FDA Approves Phase 3 Trial Plan for Prometic’s Pulmonary Fibrosis Therapy

FDA Approves Phase 3 Trial Plan for Prometic’s Pulmonary Fibrosis Therapy

The U.S. Food and Drug Administration has approved Prometic Life Sciences’ plan for a Phase 3 pivotal trial of its idiopathic pulmonary fibrosis therapy PBI-4050.

PBI-4050 is an oral compound designed to prevent inflammation and tissue scarring in several organs, including the lungs, kidneys, liver, heart and pancreas. Prometic is developing it not only as a potential treatment for IPF, but also for Alström syndrome, metabolic syndrome and chronic kidney disease associated with type 2 diabetes. A pivotal trial is one intended to provide evidence for a drug’s regulatory approval.

A 20-week Phase 2 clinical trial (NCT02538536) showed that PBI-4050, alone or in combination with Ofev (nintedanib; Boehringer Ingelheim), slowed IPF patients’ lung function decline and stabilized their breathing capacity.

The Phase 3 trial will cover people with mild to moderate IPF, including some on Ofev and some who are not. Prometic will not enroll patients on Genentech’s  Esbriet (pirfenidone) because a drug-drug interaction between Esbriet and PBI-4050 was reported in previous trials.

Using both patients who are on Ofev and patients who are not in the trial will show whether PBI-4050 is effective alone and in combination with Ofev.

Two groups of patients in the year-long trial will receive one of two doses of PBI-4050 — either 800 mg or 1,200 mg. A third group will receive a placebo.

The trial’s primary objective will be to assess PBI-4050’s ability alone, or in combination with Ofev, to slow patients’ annual rate of decline in a measure of lung function known as forced vital capacity. That is the amount of air a person can force from their lungs after taking the deepest breath possible.

Researchers will do an interim analysis of results after six months.

Prometic expects to begin recruiting patients for the trial in the middle of 2018. They will come from the United States, Canada, Australia, Britain and other places in Europe.

“The recommendations provided by the FDA will allow us to conduct a clinical trial that is much more reflective of current treatment of IPF patients,” Joseph Parker, Prometic’s senior director of clinical affairs, who will supervise the trial, said in a press release.

“Prometic is now exceptionally well positioned to address the significant unmet medical needs in IPF. We have two late-stage clinical candidates tackling different aspects of this devastating disease which affects more than 130,000 patients in the US alone,” added Pierre Laurin, Prometic’s president and chief executive officer.

Prometic is also developing a therapy called Ryplazim to manage acute IPF flare-ups.

10 comments

  1. Sandie says:

    So thankful for this encouraging news! God bless those working on this promising life changer. Sadly, it just cant come fast enough. Plz dont delay in any way, plz!

  2. Lisa Ann Balistrieri says:

    How can I get involved in any study? Willing to be a participant. I have Bronchiectasis from a lung infection NTM that I had over 8 years ago. Any information would be appreciated.
    Thank you,
    Lisa Ann Balistrieri

  3. Ric says:

    re-listened to Wednesday’s webcast and I understood indirectly that Fibrogen was out of the race for IPF and could offer at the best a complement to existing therapies. I also understood that Prometic has a leading compound…better than Nintedanib!

    Let me explain: Fibrogen compound (FG-3019) targets only the CTGF (connecting tissue growth factor) while the PBI-4050 targets the CTGF and several other factors at once. This is specifically what Dr. Martin Kolb likes about PBI-4050, it attacks on several pathways and even more than Nintedanib who does it on 7 to 8 ways. So, even if FG-3019 has been successful in PH2 clinical trials, it is specifically because it has been given in combination with Nintedanib and Pirfenidone. Their study was designed this way, for patients who already are receiving one of the two drugs available. So, if the FG-3019 is not administered alone, as the PBI-4050 was in PH2 and will be in PH3, it will not be able to become the “Standard of Care” and surpass the results of its competitors. At the best it will become a well-tolerated supplement.

