On December 18, 2019 FerGene, a new gene therapy company launched by Ferring Pharmaceuticals and Blackstone Life Sciences in November, reported the appointment of David Meek as President and Chief Executive Officer, effective January 14, 2020 (Press release, FerGene, DEC 18, 2019, View Source [SID1234552477]).

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Mr. Meek has 30 years of industry experience. Most recently, he has served as CEO of Ipsen, a leading global biopharmaceutical company focused on innovation and specialty care and dedicated to improving lives through the discovery of new medicines in oncology, neuroscience and rare diseases.

"I’m honored to lead FerGene in this exciting role and spearhead the effort to develop and commercialize this critical and innovative therapy and advance its clinical development. We will build a committed, patient-centric team and with the support from Ferring and Blackstone Life Sciences, I am confident we can efficiently and effectively bring this life changing therapy to patients in need," said Mr. Meek.

Jean Duvall, Co-Chair of FerGene and Executive Committee member of Ferring Pharmaceuticals, said, "We are extremely confident in David and his ability to lead and build FerGene as it seeks to bring a promising therapy to market for bladder cancer patients and improve the standard of care. With an impressive track record of corporate leadership experience, David is poised to excel in this role."

Paris Panayiotopoulos, Co-Chair of FerGene and Blackstone Life Sciences Managing Director, said, "We are thrilled to welcome David as FerGene’s new CEO. David’s proven track record of growing businesses across both Biotech and Pharma, as well as his deep knowledge of the US Oncology market, make him the ideal fit to lead FerGene. David’s experience will be instrumental as we advance a breakthrough investigational gene therapy that offers the potential to meaningfully improve the standard of care for a patient population which has seen little innovation over the past twenty years."

FerGene was launched in November 2019 with a focus on US commercialization of nadofaragene firadenovec (rAd-IFN/Syn3), an investigational novel gene therapy in late stage development for patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC).

FerGene also recently announced Phase 3 study results of nadofaragene firadenovec, which FerGene reports met its primary endpoint with more than half of patients with high-grade, BCG unresponsive non-muscle invasive bladder cancer (cis ± ta/t1) achieving a complete response at three months.

While at Ipsen, Mr. Meek has led a growth strategy of financial results and pipeline advancement. He has also transformed Ipsen into a global biopharma growth leader with initiatives to transform external innovation, Ipsen’s R&D operations and build out the company’s footprint in countries like the U.S. and China. In addition to his time as CEO of Ipsen, Mr. Meek’s prior leadership roles have included serving as Executive Vice President & President of Oncology at Baxalta from 2014 – 2016, following its spin-off from Baxter. He was also Chief Commercial Officer of Endocyte from 2012 – 2014. Prior to that, Mr. Meek served in various executive leadership roles at Novartis Pharma and Novartis Oncology after beginning his career at Johnson & Johnson and Janssen from 1989 – 2004.

About nadofaragene firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG unresponsive, NMIBC. It is an adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder every three months. The vector enters the cells of the bladder wall, where, it breaks down, releasing the active gene to do its work. The internal gene/DNA machinery of the cells ‘picks up’ the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into multiple interferon microfactories, enhancing the body’s natural defenses against the cancer.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is an early form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.1 It is estimated that there will be 80,000 new cases of bladder cancer in the U.S. in 2019; more than 70% of these cases present as NMIBC.2,,3 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, between 30% and 50% cases with high-grade disease will recur.4 The outcome for BCG unresponsive patients is poor, with total cystectomy (complete removal of the bladder) often being the next treatment option.5

On December 18, 2019 The Jackson Laboratory (JAX) reported that it has received $2.5 million from The Mark Foundation for Cancer Research to study in mice the influence of host genetics on response to immunotherapy (Press release, The Mark Foundation For Cancer Research, DEC 18, 2019, View Source [SID1234553264]). The goal of this project is to generate insights that will empower future decisions about the best treatments for cancer patients based on their genetic backgrounds.

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Immunotherapy, which uses the body’s own immune system to fight cancer, is one of the most promising approaches to cancer treatment today, and expanding its effectiveness to a wider population of patients is a high priority for researchers across the globe.

