Alnylam Pharmaceuticals : Q1 2026 Presentation ›

Published: 2026-05-04 17:28:17 ET ALNY

Published on 05/04/2026 at 05:28 pm EDT

April 30, 2026

Welcome

Overview

Commercial Highlights 04 Pipeline

Financial Summary

Q&A Session

Christine Akinc

Chief Corporate Communications Officer

Yvonne Greenstreet, M.D.

Chief Executive Officer

Tolga Tanguler

Chief Commercial Officer

Pushkal Garg, M.D.

Chief Research & Development Officer

Jeff Poulton

Chief Financial Officer

This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding: Alnylam's ability to achieve the goals in its "Alnylam 2030" strategy, including to achieve global TTR leadership, grow through sustainable innovation, and scale with discipline and agility, and to become the leading science-driven, fully integrated biopharmaceutical company and to maximize the potential of RNAi therapeutics for patients; Alnylam's ability to achieve sustained long-term growth; the size and future growth of the patient population with ATTR-CM; the future success of the launch of AMVUTTRA in ATTR-CM; AMVUTTRA's potential as a first-line therapy for ATTR-CM; Alnylam's ability to expand the number of prescribers of AMVUTTRA in the future and for prescribers' experience with AMVUTTRA to translate into a durable preference; Alnylam's ability to obtain regulatory approvals for AMVUTTRA in ATTR-CM in additional territories and to expand AMVUTTRA's patient reach around the world; the potential for any of Alnylam's collaborations to achieve the goals for which they were established; the number of patients who will be enrolled in Alnylam's clinical trials, including the TRITON-CM trial; the timing of initiation of, completion of enrollment in, or announcement of results from, any of Alnylam's clinical trials; the potential for any of Alnylam's product candidates to successfully complete clinical development and to receive regulatory approval and launch commercially, and the timing of any such commercial launches; the potential efficacy, treatment effect and product profiles of any of Alnylam's products and product candidates, including nucresiran, ALN-6400, zilebesiran, and ALN-HTT02; the potential for the programs in Alnylam's pipeline to change the practice of medicine, to propel Alnylam into its next phases of growth, and to accelerate growth through innovation at Alnylam in the years to come; the potential for Alnylam to achieve more substantial quarter-over-quarter growth in U.S. TTR revenues and across the world during the remainder of 2026; and Alnylam's projected commercial and financial performance, including the expected range for 2026 of TTR net product revenues, Rare net product revenues, total net product revenues, net revenues from collaborations and royalties, and non-GAAP R&D and SG&A expenses should be considered forward-looking statements.

Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam's ability to successfully execute on its "Alnylam 2030" strategy; Alnylam's ability to successfully launch, market and sell Alnylam's approved products globally, including AMVUTTRA; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam's marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the "Risk Factors" filed with Alnylam's most recent periodic report (Quarterly Report on Form10-Q or Annual Report on Form 10-K) filed with the SEC and in its other SEC filings. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

This presentation references non-GAAP financial measures. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. Percentage changes in revenue growth at Constant Exchange Rates, or CER, is a non-GAAP financial measure which is presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. CER represents growth calculated as if the exchange rates had remained unchanged from those used during the prior fiscal year.

Yvonne Greenstreet, M.D.

Chief Executive Officer

Pipeline Progress

TTR Leadership Strong Financial Performance

$910M

(+153% YoY)

Q1 Total TTR Net Revenues

$1,036M

(+121% YoY)

Q1 Total Net Product Revenues

New Data Further Support Benefits

of Vutrisiran in ATTR-CM and Potential of Zilebesiran for Hypertension Management

First Quarter >$1B Product Revenues

Strategic Investments for TTR Leadership

$4,900M

Initiated Phase 1 Trial of ALN-2232,

First Adipose Directed RNAi Therapeutic Targeting ACVR1C

to $5,300M

(+71% midpoint)

Reiterated 2026 Total Net Product Revenue Guidance

Established, Sustainable Innovation Engine and Commercial Excellence to Drive Durable Growth

Leadership in RNAi, with a Proven, Organic Product Engine

High-Yielding Pipeline with

>25 Active Clinical Programs

6 Medicines Collectively Generating

Multi-Billion-Dollar Revenues

Accelerating Innovation. Scaling Impact.

