Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit…

TOKYO--(BUSINESS WIRE)--SanBio Co., Ltd. (headquarters: Chuo-ku, Tokyo, Representative Director and President: Keita Mori, hereafter SanBio) hereby announces that it has obtained new analytical results from the Phase 2b clinical trial (the trial) of SB623 for the treatment of chronic motor deficit resulting from ischemic stroke the SanBio Group (SanBio Co., Ltd. and its subsidiary SanBio, Inc.) conducted in the US. It also announces that based on the newly obtained results, it has updated its development plans, including in regard to late-stage clinical trials for the ischemic stroke and hemorrhagic stroke programs of SB623 in Japan.

The trial evaluated efficacy and safety of SB623 in 163 patients suffering from chronic motor dysfunction from ischemic stroke. On January 29, 2019, SanBio announced that the trial did not meet its primary endpoint, as it failed to demonstrate statistical significance in the difference in the proportion of patients whose Fugl-Meyer Motor Scale (FMMS) score improved by 10 or more points from the baseline (primary endpoint) between the treatment group that received SB623 and the control group. Since then, the SanBio Group had continued to work on additional analysis of the trial data, and results of the additional analysis are as follows.

In conducting the additional analysis, from the perspective of minimal clinically important difference (MCID, or the minimal change in scores or other metrics that could be interpreted to mean the change in a patient is clinically meaningful) and based on the results of the Phase 2 clinical trial of SB623 for the treatment of chronic motor deficit from traumatic brain injury (TBI; STEMTRA trial), the company reevaluated trial data using composite FMMS. Of the total 163 patients enrolled in the trial, the company specifically looked at 77 patients who had infarct areas smaller than a certain size (47% of all patients enrolled in this trial). The SanBio Group evaluated the proportion of patients that met one or more of the following FMMS score improvement criteria 24 weeks after treatment: 6-point improvement on FMMS score for upper extremity, 4-point improvement on FMMS score for lower extremity, and 9-point improvement on FMMS total score (all from the baseline). Of the 51 patients in the treatment group that received SB623, improvement was seen in 49%, versus in 19% of 26 patients in the control group that received sham surgery, the difference between the two groups being statistically significant (p-value of 0.02). SanBio Group thinks that even compared to the primary endpointthe proportion of patients whose FMMS score improved by 10 or more points over the baseline six months after treatmentthe endpoint using composite FMMS can adequately explain clinical significance of the treatment efficacy. Details of the additional analysis results will be announced at the financial results briefing for institutional investors and the media held on September 15, 2020. The briefing video will be made available to the public on our website on the 16th of September or thereafter.

Based on the above results, the SanBio Group has begun preparations for the next late-stage clinical trials in the ischemic stroke and hemorrhagic stroke programs of SB623. 2021. Specific designs of the clinical trials and the contents of development for those two programs will be announced promptly upon being finalized. To maximize the value of SB623 at an early stage by selecting areas to focus the Groups management resources on, the SanBio Group plans to prioritize the development of the ischemic stroke and hemorrhagic stroke programs in Japan at the same time as it prepares to file for approval of SB623 for the treatment of chronic motor deficit resulting from TBI in Japan by the end of the current fiscal year (ending January 2021). The Group, however, postponed the global Phase 3 clinical trial for the TBI program of SB623 it had planned to commence this fiscal year to the next or subsequent fiscal years.

Many patients suffering from the chronic effects of ischemic stroke are said to be regularly taking drugs to prevent recurrence. However, because there is no drug that can fundamentally cure motor dysfunction, there is high unmet need for therapeutic drugs to restore motor functions for patients in the chronic phase of stroke. The SanBio Group aims to contribute to improving the lives of these patients, as well as of their family members, suffering from motor impairment and difficulties it causes in carrying out their daily lives through SB623.

About SB623

SB623 is an allogeneic mesenchymal stem cell produced by modifying and culturing bone marrow derived from healthy donors. Implantation of SB623 cells into nerve tissues is expected to promote regeneration of damaged nerve cells. Because SB623 is made from allogeneic cells, large-scale production is possible and there is no need for complex cell processing required for treatments using autologous cells, e.g., cell preparation for each patient at medical institutions. Hence, pharmaceutical products made from allogeneic cells, such as SB623, can be provided to many patients in uniform quality.

About SanBio Co., Ltd. and SanBio, Inc.

SanBio Group is engaged in the regenerative cell medicine business, spanning research, development, manufacture, and sales of regenerative cell medicines. The Companys propriety regenerative cell medicine product, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from traumatic brain injury and ischemic stroke. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

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Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit...

Outcomes After Daratumumab Addition to Induction Therapy in Newly Diagnosed Multiple Myeloma – Hematology Advisor

The addition of daratumumab (D) to standard frontline lenalidomide, bortezomib, and dexamethasone (RVd) induction therapy prior to autologous stem cell transplantation (ASCT) yielded higher rates of stringent complete response (sCR) and minimal residual disease (MRD) negativity compared with RVd in patients with newly diagnosed multiple myeloma (MM), according to results from the phase 2 GRIFFIN trial (ClinicalTrials.gov Identifier, NCT02874742), which was published in Blood.

