Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

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Drug Discovery World

DECEMBER 4, 2023

Smoking continues to be the leading cause of preventable death and disease, leading to almost eight million direct and indirect deaths annually. The results from Qnovia’s first in-human study demonstrate a superior pharmacokinetic profile compared to existing NRTs. “We

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Drug Discovery World

OCTOBER 10, 2023

Rejuvenate Biomed, a clinical-stage company advancing therapeutics to delay the onset of age-related diseases, has completed a six-week clinical proof-of-mechanism study with lead candidate RJx-01 for the treatment of sarcopenia.

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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. The study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical efficacy of SPG302 in adult AD participants.

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Drug Target Review

DECEMBER 12, 2023

We use hydrophilic linkers, which prevent ADC aggregation and generate highly stable ADCs, in combination with a unique attachment site on the antibody to create ADCs that retain pharmacokinetic properties similar to the original unconjugated antibody. This helps to maximize the targeted payload delivery to tumor cells.

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Drug Discovery World

DECEMBER 12, 2023

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Drug Discovery World

JANUARY 10, 2024

Tony Wood, Chief Scientific Officer, GSK, said: “We have a proud heritage and deep development expertise in respiratory medicines, especially addressing diseases driven by IL-5 with high levels of eosinophils or high T2 inflammation. TSLP is a clinically validated target in the treatment of asthma regardless of biomarker status.

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Drug Discovery World

NOVEMBER 10, 2023

Dense aggregates of immune cells, including CD8 positive granzyme B positive cytotoxic T cells, dendritic cells, and B cells, were observed in PDAC tissue after CAN-2409 treatment, confirming activation of a robust antitumoral immune response.

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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Drug Discovery World

AUGUST 13, 2024

They are assumed to closely mimic human disease progression and act as a good testing ground for toxicology studies and pharmacokinetics 1. A lack of translatability The most obvious argument against the continued blanket requirement for AMs is that they are not very translational for many disease areas.

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Drug Discovery World

APRIL 16, 2024

ALS is a fatal neurodegenerative disease affecting 50,000 individuals in Europe at any time, resulting in 10,000 deaths each year. Currently, there is no cure for ALS and the only approved drug in the EU for the disease is Riluzole, prolonging survival for a median of just two months.

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Drug Discovery World

FEBRUARY 2, 2024

The study aims to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary anti-tumoural activity of OT-A201. The findings were published in the New England Journal of Medicine.

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Drug Discovery World

MARCH 8, 2024

The study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of IBI3002 in healthy participants and participants with mild to moderate asthma. The potential synergistic effect in suppressing T2 inflammation is believed to be the basis of its superior efficacy in the treatment of T2 inflammatory disorders.

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Drug Discovery World

SEPTEMBER 6, 2022

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Avacta Group’s chemotherapy drug, AVA6000, for the treatment of soft tissue sarcoma. . Despite the successful advancement of localised therapies, such as surgery and radiotherapy, these tumours can recur, often with metastatic disease. .

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Drug Discovery World

JUNE 27, 2023

Alchemab Therapeutics, an antibody discovery company identifying naturally occurring antibodies from individuals resilient to disease, has unveiled data on ATLX-1088, its newly-discovered preclinical Alzheimer’s candidate. The data was announced at the Antibody Industrial Symposium in Tours, France, June 22-23, 2023.

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Drug Discovery World

DECEMBER 21, 2022

Biopharmaceutical company Rezolute has initiated a Phase II proof-of-concept study for RZ402, a plasma kallikrein inhibitor (PKI) being developed as an oral therapy for the treatment of diabetic macular oedema (DMO). . The post Oral therapy for diabetic macular oedema progresses to Phase II appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 10, 2023

The approval is in patients with locally advanced or metastatic urothelial cancer (la/mUC) who have received prior treatment with a PD-1/L1 inhibitor and platinum-based chemotherapy. “In

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Drug Discovery World

MAY 24, 2023

The ongoing global Phase I/II clinical trial is a multicentre, open-label study designed to assess the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants aged one year and older with genetically confirmed PA. Currently, there are no effective therapies for PA that target the underlying root cause of the disease.

