ALNY Stock — Alnylam Pharmaceuticals Inc.: Cash, EV, Pipeline

What they do

We believe this is a revolutionary approach with the potential to transform the care of patients across a broad range of disease areas and indications. To date, our efforts to advance this revolutionary approach have yielded the approval of six first-in-class RNAi-based medicines: AMVUTTRA ® (vutrisiran), ONPATTRO ® (patisiran), GIVLAARI ® (givosiran), OXLUMO ® (lumasiran), Leqvio ® (inclisiran) and Qfitlia ® (fitusiran).

Source: 10-K FY2025 via SEC EDGAR

Share price

$155.94

Market cap

$20,886.2M

Cash

$3,587.3M

Enterprise value

$17,298.9M

Balance sheet

Cash	$3,587.3M
Debt	$0.0M
Net cash	$3,587.3M
Enterprise value	$17,298.9M
Basic shares	133.9M shares
Fully diluted shares	143.4M shares
Fully diluted market cap	$22,365.6M
Cash per share	$26.78
Quarterly burn	+$163.6M generated

Cash figures as of 2026-09-30. Database snapshot: 2026-11-07.

Share structure

Common

133.8M

Pre-funded warrants

—

Options

3.5M

RSUs

2.5M

Warrants

0.0M

Converts

3.2M

Conv preferred

0.0M

ESPP reserve

0.4M

FDSO

143.4M

Cap table as of 2026-10-23.

Price history

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Next catalyst

ALN-2232 · Est. Nov 7, 2026 · half midpoint · guided Now

Recently reported

ALN-HTT02 Phase 1 Positive · 2026-10-23

ALN-HTT02 · huntington's disease

Primary: Safety and tolerability, with target engagement (mutant huntingtin lowering in cerebrospinal fluid) - Phase 1b single ascending dose study focused on assessing safety and tolerability as well as target engagement (HTT-lowering) — Mean reduction from baseline of 60-64% at month 3 in the highest dose group evaluated to date, with a maximum individual reduction of 83%

· Dose-dependent reductions of CSF mHTT were observed across the first five cohorts, with target engagement demonstrated using two independent assays

· Reported data include a total of 48 enrolled participants across five single-dose cohorts

· Initial data include individuals followed for up to 12 months

· Observed durability of mHTT-lowering shows the potential for a twice-yearly dosing paradigm

· Brezosiran is designed to lower all forms of HTT, including full-length mutant HTT and the shorter mutant HTT1a

· Alnylam plans to initiate a placebo-controlled pivotal study in 2027, pending regulatory alignment

Safety: Encouraging safety and tolerability profile observed with no dose-dependent increases in CSF inflammatory markers, CSF neurofilament light chain (NfL) or brain ventricular volume

Source release →

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
GUIDEDEst. Nov 7, 2026 Now half midpoint	ALN-2232 Phase 1 in obesity and weight management, results	Phase 1	obesity and weight management	H2 2026
GUIDEDEst. Nov 7, 2026 Now half midpoint	ALN-6400 Phase 1 and Phase 2 in healthy volunteers and HHT patients, results · NCT06659640	Phase 1	healthy volunteers and HHT patients	H2 2026
Feb 9 - Mar 23 in 13 wks	ALN-4285	Phase 1	Healthy Volunteers	—
Jun 8 - Jul 20 in 7 mo	ALN-2232	Phase 2	Obesity	—
Jul 9 - Aug 20 in 8 mo	ALN-4324	Phase 2	Type 2 Diabetes Mellitus (T2DM)	—
Aug 11 - Sep 22 in 9 mo	ALN-6400	Phase 2	Von Willebrand Disease (VWD); Heavy Menstrual Bleeding (HMB)	—
Sep 11 - Oct 23 in 10 mo	ALN-4915	Phase 1	Healthy Volunteers	—
Sep 20 - Nov 1 in 11 mo	ALN-APP	Phase 2	Cerebral Amyloid Angiopathy	—
Oct 12 - Nov 23 in 11 mo	ALN-4324	Phase 2	Obese or Overweight Healthy Volunteers; Type 2 Diabetes Mellitus (T2DM)	—
Nov 11 - Dec 23 in 12 mo	ALN-BCAT	Phase 1	Advanced Hepatocellular Carcinoma; Metastatic Hepatocellular Carcinoma	—
Jan 25 - Mar 7 in 15 mo	ALN-6222	Phase 1	Obesity	—
Feb 7 - Mar 20 in 15 mo	Nucresiran	Phase 3	Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy; hATTR-PN	—
Feb 16 - Mar 29 in 16 mo	ALN-6400	Phase 2	Hereditary Hemorrhagic Telangiectasia	—
May 12 - Jun 23 in 18 mo	Vutrisiran	Phase 3	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy	—
May 13 - Jun 24 in 18 mo	ALN-AGT01 RVR	Phase 2	Hypertension	—
Jun 1 - Jul 13 in 31 mo	ALN-APP	Phase 1	Early-Onset Alzheimer Disease	—
Apr 17 - May 29 in 42 mo	ALN-5288	Phase 1	Alzheimer's Disease	—
Jun 29 - Aug 10 in 44 mo	Mivelsiran	Phase 2	Down Syndrome-Associated Alzheimer's Disease (DS-AD)	—
Jul 9 - Aug 20 in 45 mo	Nucresiran	Phase 3	Transthyretin Amyloidosis With Cardiomyopathy	—
Nov 11 - Dec 23 in 49 mo	Zilebesiran	Phase 3	High Risk Cardiovascular Disease; Hypertension; High Cardiovascular Risk	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

AMVUTTRA

Approved · Active Modality: siRNA

Aliases vutrisiran

Target TTR

Indications

ATTR amyloidosis with polyneuropathy (Approved)
ATTR amyloidosis with cardiomyopathy (Approved)

Status note Subcutaneously administered RNAi therapeutic; company pursuing regulatory approval for cardiomyopathy indication in additional jurisdictions outside the US.

