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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported financial results for the first quarter ended March 31, 2022 (Press release, NanoString Technologies, MAY 10, 2022, View Source [SID1234614083]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

First Quarter Financial Highlights

Product and service revenue of $30.8 million
GeoMx Digital Spatial Profiler (DSP) revenue of $9.7 million. GeoMx DSP revenue includes:
Instrument revenue of $4.8 million, 31% year-over-year decline
Consumables revenue of $4.9 million, 78% year-over-year growth, annualized pull-through of approximately $76,000 per installed system
nCounter revenue, inclusive of all service revenue, of $21.1 million. nCounter revenue includes:
Instrument revenue of $4.3 million, 11% year-over-year decline
Consumables revenue of $12.6 million, 5% year-over-year decline, annualized pull-through of approximately $48,000 per installed system
Service revenue of $4.3 million, 16% year-over-year growth
Cash, cash equivalents and short-term investments balance of $312.1 million
"We have begun to address the challenges we experienced in the first quarter, and we believe we have the sales team and commercial structure in place to execute our long-term growth strategy" said Brad Gray, president & CEO of NanoString. "Overall growth and customer interest in spatial biology remains robust, as evidenced by the strong demand for CosMx SMI that we experienced during the first quarter."

Operational Highlights

GeoMx DSP

GeoMx Installed Base: Grew installed base to approximately 295 GeoMx DSP Systems at March 31, 2022, representing 84% growth over the prior year
Workflow Automation: Partnered with Leica, a division of Danaher, to provide a fully automated workflow for using the Leica Bond RX system to prepare slides for GeoMx DSP
American Association of Cancer Research: Highlighted a record body of more than 120 Nanostring-enabled abstracts, including more than 35 abstracts focused on spatial biology
Publications: Continued growth of peer-reviewed publications utilizing GeoMx DSP technology, with approximately 20 new publications in the first quarter, bringing the cumulative total to approximately 110 peer-reviewed publications as of March 31, 2022
CosMx SMI

CosMx Orders: Secured customer orders for more than 15 CosMx Spatial Molecular Imager (SMI) systems, bringing total orders to date to more than 35 systems
Development Status: First CosMx beta systems placements expected during the second quarter and the program remains on track for full commercial launch in the fourth quarter of 2022.
nCounter

nCounter Installed Base: Grew installed base to approximately 1,070 nCounter Analysis Systems at March 31, 2022, representing 8% growth over the prior year
nCounter Pro Analysis System Launch: Launched the nCounter Pro Analysis System, the next generation of the nCounter instrument platform, which includes a new operating system, modern cybersecurity improvements and an expansive menu to support biomanufacturing and translational research
Publications: Surpassed 5,250 cumulative peer-reviewed publications utilizing nCounter technology at March 31, 2022
Corporate

Appointment of New Board Member: Expanded the Board of Directors with the appointment of Dr. Teresa Foy, senior vice president, Immuno-Oncology and Cell Therapy, Bristol Myers Squibb
2022 Outlook

The company updated its 2022 outlook, with results expected as follows:

Total product and service revenue of $150 to $160 million, as compared to previous guidance of $170 to $180 million
GeoMx DSP revenue of $60 to $65 million, as compared to previous guidance of $73 to $78 million
nCounter revenue, inclusive of all service revenue, of $90 to $95 million, as compared to previous guidance of $97 to $102 million
Adjusted EBITDA loss of $65 to $75 million, as compared to previous guidance of $55 to $65 million
Financial Results

We have elected to present selected non-GAAP, or adjusted, financial measures, including Adjusted EBITDA. These adjusted financial measures are calculated excluding certain items that may make it more challenging to compare our GAAP operating results across periods. Such items may include collaboration revenue, stock-based compensation, depreciation and amortization, or one-time charges such as transaction related fees and expenses or restructuring charges and severance costs. A reconciliation of adjusted financial measures to the nearest comparable GAAP financial measure can be found in the notes and table at the end of this press release.

