On May 5, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2021 and shared recent operational progress (Press release, bluebird bio, MAY 5, 2021, View Source [SID1234579190]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Undoubtedly the highlight of last quarter at bluebird was the approval of Abecma, the first and only CAR T therapy approved for the treatment of relapsed or refractory multiple myeloma," said Nick Leschly, chief bluebird. "We and our colleagues at BMS are now full speed ahead and on track to begin treating patients this quarter. It has been an amazing journey and in many ways, we’re just getting started. While the oncology team has been delivering on Abecma, the severe genetic disease team met the moment. We quickly completed an investigation of the SUSAR of AML in our HGB-206 study of LentiGlobin gene therapy for SCD and determined that it was highly unlikely to be due to BB305 lentiviral vector. With these data and the other event changed from an MDS diagnosis to transfusion-dependent anemia, we are now quickly moving to engage with regulators with a goal of lifting the clinical holds in mid-2021. Amidst these challenges and work towards the planned separation, I want to commend and thank all birds for truly demonstrating anti-fragility and continuing to keep patients at the center of everything we do."

BUSINESS SEPARATION UPDATE

Today, bluebird bio is providing additional detail regarding the company’s planned business separation, which is targeted for completion by year-end 2021.

bluebird bio

bluebird bio is announcing that Tom Klima will join the company as chief commercial officer. Tom brings a track record of success across multiple commercial roles in oncology and rare diseases, most recently at Gamida Cell Ltd., where he served as chief commercial officer.

Additional members of the bluebird bio leadership team focused on severe genetic diseases will include:

Andrew Obenshain, chief executive officer (previously-disclosed)
Jason Cole, chief business officer
Rich Colvin, interim chief medical officer
Anne-Virginie Eggiman, senior vice president, regulatory science
"As we move towards separation, I’m pleased to have key leadership team members in place that are poised to bring bluebird bio to its next phase of success," said Andrew Obenshain. "With the addition of Tom Klima to lead our commercial efforts, we are rounding out our team of experts with a deep level of expertise from early stage clinical development to commercial delivery. I’m excited about our path forward and look forward to continuing to expand the bluebird team to bring gene therapy to patients with severe genetic diseases."

Oncology NewCo – 2seventy bio

Today, the company is announcing that Oncology NewCo will be named 2seventy bio and members of the leadership team will include:

Nick Leschly, chief executive officer (previously-disclosed)
Chip Baird, chief financial officer
Philip Gregory, chief scientific officer
Nicola Heffron, chief operating officer
"Two hundred seventy miles per hour is the maximum speed of human thought," said Nick Leschly, chief executive officer. "The name 2seventy was selected to signify this speed and our team’s translation of thought to action as we advance our next generation pipeline of transformative cell therapies to help cancer patients urgently in need."

RECENT HIGHLIGHTS

MULTIPLE MYELOMA

ABECMA FDA APPROVAL – On March 26, 2021, bluebird bio and Bristol-Myers Squibb announced that the U.S. Food and Drug Administration (FDA) approved Abecma (idecabtagene vicleucel; ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is a personalized immune cell therapy approved as a one-time infusion with a recommended dose range of 300 to 460 x 106 CAR-positive T cells.
KARMMA NEJM PUBLICATION– On February 24, 2021, bluebird bio and Bristol-Myers Squibb announced that results from the pivotal Phase 2 KarMMa study were published in The New England Journal of Medicine. The KarMMa study met its primary endpoint of overall response rate and key secondary endpoint of complete response rate. The data from the study demonstrates deep and durable responses with ide-cel treatment in triple-class exposed RRMM patients (n=128).
SICKLE CELL DISEASE

CLINICAL STUDIES UPDATE – On April 20, 2021, bluebird bio announced a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111). Upon further assessment, the treating investigator concluded this is not a case of MDS and revised the diagnosis to transfusion-dependent anemia. In addition, on March 10, 2021, bluebird bio reported that it is very unlikely the suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector (LVV). bluebird bio continues to work with regulators to resume its clinical studies in sickle cell disease as well as to remove the clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel for β-thalassemia, with potential lift of all clinical holds in mid-2021.
CEREBRAL ADRENOLEUKODYSTROPHY

