The Pharma Data

JULY 22, 2021

The prospective, phase 3, open-label, international multicenter study included 23 patients with severe von Willebrand disease (VWD). 1 The pharmacokinetics (PK) of VWF:ristocetin cofactor (VWF:RCo) and FVIII pharmacodynamics (PD) [PB0917] were also studied and presented at the congress. About von Willebrand Disease (VWD).

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Agency IQ

SEPTEMBER 15, 2023

The committee also made recommendations regarding pharmacokinetic and safety assessments. When looking at several pharmacokinetic studies, the FDA found that the 10 mg oral dose of phenylephrine has a very low bioavailability of less than 1% and, subsequently, low systemic alpha-1 adrenergic activity.

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The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. IPF, unclassifiable interstitial lung diseases (uILDs), Long COVID respiratory complications and related sequelae) and lymphedema. 1 Cottin, V.,

Pharmacokinetics 52

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New Drug Approvals

SEPTEMBER 13, 2024

14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 30 January 2018. nM vs. 24.0

Treatment 62

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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. UNITY is a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging. SUZHOU, China and ROCKVILLE, Md. , Ascentage Pharma (6855.HK)

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Drug Discovery World

FEBRUARY 15, 2023

Cancer is a complex heterogenous multistep disease characterised by uncontrolled cell proliferation combined with a dysregulated immune response. The number of new cases recorded between 2016 and 2018 was 375,400, with 167,142 deaths caused by cancer between 2017 and 2019. This continues to be the standard of care.

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The Pharma Data

APRIL 8, 2021

OCREVUS data show its consistent benefit on slowing disease progression in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Data for ENSPRYNG in neuromyelitis optica spectrum disorder (NMOSD) reinforce safety and efficacy, including in patients with concomitant autoimmune diseases (CAIDs). Following U.S.

Clinical Trials 52

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Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution.

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The Pharma Data

FEBRUARY 8, 2021

1 Quality of life was comparable between treatment arms per Functional Assessment of Cancer Therapy–Prostate (FACT-P Total). Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. Grade 3/4 treatment emergent adverse events (TEAEs) were reported in 63.3

Pharmaceutical Companies 52

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The Pharma Data

FEBRUARY 25, 2021

FIREFISH Part 1 data show treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support, a key motor milestone not normally seen in the natural course of the disease. Enrollment for Part 2 was completed in November 2018. mg/kg for Part 2.

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The Pharma Data

MARCH 20, 2023

Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. This trial included people with Type 2 and 3 SMA, including patients with advanced disease. Adverse events (AEs) and serious adverse events (SAEs) were reflective of the underlying disease. Enrolment for Part 2 was completed in November 2018.

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The Pharma Data

MARCH 16, 2021

SUNFISH Part 2 study population includes broad range of ages and disease severities, representing a real-world spectrum of people living with Type 2 or 3 SMA. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. The study has completed recruitment (n=174).

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The Pharma Data

MARCH 30, 2021

SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation in 2019. Enrolment for Part 2 was completed in November 2018. The study met its primary endpoint.

Treatment 52

Treatment Clinical Trials Pharmacokinetics Trials 52

The Pharma Data

SEPTEMBER 27, 2020

Of the 17 infants treated with the therapeutic dose, two experienced fatal complications of their disease at 8 and 13 months of treatment and one infant was withdrawn from the study and sadly died 3.5 Enrolment for Part 2 was completed in November 2018. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal.

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