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Drug Discovery World

JANUARY 9, 2023

Uveal melanoma is a disease in which cancer originates in the tissues of the eye, causing symptoms such as blurred vision or a dark spot on the iris. . When the cancer metastases, which it does in approximately 50% of patients, there are limited treatment options and projected overall survival is only a year. .

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Drug Discovery World

DECEMBER 14, 2023

Antibody drug conjugate MBRC-101 MBrace Therapeutics presented preclinical data demonstrating the potential of its novel antibody drug conjugate MBRC-101 for the treatment of solid tumours including hormone receptor-positive (HR+) and triple negative breast cancers. months of additional follow-up and 78.3% The results show a 25.1%

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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. In January 2023, QN-302 received Orphan Drug Designation by the FDA for the potential treatment of pancreatic cancer. This deadly cancer has a 5-year survival rate of just 7%.

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Drug Discovery World

JULY 25, 2023

The pharmaceutical industry is constantly developing evidence-based treatments for children while there continue to be scientific advancements related to new modalities and disease understanding. The voluntary pilot scheme will start with eight sPIPs and will target medicines where elements of the PIP cannot be defined.

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Drug Discovery World

NOVEMBER 20, 2023

They are well suited to rapidly responding to emerging infectious disease threats – a growing need as the rate of pandemic-capable diseases increases 6. Their speed also allows for their potential use in personalised therapy for diseases such as cancer.

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Drug Discovery World

MAY 31, 2023

In the Phase I/II trial, three of the four tumour evaluable patients had a best response of stable disease, and two patients continue treatment as of the data cut-off date. This allows Teff and NK cells to expand and kill tumour cells.

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule 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.

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The Pharma Data

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). About Huntington’s disease. Survival ranges from approximately 10-20 years following motor onset of the disease.

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The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. Donations include the impact of treatment with bulevirtide, an investigational treatment for people with habitual hepatitis delta contagion (HDV) in theU.S.

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Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

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Drug Discovery World

SEPTEMBER 9, 2024

Vesper Bio has completed a Phase I study for its lead candidate, VES001, a potentially disease-modifying treatment for frontotemporal dementia patients with mutations in the progranulin gene (FTD(GRN)). Frontotemporal dementia is the most common cause of dementia in people under the age of 60.

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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. In January 2023, QN-302 received Orphan Drug Designation by the FDA for the potential treatment of pancreatic cancer. This deadly cancer has a 5-year survival rate of just 7%.

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ASPET

SEPTEMBER 7, 2023

Awareness of drug interactions involving opioids is critical for patient treatment as they are common therapeutics used in numerous care settings including both chronic and disease related pain. Opioids are the classical pain treatment for patients who suffer from moderate to severe pain.

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Drug Discovery World

JULY 11, 2024

The funding will help advance Mission’s potential disease-modifying treatment for Parkinson’s disease (PD), MTX325. for disease-modifying Parkinson’s treatment appeared first on Drug Discovery World (DDW). Mission Therapeutics has been awarded $5.2 million from The Michael J.

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The Pharma Data

AUGUST 19, 2021

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].

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The Pharma Data

MARCH 13, 2021

HAE is unpredictable, debilitating and potentially life-threatening, and recognition of the disease remains low in Japan, meaning there are significant challenges relating to diagnosis and access to effective therapies,” said Naoyoshi Hirota, General Manager, Takeda Development Center, Japan. Takeda in Hereditary Angioedema.

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The Pharma Data

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ?12 12 years of age with severe hemophilia A.

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The Pharma Data

NOVEMBER 5, 2021

With presently no approved treatments, there’s a major unmet need for curatives that can delay progression to order failure, “ said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis. In our hunt to find new drugs, we constantly rank among the world’s top companies investing in exploration and development.

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Drug Target Review

MARCH 6, 2024

Based on preclinical data, we expect broad opportunities in haematology and oncology for LSD1 inhibitors, pointing towards potential efficacy in SCLC and AML in combination with standard of care treatments. This results in the induction of leukaemic blast cell differentiation which can slow or stop the expansion of tumour cells.

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