On May 10, 2022 TriSalus Life Sciences, an immunotherapy company on a mission to extend and improve the lives of patients living with liver and pancreatic tumors, reported the enrollment of the first patient in its Pressure-Enabled Regional Immuno-Oncology (PERIO-02) clinical study (Press release, TriSalus Life Sciences, MAY 10, 2022, View Source [SID1234614068]).

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The trial is evaluating SD-101, an investigational toll-like receptor 9 (TLR9) agonist, in adults with locally advanced, metastatic, or unresectable hepatocellular carcinoma (HCC) and intrahepatic cholangiocarcinoma (ICC). SD-101 will be administered using the Pressure-Enabled Drug Delivery (PEDD) method in combination with systemic checkpoint inhibitors.

Initiated at The University of Texas MD Anderson Cancer Center, with additional sites anticipated, the study is the second in a series of clinical trials assessing TriSalus’ immunotherapy platform across multiple indications. The platform integrates an immunotherapeutic and proprietary drug delivery technology to address the unique challenges facing patients with tumors in the liver. The PERIO-02 clinical trial will first evaluate the safety of SD-101 delivered by PEDD in varying doses and with checkpoint inhibitors in patients with HCC or ICC. Efficacy will be evaluated based on the tumor response.

The initial trial using this platform, the PERIO-01 study, is actively enrolling and is evaluating the safety of SD-101 administered by PEDD in combination with checkpoint inhibitors in patients with uveal melanoma with liver metastases. The PERIO-01 study has provided initial safety validation for the platform, and early data indicate the ability of SD-101 to favorably reprogram the tumor microenvironment of liver tumors.

"Patients with advanced HCC or ICC often have limited options when seeking treatment, as checkpoint inhibitors have had some success in these indications but results are not what we want them to be in most cases," said Steven C. Katz, MD, FACS, chief medical officer at TriSalus. "The PERIO-02 clinical trial has potential to advance the scientific foundation required to help address this unmet need, deliver new therapies to improve clinical outcomes, and ultimately, give patients a better chance to respond more reliably to different forms of immunotherapy."

While immunotherapy has yielded significant advances in cancer treatment, unique properties of liver tumors, including immune response suppression and high intratumoral pressure, can prevent optimal delivery and performance of therapeutics and limit the overall effectiveness of immunotherapy for patients with liver cancers.|[1]-4|

TriSalus’ platform is designed to address these treatment challenges. The platform consists of an investigational immunotherapy, SD-101, that aims to reactivate the immune system within the liver, and a proprietary drug delivery method, PEDD, that modulates pressure and flow within blood vessels to improve therapy uptake and tumor response.

"With the PERIO-02 trial, we’re striving to enable immunotherapy for the most common primary liver tumors," said Dr. Katz. "The study is implementing a multifaceted approach by testing the integration of an immunotherapeutic, SD-101, with an FDA-cleared delivery device, to hopefully induce the type of immune response that we’re so eager to see for patients with HCC and ICC."

As the second of three Pressure-Enabled Regional Immuno-Oncology studies planned, PERIO-02 builds upon TriSalus’ collaboration with leading cancer research centers to further develop the company’s organ-specific platform and rapidly bring new treatments to patients. The phase 1b/2 study will enroll up to 89 patients with HCC or ICC, while future studies will seek to validate this platform in additional liver and pancreatic tumor types.

Milind Javle, MD, professor in the Department of Gastrointestinal Medical Oncology at MD Anderson, will serve as principal investigator on the PERIO-02 trial.

On May 10, 2022 Catalyst Clinical Research, a market-leading provider of clinical research services, reported its selection into Inc. Magazine’s Best Places to Work (Press release, Catalyst Clinical Research, MAY 10, 2022, View Source [SID1234614067]). Catalyst was identified as an organization with Established Excellence for companies in the 5-14 years category for business. This is Catalyst’s first year being named to the list and Inc.’s most competitive selection process; only a small fraction of the thousands of applicants are featured.

