On June 30, 2022, Immatics N.V. (the "Company") Presented the investor presentation (Presentation, Immatics, JUN 30, 2022, View Source [SID1234616409]).

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On June 30, 2022 Genmab A/S (Nasdaq: GMAB) reported its intent to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) for subcutaneous epcoritamab (DuoBody-CD3xCD20), an investigational bispecific antibody, for the treatment of patients with relapsed/refractory large B-cell lymphoma (LBCL), in the second half of 2022 (Press release, Genmab, JUN 30, 2022, View Source [SID1234616408]).

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The BLA submission is supported by results from the large b-cell lymphoma (LBCL) cohort of the pivotal EPCORE NHL-1 open-label, multi-center trial evaluating the safety and preliminary efficacy of epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin lymphoma (B-NHL). In April 2022, Genmab and AbbVie announced the topline results from EPCORE NHL-1 trial. In June 2022, primary results were presented in a late-breaking oral presentation as part of the Presidential Symposium at the 27th Annual Meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2022) in Vienna, Austria.

"Relapsed or refractory large B-cell lymphoma is often difficult to treat, and patients are in need of novel therapies that are effective, tolerable and accessible," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "The results from the EPCORE NHL-1 trial, and other clinical trials evaluating epcoritamab in a variety of patients and treatment settings, have demonstrated that epcoritamab has the potential to offer people living with LBCL a new therapeutic advance with a manageable safety profile."

Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies are committed to evaluating epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies, including an ongoing phase 3, open-label, randomized trial evaluating epcoritamab as a monotherapy in patients with relapsed/refractory DLBCL (NCT: 04628494).

About Large B-cell Lymphoma (LBCL)
LBCL is a fast-growing type of non-Hodgkin’s lymphoma (NHL), a cancer that develops in the lymphatic system and affects B-cell lymphocytes, a type of white blood cell. There are an estimated 150,000 new LBCL cases each year globally. LBCL includes DLBCL, which is the most common type of NHL worldwide and accounts for approximately 31 percent of all NHL cases.i,ii,iii,iv

About the EPCORE NHL-1 Trial
EPCORE NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab including a phase 1 first-in-human, dose escalation part; a phase 2 expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-NHL, including LBCL and DLBCL. Data from the dose escalation part of the study, which determined the recommended phase 2 dose, were published in The Lancet in 2021. In the phase 2 expansion part, additional patients are treated with epcoritamab to further explore the safety and efficacy of epcoritamab in patients with different types of relapsed/refractory B-NHLs who had limited therapeutic options.

The primary endpoint of the phase 2 expansion part was overall response rate (ORR) as assessed by an IRC. Secondary efficacy endpoints included duration of response, complete response rate, progression-free survival, overall survival, time to response, time to next therapy, and rate of minimal residual disease negativity.

About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response towards target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T cell mediated killing of CD20+ cells.v CD20 is expressed on B-cells and a clinically validated therapeutic target in many B-cell malignancies, including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma and chronic lymphocytic leukemia.vi,vii

On June 30, 2022 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that research data on its novel immune-evasive universal CAR T-cells in Nature Communications, following an oral presentation at the American Society of Cell and Gene Therapy (ASGCT) (Free ASGCT Whitepaper) on May 16 (Press release, Cellectis, JUN 30, 2022, View Source [SID1234616406]).

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Cellectis’ next generation of universal CAR T-cells have the potential to improve the persistence and to allow large-scale deployment of T-cell product candidates in allogeneic settings against multiple malignancies.

Universal CAR T-cell therapies are poised to revolutionize the treatment of selected hematologic malignancies. However, realizing the full potential of CAR T in an allogeneic setting requires universal CAR T-cells that can kill target tumor cells, avoid depletion by the host immune system via the host versus graft reaction (HvG), and proliferate without attacking host tissues via the graft versus host reaction (GvH).

While the prevention of GvH can be readily addressed by the inactivation of T cell receptor T αβ (TCRαβ) expression in a CAR T-cell, prevention of HvG remains a major challenge.

To address this challenge, the Cellectis investigators engineered CAR T-cells to be deficient in Class 1 major histocompatibility complex (MHC-1) and to express the Natural Killer (NK) inhibitor HLA-E, in order to endow them with immune-evasive properties toward alloreactive Tc ells and NK cells.

"This engineering approach is very promising and could enable the universal CAR T-cells to become transiently invisible to NK and alloreactive T-cells, allowing them to eradicate tumor cells before being rejected by the patient’s immune system. This could enable the broad use of universal CAR T-cells in allogeneic settings, for the benefit of a wider population of patients." said Julien Valton, Ph.D., Vice President Gene Therapy at Cellectis.

