On May 11, 2020 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported financial results for the first quarter of 2020 ended March 31, 2020 and provided an update on the Company’s recent and future developments (Press release, Pieris Pharmaceuticals, MAY 11, 2020, View Source [SID1234557451]).

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"We continue to stay the course with regard to the advancement of our lead assets, PRS-060 and PRS-343, despite the general disruptions caused by the coronavirus pandemic. We have been preparing for the start of the phase 2a trial of PRS-060 with AstraZeneca, which we still anticipate initiating in the second half of this year. Additionally, we have seen further clinical benefit in the monotherapy study of PRS-343, and we will be advancing that asset into a phase 2 study in combination with ramucirumab and paclitaxel in second-line gastric cancer in the second half of this year," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "I am very proud of the diligence and resolve of our team during these trying times."

PRS-060: Pieris and AstraZeneca are preparing to initiate the phase 2a study of PRS-060/AZD1402 in moderate-to-severe asthmatics in the second half of 2020. The study will be sponsored, funded, and delivered by AstraZeneca and upon completion of that study, Pieris will have the options to co-develop and, subsequently, co-commercialize PRS-060/AZD1402 in the United States.
PRS-343: Based on the totality of the data in the phase 1 dose-escalation monotherapy study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors, Pieris will initiate a phase 2 single-arm study of PRS-343 in combination with ramucirumab and paclitaxel in the second-line of treatment for gastric cancer in the second half of this year. At the active dose levels for which the Company presented interim data last year in cohorts 9 (2.5 mg/kg Q3W) through 11b (8 m/kg Q2W), a partial response was observed in three patients and stable disease was observed in 11 patients as best response out of 21 evaluable patients, translating to an objective response rate (ORR) of 14% and a disease control rate (DCR) of 67%. All three objective responses in these cohorts were observed in cohort 11b, in which disease stabilization was also observed in three patients out of seven evaluable patients, translating to an ORR of 43% and a DCR of 86%. Additional clinical benefit, including complete response, was also observed in the higher dose cohorts, which are still open for enrollment to generate a larger data set. Pieris plans to present detailed data from both the monotherapy study and atezolizumab combination study at a medical meeting in the second half of this year.
Immuno-oncology Pipeline: Pieris and Servier have been working on furthering the development of PRS-344 and PRS-352. Due to scale-up challenges recently encountered with the manufacture of drug product for PRS-344, a 4-1BB/PD-L1 bispecific, Pieris and Servier have jointly decided to invest in additional CMC activities, given the strategic importance of this program. As a result, Pieris now anticipates filing an IND application for PRS-344 next year. The Company holds exclusive commercialization rights for PRS-344 in the United States and will receive royalties on ex-U.S. sales for this program. Pieris is also focused completing the non-GLP work for PRS-352, a preclinical stage program addressing undisclosed targets, and expects to hand it over to Servier this year. Pieris continues to make progress in the Seattle Genetics collaboration.
Preclinical Respiratory Pipeline: Beyond PRS-060, Pieris continues to advance three discovery programs in its five-program respiratory collaboration with AstraZeneca. Pieris expects AstraZeneca will initiate the fourth discovery program in the collaboration later this year. The Company also continues to advance several proprietary discovery-stage respiratory programs. Pieris expects to share data and rationale for advancement of one of its proprietary programs in the second half of this year.
Board of Directors Transition: Jean-Pierre Bizzari, M.D. transitioned from the Board of Directors to serve as an advisor to the Company on oncology development strategies.
Fiscal Year Financial Update:

Cash Position – Cash, cash equivalents, and investments totaled $86.8 million for the quarter ended March 31, 2020, compared to a cash, cash equivalents, and investments balance of $104.2 million for the quarter ended December 31, 2019. The decrease was due to operating cash expenses, annual bonus payments, and both capital and one-time expenditures associated with the move to a new facility in Hallbergmoos.

