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Aandeel Pharnext PSE:ALPHA.FR, FR0011191287

  • 0,525 17 sep 2021 17:35
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Forum Pharnext geopend

161 Posts
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  1. forum rang 4 AdeVries 10 juni 2020 09:38
    www.benzinga.com/pressreleases/20/06/...

    US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study

    PARIS, FRANCE / ACCESSWIRE / June 10, 2020 / Pharnext SA (FR0011191287 - ALPHA), a biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence, today provided an update on the regulatory and clinical status of PXT3003, its lead program in Charcot-Marie-Tooth Type 1A ("CMT1A").

    PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration ("FDA"). In Pharnext's previous interactions with FDA, the agency provided guidance that an additional Phase III study would be required. Based on our most recent interactions with FDA through a Type C meeting, FDA has now provided a very clear path to NDA submission for approval of our lead clinical program.
    The FDA has agreed with the key elements of Pharnext's approach for the developmental pathway to approval for PXT3003. Specifically, the FDA has indicated that a single pivotal Phase III study in CMT1A delivering robust results could be sufficient for approval of PXT3003. This study design will be similar to the earlier Phase III study of PXT3003 that yielded encouraging top line results in October 2018. The FDA has agreed that the primary endpoint will again be Overall Neuropathy Limitations Scale (ONLS). Notably, the FDA has also agreed that the factorial study requirement for combination drugs can be carried out in a preclinical CMT1A disease animal model, and not in a human Phase III clinical trial as typically required. The animal factorial study, a requirement for NDA filing, will be done under GLP or GLP-like conditions and will have a similar study design and use the same CMT1A model as our previously successful preclinical factorial study.

    Phase III Trial Design Update

    For the upcoming Phase III pivotal study, as recommended by FDA, Pharnext will use ONLS as the primary endpoint, as was used in the previous Phase III study. We will have two arms in the study which will compare the high dose vs placebo. The high dose showed encouraging results in the earlier Phase III study. In addition, we have resolved the past manufacturing issue with our high dose oral solution and will now use the earlier successful low dose oral solution formulation in a higher volume to deliver the high dose in our upcoming Phase III trial. We will also be utilizing a more convenient new unit dose package format, stick packs (liquid sachets), that will assure optimal patient compliance and more accurate dosing.

    Pharnext plans to initiate the final pivotal Phase III clinical study before the end of Q1 2021.

    Dr David Horn Solomon, Chief Executive Officer commented, "We are grateful that the FDA has provided strong and specific guidance to complete pivotal studies towards NDA submission and approval for PXT3003 in CMT1A. Both the Phase III clinical trial as well as the preclinical animal study will be informed in their design by the earlier Phase III clinical trial that provided encouraging top-line results, and the earlier animal study that was successful, respectively. We have addressed earlier manufacturing issues and look forward to initiating the Phase III clinical trial before the end Q1 2021. Our goal at Pharnext is to provide CMT1A patients and their caregivers a new therapeutic to treat this disease where no therapy currently exists."

    About Charcot-Marie-Tooth Disease Type 1A (CMT1A)

    Charcot-Marie-Tooth (CMT) disease encompasses a heterogeneous group of inherited, progressive, chronic peripheral neuropathies. CMT type 1A (CMT1A), the most common type of CMT, is an orphan disease affecting more than 100,000 people in the U.S. and E.U. The genetic mutation responsible for CMT1A is a duplication of the PMP22 gene coding for a peripheral myelin protein. Overexpression of this gene causes degradation of the neuronal sheath (myelin) and nerve dysfunction. As a result, patients have a significantly altered quality of life, suffering from progressive muscle atrophy of the limbs causing problems with walking, running balance and hand function. They may have loss of sensation, pain and cramps, and at least 5% need wheelchairs. First symptoms usually appear during childhood and progressively evolve throughout patients' lives. To date, no curative or symptomatic medications have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery.

