On March 29, 2023 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported that management will participate in a fireside chat at the H.C. Wainwright Autoimmune & Inflammatory Disease Virtual Conference on Thursday, March 30th at 11:30 am ET (Press release, Celldex Therapeutics, MAR 29, 2023, View Source [SID1234629507]). A webcast of the presentation will be available on the "Events & Presentations(opens in a new tab)" page of the "Investors & Media(opens in a new tab)" section of the Celldex website. A replay will be available for 90 days following the event.

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On March 29, 2023 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that the first patient has been dosed in the Phase 1/1b dose escalation/dose expansion study (clinical trial identifier: NCT05694364) of PRGN-3007 in advanced ROR1-positive (ROR1+) hematological and solid tumors (Press release, Precigen, MAR 29, 2023, View Source [SID1234629506]). The target patient population for the study includes chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL) and solid tumors, including breast adenocarcinomas encompassing triple negative breast cancer (TNBC). There are estimated to be more than 100,000 patients diagnosed in both the hematological and TNBC target populations in the United States, European Union and Japan in 2023.a

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PRGN-3007 UltraCAR-T is a first-in-class investigational multigenic, autologous CAR-T cell therapy utilizing Precigen’s clinically validated advanced non-viral gene delivery system and well-established overnight, decentralized manufacturing process. Precigen has further advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment by incorporating intrinsic checkpoint blockade without the need for complex and costly gene editing techniques. PRGN-3007 is engineered using a single multicistronic transposon plasmid to simultaneously express a chimeric antigen receptor (CAR) targeting ROR1, membrane-bound interleukin–15 (mbIL15), a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression. The innovative design of PRGN-3007, where the blockade of PD-1 expression is intrinsic and localized to UltraCAR-T cells, is aimed at avoiding systemic toxicity and the high cost of checkpoint inhibitors by eliminating the need for combination treatment.

The Phase 1/1b clinical trial is an open-label study designed to evaluate the safety and efficacy of PRGN-3007 in patients with advanced ROR1+ hematological (Arm 1) and solid (Arm 2) tumors. The study is enrolling in two parts: an initial 3+3 dose escalation in each arm followed by a dose expansion at the maximum tolerated dose (MTD). Arm 1 and Arm 2 are enrolling in parallel. The investigator-initiated study is being conducted in collaboration with the H. Lee Moffitt Cancer Center & Research Institute (Moffitt).

"We are excited to work with Precigen and announce that the first patient, a CLL patient, has been dosed in the first-in-human study of PRGN-3007 UltraCAR-T," said Javier Pinilla-Ibarz, MD, PhD, Senior Member, Lymphoma Section Head and Director of Immunotherapy, Malignant Hematology Department, Moffitt, and Principal Investigator for the PRGN-3007 clinical study. "ROR1 is a promising target for addressing a wide variety of tumors and we are hopeful that the PRGN-3007 study will further the development of this novel CAR-T treatment, which combines intrinsic PD-1 inhibition and ease of administration from the validated overnight manufacturing of UltraCAR-T performed at our medical center bringing therapy to patients within one day."

"Dosing the first patient with PRGN-3007, the next generation of UltraCAR-T incorporating PD-1 inhibition, is a significant milestone for the UltraCAR-T platform," said Helen Sabzevari, PhD, President and CEO of Precigen. "The PRGN-3007 study targets a broad range of hematological and solid tumor indications and this milestone helps us move closer to our vision for UltraCAR-T, which aims to deliver a library of personalized autologous UltraCAR-T therapies using overnight manufacturing at the patient’s medical center."

Precigen: Advancing Medicine with Precision
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen, LinkedIn or YouTube.

