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Expert Update: The Status of JAK Inhibitors in Development

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Published on July 13, 2016

Are there any new drug therapies for myeloproliferative neoplasm (MPN) patients? Patient Power founder, host and myelofibrosis (MF) patient, Andrew Schorr, leads a discussion with MPN experts, Dr. Mark Heaney of Columbia University Medical Center and Dr. John Mascarenhas of Mt. Sinai School of Medicine. The active areas of MPN drug research include ruxolitinib (Jakafi), pacritinib (currently on FDA hold), momelotinib (JAK 2 inhibitor currently in clinical trials) and PERSIST-1 (randomized clinical trial studying the efficacy and safety of pacritinib).

Editor's Note:
3/14/16: While pacritinib has been placed on full clinical hold, the FDA has suggested that they will allow those individuals that were seeing benefit from pacritinib to resume treatment through a Single Patient IND (SPI)/Compassionate Use program. CTI BioPharma is working to develop a program and will be providing clinical investigators with specific details to assist in submitting SPIs to the FDA for consideration in the near future.

Update 1/5/17: The FDA has removed the full clinical hold on the clinical trial for pacritinib. The maker of pacritinib, CTI BioPharma, will begin a new trial, PAC203, in the second quarter of 2017 for patients with primary myelofibrosis who have failed prior ruxolitinib therapy to evaluate the safety and efficacy of this therapy. For more information, please contact your doctor.

 

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Transcript | Expert Update: The Status of JAK Inhibitors in Development

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That's how you’ll get care that's most appropriate for you.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

Dr. Heaney:

There are a number of JAK inhibitors besides ruxolitinib (Jakafi) that have been investigated clinically. 

I've been involved in a clinical study with pacritinib, which is a drug that was very promising and may still be useful, and another drug called momelotinib, which is also in Phase III trials. Although ruxolitinib is a drug that has changed the lives of many patients with myelofibrosis, we know that ruxolitinib can suppress the blood counts, and many patients with myelofibrosis suffer with anemia and low platelet count.

And so there’s really a need for drugs that don’t have that particular side effect. And so some of the new JAK2 inhibitors seem like they don’t have as much suppressive effect on the red blood cells and the platelets and may offer an alternative for patients whose blood counts are too low. 

Andrew Schorr:                  

Let’s just get an update for a second. The drug pactritinib, it had been very promising. And then the FDA said hey, wait a minute, maybe there’s a safety concern; we’re not sure. We’re going to wait. Trials were stopped. Do you have a feeling of whether that may go forward, or we don’t know or whether it will have a more limited place? 

Dr. Heaney:         

I think historically when drugs are put on hold by the FDA that they end up in a more limited place. I think that on the basis of the Phase III trials, the drug appears to be promising. But they did see a safety effect that raises some concern. And so I think we’re trying to understand what that safety effect is, and then whether we can identify the patient population that might not have the same risk.

Andrew Schorr:                  

Safety first. Any comment from you, Dr. Mascarenhas?

Dr. Mascarenhas:               

It’s no secret. I still think that’s a valuable drug to add to the armamentarium. If you look at the results of the PERSIST-1, which was the initial Phase III study, it was a very positive study with some very interesting points. For example, transfusion independence in select groups of patients—it was much gentler on the platelets. And as we’ve discussed before, low platelets can be a real issue for a lot of patients.

That can limit the ability to have access to therapies, because many of the therapies will reduce the platelets. So I do think that there’s a select subgroup that can benefit from this drug, but it has to really be weighed against the potential risk and likely optimized in each individual patient.

Andrew Schorr:                  

Dr. Heaney, you mentioned momelotinib, if I got it right. First of all, what does the “ib” mean; ruxolitinib, pacritinib, what does the “ib” mean?

Dr. Heaney:         

The “ib” means that it’s in a class of drugs that is an inhibitor. So imatinibwas one of the first “ibs.”

Andrew Schorr:                  

Right. And if you watch the ads on TV, you’ll see ads for “mabs,” monoclonal antibodies and they’re used widely in medicine.

Dr. Heaney:         

Right.

Andrew Schorr:                  

So momelotinib, what’s the promise of that? 

Dr. Heaney:         

Momelotinib has less of an effect in suppressing the hemoglobin and some of the earlier clinical studies, some patients who even required regular transfusions were able to take momelotinib and no longer required transfusion. That’s a little bit different than ruxolitinib, which typically reduces the hemoglobin in patients with myelofibrosis.

Andrew Schorr:                  

So as we move on later, you’re going to have to assess a patient’s individual situation, how they’re responding to certain drugs, and whether with approved therapies or sometimes offering a clinical trial,will it be right for their specific situation and the way their body responds? 

Dr. Heaney:         

Yes. So we’re really looking forward to the day when we have the luxury of being able to pick and choose different medicines to really help us to tailor therapy better than we can right now. And we’re doing that for patients with another MPN, chronic myeloid leukemia. We think the chance of the timeline where we’ll be able to do that for myelofibrosis is hopefully just around the corner.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

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