Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

Drug Target Review

APRIL 19, 2024

And so, I have been the managerial co-founder and CEO of Sibylla since 2017. The discovery of the folding intermediate states to target has been enabled by our proprietary computational platform, and the development of small molecule folding interfering degraders is now accelerated by a proprietary AI platform.

Molecular Biology 111

Molecular Biology Research Pharmaceuticals Small Molecule 111

The Pharma Data

NOVEMBER 15, 2020

Urovant’s lead drug candidate, vibegron, is an oral, once-daily small molecule beta-3 agonist being evaluated for the treatment of OAB, abdominal pain associated with irritable bowel syndrome (IBS) in men with OAB, and for men suffering from OAB with benign prostatic hyperplasia (BPH).

Science 52

Science Small Molecule FDA Approval Therapies 52

Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

Drugs 75

Drugs Small Molecule Production Disease 75

New Drug Approvals

SEPTEMBER 13, 2024

2] Like other kappa opioid antagonists currently under clinical investigation for the treatment of major depression, its efficacy may be compromised by the countervailing activation of pro-inflammatory cytokines in microglia within the CNS. [7] 2] Aticaprant is taken by mouth. [1] nM vs. 24.0 13] [14] Occupancy was 35% for a 0.5 nM vs. 24.0

Treatment 62

Treatment Clinical Trials Pharmacokinetics Pharmaceuticals 62

The Pharma Data

JUNE 22, 2023

in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. We look forward to advancing new treatments in oncology with the potential to positively impact the lives of people with cancer in Japan. .–(BUSINESS WIRE)– Kite Pharma, Inc.,

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The Pharma Data

MARCH 1, 2022

This acquisition gives AbbVie access to Syndesi’s portfolio of novel modulators of the synaptic vesicle protein 2A (SV2A), including its lead molecule SDI-118. ” The lead molecule, SDI-118, is a small molecule currently in Phase 1b studies, which is being evaluated to target nerve terminals to enhance synaptic efficiency. .

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Small Molecule Licensing Licensing Disease 52

The Pharma Data

DECEMBER 15, 2020

Treatment induced sustainable clinical responses and reduced systemic inflammation. Daratumumab already is approved for the treatment of multiple myeloma. The company is developing its platform for the treatment of chronic, inflammation-mediated autoimmune diseases, and is initially focused on the treatment of RA.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

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. Tukey, Ryoichi Fujiwara. Kowalczyk I, Hawes EM, McKay G. Oncotarget.

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Small Molecule Metabolic Stability Treatment Drugs 59

KIF1A

JUNE 30, 2023

KAND and Spasticity : We created this resource to help families understand mechanisms of spasticity in KAND, including considerations between different treatment options. With your support, KIF1A.ORG continues to bring together the expertise and innovation needed to rapidly discover treatments and cures for KAND.

Small Molecule 52

Small Molecule Treatment Doctors Research 52

The Pharma Data

DECEMBER 21, 2020

Program focused on identifying small molecules that target a GPCR for degradation as potential therapeutic agents for gastrointestinal disorders. The principle of TPD is to use small molecules to commit the target protein into the E3 ligase-mediated degradation pathway thereby eliminating or reducing its activity.

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Targeted Protein Degradation Small Molecule Structure Based Drug Design (SBDD) Drugs 40

KIF1A

MAY 31, 2024

From hosting a conference to scientific projects and collaborations to the dedicated efforts of our team, this review encapsulates the relentless pursuit to accelerate treatments and a cure for all KAND warriors. The Chung Lab spearheads a robust KAND program to better understand the disorder and discover treatments.

Treatment 52

Treatment Small Molecule International Research 52

The Premier Consulting Blog

JULY 9, 2023

During the development of new small molecule drug products, developers must conduct impurity and degradant evaluation at several points in the program and to varying degrees. The TTC varies based on the clinical treatment duration and on whether multiple mutagenic impurities are present. link] ICH M7 (R2)- Q&A.

