On May 29, 2024 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, reported that the Company will present at the 2024 Jefferies Global Healthcare Conference on Wednesday, June 5, 2024 at 3:00 PM ET in New York, New York (Press release, Fate Therapeutics, MAY 29, 2024, View Source [SID1234643802]).

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A live webcast, if recorded, of each presentation can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website shortly after the event.

On May 29, 2024 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported new interim tumor response data from the ongoing randomized, controlled Phase 2b study of VBI-1901, the Company’s immunotherapeutic cancer vaccine candidate, in recurrent glioblastoma (rGBM) patients (Press release, VBI Vaccines, MAY 29, 2024, View Source [SID1234643801]). These data will be presented in a poster session at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Saturday, June 1, 2024.

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David E. Anderson, Ph.D., VBI’s Chief Scientific Officer, said: "The tumor responses we have seen to date are incredibly encouraging, and, like the tumor responses did in the Phase 1/2a study, I am excited to see how this translates to clinical and survival outcomes later this year. The approved treatments for recurrent GBM patients have limited to no efficacy, which is consistent with the data seen in the standard-of-care arm in this study. VBI-1901’s ability to stimulate an immune response capable of generating a disease control rate of 43% at this interim stage of the study, including a partial response, is hopefully an indication of the potential of this candidate to make a meaningful difference in the lives of patients, providers, and families."

Jeff Baxter, VBI’s President and CEO, said: "These data represent a considerable advancement in our effort to make a difference in the fight against GBM. Throughout the remainder of 2024, we expect to have additional tumor response data and initial survival data. Pending the strength of these clinical data, we believe we can start discussions with the FDA around what an accelerated development and approval pathway looks like, under our Fast Track and Orphan Drug Designations."

Phase 2b Data Poster Highlights

As of May 15, 2024, 23 patients had been randomized 1:1 to either the active treatment arm, VBI-1901, or to the control treatment arm (standard-of-care).

Active Study Arm: VBI-1901 + Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)
11 patients have been randomized – tumor response data was available for 7 of those patients as of May 15, 2024
One (1) PR and two (2) SD observations seen to date
The patient with the PR saw a 67% tumor reduction compared to baseline at week 6, after receiving two monthly doses of VBI-1901
43% (n=3/7) disease control rate achieved in evaluable patients to date
2 additional patients appear to be experiencing stabilization of tumor growth after their second dose of VBI-1901, but do not yet meet the threshold to qualify as stable disease
Control Study Arm: Standard-of-Care (SoC) Therapy – Carmustine or Lomustine
12 patients have been randomized – tumor response data was available for 6 of those patients as of May 15, 2024
No tumor responses have been observed in the SoC arm (n=0/6; 0% DCR)
5 evaluable patients have experienced tumor progression of 2-8x increase in tumor size and have been taken off study protocol
Tumor response rates are an objective measure of treatment efficacy in oncology trials. In VBI’s previous Phase 1/2a study of VBI-1901 in rGBM, a 44% disease control rate was observed (n=7/16), which translated to clinical improvements in overall survival, with a median overall survival (mOS) of 12.9 months after treatment with VBI-1901 compared to the 8-month mOS historical benchmark for standard-of-care chemotherapy treatment.

ASCO Poster Details

Title: Randomized Phase 2b trial of a CMV vaccine immunotherapeutic candidate (VBI-1901) in recurrent glioblastomas
Abstract Number: TPS2100
Date & Time: 9:00 AM – 12:00 PM CDT, Saturday, June 1, 2024
Poster Session: Central Nervous System Tumors
VBI’s poster presentation will be made available on the Posters page of VBI’s website, under News and Resources, after the Central Nervous System Tumors poster session concludes on June 1, 2024.