Eligible patients with newly diagnosed MM (207 patients) were randomly assigned (1:1) to receive 4 cycles of D-RVd or RVd induction, 2 cycles of ASCT, D-RVd or RVd consolidation, and 26 cycles of lenalidomide plus D or lenalidomide maintenance. The primary endpoint was sCR rate by the end of post-ASCT consolidation.

The median patient age was 59 years (range, 29-70 years) in the D-RVd arm and 61 years (range, 40-70 years) in the RVd arm. Other patient and disease characteristics were also well balanced among the arms.

The sCR rate by the end of post-ASCT consolidation was higher in the D-RVd arm compared with the RVd arm (42.4% vs 32.0%; odds ratio, 1.57; 95% CI, 0.87-2.82; 1-sided P =.068; meeting the prespecified 1-sided a of 0.10). The rate of MRD negativity (10-5 threshold) was also higher in the D-RVd arm compared with the RVd arm (21.2% vs 5.8%; P =.0019) in the intent-to-treat population.

At a median follow up of 22.1 months, the responses deepened in both arms. The sCR rates improved to 62.6% for D-RVd and 45.4% for RVd (P =.0177); the MRD negativity rates also improved (51.0% vs 20.4%, respectively; P <.0001).

Neither median progression-free survival (PFS) nor overall survival were reached in either arm. The Kaplan-Meier estimate of the 24-month PFS rates were 95.8% and 89.8% in the D-RVd and RVd arms, respectively. Disease progression occurred in 3.8% and 6.8% of patients in the D-RVd (4 patients) and RVd arm (7 patients), respectively.

No new safety concerns were reported. Grade 3/4 hematologic adverse events were more common with D-RVd compared with RVd (neutropenia, 41.4% vs 21.6%; lymphopenia, 23.2% vs 21.6%; thrombocytopenia, 16.2% vs 8.8%; leukopenia, 16.2% vs 6.9%; anemia, 9.1% vs 5.9%). Infections were more common with D-RVd compared with RVd (90.9% vs 61.8%); however, grade 3/4 infection rates were similar between the arms (23.2% vs 21.6%).

Study results from GRIFFIN are promising and practice informing; this randomized phase 2 study was designed to expediently provide efficacy and safety information on a new regimen of great interest to myeloma clinicians, wrote the authors.

These results provide a support for the ongoing phase 3 PERSEUS registration study (ClinicalTrials.gov Identifier: NCT03710603), which is assessing PFS in transplant-eligible patients with newly diagnosed MM receiving D-RVd or RVd.

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Voorhees PM, Kaufman JL, Laubach JP, et al. Daratumumab, Lenalidomide, Bortezomib, & Dexamethasone for Transplant-eligible Newly Diagnosed Multiple Myeloma: GRIFFIN. Blood. 2020;136(8):936-945. doi:10.1182/blood.2020005288

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Outcomes After Daratumumab Addition to Induction Therapy in Newly Diagnosed Multiple Myeloma - Hematology Advisor

What Certain Patients with DLBCL Need To Know About Monjuvi-Revlimid – Curetoday.com

The combination of Monjuvi (tafasitamab-cxix) and Revlimid (lenalidomide) is a recently approved treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are unable to undergo autologous stem cell transplant, and while there are still questions to be answered about its sequencing, it may work best just after stopping a previous therapy, according to the lead researcher of the study that led to its approval.

In an interview with CURE, Dr. Gilles Salles, head of the hematology department of the Centre Hospitalier Lyon-Sud in France, discusses the key factors patients should know about the treatment and what physicians should discuss with them before they start taking the combination.

Transcription:

They should probably know that the sooner this form of therapy is installed, the better the result. That is classical in cancer, but when you are at the first failure of your previous line, it works a little bit better than when you use it later.

What they should know is that other agents have been approved, essentially in the third-line setting, and we have been talking about CAR-T cells. And CAR-T cells are genetically engineered immune cells, T cells that fight the disease. This is a great tool for patients. We don't know exactly at this time how we should optimally sequence, if needed, the combination of tafasitamab/lenalidomide and CAR-T cell, whether it's better to use one before, like tafasitamab, or whether it may or not diminish the potential efficacy of CAR-T cell in the future.

So, it's the ideas and research here, so we need to discuss that with patients. Other than that, what they should know is that, at the present time for patients that have got benefit of (therapy for) this disease, there is a continuous treatment. Tafasitamab is delivered as an IV infusion every two weeks, so they have to continue to come to the hospital every two weeks to receive an infusion.

But this infusion is given, it's usually much better tolerated when we go further down the road. We actually stop lenalidomide, the second drug, after one year and we use tafasitamab alone; that has much less side effect and the infusion lasts 90 minutes. And we do hope that continuing this treatment will prevent any recurrence of the disease.

