Pfizer Seeks Patent Covering a Friedreich’s Ataxia Therapy That Resverlogix Has Already Developed

Magdalena Kegel avatar

by Magdalena Kegel |

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Friedreich’s ataxia patent

Pfizer is seeking a European patent on a family of compounds that can treat Friedreich’s ataxia by increasing levels of the frataxin protein that is lacking in the disease.

It noted in its patent application that Resverlogix has already developed a therapy called apabetalone (RVX-208) that is based on the compounds. Pfizer indicated that it wants the patent to cover all such compounds, including therapies based on them that other companies have already developed, such as apabetalone.

The patent asks that New York City-based Pfizer have the exclusive right to use the BET family of bromodomain inhibitors to increase frataxin in Friedreich’s ataxia patients. BET stands for bromodomain and extra-terminal. Apabetalone is a BET compound.

Resverlogix, which is based in Calgary, Canada, responded to news of the patent application in an understated way. Instead of indicating it would challenge the patent request, the company’s president and chief executive officer, Donald McCaffrey, said that “we welcome the attention drawn to Resverlogix and apabetalone from significant industry groups such as Nature Reviews Nephrology and Pfizer.”

“Due to the dramatic growth of BET Bromodomain [scientific journal] publications over the past decade, it is not surprising that our advanced Phase 3 compound, apabetalone, is now drawing expanded attention from the global academic and pharmaceutical communities,” McCaffrey added in a press release.

The Pfizer patent request, titled “Regulators of Frataxin” (WO 2017/037567 A1), includes the use of BET-family bromodomain inhibitors to boost the expression of frataxin. Expression is the process by which information from a gene is used to create a functional product like a protein.

Although the patent covers BET bromodomain inhibitors as a group, it specifically lists apabetalone as a compound that could be effective in treating Friedreich’s ataxia.

BET Bromodomain inhibitors can alter gene activity by impacting what are called epigenetic mechanisms. Those mechanisms are modifications to DNA that turn genes on or off. An example would be adding a chemical flag to a DNA molecule that would let the protein-making machinery know if a gene were available for protein production, or not.

These mechanisms, Resverlogix said, could be used to treat several diseases, including chronic kidney disease and Alzheimer’s.

Apabetalone is the first of this type of compound to reach the clinical trial stage.

A Phase 3 clinical trial (NCT02586155) is evaluating its ability to help patients with high-risk type 2 diabetes who also have coronary artery heart disease. And a Phase 2a trial (NCT03160430) is looking at its ability to benefit patients with end-stage kidney disease who are on dialysis.

Meanwhile, Resverlogix said a study of apabetalone’s ability to help patients with chronic kidney disease was published in a scientific journal recently. It supported the drug’s effectiveness against the condition, the company said.



Daniel Theobald avatar

Daniel Theobald

Is this compound currently available to be used for people suffering from Friedreich's? When is the soonest it would be available? My brother is 33 and has the disease. - Syracuse, NY

Magdalena Kegel avatar

Magdalena Kegel

Hi Daniel,
I am sorry to hear about your brother. Unfortunately, the compound has not been tried in patients with Friedreich's ataxia. It is currently in clinical trials for other conditions, but at this moment, it is impossible to say when, or even if, it will become available.

Eileen avatar


My son is 30 and has Friedreich's. Sure wish there could be some kind of cure or at least a treatment to slow the progression

Katie Schultz avatar

Katie Schultz

I am the Patient Engagement Director for the Friedreich's Ataxia Research Alliance (FARA) and am looking for FA patients to complete a profile in our Global Patient Registry This is the only worldwide registry for FA patients and we use it to recruit patients for research studies and clinical trials. Since FA is a rare disease, it is critical that we continue to build the registry so that we have enough people to enroll in these important studies. This is the way to finding meaning treatments and/or a cure for FA. Thank you!!

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