On May 11, 2021 Quanterix Corporation (NASDAQ: QTRX), a company helping to transform healthcare with digital protein biomarker technology enabling precision health, reported it has named Masoud Toloue to the position of President of Quanterix and Diagnostics, effective June 9, 2021 (Press release, Quanterix, MAY 11, 2021, View Source [SID1234579656]).

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"We are excited to welcome Masoud to our team at such an important moment for Quanterix," said Kevin Hrusovsky, Chairman and Chief Executive Officer Quanterix. "We are making important strides in advancing our diagnostic capability and assays, given the NIH RADx funding, our COVID-19 Emergency Use Authorizations and advances in our neurology suite of assays for Multiple Sclerosis, Alzheimer’s and Parkinson’s research as well as phase III drug trials. Masoud’s strong growth track record of doubling PerkinElmer’s diagnostics franchise, wealth of experience, deep roots in the life sciences and business acumen will be instrumental as we continue implementing our value creation strategy of entering phase III drug trials with leading protein biomarkers, such as Neurofilament Light (Nf-L), Phosphorylated Tau, Amyloid Beta 40 / 42, GFAP and others, that we believe will ultimately have productive diagnostic and health screen utility in the fields of infectious disease, neurology and immunology."

Toloue brings a wealth of industry experience and high growth track record to Quanterix. He joins Quanterix from PerkinElmer, where he most recently served as senior vice president, diagnostics. He previously led that company’s applied genomics business. Prior to this role, he founded and led Bioo Scientific’s next generation sequencing business which was acquired by PerkinElmer in 2016. He also co-founded and led Genohub, where he transformed that company from a supplier of next generation sequencing matching technology to a leading platform provider for manging sequencing projects globally. He holds a doctoral degree in molecular cell biology from the University at Buffalo and was a postdoctoral fellow in biochemistry at The University of Texas Health Science Center at San Antonio.

In his new role, Toloue will oversee Quanterix’ growing diagnostics (Dx) business and assume responsibility for the Company’s Accelerator Lab Services, strategic partnerships and corporate development.

"Quanterix is empowering a truly innovative approach to highly-sensitive diagnostics that has incredible potential to change the way healthcare in administered," said Toloue. "I am eager to roll up my sleeves and join this exceptional leadership team to support the company’s continued diagnostics expansion."

On May 11, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported program updates and financial results for the first quarter ended March 31, 2021 (Press release, Poseida Therapeutics, MAY 11, 2021, View Source [SID1234579671]).

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"We are encouraged by our continued progress in first quarter of 2021, building on the accomplishments from the prior year as we moved multiple programs forward to important inflection points, including our ongoing P-BCMA-101 and P-PSMA-101 CAR-T programs, on which we plan to provide further clinical updates later in the year," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida. "Additionally, we intend to advance two INDs in our allogeneic CAR-T portfolio this year, one for P-BCMA-ALLO1, targeting BCMA in relapsed/refractory multiple myeloma; and the other for P-MUC1C-ALLO1, targeting MUC1-C in a variety of solid tumor indications, including triple negative breast cancer and ovarian cancer. Though still early, initial clinical activity in patients with prostate cancer following treatment with P-PSMA-101 increases our excitement and confidence in the probability of success for this pan-solid tumor program."

Program Updates

BCMA Program

P-BCMA-101 is an autologous CAR-T product candidate in an ongoing Phase 1 dose expansion trial and Phase 2 trial in development for the treatment of relapsed/refractory multiple myeloma. Phase 1 dose expansion enrollment continues, with an expected update on this program later in 2021.

P-BCMA-ALLO1, the Company’s first allogeneic CAR-T product candidate, is in development for the treatment of relapsed/refractory multiple myeloma and is designed to be fully allogeneic, with genetic edits intended to reduce or eliminate both host-vs-graft and graft-vs-host alloreactivity. The program is proceeding toward an IND filing. Due to a suspected equipment failure at the contract manufacturer for P-BCMA-ALLO1, the IND is now expected in the third quarter of 2021.