ELI-CEL DATA AT EBMT– On March 15, 2021, bluebird bio presented new data suggesting durability of response and a strong safety profile post elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021). Long-term results from the Phase 2/3 Starbeam study of eli-cel, showed that ninety percent of patients (27/30) are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up. In the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104) there were no reports of graft failure, graft rejection, GVHD, replication competent lentivirus or insertional oncogenesis.
PIPELINE

BILL & MELINDA GATES FOUNDATION GRANT TO EXPLORE IN-VIVO LVV APPLICATIONS – bluebird bio is announcing today that it has received a grant from the Bill & Melinda Gates Foundation to explore new, potentially transformative in vivo treatments for SCD using the Company’s proprietary lentiviral vector (LVV) platform. The funding will support the research and development of novel LVVs that target hematopoietic stem cells (HSCs) for in vivo administration to bring gene-based therapies for SCD and other potential indications to patients around the world who may have limited access to ex vivo and other emerging therapies. This research may also enable the application of in-vivo LVV approaches in other severe genetic diseases.
PSIOXUS PRE-CLINICAL DATA – On April 14, 2021, bluebird bio and PsiOxus Therapeutics presented preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021. The results showed synergistic activity between PsiOxus’ T-SIGn vector and bluebird bio’s CAR-T therapy in primary and metastatic solid tumors. A single IV cycle of PsiOxus’ T-SIGn vector enabled an otherwise non-effective dose of CAR-T cell therapy to clear primary and metastatic tumors in vivo.
COMPANY

BUSINESS OPERATIONS – On April 20, 2021 bluebird bio announced its decision to withdraw ZYNTEGLO (betibeglogene autotemcel, beti-cel) for transfusion-dependent β-thalassemia (TDT) from the German market because reimbursement negotiations in Germany did not result in a price for ZYNTEGLO that reflects the value of this one-time gene therapy with potential life-long benefit for people living with TDT. Due in part to this decision, the company also announced a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs. This reduction and reallocation of resources will allow the company to focus on priority European markets and streamline global operations going forward to ensure its ability to deliver gene therapies to patients.
UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

SCD: The company is investigating the recently-reported safety events and plans to continue to work closely with the FDA in their review of these events to provide an update on the Company’s development plan and timeline for submission for regulatory approval by year end.
TDT: The company is on track to complete its rolling BLA submission to the U.S. FDA for beti-cel in mid-2021, contingent upon successful resolution of any U.S. FDA concerns applicable to the program arising out of the recently-reported safety events in the SCD program. This submission is anticipated to include adult, adolescent and pediatric patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
CALD: The company is on track to complete its BLA submission to the U.S. FDA for eli-cel in mid-2021. The company plans to receive European approval for eli-cel in patients with CALD in mid-2021.
Clinical Updates and Milestones

Updated data from ongoing clinical study in patients with SCD by the end of 2021.
Updated data from ongoing clinical studies in patients with TDT in mid-2021.
Updated clinical data from the ongoing pivotal Phase 2 KarMMa study of Abecma (ide-cel, bb2121) in patients with relapsed and refractory multiple myeloma to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 (ASCO21) Virtual Scientific Program on June 4.
bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2021.
Submission of 1 – 2 investigational new drug (IND) applications by the end of 2021.
Commercial and Foundation Building

