Planned Phase 3 Trial of PBI-4050 in IPF Patients Among Top Priorities, Prometic Says

Planned Phase 3 Trial of PBI-4050 in IPF Patients Among Top Priorities, Prometic Says

A Phase 3 clinical trial in idiopathic pulmonary fibrosis (IPF) patients will be a top clinical development priority for Prometic Life Sciences in 2018, the company announced.

The realignment of the company’s research priorities follows a meeting held with the U.S. Food and Drug Administration (FDA) in January 2018. Prometic will test its oral anti-fibrotic candidate PBI-4050 in an “all comers” study in mild-to moderate IPF patients, eligible regardless of whether they are taking the standard-of-care medication Ofev (nintedanib, Boehringer Ingelheim). Patients taking  Esbriet (pirfenidone, Genentech) will be excluded, however, as previous studies reported a drug-drug interaction between PBI-4050 Esbriet.

The Phase 3 trial will assess the efficacy of PBI-4050 both as a stand-alone treatment and as an add-on to Ofev, and will integrate a dataset supporting a simple, all-inclusive indication for IPF treatment. The multi-center study will be conducted at sites across the U.S., Canada, Australia, the U.K., and Europe. Prometic anticipates starting patient enrollment in mid-2018.

“Idiopathic pulmonary fibrosis (IPF) is now priority No.1 for PBI-4050” Pierre Laurin, president and CEO of Prometic, said in a press release.

“IPF remains to this day a significant unmet medical need affecting hundreds of thousands of patients … The clinical efficacy demonstrated so far in multiple phase 2 clinical trials combined with an impressive safety and tolerability profile gives us great confidence in PBI-4050’s ability to efficiently address fibrotic diseases,” Laurin added.

A Phase 2 study (NCT02538536) in 40 IPF patients reported a stabilization in lung function among those given PBI-4050 as a stand-alone therapy or in combination with Ofev after 12 weeks of treatment, while a significant further decline was seen in those taking PBI-4050 plus Esbriet.

As part of its clinical realignment, Prometic terminated clinical trials of PBI-4050 in cystic fibrosis-related diabetes and is evaluating other programs underway. The company is also analyzing the development of PBI-4050 analogs (similar compounds), which demonstrated similar effects or even outperformed PBI-4050 in preclinical studies. These analogs could enable targeting other fibrotic-related indications, the company said.

Prometic also hopes to bring Ryplazim (plasminogen) to the market to treat as a treatment of congenital plasminogen deficiency. It is awaiting the review of its Biologic License Application submitted to the U.S. Food and Drug Administration (FDA).

Patients with IPF may also benefit from Ryplazim, Prometic states. The body converts plasminogen to the enzyme plasmin, which is key in wound healing and could work to  reduce IPF flares. The company plans to test Ryplazim in IPF patients in upcoming clinical trials. The treatment candidate recently received orphan drug status by the FDA, a designation for medications that intend to treat disorders affecting fewer than 200,000 patients in the U.S

In addition, Prometic is developing PBI-4050 as a treatment for Alström syndrome, a rare, genetic, multi-organ disease. Current data from prolonged treatment with PBI-4050 in a Phase 2 trial (NCT02739217) in the U.K. supports the treatment candidate’s use as a stand-alone medication in this patient population. The company plans to meet with regulatory agencies in the U.S. and Europe regarding this program.

“Safety and efficacy data collected and demonstrated to date in such a challenging patient population will also contribute to the clinical design and potential treatment for future clinical development initiatives of the Corporation with respect to indications such as heart, kidney & liver fibrosis,” Laurin said.

23 comments

  1. Joyce R. Jurkiw says:

    I think I would be willing to take part in this trial. I am on OFEV now.
    Not being able to breath well is just the pits but I can say I am grateful to be here and breathing!

  2. Bernd Felgner says:

    I am on OFEV, the side effects are not pleasant, however, I am still breathing with difficulties.
    I still maintain that this disease is work related. Worked in the metal industry for over 40 years, welding, grinding and lots of small molecules (polluted air) around at all times while at work.

  3. Karin Roth says:

    I have been on OFEV for 7 months now. I am functioning well, and would be grateful for any any additional med that lengthens my life. I want in!

  4. Karin Roth says:

    I have been on OFEV for 7 months now,and would be grateful for any any additional med that lengthens my life. I want in!

  5. ilene says:

    I agree with Bernd Felgner about the disease being work related. I worked in a glass bottle manufacturing plant, years of breathing glass dust with no mask has to shred the lungs to pieces.

  6. Dania Parisé says:

    MON PÈRE est le candidat idéal pour l essai de ce nouveau médicament,,,et prêt à se déplacer…car top vieux pour la transplantation mais trop jeune pour mourir de la fibrose pulmonaire…et le plus tôt sera le mieux

  7. i am 76 years old and was diagnosed in 2009 with interstitial pneumonitis. apart from an initial cocktail of drugs which didn’t help i have received no further treatment until 2016, by which time i was on oxygen therapy, 2ltrs at rest and 6 ltrs walking, when i was offered a trial place for nantidinab. half way through I fell down the stairs and broke all three bones in my ankle, and was laid up for a few months and the trial was suspended. In july 2017 I started back on the drug trial but suffered a mild heart attack and had to come off it. during the period of being laid up my breathing deteriorated and I am now on 3.5 ltrs at rest and 15 ltrs for walking. I am told that my lung function is not bad enough to warrant any treatment on the nhs. I have read that early diagnosis is vital but I don’t understand why.

  8. Dania Parisé says:

    l’essai clinique est prévu quand pour la phase 3 de PBI-4050,,,,MON PÈRE serait un excellent candidat,,,ça presse

  9. Melissa Smith says:

    Please help find a cure for this awful disease. It is as deadly as most late stage cancer diagnosis and needs more research and attention to help solve this crisis.

  10. vital says:

    Je suis traité depuis 2 mois avec ofev je serais volontaire pour faire l’essai du P B I 4050 à qui doit on s’adresser.J’ai 74 ans et atteint d’une pneumopathie interstitielle idiopatique .Merci de m’informer

  11. kathy says:

    Yes, PLEASE hurry to get approved. All IPF patients are desperate to avoid the horror of “death by IPF”.

  12. Dania Parisé says:

    comment faire pour faire partie de cet essai clinique,,,mon père est le candidat idéal…..SVP…

  13. ROBERT A SEABOLD says:

    I was on Ofev for 4 months but could not tolerate the side effects. I would be interested in learning more about PBI4050. Thanks

  14. Terry Doherty says:

    I am a moderate IPF patient recently diagnosed. Am only on O2- have been referred to the U Wash ILD COE. Interested in treatment but a bit leery of being a Placebo patient.

  15. Chuck Doria says:

    Chuck – In South Carolina and 72 with IPF. It looks like with all I’ve read PBI-4050 IS the best hope for those of us with IPF
    PLEASE HURRY!!!

  16. Ecole de danse Danipier Dania Parisé says:

    je vous suis depuis 2 ans,,,,,et j’espère depuis ce jour que mon père puisse participer à cette phase 3…..ou peut-on se renseigner….pour avoir plus d informations

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