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"At Catalyst, our people remain our greatest differentiator and we champion them every day. This honor provides strong evidence of Catalyst’s commitment to a people-first culture and reinforces that the work we are doing to create an intentional, healthy culture is truly making an impact," said Marshall Skelton, Senior Vice President of People and Culture at Catalyst Clinical Research. A big thanks to each of our employees for the role they play in helping Catalyst achieve this honor."

Results were based on an employee-based survey. Some of the insights gleaned during the selection process included:

More than 98% of our team members were judged engaged advocates of Catalyst.
In the open-ended commentary, many of the team members commented on our people-first culture and how it’s truly an action and not just a phrase. Example: "Catalyst says they put people first, and they really do. I’ve worked at other large CROs who have said the same thing, but in actuality, you are just a cog in a machine, and the only way you would get noticed there is if you mess up. They don’t just care for you while you work. If you have an illness with you or your family, they will reach out to make sure everything is ok, and offer to helo. They offer unlimited PTO, and constantly urge us to take advantage of it. I don’t view the people at Catalyst as my co-workers. I view them as an extension of my family."
More than three-quarters of the company’s employees agreed that "they see professional growth and career development opportunities for themselves in the organization."
The word that best described our work environment from survey takers was "Flexible."
Catalyst was in the top 10% of all companies for senior leaders visibly valuing people as the most important asset of the company.
"This recognition is a huge accomplishment as we emerge from a two-year pandemic and associated challenges, and creates a great opportunity to continue building a differentiated culture as we expand internationally both organically and via the acquisition of Aptus Clinical," said Nick Dyer, CEO of Catalyst Clinical Research. "I am honored on behalf of all our employees and grateful to our entire management group who engage with our teams daily, creating the foundation to make Catalyst an attractive company to work at and to grow a sustained career."

Additional recent awards and recognitions given to Catalyst Clinical Research include: Triangle Business Journal’s Fast 50 (2017, 2020, 2021), Inc.’s 5000 Fastest-Growing Private Companies (2020, 2021), CRO Leadership Awards (2020) and Clearlyrated Best Staffing – Talent Satisfaction (2021).

On May 10, 2022 GeneCentric Therapeutics, a company making precision medicine more precise through RNA-based diagnostics, reported an upcoming presentation of initial results from the GARNER (Genomic Analysis of high-Risk Non-muscle invasive bladder cancer) real-world study at the 2022 American Urological Association (AUA) Annual Meeting, which is being held in New Orleans, Louisiana, May 13-16, 2022 (Press release, GeneCentric Therapeutics, MAY 10, 2022, View Source [SID1234614066]). In this moderated poster presentation, the frequency of fibroblast growth factor receptor (FGFR) alterations in high-risk non-muscle invasive bladder cancer (HR-NMIBC) and the association with bacillus Calmette-Guérin (BCG) outcome will be discussed.

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The GARNER Study is the largest HR-NMIBC real-world patient cohort ever assembled with both clinical and genomic detail and the first study of the broader GARNER Bladder Cancer Program. The study is a collaboration between the Department of Urology at Erasmus MC Cancer Institute (EMC), Janssen Research & Development, LLC (Janssen) and GeneCentric Therapeutics. The collaboration, led at EMC by Tahlita Zuiverloon, MD, PhD, Principal Investigator at the Erasmus MC Urothelial Cancer Research Group (EUCRG), involves retrospective longitudinal, genomic analysis of samples from a cohort of almost 600 NMIBC patients who underwent surgery and adjuvant BCG treatment.

"The initial results from the GARNER Study provide a comprehensive picture of FGFR alteration frequency and other findings and provides a deeper understanding of drivers of disease progression, as well as potential factors related to treatment response and failure or drug resistance," said Dr Zuiverloon. "We look forward to presenting the initial findings from the study with our collaborators at GeneCentric and Janssen as we continue to explore the complexities of FGFR in HR-NMIBC."

While topline clinicogenomic results from this study will be presented at AUA2022, further results will be presented as part of a subsequent publication.

