Ionis reports second quarter 2021 financial results and recent business achievements

Key Phase 3 milestones move tofersen, eplontersen and pelacarsen closer to the market

Exclusive license to Bicycle Therapeutics' technology potentially expands LICA technology capabilities

Webcast today, August 4, 2021, at 11:30 a.m. Eastern Time

CARLSBAD, Calif., Aug. 4, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial results for the second quarter of 2021 and recent business achievements.

"Since our last quarterly update, we continued to execute on our strategic objectives to prepare for multiple Ionis commercial launches, expand our drug delivery capabilities and advance new products towards the market. 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," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "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."

Second Quarter 2021 and Recent Summary Financial Results

• Second quarter results reflect focus on Ionis' strategic objectives
• $126 million in total revenues
• $154 million of operating expenses on a non-GAAP basis(1) and $199 million on a GAAP basis
• Net loss of $36 million on a non-GAAP basis(1) and $81 million on a GAAP basis
• Well capitalized with cash and investments of $2.1 billion at the end of the second quarter

"In addition to advancing our pipeline and expanding our drug discovery capabilities, we have taken multiple steps to streamline our operations in support of our wholly owned medicines. We have completed the integration of Akcea, entered distribution arrangements with Sobi and restructured our commercial operations. These steps enabled us to unlock significant resources that we are redirecting towards our highest priority programs," said Elizabeth L. Hougen, chief financial officer of Ionis. "We remain on track to achieve our 2021 revenue guidance of more than $600 million. We continue to expect increased R&D revenue in the second half of this year. Already in the third quarter, we earned $25 million from Novartis for the pelacarsen enrollment milestone. We are revising our 2021 operating expense and net loss guidance because of our license of Bicycle's technology. Importantly, we remain well-capitalized with the resources we need to achieve our strategic objectives."  

Revised 2021 Financial Guidance

• Updated operating expense and net loss guidance because of Ionis' license of Bicycle's technology(1)