About Receptor Tyrosine Kinase-like Orphan Receptor 1 (ROR1)
ROR1 is a type I orphan-receptor that is expressed during embryogenesis and by certain hematological and solid tumors but is undetectable on normal adult tissues.1-3 ROR1 plays an important role in oncogenesis by activating cell survival signaling events, particularly the non-canonical WNT signaling pathway.4 Aberrant expression of ROR1 is detected in multiple hematological malignancies including CLL5, MCL6, ALL7, and DLBCL.8 Elevated ROR1 expression is detected in various solid tumors, including breast adenocarcinoma encompassing TNBC, pancreatic cancer, ovarian cancer, Ewing’s sarcoma and lung adenocarcinoma.9-14 Many human breast adenocarcinomas express high levels of ROR1, which is not expressed by normal breast tissue.15

UltraCAR-T
UltraCAR-T is a multigenic autologous CAR-T platform that utilizes Precigen’s advanced non-viral Sleeping Beauty system to simultaneously express an antigen-specific CAR to specifically target tumor cells, mbIL15 for enhanced in vivo expansion and persistence, and a kill switch to conditionally eliminate CAR-T cells for a potentially improved safety profile. Precigen has advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment by incorporating a novel mechanism for intrinsic checkpoint blockade without the need for complex and expensive gene editing techniques. UltraCAR-T investigational therapies are manufactured via Precigen’s overnight manufacturing process using the proprietary UltraPorator electroporation system at the medical center and administered to patients only one day following gene transfer. The overnight UltraCAR-T manufacturing process does not use viral vectors and does not require ex vivo activation and expansion of T cells, potentially addressing major limitations of current T cell therapies.

UltraPorator
The UltraPorator system is an exclusive device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T therapies and potentially represents a major advancement over current electroporation devices by significantly reducing the processing time and contamination risk. The UltraPorator device is a high-throughput, semi-closed electroporation system for modifying T cells using Precigen’s proprietary non-viral gene transfer technology. UltraPorator is being utilized for clinical manufacturing of Precigen’s investigational UltraCAR-T therapies in compliance with current good manufacturing practices.

Trademarks
Precigen, UltraCAR-T, UltraPorator, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

On March 29, 2023 Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, reported an upcoming presentation highlighting WU-NK-101, the company’s lead memory natural killer (NK) cell therapy product (Press release, Wugen, MAR 29, 2023, View Source [SID1234629505]). The data support the potential of WU-NK-101 to overcome resistance to immune checkpoint blockade (ICB) therapy and will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 taking place April 14 – 19, 2023 in Orlando, Florida.

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The details of Wugen’s presentation at AACR (Free AACR Whitepaper) are as follows:

Title: WU-NK-101 as Salvage Therapy Post Immune Checkpoint Blockade (ICB)

Session: Immunotherapy Strategies and Mechanisms

Session Date and Time: Wednesday, April 19, 2023, 9:00 a.m. – 12:30 p.m. ET

Location: Section 25

Poster Number: 5

Abstract Number: 6418

Presenter: Tom Leedom

Additional meeting information can be found at View Source

About WU-NK-101

WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This rare cell population has a superior phenotype, proliferation capacity, and metabolic fitness that makes it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML) and solid tumors.

On March 29, 2023 Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, reported financial results for the fourth quarter and full year ending December 31, 2022 and provided a business update (Press release, Synlogic, MAR 29, 2023, View Source [SID1234629504]).

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"Last year was a tremendous year for Synlogic and our Synthetic Biotic platform given our three positive clinical readouts in three different diseases in the fourth quarter alone," said Aoife Brennan, M.B. Ch.B., Synlogic President and Chief Executive Officer. "We are on track to initiate Synpheny-3, the pivotal Phase 3 study for SYNB1934 in PKU, our lead program, and we were thrilled to present three data presentations – including Phase 2 PKU data as a platform presentation – at the SIMD Annual Meeting."