Phase 2b Patient Enrollment Update

26 patients have been randomized as of May 28, 2024
Patient enrollment rate continues to increase, with six (6) patients randomized in May
VBI expects the study to be fully enrolled (n=60) by year-end 2024, subject to continued pace of enrollment
Phase 2b Study Design

Multi-center, randomized, controlled, open-label study in up to 60 patients with first recurrent GBM

Patients will be randomized in a 1:1 ratio across two study arms:
Intradermal VBI-1901 + GM-CSF: 10 µg dose every 4 weeks until clinical disease progression
Monotherapy standard-of-care: either intravenous carmustine or oral lomustine, every 6 weeks until disease progression or intolerable toxicity
Endpoints include:
Safety and tolerability
Overall survival (OS) – median and overall
Tumor response rate (TRR)
Progression-free survival (PFS)
Immunologic responses
Reduction in corticosteroid use relative to baseline
Change in quality of life compared to baseline
The randomized, controlled Phase 2b study is evaluating overall survival, tumor response rates, and safety and tolerability of VBI-1901 as a monotherapy treatment in rGBM patients. There are no effective, approved treatments available for patients with rGBM, and median overall survival remains low at approximately eight (8) months.

The U.S. Food and Drug Administration (FDA) has considered demonstration of a statistically significant improvement in overall survival relative to a randomized control arm to be clinically significant and has recognized this as criteria to support the approval of new oncology drugs.1

For more information about the Phase 2b study, visit clinicaltrials.gov and reference trial identifier: NCT03382977.

Phase 1/2a Study Data Highlights – VBI-1901 10µg + GM-CSF Study Arms

(n=16)

44% disease control rate achieved (n=7/16) – disease control rate is defined as stable disease (SD) + partial tumor response (PR) + complete tumor response (CR)
2 partial responses (PR) were observed – 1 patient was on treatment for more than 28 months (2.33 years), surviving at least 40 months (3.33 years) as of August 1, 2023, with a maximum tumor reduction of 93% relative to baseline
5 additional patients demonstrated stable disease (SD) for a sustained period of time
All patients with a tumor response (PR or SD) (n=7/16) reached a minimum survival of 12 months
Median overall survival (mOS) was 12.9 months, comparing favorably to 8-month mOS for monotherapy standard-of-care2
About GBM and VBI-1901

Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. The FDA has granted VBI-1901 Fast Track Designation and Orphan Drug Designation for the treatment of recurrent glioblastoma. These designations are intended to provide certain benefits to drug developers, including more frequent meetings with the FDA, and Accelerated Approval and Priority Review, if relevant criteria are met, among other benefits.

References

1. Oncology Center of Excellence, Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics; Guidance for Industry. FDA.gov. December, 2018

2. Taal W, Oosterkamp HM, Walenkamp AME, et al. Single-agent bevacizumab or lomustine versus a combination of bevacizumab plus lomustine in patients with recurrent glioblastoma (BELOB trial): a randomized controlled phase 2 trial. Lancet Oncol. 2014; 15: 943-953

On May 29, 2024 StarkAge Therapeutics, a French biotechnology company, reported a €1.2 million fundraising round conducted with biotech focused business angels and BPI (Press release, StarkAge Therapeutics, MAY 29, 2024, View Source [SID1234643800]). The company, founded in 2018, specializes in the targeting of cellular senescence in the onctext of age-related pathologies, especially cancer.

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Currently, cancers are mainly treated with radiotherapy, chemotherapy and/or targeted therapy. These therapies can lead to the emergence of senescent cells within the tumor and in adjacent normal tissues. Senescent cancer cells are implicated in treatment resistance, metastasis, and numerous side-effects including fibrosis. StarkAge Therapeutics’ mission is to identify specific targets expressed at the plasma membrane of senescent cells to develop targeted therapies and thus improve patients’ response to conventional therapies.

This funding round will enable the company to finalize in vivo testing of its first lead candidate, STX-1, an Antibody-Drug Conjugated (ADC) directed against the transmembrane protein DPP4 (CD26), highly overexpressed by senescent cells. The preliminary in vitro and in vivo results already obtained, particularly in liver and prostate cancer models, are extremely promising and applicable to a wide variety of other types of cancer expressing DPP4.

