Analyst Conference Summary

biotechnology

conference date: November 2, 2023 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2023 (third quarter, Q3)

Forward-looking statements

Overview: Continued strong revenue growth and pipeline development. Numbers jumped from high collaboration income.

Basic data (GAAP):

Revenue was $751 million, down 235% sequentially from $319 million, and up 184% from $264 million year-earlier.

Net income was $148 million, up sequentially from negative $276 million, and up from negative $406 million year-earlier.

Diluted EPS was $1.15, down sequentially from negative $2.21, and down from negative $3.32 year-earlier.

Guidance:

Update to 2023 guidance: net reveue from collaborations and royalties increased to 4575 to $625 million.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "While we were disappointed by the recent Complete Response Letter for Oboattri for the treatment of the cardiomyopathy of ATTR amyloidosis, we remain confident in the HELIOS-B Phase 3 study of vutrisiran, which is on track to deliver topline results in early 2024. We're also excited about advancements across our broad pipeline of RNAi therapeutics, including recent positive topline results from the KARDIA-1 Phase 2 study of zilebesiran, as well as upcoming Phase 1 results for ALN-TTRsc04 and ALN-KHK by year-end. With this continued execution we remain on track to meet our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial results."

On July 24, 2023 Alnylam announced an agreement with Roche to develop zilebesiran for hypertension in patients with high cardiovascular risk. Will receive $310 upfront and is eligible for $2.8 billion in milestones. Profit sharing 50/50 in the U.S. and royalties on ex-US sales. The drug is currently in a Phase 2 trial. Positive Phase 1 results were published in Q1 2023.

By the end of 2023 Alnylam expects topline Phase 1 ALN-TTRsc04, Phase 1 ALN-KHK, and Phase 2 ALN-HBV02 data.

Zilebesirin for hypertension reported positive Phase 2 topline results in October 2023. Will partner with Roche for global development and distribution. Results to be announced November 11, 2023 at American Heart Association meeting.

In Q3 2023 Leqvio (inclisiran) was approved in China and Japan. US launch ongoing. Leqvio results in royalty income.

On August 3, 2023 Alnylam announced an exclusive worldwide license agreement with Agios Pharmaceuticals to develop and commercialize an siRNA targeting TMPRSS6 for polycythemia vera and related iron-overload disorders.

Vir is conducting multiple trials evaluating the potential for ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional cure for chronic hepatitis B. Phase 2 data readouts are on track for Q4 2023.

In Q3 2023 the FDA issued a Complete Response Letter (CLR) for Onpattro (patisiran) for in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy. Alnylam had announced topline results were positive in Q3 2022.

Amvuttra (Vutrisiran) Phase 3 for ATT cardiomyopathy is expected to have 30 month topline results in early 2024.

Alnylam will not proceed with regulatory filings for biannual dosing of Amvuttra, instead advancing ALN-TTRsc04, which could work with annual dosing. Began dosing in the Phase 1 trial in Q1 2023.

In August 2023, Massachusetts closed its case on Onpattro marketing with no action taken.

Net revenue from collaborations was $427 million, up from $29 million year-earlier. The spike was mainly from an upfront payment from Roche for zilebesirin. Royalty revenue was $10 million, up from $3 million year-earlier.

Non-GAAP net income $228 million, up sequentially from negative $202 million, and up from negative $193 million year-earlier. EPS $1.83, up sequentially from negative $1.62, and up from negative $1.58 year-earlier.

Cash and equivalents balance at the end of the quarter was $2.40 billion, up sequentially from $2.06 billion. Debt increased to $1.02 billion.

See also Alnylam pipeline.

Operating expenses of $537 million consisted of: $79 million for cost of goods sold; $253 million for research and development; and $199 million for general and administrative expense; $5 million cost of collaboration. Operating income $214 million. Interest & other expense was $58 million. $3 million income tax.

Q&A Selective Summary:

Statistical plan for Helios-B cardiovascular ATTR Amvuttra? Focus is on deaths and hospitalizations. We have differential follow up.

Helios-B advisory committee, do you need better outcomes than tafamadis? Patisiran trend bodes well for Helios-B. Add on question for Onpattro was because tafamadis had a mortality claim but Onpattro was a functional, 6 minute walk path claim. That raises questions about using the drugs in combination or in sequence. Contrast expected outcomes results from Helios B, with larger population and longer follow up.

Helios-B data will inform how we position against tafamadis, and with payers.

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