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.
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 2021, there were 29,000 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 common method of administration is the oral route.
- High-impact upcoming events for drugs in the ALS space comprise topline Phase II, Phase IIa, Phase IIb, Phase II/III, and Phase III trial results, an expected PDUFA date, a CHMP opinion, an estimated European approval decision, and an FDA advisory panel brief as well as panel meeting.
- The overall likelihood of approval of a Phase I ALS asset is 9%, and the average probability a drug advances from Phase III is 33.3%. ALS drugs, on average, take 7.3 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 83% of trials in Phase I–II, and only 17% 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 15 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 AMX0035 for ALS (March 30, 2022)
27 AMX0035 for ALS (March 28, 2022)
31 CNM-Au8 for ALS (November 2, 2021)
33 IONIS-SOD1Rx for ALS (October 17, 2021)
35 Ultomiris for ALS (August 20, 2021)
36 Miplyffa for ALS (May 7, 2021)
39 KEY UPCOMING EVENTS
40 KEY REGULATORY EVENTS
40 Will Amylyx’s ALS Drug Benefit From US FDA’s Aduhelm Experience?
40 US FDA’s ALS Science Strategy Includes Near-Term Rare Neurodegenerative Disease Task Force
41 Mitsubishi’s Oral Radicava Provides Convenience Option For ALS
41 Amylyx Betting On Regulatory Flexibility In ALS
41 Amylyx’s ALS Drug Should Wait For Phase III Results, US FDA Panel Says
42 New EU Filings Include Amylyx’s AMX0035
42 Mitsubishi Tanabe Bids To Take Radicava Oral
42 House ALS Bill Would Require US FDA ‘Action Plan’ – But Not Conditional Approval
43 AMX0035 NDA Rides On CENTAUR, Waits For PEGASUS To Land
44 PROBABILITY OF SUCCESS
45 LICENSING AND ASSET ACQUISITION DEALS
45 Biogen Returns ALS Drug To Karyopharm
45 Pasithea Therapeutics Acquires Alpha-5 Integrin
45 Pfizer Acquires Biohaven Pharmaceutical For $11.6bn
45 Aquestive Enters Into Licensing Agreement With Haisco
45 Roche Partners With Aquinnah On Oral Therapies For ALS
46 GSK Dives Into Neurology With £30m Oxford University Pact
46 Neumora Launches With $500m To Advance Personalized Neuroscience Drugs
46 Ultomiris And Successor Key To AstraZeneca’s Rare Disease Ambitions
47 Abbiska Partners With Sperogenix To Develop ABSK021
47 Lilly/Verge Pact Shows Resistance To Biopharma AI Is Futile
47 GSK Looks To Immuno-Neurology In $700m Alector Tie-up
49 REVENUE OPPORTUNITY
50 CLINICAL TRIAL LANDSCAPE
51 Sponsors by status
52 Sponsors by phase
53 Recent events
55 BIBLIOGRAPHY
56 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in incident cases of ALS, 2019–28
16 Figure 2: Overview of pipeline drugs for ALS in the US
16 Figure 3: Pipeline drugs for ALS, by company
17 Figure 4: Pipeline drugs for ALS, by drug type
17 Figure 5: Pipeline drugs for ALS, by classification
33 Figure 6: CNM-Au8 for ALS (November 2, 2021): Phase II – RESCUE-ALS
35 Figure 7: IONIS-SOD1Rx for ALS (October 17, 2021): Phase III – VALOR
38 Figure 8: Miplyffa for ALS (May 7, 2021): Phase III – ORARIALS-01
39 Figure 9: Key upcoming events in ALS
44 Figure 10: Probability of success in the ALS pipeline
50 Figure 11: Clinical trials in ALS
50 Figure 12: Top 10 drugs for clinical trials in ALS
51 Figure 13: Top 10 companies for clinical trials in ALS
51 Figure 14: Trial locations in ALS
52 Figure 15: ALS trials status
53 Figure 16: ALS trials sponsors, by phase
LIST OF TABLES
11 Table 1: Incident cases of ALS, 2019–28
13 Table 2: Incidence rates of ALS, 2019–28
15 Table 3: Marketed drugs for ALS
18 Table 4: Pipeline drugs for ALS in the US
25 Table 5: AMX0035 for ALS (March 30, 2022)
28 Table 6: AMX0035 for ALS (March 28, 2022)
32 Table 7: CNM-Au8 for ALS (November 2, 2021)
34 Table 8: IONIS-SOD1Rx for ALS (October 17, 2021)
36 Table 9: Ultomiris for ALS (August 20, 2021)
37 Table 10: Miplyffa for ALS (May 7, 2021)
49 Table 11: Historical global sales, by drug ($m), 2017–21
49 Table 12: Forecasted global sales, by drug ($m), 2022–26
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