Elias Kouchakji, MD

“Although our primary end point is to assess muscle function by the PUL, we are looking at these other parameters that we think collectively as combined data have additional benefits. We are hoping that this combined data can indicate that the drug can slow the progression of the disease.”

Pamrevlumab, a first-in-class antibody developed by FibroGen, will be evaluated in a phase 3 study that will explore the effects of the drug on muscle function in nonambulatory patients with Duchenne muscular dystrophy (DMD). Dubbed LELANTOS (NCT04371666), the recently initiated study will use change in the total score of performance of upper limb (PUL) assessment as the primary end point over a 52-week treatment period.

Approximately 90 patients will be randomized 1:1 to receive pamrevlumab plus systemic corticosteroids or placebo plus corticosteroids during the double-blind treatment period. Elias Kouchakji, MD, senior vice president of clinical development, drug safety, and pharmacovigilance, FibroGen, noted that the design of the study was inspired by previous results from a phase 2 study, which ultimately showed pamrevlumab’s ability to decrease fibrosis in muscles, leading to increased muscle function.

 to provide insight on the parameters of the study, including how they formulated the end points and other important secondary objectives.

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The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen provides details of the phase 3 LELANTOS trial of pamrevlumab in Duchenne muscular dystrophy.

Elias Kouchakji, MD: Investigating Pamrevlumab for DMD in Phase 3 LELANTOS Study

"Putting an estimate of survival, or how long they’re going to be living, is to also take a look at how long you’re going to maintain their living into more functional.”

An important phase in Duchenne muscular dystrophy (DMD) is the loss of ambulation, which can usually occur between the ages of 7 and 13 years, leaving patients in a wheelchair and damaging their quality of life. During that time, their lungs may weaken, which can lead to difficulties in breathing and an increased risk of chest infections. For this reason, DMD gene therapy trials over the years have often been tailored towards ambulatory patients.

Elias Kouchakji, MD, senior vice president of clinical development, drug safety, and pharmacovigilance, FibroGen feels as though these patient populations have been underrepresented but are slowly making their way into more clinical trials. Pamrevlumab, Fibrogen’s investigational DMD agent, is expected to be assessed in a phase 3 setting that will include both ambulatory and non-ambulatory patients.

Like many others, Kouchakji has stressed that a more rigorous examination of this patient population will support the general hypothesis with neurological disorders in that, earlier treatment can improve quality of life features further down the clinical disease progression. In this interview with 

, Kouchakji takes a closer look at this population, and why he feels it holds a sacred place within the DMD community and research settings.

The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen discussed why the representation of non-ambulatory patient populations is necessary in DMD trials.

Elias Kouchakji, MD: Importance of Non-Ambulatory Patients in DMD Trials

"I’m looking forward for the patient to live with Duchenne, but not die from Duchenne. That will be the greatest accomplishment.”

FibroGen’s pamrevlumab enters a phase 3 trial as another drug hoping to become FDA-approved for the treatment of Duchenne muscular dystrophy (DMD), adding to the growing number of treatments available for patients. The drug will be assessed in both ambulatory and non-ambulatory populations, addressing 2 major types of patients with DMD that are at different stages of disease progression.

As the momentum within the DMD research community continues to surge, there still remains a great unmet need according to Elias Kouchakji, MD. Kouchakji, who is the senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen, feels as though transforming the fatal disease to a chronic one is among the most pressing needs at the moment.

Kouchakji compared the goals of DMD research to that of HIV, where instead of dying from it, patients are dying with it. In an interview with 

, Kouchakji explained what he feels is the greatest unmet need, and how close the DMD research community is to achieving that goal.

The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen discussed why turning Duchenne muscular dystrophy into a chronic disease rather than a fatal one, is a top priority.

Author | Elias Kouchakji, MD

Articles

Elias Kouchakji, MD: Investigating Pamrevlumab for DMD in Phase 3 LELANTOS Study

Elias Kouchakji, MD: Importance of Non-Ambulatory Patients in DMD Trials

Elias Kouchakji, MD: Greatest Unmet Need in Duchenne Muscular Dystrophy