EMA

Draft revised consolidated 3-year work plan for the Methodology Working Party (MWP)

Retrieved on: 
Wednesday, February 14, 2024

Industry level .................................................................................................. 13

Key Points: 
    • Industry level .................................................................................................. 13

      Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 2/14

      1.

    • A reflection paper
      (RP) on the clinical pharmacology package for oligonucleotides is a prioritised activity in the MWP work

      Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 3/14

      plan, and it is envisaged that something similar may be needed for other emerging treatment
      modalities (e.g., peptides).

    • Guideline work led by other working parties
      ?

      Revision of the guideline on the requirements for clinical documentation for orally inhaled
      products (CPMP/EWP/4151/00 Rev.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 4/14

      The planned concept papers (CPs) will formulate problem statements for potential workshops and
      subsequent guidance documents will be informed and enriched by the outcome of discussions of
      workshops to be held in 2024.

    • Guideline work led by other working parties and committees
      ?

      Revision of Guidance on the investigation of medicinal products in the term and preterm
      neonate (EMEA/536810/2008).

    • There is a need for
      Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 5/14

      new guidance in these areas to ensure these novel approaches meet the required evidentiary
      standards and facilitate their evaluation.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 6/14

      ?

      Revision of Guideline on clinical evaluation of diagnostic agents (CPMP/EWP/1119/98/Rev.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 7/14

      ?

      Provide appropriate support to the EU network for generic and hybrid medicines including
      product-specific requirements.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 8/14

      2.3.

    • ?

      Cross disciplinary work with Quality Working Party and other stakeholders on physiologically
      based biopharmaceutics modelling (PBBM).

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 9/14

      ?

      In order to support adequate evaluation of all methodology MWP will aim to facilitate an
      increase in presence and visibility in relevant committees of methodological expertise from
      across the EU network such as CHMP, PRAC, PDCO, CMD(h), ETF and CAT.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 10/14

      ?

      Product Specific Bioequivalence Guidelines (PSBGLs) (multiple) in liaison with CMD(h): for
      2024, azacitidine, budesonide (LALA GIT), trametinib, dabrafenib, paliperidone palmitate (3M
      depot) and melatonin have been prioritised as the next in series for drafting.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 11/14

      4.2.

    • ?

      Cross disciplinary work with Quality Working Party and other stakeholders on PBBM model
      assessment.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 12/14

      ?

      To deliver an improved access to raw data (e.g.

    • ?

      Propose regulatory research priorities for funders in across the activities of Methodology
      Working Party, including in the big data area.

    • ?

      Establish key communication points in national competent authorities and build a resource of
      key messages and communication materials on regulation and methodology.

    • The timing of workshops may need to be arranged according to the

      Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 13/14

      specific needs of the guidance ? either before the guidance is finalised to gather views and
      expertise; or once it is finalised for training purposes.

    • Revised consolidated 3-year work plan for the Methodology Working Party (MWP)
      EMA/CHMP/58124/2023

      Page 14/14

Blacksmith Medicines and Zoetis Announce Research Collaboration for Novel Animal Health Antibiotics

Retrieved on: 
Wednesday, February 14, 2024

SAN DIEGO, Feb. 14, 2024 /PRNewswire/ -- Blacksmith Medicines, Inc. (Blacksmith), a leading biopharma dedicated to discovering and developing medicines targeting metalloenzymes, and Zoetis, the world's leading animal health company, announced today a collaboration to discover and develop novel antibiotics for animal health.

Key Points: 
  • SAN DIEGO, Feb. 14, 2024 /PRNewswire/ -- Blacksmith Medicines, Inc. (Blacksmith), a leading biopharma dedicated to discovering and developing medicines targeting metalloenzymes, and Zoetis, the world's leading animal health company, announced today a collaboration to discover and develop novel antibiotics for animal health.
  • Blacksmith and Zoetis will collaborate to discover and develop novel antibiotics to selectively target bacteria for animal health," said Zachary Zimmerman, Ph.D., CEO and co-founder of Blacksmith.
  • "As part of our commitment to reduce the dependency on antibiotic classes shared with human health, we are pleased to leverage our veterinary expertise along with our extensive collection of pathogens in this collaboration with Blacksmith," said Dr. Jeff Watts, Research Director, External Innovation at Zoetis.
  • "Through our research, we aim to ultimately provide new options for our customers to treat life-threatening infections in livestock."

