The Pharma Data

DECEMBER 20, 2020

ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus. Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. About ALS-4.

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Drug Target Review

JULY 13, 2023

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. They found that although the targets themselves may not be easily druggable due to their disordered nature, their ability to condense can be modulated using small molecules.

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The Pharma Data

JANUARY 19, 2021

As part of Aptorum Group’s Acticule infectious disease platform, ALS-4 is a novel orally administered first-in-class small molecule that was developed based on an anti-virulence approach targeting Staphylococcus aureus including MRSA. About ALS-4.

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The Pharma Data

JUNE 24, 2025

To date, more than 70 joint research projects have been initiated, and over 10 co-authored publications have appeared in top-tier international academic journals. A recent example is Bayer’s global license agreement with Puhe BioPharma , focused on an oral, small molecule PRMT5 inhibitor designed to selectively target MTAP-deleted tumors.

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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.

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New Drug Approvals

JULY 10, 2025

g/mol O5ZD2TU2B7 FDA 7/3/2025, Ekterly, To treat acute attacks of hereditary angioedema N -[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-[[4-[(2-oxopyridin-1-yl)methyl]phenyl]methyl]pyrazole-4-carboxamide Sebetralstat , sold under the brand name Ekterly , is a medication used for the treatment of hereditary angioedema. [1]

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New Drug Approvals

MAY 26, 2025

M in tetrahydrofuran) was added at room temperature at a rate to maintain the internal temperature below 30 C. “Discovery of Ervogastat (PF-06865571): A Potent and Selective Inhibitor of Diacylglycerol Acyltransferase 2 for the Treatment of Non-alcoholic Steatohepatitis” Journal of Medicinal Chemistry. equiv, 1.3

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New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment. Suzetrigine is taken by mouth. [1]

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The Premier Consulting Blog

NOVEMBER 20, 2024

In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients.

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The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the U.S. BUFFALO, N.Y.,

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New Drug Approvals

APRIL 18, 2025

Formula C 27 H 29 Cl 2 FN 2 OS Crinecerfont , sold under the brand name Crenessity , is a medication used for the treatment of congenital adrenal hyperplasia. [1] kg, the preparation of Compound 8A has been previously described in International Publication Number WO2010/125414) were added to the reactor and heated to 85 C. kg, 1 eq.)

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Drug Target Review

APRIL 19, 2024

The discovery of the folding intermediate states to target has been enabled by our proprietary computational platform, and the development of small molecule folding interfering degraders is now accelerated by a proprietary AI platform. As a woman in STEM, what unique perspectives or strengths do you believe you bring to your work?

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The Pharma Data

JANUARY 31, 2021

Convergent is currently conducting advanced human trials relating to prostate cancer treatments involving peptide receptor radionuclide therapy (“PRRT”) that targets the prostate-specific membrane antigen (“PSMA”) present on prostate cancer cells. or its ability to internalize PSMA.

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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

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.

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The Pharma Data

DECEMBER 15, 2020

Treatment induced sustainable clinical responses and reduced systemic inflammation. Daratumumab already is approved for the treatment of multiple myeloma. The company is developing its platform for the treatment of chronic, inflammation-mediated autoimmune diseases, and is initially focused on the treatment of RA.

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The Pharma Data

OCTOBER 18, 2020

DUBLIN–( BUSINESS WIRE )– Inflection Biosciences Ltd , a company developing innovative therapeutics for the treatment of cancer, today announced the appointment of industry veteran Gregory I. Inflection Biosciences Ltd is developing small molecule therapeutics for the treatment of cancer. 19, 2020 08:02 UTC.

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The Pharma Data

MARCH 1, 2022

This acquisition gives AbbVie access to Syndesi’s portfolio of novel modulators of the synaptic vesicle protein 2A (SV2A), including its lead molecule SDI-118. ” The lead molecule, SDI-118, is a small molecule currently in Phase 1b studies, which is being evaluated to target nerve terminals to enhance synaptic efficiency. .

