Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
$1,318.00
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 majority of industry-sponsored drugs in active clinical development for ALS are in Phase II, with only one drug in the NDA/BLA phase.
- 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 topline Phase II, Phase IIa, Phase II/III, and Phase III trial results, expected PDUFA dates, a CHMP opinion, and an estimated European approval decision.
- The overall likelihood of approval of a Phase I ALS asset is 9.1%, and the average probability a drug advances from Phase III is 33.3%. ALS drugs, on average, take 7.5 years from Phase I to approval, compared to 10.2 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 20 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 CNM-Au8 for ALS (November 2, 2021)
26 IONIS-SOD1Rx for ALS (October 17, 2021)
28 Ultomiris for ALS (August 20, 2021)
29 Miplyffa for ALS (May 7, 2021)
31 NurOwn for ALS (November 17, 2020)
32 AMX0035 for ALS (September 2, 2020)
35 KEY UPCOMING EVENTS
36 KEY REGULATORY EVENTS
36 Mitsubishi Tanabe Bids To Take Radicava Oral
36 House ALS Bill Would Require US FDA ‘Action Plan’ – But Not Conditional Approval
36 AMX0035 NDA Rides On CENTAUR, Waits For PEGASUS To Land
37 BrainStorm’s Phase III Data Do Not Support Clinical Benefit Of NurOwn In ALS, US FDA Says
38 PROBABILITY OF SUCCESS
39 LICENSING AND ASSET ACQUISITION DEALS
39 GSK Dives Into Neurology With £30m Oxford University Pact
39 Neumora Launches With $500m To Advance Personalized Neuroscience Drugs
39 Ultomiris And Successor Key To AstraZeneca’s Rare Disease Ambitions
40 Abbiska Partners With Sperogenix To Develop ABSK021
40 Lilly/Verge Pact Shows Resistance To Biopharma AI Is Futile
40 GSK Looks To Immuno-Neurology In $700m Alector Tie-up
40 CRISPR Therapeutics Enters Partnership With Capsida Biotherapeutics To Develop Gene-Editing Therapies
41 Lilly Broadens Its CNS Reach With Rigel RIPK1 Deal
41 Helixmith Ties Up With Wacker, Sells US DNA Manufacturing Firm
41 Mitsubishi Tanabe To Market Aquestive ALS Drug In US
42 Eli Lilly Acquires Prevail Therapeutics In $880m Deal
43 REVENUE OPPORTUNITY
44 CLINICAL TRIAL LANDSCAPE
45 Sponsors by status
46 Sponsors by phase
47 Recent events
49 BIBLIOGRAPHY
50 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in incident cases of ALS, 2019–28
16 Figure 2: Overview of pipeline drugs for ALS in the US
16 Figure 3: Pipeline drugs for ALS, by company
17 Figure 4: Pipeline drugs for ALS, by drug type
17 Figure 5: Pipeline drugs for ALS, by classification
26 Figure 6: CNM-Au8 for ALS (November 2, 2021): Phase II – RESCUE-ALS
28 Figure 7: IONIS-SOD1Rx for ALS (October 17, 2021): Phase III – VALOR
31 Figure 8: Miplyffa for ALS (May 7, 2021): Phase III – ORARIALS-01
32 Figure 9: NurOwn for ALS (November 17, 2020): Phase III – BCT-002-US (US/Canada)
35 Figure 10: Key upcoming events in ALS
38 Figure 11: Probability of success in the ALS pipeline
44 Figure 12: Clinical trials in ALS
44 Figure 13: Top 10 drugs for clinical trials in ALS
45 Figure 14: Top 10 companies for clinical trials in ALS
45 Figure 15: Trial locations in ALS
46 Figure 16: ALS trials status
47 Figure 17: ALS trials sponsors, by phase
LIST OF TABLES
11 Table 1: Incident cases of ALS, 2019–28
13 Table 2: Incidence rates of ALS, 2019–28
15 Table 3: Marketed drugs for ALS
18 Table 4: Pipeline drugs for ALS in the US
25 Table 5: CNM-Au8 for ALS (November 2, 2021)
27 Table 6: IONIS-SOD1Rx for ALS (October 17, 2021)
29 Table 7: Ultomiris for ALS (August 20, 2021)
30 Table 8: Miplyffa for ALS (May 7, 2021)
31 Table 9: NurOwn for ALS (November 17, 2020)
33 Table 10: AMX0035 for ALS (September 2, 2020)
43 Table 11: Historical global sales, by drug ($m), 2016–20
43 Table 12: Forecasted global sales, by drug ($m), 2022–26
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