Fourth Quarter 2022 and Recent Highlights

Received positive opinion on orphan designation from the European Medicines Agency (EMA) for SYNB1934 for PKU
Presentation of data from both the Phase 2 Synpheny-1 study in PKU and the Phase 1 study with SYNB1353 for HCU at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting
Received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for both:
SYNB1934 for PKU
SYNB1353 for HCU
Announced positive top-line Phase 2 data for PKU; advancing SYNB1934 to Phase 3
Demonstrated proof of mechanism for SYNB1353 for HCU, based on a top-line data from a Phase 1 study using a methionine meal challenge in healthy volunteers
Proof of concept achieved with Phase 1b top-line data for SYNB8802 for enteric hyperoxaluria
Anticipated Upcoming Milestones

Initiation of Phase 3 clinical trial of SYNB1934 for PKU in the first half of 2023
Advancing SYNB1353 to Phase 2 study in patients with HCU
Presentation of data from Phase 1b studies of SYNB8802, in development for enteric hyperoxaluria
Progression of preclinical pipeline programs, including partnerships
Corporate Updates

In January 2023, the company announced the appointment of Dr. Dave Hava as Head of Research and Development. Through the integration of the company’s research and development teams, Dr. Hava oversees the advancement of clinical stage programs in addition to progressing our collaboration with Roche and select preclinical programs. Dr. Hava joined Synlogic in 2020, as Chief Scientific Officer, and continues to assume this position within the company.

Fourth Quarter 2022 Financial Results

As of December 31, 2022, Synlogic had cash, cash equivalents, and short-term marketable securities of $77.6 million.

Revenue was $0.1 million for the three months ended December 31, 2022, compared to $0.6 million for the corresponding period in 2021. Revenue in both periods was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic medicine for the treatment of IBD.

Research and development expenses were $13.6 million for the three months ended December 31, 2022, compared to $11.9 million for the corresponding period in 2021.

General and administrative expenses for the three months ended December 31, 2022 were $3.8 million compared to $3.9 million for the corresponding period in 2021.

For the three months ended December 31, 2022, Synlogic reported a consolidated net loss of $16.7 million, or $0.24 per share, compared to a consolidated net loss of $15.1 million, or $0.21 per share, for the corresponding period in 2021.

Full Year 2022 Financial Results

Revenues were $1.2 million for the year ended December 31, 2022, compared to $1.8 million for the same period in 2021. Revenue in both periods was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic medicine for the treatment of IBD. Operating expenses were $68.6 million for the year ended December 31, 2022, compared to $62.5 million for the same period in 2021. For the year ended December 31, 2022, consolidated net loss was $66.1 million, or $0.92 per share, compared to a consolidated net loss of $60.6 million, or $1.09 per share, for the year ended December 31, 2021.

Financial Outlook

Based upon its current operating plan and balance sheet as of December 31, 2022, Synlogic expects to have sufficient cash to be able to fund operations into the second half of 2024.

On March 29, 2023 CVS Health (NYSE: CVS) reported that it has completed its acquisition of Signify Health (Press release, Signify Health, MAR 29, 2023, View Source [SID1234629503]).

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Signify Health is a leading technology and services company, focused on provider enablement and bringing clinicians into the home to identify chronic conditions, close gaps in care, and address social determinants of health. Signify has a network of more than 10,000 clinicians in all 50 states, who spend an average of 2.5 times longer with a member during home visits than an average visit with a primary care provider.

"This transaction advances our value-based care strategy by enhancing our presence in the home," said CVS Health President and CEO Karen S. Lynch. "Our expanded capabilities will bring us closer to the consumer as we continue to redefine how people access and experience care that is more affordable, convenient and connected."

The combined company will work to improve care delivery capabilities, lower costs, improve levels of engagement and enable broad access to high-quality care, especially for Medicare Advantage customers. Signify clinicians can have an even greater impact by engaging with CVS Health’s unique collection of assets and connecting consumers to care how and when they need it.

"Our mission has always been to build trusted relationships to make people healthier," said Kyle Armbrester, who will continue to lead Signify Health. "As part of CVS Health, we will be uniquely positioned as a payor-agnostic business to support a more proactive, preventive and holistic approach to patient care.

"We’re excited to welcome the Signify team to CVS Health," said Lynch. "I’m confident our shared values and complementary capabilities will work together to better address patient needs."

Under the terms of the transaction, each outstanding share of Signify Health common stock is being exchanged for $30.50 in cash, representing a total transaction value of approximately $8 billion. CVS Health funded the transaction price with existing cash and available resources and is committed to maintaining its current credit ratings.