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MarketScreener Homepage  >  Equities  >  Nasdaq  >  Alnylam Pharmaceuticals, Inc.    ALNY

ALNYLAM PHARMACEUTICALS, INC.

(ALNY)
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Alnylam Pharmaceuticals : Conference Call to Discuss FDA Approval of OXLUMO™ (lumasiran) Presentation ›

11/24/2020 | 08:25am EST

Conference Call to Discuss FDA Approval of OXLUMO™ (lumasiran)

November 24, 2020

© 2020 Alnylam Pharmaceuticals, Inc.

Agenda

Welcome

  • Christine Lindenboom
    Senior Vice President, Investor Relations & Corporate Communications

Introduction

  • John Maraganore, Ph.D. Chief Executive Officer

OXLUMO™ (lumasiran) Label & Data

  • Akshay Vaishnaw, M.D., Ph.D. President of R&D

Commercialization Strategy

  • Andy Orth
    Senior Vice President, Head of U.S. Business

Q&A Session

2

Alnylam Forward Looking Statements

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. There are a number of important factors that could cause actual results to differ materially from the results anticipated by these forward-looking statements. These important factors include: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by our products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to our business, the effectiveness or timeliness of steps taken by us to mitigate the impact of the pandemic, and our ability to execute business continuity plans to address disruptions caused by the COVID- 19 or any future pandemic; our ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of our product candidates, pre-clinical and clinical results for our product candidates; actions or advice of regulatory agencies; delays, interruptions or failures in the manufacture and supply of our product candidates and our marketed products, including OXLUMOTM (lumasiran); intellectual property matters including potential patent litigation relating to our platform, products or product candidates; our and our partner's ability to obtain regulatory approval for our product candidates, including inclisiran, and our ability to maintain regulatory approval and obtain pricing and reimbursement for products, including ONPATTRO® (patisiran), GIVLAARI® (givosiran) and OXLUMO; our ability to successfully launch, market and sell our approved products globally, including ONPATTRO, GIVLAARI and OXLUMO, and achieve net product revenues for ONPATTRO within our further revised expected range during 2020; our ability to successfully expand the indication for ONPATTRO in the future; competition from others using similar technology and developing products for similar uses; our ability to manage our growth and operating expenses within the reduced ranges of guidance provided by us through implementation of further discipline in operations to moderate spend and our ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; our ability to establish and maintain business alliances; our dependence on third parties, including Novartis, for the development, manufacture and commercialization of inclisiran, Regeneron, for development, manufacture and commercialization of certain products, including eye and CNS products such as ALN-APP and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; and the risk of government investigations; as well as those risks and other factors more fully discussed in our most recent annual, quarterly and current reports filed with the SEC. If one or more of these factors materialize, or if any underlying assumptions prove incorrect, our actual results, performance or achievements may vary materially from any future results, performance or achievements expressed or implied by these forward-looking statements. All forward-looking statements speak only as of the date of this presentation and, except as required by law, we undertake no obligation to update such statements.

3

John Maraganore, Ph.D. Chief Executive Officer

Introduction

4

The third RNAi therapeutic is

NOW APPROVED IN THE U.S. & EU

5

Additional Launches Planned Over Next 18 Months

2018

ONPATTRO is indicated in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults1

2019

GIVLAARI is indicated in the U.S. for the treatment of adults with acute hepatic porphyria2

2020

OXLUMO is indicated in the

U.S. for the treatment of

primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients3

2020

LEQVIO®*

Hypercholesterolemia

Ph3

NDA/MAA accepted

Received positive

CHMP opinion

2021-2022

Vutrisiran

Fitusiran*

ATTR amyloidosis

Hemophilia

HELIOS-A Phase 3 topline

Two of three Phase 3

results in early 2021

studies fully enrolled

6

Robust pipelinefuels sustainableproduct launches beyond 2021,leveraging global commercialinfrastructure

* Novartis is leading and funding development of inclisiran and will commercialize inclisiran, assuming regulatory approvals; Sanofi Genzyme is leading and funding development of fitusiran and will commercialize fitusiran, if successful

