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
Amyotrophic lateral sclerosis (ALS) is a fatal late-onset neurodegenerative disorder characterized by progressive muscular paralysis. It leads to damage of the upper and lower motor neurons present in the corticospinal tracts, brainstem, primary motor cortex, and spinal cord. The first onset of symptoms is generally seen in patients aged 50–65 years. Common symptoms reported by patients with ALS include muscle twitching, weakness, and cramping, which subsequently lead to muscle impairment. Furthermore, patients in the final stages of ALS will develop symptoms of dysphagia and dyspnea.
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 for CNM-Au8, topline Phase II trial results for Radicava and AT-1501, topline Phase IIa trial results for ANX-005, topline Phase II/III trial results for Cu(II)ATSM and verdiperstat, and topline Phase III trial results for arimoclomol, IONIS-SOD1Rx, and Ultomiris.
- The overall likelihood of approval of a Phase I ALS asset is 9.4%, and the average probability a drug advances from Phase III is 42.9%. ALS drugs, on average, take 7.0 years from Phase I to approval, compared to 9.8 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 79% of trials in Phase I–II, and only 21% 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 14 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
24 RECENT EVENTS AND ANALYST OPINION
24 NurOwn for ALS (November 17, 2020)
25 AMX0035 for ALS (September 2, 2020)
27 Simdax for ALS (July 28, 2020)
28 AMX0035 for ALS (December 17, 2019)
30 MN-166 for ALS (November 8, 2019)
31 Radicava for ALS (September 17, 2019)
33 KEY UPCOMING EVENTS
34 PROBABILITY OF SUCCESS
35 LICENSING AND ASSET ACQUISITION DEALS
35 Bristol Seeks Novel Targets, Candidates For ALS, FTD
35 Scribe Inks CRISPR-Based ALS Partnership With Biogen
35 Novus Acquires Anelixis, ALS Candidate In Stock Swap Transaction
35 Deal Watch: Merck Gets Rights To Yumanity’s Preclinical ALS, FTLD Candidates
37 CLINICAL TRIAL LANDSCAPE
38 Sponsors by status
39 Sponsors by phase
40 Recent events
42 BIBLIOGRAPHY
42 Prescription information
43 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
25 Figure 6: NurOwn for ALS (November 17, 2020): Phase III – BCT-002-US (US/Canada)
28 Figure 7: Simdax for ALS (July 28, 2020): Phase III – REFALS
30 Figure 8: AMX0035 for ALS (December 17, 2019): Phase II – CENTAUR
31 Figure 9: MN-166 for ALS (November 8, 2019): Phase IIa – ALS Biomarker
33 Figure 10: Key upcoming events in ALS
34 Figure 11: Probability of success in the ALS pipeline
37 Figure 12: Clinical trials in ALS
37 Figure 13: Top 10 drugs for clinical trials in ALS
38 Figure 14: Top 10 companies for clinical trials in ALS
38 Figure 15: Trial locations in ALS
39 Figure 16: ALS trials status
40 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
24 Table 5: NurOwn for ALS (November 17, 2020)
26 Table 6: AMX0035 for ALS (September 2, 2020)
27 Table 7: Simdax for ALS (July 28, 2020)
29 Table 8: AMX0035 for ALS (December 17, 2019)
30 Table 9: MN-166 for ALS (November 8, 2019)
32 Table 10: Radicava for ALS (September 17, 2019)
© Informa UK Ltd. This document is a licensed product and is not to be reproduced or redistributed
Do you have a subscription to Datamonitor Healthcare, Biomedtracker or Meddevicetracker? You may already have access to these reports, contact your account manager or email pharma@informabi.com for further help or assistance.
Market Spotlight: Tuberculosis (TB) 
Sign up to the