- Forum veteraan
- Berichten: 1259
- Lid geworden op: 28 jan 2018 18:54
- waarderingen: 662
Ik lees in de corporate presentatie dat men verwacht 20.000 patienten te bereiken in de VS. In welke orde van grote moeten we denken als het gaat om de prijs waar tegen het medicijn in de markt gezet gaat worden?
Verder hebben we natuurlijk Japan en later mogelijk Europa.
Ik zou het wel interessant vinden iets van een waarderingsmodel te zien. Als hierin de hele pijplijn behandeld wordt is dat natuurlijk een plus
- Forum actieveling
- Berichten: 618
- Lid geworden op: 06 jun 2012 16:57
- waarderingen: 272
maar beperkt opwaarts potentieel lijkt 15-25, spreken we vandaag over slechts 10%-15%, bij goede phase 3 resultaten. Lijkt me naar de lage kant, of denk ik verkeerd?Stofke schreef: ↑21 mei 2020 14:54Revisiting Key Debates Ahead of Phase 3 gMG Readout
https://pbs.twimg.com/media/EYia4XOWoAA ... name=large
- Berichten: 910
- Lid geworden op: 13 okt 2017 16:18
- waarderingen: 547
We zijn ook al wat vooruit gelopen op komend nieuws denk ik, nog niet zo lang geleden bleven we hangen rond de 130, naarmate het nieuws nadert hebben we er al 20 bijgekregen deze week. Bij goed nieuws pieken tot 160-170 om daarna tussen de 150 en 160 te blijven hangen tot het volgend nieuws. Garantie tot aan de deur.Gentse strop schreef: ↑21 mei 2020 16:18maar beperkt opwaarts potentieel lijkt 15-25, spreken we vandaag over slechts 10%-15%, bij goede phase 3 resultaten. Lijkt me naar de lage kant, of denk ik verkeerd?Stofke schreef: ↑21 mei 2020 14:54Revisiting Key Debates Ahead of Phase 3 gMG Readout
https://pbs.twimg.com/media/EYia4XOWoAA ... name=large
Ik vind het wel spannend worden, met de Phase 3 resultaten 'around the corner'.
Phase 2 waren slechts 24 patienten, dus in hoeverre houden de goede phase 2 resultaten stand in de phase 3 resultaten..
Ter reminder Phase 2 resultaten (24 pts):
* 75% van de patienten in de phase 2 studie hadden significante verbetering (min. 2-punt reductie) van MG_ADL score vs 25% op placebo.
* snelle onset, 1 week na infusie.
We gaan het snel meemaken, op 15 mei hadden alle patienten de 26 weken doorlopen, dus wellicht ergens eind juni/juli resulaten.
- Forum actieveling
- Berichten: 285
- Lid geworden op: 21 nov 2017 10:16
- waarderingen: 275
https://seekingalpha.com/article/434799 ... art=single
"By the time the shelter-in-place restrictions went into effect, all 167 patients in ADAPT had already passed the eight-week time point for the analysis of the primary end point and the majority of patients had already rolled over into the open-label extension. As an update today, all patients have now completed the 26-week primary trial. We will be reporting top line data mid this year, which sets us up to file a BLA before the end of 2020, assuming success and to launch in 2021 in the United States. Additionally, we are on track to file in Japan in 2021."
- Forum gebruiker
- Berichten: 161
- Lid geworden op: 27 jan 2018 11:38
- waarderingen: 194
Highlights of topline ADAPT data
67.7% of AChR-Ab+ patients treated with efgartigimod achieved the primary endpoint compared with 29.7% on placebo (p<0.0001).
63.1% of AChR-Ab+ patients responded to efgartigimod compared with 14.1% on placebo on the Quantitative Myasthenia Gravis (QMG) score (p<0.0001); responder defined as having at least a three-point improvement on the QMG score for at least four consecutive weeks.
40.0% of efgartigimod-treated AChR-Ab+ patients achieved minimal symptom expression defined as MG-ADL scores of 0 (symptom free) or 1, compared to 11.1% treated with placebo.
Efgartigimod was well-tolerated with a safety profile that was comparable to placebo.
