No better example of a modest Phase 2 trial and subsequent FDA accelerated marketing approval....than this PI3K lymphoma drug umbralisib. The FDA approval took 7 months from the time the application was submitted, to approval. All layed out here.
Double the number of patients in their trials, compared to GDC 0084/paxalisib Phase 1 and Phase 2 patients....... But unlike GBM, a number of other drugs are already approved for this cancer class......and TG Therapeutics most certainly did not have an absolute dream team / of FDA peer types like this research:
First-in-Human Phase I Study to Evaluate the Brain-Penetrant PI3K/mTOR Inhibitor GDC-0084 in Patients with Progressive or Recurrent High-Grade GliomaP
atrick Y. Wen, Timothy F. Cloughesy, Alan G. Olivero, Kari M. Morrissey, Timothy R. Wilson, Xuyang Lu, Lars U. Mueller, Alexandre F. Coimbra, Benjamin
M. Ellingson, Elizabeth Gerstner, Eudocia Q. Lee and Jordi Rodon
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If you have a close look at this info below - it would seen 18 months from now until Pax GBM approval is very realistic.
So again what is an approved paxalisib worth : 4 BILLION / 5 BILLION what difference would it make, when the cap today is US$150million
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Kazia in fact noting the approval - of another PI3K drug via twitter.
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TG Therapeutics Announces FDA Accelerated Approval of UKONIQ™ (umbralisib)February 05, 2021 13:55 ET | Source:
TG Therapeutics, Inc.EFFICACY & SAFETY DATA IN RELAPSED/REFRACTORY MZL AND FLThe efficacy of UKONIQ monotherapy was evaluated in two single-arm cohorts, within the Phase 2 UNITY-NHL clinical trial, in 69 patients with MZL who received at least 1 prior therapy, including an anti-CD20 regimen, and in 117 patients with FL who received at least 2 prior systemic therapies, including an anti-CD20 monoclonal antibody and an alkylating agent. The UNITY-NHL Phase 2 trial is an open-label, multi-center, multi-cohort study with patients receiving UKONIQ 800 mg once daily. The primary endpoint was independent review committee (IRC) assessed overall response rate (ORR) according to the Revised International Working Group Criteria.
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Twitter from Kazia - this drug approval.
Kazia Therapeutics
@KaziaTx
·
Feb 8Congratulations to
@TGTherapeutics on
@US_FDA approval for Ukoniq (umbralisib) as a treatment for patients with previously treated marginal zone lymphoma or follicular lymphoma, making it the 5th commercial product in the PI3K inhibitor class. Read here:
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Jun 17, 2020
NEW YORK, June 17, 2020 (GLOBE NEWSWIRE) -- TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases, today announced the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) requesting accelerated approval of umbralisib, the Company’s investigational once-daily, oral, dual inhibitor of PI3K-delta and CK1-epsilon, as a treatment for patients with previously treated marginal zone lymphoma (MZL) and follicular lymphoma (FL). The FDA previously granted umbralisib breakthrough therapy designation (BTD) for MZL and orphan drug designation (ODD) for MZL and FL.
Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "The completion of this NDA submission marks an important milestone in bringing us one step closer to providing umbralisib as a potential treatment option for patients with relapsed/refractory MZL and FL. As a company this is a very exciting moment for us, as it marks our very first NDA submission, and I commend our team for all their efforts to get to this point.” Mr. Weiss continued, “Importantly, I also want to thank the patients, their families and the research teams who participated in these trials. This has been an incredibly impactful year for TG thus far, with several important milestones yet to come, including topline data from the