1 ONPATTRO is approved in the U.S. and Canada for the PN of hATTR amyloidosis in adults, and in the EU, Japan and other countries for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. For additional information on ONPATTRO, see Full Prescribing Information

2 GIVLAARI is approved in the EU for the treatment of acute hepatic porphyria (AHP) in adults and adolescents over 12 years old, and in Brazil for the treatment of AHP in adults; Alnylam has filed or plans to file for marketing authorization for givosiran in Japan and other countries in 2020; For additional information on GIVLAARI, see Full Prescribing Information

3 For additional information on OXLUMO, see Full Prescribing Information

Anticipated dates of launch based on current development timelines for investigational therapeutics and assuming positive pivotal study data and regulatory approval

Akshay Vaishnaw, M.D., Ph.D.

President of R&D

OXLUMO™ (lumasiran) Label & Data

7

Primary Hyperoxaluria Type 1

Lumasiran

Description

Rare autosomal recessive disorder of increased oxalate synthesis resulting in kidney stones and renal failure, with subsequent oxalate accumulation in extra-renal tissues

Onset generally

pediatric

very limited treatment options

Patient Population

~3,000

potentially symptomatic in U.S./EU1

Benson

Living with Primary Hyperoxaluria Type 1

Retinal Oxalosis

Cardiomyopathy

Nephrocalcinosis Renal stones

ESRD

Skeletal

Involvement

1 Includes patients that are presymptomatic, subclinical, or symptomatic

Lumasiran

Phase 3 Study

Met Primary and All Tested Secondary Endpoints with Encouraging Safety and Tolerability Profile

Rapid and sustained reduction in 24hr urinary

oxalate levels from baseline to Month 6

Urinary

± SEM)

20

0

from Baseline in 24hr

Corrected for BSA (%;

-20

-40

-60

-80

LS Mean Average (M3 - M6)

-11.8%

Placebo (N=13)

Change

Oxalate

-100

-65.4%

Lumasiran (N=26)

BL

M1

M2

M3

M4

M5

M6

Dosing

Patients (N)

13

13

12

13

13

13

13

26

24

26

24

23

25

25

-53%

-76%

Majority of patients achieved near-normalization (≤1.5 x ULN) or

normalization (≤ULN) in 24hr urinary oxalate levels at Month 6

100

84%

Lumasiran

75

Placebo

p*=8.3 x 10-7

52%

(%)

50

Patients

p*=0.0010

25

0%

0%

0

Near-Normalization

Normalization

Safety

  • No deaths, severe, or serious AEs. All AEs mild or moderate
  • Most common related AEs were injection-site reactions (N=10; 38%)
    • All generally mild; no discontinuation of treatment
    • Most common symptoms: erythema, pain, pruritus, and swelling

Difference in LS mean average M3-M6

Mean maximal reduction

No hepatic AEs reported in either group

No clinically relevant changes in laboratory measures, vital signs, or

(Lumasiran-Placebo) (p=1.7 x 10-14)

electrocardiograms related to lumasiran observed

9

BL, baseline; BSA, body surface area; hr, hour; LS, least squares; M, month; ULN, upper limit of normal; AE, adverse event

* p-value is based on Cochran-Mantel-Haenszel test stratified by baseline 24 hr urinary oxalate corrected for BSA (≤1.70 vs >1.70 mmol/24 hr/1.73 m2)

Lumasiran

Phase 3 Study

Efficacy Results and Safety Profile in Pediatric Patients Similar to Those Observed in ILLUMINATE-A

Rapid and sustained reduction in spot urinary

Primary Endpoint Result

oxalate:creatinine ratio across all weight groups

ChangePercentfrom Baseline in

Oxalate:CreatinineUrinarySpot

0

-72%

(mmom/mmol)Ratio

-90

<10 kg (N=3)

lumasiran1

-10

-20

10 to <20 kg (N=12)

LS mean reduction in spot urinary

-30

oxalate:creatinine ration from BL to M6

≥20 kg (N=3)