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What Certain Patients with DLBCL Need To Know About Monjuvi-Revlimid - Curetoday.com

LAVA Therapeutics, Amsterdam UMC and Monash University Announce Publication of a Novel Cancer Immunotherapy Approach in Nature Cancer – Business Wire

UTRECHT, The Netherlands & PHILADELPHIA--(BUSINESS WIRE)--LAVA Therapeutics B.V., a biotech company pioneering bispecific gamma-delta T cell engagers for cancer, Amsterdam UMC and the Monash Biomedicine Discovery Institute in Australia, today announced that Nature Cancer has published their co-authored paper titled, A single domain bispecific antibody targeting CD1d and the NKT T cell receptor induces a potent antitumor response, detailing a novel immune-oncology approach for the potential treatment of cancer.

Findings in the paper highlight the potential for a LAVA-derived antibody fragment, known as a single domain antibody, that acts as a bridge to link together two key immune cell receptors the CD1d receptor and the T cell receptor of natural killer T (NKT) cells in order to enhance the bodys immune response to cancer. The CD1d receptor is frequently expressed by tumor cells and NKT cells are positioned at the interface between the innate and adaptive immune system and play an important role in the host-rejection of both tumors and virally infected cells. Preclinical research through the companies collaboration demonstrates that a CD1d-NKT cell targeting antibody resulted in significant activation of NKT cells and the subsequent killing of CD1d-expressing tumor cells in multiple tumor samples, including multiple myeloma and acute myeloid leukemia.

These data underscore the potential of LAVAs bispecific antibody approach to target and activate NKT cells for the treatment of cancer, said Hans van der Vliet, professor in medical oncology at Amsterdam UMC and chief scientific officer of LAVA Therapeutics. By targeting and boosting natural immune cells that are inherent in all humans, such as NKT and gamma-delta T cells, for an enhanced therapeutic effect, our approach could translate into a broadly applicable immunotherapeutic strategy for treating a range of cancer indications.

Using the Australian Synchrotron, the team at Monash University provided detailed atomic insight into how the single domain antibodies exerted their effect on immune cells in a cancer model. Through this, we were able to precisely visualize how the single domain antibody simultaneously recognized CD1d and the NKT T cell receptor, thereby providing a molecular basis for their anti-tumor properties, professor Jamie Rossjohn, Australian Research Council Laureate Fellow at Monash University stated. The collaboration with LAVA Therapeutics on this ground-breaking approach was very effective.

Instrumental to the study were joint first authors Dr. Roeland Lameris from Amsterdam UMC and Dr. Adam Shahine from Monash University.

About LAVA Therapeutics

LAVA Therapeutics, B.V., is developing a proprietary bispecific antibody platform that engages gamma-delta T cells for the treatment of hematological and solid cancers. The companys first-in-class immuno-oncology approach activates V9V2 T cells upon binding to membrane-expressed tumor targets. LAVA was founded in 2016 based on intellectual property originating from Hans van der Vliet`s group at the Amsterdam UMC, and is backed by Lupus Ventures, Biox Biosciences, Versant Ventures, Gilde Healthcare and MRL Ventures Fund. The company has established a highly experienced antibody research and development team located in Utrecht, the Netherlands (headquarters) and Philadelphia. For more information, please visit http://www.lavatherapeutics.com.

About the Monash Biomedicine Discovery Institute at Monash University

Committed to making the discoveries that will relieve the future burden of disease, the newly established Monash Biomedicine Discovery Institute at Monash University brings together more than 120 internationally-renowned research teams. Spanning six discovery programs across Cancer, Cardiovascular Disease, Development and Stem Cells, Infection and Immunity, Metabolic Disease and Obesity, and Neuroscience, Monash BDI is one of the largest biomedical research institutes in Australia. Our researchers are supported by world-class technology and infrastructure, and partner with industry, clinicians and researchers internationally to enhance lives through discovery.

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LAVA Therapeutics, Amsterdam UMC and Monash University Announce Publication of a Novel Cancer Immunotherapy Approach in Nature Cancer - Business Wire

Stem Cell Therapy Market To 2026: Growth Analysis By Manufacturers, Regions, Types And Applications – Kewaskum Statesman News Journal

IndustryGrowthInsights, one of the worlds prominent market research firms has announced a novel report on the Stem Cell Therapy market. The report is integrated with imperative insights on the market which will support the clients to make precise business decisions. This research will help both existing and new aspirants for Global Stem Cell Therapy Market to figure out and study market requirements, market size, and competition. The report incorporates data regarding the supply and demand situation, the competitive scenario, and the challenges for market growth, market opportunities, and the threats encountered by key players during the forecast period of 2020-2027.

Request a sample before buying this report @ https://industrygrowthinsights.com/request-sample/?reportId=168110

Impact of COVID-19

The report also incorporates the impact of the ongoing global crisis i.e. COVID-19 on the Stem Cell Therapy market and explains how the future is going to unfold for the global market. The report also provides an analysis of the effects of the pandemic on the global economy. The outbreak has directly affected production and demand disrupted the demand and supply chain. The report also computes the financial impact on firms and financial markets. IndustryGrowthInsights has accumulated insights from various delegates of the industry and got involved in the primary and secondary research to offer the clients data & strategies to combat the market challenges during and after the COVID-19 pandemic.

