Catalytic Protein Inhibitors
Covalent Modifiers
JANUARY 13, 2024
Thomas Kodadek Angewandte Chemie International Edition 2024 e202316726 [link] Many of the highest priority targets in a wide range of disease states are difficult-to-drug proteins.
Disease International Small Molecule 
Condensate biology: advancing drug discovery for complex diseases
Drug Target Review
JULY 13, 2023
In this article, we will delve into the world of condensate biology and explore the groundbreaking research projects undertaken to discover the potential they hold for diseases such as amyotrophic lateral sclerosis ( ALS ) and colorectal cancer. Klein notes the importance of Dewpoint’s disease-agnostic approach.
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Rare autoantibody diseases: an innovative targeted pathway
Drug Target Review
JULY 7, 2023
Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.
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Unlocking Undruggable Targets: Shifting Paradigms in Modern Drug Discovery
DrugBank
MAY 15, 2024
Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.
Targeted protein degradation: turning undruggable targets into druggable targets
Drug Target Review
JULY 6, 2023
TPD is a rapidly evolving therapeutic modality to degrade a disease-causing proteins, thereby eliminating their function specifically. 1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules.
Sentiment & Themes Emerging From JPM 2024
LifeSciVC
JANUARY 16, 2024
The expectation that Pharma will continue buy-ups here is helping to fuel conviction for another strong year ahead for M&A, especially for those acquirers who will rely on inorganic acquisition to build in areas under-represented by internal R&D (e.g., Small molecule GLP1s? Additional trials (e.g.,
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The power of combinations in blood cancers
Drug Target Review
NOVEMBER 6, 2023
Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers.
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Therapeutic frontiers: the potential of RRx-001 and AdAPT-001
Drug Target Review
AUGUST 25, 2023
However, in tissues where the 3 Hs are not present, the small molecule 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. Journal of Cancer Research and Clinical Oncology.
Sosei Heptares’ COVID-19 Program Identifies Potent Broad Spectrum Anti-Viral Small Molecules for Further Development
The Pharma Data
NOVEMBER 19, 2020
Program has advanced rapidly under an international collaboration of companies led by Sosei Heptares as part of its commitment to socially responsible investing. Sosei Heptares has successfully designed three distinct series of compounds with attractive anti-viral drug-like properties and advanced the most promising molecules.
Sosei Heptares and Biohaven Enter Global Collaboration and License Agreement to Advance Novel Small-Molecule CGRP Antagonist Portfolio
The Pharma Data
NOVEMBER 30, 2020
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.,
Advances in the Battle Against Autoimmune Disease
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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Deconstructing the Diligence Process: An Approach to Vetting New Product Theses
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? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?
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#WhyIScience Q&A: A research scientist uses mass spectrometry to discover how metabolites affect human health
Broad Institute
OCTOBER 17, 2023
Deik received her bachelor's degree in botany from Connecticut College and immediately went to work at International Paper, a paper manufacturing company, where she engineered trees to grow faster. A lot of what we do at the Metabolomics Platform is look for early indicators of disease and use metabolomics to monitor disease progression.
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Fearlessly Forward to our Destination
Dark Matter Blog
JANUARY 16, 2023
Our terra firma is small-molecule medicines. As we know, small molecules have been the mainstay of our pharmacopeia for several centuries and have, almost without exception, targeted proteins. This is why Arrakis set out in 2015 on an expedition to figure out how to develop small molecules that can target RNA.
Women in Stem with Lidia Pieri
Drug Target Review
APRIL 19, 2024
The scientific founders have developed a pioneering method for drug discovery that applies to the research and design of new drugs effective against diseases that are incurable to date. Our ongoing research and development are poised to explore these avenues, aiming to address complex diseases with novel therapeutics.
The Opportunities Waiting on Big Pharma Shelves
LifeSciVC
JULY 6, 2023
Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.
AbbVie to Showcase Depth of Gastroenterology Portfolio and Pipeline at Digestive Disease Week
The Pharma Data
MAY 16, 2022
AbbVie (NYSE: ABBV) today announced that 27 abstracts across its gastroenterology portfolio will be presented at the Digestive Disease Week (DDW) Annual Meeting, May 21-24, 2022, in San Diego and virtually. vice president, head of US medical affairs, AbbVie. The details of AbbVie’s oral presentations at DDW 2022 are outlined below.
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Sosei Heptares and Captor Therapeutics Enter Strategic Technology Collaboration Focused on Targeted GPCR Degradation as Novel Approach for Drug Design
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.
