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EDAP Announces Publication of Positive Pre-clinical Results Using Intraoperative HIFU Ablation of the Pancreas in the Journal Cancers

On January 13, 2022 EDAP TMS SA (Nasdaq: EDAP) ("the Company"), the global leader in robotic energy-based therapies, reported the publication of positive pre-clinical results using intraoperative high intensity focused ultrasound (HIFU) ablation of the pancreas in the peer-reviewed journal Cancers (Press release, EDAP TMS, JAN 13, 2022, View Source [SID1234605466]).

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The paper, entitled, Intraoperative HIFU Ablation of the Pancreas Using a Toroidal Transducer in a Porcine Model. The First Step towards a Clinical Treatment of Locally Advanced Pancreatic Cancer, describes a pre-clinical study designed to assess the feasibility of using HIFU under Doppler guidance to treat the pancreatic parenchyma and tissues surrounding the superior mesenteric vessels in vivo, in an animal model.

Pancreatic adenocarcinoma is among the most aggressive of all cancers. Regardless of treatment, the overall 5-year survival rate for patients with locally advanced pancreatic adenocarcinoma ("LAPA") is less than 5%. A HIFU approach could help in the treatment of such pathology that is inaccessible by other known therapeutic methods. Apart from palliative chemotherapy, no other curative options have been proven effective for the treatment of LAPA.

During the study, HIFU treatments were performed using an intraoperative HIFU probe which caused irreversible tissue necrosis in only a few seconds by producing sufficiently strong heat in the focal area. This study is the first step toward the launch of a Phase I study to evaluate the safety and feasibility of such a HIFU approach in patients with LAPA and to further confirm these encouraging results.

"The possibility of treating the pancreas using HIFU holds great promise for the treatment of locally advanced pancreatic cancers. If clinically successful, chemotherapy followed by HIFU treatment could rapidly become a novel treatment option of LAPA," concluded the paper’s authors.

Marc Oczachowski, EDAP’s Chairman and Chief Executive Officer, said: "We are very excited with these pre-clinical results and the promise they demonstrate for HIFU as a viable treatment for pancreatic cancer and other severe pathologies with low overall survival rates and no effective treatment options. We are proud of the ongoing work of the EDAP research team, who, together with Inserm and Centre Leon Bérard, are advancing the development of HIFU to offer patients a potentially effective treatment alternative for these difficult to treat cancers."

The full text of the paper can be found here.

AMPLIA COMPLETES MANUFACTURE OF AMP945 FOR PLANNED TRIALS

On January 13, 2022 Amplia Therapeutics Limited (ASX: ATX) ("Amplia", the "Company") reported a GMP* manufacturing run of its clinical-stage drug candidate AMP945 has been successfully completed (Press release, Amplia Therapeutics, JAN 13, 2022, View Source;[email protected] [SID1234605465]). This provides additional drug substance to support toxicology studies and Phase 2 clinical trials in pancreatic cancer and lung fibrosis which are scheduled to start during 2022.

Amplia’s contract manufacturing organisation (CMO) has successfully completed the manufacture of a 2 kg GMP batch of AMP945 within budget and on time. Previously, the maximum batch size of AMP945 that had been manufactured was 1 kg, meaning that the Company has now demonstrated a doubling of the scale at which AMP945 can be manufactured under GMP conditions. During the recently completed manufacturing run, the Company also implemented improvements to the manufacturing process for AMP945 that will support its future clinical and commercial development. Of note, long-term stability testing data has shown that AMP945 can be stored for periods of more than 24 months without detectable deterioration, an outcome that further supports commercial development of AMP945.

Amplia’s CEO, Dr John Lambert commented that "The successful doubling of our manufacturing capacity for clinical-grade AMP945 and the implementation of manufacturing improvements are outstanding outcomes which have exceeded our initial expectations. As well as providing material for our near-term development programs, these improvements lay a strong foundation for the AMP945 data package that will be required for future regulatory applications and its commercial development."

The Phase 2 clinical trial of AMP945 in patients with pancreatic cancer remains on track to commence during the current quarter. In addition, the extended (3-month) toxicology studies required to support the Phase 2 clinical trial of AMP945 in patients with fibrotic lung disease are on schedule to commence in February 2022 as previously advised.

* GMP: Good Manufacturing Practice

This ASX announcement was approved and authorised for release by the CEO of Amplia Therapeutics.

Rubius Therapeutics Announces Dosing of First Patient in Phase 1/2 Trial of RTX-224, a Broad Immune Agonist, for the Treatment of Certain Solid Tumors

On January 13, 2022 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is biologically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune disease, reported that the first patient has been dosed in its Phase 1/2 clinical trial of RTX-224 for the treatment of patients with certain relapsed/refractory or locally advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer (Press release, Rubius Therapeutics, JAN 13, 2022, View Source [SID1234605463]).

"While our lead oncology product candidate, RTX-240, is designed to broadly stimulate the immune system by activating and expanding NK and CD8+ memory T cells, we expect RTX-224 to produce a broad and potent anti-tumor T cell response, an innate immune response and have anti-tumor activity in those tumor types with known sensitivity to T cell killing, including tumor types with high mutational burden, PD-L1 expression and known responsiveness to checkpoint inhibitors," said Larry Turka, M.D., chief scientific officer and head of research and translational medicine of Rubius Therapeutics. "Given the mechanism of action of RTX-224 and totality of our preclinical data generated to date, we believe RTX-224 will be an effective treatment for select advanced solid tumors."

RTX-224 is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to express hundreds of thousands of copies of 4-1BB ligand (4-1BBL) and interleukin-12 (IL-12) on the cell surface.

About the RTX-224 Phase 1/2 Clinical Trial
This is a Phase 1/2 open label, multicenter, multidose, first-in-human dose-escalation and expansion study to determine the safety and tolerability, pharmacokinetics, maximum tolerated dose and a recommended Phase 2 dose and dosing regimen of RTX-224 in adult patients with certain relapsed/refractory or locally advanced solid tumors including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer. The trial will also assess pharmacodynamic changes in immune cell populations relative to baseline and anti-tumor activity. The study will include a monotherapy dose escalation phase followed by an expansion phase in specified tumor types during the Phase 2 portion of the trial.

Regeneron to Report Fourth Quarter and Full Year 2021 Financial and Operating Results and Host Conference Call and Webcast on

On January 13, 2022 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will report its fourth quarter and full year 2021 financial and operating results on Friday, February 4, 2022, before the U.S. financial markets open (Press release, Regeneron, JAN 13, 2022, View Source [SID1234605462]). The Company will host a conference call and simultaneous webcast at 8:30 AM Eastern Time that day.

Conference Call Information
Participants may access the conference call live via webcast on the ‘Investors and Media’ page of Regeneron’s website at View Source To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company’s website for at least 30 days.

Quanterix Provides Operational and Preliminary Financial Highlights

On January 13, 2022 Quanterix Corporation (NASDAQ: QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, reported that operational and preliminary financial highlights for the fourth quarter and fiscal year ending December 31, 2021 (Press release, Quanterix, JAN 13, 2022, View Source [SID1234605461]).

"The healthcare industry will reach its next inflection point in 2022 requiring novel solutions to combat COVID-19 and its new variants like Omicron, in addition to treating challenging neurodegenerative disorders, including Alzheimer’s and Parkinson’s, which affect more than seven million people in the United States alone," said Kevin Hrusovsky, Chairman and Chief Executive Officer, Quanterix.

"Our ultra-sensitive biomarker detection technology has been making unprecedented progress in these key therapeutic areas, particularly with the recent Breakthrough Device Designation for our plasma pTau-181 Alzheimer’s test and COVID-19 Antigen EUA label expansion for asymptomatic and saliva testing. Quanterix continues to empower drug developers with tests that improve drug trial efficiency, and which also show promise to payors for early disease detection, treatment for improved outcomes, and cost-effective patient monitoring to deliver clinical evidence in support of coverage decisions. We believe that the high sensitivity and accuracy of these tests ultimately have the potential to transform reactive, symptomatic medicine, or ‘sick-care,’ to proactive, asymptomatic healthcare," continued Hrusovsky.

