Month: August 2017

Immune Design Announces New CMB305 + Checkpoint Inhibitor Topline Data from an Upcoming Presentation at the ESMO 2017 Congress

On August 30, 2017 Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, reported positive topline data from its interim analysis of the ongoing, randomized Phase 2 trial evaluating CMB305 in combination with atezolizumab (TECENTRIQ) or atezolizumab alone in 88 soft tissue sarcoma patients (Press release, Immune Design, AUG 30, 2017, View Source [SID1234520357]). The data will be presented in a poster discussion session at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress, September 8-12, 2017 in Madrid, Spain.

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"The two main goals of this study are (1) to determine whether combining a next-generation vaccine like CMB305 with a checkpoint inhibitor (such as an anti-PD-L1 antibody) provides improved clinical benefit compared to that of the checkpoint inhibitor alone, particularly in a PD-L1-low or -negative tumor, and (2) in a randomized setting, to determine the biological activity of CMB305," said Carlos Paya, M.D., Ph.D., President and Chief Executive Officer of Immune Design. "We believe these interim data constitute the first steps towards answering both questions in a favorable manner."

Data to be presented at ESMO (Free ESMO Whitepaper) build upon those data on the first 36 patients summarized in the abstract, and include a greater number of patients enrolled.

Clinical Benefit: Data from the larger patient population show that patients receiving CMB305 and atezolizumab experienced greater clinical benefit in the form of Disease Control Rate (including objective responses), Progression Free Survival and Time to Next Treatment than those receiving atezolizumab alone.
Immune Response: Patients who received the combination of CMB305 and atezolizumab demonstrated an increased frequency of induced immune responses to NY-ESO-1, including NY-ESO-1-specific T cells, NY-ESO-1 antibodies, and antigen spreading, in comparison to patients who received atezolizumab alone
Biomarkers: In an exploratory analysis, a trend towards improved overall survival was observed in patients in the CMB305 and atezolizumab combination arm who had pre-existing and treatment-induced anti-NY-ESO-1 immunity, compared to the atezolizumab alone arm. Pre-treatment tumor biopsy analysis showed negligible levels of PD-L1 expression.
Safety: No new safety signals have been observed in either arm.
The fully enrolled trial is evaluating the safety, immunogenicity and efficacy of CMB305 in combination with atezolizumab, or atezolizumab alone, in a total of 88 patients with locally advanced, relapsed, or metastatic NY-ESO-1+ synovial sarcoma or myxoid/round-cell liposarcoma. Atezolizumab is a monoclonal antibody designed to target and bind to a protein called PD-L1 (programmed death ligand-1) and is being developed by Genentech, a member of the Roche Group. The trial is being conducted pursuant to a clinical collaboration with Genentech. Immune Design intends to present survival data in 2018 once all patients have at least one year of follow up (current data are preliminary: median duration of observation

The ESMO (Free ESMO Whitepaper) Poster Discussion session presentation details are as follows:

A Phase 2 Study of CMB305 and Atezolizumab in NY-ESO-1+ Soft Tissue Sarcoma: Interim Analysis of Immunogenicity, Tumor Control and Survival
Abstract # 1480PD
Session Title: Sarcoma Poster Discussion Session
Date: Monday, Sept. 11, 2017
Time: 11 a.m. — 12:30 p.m.
Location: Bilbao Auditorium
Poster Presenter: Dr. Sant Chawla
Poster Discussant: Dr. Robert Maki

About CMB305

CMB305 is a prime-boost vaccine approach against NY-ESO-1-expressing tumors, designed to generate an integrated, anti-NY-ESO-1 immune response in vivo via a targeted, specific interaction with dendritic cells, a mechanism of action Immune Design believes differs from traditional cancer vaccines. CMB305 is being evaluated in soft tissue sarcoma patients in ongoing Phase 1 monotherapy and 2 combination studies. Immune Design has received Orphan Drug Designation for CMB305 from the U.S. Food and Drug Administration (FDA) for the treatment of soft tissue sarcoma, as well as from the FDA and European Medicines Agency for each of the components of CMB305 for the treatment of soft tissue sarcoma.