New targeted therapies show promise in lung cancer treatment
Drug Target Review
DECEMBER 13, 2024
1 Lung cancer is primarily classified into two types based on histological examination: Non-Small Cell Lung Cancer (NSCLC) and Small Cell Lung Cancer (SCLC). These types differ in their etiology, natural history, and present distinct challenges in disease management. percent of total cancer fatalities.
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AI Tool CHIEF Paints a Landscape of a Cancer, Refining Diagnosis, Treatment, and Prognosis
PLOS: DNA Science
SEPTEMBER 5, 2024
Gleevec is a small molecule that interferes with entry of an enzyme – a tyrosine kinase – that enables growth signals to enter specific cells and trigger division. A name ending in “nib” means a small molecule that inhibits an enzyme called a kinase, and is short for “inhibit.”
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Gilead Strengthens Early Pipeline in Oncology and Inflammation Through the Acquisition of XinThera
The Pharma Data
MAY 15, 2023
Through the acquisition, Gilead gains rights to a portfolio of small molecule inhibitors targeting PARP1 for oncology and MK2 for inflammatory diseases that could enter clinical trials later this year. XinThera is backed by a group of international investors including Foresite Capital, OrbiMed Advisors, LLC and TTM Capital.
Defense-Forward Biosecurity
Codon
AUGUST 27, 2024
Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. Sometimes, though, novel diseases do emerge, and as COVID-19 demonstrated, they can surprise us. This is the third essay of four in our pandemic mini-issue.
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Eloxx Pharmaceuticals to Report Third Quarter 2020 Financial Results and Provide Business Update on November 5, 2020
The Pharma Data
OCTOBER 14, 2020
International Dial-in Number: (210) 874-7715. 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. WALTHAM, Mass., Time : 4:30 p.m. Conference ID : 9467336.
Mission Therapeutics Appoints Dr Suhail Nurbhai as Chief Medical Officer
The Pharma Data
NOVEMBER 1, 2020
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. The Company is managed by a team with broad international, commercial and clinical-science experience.
Cullgen Announces “Featured Article” Publication of First in Class TRK Protein Degraders in Journal of Medicinal Chemistry
The Pharma Data
OCTOBER 15, 2020
a leading biotechnology company developing small molecule therapeutics based on its proprietary uSMITE platform of targeted protein degradation technology, today announced that the company’s internal program to develop selective degraders that target key proteins within the TRK family has been published by the Journal of Medicinal Chemistry.
AbbVie Acquires Syndesi Therapeutics, Strengthening Neuroscience Portfolio
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.
Tonix Pharmaceuticals Announces Publication of Patent Application for TNX-1500 (Monoclonal Antibody Anti-CD40-Ligand) in Development for Preventing and Treating Organ Transplant Rejection and Treating Autoimmune Conditions
The Pharma Data
JANUARY 13, 2021
The patent application is titled “Anti-CD154 Antibodies and Uses Thereof” and published under International Publication No. International Immunol. (11):1583 The CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. WO 2021/001458 A1. Structure.
FDA Action Alert: Merck, Aurinia and Amgen
The Pharma Data
JANUARY 17, 2021
People living with LN are in need of an advanced therapy that quickly drives the disease into remission and mediates kidney damage,” said Peter Greenleaf, president and chief executive officer of Aurinia, in July 2020 when the FDA accepted the NDA to review. “We The bone marrow suppression inhibits the production of blood cells.
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Extension Of Innovation Partnering Office at Monash University In Collaboration With Victorian State Government To Accelerate The Life-Sciences Hub
The Pharma Data
JULY 15, 2021
. “Several high impact Victorian healthcare collaborations have been signed since our launch, including those novel medical technologies and treatments for neurodegenerative and autoimmune conditions, and solutions for significant unmet needs in global public health including maternal mortality and infectious diseases.”
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OBI Pharma Announces Presentations at ESMO Asia 2020 Virtual Annual Meeting for OBI-833, a Novel Anti-Globo H Targeted Therapeutic Cancer Vaccine
The Pharma Data
NOVEMBER 18, 2020
In addition, OBI-833 can elicit a beneficial immune response in NSCLC patients and had rendered some TKI-treated patients a durable stable disease status. Title: A phase I cohort expansion trial of OBI-833 in non-small cell lung cancer patients. Department of Internal Medicine, Tri-Service General Hospital, Taipei, Taiwan.
