ALNY Alnylam Pharmaceuticals Inc

− Achieved Third Quarter 2024 Global Net Product Revenues of $420 Million, Representing 34% Year-Over-Year Growth Compared to Q3 2023, Driven by Continued Momentum from TTR Business –

− Submitted Regulatory Applications in U.S. and EU for AMVUTTRA® (vutrisiran) for the Treatment of Transthyretin Amyloidosis with Cardiomyopathy –

− Reiterated 2024 Financial Guidance, Including Combined Net Product Revenues of $1,575 Million to $1,650 Million –

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2024 and reviewed recent business highlights.

“Alnylam continued to deliver robust results commercially and clinically in the third quarter. We achieved 34% year-over-year growth, with global net product revenues of $420 million, primarily driven by our TTR business. Further with TTR, we continued to share data for vutrisiran in ATTR-CM, with detailed HELIOS-B results presented at the European Society of Cardiology Congress, and supportive echocardiographic and biomarker data presented at the Heart Failure Society of America Annual Scientific Meeting 2024. We’re pleased to have submitted regulatory filings in the U.S. and EU, keeping us on track for a potential U.S. launch in early 2025 and other markets later in the year,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. “We also made great strides with our pipeline, sharing new results today from the Phase 1 study of mivelsiran in patients with Alzheimer’s disease and initiating a Phase 1 study for ALN-HTT02 in adult patients with Huntington’s disease earlier in the third quarter. With this execution, we have continued to progress toward achieving our Alnylam P5x25 goals and becoming a leading global biotech company.”

Third Quarter 2024 and Recent Significant Business Highlights

Commercial Performance

Total TTR: ONPATTRO® (patisiran) & AMVUTTRA® (vutrisiran)

• Continued growth momentum in total TTR, achieving global net product revenues for ONPATTRO and AMVUTTRA for the third quarter of $50 million and $259 million, respectively, which combined represent 34% TTR annual growth compared to Q3 2023.

Total Rare: GIVLAARI® (givosiran) & OXLUMO® (lumasiran)

• Achieved global net product revenues for GIVLAARI and OXLUMO for the third quarter of $71 million and $40 million, respectively, which combined represent 34% Rare annual growth compared to Q3 2023.

R&D Highlights

• Presented detailed results from the positive HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) at the European Society of Cardiology Congress (ESC).
  • Additional data from HELIOS-B, including echocardiographic and biomarker data, were presented at the Heart Failure Society of America Annual Scientific Meeting 2024 (HFSA).
  • Based on these positive results, Alnylam submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) using a Priority Review Voucher, as well as a Type II Variation to the European Medicines Agency (EMA), for vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy.
• Today announced positive initial results from the multiple dose portion of the Phase 1 study of mivelsiran in patients with Alzheimer’s disease.
  • Following a second 50 mg dose of mivelsiran at month 6, further reductions of sAPPβ were observed at month 7, with patients achieving greater than 90% lowering of sAPPβ.
  • No new safety signals have been identified, and no significant abnormalities have been seen on CSF safety labs such as total protein and CSF white cell count, or on the exploratory biomarker neurofilament light chain.
• Presented the design of the cAPPricorn-1 Phase 2 study of mivelsiran in patients with cerebral amyloid angiopathy (CAA) at the 2024 International CAA Conference.
• Initiated a Phase 1 study of ALN-HTT02 in adult patients with Huntington’s disease.
  • Exon1 targeting strategy and design of the Phase 1 study were presented at the European Huntington's Disease Network (EHDN) & Enroll-HD 2024 Congress.
  • Nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates after single and repeated intrathecal administration of ALN-HTT02 were also shared.
• Today announced the decision to suspend further clinical development of ALN-KHK, an investigational RNAi therapeutic targeting ketohexokinase for the treatment of type 2 diabetes mellitus. This decision reflects portfolio prioritization efforts to allocate clinical, managerial and financial resources carefully to bring transformative medicines to patients.

