Drug Discovery World

NOVEMBER 8, 2023

Deepika Khedekar , Associate Centralized Clinical Lead at IQVIA, explains how artificial intelligence could make clinical trials more accurate, accessible and standardised. Tapping into the capabilities of AI One reason AI may accelerate clinical trials is its ability to rapidly process and analyse vast amounts of data.

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Drug Discovery World

MAY 26, 2023

AM1476, a highly selective small molecule, delivered as an orally administered tablet, was found to be safe and well-tolerated at all relevant doses in a trial of approximately 100 healthy subjects. There are currently no treatments on the market that effectively reverse scarring.

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Drug Discovery World

AUGUST 14, 2023

Research partners in the UK are progressing their orally available small molecule cancer immunotherapy drug into clinical development as a treatment for advanced solid tumours. Chief Investigator Dr Bristi Basu, University of Cambridge, and Co-Chief Investigator Dr Debashis Sarker, King’s College London, are leading the trial.

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Drug Discovery World

JUNE 26, 2024

Spinogenix has initiated a Phase II trial to evaluate SPG302 for the treatment of adult participants with mild-to-moderate Alzheimer’s disease (AD), following approval for the trail in Australia. SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases.

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Drug Discovery World

MARCH 8, 2023

Trop2 is a validated drug target with one approved drug for the treatment of solid tumours and is a target of high interest for new drug discovery. The post Company launches ADC Phase I trial in solid tumours appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 15, 2024

We believe that the results from the Phase Ib MAD study directly support larger scale Phase II clinical development to more fully understand the potential for ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases.”

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Drug Discovery World

JUNE 1, 2023

Based on the positive DMC review and confirmed financial commitment from its investors, the company will expand the trial to Belgium, France, and Italy. AP-325 is a small molecule drug candidate that binds and modulates the GABAA receptor, reducing the excitability of the respective neurons.

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Drug Discovery World

SEPTEMBER 12, 2024

Childhood cancers often have a different biology than those in adults, yet targeted treatment options are mostly based on adult therapies. In addition, small patient populations make clinical trials difficult and reduce incentives for investment.

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Drug Discovery World

NOVEMBER 9, 2023

The positive human qEEG biomarker results with apimostinel are consistent with prior biomarker observations with zelquistinel, Gate’s lead programme, which is currently being developed as a rapid-acting, once weekly, oral treatment for major depressive disorder (MDD). “We

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Drug Discovery World

AUGUST 15, 2024

A Phase IIa clinical trial has begun evaluating the safety and efficacy of Pelage Pharmaceuticals’ PP405, a novel topical small molecule, for the treatment of androgenetic alopecia (pattern baldness). The Phase IIa trial is a randomised, placebo-controlled study of PP405 in women and men with androgenetic alopecia.

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Drug Discovery World

MARCH 15, 2023

The primary endpoint has been achieved in a Phase II trial of AP30663, a first-in-class SK ion channel inhibitor for conversion of atrial fibrillation (AF) to normal sinus rhythm. Acesion Pharma enrolled 63 patients with a current episode of AF in the trial and tested two intravenous doses of AP30663, 3 and 5mg/kg, compared to placebo.

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Drug Discovery World

MAY 30, 2024

A new research paper has validated the mechanism of action of the Atrogi’s first-in-class, small molecule, ATR-127, in combating obesity and metabolic complications. We are now advancing our next generation small molecules for the treatment of obesity to IND preparatory studies in H1 next year and expect to enter the clinic in 2026.”

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Drug Target Review

OCTOBER 11, 2023

Most of these conditions are genetic in origin and the majority have no effective treatment. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. That includes countless rare peripheral diseases, including many for which there are currently no treatments.

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Drug Hunter

AUGUST 15, 2023

Clinical advancement of LRRK2 inhibitors was initially stalled by concerns about on-target lung findings in primates, but these were ameliorated by a Merck/Genentech/Pfizer/MJFF study showing that these lung changes were reversible, and Biogen/Denali has currently a small molecule (BIIB122/DNL151) in Ph. I ( NCT04557800 ).

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Drug Discovery World

MARCH 15, 2024

The top stories: New drug shows promising neuroprotective effects Galimedix Therapeutics has announced data showing small molecule GAL-201 has neuroprotective and symptomatic alleviation potential in models of Alzheimer’s disease (AD). million raised to progress Parkinson’s treatment Mission Therapeutics has raised £25.2 ($32.1)

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Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza.

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Drug Discovery World

AUGUST 16, 2024

Misetionamide is a tumour cell selective small molecule drug that is broadly active in multiple cancer models. New published data have demonstrated positive monotherapy results for misetionamide as well as synergistic activity with PARP inhibitors (PARPis) and bevacizumab antineoplastic in ovarian cancer.

