New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., SCHEME PATENT PTC Therapeutics Inc.,

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NIH Director's Blog: Drug Development

OCTOBER 30, 2018

Now comes word of another absolutely incredible use of cryo-EM: determining with great ease and exquisite precision the structure of the smaller organic chemical compounds, or “small molecules,” that play such key roles in biological exploration and drug development. Also analyzed were eight less-familiar small molecules.

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New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. 2018; 32(9): 849–861. [2]. 2018 Mar;57(3):315-333. 2007 ; Kocis et al., 2016 ; Abushakra et al., Hey JA, et al. Clin Pharmacokinet.

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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. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 1 , 33–43 (2018). & Ohi, R.)

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New Drug Approvals

SEPTEMBER 29, 2024

link] 01 Aug 2022 Cortexyme is now called Quince Therapeutics You need to be a logged in or subscribed to view this content This small molecule is an orally available protease inhibitor targeting the lysine proteases of the periodontal pathogen Porphyromonas gingivalis. 2018 ; Ding et al., 2017 ; Ilievski et al.,

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Drug Target Review

JUNE 19, 2024

There are many opportunities to address conditions of the eye, such as retinitis pigmentosa, dry age-related macular degeneration or any of the hundreds of inherited retinal diseases. Just like small molecules and antibodies, cell therapies are changing how we treat patients.

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Drug Target Review

NOVEMBER 7, 2024

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. 1) GLOBOCAN 2018: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2018. Noria) and PSMA Therapeutics Inc.

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The Pharma Data

JANUARY 6, 2021

“IPF is a devastating condition with an estimated mean survival of 2-5 years from time of diagnosis, and currently there are no available therapies that stop the progression of fibrosis or treat the underlying causes of the disease,” said John Hood, Ph.D., Taladegib is a small-molecule inhibitor of the Hedgehog signaling pathway.

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The Pharma Data

JANUARY 13, 2021

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma.

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Drug Target Review

MAY 23, 2023

Inflammatory bowel disease (IBD) is an umbrella term that describes complex disorders that cause chronic inflammation in the digestive tract with alternating periods of relapse and remission. A changed composition of the gut microbiota is a characteristic trait of inflammatory bowel disease. More than 6.8

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The Pharma Data

NOVEMBER 1, 2020

Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of kidney disease, neurodegenerative disease, rare mitochondrial diseases and fibrosis. The Company is managed by a team with broad international, commercial and clinical-science experience.

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The Pharma Data

MARCH 1, 2022

The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.

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The Pharma Data

JULY 15, 2021

JJIPO@Monash was originally launched in 2018 to provide crucial support to researchers and companies in the Victorian life-science sector as part of the Government’s commitment to the medical technologies and pharmaceuticals sector – one of eight priority growth sectors. .

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The Pharma Data

JANUARY 13, 2021

Structure 3:1031-1039 (1995) 3 Waters J, Biocentury ; October 26, (2018) 4 NCT02273960; ClinicalTrials.gov ; “Study to Evaluate Safety and Efficacy in Adult Subjects With ITP (ITP)”; results posted April 1, 2019, updated July 29, 2019 and accessed Jan 11, 2021 5 Ferrant JL et al., 1 Lederman, S. International Immunol. (11):1583

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The Pharma Data

NOVEMBER 17, 2020

LYT-100 is a deuterated, oral small molecule designed to overcome the challenges associated with pirfenidone, an approved and marketed anti-inflammatory and anti-fibrotic drug. IPF, unclassifiable interstitial lung diseases (uILDs), Long COVID respiratory complications and related sequelae) and lymphedema. Photo: Business Wire).

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Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

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The Pharma Data

MARCH 23, 2022

Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase. But targeting Ebola virus polymerase has proven tough.

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The Pharma Data

DECEMBER 2, 2020

Food and Drug Administration (FDA) for Onivyde (irinotecan liposome injection) for study patients with small cell lung cancer (SCLC) who progressed following a first-line platinum-based regimen. Previously, the company announced positive results of the previous Phase IIa induction study in UC in September 2018.

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Metabolite Tales Blog

DECEMBER 13, 2023

Conjugation of small molecule drugs to glucuronic acid is catalysed by several UGTs to frequently form N – and O -glucuronides. As in this example, most N -glucuronides of small molecule drugs are innocuous and possess no pharmacological activity. PMID: 9733662. [8] Antimicrob Agents Chemother.

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The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. AB2 Bio – Swiss pharma company AB2 Bio Ltd. named Djordje Filipovic as its new chief commercial officer.

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The Pharma Data

OCTOBER 14, 2020

2018) PLoS One. Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. TNX-102 SL is also in development for agitation in Alzheimer’s disease and alcohol use disorder (AUD).

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LifeSciVC

APRIL 27, 2023

Fourteen years on, this corporate experiment has gone far beyond the initial idea, and has established an R&D engine more effective than most big pharma R&D groups at producing best-in-class small molecules against targets that matter in human disease biology. Would we wind down and exit, or chart some different path?

