The Pharma Data

DECEMBER 28, 2020

The AMBITION trial is the first placebo-controlled study of CRV431 in NASH patients with evidence of moderate-to-severe fibrosis. The primary objectives of the AMBITION trial are to assess safety and tolerability of CRV431, as well as to delineate pharmacokinetics. To date, there are no approved drugs to treat NASH.

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The Pharma Data

NOVEMBER 8, 2020

The New Drug Application (NDA) for the drug is based on data from four Phase III trials in pediatric patient populations from the age of 6 to 17 years, two Phase II trials, several Phase I trials, a long-term open label extension study, preclinical testing, and drug manufacturing data. The NDA was resubmitted on May 2020.

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FDA Law Blog: Drug Discovery

FEBRUARY 2, 2023

Since 1962, the FD&C Act has authorized FDA to require that sponsors of clinical trials submit data from “preclinical tests (including tests on animals)” in order to demonstrate that their drug is safe enough to advance to testing in humans. For more on FDORA’s other provisions, see HPM’s complete summary here ). FDORA § 3209(a)(1).

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PerkinElmer

JANUARY 7, 2022

There are many reasons that promising drug candidates are discontinued, including poor pharmacokinetics, lack of clinical efficacy, and toxicity. 2 Toxic to the Heart One of the top reasons for drug withdrawal from clinical trials and the market is cardiotoxicity, which can be defined as toxicity that has a detrimental impact on the heart.

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New Drug Approvals

SEPTEMBER 13, 2024

2] As of July 2022, it is in phase 3 clinical trials for major depressive disorder. [2] hours following a single dose of 10 mg, which supported the 4 mg to 25 mg dosages that aticaprant is being explored at in clinical trials. [13] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] nM vs. 24.0 nM vs. 24.0

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The Pharma Data

JANUARY 17, 2021

The companies announced in January 2019 a collaboration agreement to develop, manufacture and commercialize SPR206 in the Territory. Spero plans to initiate additional Phase 1 studies to assess the penetration of SPR206 into the pulmonary compartment and pharmacokinetics in subjects with renal impairment in 2021. SHANGHAI , Jan.

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The Pharma Data

DECEMBER 13, 2020

A biosimilar is like a generic version of a biologic, but unlike a generic, which is a direct copycat of a branded drug, a biosimilar is “similar,” and as a result has to undergo a regulatory and clinical trial process similar to that of new drugs. The studies found no clinically meaningful differences between ABP 798 and Rituxan.

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Drug Target Review

SEPTEMBER 25, 2024

8 Preliminary data from the trial have shown good safety and tolerability for TT125-802, with confirmed preliminary target engagement in surrogate tissues such as hair follicles and peripheral blood mononuclear cells. 2019 Dec 13 [cited 2024 Sep 4];122(4):465–72. Nature Reviews Drug Discovery [Internet]. Available from: [link] 2.Bell

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New Drug Approvals

OCTOBER 24, 2023

This approval was based on favorable results obtained from Pfizer’s Elevate UC Phase III registrational program, consisting of the Elevate UC 52 and Elevate UC 12 clinical trials, that investigates the efficacy of a 2-mg daily dose regimen of etrasimod, with a 32% and 26% remission rate observed in UC 52 and UC 12 trials respectively.

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Alta Sciences

APRIL 17, 2024

Ketamine is a dissociative anesthetic approved in 2019 by the U.S. Fifteen oligonucleotides have so far received market authorization in different countries, and several others are being test in clinical Phase I to III trials. Food and Drug Administration (FDA) as a nasal spray called esketamine, for treatment-resistant depression.

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Agency IQ

FEBRUARY 2, 2024

The revisions flesh out considerations for use of biomarkers at all stages of product development and add new content on rare cancers and platform trial designs. In 2019 more than one in four (28%) of deaths in the E.U. This enables patient selection, stratification and other adjustment in clinical trial design planning.