    About PXT3003

    Pharnext's first-in-class PLEODRUG(TM) PXT3003, developed using Pharnext's R&D platform, PLEOTHERAPY(TM), is a novel oral fixed-dose combination of baclofen, naltrexone and sorbitol, with Orphan Drug Designation in the U.S. and E.U. PXT3003, Pharnext's lead PLEODRUG(TM), has shown positive results both in non-clinical pharmacology and clinical studies for the treatment of CMT1A. In non-clinical pharmacology studies, PXT3003 inhibited the overexpression of the PMP22 gene, improved myelination of peripheral nerves and motor / sensory impairments. In a Phase II clinical study in 80 adult patients with CMT1A, PXT3003 was safe and well tolerated. In addition, PXT3003 showed trends in multiple efficacy endpoints beyond stabilization, particularly the ONLS scale. These results were published in the Orphanet Journal of Rare Diseases (OJRD) in December 2014. In October 2018, PXT3003 completed an international Phase III trial in 323 patients 16 years and older for the treatment of CMT1A, confirming the excellent safety profile of the combination and demonstrating an encouraging efficacy profile. The Phase III extension study is currently ongoing. An additional international pivotal Phase III trial is planned to be initiated in the first quarter of 2021.
  2. rodney007 29 april 2021 08:45
    Zou wel eens de ster van de dag kunnen worden.

    Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

    New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale (‘ONLS’), after 4.5 years of total trial time.

    A live conference call and webcast will be held tomorrow, Thursday April 29th at 2:00 p.m. CET
    (8:00 a.m. ET)

    PARIS, France, April 28, 2021, 6:00 p.m. CET – Pharnext SA (FR0011191287 - ALPHA) (the ‘Company’), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY™ platform, today announces new results from an interim analysis of an ongoing open-label follow-up extension study (‘PLEO-CMT-FU trial’) following the first double-blind, placebo-controlled Phase III study (‘PLEO-CMT trial’) of PXT3003 for the treatment of Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’). There is currently no approved drug treatment for CMT1A.

    In January 2020, the company reported interim results suggesting sustained safety and efficacy of PXT3003 in patients with mild-to-moderate CMT1A after 24 months of total trial time (PLEO-CMT and PLEO-CMT-FU Period 1 trials). The new results announced today continue to show sustained treatment benefits for CMT1A patients treated with PXT3003 at High Dose (‘HD’) in the PLEO-CMT-FU Period 2 trial with a data readout at 54 months of total trial time (double-blind + open-label). Highlights from an analysis of available data include:

    PXT3003 was safe and well tolerated. Data are consistent with observed safety profile in prior clinical trials.

    Data on the Overall Neuropathy Limitations Scale (‘ONLS’), which measures patients’ functional motor disability, are as follows (please refer to the diagram below for graphical illustration):

    During PLEO-CMT (double-blind Phase III study), patients treated with placebo on average declined on ONLS while patients treated with PXT3003 on average improved. The best efficacy signal was observed in the cohort of patients treated with PXT3003 HD.
  3. Chasse_Patate 8 juni 2021 11:45
    quote:

    twopence schreef op 28 mei 2021 10:32:


    Moet wel heel veel geduld hebben met dit Aandeel. Na eet nuen lange periode van zijwaarts bewegen gaat het nu duidelijk lager.


    Dat was vast voorkennis:

    pharnext.com/en/press-releases/pharne...

    Dit soort financiële constructies zorgt voor veel verwatering en alleen winst voor de partij die hier komt "investeren".
    De zittende aandeelhouders zijn meestal de dupe.
    Vanaf nu een nog veel lagere koers als er geen goed nieuws komt.

  4. Kojo2 26 juni 2021 12:02
    Persoonlijk vind ik het altijd een spijtige zaak dat biotechbedrijven bijna uitsluitend gebruik maken van de uitgifte van converteerbare obligaties. Vooreerst omdat niet alle aandeelhouders op dezelfde wijze worden behandeld. Ten tweede omdat dit die bedrijven in een positie brengt waarbij ze volledig afhankelijk worden van de bevoorrechte investeerders. En die bepalen finaal tegen welke prijs de conversie in aandelen gebeurt. En aangezien ze geen Sinterklaas spelen, maar uit zijn op winstmaximalisatie, is de kleine aandeelhouder altijd de dupe.

    Ik volg Pharnext al een tijd. Het ontwikkelt een beloftevol medicijn, maar heeft nog een paar jaar nodig om te slagen in het opzet. Het blijft evenwel een risicovolle onderneming. Alles inzetten op één kanshebber is riskant. Met de uitgifte van de concerteerbare obligaties kan Pharnext wellicht de periode tot aan de eventuele erkenning overbruggen.

    De koersevolutie lijkt me normaal. Ik begrijp niet waarom de ceo moord en brand schreeuwt omwille van een halvering van de koers. Als alle nieuw uitgegeven obligaties omgezet worden, herleidt men de aandelen van de andere beleggers tot ongeveer een derde. In die context is een koersdaling tot een pennystock redelijk normaal.
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