« We would like to thank all our partners for their support and confidence. Our ambition is to develop a highly innovative therapeutic approach based on the elimination of senescent cells in patients with refractory cancers, to increase their chances of recovery while limiting functional side effects. With this fundraising, we will finalize the in vivo experiments and initiate our second research program, STX-2. We hope to initiate a new round of financing in September 2024 to complete the preclinical studies for STX-1, prepare our entry into the clinic and continue the preclinical development of our STX-2 program, » explains Dr Thierry Mathieu, Founder and CEO of StarkAge Therapeutics.

The company is also focusing on research program, notably through a partnership with Professor Myriam Gorospe at the National Institute of Health (NIH), a world-renowned specialist in the field of cellular senescence. This collaboration will enable us to better understand the involvement of the DPP4 protein in age-related diseases, and to extend the STX-1 program to other therapeutic indications.

StarkAge Therapeutics’ research associated to an increasing number of scientific publications, demonstrate that the accumulation of senescent cells during disease progression is often extremely deleterious for patients. Many age-related diseases, such as pulmonary fibrosis, neurodegenerative and cardiovascular diseases, metabolic dysfunctions, liver fibrosis and cancer, are directly related to senescent cells and their accumulation in the human body. Targeting them opens the way to numerous innovative targeted therapies.

On May 29, 2024 Scorpius Holdings, Inc (NYSE American: SCPX) (‘Scorpius" or "the Company"), an integrated contract development and manufacturing organization ("CDMO"), reported strategic, financial, and operational updates for the first quarter ended March 31, 2024 (Press release, Scorpius BioManufacturing, MAY 29, 2024, View Source [SID1234643799]).

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Jeff Wolf, CEO of Scorpius Holdings, Inc., stated, "We are successfully executing our strategy to enhance revenue and reduce costs as we seek to become cash flow positive in the near future. This quarter is indicative of this goal as we achieved a 359% increase in revenue while reducing operating expenses by 34% over the same period last year. We believe this performance reinforces the growing demand for our services and our prudent financial management. Moreover, we have built a highly scalable business model poised to generate meaningful cash flow as we continue to grow our sales and increase utilization of our state-of-the-art San Antonio campus, which we anticipate will allow us to achieve meaningful operating leverage. We are very confident that the future for Scorpius is brighter than ever, with significant industry-wide capacity shortages, and our growing revenue backlog, which stood at $10.8 million as of March 31, 2024."

2023 Financial Results

For the three months ended March 31, 2024, the Company recognized $3.5 million of revenue from process development. For the three months ended March 31, 2023, the Company recognized $0.7 million of process development revenue and $0.1 million of license revenue. The increase in process development revenue was attributable to the expanded biomanufacturing operations and service offerings of the CDMO.

Cost of revenues were $0.9 million and $0.6 million for the three months ended March 31, 2024, and 2023, respectively, and primarily consisted of the direct cost of labor, overhead and material costs at Scorpius. The increase in cost of revenues was due to the expanded service offerings and completed milestone work on multiple CDMO contracts.

Research and development expenses were $3.9 million for the three months ended March 31, 2024, compared to $6.3 million for the three months ended March 31, 2023.

Selling, general and administrative expenses were $5.0 million and $6.5 million for the three months ended March 31, 2024, and 2023, respectively. The decrease of $1.5 million was primarily due to decreases in marketing expense of $0.5 million, consultant labor of $0.5 million, and stock-based compensation of $0.5 million.

For the three months ended March 31, 2024, the change in fair value of contingent earn-out receivable, related party, was $1.0 million. This change was primarily due to an increase in expected value of the earn-out due to a new contract received by Elusys Therapeutics.

Total non-operating income was $0.7 million for the three months ended March 31, 2024, which primarily consisted of $1.0 million from the sale of an intellectual property license, partially offset by $0.2 million of interest expense on finance leases, and $0.1 million change in fair value of convertible promissory note, related party. Total non-operating income was $0.1 million for the three months ended March 31, 2023, which primarily consisted of $0.2 million of interest income, $0.1 million of unrealized gain on short-term investment balances, partially offset by $0.2 million of interest expense on finance leases and other expense.

