Oncology Drug Advisory Committee

Panbela Provides Business Update and Reports Q3 2023 Financial Results

Retrieved on: 
Thursday, November 9, 2023

MINNEAPOLIS, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today provides a business update and reports financial results for the quarter ended September 30, 2023.

Key Points: 
  • MINNEAPOLIS, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today provides a business update and reports financial results for the quarter ended September 30, 2023.
  • The independent Data Safety Monitoring Board (DSMB) of the ASPIRE trial completed its pre-specified review of safety data for treated patients in the trial.
  • The aggregate gross proceeds from the exercise of the existing warrants totaled approximately $1.9 million, before deducting financial advisory fees.
  • Third Quarter ended September 30, 2023 Financial Results
    General and administrative expenses were $1.1 million in the third quarter of 2023, compared to $1.3 million in the third quarter of 2022.

Abecma Delivers Sustained Progression-Free Survival Versus Standard Regimens in Earlier Lines of Therapy for Relapsed and Refractory Multiple Myeloma Based on Longer-Term Follow-up from KarMMa-3

Retrieved on: 
Tuesday, December 12, 2023

With extended follow-up, treatment with Abecma (n=254) continued to demonstrate higher overall response rates (ORR) and a deepening of responses versus standard regimens.

Key Points: 
  • With extended follow-up, treatment with Abecma (n=254) continued to demonstrate higher overall response rates (ORR) and a deepening of responses versus standard regimens.
  • Due to the median PFS observed with standard regimens, more than half (56%) of patients in the standard regimens arm crossed over to receive Abecma as a subsequent therapy.
  • Historically, based on real-world evidence, median OS for patients with triple-class exposed relapsed and refractory multiple myeloma is approximately 13 months.
  • Regulatory applications for Abecma in earlier lines of therapy for triple-class exposed relapsed and refractory multiple myeloma are also under review with the European Medicines Agency and Swissmedic.

Bristol Myers Squibb and 2seventy bio Provide Update on U.S. FDA Review of sBLA for Abecma (idecabtagene vicleucel) in Earlier Lines of Therapy for Triple-Class Exposed Relapsed or Refractory Multiple Myeloma

Retrieved on: 
Monday, November 20, 2023

The date of the ODAC meeting has not yet been confirmed by the FDA.

Key Points: 
  • The date of the ODAC meeting has not yet been confirmed by the FDA.
  • The FDA also informed the companies that a decision on the application will not be made by the Prescription Drug User Fee Act (PDUFA) target action date of December 16, 2023.
  • The companies anticipate that the committee will review data related to the secondary endpoint of overall survival (OS).
  • The ODAC meeting has no impact on the currently approved indication for Abecma for adult patients with triple-class exposed RRMM after four or more prior lines of therapy.

Stupid Strong Charitable Foundation Pledges $450,000 Grant to Advance Gastric Cancer Family Study

Retrieved on: 
Wednesday, November 1, 2023

This grant will propel the "Cancer Patients' Family Matters" study led by Dr. Jaffer Ajani, aimed at unraveling the mysteries behind the hereditary nature of gastric cancer and developing strategies for early detection and prevention.

Key Points: 
  • This grant will propel the "Cancer Patients' Family Matters" study led by Dr. Jaffer Ajani, aimed at unraveling the mysteries behind the hereditary nature of gastric cancer and developing strategies for early detection and prevention.
  • The research, supported by Stupid Strong, will focus on enrolling gastric cancer patients and their family members, totaling 500 patients and 5,000 individuals overall.
  • This generous grant from the Stupid Strong Charitable Foundation will enable Dr. Ajani to hire staff, identify study participants, administer detailed lifestyle questionnaires, and collect and process blood samples.
  • For more information about the Stupid Strong Charitable Foundation, please visit www.stupidstrong.org .

Alnylam Stock Trading Halted Today; FDA Advisory Committee to Review Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Wednesday, September 13, 2023

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that NASDAQ has halted trading of the Company’s common stock.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that NASDAQ has halted trading of the Company’s common stock.
  • The U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee is meeting today to review the supplemental New Drug Application for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
  • As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.
  • Patisiran is the established name for ONPATTRO®, which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.

Ajax Therapeutics Appoints Dr. David Steensma as Chief Medical Officer

Retrieved on: 
Wednesday, August 23, 2023

Ajax Therapeutics, Inc., a biopharmaceutical company applying computational chemistry and structure-based technologies to develop next generation JAK inhibitors for patients with myeloproliferative neoplasms (MPNs), today announced the appointment of David P. Steensma, MD, FACP, as Chief Medical Officer.

