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Cstone's Partner Blueprint Medicines Presented Updated Part 1 Data from PIONEER Trial of Avapritinib
Hong Kong Exchanges and Clearing Limited and The Stock Exchange of Hong Kong Limited take no responsibility for the contents of this announcement, make no representation as to its accuracy or completeness and expressly disclaim any liability whatsoever for any loss howsoever arising from or in reliance upon the whole or any part of the contents of this announcement. The forward-looking statements made in this announcement relate only to the events or information as of the date on which the statements are made in this announcement. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this announcement completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this announcement, statements of, or references to, our intentions or those of any of our directors and/or our Company are made as of the date of this announcement. Any of these intentions may alter in light of future development. CStone Pharmaceuticals 基 石 藥 業 (Incorporated in the Cayman Islands with limited liability) (Stock Code: 2616) VOLUNTARY ANNOUNCEMENT CSTONE’S PARTNER BLUEPRINT MEDICINES PRESENTED UPDATED PART 1 DATA FROM PIONEER TRIAL OF AVAPRITINIB SHOWING ROBUST REDUCTIONS IN CUTANEOUS DISEASE SYMPTOMS IN PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS The partner of CStone Pharmaceuticals (the “Company” or “CStone”), Blueprint Medicines Corporation (NASDAQ: BPMC) (“Blueprint Medicines”), announced on June 6 updated clinical data from part 1 of the PIONEER trial showing robust and consistent clinical activity for avapritinib across multiple qualitative and quantitative measures of cutaneous disease in patients with indolent systemic mastocytosis (“ISM”). -
Entrectinib (Interim Monograph)
Entrectinib (interim monograph) DRUG NAME: Entrectinib SYNONYM(S): RXDX-1011, NMS-E6282 COMMON TRADE NAME(S): ROZLYTREK® CLASSIFICATION: molecular targeted therapy Special pediatric considerations are noted when applicable, otherwise adult provisions apply. MECHANISM OF ACTION: Entrectinib is an orally administered, small molecule, multi-target tyrosine kinase inhibitor which targets tropomyosin- related kinase (Trk) proteins TrkA, TrkB, and TrkC, proto-oncogene tyrosine-protein kinase ROS (ROS1) and anaplastic lymphoma kinase (ALK). TrkA, TrkB, and TrkC are receptor tyrosine kinases encoded by the neurotrophic tyrosine receptor kinase (NTRK) genes NTRK1, NTRK2, and NTRK3, respectively. Fusion proteins that include Trk, ROS1, or ALK kinase domains drive tumorigenic potential through hyperactivation of downstream signalling pathways leading to unconstrained cell proliferation. By potently inhibiting the Trk kinases, ROS1, and ALK, entrectinib inhibits downstream signalling pathways, cell proliferation and induces tumour cell apoptosis.3-5 PHARMACOKINETICS: Oral Absorption bioavailability = 55%; Tmax = 4-6 hours; Tmax delayed 2 h by high-fat, high-calorie food intake Distribution highly bound to human plasma proteins cross blood brain barrier? yes volume of distribution 551 L (entrectinib); 81.1 L (M5) plasma protein binding >99% (entrectinib and M5) Metabolism primarily metabolized by CYP 3A4 active metabolite(s) M5 inactive metabolite(s) M11 Excretion primarily via hepatic clearance urine 3.06% feces 82.9% (36% as unchanged entrectinib, 22% as M5) terminal half life 20 h (entrectinib); 40 h (M5) clearance 19.6 L/h (entrectinib); 52.4 L/h (M5) Elderly no clinically significant difference Children comparable pharmacokinetics of entrectinib and M5 in adults and children Ethnicity no clinically significant difference Adapted from standard reference3,4 unless specified otherwise. -
07052020 MR ASCO20 Curtain Raiser