R&D Expense – R&D expenses were $12.8 million for the quarter ended March 31, 2020, compared to $14.3 million for the quarter ended March 31, 2019. The decrease in research and development expenses reflects lower manufacturing spending on PRS-344. Partially offsetting this decrease were higher personnel expenses due to an overall increase in R&D headcount and an increase in allocated facility costs due to the new Hallbergmoos site, both associated with the advancement of our preclinical and clinical programs.

G&A Expense – G&A expenses were $4.4 million for the quarter ended March 31, 2020, compared to $4.9 million for the quarter ended March 31, 2019. The decrease in G&A expenses reflects lower personnel costs and a reduction in audit and tax professional fees.

Net Loss – Net loss was $3.6 million or $(0.07) per share for the quarter ended March 31, 2020, compared to a net loss of $10.3 million or $(0.20) per share for the quarter ended March 31, 2019.

Conference Call:

Pieris management will host a conference call beginning at 8:00 AM EDT on Monday, May 11, 2020, to discuss the first quarter financial results and provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). An archived replay of the call will be available by dialing +1-877-660-6853 (US & Canada) or +1-201-612-7415 (International) and providing the Conference ID #: 13661472.

On May 11, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2020 and provided an operational update (Press release, bluebird bio, MAY 11, 2020, View Source [SID1234557450]).

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"We remain grounded in our core values and priorities: our patients, our people, our community and our business," said Nick Leschly, chief bluebird. "This quarter, we successfully submitted the ide-cel BLA with our partners at BMS and I am pleased to announce today that we have alignment with FDA on our clinical data package and filing path for LentiGlobin for sickle cell disease, which accelerates our planned base case filing timeline into 2021. Additionally, we are amending our co-promotion/co-development agreement with BMS to enable both companies to focus their efforts on efficient commercialization of ide-cel and generate non-dilutive capital for our business. Lastly, after a rigorous review of all operational plans to reflect COVID-19 uncertainties and recent program shifts, we have prioritized our core four programs to drive ide-cel launch and three filings in 2021 for CALD, TDT and SCD and continue to drive forward high potential pipeline assets. This prioritization effort and operational review has led to significant efficiencies and spend reduction across our company and will extend our cash runway into 2022. The fundamentals of our business remain sound and our newly revised operating plan enables us to execute on the 2022 vision while putting us on a path towards financial sustainability. Our team remains excited and committed to our mission with the belief we will emerge from a very tough period in history stronger than ever to deliver for patients. Thank you birds!"

SCD REGULATORY PATH

Today, bluebird bio announced general agreement with FDA that the clinical data package required to support a BLA submission for LentiGlobin for sickle cell disease (SCD) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint and at least 18 months of follow-up post drug product infusion. Globin response will be used as a key secondary endpoint. The company anticipates additional guidance from FDA regarding the commercial manufacturing process, including suspension lentiviral vector. The company is planning to seek an accelerated approval and expects to submit the U.S. Biologics Licensing Application (BLA) for sickle cell disease in the second half of 2021. bluebird bio plans to present updated data from the HGB-206 study at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting.

Additionally, to enhance its strategic, clinical and commercial manufacturing platform, bluebird bio has entered into expanded relationships with two subsidiaries of Hitachi Chemical Co., Ltd. (Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma GmbH). These agreements will support late-stage and commercial drug product manufacturing of LentiGlobin for SCD in both the United States and Europe, expand commercial drug product manufacturing capacity for ZYNTEGLO (betibeglogene autotemcel) in Europe, and expand clinical and commercial manufacturing capacity for Lenti-D for cerebral adrenoleukodystrophy (CALD) in Europe.

