Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
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This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Key Takeaways
- Datamonitor Healthcare estimates that in 2017, there were 47,190 incident cases of amyotrophic lateral sclerosis (ALS) in adults aged 40 years and older worldwide, and forecasts that number to increase to 56,070 incident cases by 2026.
- Oceania and Europe are estimated to have the highest disease incidence (2.33 and 2.16 cases per 100,000 people, respectively), while Asia and Africa have the lowest incidence (0.62 and 0.89 cases per 100,000 people, respectively).
- Italfarmaco’s Tiglutik, Mitsubishi Tanabe’s Radicava, Advanz’s Rilutek, and Corestem’s Neuronata-R are the only marketed drugs available for ALS. These drugs are administered via the intravenous and oral routes.
- The greatest proportion of industry-sponsored drugs in active clinical development for ALS are in Phase II, with two drugs in the NDA/BLA phase.
- Therapies in mid-to-late-stage development for ALS focus on a wide variety of targets. The most popular method of administration is the oral route.
- High-impact upcoming events for drugs in the ALS space comprise topline Phase II and Phase III trial results for a number of drugs, and expected PDUFA dates for BHV-0223 and Exservan.
- The overall likelihood of approval of a Phase I ALS asset is 7.1%, and the average probability a drug advances from Phase III is 42.9%. Drugs, on average, take 7.9 years from Phase I to approval, compared to 9.7 years in the overall neurology space.
- There have been 26 licensing and asset acquisition deals involving ALS drugs during 2014–19. The $1,225m exclusive collaboration and license agreement signed in October 2018 between Denali and Sanofi to develop and commercialize products containing receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitors for neurological indications such as Alzheimer’s disease, ALS, and multiple sclerosis was the largest deal.
- The distribution of clinical trials across Phase I–IV indicates that the majority of trials for ALS have been in the early and midphases 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 ALS clinical trials globally. The UK leads the major EU markets, while Japan has the top spot in Asia.
- Clinical trial activity in the ALS space is dominated by completed trials. Otsuka/Avanir have the highest number of completed clinical trials for ALS, with 11 trials.
- Biogen leads industry sponsors with the highest overall number of clinical trials for ALS.
CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
8 Subtypes
9 TREATMENT
9 Symptomatic treatments
9 Ventilatory management
9 Nutritional management
9 Disease-modifying therapies
11 EPIDEMIOLOGY
14 MARKETED DRUGS
16 PIPELINE DRUGS
24 RECENT EVENTS AND ANALYST OPINION
24 IONIS-SOD1Rx for ALS (May 7, 2019)
25 CK-2127107 for ALS (May 5, 2019)
27 Masitinib for ALS (May 28, 2018)
28 NP001 for ALS (April 26, 2018)
30 IONIS-C9Rx for ALS (April 20, 2018)
31 IONIS-SOD1Rx for ALS (April 20, 2018)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
35 Wanted: Orphan Drugs, Antivirals Dominate China’s Latest Fast Track List
35 EMA Decides Fast-Track Fate Of Masitinib And Anthrax Antitoxin
35 ITF Pharma Plans October US Roll-Out Of Liquid Riluzole Formulation For ALS
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 Deals Shaping The Medical Industry, February 2019
37 Sanofi Stumps Up $125m To Access Denali RIPK1 Inhibitors
38 $27.5m For AliveGen Neuromuscular Candidates
38 Celgene Options Neuro Candidates From Skyhawk, Participates In Series A Round
39 PARENT PATENTS
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
45 BIBLIOGRAPHY
45 Prescription information
46 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in incident cases of ALS, 2017–26
16 Figure 2: Overview of pipeline drugs for ALS in the US
17 Figure 3: Pipeline drugs for ALS, by company
17 Figure 4: Pipeline drugs for ALS, by drug type
18 Figure 5: Pipeline drugs for ALS, by classification
25 Figure 6: IONIS-SOD1Rx for ALS (May 7, 2019): Phase I/II – 233AS101
27 Figure 7: CK-2127107 for ALS (May 5, 2019): Phase II – FORTITUDE-ALS
30 Figure 8: NP001 for ALS (April 26, 2018): Phase II – North America
31 Figure 9: IONIS-C9Rx for ALS (April 20, 2018): Partnership – Amendment/Restructuring
33 Figure 10: IONIS-SOD1Rx for ALS (April 20, 2018): Partnership – Amendment/Restructuring
34 Figure 11: Key upcoming events in ALS
36 Figure 12: Probability of success in the ALS pipeline
37 Figure 13: Licensing and asset acquisition deals in ALS, 2014–19
39 Figure 14: Parent patents in ALS
40 Figure 15: Clinical trials in ALS
40 Figure 16: Top 10 drugs for clinical trials in ALS
41 Figure 17: Top 10 companies for clinical trials in ALS
41 Figure 18: Trial locations in ALS
42 Figure 19: ALS trials status
43 Figure 20: ALS trials sponsors, by phase
LIST OF TABLES
11 Table 1: Incident cases of ALS, 2017–26
13 Table 2: Incidence rates of ALS, 2017–26
15 Table 3: Marketed drugs for ALS
19 Table 4: Pipeline drugs for ALS in the US
24 Table 5: IONIS-SOD1Rx for ALS (May 7, 2019)
26 Table 6: CK-2127107 for ALS (May 5, 2019)
28 Table 7: Masitinib for ALS (May 28, 2018)
29 Table 8: NP001 for ALS (April 26, 2018)
30 Table 9: IONIS-C9Rx for ALS (April 20, 2018)
32 Table 10: IONIS-SOD1Rx for ALS (April 20, 2018)
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