Additional Business Updates

• Alnylam hosted a TTR Investor Day featuring presentations from Alnylam senior leaders and an external expert related to the Company's TTR business. A replay of the webcast is available on the Company's website.
• Appointed Robert Hesslein as Chief Legal Officer and Corporate Secretary.
• Named one of Fast Company Top 100 Workplaces for Innovators in 2024.
• Recognized by Science magazine as a Top Employer for the sixth consecutive year.

Upcoming Events

Alnylam will present additional findings from the ongoing Phase 1 study of ALN-TTRsc04 in patients with ATTR amyloidosis at the American Heart Association Scientific Sessions 2024. The Company plans to share its Phase 3 development plan for ALN-TTRsc04 in the first quarter of 2025.

Alnylam intends to initiate a Phase 2 study of mivelsiran in patients with Alzheimer’s disease at or around year-end.

Financial Results for the Quarter Ended September 30, 2024

 

Three Months Ended September 30,

(In thousands, except per share amounts)

 

2024

 

 

2023

Net product revenues

$

420,146

 

 

$

313,153

Net revenue from collaborations

$

57,387

 

 

$

427,472

Royalty revenue

$

23,386

 

 

$

9,905

 

 

 

 

GAAP Operating (loss) income

$

(76,905

)

 

$

213,867

Non-GAAP Operating (loss) income

$

(31,101

)

 

$

277,804

 

 

 

 

GAAP Net (loss) income

$

(111,570

)

 

$

147,753

Non-GAAP Net (loss) income

$

(64,199

)

 

$

228,534

 

 

 

 

GAAP Net (loss) income per common share - basic

$

(0.87

)

 

$

1.18

Non-GAAP Net (loss) income per common share - basic

$

(0.50

)

 

$

1.83

 

 

 

 

GAAP Net (loss) income per common share - diluted

$

(0.87

)

 

$

1.15

Non-GAAP Net (loss) income per common share - diluted

$

(0.50

)

 

$

1.74

 

 

 

 

For an explanation of our use of non-GAAP financial measures refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release.

Net Product Revenues

 

Three Months Ended September 30,

 

Year over Year % Growth

(In thousands, except percentages)

2024

 

2023

 

As Reported

 

At CER*

ONPATTRO net product revenues

$

50,293

 

$

81,589

 

(38)%

 

(38)%

AMVUTTRA net product revenues

 

258,590

 

 

148,680

 

74%

 

75%

Total TTR net product revenues

 

308,883

 

 

230,269

 

34%

 

35%

 

 

 

 

 

 

 

 

GIVLAARI net product revenues

 

71,043

 

 

54,148

 

31%

 

31%

OXLUMO net product revenues

 

40,220

 

 

28,736

 

40%

 

40%

Total Rare net product revenues

 

111,263

 

 

82,884

 

34%

 

34%

 

 

 

 

 

 

 

 

Total net product revenues

$

420,146

 

$

313,153

 

34%

 

35%

 

 

 

 

 

 

 

 

* CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in the third quarter 2023. CER is a non-GAAP measure.

• Total net product revenues increased 34% and 35% at actual currency and CER, respectively, during the three months ended September 30, 2024, as compared to the same period in 2023, due to an increased number of patients receiving AMVUTTRA, GIVLAARI, and OXLUMO, partially offset by a decrease in patients receiving ONPATTRO primarily driven by switching to AMVUTTRA.

Net Revenues from Collaborations

• Net revenues from collaborations decreased during the three months ended September 30, 2024, as compared to the same period in 2023, due to less revenue recognized under our collaborations with Roche and Regeneron. During 2023, we recognized $310 million of revenue from the upfront payment we received from Roche and a $65 million cumulative adjustment from the $100 million milestone we received in connection with our Regeneron Collaboration for achieving certain criteria during early clinical development for mivelsiran (formerly ALN-APP).