PSMA Program

P-PSMA-101 is a solid tumor autologous CAR-T product candidate being developed to treat patients with metastatic castrate resistant prostate cancer (mCRPC) currently in an ongoing Phase 1 dose escalation trial. Following a previously disclosed serious adverse event in cohort 1 (0.75X10E6 cells/kg), the Company elected to de-escalate dosing and treat at least three patients at the protocol preestablished cohort -1 (0.25X10E6 cells/kg) dose. All three patients have been treated, with significant improvements in activity measures seen in the first two patients, one with a >50% decline in prostate-specific antigen (PSA) at about two weeks post CAR-T treatment; and the second with >96% decline in PSA and a 70% reduction in standard uptake value in PSMA PET imaging of target lesions at four weeks post CAR-T treatment. The third patient was only recently treated. One patient demonstrated Grade 1 cytokine release syndrome, or CRS. Assuming no dose limiting toxicities are observed, re-escalation to the cohort 1 dose and beyond is expected. The Company intends to provide an additional update on this program in the second half of 2021.

MUC1-C Program

P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in preclinical development with the potential to treat a wide range of solid tumors, including breast and ovarian cancers. P-MUC1C-ALLO1 is proceeding as planned, with an anticipated IND filing and initiation of Phase 1 clinical trial by the end of 2021.

Liver-Directed Gene Therapy Programs

P-OTC-101 is the Company’s first liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, a condition characterized by high unmet medical need. The Company expects an IND submission and initiation of a Phase 1 clinical trial in 2022.

Early-Stage Development Programs

For programs in early development, including P-FVIII-101, a liver-directed gene therapy for the in vivo treatment of hemophilia A, preclinical studies are ongoing that will inform the development plans and timelines to IND filings.

For discovery programs, the Company may seek partnerships or collaborations to advance development in the near term.

Other Operational Updates and Upcoming Events

Launch of Immuno-Oncology Scientific Advisory Board (SAB)

In March, the Company announced the appointment of distinguished scientist and pioneer in the field of CAR-T therapy, Carl June, M.D., to chair the newly expanded immuno-oncology scientific advisory board, which will provide advice and counsel on the research and development efforts that drive the Company’s innovative cell and gene therapies.

Dr. June has since been joined by Luca Gattinoni, M.D., Chair for Functional Immune Cell Modulation at the Regensburg Center for Interventional Immunology at the University of Regensburg in Germany; Christine Brown, Ph.D., Heritage Provider Network Professor of Immunotherapy at City of Hope Comprehensive Cancer Center; and J. Joseph Melenhorst, Ph.D., Adjunct Associate Professor of Pathology & Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania.

The Company is also in the process of establishing a second SAB focused on additional applications for its proprietary gene therapy platform technologies.

American Society of Gene and Cell Therapy 2021 Virtual Annual Meeting

The Company gave multiple oral and poster presentations earlier today, May 11, 2021, at the American Society of Gene and Cell Therapy 2021 Virtual Annual Meeting. The Company’s oral presentation highlighted new data demonstrating the potential of its proprietary piggyBac DNA Delivery System for the treatment of genetic liver disorders in children and infants. Two additional presentations highlighted preclinical data supporting Poseida’s first allogeneic CAR-T product candidate, P-BCMA-ALLO1 for R/R multiple myeloma, as well as preclinical data supporting the Company’s anti-c-kit CAR-T program as a potentially safer preconditioning regimen for hematopoietic stem cell transplantation in patients with AML. The presentation materials can be accessed on the Poseida website.

BofA Securities 2021 Virtual Health Care Conference

The Company is participating in a virtual conference at 2:45pm ET/11:45am PT on Wednesday, May 12, 2021. The audio recording of the presentation, formatted as a fireside chat, will be made available on the Company’s website.

BofA Securities 2021 Napa Biopharma Virtual Conference

The Company is participating in a virtual conference at 4:30pm ET/1:30pm PT on Monday, June 14, 2021. The audio recording of the presentation, formatted as a fireside chat, will be made available on the Company’s website.