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Some of the major companies that are covered in this report:

Osiris Therapeutics NuVasive Chiesi Pharmaceuticals JCR Pharmaceutical Pharmicell Medi-post Anterogen Molmed Takeda (TiGenix) Stem Cell Therap

*Note: Additional companies can be included on request

The market scenario is likely to be fairly competitive. To analyze any market with simplicity the market is fragmented into the following segments:

By Application:

Musculoskeletal Disorder Wounds & Injuries Cornea Cardiovascular Diseases Others

By Type:

Autologous Allogeneic Stem Cell Therap

By Geographical Regions

Asia Pacific: China, Japan, India, and Rest of Asia Pacific Europe: Germany, the UK, France, and Rest of Europe North America: The US, Mexico, and Canada Latin America: Brazil and Rest of Latin America Middle East & Africa: GCC Countries and Rest of Middle East & Africa

Segmenting the market into smaller components helps in analyzing the dynamics of the market with more clarity. Another key component that is integrated into the report is the regional analysis to assess the global presence of the Stem Cell Therapy market. You can also opt for a yearly subscription of all the updates on the Stem Cell Therapy market.

Below is the TOC of the report:

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Stem Cell Therapy Market Overview

Global Stem Cell Therapy Market Analysis and Forecast by Type

Global Stem Cell Therapy Market Analysis and Forecast by Application

Global Stem Cell Therapy Market Analysis and Forecast by Sales Channel

Global Stem Cell Therapy Market Analysis and Forecast by Region

North America Stem Cell Therapy Market Analysis and Forecast

Latin America Stem Cell Therapy Market Analysis and Forecast

Europe Stem Cell Therapy Market Analysis and Forecast

Asia Pacific Stem Cell Therapy Market Analysis and Forecast

Asia Pacific Stem Cell Therapy Market Size and Volume Forecast by Application

Middle East & Africa Stem Cell Therapy Market Analysis and Forecast

Competition Landscape

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Stem Cell Therapy Market To 2026: Growth Analysis By Manufacturers, Regions, Types And Applications - Kewaskum Statesman News Journal

$14.6M Grant to Explore a Therapy to Control HIV Without Meds – POZ

In nearly 40 years of the HIV epidemic, only two people have likely been cured of the virus. Both scenarios resulted from stem cell transplants needed to fight blood cancers such as leukemia. Inspired by these two cases, a team of scientists is studying a multipronged way to potentially control HIV without medication. It involves two different genetic alterations of immune cells and with a safer method of stem cell transplants, also referred to as bone marrow transplants, a procedure that is generally toxic and dangerous.

The research is being funded by a five-year $14.6 million grant from the National Institutes of Health. The scientists coleading the preclinical studies are Paula Cannon, PhD, a distinguished professor of molecular microbiology and immunology at the Keck School of Medicine of the University of Southern California, and Hans-Peter Kiem, MD, PhD, who directs the stem cell and gene therapy program at the Fred Hutchinson Cancer Research Center, also known as Fred Hutch. According to a Keck School of Medicine press release, the two other main partners are David Scadden, MD, a bone marrow transplant specialist and professor at Harvard University and the Harvard Stem Cell Institute, and the biotechnology company Magenta Therapeutics.

In the HIV cure scenariosinvolving the so-called Berlin and London patientsboth men received stem cell transplants from donors with a natural genetic mutation that made them resistant to HIV. Specifically, their genes resulted in immune cells that lack CCR5 receptors on their surface (HIV latches onto these receptors to infect cells). Unfortunately, this method isnt viable for the nearly 38 million people worldwide living with HIV. Not only is it expensive, toxic and riskyit involves wiping out the patients immune system and replacing it with the new immune cellsbut it also requires matched donors who are CCR5 negative. According to the press release, about 1% of the population have this mutation.

With funding from this new grant, researchers hope to overcome these challenges in several ways. First, Cannon has already developed a gene-editing method to remove the CCR5 receptors from a patients own stem cells. She now hopes to further genetically engineer stem cells so they release antibodies that block HIV.

Our engineered cells will be good neighbors, Cannon said in the press release. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5 negative, have some chance of being protected.

Fred Hutchs Kiem will use CAR-T therapya new method of genetically modifying immune cells that is emerging out of cancer researchwith the goal of creating T cells that attack HIV-infected cells.

In addition, other scientists involved in the federal grant aim to develop less toxic methods of bone marrow transplantationfor example, by reducing the amount of chemotherapy required and speeding up the process of creating the new immune system.

