On January 7, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), have been selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy ("ASTCT") and Center for International Blood & Marrow Transplant Research ("CIBMTR"), taking place February 2 – 6, 2022 in Salt Lake City, Utah ("2022 Tandem Meetings") (Press release, Mustang Bio, JAN 7, 2022, View Source [SID1234598444]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center ("Fred Hutch").

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!

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The compelling clinical activity and favorable safety profile that MB-106 continues to demonstrate in the ongoing Phase 1/2 trial at Fred Hutch highlight its potential as an outpatient therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia. We look forward to the updated data that will be presented by Fred Hutch at the 2022 Tandem Meetings, in particular for patients with diffuse large B cell lymphoma and Waldenstrom’s macroglobulinemia. We also look forward to advancing our MB-106 CD20-targeted CAR T cell therapy program towards a multicenter trial under Mustang’s IND in the current quarter."

Details of the presentation are as follows:

Title: High Efficacy and Low Toxicity of MB-106, a Third Generation CD20 Targeted CAR-T for Treatment of Relapsed/Refractory B-NHL and CLL
Poster Number: 225
Dates and Times: Thursday, February 3, 6:45 pm to 8:15 pm and Saturday, February 5, 6:15 pm to 7:45 pm
Presenter: Mazyar Shadman, M.D., M.P.H., Associate Professor, Clinical Research Division, Fred Hutch, Seattle, WA; Physician at Seattle Cancer Care Alliance; Associate Professor, Division of Medical Oncology, University of Washington School of Medicine

For more information, please visit the 2022 Tandem Meetings website at View Source

Note: Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About MB-106 (CD20-targeted CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody, and MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the planned multicenter phase 1/2 clinical trial to be initiated shortly under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trial can be found at View Source using the identifier NCT03277729.

On January 7, 2022 Alexion, AstraZeneca’s Rare Disease group, reported that it has entered into an exclusive global collaboration and licence agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody currently in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) (Press release, AstraZeneca, JAN 7, 2022, View Source [SID1234598393]). NI006 specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart, with the potential to treat patients with advanced ATTR-CM.

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!

Under the agreement, Alexion will be granted an exclusive worldwide licence to develop, manufacture and commercialise NI006.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis.1 It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.2

Marc Dunoyer, Chief Executive Officer, Alexion, said: "With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed. We look forward to applying this expertise to the development of NI006, which is designed to clear cardiac amyloid fibril deposits with the potential to improve cardiac function for patients living with advanced ATTR-CM, who are currently underserved by existing treatment options."

There is a significant unmet medical need for patients with various types and levels of severity of amyloidosis that may require multiple mechanisms of action to address those needs. NI006, an ATTR depleter, adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens our broader commitment to addressing cardiomyopathies that can lead to heart failure.

Financial considerations
Alexion will pay Neurimmune an upfront payment of $30m with the potential for additional contingent milestone payments of up to $730m upon achievement of certain development, regulatory and commercial milestones, as well as low-to-mid teen royalties on net sales of any approved medicine resulting from the collaboration.

Neurimmune will continue to be responsible for completion of the current Phase Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing and commercialisation.

The transaction is expected to close following satisfaction of customary closing conditions and regulatory clearances.

Notes

Heart failure
Heart failure (HF) is a life-threatening chronic disease that prevents the heart from pumping sufficient levels of blood around the body. HF affects approximately 64 million people worldwide. HF remains as fatal as some of the most common cancers in both men (prostate and bladder cancers) and women (breast cancer).5 Chronic HF is the leading cause of hospitalisation for those over the age of 65 and represents a significant clinical and economic burden.6

AstraZeneca’s ambition is to be the leading company in HF, expanding from Forxiga today in heart failure with reduced ejection fraction (HFrEF), to the full HF spectrum including cardiomyopathies. AstraZeneca is investing in multiple investigational therapies with diverse mechanisms of action to address the spectrum of patient need in this area.

ATTR-CM
Cardiomyopathy due to ATTR is caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium. In patients with ATTR-CM, both the mutant and wild type TTR protein builds up as fibrils in tissues, including the heart. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.

Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM3,4; however, many of those patients remain undiagnosed.

NI006
NI006 is an investigational human monoclonal antibody that specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart.

On January 7, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, reported that it will present data on Iovance tumor-infiltrating lymphocyte (TIL) cell therapy at the upcoming Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, to be held February 2 – 6, 2022 in Salt Lake City, Utah, and virtually (Press release, Iovance Biotherapeutics, JAN 7, 2022, View Source [SID1234598409]). Details for the abstracts are as follows:

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!

