Given the number of successfully treated patients in the Mim8 clinical trial, the breakthrough may open even more doors for hemophilia patients, their treatments, and the overall understanding of the disease.
A clinical trial conducted on an investigational hemophilia treatment known as Mim8, a drug developed by Novo Nordisk, has found a new way to treat people with the disease.
Dr. Anthony Chan, a physician working at McMaster University McMaster Children’s Hospital, specializes in treating hemophilia is excited to have another potential tool in the arsenal to provide adequate care to people living with the disease, especially since the results are highly promising.
“The percentage of patients on this trial … that will get down to zero bleeding is probably somewhere around 85 per cent,” Dr. Chan said.
Given the number of successfully treated patients in the trial, the breakthrough may open even more doors for hemophilia patients, their treatments, and the overall understanding of the disease.
What is hemophilia?
Hemophilia is a rare, mainly hereditary disease that causes bleeding. It develops due to gene mutations, specifically in the genes that create clotting factors. These proteins drive clotting to prevent excessive bleeding outside or within the body.
There are several types of hemophilia: A, B, C, and acquired. Hemophilia A and B are the two most severe types and develop when a person doesn’t have enough clotting factors 8 and 9, respectively.
The condition can range in severity, and typically, if a person has hemophilia, they are diagnosed before 18 months of age. In milder cases, it can take up to adulthood to be diagnosed.
People living with hemophilia can experience different types of bleeding, from spontaneous joint bleeds and nosebleeds to excessive bleeding after an injury or during surgery. Joint bleeds can lead to joint damage and disability. Life-threatening brain bleeds can also occur.
“When they (people with hemophilia) don’t have any therapy, the significant impact is bleeding into a significant area, like the brain or the airways,” Dr. Chan said. “And those can cause death. So, in general, they do have a short life expectancy if they don’t have any treatment.”
The recent results from the study show a new and viable way to help prevent bleeding in people with hemophilia. The current therapies, including replacement therapy and prophylactic measures, revolve around reintroducing clotting factors into the body.
While treatment has improved over the last few decades, the new results show even more progress in the right direction when it comes to treatments for hemophilia because, if left untreated, the disease can lead to severe disability and death.
New research geared toward improving quality of life in hemophilia
The phase 3 trial, known as FRONTIER2, had 254 adults and adolescent participants over the age of 12 with a specific type of hemophilia, hemophilia A. They assessed the efficacy of the drug on these groups, some of which had prior prophylactic treatment, a therapy designed to prevent “prolonged and spontaneous bleeding,” while others did not.
The participants were split up into groups of people who used prophylactic treatment and people who did not, and then again into groups that took Mim8 once weekly or once monthly.
The participants in the trial who were not on prophylactic treatment prior to starting Mim8 experienced a significant reduction in bleeds measured against annualized bleeding rate (ABR), which is a measure of determining how often a person with hemophilia has a bleed. The reduction rates were 97.1 per cent in those taking Mim8 weekly and 98.7 per cent in those taking it once monthly.
In the group that had taken prophylactics before the trial, the participants still experienced a reduction in bleeds measured against the ABR, but at rates of 48 per cent for once-weekly treatment and 42.6 per cent for once-monthly.
Other promising results showed that those taking Mim8 in the non-prophylaxis group experienced no bleeds at a rate of 85.7 per cent with once-weekly treatments and 95 per cent in once-monthly treatments. Those in the prophylaxis group also saw zero bleeds at rates of 66.3 per cent of participants in the once-weekly treatment and 65.3 per cent with one-monthly.
These results show that Mim8 could be a viable new therapy option for people living with hemophilia, more so than what is already currently available.
Along with the reduction of bleeds, the new Mim8 drug also reduces the number of treatments a person must adhere to control their disease. Mim8 showed viability in both once-weekly and once-monthly therapies, whereas current treatments are given more often.
“In order to prevent long-term disability that is caused by bleeding into organs like the brain or the airway, we give them back the protein because they lack the factor 8,” said Dr. Chan. “So, we give that back to them, but that has to be done by intravenous injections and up until a few years ago, we have to give those infusions very frequently.”
He continued, “For most of the patients, they will require it to be given twice or three times a week, but even as frequent as every other day in order to prevent most of the bleed.”
Now, however, with Mim8 on the horizon, the once-weekly or once-monthly medication will work to prevent bleeds and reduce how often a person has to take their medicine, making it more viable by lowering the obstacle to therapy.
The impact of hemophilia
Facing health challenges from infancy can impact a person in significant ways, and other research has found that as many as 14.5 per cent of people living with hemophilia are also diagnosed with depression.
The prevalence of anxiety in those with hemophilia is also fairly high at 16 per cent, and attention-deficit hyperactivity disorder was also found as a common co-condition in 15.3 per cent of people living with hemophilia.
The combination of symptoms, a lowered quality of life, and mental health disorders in those living with hemophilia can lead to long-term complications that affect them for life.
“They (people with hemophilia) would usually bleed into the joint, especially when they start weight bearing and start walking around,” said Dr. Chan. “Bleeding into the joint would cause long-term joint damage that would require joint replacement because the joint is so damaged… and that can cause long-term disability.”
Not just a male disease
Hemophilia has been notoriously dubbed a male disease, as it occurs more often in males than females. The reason for this thinking comes down to the number of male versus female cases of hemophilia driven by biological science.
“It mostly impacts males because these genes are carried in the X chromosome, and males only have one X chromosome,” Dr. Chan said.
“Having said that, I think over time we do recognize some of the females, even if only one gene is being impacted that although they have normal X chromosomes with a normal factor 8 or 9 gene, they can still be impacted by the disease and then they will not just be classified as a carrier but truly have a bleeding problem related to hemophilia.”
The more medical scientists, researchers, and clinicians understand female hemophilia, the better they will be able to provide diagnostic processes and new therapies that work for both sexes.
What this means for the future of hemophilia treatment in Canada
With the results of the Mim8 trials showing such promise, people living with hemophilia and the clinicians that treat them are better able to provide more options to prevent bleeds and improve care of patients with hemophilia.
According to Dr. Elisa in the press release, “We have the potential of offering a substantial proportion of patients the prospect of zero bleeds and convenient dosing flexibility.”
The next steps in the trial involve getting it approved for use and in the hands of patients because, according to Dr. Chan, “no matter how good the therapy is, if the patients cannot access it, it would not be useful.”