Why did total revenue jump so much in Q2 2024?

Net revenues from collaborations rose to $227.3 million from $5.8 million, largely because about $185 million of previously deferred revenue was recognized when the Regeneron collaboration was modified. This was a one-time accounting effect, so it should be excluded when assessing underlying growth. Net product revenues, which grew 34%, are the better measure of commercial momentum.

What did the HELIOS-B results show and why does it matter?

HELIOS-B met its primary endpoint, showing a statistically significant 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events overall, and 33% in the monotherapy population. All-cause mortality fell 36% (overall) and 35% (monotherapy). The results could support an expansion of AMVUTTRA into ATTR cardiomyopathy, a large patient population, but the indication is not yet approved and the company plans a supplemental NDA using a Priority Review Voucher, so regulatory outcome and timing remain uncertain.

Is Alnylam now profitable?

Not on a GAAP basis for the full period. Q2 2024 GAAP operating income was $48.6 million and the GAAP net loss was $16.9 million, while six-month GAAP net loss was $82.8 million. The quarter was helped by the one-time collaboration revenue. Non-GAAP net income was $73.8 million, which excludes stock-based compensation, a measure management uses to evaluate ongoing performance.

What drove the guidance increase and what are the risks?

The increase in combined net product revenue guidance to $1.575-1.650 billion reflects strong AMVUTTRA uptake and continued growth in GIVLAARI and OXLUMO. Risks include the pace of AMVUTTRA adoption in the ATTR-CM market, competition, reimbursement and pricing, dependence on partners such as Sanofi, Regeneron, Vir and Novartis, and the regulatory review timeline. Operating expense guidance also rose, to $2.0-2.15 billion GAAP for R&D and SG&A.

What catalysts should investors watch in the second half of 2024?

Key catalysts include the presentation of detailed HELIOS-B results at ESC on August 30, 2024, and the accompanying webcast; the planned sNDA submission for vutrisiran in ATTR-CM; the TTR Investor Day on October 9, 2024; interim results from the mivelsiran Phase 1 Alzheimer's study; and initiation of a Phase 3 ALN-TTRsc04 study in ATTR-CM at or around year-end.

ALNY 8-K SEC Disclosure

01—Abstract

Summary

Summary of the 8-K filed August 1, 2024. Cited figures are marked; the original filing is the authoritative source.

Alnylam Pharmaceuticals reported second quarter 2024 results on August 1, 2024, led by strong commercial execution in its TTR franchise.

Global net product revenues reached $410 million^a, up 34% year over year (35% at constant exchange rates), with AMVUTTRA growing 74% to $230 million and total TTR revenues rising 37% to $307 million. Rare disease products (GIVLAARI and OXLUMO) grew 25% to $103 million. Total revenues were $660 million, but this figure was inflated by roughly $185 million of previously deferred collaboration revenue recognized upon the amended Regeneron agreement, so the headline top line overstates underlying run-rate growth.

Profitability improved markedly. GAAP operating income was $48.6 million^b versus a loss of $229.8 million a year earlier, and GAAP net loss narrowed to $16.9 million ($0.13 per share) from $276.0 million ($2.21 per share). Non-GAAP net income was $73.8 million ($0.56 per diluted share).

Cost of goods sold fell to 16.4% of net product revenues from 24.6%. Operating expenses rose, with GAAP R&D up 18% and GAAP SG&A up 16%, reflecting clinical startup costs for zilebesiran and mivelsiran, higher TTR marketing spend, and higher stock-based compensation. Cash, cash equivalents and marketable securities totaled $2.62 billion^c at June 30, 2024.

The most consequential development is positive topline data from the HELIOS-B Phase 3 study of vutrisiran in ATTR cardiomyopathy, which met its primary endpoint with a 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events (33% in the monotherapy population) and a 35-36% reduction in all-cause mortality. Management raised full-year 2024 combined net product revenue guidance to $1.575^d-1.650 billion from $1.400-1.500 billion. Investors should note that the revenue guidance raise is driven by the strong AMVUTTRA trajectory, while full data from HELIOS-B are not yet available and the cardiomyopathy indication is not approved.

02—Key points

Key Highlights

Q2 2024 global net product revenues of $410.1 million, up 34% YoY (35% at CER), driven by AMVUTTRA at $230.1 million (+74% YoY).
Total TTR franchise revenue of $307.4 million (+37% YoY), with U.S. TTR growth of 40%.
Total revenues of $659.8 million included about $185 million of previously deferred collaboration revenue tied to the amended Regeneron agreement, a one-time boost to the quarter.
GAAP net loss narrowed to $16.9 million ($0.13 per share) from $276.0 million a year earlier; non-GAAP net income was $73.8 million ($0.56 per diluted share).
HELIOS-B Phase 3 met its primary endpoint (28% reduction overall, 33% monotherapy) and showed a 35-36% all-cause mortality reduction; detailed results to be presented at ESC on August 30, 2024.
2024 combined net product revenue guidance raised to $1.575-1.650 billion from $1.400-1.500 billion; collaboration and royalty revenue guidance raised to $575-650 million.
Cash, cash equivalents and marketable securities of $2.62 billion at June 30, 2024, up from $2.44 billion at year-end 2023.

03—Questions

Frequently Asked Questions

Why did total revenue jump so much in Q2 2024?
Net revenues from collaborations rose to $227.3 million from $5.8 million, largely because about $185 million of previously deferred revenue was recognized when the Regeneron collaboration was modified. This was a one-time accounting effect, so it should be excluded when assessing underlying growth. Net product revenues, which grew 34%, are the better measure of commercial momentum.
What did the HELIOS-B results show and why does it matter?
HELIOS-B met its primary endpoint, showing a statistically significant 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events overall, and 33% in the monotherapy population. All-cause mortality fell 36% (overall) and 35% (monotherapy). The results could support an expansion of AMVUTTRA into ATTR cardiomyopathy, a large patient population, but the indication is not yet approved and the company plans a supplemental NDA using a Priority Review Voucher, so regulatory outcome and timing remain uncertain.
Is Alnylam now profitable?
Not on a GAAP basis for the full period. Q2 2024 GAAP operating income was $48.6 million and the GAAP net loss was $16.9 million, while six-month GAAP net loss was $82.8 million. The quarter was helped by the one-time collaboration revenue. Non-GAAP net income was $73.8 million, which excludes stock-based compensation, a measure management uses to evaluate ongoing performance.
What drove the guidance increase and what are the risks?
The increase in combined net product revenue guidance to $1.575-1.650 billion reflects strong AMVUTTRA uptake and continued growth in GIVLAARI and OXLUMO. Risks include the pace of AMVUTTRA adoption in the ATTR-CM market, competition, reimbursement and pricing, dependence on partners such as Sanofi, Regeneron, Vir and Novartis, and the regulatory review timeline. Operating expense guidance also rose, to $2.0-2.15 billion GAAP for R&D and SG&A.
What catalysts should investors watch in the second half of 2024?
Key catalysts include the presentation of detailed HELIOS-B results at ESC on August 30, 2024, and the accompanying webcast; the planned sNDA submission for vutrisiran in ATTR-CM; the TTR Investor Day on October 9, 2024; interim results from the mivelsiran Phase 1 Alzheimer's study; and initiation of a Phase 3 ALN-TTRsc04 study in ATTR-CM at or around year-end.

04—Continue

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