The project, "Dissecting the Genetic Control of Response to Immune Checkpoint Inhibitors in Cancer," has major implications for how oncologists may be able to predict whether their patients will respond positively to immunotherapy before prescribing a treatment regimen.

A unique opportunity

Immunotherapy has resulted in some remarkable outcomes for patients whose cancers would have otherwise been fatal. Nonetheless, the overall efficacy rates for this class of treatment are not high, and many patients experience serious or even life-threatening side effects. The determination of which patients will respond well to immunotherapy is still uncertain. Probing factors that may influence response to immunotherapy, such as the personal genetics of each individual, tumor characteristics, and the cross-talk among these profiles is a critical step toward designing immune therapies that will have broader impact.

JAX President and CEO Edison T. Liu, MD and Associate Professor Laura Reinholdt, PhD, co-principal investigators of the study, will use genetically diverse JAX mouse models to untangle the relationship between genetics and response to immunotherapies.

"This research may one day allow doctors to use genetic sequencing to predict patient response to immunotherapy, as well as help scientists develop more effective anti-cancer drugs that fight tumors by activating an immune response," said Liu. "We are so grateful to The Mark Foundation for enabling this extraordinary opportunity, which we hope will have a significant impact on patients and the field of immunotherapy," said Liu.

The mission of The Mark Foundation for Cancer Research is to support scientists tackling the toughest challenges in cancer research. Multi-disciplinary groups such as the JAX team, which brings together experts in genetics, bioinformatics, mouse models, and immunology, are well positioned to take on these challenges. By funding this type of collaborative project, The Mark Foundation ensures that the right expertise is brought to bear on a substantial unmet need, ultimately accelerating cancer research to the benefit of patients.

"This project addresses critical limitations in cancer research. First, most mouse models for studying therapeutic response lack the genetic diversity that exists in the human population, often leading to disappointment when treatments that show promise in these models are deployed broadly in patients. Second, the biology of how genetics impact tumor response is not well understood due to a lack of adequate experimental systems for uncovering new knowledge", said Michele Cleary, PhD, CEO of The Mark Foundation. "Solving both of these issues requires the type of complex and nuanced work in mouse genetics for which JAX is world-renowned."

The power of genetic diversity

The grant to the Jackson Laboratory research team, which also includes Assistant Professor Chih-Hao "Lucas" Chang, PhD, Associate Research Scientist John P. Graham, PhD, Professor Karolina Palucka, MD, PhD, and Research Scientist Daniel Skelly, PhD, will examine four different tumor lines: a melanoma tumor, a triple-negative breast cancer tumor, and two types of colon cancer tumors. The tumor types were chosen to represent a wide range of responses to immunotherapy. For example, melanoma tumors tend to respond quite well to immunotherapy, whereas triple-negative breast cancer tumors typically do not.

The scientists will use JAX’s genetically diverse mouse strains in their studies, which will allow them to identify the genetic differences that underlie the variation in response to immunotherapy. Such an approach is inordinately difficult to achieve in human clinical trials.

"The increasing adoption of genetically diverse mice in biomedical research is really a paradigm shift driven by new technologies and biological resources, and by the recognition that an inbred mouse strain only represents one genome," said Reinholdt.

"This project is providing a unique opportunity to harness natural genetic variation in laboratory mice to more accurately model the biological complexity of cancer treatment response. The power of these genetically diverse mouse strains is that they facilitate genetic discovery and they provide tractable in vivo systems for dissecting molecular pathways, which are essential for the identification of biomarkers and potentially new therapeutic targets."

The research team plans to map the genetic differences found in the mice to specific genetic variants in humans. Ultimately, they hope that doctors will be able to test and determine a given individual’s likely response to immunotherapy to help guide the selection of a treatment regimen.

On December 18, 2019 BeyondSpring Inc. (the "Company" or "BeyondSpring") (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative cancer therapies, reported an update on the Company’s operations and announced its financial results for the three months ended September 30, 2019 (Press release, BeyondSpring Pharmaceuticals, DEC 18, 2019, View Source [SID1234552462]).