Operating Expenses

 

Three Months Ended September 30,

(In thousands, except percentages)

 

2024

 

 

 

2023

 

Cost of goods sold

$

81,980

 

 

$

79,473

 

Cost of goods sold as a percentage of net product revenues

 

19.5

%

 

 

25.4

%

 

 

 

 

Cost of collaborations and royalties

$

3,925

 

 

$

4,836

 

 

 

 

 

GAAP research and development expenses

$

270,926

 

 

$

253,179

 

Non-GAAP research and development expenses

$

251,132

 

 

$

224,024

 

 

 

 

 

GAAP selling, general and administrative expenses

$

220,993

 

 

$

199,175

 

Non-GAAP selling, general and administrative expenses

$

194,983

 

 

$

164,393

 

 

Cost of Goods Sold

• Cost of goods sold as a percentage of net product revenues decreased during the three months ended September 30, 2024, as compared to the same period in 2023, primarily due to higher costs in 2023 associated with cancelled manufacturing commitments and the impairment of ONPATTRO inventory that had been manufactured for future demand associated with the use of patisiran for the treatment of patients with ATTR amyloidosis with cardiomyopathy for which we did not receive regulatory approval in the U.S., offset by increased rate of royalties payable on net sales of AMVUTTRA.

Research & Development (R&D) Expenses

• GAAP and non-GAAP R&D expenses increased during the three months ended September 30, 2024, as compared to the same period in 2023, primarily due to increased costs associated with our preclinical activities as we develop our clinical pipeline of RNAi therapeutics targeting multiple tissues, increased clinical trial expenses associated with increased Phase 2 activities for the zilebesiran KARDIA-3 clinical trial, and increased employee compensation expenses.

Selling, General & Administrative (SG&A) Expenses

• GAAP and non-GAAP SG&A expenses increased during the three months ended September 30, 2024, as compared to the same period in 2023, primarily due to higher costs associated with marketing investments to promote our TTR therapies and prepare for the potential launch of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy, and employee compensation.

Other Financial Highlights

• Cash, cash equivalents and marketable securities were $2.78 billion as of September 30, 2024, as compared to $2.44 billion as of December 31, 2023, with the increase primarily due to improved operating performance and increased net proceeds from the issuance of common stock in connection with stock option exercises.

A reconciliation of our GAAP to non-GAAP financial results for the quarter is included in the tables at the end of this press release.

2024 Financial Guidance

Full year 2024 financial guidance is reiterated and consists of the following:

Combined net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO1

 

$1,575 million - $1,650 million

Net Product Revenue Growth vs. 2023 at reported FX rates1

 

27% to 33%

Net Product Revenue Growth vs. 2023 at CER*

 

28% to 34%

Net revenues from collaborations and royalties

 

$575 million - $650 million

GAAP R&D and SG&A expenses

 

$2,000 million - $2,150 million

Non-GAAP R&D and SG&A expenses2

 

$1,775 million - $1,875 million

 

 

 

1 Uses June 30, 2024 FX rates including: 1 EUR = 1.07 USD and 1 USD = 161 JPY

2 Primarily excludes $225 - $275 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses

*CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in the twelve months ended December 31, 2023. CER is a non-GAAP measure.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. 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.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses and realized and unrealized losses on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the realized and unrealized losses on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet.

Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

Conference Call Information

Management will provide an update on the Company and discuss third quarter 2024 results as well as expectations for the future via conference call on Thursday, October 31, 2024 at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About ONPATTRO® (patisiran)

ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com.

About AMVUTTRA® (vutrisiran)

AMVUTTRA (vutrisiran) is an RNAi therapeutic approved in the United States for the treatment of adults with hATTR amyloidosis with polyneuropathy. It is a double-stranded small interfering RNA (siRNA) that targets mutant and wild-type transthyretin (TTR) messenger RNA (mRNA). Using Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, AMVUTTRA is designed for increased potency and high metabolic stability to allow for subcutaneous injection once every three months (quarterly). Results from the pivotal HELIOS-A Phase 3 study demonstrate AMVUTTRA rapidly reduces serum TTR levels, has the potential to reverse neuropathy impairment relative to baseline and improves other key measures of disease burden relative to external placebo in patients with the polyneuropathy of hATTR amyloidosis. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com.

About GIVLAARI® (givosiran)

GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal study, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, including the full U.S. Prescribing Information, visit GIVLAARI.com.