2021 Operational Highlights

Quanterix’ pTau-181 assay was granted Breakthrough Device Designation from the U.S. FDA as an aid in the diagnosis of Alzheimer’s disease.
Eli Lilly presented new data from its Phase 2 TRAILBLAZER-ALZ study, which employed Quanterix’ ultra-sensitive Simoa HD-X technology to measure plasma pTau-217, using antibodies developed by Lilly. Lilly reported a significant reduction in blood levels of phosphorylated Tau protein after treatment with donanemab, and that a reduction in plasma pTau-217 levels correlated with the slowing of cognitive decline.
Following the FDA approval of ADUHELM, Biogen conducted Simoa biomarker studies on Phase 3 EMERGE and ENGAGE trial samples, utilizing Quanterix’ Simoa HD-X to measure plasma pTau-181. Dr. Oskar Hansson from University of Lund reported preliminary data showing a dose-dependent reduction in plasma pTau-181 levels following treatment with ADUHELM, which correlated with decreases in amyloid PET and a slowing of cognitive decline across four independent assessment tools.
Quanterix received label expansion on the Emergency Use Authorization for its COVID Antigen Test.
Instrument installations increased by 32% in 2021 to 708, with many HD-X instruments being used for neuro-related applications.
Data presented at 2021 Clinical Trials on Alzheimer’s Disease (CTAD) conference described a prototype Simoa plasma pTau-231 assay and the potential role for this emerging biomarker in the detection of Alzheimer’s disease pathology even earlier in the disease continuum, when patients are asymptomatic and not yet exhibiting brain pathology in PET imaging studies.
Academic publication pull-through performance continued to be strong. Quanterix’ Simoa technology was highlighted in a record 465 new publications in 2021, bringing total Simoa-specific inclusions to over 1,585.
The Company strengthened its balance sheet by successfully raising $287.5 million in gross proceeds through a follow-on offering. Quanterix had $411 million in cash and cash equivalents on the balance sheet at the end of Q3 FY21.
Hired President and CCO in 2021 to accelerate the operational scaling of Quanterix’ RUO opportunity and to catalyze the symbiotic relationship between Research and Diagnostic.
In an interview with Managed Healthcare Executive for the "Tuning into the C-Suite Podcast," Hrusovsky discussed successful tools to improve precision health. The podcast was named one of the most popular multimedia posts for 2021.
Quanterix was named a finalist for the Deloitte Fast 500 list, which ranks the fastest-growing technology, media, telecommunications and life sciences companies based in North America. The annual ranking is based on percentage revenue growth over the Company’s last three fiscal years.
Preliminary Non-Audited Financial Highlights:

Q4 2021

Expect total revenue, which includes one-time grant revenue, of $29-31 million(1); product and service revenue of $28-30 million; and product (consumables and instruments) revenue of $21-23 million.
Product and service revenue represents an increase of approximately 35% over prior Q4.
Product revenue represents an increase of approximately 45% over prior Q4.
Full Year 2021

Expect total revenue, which includes one-time grant revenue, of $109-111 million(2); product and service revenue of $104-106 million; and product revenue of $80-82 million.
Product and service revenue growth represents an increase of approximately 53% year over year.
Product revenue growth represents an increase of approximately 84% year over year.
(1) Total revenue for Q4 2021 includes approximately $1 million in RADx-related grant revenue, which is subject to adjustment pending finalization of contract accounting for Q4. Total revenue for Q4 2020 includes approximately $4.5 million in RADx-related grant revenue.

(2) Total revenue for full year 2021 includes the approximate $1 million in Q4 RADx-related grant revenue noted above plus an additional $4 million of RADx revenue that was recognized in the first three quarters of 2021. Total revenue for full year 2020 includes (i) $6.4 million in RADx revenue; and (ii) $11.2 million revenue recognized in connection with certain license agreements with Abbott laboratories.

The estimated unaudited financial results as of and for the fourth quarter and full fiscal year ended December 31, 2021 presented above are preliminary and are subject to completion of our quarter-end and year-end closing procedures and further financial review. This preliminary financial information has not been audited by our independent auditing firm. Our actual results may differ from these estimates as a result of the completion of our quarter-end and year-end closing procedures, review adjustments and other developments that may arise between now and the time our financial results for the fourth quarter and year are finalized.

Presentation at JP Morgan Healthcare Conference

As previously announced, Hrusovsky will be presenting the Company’s growth and vision for the sixth consecutive year at the 40th Annual J.P. Morgan Healthcare Conference. Hrusovsky’s session will take place on Thursday, Jan. 13, 2022, at 8:15 a.m., EST (5:15 a.m., PST) and will be made available to attendees and the general public. In addition to Hrusovsky’s session, he will also be hosting one-on-one meetings with institutional investors during the conference.

To access the live webcast of Quanterix’ presentation, please visit: View Source;kiosk=true