Financial Results for the First Quarter 2021

Research and Development Expenses

Research and development expenses were $29.1 million for the first quarter ended March 31, 2021, compared to $23.4 million for the same period in 2020. The increase was primarily due to increased headcount, external costs related to our preclinical programs and clinical stage programs, including the ongoing enrollment and manufacturing associated with our P-BCMA-101 and P-PSMA-101 clinical trials, and internal costs related to facilities development.

General and Administrative Expenses

General and administrative expenses were $8.4 million for the first quarter ended March 31, 2021, compared to $4.9 million for the same period in 2020. The increase was primarily due to increased headcount and professional fees associated with becoming a publicly traded company.

Net Loss

Net loss was $38.3 million for the first quarter ended March 31, 2021 compared to $28.8 million for the first quarter ended March 31, 2020.

Cash Position

As of March 31, 2021, cash, cash equivalents and short-term investments were $270.0 million.

On May 11, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported positive new in vivo data showing that PH-762 significantly enhanced the antitumor efficacy of HER2-targeted CAR-T cells (HER2CART) in solid tumors (Press release, Phio Pharmaceuticals, MAY 11, 2021, View Source [SID1234579687]). Compared to untreated HER2CART cells, HER2CART cells treated with PH-762 showed a statistically significant and durable inhibition of tumor growth. These data, using a HER2-targeted CAR-T cell product against a HER2-expressing ovarian cancer xenograft model, provide proof-of-concept for the application of PD-1 checkpoint silencing with INTASYL in CAR-T cells prior to adoptive cell therapy to enhance the therapeutic efficacy of CAR-T cell therapy in solid tumors.

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"The in vivo data announced today further support advancing the development of PH-762 into the clinic as a viable approach to improve various forms of T cell based immunotherapy. These data are especially impressive considering that to date CAR T-cell therapy effectiveness in solid tumors has been disappointing and the use of additional genetic engineering to address these issues has proven challenging and costly. Indeed, in a recent clinical study it was shown that CRISPR-Cas9 mediated PD-1 disruption resulted in low efficiency, namely an editing efficiency of less than 6% and a median disruption of PD-1 expression of less than 50% in the edited T cells. This compares sharply with our results where we show that our INTASYL compound, PH-762, achieves PD-1 silencing efficiency of ~90% in nearly 100% of the HER2CART cells used in this study," stated Dr. Simon Fricker, Phio’s VP of Research. "In addition, these results are achieved by merely adding PH-762 to the HER2CART cell culture media, without the need for cell delivery vehicles or vectors, and without negative impact on cell growth/survival. In a prior presentation we also showed how PH-762 can improve the tumor cell killing activity of another adoptive cell therapy platform, namely tumor infiltrating lymphocytes. Taken together, you can start to see the full picture of the potential advancement that INTASYL could provide in adoptive cell therapy."

In this study the Company assessed the potential of PH-762, a PD-1 targeting INTASYL compound, to enhance the therapeutic efficacy of HER2CART cells in the treatment of a subcutaneous HER2-expressing SKOV3 model of human ovarian cancer in mice. On-target silencing of PD-1 in vitro was demonstrated in a dose associated manner in activated HER2CART cells, without significant impact on viability, and resulted in an enrichment of CD8+ and CD25+ cells. Analyses of PH-762-treated HER2CART cells isolated from tumors suggest that PH-762 enhances CAR-T function through multiple mechanisms including enhanced efficiency, degranulation, decreased suppressive potential, and promotion of memory/stem populations.

These data were presented today during the 24th Annual Meeting of the ASGCT (Free ASGCT Whitepaper) in a poster titled "INTASYL PH-762 Self-Delivering RNAi Targeting PD-1 Enhances the Therapeutic Efficacy of Systemically Administered HER2-Targeted CAR-T Cells in a SKOV3 Model of Human Ovarian Adenocarcinoma in NCG Mice". An archived version of the poster presentation will be made available on the "Investors – Events and Presentations" section of the Company’s website (click here).

