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 Inceptor Bio, a biotechnology company advancing cell therapies for difficult-to-treat cancers, reported a collaboration with University of Minnesota (Press release, Inceptor Bio, JUN 30, 2022, View Source [SID1234616422]). The aim of this collaboration is to build a novel induced pluripotent stem cells (iPSC) platform that will accelerate Inceptor Bio’s best-in-class next-generation cell therapies platforms. Under the terms of the agreement, Inceptor Bio will receive an exclusive license to the technology developed under this collaboration.

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Inceptor Bio plans to advance multiple cell therapy products into clinical studies incorporating the iPSC platform into its proprietary K62 platform for CAR-M therapy, which increases the phagocytic capabilities of macrophages and supports an M1 anti-tumor phenotype, as well as its novel co-stimulatory domain, M83, for CAR-NK therapies.

"iPSC-derived cell therapies have the potential to enable the next frontier of cell therapies. We are excited to work with Dr. Beau Webber at University of Minnesota and his team to develop this unique platform," said Mike Nicholson, Ph.D., President and Chief Operating Officer at Inceptor Bio.

"The team at University of Minnesota is confident that Inceptor Bio is the right partner for building a differentiated iPSC platform to advance novel cell therapies," said Beau Webber, Ph.D., Assistant Professor in the Department of Pediatrics, Division of Hematology and Oncology. "We are deeply encouraged by Inceptor Bio’s progress in the cell therapy arena, and we look forward to being part of future developments to help cure difficult-to-treat cancers."

"This partnership is an important step in continuing to execute on our strategy of advancing cell therapies to bring a more positive prognosis and quality of life to patients with difficult-to-treat cancers," said Abe Maingi, Vice President, Business Development at Inceptor Bio. "We are thrilled to be able to develop and deliver on the promise of iPSC-derived cell therapies."

On June 30, 2022 Provectus (OTCQB: PVCT) reported that updated data from the Company’s initial expansion cohort of patients with uveal melanoma metastatic to the liver (mUM) in its cancers-of-the-liver Phase 1 trial of investigational immunocatalyst PV-10 (NCT00986661) were part of two oral presentations at the 20th Congress of the International Society of Ocular Oncology (ISOO), held June 17-21, 2022 in Leiden, The Netherlands (Press release, Provectus Biopharmaceuticals, JUN 30, 2022, View Source [SID1234616443]).

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The first presentation, given by Krysta McVay, Research Nurse, Department of Melanoma Medical Oncology, Division of Cancer Medicine at MD Anderson Cancer Center (MDACC), was entitled "A phase 1 study of percutaneous autolytic rose bengal disodium for metastatic uveal melanoma patients with hepatic metastases." A copy of her presentation is available on Provectus’ website at: View Source

Sapna Patel, MD, Associate Professor, Department of Melanoma Medical Oncology, Division of Cancer Medicine and Director of the Uveal Melanoma Program at MDACC and Chair, Melanoma Committee, SWOG Cancer Research Network, made the second presentation entitled "Metabolic complete responses (mCR) in metastatic uveal melanoma (mUM) patients treated with image-guided injection of PV-10." A copy of her presentation is available on the Company’s website at: View Source

This ongoing single-center mUM study at MDACC has been led since inception by Dr. Patel. Up to three hepatic mUM tumors can be injected per PV-10 treatment cycle. Response assessments are performed at Day 28, and then every three months. Patients with additional, injectable, visceral hepatic mUM disease may receive additional cycles of PV-10 after Day 28. Eligible patients may also receive standard of care immune checkpoint blockade during and after PV-10 treatment.

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 Nanostics Inc., a precision health diagnostics company, reported that positive data from a pre-biopsy clinical validation study of its ClarityDX Prostate test designed to improve the accuracy of detecting clinically significant prostate cancer in men that are at risk of the disease (Press release, Nanostics, JUN 30, 2022, View Source [SID1234616423]).

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The ClarityDX Prostate test uses a proprietary machine-learning algorithm that combines data from two biological biomarkers and three clinical biomarkers to generate a risk score for clinically significant prostate cancer. The ClarityDX Prostate test is intended to be used as a reflex test for men with elevated levels of PSA, the current prostate cancer screening test, and is designed to help physicians and patients make a more informed decision to proceed with biopsy or not.

"We are extremely excited to announce positive results from the clinical validation study of the ClarityDX Prostate test in Alberta", John Lewis, CEO of Nanostics said, "Implementing ClarityDX Prostate as a reflex test for men with elevated PSA levels will improve decision-making for men and their families, while providing substantial savings for healthcare systems and resulting in better health outcomes for men with prostate cancer."

A total of 1,437 men between 40-75 years of age, with elevated PSA levels, no prior prostate cancer diagnosis, and who were referred for prostate biopsy, were recruited for this clinical study. Algorithmic risk models to predict prostate cancer or clinically significant (grade group ≥2) prostate cancer were generated using data from 1036 men recruited at the Kipnes Urology Center in Edmonton, AB, and a site in Baltimore, USA. These models were then tested in a validation cohort of 401 men recruited at the Prostate Cancer Center in Calgary, AB.

The ClarityDX Prostate test provided 94% sensitivity, 37% specificity, 49% positive predictive value, and 90% negative predictive value for predicting clinically significant (grade group ≥2) prostate cancer. The potential impact of the ClarityDX Prostate test is considerable; implementation could eliminate up to 37% of unnecessary biopsies and significantly reduce the number of unnecessary treatments for prostate cancer. The results from the study are being submitted for peer-reviewed publication. Beyond the immediate cost savings to the healthcare system, the launch of the ClarityDX Prostate test will positively impact the overall healthcare experience and quality of life for men with prostate cancer.

The study was conducted in partnership with DynaLIFE Medical Labs and the Alberta Prostate Cancer Research Initiative (APCaRI) at the University of Alberta. Funding for the clinical study comes in part from The Bird Dogs, Motorcycle Ride for Dad, Alberta Cancer Foundation, Alberta Innovates, the University Hospital Foundation Kaye Fund, and the Frank and Carla Sojonky Chair in Prostate Cancer Research funded by the Alberta Cancer Foundation, held by Dr. John Lewis from 2012 to 2022.