The research finding could translate to other illnesses, such as cancer, sickle cell anemia and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

For the latest on the cure cases, see Famed London Man Probably Cured of HIV from earlier this year. And in related news, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

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$14.6M Grant to Explore a Therapy to Control HIV Without Meds - POZ

Talaris Therapeutics Announces Collaboration With Kentucky Organ Donor Affiliates to Advance Preclinical Study of Tolerance Induction to Organs From…

BOSTON & LOUISVILLE, Ky.--(BUSINESS WIRE)--Talaris Therapeutics, Inc., a privately held biotechnology company developing transformative cell therapies that have the potential to induce durable immune tolerance across a range of indications, today announced a collaboration with Kentucky Organ Donor Affiliates (KODA). KODA is an independent, non-profit organ and tissue procurement organization that facilitates deceased donor transplants throughout Kentucky, southern Indiana and western West Virginia. The collaboration will advance preclinical studies of the potential of Talaris allogeneic cell therapy to induce immune tolerance to an organ from a deceased donor.

Organ donation from deceased donors makes possible more than 80 percent of solid organ transplants in the U.S. These transplants are frequently lifesaving, but they bring the burden of lifelong immunosuppression for organ recipients, which puts patients at heightened risk of infection as well as a number of other potentially serious side effects. Additionally, immunosuppressant drugs are toxic to the kidneys over time, which can result in declining kidney function and necessitate another organ transplant.

Talaris novel cell therapy, FCR001, has shown promising potential to eliminate the need for immunosuppression among recipients of kidney transplants from living donors. Our collaboration with KODA will be an important step toward potentially extending this promise to recipients of organ transplants from deceased donors as well, said Scott Requadt, Chief Executive Officer of Talaris.

Within the collaboration, KODA, after authorization from the donors family, will recover vertebrae from deceased organ donors. Researchers at Talaris will then isolate stem cells from the vertebrae, with the goal of demonstrating the feasibility of producing FCR001 from vertebral bone marrow. These preclinical studies will serve as a first step toward enabling future clinical studies to evaluate whether FCR001 administered alongside organ transplantation can induce durable immune tolerance to an organ from a deceased donor.

The generosity organ donors and their families display is measureless. By initiating this collaboration with Talaris, we hope to further honor their gift by advancing an important treatment for transplant recipients with the potential to improve many individuals lives, said Julie Bergin, RN, BSN, MHA, President & Chief Executive Officer of KODA.

About Talaris Therapeutics

Talaris Therapeutics, Inc. is a late-clinical stage biotechnology company that is developing transformative cell therapies with the potential to eliminate the burden of chronic immunosuppression for organ transplant recipients as well as induce durable remissions in patients with severe auto-immune and immune-mediated disorders. Talaris was founded on technology discovered and developed by Dr. Suzanne Ildstad and operates its own cell processing facility in Louisville, KY. Talaris is backed by leading life sciences investors Blackstone Life Sciences, Longitude Capital and Qiming Venture Partners USA and maintains corporate offices in Boston, MA and Louisville, KY. http://www.TalarisTx.com.

About Kentucky Organ Donor Affiliates (KODA)

Kentucky Organ Donor Affiliates is dedicated to saving lives through organ and tissue donation and transplantation. KODA is an independent, federally designated, non-profit organ and tissue procurement organization formed in 1987. KODA was recognized by Louisville Business First as the Nonprofit of the Year in 2011. KODAs mission is to provide organ and tissues to those in need and to maintain a profound respect for those who gave. KODA serves 114 counties in Kentucky, 4 counties in southern Indiana and 2 counties in western West Virginia. For more information visit donatelifeky.org or call 1-800-525-3456.

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Talaris Therapeutics Announces Collaboration With Kentucky Organ Donor Affiliates to Advance Preclinical Study of Tolerance Induction to Organs From...

Alexion and Caelum Biosciences Announce Start of Phase 3 Studies of CAEL-101 in AL Amyloidosis – Business Wire

BOSTON & BORDENTOWN, N.J.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Caelum Biosciences today announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase 3 studies one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.

In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function. While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes, said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs. Data from Phase 1 studies suggest that this treatment approach may improve organ function and long-term survival. We look forward to investigating this further in the Phase 3 clinical program.

AL amyloidosis is particularly devastating when it affects the heart, with median survival in these patients of less than one year following diagnosis, said Michael Spector, President and Chief Executive Officer of Caelum. Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study showed that 78 percent were still alive after a median follow-up time of more than three years. We recognize the urgent need for new treatments that address the organ damage caused by AL amyloidosis and are working together with the AL amyloidosis community and Alexion to advance the Phase 3 clinical program as quickly as possible.

About the CARES Phase 3 Clinical Program

The CARES clinical program consists of two parallel double-blind, randomized, event-driven global Phase 3 studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and nave to standard of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). One study is enrolling approximately 260 patients with Mayo stage IIIa disease and one study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan, and Australia.

In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks.

The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain, or GLS).

Phase 2 Study Results

The Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.

Phase 1a/1b Long-Term Follow-Up Results Presented at ISA 2020

As previously reported, the Phase 1a/1b study of CAEL-101 was the first clinical trial to demonstrate improvement in cardiac function via GLS after treatment with an amyloid fibril targeted therapy in AL amyloidosis patients with amyloid cardiac involvement. New long-term follow-up data from the Phase 1a/1b study will be presented at the virtual International Symposium on Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled, Long term follow-up of patients with AL amyloidosis treated on a phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101 (Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University Medical Center). These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-P]

About Caelum Biosciences

Caelum Biosciences, Inc. (Caelum) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelums lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (AL) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.caelumbio.com.