About OXLUMO® (lumasiran)

OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C study, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions (ISRs) were the most common drug-related adverse reaction. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, including the full U.S. Prescribing Information, visit OXLUMO.com.

About LNP Technology

Alnylam has licenses to Arbutus Biopharma lipid nanoparticle (LNP) intellectual property for use in RNAi therapeutic products using LNP technology.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.

Alnylam Forward Looking Statements

This press release 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 expectations regarding the potential launch of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy in the U.S. in early 2025 and in other countries later in 2025; Alnylam’s advancement towards its “Alnylam P5x25” strategy and aspiration to become a leading global biotech company; the potential for Alnylam to advance its research and development programs, including the timing of Alnylam’s presentation of data and sharing of the development plan for ALN-TTRsc04 and initiation of a Phase 2 clinical trial for mivelsiran in patients with Alzheimer’s Disease; and Alnylam’s projected commercial and financial performance, including the expected range of net product revenues and net revenues from collaborations and royalties for 2024, and the expected range of aggregate annual GAAP and non-GAAP R&D and SG&A expenses for 2024, 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 P5x25” strategy; 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, including vutrisiran, zilebesiran, ALN-TTRsc04 and mivelsiran; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to successfully expand the approved indications for AMVUTTRA in the future; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, Regeneron and Vir; the outcome of litigation; the risk of future government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. 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 release discusses investigational RNAi therapeutics and uses of previously approved RNAi therapeutics in development and is not intended to convey conclusions about efficacy or safety as to those investigational therapeutics or uses. Vutrisiran has not been approved by any regulatory agency for the treatment of ATTR amyloidosis with cardiomyopathy. No conclusions can or should be drawn regarding its safety or effectiveness in treating cardiomyopathy in this population. There is no guarantee that any investigational therapeutics or expanded uses of commercial products will successfully complete clinical development or gain health authority approval.

 

ALNYLAM PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except per share amounts)

   

 

September 30, 2024

 

December 31, 2023

ASSETS

(Unaudited)

 

 

Current assets:

 

 

 

Cash and cash equivalents

$

1,099,920

 

 

$

812,688

 

Marketable debt securities

 

1,671,993

 

 

 

1,615,516

 

Marketable equity securities

 

8,322

 

 

 

11,178

 

Accounts receivable, net

 

353,852

 

 

 

327,787

 

Inventory

 

75,987

 

 

 

89,146

 

Prepaid expenses and other current assets

 

145,350

 

 

 

126,382

 

Total current assets

 

3,355,424

 

 

 

2,982,697

 

Property, plant and equipment, net

 

506,997

 

 

 

526,057

 

Operating lease right-of-use assets

 

196,408

 

 

 

199,732

 

Restricted investments

 

68,592

 

 

 

49,391

 

Other assets

 

77,618

 

 

 

72,003

 

Total assets

$

4,205,039

 

 

$

3,829,880

 

LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT)

 

 

 

Current liabilities:

 

 

 

Accounts payable

$

70,805

 

 

$

55,519

 

Accrued expenses

 

946,168

 

 

 

713,013

 

Operating lease liability

 

42,144

 

 

 

41,510

 

Deferred revenue

 

71,030

 

 

 

102,753

 

Liability related to the sale of future royalties

 

90,516

 

 

 

54,991

 

Total current liabilities

 

1,220,663

 

 

 

967,786

 

Operating lease liability, net of current portion

 

236,030

 

 

 

243,101

 

Deferred revenue, net of current portion

 

1,012

 

 

 

188,175

 

Convertible debt

 

1,023,654

 

 

 

1,020,776

 

Liability related to the sale of future royalties, net of current portion

 

1,333,849

 

 

 

1,322,248

 

Other liabilities

 

357,477

 

 

 

308,438

 

Total liabilities

 

4,172,685

 

 

 

4,050,524

 

Commitments and contingencies (Note 13)

 

 

 

Stockholders’ equity (deficit):

 

 

 

Preferred stock, $0.01 par value per share, 5,000 shares authorized and no shares issued and outstanding as of September 30, 2024 and December 31, 2023

 

—

 

 

 

—

 