On March 11, 2021 Catamaran Bio, Inc., a biotechnology company developing allogeneic CAR-NK cell therapies to treat solid tumors, reported that an oral presentation with results generated using an advanced transposon technology for non-viral engineering of CAR-NK cells will be highlighted at the virtual annual meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), based on work from the laboratory of the company’s scientific founder, Branden Moriarity, PhD (Press release, Catamaran Bio, MAY 11, 2021, View Source [SID1234579703]). This is the first presentation of data for this novel transposon technology in NK cells, demonstrating high efficiency transposon integration in primary NK cells, subsequent cell expansion, and in vitro activity of the resulting CAR-NK cells. Dr. Moriarity, an Assistant Professor at the Medical School at the University of Minnesota in the Division of Pediatric Hematology/Oncology, is a pioneer in the fields of genome engineering and non-viral cell engineering and a scientific co-founder of Catamaran. In addition to an ongoing research collaboration with Dr. Moriarity, Catamaran also received support through an early investment from the University of Minnesota Discovery Capital investment program.

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"We congratulate our collaborators at the University of Minnesota for this landmark presentation, which provides the first demonstration that this transposon technology efficiently generates active CAR-NK cells and offers potential advantages compared to viral vectors in the manufacture of CAR-NK cells," said Vipin Suri, PhD, Chief Scientific Officer of Catamaran Bio. "This novel transposon technology demonstrates important capabilities for improved manufacturability, which will be essential in developing safe and effective cell therapies for solid tumors. We look forward to working with Dr. Moriarity as we develop and advance our CAR-NK programs."

The oral presentation, entitled "Non-Viral Engineering of CAR-NK Cells Using the TC Buster Transposon System," will occur on Thursday, May 13, 2021, at 6:30 p.m. ET during the Advances in Cellular and Immunotherapies session of the ASGCT (Free ASGCT Whitepaper) virtual meeting. The data presented highlights the following research findings:

Approximately 50% transposon integration efficiency using the transposon technology to deliver a multi‑cistronic chimeric antigen receptor (CAR) cassette into primary human peripheral blood NK cells.
Resulting cells underwent greater than 800-fold expansion and demonstrated efficacy in an in vitro cell-killing assay.
In contrast to the viral gene delivery methods commonly used in cell therapy manufacturing today, DNA transposon technology has the capacity to efficiently deliver large multi‑cistronic DNA cargoes and drive multiplex genome engineering via a single electroporation step. Transposons have the potential for more convenient and cost-effective manufacture of cell therapy products.

"We are pleased to present these data showing the effective transposon integration in primary NK cells with our transposon technology, the rapid expansion of the resulting CAR-NK cells and their activity in an in vitro model," said Dr. Moriarity. "I look forward to continuing to work closely with the team at Catamaran to utilize this technology in conjunction with their CAR-NK cell therapy platform as they advance new cell therapy approaches to treat solid tumors."

About the TAILWIND Platform

Catamaran’s TAILWIND Platform integrates proprietary capabilities to create novel, allogeneic CAR‑NK cell therapies by harnessing the natural cancer-fighting properties of natural killer (NK) cells and enhancing them with the power of synthetic biology and innovative NK cell engineering and manufacturing. With the TAILWIND Platform, CAR-NK cells are programmed with NK cell-specific CAR architectures and potency-boosting switches to neutralize the hostile tumor microenvironment and enable efficacy against diverse cancer types, especially solid tumors. Additionally, the TAILWIND Platform includes proprietary, non-viral NK cell engineering technology for efficient modification of NK cells with customized genetic programs enabled by synthetic biology. Catamaran’s CAR-NK cell therapies use healthy donor cells that are engineered and manufactured for off‑the‑shelf use, unlike current CAR-T cell therapies that use a patient’s own genetically modified T cells and require a customized, multi-week manufacturing process.