FINANCIAL AND BUSINESS UPDATES

IDE-CEL ROYALTY MONETIZATION AND BCMA RELATIONSHIP AMENDMENT

Today, bluebird bio announced in a separate press release that it has amended its existing co-promotion/co-development agreement with Bristol Myers Squibb (BMS) to enable the companies to focus their efforts on efficient commercialization of idecabtagene vicleucel (ide-cel; bb2121) in the U.S., the companies’ lead investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, currently in review with the FDA. The companies will continue to share equally profits and losses in the U.S. Under the terms of the amended agreement, BMS will buy out its obligations to pay bluebird bio future ex-U.S. milestone and royalty payments for ide-cel and bb21217, the companies’ second BCMA-directed CAR T immunotherapy, for a one-time upfront payment of $200 million. bluebird bio is currently in the process of building out and qualifying its wholly-owned manufacturing facility in Durham, North Carolina for the production of lentiviral vector (LVV) to support the U.S. commercial market for ide-cel and for bluebird bio’s pipeline. Over time, BMS will assume responsibility for manufacturing of LVV outside the U.S. In partnership with BMS, bluebird bio is planning to present updated ide-cel clinical data from the Phase 2 KarMMa study at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting.

REVISED BUSINESS PRIORITIES AND OPERATING PLAN

Given the ongoing impact of the COVID-19 global pandemic and recent shifts in regulatory timelines, bluebird bio has undertaken a comprehensive business review with the goal of ensuring the ability to achieve its 2022 vision with a path towards financial sustainability. Under the revised business priorities and operating plan, bluebird remains on track for potential regulatory approval and commercial launch for ZYNTEGLO, ide-cel, Lenti-D for CALD, and LentiGlobin for SCD by 2022.

Through this comprehensive business review, bluebird bio has prioritized key research and development programs and has made a number of changes to the future cost structure relative to the prior long-range plan, including:

Reduced investment in selling, general and administrative expenses, including a deferred investment in building a U.S. commercial organization, reduced facilities and IT infrastructure, and other cost-reduction measures.
Prioritized investment in R&D expenses, including an indefinite pause of the HGB-211 clinical study in SCD patients at high risk of stroke, adjustment to the timing of investment in ongoing clinical studies to reflect COVID-19 related delays in enrollment, reduction or elimination of investment in certain preclinical programs, and other cost-reduction measures.
Nick Leschly, chief bluebird, will decline nearly 100% of his salary for the next 12 months. Similarly, additional members of the bluebird bio senior leadership team and all members of the Company’s Board of Directors will forgo 20% of their salaries or Board cash retainers for the next 12 months. All will receive a grant of restricted stock units equal to 80% of the value of the released cash compensation, which will vest over one year.
In total, these changes are expected to result in over $500 million of net cash savings through 2022 compared to the prior long-range plan. As a result, bluebird bio expects cash, cash equivalents, and marketable securities of $1.02 billion as of March 31, 2020, together with projected revenue generated under collaborative arrangements, projected sales of products and cash inflows associated with the amended agreements with BMS, to fund the revised operating plan into 2022. bluebird bio expects to continue to drive additional savings through rigorous prioritization and focus on expenses, real estate optimization, and exploration of additional sources of funding to further strengthen its financial position.

PREVIOUSLY DISCLOSED RECENT HIGHLIGHTS

IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On March 31, 2020, bluebird bio and BMS announced the submission of their BLA to the U.S. FDA for ide-cel, the companies’ lead investigational BCMA-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The BLA submission includes results from pivotal KarMMa study evaluating ide-cel in a heavily pre-treated patient population with relapsed and refractory multiple myeloma.
COVID-19 IMPACT – On March 26, 2020, bluebird bio provided an assessment of the impact of the COVID-19 pandemic and outlined steps the company has taken to ensure the safety of its patients and employees, while working to ensure the sustainability of its business operations as this unprecedented situation continues to evolve. Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months and expects timing shifts to vary by clinical trial and by program.
UPCOMING ANTICIPATED MILESTONES