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New Drug Approvals

SEPTEMBER 13, 2024

30 January 2018. “LY2456302 is a novel, potent, orally-bioavailable small molecule kappa-selective antagonist with activity in animal models predictive of efficacy in mood and addictive disorders” Neuropharmacology. Reed B, Butelman ER, Fry RS, Kimani R, Kreek MJ (March 2018). McHugh KL, Kelly JP (2018).

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Drug Target Review

MAY 31, 2024

I currently lead a talented team of multi-disciplinary scientists who are dedicated to discovering a treatment for Parkinson’s Disease, a debilitating condition with significant unmet medical need. About the author Dr H Rachel Lagiakos Senior Principal Scientist, Therapeutics Group at Schrodinger Dr Lagiakos joined the company in 2018.

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Agency IQ

OCTOBER 6, 2023

This is a significant jump from any previous year, with the former peak at 15 in FY 2018, followed by several years in the 10-14 range. Taking a closer look, nearly half of the products were reviewed by the Office of Oncologic Diseases. Amanda Conti, AgencyIQ What kind of biologic products entered the market?

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The Pharma Data

OCTOBER 26, 2020

The filings were based on the results of a robust Phase 3 clinical trial program, across which abrocitinib demonstrated statistically superior improvements in skin clearance, disease extent, and severity, as well as rapid improvements (measured as early as Week 2) in itch versus placebo. About Atopic Dermatitis. Pfizer Inc.:

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The Pharma Data

DECEMBER 27, 2020

This year has been difficult, but it has reinforced the importance of our mission: to treat and potentially even reverse the effects of serious diseases and conditions by advancing our novel cell therapy product candidates through clinical trials and into the hands of physicians.

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The Pharma Data

APRIL 23, 2021

Dr Hal Barron, Chief Scientific Officer and President R&D, GSK, said: “Women with recurrent endometrial cancer, or advanced disease that has progressed on or after chemotherapy, currently have limited treatment options and a poor prognosis. and a disease control rate of 55.6% (95% CI; 45.7-65.1). Published 2018.

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The Pharma Data

FEBRUARY 4, 2021

AbbVie announced positive top-line results from the Phase 3 ADVANCE and MOTIVATE studies, which evaluated the efficacy and safety of Skyrizi (risankizumab) for induction therapy in adult patients with moderate to severe Crohn’s disease (CD). AbbVie and Frontier Medicines, Corp.,

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Agency IQ

NOVEMBER 3, 2023

Synthetic biotics have been engineered to perform a variety of functions: “ Several classes of effectors have been engineered into bacteria, including enzyme pathways that metabolize disease-causing toxins, production of protein effectors, and production of small-molecule effectors,” according to a recent article on the topic.

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The Pharma Data

APRIL 27, 2021

Rare Disease. Others Rare Disease. Total Rare Disease. In the first quarter, Rare Disease sales increased 4.4% up 60%) driven by increase disease and product awareness as well as adoption of new ISTH (International Society on Thrombosis and Haemostasis) TTP guidelines. Net sales (€ million). Change at CER.

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New Drug Approvals

APRIL 18, 2023

H]Cl.NCCCCN(CC1=NC2=CC=CC=C2N1)[C@@H]3C4=NC=CC=C4CCC3 AMD-070 is a small molecule drug candidate that belongs to a new investigational class of anti-HIV drugs known as entry (fusion) inhibitors. 2018 Jul;40(1):303-308. Molecular Weight 349.47 Appearance Solid Formula C 21 H 27 N 5 CAS No. 2309699-17-8 SMILES [H]Cl.[H]Cl.[H]Cl.NCCCCN(CC1=NC2=CC=CC=C2N1)[C@@H]3C4=NC=CC=C4CCC3

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Drug Target Review

FEBRUARY 15, 2024

Rising demand for animal protein has led to more factory farming, increasing the likelihood that diseases can spread among these animals then into humans. 5 Furthermore, ever larger numbers of humans are living in cities or travelling across borders, increasing the risk of diseases being transmitted person-to-person over a long range.

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The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase III clinical trial programme for fenebrutinib, an investigational medicine designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, which may offer novel approach to suppress disease activity and slow disease progression in MS.

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The Pharma Data

APRIL 7, 2023

Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. “Through our comprehensive development program, the BRAFTOVI and MEKTOVI combination has shown the potential to help more patients, such as those living with BRAF V600E-mutant non-small cell lung cancer.

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Metabolite Tales Blog

NOVEMBER 13, 2024

Major biotransformation routes of aficamten involving hydroxylation, onwards glucuronidation and gut metabolism Hypertrophic cardiomyopathy is a surprisingly common genetic heart disease, affecting 1:200 to 1:500 people in the US. Drug Metabolism and Disposition April 1, 2020, 48 (4) 317-325; DOI: [link]. [5] Watanabe, A., Deguchi, T.,

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