Media Release New data at the ASCO20 Virtual Scientific Program reflects Roche’s commitment to accelerating progress in cancer care First clinical data from tiragolumab, Roche’s novel anti-TIGIT cancer immunotherapy, in combination with Tecentriq® (atezolizumab) in patients with PD-L1-positive metastatic non- small cell lung cancer (NSCLC) Updated overall survival data for Alecensa® (alectinib), in people living with anaplastic lymphoma kinase (ALK)-positive metastatic NSCLC Key highlights to be shared on Roche’s ASCO virtual newsroom, 29 May 2020, 08:00 CEST Basel, 7 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from clinical trials of 19 approved and investigational medicines across 21 cancer types, will be presented at the ASCO20 Virtual Scientific Program organised by the American Society of Clinical Oncology (ASCO), which will be held 29-31 May, 2020. A total of 120 abstracts that include a Roche medicine will be presented at this year's meeting. "At ASCO, we will present new data from many investigational and approved medicines across our broad oncology portfolio," said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development. “These efforts exemplify our long-standing commitment to improving outcomes for people with cancer, even during these unprecedented times. By integrating our medicines and diagnostics together with advanced insights and novel platforms, Roche is uniquely positioned to deliver the healthcare solutions of the future." Together with its partners, Roche is pioneering a comprehensive approach to cancer care, combining new diagnostics and treatments with innovative, integrated data and access solutions for approved medicines that will both personalise and transform the outcomes of people affected by this deadly disease. -
Cogent Biosciences Announces Creation of Cogent Research Team
Cogent Biosciences Announces Creation of Cogent Research Team April 6, 2021 Names industry veteran John Robinson, PhD as Chief Scientific Officer New Boulder-based team with exceptional track record of drug discovery and development focused on creating novel small molecule therapies for rare, genetically driven diseases Strong year-end cash position of $242.2 million supports company goals into 2024, including three CGT9486 clinical trials on-track to start this year, beginning with ASM in 1H21 BOULDER, Colo. and CAMBRIDGE, Mass., April 6, 2021 /PRNewswire/ -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced the formation of the Cogent Research Team led by newly appointed Chief Scientific Officer, John Robinson, PhD. "Today marks an important step forward for Cogent Biosciences as we announce the formation of the Cogent Research Team with a focus on discovering and developing new small molecule therapies for patients fighting rare, genetically driven diseases," said Andrew Robbins, President and Chief Executive Officer of Cogent Biosciences. "I am thrilled to welcome John onboard as Cogent Biosciences' Chief Scientific Officer. John's expertise and seasoned leadership make him ideally suited to lead this new team of world class scientists. Given the team's impressive experience and accomplishments, we are excited for Cogent Biosciences' future and the opportunity to expand our pipeline and deliver novel precision therapies for patients." With an exceptional track record of innovation, the Cogent Research Team will focus on pioneering best-in-class, small molecule therapeutics to both improve upon existing drugs with clear limitations, as well as create new breakthroughs for diseases where others have been unable to find solutions. -
Tabrecta (Capmatinib)
Tabrecta (capmatinib) NEW PRODUCT SLIDESHOW Introduction . Brand name: Tabrecta . Generic name: Capmatinib . Pharmacologic class: Kinase inhibitor . Strength and Formulation: 150mg, 200mg; tablets . Manufacturer: Novartis . How supplied: Tabs—56 . Legal Classification: Rx Tabrecta Indication Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA- approved test . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s) Dosage and Administration Confirm presence of a mutation that leads to MET exon 14 skipping in tumor specimens Swallow whole 400mg twice daily Recommended dose reductions for adverse reactions: . First: 300mg twice daily . Second: 200mg twice daily . Permanently discontinue in patients unable to tolerate 200mg twice daily Dosage Modifications for Adverse Reactions Interstitial lung disease/pneumonitis . Any grade: permanently discontinue Increased ALT and/or AST without increased total bilirubin . Grade 3: withhold until recovery to baseline ALT/AST; if recovery to baseline within 7 days, then resume at same dose, otherwise resume at a reduced dose . Grade 4: permanently discontinue Increased ALT and/or AST with increased total bilirubin in the absence of cholestasis or hemolysis . ALT and/or AST >3xULN with total bilirubin >2xULN: permanently discontinue Dosage Modifications for Adverse Reactions Increased total bilirubin without concurrent increased ALT and/or AST . Grade 2: withhold until recovery to baseline bilirubin; if recovered to baseline within 7 days, then resume at same dose, otherwise resume at reduced dose . Grade 3: withhold until recovery to baseline bilirubin; if recovered to baseline within 7 days, then resume at reduced dose, otherwise permanently discontinue . -
