Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by recurrent bleeding in the joints.
The private investment firm Gurnet Point Capital also will support other aspects of TRM-201‘s clinical development, though none of the terms of the agreement were disclosed. The investigational therapy was designed to ease the pain of severe joint disease in people with hemophilia, for which there currently is no approved treatment.
“Hemophilia treatment has advanced significantly but hemophilic arthropathy hasn’t gone away,” David Moore, a partner at Gurnet Point Capital, said in a press release. “We made this investment because we see an opportunity to provide a pain management option that’s long overdue to the bleeding disorder community.”
RESET-HA is expected to start early next year. After a screening period, the study will include a 12-week (about three months) treatment program in which participants will be randomly assigned to receive either TRM-201 or a placebo. That will then be followed by a one-year extension period in which only the potential therapy will be given.
Eligible participants, ages 12-75, will have hemophilia A or B and chronic pain in one or more joints. Study locations will open soon, according to Tremeau; patients can sign up here to receive notifications.
Hemophilic arthropathy is the most common complication in hemophilia — however, no medications exist in the U.S. to treat joint pain in people with bleeding disorders.
At present, opioids are the most frequently prescribed medication to treat joint pain in these patients. However, these pain-relieving medicines are associated with a risk of addiction, among other health concerns.
In the general population, pain is commonly managed with non-steroid anti-inflammatory drugs (NSAIDs), which include medications such as ibuprofen (sold as Advil and Motrin, among others) and aspirin. These therapies work by blocking the activity of two proteins, called COX-1 and COX-2.
TRM-201 is a NSAID that only blocks the activity of COX-2, and not COX-1, which is involved in blood clotting. As such, it is believed that TRM-201 may be a safer alternative for people with hemophilia.
In a clinical trial conducted in people with rheumatoid arthritis — an autoimmune disease characterized by joint pain — treatment with rofecoxib, the active component of TRM-201, caused fewer bleeds in the intestinal tract than treatment with a non-selective NSAID called naproxen. Naproxen is sold under the brand name Aleve, among others.
“Patients with hemophilic arthropathy need an alternative to opioids,” said Bradford C. Sippy, CEO of Tremeau. “TRM-201 could be this much-needed option, and the investment and support from Gurnet Point Capital will enable us to make our vision a reality.”
Rofecoxib was previously marketed under the brand name Vioxx. Due to concerns about the medication’s effect on heart health, Vioxx was voluntarily withdrawn from the market in 2004. Since then, research has shown that all NSAIDs, not just rofecoxib, can impact heart health, if taken at certain doses for long enough lengths of time.
“For many of my patients with bleeding disorders, the joint pain they experience has a profound negative impact on their lives,” said Tyler Buckner, MD, a practicing hematologist in Denver.
“The withdrawal of VIOXX took away an important treatment option for many of our patients,” Buckner said.
Tremeau has previously received guidance from the U.S. Food and Drug Administration on how to conduct a pivotal Phase 3 clinical trial to test TRM-201 in people with hemophilia-associated joint pain. The agency has since considered that available evidence shows that levels of rofecoxib exposure between TRM-201 and the previously marketed version of VIOXX are comparable.
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