ONPATTRO

Approved · Active Modality: siRNA

Aliases patisiran

Target TTR

Indications

hATTR amyloidosis with polyneuropathy (Approved)

Status note Intravenously administered RNAi therapeutic.

GIVLAARI

Approved · Active Modality: siRNA

Aliases givosiran

Target ALAS1

Indications

acute hepatic porphyria (Approved)

OXLUMO

Approved · Active Modality: siRNA

Aliases lumasiran

Target HAO1

Indications

primary hyperoxaluria type 1 (Approved)

Leqvio

Approved · Active Modality: siRNA

Aliases inclisiran

Target PCSK9

Partnerships Novartis (commercialized by Novartis; ex-US rights via MDCO license)

Indications

hypercholesterolemia (Approved)

Status note July 2025 FDA label update enabled monotherapy use with diet and exercise to reduce LDL-C in adults with hypercholesterolemia.

Qfitlia

Approved · Active Modality: siRNA

Aliases fitusiran

Target AT (antithrombin)

Partnerships Sanofi (Genzyme; commercialized by Sanofi)

Indications

hemophilia A or B (Approved)

Status note FDA approved March 2025 for routine prophylaxis in patients 12+ with hemophilia A or B with or without inhibitors; NMPA approved December 2025.

zilebesiran

Phase 3 · Active Modality: siRNA

Target angiotensinogen (AGT)

Partnerships Roche (worldwide strategic collaboration for joint development; Alnylam received $675M license obligation transaction price)

Indications

hypertension (Phase 3)

Status note Investigational siRNA targeting liver-expressed angiotensinogen, in Phase 3 for hypertension.

mivelsiran

Stage not disclosed · Active Modality: siRNA

Aliases ALN-APP

Target amyloid precursor protein (APP)

Indications

cerebral amyloid angiopathy (CAA) (Stage not disclosed)
Alzheimer's disease (Stage not disclosed)

Status note Intrathecally administered RNAi therapeutic; filing places it under early-stage clinical development programs; current phase not stated.

nucresiran

Phase 1 · Active Modality: siRNA

Target TTR

Indications

ATTR amyloidosis (Phase 1)

Catalysts

— by 2028
— by 2030

Status note Next-generation TTR silencer using IKARIA technology; positive interim Phase 1 results announced November 2024 (rapid TTR knockdown sustained six months after a single dose).

cemdisiran

Phase 3 · Active Modality: siRNA

Target C5

Partnerships Regeneron (solely responsible for development, manufacturing and commercialization; Alnylam receives milestones and tiered double-digit royalties)

Indications

paroxysmal nocturnal hemoglobinuria (PNH) (Phase 3)
myasthenia gravis (Phase 3)
geographic atrophy (Phase 3)

Status note Regeneron advancing cemdisiran in combination with its anti-C5 antibody pozelimab in Phase 3 for PNH, and as monotherapy and in combination with pozelimab in Phase 3 for myasthenia gravis and geographic atrophy.

Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	Vutrisiran
Lead indication	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Lead phase	Phase 3
Active trials	26
Collaborator trials	1

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT07223203	Phase 3	Recruiting	Sponsor	ALNY	—	Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy; hATTR-PN	—
NCT06679946	Phase 3	Enrolling by invitation	Sponsor	ALNY	—	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy	—
NCT07052903	Phase 3	Recruiting	Sponsor	ALNY	—	Transthyretin Amyloidosis With Cardiomyopathy	—
NCT07181109	Phase 3	Recruiting	Sponsor	ALNY	—	High Risk Cardiovascular Disease; Hypertension; High Cardiovascular Risk	—
NCT04153149	Phase 3	Active, not recruiting	Sponsor	ALNY	—	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy	—
NCT07463846	Phase 2	Recruiting	Sponsor	ALNY	—	Obesity	—
NCT07465224	Phase 2	Recruiting	Sponsor	ALNY	—	Type 2 Diabetes Mellitus (T2DM)	—
NCT07575308	Phase 2	Recruiting	Sponsor	ALNY	—	Von Willebrand Disease (VWD); Heavy Menstrual Bleeding (HMB)	—
NCT06393712	Phase 2	Active, not recruiting	Sponsor	ALNY	—	Cerebral Amyloid Angiopathy	—
NCT06845202	Phase 2	Active, not recruiting	Sponsor	ALNY	—	Obese or Overweight Healthy Volunteers; Type 2 Diabetes Mellitus (T2DM)	—
NCT06659640	Phase 2	Active, not recruiting	Sponsor	ALNY	—	Hereditary Hemorrhagic Telangiectasia	H2 2026 Est. Dec 31, 2026 · PCD + 6 wks (Dec 31-Dec 31)
NCT07553442	Phase 2	Recruiting	Sponsor	ALNY	—	Hypertension	—

Sponsor-matched active trials from ClinicalTrials.gov (26 as sponsor, 1 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 27 active trials for Alnylam Pharmaceuticals on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
Casdin Capital	75,000	no change	1.13%	2026-08-14
Deerfield Management	67,935	no change	0.21%	2026-08-14
OrbiMed Advisors	17,000	-102,000	0.10%	2026-08-14
Sphera Biotech	10,800	+10,800	0.47%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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ALNY — Alnylam Pharmaceuticals Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Recently reported

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

ALNY — Alnylam Pharmaceuticals Inc.