On February 14, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer and Brian Mueller, Executive Vice President, Chief Financial Officer of BioMarin will participate virtually in the SVB Securities Global Biopharma Conference on Thursday, February 16, 2023, at 11:20 a.m. ET (Press release, BioMarin, FEB 14, 2023, View Source,-2023 [SID1234627170]).

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To access the live webcast, please visit: View Source An archived version of the presentation will also be available through the Company’s website for a limited time following the conference.

On February 14, 2023 Bio-Techne Corporation (NASDAQ: TECH) and Cell Signaling Technology (CST) reported the addition of Simple Western validation to CST antibodies (Press release, Bio-Techne, FEB 14, 2023, View Source [SID1234627169]). This will allow researchers in various disciplines an easier way to study important molecular signaling pathways on a trusted and validated platform. Obtaining accurate data efficiently and fast is key to advancing drug discovery and development. This ongoing partnership will allow for an expanded menu of Simple Western validated antibodies for various targets across multiple disciplines.

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Simple Western systems from ProteinSimple, a Bio-Techne brand, are the only fully automated western blotting solutions for protein detection and characterization, providing results in as little as 3 hours. Cell Signaling Technology (CST) is a leader in the development of antibodies and other related reagents used to elucidate cell signaling pathways that dictate cellular behavior and impact human health. CST has completed assay optimization and validation on the Simple Western platform for a number of their antibodies, making it easier than ever to develop new robust and quantitative immunoassays for the detection and quantification of phospho and total proteins. Researchers who choose CST primary antibodies with Simple Western validation can simply dilute the antibody to the recommended dilution range found on the data sheet and product page. CST antibodies are compatible with the standard Simple Western protocol, which helps users seamlessly incorporate these rigorously validated antibodies into their existing workflows.

"We are excited to partner with Cell Signaling Technology as it combines the strengths of our respective companies to provide researchers with new, more quantitative ways to interrogate signaling pathways," commented Will Geist, President of Bio-Techne’s Protein Sciences Segment. "The assay optimization efforts completed for this additional antibody catalog will help scientists get groundbreaking results even faster."

"Reproducibility has never been more important as more biotherapeutics advance to the clinic. We continue to strive to help scientists understand the disease mechanisms and signaling events responsible, and this partnership will help get the needed data faster. Our partnership with Bio-Techne, and their Simple Western platforms, shows how we can form bridges for our common customers," said Roberto Polakiewicz, Cell Signaling Technology, Chief Scientific Officer.

On February 14, 2023 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, reported financial results for the fourth quarter and full year ended December 31, 2022 (Filing, 8-K, Adaptive Biotechnologies, FEB 14, 2023, View Source [SID1234627162]).

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"We finished the year with 20% revenue growth, driven by both our MRD and Immune Medicine business areas," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "2023 has started strong and I am encouraged to see how momentum is building. We are growing revenue, advancing our pipeline and managing operating expenses with the capital to fuel sustainable growth and execute towards our goals."

Recent Highlights

Revenue of $55.2 million for the fourth quarter and $185.3 million for the full year of 2022, representing a 46% increase and 20% increase over the corresponding periods in 2021, respectively.

clonoSEQ test volume increased 54% to 10,526 tests delivered in the fourth quarter of 2022, compared to the fourth quarter 2021 and ended the year with 36,871 tests delivered, up 51% versus 2021.

Launched clonoSEQ to assess MRD in patients with diffuse large B-cell lymphoma (DLBCL), the most common type of non-Hodgkin’s lymphoma, with Medicare coverage.

Delivered 2 additional TCR data packages to Genentech.

Strengthened our capital position, ending the year with $498.2 million in cash, cash equivalents and marketable securities.

Provided long-term guidance including a 20%-30% revenue CAGR, positive adjusted EBITDA in 2025 and cash flow breakeven in 2026.
Fourth Quarter 2022 Financial Results

Revenue was $55.2 million for the quarter ended December 31, 2022, representing a 46% increase from the fourth quarter in the prior year. Immune Medicine revenue was $27.1 million for the quarter, representing a 27% increase from the fourth quarter in the prior year. MRD revenue was $28.1 million for the quarter, representing a 70% increase from the fourth quarter in the prior year.

Operating expenses were $94.4 million for the fourth quarter of 2022, compared to $99.5 million in the fourth quarter of the prior year, representing a decrease of 5%. Interest expense from our revenue interest purchase agreement was $3.6 million in the fourth quarter of 2022.

Net loss was $40.2 million for the fourth quarter of 2022, compared to $61.4 million for the same period in 2021.

Adjusted EBITDA (non-GAAP) was a loss of $19.6 million for the fourth quarter of 2022, compared to a loss of $44.9 million for the fourth quarter of the prior year.

Full Year 2022 Financial Results

Revenue was $185.3 million for the year ended December 31, 2022, representing a 20% increase from the prior year. Immune Medicine revenue was $98.2 million in 2022, representing an 11% increase from 2021. MRD revenue was $87.1 million in 2022, representing a 32% increase from the prior year.

Operating expenses for 2022 were $385.5 million, compared to $363.3 million for 2021, representing an increase of 6%. Interest expense from our revenue interest purchase agreement was $4.2 million in 2022.

Net loss was $200.4 million in 2022, compared to $207.3 million in 2021.

Adjusted EBITDA (non-GAAP) was a loss of $121.6 million for 2022, compared to a loss of $151.7 million in the prior year.

Cash, cash equivalents and marketable securities was $498.2 million as of December 31, 2022.

2023 Financial Guidance

Adaptive Biotechnologies expects full year 2023 revenue to be in the range of $205 million to $215 million.

We expect operating expenses, including cost of revenue, to be below full year 2022 operating expenses of $385.5 million.

Management will provide further details on the 2023 outlook during the conference call.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its fourth quarter and full year 2022 financial results after market close on Tuesday, February 14, 2023 at 4:30 PM Eastern Time. The conference call can be accessed at View Source The webcast will be archived and available for replay at least 90 days after the event.

On February 14, 2023 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in NASH and viral diseases, reported that the company will deliver several oral and poster presentations at the 32nd Conference of the Asian Pacific Association for the Study of the Liver (APASL), being held Feb 15 – 19 in Taipei, Taiwan at the Taipei International Convention Center (Press release, Aligos Therapeutics, FEB 14, 2023, View Source [SID1234627156]). Aligos will present data for ALG-055009, its THR-β agonist in development for nonalcoholic steatohepatitis (NASH), as well as for several clinical and nonclinical investigational agents from its chronic hepatitis B (CHB) portfolio.

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"At this year’s APASL meeting, we look forward to presenting data from several of our ongoing programs including ALG-055009, our thyroid hormone receptor-beta (THR-β) agonist in development for NASH for which we are conducting Phase 2-enabling activities to enable a Phase 2 filing by the end of this year," said Lawrence Blatt, Ph.D., MBA, CEO and Chairman of the Board at Aligos. "In addition, we are pleased to show exciting data for our CAM-E, ALG-000184, demonstrating notable reductions in HBsAg levels in patients with HBeAg-positive CHB."

Presentation details are as follows. Posters will be available for viewing throughout the conference.

NASH program

Poster presentation

Title: Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) of Single and Multiple Ascending Oral Doses of ALG-055009, a Thyroid Hormone Receptor Beta (THR-β) Agonist for the Treatment of Non-Alcoholic Steatohepatitis (NASH), in Healthy Volunteers and Subjects with Hyperlipidaemia
Presenter: Hakim Charfi, M.D.
Poster number: PH-025

Chronic hepatitis B program

Oral presentations

Title: The Capsid Assembly Modulator ALG-000184 Dosed for 28 Days Was Well Tolerated and Rapidly Reduced Viral Markers in Subjects with Chronic Hepatitis B, Including HBsAg in a Subset of HBeAg Positive Subjects with Elevated Baseline ALT
Presenter: Ed Gane, MBChB, M.D.
Presentation date/time: Thursday, February 16, 14:40 – 14:55 Taipei Standard Time (TST)
Presentation location: 3F North Lounge
Session title: HBV (Clinical) and HCV
Presentation number: FP03-16

Title: ALG-000184, a Capsid Assembly Modulator, Demonstrates Superior Antiviral Activity in Combination with Entecavir Compared to Entecavir in HBeAg Positive Subjects with Chronic Hepatitis B infection
Presenter: Jinlin Hou, M.D.
Presentation date/time: Thursday, February 16, 14:55 – 15:10 TST
Presentation location: 3F North Lounge
Session title: HBV (Clinical) and HCV
Presentation number: FP03-17

Title: A Preclinical Profile of ALG-125755, a GalNAc-siRNA Targeting HBV
Presenter: Jin Hong, Ph.D.
Presentation date/time: Saturday, February 18, 11:35 – 11:50 TST
Presentation location: 3F South Lounge
Session title: HBV (Basic)
Presentation number: FP11-59

Poster presentations

Title: Effect of the Capsid Assembly Modulator (CAM) ALG-000184 on HBsAg Levels in Subjects with HBeAg Positive Chronic Hepatitis B (CHB)
Presenter: Jinlin Hou, Ph.D.
Poster number: PC-019

Title: Preclinical Antiviral, Pharmacological and Toxicological Characteristics of ALG-000184, a Prodrug of the Novel HBV Capsid Assembly Modulator ALG-001075
Presenter: Andreas Jekle, Ph.D.
Poster number: PB-007

Title: Safety, Tolerability and Pharmacokinetics (PK) of Single Ascending Doses of ALG-125755, a GalNAc-Conjugated Small Interfering RNA (siRNA), in Healthy Volunteers (HV)
Presenter: Ed Gane, MBChB, M.D.
Poster number: PPB-043

Title: Discovery of a Liver-Targeted PD-L1 Small Molecule Inhibitor for the Treatment of Chronic Hepatitis B and Liver Cancer
Presenter: Heleen Roose, Ph.D.
Poster number: PB-010Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in NASH and viral diseases, reported that the company will deliver several oral and poster presentations at the 32nd Conference of the Asian Pacific Association for the Study of the Liver (APASL), being held Feb 15 – 19 in Taipei, Taiwan at the Taipei International Convention Center. Aligos will present data for ALG-055009, its THR-β agonist in development for nonalcoholic steatohepatitis (NASH), as well as for several clinical and nonclinical investigational agents from its chronic hepatitis B (CHB) portfolio.

"At this year’s APASL meeting, we look forward to presenting data from several of our ongoing programs including ALG-055009, our thyroid hormone receptor-beta (THR-β) agonist in development for NASH for which we are conducting Phase 2-enabling activities to enable a Phase 2 filing by the end of this year," said Lawrence Blatt, Ph.D., MBA, CEO and Chairman of the Board at Aligos. "In addition, we are pleased to show exciting data for our CAM-E, ALG-000184, demonstrating notable reductions in HBsAg levels in patients with HBeAg-positive CHB."

Presentation details are as follows. Posters will be available for viewing throughout the conference.

NASH program

Poster presentation

Title: Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) of Single and Multiple Ascending Oral Doses of ALG-055009, a Thyroid Hormone Receptor Beta (THR-β) Agonist for the Treatment of Non-Alcoholic Steatohepatitis (NASH), in Healthy Volunteers and Subjects with Hyperlipidaemia
Presenter: Hakim Charfi, M.D.
Poster number: PH-025

Chronic hepatitis B program

Oral presentations

Title: The Capsid Assembly Modulator ALG-000184 Dosed for 28 Days Was Well Tolerated and Rapidly Reduced Viral Markers in Subjects with Chronic Hepatitis B, Including HBsAg in a Subset of HBeAg Positive Subjects with Elevated Baseline ALT
Presenter: Ed Gane, MBChB, M.D.
Presentation date/time: Thursday, February 16, 14:40 – 14:55 Taipei Standard Time (TST)
Presentation location: 3F North Lounge
Session title: HBV (Clinical) and HCV
Presentation number: FP03-16

Title: ALG-000184, a Capsid Assembly Modulator, Demonstrates Superior Antiviral Activity in Combination with Entecavir Compared to Entecavir in HBeAg Positive Subjects with Chronic Hepatitis B infection
Presenter: Jinlin Hou, M.D.
Presentation date/time: Thursday, February 16, 14:55 – 15:10 TST
Presentation location: 3F North Lounge
Session title: HBV (Clinical) and HCV
Presentation number: FP03-17

Title: A Preclinical Profile of ALG-125755, a GalNAc-siRNA Targeting HBV
Presenter: Jin Hong, Ph.D.
Presentation date/time: Saturday, February 18, 11:35 – 11:50 TST
Presentation location: 3F South Lounge
Session title: HBV (Basic)
Presentation number: FP11-59

Poster presentations

Title: Effect of the Capsid Assembly Modulator (CAM) ALG-000184 on HBsAg Levels in Subjects with HBeAg Positive Chronic Hepatitis B (CHB)
Presenter: Jinlin Hou, Ph.D.
Poster number: PC-019

Title: Preclinical Antiviral, Pharmacological and Toxicological Characteristics of ALG-000184, a Prodrug of the Novel HBV Capsid Assembly Modulator ALG-001075
Presenter: Andreas Jekle, Ph.D.
Poster number: PB-007

Title: Safety, Tolerability and Pharmacokinetics (PK) of Single Ascending Doses of ALG-125755, a GalNAc-Conjugated Small Interfering RNA (siRNA), in Healthy Volunteers (HV)
Presenter: Ed Gane, MBChB, M.D.
Poster number: PPB-043

Title: Discovery of a Liver-Targeted PD-L1 Small Molecule Inhibitor for the Treatment of Chronic Hepatitis B and Liver Cancer
Presenter: Heleen Roose, Ph.D.
Poster number: PB-010

On February 14, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported favourable imaging data from its Phase I diagnostic trial of 64Cu SAR-bisPSMA in prostate cancer, PROPELLER (NCT 048393671)1. This follows the announcement of top line data in December 2022 (Press release, Clarity Pharmaceuticals, FEB 14, 2023, View Source [SID1234627155]).

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This data is currently being presented via a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Symposium in San Francisco. The poster will be available on Clarity’s website once released at ASCO (Free ASCO Whitepaper) GU.

The PROPELLER trial evaluated 30 patients with confirmed prostate cancer prior to undergoing radical prostatectomy (surgical removal of the prostate) and lymph node dissection (removal). In addition to the primary (safety, tolerability, imaging efficacy) and secondary (determining the optimal dose for subsequent investigation) endpoints, the study also compared the diagnostic properties of Clarity’s 64Cu SAR-bisPSMA product to 68Ga PSMA-11, an approved standard-of-care (SOC) product for prostate cancer imaging in Australia and the US, as an exploratory objective.

The comparison evaluated prostate cancer detection and the intensity of product uptake within the same lesions (the higher the uptake, the brighter and more visible the lesion appears on the scan). The uptake of the products was measured by maximum Standardised Uptake Values (SUVmax) on the PET scans. Importantly, all scans were evaluated by two independent, blinded, central readers.

Clarity’s Executive Chairman, Dr Alan Taylor, commented, "64Cu SAR-bisPSMA detected prostate cancer lesions that are more defined and brighter on the scans than the current SOC product, 68Ga PSMA-11. This may enable detection of smaller lesions that would have otherwise gone undetected. Arming clinicians with more accurate diagnostic information helps them determine the best course of treatment for their patients and may sometimes make the difference between the removal of the prostate, a severe and life-changing surgery, and other options that may be more effective in treating the patient’s cancer while enabling better quality of life post treatment. This patient-centric approach, reinforced by the flexibility in the timing of the scan, centralised product manufacture and its broad distribution to any imaging centre with a PET camera, would be a true paradigm shift in the management of prostate cancer. We are continuing to work diligently towards the start of the diagnostic Phase III trial of SAR-bisPSMA in the US in order to make this product available to the patients who need it most."

Prof Louise Emmett, (St Vincent’s Hospital Sydney), Principal Investigator in the PROPELLER trial, commented, "It is very encouraging to see such positive imaging results from 64Cu SAR-bisPSMA, an agent we are very pleased to work with. The higher SUVmax was consistent across both independent, blinded, central readers, and, importantly, as shown on the images on the poster, 64Cu SAR-bisPSMA was able to detect disease outside of the primary lesion that was not detected with 68Ga PSMA-11. This is a very important result when it comes to patient management, and we are looking forward to further exploring these findings as we aim to better understand the benefits of 64Cu SAR-bisPSMA during the Phase III trial. With the high uptake of the product in tumours as well as the additional benefits of later imaging timepoints, we will continue to be involved in SAR-bisPSMA trials with the ultimate purpose of improving outcomes for our patients."

Results
64Cu SAR-bisPSMA was shown to be safe and well tolerated across all patients, with only 1 patient in 30 reporting a metallic taste that was mild in nature (Grade 1).

A dose of 200 MBq was determined to be the optimal dose compared to other dose levels. All trials with 64Cu SAR-bisPSMA, both currently and in the future, will be undertaken at this dose level.

64Cu SAR-bisPSMA reported higher SUVmax values compared to 68Ga PSMA-11 in the 200 MBq dose cohort (n=18), according to both readers. Images from two patients comparing the 64Cu SAR-bisPSMA and 68Ga PSMA-11 scans are depicted below in Figure 1.

In this cohort, Reader 1 was able to detect primary prostate cancer in 100% of patients when 64Cu SAR-bisPSMA was used, while 68Ga PSMA-11 showed the cancer in 77.8% of patients. Similarly, Reader 2 detected primary prostate cancer in 85.7% of patients using 64Cu SAR-bisPSMA and in 83.3% of patients when using 68Ga PSMA-11.

In one patient, secondary disease was detected by 64Cu SAR-bisPSMA in a pelvic lymph node that was not detected by 68Ga PSMA-11 (see Figure 2). The lymph node was subsequently determined to be positive for prostate cancer by histopathology.