Details regarding the presentation are provided below and will be available following the meeting at View Source

Title: Frequency of Fibroblast Growth Factor Receptor Alterations and Association with Bacillus Calmette-Guérin Outcomes in a Real-World Genomic Analysis of High-Risk Non-Muscle-Invasive Bladder Cancer (GARNER) Study

First Author: Tahlita C.M. Zuiverloon, MD, Department of Urology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands

On May 10, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage company focused on DNA-based immunotherapy and next-generation vaccines, reported an update on the progress made in the Company’s two lead development programs (Press release, Celsion, MAY 10, 2022, View Source [SID1234614059]).

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"We continue to make important progress in both of our lead development programs, and I am very pleased by the encouraging results to date," said Michael H. Tardugno, chairman, president and chief executive officer of Celsion. "Our PLACCINE DNA-based vaccine platform was recently highlighted at the 2022 World Vaccine Congress and is demonstrating its potential for rapid design and capability for targeting two or more different COVID variants in one vaccine. We have demonstrated a proof-of-concept utilizing a standard mouse model showing that PLACCINE can target two variants and produce robust levels of IgG, neutralizing antibodies, and t-cell responses. This proof-of-concept data is comparing favorably to commercial vaccines in mouse models with this data recently reported at the World Vaccine Congress. A vaccine that targets multiple strains at once and designed to provide long lasting immunity, is important in a future COVID-19 vaccination strategy. We are moving this program forward quickly and anticipate confirming our proof-of-concept in non-human primates over the next several months, with durability results later this year."

Mr. Tardugno continued, "OVATION 2, the Phase II study of our GEN-1 immunotherapy in ovarian cancer is 85% enrolled. In spite of all of the challenges presented by COVID 19, we are hopeful to complete enrollment in the third quarter of this year. Preclinical and clinical data gives us every reason to believe in GEN-1’s promise for ovarian cancer patients along with the support from leading medical researchers of the Gynecological Oncology Group (GOG). The GOG’s interest in forging a partnership to develop GEN-1 in ovarian cancer will assist Celsion in its plans for an accelerated registrational program. Meanwhile, FDA has approved our protocol for a second Phase II clinical trial to evaluate GEN-1 in combination with Avastin (bevacizumab) in patients with advanced ovarian cancer. Our preclinical tumor inhibition data provides a convincing basis for this study. We look forward to initiating this study at major comprehensive cancer hospitals later this year."

About PLACCINE

PLACCINE is a first in class DNA vaccine platform that can target multiple antigens and be administered with a standard intramuscular injection. PLACCINE was derived from the Company’s proprietary TheraPlas platform.

About GEN-1

GEN-1, an IL-12 DNA plasmid vector formulated into nanoparticles with a lipopolymeric delivery system, is the first product designed via the TheraPlas platform technology. GEN-1 may prove to be a safe and effective immunotherapy for treating various types of tumors by producing high levels of interleukin-12 (IL-12) at the site of tumors. IL-12 is one of the most active cytokines for stimulating an immune response against cancer. However, when administered as a recombinant protein requiring systemic administration, the pharmacokinetics of IL-12 requires that it be given by frequent, large bolus injections, resulting in serious toxicities that limit its use. GEN-1 addresses the toxicity issues associated with systemic IL-12. GEN-1’s nanoparticle design enables local administration (into the abdominal cavity) and cell transfection followed by persistent, local secretion of IL-12 at therapeutic levels, while avoiding the toxicities associated with recombinant IL-12.

On May 10, 2022 Prelude Therapeutics Incorporated ("Prelude") (Nasdaq: PRLD), a clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2022, and provided an update on recent clinical and development pipeline progress (Press release, Prelude Therapeutics, MAY 10, 2022, View Source [SID1234614058]).

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"Prelude continues to make great progress in discovering and advancing a diverse pipeline of differentiated small molecules, and with our current cash runway, we have the opportunity to deliver on numerous meaningful milestones. I’m delighted to have Jane onboard and am confident in her leadership to guide focused clinical development of our pipeline and organizational growth," said Kris Vaddi, Ph.D., Chief Executive Officer.

