OUR SCIENCE

Amyotrophic Lateral Sclerosis (ALS)

ALS is a progressive motor neuron disease characterized by muscle weakness, paralysis, and respiratory failure. Most patients survive for 2–5 years after diagnosis. Existing treatments offer limited clinical benefit, and there is a significant unmet need. PhenoNet is developing treatments to slow disease progression and improve clinical outcomes.

Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder that severely impacts motor neuron function, leading to muscle weakness, loss of movement, and diminished physical abilities. These changes significantly compromise quality of life and life expectancy. Current treatment options provide only limited benefit, leaving a critical gap in effective care and support for those affected by ALS. PhenoNet’s mission is to close this gap by developing innovative therapies designed to slow disease progression and improve clinical outcomes for patients worldwide.

*Reference: ALS Association; https://www.als.org/understanding-als/what-is-als

Most Prevalent 
Motor Neuron Disease

Our current clinical study targets early-stage patients with defined progression profiles, representing a more precise and promising approach to ALS drug development.


Roughly 90% of ALS cases are sporadic (sALS), with no known family history. The remaining 10% are familial ALS (fALS), usually inherited in an autosomal dominant pattern. ALS affects individuals of all ethnicities and races, though studies show a higher prevalence among Caucasians.


PHENOGENE-1A in Alzheimer Disease:


Mechanism of Action

Targets Aβ - amyloid monomer aggregation

Modulates neuroinflammatory pathways

Enhances synaptic and neurite function

Potential benefit in APOE4-negative gene carrier subgroups.

Most Prevalent Motor Neuron Disease

Our current clinical study targets early-stage patients with defined progression profiles, representing a more precise and promising approach to ALS drug development.


Roughly 90% of ALS cases are sporadic (sALS), with no known family history. The remaining 10% are familial ALS (fALS), usually inherited in an autosomal dominant pattern. ALS affects individuals of all ethnicities and races, though studies show a higher prevalence among Caucasians.

PHENOGENE-1A in Amyotrophic Lateral Sclerosis:

PHENOGENE-1A, an investigational therapy containing cromolyn as its active ingredient, is being developed as an adjunct treatment for mild to moderate amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease with limited effective treatments. Cromolyn, a mast cell stabilizer with a long-established safety profile, has been used since the 1970s to treat asthma and related conditions.

Emerging evidence supports the use of cromolyn in ALS due to its anti-inflammatory and neuroprotective properties. Preclinical studies suggest that cromolyn modulates neuroinflammation, inhibits cytokines and chemokines, promotes neurite outgrowth, and suppresses fibrosis (Elmaleh et al., 2019; Wang et al., 2021a; Wang et al., 2021b). In addition to its mast cell-stabilizing properties, cromolyn has been shown to prevent degranulation of mast cells, modulate ion transport by affecting chloride and calcium channels (Franzius et al., 1994; Romanin et al., 1991), and inhibit the production of inflammatory mediators associated with neurodegeneration.

By Dr Jacqueline Gosink, EUROIMMUN AG, Luebeck, Germany

Alzheimer's Disease 
(AD)

Alzheimer's Disease (AD) Recent advances in AD research have highlighted two convergent biological pathways—amyloid-beta (Aβ) aggregation and neuroinflammation—that play central roles in early disease pathophysiology. Cromolyn directly interacts with Aβ peptides to inhibit their polymerization into neurotoxic oligomers and fibrils, while also enhancing clearance mechanisms. In parallel, it modulates immune responses by stabilizing mast cells, inhibiting proinflammatory cytokines and altering microglial activation, thereby reducing chronic neuroinflammatory signaling that exacerbates neurodegeneration. Together, these mechanisms provide a dual anti-amyloid and anti-inflammatory approach, aligning PHENOGENE-1A with current precision medicine strategies that aim to target upstream drivers of AD progression.

PHENOGENE-1A in Alzheimer Disease:

PHENOGENE-1A

Unique Properties


Targets PI3Kdelta a regulator of immune cell function



  • Inhibits pro-inflammatory cytokine production
  • Increase protective cytokines
  • Shifts microglia into neuroprotective, phagocytic state
  • Targets production of neurotoxic peptides by inhibiting the accumulation and oligomerization of Aβ dimers and trimers in nM concentration
  • Low dose allows chronic administration with minimal side effects


Mechanism of Action


  • Targets Aβ - amyloid monomer aggregation
  • Modulates neuroinflammatory pathways
  • Enhances synaptic and neurite function
  • Potential benefit in APOE4-negative gene carrier subgroups.


This precision-medicine strategy may finally overcome the limitations of past trials by reducing patient heterogeneity and targeting upstream molecular mechanisms.


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