This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
Smallmolecule drugs make up most of the drugs we take conveniently as pills, including painkillers like ibuprofen (Advil), antibiotics like penicillin and amoxicillin, or cholesterol-lowering drugs like atorvastatin (Lipitor). The smallmolecules drugs of today look nothing like the molecules of the 1970s.
By accelerating timelines, anticipating challenges and alleviating bottlenecks, pharmaceutical companies and combined CDMO and CRO partners together can help ensure those potential treatments reach the patients who need them as efficiently as possible.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. 2 However, when dosed at the MTD, ADCs display improved efficacy over smallmolecules in oncology trials. 3D rendering of Antibody Drug Conjugate Molecules.
In May 2025, Altasciences attended CPHI Americas in Philadelphia, where industry leaders, innovators, and stakeholders gathered to explore the latest advancements in pharmaceutical development technologies and connect with providers. Why does that matter? Beyond the science, what’s exciting is how this technology opens doors. The response?
Clinical genomics, powered by NGS, enables more precise target validation, improved patient stratification and optimised trial design, ultimately aiming to increase PoS. Only in this way can AI reach its full potential in accelerating drug discovery and improving trial success rates. Highlighting data integration.
Orforglipron ( LY-3502970 ) is an oral, non-peptide, small-molecule GLP-1 receptor agonist developed as a weight loss drug by Eli Lilly and Company. [1] 1] It was discovered by Chugai Pharmaceutical Co. 1] It was discovered by Chugai Pharmaceutical Co. 1] It was discovered by Chugai Pharmaceutical Co.
As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted smallmolecules aimed at revolutionising cancer treatment. Additionally, we are preparing to launch several other trials over the coming months.
By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new smallmolecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.
It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. Because LLMs are trained on extensive, internet-scale datasets, they can learn to identify contexts linking words and language.
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional smallmolecules. 1 The concept of TPD was first demonstrated with a heterobifunctional smallmolecule to degrader a protein of interest in the early 2000s.
It is a smallmolecule delivered orally. [1] mmol) was added tert-butyl piperidin-4-ylcarbamate (841 mg, 98% Wt, 1.2 mmol) and DIPEA (1.19 mL, 3 Eq, 10.3
The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4. About Aptorum Group.
By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.
Bayer and Tsinghua University Deepen Strategic Alliance to Advance Pharmaceutical Innovation in China Bayer and Tsinghua University (THU), two powerhouses in global pharmaceutical innovation and academic research, have announced a renewed commitment to their long-standing partnership.
Symptoms were reversed in mouse models and a clinical trial is planned for later this year. 5 This drug candidate is progressing towards the clinic, with phase 1 trials due to start within 18 months. Autophagy boosters, in contrast, are conventional smallmolecule drugs. References England Rare Diseases Action Plan 2022.
Our joint goal is to bring innovation to cancer patients by building a robust, sustainable pipeline in oncology,” said Dominik Ruettinger, Global Head of Research and Early Development for Oncology, Pharmaceuticals Division, Bayer AG.
Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists.
The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials.
Developing pharmaceuticals is a complex process, requiring stringent bioanalytical method validation guidelines to ensure drug safety and efficacy. To help integrate these various regulatory standards, the International Council for Harmonization (ICH) of Technical Requirements for Pharmaceuticals for Human Use was founded in 2016.
The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral smallmolecules against predicted future variants of SARS-CoV-2 and other related human viruses.
The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinical trials. We have several exciting projects in various stages of discovery, but a small-molecule drug that is causing a lot of excitement at the moment is Capivasertib.
1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] The FDA granted approval of Journavx to Vertex Pharmaceuticals Incorporated.
However, in tissues where the 3 Hs are not present, the smallmolecule has anti-toxicity activity through 1) direct inhibition of the NLRP3 inflammasome, an inflammation starter and perpetuator, and 2) upregulation of the master antioxidant transcription factor, Nrf2. References Oronsky B, Scribner C, Aggarwal R, Cabrales P.