Regulatory
Submission of a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical
Presentation of ide-cel clinical data from the KarMMa study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting later this month, in partnership with Bristol-Myers Squibb.
Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes at the 2020 Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Updated data presentation from HGB-206 clinical study in patients with SCD at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.
Updated data presentation from ALD-102 in patients with CALD by the end of 2020.
Commercial and Foundation Building
ZYNTEGLO first commercial patients treated in Europe in the second half of 2020.
ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
FIRST QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2020 and December 31, 2019 were $1.02 billion and $1.24 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $21.9 million for the three months ended March 31, 2020 compared to $12.5 million for the three months ended March 31, 2019. The increase was primarily attributable to an increase in ide-cel license and manufacturing service revenue under our agreement with BMS, as well as an increase in royalty revenue.
R&D Expenses: Research and development expenses were $154.1 million for the three months ended March 31, 2020 compared to $122.6 million for the three months ended March 31, 2019. The increase was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $73.2 million for the three months ended March 31, 2020 compared to $60.3 million for the three months ended March 31, 2019. The increase was largely attributable to costs incurred to support the company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $202.6 million for the three months ended March 31, 2020 compared to $164.4 million for the three months ended March 31, 2019.
CONFERENCE CALL DETAILS

bluebird bio will hold a conference call to discuss business updates and first quarter 2020 financial results on Monday, May 11 at 8:00AM ET.

Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 335-5158.

On May 11, 2020 Bristol Myers Squibb (NYSE: BMY) reported the presentation of data across its portfolio, aimed at addressing solid tumor and hematologic malignancies in 28 types of cancer, at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO20) Virtual Scientific Program, which will take place from May 29 to May 31, 2020 (Press release, Bristol-Myers Squibb, MAY 11, 2020, View Source [SID1234557449]).

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Presentations will feature clinical and non-clinical studies highlighting the potential role of immunotherapy, including combination approaches, to deliver durable treatment outcomes in multiple hard-to-treat solid tumors. In hematology, presentations will demonstrate the company’s innovative research in multiple myeloma, through both BCMA-targeted CAR T and proof-of-concept CELMoD data, suggesting targeted protein degradation as a potential new treatment approach. In addition, precision treatment approaches will explore how new biomarker insights may aid in the selection of optimal therapies for patients based on disease biology.

Accepted abstracts will be available on the ASCO (Free ASCO Whitepaper) conference website on Wednesday, May 13 at 5 p.m. EDT and the embargo will lift for all data included in these presentations at that time. Overall, data from more than 50 company-sponsored studies and collaborations will be featured at the meeting:

Solid Tumor

First presentation of results from CheckMate -9LA evaluating Opdivo (nivolumab) plus Yervoy (ipilimumab) with limited chemotherapy, and three-year follow-up data from CheckMate -227 evaluating Opdivo plus Yervoy combination in first-line metastatic non-small cell lung cancer
Treatment-free survival data from analyses of Opdivo plus Yervoy in advanced melanoma and new data in patients with immunotherapy-resistant renal cell carcinoma (RCC)
Analyses of multiple biomarkers associated with Opdivo plus Yervoy or Opdivo in patients with RCC
Cell Therapy

Pivotal trial results from KarMMa study of idecabtagene vicleucel (ide-cel; bb2121), a potential first-in-class BCMA CAR T in multiple myeloma being developed with bluebird bio
Updated data from EVOLVE Phase 1 study of orva-cel (orvacabtagene autoleucel), a fully human BCMA CAR T being developed by Juno Therapeutics, a Bristol-Myers Squibb Company, in heavily pre-treated multiple myeloma patients
Hematology

First clinical disclosure for CC-92480, a novel CELMoD agent evaluated in combination with dexamethasone in patients with relapsed and refractory multiple myeloma
Multiple analyses from the QUAZAR-001 pivotal Phase 3 study evaluating CC-486 maintenance therapy in acute myeloid leukemia
"This year’s ASCO (Free ASCO Whitepaper) meeting underscores the science-driven approach of our development program in solid tumor and hematologic malignancies, our dedication to precision medicine, as well as our commitment to helping to deliver durable improvement in patient outcomes through potentially transformative therapies," said Samit Hirawat, M.D., executive vice president, chief medical officer, global drug development, Bristol Myers Squibb. "With the impact of COVID-19, we are committed to supporting patients with cancer and overcoming challenges that patients, physicians and the research community may face during this time. We look forward to coming together virtually to share important data that continues to advance innovation for patients."

