Drug Target Review

JULY 1, 2024

SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. He is spearheading the scientific and clinical development and fundraising of South Rampart Pharma and technology development efforts.

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The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. The full range of data from Roche’s clinical development programme in neuroscience being presented at 2021 AAN include: Medicine and/or Therapeutic Area.

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The Pharma Data

OCTOBER 18, 2020

Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML.

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The Pharma Data

OCTOBER 25, 2020

In addition, this indication offers synergy with the commercial infrastructure being developed to support our first New Drug Application.”. This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995.

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The Pharma Data

DECEMBER 29, 2020

” The Phase III clinical development program consists of three studies, the BRIDGE study, the BALANCE study and the BRIGHT study. Patients had the option to receive PRX-102 infusions in a home care setting based on infusion tolerability and country regulation. About Fabry Disease. Galactosidase-A enzyme.

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The Pharma Data

DECEMBER 15, 2020

The researchers regulated the abnormal immunological memory processes found in these patients. Treatment induced sustainable clinical responses and reduced systemic inflammation. The secondary outcome involves pharmacokinetic endpoints. The human monoclonal antibody targets specific immune plasma cells. Bioelectronic Platform.

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The Pharma Data

APRIL 11, 2023

Bayer will present the first clinical Phase 1 results on aryl hydrocarbon receptor (AhR) inhibitor BAY 2416964, the company’s most advanced Immuno-Oncology program. The company has the passion and determination to develop innovative medicines that help improve and extend the lives of people living with cancer.

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Metabolite Tales Blog

APRIL 4, 2023

Tapinarof is a topical aryl hydrocarbon receptor–modulating agent approved for the treatment of psoriasis by downregulating proinflammatory interleukin-17 and regulating expression of the skin-barrier proteins filaggrin and loricrin. Dermavant’s tapinarof is one such friend. 8 This is not the only point of interest. Acta Pharm Sin B.

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The Pharma Data

AUGUST 29, 2020

3,4 LNP023 is currently in clinical development for PNH and a number of renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy, complement 3 glomerulopathy (C3G), atypical hemolytic uremic syndrome and membranous nephropathy.

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Agency IQ

SEPTEMBER 15, 2023

BY AMANDA CONTI SEP 13, 2023 1:58 PM CDT Quick background on nonprescription drug regulation Nonprescription drugs, also known as over-the-counter (OTC) drugs, are regulated differently than traditional prescription drugs. developing or amending a monograph) or sponsor-initiated operations (e.g.,

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The Pharma Data

DECEMBER 13, 2020

AMPK is a master regulator of several important metabolic pathways, including lipid metabolism, glucose control and inflammation, and is a novel target for NASH and additional chronic and rare metabolic diseases. PXL770 is a first-in-class, oral direct adenosine monophosphate-activated protein kinase (AMPK) activator. About PXL770.

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Agency IQ

AUGUST 9, 2024

estimated prevalence of the condition of interest, any relevant pharmacokinetic or pharmacogenomic data, demographic factors) and how they intend to meet those goals, as well as operational and process factors such as how they should be submitted and how the “action plan” may be updated over time. What about combination products?

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PPD

APRIL 14, 2025

To that end, the PI can ensure patients understand the science of appetite and metabolic regulation, for example, to relieve some of the self-imposed expectations for instant weight loss or other quick signs of success. Were applying that expertise and commitment to every aspect of developing and running successful GLP-1 studies.

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. This progress in clinical development qualifies Ascentage Pharma for a milestone payment according to the terms of the licensing agreement. SUZHOU, China and ROCKVILLE, Md. ,

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The Pharma Data

JULY 21, 2021

As part of our commitment to understanding the potential for our HIV medicines in a broad range of patients, we focused on the enrollment of diverse patient populations at risk for HIV, including women, who have one of the highest unmet needs in HIV prevention.”.

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DrugBank

JUNE 18, 2025

Drug development is a complex and highly regulated process. Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. While these regulations protect public health, they also introduce significant challenges for pharmaceutical researchers.

