Oral. Scalable. Precise. Designed for chronic conditions, where access and patient convenience matter most.
THE SCIENCE
Small molecules offer both practical and scientific advantages; they’re oral, scalable, and cost-effective, while also achieving tissue penetration and precise targeting. These strengths make them especially well-suited for chronic, multisystemic rare diseases where access, convenience, and long-term patient care are essential.
Patient-Friendly Administration
Most small molecules can be formulated as convenient oral pills or capsules, offering a comfortable and patient-friendly option for long-term use.
Broad Tissue Penetration
Small molecules can often cross biological barriers. This is crucial since many rare diseases are neurological or metabolic, affecting multiple organs.
Small molecules are easier and less expensive to manufacture, with shorter production times, which makes therapies more accessible for patient groups.
Manufacturing & Access
Regulatory & Clinical Advantages
Small molecules benefit from well-established regulatory pathways that offer a clear framework for development and evaluation. When supported by robust nonclinical data to guide study design, this structure enables more efficient development timelines.
LEAD PROGRAM
Hypophosphatasia (HPP)
HPP patients lack alkaline phosphate enzyme, which leads to PPI buildup and affects multiple processes. PPi accumulation also occurs outside of bone, with deleterious consequences resulting in a spectrum of disease.
​ALE1 is an oral small molecule designed to inhibit a novel target that regulates levels of PPi, the metabolite at the center of HPP pathology. By lowering excess PPi, ALE1 aims to restore healthier bone and mineral metabolism across the full spectrum of HPP.
Preclinical studies have shown that ALE1 consistently reduces PPi levels across multiple animal models following oral dosing, supporting its potential as a first-in-class, conveniently administered therapy with broad applicability.
THE IMPACT ON HPP
ALE1 has the potential to address the entire spectrum of HPP and fundamentally transform patient care across this underserved disease.
Less invasive procedures enhance patient comfort and increase treatment adherence. While enzyme replacement therapies offer life-saving benefits for infants and children with HPP, the need for frequent and often painful injections places a significant burden on patients and families, affecting comfort, emotional well-being, and quality of life. Over time, the treatment routine may become overwhelming, especially in chronic lifelong conditions.
Addresing the full spectrum of HPP
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With enzyme replacement therapies not approved for adults, a large segment of the HPP community remains without a sustainable long-term treatment option.
Remove the cost barrier
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Access to Enzyme replacement therapies can be limited due to factors such as cost, insurance coverage, and regional healthcare policies.
Long-term impact
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As effective or potentially more effective than current treatments due to the simple mechanism of delivery and patient access
PATIENTS FOCUSED