"We are pleased with the progress we are seeing in our clinical programs surrounding our lead compound, Plinabulin, for the treatment of non-small cell lung cancer (NSCLC) and CIN," said Dr. Lan Huang, BeyondSpring’s Co-Founder, Chairman and Chief Executive Officer. "Our goal from day one has been to develop transforming treatments for cancer patients. Between the enrollment of the first patient in our global Phase 3 Study 106, the publication of Plinabulin’s immune mechanism as a potent Antigen Presenting Cell (APC) inducer in three peer-review journals and continual positive data being presented at medical meetings world-wide, we remain on track to address large unmet medical needs among these cancer patients."

"The combination of our clinical experience – over 900 patients enrolled globally – and the deep insight into Plinabulin’s immune mechanism – a potent APC inducer – lead us to believe Plinabulin has potential to be a ’pipeline in a drug’. Our life cycle plan for Plinabulin begins with the foundational indications in NSCLC and CIN. From here we expect to expand into a broader chemotherapy combination footprint, and eventually we expect to transform Plinabulin’s potential in combination with checkpoint inhibitors and radiation or chemotherapy. This triple therapy combination could be a powerful treatment in multiple cancer types with significant market opportunities," added Dr. Huang. "We anticipate filing NDAs for Plinabulin in the U.S. and China for both NSCLC and CIN over the next 18 months. The success of our recent round of financing ensures that we will have the necessary resources to achieve the clinical milestones moving forward."

Recent Operational Highlights

Novel Study Design of Plinabulin in NSCLC Phase 3

In October 2019, BeyondSpring announced that its abstract, titled, "DUBLIN-3, a Stage IIIb / IV NSCLC Phase 3 Trial Comparing the Plinabulin / Docetaxel Combination with Docetaxel Alone," was presented at this year’s European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. The abstract highlighted the study’s unique design, which positively addressed the challenges associated with trial failure risk in NSCLC studies.

In November 2019, BeyondSpring presented a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, titled, "Validation of a Single-Blinded (Patients Only) Study Design for the Prevention of Premature Patient Consent Withdrawal in the Immuno-Oncology Trial DUBLIN-3." This abstract explained that cancer patients generally prefer immunotherapy over chemotherapy, which can lead to premature withdrawal of their consent to participate in a clinical trial if they are allocated to the chemotherapy comparator arm, and can negatively impact the study’s outcome. Specifically, the DUBLIN-3 trial was a single-blinded study, while the Javelin trial was an open-label study, which led investigators to their conclusion.

1
Plinabulin’s Superior Profile in CIN Prevention

In September 2019, BeyondSpring announced that an abstract, titled, "The Effect of Increasing Doses of Pegfilgrastim on Thrombocytopenia in Breast Cancer Patients Receiving Taxotere, Doxorubicin, Cyclophsophamide and Plinabulin," was accepted for presentation at ESMO (Free ESMO Whitepaper). The abstract evaluated the effects of pegfilgrastim combined with Plinabulin on absolute neutrophil and platelet counts compared to pegfilgrastim alone. The data showed that the combination appears to have a superior product profile over pegfilgrastim in CIN control, platelet counts and bone pain.

In October 2019, BeyondSpring announced the enrollment of the first patient in its Study 106, a global Phase 3 trial with Plinabulin in combination with G-CSF to prevent CIN. The study is the key platform to confirm this finding and bring improved care to patients.

Most recently at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s (ASH) (Free ASH Whitepaper) annual meeting, BeyondSpring presented two e-publications on Study 105 and a poster on Study 106, highlighting Plinabulin’s mechanism of action in preventing CIN.

The Study 105 e-publications titled, "Clinical Evidence of Granulocyte-Monocyte Progenitor (GMP) Stem Cell Involvement in Plinabulin’s Mechanism of Action (MoA) for the Prevention of Docetaxel (Doc) Chemotherapy (Chemo)-Induced Neutropenia (CIN)," and "Clinical Evidence Against the Continuum of Low-Primed Uncommitted Hematopoietic and Progenitor Cells (CLOUD-HSPC) Concept for Hematopoiesis," highlight Plinabulin’s ability to protect hematopoietic stem cells, which differentiates into neutrophils and other white blood cells.

