PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS

Inclusion Criteria:

1. Diagnosis of ALS; the diagnosis of ALS defined by revised El Escorial criteria asfollows:

2. Evidence of lower motor neuron (LMN) degeneration by clinical,electrophysiological, or neuropathological examination.

3. Evidence of upper motor neuron (UMN) degeneration by clinical examination.

4. Progressive spread of symptoms or signs within a region or to other regions, asdetermined by clinical examination or the history of disease progression.

5. Absence of electrophysiological, neuroimaging, or pathological evidence ofother diseases that might explain the UMN or LMN degeneration and exclusion ofother causes.

6. Male or female subjects aged 18 to 75 years inclusive.

7. Must provide written informed consent for study-related procedures.

8. Must be capable of completing all study-related procedures, assessments, and visitsin the judgment of Investigator.

9. Disease duration from ALS symptom onset of motor weakness ≤24 months.

10. ALSFRS-R total score ≥38 at screening visit.

11. ALSFRS-R Breathing subscore should be ≥9 at the time of screening.

12. ALSFRS-R Bulbar subscore should be ≥9 at the time of screening.

13. Forced vital capacity >70% of predicted value.

14. PIFR ≥100 L/minute.

15. Must be receiving a stable dose of standard-of-care treatment Riluzole for 4-weeksbefore signing informed consent.

16. Female subjects who are of childbearing potential must agree to use of highlyeffective methods of contraception consistent with local regulations during thestudy, and for 3 months after the study drug administration. Examples include thefollowing, but not limited to:

17. Combined (estrogen and progestogen containing) or progestogen-only hormonalcontraceptives;

18. Intrauterine device or intrauterine hormone-releasing system; OR

19. Post-menopausal status must have experienced their last menstrual periodminimum of 1 year prior to study drug administration; OR

20. Surgically sterilized. Female subject should be willing to not donate eggduring the trial and for 3 months after the last dose of the study drug.

21. Male subjects who are sexually active with a female of childbearing potential mustagree to use highly effective contraception as described above, or a combination of 2 acceptable methods of contraception (e.g., a barrier method along with a femalepartner using a hormonal contraceptive method), in accordance with localregulations, throughout the duration of the study, and for 3 months after the lastdose of the study drug.

(Male subject should be willing to not donate sperm during the trial and for 3 months after the last dose of the study drug.)

Exclusion Criteria:

1. ALSFRS-R score change (decrease) by 2.5 or more points between the screening visitand Day 1 (baseline) score.

2. Bulbar onset ALS (<9 bulbar subscore)

3. Any use of non-invasive ventilation (e.g., continuous positive airway pressure,non-invasive bi-level positive airway pressure or non-invasive volume ventilation)for any portion of the day, or mechanical ventilation via tracheostomy, or on anyform of oxygen supplementation.

4. Any other significant neurological disorder which can interfere with studyassessments, e.g., significant cognitive impairment and/or clinical dementia.

5. Significant psychiatric illness like schizophrenia, bipolar disorder etc. Subjectswith depression can be included, only if the depression has been stable and noepisode of major depression has occurred in the past year.

6. Severe cardiac disease (e.g., QTc>500 ms), Torsade de Pointes, evidence ofsignificant heart failure (New York Heart Association [NYHA] Class 3 or greater,myocardial infarction or unstable angina in the 6 months prior to screening).

7. Any moderate-to-severe pulmonary disease or difficulty taking inhaled drugs.

8. Inability to tolerate the administration of an oral inhaled powder via DPI.

9. Has taken any investigational product within 30 days or 5 half lives of the drug,whichever is longer, prior to dosing.

10. Taking inhaled protein products on a chronic basis (such as insulin, parathyroidhormone, etc).

11. Subjects with a body weight of 32 kg or less, or a body mass index of <17.5 or >35.0at time of screening.

12. Moderate-to-severe liver disease: aspartate aminotransferase (AST), alanineaminotransferase (ALT) >3 times the upper limit of normal; total bilirubin > 1.5 xULN.; subjects with hepatic diseases such as hepatic cirrhosis, hepatic cancer andactive hepatitis.

13. Moderate-to-severe renal disease: creatinine clearance <45 mL/min/1.73 m2 (byCockcroft-Gault calculation).

14. Any clinically significant disorder or laboratory abnormality that, in theInvestigator's opinion, could interfere with the subject's participation in thestudy, place the subject at increased risk, or confound interpretation of the studyresults.

15. Pregnant or breast-feeding females.