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Agency IQ

SEPTEMBER 22, 2023

Traditionally, FDA has interpreted the need for “well-controlled investigations” to mean at least two clinical trials for new drugs, or applications for supplemental indications. When looking at all drugs approved between 2011 and May 2020, 136 drugs were approved on the basis of a single pivotal trial, according to AgencyIQ’s research.

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The Pharma Data

SEPTEMBER 26, 2021

The positive CHMP opinion was supported data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to determine bioequivalent exposure of monomethyl fumarate, the active metabolite, and relied partially on the well-established long-term safety and efficacy profile of TECFIDERA.

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The Pharma Data

DECEMBER 21, 2020

As UNITY reported in its press release, this “Phase I, first-in-human, open-label, single-ascending dose study being conducted by UNITY is designed to evaluate the safety, tolerability, and pharmacokinetics of UBX1325 in patients with advanced DME. Ascentage Pharma (6855.HK)

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The Pharma Data

APRIL 27, 2021

The NDA for mobocertinib is primarily based on results from the Phase 1/2 trial , which is evaluating the safety and efficacy of oral mobocertinib in patients with mNSCLC. In 2019, the U.S. About the Phase 1/2 Trial. and around the globe.”. The application was submitted under the FDA’s accelerated approval program.

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The Pharma Data

APRIL 11, 2021

” In the Phase 1/2 LIBRETTO-001 trial, 32 adult patients with 12 unique RET fusion-positive advanced cancer types were enrolled by the efficacy cutoff date of September 19, 2020 (with follow-up through March 19, 2021). Retevmo was approved based on the Phase 1/2 LIBRETTO-001 trial’s endpoints of ORR and DoR.

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The Pharma Data

JANUARY 4, 2021

Cell 2019 ). Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 20-F for the year ended December 31, 2019, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public, by the Company.

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Drug Target Review

JULY 12, 2023

The goal of DCI in preclinical and clinical assessments is to achieve a manyfold higher drug free-fraction pharmacokinetic (PK) C max level (the plasma concentration of therapy in a specific area of the body) to break tumour addiction to the MAPK signalling pathway followed by a rapid drop off of drug levels enabled by a short plasma half-life.

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The Pharma Data

JANUARY 10, 2021

In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches. COPENHAGEN, Denmark, Jan. Morgan Healthcare Conference. “In . ?. . . .

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The Pharma Data

SEPTEMBER 15, 2020

Limited blinded tolerability data from the ongoing Phase 3 trial, confirming the mostly mild to moderate tolerability profile as was observed in Phase 1. In the blinded data presented, 50% of trial participants received placebo and 50% received BNT162b2. A Phase 3 trial is ongoing. Protease Inhibitor Program. Rare Disease.

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The Pharma Data

OCTOBER 27, 2020

2019. 2019. million for the three months ended September 30, 2020 , compared to the same period in 2019. In addition, as previously disclosed, our 2019 revenues were impacted by a mistake by one of our U.S. million in total, during the third quarter of 2019. 2019.

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The Pharma Data

DECEMBER 5, 2020

Potentially registrational Phase 2 portion of the trial has been initiated and is enrolling patients. NASDAQ: REGN) today announced updated data for REGN5458, a BCMAxCD3 bispecific antibody, from the Phase 1 portion of a Phase 1/2 trial in patients with relapsed or refractory (R/R) multiple myeloma. Regeneron Pharmaceuticals, Inc.

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The Pharma Data

FEBRUARY 8, 2021

Across clinical trials (PALOMA-1, PALOMA-2, PALOMA-3), 1.0% The pharmacokinetics of IBRANCE have not been studied in patients requiring hemodialysis. of IBRANCE-treated patients had ILD/pneumonitis of any grade, 0.1% had Grade 3 or 4, and no fatal cases were reported. About Pfizer Oncology.