On May 11, 2021 NANOBIOTIX (Euronext : NANO – NASDAQ: NBTX), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported a partnership with LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and major Asian markets, to develop and commercialize Nanobiotix lead product candidate NBTXR3, a potential first-in-class radioenhancer, in Greater China (mainland China, Hong Kong, Taiwan, and Macau), South Korea, Singapore and Thailand. (Press release, Nanobiotix, MAY 11, 2021, View Source [SID1234579720])

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Shanghai and Princeton-based LianBio was founded by Perceptive Advisors with an innovative partnership model to develop and commercialize therapeutics in China and other Asian markets. LianBio’s cross-border development and commercialization expertise includes strong capabilities in oncology and NBTXR3 is the third investigational cancer treatment in the company’s portfolio. Given the global high unmet need in locally advanced and metastatic cancers, along with data supporting the potential of NBTXR3 across solid and metastatic tumor types, Nanobiotix continues to leverage strategic relationships with world class collaborators to expand and accelerate its pipeline while prioritizing Company resources to support development in head and neck cancer and immunotherapy.

"Discovery of practice-changing therapeutics for major diseases through a physics-based approach, with the support of people committed to making a difference for humanity, is our mission at Nanobiotix. We have long believed that NBTXR3 will significantly improve treatment outcomes for patients with any solid or metastatic tumor, and we are grateful that a partner with the talent and capability of LianBio has agreed to walk with us in our journey. Cancer is a disease that knows no borders and bringing our innovation to patients in Greater China with speed and determination is an absolute necessity," said Nanobiotix CEO and co-founder Laurent Levy.

"We purpose-built LianBio with a next-generation licensing model that enables us to meaningfully contribute to our partners’ global development initiatives in order to accelerate the availability of transformative therapeutics for patients throughout Asia," said Konstantin Poukalov, Executive Chairman of LianBio and Managing Director of Perceptive Advisors. "We believe NBTXR3 has a highly targeted nature and broadly applicable mechanism of action and thus has the potential to change radiotherapy and immuno-oncology treatment paradigms by addressing key limitations of current standards of care."

"The NBTXR3 therapeutic approach offers a promising avenue to address the significant burden of disease across multiple tumor types," said Debra Yu, M.D., President and Chief Business Officer, LianBio. "Our partnership will provide Nanobiotix with access to LianBio’s regional expertise and is designed to enable Nanobiotix to reach the growing number of cancer patients in China and other Asian markets who are in need of improved treatment options."

Under the terms of the agreement, LianBio will obtain exclusive rights to develop and commercialize NBTXR3 in Greater China, South Korea, Singapore, and Thailand. Nanobiotix will receive a $20 million upfront payment and is entitled to receive up to an aggregate of $220 million in potential contingent, development and commercialization milestone payments. Nanobiotix will also be eligible to receive tiered, low double-digit royalties based on net sales of NBTXR3 in the licensed territories. LianBio will participate in the Nanobiotix global phase III registrational study evaluating NBTXR3 for patients with locally advanced head and neck squamous cell carcinoma (HNSCC; head and neck cancer) by enrolling 100 patients in China in the study. In addition to the phase III head and neck cancer study, LianBio has committed to enroll patients in four additional registrational studies conducted by Nanobiotix across indications and therapeutic combinations potentially including immunotherapy. LianBio will fund all development and commercialization expenses in the collaboration territory, and Nanobiotix will continue to fund all development and commercialization expenses in all other geographies.

About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

NBTXR3 is being evaluated primarily in locally advanced head and neck squamous cell carcinoma (HNSCC). The company-sponsored phase I dose escalation and dose expansion study has produced consistently favorable safety data and early signs of efficacy; and a phase III global registrational study is planned to launch in 2021. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the planned phase III study.

Nanobiotix has also prioritized an Immuno-Oncology development program—beginning with a Company-sponsored phase I clinical study evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/metastatic HNSCC and lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a strategic collaboration strategy with world class partners to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 The University of Texas MD Anderson Cancer Center entered into a broad, comprehensive clinical research collaboration with Nanobiotix to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations.