Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04534842 | Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria | PHASE2 | COMPLETED | 20 | — | — | Aug 25, 2020 | Oct 7, 2022 | Oct 17, 2022 | 11 | United States |
| NCT03516487 | Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria | PHASE1 | COMPLETED | 70 | — | — | Apr 17, 2018 | Jun 21, 2019 | May 13, 2021 | 4 | United States |
The primary efficacy outcome will be assessed by measuring change from baseline in plasma D5-Phe AUC over 24 hours after D5-Phe administration
Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Adverse events (AEs) are reported based on clinical laboratory tests, vital sign and weight measurements, physical examinations, electrocardiograms, and any other medically indicated assessments, including subject interviews, from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if they occur or worsen in severity after the first dose of study treatment. TEAEs are considered treatment-related if relationship to study drug is possibly related, probably related, or definitely related.
| Arm | Type | Description |
|---|---|---|
| SYNB1618 | EXPERIMENTAL | Dose ramp of SYNB1618 |
| SYNB1934 | EXPERIMENTAL | Dose ramp of SYNB1934 |
| SAD HV: SYNB1618 (1 x 10^10 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (1 x 10\^10 colony-forming units \[CFU\]) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD HV: SYNB1618 (5 x 10^10 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (5 x 10\^10 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD HV: SYNB1618 (1 x 10^11 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (1 x 10\^11 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD HV SB: SYNB1618 (1 x 10^11 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (1 x 10\^11 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). On Day 1, subjects in this cohort receive a solid breakfast (SB) that contains approximately the same amount of calories and protein as the meal supplement shake given to subjects in the other SAD cohorts. |
| SAD HV: SYNB1618 (2 x 10^11 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (2 x 10\^11 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD HV: SYNB1618 (5 x 10^11 CFU) | EXPERIMENTAL | HV subjects receive a single oral dose of SYNB1618 (5 x 10\^11 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD HV: Placebo | PLACEBO_COMPARATOR | HV subjects receive a single oral dose of placebo in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD PKU: SYNB1618 (7 x 10^10 CFU) | EXPERIMENTAL | Subjects with PKU receive a single oral dose of SYNB1618 (7 x 10\^10 CFU) in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| SAD PKU: Placebo | PLACEBO_COMPARATOR | HV subjects receive a single oral dose of placebo in a chilled buffered solution on Day 1 in the SAD study (Part 1). |
| MAD HV: SYNB1618 (1 x 10^10 CFU) | EXPERIMENTAL | HV subjects receive oral SYNB1618 (1 x 10\^10 CFU) in a chilled buffered solution 3 times per day (TID) for 7 days in the MAD study (Part 2). |
| MAD HV: SYNB1618 (5 x 10^10 CFU) | EXPERIMENTAL | HV subjects receive oral SYNB1618 (5 x 10\^10 CFU) in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| MAD HV: SYNB1618 (7 x 10^10 CFU) | EXPERIMENTAL | HV subjects receive oral SYNB1618 (7 x 10\^10 CFU) in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| MAD HV: SYNB1618 (1 x 10^11 CFU) | EXPERIMENTAL | HV subjects receive oral SYNB1618 (1 x 10\^11 CFU) in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| MAD HV: Placebo | PLACEBO_COMPARATOR | HV subjects receive oral placebo in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| MAD PKU: SYNB1618 (7 x 10^10 CFU) | EXPERIMENTAL | Subjects with PKU receive oral SYNB1618 (7 x 10\^10 CFU) in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| MAD PKU: Placebo | PLACEBO_COMPARATOR | Subjects with PKU receive oral placebo in a chilled buffered solution TID for 7 days in the MAD study (Part 2). |
| Name | Type | Description |
|---|---|---|
| SYNB1618 | DRUG | 15-day dose-ramp regimen (4 dose levels) of orally administered SYNB1618 |
| SYNB1934 | DRUG | 15-day dose-ramp regimen (4 dose levels) of orally administered SYNB1934 |
| Placebo | DRUG | Subjects receive placebo orally in a chilled buffered solution (100 mL). |
Inclusion Criteria: 1. Age ≥ 18 years. 2. Able and willing to voluntarily complete the informed consent process. 3. Diagnosis of classic PKU based on medical history as assessed by the investigator (e.g., Phe concentration of \>1200 µmol/L at any time, low dietary Phe tolerance, or genetic diagnosi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase, BMN 307 |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923, Sepiapterin |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | — | HMI-102 |