Common stock, $0.01 par value per share, 250,000 shares authorized; 128,841 shares issued and outstanding as of September 30, 2024; 125,794 shares issued and outstanding as of December 31, 2023

 

1,289

 

 

 

1,259

 

Additional paid-in capital

 

7,259,876

 

 

 

6,811,063

 

Accumulated other comprehensive loss

 

(24,826

)

 

 

(23,375

)

Accumulated deficit

 

(7,203,985

)

 

 

(7,009,591

)

Total stockholders’ equity (deficit)

 

32,354

 

 

 

(220,644

)

Total liabilities and stockholders’ equity (deficit)

$

4,205,039

 

 

$

3,829,880

 

 

This selected financial information should be read in conjunction with the consolidated financial statements and notes thereto included in Alnylam’s Annual Report on Form 10-K which includes the audited financial statements for the year ended December 31, 2023.

 

ALNYLAM PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except per share amounts) (Unaudited)

   

 

Three Months Ended

 

Nine Months Ended

 

September 30, 2024

 

September 30, 2023

 

September 30, 2024

 

September 30, 2023

Statements of Operations

 

 

 

 

 

 

 

Revenues:

 

 

 

 

 

 

 

Net product revenues

$

420,146

 

 

$

313,153

 

 

$

1,195,397

 

 

$

895,186

 

Net revenues from collaborations

 

57,387

 

 

 

427,472

 

 

 

403,273

 

 

 

469,778

 

Royalty revenue

 

23,386

 

 

 

9,905

 

 

 

56,407

 

 

 

23,610

 

Total revenues

 

500,919

 

 

 

750,530

 

 

 

1,655,077

 

 

 

1,388,574

 

 

 

 

 

 

 

 

 

Operating costs and expenses:

 

 

 

 

 

 

 

Cost of goods sold

 

81,980

 

 

 

79,473

 

 

 

203,864

 

 

 

196,241

 

Cost of collaborations and royalties

 

3,925

 

 

 

4,836

 

 

 

16,689

 

 

 

28,307

 

Research and development

 

270,926

 

 

 

253,179

 

 

 

826,063

 

 

 

732,274

 

Selling, general and administrative

 

220,993

 

 

 

199,175

 

 

 

680,187

 

 

 

597,523

 

Total operating costs and expenses

 

577,824

 

 

 

536,663

 

 

 

1,726,803

 

 

 

1,554,345

 

(Loss) income from operations

 

(76,905

)

 

 

213,867

 

 

 

(71,726

)

 

 

(165,771

)

Other (expense) income:

 

 

 

 

 

 

 

Interest expense

 

(34,376

)

 

 

(30,893

)

 

 

(102,887

)

 

 

(89,883

)

Interest income

 

32,146

 

 

 

25,425

 

 

 

90,973

 

 

 

65,155

 

Other expense, net

 

(29,528

)

 

 

(57,658

)

 

 

(99,777

)

 

 

(105,331

)

Total other expense, net

 

(31,758

)

 

 

(63,126

)

 

 

(111,691

)

 

 

(130,059

)

(Loss) income before income taxes

 

(108,663

)

 

 

150,741

 

 

 

(183,417

)

 

 

(295,830

)

Provision for income taxes

 

(2,907

)

 

 

(2,988

)

 

 

(10,977

)

 

 

(6,542

)

Net (loss) income

$

(111,570

)

 

$

147,753

 

 

$

(194,394

)

 

$

(302,372

)

 

 

 

 

 

 

 

 

Net (loss) income per common share - basic

$

(0.87

)

 

$

1.18

 

 

$

(1.53

)

 

$

(2.43

)

Net (loss) income per common share - diluted

$

(0.87

)

 

$

1.15

 

 

$

(1.53

)

 

$

(2.43

)

 

 

 

 

 

 

 

 

Weighted-average common shares - basic

 

128,590

 

 

 

125,220

 

 

 

127,159

 

 

 

124,667

 

Weighted-average common shares - diluted

 

128,590

 

 

 

131,337

 

 

 

127,159

 

 

 

124,667

 

 