Forward-Looking Statement

This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and Caelum, including statements related to: the safety and efficacy CAEL-101 as a treatment for AL amyloidosis; CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from the heart, kidney and other organs; data from the Phase 1 studies suggest that the treatment approach may improve organ function and long-term survival and enrollment of the Phase 3 trials. Forward-looking statements are subject to factors that may cause Alexion's and Caelums results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated safety profile and the benefits of the CAEL-101 may not be realized (and the results of the clinical trials may not be indicative of future results); the inability to enroll and complete the Phase 3 trial; results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexions or Caelums business, including on commercial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us; the risk that third party payors (including governmental agencies) will not reimburse for the use of our products at acceptable rates or at all; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including COVID-19 and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2020 and in their other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

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Alexion and Caelum Biosciences Announce Start of Phase 3 Studies of CAEL-101 in AL Amyloidosis - Business Wire

Seattle Genetics and Merck Announce Two Strategic Oncology Collaborations – BioSpace

Sept. 14, 2020 10:45 UTC

BOTHELL, Wash. & KENILWORTH, N.J.--(BUSINESS WIRE)-- Seattle Genetics, Inc. (Nasdaq: SGEN) and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced two new strategic oncology collaborations.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200914005237/en/

The companies will globally develop and commercialize Seattle Genetics ladiratuzumab vedotin, an investigational antibody-drug conjugate (ADC) targeting LIV-1, which is currently in phase 2 clinical trials for breast cancer and other solid tumors. The collaboration will pursue a broad joint development program evaluating ladiratuzumab vedotin as monotherapy and in combination with Mercks anti-PD-1 therapy KEYTRUDA (pembrolizumab) in triple-negative breast cancer, hormone receptor-positive breast cancer and other LIV-1-expressing solid tumors. Under the terms of the agreement, Seattle Genetics will receive a $600 million upfront payment and Merck will make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics is eligible for progress-dependent milestone payments of up to $2.6 billion.

Separately, Seattle Genetics has granted Merck an exclusive license to commercialize TUKYSA (tucatinib), a small molecule tyrosine kinase inhibitor, for the treatment of HER2-positive cancers, in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics will receive $125 million from Merck as an upfront payment and is eligible for progress-dependent milestones of up to $65 million.

Collaborating with Merck on ladiratuzumab vedotin will allow us to accelerate and broaden its development program in breast cancer and other solid tumors, including in combination with Mercks KEYTRUDA, while also positioning us to leverage our U.S. and European commercial operations, said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. The strategic collaboration for TUKYSA will help us reach more patients globally and benefit from the established commercial strength of one of the worlds premier pharmaceutical companies.

These two strategic collaborations will enable us to further diversify Mercks broad oncology portfolio and pipeline, and to continue our efforts to extend and improve the lives of as many patients with cancer as possible, said Dr. Roger M. Perlmutter, President, Merck Research Laboratories. We look forward to working with the team at Seattle Genetics to advance the clinical program for ladiratuzumab vedotin, which has shown compelling signals of efficacy in early studies, and to bring TUKYSA to even more patients with cancer around the world.

Ladiratuzumab Vedotin Collaboration Details

Under the terms of the agreement, Seattle Genetics and Merck will collaborate and equally share costs on the global development of ladiratuzumab vedotin and other LIV-1-targeting ADCs. The companies have agreed to jointly develop and share future costs and profits for ladiratuzumab vedotin on a 50:50 basis worldwide. Merck will pay Seattle Genetics $600 million upfront and make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics will be eligible to receive up to $2.6 billion in milestone payments, including $850 million in development milestones and $1.75 billion in sales milestones.

The companies will jointly develop and commercialize ladiratuzumab vedotin and equally share profits worldwide. The companies will co-commercialize in the U.S. and Europe. Seattle Genetics will be responsible for marketing applications for approval in the U.S. and Canada, and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in Europe and in countries outside the U.S. and Canada, and will record sales in countries outside the U.S., Europe and Canada. Including the upfront payment, equity investment proceeds and potential milestone payments, Seattle Genetics is eligible to receive up to $4.2 billion.

The closing of the equity investment is contingent on completion of review under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act).

TUKYSA Collaboration Details

Under the terms of the agreement, Merck has been granted exclusive rights to commercialize TUKYSA in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics retains commercial rights and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in its territory, supported by the positive results from the HER2CLIMB clinical trial.

Merck will also co-fund a portion of the TUKYSA global development plan, which encompasses several ongoing and planned trials across HER2-positive cancers, including breast, colorectal, gastric and other cancers set forth in a global product development plan. Seattle Genetics will continue to lead ongoing TUKYSA global development planning and operational execution. Merck will solely fund and conduct country-specific clinical trials necessary to support anticipated regulatory applications in its territory.