“The efgartigimod data showed rapid and robust responses in people with gMG, as well as a favorable tolerability profile,” said James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the ADAPT trial. “Patients with this devastating disease can experience chronic and potentially life-threatening muscle weakness that has a major impact on their quality of life, and more treatment options are needed. These data are very encouraging as they show efgartigimod has potential to make a meaningful impact on daily living activities, and we are hopeful they will lead to a new treatment being available for the gMG community.”
“With the ADAPT trial, we set out to evaluate efgartigimod’s ability to redefine the treatment paradigm for people living with gMG. The data showed that efgartigimod drove fast and deep responses, including in a proportion of patients who achieved minimal or no symptoms after treatment. In addition, we saw responses that lasted beyond eight or 12 weeks, supporting our plans to offer individualized dosing schedules that are purpose-fit to the variability in disease course that gMG patients experience,” commented Wim Parys, M.D., Chief Medical Officer of argenx. “Based on these data, we intend to submit a BLA for efgartigimod to the FDA before the end of the year, taking us one step closer to potentially making efgartigimod available to patients in 2021. All of us at argenx want to thank the patients and healthcare providers who participated in the ADAPT trial. ADAPT is the first pivotal trial of efgartigimod and these data further our confidence in its broad opportunity in other severe, IgG-mediated autoimmune diseases.”
Additional ADAPT results, including secondary endpoints and prespecified analyses
In the ADAPT trial, the secondary endpoints listed below also demonstrated statistically significant differences in the efgartigimod arm for AChR-Ab+ patients, unless otherwise noted, compared to placebo:
MG-ADL responders in the overall population, including both AChR-Ab+ and AChR-antibody negative patients (p<0.0001).
Time on trial in clinically meaningful improvement (MG-ADL improvement ≥2) (p=0.0001).
Fast onset of response on MG-ADL score (onset observed in first two weeks) (p=0.0004).
Time to qualify for retreatment endpoint did not meet statistical significance.
In AChR-Ab+ patients who met the primary endpoint, the majority showed a sustained response, including 88.6% who achieved a response for at least six weeks, 56.8% for at least eight weeks and 34.1% for at least 12 weeks.
Of AChR-Ab+ patients who received a second treatment cycle, 70.6% were MG-ADL responders compared to 25.6% of placebo patients.
90% of patients enrolled in the ADAPT trial continued to the ADAPT-Plus open-label extension study.
Percentage of efgartigimod responders on the MG-ADL score in the AChR-antibody negative patient population was consistent with the AChR-Ab+ patient population, but a greater placebo response was observed in this cohort.
Detailed data from the ADAPT trial will be submitted for presentation at a future medical meeting.
Phase 3 ADAPT Trial Design
The Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial evaluating the safety and efficacy of efgartigimod in patients with gMG. A total of 167 adult patients with gMG in North America, Europe and Japan enrolled in the trial and were treated. Enrolled patients had a confirmed gMG diagnosis and an MG-ADL total score of five or greater. Patients were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs, and were required to remain on that stable dose throughout the primary trial. Patients were eligible to enroll in ADAPT regardless of antibody status, including patients with AChR antibodies (AChR-Ab+) and patients where AChR antibodies were not detected.
Patients were randomized in a 1:1 ratio to receive efgartigimod or placebo for a total of 26 weeks as part of the primary trial. ADAPT was designed to enable an individualized treatment approach with an initial treatment cycle followed by a variable number of subsequent treatment cycles. Treatment cycles consist of four infusions of efgartigimod (10mg/kg IV) or placebo at weekly intervals. Retreatment with additional treatment cycles was initiated according to clinical response. The primary endpoint was the number of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for four or more consecutive weeks.
After the 26-week primary ADAPT trial, patients were eligible to roll-over into an open-label extension, ADAPT Plus.
Efgartigimod is a first-in-class antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.
About Myasthenia Gravis (MG)
MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility. In more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed AChR antibodies account for 80-90% of the total gMG population. There are approximately 65,000 people in the United States and 20,000 people in Japan living with the disease.
Conference Call Details
Management will host a conference call and webcast presentation today at 2:30 p.m. Central European Summer Time (CEST) / 8:30 a.m. Eastern Daylight Time (EDT). To participate in the conference call, please select your phone number below and use the confirmation code 6295982. The webcast may be accessed on the Investors page of the argenx website at www.argenx.com or by clicking here.
Please dial in 5–10 minutes prior to 2:30 p.m. CEST / 8:30 a.m. EDT using the number and conference ID below.
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