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Alnylam Pharmaceuticals Reports First Quarter 2022 Financial Results and Highlights Recent Period Activity

− Achieved First Quarter 2022 Global Net Product Revenues of $187 Million for ONPATTRO®, GIVLAARI®, and OXLUMO®

− Reported Positive 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in hATTR Amyloidosis Patients with Polyneuropathy –

− Announced 3-Month Extension of Review Period for New Drug Application for Vutrisiran due to Amendment to Address Pending Inspection Classification at Third-Party Secondary Packaging and Labeling Facility –

– Decreased 2022 Guidance Range for Combined Net Product Revenues from $900 Million – $1 Billion to $870 – $930 Million –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2022 and reviewed recent business highlights.

“The first quarter of 2022 saw a steady increase in patients on therapy across all products in our commercial portfolio, despite combined product revenues that were down compared to Q4 2021, as anticipated, given several non-recurring revenue benefits in the fourth quarter of 2021, as well as some pandemic-related impact experienced early in the quarter. We continue to make progress with our pipeline programs, including our TTR franchise, for which we reported positive 18-month results from the HELIOS-A study of vutrisiran, and we remain on track for topline results from the APOLLO-B study of patisiran in the middle of this year,” said Yvonne Greenstreet, MBChB, MBA, Chief Executive Officer of Alnylam. “With our earlier stage programs, we’re excited to have initiated our first clinical study of an Alnylam investigational RNAi therapeutic targeting a CNS disorder with the start of our Phase 1 study for ALN-APP in patients with early-onset Alzheimer’s disease, and are announcing today that the Phase 1 study of ALN-XDH in patients with gout also has been initiated. These programs underscore our commitment to advancing innovative new medicines into the clinic, and we anticipate data from both programs later this year. We look forward to continuing our steady execution across our commercial products and advancement of our clinical development pipeline, with many key upcoming milestones to mark our progress toward achieving our Alnylam P5x25 strategy.”

First Quarter 2022 and Recent Significant Corporate Highlights

Commercial Performance

ONPATTRO® (patisiran)

GIVLAARI® (givosiran)

OXLUMO® (lumasiran)

Leqvio® (inclisiran)

R&D Highlights

Vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis and Stargardt disease

Lumasiran (the non-proprietary name for OXLUMO), for the treatment of primary hyperoxaluria type 1 (PH1), and in development for the treatment of recurrent kidney stone disease

Early- and mid-stage investigational RNAi therapeutic pipeline programs and RNAi platform

Additional Business Updates

Upcoming Events

In early and mid-2022, Alnylam intends to:

Financial Results for the Quarter Ended March 31, 2022

Financial Highlights 

(in thousands, except per share amounts)

 

Three Months Ended
March 31,

 

 

2022

 

 

 

2021

 

 

 

 

 

ONPATTRO net product revenues

$

137,009

 

 

$

101,951

 

GIVLAARI net product revenues

 

35,277

 

 

 

24,673

 

OXLUMO net product revenues

 

14,586

 

 

 

9,145

 

Total net product revenues

$

186,872

 

 

$

135,769

 

 

 

 

 

Net revenue from collaborations

$

25,945

 

 

$

41,797

 

 

 

 

 

Royalty revenue

$

442

 

 

$

 

 

 

 

 

GAAP operating loss

$

(146,732

)

 

$

(186,254

)

Non-GAAP operating loss

$

(117,439

)

 

$

(130,564

)

 

 

 

 

GAAP other expense

$

(92,624

)

 

$

(13,021

)

Non-GAAP other expense

$

(61,463

)

 

$

(60,037

)

 

 

 

 

GAAP net loss

$

(240,341

)

 

$

(200,291

)

Non-GAAP net loss

$

(179,887

)

 

$

(191,617

)

 

 

 

 

GAAP net loss per common share – basic and diluted

$

(2.00

)

 

$

(1.71

)

Non-GAAP net loss per common share – basic and diluted

$

(1.49

)