Small Molecule 52

Small Molecule DNA Drugs Clinical Development 52

The Pharma Data

DECEMBER 9, 2020

333-222268) previously filed with the Securities and Exchange Commission (the “SEC”) on December 22, 2017 and declared effective by the SEC on January 5, 2018. The shares of common stock are being offered by Oncternal pursuant to a “shelf” registration statement on Form S-3 (File No. More information is available at.

Clinical Trials 52

Clinical Trials Small Molecule Therapies Treatment 52

NIH Director's Blog: Drug Development

JULY 20, 2017

Specifically, after 2 to 4 weeks of treatment with this combination of two correctors and a potentiator, such patients experienced about a 10 percentage point improvement in forced expiratory volume in 1 second (FEV1), which is a key measure of lung capacity. Cystic Fibrosis Foundation News Release, July 18, 2017.

Clinical Trials 52

Clinical Trials Small Molecule Therapies Disease 52

The Pharma Data

JANUARY 4, 2021

SENS-401 has demonstrated highly encouraging efficacy in preclinical models and we are excited to progress this small molecule towards clinical trials to address this significant unmet medical need in cancer patients undergoing cisplatin treatment. 2017 Oct;38(9):1355-1361. D’heygere, R. Kuhweide, H. Source link.

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Clinical Trials Small Molecule Therapies Treatment 40

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. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics.

Small Molecule 52

Small Molecule Clinical Development Engineering Pharmaceuticals 52

The Pharma Data

NOVEMBER 8, 2020

In 2017 we bet on refocusing our strategy on medical dermatology to best address patients’ unmet needs. All Almirall’s production sites continue to operate at full capacity in order to be able to ensure the supply of medicines to all patients requiring treatment. In key markets such as Germany , Ilumetri ® gained c.

Licensing 52

Licensing Licensing Marketing Production 52

The Pharma Data

SEPTEMBER 2, 2020

New data further reinforce OCREVUS (ocrelizumab) as a highly effective treatment option offering a favourable and consistent benefit:risk profile, with high treatment persistence and adherence. Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations.

Clinical Trials 52

Clinical Trials Treatment Disease Therapies 52

Drug Target Review

MAY 31, 2024

One of the first programs I worked on in my career was drugging a novel, epigenetic target which had potential implications for the treatment of cancers. Could you share an example of a specific project or research that you have worked on and the impact it has had in your field?

Organic Chemistry 45

Organic Chemistry Computational Chemistry Small Molecule Disease 45

The Pharma Data

NOVEMBER 5, 2020

has been appointed to the additional role of president and will oversee the growth of the company’s technology portfolio for the treatment of ischemic and hemorrhagic stroke. Prior to joining Navitor, Hughes served as president and CSO of Zafgen and previously led Zafgen as CEO from 2008 to 2017. Schijns, a former professor at N.C.

Analytical Chemistry 40

Analytical Chemistry Pharmaceuticals Small Molecule Therapies 40

Agency IQ

FEBRUARY 23, 2024

As the name implies, the MTD seeks to find the highest dose of a treatment that can both be tolerated and treat the cancer. Additionally, this approach is generally no longer applicable to new treatments that are more targeted in their effects, such as immunotherapies and targeted small molecule therapies.

Science 40

Science FDA Trials Clinical Pharmacology 40

The Pharma Data

MARCH 4, 2021

Under the 2017 collaboration agreement, Takeda received equity in Ovid and was eligible to receive up to $85 million in payments for regulatory milestones, including the initiation of Phase 3 clinical trials. “We Advancing New Treatment Options for DS and LGS Patients. selective GABA A receptor agonist.

Treatment 52

Treatment Clinical Trials Pharmaceutical Companies Trials 52

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. 2017 ; Ilievski et al., 2018 ; Ding et al., and Europe.

Small Molecule 62

Small Molecule DNA Disease Trials 62

NIH Director's Blog: Drug Discovery

APRIL 11, 2017

Now, NIH researchers and their collaborators have discovered a new kind of treatment that holds promise for fighting parasitic roundworms. A bonus of this result is that this same treatment might work also for certain deadly kinds of bacteria. 2017 Apr 3;8:14932. All of their hard work came up empty. PLoS Negl Trop Dis.

