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These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 3D rendering of Antibody Drug Conjugate Molecules.
Metabolism of 2022 FDA approved smallmolecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 smallmolecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. References Iversen et al., link] Tang e t al.,
Metabolism of 2023 FDA Approved SmallMolecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 smallmolecules out of a total of 55 new drugs [1]. References [1] 2023 Novel SmallMolecule FDA Drug Approvals. Blood 129(13): 1823-1830.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.
The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions. References Sun D, et al. Acta Pharmaceutica Sinica B , 12(7), pp.
recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Smallmolecule GLP1s? Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. Additional trials (e.g.,
Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a smallmolecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.
2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 2020 Jan 1;57(1):372–92. References: Ninou I, Magkrioti C, Aidinis V.
15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. Conference Call Information : Date : Thursday, November 5, 2020. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.
Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists.
We are now committed to pursuing other CGRP-mediated diseases through advancing novel investigational agents such as HTL0022562 into human studies.”
TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,
As part of Aptorum Group’s Acticule infectious disease platform, ALS-4 is a novel orally administered first-in-class smallmolecule that was developed based on an anti-virulence approach targeting Staphylococcus aureus including MRSA. About ALS-4.
20, 2020 /PRNewswire/ — Sosei Group Corporation (“the Company”) (TSE: 4565) announces a progress update for its R&D program focused on the design and development of novel drugs targeting the SARS-CoV-2 coronavirus and to treat COVID-19. The program was initiated in April 2020. References. Hilgenfeld, Febs J.
Applying our technology has enabled us to develop a new smallmolecule, TT125-802, and to assign a new role to the epigenetic regulator CBP/p300 as a novel master regulator of non-oncogene resistance. This orally available smallmolecule binds to the bromodomain of CBP/p300 in a highly specific manner.
21, 2020 11:00 UTC. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented: “Further to our previous update in September 2020, we are pleased to announce this CTA submission which represents a major development milestone for our ALS-4 antimicrobial program. About ALS-4.
Smallmolecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Multiple System Atrophy.
BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.
Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted smallmolecule therapeutics. This is because many proteins do not have small-molecule binding sites. This is not the first deal for RNA therapeutics for Genentech.
A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.
Presentations to highlight the OBI-833 Phase 1 clinical study results in non-small cell lung cancer (NSCLC) and the dose escalation cohort. 19, 2020 /PRNewswire/ — OBI Pharma, Inc. Session Date and Time: Friday, November 20, 2020. Session Date and Time: Friday, November 20, 2020. TAIPEI, Taiwan , Nov.
AL01811 is a preclinical selective GBA2 inhibitor with first-in-class potential as an oral disease modifying treatment for Parkinson’s Disease Alectos to receive a $15 million upfront payment and is eligible to receive potential future development and commercial milestone payments. Biogen Inc. President and CEO at Alectos Therapeutics.
30, 2020 07:00 UTC. Headquarters: Cambridge, UK, “Wren”) today announced that the companies have entered into an exclusive research collaboration agreement aiming to advance the discovery of novel smallmolecules that target ? synuclein, which is associated with the onset and progression of these diseases.
5, 2020 11:00 UTC. NASDAQ: CNCE) today reported financial results for the third quarter of 2020. Our team has diligently and effectively executed on our plan, and I’m pleased that we have achieved our stated 2020 pipeline milestones.
Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. LEXINGTON, Mass.–(
23, 2020 13:30 UTC. SAN DIEGO–( BUSINESS WIRE )– Vividion Therapeutics , a biotechnology company discovering and developing selective smallmolecule medicines that drug traditionally inaccessible targets, today announced that Jeffrey Hatfield has been appointed as the company’s chief executive officer.
3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. AbbVie hosted an Immunology Strategic Update event on December 14, 2020 for members of the investment community. NORTH CHICAGO, Ill., Gonzalez , chairman and chief executive officer, AbbVie.
12, 2020 14:00 UTC. As an orally bioavailable smallmolecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”.
LYT-100 is an anti-fibrotic and anti-inflammatory oral smallmolecule that has been designed to overcome the tolerability challenges associated with pirfenidone, an anti-inflammatory, anti-fibrotic drug currently on the market. The trial is ongoing in the U.S. and Europe, and PureTech Head of Innovation, Dr. Michael C.
This donation of the vaccine is another tool in our toolbox of measures to help make the Olympic and Paralympic Games Tokyo 2020 safe and secure for all participants and to show solidarity with our gracious Japanese hosts,” said IOC President Thomas Bach. “We This press release features multimedia. View the full release here: [link].