Seattle Genetics will receive from Merck $125 million as an upfront payment and is eligible to receive progress-dependent milestones of up to $65 million. Seattle Genetics will also receive $85 million in prepaid research and development payments to be applied to Mercks global development funding obligations. In addition, Seattle Genetics would receive tiered royalties on sales of TUKYSA in Mercks territory.

The financial impact of these collaborations is not included in Seattle Genetics 2020 guidance.

Seattle Genetics Conference Call Details

Seattle Genetics management will host a conference call to discuss these collaborations today at 6:00 a.m. Pacific Time (PT); 9:00 a.m. Eastern Time (ET). The event will be simultaneously webcast and available for replay from the Seattle Genetics website at http://www.seattlegenetics.com, under the Investors section. Investors may also participate in the conference call by calling 844-763-8274 (domestic) or +1 412-717-9224 (international). The conference ID is 10147850.

About Ladiratuzumab Vedotin

Ladiratuzumab vedotin is a novel investigational ADC targeted to LIV-1. Most metastatic breast cancers express LIV-1, which also has been detected in several other cancers, including lung, head and neck, esophageal and gastric. Ladiratuzumab vedotin utilizes Seattle Genetics proprietary ADC technology and consists of a LIV-1-targeted monoclonal antibody linked to a potent microtubule-disrupting agent, monomethyl auristatin E (MMAE) by a protease-cleavable linker. This novel ADC is designed to bind to LIV-1 on cancer cells and release the cell-killing agent into target cells upon internalization. Ladiratuzumab vedotin may also cause antitumor activity through other mechanisms, including activation of an immune response by induction of immunogenic cell death.

About TUKYSA (tucatinib)

TUKYSA is an oral, small molecule tyrosine kinase inhibitor (TKI) of HER2, a protein that contributes to cancer cell growth. TUKYSA in combination with trastuzumab and capecitabine was approved by the U.S. Food and Drug Administration (FDA) in April 2020 for adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting. In addition, TUKYSA received approval in Canada, Singapore, Australia and Switzerland under the Project Orbis initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners. A marketing application is under review in the European Union.

TUKYSA is being evaluated in several ongoing clinical trials and additional studies are planned. Current trials include the following:

For additional information, visit http://www.clinicaltrials.gov.

TUKYSA Important Safety Information

Warnings and Precautions

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Adverse Reactions

Serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in 2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock.

Adverse reactions led to treatment discontinuation in 6% of patients who received TUKYSA; those occurring in 1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions led to dose reduction in 21% of patients who received TUKYSA; those occurring in 2% of patients were hepatotoxicity (8%) and diarrhea (6%).

The most common adverse reactions in patients who received TUKYSA (20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, fatigue, hepatotoxicity, vomiting, stomatitis, decreased appetite, abdominal pain, headache, anemia, and rash.

Lab Abnormalities

In HER2CLIMB, Grade 3 laboratory abnormalities reported in 5% of patients who received TUKYSA were: decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

Drug Interactions

Use in Specific Populations

For more information, please see the full Prescribing Information for TUKYSA here.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

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Seattle Genetics and Merck Announce Two Strategic Oncology Collaborations - BioSpace

Cancer Stem Cells Market to witness astonishing growth by 2026 | AbbVie, The Merck KGaA Group, Bionomics, Lonza Group – The PRNews Pulse

Global Cancer Stem Cells Market Insights by Application, Product Type, Competitive Landscape & Regional Forecast 2025 is latest research study released by HTF MI evaluating the market, highlighting opportunities, risk side analysis, and leveraged with strategic and tactical decision-making support. The study provides information on market trends and development, drivers, capacities, technologies, and on the changinginvestment structure of the Global Cancer Stem Cells Market. Some of the key players profiled in the study are Thermo Fisher Scientific, Inc. (United States), AbbVie, Inc.(United States), The Merck KGaA Group (Germany), Bionomics (Australia), Lonza Group (Switzerland), Stemline Therapeutics, Inc.(United States), Fujifilm Irvine Scientific (United States), STEMCELL Technologies Inc. (Canada), Sino Biological Inc. (United States) and BIOTIME, Inc. (United States).

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Market Snapshot: According to the World Health Organization, Cancer is the second leading cause of death globally, and is responsible for an estimated 9.6 million deaths in 2018. Globally, about 1 in 6 deaths is due to cancer. Hence, there is a need for a tremendous research on Cancer Cells. Cancer stem cells (CSCs) refers to the cells which are obtained from tumor that posses potential to reproduce all types of cancer cells found in a cancer sample.These cells are grown in tumors as a separate population and thereby it causes Deterioration and Metastasis of Existing tumor through generation of new tumor. Hence, with the Advancement in Technology Especially in Cancer Stem Cells Research area, Therapies specific to Targeting Cancer Stem Cells are anticipated to drive the Global Cancer Stem Cells Market.