ALNYLAM PHARMACEUTICALS, INC. RECONCILIATION OF SELECTED GAAP MEASURES TO NON-GAAP MEASURES (In thousands, except per share amounts) (Unaudited)

   

 

Three Months Ended

 

September 30, 2024

 

September 30, 2023

Reconciliation of GAAP to Non-GAAP Research and development:

 

 

 

GAAP Research and development

$

270,926

 

 

$

253,179

 

Less: Stock-based compensation expenses

 

(19,794

)

 

 

(29,155

)

Non-GAAP Research and development

$

251,132

 

 

$

224,024

 

 

 

 

 

Reconciliation of GAAP to Non-GAAP Selling, general and administrative:

 

 

 

GAAP Selling, general and administrative

$

220,993

 

 

$

199,175

 

Less: Stock-based compensation expenses

 

(26,010

)

 

 

(34,782

)

Non-GAAP Selling, general and administrative

$

194,983

 

 

$

164,393

 

 

 

 

 

Reconciliation of GAAP to Non-GAAP Operating (loss) income:

GAAP Operating (loss) income

$

(76,905

)

 

$

213,867

 

Add: Stock-based compensation expenses

 

45,804

 

 

 

63,937

 

Non-GAAP Operating (loss) income

$

(31,101

)

 

$

277,804

 

 

 

 

 

Reconciliation of GAAP to Non-GAAP Net (loss) income:

 

 

 

GAAP Net (loss) income

$

(111,570

)

 

$

147,753

 

Add: Stock-based compensation expenses

 

45,804

 

 

 

63,937

 

Add: Realized and unrealized loss on marketable equity securities

 

1,567

 

 

 

16,844

 

Non-GAAP Net (loss) income

$

(64,199

)

 

$

228,534

 

 

 

 

 

Reconciliation of GAAP to Non-GAAP Net (loss) income per common share- basic:

GAAP Net (loss) income per common share - basic

$

(0.87

)

 

$

1.18

 

Add: Stock-based compensation expenses

 

0.36

 

 

 

0.51

 

Add: Realized and unrealized loss on marketable equity securities

 

0.01

 

 

 

0.13

 

Non-GAAP Net (loss) income per common share - basic

$

(0.50

)

 

$

1.83

 

 

 

 

 

Reconciliation of GAAP to Non-GAAP Net (loss) income per common share- diluted:

GAAP Net (loss) income per common share - diluted

$

(0.87

)

 

$

1.15

 

Add: Stock-based compensation expenses

 

0.36

 

 

 

0.49

 

Add: Realized and unrealized loss on marketable equity securities

 

0.01

 

 

 

0.13

 

Non-GAAP Net (loss) income per common share - diluted

$

(0.50

)

 

$

1.74

 

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

 

ALNYLAM PHARMACEUTICALS, INC. RECONCILIATION OF GAAP TO NON-GAAP PRODUCT REVENUE GROWTH AT CONSTANT CURRENCY (Unaudited)

   

 

September 30, 2024

 

Three Months Ended

ONPATTRO net product revenue growth, as reported

(38

)%

Add: Impact of foreign currency translation

—

 

ONPATTRO net product revenue growth at constant currency

(38

)%

 

 

AMVUTTRA net product revenue growth, as reported

74

%

Add: Impact of foreign currency translation

1

 

AMVUTTRA net product revenue growth at constant currency

75

%

 

 

Total TTR net product revenue growth, as reported

34

%

Add: Impact of foreign currency translation

1

 

Total TTR net product revenue growth at constant currency

35

%

 

 

GIVLAARI net product revenue growth, as reported

31

%

Add: Impact of foreign currency translation

—

 

GIVLAARI net product revenue growth at constant currency

31

%

 

 

OXLUMO net product revenue growth, as reported

40

%

Add: Impact of foreign currency translation

—

 

OXLUMO net product revenue growth at constant currency

40

%

 

 

Total net product revenue growth, as reported

34

%

Add: Impact of foreign currency translation

1

 

Total net product revenue growth at constant currency

35

%

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20241031367620/en/

Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom (Investors and Media) 617-682-4340

Josh Brodsky (Investors) 617-551-8276