The Study 106 poster titled, "A Randomized Phase 3 Clinical Trial of the Combination of Plinabulin (Plin) + Pegfilgrastim (Peg) versus (vs) Peg Alone for TAC (docetaxel, doxorubicin, cyclophosphamide) Induced Neutropenia (CIN)," provides rationale for the addition of Plinabulin to pegfilgrastim (Neulasta) due to their differing mechanisms of action (MoA) for the prevention against CIN.

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Plinabulin’s Anti-cancer Immune Mechanism and CIN Prevention Mechanism Published in Peer-Reviewed Journals

In October 2019, prestigious Cell publications, Chem and Cell Reports, published novel findings focusing on the mechanism of Plinabulin to mature dendritic cells, which leads to T-cell activation.

The Chem article, titled, "Structure, Thermodynamics and Kinetics of Plinabulin Binding to Two Tubulin Isotypes," outlined research utilizing X-ray crystallography and thermodynamic calculations to demonstrate Plinabulin as a new chemical entity with differentiated binding and kinetics compared to other tubulin binding agents, which gives rise to its differentiated superior efficacy and safety profile in the clinic.

In Cell Reports, titled, "GEF-H1 Signaling Upon Microtubule Destabilization Is Required for Dendritic Cell Activation and Specific Anti-tumor Responses," authors cited Plinabulin’s ability to destabilize microtubule and release the immune defense protein GEF-H1, which is a critical signaling protein for dendritic cell maturation. This leads to the priming of CD8 T-cells. GEF-H1 high immune signature showed better survival in cancer patients, which validated GEF-H1 as a first in class target.

In early December, Cancer Chemotherapy & Pharmacology published an article that examines the mechanism-of-action of Plinabulin and its anti-CIN benefits. The article, titled, "Plinabulin Ameliorates Neutropenia Induced by Multiple Chemotherapies Through a Mechanism Distinct from G‑CSF Therapies," reports the results of an animal study that support the clinical testing of Plinabulin as a non-G-CSF-based treatment for CIN associated with chemotherapies of different mechanisms. Results also support hematopoietic stem/progenitor cells as a focal point for future mechanism-of-action work aimed at understanding the ability of Plinabulin to reduce the serious side effect of cytotoxic therapy in cancer patients.

Strong Intellectual Property Protection through 2036 Globally

In October 2019, BeyondSpring announced that the U.S. Patent and Trademark Office granted the Company a new patent for methods of treating brain tumors by administering Plinabulin. This underscores the Company’s belief that Plinabulin is a first-in-class clinical pipeline asset. Currently, surgery and radiation are the only approved treatments for brain tumors. Chemotherapy has never been approved to treat metastatic brain tumors.

We now have 75 granted Plinabulin patents in 36 jurisdictions and 17 patents granted in the U.S., including the composition of matter patent, with protection to 2036.

Equity Financing

On October 25, 2019, BeyondSpring priced an underwritten public offering of its ordinary shares. The offering was led by Decheng Capital, with gross proceeds of $25.8 million including the overallotment options, before deducting underwriting discounts, commissions and other offering expenses. The Company expects to use the net proceeds to continue clinical and pre-clinical development in addition to general corporate purposes.

Financial Results for the Three Months Ended September 30, 2019

Research and development ("R&D") expenses were $7.2 million for the quarter ended September 30, 2019, compared to $14.1 million for the quarter ended September 30, 2018. The $6.9 million decrease was largely attributable to a $3.9 million decrease in expenses incurred by clinical research organizations and other service fees related to clinical trials, a $0.5 million decrease in preclinical trial expenses and a $0.5 million decrease in non-cash share-based compensation.

General and administrative ("G&A") expenses were $2.5 million for the quarter ended September 30, 2019, compared to $1.5 million for the quarter ended September 30, 2018. The $1.0 million increase was mainly due to the $0.7 million of market research expenses incurred during the quarter ended September 30, 2019.

Net loss attributable to the Company was $9.4 million for the quarter ended September 30, 2019, compared to $14.9 million for the quarter ended September 30, 2018.

