Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Apilimod Dimesylate Capsule, LAM-002A
Apilimod Dimesylate · 3 trials · 4 indications
Number of Participants with Treatment-Related Adverse Events (TEAEs) and Serious Adverse Events
The percentage of participants who complete 12 weeks on study treatment during the Core Study.
The concentration of LAM-002A and its major metabolites (STA-5908, STA-5944, and STA-6048) in plasma.
The concentration of LAM-002A and its major metabolites (STA-5908, STA-5944, and STA-6048) cerebral spinal fluid (CSF).
The primary efficacy outcome measure evaluated change in SARS-CoV-2 viral load at Day 4 from Day 1, of LAM-002A or placebo-treated participants. SARS-CoV-2 viral load was measured by a real-time quantitative reverse transcription polymerase chain reaction (qRT-PCR) test of nasopharyngeal samples. Analysis focused on log10 viral load on Day 4 compared to baseline viral load at Day 1 in participants with baseline viral load \>100,000 copies/mL
MTD was determined by testing increasing doses up to 125 mg twice a day or 75 mg three times a day orally on dose escalation cohorts with 3 to 6 participants each. In the dose escalation, the cohort sizes of 3 to 6 subjects allow evaluation of regimen safety using a standard definition of MTD (ie, the highest starting dose associated with DLT in \<33% of subjects during the first cycle of therapy) when administered continuously (daily administration) and then when administered intermittently (repeated courses of 3 days on and 4 days off).
| Arm | Type | Description |
|---|---|---|
| LAM-002A | EXPERIMENTAL | LAM-002A will be administered orally in five 25 mg capsules twice a day (250 mg total daily dose). |
| Placebo | PLACEBO_COMPARATOR | Placebo matching LAM-002A will be administered orally in 5 capsules twice a day. |
| Continuous monotherapy | EXPERIMENTAL | All patients will take LAM-002A two times daily by mouth every day until cancer progression or intolerability. |
| Intermittent monotherapy | EXPERIMENTAL | All patients will receive LAM-002A at escalating dose levels two times daily by mouth for 3 days on therapy followed by 4 days off therapy every week until cancer progression or intolerability. |
| LAM-002A + rituximab | EXPERIMENTAL | All patients will receive LAM-002A 125mg two times daily by mouth every day until cancer progression or intolerability and rituximab 375 mg/m2 by vein every week for 4 weeks and then every 8 weeks for 4 times (total of 8 infusions) |
| LAM-002A + atezolizumab | EXPERIMENTAL | All patients will receive LAM-002A 125mg two times daily by mouth every day until cancer progression or intolerability and atezolizumab 1200 mg by vein every 3 weeks until cancer progression or intolerability |
| Name | Type | Description |
|---|---|---|
| LAM-002A | DRUG | LAM-002A (apilimod dimesylate) is formulated in capsules containing 25 mg of apilimod dimesylate. The capsule is Swedish orange, size 0. |
| Placebo | OTHER | Microcrystalline cellulose in Swedish orange, size 0 capsules. |
| Apilimod Dimesylate Capsule | DRUG | LAM-002A is formulated in capsules containing 25 mg of apilimod dimesylate. The capsule is Swedish orange, Size 0. |
| Rituximab | DRUG | 375 mg/m2 by vein |
| Atezolizumab | DRUG | 1200 mg by vein |
Inclusion Criteria: 1. Diagnosis of C9ORF72-associated ALS with BOTH of the following: 1. Documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72. If there is a strong clinical suspicion for C9ALS based on C9-positive family history and El...