Achieve Global TTR Leadership

BUILD A DURABLE TTR FRANCHISE

Lead TTR market in revenue by 2030 and cumulatively across 5-year period

Launch best-in-class, next-gen silencer, nucresiran, in PN by 2028 and CM by 2030

Grow Through Sustainable Innovation

DELIVER THERAPIES THAT PREVENT, HALT, OR REVERSE DISEASE

Deliver 2+ new transformative medicines beyond TTR with blockbuster potential

Expand to 10 tissue types and > 40 clinical programs

Invest ~30% of revenues in non-GAAP R&D, including select external innovation

Scale with Discipline & Agility

DRIVE SUSTAINED, PROFITABLE GROWTH

Achieve 25%+ total revenue CAGR through YE 2030

Deliver ~30% non-GAAP operating margin

Tolga Tanguler

Chief Commercial Officer

Q1 2026 Overall Portfolio

$1,036M

Combined Net Product Revenues

+121%

YoY growth1 vs. Q1'25

+4%

QoQ growth1 vs. Q4'25

TTR Franchise

Rare Franchise

1 With FX impact. For growth at CER = constant exchange rate - see the Financial Summary slide for more information. 9

$126M

Total Rare Global Q1 2026

Net Product Revenues

Q1 2026 Rare Franchise Highlights

Revenues ($M)

$109M

+15%

$38M

$44M

$49M

$56M

$50M

$55M

$32M

$31M

$32M

$34M

$23M

$18M

$16M

$21M

$17M

$30M

$14M

$26M

$23M

$25M

$128M $127M

$136M

QoQ % Growth

YoY % Growth

GIVLAARI

-14%

11%

OXLUMO

22%

TOTAL Rare

-8%

15%

$126M

Q1 2025 Q2 2025 Q3 2025 Q4 2025 Q1 2026

GIVLAARI YoY +11% growth highlights:

~16% YoY increase in global patients on therapy

Partially offset by higher gross-to-net deductions in our US and International markets

OXLUMO YoY +22% growth highlights:

~21% YoY increase in global patients on therapy

Favorable impact of timing of orders in partner markets offset by higher gross-to-net deductions in our US and International markets

FX tailwind +5% (YoY CER1 growth = 10%)

1 CER = Constant exchange rate, which is a non-GAAP financial measure that represents growth calculated as if exchange rates had remained unchanged from those used during 2025 - see the Financial Summary slide for more information. 10

$910M

Total TTR Global Q1 2026

Net Product Revenues

Q1 2026 TTR Franchise Highlights

QoQ % Growth

YoY % Growth

U.S.

234%

ROW

-3%

35%

Global

153%

Revenues ($M)

Q1 2025 Q2 2025 Q3 2025 Q4 2025 Q1 2026

U.S. Q1'26 vs. Q4'25 (QoQ) +9% growth highlights:

+153%

$910M

+$52M Global TTR

vs. Q4

$858M

$187M

$724M

$185M

-$7M ROW TTR

vs. Q4

$20M

$544M

$359M

+$59M U.S. TTR

vs. Q4

$22M

$198M

$34M

$361M

$112M

$30M

$530M

$131M

$27M

$642M

$703M

$10M

$155M

$10M

$12M

$16M

Continued demand growth (+9%) driven by ATTR-CM uptake, despite Q1 headwinds related to benefits reverification and shipping dynamics

Favorable impact of inventory channel build fully offset by increase in gross-to-net deductions

U.S. Q1'26 vs. Q1'25 (YoY) +234% growth highlights:

Strong demand growth driven by ATTR-CM launch

Partially offset by lower net price

ROW YoY +35% growth primarily driven by uptake in ATTR-CM sales in Japan, continued ATTR-PN patient growth, both partially offset by negative price impact primarily driven by German launch in ATTR-CM