Abecma first commercial patients treated in the first half of 2021.
Company

bluebird bio anticipates the separation of its severe genetic disease and oncology businesses into two independent, publicly traded companies (bluebird bio and 2seventy bio) to be completed by the end of 2021.
FIRST QUARTER 2021 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2021 and December 31, 2020 were $1.09 billion and $1.27 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities.
Revenues: Total revenues were $12.8 million for the three months ended March 31, 2021 compared to $21.9 million for the three months ended March 31, 2020. The decrease was primarily driven by a decrease in ide-cel license and manufacturing services revenue and a decrease in revenue recognized in connection with treating patients in the Phase 1 CRB-402 study of bb21217 under our agreements with BMS.
R&D Expenses: Research and development expenses were $154.5 million for the three months ended March 31, 2021 compared to $154.1 million for the three months ended March 31, 2020. The increase was primarily driven by increased costs incurred through the amended BMS collaboration as well as an increase in employee compensation, benefit, and other headcount related expenses. These increased costs were partially offset by a decrease in manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses were $86.9 million for the three months ended March 31, 2021 compared to $73.2 million for the three months ended March 31, 2020. The increase was primarily driven by increased employee compensation, benefit, and other headcount related expenses, as well as an increase in consulting fees associated with the ongoing project to separate the Company’s severe genetic disease and oncology programs into two independently traded companies. These increased costs were partially offset by a decrease in costs related to commercial readiness activities due to delays in commercialization as a result of the COVID-19 pandemic and in light of safety events in the HGB-206 study of LentiGlobin gene therapy for SCD.
Net Loss: Net loss was $205.8 million for the three months ended March 31, 2021 compared to $202.6 million for the three months ended March 31, 2020.

On May 5, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported business updates and financial results for the first quarter ended March 31, 2021 (Press release, Alector, MAY 5, 2021, View Source [SID1234579189]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Positive momentum continues to build throughout our early and late-stage product pipeline," said Arnon Rosenthal, Ph.D., co-founder and chief executive officer of Alector. "Our pivotal AL001 INFRONT-3 Phase 3 trial and AL002 INVOKE-2 placebo-controlled Phase 2 trial continue to enroll patients, and we look forward to sharing additional data from the AL001 Phase 2 clinical development program in people with FTD-GRN this summer at AAIC. In parallel, we continue to prepare for the initiation of a Phase 2 study evaluating AL001 in people with amyotrophic lateral sclerosis and advance our early-stage novel immuno-oncology candidates toward first-in-human studies. This is an exciting time for Alector and we look forward to continued execution on our clinical and corporate objectives."

Key Business Updates

Progranulin Portfolio:

Alector plans to present updated data from the ongoing AL001 Phase 2 open-label study at the upcoming Alzheimer’s Association International Conference (AAIC), taking place July 26-30, 2021.

Enrollment is ongoing in INFRONT-3, a randomized, placebo-controlled, pivotal Phase 3 trial evaluating AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). The ongoing pivotal trial is evaluating the efficacy and safety of AL001 in at-risk and symptomatic participants with FTD-GRN. Participants in the trial will be given the option to continue receiving treatment in an open-label extension study.

The Company is on track to initiate a Phase 2 study evaluating AL001 in people with amyotrophic lateral sclerosis (ALS) caused by C9orf72 repeats, which share TDP-43 pathology with FTD-GRN in the second half of 2021.
Alzheimer’s Disease Portfolio:

Alector continues patient enrollment in INVOKE-2, a Phase 2 trial evaluating AL002 in people with early Alzheimer’s disease. The randomized, double-blind, placebo-controlled, dose-ranging, multi-center Phase 2 study is expected to enroll approximately 265 participants with early Alzheimer’s disease (AD) at up to 90 sites globally. The AL002 clinical program is being developed in collaboration with AbbVie.

Alector plans to present two posters highlighting AL002 at AAIC, taking place July 26-30, 2021. The first poster will include the results of the AL002 Phase 1 study in healthy volunteers and the second poster will highlight the study design of the ongoing INVOKE-2 trial in people with early AD.

Data from the Phase 1b study evaluating AL003 in participants with AD is expected in 2021. Alector initiated the Phase 1b study in January 2020 and completed enrollment in 2020. The AL003 clinical development program is being developed in collaboration with AbbVie. Preliminary results from the study are expected to be presented at a scientific medical meeting in the second half of 2021.
Early-Stage Pipeline with Potential in Neurodegenerative Diseases and Oncology

The Company continues to progress AL044, a first-in-class antibody targeting the MS4A4A receptor. MS4A4A is a major risk gene for AD that encodes a transmembrane receptor protein that is expressed selectively in microglia in the brain and is associated with control of microglia functionality and potential viability.

Planning is underway to initiate first-in-human studies for AL008 and AL009, two oncology programs, in 2022. AL008 is a novel, investigational, antibody product candidate with a dual mechanism of action that combines inhibition of the CD47-SIRP-alpha (SIRPα) pathway with stimulation of activating Fc receptors and has the potential to yield a best-in-class product. AL009 is a first-in-class multi-Siglec inhibitor that works to enhance the innate and adaptive immune system response by blocking a critical glycan checkpoint pathway that drives immune inhibition.
First Quarter 2021 Financial Results

Revenue. Collaboration revenue for the quarter ended March 31, 2021, was $4.1 million, compared to $7.2 million for the same period in 2020. Revenue is recognized as the program costs are incurred by measuring actual costs incurred to date compared to the overall total expected costs to satisfy the performance obligation. Changes in estimates for revenue recognized over time are recognized on a cumulative basis.

R&D Expenses. Total research and development expenses for the quarter ended March 31, 2021, were $45.7 million, compared to $34.6 million for the same period in 2020. This increase was mainly driven by an increase in expenses to support the advancement of clinical and pre-clinical programs across several therapeutic initiatives as well as an increase in personnel-related expenses.

G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2021, were $11.1 million, compared to $14.6 million for the same period in 2020. This decrease was primarily due to a decrease in legal fees related to the conclusion of our arbitration proceedings for certain intellectual property matters.

Net Loss. For the quarter ended March 31, 2021, Alector reported a net loss of $52.2 million, compared to a net loss of $40.0 million for the same period in 2020.

Cash Position. Cash, cash equivalents, and marketable securities were $362.7 million as of March 31, 2021.

On May 5, 2021 Emergent BioSolutions Inc. (NYSE: EBS) reported that members of the company’s senior management team will participate in the following investor conferences (Press release, Emergent BioSolutions, MAY 5, 2021, View Source [SID1234579188]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Goldman Sachs Credit and Leveraged Finance Conference
May 17, 2021
1-on-1 meetings only
Benchmark Company Healthcare House Call Conference
May 26, 2021
1-on-1 meetings only
Singular Research Spring Select Webcall
May 27, 2021
Company presentation scheduled at 8:00 am Pacific / 11:00 am Eastern
For conferences where a presentation is planned, the company’s webcast presentation may include a discussion of the company’s recent business developments as well as its financial results and guidance. The webcast will be available both live, if possible, and by replay, and will be accessible from the Emergent website.

On May 5, 2021 MorphoSys AG (FSE:MOR; NASDAQ:MOR) reported its financial results for the first quarter of 2021 (Press release, MorphoSys, MAY 5, 2021, View Source [SID1234579187]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"For 2021, our focus is on three key areas: executing on the Monjuvi launch; rapidly advancing the tafasitamab backbone strategy through additional clinical studies; and expanding our pipeline," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "While we experienced headwinds from the pandemic in the first quarter, we are cautiously optimistic that the COVID-19 impact in the U.S. will start to diminish in the second half of 2021. We are confident in the potential of Monjuvi given its broad second-line label and overall profile in the r/r DLBCL setting. We are also making important progress initiating key trials for both tafasitamab and felzartamab this year."

Tafasitamab Highlights

Monjuvi(R) (tafasitamab-cxix) U.S. net product sales of € 12.9 million (US$ 15.5 million).
Monjuvi was granted a product-specific HCPCS J-Code, effective April 1, 2021.
On January 5, 2021, MorphoSys and Incyte announced that the Swiss Agency for Therapeutic Products (Swissmedic) had accepted the marketing authorization application (MAA) for tafasitamab and on January 12, 2021, MorphoSys and Incyte announced that Health Canada had accepted the New Drug Submission (NDS) for tafasitamab. Both applications seek approval for tafasitamab, in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from low grade lymphoma, who are not eligible for, or refuse, autologous stem cell transplantation (ASCT).
Pipeline Highlights

Felzartamab:

M-PLACE study of felzartamab in autoimmune membranous nephropathy ongoing: the safety run-in phase was completed and the full enrollment phase opened.
In February 2021, the first patient with autoimmune membranous nephropathy was dosed with felzartamab in the New-PLACE study, a phase 2 study evaluating different treatment schedules to identify the regimen for the pivotal study.
Otilimab:

On March 2, 2021, we announced that our partner GSK reported preliminary results of the OSCAR study using otilimab for the treatment of severe pulmonary COVID-19 related disease. Given these data suggest an important clinical benefit in a pre-defined sub-group of high-risk patients and the urgent public health need, GSK has amended the OSCAR study to expand this cohort to confirm these potentially significant findings. The dosing of the first patient in the expanded study triggered milestone payments totaling € 16 million to MorphoSys.
MOR210:

On January 25, 2021, MorphoSys and I-Mab announced that the first patient has been dosed in a phase 1 dose escalation study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of MOR210/TJ210 monotherapy in patients with relapsed or refractory advanced solid tumors in the United States.
Corporate Updates

On January 6, 2021, MorphoSys announced the appointment of Sung Lee as Chief Financial Officer, effective as of February 2, 2021.
Significant Events After The Reporting Period

On April 19, 2021, MorphoSys and Incyte announced that the first patient has been dosed in the placebo-controlled Phase 3 inMIND study evaluating the efficacy and safety of tafasitamab or placebo in combination with lenalidomide and rituximab in patients with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL).

First Quarter 2021 Financial Results (IFRS)

Total revenues for the quarter ended March 31, 2021 were € 47.2 million compared to € 251.2 million for the comparable period in 2020. The year-over-year decline was driven by the upfront payment of the collaboration and license agreement with Incyte in the first quarter 2020 for the out-licensing of tafasitamab outside the USA.

Cost of Sales: In the first three months of 2021, cost of sales increased to € 5.0 million (3M 2020: € 3.3 million).

Research and Development (R&D) Expenses: In the first three months of 2021, research and development expenses were € 33.3 million (3M 2020: € 21.5 million). Growth over 2020 reflects the increased investment to support the advancement of proprietary programs and consisted primarily of expenses for external laboratory services and personnel expenses.

Selling, General and Administrative (SG&A) Expenses: Selling expenses increased in the first three months of 2021 to € 28.2 million (3M 2020: € 12.8 million) and general and administrative expenses remained almost unchanged at € 10.3 million (3M 2020:€ 10.1 million). The year-over-year increase in selling expenses was primarily driven by the full quarter impact of the expenses for services provided by Incyte as part of the joint U.S. marketing activities for Monjuvi.

Operating Loss: Operating loss amounted to € 29.6 million in the first three months of 2021 (3M 2020: operating profit of € 203.5 million).

Consolidated Net Loss: For the first three months of 2021, consolidated net loss was € 41.6 million (3M 2020: consolidated net profit of € 195.5 million).

Cash and Investments: As of March 31, 2021, the Company had cash and investments of € 1,215.0 million compared to € 1,244.0 million on December 31, 2020.

Number of shares: The number of shares issued remained unchanged since year-end 2020 and totaled 32,890,046.

Financial Guidance and Operational Outlook for 2021

*Group revenues includes the announced € 16 million milestone payments from GSK, but excludes other potential significant milestones from development partners and/or licensing partnerships. This revenue guidance is subject to a number of uncertainties including the potential for variability from the first full year of the Monjuvi product launch, the limited visibility that MorphoSys has on the Tremfya royalty stream as well as the ongoing COVID-19 pandemic and the impact on our as well as our partner’s business operations.