Reflection paper on investigation of pharmacokinetics in the obese population - Scientific guideline

Retrieved on: 
Wednesday, February 14, 2024

Reflection paper on investigations of pharmacokinetics in

Key Points: 
    • Reflection paper on investigations of pharmacokinetics in
      the obese population
      Table of contents
      1.
    • References .............................................................................................. 9

      Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 2/12

      1.

    • This is considered
      a shortcoming that is potentially compounded by obese patients often being poorly represented in
      clinical studies.
    • The specific aims of this reflection paper are to:
      ?

      describe how the effects of obesity can be investigated during clinical medicinal product
      development.

    • ?

      provide recommendations on when investigations of the effect of obesity on the PK of a
      medicinal product should be particularly considered.

    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 3/12

      ?

      discuss how to reflect PK (and/or PK/PD) findings in weight/weight-based dosing
      recommendations.

    • Absorption
      Reduced rate of absorption linked to locally reduced blood flow (8) is reported for the subcutaneous
      and transdermal routes in obese subjects.
    • Distribution
      The distribution of medicinal products is driven by body composition, regional blood flow and binding to
      tissue and plasma proteins.
    • Obese subjects have a larger absolute lean body weight (LBW) as well as fat mass.
    • The physicochemical properties of a medicinal product (lipophilicity, polarity, molecular size, and
      degree of ionization) influence its distribution in the body.
    • In BMI class III obese
      subjects, the blood flow per gram of fat is significantly lower than that observed in class I obese or
      lean subjects (4).
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 4/12

      An increased amount of alpha-1-acid-glycoprotein (AAG), linked to a chronic inflammatory state, is
      reported in obese individuals.

    • Fatty infiltrations are present in the liver for 90% of obese subjects, with the extent of the infiltrations
      being proportional to the degree of obesity.
    • In some cases, in particular for CYP3A4 metabolized medicinal products,
      bodyweight normalized clearance can be lower in obese patients (23).
    • Based on presently available data, it has been suggested that uptake transporters

      Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 5/12

      are downregulated while efflux transporters may be upregulated (31).

    • Platelet hyper-reactivity is also observed,
      which can impair the response to anti-platelet medicinal products in obese patients (42, 43).
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 6/12

      3.

      the medicinal product properties and scientific literature indicate that obesity may lead to a
      marked effect on elimination and/or distribution or on the PK/PD relationship.

    • These
      models may aid in extrapolating the known efficacy and safety in the non-obese population to the
      obese population.
    • The Pharmacokinetics of the CYP3A Substrate Midazolam in Morbidly Obese Patients
      Before and One Year After Bariatric Surgery.
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 11/12

      41.

    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 12/12

Medicenna Therapeutics Reports Third Quarter Fiscal 2024 Financial Results and Corporate Update

Retrieved on: 
Wednesday, February 14, 2024

Today, the Company reports promising clinical data from the on-going monotherapy escalation and expansion arms of the ABILITY-1 study.

Key Points: 
  • Today, the Company reports promising clinical data from the on-going monotherapy escalation and expansion arms of the ABILITY-1 study.
  • Clinical update from the combination arm of the ABILITY-1 study evaluating MDNA11 in with KEYTRUDA® expected in H1 and H2 of 2024.
  • Research and development expenses of $3.0 million were incurred during the quarter ended December 31, 2023, compared with $2.9 million incurred in the quarter ended December 31, 2022.
  • General and administrative expenses of $1.8 million were incurred during the quarter ended December 31, 2023, compared with $2.0 million during the quarter ended December 31, 2022.

Outlook Therapeutics® Reports Financial Results for First Quarter Fiscal Year 2024 and Provides Corporate Update

Retrieved on: 
Wednesday, February 14, 2024

ISELIN, N.J., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company working to achieve regulatory approval for the first ophthalmic formulation of bevacizumab for the treatment of retinal diseases, today announced financial results for first quarter fiscal year 2024 and provided a corporate update.

Key Points: 
  • ISELIN, N.J., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company working to achieve regulatory approval for the first ophthalmic formulation of bevacizumab for the treatment of retinal diseases, today announced financial results for first quarter fiscal year 2024 and provided a corporate update.
  • Importantly, the first subject was enrolled in NORSE EIGHT in January 2024,” commented Russell Trenary, President and Chief Executive Officer.
  • In January 2024, Outlook Therapeutics announced that it received written agreement on the NORSE EIGHT trial protocol from the FDA under a SPA for NORSE EIGHT.
  • Outlook Therapeutics expects NORSE EIGHT topline results and resubmission of the ONS-5010 BLA by the end of calendar year 2024.

