MarketVue® Report
Geographic Atrophy (U.S.), 2023

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Geographic Atrophy (U.S.), July 2023

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The MarketVue®: Geographic Atrophy market landscape report combines primary (KOL interviews and survey data) and secondary market research to empower strategic decision-making and provide a complete view of the market.
 
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
 
Topics covered in this report:
  • Disease overview: Review the disease pathophysiology and potential druggable targets
  • Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
  • Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
  • Unmet needs: Identify opportunities to address treatment or disease management gaps
  • Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
  • Value and access: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets
 
Methodology:
Research for the MarketVue®: Geographic Atrophy report is supported by 4 qualitative interviews with key opinion leaders, a quantitative survey with 25 U.S. physicians and secondary research.
 
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
 
Key companies mentioned:
  • Apellis Pharmaceuticals
  • Astellas Pharma
  • Iveric Bio
  • Novartis Pharmaceuticals
  • Alexion/AstraZeneca
  • Alkeus Pharmaceuticals
  • Belite Bio
  • ONL Therapeutics
  • Genentech
  • Ionis Pharmaceuticals
  • Evergreen Therapeutics
  • Janssen
  • Aviceda Therapeutics
  • Perceive Biotherapeutics
  • Cognition Therapeutics
 
Key drugs mentioned:
  • Pegcetacoplan (Syfovre)
  • AREDS/AREDS2
  • Avacincaptad pegol (Zimura)
  • Iptacopan (Fabhalta)
  • Danicopan (Voydeya)
  • ALK-001
  • LBS-008 / Tinlarebant
  • ONL1204
  • GT005
  • IONIS-FB-LRx
  • ASP7317
  • EG-301
  • JNJ-81201887
  • AVD-104
  • VOY-101
  • CT1812
  • OpRegen
 
Key takeaways from the report:
Prior to the approvals of Syfovre and Izervay, the treatment for GA consisted of nonspecific therapies that do not target GA pathophysiology directly (e.g., dietary supplements and surgeries).
 
Despite the availability of Syfovre for GA and significant number of patients receiving the drug already, interviewed ophthalmologists feel the functional benefit and safety profile of the drug could be improved.
 
Ophthalmologist, U.S.: “With Syfovre, the vision’s not getting better. In fact, the trials show it gets worse, not because of the drug but because of the natural history of the disease. Unfortunately, you can’t measure improvement on visual acuity but if you did microperimetry you would see that you’re maintaining these patients’ vision, but not improving.”
 
When asked about what improvements they’d like to see over Syfovre and Izervay, ophthalmologists report they would like to see less frequently administered products with fewer adverse events and treatments able to demonstrate improved functional benefit.
 
Fortunately for GA patients, the pipeline is full of novel incoming treatment options in development. Ongoing phase 3 trials include:
  • Alkeus’ oral chemically modified Vitamin A – ALK-001
  • Belite’s oralanti-RBP4– LBS-008
Bayley Koopman, Research Associate at REACH: “While approximately once monthly intravitreal injections represent the current standard of care, orally administered or ‘one-and-done’ treatments such as cell and gene therapies are in development for GA and could potentially reduce the burden of treatment if these therapies are able to demonstrate efficacy.”
1. DISEASE OVERVIEW
A progressive macular degeneration of the retina characterized by irreversible central vision loss
Figure 1.1. Risk factors for GA
Figure 1.2. Physician-reported proportion of their AMD patients that progress to GA
Disease mechanism
Figure 1.3. Overactivation of the complement cascade in GA leads to retinal degeneration
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1. U.S. and EU5 diagnosed prevalent cases of GA by region
Table 2.1. Prevalent cases of GA in populations aged 50-85 years in the U.S. and EU5
3. DIAGNOSIS & CURRENT TREATMENT
Overview
Figure 3.1. Referral and treatment pathway for GA
Physician insights on diagnosis of GA
Before Syfovre, physicians had no treatment options besides convincing patients to enroll in clinical trials
Figure 3.2. Current treatment patient share
Figure 3.3. Evolving treatment goals for GA, pre- and post-Syfovre approval
Syfovre approval is viewed as a first step in GA management; physicians find efficacy underwhelming
Figure 3.4. Physician-reported experience/impression of Syfovre
Overview of Syfovre’s OAKS and DERBY trial data
Syfovre demonstrates increasing effects over time with accelerated treatment effect between 18-24 months
Table 3.1. Lesion growth reduction was similar in pts with extrafoveal & subfoveal lesions at 18-24 mos
Table 3.2. Key insights from OAKS and DERBY trials
Ophthalmologists’ perspectives on Syfovre
Physicians report that there are few but significant barriers to Syfovre uptake
Figure 3.5. Ophthalmologist-reported most impactful barriers to receiving treatment with Syfovre
Figure 3.6. Percentage of non-prescribers citing reasons for not yet prescribing Syfovre
Key treatment dynamics that will shape disease management and drug use in GA
Figure 3.7. Important dynamics of GA market evolution
4. UNMET NEED
Overview
Figure 4.1. Ophthalmologist-reported unmet needs in GA patients
Physicians emphasize on the need for txs that not only slow down lesion growth but also provide functional benefit
5. PIPELINE ANALYSIS
Overview
Figure 5.1. Number of emerging therapies in Ph 3 and Ph 2 addressing key unmet needs in GA
Figure 5.2. Ophthalmologist-reported most promising mechanisms of action in the pipeline
GA pipeline is active with several therapies in Ph 2 and 3 development
Table 5.1. Key Phase 3 trials of drugs in development for GA
Complement inhibitors and cell/gene therapies dominate the mid-late stage GA pipeline
Table 5.2. Ongoing key Phase 2 trials of therapies in development for GA
Complement inhibitors and cell/gene therapies appear to be the future of GA management
Table 5.2. (contd.) Ongoing key Phase 2 trials of therapies in development for GA
Astellas Pharmaceutical’s Zimura is poised to compete for GA patient share, if approved
Figure 5.3. Select results from Phase 3 trials of Zimura in GA patients
There have been several failures of complement-targeted therapies in clinical development for GA
Table 5.3. Clinical trial failures of complement-targeted therapies
Figure 5.4. Physician-reported most meaningful trial outcomes to assess functional benefit
Physician perspectives on patient selection and outcomes in clinical trials
6. VALUE AND ACCESS
Overview
Table 6.1. Current pricing of select GA treatments
Table 6.2. Typical U.S. commercial payer coverage of Syfovre
Syfovre access and reimbursement dynamics in GA
Figure 6.1. GA patients by insurance type, U.S.
Figure 6.2. Figure 6.2. Reimbursement and Access Considerations for Emerging Therapies in GA
Figure 6.3. Ophthalmologist-reported percentage of patients who have difficulty accessing and/or staying on Syfovre due to insurance restrictions, high OOP costs, or prior authorizations
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
Disease definition
Diagnosed prevalence estimates
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