Small Molecule 52

Small Molecule Science Drug Development Disease 52

The Pharma Data

NOVEMBER 29, 2020

1 Meanwhile, these MDM2 inhibitors present some challenges, including dose-limiting hematological toxicities, thus the urgent need to develop new generation of MDM2-targeting therapies in the treatment of cancer. Small-molecule inhibitors of the MDM2-p53 protein-protein interaction (MDM2 inhibitors) in clinical trials for cancer treatment.

Licensing 40

Licensing Licensing Small Molecule Clinical Trials 40

The Open Targets Blog

APRIL 5, 2023

One treatment angle for neurodegenerative diseases is to dampen the Unfolded Protein Response to rescue the neurons. The Druggable Genome The Druggable Genome is the subset of all human genes encoding proteins that could be targeted with a small molecule or antibody drug [4]. 2017 Apr 11;8(4):803-812. Stem Cell Reports.

Small Molecule 52

Small Molecule Disease RNA Treatment 52

The Premier Consulting Blog

APRIL 25, 2023

In the US, new small molecule drugs are developed under the requirements of Sections 505(b)(1) and 505(b)(2) of the FFDCA. Synthroid® oral tablets) were marketed as unapproved drugs for the treatment of hypothyroidism in the US. This strategy can reduce the overall study requirements for approval. [1]

FDA 52

FDA Animal Testing Pharmacokinetics Pharmaceuticals 52

Agency IQ

DECEMBER 11, 2023

The areas within oncology that have seen the most success in terms of new and effective treatment options are now entering a new paradigm where the terms “success” and “harm” are being redefined. In parallel, stakeholders have advocated for recognizing the importance of treatment tolerability when determining doses and regimens.

FDA 40

FDA Science Clinical Trials Trials 40

New Drug Approvals

JULY 10, 2023

g/mol BASE Vazegepant BMS-742413 BHV-3500 FDA APPR 3/9/2023 Zavzpret N -[(2 R )-3-(7-methyl-1 H -indazol-5-yl)-1-[4-(1-methylpiperidin-4-yl)piperazin-1-yl]-1-oxopropan-2-yl]-4-(2-oxo-1 H -quinolin-3-yl)piperidine-1-carboxamide ZAVZPRET is indicated for the acute treatment of migraine with or without aura in adults. 1] It is sold by Pfizer. [1]

Clinical Trials 40

Clinical Trials Treatment Trials FDA 40

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.

Small Molecule 127

Small Molecule Drugs Immune Response Targeted Protein Degradation 127

The Pharma Data

SEPTEMBER 15, 2020

The prophylactic steroid treatment was also changed from 1 mg/kg to an intermediate dose of 2mg/kg. The most frequent treatment-emergent adverse events were nasopharyngitis and atopic dermatitis.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

Drug Target Review

APRIL 11, 2025

This promises to significantly improve the probability of success for new drugs, accelerating their development and ultimately delivering life-saving treatments to patients faster. Traditional drugs for eye diseases like glaucoma are small molecules delivered by simple eye drops.

Clinical Development 72

Clinical Development Drugs Pharmaceuticals Clinical Trials 72

Drug Target Review

NOVEMBER 7, 2024

This targeted enhancement not only boosts the body’s natural mechanisms but also holds promise for developing novel treatments in immunology, where modulating immune responses can lead to more effective and durable therapeutic outcomes. References 1 Tinoco R, Otero DC, Takahashi AA, Bradley LM. Trends in immunology , 38(5), 323-335.

Disease 52

Disease Immune Response Regulations Engineering 52

Drug Target Review

FEBRUARY 15, 2024

Protection from a vaccine may not be durable or complete, and persons may still require treatment with medications for their infection. 11 Health agencies globally must collaborate with academic research centres and industry to address this important gap in drug treatments for pandemic-prone pathogens. 2017 Apr 10;114(16):4031–3.

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