Key Points: 
  • Ajax Therapeutics, Inc., a biopharmaceutical company applying computational chemistry and structure-based technologies to develop next generation JAK inhibitors for patients with myeloproliferative neoplasms (MPNs), today announced the appointment of David P. Steensma, MD, FACP, as Chief Medical Officer.
  • View the full release here: https://www.businesswire.com/news/home/20230823046723/en/
    Ajax Therapeutics CMO David Steensma, MD, FACP (Photo: Business Wire)
    “We are very fortunate to have such a veteran hematology drug developer as David join the Ajax team as we near the clinic with our next generation Type II JAK2 inhibitor program for MPNs,” said Martin Vogelbaum, CEO of Ajax Therapeutics.
  • Dr. Steensma has published over 200 original research papers as well as numerous reviews, editorials and book chapters.
  • He received his medical degree from the University of Chicago’s Pritzker School of Medicine.

AMGEN TO DISCUSS APPLICATION FOR LUMAKRAS® (SOTORASIB) FOR THE TREATMENT OF KRAS G12C-POSITIVE NSCLC AT FDA ADVISORY COMMITTEE MEETING

Retrieved on: 
Monday, August 21, 2023

LUMAKRAS/LUMYKRAS is approved in several markets outside the United States including Europe, South America, Asia and the European Union.

Key Points: 
  • LUMAKRAS/LUMYKRAS is approved in several markets outside the United States including Europe, South America, Asia and the European Union.
  • To date, over 6,500 patients around the world have received LUMAKRAS/LUMYKRAS through the clinical development program and commercial use.
  • Lumakras has demonstrated a favorable benefit/risk profile in multiple studies in non-small lung cancer and other tumor types such as colo-rectal cancer.
  • Amgen is progressing the largest and broadest global KRASG12C inhibitor development program exploring multiple combination regimens, with clinical trial sites spanning five continents.

Mesoblast Receives Complete Response From U.S. Food and Drug Administration for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in Children

Retrieved on: 
Friday, August 4, 2023

To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality.

Key Points: 
  • To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality.
  • This adult study is in line with our overall commercial strategy, which envisioned a sequenced progression from pediatric to adult SR-aGVHD indications.
  • Mesoblast has successfully met the pre-specified primary endpoint, prospectively agreed with FDA, of a single-arm Phase 3 trial in 54 children with SR-aGvHD.
  • It can be accessed via: https://webcast.openbriefing.com/msb-mu-2023/
    The archived webcast will be available on the Investor page of the Company’s website: www.mesoblast.com

FDA ADVISORY COMMITTEE UNANIMOUSLY CONFIRMS EFFICACY AND CLINICAL BENEFIT OF LEQEMBI FOR EARLY ALZHEIMER'S DISEASE

Retrieved on: 
Friday, June 9, 2023

"The Alzheimer's Association welcomes and celebrates this action by the FDA Advisory Committee to support traditional approval of Leqembi for people with early Alzheimer's," said Joanne Pike, DrPH, Alzheimer's Association president and CEO.

Key Points: 
  • "The Alzheimer's Association welcomes and celebrates this action by the FDA Advisory Committee to support traditional approval of Leqembi for people with early Alzheimer's," said Joanne Pike, DrPH, Alzheimer's Association president and CEO.
  • The Alzheimer's Association and members of the Association's Early Stage Advisory Group each submitted comments urging the Advisory Committee to recommend traditional approval of Leqembi, which was granted accelerated approval in January 2023.
  • "We are in full agreement with the FDA Advisory Committee that Leqembi provides clinical benefit and that this benefit outweighs the risks.
  • Leqembi, an anti-amyloid treatment, changes the course of Alzheimer's and delivers clear clinical benefit for people in the early stages of the disease.

Health Canada Accepts New Drug Submission for Lecanemab as Treatment for Early Alzheimer's Disease

Retrieved on: 
Tuesday, May 16, 2023

Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.

Key Points: 
  • Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.
  • As such, lecanemab may have the potential to have an effect on disease pathology and to slow down the progression of the disease.
  • The Clarity AD study of lecanemab met its primary endpoint and all key secondary endpoints with highly statistically significant results.
  • Lecanemab was approved under the accelerated approval pathway in the U.S. and was launched in the U.S. on January 18, 2023.