Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

This Phase 3b extension study will assess the long-term safety of Ace-ER in patients who
participated in and completed study UX001-CL301 (NCT02377921), study UX001-CL202
(NCT01830972), and study UX001-CL203 (NCT02731690).

Inclusion Criteria:

- Have completed UX001-CL202, UX001-CL301 or UX001-CL203 study

- Willing and able to provide written, signed informed consent after the nature of the
study has been explained, and before any research-related procedures are conducted

- Willing to comply with all study procedures

- Female participants of child‐bearing potential or male participants with female
partners of child-bearing potential who have not undergone a bilateral
salpingo‐oophorectomy and are sexually active must consent to use a highly effective
method of contraception as determined by the site investigator (i.e., oral hormonal
contraceptives, patch hormonal contraceptives, vaginal ring, intrauterine device,
physical double-barrier methods, surgical hysterectomy, vasectomy, tubal ligation or
true abstinence [when this is in line with the preferred and usual lifestyle of the
subject], which means not having sex because the subject chooses not to), from the
period following the signing of the informed consent through 30 days after last dose
of study drug

- Females of childbearing potential must have a negative pregnancy test at Screening and
be willing to have additional pregnancy tests during the study. Females considered not
of childbearing potential include those who have been in menopause for at least two
years, have had tubal ligation at least one year prior to Screening, or who have had a
total hysterectomy or bilateral salpingo oophorectomy

Exclusion Criteria:

Individuals who meet any of the following exclusion criteria will not be eligible to
participate in the study:

- Ingestion of N-acetyl-D-mannosamine (ManNAc) or related metabolites; intravenous
immunoglobulin (IVIG); or anything that can be metabolized to produce SA in the body
within 60 days prior to the Screening Visit

- Has had any hypersensitivity to sialic acid (SA) or its excipients that, in the
judgment of the investigator, places the subject at increased risk for adverse effects

- Pregnant or breastfeeding at Screening or planning to become pregnant (self or
partner) at any time during the study

- Use of any investigational product or investigational medical device within 30 days
prior to Screening, or anticipated requirement for any investigational agent prior to
completion of all scheduled study assessments

- Has a condition of such severity and acuity, in the opinion of the investigator, that
it warrants immediate surgical intervention or other treatment or may not allow safe
participation in the study

- Has a concurrent disease, active suicidal ideation, or other condition that, in the
view of the investigator, places the subject at high risk of poor treatment compliance
or of not completing the study, or would interfere with study participation or would
affect safety