"I’m excited to be a part of Prelude’s continued progress," said Jane Huang, M.D., President and Chief Medical Officer. "Looking ahead, we remain on track with ongoing development of our PRMT5 program, that will drive strategic decisions in the second half of the year. In parallel, we are focused on rapidly progressing our MCL1 candidate, PRT1419, into expansion and combination cohorts, and identifying a Phase 2 dose for PRT2527, our CDK9 inhibitor. We are also on track for Investigational New Drug (IND) submissions for both our SMARCA2 degrader and PRT3645, our brain penetrant CDK4/6 inhibitor, in the second half of the year. It’s clear that Prelude’s discovery engine, depth and breadth of the pipeline, coupled with an experienced management team, will position us to deliver potential medicines for patients with underserved cancers."

Recent Highlights and Upcoming Objectives

2022 AACR (Free AACR Whitepaper) Annual Meeting: During the quarter, Prelude participated in the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Four posters and one oral presentation providing data on Prelude’s clinical and preclinical pipeline molecules, with highly potent, selective and differentiated properties, were presented as part of the scientific conference.

PRMT5 Inhibitor Program: As previously announced, Prelude has prioritized PRT811 for clinical development in select expansion cohorts. Prelude intends to complete the data analyses of the ongoing expansion cohorts and expects to announce next steps for the PRMT5 program in 2H2022.

MCL1 Inhibitor Program: As previously announced, Prelude has prioritized development of the intravenous formulation of PRT1419, which demonstrated a desirable pharmacokinetic, pharmacodynamic and safety profile, with potential for differentiation from competitor compounds. Prelude remains on track to begin evaluating combinations with PRT1419 by mid-year.

CDK9 Inhibitor Program: Prelude remains on track to complete enrollment in the Phase 1 dose escalation study of PRT2527 and identify a recommended Phase 2 dose by 2H2022.

CDK4/6 Inhibitor Program: Prelude continues to expect to file an IND application mid-year, with the initiation of a Phase 1 trial of PRT3645 to follow in 2H2022.

SMARCA2/BRM Protein Degrader Program: Prelude remains on track to complete IND-enabling studies and submit an IND application by year-end 2022.
Corporate Update

In March 2022, Prelude announced the appointment of Jane Huang, M.D., effective April 4, 2022, to the newly created position of President and Chief Medical Officer. Dr. Huang joins Prelude from BeiGene Ltd., where she served as Chief Medical Officer, Hematology. Currently, Dr. Huang serves as an Adjunct Clinical Assistant Professor in Thoracic Oncology at Stanford University.
First Quarter 2022 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents, and marketable securities as of March 31, 2022, were $266.2 million. Prelude anticipates that its existing cash, cash equivalents and marketable securities will fund Prelude’s operations into the second half of 2024.

Research and Development (R&D) Expenses: For the first quarter of 2022, R&D expense increased to $22.8 million from $16.5 million for the prior year period. Included in research and development expenses for the quarter ended March 31, 2022, was $3.2 million of non-cash expense related to stock-based compensation expense, including employee stock options, as compared to $1.8 million for the prior year period. The increase in research and development expense was primarily due to an increase in discovery-stage program expenses and from the growth and advancement of our clinical pipeline. We expect our research and development expenses to vary from quarter to quarter, primarily due to the timing of our clinical development activities.

General and Administrative (G&A) Expenses: For the first quarter of 2022, G&A expense increased to $7.5 million from $5.5 million for the prior year period. Included in the G&A expenses for the quarter ended March 31, 2022, was $3.6 million of non-cash expense related to stock-based compensation expense, including employee stock options, as compared to $2.0 million for the prior year period. The increase in G&A expense was primarily due to an increase in our non-cash stock compensation expense along with professional fees as we expanded our operations to support our research and development efforts.

Net Loss: For the three months ended March 31, 2022, net loss was $29.5 million, or $0.63 per share of common stock, basic and diluted compared to $21.3 million, or $0.47 per share, respectively, for the prior year period. Included in the net loss for the quarter ended March 31, 2022, was $6.8 million of non-cash expense related to the impact of expensing share-based payments, including employee stock options, as compared to $3.9 million for the prior year period.