Summary of Presentations:

Selected Bristol Myers Squibb studies at the 2020 ASCO (Free ASCO Whitepaper) Virtual Annual Meeting include:

Gastrointestinal Malignancies

Nivolumab (NIVO) + low-dose ipilimumab (IPI) as first-line (1L) therapy in microsatellite instability-high/mismatch repair-deficient (MSI-H/dMMR) metastatic colorectal cancer (mCRC): Two-year clinical update
Author: Heinz-Josef Lenz
Abstract: #4040
Poster Session: Gastrointestinal Cancer—Colorectal and Anal (Poster: #32)
Phase 3 APACT trial of adjuvant nab-paclitaxel plus gemcitabine (nab-P + gem) vs. gem alone for patients with resected pancreatic cancer (PC): outcomes by geographic
Author: Reni
Abstract: #4515
Poster Session: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary (Poster: #123)
Genitourinary Malignancies

Biomarker analyses from the Phase 3 CheckMate -214 trial of Opdivo plus Yervoy or sunitinib in aRCC
Author: Motzer
Abstract: #5009
Clinical Science Symposium: Updates on Immunotherapy Biomarkers Development in Kidney and Bladder Cancers
Evaluation of predictive biomarkers for Opdivo in patients with mccRCC from the CheckMate -025 (CM -025) trial
Author: Ficial
Abstract: #5023
Poster Session: Genitouinary Cancer—Kidney and Bladder (Poster: #92)
FRACTION-RCC: Innovative, high-throughput assessment of Opdivo plus Yervoy for treatment refractory aRCC
Author: Choueiri
Abstract: #5007
Oral Session: Genitourinary Cancer—Kidney and Bladder
Immunogenomic characterization of advanced clear cell RCC treated with PD-1 blockade
Author: Braun
Abstract: #5010
Clinical Science Symposium: Updates on Immunotherapy Biomarkers Development in Kidney and Bladder Cancers
Leukemia

CC-486 is safe and well-tolerated as maintenance therapy in elderly patients (≥75 years) with acute myeloid leukemia (AML) in first remission following induction chemotherapy: Results from the phase III QUAZAR AML-001 trial
Author: Ravandi
Abstract: #7530
Poster Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant (Poster: #303)
Enasidenib plus azacitidine significantly improves complete remission and OR vs azacitidine alone in patients with newly diagnosed AML with isocitrate dehydrogenase 2 (IDH2) mutations: Results of a randomized Phase 2 study
Author: DiNardo
Abstract: #7501
Oral Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant
Escalated dosing schedules of CC-486 for patients experiencing first acute myeloid leukemia (AML) relapse: Results from the phase III QUAZAR AML-001 maintenance trial
Author: Doehner
Abstract: #7513
Poster Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant (Poster: #286)
Health-related quality of life (HRQoL) in the Phase III QUAZAR-AML-001 trial of CC-486 maintenance therapy for patients with acute myeloid leukemia (AML) in first remission following induction chemotherapy (IC)
Author: Roboz
Abstract: #7533
Poster Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant (Poster: #306)
Longer-term RBC transfusion reduction in the Phase 3 MEDALIST study of luspatercept in patients with lower-risk MDS with ring sideroblasts (RS)
Author: Komrokji
Abstract: #7518
Poster Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant (Poster: #291)
Lymphoma

Burden of cytokine release syndrome (CRS) and neurologic events (NE) in patients (Pts) with relapsed/refractory non-Hodgkin lymphoma (NHL) receiving lisocabtagene maraleucel (Liso-cel; JCAR017) in TRANSCEND NHL 001
Author: Abramson
Abstract: #6637
Poster Session: Health Services Research, Clinical Informatics, and Quality of Care (Poster: #328)
Lisocabtagene maraleucel (liso-cel) for treatment of second-line (2L) transplant noneligible (TNE) relapsed/refractory (R/R) aggressive large B-cell non-Hodgkin lymphoma (NHL): updated results from the PILOT study
Author: Sehgal
Abstract: #8040
Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia (Poster: #373)
Opdivo plus brentuximab vedotin for R/R classical Hodgkin lymphoma (cHL) in children, adolescents, and young adults (CAYA)
Author: Cole
Abstract: #8013
Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia (Poster: #346)
Outpatient treatment with lisocabtagene maraleucel (liso-cel) across a variety of clinical sites from 3 ongoing clinical studies in relapsed/refractory (R/R) large B-cell lymphoma (LBCL)
Author: Bachier
Abstract: #8037
Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia (Poster: #370)
Melanoma

Estimating treatment-free survival (TFS) over extended follow-up in patients (pts) with advanced melanoma (MEL) treated with immune-checkpoint inhibitors (ICIs): Five-year follow-up of CheckMate 067
Author: Regan
Abstract: #10043
Poster Session: Melanoma/Skin Cancers (Poster: #392)
Integrative tumor and immune cell multi-omic analyses predict melanoma response to immune checkpoint blockade 
Author: Anagnostou
Abstract: #10009
Clinical Science Symposium: Systems Biology Approaches to Immunotherapy Response and Toxicity
Multiple Myeloma

First-in-human phase I study of the novel CELMoD agent CC-92480 combined with dexamethasone (DEX) in patients (pts) with relapsed/refractory multiple myeloma (RRMM)
Author: Richardson
Abstract: #8500
Oral Session: Hematologic Malignancies—Plasma Cell Dyscrasia
Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T-cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): Initial KarMMa results
Author: Munshi
Abstract: #8503
Oral Session: Hematologic Malignancies—Plasma Cell Dyscrasia
Orvacabtagene autoleucel (orva-cel), a B-cell maturation antigen (BCMA)-directed CAR T cell therapy for patients (pts) with relapsed/refractory multiple myeloma (RRMM): update of the phase 1/2 EVOLVE study (NCT03430011)
Author: Mailankody
Abstract: #8504
Oral Session: Hematologic Malignancies—Plasma Cell Dyscrasia
KarMMa-RW: a study of real world treatment patterns in heavily pretreated patients with relapsed and refractory multiple myeloma (RRMM) and comparison of outcomes to KarMMa
Author: Jagannath
Abstract: #8525
Poster Session: Hematologic Malignancies—Plasma Cell Dyscrasia (Poster: #425)
Non-Small Cell Lung Cancer

Nivolumab + ipilimumab versus platinum-doublet chemotherapy as first-line treatment for advanced non-small cell lung cancer: Three-year update from CheckMate -227 Part 1
Author: Ramalingam
Abstract: #9500
Oral Session: Lung Cancer—Non-Small Cell Metastatic
Nivolumab (NIVO) + ipilimumab (IPI) + 2 cycles of platinum-doublet chemotherapy (chemo) vs 4 cycles chemo as first-line (1L) treatment (tx) for stage IV/recurrent non-small cell lung cancer (NSCLC): CheckMate -9LA
Author: Reck
Abstract: #9501
Oral Session: Lung Cancer—Non-Small Cell Metastatic
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 patients’ quality of life, 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.

On May 11, 2020 Cardinal Health (NYSE: CAH) reported financial results for the third quarter of fiscal 2020 ended March 31, 2020 (Press release, Cardinal Health, MAY 11, 2020, View Source [SID1234557448]).Third quarter revenue was $39.2 billion, an increase of 11% from the third quarter of last year.

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Third quarter GAAP operating earnings increased 30% to $562 million. Non-GAAP operating earnings increased 8% to $719 million. GAAP diluted earnings per share (EPS) increased 20% to $1.19, while non-GAAP diluted EPS increased 2% to $1.62.