Modest FX tailwind +3% (YoY CER1 growth = 150%)

Established 1L leadership among HCPs who have tried AMVUTTRA

>90% first-line access;

improved vs. 2025

Majority pay $0 OOP

~90% of patients in the

U.S. can receive AMVUTTRA within

~10 miles of home

Robust category growth

Source: Data on file 12

>1,200

new AMVUTTRA prescribers since CM launch

+77%

since competitive launches

(CAGR: Dec'24 - Dec'25)

>90%

patient retention rate QoQ

Significant opportunity

U.S. metrics 13

Commercial Access Unlocked

Launched in U.S., Japan, Germany, Austria, UK, Switzerland, and Italy

Strong Forward Momentum

Advancing pricing and reimbursement negotiations

LAUNCHED IN PROCESS

and others

Pushkal Garg, M.D.

Chief Research & Development Officer

PHASE 1

PHASE 2

PHASE 3

TTR

Nucresiran

ATTR Amyloidosis with Cardiomyopathy

Nucresiran

hATTR Amyloidosis with Polyneuropathy

CARDIOVASCULAR

Zilebesiran2

Hypertension

Zilebesiran REVERSIR2

Hypertension

METABOLIC

Rapirosiran (ALN-HSD)1

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

ALN-ANG31

Diabetic Kidney Disease

ALN-4324 (GRB14)

Type 2 Diabetes Mellitus

ALN-2232 (ACVR1C)

Obesity & Weight Management

ALN-PNP3

Non-Alcoholic Fatty Liver Disease (NAFLD)

ALN-APOC31

Dyslipidemia

ALN-CIDEB1

MASH

NEUROSCIENCE

Cemdisiran1

Myasthenia Gravis

Mivelsiran

Cerebral Amyloid Angiopathy

Mivelsiran

Alzheimer's Disease

ALN-HTT024

Huntington's Disease

ALN-5288 (MAPT)4

Alzheimer's Disease

ALN-SOD3

SOD1 Amyotrophic Lateral Sclerosis

ALN-SNCA1

Parkinson's Disease

HEMATOLOGY

Cemdisiran1

Paroxysmal Nocturnal Hemoglobinuria

ALN-6400 (PLG)

Bleeding Disorders

AG-236 (ALN-TMP)1

Polycythemia Vera

ALN-CFB1

Paroxysmal Nocturnal Hemoglobinuria

OTHER

Cemdisiran1

Geographic Atrophy

Elebsiran1

Hepatitis D Virus Infection

ALN-BCAT

Hepatocellular Carcinoma

ALN-4285

Healthy Volunteers

ALN-4915

Healthy Volunteers

ALN-F12021

Healthy Volunteers

1 Out-licensed with milestones and/or royalties; 2 Partnered, Alnylam-led development with U.S. profit split and milestones/royalties ex-U.S.; 3 Partner-led with profit split; 4 Partnered, Alnylam-led with profit split 16

12.5%

≥90-day gap

13.4%

≥60-day gap

Percentage of patients (%)

100

93.8%

Adherent

87.5%

86.6%

100

6.2%

Non-adherent

Rate of persistence

Rate of discontinuation

Real-world data demonstrate

high adherence and persistence of quarterly HCP-administered dosing with vutrisiran1

120

Rates of persistence and

discontinuation at 12 months3

Adherence to vutrisiran

over treatment period2

Percentage of patients (%)

Vutrisiran reduced the risk of all-cause mortality and recurrent CV events and improved HELIOS-B endpoints irrespective of patients' baseline diastolic dysfunction grade4

1 Hefferman, et al. ACC 2026; 2 Patients were considered adherent if PDC ≥0.8 (PDC, proportion of days covered); 3 The rate of persistence was calculated as 100% minus the rate of discontinuation; 4 Cuddy et al. ACC 2026 17

Next-generation silencer with potential for greater TTR knockdown, improved efficacy, and biannual dosing

Targeting Launch

by 2030

Randomized, double-blind, event-driven outcomes study of nucresiran versus placebo

