Dive Brief:
- Armetheon, a specialty pharmaceutical company, has reached an agreement with the Food and Drug Administration to do a single 1,000-patient Phase 3 clinical trial for its lead drug candidate tecarfarin, a novel oral anticoagulant, before filing a New Drug Application (NDA).
- The privately held, California-based company said it expects to file the NDA in 2019 for tecarfarin, being investigated for use as a vitamin K antagonist.
- The FDA indicated that Armetheon could amend the existing Special Protocol Assessment (SPA), or detailed guidance, for the 1000-patient TACT study, the company said.
Dive Insight:
Armetheon is aiming to find its niche among numerous players in a huge market. The global market for antithrombotic/anticoagulant drugs is expected to reach $27.3 billion by 2021, up from $18.9 billion in 2016, according to Dublin-based Research and Markets.
Armetheon conceded that warfarin—FDA approved in 1954—has been the standard of care for oral anticoagulants for decades. But the company stressed that warfarin has its limitations because of the enzyme that metabolizes it, making it difficult to achieve stable anticoagulation control in certain patients, including people with chronic kidney disease.
In 2009, tecarfarin failed a clinical trial when it couldn’t beat warfarin head to head, ultimately resulting in the 2011 closure of its original owner, ARYx Therapeutics. That same year the drug went to Armetheon, with the startup deciding to target a specific group of patients that doesn’t respond well to warfarin.
It’s been two years since Armetheon raised $24 million in Series B financing to further tecarfarin’s development. In August 2016, Armetheon cited positive results from a Phase 1 pharmacokinetics study of tecarfarin vs. warfarin in patients with severe chronic kidney disease who required anticoagulation therapy—and announced it was planning the Phase 3 pivotal TACT trial.
Armetheon said the forthcoming pivotal trial will enroll patients with all indications for anticoagulation, which would support a potentially broad label if its product is approved.
Armetheon's President and Interim CEO M. Kengatharan described the FDA’s agreement, allowing his company to conduct a 1000-patient final Phase 3 study prior to a potential NDA filing, as being "unprecedented for an anti-thrombosis/cardiovascular drug."
Tecarfarin has been studied in nearly 900 patients in 10 different clinical trials, including a prior pivotal trial and a study in chronic kidney disease patients "where tecarfarin was observed to have potential benefits based on pharmacokinetics when compared to warfarin," Armetheon said.
In addition to its work on tecarfarin, Armetheon is developing its oral anti-arrhythmic drug candidate, budiodarone, to treat ventricular tachycardia in patients with implantable cardioverter defibrillators. Armetheon said Feb. 13 the Patent and Trademark Office issued a new patent Jan. 24 covering budiodarone, which the company described as the only known novel anti-arrhythmic in clinical development.
To date, budiodarone has been studied in nearly 129 subjects in six different clinical trials, including two Phase 2a studies, Armetheon said. The company said it plans to initiate two new Phase 2 clinical studies with budiodarone in the second half of 2017.