"One potential advantage of this approach is to spare endogenous immune effectors and allow them to work in concert with CAR T-cells in the fight against hard-to-treat cancers, including solid tumors" said Laurent Poirot, Ph.D. Senior Vice President Immuno-Oncology at Cellectis.

The research data show that:

· Endowing universal CAR T-cells with immune-evasive properties via TALEN-mediated gene editing and adeno-associated virus (AAV)-dependent gene insertion, is highly efficient and specific, transferable to different CAR constructs and adaptable to conventional CAR T-cell manufacturing processes.

· Immune-evasive universal CAR T-cells overcame alloresponsive T-cell and NK cells attacks and exhibit prolonged antitumor activity, even in the presence of highly active NK cells.

· Immune-evasive properties against NK cells from most healthy donors and acute myeloid leukemia (AML) patients were similar, demonstrating the robustness and transportability of this hypoimmunogenic approach to persistence and warranting further evaluation in preclinical and clinical settings

On June 30, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported strategic financing collaborations with Sixth Street and Royalty Pharma (NASDAQ: RPRX) for up to $1.25 billion, bringing significant non-dilutive, low-cost capital to drive innovation and growth (Press release, Blueprint Medicines, JUN 30, 2022, View Source [SID1234616405]).

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These tailored investments by two highly respected life sciences-focused investors capitalize on Blueprint Medicines’ significant accomplishments to date and add strategic financial partners who are aligned with the company’s growth ambitions and confidence in the anticipated commercial opportunity and launch performance of AYVAKIT/AYVAKYT (avapritinib) and GAVRETO (pralsetinib). The financings provide capital to expand and advance the company’s robust and diverse pipeline towards commercialization and to continue pursuing strategic and synergistic business development opportunities.

"This attractive deal puts Blueprint Medicines in a very strong financial position to drive rapid growth while maintaining our path to profitability in the coming years. The combination of our strong cash position, multiple drivers of top-line revenue, and diversity of important pipeline programs uniquely positions us to continue building a leading precision therapy company and bring transformative medicines to patients worldwide," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Executing this deal with such favorable terms in the current market environment speaks to the quality of the assets, the aligned confidence in the commercial opportunities, and the investment opportunity that Blueprint Medicines represents overall for firms like Sixth Street and Royalty Pharma, with whom we are building long-term strategic relationships."

This multi-component deal is comprised of the following elements:

The agreement with Sixth Street has three parts:
$250 million cash upfront in exchange for future AYVAKIT/AYVAKYT and BLU-263 royalties at a rate of 9.75 percent subject to an annual cap of $900 million in net sales and a cumulative cap of 1.45 times invested capital;
Up to $400 million in a senior secured credit facility, of which Blueprint Medicines will draw $150 million initially with an additional $250 million available in delayed draw tranches at Blueprint Medicines’ election; and
$260 million in a potential credit facility to support buy-side business development opportunities, subject to mutual agreement between Sixth Street and Blueprint Medicines.
The agreement with Royalty Pharma monetizes royalties receivable from GAVRETO net sales by Roche outside of the US, not including Greater China, with $175 million cash paid to Blueprint Medicines upfront and up to $165 million in potential milestone payments based on future sales.
"Blueprint Medicines is an impressive and differentiated biopharmaceutical company, with a proven track record of success in developing and commercializing precision therapies. We are particularly excited about the opportunity for AYVAKIT to meet the substantial need in patients with non-advanced systemic mastocytosis," said Vijay Mohan and Jeff Pootoolal, Partners at Sixth Street. "We believe that investing now, when the company is already in a strong financial position, is the first step toward a long-term relationship that will open the door to further potential opportunities for growth and partnership."

"GAVRETO is an important precision therapy that has been incredibly meaningful for patients with metastatic, RET fusion-positive non-small cell lung cancer who may have otherwise had limited options," said Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer. "We are pleased to establish a partnership with the experienced team at Blueprint Medicines to help fuel their execution on the significant commercial and development opportunities they have ahead."

Cowen and Company served as financial advisor and Goodwin Procter LLP served as legal advisor to Blueprint Medicines. Cooley LLP acted as legal advisors to Sixth Street. Gibson Dunn acted as legal advisors to Royalty Pharma.

Investor Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss the collaborations. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 658541. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit AYVAKIT.com. Under the brand name AYVAKYT (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S. or Europe.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and non-advanced SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis.

Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT.

About GAVRETO (pralsetinib)

GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, and adults and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials. In addition, GAVRETO is approved by the National Medical Products Administration (NMPA) of China for the treatment of adult patients with locally advanced or metastatic RET fusion-positive NSCLC after platinum-based chemotherapy.