As of September 30, 2019, the Company had a cash balance of $24.7 million. With the additional $25.8 million gross proceeds from the recent public offering described above, the Company believes it has sufficient cash to support its clinical trials and submit NDAs in the U.S. and China for Plinabulin for the treatment of NSCLC and CIN, as well as to advance its immuno-oncology pipeline and its ubiquitination protein degradation research platform.

Anticipated Milestones

The following outlines the Company’s anticipated upcoming milestones and projected timelines:

NDA submission to China’s National Medical Products Administration (NMPA) for Plinabulin for CIN – Q1 2020

Final data readout for Study 105 Phase 3 for CIN – H1 2020

Final data readout for Study 106 Phase 3 for CIN – H1 2020

Final data readout for Study 103 Phase 3 for NSCLC – H2 2020

NDA submission to China’s NMPA for Plinabulin for NSCLC – H2 2020

NDA submission to the U.S. Food and Drug Administration (FDA) for Plinabulin for CIN – H2 2020

NDA submission to the U.S. FDA for Plinabulin for NSCLC – H1 2021

Conference Call and Webcast Information

BeyondSpring’s management will host a conference call and webcast today at 8 a.m. Eastern Time to discuss the financial results and provide a corporate update. The dial-in numbers for the conference call are 1-877-451-6152 (U.S.) or 1-201-389-0879 (international). Please reference conference ID 13697474. A live webcast will be available on BeyondSpring’s website at www.beyondspringpharma.com under "Events & Presentations" in the Investors section. An archived replay of the webcast will be available for 30 days.

On December 18, 2019 Immunitas Therapeutics ("Immunitas"), an innovative single cell genomics discovery platform company focused on developing breakthrough immuno-oncology therapies, reported it has appointed Jeffrey M. Goldberg as Chief Executive Officer and Director (Press release, Immunitas Therapeutics, DEC 18, 2019, View Source [SID1234552478]). He succeeds founding CEO, Christoph Westphal, M.D, Ph.D., who assumes the role of Chairman of the Board of Directors.

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"One of the central challenges of drug development has been bridging the gap between laboratory research in model organisms to meaningful clinical advances in humans," said Jeff Goldberg. "Immunitas and our scientific co-founders use single cell genomic sequencing and sophisticated computational biology techniques to look at human biology directly. I believe this innovative approach can help to accelerate the development of new therapies for patients. I am excited to be joining the Immunitas team as we discover and develop these highly targeted new immuno-oncology therapies."

Immunitas identifies novel, promising oncology targets with potential applicability across both solid and liquid tumors. Additionally, as part of the discovery process, Immunitas develops key biomarkers to guide the selection of patients who may benefit from its new drugs. The company leverages its expertise in antibody discovery and engineering to create therapies that modulate these targets. Immunitas is currently advancing a number of programs toward early human studies, including a lead program with fully-human monoclonal antibodies that will be developed as single agents using a clinical biomarker strategy to guide early efficacy studies.

"Jeff Goldberg has over 20 years of industry experience driving programs from discovery through all phases of drug development to commercialization in multiple therapeutic areas, including oncology, neurology, renal, and rare diseases," said Lea Hachigian, President and Director, Immunitas Therapeutics. "We are fortunate to have his demonstrated ability leading and building teams as we create an oncology company powered by our human biology-focused approach to immunology."

Mr. Goldberg joins the Immunitas Board of Directors, which includes Dr. Laura Brass, Managing Director at Novartis Venture Fund, Dr. Jürgen Eckhardt, Head of Leaps by Bayer, Bayer’s strategic venture capital unit, Dr. Lea Hachigian, Principal, Longwood Fund, Dr. Lucio Iannone, Director of Venture Investments of Leaps by Bayer, Dr. Christoph Westphal, co-founder and General Partner of Longwood Fund, and Dr. Vincent Xiang, Managing Director at Hillhouse Capital.

