Market Spotlight: Amyotrophic Lateral Sclerosis (ALS)

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Report
44 Pages
February 2021
Region: Global
Citeline
ID: 4421383

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

The publisher 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.

OVERVIEWKEY TAKEAWAYS

DISEASE BACKGROUND

Subtypes

TREATMENT

Symptomatic treatments
Ventilatory management
Nutritional management
Disease-modifying therapies

EPIDEMIOLOGYMARKETED DRUGSPIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

NurOwn for ALS (November 17, 2020)
AMX0035 for ALS (September 2, 2020)
Simdax for ALS (July 28, 2020)
AMX0035 for ALS (December 17, 2019)
MN-166 for ALS (November 8, 2019)
Radicava for ALS (September 17, 2019)

KEY UPCOMING EVENTSPROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

Bristol Seeks Novel Targets, Candidates For ALS, FTD
Scribe Inks CRISPR-Based ALS Partnership With Biogen
Novus Acquires Anelixis, ALS Candidate In Stock Swap Transaction
Deal Watch: Merck Gets Rights To Yumanity’s Preclinical ALS, FTLD Candidates

CLINICAL TRIAL LANDSCAPE

Sponsors by status
Sponsors by phase
Recent events

BIBLIOGRAPHY

Prescription information

APPENDIX

LIST OF FIGURES

Figure 1: Trends in incident cases of ALS, 2019–28
Figure 2: Overview of pipeline drugs for ALS in the US
Figure 3: Pipeline drugs for ALS, by company
Figure 4: Pipeline drugs for ALS, by drug type
Figure 5: Pipeline drugs for ALS, by classification
Figure 6: NurOwn for ALS (November 17, 2020): Phase III - BCT-002-US (US/Canada)
Figure 7: Simdax for ALS (July 28, 2020): Phase III - REFALS
Figure 8: AMX0035 for ALS (December 17, 2019): Phase II - CENTAUR
Figure 9: MN-166 for ALS (November 8, 2019): Phase IIa - ALS Biomarker
Figure 10: Key upcoming events in ALS
Figure 11: Probability of success in the ALS pipeline
Figure 12: Clinical trials in ALS
Figure 13: Top 10 drugs for clinical trials in ALS
Figure 14: Top 10 companies for clinical trials in ALS
Figure 15: Trial locations in ALS
Figure 16: ALS trials status
Figure 17: ALS trials sponsors, by phase

LIST OF TABLES

Table 1: Incident cases of ALS, 2019–28
Table 2: Incidence rates of ALS, 2019–28
Table 3: Marketed drugs for ALS
Table 4: Pipeline drugs for ALS in the US
Table 5: NurOwn for ALS (November 17, 2020)
Table 6: AMX0035 for ALS (September 2, 2020)
Table 7: Simdax for ALS (July 28, 2020)
Table 8: AMX0035 for ALS (December 17, 2019)
Table 9: MN-166 for ALS (November 8, 2019)
Table 10: Radicava for ALS (September 17, 2019)

Table of Contents

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