Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. Blood 129(13): 1823-1830.

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Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. References Iversen et al., Curr Drug Metab. 20(4): 254-265.

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LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). 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? Small molecule GLP1s? Join the club. Additional trials (e.g.,

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Broad Institute

NOVEMBER 2, 2023

Combining Broad’s expertise in cancer biology and state-of-the-art drug discovery methods with Bayer’s expertise in drug development greatly increases our power to bring transformative medicines to cancer patients.” “We As a result of this collaboration, Bayer’s mutant EGFR/HER2 inhibitor is currently in Phase I of clinical trials.

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The Pharma Data

NOVEMBER 30, 2020

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. Vlad Coric , M.D.,

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The Pharma Data

NOVEMBER 19, 2020

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 small molecules against predicted future variants of SARS-CoV-2 and other related human viruses.

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The Pharma Data

MAY 31, 2022

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.

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LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? I will note that “de-risked” is never fully risk-free in therapeutics investing, as even precedented mechanisms hit stumbling blocks in preclinical or clinical development.

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The Pharma Data

DECEMBER 15, 2020

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.

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Drug Target Review

JULY 18, 2023

AI is being used to find the targets themselves, design the drugs to manipulate that biology, and thirdly, AI is being used to support validation of those targets and drugs as part of pre-clinical development. Cavlan explains that “they are brilliant at developing and bringing new drugs to market.”

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The Pharma Data

NOVEMBER 11, 2020

“These data provide further evidence of efficacy and high potency of SLV213 against SARS-CoV-2 and support the clinical development of SLV213 as a potential oral treatment for COVID-19,” said Ted Daley, President and CEO, Selva Therapeutics. “As Viruses work by infecting host cells and hijacking the cell’s replication machinery.

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The Pharma Data

SEPTEMBER 29, 2020

It is a synthetic small molecule that would be self-administered via nasal spray, taken once or twice a week for the prevention of COVID-19. from Australian investors to progress the INNA-051 clinical development programme. So far, Ena has raised AU$11.7m Source link.

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The Pharma Data

JANUARY 13, 2021

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma.

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. Xofigo is currently under further evaluation in a broad clinical development program in prostate cancer and beyond.

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The Pharma Data

DECEMBER 10, 2020

executive vice president of research and development. “We We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible.”. About LX9211. Safe Harbor Statement.

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The Pharma Data

MAY 15, 2023

The acquisition complements Gilead’s existing clinical development priorities by adding additional pipeline assets for well-validated targets in oncology and inflammation. About XinThera XinThera is a private biotechnology company whose mission is to develop best-in-class small molecule drugs to treat cancer and immunologic diseases.

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The Pharma Data

SEPTEMBER 25, 2020

Monte Rosa Therapeutics has raised $96m in Series B financing to support further develop of its pipeline of small-molecule protein degraders. ICR and Cancer Research UK spinout closes Series B financing. Source link.

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The Pharma Data

OCTOBER 27, 2020

The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases,” Cumbo said. and China. said of B cells’ potential. “We

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The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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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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The Pharma Data

APRIL 6, 2021

Approvals were based on the overall response rate (ORR) from the open-label, single-arm Phase II CHRONOS-1 (NCT01660451) trial of copanlisib monotherapy in 104 adult patients with follicular B-cell NHL who had relapsed disease following at least two prior systemic therapies. Filings in other regions are underway or planned.

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The Pharma Data

FEBRUARY 21, 2022

At its annual Pharma Media Day 2022, Bayer presented the latest developments in the ongoing transformation of its pharmaceuticals business, which is aimed at delivering long-term, sustainable business growth by bringing forward new options for patients. “We

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The Pharma Data

MARCH 1, 2022

The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.

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The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group.

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LifeSciVC

MARCH 1, 2023

They also highlighted why targeting the CD28 pathway overcomes some of these challenges and why CBL-B modulation, downstream of CD28 and other signaling receptors with clinical validation, may be more advantageous.

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The Pharma Data

NOVEMBER 10, 2020

11, 2020 (GLOBE NEWSWIRE) — Calithera Biosciences, Inc. , (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, today announced its participation at two upcoming healthcare investor conferences in November.

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Alta Sciences

APRIL 17, 2024

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. It is a growing field where new technology, and an increasing understanding of the science, are bringing advancements in drug development. Read The Altascientist , “Nonclinical Studies in Cell and Gene Therapy”. 3.

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The Pharma Data

JANUARY 7, 2021

Prener has led companies and teams across several therapeutic areas, including a focus on rare diseases. a gene therapy company focused on eye diseases. Before that, Dr. Prener was Global Therapeutic Area Head of Hematology and Vice President, Clinical Research Hematology at Baxalta.

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The Pharma Data

JANUARY 19, 2021

Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 CAMBRIDGE, Mass., Forward-Looking Statements.

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The Pharma Data

DECEMBER 12, 2020

Dedicated rare disease unit to be headquartered in Boston. 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. This acquisition allows us to enhance our presence in immunology.

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The Pharma Data

JANUARY 19, 2021

Its lead candidate is CLN-081, an oral small molecule designed to be a next-generation, irreversible EGFR inhibitor. The compound was developed in the laboratory of Richard Childs, chief of the Laboratory of Transplantation Immunotherapy with the National Heart, Lung, and Blood Institute (NHLBI).

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The Pharma Data

JANUARY 3, 2021

Based on the strong scientific rationale for telaglenastat in KEAP1/NRF2 mutant non-small cell lung cancer patients, and the safety profile observed in CANTATA, we remain dedicated to advancing our randomized KEAPSAKE trial.”. SOUTH SAN FRANCISCO, Calif., Calithera is headquartered in South San Francisco, California.

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The Pharma Data

JANUARY 10, 2021

“We expect 2021 will mark a number of key clinical and commercial milestones. By the end of the year, we expect to have five independent TransCon product candidates in clinical development leveraging TransCon technologies through our algorithm for product innovation. Pipeline Updates. •. .

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The Pharma Data

OCTOBER 14, 2020

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. TNX-102 SL is also in development for agitation in Alzheimer’s disease and alcohol use disorder (AUD).

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The Pharma Data

AUGUST 29, 2020

Seven of ten patients discontinued eculizumab and remained on LNP023 as monotherapy, retaining hemoglobin (Hb) levels with no changes in biomarkers of disease activity and with no signs or symptoms of breakthrough hemolysis . Peffault de Latour.

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Advarra

FEBRUARY 24, 2023

In short, FDA is acknowledging this is complex area, so make sure all clinical development and biostatistical experts are well-versed on how to design these types of trials to ensure startup and/or regulatory approval is not delayed.

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