Cure SMA Updates on ZolgensmaAugust 2019

AGENDA

1. Introduction2. Food and Drug Administration3. Cure SMA4. AveXis/Novartis

-What, When, Impact. -US Only.-Not Why, Who.

Peter Marks, M.D., Ph.D.Director, Center for Biologics Evaluation and Research

August 28, 2019

Update on Zolgensma

1. Summary of approval2. Background on data issue3. Current status of Zolgensma4. Responses to questions

FDA-Approved Label: INDICATIONS AND USAGE

ZOLGENSMA (onasemnogene abeparvovec-xioi)

• IV delivery AAV9 vector-based gene therapy Approved for: • patients

Data Issues Impact

1. Clinical Trials 2. MAP3. Current Commercially Approved4. Future Trials and Label Expansion

Current Dosing Access Metrics

A. 44 sites in 25 states (including DC) are publicly identified as offering Zolgensma

B. 31 commercial plans and 13 Medicaid plans have policies in place

Zolgensma Treatment Sites:44 sites in 25 states (including DC)

https://www.curesma.org/find-a-location/Send sites and contacts to patientcare@curesma.org

https://www.curesma.org/find-a-location/

Trial vs Label vs Policy-FDA label includes:

• < 2 years of age at dosing• All copy numbers of SMN2• All types

-InsuranceCoveragePolicies?

-IV Clinical trials included:• < 9 months of age at dosing• Two copies of SMN2

Insurance Coverage

Insurance Coverage Status1. Don’t get discouraged

A. Policies are not fixed1) Updated and individually implemented

B. Denials expected at this stage1) Appeals2) Precedents.

C. Let us know

Cure SMA Coverage & Payment Activities 1. Letters and Information

A. CEOs and CMOs of all major insurance plans and companies

B. All of the state Medicaid directors and governors

2. Next Steps:A. Meetings with various national organizations and

government agencies involved in recommending, influencing, and otherwise determining coverage and payment policy1) Testimony Opportunities

B. Focus on any restrictions to label

Future Steps for Gene Therapy

1. Intrathecal (IT) delivery for older and bigger patients:A. Differences to IVB. Current Clinical Trial: Study of Intrathecal Administration

of AVXS-101 for Spinal Muscular Atrophy 1) Clinicaltrials.gov Identifier: NCT033817292) Also known as STRONG3) Phase1: 51 subjects at 11 sites in the US4) 6 months to 60 months of age and three copies of SMN2 and

symptoms < 12 months of age5) Sit independently and not standing or walking independently6) Three different dose cohorts

C. Potentially additional trialsD. Data for FDA and Community and Coverage

Choices and Waiting

1. Discuss the following with your neurologistA. All available safety and efficacy dataB. Method of administrationC. Practical and process timing issues

2. Cure SMA recommends not waiting for a future treatment or trial option if other options are available nowA. Earliest possible administration of SMN enhancing drugs

yields best resultsB. Timing is one of the biggest factors in degree of response

to SMN upregulation

Care and Monitoring

• Continue close follow up by an experienced healthcare provider:• Monitor for symptoms of SMA• Monitor for side effects of treatments• Optimize the benefits of treatment• Discuss new information about SMA

Resources• General Information and current insurance policies:

– https://www.curesma.org/Zolgensma/• Administration Site List:

– https://www.curesma.org/find-a-location/• Choice and Connection to Care Booklet (general

information on choosing and navigating insurance coverage)– https://www.curesma.org/care-series-booklets/

• Additional questions about Zolgensma or other treatment-related inquiries:– Email treatments@curesma.org

https://www.curesma.org/Zolgensma/https://www.curesma.org/find-a-location/https://www.curesma.org/care-series-booklets/mailto:treatments@curesma.org

AVEXIS UPDATEDave Lennon, PhD, President

CONFIDENTIAL 1

Avexis update

Dave Lennon, PhD, President

CONFIDENTIAL

1

Cure SMA Updates on Zolgensma�August 2019AGENDAUpdate on Zolgensma FDA-Approved Label: �INDICATIONS AND USAGE Data Issues ImpactCurrent Dosing Access MetricsZolgensma Treatment Sites:�44 sites in 25 states (including DC)Trial vs Label vs PolicySlide Number 9Insurance Coverage StatusCure SMA Coverage & Payment Activities Future Steps for Gene TherapyChoices and WaitingCare and MonitoringResourcesSlide Number 16Slide Number 17