Jeff Goldberg is an experienced biotech program and brand leader with over 20 years of industry experience. He has driven programs from discovery and pre-clinical through IND, clinical trials, NDA, and commercialization in multiple therapeutic areas, including oncology, neurology, renal, and other rare and orphan diseases. Mr. Goldberg joins Immunitas from Akcea Therapeutics, where he was Chief Operating Officer from the time of its formation in January 2015. Previously, Mr. Goldberg was VP of Business Operations, leading both program management and business development at Proteostasis Therapeutics, Inc., a biotech company focusing on neurology and rare diseases. He also spent more than 11 years in positions of increasing responsibility with Genzyme and Sanofi, providing brand management for two marketed products within Sanofi Oncology. Prior to joining Sanofi Oncology, Mr. Goldberg served as Global Program Lead for Genzyme’s stem cell mobilization agent Mozobil, leading the global launch team and overseeing the program management and marketing functions for the product. He began his career at Genzyme as Director, Program Management overseeing the development and launch of Renvela in patients undergoing dialysis. Mr. Goldberg has both an MBA and a Master’s degree in Chemical Engineering from the Massachusetts Institute of Technology, and a B.S. in Chemical Engineering from Cornell University.

On December 18, 2019 Bristol-Myers Squibb Company (NYSE: BMY) reported the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lisocabtagene maraleucel (liso-cel), its autologous anti-CD19 chimeric antigen receptor (CAR) T‑cell immunotherapy comprising individually formulated CD8+ and CD4+ CAR T cells for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) after at least two prior therapies (Press release, Bristol-Myers Squibb, DEC 18, 2019, View Source [SID1234552463]).

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The submission is based on the safety and efficacy results from the TRANSCEND NHL 001 trial, evaluating liso-cel in 269 patients with relapsed/refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL). Bristol-Myers Squibb recently presented data from this pivotal study at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.

Liso-cel has been granted Breakthrough Therapy (BT) and Regenerative Medicine Advanced Therapy (RMAT) designations by the FDA for relapsed/refractory aggressive large B-cell non-Hodgkin lymphoma (NHL), including DLBCL, not otherwise specified (de novo or transformed from indolent lymphoma), primary mediastinal B-cell lymphoma (PMBCL) or Grade 3B follicular lymphoma (FL) and Priority Medicines (PRIME) scheme by the European Medicines Agency for relapsed/refractory DLBCL.

Liso-cel is an investigational compound that is not approved for use in any country.

About Diffuse Large B-cell Lymphoma

Diffuse large B-cell lymphoma (DLBCL) is the most common and aggressive NHL, accounting for three out of every five cases. Approximately one-third of patients with DLBCL relapse after receiving first-line treatment, and about 10% have refractory disease. Historically, median life expectancy for patients who relapse or are refractory to current standard of care treatments is approximately six months.

Bristol-Myers Squibb: Advancing Cancer Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase quality, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Lisocabtagene Maraleucel (liso-cel)

Liso-cel is an investigational chimeric antigen receptor (CAR) T-cell therapy designed to target CD19, which is a surface glycoprotein expressed during normal B-cell development and maintained following malignant transformation of B cells. Liso-cel aims to target CD19-expressing cells through a CAR construct that includes an anti-CD19 single-chain variable fragment (scFv) targeting domain for antigen specificity, a transmembrane domain, a 4-1BB costimulatory domain hypothesized to increase T-cell proliferation and persistence, and a CD3-zeta T-cell activation domain. The defined composition of CAR-positive viable T-cells (consisting of CD8 and CD4 components) in liso-cel may reduce product variability; however, the clinical significance of defined composition is unknown.

About TRANSCEND NHL 001

TRANSCEND NHL 001 is an open-label, multicenter, pivotal phase 1 study to determine the safety, pharmacokinetics, and antitumor activity of liso-cel in patients with relapsed/refractory B-cell NHL, including DLBCL, PMBCL, Grade 3B FL. Mantle cell lymphoma is investigated in a separate cohort. The primary outcome measures included treatment-related adverse events, dose-limiting toxicities and objective response rate. Secondary outcome measures included complete response rate, duration of response, and progression-free survival. The TRANSCEND program is a broad clinical program evaluating liso-cel in multiple disease states and treatment stages.