Market Drivers Increasing Prevalence of Cancer leading to rapidly rising burden of the mortality rate of Cancer among Patients The Continuous Rise in the number of Research Studies and Development on Cancer Stem Cells (CSCs) The Government initiatives to boost the Cancer Research activities and availability of funds.

Market Trend Improvements in experimental approaches by the Researchers such as, In vitro assay has enabled them to establish a relationship between different cell types in a tumor and their microenvironment This has led to the Development of a Broad Therapeutic Portfolio for CSCs and their associated key pathways.

Restraints High Costs related to Cancer Stem Cell Therapeutics may hamper market growth.

Cancer Stem Cells Market: Demand Analysis & Opportunity Outlook 2025

Cancer Stem Cells research study is to define market sizes of various segments & countries by past years and to forecast the values by next 5 years. The report is assembled to comprise each qualitative and quantitative elements of the industry facts including: market share, market size (value and volume 2014-19, and forecast to 2025) which admire each countries concerned in the competitive examination. Further, the study additionally caters the in-depth statistics about the crucial elements which includes drivers & restraining factors that defines future growth outlook of the market.

Important years considered in the study are: Historical year 2014-2019 ; Base year 2019; Forecast period** 2020 to 2025 [** unless otherwise stated]

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The segments and sub-section of Cancer Stem Cells market are shown below:

The Study is segmented by following Product Type: Cell Culturing, Cell Separation, Cell Analysis, Molecular Analysis and Others

Major applications/end-users industry are as follows: Breast Cancer Diagnosis and Treatment , Prostate Cancer Diagnosis and Treatment , Colorectal Cancer Diagnosis and Treatment , Lung Cancer Diagnosis and Treatment and Other Cancers Diagnosis and Treatment

Some of the key players/Manufacturers involved in the Market are Thermo Fisher Scientific, Inc. (United States), AbbVie, Inc.(United States), The Merck KGaA Group (Germany), Bionomics (Australia), Lonza Group (Switzerland), Stemline Therapeutics, Inc.(United States), Fujifilm Irvine Scientific (United States), STEMCELL Technologies Inc. (Canada), Sino Biological Inc. (United States) and BIOTIME, Inc. (United States)

If opting for the Global version of Cancer Stem Cells Market analysis is provided for major regions as follows: North America (USA, Canada and Mexico) Europe (Germany, France, the United Kingdom, Netherlands, Russia , Italy and Rest of Europe) Asia-Pacific (China, Japan, Australia, New Zealand, South Korea, India and Southeast Asia) South America (Brazil, Argentina, Colombia, rest of countries etc.) Middle East and Africa (Saudi Arabia, United Arab Emirates, Israel, Egypt, Nigeria and South Africa)

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Key Answers Captured in Study are Which geography would have better demand for product/services? What strategies of big players help them acquire share in regional market? Countries that may see the steep rise in CAGR & year-on-year (Y-O-Y) growth? How feasible is market for long term investment? What opportunity the country would offer for existing and new players in the Cancer Stem Cells market? Risk side analysis involved with suppliers in specific geography? What influencing factors driving the demand of Cancer Stem Cells near future? What is the impact analysis of various factors in the Global Cancer Stem Cells market growth? What are the recent trends in the regional market and how successful they are?

Read Detailed Index of full Research Study at @https://www.htfmarketreport.com/reports/2134979-global-cancer-stem-cells-market-2

There are 15 Chapters to display the Global Cancer Stem Cells market. Chapter 1, About Executive Summary to describe Definition, Specifications and Classification of Global Cancer Stem Cells market, Applications [Breast Cancer Diagnosis and Treatment , Prostate Cancer Diagnosis and Treatment , Colorectal Cancer Diagnosis and Treatment , Lung Cancer Diagnosis and Treatment and Other Cancers Diagnosis and Treatment], Market Segment by Types Cell Culturing, Cell Separation, Cell Analysis, Molecular Analysis and Others; Chapter 2, objective of the study. Chapter 3, to display Research methodology and techniques. Chapter 4 and 5, to show the Cancer Stem Cells Market Analysis, segmentation analysis, characteristics; Chapter 6 and 7, to show Five forces (bargaining Power of buyers/suppliers), Threats to new entrants and market condition; Chapter 8 and 9, to show analysis by regional segmentation[North America, Europe, Asia-Pacific etc ], comparison, leading countries and opportunities; Regional Marketing Type Analysis, Supply Chain Analysis Chapter 10, to identify major decision framework accumulated through Industry experts and strategic decision makers; Chapter 11 and 12, Global Cancer Stem Cells Market Trend Analysis, Drivers, Challenges by consumer behavior, Marketing Channels Chapter 13 and 14, about vendor landscape (classification and Market Ranking) Chapter 15, deals with Global Cancer Stem Cells Market sales channel, distributors, Research Findings and Conclusion, appendix and data source.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia or Oceania [Australia and New Zealand].

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Cancer Stem Cells Market to witness astonishing growth by 2026 | AbbVie, The Merck KGaA Group, Bionomics, Lonza Group - The PRNews Pulse