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 marketed and pipeline drugs, clinical trials, upcoming and regulatory events, recent events and analyst opinion, a 10-year disease incidence forecast, probability of success, and licensing and acquisition deals.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key marketed and pipeline drugs, clinical trials, upcoming and regulatory events, recent events and analyst opinion, a 10-year disease incidence forecast, probability of success, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Key Takeaways
- Datamonitor Healthcare estimates that in 2019, there were 28,100 incident cases of amyotrophic lateral sclerosis (ALS) in adults aged 40 years and older worldwide, and forecasts that number to increase to 32,600 incident cases by 2028.
- Oceania and Europe are estimated to have the highest disease incidence rates (2.33 and 2.16 cases per 100,000 people, respectively), while Asia and Africa have the lowest incidence rates (0.62 and 0.89 cases per 100,000 people, respectively).
- Approved drugs in the ALS space target glutamine, stem cells/other cell therapies, and mitochondria. These drugs are administered via the sublingual, oral transmucosal, intrathecal, intravenous, and oral routes.
- The largest proportion of industry-sponsored drugs in active clinical development for ALS are in Phase II. Therapies in active clinical 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 updated Phase II trial results and topline Phase II, Phase IIa, Phase II/III, and Phase III trial results.
- The overall likelihood of approval of a Phase I ALS asset is 7.8%, and the average probability a drug advances from Phase III is 37.5%. ALS drugs, on average, take 7.0 years from Phase I to approval, compared to 10.1 years in the overall neurology space.
- 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 80% of trials in Phase I–II, and only 20% in Phase III–IV.
- The US has a substantial lead in the number of ALS clinical trials globally. The UK leads the major European markets, while Japan has the top spot in Asia.
- Clinical trial activity in the ALS space is dominated by completed trials. Mitsubishi Tanabe has the highest number of completed clinical trials for ALS, with 16 trials.
- Mitsubishi Tanabe leads the 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
25 RECENT EVENTS AND ANALYST OPINION
25 Miplyffa for ALS (May 7, 2021)
26 NurOwn for ALS (November 17, 2020)
27 AMX0035 for ALS (September 2, 2020)
29 Simdax for ALS (July 28, 2020)
31 KEY UPCOMING EVENTS
32 KEY REGULATORY EVENTS
32 BrainStorm’s Phase III Data Do Not Support Clinical Benefit Of NurOwn In ALS, US FDA Says
33 PROBABILITY OF SUCCESS
34 LICENSING AND ASSET ACQUISITION DEALS
34 Ultomiris And Successor Key To AstraZeneca’s Rare Disease Ambitions
34 Abbiska Partners With Sperogenix To Develop ABSK021
34 Lilly/Verge Pact Shows Resistance To Biopharma AI Is Futile
34 GSK Looks To Immuno-Neurology In $700m Alector Tie-up
35 CRISPR Therapeutics Enters Partnership With Capsida Biotherapeutics To Develop Gene-Editing Therapies
35 Lilly Broadens Its CNS Reach With Rigel RIPK1 Deal
35 Helixmith Ties Up With Wacker, Sells US DNA Manufacturing Firm
36 Mitsubishi Tanabe To Market Aquestive ALS Drug In US
36 Eli Lilly Acquires Prevail Therapeutics In $880m Deal
36 Bristol Seeks Novel Targets, Candidates For ALS, FTD
36 Scribe Inks CRISPR-Based ALS Partnership With Biogen
37 Novus Acquires Anelixis, ALS Candidate In Stock Swap Transaction
38 REVENUE OPPORTUNITY
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
45 BIBLIOGRAPHY
46 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in incident cases of ALS, 2019–28
16 Figure 2: Overview of pipeline drugs for ALS in the US
16 Figure 3: Pipeline drugs for ALS, by company
17 Figure 4: Pipeline drugs for ALS, by drug type
17 Figure 5: Pipeline drugs for ALS, by classification
26 Figure 6: Miplyffa for ALS (May 7, 2021): Phase III – ORARIALS-01
27 Figure 7: NurOwn for ALS (November 17, 2020): Phase III – BCT-002-US (US/Canada)
30 Figure 8: Simdax for ALS (July 28, 2020): Phase III – REFALS
31 Figure 9: Key upcoming events in ALS
33 Figure 10: Probability of success in the ALS pipeline
40 Figure 11: Clinical trials in ALS
40 Figure 12: Top 10 drugs for clinical trials in ALS
41 Figure 13: Top 10 companies for clinical trials in ALS
41 Figure 14: Trial locations in ALS
42 Figure 15: ALS trials status
43 Figure 16: ALS trials sponsors, by phase
LIST OF TABLES
11 Table 1: Incident cases of ALS, 2019–28
13 Table 2: Incidence rates of ALS, 2019–28
15 Table 3: Marketed drugs for ALS
18 Table 4: Pipeline drugs for ALS in the US
25 Table 5: Miplyffa for ALS (May 7, 2021)
26 Table 6: NurOwn for ALS (November 17, 2020)
28 Table 7: AMX0035 for ALS (September 2, 2020)
29 Table 8: Simdax for ALS (July 28, 2020)
38 Table 9: Historical global sales, by drug ($m), 2016–20
39 Table 10: Forecasted global sales, by drug ($m), 2021–25
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