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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.

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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.

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The Pharma Data

OCTOBER 14, 2021

Reducing the amount of time patients need to be in hospital has the potential to improve their treatment experience whilst also increasing efficiency for institutions and healthcare systems.”. Roche’s Chief Medical Officer and Head of Global Product Development. About the GAZELLE study. About follicular lymphoma.

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The Premier Consulting Blog

MAY 31, 2023

In this blog post, we outline typical elements of an IND-enabling package for a single ascending dose (SAD)/multiple ascending dose (MAD) study of an oral small molecule for a non-cancer indication with a maximum of 14 days of dosing.

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The Pharma Data

OCTOBER 6, 2021

The companies do not expect the introduction of booster doses in the United States and the EU, if authorized, to impact the existing supply agreements in place with governments and international health organizations around the world.

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The Pharma Data

NOVEMBER 18, 2020

Department of Internal Medicine, Tri-Service General Hospital, Taipei, Taiwan. Title: OBI-833 was safe and immunogenic, without treatment-related SAEs, in a Phase 1 dose-escalation trial. Session Title: E-poster Display session – Thoracic tumours, metastatic. Session Date and Time: Friday, November 20, 2020.

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KIF1A

MAY 31, 2024

From hosting a conference to scientific projects and collaborations to the dedicated efforts of our team, this review encapsulates the relentless pursuit to accelerate treatments and a cure for all KAND warriors. The Chung Lab spearheads a robust KAND program to better understand the disorder and discover treatments.

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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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KIF1A

JUNE 30, 2023

KAND and Spasticity : We created this resource to help families understand mechanisms of spasticity in KAND, including considerations between different treatment options. With your support, KIF1A.ORG continues to bring together the expertise and innovation needed to rapidly discover treatments and cures for KAND.

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The Pharma Data

MAY 16, 2022

About Digestive Disease Week® Digestive Disease Week (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery.

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The Premier Consulting Blog

JULY 9, 2023

During the development of new small molecule drug products, developers must conduct impurity and degradant evaluation at several points in the program and to varying degrees. The TTC varies based on the clinical treatment duration and on whether multiple mutagenic impurities are present.

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New Drug Approvals

JANUARY 31, 2024

Sotuletinib (BLZ945) is an experimental drug in development for the treatment of amyotrophic lateral sclerosis (ALS). 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. PCT/US2007/066898 filed on Apr. 36 (1): 456–467.

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The Pharma Data

DECEMBER 12, 2020

AstraZeneca has developed a broad range of technologies, initially focused on small molecules and biologics and with a growing focus in precision medicine, genomics, oligonucleotides and epigenetics. 5% have US Food and Drug Administration -approved treatments. Click here for available international numbers.

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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.

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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.

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Drug Target Review

NOVEMBER 22, 2023

How does iOnctura plan to design novel combination treatments that effectively target both autotaxin and the TGF pathway while ensuring safety for patients? We will present some of the data at the end of the study at an international conference next year. Are there any unique strategies or approaches being considered?

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The Pharma Data

AUGUST 29, 2020

Phase II results are promising for treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder; 1,2 second Phase II study with LNP023 monotherapy in anti-C5 naïve PNH patients ongoing . All patients required RBC transfusions prior to starting LNP023 treatment. “In Peffault de Latour.

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The Pharma Data

JANUARY 3, 2021

As compared to treatment with cabozantinib, the combination of telaglenastat and cabozantinib did not meet the primary endpoint of improving progression free survival (PFS) in the study population. “We The webcast will be recorded and available for replay on Calithera’s website for 30 days. About Telaglenastat. Forward Looking Statements.

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The Pharma Data

NOVEMBER 17, 2020

LYT-100 is PureTech’s wholly-owned product candidate that is being advanced for the potential treatment of conditions involving inflammation and fibrosis and disorders of lymphatic flow. The therapeutic dose of pirfenidone approved by the US Food and Drug Administration (FDA) for the treatment of IPF is 801 mg three times a day.

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