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Ataluren+ Pembrolizumab · 1 trial · 2 indications
To characterize toxicities and side effects of Ataluren when combined with pembrolizumab in patients with pMMR CRC, dMMR mCRC and dMMR EC. Recorded on Adverse Events form and ranking adverse event severity according to the NCI Common Terminology Criteria for Adverse Events v3.0
Measured by immune-related response criteria
| Arm | Type | Description |
|---|---|---|
| Phase I dMMR and pMMR | EXPERIMENTAL | 2-4 groups of 3 patients treatment with 200mg i.v. Pembrolizumab q3w and dose escalation of Ataluren in order to determine the Ataluren MTD. These patients can either be pMMR/dMMR CRC and dMMR EC patients. |
| Phase II dMMR | EXPERIMENTAL | Mismatch repair deficient CRC or EC patients treated with 200mg i.v. pembrolizumab q3w and Ataluren at MTD. |
| Phase II pMMR | EXPERIMENTAL | Mismatch repair proficient CRC patients treated with 200mg i.v. pembrolizumab q3w and Ataluren at MTD. |
| Name | Type | Description |
|---|---|---|
| Ataluren + Pembrolizumab | DRUG | Ataluren and Pembrolizumab combination therapy |
In order to be eligible for participation in this trial, the subject must: * Have at least one lesion with measurable disease as defined by 10mm in longest diameter for a soft tissue lesions or 15mm in short axis for a lymph node by RECIST 1.1 and irRC criteria for response assessment. * Have recei...
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Ataluren is an investigational small molecule being studied for Duchenne muscular dystrophy (DMD), specifically nonsense mutation Duchenne muscular dystrophy, as well as aniridia, colorectal cancer, and endometrial cancer. It is being developed by PTC Therapeutics, Inc. (NASDAQ: PTCT) and is currently in Phase 2 clinical development.
Ataluren is designed to enable the read-through of premature stop codons in messenger RNA, allowing the production of full-length, functional proteins in patients with nonsense mutations. This mechanism is relevant for genetic disorders like Duchenne muscular dystrophy caused by nonsense mutations, though the specific molecular target has not been disclosed.
Ataluren is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PTCT. The company is conducting clinical trials of Ataluren across multiple indications, including Duchenne muscular dystrophy and certain cancers.
Ataluren is in Phase 2 clinical development. It has completed Phase 3 trials for nonsense mutation Duchenne muscular dystrophy, and a Phase 1 trial combining Ataluren with pembrolizumab is currently recruiting patients with metastatic colorectal or endometrial cancer. Ataluren remains investigational and is not FDA approved.
Ataluren has been studied in several clinical trials, including NCT01826487, a completed Phase 3 study in nonsense mutation Duchenne muscular dystrophy with 230 participants, and NCT03179631, a completed Phase 3 long-term outcomes study with 360 participants. An active Phase 1 trial, NCT04014530, is evaluating Ataluren with pembrolizumab in colorectal and endometrial cancers.
Ataluren plus Pembrolizumab is a combination regimen being studied in clinical trials, not a separate drug name. The combination is being evaluated in the ATAPEMBRO study (NCT04014530) for metastatic colorectal and endometrial cancers, where Ataluren is administered alongside the immunotherapy pembrolizumab.