"I want to express my gratitude to our employees and our partners across the healthcare industry for their incredible efforts to help us perform our critical role in the supply chain during this unprecedented time," said Mike Kaufmann, CEO of Cardinal Health. "We remain committed to delivering products and solutions to front-line health care providers so they can safely serve and treat patients around the world. As we look toward and beyond the fourth quarter, we will continue to take strategic actions to fulfill this mission."

During the third quarters of fiscal 2020 and 2019, GAAP effective tax rates were 26.8% and 20.0%, respectively. Non-GAAP effective tax rates were 25.7% and 21.6%

Third quarter revenue for the Pharmaceutical segment increased 12 percent to $35.1 billion, due to sales growth from Pharmaceutical Distribution customers and, to a lesser extent, Specialty Solutions customers. This growth included an acceleration in overall pharmaceutical sales in March, which the company believes was due to the COVID-19 pandemic.

Pharmaceutical segment profit was flat at $534 million in the third quarter. This reflects strong performance in the company’s generics program and the adverse impact of Pharmaceutical Distribution customer contract renewals.

Third quarter revenue for the Medical segment increased 5 percent to $4.1 billion, due to growth in products and distribution and Cardinal Health at Home.

Medical segment profit increased 15 percent to $178 million in the third quarter due to an increase in products and distribution, including benefits from global manufacturing and cost savings initiatives.

Fiscal year 2020 Outlook
While both segments experienced a modest net positive impact in the third quarter from increased volume related to the COVID-19 pandemic, the company expects a significant net negative impact to fourth quarter financial results in both segments. This is driven most meaningfully by a decrease in volume related to the cancellation or deferral of elective medical procedures.

The company does not provide forward-looking guidance on a GAAP basis as certain financial information, the probable significance of which cannot be determined, is not available and cannot be reasonably estimated. See "Use of Non-GAAP Measures" following the attached schedules for additional explanation.

The company reaffirms its fiscal year 2020 guidance range for non-GAAP diluted earnings per share attributable to Cardinal Health, Inc. of $5.20 to $5.40.

Recent highlights

Jason Hollar joined the company on April 27 and will become Chief Financial Officer on May 26. Jason previously served as CFO of Tenneco Inc. and Sears Holdings Corporation.

Cardinal Health board of directors approved a quarterly dividend of $0.4859 per share. The dividend will be payable on July 15, 2020 to shareholders of record at the close of business on July 1, 2020.

The company was recognized as a "2020 NAFE Top Companies for Executive Women" by the National Association for Female Executives for the ninth consecutive year.

Webcast
Cardinal Health will host a webcast today at 8:30 a.m. Eastern to discuss third quarter results. To access the webcast and corresponding slide presentation, go to the Investor Relations page at ir.cardinalhealth.com. No access code is required.

Presentation slides and a webcast replay will be available until May 10, 2021.

On May 11, 2020 AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo)’s Enhertu (trastuzumab deruxtecan) reported that it has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of patients with HER2-positive unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma who have received two or more prior regimens including trastuzumab (Press release, AstraZeneca, MAY 11, 2020, View Source [SID1234557441]).

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Gastric cancer is the third leading cause of cancer mortality with a five-year survival rate of 5% for metastatic disease.1,2 Approximately one in five gastric cancers are considered HER2 positive.3,4

The Food and Drug Administration’s (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging early clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

José Baselga, Executive Vice President, R&D Oncology, said: "Current therapy options are limited for patients with HER2-positive metastatic gastric cancer and for those who relapse, there are no approved HER2-targeted medicines. We look forward to working with the FDA to further explore the potential of Enhertu to become an important new treatment and the first antibody drug conjugate for this devastating disease."

Gilles Gallant, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo, said: "DESTINY-Gastric01 represents the first randomised trial of Enhertu to demonstrate clinically meaningful and statistically significant results, including objective response and survival increases compared to physician’s choice of chemotherapy. We are thrilled that the FDA has granted Enhertu a second Breakthrough Therapy Designation."