Supplemental Information

As a supplement to the table above, we have posted to the investor relations section of our website, at www.nanostring.com/Investor Relations/Financials/Quarterly Results, supplemental financial data that include our adjusted financial measures as compared to the nearest comparable GAAP financial measures, for the first quarter of 2022 and for each quarter and the full year of 2021.

Conference Call

Management will host a conference call today beginning at 1:30 pm PT / 4:30 pm ET to discuss these results and answer questions. Investors and other interested parties can register for the call in advance by visiting View Source After registering, an email confirmation will be sent including dial-in details and unique conference call codes for entry. Registration is open throughout the call, but to ensure connection for the full call, registration in advance is recommended. The link to the webcast and audio replay will be made available at the Investor Relations website: www.nanostring.com. A replay of the call will be available beginning May 10, 2022 at 7:30pm ET through midnight ET on May 17, 2022. To access the replay, dial (866) 813-9403 or (929) 458-6194 and reference Conference ID: 221442. The webcast will also be available on our website for one year following the completion of the call.

Non-GAAP, or Adjusted, Financial Information

We believe that the presentation of non-GAAP, or adjusted, financial information provides important supplemental information to management and investors regarding financial and business trends relating to our financial condition and results of operations. Reconciliation of adjusted financial measures to the most directly comparable financial result as determined in accordance with GAAP are included at the end of this press release following the accompanying financial data. A reconciliation of adjusted guidance measures to corresponding GAAP measures is not available on a forward-looking basis without unreasonable effort due to the uncertainty regarding certain expenses that may be incurred in the future. For further information regarding why we believe that these adjusted measures provide useful information to investors, the specific manner in which management uses these measures and some of the limitations associated with the use of these measures, please refer to "Notes Regarding Non-GAAP Financial Information" at the end of this press release.

On May 10, 2022 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the first quarter of 2022 and provided a corporate update (Press release, Aclaris Therapeutics, MAY 10, 2022, View Source [SID1234614099]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We have continued to progress our clinical programs, including activating multiple clinical sites in our Phase 2b trial of ATI-1777 in subjects with moderate to severe atopic dermatitis," said Dr. Neal Walker, President and CEO of Aclaris. "We look forward to advancing all of our clinical and preclinical programs."

Research and Development Highlights:

Clinical Programs

Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
Currently being developed as a potential treatment for immuno-inflammatory diseases

ATI-450-RA-202: This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe rheumatoid arthritis (RA) is ongoing.
Aclaris expects topline data in 2023.

ATI-450-HS-201: This Phase 2a trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of zunsemetinib (50 mg twice daily) in subjects with moderate to severe hidradenitis suppurativa (HS) is ongoing.
Aclaris expects topline data in the first half of 2023.

ATI-450-PsA-201: Aclaris expects to activate clinical sites in the coming weeks in this Phase 2a trial of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis.
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developing as a potential treatment for moderate to severe atopic dermatitis (AD)

ATI-1777-AD-202: Aclaris activated multiple clinical sites in May 2022 in this Phase 2b trial to determine the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with moderate to severe AD. In this trial, Aclaris will explore multiple concentrations of twice daily treatment with ATI-1777 and a single concentration of once daily treatment with ATI-1777, in patients 12 years and older.
Aclaris expects topline data in the first half of 2023.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor:
Currently being developed as a potential treatment for T cell-mediated autoimmune diseases

ATI-2138-PKPD-101: This Phase 1 single ascending dose (SAD) trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-2138 in healthy subjects is ongoing.
Aclaris expects topline data in 2022.

If the Phase 1 SAD trial is successful, Aclaris currently plans to initiate a two-week Phase 1 multiple ascending dose trial of ATI-2138 in subjects with psoriasis in 2022. Aclaris is also currently exploring alternative indications to the planned indication that are relevant to the mechanism of action.
Preclinical Programs

ATI-2231, an investigational oral MK2 inhibitor compound:
Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer

Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long half-life.

IND-enabling studies are underway, and Aclaris expects to submit an IND by the end of 2022.
Discovery Programs

Currently developing oral gut-biased JAK inhibitors with limited systemic exposure as potential treatments for inflammatory bowel disease.
Central nervous system (CNS) kinase inhibitor targets:
Currently engaged in research to identify brain penetrant kinase inhibitor candidates and assess their impact on neuronal pro-inflammatory cytokine production, microglia growth and survival, and neurodegeneration.
Other Highlights

Aclaris continues to expand its senior R&D team and recently appointed Ian Anderson, Ph.D., as Executive Vice President, Translational Research & Development, and Rob Ortmann, M.D., as Vice President, Clinical Development. Dr. Anderson brings more than 30 years of immunology research experience in drug development, from discovery through Phase 2. He previously held senior scientific leadership roles at Flame Biosciences, Janssen Pharmaceutical, MedImmune and Cambridge Antibody Technology. Dr. Ortmann is a board-certified rheumatologist with more than 10 years of clinical research experience in autoimmune-related therapeutic areas. He previously held clinical development positions at Horizon Therapeutics and Eli Lilly and Company.

Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2022, Aclaris had aggregate cash, cash equivalents and marketable securities of $204 million compared to $226 million as of December 31, 2021. Additionally, in April 2022, Aclaris sold approximately 4.8 million shares under its ATM facility for aggregate net proceeds of $73 million.

Aclaris now anticipates that its cash, cash equivalents and marketable securities as of March 31, 2022 in combination with the $73 million in net proceeds from the April 2022 ATM sale will be sufficient to fund its operations through the end of 2025, without giving effect to any additional potential business development transactions or financing activities.

Financial Results

First Quarter 2022

Net loss was $18.8 million for the first quarter of 2022 compared to $28.8 million for the first quarter of 2021.
Total revenue was $1.5 million for the first quarter of 2022 compared to $1.8 million for the first quarter of 2021.
Research and development (R&D) expenses were $14.3 million for the quarter ended March 31, 2022 compared to $7.8 million for the prior year period.
The $6.5 million increase was primarily the result of:
Higher zunsemetinib development expenses, including costs associated with clinical activities for a Phase 2b trial for RA and a Phase 2a trial for HS.
Higher ATI-1777 development expenses related to drug candidate manufacturing and other preclinical activities and start-up costs associated with a Phase 2b clinical trial.
Higher preclinical development activities related to ATI-2231.

General and administrative (G&A) expenses were $6.1 million for the quarter ended March 31, 2022 compared to $4.8 million for the prior year period.
The $1.3 million increase was primarily the result of higher compensation-related costs, including stock-based compensation, due to increased headcount and the impact of new equity awards granted during the first quarter of 2022.
Revaluation of contingent consideration resulted in a $1.2 million credit for the quarter ended March 31, 2022 compared to a contingent consideration charge of $16.4 million for the prior year period.

On May 10, 2022 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, reported that Chief Executive Officer John P. Furey will be presenting at the following upcoming investor conferences (Press release, Imvax, MAY 10, 2022, View Source [SID1234614131]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

2022 RBC Capital Markets Global Healthcare Conference
Date: May 18, 2022 Time: 2:05 p.m. ET
Location: New York City, NY

UBS Global Healthcare Conference 2022
Date: May 24, 2022 Time: 12:15 p.m. ET
Location: New York City, NY

On May 10, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that the phase I trial interim results of an investigator-initiated trial of CT041 have been published in Nature Medicine (View Source), which is one of the top international medical journals in the Nature Portfolio (2-year Impact Factor of 53.44) (Press release, Carsgen Therapeutics, MAY 10, 2022, View Source [SID1234614148]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The publication, titled "Claudin18.2-specific CAR T cells in gastrointestinal cancers: phase I trial interim results" presented results from a multi-center, open-label Phase I clinical trial conducted in China to explore the safety, efficacy, and cellular pharmacokinetics of CT041 in patients with advanced CLDN18.2-positive gastrointestinal cancers. The interim trial results showed that among 37 patients with advanced gastrointestinal cancers, CT041 was well tolerated with a manageable safety profile including no dose-limiting toxicity in 28 days post infusion, no grade ≥3 cytokine release syndrome, no neurotoxicity, and no treatment-related death. The objective response rate (ORR) and disease control rate (DCR) in patients with gastrointestinal cancers were 48.6% and 73.0%, respectively. In patients with gastric cancer or gastroesophageal junction cancer (GC/GEJ), the ORR and DCR reached 57.1% and 75.0%, respectively.

