Analyst Conference Summary

biotechnology

conference date: October 27, 2022 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2022 (third quarter, Q3)

Forward-looking statements

Overview: Revenue continues to expand rapidly, but still deep in the red.

Basic data (GAAP):

Revenue was $264 million, up 17% sequentially from $225 million, and up 40% from $188 million year-earlier.

Net income was negative $406 million, down sequentially from negative $277 million, and down from negative $205 million year-earlier.

Diluted EPS was negative $3.32, down sequentially from negative $2.29, and down from negative $1.72 year-earlier.

Guidance:

For 2022 reduced expected royalty and collaboration revenue from $175 to $225 million, now $100 to $150 million. Otherwise unchanged.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "The third quarter of 2022 was one of strong execution across our commercial and clinical development operations. Amvuttra has completed its first full quarter on the market since its U.S. approval and launch in June and is off to a great start, demonstrating the potential of our RNAi therapeutics portfolio in patients with hATTR amyloidosis with polyneuropathy which achieved 30% U.S. growth compared to Q2. We’re also looking forward to potentially expanding the opportunity for this franchise and are encouraged by the positive APOLLO-B Phase 3 results for patisiran in patients with ATTR amyloidosis with cardiomyopathy presented recently. We are on track to submit an sNDA for ONPATTRO by year-end and hope to bring this treatment option to patients in 2023 assuming successful regulatory review and approval." Some negative impact from FX. Believes cash is sufficient to reach cash-flow break even.

In Q3 2022, based on the successful outcome of the ILLUMINATE-C study in children and adults with advanced PH1, received approval from the FDA of an sNDA for Oxlumo, expanding the indication for the treatment of PH1 to lower urinary oxalate and plasma oxalate levels in pediatric and adult patients, also received approval from the EMA of a Type II variation to include the ILLUMINATE-C data in the label.

In Q3 2022 Alnylam filed a Clinical Trial Application for ALN-TTRsc04, the first clinical program from the IKARIA platform, aimed at achieving an annual dosing regimen with highly potent and reversible effects of TTR lowering.

Onpattro (patisiran) Apollo-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy topline results were positive (Q3 2022) and sNDA on track to file by end of 2023.

In Q2 2022 Alnylam started a Phase 1 study of ALN-XDN for gout and a Phase 1 trial of ALN-APP for Alzheimer's. Could have top line results in late 2022.

In Q3 enrollment and dose escalation continue in the Phase 1 study of ALN-APP in patients with early onset Alzheimer's Disease, in collaboration with Regeneron, and initial results are now expected in early 2023.

In Q3 2022 Alnylam issued $1.04 billion convertible senior notes, with proceeds primarily used to pay down the Blackstone $700 million credit facility and $200 million in prepayment premiums under the credit facility, the purchase of capped call transactions, and underwriter fees.

Non-GAAP net income negative $193 million, up sequentially from negative $246 million, and down from negative $190 million year-earlier. EPS negative $1.58, down sequentially from negative $2.03, and down from negative $1.59 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.27 billion, up sequentially from $2.11 billion.

In Q2 2022 the FDA approved Amvuttra (Vutrisiran or ALN-TTRsc02) for polyneuropathy of hATTR amyloidosis. Expects to launch in EU in 2022, already has a positive opinion from the CHMP. Will now seek to expand the label. Helios-A Phase 3 study reported positive topline results for polyneuropathy in April 2022. Also current Phase 3 Helios-B study for cardiomyopathy is fully enrolled, readout in early 2024.

Vir Biotechnology reported ALN-HBV02 (VIR-2218) had good Phase 2 results for HBV in Q2 2022.

In Q2 2022 Cemdisiran for complement mediated diseases reported positive topline Phase 2 results for immunoglobin A nephropathy. Working with Regeneron to finalize Phase 3 trial plans.

ALN-APP Phase 1 topline results are expected in late 2022.

Leqvio (inclisiran) royalties from Novartis could rise after FDA approval for the treatment of adults with clinical atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia who require additional lowering of LDL-C.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS-PPX study reported positive results in Q2 2022. Sanofi Genzyme is a partner in the program. In Q4 2020 received Fast Track Designation from the FDA. Filing date moved back to 2024 to allow for a lower dose cohort.

Novartis launch of Leqvio (inclisiran) for is ongoing. Guidance for 2022 was lowered in part because collaboration and royalty revenues from this partnership are trending lower than expected as of late October 2022.

ALN-HSD reported preliminary Phase 1 results for NASH in Q3 2022.

In Q2 ALN-TTRsc04 was introduced for a potential annual dosing regimen, with IND filing planned for end of 2022.

See also Alnylam pipeline.

Operating expenses of $522 million consisted of: $37 million for cost of goods sold; $245 million for research and development; and $236 million for general and administrative expense; $5 million cost of collaboration. Operating loss $258 million. Interest & other expense was $148 million. $0 million income tax.

Q&A Selective Summary:

FDA feedback on Apollo-B data? Delighted with study results, meaningful and significant benefits and safety. Confident in our data package. Filing by year end. There will be a 180 day safety update. KOL feedback is good.

Decision to skip Helios-B interim? Positive Apollo-B data reinforces our view of strenth of Helios-B data. Interim cuts into alpha. Also study is overenrolled, so full readout highly likely positive.

Cardiomyopathy potential update v. tafamidis? Majority in real world has no drug right now, about 25% have been on tafamidis. Study readouts are not about subgroups. But believes diagnosis will continue to climb, and patients on tafamidis continue to progress. Won't know more until we get the FDA label.

Amvuttra v. Onpattro for new patients? About half of new patients on each at present. Expect to grow the market and get earlier treatment. Believe trend should be similar ex-US. Still a significant unmet need, Amvuttra has an attractive profile.

Apollo-B approvability given only one study, etc.? Seventh phase 3 study by Alnylam, all work is guided by regulators. Original Apollo cardiac subgroup was encouraging, regulators agreed, Apollo-B is the follow up.

The Inflation Reduction Act does change incentives, including for orphan drugs with multiple targets. Evaluating implications, but believe Alnylam is well-positioned.

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