Clinical Development, Events, Pharmacokinetics and Regulations

Analysis Life Sciences Thank You FDA’s nonprescription advisors find no efficacy for phenylephrine

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.,

Science

Science FDA Clinical Trials Pharmacy

Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

The Pharma Data

AUGUST 29, 2020

LNP023 also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events. Following the cut-off date for the presented data, one participant, who had severe lymphopenia at study entry, discontinued treatment due to a serious adverse event (AE) of lymphoproliferative disorder.

Treatment

Treatment Disease Small Molecule Production

Poxel Announces Additional Positive Phase 2a Results, and Phase 2b Plan for PXL770, an Oral First-in-Class AMPK Activator, in NASH

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. NASH Investor Event Information. A replay of the event will be available on Poxel’s website following the presentation.

Trials

Trials Disease Treatment Pharmacokinetics

BioXcel Therapeutics Receives FDA Clearance of IND for Phase 2 Trial with BXCL501 for the Treatment of Agitation Associated with DeliriumFifth potential indication for BXCL501, an orally dissolving thin film

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.

Treatment

Treatment Trials FDA Hospitals

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

LifeSciVC

APRIL 24, 2024

There is a continuum of evidence for a given target – at one end are novel targets with some evidence of importance in disease, and at the other end are “de-risked” targets where the biology is precedented with an approved product or late-stage clinical asset(s). One example of such a genetics exercise is represented in Fig.

Production

Production Small Molecule Regulations Trials

Ascentage Pharma’s Licensee UNITY Biotechnology Announces Milestone Reached in Clinical Development of Treatments for Age-Related Disease, Leading to Milestone Payment

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. ,

Clinical Development

Clinical Development Disease Treatment Licensing

Merck Presents New Data from Ongoing Phase 2a Clinical Trial Evaluating the Safety, Tolerability and Pharmacokinetics of Investigational, Once-Monthly, Oral Islatravir for HIV-1 Prevention at IAS 2021

The Pharma Data

JULY 21, 2021

Most adverse events (AEs) were mild and there were no serious drug-related AEs in people who received islatravir. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.

Clinical Trials

Clinical Trials Pharmacokinetics Trials International

SRP-001: redefining pain treatment with a safer, non-opioid analgesic

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. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety.

Treatment

Treatment Clinical Trials Molecular Biology Therapies

GSK and Vir Biotechnology announce the start of the EMA rolling review of VIR-7831 (sotrovimab) for the early treatment of COVID-19

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.

Treatment

Treatment Virus Vaccine Trials

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

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.

Clinical Trials

Clinical Trials Trials Disease Nurses

Aptose Initiates Dosing of CG-806 in Patients with Acute Myeloid LeukemiaPhase 1 a/b Study of CG-806 in AML Initiates with Starting Dose of 450mg

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. .

Small Molecule

Small Molecule Clinical Trials Trials Therapies

Analysis Life Sciences Thank You FDA’s Diversity Action Plan: Questions, answers, and what we (don’t) know so far

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?

Science

Science FDA Production Trials

Analysis Life Sciences Thank You FDA solidifies its advice on dose optimization for oncology products

Agency IQ

AUGUST 16, 2024

intermediate or surrogate) endpoints and clinical outcome endpoints (e.g., Further, some newer therapies are associated with immune-mediated adverse events such as cytokine release syndrome that are not always dose-related. Today, though, patients, clinicians, and regulators expect more.

Production

Production FDA Science Clinical Trials

Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

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.

FDA

FDA Science Small Molecule Immune Response

Better Late Than Never – Unpacking FDA’s Highly Anticipated (and Long Overdue) Draft Guidance on Diversity Action Plans

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)).

FDA

FDA Clinical Trials Disease Trials

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease

The Pharma Data

DECEMBER 29, 2020

PRX-102 was well-tolerated in the study, with all adverse events being transient in nature without sequelae. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. In clinical studies, PRX-102 has been observed to have a circulatory half-life of approximately 80 hours.

Clinical Trials

Clinical Trials Disease Treatment Trials

Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy

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.

Treatment

Treatment Clinical Trials Pharmacokinetics Trials

Analysis Life Sciences Thank You FDA just published its Diversity Action Plan guidance. Here’s what you need to know.

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.)

FDA

FDA Science Clinical Trials Production

Drug Discovery Digest

Analysis Life Sciences Thank You FDA’s nonprescription advisors find no efficacy for phenylephrine

Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Trending Sources

Poxel Announces Additional Positive Phase 2a Results, and Phase 2b Plan for PXL770, an Oral First-in-Class AMPK Activator, in NASH

BioXcel Therapeutics Receives FDA Clearance of IND for Phase 2 Trial with BXCL501 for the Treatment of Agitation Associated with DeliriumFifth potential indication for BXCL501, an orally dissolving thin film

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

Ascentage Pharma’s Licensee UNITY Biotechnology Announces Milestone Reached in Clinical Development of Treatments for Age-Related Disease, Leading to Milestone Payment

Merck Presents New Data from Ongoing Phase 2a Clinical Trial Evaluating the Safety, Tolerability and Pharmacokinetics of Investigational, Once-Monthly, Oral Islatravir for HIV-1 Prevention at IAS 2021

SRP-001: redefining pain treatment with a safer, non-opioid analgesic

GSK and Vir Biotechnology announce the start of the EMA rolling review of VIR-7831 (sotrovimab) for the early treatment of COVID-19

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

Aptose Initiates Dosing of CG-806 in Patients with Acute Myeloid LeukemiaPhase 1 a/b Study of CG-806 in AML Initiates with Starting Dose of 450mg

Analysis Life Sciences Thank You FDA’s Diversity Action Plan: Questions, answers, and what we (don’t) know so far

Analysis Life Sciences Thank You FDA solidifies its advice on dose optimization for oncology products

Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

Better Late Than Never – Unpacking FDA’s Highly Anticipated (and Long Overdue) Draft Guidance on Diversity Action Plans

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease

Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy

Analysis Life Sciences Thank You FDA just published its Diversity Action Plan guidance. Here’s what you need to know.

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