 2 for Alexander disease.

In Q1 2021 advanced ION541 into Phase 2 for ALS, resulting in a $10 million payment from Biogen.

In Q2 2021 made a license agreement with Bicycle Therapeutics for rights to peptide technology to enhance LICA.

The Phase 3 study for the AKCEA-TTR-LRx continued enrollment.

Ionis continues to develop technologies that allow RNA therapies to almost any part of the body, including inhaled agents.

Ionis expects to initiate 8 Phase 2 or 3 studies in 2021.

Ionis has a pipeline of 45 potential drugs. A growing number are wholly-owned.

GAAP Operating expense was $199 million, consisting of $3 million for cost of goods sold; $139 million for R&D and $57 million for selling, general and administrative. Operating loss was $73 million. Other expense was $8 million. Income tax $0 million.

Expects $59 million in savings in 2021. However, operating expenses will increase in 2H 2021 as new trials continue to start.

Q&A summary:

Huntington's disease plan? Roche is analyzing the data. Next steps by year end.

Vupanorsen? Pfizer expects Phase 2b data this year. High cholesterol and triglyceride patient population, a very large population.

Respiratory franchise plan? We had a preclincial issue, cannot speak to competition. We are making good preclinical progress that could move the pulmonary program back to the clinic in the future.

Phase 3 HAE study steps? Finalizing Phase 3 trial design, discussing logistics, talking to regultaors. Hope to start by end of year, could be Q1 2022.

Bicycle compared to other approaches? Yes, we did considerable comparable work before making the deal. We are excited because we wanted better ligands and they have developed them for multiple proteins. Key advantage is small size of the peptides, much smaller than antibodies, if it works.

Your tau approach v. antibody approach? Biogen plans to advance into a larger Phase 2 study to look for clinical benefits. Tau is intracellular, which is where RNAi works, but antibodies do not reach the interior of the cells. That only works in the CFS, but you need to lower tau in the cells throughout the brain.

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