Delaying Onset Type 1 Diabetes (U.S.), March 2023
The MarketVue®: delaying onset of Type 1 Diabetes 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®: delaying onset of Type 1 Diabetes report is supported by 5 qualitative interviews with key opinion leaders, a quantitative survey with 24 U.S. physicians and secondary research.
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
Key companies mentioned:
- Sanofi
- Provention Bio / Sanofi
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Diamyd Medical
- Dompe Farmaceutici
- Novartis
- Imcyse
- Throne Biotechnologies
- Avotres Inc.
- Creative Medical Technology Holdings, Inc.
- CRISPR Therapeutics
- Vertex Pharmaceuticals
Key drugs mentioned:
- Teplizumab (Tzield)
- Metformin (Glucophage)
- Pramlintide (Symlin)
- Insulin
- Diamyd
- Ladarixin
- Anti-thymocyte globulin
- CFZ533 / iscalimab
- IMCY-0098
- Stem cell educator therapy
- AVT001
- CELZ-201
- VCTX211
- VCTX210A
- VX-264
- VX-880
- Verapamil (Calan, Isoptin)
- Imatinib (Gleevec)
Key takeaways from the report:
Type 1 Diabetes (T1D) is a life-long autoimmune disease where the immune system destroys the insulin-producing islet cells in the pancreas until they no longer produce insulin. To date, insulin replacement therapy has been the backbone of T1D treatment but has a significant treatment burden on patients. Recently, clinical development appears to be shifting towards therapies that could delay the need for insulin and preserve beta cell function.
The launch of Tzield is an important milestone in T1D disease management in that patients in the pre-diabetic stage now have an option to delay T1D onset by ~2 years, thereby delaying insulin use. Further, the approval has increased interest and awareness around screening patients who may be at risk for the disease.
While the approval of Tzield has created excitement amongst clinicians, the response to its efficacy and dosing is underwhelming; less than 30% of interviewed doctors reported that they found the dosing impressive, according to REACH Market Research’s MarketVue® assessment.
Endocrinologist, U.S.: “What are the potential benefits of a drug like teplizumab in a person who has stage 2 diabetes? It is not a magical bullet, but it’s a good start.”
The current T1D pipeline consists largely of insulin-based treatments with few therapies in development to delay T1D and insulin use in recent-onset patients, including:
- Sanofi’s teplizumab label extension for patients 0-7 yrs and recent-onset T1D
- NIDDK’s anti-thymocyte globulin and granulocyte colony-stimulating factor
- Diamyd Medical AB’s Diamyd®, a glutamic acid decarboxylase 65 (GAD65)-specific immunotherapy
- Dompe Farmaceutici S.p.A’s ladarixin, an anti-IL-8, CXCR1 and CXCR2 inhibitor
- Novartis’ iscalimab, an anti-CD40 mAb
Endocrinologists interviewed by REACH are eager for preventative treatments that offer a longer time to T1D progression and more convenient dosing than Tzield.
Meghana Pandit, REACH Analyst: “Tzield as a concept is viewed favorably by physicians, however, there are concerns around its dosing, and efficacy profile which is reported as marginal.”
1. DISEASE OVERVIEW
A heterogeneous, chronic, immune-mediated disease associated with the destruction of insulin-producing pancreatic beta cells
Figure 1.1. Endocrinologist-reported age range of T1D patients n=24
Figure 1.2. Endocrinologist-reported impact of T1D
Figure 1.3. T1D Beta-cell function by stage
Figure 1.4. Pathogenesis of T1D and associated drug targets
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Table 2.1. Incident populations of T1D and prevalent populations of stage 2 T1D in the U.S. and EU
3. DIAGNOSIS & CURRENT TREATMENT
Overview
Figure 3.1. Referral and diagnostic pathway for T1D prediabetic and diabetic patients
Delayed diagnosis of T1D is largely due to inadequate pediatric screening
Figure 3.2. Physician-reported proportion of T1D patients in each category at presentation
All T1D patients are treated with insulin to achieve control of blood sugar levels
Table 3.1. Treatment goals for T1D
Figure 3.3. Endocrinologist-reported percentage of T1D patients receiving some form of drug therapy besides insulin
Treatment flow for Type 1 Diabetes
Teplizumab approval is viewed as an important milestone in T1D disease management
Figure 3.4. Physician-reported experience/impression of Tzield
Physician perspectives on teplizumab attributes
Key treatment dynamics that will shape disease management and drug use in T1D
Figure 3.5. Important dynamics of T1D market evolution
4. UNMET NEED
Overview
Figure 4.1. Top unmet needs in Type 1 Diabetes
Figure 4.2. Physician-reported unmet needs in Type 1 Diabetes
Physician perspectives on unmet needs in T1D
5. PIPELINE ANALYSIS
Overview
Figure 5.1. Physician-reported perspectives on promising mechanisms of action for delaying T1D onset
Figure 5.2. Physician-reported perspectives on promising mechanism of action for recent-onset T1D
Pipeline for delaying onset of T1D is sparse
Table 5.1. Comparison of key late-stage trials for delaying onset of T1D
Reducing and delaying insulin use is the main focus of new-onset T1D treatments
Table 5.2. Comparison of key late-stage trials for new onset of T1D
Development in the T1D space is active
Table 5.3. Table 5.3. Competitive landscape for T1D
Physician perspectives on drug development in T1D
6. VALUE & ACCESS
Overview
Table 6.1. Current T1D therapy pricing, U.S.
Figure 6.1. T1D patients by insurance type
Table 6.2. Typical U.S. commercial payer coverage of teplizumab
Provention Bio (now under Sanofi) offers a Tzield patient support and access program
Table 6.3. COMPASS Navigator Benefits for Tzield patients
Table 6.4. Provention Bio’s road map for payer access success with teplizumab
Sanofi’s acquisition of Provention Bio will expand Teplizumab access
Table 6.2. Sanofi-provided benefits to Provention Bio in initial collaboration agreement
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
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