Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)
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
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.
- 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.
- Industry-sponsored drugs in active clinical development for ALS are spread evenly across Phase I and 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 topline Phase II trial results for Radicava, topline Phase II/III trial results for Cu(II)ATSM, and topline Phase III trial results for NurOwn, arimoclomol, and IONIS-SOD1Rx.
- The overall likelihood of approval of a Phase I ALS asset is 10.6%, and the average probability a drug advances from Phase III is 42.9%. ALS drugs, on average, take 7.3 years from Phase I to approval, compared to 9.8 years in the overall neurology space.
- There have been 23 licensing and asset acquisition deals involving ALS drugs during 2015–20. 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 European 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 and Mitsubishi Tanabe lead the industry sponsors with the highest overall number of clinical trials for ALS
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
9 Symptomatic treatments
9 Ventilatory management
9 Nutritional management
9 Disease-modifying therapies
14 MARKETED DRUGS
16 PIPELINE DRUGS
24 RECENT EVENTS AND ANALYST OPINION
24 AMX0035 for ALS (December 17, 2019)
25 MN-166 for ALS (November 8, 2019)
27 Radicava for ALS (September 17, 2019)
28 IONIS-SOD1Rx for ALS (May 7, 2019)
30 Reldesemtiv for ALS (May 5, 2019)
32 KEY UPCOMING EVENTS
33 KEY REGULATORY EVENTS
33 US FDA Reassures ALS Patients In Final Development Guidance
33 Hello NMPA: New Drug Approvals, Online Database And More
33 Wanted: Orphan Drugs, Antivirals Dominate China’s Latest Fast Track List
34 PROBABILITY OF SUCCESS
35 LICENSING AND ASSET ACQUISITION DEALS
35 Deals Shaping The Medical Industry, November 2019
35 Merck Acquires Novel Neurodegenerative Disease Target With Calporta Buy
36 Device/Diagnostics Quarterly Dealmaking Statistics, Q2 2019
36 Mitsubishi Tanabe/Daiichi Sankyo Link For Edaravone In Brazil
37 PARENT PATENTS
38 CLINICAL TRIAL LANDSCAPE
39 Sponsors by status
40 Sponsors by phase
41 Recent events
43 Prescription information
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: AMX0035 for ALS (December 17, 2019): Phase II – CENTAUR
27 Figure 7: MN-166 for ALS (November 8, 2019): Phase IIa – ALS Biomarker
30 Figure 8: IONIS-SOD1Rx for ALS (May 7, 2019): Phase I/II – 233AS101
31 Figure 9: Reldesemtiv for ALS (May 5, 2019): Phase II – FORTITUDE-ALS
32 Figure 10: Key upcoming events in ALS
34 Figure 11: Probability of success in the ALS pipeline
35 Figure 12: Licensing and asset acquisition deals in ALS, 2015–20
37 Figure 13: Parent patents in ALS
38 Figure 14: Clinical trials in ALS
38 Figure 15: Top 10 drugs for clinical trials in ALS
39 Figure 16: Top 10 companies for clinical trials in ALS
39 Figure 17: Trial locations in ALS
40 Figure 18: ALS trials status
41 Figure 19: 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: AMX0035 for ALS (December 17, 2019)
25 Table 6: MN-166 for ALS (November 8, 2019)
28 Table 7: Radicava for ALS (September 17, 2019)
29 Table 8: IONIS-SOD1Rx for ALS (May 7, 2019)
30 Table 9: Reldesemtiv for ALS (May 5, 2019)
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