Stemline Therapeutics to Present SL-801 Phase 1 Data at Upcoming ESMO Congress

On August 30, 2017 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported that data from SL-801’s ongoing Phase 1 trial in patients with advanced solid tumors has been selected by the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) for poster presentation at their Annual Congress 2017, to be held September 8-12, 2017 in Madrid, Spain (Press release, Stemline Therapeutics, AUG 30, 2017, View Source [SID1234520358]). SL-801 is a novel, potent and reversible Exportin-1 (XPO1) inhibitor.

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Details on the presentation are as follows:

Title: Ongoing Phase 1 Trial of SL-801, a Novel XPO-1 Inhibitor, in Patients with Advanced Solid Tumors; Interim Results
First Author: Judy Wang, MD; Florida Cancer Specialists & Research Institute
Abstract: 406P

Date/Time: Monday, September 11, 2017 – 1:15-2:15 PM CET
Location: Hall 8

Karyopharm Announces the Presentation of Selinexor and KPT-9274 Clinical Data at the European Society of Medical Oncology 2017 Annual Meeting

On August 30, 2017 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported two upcoming poster presentations at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Annual Meeting, taking place September 8-12, 2017 in Madrid, Spain (Press release, Karyopharm, AUG 30, 2017, View Source [SID1234520355]). One poster will describe Phase 1 data from an ongoing investigator-sponsored trial (IST) evaluating the safety and tolerability of the Company’s lead product candidate, selinexor (KPT-330), an oral Selective Inhibitor of Nuclear Export / SINE compound, in combination with paclitaxel and carboplatin in patients with advanced ovarian or endometrial cancers. The other poster will describe Phase 1 clinical data from an ongoing study evaluating the safety and tolerability of KPT-9274, Karyopharm’s oral, dual inhibitor of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT) in patients with advanced solid malignancies or non-Hodgkin’s lymphoma (NHL).

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"We look forward to the presentations of the findings from both of these early-stage clinical trials at ESMO (Free ESMO Whitepaper) this year, the results of which will continue to inform and guide our ongoing clinical programs," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm.

Details for the Poster Presentations at ESMO (Free ESMO Whitepaper) 2017:

Title: A Phase 1 Study of Selinexor (S) in Combination with Paclitaxel (P) and Carboplatin (C) in Patients (pts) with Advanced Ovarian (OC) or Endometrial Cancers (EC)
Presenter: Vicky Makker, Memorial Sloan Kettering Cancer Center, New York, New York, USA
Abstract code: 970P
Session: Poster Display Session
Location: Hall 8
Date and Time: Saturday, September 9, 2017 from 13:15 – 14:15 CET

Title: A First in Human Phase 1 Study of KPT-9274, a First in Class Dual Inhibitor of PAK4 and NAMPT, in Patients with Advanced Solid Malignancies or NHL
Presenter: Aung Niang, MD Anderson Cancer Center, Houston, Texas, USA
Abstract code: 374PD
Session: Poster Discussion Session — Developmental Therapeutics
Location: Alicante Auditorium
Date and Time: Saturday, September 9, 2017 from 16:30 – 18:00 CET

About Selinexor

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 2,100 patients have been treated with selinexor and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform the Company’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

About KPT-9274

KPT-9274 is a first-in-class, orally bioavailable, small molecule immunometabolic modulator that works through non-competitive dual inhibition of p21-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase (NAMPT). Co-inhibition of these targets is believed to lead to synergistic anti-tumor effects through suppression of ß-catenin by blocking PAK4, leading to both immune cell activation and inhibition of tumor growth, energy depletion through NAMPT inhibition, blockade of DNA repair, cell cycle arrest and ultimately apoptosis. KPT-9274 may therefore have both immune-activating and direct antitumor effects. In contrast, normal cells are less sensitive to inhibition by KPT-9274 due in part to their relative genomic stability and lower metabolic demands.