    Regarding the future of IPF, PBI-4050, Ryplazim, Nintedanib and Pirfenidone, this is what Dr. Martin Kolb had to say Wednesday:

    «I am a physician scientist, I work with a lot of different companies, I have a research lab, I do basic science, I do animal models and mechanism of disease, I also have a large clinic and follow about 1000 patients with different fibrotic lung diseases, about 250 of them are on the approved drugs, so I think I know what patients need and what we, as a community, need to make this disease better. » (I think this sets how credible he is.)

    «I’m pretty sure that in 15 or 20 years we’ll have different color pill that stop IPF and other chronic diseases and this is why we are working so hard» (He means that the solution for treating IPF will be a drug cocktail, not a miracle one, like we see now for AIDS)

    « Those 2 drugs (Nintedanib and Pirfenidone) they don’t stop the disease, they slow it down, they have very comparable effects, but they have a very different side effect profile, so when I see a patient, I give them the choice, will you pick diarrhoea or vomiting? »

    «The side effect spectrum is really what determines, in my clinic, the choice of a drug. »

    « We know that just about a third of all patients with IPF are actually on one of these drugs because they hesitate. » (Between diarrhoea and vomiting, 2/3 choose none)

    « Patients have a lot of comorbidities as well and they make it challenging to tolerate high effect rich drugs (…) that’s another reason for looking at something that is much better tolerated. » (Phase 2 trial of PBI-4050: Very well tolerated whether used alone or in combination. Most TEAE was diarrhoea, with the lowest frequency in the PBI-4050 alone group)

    « There’s a few posters presented on meetings (about PBI-4050) I’ve seen the stuff, that looks pretty cool and what this drug does, it interferes with a lot of different pathways (…) and we know from the research that folks at Prometic have done is that this molecule interferes with a lot of them. » (Meaning that if the drug targets only one pathway, the disease will overcome the treatment…so that’s not the case for PBI-4050 witch targets multiple pathways)

    « If you have these chronic disrupting diseases (like IPF), you want to have 2 strategies, stop the destruction and support the rebuilt, and PBI-4050 is active in both of these things. » (I will copy a part of the Prometic corporate presentation: The biology of healing: We are the company that can effectively address the entire healing process in a groundbreaking way using both small molecule drugs and plasma protein therapies.)

    « As a scientist I want to read about: what does a drug do? When I talk to my fellow expert in the field they don’t know much about PBI-4050, because there’s not much published. » (The MOA will be publish starting mid-February. There will be 15 to 18 publications in scientific journals. 15 to 18? What do we need to understand? That PBI-4050 interacts against fibrosis on 15 to 18 pathways? Simple speculation here.)

    To conclude, Prometic has a lot to offer! There’s no doubt in my mind that PBI-4050 will become the standard of care, because of its efficacy but mainly for its safety profile! People with IPF will have choice between: diarrhoea, vomiting or PBI-4050! Really? It’s becoming obvious that Prometic has the lead in the race. Let’s wait and see! That’s my bet.

  4. Keith Leabo says:

    I would be very interested in this study as well. I have been diagnosed with early IPF in September of 2017.Please keep me in formed. Thank you.

  5. Andrew says:

    I am highly interested in this study. I have IPF diagnosed 5 years ago. I am currently oxygen dependent with FVC 31%, stable from february 2017. I will appreciate any information you have.

  6. John Latham says:

    Hi
    I would also like to be involved in this study. I have IPF and into my 5th year. Nintedanib has slowed progression but finding it harder every day. I also run Bolton pulmonary support group with 2 mates. I am aged 63 with a wife and an 11-year-old son and it breaks my heart to think I may have to leave them soon.

  7. Richard Hein says:

    I’m curious as to how I can get involved with the study as well. I’m 53, diagnosed with mild IPF 2 months ago. Dr. has prescribed Esbriet but I have decided against taking it at this time as I’m looking for other alternatives.

  8. Chuck Doria says:

    I am 71 diagnosed with IPF 2 months ago. With much reading there is little hope for IPF patients. This study provides that hope. Please keep me informed and allow me to become involved.

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