Jazz Pharmaceuticals Acquires Chimerix for Promising Brain Cancer Drug

Brain cancers are known for being hard to treat, with surgery and radiation being the usual options. However, a rare and aggressive type of brain tumor may soon have its first approved drug. Jazz Pharmaceuticals is investing $935 million to add this potential breakthrough to its collection of therapies.

The drug in question is called dordaviprone, which has been developed by Chimerix, a biotech company based in Durham, North Carolina. The specific cancer it targets is known as H3 K27M-mutant diffuse glioma, a severe form of glioma that primarily impacts children and young adults. Conventional treatments, such as surgery, pose significant risks for these patients as the tumors are located in critical areas of the brain and spinal cord. Similarly, while radiation therapy is an option, it is not curative and carries its own set of complications.

Speaking at TD Cowen's annual healthcare conference, Jazz's Chief Medical Officer, Robert Iannone, emphasized the urgent need for new treatments in this area. He expressed the difficulty he and his medical colleagues face when they can only offer radiation and surgery to patients suffering from such a challenging condition.

According to estimates by Chimerix, approximately 2,000 patients in the U.S. are diagnosed with H3 K27M-mutant gliomas each year. Dordaviprone belongs to a new class of cancer medications known as imipridones. These drugs, which are small molecules, work by targeting specific G protein-coupled receptors and enzyme targets to lead to the destruction of cancer cells. Dordaviprone, which was previously known as ONC201, is designed to specifically target mitochondrial protease ClpP and dopamine receptor D2.

The Phase 2 clinical trial results for dordaviprone have shown promise, indicating that the drug may cause tumor shrinkage in patients with glioblastoma carrying the H3 K27M mutation. Among the 50 patients analyzed for its effectiveness, there was a reported 28% objective response rate, with an average duration of response lasting 10.4 months. Additionally, a Phase 3 trial is underway that will enroll newly diagnosed patients with this brain cancer type to pave the way for its first-line use; interim results are expected by the third quarter of 2025. Based on the encouraging Phase 2 data, Chimerix is actively pursuing accelerated approval for dordaviprone to treat recurrent H3 K27M-mutant diffuse glioma. The FDA has accepted the application for priority review, with a decision expected by August 18.

While cancer treatment is the focus of Chimerix, it is a relatively new venture for the company, which has a long history within North Carolina's Research Triangle. The development of dordaviprone was part of Chimerix's acquisition of Oncoceutics in 2021, which primarily offered research into imipridones. The acquisition also introduced ONC206, another imipridone currently in Phase 1 testing for central nervous system tumors. Following the Oncoceutics deal, Chimerix agreed to terms that could yield up to $360 million in milestone payments plus royalties from sales for its shareholders.

Chimerix was founded in 2000 and started as a developer of antiviral medications. It originally centered its efforts on brincidofovir, aimed at treating cytomegalovirus infections. While that attempt did not succeed, the compound received FDA approval in 2021 as a treatment for smallpox and is marketed under the name Tembexa. In 2022, Chimerix sold the rights to Tembexa for $225 million upfront and potential additional milestones of $100 million, using the funds to further develop dordaviprone.

In the past year, Chimerix's stock has primarily traded below $1. However, the announcement in December for its plans to seek accelerated approval for dordaviprone caused a notable rise in share price. According to the terms of the recent acquisition, Dublin-based Jazz will pay $8.55 in cash for each Chimerix share, a 72% premium over the closing stock price the day prior. There is also room for future financial gain due to the prospect of obtaining a pediatric drug priority review voucher if dordaviprone is approved. Such vouchers can be valuable, historically selling in the range of $100 million to $150 million.

Jazz currently has five cancer treatments in its portfolio and views dordaviprone as an excellent addition to its oncology efforts. The Chief Financial Officer, Philip Johnson, expressed that the drug can benefit from Jazz’s existing commercial resources and has extended patent protection that could last until 2037 or beyond.

The acquisition aligns with Jazz's strategy to pursue assets that address rare medical needs in niche markets, according to Marc Goodman, an analyst at Leerink Partners. In addition to meeting a significant unmet need, this acquisition draws on Jazz's expertise in rare diseases and oncology. Goodman noted that Jazz prefers to acquire late-stage assets poised for near-term market introduction, mentioning that due to the small patient pool for this specific cancer type, dordaviprone might be priced between $300,000 and $800,000 for patients.

Both companies have approved the acquisition, which awaits customary closing procedures, such as acquiring a majority of Chimerix's outstanding shares. The deal is expected to finalize in the second quarter of this year.

Cancer, Pharmaceutical, Acquisition