Incyte Corporation
Building Value through Innovative Medicines 2019 First Quarter Financial and Corporate Update April 30, 2019 Forward-looking Statements Except for the historical information set forth herein, the matters set forth in this presentation contain predictions, estimates and other forward-looking statements, including without limitation statements regarding: expectations regarding ruxolitinib, ruxolitinib cream, itacitinib, pemigatinib, parsaclisib and INCMGA0012 trial results and timing of the receipt and presentation of those results; the expected timing of the NDA submissions for pemigatinib and capmatinib; our belief that certain of our projects, such as the acceleration of vitiligo development and opportunities with pemigatinib and itacitinib, warrant increased near-term funding; expectations regarding planned regulatory updates, planned pivotal clinical updates and planned clinical trial initiations; expectations by our collaborative partners regarding timing of NDA submission for capmatinib and announcement of baricitinib trial results; our plans and expectations for development of, and clinical trials involving, our other product candidates, including the potential timing for regulatory submissions; our plans for immediate launch of ruxolitinib for steroid-refractory acute GVHD should the FDA approve our sNDA; our updated 2019 GAAP and non-GAAP guidance; our expectations regarding baricitinib royalties; and our expected 2019 newsflow events. These forward-looking statements are based on our current expectations and are subject to risks -
First-Line Treatment Options for Patients with Stage IV Non-Small Cell Lung Cancer with Driver Alterations
First-Line Treatment Options for Patients with Stage IV Non-Small Cell Lung Cancer with Driver Alterations Patients with stage IV non-small cell lung cancer Nonsquamous cell carcinoma and squamous cell carcinoma Activating EGFR mutation other Sensitizing (L858R/exon 19 MET exon 14 skipping than exon 20 insertion mutations, EGFR exon 20 mutation ALK rearrangement ROS1 rearrangement BRAF V600E mutation RET rearrangement NTRK rearrangement mutations KRAS alterations HER2 alterations NRG1 alterations deletion) EGFR mutation T790M, L858R or Ex19Del PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options Emerging target; no Emerging target; no Emerging target; no Platinum doublet † † † Osimertinib monotherapy S Afatinib monotherapy M M Alectinib S Entrectinib M Dabrafenib/trametinib M Capmatinib M Selpercatinib M Entrectinib M conclusions available conclusions available conclusions available chemotherapy ± bevacizumab Gefitinib with doublet Standard treatment based on Standard treatment based on Standard treatment based on M M M Brigatinib S Crizotinib M M Tepotinib M Pralsetinib* W Larotrectinib M chemotherapy non-driver mutation guideline non-driver mutation guideline non-driver mutation guideline If alectinib or brigatinib are not available If entrectinib or crizotinib are not available Standard treatment based on Standard treatment based on Standard treatment based on Dacomitinib monotherapy M Osimertinib W M M M Ceritinib S Ceritinib W non-driver mutation guideline non-driver mutation guideline non-driver mutation guideline Monotherapy with afatinib M Crizotinib S Lortlatinib W Standard treatment based on Erlotinib/ramucirumab M M non-driver mutation guideline Erlotinib/bevacizumab M Monotherapy with erlotinib M Strength of Recommendation Monotherapy with gefitinib M S Strong M Moderate W Weak Monotherapy with icotinib M Notes. -
CDER Breakthrough Therapy Designation Approvals Data As of December 31, 2020 Total of 190 Approvals