(average change of M3-M6)

-40

All lumasiran treated (N=18)

-50

Safety

-60

No deaths, discontinuations or withdrawals, or severe

-70

AEs

-80

One serious AE occurred, considered not related to

BL

M1

M2

M3

M4

M5

M6

Most common related AE was injection-site reactions

Visit

- All mild and transient in severity

No clinically relevant changes in laboratory measures,

N=

3

3

3

3

3

3

3

vital signs, or electrocardiograms related to lumasiran

N=

12

12

12

12

12

12

12

observed

N=

3

3

3

3

3

3

3

No hepatic events reported

N=

18

18

18

18

18

18

18

Data in graph are presented as mean ± SEM of observed values

10 1Viral infection, considered not related to the study drug by the Investigator

BL, baseline; M, month; SEM, standard error of the mean; ULN, upper limit of normal; AE, adverse event; SAE, serious adverse event

OXLUMO™ (lumasiran) Label for U.S.

Indication

Dosing & Administration

Safety

OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients

The recommended dosing regimen of OXLUMO consists of loading doses followed by maintenance doses administered subcutaneously.

Dosing is based on actual body weight.

Contraindications

  • None

Warnings and Precautions

  • None
  • For additional Important Safety Information on OXLUMO, see full Prescribing Information

11

Lumasiran Phase 3 Studies

Robust Registrational Program Evaluating Lumasiran Across all Ages and Full PH1 Disease Spectrum

Double-blind, placebo-

Single arm, open-label study

Single arm, open-label study

controlled trial in PH1

in PH1 patients less than 6

in PH1 patients with impaired

patients at least 6 years old

years old with preserved

renal function, including

with preserved renal function

renal function

advanced disease

Full results presented

Full results presented

Topline results

June 2020

October 2020

expected in 2021

Expanded Access Protocol (EAP) for PH1 patients initiated in U.S. and Europe

12

Andy Orth

Senior Vice President, Head of U.S. Business

Commercialization Strategy

13

OXLUMO Market Opportunity

Estimated U.S./EU Prevalence and Addressable Population

Patients in the U.S. / EU

MUTATION

PREVALENCE1

~4.3

per Million

(U.S./EU)

POTENTIALLY

DIAGNOSED

DIAGNOSED

SYMPTOMATIC

& NON-

PREVALENCE2-4

PREVALENCE^

TRANSPLANTED5

~3.5 to 4.0

~1.5 to 2.5

~1.2 to 2.0

per Million

per Million

per Million

(U.S./EU)

(U.S./EU)

(U.S./EU)

POTENTIALLY SYMPTOMATIC

~2,900 - 3,400

DIAGNOSED ~1,300 - 2,100

NON-

TRANSPLANTED

~1,000 -

1,700

Opportunity for growth with increased awareness and diagnosis, as well as global commercial expansion

† US population=328MM, EU population (including UK) = 513MM

^ Includes patients that are presymptomatic, subclinical, or symptomatic

Sources: 1. Hopp K, et al. J Am Soc Nephrol. 2015 Feb 2; 2. Cochat et al. Nephrol Dial Transplant. 1995; 10: 3-7; 3. Kopp and Leumann. Nephrol Dial Transplant. 1995; 10: 2224-2227; 4. van Woerden, et al. Nephrol Dial Transplant. 2003; 18: 273-279; 5. Data on

14 file. Alnylam chart review studies (US and EU) estimated 17% transplant rate, rounded up to 20%; 6. Cochat P, et al. N Engl J Med. 2013 Nov 28;369(22):2163; 7. Harambat J. Clin J Am Soc Nephrol. 2012 Mar;7(3):458-65; 8. Kamoun A. Pediatr Nephrol. 1996 Aug;10(4):479-82.

