Chimerix, Inc. (NASDAQ:CMRX) is a biopharmaceutical company focused on developing innovative medicines to improve and extend the lives of patients facing deadly diseases. The company's lead clinical candidate, dordaviprone (ONC201), is currently in a pivotal Phase 3 study for the treatment of H3 K27M-mutant diffuse glioma, a rare and aggressive form of brain cancer with a significant unmet medical need.

In the first quarter of 2024, Chimerix continued to make strong progress on the execution of its strategic priorities, highlighted by the ongoing advancement of the dordaviprone Phase 3 ACTION study and the emergence of promising regulatory pathways for potential accelerated approval.

Financials

For the first quarter of 2024, Chimerix reported a net loss of $21.9 million, or $0.25 per basic and diluted share, compared to a net loss of $21.4 million, or $0.24 per basic and diluted share, in the same period of 2023. The company's research and development expenses remained relatively flat at $18.8 million, while general and administrative expenses decreased to $5.5 million from $5.7 million in the prior year period.

Chimerix ended the first quarter of 2024 with $188.2 million in capital available to fund operations, providing a strong financial foundation to advance its pipeline. The company's rolling 4-quarter burn rate of $58 million positions it among the most capital-efficient Phase 3 companies in its peer group, and Chimerix continues to expect its cash balance to be sufficient to support operations into the fourth quarter of 2026.

Dordaviprone Phase 3 ACTION Study Progresses

The pivotal Phase 3 ACTION study evaluating dordaviprone for the treatment of H3 K27M-mutant diffuse glioma remains on track, with the first interim overall survival (OS) and progression-free survival (PFS) analyses expected in 2025. The study is currently enrolling patients at over 135 sites across 13 countries in North America, Europe, the UK, Israel, Australia, and Asia.

Chimerix is pleased with the steady state accrual being achieved in the ACTION study and remains confident in its ability to reach the targeted enrollment of 450 patients. The primary endpoints of the study are OS and PFS, with key secondary endpoints including corticosteroid response, performance status response, quality of life assessments, and neurologic function.

Accelerated Regulatory Pathways Emerging

In addition to the ongoing pivotal Phase 3 study, Chimerix is actively exploring accelerated regulatory pathways for dordaviprone, particularly in regions with high unmet need and supportive regulatory frameworks.

The company recently engaged with the Therapeutic Goods Administration (TGA) in Australia, where the TGA agreed that dordaviprone meets the criteria to advance to the second step of the Provisional Registration process. This pathway is designed to provide accelerated approval for medicines that address significant unmet medical needs, and the TGA's positive assessment of dordaviprone's data package and the status of the ACTION study was an encouraging validation of the program.

Chimerix plans to submit the Provisional Determination application in the coming months, which will initiate a 20-working day review process. If successful, the company would then submit an application for Provisional Registration, which is expected to take approximately 1 year. Chimerix anticipates a potential filing around the end of 2024, with possible commercial availability in Australia as early as 2026.

This engagement with the TGA is emblematic of Chimerix's broader strategy to accelerate global access to dordaviprone, leveraging regulatory pathways that can provide earlier approval in regions with significant unmet need. The company remains focused on the successful execution of the pivotal Phase 3 ACTION study, which will serve as the primary basis for regulatory submissions worldwide, while also exploring complementary accelerated approval opportunities.

ONC206 Development Advancing

In addition to the progress with dordaviprone, Chimerix is also advancing its second-generation imipridone compound, ONC206, which is currently in Phase 1 dose escalation trials in patients with advanced central nervous system (CNS) tumors.

The ONC206 program has demonstrated an encouraging safety profile to date, with no dose-limiting toxicities identified. Chimerix is now evaluating a more frequent dosing schedule to increase the duration of therapeutic exposure, and the company expects to report preliminary safety and pharmacokinetic data in mid-2024.

Parallel to the ongoing clinical work, Chimerix is also conducting preclinical studies to identify and evaluate potential oncology indications and predictive biomarkers for ONC206. The company plans to provide an update on the development strategy for this program before the end of the year, as it nears a decision on advancing ONC206 into Phase 2 studies.

Robust Financial Position to Support Continued Advancement

Chimerix's strong financial position, with $188.2 million in capital available to fund operations as of March 31, 2024, provides the company with the resources necessary to continue advancing its pipeline of innovative oncology therapies.

The company's disciplined financial management, with a rolling 4-quarter burn rate of $58 million, positions Chimerix among the most capital-efficient Phase 3 companies in its peer group. This financial strength, combined with the company's strategic focus and execution, gives Chimerix confidence in its ability to achieve its key milestones and deliver value for shareholders.

Significant Unmet Need in H3 K27M-Mutant Glioma

H3 K27M-mutant diffuse glioma is a rare and aggressive form of brain cancer with a significant unmet medical need. Patients diagnosed with this disease typically have a median overall survival of less than 1 year, and there are currently no approved treatment options that have demonstrated a clinical benefit beyond standard radiation therapy.

The high unmet need in this patient population, coupled with the promising data generated with dordaviprone in earlier studies, has driven Chimerix's focus on accelerating the development and potential approval of this investigational therapy. The company's engagement with the TGA in Australia, as well as its ongoing discussions with other regulatory agencies, underscores the recognition of the urgent need for new treatment options for patients with H3 K27M-mutant glioma.

Chimerix's commitment to this patient population is further evidenced by the continued execution of the pivotal Phase 3 ACTION study, which the company believes represents the best path to a definitive assessment of dordaviprone's safety and efficacy in the frontline setting. The company's parallel efforts to explore accelerated approval pathways in regions with supportive regulatory frameworks demonstrate its multi-pronged approach to bringing this potentially transformative therapy to patients as quickly as possible.

Conclusion

Chimerix's first quarter of 2024 was marked by significant progress across its key strategic initiatives, highlighted by the continued advancement of the dordaviprone Phase 3 ACTION study and the emergence of promising regulatory pathways for potential accelerated approval.

The company's financial strength, with $188.2 million in capital available to fund operations, provides a solid foundation to support the continued development of dordaviprone and its earlier-stage pipeline candidate, ONC206. Chimerix's disciplined financial management and capital-efficient approach position the company well to execute on its strategic priorities and deliver value for shareholders.

As Chimerix navigates the regulatory landscape and works to bring dordaviprone to patients with H3 K27M-mutant glioma, a disease with significant unmet need, the company remains committed to its mission of developing innovative medicines that can meaningfully improve and extend the lives of those facing deadly diseases.