Net loss attributable to Scorpius was approximately $4.4 million, or ($0.16) per basic and diluted share, for the three months ended March 31, 2024, compared to approximately $12.8 million, or ($0.49) per basic and diluted share, for the three months ended March 31, 2023.

As of March 31, 2024, the Company had approximately $1.7 million in cash, cash equivalents, and short-term investments. On May 16, 2024, the Company consummated a public offering resulting in aggregate gross proceeds of approximately $6.0 million, before deducting underwriting discounts and other offering expenses.

On May 29, 2024 QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported the launch of its QIAseq Multimodal DNA/RNA Lib Kit (Press release, Qiagen, MAY 29, 2024, View Source [SID1234643798]). The new kit enables seamless preparation of DNA and RNA libraries for next-generation sequencing (NGS), such as whole genome sequencing (WGS) and whole transcriptome sequencing (WTS), as well as downstream target enrichment based on hybrid-capture from a single sample.

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The QIAseq Multimodal DNA/RNA Lib Kit facilitates multiomics, the studies of several omic fields like genomics, transcriptomics and proteomics, aiming to gain a deeper understanding of biological processes and systems – something crucial for studying diseases like cancer. The kit offers a streamlined and rapid workflow to generate WGS and WTS libraries from a single sample by combining chemistry optimized for DNA and RNA simultaneously. Using traditional methods, separate workflows for DNA and RNA sequencing require a large amount of sample material, labor-intensive library preparation procedures, and long turn-around times.

"With the introduction of the QIAseq Multimodal DNA/RNA Lib Kit, we are addressing a direct need in the scientific community for a more efficient way to conduct multiomic studies," said Nitin Sood, Senior Vice President, Head of the Life Sciences Business Area at QIAGEN. "Studying and understanding the complexity of biological systems better will facilitate identifying potential biomarkers for diseases or therapeutic targets for drug development and finally advance precision medicine and healthcare."

Researchers can also use the QIAseq Multimodal DNA/RNA Lib Kit for generating DNA-only or RNA-only libraries. It is the first NGS multimodal kit on the market that is compatible with a wide range of input samples, including blood, Formalin-Fixed Paraffin-Embedded (FFPE) samples, and cell-free DNA (cfDNA). This is particularly relevant in translational research, such as in the study of cancers, where different types of samples may be available. The kit is highly sensitive, enabling detection of both DNA and RNA rare variants. The DNA and RNA libraries generated using the QIAseq Multimodal DNA/RNA Lib Kit are directly compatible with different sequencing platforms such as Illumina instruments and Element Aviti and can be sequenced on other sequencers with an added conversion step (Complete Genomics/MGI, Singular Genomics, and Ultima Genomics).

Additionally, the flexibility of generating DNA only or RNA only libraries, as well as DNA and RNA libraries compatible with further target enrichment by hybrid capture, consolidates an NGS lab’s multiple workflows into a single kit, saving lab resources while enhancing efficiency. For target enrichment, the QIAseq xHYB Human panels can be used for WES (Whole Exome Sequencing), or targeted sequencing with custom designed content. The libraries are also compatible with hybrid capture panels from other providers.

The new kit adds to QIAGEN’s growing portfolio that harnesses the true potential of NGS in biological research by offering fast and efficient solutions that reduce turnaround times and maximize sequencing capacity. With a focus on front-end sample processing and downstream bioinformatics, QIAGEN provides innovative NGS technologies for genomics, transcriptomics, epigenomics, and metagenomics applications.

QIAGEN’s QIAseq kits have processed over four million NGS samples, enabling biomarker research, gene expression studies, viral epidemiology, and disease surveillance. From DNA and RNA sequencing to multimodal sequencing and epigenomics, the comprehensive range of QIAseq kits delivers reliable data on any sequencing instruments.

More information about the QIAseq Multimodal DNA/RNA Lib Kit can be found here: View Source