As of the date of this announcement, CT041 is the world’s first and only CAR T-cell candidate for the treatment of solid tumors entering a confirmatory Phase II clinical trial.

The corresponding author for the article, Professor Lin Shen of Beijing Cancer Hospital, said, "CAR T-cell therapy has successfully treated a variety of hematologic malignancies, while few breakthroughs have been made in solid tumors. CT041 is the first-in-class CAR T-cell product candidate against Claudin18.2. In this clinical trial, CT041 showed promising efficacy and manageable safety for patients with gastrointestinal cancers, particularly gastric cancer, who have failed prior lines of conventional therapies. As the largest clinical trial for solid tumors to date, the CAR T-cell therapy showed unprecedented efficacy against solid tumors. The publication of the interim trial results in Nature Medicine enables us to better share the results with our peers and advance CAR T-cell therapy in solid tumors. It also demonstrates the potential for more innovative, China-developed medicines to reach and benefit patients in global markets."

"This study is made possible by the joint efforts of CARsgen’s research and development team and our investigators, as well as the support and trust from the patients and their families. For CT041, the world’s first solid tumor CAR T-cell product candidate that has entered a confirmatory Phase II clinical trial, we will spare no efforts to advance the clinical trials and benefit patients as soon as possible," added Shen.

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, said, "Congratulations to the research team led by Prof. Shen. We thank the patients participating in the trial for their trust and thank all the researchers for their dedication. Publication of the interim CT041 trial results in Nature Medicine demonstrates the high quality and value of this trial. The development of innovative therapies has always been an arduous journey, particularly for effective CAR T-cell therapies against solid tumors. Despite these challenges, we started to collaborate with our investigators in 2015 for the treatment of hepatocellular carcinoma and glioblastoma. Since then, we have continued to explore innovative technologies and products. I am very glad to see the breakthrough interim results of CT041 in the research led by Prof. Shen. We will continue the joint efforts with our investigators in the global CT041 clinical development."

About CT041

CT041 is an autologous CAR T-cell product candidate against the protein CLDN18.2 that has the potential to be the first-in-class globally. CT041 targets the treatment of CLDN18.2-positive solid tumors with a primary focus on GC/GEJ and pancreatic cancer (PC).

In addition to the investigator-initiated trials in China, CARsgen has initiated a Phase Ib clinical trial for advanced GC/GEJ and PC, confirmatory Phase II clinical trial for advanced GC/GEJ in China and initiated a Phase 1b clinical trial for advanced gastric or pancreatic adenocarcinoma in North America. CARsgen also intends to initiate a pivotal Phase 2 clinical trial in North America in 2022.

CARsgen plans to submit an NDA to the NMPA in China in the first half of 2024 and to submit the BLA to the U.S. FDA in 2024.

In 2020 and 2021, CT041 received Orphan Drug designation from the U.S. FDA for the treatment of GC/GEJ and Orphan Medicinal Product designation from the EMA for the treatment of advanced gastric cancer. In November 2021, CT041 was granted PRIME Eligibility by the EMA for the treatment of advanced gastric cancer. In January 2022, CT041 was granted Regenerative Medicine Advanced Therapy (RMAT) Designation by the U.S. FDA for the treatment of advanced gastric or gastroesophageal junction adenocarcinoma with CLDN18.2-positive tumors.

As of the date of the announcement, CT041 is the only CLDN18.2-targeted CAR T-cell product candidate globally that is being studied in clinical trials with IND/CTA approvals from the FDA, the NMPA, and Health Canada.