GAVRETO is not approved for the treatment of any other indication in the U.S. by the FDA or in China by the NMPA, or for any indication in any other jurisdiction by any other health authority.

GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In preclinical studies, GAVRETO inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2 and JAK2. For more information, visit GAVRETO.com.

Blueprint Medicines and Roche are co-developing GAVRETO globally (excluding Greater China) for the treatment of patients with RET-altered NSCLC, various types of thyroid cancer and other solid tumors. The European Medicines Agency validated a marketing authorization application for GAVRETO for the treatment of RET fusion-positive NSCLC. The FDA granted breakthrough therapy designation to GAVRETO for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

Please click here to see the full U.S. Prescribing Information for GAVRETO.

On June 30, 2022 Royal Philips (NYSE: PHG, AEX: PHIA), a global leader in health technology, reported it has teamed up with Biodesix, Inc. (Colorado, U.S.) (Nasdaq: BDSX), a leading data-driven diagnostic solutions company, to incorporate the results of Biodesix’s Nodify Lung blood-based lung nodule risk assessment testing into Philips Lung Cancer Orchestrator lung cancer patient management system (Press release, Biodesix, JUN 30, 2022, View Source [SID1234616404]). The incorporation of proteomics data, along with the radiologic and patient history data currently used to determine the treatment decisions, can help create diagnostic efficiency for cancer care centers in the management of a growing number of lung nodule cases. Philips Lung Cancer Orchestrator solution is designed to enable health systems to operationalize lung cancer screening and lung nodule management programs at scale.

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Lung cancer remains the leading cause of cancer deaths worldwide [1], with current 5-year survival rates after diagnosis typically less than 20% [2]. If detected and treated early, however, research shows 10-year survival rates could increase to more than 90% [3]. Thanks to the adoption of low-dose CT (computer tomography) screening and better management of incidental lung nodule findings, early-stage diagnosis and treatment is now possible, but has resulted in rapidly increasing caseloads. For example, recent changes in U.S. guidelines have nearly doubled the number of people now eligible for lung cancer screening [4].

"By incorporating Biodesix’s Nodify Lung testing, we take another step in leveraging integrated diagnostics from imaging, genomics, and now proteomic results from a simple blood draw to address key moments in the lung cancer patient journey, support care team decision-making, and help health systems learn from their practice patterns," said Louis Culot, General Manager Oncology Informatics and Genomics at Philips. "We expect the inclusion of Biodesix’s ground-breaking technology in Philips Lung Cancer Orchestrator to help drive more confident decisions for the care team, and ultimately benefit patients."

As a next-step after radiologic lung nodule assessment, minimally-invasive biopsy procedures such as endobronchial biopsy (accessing a nodule via the patient’s airways), are already helping early-stage diagnosis. With 62% of biopsies conducted on benign nodules, by detecting the presence of blood-based biomarkers combined with clinical and radiomic factors, Biodesix’s Nodify Lung blood-based proteomic tests help clinicians to reclassify the risk of malignancy to better target resources to those who need them. If a nodule is malignant, the delay in surgical care for cancer patients by four weeks increases the mortality by 6-8% [5].

"We are delighted that our tests are being incorporated into Philips’ vision for end-to-end cancer care management using a multi-diagnostic approach," said Scott Hutton, Chief Executive Officer at Biodesix. "By integrating our Nodify tests in Philips Lung Cancer Orchestrator we hope to make these tests more accessible to physicians and patients and more easily utilized by care teams with the ultimate goal of improving patient care and outcomes."

Philips Lung Cancer Orchestrator is an integrated patient management system for CT lung cancer screening programs and incidental lung nodule findings that keeps track of patients, appointments, diagnostic images, test results, and clinical decisions for every step of a lung cancer patient’s screening and treatment journey. Fitting seamlessly into conventional screening and diagnostic workflows, it coordinates the end-to-end patient journey to create a fully traceable, fully documented timeline of scheduled actions, and aggregates data on tumor staging, radiology, pathology, and biomarker analyses for informed decision-making. The ability to order and receive the results expedites Biodesix’s proteomic testing directly to and from the Lung Cancer Orchestrator at the same time it adds new insights on nodule risk of malignancy to further support the decision-making process.

Lung Cancer Orchestrator is part of Philips’ expanding portfolio of integrated cancer care solutions that seamlessly connect data, technology, operations, and clinical workflows to enable pivotal decision-making. Visit Philips Oncology to learn how Philips combines smart diagnostic and imaging technologies with connected workflows that integrate patient data from disparate systems, to provide solutions that put expert information at clinicians’ fingertips to help expand the quality and reach of personalized cancer care.