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
Dr. Vickers takes the helm of Faze shortly after the company launched in December 2020 with an $81 million Series A financing. Preclinical and clinical programs at Northern Biologics were acquired by big pharma companies in 2020. In addition to his role as CEO, Dr. Vickers will also join Faze’s board of directors. “We
When Chinook Therapeutics looks at 2021, it foresees a host of clinical trials that will bring precision medicine to the kidney disease space. Chinook’s nearest milestone is the initiation of a 300-patient Phase 3 ALIGN trial of atrasentan for IgA nephropathy (Berger’s disease) that is expected to begin early this year.
Chemistry42 is a customisable and fully automated software platform launched in 2020 that integrates generative AI algorithms with computational and medicinal chemistry methods to generate novel molecules with drug-like properties, sometimes in a matter of days. How was ISM6331 generated and optimised using Chemistry42?
16, 2020 14:00 UTC. Validated technology platform translates novel molecules from plants into first-in-class medicines. Leveraging this database of hundreds of thousands of unique chemicals and their links to biological signatures, Enveda’s machine learning algorithms predict new chemicals with the potential to treat complex diseases.
26, 2020 /PRNewswire/ — PTC Therapeutics , Inc. NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease.
Program focused on identifying smallmolecules that target a GPCR for degradation as potential therapeutic agents for gastrointestinal disorders.
The principle of TPD is to use smallmolecules to commit the target protein into the E3 ligase-mediated degradation pathway thereby eliminating or reducing its activity.
Dedicated rare disease unit to be headquartered in Boston. 12, 2020– AstraZeneca and Alexion Pharmaceuticals, Inc. Pascal Soriot , Chief Executive Officer, AstraZeneca , said: “ Alexion has established itself as a leader in complement biology, bringing life-changing benefits to patients with rare diseases.
2, 2020 08:00 UTC. 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. This press release features multimedia. View the full release here: [link].
16, 2020 09:20 UTC. The first-in-class degraders developed by Cullgen efficiently degrade mutated, disease causing forms of the TRK protein and subsequently inhibit cancer cell growth. Hence, Cullgen’s TRK degraders could offer patients a new therapeutic approach to treat TRK-mediated cancer diseases with reduced drug resistance. “We
Comparing to Non-Small Cell Lung Cancer(NSCLC), it is still not clear about tumor driving gene in the development of SCLC, and thus it is lacking of targeted therapeutic treatment for this life threaten disease. SH ) was founded in Shenzhen on 2001, specializing in the research and development of novel smallmolecule drugs.
The objective of the Phase 3 clinical trial is to evaluate safety and effectiveness of 18 F-APN-1607 to differentiate patients with Mild Cognitive Impairment (MCI) and different stages of Alzheimer’s disease (AD) from healthy subjects. SUZHOU, China , Oct.
15, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) and MBC BioLabs today announced that BigHat Biosciences and Trilo Therapeutics have each been awarded an Amgen Golden Ticket to MBC BioLabs. Our Degradomer® class of targeted protein degraders expands on the Trilomer® concept, allowing highly specific degradation of disease-causing proteins.
11, 2020 (GLOBE NEWSWIRE) — Calithera Biosciences, Inc. , (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel smallmolecule drugs for the treatment of cancer and other life-threatening diseases, today announced its participation at two upcoming healthcare investor conferences in November.
11, 2020 (GLOBE NEWSWIRE) — Lexicon Pharmaceuticals, Inc. LX9211 is a potent, orally delivered, selective smallmolecule inhibitor of adapter-associated kinase 1 (AAK1). THE WOODLANDS, Texas, Dec. Nasdaq: LXRX), announced today that it has received Fast Track designation from the U.S. About LX9211.
The offering is being made pursuant to a shelf registration statement that was filed with the Securities and Exchange Commission (“SEC”) on June 12, 2020 and declared effective by the SEC on June 22, 2020. Syros is redefining the power of smallmolecules to control the expression of genes. About Syros Pharmaceuticals.
13, 2020 /PRNewswire/ — Senhwa Biosciences, Inc. Silmitasertib is a first-in-class smallmolecule drug that targets CK2 and acts as a CK2-inhibitor. In July 2020 , Silmitasertib was granted Rare Pediatric Disease Designation to treat Medulloblastoma by the U.S. TAIPEI and SAN DIEGO , Dec.
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