 

$

(1.64

)

Net Product Revenues

Net Revenues from Collaborations

First Quarter 2022 Expenses

 

Three Months Ended March 31,

 

2022

 

2021

 

 

 

 

GAAP research and development expenses

$

169,893

 

$

185,899

Non-GAAP research and development expenses

$

158,276

 

$

161,524

 

 

 

 

GAAP selling, general and administrative expenses

$

154,471

 

$

146,859

Non-GAAP selling, general and administrative expenses

$

136,795

 

$

115,544

Research & Development (R&D) Expenses

Selling, General & Administrative (SG&A) Expenses

Other Financial Highlights

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

2022 Updated Financial Guidance 

Full year 2022 financial guidance has been updated as follows:

 

Provided 2/10/2022

($ millions)1

Updated 4/28/2022

($ millions)2

Combined net product revenues for ONPATTRO, GIVLAARI, OXLUMO and vutrisiran3

$900 – $1,000

$870 – $930

Net revenues from collaborations and royalties

$175 – $225

Unchanged

GAAP R&D and SG&A expenses

$1,630 – $1,750

$1,620 – $1,700

Non-GAAP R&D and SG&A expenses4

$1,400 – $1,500

$1,390 – $1,450

1 Prior FY 2022 Guidance utilized January 31, 2022 FX rates of: 1 EUR = 1.12 USD; 1 GBP = 1.34 USD; 1 CHF = 1.08 USD; 1 CAD = 0.79 USD, 1 USD = 115 JPY

2 Updated FY 2022 Guidance utilizes April 18, 2022 FX rates of: 1 EUR = 1.08 USD; 1 GBP = 1.31 USD; 1 CHF = 1.06 USD; 1 CAD = 0.79 USD, 1 USD = 126 JPY

3 Assuming FDA approval of vutrisiran by the July 14, 2022 PDUFA goal date

4 Primarily excludes $230-$250 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses.

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 (gains) 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 (gains) 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.

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 first quarter 2022 results as well as expectations for the future via conference call on Thursday, April 28, 2022 at 8:30 am ET. To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 2758447. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or +1-404-537-3406 (international) and refer to conference ID 2758447.

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 was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. 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) and should be administered via a healthcare professional. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, including please see the full U.S. Prescribing Information, visit ONPATTRO.com.

About GIVLAARI® (givosiran)

GIVLAARI 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, givosiran 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 is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. 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. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter 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 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 (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 20 years ago, 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), GIVLAARI® (givosiran), OXLUMO® (lumasiran) and Leqvio® (inclisiran) being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including five 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 Twitter at @Alnylam, on LinkedIn, or on Instagram.

Alnylam Forward Looking Statements

Various statements in this release concerning Alnylam’s expectations, plans, aspirations and goals, including, without limitation, our aspiration to become a leading biotech company and the planned achievement of our “Alnylam P5x25” strategy, the potential launch of vutrisiran for the treatment of hATTR amyloidosis patients with polyneuropathy, if approved by the FDA, and the ongoing review of vutrisiran by other regulatory authorities, FDA review of a supplemental NDA for OXLUMO and CHMP review of a Type II variation for lumasiran to amend the label, the achievement of additional pipeline milestones and data, including relating to ongoing clinical studies of patisiran, zilebesiran, cemdisiran, ALN-HBV02 (Vir 2218), and ALN-HSD, the initiation of clinical studies for ALN-APP and ALN-XDH, the expected impact of the conflict in Ukraine and the ongoing pandemic on enrollment in the zilebesiran KARDIA-1 Phase 2 study and timing for topline data, the expected range of net product revenues, as updated, and net revenues from collaborations and royalties for 2022, and the expected range of aggregate annual GAAP and non-GAAP R&D and SG&A expenses for 2022, as updated, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward

Contacts

Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom

(Investors and Media)

617-682-4340

Josh Brodsky

(Investors)

617-551-8276

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