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.
Process validation is a critical regulatory requirement in pharmaceutical manufacturing. In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial smallmolecule or biologic product to expedite product availability for patients.
–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?) CAMBRIDGE, Mass.–(BUSINESS
Between 2000 and 2020, approximately 30 percent of the newly introduced smallmolecule drugs were derived from natural products. In short, this hypothesis underscores the potential benefits of aligning pharmaceutical development more closely with nature.
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. .
SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,
Each fall, leading pharmaceutical patent litigators for brand-name and generic drug companies gather at the Paragraph IV Disputes Master Symposium in Chicago to receive up-to-the-minute information on the latest developments affecting Hatch-Waxman litigation and participate in significant peer-to-peer networking opportunities. s Kurt R.
15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. About Eloxx Pharmaceuticals. Eloxx Pharmaceuticals, Inc. Eloxx’s lead investigational product candidate, ELX-02, is a smallmolecule drug candidate designed to restore production of full-length functional proteins. SOURCE: Eloxx Pharmaceuticals, Inc.
Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.
11, 2020 (GLOBE NEWSWIRE) — Lexicon Pharmaceuticals, Inc. Lexicon is currently enrolling patients with diabetic peripheral neuropathic pain in a Phase 2 proof-of-concept study of LX9211 and is preparing to initiate a second Phase 2 clinical trial of LX9211 in post-herpetic neuralgia. About Lexicon Pharmaceuticals.
Also, many companies never reached the point where they received validation from big pharmaceutical companies. That would provide wonderful information as we think about then going into a first-in-human clinical trial. Just like smallmolecules and antibodies, cell therapies are changing how we treat patients.
–( BUSINESS WIRE )– Concert Pharmaceuticals, Inc. President and Chief Executive Officer of Concert Pharmaceuticals.
Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The trial will evaluate 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo at sites in the U.S., 5, 2020 11:00 UTC.
Retrieved 9 July 2025. ^ “KalVista Pharmaceuticals Announces FDA Approval of Ekterly (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema” (Press release). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87” WHO Drug Information.
Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).
If we look at the pharmaceutical industry’s discovery and development success rate over the last 20 to 30 years, it has not improved, staying stubbornly at around 10 percent. With support from pharmaceutical companies, ultimately, the answer to this question should be yes. Will systems like the NHS be able to deliver it?
Statistically significant improvements were achieved in all key secondary endpoints in the trial as well. Etrasimod was developed by Arena Pharmaceuticals, which was recently acquired by Pfizer. The safety profile was consistent with previous Phase 2 studies. Results from the ELEVATE 52 study will be available by the end of Q1.
27, 2020 /PRNewswire/ — Aridis Pharmaceuticals, Inc. Dr. Hasan Jafri , Chief Medical Officer of Aridis Pharmaceuticals, will be a speaker on a panel entitled “Direct Antivirals and Other Agents Against SARS-CoV2 Virus.” About Aridis Pharmaceuticals, Inc. Aridis Pharmaceuticals, Inc. SAN JOSE, Calif. ,
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. The proposed trial will enroll approximately 230 subjects with all receiving 18 F-APN-1607.
Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational smallmolecule developed by Abbisko Therapeutics Co., In this context, the potential approval of pimicotinib could address a large and unmet medical need , especially in China where treatment options remain limited.
With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. 2021 – and a robust pipeline that includes 3 AI-designed drugs (for IPF, COVID-19 and cancer) currently in clinical trials.
My last stop at Arrakis Therapeutics is with a company targeting RNA with smallmolecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like smallmolecules.
The results provide an opportunity for the company to pursue a pivotal Phase 2/3 clinical trial in patients with primary biliary cholangitis (PBC), based on interactions with the FDA. The trial consisted of a single ascending dose (SAD) part and a multiple ascending dose (MAD) part with dosing up to 1600mg/day. About Calliditas.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content