CDER Breakthrough Therapy Designation Approvals Data as of December 31, 2020 Total of 190 Approvals Submission Application Type and Proprietary Approval Use Number Number Name Established Name Applicant Date Treatment of patients with previously BLA 125486 ORIGINAL-1 GAZYVA OBINUTUZUMAB GENENTECH INC 01-Nov-2013 untreated chronic lymphocytic leukemia in combination with chlorambucil Treatment of patients with mantle cell NDA 205552 ORIGINAL-1 IMBRUVICA IBRUTINIB PHARMACYCLICS LLC 13-Nov-2013 lymphoma (MCL) Treatment of chronic hepatitis C NDA 204671 ORIGINAL-1 SOVALDI SOFOSBUVIR GILEAD SCIENCES INC 06-Dec-2013 infection Treatment of cystic fibrosis patients age VERTEX PHARMACEUTICALS NDA 203188 SUPPLEMENT-4 KALYDECO IVACAFTOR 21-Feb-2014 6 years and older who have mutations INC in the CFTR gene Treatment of previously untreated NOVARTIS patients with chronic lymphocytic BLA 125326 SUPPLEMENT-60 ARZERRA OFATUMUMAB PHARMACEUTICALS 17-Apr-2014 leukemia (CLL) for whom fludarabine- CORPORATION based therapy is considered inappropriate Treatment of patients with anaplastic NOVARTIS lymphoma kinase (ALK)-positive NDA 205755 ORIGINAL-1 ZYKADIA CERITINIB 29-Apr-2014 PHARMACEUTICALS CORP metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib Treatment of relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients NDA 206545 ORIGINAL-1 ZYDELIG IDELALISIB GILEAD SCIENCES INC 23-Jul-2014 for whom rituximab alone would be considered appropriate therapy due to other co-morbidities -
2020 Master Class Course Best Practices and Expanding Treatment Options in Soft Tissue Sarcomas Including GIST George D
2020 Master Class Course Best Practices and Expanding Treatment Options in Soft Tissue Sarcomas including GIST George D. Demetri, MD FACP FASCO 1 George D. Demetri, MD Faculty Disclosure • Scientific consultant with sponsored research to Dana-Farber: Bayer, Pfizer, Novartis, Epizyme, Roche/Genentech, Epizyme, LOXO Oncology, AbbVie, GlaxoSmithKline, Janssen, PharmaMar, Daiichi-Sankyo, AdaptImmune • Scientific consultant: GlaxoSmithKline, EMD-Serono, Sanofi, ICON plc, MEDSCAPE, Mirati, WCG/Arsenal Capital, Polaris, MJ Hennessey/OncLive, C4 Therapeutics, Synlogic, McCann Health • Consultant/SAB member with minor equity holding: G1 Therapeutics, Caris Life Sciences, Erasca Pharmaceuticals, RELAY Therapeutics, Bessor Pharmaceuticals, Champions Biotechnology, Caprion/HistoGeneX • Board of Directors member and Scientific Advisory Board Consultant with minor equity holding: Blueprint Medicines, Translate BIO • Patents/Royalties: Novartis royalty to Dana-Farber for use patent of imatinib in GIST • Non-Financial Interests: AACR Science Policy and Government Affairs Committee Chair, Alexandria Real Estate Equities, Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off label or investigational uses (any uses not approved by the FDA) of products or devices. 2 Soft Tissue Sarcomas: Approximate Anatomic Distribution Head and Neck 10% Upper extremities 15% Trunk 10% Retroperitoneal, pelvic and visceral Lower extremities 15% 50% (includes GIST and GYN sarcomas) Modified from Clark MA et al. NEJM 2005;353:701-11 3 Sarcomas represent an exceedingly diverse set of diseases Bone Sarcomas 10% GIST Un- differentiated Liposarcomas Leiomyo- Sarcomas 4 Ducimetiere et al. -
Overcoming MET-Dependent Resistance to Selective RET Inhibition in Patients with RET Fusion–Positive Lung Cancer by Combining Selpercatinib with Crizotinib a C Ezra Y
Published OnlineFirst October 20, 2020; DOI: 10.1158/1078-0432.CCR-20-2278 CLINICAL CANCER RESEARCH | RESEARCH BRIEFS: CLINICAL TRIAL BRIEF REPORT Overcoming MET-Dependent Resistance to Selective RET Inhibition in Patients with RET Fusion–Positive Lung Cancer by Combining Selpercatinib with Crizotinib A C Ezra Y. Rosen1, Melissa L. Johnson2, Sarah E. Clifford3, Romel Somwar4, Jennifer F. Kherani5, Jieun Son3, Arrien A. Bertram3, Monika A. Davare6, Eric Gladstone4, Elena V. Ivanova7, Dahlia N. Henry5, Elaine M. Kelley3, Mika Lin3, Marina S.D. Milan3, Binoj C. Nair5, Elizabeth A. Olek5, Jenna E. Scanlon3, Morana Vojnic4, Kevin Ebata5, Jaclyn F. Hechtman4, Bob T. Li1,8, Lynette M. Sholl9, Barry S. Taylor10, Marc Ladanyi4, Pasi A. Janne€ 3, S. Michael Rothenberg5, Alexander Drilon1,8, and Geoffrey R. Oxnard3 ABSTRACT ◥ Purpose: The RET proto-oncogene encodes a receptor tyrosine Results: MET amplification was identified in posttreatment kinase that is activated by gene fusion in 1%–2% of non–small biopsies in 4 patients with RET fusion–positive NSCLC treated with