Sage Therapeutics Announces Fourth Quarter and Full Year 2023 Financial Results and Highlights Pipeline and Business Progress

Retrieved on: 
Wednesday, February 14, 2024

Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today reported business highlights and financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today reported business highlights and financial results for the fourth quarter and full year ended December 31, 2023.
  • Revenue: Collaboration revenue from sales of ZURZUVAE was $0.8 million in the fourth quarter of 2023, the first quarter of sales.
  • Net Loss: Net loss was $32.7 million for the fourth quarter of 2023 compared to $147.1 million for the same period in 2022.
  • ET to review its fourth quarter and full year 2023 financial results and discuss recent corporate updates.

JumpCloud's Q1 2024 SME IT Trends Report Reveals AI Optimism Tempered by Security Concerns

Retrieved on: 
Wednesday, February 14, 2024

LOUISVILLE, Colo., Feb. 14, 2024 (GLOBE NEWSWIRE) -- JumpCloud Inc. , today announced the findings from its Q1 2024 small to medium-sized enterprise (SME) IT Trends Report, “ State of IT 2024: The Rise of AI, Economic Uncertainty, and Evolving Security Threats ”.

Key Points: 
  • LOUISVILLE, Colo., Feb. 14, 2024 (GLOBE NEWSWIRE) -- JumpCloud Inc. , today announced the findings from its Q1 2024 small to medium-sized enterprise (SME) IT Trends Report, “ State of IT 2024: The Rise of AI, Economic Uncertainty, and Evolving Security Threats ”.
  • The report provides updated survey results and new findings to the company’s biannual SME IT Trends Report, which was first released in June 2021.
  • But IT admins report notable concerns around their organizations’ current ability to secure against related threats—and personal concerns about AI’s impact on their career.
  • The findings from the JumpCloud Q1 2024 SME IT Trends Report can be found in “State of IT 2024: The Rise of AI, Economic Uncertainty, and Evolving Security Threats,” here .

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

Retrieved on: 
Wednesday, February 14, 2024

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Orphan medicines development - ask the European regulator, Online, from 29 February 2024, 16:00 (CET) to 29 February 2024, 17:30 (CET)

Retrieved on: 
Wednesday, February 14, 2024

Orphan medicines development - ask the European regulator

Key Points: 
  • Orphan medicines development - ask the European regulator
    EventHumanMedicines
    Date
    Location
    The EMA’s Orphan Medicines Office is hosting an interactive webinar Rare Disease Day on 29 February 2024, to answer questions that developers of products for rare diseases or researchers in this field might have on orphan designations and rare disease development.
  • The webinar will feature:
    The event is directed towards healthcare professionals, academia, industry representatives and patients and requires registration to enable active participation.
  • The event will be also broadcast and recorded.
  • The videos and presentations will be made available on the EMA website after the event.

Technical Webinar: Regulatory Procedure Management for PLM in IRIS for Network Users, Online, European Medicines Agency, Amsterdam, the Netherlands, from 27 February 2024, 14:00 (CET) to 27 February 2024, 16:00 (CET)

Retrieved on: 
Wednesday, February 14, 2024

Date

Key Points: 
  • Date
    - Tuesday, 27 February 2024, 14:00 (CET) - 16:00 (CET)
    Location
    - OnlineEuropean Medicines Agency, Amsterdam, the Netherlands
    Event summary
    EMA is currently working on the onboarding of Regulatory Procedure Management (RPM) for Product Lifecycle Management (PLM) for medicinal products on IRIS.
  • The 1st roll-out occurred on 23 January 2024, covering Variations, Article 61.3 Notifications, and Marketing Authorisation Transfers procedures for a subset of both human and veterinary medicinal products.
  • IRIS Network users keen to know more about the technical aspects of the RPM for PLM transition to IRIS and seek specific answers to their technical queries are invited to join the RPM Technical webinar.
  • Registration
    The registration is open via this link:
    Technical Webinar: Regulatory Procedure Management for PLM in IRIS for Network Users
    Video recording
    A video recording will be made available after the event.