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The Pharma Data

FEBRUARY 8, 2021

Patients in the trial received either a combination of ERLEADA ® and ZYTIGA ® plus prednisone (combination arm) or placebo and ZYTIGA ® plus prednisone (control arm). [i] Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. 2 ERLEADA ® received U.S.

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Agency IQ

JULY 26, 2024

In switching studies , a two-arm trial design is commonly employed. The new draft guidance also points applicants to 2019 draft guidance on the Development of Therapeutic Protein Biosimilars (comparative analytical assessments) and the ICH Q6B guidance on test procedures and acceptance criteria.

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The Pharma Data

MAY 4, 2021

There are limited clinical data available for baricitinib use in coronavirus 2019 (COVID-19). volunteers to evaluate the safety, tolerability, pharmacokinetics and immunogenicity. Junshi Biosciences has completed a similar Phase 1 study in healthy volunteers in China and has initiated Phase 1b/2 trials in COVID-19 patients globally.

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Agency IQ

SEPTEMBER 15, 2023

The clinical trials underpinning the original determination that phenylephrine is GRASE – that is, Generally Regarded as Safe and Effective – predate the 1994 inclusion of phenylephrine hydrochloride in the final monograph for OTC nasal decongestant drug products. Once metabolized, which occurs quickly with a half-life of approximately 1.5

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The Pharma Data

DECEMBER 3, 2020

Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single and Multiple Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study. Clinical Trials. MONARCH Phase 1/2a Current Study Design. December 6, 2020 12:00 PM – 1:30 PM; Track: 7. Antiepileptic Drugs / 7B.

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The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. The primary objective of the trial is to evaluate the safety and tolerability of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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The Pharma Data

NOVEMBER 4, 2020

. Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The THRIVE-AA1 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing in approximately 700 adult patients with moderate to severe alopecia areata.

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The Pharma Data

JULY 6, 2021

Population pharmacokinetic and pharmacodynamic modeling data to characterize the antithrombin (AT) lowering dynamics in hemophilia patients treated with fitusiran will be shared in a poster presentation. These data support the revised fitusiran dose and dosing regimen implemented in the ongoing adult and adolescent studies. FDA for ITP.

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The Pharma Data

OCTOBER 6, 2021

.” In August, 2021, AstraZeneca declared high- position results from the PROVENTpre-exposure prophylaxis trial which showed AZD7442 reduced the imminence of developing discriminating COVID-19 by 77 (95 confidence interval (CI) 46, 90), compared to placebo. The trial accrued 25 cases of discriminating COVID-19 at the primary analysis.

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The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

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The Pharma Data

OCTOBER 14, 2020

The designation of lanifibranor as a Breakthrough Therapy for the treatment of NASH follows the publication in June 2020 of positive topline results from Inventiva’s NATIVE Phase IIb clinical trial with lanifibranor in NASH patients. The trial plans to evaluate safety, tolerability, cytokine profile and efficacy parameters.

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The Pharma Data

JUNE 28, 2021

The application is based on evidence from adequate and well-controlled studies of Xarelto in adults with additional data from two Phase III clinical trials of Xarelto in pediatric patients: EINSTEIN-Jr., From November 2016 to June 2019, a total of 112 participants were enrolled across 36 sites in 10 countries. EINSTEIN-Jr.

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FDA Clinical Trials Treatment Therapies 52

Agency IQ

FEBRUARY 23, 2024

Since 2019, more than half of the population of Europe has been classified as overweight or obese. According to Eurostat, the prevalence of overweight and obesity in adults in 2019 (the most recent year with complete data) was 53%. The figures are lower for individuals meeting obesity criteria , with 2019 data showing just 16.3%

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The Pharma Data

FEBRUARY 25, 2021

FIREFISH, an open-label, two-part pivotal study, was designed to assess Evrysdi safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) in patients aged 1 to 7 months with Type 1 SMA. Evrysdi has been and continues to be studied in more than 450 people as part of a large and robust clinical trial program in SMA.

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