In the orphan drug development process, it is becoming more obvious to pharmaceutical companies that they can no longer rely mostly on scientists and doctors to get their information. Patients’ input is key to understanding a rare disease. That is why the smarter pharmaceutical companies are partnering with patient groups to help them guide their orphan drug development.
The most recent example of such a partnership is between BERG and The Dystrophic Epidermolysis Bullosa Research Association of America (debra of America) to develop a drug to treat Epidermolysis Bullosa (EB), a rare skin disorder.
In an exclusive interview with Rare Disease Report
, we talked with the CEO of BERG, Niven R. Narain, and executive director of debra of America, Brett Kopelan.
Kopelin explained that debra of America is an all-inclusive patient group. They assist EB families in all aspects of care but they also perform/invest in research that will help lead to better ways to manage the rare condition.
The drug BERG is developing for EB is a topical cream formulation of their anticancer drug BPM 31510 (Ubidecarenone). According to Narain, the drug advanced wound healing in preclinical studies and a Phase I clinical trial is currently being conducted at the University of Miami Department of Dermatology & Cutaneous Surgery. Narain also noted the product, in addition to having wound healing properties, it has anti-inflammatory effects and analgesic effects that may also benefit EB patients.
Details of the study can be found on the debra website
The primary objective of the study is to evaluate the safety and tolerability in patients with EB when treated with topical BPM31510 3% cream. The secondary outcome measures will include pharmacokinetics, pain, quality of life, time to healing, quality of healing, increased resistance to trauma, and reduction in blister formation. The study is an 18 week commitment, including a 2 week screening period, a 12 week treatment period, and a 4 week follow-up.
The cream is applied every other day to twice per week to a wound on the patient. The cream will also be applied to a section of intact skin every day.
Patients will be required to visit the trial site (University of Miami) during weeks 1, 2, 5, and 12. Patients may be eligible for a travel stipend.
Patients must be at least 12 years of age and have histologically confirmed EB.
A total of 10 patients are needed for the Phase 1 trial.
Benefits of a Partnership
The partnership between BERG and debra of America allows the 2 parties to advance the clinical trial process. Kopelan noted in debra’s discussions with BERG, it was made clear what patients want/need to participate in a clinical trial. As such, a travel stipend is now being made available.
From BERG’s perspective, the company is fairly new to EB. Most of their clinical trials are for oncology. Therefore, partnering with a well-organized, committed patient group provides the company with insights into what patients need as well as what clinicians and regulatory bodies need in regard to EB treatment. Narain said, “you can look at this in a textbook, you can read it online, you can be very academic about EB or you can actually know what it means to these people by talking and meeting with them,” adding that by meeting with the patients and their families, the company’s EB team has clearer understanding of how important their jobs are.
Narain listed the 3 positive impacts the partnership has had on his company.
It has allowed the company to truly understand the disease from the patients’ perspective.
Given them access to key opinion leaders throughout the country.
If the drug succeeds in early trials, it will be easier to recruit patients for more studies.
EB is a group of genetic conditions that cause the skin to be very fragile and to blister easily. Blisters and skin erosions form in response to minor injury or friction, such as rubbing or scratching. It can be a very painful condition and often referred to as the worst disease you have never heard of.
In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases of this condition involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical problems.
It currently affects an estimated 1 in every 20,000 children born in the United States.