Title: Successful Manufacturing of Tumor-Infiltrating Lymphocyte (TIL) Cell Therapy from Cryopreserved Melanoma Tumors Shipped from Australia
Presenting Author: Anandaraman Veerapathran, Iovance Biotherapeutics
Presentation Type: Poster
Abstract ID: 287

Title: Decitabine Treatment of Tumor-Infiltrating Lymphocytes (TIL) during Ex Vivo Expansion Induces a More Memory-like Phenotype, Reduces Inhibitory Receptor Expression, and Increases Functionality
Presenting Author: Rafael Cubas, Iovance Biotherapeutics
Presentation Type: Poster
Abstract ID: 270

Poster Display Hours: Wednesday, February 2, 2022 from 10:00 am to 8:00 pm; Thursday, February 3, 2022 from 7:00 am to 8:15 pm; Friday, February 4, 2022 from 7:00 am to 8:00 pm; and Saturday, February 5, 2022 from 7:00 am to 7:45 pm (all times MST)

Poster Receptions: Thursday, February 3, 2022 from 6:45 pm to 8:15 pm MST and Saturday, February 5, 2022 from 6:15 pm to 7:45 pm MST

On January 7, 2022 CytRx Corporation (OTCQB: CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported that John Caloz, the Company’s Chief Financial Officer, reported at the H.C. Wainwright BioConnect Virtual Conference, which is taking place virtually from Monday, January 10th through Thursday, January 13th (Press release, CytRx, JAN 7, 2022, View Source [SID1234598424]).

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!

Mr. Caloz’s presentation will focus on CytRx’s high-potential pipeline and assets, including its licensing agreements with ImmunityBio, Inc. and Orphazyme A/S. The presentation will also discuss the Company’s assessment of its Centurion BioPharma subsidiary and its LADR Platform, which aims to concentrate ultra-high-potency drugs in solid tumors and minimize systemic toxicity. In addition to the LADR Platform, the technology includes the ACDx diagnostic, which was developed to allow oncologists to determine which patients can benefit from the ultra-high-potency drugs.

The virtual presentation will be available on CytRx’s website under the News & Events section following the conference.

On January 7, 2022 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported that preliminary Monjuvi(R) U.S. net product sales for the full year of 2021 and provided financial guidance for 2022 (Press release, MorphoSys, JAN 7, 2022, View Source [SID1234598394]).

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!

Preliminary Monjuvi (tafasitamab-cxix) U.S. net product sales are US$ 23.6 million (€ 20.5 million) for the fourth quarter and US$ 79.1 million (€ 66.9 million) for the full year of 2021. Fourth quarter and full year 2021 results will be published on March 16, 2022.

"We are pleased that many patients have benefitted from Monjuvi since launch and we expect to see continued growth in 2022," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "The updated structure of our 2022 financial guidance better reflects our business model and provides greater transparency to the investment community."

Full Year 2022 Financial Guidance:

Amounts in million 2022 Financial Guidance 2022 Guidance Insights
Monjuvi U.S. Net Product Sales US$ 110m to 135m 100% of Monjuvi U.S. product sales are recorded on MorphoSys’ income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.
Gross Margin for Monjuvi U.S. Net Product Sales 75% to 80% 100% of Monjuvi U.S. product cost of sales is recorded on MorphoSys’ income statement and related profit/loss is split 50/50 between MorphoSys and Incyte.
R&D expenses € 300m to 325m 2022 growth over 2021 will be driven primarily by investment in ongoing pivotal phase-3 studies, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.
SG&A expenses € 155m to 170m 51% to 56% of mid-point of SG&A expenses represents Monjuvi U.S. selling costs of which 100% are recorded in MorphoSys’ income statement. Incyte reimburses MorphoSys for half of these selling expenses.
For 2022, we anticipate a year-over-year decline in SG&A, excluding transaction/restructuring/other charges related to Constellation acquisition recorded in 2021.

Additional information related to 2022 Financial Guidance:

– Tremfya royalties will continue to be recorded as revenue without any cost of sales in MorphoSys’ income statement. These royalties, however, will not contribute any cash to MorphoSys as 100% of the royalties will be passed on to Royalty Pharma.

– MorphoSys anticipates receiving royalties for Minjuvi(R) sales outside of the U.S. Guidance for these royalties is not being provided as MorphoSys does not receive any sales forecasts from its partner Incyte.

– MorphoSys does not anticipate any significant cash-accretive revenues from the achievement of milestones in 2022. Milestones for otilimab are passed on to Royalty Pharma. Milestones from all other programs remain with MorphoSys at 100%.

– MorphoSys anticipates sales of commercial and clinical supply of tafasitamab outside of the U.S. to its partner Incyte. Revenue from this supply is recorded in the "Licenses, milestones and other" category in MorphoSys’ income statement. These sales result in a zero gross profit/margin. As such, MorphoSys does not provide guidance for these sales.

– While R&D expense is anticipated to grow year-over-year due to investments in three pivotal studies, the growth is partially being offset by the consolidation of research/discovery activities.

– SG&A expense guidance range reflects savings from synergies following the acquisition of Constellation and streamlined commercialization efforts.

– Anticipated foreign exchange (USD/EUR) to impact operating expenses (R&D and SG&A) negatively by approximately 3%.

About Monjuvi(R) (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).
In the United States, 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 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 Europe, Minjuvi(R) (tafasitamab) received conditional approval, in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

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

Minjuvi(R) and Monjuvi(R) are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi(R) in the U.S., and marketed by Incyte under the brand name Minjuvi(R) in the EU.

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