Analyst Conference Summary

biotechnology

conference date: February 22, 2022 @ 8:30 AM Pacific Time
for quarter ending: December 31, 2022 (Q4, fourth quarter 2022)

Forward-looking statements

Overview: Revenue decline due mainly to large single license fee in 2021. Still in the red.

Basic data (GAAP):

Revenue was $152 million, down 5% sequentially from $160 million, and down 65% from $ million year-earlier.

Net income was negative $52 million, down sequentially from negative $47 million, and down from $225 million year-earlier.

EPS (diluted) was negative $0.37, down sequentially from negative $na, and down from $1.41 year-earlier.

Guidance:

For the full year 2023 Ionis expects revenue of at least $575 million. But non-GAAP op ex of $970 to $995 million will result in a net operating loss of up to $425 million. Cash and equivalents at end of 2023 should be near $2.0 billion.

Conference Highlights:

CEO Brett Monia said "We made substantial progress in 2022, marked by important achievements, including the December submission of the eplontersen NDA for people with ATTRv-PN. We also delivered multiple positive data readouts, enabling us to advance and expand our rich late- and mid-stage pipeline. And we took important steps to expand and diversify our technology, including our Metagenomi collaboration to add DNA editing to our platform." In 2023 Ionis hopes for FDA and EU approvals of Tofersen and an FDA approval of Eplontersen. Building a commercial organization for launches. Expects many data readouts in 2023. Expects olezarsen to be a substantial driver of future growth.

In October 2022 Ionis entered into a long-term lease in to construct a new manufacturing facility supporting continued growth. Also entered into a sale-leaseback transaction in for several real estate assets, generating net proceeds of $240 million plus full funding to expand the R&D campus.

In Q1 2022 Ionis increased its balance sheet with over $700 million from royalty monetization and sale and leaseback transactions.

In Q4 2023 submitted the NDA for FDA approval of eplontersen. In Q3 2022 presented 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 parnter Astra-Zeneca outside the US this year.

In Q3 2022 the NDA for tofersen was accepted and granted priority review by the FDA. An advisory committee meeting is scheduled for March 22, 2023. PDUFA date April 25, 2023. The EU accepted an MAA for review. Tofersen is licensed by Biogen for ALS.

In Q4 2022 Ionis reported positive Phase 2 data from the open label extension study of donidalorsen in patients with hereditary angioedema; data showed clinically meaningful improvement in angioedema quality of life score. The Phase 3 study is expected to complete enrollment in 2023.

In Q1 2023 GSK advanced bepirovirsen into Phase 3 development in patients with chronic hepatitis B. Ionis earned a $15 million milestone payment in Q1 from that event. In Q3 2022 GSK presented positive end of study data from the Phase 2b B-Clear study of bepirovirsen, demonstrating potential for functional cures in patients with chronic hepatitis B.

Biogen has partnered with Metagenomi to add gene editing to its platform.

In Q4 2022 Biogen sales of Spinraza were $459 million, up from $431 million in Q3. Biogen continues to work to expand sales.

Non-GAAP numbers: net income negative $168 million, down sequentially from negative $23 million, and down from $248 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.0 billion, down sequentially from $na billion. Debt was $1.17 billion in convertible senior notes.

In Q1 2023 partner Biogen initiated a Phase 2 study of IONIS-MAPTRx (BIIB080) in patients with mild cognitive impairment or mild dementia due to Alzheimer's disease

In Q1 2023 partner Roche initiated a Phase 2 study of tominersen in patients with prodromal or early manifest Huntington's disease

In Q1 2023 partner Biogen initiated a Phase 1 study of ION306 (BIIB115) for the treatment of SMA (spinal muscular atrophy) with the potential for long interval dosing

In Q4 2022 the FDA granted olezarsen Fast Track designation. In Q3 2022 Ionis initiated a confirmatory Phase 3 study of olezarsen in patients with severe hypertriglyceridemia. Also initiated a supporting Phase 3 study of olezarsen in patients with SHTG or hypertriglyceridemia and cardiovascular disease.

In Q3 2022 Ionis presented positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy. Roche plans to advance IONIS-FB-LRx into Phase 3 development in the first half of 2023.

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 $360 million, consisting of $4 million for cost of goods sold; $308 million for R&D and $48 million for selling, general and administrative. Operating income was negative $208 million. Other income was $164 million. Income tax $8 million.

In 2023 will have 6 Phase 3 clinical programs underway, which is driving expenses higher.

Q&A selective summary:

NDA acceptance timeline for eplontersen? Completed NDA process in December, cleared 60-day review period. Expect the official notice, day-74 letter will give the PDUFA date. Expects a standard review, with no Ad Com. Process has gone smoothly, no concerns expessed so far by the FDA.

Eplontersen ATTR-CM timeline? Phase 3 enrollment should complete midyear, seeing significant uptick in blinded event rates. Seeing a good patient balance. Data readout is on track for 2025.

Analyam ATTR-CM competitor? Prefer not to comment on competitor products, but not surprised their results were complicate.

We are on pause for business development activities.

Monthly dosing is one of the key competitive advantages for donidalorsen, as is efficacy.

2023 revenue is likely to be back-end loaded. Confident guidance is achievable even if eplontersen approval is delayed.

Biogen SMA return to growth plan? Starting in the US with stabilization and a bit of increase Q3/Q4. Will see those dynamics in other geographies. Teens and adults are where Spinraza has proven safety and efficacy, so that would be the growth population. Biogen sees Asia as a particular opportunity.

None of the 2023 op ex guidance is for the new manufacturing facility. That will flow into op ex as amortization starting around 2026.

We see no read-through from other HBC programs to ours; if anything it clarifies an enhanced competitive landscape for bepirovirsen.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: