Spectrochimica Acta Part B

Scholar Rock Presents New Data from Phase 1 DRAGON Trial Showing Promising Anti-Tumor Activity in Anti-PD-1 Resistant Metastatic ccRCC Patients and Supporting SRK-181 Continued Tolerability

Retrieved on: 
Friday, November 3, 2023

The second poster focuses on preliminary biomarker data from part B of the trial in patients with multiple tumor types.

Key Points: 
  • The second poster focuses on preliminary biomarker data from part B of the trial in patients with multiple tumor types.
  • Data presented continues to support proof of concept for SRK-181 in 28 heavily pretreated patients with ccRCC resistant to anti-PD-1.
  • In the biomarker analysis for ccRCC, levels of circulating granulocytic myeloid-derived suppressor cells (gMDSC) correlated with clinical activity in ccRCC patients treated with SRK-181 in combination with pembrolizumab.
  • “The DRAGON trial has successfully delivered on its objective of demonstrating proof of concept for SRK-181 by showing promising anti-tumor activity.

Y-mAbs Therapeutics Announces U.S. FDA Clearance of Investigational New Drug Application for CD38-SADA

Retrieved on: 
Tuesday, October 17, 2023

The primary study outcome will evaluate safety and initial signals of efficacy using repeated dosing.

Key Points: 
  • The primary study outcome will evaluate safety and initial signals of efficacy using repeated dosing.
  • Y-mAbs expects a total of approximately 30 patients and up to 12 U.S. sites to be included in the trial.
  • The SADA technology platform utilizes a pre-targeted payload delivery method where antibody constructs assemble in tetramers and bind to the tumor target.
  • Unbound constructs predictably disassemble into smaller antibody fragments and are predominantly excreted through the kidneys within hours after administration.

ACELYRIN, INC. Announces Top-Line Results from Placebo-Controlled Clinical Trial of Izokibep for Moderate-to-Severe Hidradenitis Suppurativa

Retrieved on: 
Monday, September 11, 2023

HiSCR75 did meet statistical significance at week 16 in a Last Observation Carried Forward sensitivity analysis.

Key Points: 
  • HiSCR75 did meet statistical significance at week 16 in a Last Observation Carried Forward sensitivity analysis.
  • Response was dose ordered, and safety was consistent with prior izokibep experience and not dose-limiting.
  • Izokibep appears to be demonstrating consistent early and high orders of response without safety or tolerability limitation.
  • The trial was conducted at 50 sites globally and assessed various efficacy endpoints, including the primary endpoint of HiSCR75 (Hidradenitis Suppurativa Clinical Response) at 16 weeks utilizing a non-responder imputation (NRI) analysis method.

Vir Biotechnology Presents New Data Evaluating the Potential for VIR-2218 and VIR-3434 as Therapies for Chronic Hepatitis B and Hepatitis D

Retrieved on: 
Saturday, June 24, 2023

SAN FRANCISCO, June 24, 2023 (GLOBE NEWSWIRE) -- Vir Biotechnology, Inc. (Nasdaq: VIR) today announced new data from its robust hepatitis B and D virus (HBV and HDV) portfolio that were presented at the EASL™ (European Association for the Study of the Liver) Congress.

Key Points: 
  • These data further support the clinical development of these investigational medicines for the treatment of HDV.
  • “I am very excited by the progress we are making toward our goal of achieving HBV functional cure.
  • Our data to-date with VIR-2218 and PEG-IFN-⍺ support our hypothesis of using a cocktail of antivirals combined with immunomodulators.
  • The majority of adverse events were consistent with the known effects of PEG-IFN-⍺ and resolved after the end of treatment.

Stepwell for School Districts Now Available to Improve Special Education Outcomes

Retrieved on: 
Tuesday, June 13, 2023

OKEMOS, Mich., June 13, 2023 /PRNewswire-PRWeb/ -- Stepwell, a collaborative web-based platform providing visibility into special education compliance and monitoring for results, is now available to school districts. The district-level solution will help education leaders plan and implement systemic changes to achieve better outcomes for students with an Individualized Education Plan (IEP) and ensure compliance with the Individuals with Disabilities Education Act (IDEA).

Key Points: 
  • Designed to make IDEA compliance easier, Stepwell helps school district leaders drive districtwide improvement and ensure results-driven accountability
    OKEMOS, Mich., June 13, 2023 /PRNewswire-PRWeb/ -- Stepwell , a collaborative web-based platform providing visibility into special education compliance and monitoring for results, is now available to school districts.
  • The district-level solution will help education leaders plan and implement systemic changes to achieve better outcomes for students with an Individualized Education Plan (IEP) and ensure compliance with the Individuals with Disabilities Education Act (IDEA).
  • Stepwell streamlines relevant information and workflows so that education leaders can focus on system-wide improvement and overcome cumbersome tasks associated with compliance management.
  • Together, Stepwell provides the structure and support education leaders need to monitor their special education systems in place—and shift the focus from compliance to quality special education districtwide.

Alnylam and Regeneron Report Positive Interim Phase 1 Clinical Data on ALN-APP, an Investigational RNAi Therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy

Retrieved on: 
Wednesday, April 26, 2023

Twenty patients have been enrolled in three single-dose cohorts in Part A of the ongoing Phase 1 study in patients with early-onset Alzheimer’s disease.

Key Points: 
  • Twenty patients have been enrolled in three single-dose cohorts in Part A of the ongoing Phase 1 study in patients with early-onset Alzheimer’s disease.
  • All adverse events were mild or moderate in severity, with available cerebrospinal fluid data for white blood cells and protein appearing similar to placebo.
  • Early data for neurofilament light chain from a subset of cohorts (2 of 3 studied) looked comparable to placebo.
  • A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events .

Regeneron and Alnylam Report Positive Interim Phase 1 Clinical Data on ALN-APP, an Investigational RNAi Therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy

Retrieved on: 
Wednesday, April 26, 2023

TARRYTOWN, N.Y. and CAMBRIDGE, Mass., April 26, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (Nasdaq: REGN) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA).

Key Points: 
  • Twenty patients have been enrolled in three single-dose cohorts in Part A of the ongoing Phase 1 study in patients with early-onset Alzheimer’s disease.
  • In this study to date, single doses of ALN-APP, which are administered by intrathecal injection, have been well tolerated.
  • All adverse events were mild or moderate in severity, with available cerebrospinal fluid data for white blood cells and protein appearing similar to placebo.
  • Early data for neurofilament light chain from a subset of cohorts (2 of 3 studied) looked comparable to placebo.

Caribou Biosciences Reports CB-010 ANTLER Phase 1 Trial Progress

Retrieved on: 
Monday, December 12, 2022

BERKELEY, Calif., Dec. 12, 2022 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today reported new 12-month clinical data from cohort 1 in the ongoing ANTLER Phase 1 trial, which show long-term durability following a single infusion of CB-010 at the initial dose level 1 (40x106 CAR-T cells). Cohort 1 results show:

Key Points: 
  • Today at the 64th Annual ASH meeting, a trial-in-progress poster is being presented to provide details of the design and objectives of the ANTLER Phase 1 trial for CB-010 in r/r B-NHL.
  • Caribou Biosciences is a clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases.
  • “Caribou Biosciences” and the Caribou logo are registered trademarks of Caribou Biosciences, Inc.
  • Except as required by law, Caribou undertakes no obligation to update publicly any forward-looking statements for any reason.

Scholar Rock Reports Third Quarter 2022 Financial Results and Highlights Business Progress

Retrieved on: 
Monday, November 14, 2022

I am excited to strengthen our experienced executive team with the addition of Scholar Rocks new CMO, Dr. Jing Marantz.

Key Points: 
  • I am excited to strengthen our experienced executive team with the addition of Scholar Rocks new CMO, Dr. Jing Marantz.
  • Revenue was $0 for the quarter ended September 30, 2022, compared to $5.5 million for the quarter ended September 30, 2021.
  • General and administrative expense was $10.5 million for the quarter ended September 30, 2022, compared to $11.3 million for the quarter ended September 30, 2021.
  • Scholar Rock is a registered trademark of Scholar Rock, Inc.

Atossa Therapeutics Announces Third Quarter 2022 Financial Results and Provides Corporate Update

Retrieved on: 
Monday, November 7, 2022

SEATTLE, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Atossa Therapeutics, Inc. (Nasdaq: ATOS), today announces financial results for the fiscal quarter ended September 30, 2022, and provides an update on recent company developments.

Key Points: 
  • SEATTLE, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Atossa Therapeutics, Inc. (Nasdaq: ATOS), today announces financial results for the fiscal quarter ended September 30, 2022, and provides an update on recent company developments.
  • As of September 30, 2022, the Company had cash, cash equivalents and restricted cash of approximately $117,477.
  • Nine Months Ended September 30, 2022 Financial Results (in thousands):
    Revenue and Cost of Revenue: For the nine months ended September30, 2022and 2021, we hadno source ofrevenue and no associated cost of revenue.
  • Operating expenses for the nine months ended September 30, 2022 consisted of R&D expenses of $10,097and G&Aexpenses of $9,456.