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]).

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"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

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.

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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.

On January 13, 2022 AstraZeneca reported that has signed a collaboration agreement with Scorpion Therapeutics to discover, develop and commercialise precision medicines against previously hard-to-target cancer proteins, with the potential to transform oncology treatment (Press release, AstraZeneca, JAN 13, 2022, View Source [SID1234598651]).

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The new collaboration focuses on a class of proteins called transcription factors, which control gene expression and can regulate important cellular processes including cell growth and survival. Many transcription factors have long been identified as important targets for new cancer treatments and as drivers of disease,1 but have previously been considered ‘undruggable’ using conventional drug discovery approaches.

To overcome the challenges of targeting transcription factors and to reach underserved patient populations, the collaboration will combine Scorpion’s fully integrated discovery platform with AstraZeneca’s leadership in developing and commercialising precision medicines for cancer treatment.

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "Unlocking potentially transformative biology is pivotal for delivering the next wave of cancer treatments. Scorpion’s innovative platform is a strong strategic fit as we explore a range of new modalities across our broad drug discovery toolbox with promise to disrupt the activity of these highly-validated cancer targets."

Axel Hoos, MD, PhD, Chief Executive Officer, Scorpion, said: "We are pleased to enter into this collaboration with AstraZeneca, whose expertise in drug development and commercialisation complements our discovery platform, which leverages cutting-edge advances in cancer biology and medicinal chemistry, including chemical proteomics, structure-based drug design and machine learning. We expect this collaboration will accelerate Scorpion’s efforts to deliver the promise of ‘Precision Oncology 2.0’: optimised, transformational therapies for more patients living with cancer."

Under the terms of the collaboration agreement, Scorpion will lead discovery and certain preclinical activities. AstraZeneca has the exclusive option to licence worldwide rights for up to three drug candidates. AstraZeneca would be responsible for development and commercialisation activities worldwide following opt-in, while Scorpion would retain the option to co-develop and co-promote up to two of these programmes in the US under certain conditions, including if AstraZeneca exercises three licence options.

Financial considerations
Scorpion will receive an upfront cash payment of $75m and is eligible to receive additional success-based payments in the form of option fees and milestone payments, as well as tiered royalties on net sales ranging from mid-single digit to low-double digits.

In the event Scorpion opts into co-developing and co-promoting a nominated programme, Scorpion will participate in the operating costs and be entitled to a proportionate share of the economics in the US, subject to certain adjustments.

Notes

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

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]).

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"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.

On January 13, 2022 APIM Therapeutics (APIM), a clinical stage biotech company, focusing on the development of novel peptide therapeutics targeting PCNA (Proliferating Cell Nuclear Antigen), reported the publication of research data of ATX-101 in glioblastoma preclinical models in the peer reviewed journal Cancers (Press release, APIM Therapeutics, JAN 13, 2022, View Source,c3485004 [SID1234605480]).

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The article entitled "ATX-101, a Peptide Targeting PCNA, has antitumor efficacy alone or in combination with Radiotherapy in murine models of human glioblastoma" investigated ATX-101, the lead compound of APIM’s development program, in a panel of human glioblastoma (GBM) and patient-derived glioma-initiating cells (GICs) in vitro and in vivo alone or in combination with Radiotherapy (RT). The study was a collaboration between APIM Therapeutics and the laboratory of Dr. Festuccia at the Department of Biotechnological and Applied Clinical Sciences, Laboratory of Radiobiology, University of L’Aquila, Italy.

In the study, it was shown that ATX-101 has anticancer activity as a single agent in vitro and in vivo. Furthermore, ATX-101 potentiated the effect of RT in both subcutaneous and intracranial xenograft tumor models. ATX-101 affected key oncogenic signaling pathways such as AKT/mTOR and DNA-PKcs; a correlation between high AKT activation and sensitivity to ATX-101 was also observed. Finally, ATX-101 increased the levels of γH2AX, DNA fragmentation, and apoptosis when combined with RT.

"The results obtained in glioblastoma models are in agreement with previous preclinical investigations of ATX-101 conducted in a large variety of tumor models" said Professor Marit Otterlei, CSO of APIM Therapeutics. "Our data suggest that targeting the stress regulatory mechanisms of PCNA holds therapeutic promise in glioblastoma tumors".

"The fact that ATX-101 exhibits therapeutic activity and potentiates the effect of RT in GBM opens up new clinical development opportunities for APIM Therapeutics" stated Dr. Kostas Alevizopoulos, CEO of APIM. "This could help provide a new therapeutic option for this deadly disease to be tested in human clinical trials."

The article co-authored by Dr M. Otterlei, Dr C. Festuccia and coworkers and published in Cancers (January 7, 2022) can be accessed here: View Source

About ATX-101

ATX-101 is a first-in-class, cell penetrating peptide featuring a novel PCNA-interacting motif (AlkB homolog 2 PCNA Interacting Motif or APIM). In preclinical experiments, it was shown that APIM-containing proteins bind to PCNA and mediate processes of escape mechanisms and survival of cancer cells. ATX-101 competitively inhibits interaction of PCNA with APIM-containing protein complexes resulting in cancer cell death and altered cellular signaling. These properties translate in anticancer effects of ATX-101 as demonstrated in several preclinical models in vitro and in vivo. Currently, ATX-101 is in clinical development for ovarian cancer (phase 1b/2a) and sarcoma (phase 2).