BBP-418 for Limb-Girdle Muscular Dystrophy

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: ML Bio Solutions, Inc.

Must not be taking: Corticosteroids, Ribose, Sugar alcohols, others

Disqualifiers: Cardiac, Pulmonary, Renal, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 90 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called BBP-418 for individuals with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), a condition that weakens muscles around the hips and shoulders. The trial aims to assess the safety and tolerability of BBP-418 for both ambulatory and non-ambulatory participants. Different doses will be administered to determine the optimal option. Individuals with a confirmed LGMD2I diagnosis who experience muscle weakness and can or cannot complete a short walk within a specific time may be suitable candidates for this trial. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that medication for common and mild conditions may be allowed after consulting with the principal investigator. You cannot use ribose, sugar alcohol supplements, or corticosteroids within 60 days before starting the trial.

Is there any evidence suggesting that BBP-418 is likely to be safe for humans?

Research has shown that BBP-418 is well-tolerated in earlier studies. In a current study with 14 participants, BBP-418 was taken for over 21 months without major problems. Most participants did not experience serious side effects, suggesting it is generally safe. The study found that the treatment helped maintain stability in certain health measures, which is encouraging. While no treatment is without risk, BBP-418 has demonstrated a promising safety record so far for individuals with Limb-Girdle Muscular Dystrophy Type 2I. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Limb-Girdle Muscular Dystrophy?

Researchers are excited about BBP-418 for Limb-Girdle Muscular Dystrophy (LGMD) because it introduces a potential new approach to treatment. Unlike current options that mainly focus on managing symptoms, BBP-418 targets the underlying cause of the muscle weakness by aiming to restore muscle function directly. This treatment involves a different active ingredient and is administered in varying doses, which might optimize its effectiveness and safety profile. This innovative mechanism of action could pave the way for more effective management of LGMD, offering hope for improved quality of life for patients.

What evidence suggests that BBP-418 might be an effective treatment for Limb-Girdle Muscular Dystrophy Type 2I?

Research has shown that BBP-418 may help treat Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). In studies, patients who took BBP-418 showed noticeable improvements in important health measures after 12 months. Early findings also suggest that BBP-418 lowers CK levels, which indicate muscle damage. Additionally, BBP-418 enhances muscle function, such as walking and movement speed. Overall, BBP-418 could become the first oral treatment to change the course of the disease, potentially offering real benefits to those with LGMD2I. Participants in this trial will receive different dosages of BBP-418, as outlined in the various treatment cohorts. ^{1 2 5 6 7}

Who Is on the Research Team?

Amy Harper, MD

Principal Investigator

Virginia Commonwealth University

Are You a Good Fit for This Trial?

This trial is for individuals over 30 kg with genetically confirmed LGMD2I, showing clinical weakness. Participants must be able to walk 10 meters unaided in ≤12 seconds or be non-ambulatory. They should agree to contraception use and not have significant other diseases or recent drug abuse history.

Inclusion Criteria

Previous enrolment in the Natural History study MLB-01-001

Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose

I have a genetic diagnosis of LGMD2I and show signs of muscle weakness.

Exclusion Criteria

Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the investigator's opinion, is likely to unfavorably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance

I have a serious health condition alongside my cancer.

I have not been on any experimental treatments or devices for the last 60 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BBP-418 with ascending dose levels to determine safety and tolerability

90 days

Dose Escalation

Participants continue receiving BBP-418 at 12 grams BID until study completion

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BBP-418

Trial Overview BBP-418 is being tested for safety and tolerability in different dose levels on ambulatory and non-ambulatory patients with LGMD2I, a condition without approved treatments. This open label study allows all participants to receive the drug.

How Is the Trial Designed?

Group I: Cohort 3 Experimental Treatment 1 Intervention

Subjects will receive 12 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.

Group II: Cohort 2 Experimental Treatment 1 Intervention

Subjects will receive 6 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.

Group III: Cohort 1 Experimental Treatment 1 Intervention

Subjects will receive 6 grams of BBP-418 once daily x 90 days, then 12 grams twice daily (BID, a least 8 hours apart) of BBP-418 daily until study completion.

Who Is Running the Clinical Trial?

ML Bio Solutions, Inc.

Lead Sponsor

Trials

3

Recruited

200+

Citations

1. investor.bridgebio.com investor.bridgebio.com/news/news-details/2025/BridgeBio-Reports-Positive-Phase-3-Results-for-Small-Molecule-BBP-418-in-LGMD2IR9-FORTIFY-Study/default.aspx

BridgeBio Reports Positive Phase 3 Results for Small ... - BBP-418 treated individuals had statistically significant, clinically meaningful improvements at 12 months in all key clinical endpoints ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05775848

Study to Evaluate the Efficacy and Safety of BBP-418 ... This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9.

3. mdaconference.org mdaconference.org/abstract-library/preliminary-results-from-mlb-01-003-an-open-label-phase-2-study-of-bbp-418-in-patients-with-limb-girdle-muscular-dystrophy-type-2i/

An Open Label Phase 2 Study of BBP-418 in Patients ... Preliminary MLB-01-003 data from patients with LGMD2i treated with BBP-418 suggests a positive effect on CK, a widely used biomarker of muscle injury.

4. bridgebio.com bridgebio.com/lgmd2i-r9/bbp-418/

BBP-418 | Glycosylation Substrate Pro-Drug For LGMD2I/R9 If approved, BBP-418 has the potential to be the first disease-modifying oral therapy available for people with LGMD2I/R9. Learn more today.

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896623006685

an open label phase 2 study of BBP-418 in patients with ... Preliminary MLB-01-003 data from patients with LGMD2I suggest a positive effect of BBP-418 on levels of glycosylated αDG, CK, NSAD, 100MTT, and 10MWT velocity.

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896624003390

a phase 3 study to evaluate efficacy & safety of BBP-418 in ... The FORTIFY study will assess the safety, tolerability, and efficacy of BBP-418 in LGMD2I/R9. In addition to evaluating the effect of BBP-418 on motor ...

7. mdaconference.org mdaconference.org/abstract-library/fortify-a-phase-3-study-to-evaluate-efficacy-safety-of-bbp-418-in-individuals-with-limb-girdle-muscular-dystrophy-2i-lgmd2i-lgmdr9-fkrp-related/

A Phase 3 Study to Evaluate Efficacy & Safety of BBP-418 ... In an ongoing open-label Phase 2 study in 14 individuals with LGMD2I/R9, BBP-418 was well-tolerated. Following 21+ months of dosing, stabilization in measures ...