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.

On June 30, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, reported new data from RATIONALE 306, a global Phase 3 trial evaluating tislelizumab plus chemotherapy in adult patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) without prior systemic treatment for advanced disease (Press release, BeiGene, JUN 30, 2022, View Source [SID1234616403]). Study results presented today as a late-breaking oral presentation at the 2022 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer (Abstract #LBA-1) showed a statistically significant and clinically meaningful improvement in overall survival (OS) for patients receiving tislelizumab in combination with chemotherapy with a median OS of 17.2 months [95% CI: 15.8,20.1] versus 10.6 months [95% CI: 9.3,12.1] for those receiving chemotherapy plus placebo. The combination of tislelizumab with chemotherapy reduced the risk of death by 34% (HR=0.66 [95% CI: 0.54,0.80, p<0.0001]) compared to chemotherapy plus placebo.

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"We are encouraged by the consistent, clinically meaningful benefit seen with tislelizumab and chemotherapy in key endpoints measuring efficacy and durability of response and across pre-specified subgroups in this 1L ESCC treatment setting," said Mark Lanasa, M.D., Chief Medical Officer, Solid Tumors at BeiGene. "We sincerely appreciate the patients with ESCC from across the world who chose to participate in this study as we search for treatment options for this challenging condition."

The OS benefit for tislelizumab plus chemotherapy was observed regardless of baseline PD-L1 expression. The median OS for patients with PD-L1 score ≥10% was 16.6 months [(95% CI: 15.3,24.4] in the tislelizumab plus chemotherapy group versus 10.0 months [95% CI: 8.6,13.0] for patients receiving chemotherapy plus placebo (HR=0.62; 95% CI, 0.44,0.86, p=0.0020). Analysis of patients with a PD-L1 score <10% showed a median OS of 16.7 months [95% CI: 13.0,20.1] for tislelizumab plus chemotherapy versus 10.4 months [95% CI: 9.1,13.0]; (HR=0.72 [95% CI: 0.55,0.94]) for chemotherapy plus placebo. This survival benefit was consistent across all other pre-specified subgroups, including race, region, and choice of chemotherapy.

"ESCC represents the majority of esophageal cancer worldwide, but unfortunately chemotherapy by itself provides a median survival in the range of only a year, so the survival benefit seen when tislelizumab was added to chemotherapy in our study is compelling" said Harry Yoon, MD, Associate Professor of Oncology and Chair of the Gastroesophageal Cancer Disease Group at Mayo Clinic in Rochester, Minnesota. "Additionally, it is encouraging to see a familiar safety and tolerability profile for the combination consistent with those established for chemotherapy in the community."

Progression-free survival was significantly improved for the tislelizumab plus chemotherapy (7.3 months) group compared to chemotherapy alone (5.6 months) (HR=0.62 [95% CI: 0.52,0.75, p<0.0001]), Additional benefit in overall response (ORR) was seen with tislelizumab and chemotherapy compared to chemotherapy [ORR 63.5% vs 42.4%; p<0.0001) and the median duration of response was 7.1 months [95% CI: 6.1,8.1] for tislelizumab plus chemotherapy versus 5.7 months [95% CI: 4.4,7.1] chemotherapy alone.

The incidence of treatment-related adverse events (TRAEs) was similar in both arms; the most commonly reported TRAEs (≥ 20%) were anemia, decreased neutrophil count, decreased white blood cell count, decreased appetite, nausea and peripheral sensory neuropathy.

Tislelizumab is currently under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for advanced or metastatic ESCC after prior chemotherapy. The EMA is also reviewing tislelizumab for advanced or metastatic non-small cell lung cancer (NSCLC) after prior chemotherapy, and in combination with chemotherapy for previously untreated advanced or metastatic NSCLC. In January 2021, BeiGene announced a collaboration with Novartis to accelerate the clinical development and marketing of tislelizumab in North America, Europe, and Japan.

Tislelizumab is approved by the China National Medical Products Administration (NMPA) as a treatment for nine indications, including a recent approval for use in patients with locally advanced or metastatic ESCC who have disease progression or are intolerant to first-line standard chemotherapy. Tislelizumab is not approved for use outside of China.

About RATIONALE 306

RATIONALE 306 (NCT03783442) is a randomized, placebo-controlled, double-blind, global Phase 3 study to evaluate the efficacy and safety of tislelizumab in combination with chemotherapy as a first-line treatment in patients with advanced or metastatic ESCC. The primary endpoint of the trial is overall survival (OS). Secondary endpoints include progression free survival, overall response rate, duration of response per RECIST v1.1, and OS in patients with PD-L1 score ≥10%, as well as health-related quality of life measures and safety.

The trial enrolled 649 patients at research centers across Asia-Pacific, Europe, and North America. Patients were randomized 1:1 to receive either tislelizumab plus chemotherapy or placebo plus chemotherapy.

About Esophageal Squamous Cell Carcinoma

There are two main types of esophageal cancer, based on the cells where cancer develop: squamous cell carcinoma (ESCC) and adenocarcinoma (EAC). ESCC is the most common subtype of esophageal cancer, accounting for more than 85% of esophageal cancers worldwide.i,ii Because many patients are diagnosed with ESCC at later stages of disease, management of ESCC is challenging and the overall prognosis remains poor.iii,iv,v

About Tislelizumab

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. In pre-clinical studies, binding to Fcγ receptors on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells.

Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

BeiGene has initiated or completed more than 20 potentially registration-enabling clinical trials in 35 countries and regions, including 17 Phase 3 trials and four pivotal Phase 2 trials. More information on the clinical trial program for tislelizumab can be found at: View Source

BeiGene Oncology

BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 16,000 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the U.S., China, the European Union, Great Britain, Canada, Australia, and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the PARP inhibitor pamiparib in China.

On June 30, 2022 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for belzupacap sarotalocan (AU-011), Aura’s first VDC product candidate, for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) (Press release, Aura Biosciences, JUN 30, 2022, View Source [SID1234616402]).

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"We are pleased that belzupacap sarotalocan has been granted Fast Track designation. We believe that, given that NMIBC presents such a high unmet medical need, the opportunity for more frequent interactions with Division of Oncology at FDA and the potential for Priority Review will be valuable as we advance further into clinical development in patients with NMIBC," said Dr. Mark De Rosch, Chief Operating Officer and Head of Regulatory Affairs of Aura.

"NMIBC has no approved targeted therapies, and patients experience high levels of recurrence and progression, ultimately leading to cystectomy or the removal of the entire bladder," said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura. "This milestone supports our goal to advance the development of belzupacap sarotalocan for patients with NMIBC in need of better and earlier targeted treatment options with the potential to help preserve the bladder."

Aura’s planned Phase 1 clinical trial with belzupacap sarotalocan in this indication will evaluate the safety and early proof of mechanism, assess distribution, local necrosis and evidence of immune activation. Aura expects to initiate the trial in the second half of 2022, with initial Phase 1 data expected in 2023.

Fast Track designation is an FDA process designed to facilitate the development of products that address high unmet medical needs and may expedite the review of drugs intended to treat serious or life-threatening diseases. Features of Fast Track designation include opportunities for more frequent interactions with the FDA review team and, if supported by clinical data, eligibility for Priority Review. Belzupacap sarotalocan has also been previously granted Fast Track and Orphan Drug designations by the FDA for the treatment of choroidal melanoma and is currently in Phase 2 clinical development in this indication.