**Operating expenses is comprised of R&D and SG&A, inclusive of Incyte’s share of Monjuvi selling costs in the USA.

MorphoSys expects for Tafasitamab the following events and activities in 2021:

Continuation of the phase 1b trial with tafasitamab in previously untreated DLBCL (firstMIND);
Initiation of a pivotal phase 3 trial of tafasitamab in previously untreated DLBCL (frontMIND);
Continuation of the phase 3 inMIND trial of tafasitamab in patients with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL);
Investigation of tafasitamab, plamotamab and lenalidomide in patients with relapsed or refractory DLBCL, first-line DLBCL and relapsed or refractory follicular lymphoma (r/r FL) jointly with Incyte and Xencor;
Continuation of the L-MIND study of tafasitamab and evaluate the long-term efficacy and safety data;
Continuation of the phase 3 B-MIND study of tafasitamab in combination with bendamustine for r/r DLBCL;
Presentation of data from the 3-year follow up of L-MIND as well as other abstracts at several scientific conferences (e.g. ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper));
Decision on the European Marketing Authorization Application (MAA), seeking approval of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with r/r DLBCL which is currently under review;
Support of Incyte in submitting marketing authorization applications in other markets.
MorphoSys will hold its conference call and webcast tomorrow, May 6, 2021, to present the results for the first quarter of 2021 and the further outlook for 2021.

A live webcast and slides will be made available at the Media and Investors section under Conferences on MorphoSys’ website at View Source and after the call, a slide-synchronized audio replay of the conference will be available at the same location.

The interim statement for the first quarter of 2021 (IFRS) is available online:
View Source/Reports

About tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi(R) is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi(R) is a registered trademark of MorphoSys AG.
XmAb(R) is a registered trademark of Xencor, Inc.

On May 20, 2021 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the first quarter ended March 31, 2021 (Press release, Tracon Pharmaceuticals, MAY 5, 2021, View Source [SID1234579186]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to be pleased with the pace of enrollment in the pivotal ENVASARC trial and remain on track to deliver interim data in the 2nd half of this year and final data in 2022," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We have now initiated 22 sites and have enrolled more than 20 patients which has triggered the initial Data Monitoring Committee review of safety data from each cohort, which we expect later this quarter."

Recent Corporate Highlights

Envafolimab

In April, we resubmitted our Orphan Drug Designation application to the FDA in response to a request for preclinical or clinical evidence of activity for envafolimab in sarcoma. We expect correspondence from the FDA this quarter based on the amended application.

As of May 5, we have initiated 22 U.S. clinical sites and enrolled more than 20 patients in the pivotal ENVASARC trial of single agent envafolimab and envafolimab combined with Yervoy, which has triggered the initial Data Monitoring Committee review of safety data from each cohort.
Expected Key Upcoming Milestones

Orphan Drug Designation for envafolimab in soft tissue sarcoma from FDA in 1H 2021.

Independent Data Monitoring Committee review of ENVASARC safety data in 1H 2021.

American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) presentation of ENVASARC pivotal trial design in 1H 2021.

ASCO presentation of TJ004309 Phase 1 data in 1H 2021.

Interim ENVASARC efficacy and safety data in 2H 2021.

Request FDA breakthrough therapy designation or Fast Track designation for envafolimab in 2H 2021.

Decision on the envafolimab New Drug Application (NDA) in MSI-H/dMMR cancer that is under priority review by the Chinese National Medical Products Administration (NMPA).
First Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments were $30.4 million at March 31, 2021, compared to $36.1 million at December 31, 2020. The Company expects that its current cash, cash equivalents and short-term investments will fund operations into the second half of 2022.

Research and development expenses for the first quarter of 2021 were $2.3 million, compared to $2.0 million for the first quarter of 2020.

General and administrative expenses for the first quarter of 2021 were $2.7 million, compared to $1.9 million for the first quarter of 2020.

Net loss for the first quarter of 2021 was $5.1 million, compared to $4.0 million for the first quarter of 2020.
Conference Call Details

Wednesday, May 5, at 4:30 PM Eastern Time / 1:30 PM Pacific Time
Domestic: 855-779-9066
International: 631-485-4859
Conference ID: 8852857
A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, has been studied in a completed Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients in China and is being studied in an ongoing Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China, with both Chinese trials sponsored by 3D Medicines. TRACON’s partners Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of the KEYNOTE-164 clinical trial.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multi-center, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.

About TRC102

TRC102 (methoxyamine) is a novel, small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the U.S. FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.