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Drug Discovery World

MARCH 26, 2024

Additional approaches, such as profiling of RNA and proteins, and the use of artificial intelligence (AI), could be key to providing a more comprehensive picture of a patient’s tumour and enable the use of optimised treatments. MT: Why was STORM attending the conference? We were there to present the latest findings from our research.

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The Pharma Data

JANUARY 19, 2021

Precision medicine developer Kura Oncology is positioned to make a meaningful difference in the treatment of cancer in the next few years with its two, wholly-owned, small molecule compounds. Kura Oncology CEO Troy Wilson pictured above. Photo courtesy of Kura. Tipifarnib is a farnesyltransferase inhibitor (FTI).

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Drug Discovery World

FEBRUARY 12, 2024

Cameron Ross, SVP, Generative AI, Elsevier “AI has the potential to dramatically shorten one of the most costly areas of early drug development – small molecule discovery. We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials.

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PPD

JUNE 27, 2024

In recent years, AA has followed in the footsteps of other dermatology indications — including psoriasis and atopic dermatitis — with growing research and clinical studies focusing on small molecules with broad cellular effects (JAK and PDE4 inhibitors) and cytokine-specific molecules antagonists (IL-23, Th2 and IL17) to treat the condition.

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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

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Olympian Clinical Research

MAY 6, 2024

The journey toward effective treatments has been long and evolving, with recent breakthroughs offering new hope to those living with this challenging condition. From Coal Tar to Biologics: A Historical Perspective The history of psoriasis treatment is as old as the condition itself, with records dating back to ancient times.

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Drug Discovery World

MARCH 12, 2024

Lastly, for treatment of substance use disorders, few pipeline drugs are currently undergoing clinical trials, yet those in trial only address the withdrawal symptoms but not the underlying reward circuitry and the cravings. RA: What opportunity is there for better treatments in pain and mental health conditions?

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Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 27, 2023

. “The NMPA approval of vebreltinib is an important milestone toward providing a new treatment option for patients with MET exon 14 skipping NSCLC in China. Vebreltinib is a potent, small molecule, orally bioavailable and highly selective c-Met inhibitor.

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.

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DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

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Drug Discovery World

NOVEMBER 11, 2022

BioSenic’s arsenic trioxide (ATO) autoimmune disease platform has completed a successful Phase IIb trial in chronic Graft vs Host Disease (cGVHD) and is preparing to move to a Phase III trial. . The Phase II trial demonstrated efficacy of more than 75% and the Phase III trial is expected to receive accelerated examination by the FDA.

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ProRelix Research

NOVEMBER 23, 2022

Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with small molecule, chemically synthesized drug molecules remain suboptimal. […].

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Drug Discovery World

MARCH 15, 2023

CADD522 is a small molecule which blocks the RUNX2 protein from having an effect. First new treatment in 45 years Lead researcher Dr Darrell Green, from UEA’s Norwich Medical School, was inspired to study childhood bone cancer after his best friend died from the disease as a teenager.

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Drug Discovery World

NOVEMBER 10, 2023

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). There was a mean improvement in visual acuity of 0.30

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Drug Discovery World

MARCH 12, 2024

Advancements in antibody-drug conjugates are also expected, enhancing targeted delivery for cancer treatments.” Carole Nicco, CSO, BioSenic “mAb immunotherapy is the most widely used approach and has achieved tremendous success. Other ACTs such as TIL, TCR, NK cell, Treg, and MDSC therapies are now emerging.

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.

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Drug Discovery World

OCTOBER 4, 2022

Using its proprietary compound screening platform, Oligomerix discovers and develops differentiated, oral, small molecule inhibitors of tau self-association that are easy to administer and cost-effective. . With its recent award of $3.35 With its recent award of $3.35 Focused on opportunity .

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Drug Hunter

JUNE 26, 2023

This article compiles recent high-profile clinical readouts and related news with small molecules of general interest and structures where they are available. III trial ( NCT05099640 ; n=98, 7.5-60mg/kg Get ahead now by requesting a trial. III in Phenylketonuria sepiapterin (PTC923, CNSA-001) oral BH 4 precursor 7.5-60mg/kg

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Drug Discovery World

JULY 17, 2023

IRBM, Rainwater Charitable Foundation and Weill Cornell Medicine will start of a multi-year drug discovery collaboration to identify small molecule cGAS (cyclic GMP-AMP synthase) inhibitors for the treatment of tauopathies and other neurodegenerative diseases.

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