Roche to present new data from its oncology portfolio at the 2017 European Society for Medical Oncology (ESMO) Congress

On August 30, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that new results from a number of studies across 18 approved and investigational medicines will be presented during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress from 8-12 September in Madrid, Spain. These include phase III results from Roche’s targeted medicines portfolio, including Zelboraf and Alecensa, updates from our cancer immunotherapy development programme across multiple tumour types and important new insights into the biology of cancer that will help further our understanding of this highly complex disease.

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"Progress against cancer is accelerating as our understanding of the disease is enhanced by more sophisticated diagnostics, leading to increasingly tailored treatment approaches," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "Continuing this progress will require both incremental and ground-breaking advancements as we collectively pursue cures for the many types of this incredibly complex disease. At ESMO (Free ESMO Whitepaper), we are proud to share data from across our broad cancer programme and we look ahead to an unprecedented number of key milestones over the next 12 months."

First results from the phase III BRIM8 study of Zelboraf for adjuvant (after surgery) treatment of BRAF V600 mutation-positive melanoma will be presented during ESMO (Free ESMO Whitepaper)’s Presidential Symposium on Monday 11 September 2017.

Additional highlights from the Roche oncology portfolio include new data from the phase III ALEX study investigating Alecensa in anaplastic lymphoma kinase (ALK)-positive NSCLC in the first-line setting. These new data further characterise the impact of Alecensa treatment on lung cancer that has the potential to, or has, spread to the central nervous system, building on the study’s positive primary results that were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting earlier this year. In addition, Roche will present new results from the phase III ALUR study of Alecensa in ALK-positive advanced NSCLC that compared Alecensa to chemotherapy in people who had been previously treated with chemotherapy as well as crizotinib.

Results from the phase II LORELEI study1 evaluating neoadjuvant (pre-surgery) use of taselisib in HER2-negative/oestrogen receptor (ER)-positive early breast cancer will provide additional insights into the potential role of PI3K inhibition in HER2-negative/ER-positive disease. These data from BRIM8, ALEX, ALUR, and LORELEI are included in the official ESMO (Free ESMO Whitepaper) press programme.

Roche will also present updates on the progress made in the understanding of the biology and immunology of cancer, including data generated in collaboration with Foundation Medicine. At ESMO (Free ESMO Whitepaper), data will be presented for the first time for a blood-based assay that is used to measure tumour mutational burden (TMB). Measuring TMB by comprehensive genomic profiling summarises how many mutations are present in a person’s tumour and may be a way to predict responses to certain cancer immunotherapies. The validation study being presented at ESMO (Free ESMO Whitepaper) was conducted using samples from the phase II POPLAR and phase III OAK studies of TECENTRIQÒ (atezolizumab) and provides initial, retrospective evidence of an association between TMB in the blood (bTMB) and TECENTRIQ activity. These early data will inform ongoing and future prospective research to better understand the role of both TMB and bTMB as it relates to treatment with cancer immunotherapy.

Follow Roche on Twitter via @Roche and keep up to date with ESMO (Free ESMO Whitepaper) 2017 congress news and updates by using the hashtag #ESMO17.

For more information on Roche’s approach to cancer, visit Roche.com.
Overview of key presentations featuring Roche medicines at ESMO (Free ESMO Whitepaper) 2017
About Roche in Oncology

Roche has been working to transform cancer care for more than 50 years, bringing the first specifically designed anti-cancer chemotherapy drug, fluorouracil, to patients in 1962. Roche’s commitment to developing innovative medicines and diagnostics for cancers remains steadfast.

The Roche Group’s portfolio of innovative cancer medicines includes: Alecensa (alectinib); Avastin (bevacizumab); Cotellic (cobimetinib); Erivedge (vismodegib); Gazyva/Gazyvaro (obinutuzumab); Herceptin (trastuzumab); Kadcyla (trastuzumab emtansine); MabThera/Rituxan (rituximab); Perjeta (pertuzumab); Tarceva (erlotinib); TECENTRIQ (atezolizumab); Venclexta/Venclyxto (venetoclax); Xeloda (capecitabine); Zelboraf (vemurafenib). Furthermore, the Roche Group has a robust investigational oncology pipeline focusing on new therapeutic targets and novel combination strategies.

