Dublin, June 24, 2019 (GLOBE NEWSWIRE) -- The "Market Spotlight to 2027: Amyloidosis" report has been added to ResearchAndMarkets.com's offering.

This Market Spotlight report covers the amyloidosis market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, key upcoming events, probability of success, patent information, a 10-year disease incidence forecast, and licensing and acquisition deals.

Key Takeaways

• The author estimates that in 2017, there were approximately 90,500 incident cases of amyloidosis worldwide, with the highest proportion being the primary (amyloid light-chain; AL) amyloidosis type.
• Among incident multiple myeloma cases, the author estimates there were approximately 19,300 cases of AL amyloidosis worldwide in 2017.
• Pfizer's Vyndaqel was the first therapy to gain approval in the EU for hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. The drug is not approved in the US, where it received a complete response letter from the US Food and Drug Administration (FDA) in 2012. Alnylam's Onpattro and Akcea's Tegsedi are FDA-approved treatments in the US for hATTR amyloidosis with polyneuropathy in adults. All of these drugs are designed to block the production of TTR protein.
• The majority of industry-sponsored drugs in active clinical development for amyloidosis are in Phase I. Therapies in development for amyloidosis focus on targets such as transthyretin, amyloid-light chain, cluster of differentiation 38, proteasome, serum amyloid P component, alpha-synuclein, amyloid beta/amyloid plaques, tau proteins, and catechol-Omethyltransferase. These therapies are administered via the intravenous, oral, and subcutaneous routes.
• High-impact upcoming events for drugs in the amyloidosis space comprise topline Phase II trial results for dezamizumab and GSK2315698A.
• The overall likelihood of approval of a Phase I metabolic-other asset is 7.2%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 9.3 years from Phase I to approval, compared to 8.9 years in the overall metabolic space.
• There have been only seven licensing and asset acquisition deals involving amyloidosis drugs during 2013-18. The largest deal was the $1,740m agreement in 2018 between Ionis Pharmaceuticals and Akcea Therapeutics, pursuant to which Akcea was granted an exclusive worldwide license to Ionis's inotersen and AKCEA-TTR-LRx (formerly IONIS-TTR-LRx).
• The clinical trials distribution across Phase I-IV indicates that the majority of trials for amyloidosis have been in the early and mid-phases of development, with 81% of trials in Phase I-II, and only 19% in Phase III-IV.
• The US has a substantial lead in the number of amyloidosis clinical trials globally, while the UK leads the major EU markets. - GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with four trials.
• GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis, followed by Pfizer.

Key Topics Covered

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND
Amyloidosis subtypes

TREATMENT
Chemotherapy
Targeted therapy
Surgery
Bone marrow/stem cell transplantation
Palliative or supportive care

EPIDEMIOLOGY
Amyloidosis incidence methodology
Amyloidosis in multiple myeloma incidence methodology
TTR amyloidosis

MARKETED DRUGS
Approvals by country

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION
AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)
Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
Onpattro for hATTR Amyloidosis with Polyneuropathy (April 24, 2018)
NEOD001 for AL Amyloidosis (April 23, 2018)
CAEL-101 for AL Amyloidosis (April 23, 2018)
Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
ALN-TTRsc02 for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017)
Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017)
Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017)
Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS
US FDA Marches Toward Record Novel Approvals With Ionis' Tegsedi, Leadiant's Revcovi
Aimovig And Tegsedi In Raft Of Products To Get EMA Thumbs up

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS
GSK Passes On Taking Inotersen To FDA With Ionis
Fortress Subsidiary Licenses Amyloidosis Candidate From Columbia

PARENT PATENTS

CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Recent events

BIBLIOGRAPHY
Prescription information

APPENDIX

LIST OF FIGURES
Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2017-26
Figure 2: Overview of pipeline drugs for amyloidosis in the US
Figure 3: Pipeline drugs for amyloidosis, by company
Figure 4: Pipeline drugs for amyloidosis, by drug type
Figure 5: Pipeline drugs for amyloidosis, by classification
Figure 6: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018): Phase II - Dose-Ranging (201)
Figure 7: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
Figure 8: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 24, 2018): Phase III - APOLLO
Figure 9: NEOD001 for AL Amyloidosis (April 23, 2018): Phase IIb - PRONTO, Phase IIb - PRONTO (OLE), Phase III - VITAL
Figure 10: Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018): Phase III - ATTR-ACT
Figure 11: Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017): Phase II/III - NEURO-TTR
Figure 12: Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017): Phase III - APOLLO
Figure 13: Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017): Phase II/III - NEURO-TTR
Figure 14: Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017): Phase III - APOLLO
Figure 15: Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017): Trial Data - Top-Line Results
Figure 16: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017): Phase II - 002 (Ext.)
Figure 17: Key upcoming events in amyloidosis
Figure 18: Probability of success in the amyloidosis pipeline
Figure 19: Licensing and asset acquisition deals in amyloidosis, 2013-18
Figure 20: Parent patents in amyloidosis
Figure 21: Clinical trials in amyloidosis
Figure 22: Top 10 drugs for clinical trials in amyloidosis
Figure 23: Top 10 companies for clinical trials in amyloidosis
Figure 24: Trial locations in amyloidosis
Figure 25: Amyloidosis trials status
Figure 26: Amyloidosis trials sponsors, by phase

LIST OF TABLES
Table 1: Global incident cases of amyloidosis in adults, by type, 2017-26
Table 2: Global incident cases of primary amyloidosis in multiple myeloma, 2017-26
Table 3: Marketed drugs for amyloidosis
Table 4: Approvals by country for amyloidosis
Table 5: Pipeline drugs for amyloidosis in the US
Table 6: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)
Table 7: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
Table 8: Onpattro for hATTR Amyloidosis With Polyneuropathy (April 24, 2018)
Table 9: NEOD001 for AL Amyloidosis (April 23, 2018)
Table 10: CAEL-101 for AL Amyloidosis (April 23, 2018)
Table 11: Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
Table 12: ALN-TTRsc02 for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
Table 13: Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
Table 14: Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
Table 15: Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017)
Table 16: Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017)
Table 17: Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017)
Table 18: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017)

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