cell lung cancers (NSCLC) and rarely in other cancer types. selpercatinib. In at least one case, MET amplification was clearly Selpercatinib is a highly selective RET kinase inhibitor that has evident prior to therapy with selpercatinib. We demonstrate that recently been approved by the FDA in lung and thyroid cancers increased MET expression in RET fusion–positive tumor cells causes with activating RET gene fusions and mutations. Molecular resistance to selpercatinib, and this can be overcome by combining mechanisms of acquired resistance to selpercatinib are poorly selpercatinib with crizotinib. Using SPPs, selpercatinib with crizo- understood. -
CHMP Agenda of the 19-22 April 2021 Meeting
28 July 2021 EMA/CHMP/220334/2021 Corr.11 Human Medicines Division Committee for medicinal products for human use (CHMP) Agenda for the meeting on 19-22 April 2021 Chair: Harald Enzmann – Vice-Chair: Bruno Sepodes 19 April 2021, 09:00 – 19:30, virtual meeting/ room 1C 20 April 2021, 08:30 – 19:30, virtual meeting/ room 1C 21 April 2021, 08:30 – 19:30, virtual meeting/ room 1D 22 April 2021, 08:30 – 19:00, virtual meeting/ room 1C Disclaimers Some of the information contained in this agenda is considered commercially confidential or sensitive and therefore not disclosed. With regard to intended therapeutic indications or procedure scopes listed against products, it must be noted that these may not reflect the full wording proposed by applicants and may also vary during the course of the review. Additional details on some of these procedures will be published in the CHMP meeting highlights once the procedures are finalised and start of referrals will also be available. Of note, this agenda is a working document primarily designed for CHMP members and the work the Committee undertakes. Note on access to documents Some documents mentioned in the agenda cannot be released at present following a request for access to documents within the framework of Regulation (EC) No 1049/2001 as they are subject to on- going procedures for which a final decision has not yet been adopted. They will become public when adopted or considered public according to the principles stated in the Agency policy on access to documents (EMA/127362/2006). 1 Correction in section 8.1.1 Official address Domenico Scarlattilaan 6 ● 1083 HS Amsterdam ● The Netherlands Address for visits and deliveries Refer to www.ema.europa.eu/how-to-find-us Send us a question Go to www.ema.europa.eu/contact Telephone +31 (0)88 781 6000 An agency of the European Union © European Medicines Agency, 2021. -
Rxoutlook® 1St Quarter 2019
® RxOutlook 1st Quarter 2020 optum.com/optumrx a RxOutlook 1st Quarter 2020 Orphan drugs continue to feature prominently in the drug development pipeline In 1983 the Orphan Drug Act was signed into law. Thirty seven years later, what was initially envisioned as a minor category of drugs has become a major part of the drug development pipeline. The Orphan Drug Act was passed by the United States Congress in 1983 in order to spur drug development for rare conditions with high unmet need. The legislation provided financial incentives to manufacturers if they could demonstrate that the target population for their drug consisted of fewer than 200,000 persons in the United States, or that there was no reasonable expectation that commercial sales would be sufficient to recoup the developmental costs associated with the drug. These “Orphan Drug” approvals have become increasingly common over the last two decades. In 2000, two of the 27 (7%) new drugs approved by the FDA had Orphan Designation, whereas in 2019, 20 of the 48 new drugs (42%) approved by the FDA had Orphan Designation. Since the passage of the Orphan Drug Act, 37 years ago, additional regulations and FDA designations have been implemented in an attempt to further expedite drug development for certain serious and life threatening conditions. Drugs with a Fast Track designation can use Phase 2 clinical trials to support FDA approval. Drugs with Breakthrough Therapy designation can use alternative clinical trial designs instead of the traditional randomized, double-blind, placebo-controlled trial. Additionally, drugs may be approved via the Accelerated Approval pathway using surrogate endpoints in clinical trials rather than clinical outcomes.