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INAXAPLIN
New Drug Approvals
JANUARY 31, 2024
INAXAPLIN, 2446816-88-0 2446816-88-0 S2SJ2RVZ6Y UNII-S2SJ2RVZ6Y C 21 H 18 F 3 N 3 O 3 3-[5,7-difluoro-2-(4-fluorophenyl)-1 H -indol-3-yl]- N -[(3 S ,4 R )-4-hydroxy-2-oxopyrrolidin-3-yl]propanamide Inaxaplin ( VX-147 ) is a small-molecule apolipoprotein L1 inhibitor developed by Vertex Pharmaceuticals for APOL1-mediated kidney disease.
The Year In Review: 2023
KIF1A
MAY 31, 2024
ASCEND Online and KOALA In-Person Natural History Study Updates Understanding KAND and the progression of the disease informs our path to treatment. This international study supports families who speak English, Spanish, Portuguese, French, Dutch, German, and Italian, and KAND patients can be verbal or non-verbal.
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Article FDA Thank You FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma
Agency IQ
APRIL 19, 2024
FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.
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Sotuletinib HCl
New Drug Approvals
JANUARY 31, 2024
PATENT The free base and salts of the compound of formula (I) may be prepared for example, according to the procedures given in International Patent Application No. It works as a colony-stimulating factor 1 ( CSF1 ) receptor inhibitor. [1] PCT/US2007/066898 filed on Apr. 18, 2007 and published as WO2007/121484 on Oct.
Enlisting Monoclonal Antibodies in the Fight Against COVID-19
NIH Director's Blog: Drug Development
MAY 21, 2020
Science, 2020 We now know that the immune system of nearly everyone who recovers from COVID-19 produces antibodies against SARS-CoV-2, the novel coronavirus that causes this easily transmitted respiratory disease [1].
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Chimerix Acquires Oncoceutics to Expand Pipeline with Late-Stage Oncology Program
The Pharma Data
JANUARY 7, 2021
08, 2021 (GLOBE NEWSWIRE) — Chimerix (NASDAQ:CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that the Company has acquired Oncoceutics, Inc. , Compelling responses at this stage of disease are rare and lack durability.
2021 Biotech IPOs Get Off to a Roaring Start
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. Qilian International – Jiuquan, China-based Qilian International holding Group Limited, a pharmaceutical and chemical products manufacturer, announced its IPO of 5 million ordinary shares at $5 per share on January 14.
Syros Announces Pricing of $75.6 Million Public Offering of Common Stock
The Pharma Data
JANUARY 19, 2021
Syros is redefining the power of small molecules to control the expression of genes. Based on its unique ability to elucidate regulatory regions of the genome, Syros aims to develop medicines that provide a profound benefit for patients with diseases that have eluded other genomics-based approaches. About Syros Pharmaceuticals.
Navigating the Challenges in Clinical Research for Hepatitis C Therapies
Vial
DECEMBER 11, 2023
Pathophysiological evidence links HCV infections to the risk of liver diseases such as hepatocellular carcinoma (HCC) and liver cirrhosis. The Food and Drug Administration (FDA) approved and recommended dozens of small-molecule drugs. Patients at advanced stages of liver disease can still be treated.
Ascentage Pharma Enters into an Agreement with University of Michigan to obtain an exclusive license for a MDM2 Degrader using PROTAC Technology
The Pharma Data
NOVEMBER 29, 2020
C-Founder of Ascentage Pharma and Chairman of its Scientific Advisory Board, and Warner-Lambert/Parke-Davis Professor in Medicine, Professor of Internal Medicine, Pharmacology and Medicinal Chemistry, Director of Michigan Center for Therapeutic Innovation, University of Michigan , is a leading researcher in the field. Shaomeng Wang, Ph.D.,
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A backpack full of multiple sclerosis therapy
The Pharma Data
APRIL 22, 2023
Multiple sclerosis (MS) is a devastating autoimmune disease that destroys the protective myelin covering around nerves, disrupting communication between the brain and body, and causing patients’ ability to move and function to progressively decline. million individuals that currently have to live with the disease.
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The Year (and a Half) in Review: 2022
KIF1A
JUNE 30, 2023
Those who lead the way in the world of rare disease are those who are impacted each and every day. Natural History Study Update Understanding KAND and the progression of the disease informs our path to treatment. With these models established, we are preparing to test our first batch of small molecules drugs later this year!
BioSpace Movers & Shakers, Dec. 18
The Pharma Data
DECEMBER 17, 2020
Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. AB2 Bio – Swiss pharma company AB2 Bio Ltd. named Djordje Filipovic as its new chief commercial officer.
Calithera Biosciences Reports CANTATA Study of Telaglenastat in Renal Cell Carcinoma Did Not Achieve Primary Endpoint
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., About Telaglenastat. Calithera is headquartered in South San Francisco, California.
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