Chimerix to Submit Dordaviprone for Accelerated Approval to U.S. FDA for Patients with Recurrent H3 K27M-Mutant Diffuse Glioma Before Year-End
CMRX 12.09.2024
Drug:H3-K27M dordaviprone
Diseases:H3 K27M-mutant glioma
Date of Upcoming Event:2024-12-10
Name of Upcoming Event:Chimerix Conference Call
About Gravity Analytica
Potential Approval in Q3 2025 in Recurrent H3 K27M-Mutant Diffuse Glioma
Submission Plan Follows Productive and Collaborative Pre-NDA Interactions with FDA
Company to Host Conference Call on
“We expect that, if approved, dordaviprone will fundamentally change the treatment landscape for patients suffering from this lethal form of brain cancer who have extremely limited treatment options. We have worked collaboratively with the
“As a pediatric oncologist, this program is particularly meaningful given the impact a potential approval would have on children and young adults devastated by this disease. We are confident that the data generated to date could support an accelerated approval for this urgent unmet medical need,” said
The following recent program milestones and additional supportive data were extensively discussed with the FDA and will be included in the NDA:
- Substantial enrollment of the Phase 3 ACTION study
- Phase 2 objective response rate of the 50-patient primary efficacy analysis assessed by blinded independent central review (BICR) as the primary basis of efficacy in the NDA
- Several response assessments, including the most contemporary response assessment criteria for gliomas, Response Assessment in Neuro-Oncology 2.0 (RANO 2.0), under which dordaviprone demonstrated an objective response rate of 28%, a median duration of response of 10.4 months and a median time to response of 4.6 months
- Additional clinical data sets and patient narratives supportive of the primary efficacy analysis
- Clinical and nonclinical demonstration of dordaviprone-driven reversal of the central hallmark of H3 K27M-mutant glioma, H3K27 trimethyl loss
- Comprehensive safety database of glioma patients and healthy volunteers that supports a favorable benefit/risk profile
- Comprehensive clinical pharmacology and chemistry, manufacturing, and controls (CMC) studies
Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and is eligible to apply for a Rare Pediatric Disease Priority Review Voucher (PRV). Chimerix intends to apply for a Rare Pediatric Disease PRV in the upcoming NDA submission.
Conference Call and Webcast
About
About DordaviproneDordaviprone (ONC201) is a novel first-in-class small molecule imipridone that selectively targets the mitochondrial protease ClpP and dopamine receptor D2 (DRD2).
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things: the possible regulatory path forward for dordaviprone, including the potential to seek accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers and approval for marketing authorization; timing and consequences of an NDA submission to FDA; FDA’s acceptance for filings; the timeline of related discussions with the FDA; the initial potential PDUFA timing; the potential commercial opportunity; the ability of dordaviprone to attain significant market acceptance among disease experts, patient advocates and their patients; and the expected impact of dordaviprone on patients. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks related to the ability to obtain and maintain accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers, and approval for marketing authorization; uncertainty on the response of regulators to including additional supportive data to be submitted in the NDA filing, including RANO 2.0 assessments, and uncertainty with respect to the initial potential PDUFA timing; risks related to the timing, completion and outcome of the Phase 3 ACTION study of dordaviprone; risks associated with market acceptance; risks associated with repeating positive results obtained in prior preclinical or clinical studies in future studies; risks related to the clinical development of our clinical candidates; and additional risks set forth in the Company’s filings with the
CONTACT:Will O’ConnorStern Investor Relations212-362-1200
Source: Chimerix, Inc.