- Enrollment expanded from N ~1,250 to ~1,750

Primary endpoint of all-cause mortality and recurrent CV events

Targeting Launch

by 2028

Randomized, open-label study of nucresiran versus vutrisiran internal reference (N ~125)

Primary endpoint of mNIS+7 at Month 9 in nucresiran versus APOLLO placebo (similar to HELIOS-A)

- Continued data collection through Month 18

Nucresiran is an investigational therapeutic in development for the treatment of ATTR amyloidosis. The safety and efficacy of nucresiran have not been established or approved by the FDA, EMA or any other health authority. 18

Delivering Therapies that Prevent, Halt, or Reverse Disease

Deliver 2+ new transformative medicines

beyond TTR with blockbuster potential

Expand to 10 tissue types & >40 clinical programs

Invest ~30% of revenues in non-GAAP R&D, including select external innovation

Nucresiran

ATTR Amyloidosis

Advance TRITON-CM Phase 3 Trial

Ongoing

Advance TRITON-PN Phase 3 Trial

Ongoing

Zilebesiran

Hypertension

Advance ZENITH Phase 3 Trial

Ongoing

Mivelsiran

Cerebral Amyloid Angiopathy

Complete Enrollment of cAPPricorn-1 Phase 2 Trial

Alzheimer's Disease

Initiate Phase 2 Trial

ALN-6400

Bleeding Disorders

Initiate Phase 2 Trial in Second Bleeding Disorder

Phase 1 Data in Healthy Volunteers

Phase 2 Results in HHT

ALN-4324

Type 2 Diabetes Mellitus

Initiate Phase 2 Trial

ALN-HTT02

Huntington's Disease

Phase 1 Data

ALN-2232

Obesity & Weight Management

Phase 1 Data

Additional Programs

File 3-4 INDs

2026

Products mentioned above are not approved for any indication and conclusions regarding the safety or effectiveness of these drugs have not been established. 20

HEMATOLOGY

CARDIOVASCULAR

+ METABOLIC

NEUROSCIENCE

ALN-6400

ZILEBESIRAN

ALN-HTT02

Targeting Plasminogen to Address Wide Range of Bleeding Disorders

Targeting Angiotensinogen to Achieve Continuous Control of Blood Pressure

Targeting Exon 1 of Huntington Gene to

Reduce Progression of Huntington's Disease

>3M people in U.S. estimated to have inherited bleeding disorders1

>60M patients in 7 major markets with uncontrolled hypertension and high CV risk5

>100K symptomatic HD patients globally7

Hereditary Hemorrhagic Telangiectasia (HHT) 2nd most common inherited bleeding disorder2 70K estimated HHT patients in the U.S.3

2 doses per year

>75% reductions in HTT8

>90% Reductions in circulating PLG4 and clinical Proof of Mechanism

Phase 2 HHT results and Phase 1 healthy volunteer data in 2H26

Continuing enrollment in ZENITH Phase 3 CVOT; Launch expected ~20306

Initial Phase 1 data expected 2H26

The safety and efficacy of ALN-6400, zilebesiran, and ALN-HTT02 have not been established or approved by the FDA, EMA or any other health authority. 1 www.bleeding.org/make-a-difference/join-us/bleeding-disorders-awareness-month. 2 Blood (2023) 142 (Supplement 1): 28. 3

Internal estimate based on market research. 4 Data in non-human primates. 5 Internal estimate based on NCD-RisC, 2021; Nguyen T & Chow C, Lancet (2021); Muntner P et al., Hypertension (2020). 6 Launch assumes positive clinical trial results and regulatory approval.

Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche. 7 Internal estimate based on market research. 8 Data in non-human primates. ALN-HTT02 is being developed in partnership with Regeneron Pharmaceuticals. 21

HEMATOLOGY

ALN-6400

Potential pipeline-in-a-product to treat many bleeding disorders

CARDIOVASCULAR

+ METABOLIC

NEUROSCIENCE

ZILEBESIRAN

Potential to reduce the risk of cardiovascular events by providing continuous control of blood pressure

ALN-HTT02

Unique targeting strategy aiming to reduce progression of Huntington's disease

Jeff Poulton

Chief Financial Officer

($ in millions except where noted as percentages)

Q1 2025

Q1 2026

Q1 2026 vs

Q1 2025

(Reported)

Q1 2026 vs Q1 2025 (CER2)

Total Net Product Revenues

$469

$1,036

121%

117%

Net Revenues from Collaborations & Royalties

126

131

Collaboration Revenue

-17%

Royalty Revenue

85%

Total Revenues

594

1,167

96%

93%

Total Cost of Goods Sold, Collaborations & Royalties

211

Gross Margin on Product Revenues

85%

80%

Gross Margin on Total Revenues

88%

82%

Non-GAAP Combined R&D and SG&A Expenses1

448

617

38%

R&D

241

335

39%

SG&A

207

283

36%

Non-GAAP Operating Income1

339

353%

Non-GAAP Operating Margin1

13%

29%

Non-GAAP Net Income1

273

($ in millions)

Q4 2025

Q1 2026

Cash, Cash Equivalents & Marketable Securities (period end)

2,908

3,009

1 Non-GAAP R&D expenses, Non-GAAP SG&A expenses, Non-GAAP operating income / (loss), Non-GAAP Operating Margin, and Non-GAAP Net Income are non-GAAP financial measures that exclude from the corresponding GAAP measures costs related to stock-based compensation expense and realized and unrealized gains or losses on marketable equity securities. A reconciliation of these non-GAAP financial measures to the comparable GAAP measures, as well as additional information regarding our use of non-GAAP financial measures, are included in the Appendix to this presentation and in our press release dated April 30, 2026, which is accessible in the Investors section of our website at https://www.alnylam.com.

2 CER growth rates represent growth at Constant Exchange Rates, a non-GAAP financial measure determined by comparing Q1 2026 performance (restated using Q1 2025 exchange rates) to actual Q1 2025 reported performance. 24

Item

FY 2026 Guidance

Key Assumptions

Total Net Product Revenues1

$4,900 to $5,300 million

Total Rare Net Product Revenues

(GIVLAARI, OXLUMO)

$500 to $600 million

Total TTR Net Product Revenues (AMVUTTRA, ONPATTRO)

$4,400 to $4,700 million

U.S. TTR category growth consistent with recent run rate

U.S. AMVUTTRA mid-single digit net price decrease

International markets TTR $ growth similar to 2025

Net Product Revenues Growth vs. 2025

at Reported FX Rates1

64% to 77%

Uses December 31, 2025 FX rates

Net Product Revenues Growth vs. 2025

at constant exchange rates (i.e., operational growth)2

64% to 77%

Uses 2025 actual FX rates

Net Revenues from Collaborations & Royalties

$400 to $500 million

Non-GAAP Combined R&D and SG&A Expenses3

$2,700 to $2,800 million

1 Our 2026 FY Guidance is based upon December 31, 2025 FX rates including 1 EUR = 1.17 USD and 1 USD = 157 JPY

2 CER = constant exchange rate, representing growth calculated as if exchange rates had remained unchanged from those used in 2025. CER is a non-GAAP financial measure

3 2026 Non-GAAP Combined R&D and SG&A Expenses guidance is a non-GAAP financial measure that excludes from the corresponding GAAP measure stock-based compensation expense estimated at $300M - $400M. 25

Q1 2026 Financial Results

‌27

Q1 2026 Financial Results

‌Alnylam Pharmaceuticals, Inc.

Reconciliation of Selected GAAP Measures to Non-GAAP Measures

(In thousands)

Please note that the figures presented above may not sum exactly due to rounding 29

‌Alnylam Pharmaceuticals, Inc.

Reconciliation of Product Revenue and Growth at Constant Currency

Constant Currency = Constant Exchange Rate, or CER 30

Disclaimer

Alnylam Pharmaceuticals Inc. published this content on May 04, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 04, 2026 at 21:24 UTC.