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Agency IQ

SEPTEMBER 22, 2023

In 1972, when regulatory authority for biologic products was transferred from the National Institutes of Health (NIH) to the FDA, the agency extended the two-study framework to biologic regulation as well. When should this occur? at the end-of-phase 2 meeting).

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Agency IQ

JUNE 9, 2023

For this reason, animal models are still used both during drug discovery and the more intensive nonclinical studies conducted during clinical development. CBER-regulated products are no longer within scope of the guidance. Furthermore, nonhuman primates are not required nor indicated in the new guidance for any studies.

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Agency IQ

DECEMBER 11, 2023

Previously, burdensome side effects may have been written off as a necessary part of treatment and benefits measured in additional weeks or months of survival time; today, though, patients, clinicians, and regulators expect more. In July, a new contract notice indicated that this is an area of interest for FDA as well.

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FDA Law Blog: Drug Discovery

JULY 9, 2024

Tobolowsky & Véronique Li, Senior Medical Device Regulation Expert & Sarah Wicks & Deborah L. DAP Content In developing DAPs, the Draft Guidance recommends that sponsors consider whether certain demographic groups may have a different response to a medical product regarding either effectiveness or safety. 360j(g)(9)(A)).

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Agency IQ

FEBRUARY 2, 2024

EMA finalizes clinical anticancer therapeutic guidance update In a newly finalized revision, the EMA’s guideline covering all aspects of clinical development of anticancer therapeutics has expanded its already broad scope. The EMA started providing guidance on the clinical development of anticancer therapeutics in 1996.

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PPD

JUNE 20, 2023

The staff transfer team at the PPD clinical research business of Thermo Fisher Scientific provides transfer success rates that average nearly 90% for FSP engagements and are experts in Acquired Rights Directive laws and regulations. Does your CRO leverage data-backed intelligence to accelerate your clinical development strategy?

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Agency IQ

JUNE 28, 2024

estimated prevalence of the condition of interest, any relevant pharmacokinetic or pharmacogenomic data, demographic factors) and how they intend to meet those goals, as well as operational and process factors such as how they should be submitted and how the “action plan” may be updated over time. ANNA ESHOO (D-Calif.)

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Agency IQ

AUGUST 16, 2024

Today, though, patients, clinicians, and regulators expect more. Another addition to FDA’s general advice is a recommendation for sponsors to engage early with the FDA “early in clinical development” (as opposed to “at relevant milestone meetings”).

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The Pharma Data

MARCH 30, 2021

It is vital that we work together with health authorities, regulators and industry to ensure we can get this medicine to the patients who need it as soon as possible.’’. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About FIREFISH.

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The Pharma Data

MAY 7, 2021

GSK and Vir continue discussions with global regulators to make sotrovimab available to patients with COVID-19. Sotrovimab is also under review by other global regulators including Health Canada under the expedited Interim Order application pathway for COVID-19 drugs. About the Sotrovimab Clinical Development Program.

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The Premier Consulting Blog

FEBRUARY 11, 2025

Despite this, drug developers must still meet regulatory requirements. Engaging early and actively with regulators, leveraging incentive programs, and discussing the benefit:risk balanceespecially for severe diseases with no available treatmentsare key strategies for advancing novel therapies in rare diseases.

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The Pharma Data

MARCH 26, 2021

GSK and Vir will continue discussions with the European Medicines Agency (EMA) and other global regulators to make VIR-7831 available to patients with COVID-19 as soon as possible. About the VIR-7831 Clinical Development Programme. About COMET-ICE.

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New Drug Approvals

MAY 11, 2025

5] This disease tends to develop in children and teenagers and gradually worsens over time. [5] 10] Nrf2 transcriptionally regulates multiple genes that play both direct and indirect roles in producing antioxidative potential and the production of cellular energy ( i.e. , adenosine triphosphate or ATP ) within the mitochondria.

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