The FDA granted BTD based on data from the registrational Phase II DESTINY-Gastric01 trial and data from the Phase I trial published in The Lancet Oncology.5,6 In DESTINY-Gastric01, patients treated with Enhertu, a HER2-directed antibody drug conjugate (ADC), demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR), the primary endpoint, and overall survival (OS), a key secondary endpoint, versus patients treated with investigator’s choice of chemotherapy (irinotecan or paclitaxel monotherapy).

The overall safety and tolerability profile of Enhertu (6.4 mg/kg) in DESTINY-Gastric01 was consistent with that seen in the Phase I trial. The most common adverse events were haematologic and gastrointestinal including neutrophil count decrease, anaemia, nausea and decreased appetite. There were cases of drug-related interstitial lung disease (ILD) and pneumonitis, the majority of which were Grade 1 and 2, with two Grade 3 and one Grade 4. No ILD-related deaths (Grade 5) occurred in patients with gastric cancer in the Phase I trial or in the Phase II DESTINY-Gastric01 trial.

The full results of DESTINY-Gastric-01 will be presented during the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program.

Enhertu received SAKIGAKE designation in March 2018 by Japan’s Ministry of Health, Labour and Welfare (MHLW) for potential use in the same HER2-positive gastric cancer patient population and was recently submitted to the MHLW for approval. This is the second BTD granted for Enhertu in the US. Enhertu received BTD in 2017 for HER2-positive metastatic breast cancer and received approval in December 2019.

Gastric cancer

Gastric (stomach) cancer is the fifth most common cancer worldwide and the third leading cause of cancer mortality; there were approximately one million new cases reported in 2018 and 783,000 deaths.1 In the US, it is estimated that 27,600 new cases of gastric cancer will be diagnosed in 2020 and more than 11,000 people will die from the disease.7

Approximately one in five gastric cancers are HER2 positive.2,4 HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumours including gastric, breast and lung cancers. Gastric cancer is usually diagnosed in the advanced stage, but even when diagnosed in earlier stages of the disease the survival rate remains modest.8 Recommended 1st-line treatment for HER2-positive advanced or metastatic gastric cancer is combination chemotherapy plus trastuzumab, an anti-HER2 medicine, which has been shown to improve survival outcomes when added to chemotherapy. For gastric cancer that progresses on 1st-line treatment, there are no other approved HER2-targeting medicines.9

DESTINY-Gastric01

DESTINY-Gastric01 is a registrational Phase II, open-label, multi-centre trial testing the safety and efficacy of Enhertu in a primary cohort of 188 patients from Japan and South Korea with HER2-expressing, advanced gastric cancer or gastroesophageal junction adenocarcinoma (defined as IHC3+ or IHC2+/ISH+) who have progressed on two or more prior treatment regimens including fluoropyrimidine and platinum chemotherapy and trastuzumab. Patients were randomised 2:1 to receive Enhertu or investigator’s choice of chemotherapy (paclitaxel or irinotecan monotherapy). Patients were treated with Enhertu 6.4 mg/kg once every three weeks or chemotherapy. The primary endpoint of the trial is ORR, as assessed by an independent review committee. Secondary endpoints include OS, progression-free survival, duration of response, disease control rate and time to treatment failure as well as pharmacokinetic and safety endpoints.5

Enhertu

Enhertu (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the US) is a HER2-directed ADC and is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells.

Enhertu is approved in the US and Japan for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the DESTINY-Breast01 trial.

Enhertu clinical development

A comprehensive development programme is underway globally with six registrational trials evaluating the efficacy and safety of Enhertu monotherapy across multiple HER2-driven cancers including breast, gastric and lung cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.

Collaboration between AstraZeneca and Daiichi Sankyo

In March 2019, AstraZeneca and Daiichi Sankyo entered into a global collaboration to jointly develop and commercialise Enhertu worldwide, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is solely responsible for manufacturing and supply.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With six new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investment that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and ADCs – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.