The market for drugs to treat amyotrophic lateral sclerosis (ALS) in major markets is expected to surge from US$317M in 2019 to US$1.28B in 2029, at a CAGR of 15%.
It is understood that this increase is caused by a variety of factors A new report from GlobalData. Chief among these, according to interviews with key opinion leaders, is the need for disease-modifying therapies and the need to diagnose disease earlier, which will not only increase the number of people treated but also improve outcomes.
Momna Ali, Healthcare Analyst at GlobalData, noted: “Early intervention can extend treatment duration, further increase revenue, and ultimately improve patient outcomes, thereby enhancing product reputation and driving demand and market share.”
Currently, there are only six treatments for ALS, also known as Lou Gehrig’s disease. The latest approved is Biogen’s (NASDAQ:BIIB) Qalsody (tofersen), approved in 2023.Nasdaq: IONS), bringing in $5.9 million in revenue in 2023.
In April, Amylyx Pharmaceuticals (AMLX) said it would withdraw its drug Relyvrio (sodium phenylbutyrate and taurodiol) from the market after Phase 3 trial results failed to benefit patients, causing a major hit to the treatment market. frustrated.
However, there are several candidates in the pipeline that show promise. Ali identified Ulefnathan of Ioannis as one of them. The drug candidate is an antisense drug, currently in phase III, that works by reducing the production of the sarcoma fusion protein (FUS).
Biogen collaborated with Ionis to develop BIIB105, an antisense oligonucleotide in Phase 2 development. Regulator, TDP-43 pathology is an important pathology in approximately 95% of ALS cases. Preclinical studies show that by reducing ATXN2, it improves patient function and potentially slows disease progression and improves survival.
Partnering with privately held Calico Life Sciences, AbbVie (NYSE:ABBV) owns ABBV-CLS-7262, an eIF2B activator in Phase 2 development.
Denali Therapeutics, Inc. (NASDAQ:DNLI) has DNL243 in late-stage clinical development. Like ABBV-CLS-7262, it also targets eF2B.
Utreloxastat (PTC-857), in Phase 2 development by PTC Therapeutics (PTCT), inhibits 15-lipoxygenase, an enzyme that mediates oxidative stress and ferroptosis (ie, iron-dependent cell death). Interim data from the CardinALS study are expected in the fourth quarter.