OXLUMO Market Opportunity

First-in-Class Product in Ultra-Rare Orphan Disease

PREVALENCEDIAGNOSISDISEASE BURDEN COST BURDEN

~1,300-2,100

~50%

30-65%

$1M+

patients with a confirmed PH1

currently diagnosed4; mean time

reach end-stage renal

average cost (transplant &

diagnosis in U.S./EU1-3

to diagnosis ~6 years5

disease before diagnosis5

lifelong immunosuppression)

LUMASIRAN | PRIMARY HYPEROXALURIA TYPE 1

>$500M potential market opportunity

15 1. Cochat et al. Nephrol Dial Transplant. 1995; 10: 3-7; 2. Kopp and Leumann. Nephrol Dial Transplant. 1995; 10: 2224-2227; 3. van Woerden, et al. Nephrol Dial Transplant. 2003; 18: 273-279; 4.Hopp R, et al. J Am Soc Nephrol. 2015;26:2559-2570; 5. Harambat J, et al. Kidney Int. 2010;77(5):443-449.

Alnylam's Patient Access Philosophy

Critical excerpts from Alnylam's Patient Access

Philosophy

• Demonstrate evidence-based value objectively and transparently

• Establish responsible pricing that reflects value delivered to patients, caregivers and society

• Proactively pursue reimbursement through value-based agreements and other innovative approaches

• Commit to growth through continuous innovations, not arbitrary price increases in the U.S.

Help Patients

Deliver Value

Be Proactive

16

Pricing, Cost & Access Considerations

DEBILITATING DISEASE, CHILDHOOD ONSET

  • Inherited disease typically presenting in childhood
  • Oxalate overproduction causes painful, recurrent kidney stones leading to irreversible renal failure & multi-organ damage

RISKS OF RENAL FAILURE & TRANSPLANT WITH SoC

  • Inevitable progression to renal failure can require 6-7 sessions of dialysis per week
  • Some patients endure 10-12 hours of dialysis per day and night, until they can undergo a dual or sequential liver/kidney transplant

ULTRA-RARE POPULATION

  • Estimated genetic prevalence in the range of 4 individuals per million in the US and EU
  • OXLUMO may be a good option for ~1000-1700 of these patients with a confirmed PH1 diagnosis who have not received a transplant

FIRST THERAPY TO TREAT PH1

  • 1st FDA-approved therapy to substantially reduce oxalate production in PH1
  • 53% reduction in urinary oxalate (vs placebo)
  • Safety profile understood in pediatric and adult patients

REDUCED BARRIERS TO ACCESS

  • New value-based agreement (VBA) framework and innovative Patient Need Adjustment (PNA) seek to mitigate barriers to access
  • Agreements aim to address payers' concerns on budget predictability:
    1. Administration across a broad age range of patients
    2. Unpredictable prevalence in rare and ultra-rare diseases

Average Annual WAC

$490,000 - before mandatory

rebates to government

institutions

Average Effective Net Price

$380,000

Price may vary per individual

dosing, since OXLUMO is

indicated for both pediatric and

adult patients and is dosed according to body weight

17

Launching in a Pandemic

Execution Tailored to Meet Needs of Customers

Leading with

New Engagement

Digital

Models

Disease awareness being

driven by digital and in-person

education

Sales representatives

Medical Science Liaisons

Field Reimbursement

Specialists

Broad Support

Proactive Payer

Services

Engagement

Comprehensive patient

Value-Based Agreements tied to

services program offering

clinical experience

support to patients throughout

treatment via wide range of

Price increase protection

personalized services:

Patient Need Adjustment to

In-house Case Managers

support patient access to therapy

Field-based Patient

by providing budget predictability

Education Liaisons

for payers

Includes financial support for

Prevalence-Based Adjustment to

eligible patients

address uncertainty in plan

patient prevalence

18

OXLUMO™ (lumasiran) FDA Approval

Q&A Session

19

To those who say "impossible, impractical, unrealistic," we say:

CHALLENGE ACCEPTED

© 2020 Alnylam Pharmaceuticals, Inc.

Disclaimer

Alnylam Pharmaceuticals Inc. published this content on 24 November 2020 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 24 November 2020 13:24:00 UTC


© Publicnow 2020
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