In addition to Roche’s innovative portfolio of cancer medicines, Roche is constantly developing new diagnostic tests that will have a significant impact on disease management for cancer patients. Within the Roche Group there are more than 350 pharmaceutical and diagnostic collaborations, far more than half of which are in the field of oncology.

With a broad portfolio of tumour markers for prostate, colorectal, liver, ovarian, breast, stomach, pancreatic and lung cancer, as well as a range of tissue and molecular oncology tests that contribute to personalised cancer care today, Roche is leading a new era of innovation in the fight against cancer.

Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah(TM) (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice

On August 30, 2017 – Novartis announced today that the US Food and Drug Administration (FDA) has approved Kymriah(TM)(tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, the first chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient’s own T cells to fight cancer (Press release, Novartis, AUG 30, 2017, View Source [SID1234520344]). Kymriah is the first therapy based on gene transfer approved by the FDA.

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"At Novartis, we have a long history of being at the forefront of transformative cancer treatment," said Joseph Jimenez, CEO of Novartis. "Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care."

"We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program," said Bruno Strigini, CEO of Novartis Oncology. "As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated."

The FDA has approved a Risk Evaluation and Mitigation Strategy (REMS) for Kymriah. The REMS program serves to inform and educate healthcare professionals about the risks that may be associated with Kymriah treatment. To support safe patient access, Novartis is establishing a network of certified treatment centers throughout the country which will be fully trained on the use of Kymriah and appropriate patient care.

There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory (r/r) B-cell precursor ALL, whose prognosis is poor. Patients often undergo multiple treatments including chemotherapy, radiation, targeted therapy or stem cell transplant, yet less than 10% of patients survive five years [2], [3].

Kymriah is an innovative immunocellular therapy that is a one-time treatment. Kymriah uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence. In 2012, Novartis and the University of Pennsylvania (Penn) entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

"This therapy is a significant step forward in individualized cancer treatment that may have a tremendous impact on patients’ lives," said Carl June, MD, the Richard W. Vague Professor of Immunotherapy, Director of the Center for Cellular Immunotherapies in Penn’s Perelman School of Medicine, who is a pioneer of this new treatment. "Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types."

In close collaboration with Novartis and Penn, Children’s Hospital of Philadelphia (CHOP) was the first institution to investigate Kymriah in the treatment of pediatric patients leading the single site trial.

"Tisagenlecleucel is the first CAR-T therapy to demonstrate early, deep and durable remission in children and young adults with relapsed or refractory B-cell ALL," said Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at Penn, and Director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia (CHOP). "We’ve never seen anything like this before and I believe this therapy may become the new standard of care for this patient population."

Novartis is committed to ensuring eligible patients have access to Kymriah, and is working to ensure payers understand the value of Kymriah and provide coverage for patients. To address the unique aspects of the therapy, Novartis has also developed various patient access programs to support safe and timely access for patients. Novartis is also providing traditional support to patients by helping them navigate insurance coverage and providing financial assistance for those who are uninsured or underinsured.

Novartis plans additional filings for Kymriah in the US and EU later this year, including applications with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Additional filings beyond the US and EU are anticipated in 2018.

Groundbreaking Collaboration with the Centers for Medicare and Medicaid Services
Novartis also announced a novel collaboration with the United States Centers for Medicare and Medicaid Services (CMS) focused on improving efficiencies in current regulatory requirements in order to deliver value-based care and ensure access for this specific patient population.

This approach is intended to include indication-based pricing for medicines and supports payments for a medicine, such as Kymriah for its initial indication, based on the clinical outcomes achieved, which would eliminate inefficiencies from the healthcare system. Other value-based approaches related to future indications for Kymriah and CAR-T cell therapies are under discussion.

Furthermore, Novartis is collaborating with CMS to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach.

"Novartis has been at the forefront of outcomes-based pricing and is very pleased to work with CMS on this first-of-its-kind collaboration with a technology that has the potential to transform cancer care," said Joseph Jimenez, CEO of Novartis. "We look forward to continuing to work with CMS to potentially expand this approach to other products and disease states."