New Drug Approvals

JULY 21, 2025

Hetrombopag olamine (6), an oral nonpeptide thrombopoietin receptor (TpoR)agonistdevelopedby JiangsuHengruiPharmaceutical, was approved in China in June2021 for treatment of adult patients with chronic primary immune thrombocytopenia (ITP) and severe aplastic anemiawhohave not responded well to other treatments.46Hetrombopag,

Small Molecule

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.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

4 Another related and well described non-oncogene resistance mechanism is the histological transformation of EGFR-mutated non-small cell lung cancer (NSCLC) to small-cell lung cancer upon treatment with an EGFR inhibitor. This orally available small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

Small Molecule

DrugBank

OCTOBER 17, 2024

By utilizing high-resolution protein structures obtained through X-ray crystallography, researchers could design small molecules that potently inhibit the SARS-CoV-2 main protease, a key enzyme essential for viral replication.

Drug Development

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. In addition, we have advanced zavegepant, our intra-nasal CGRP receptor antagonist, successfully through its first pivotal trial, also for the acute treatment of migraine. TOKYO and CAMBRIDGE, England , Dec.

Small Molecule

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.

Small Molecule

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.

Small Molecule

The Pharma Data

NOVEMBER 19, 2020

Structure-based design approach has identified potent compounds for further development as oral treatments for SARS-CoV-2 infection and related human coronaviruses. We disclaim any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Small Molecule

The Pharma Data

JANUARY 13, 2021

Preliminary analyses showed no clinically meaningful differences in safety results between the dexpramipexole and placebo groups, with no serious adverse events and 96% of subjects completing the primary assessment phase. Background standard of care was inhaled corticosteroids in combination with a long-acting ?2 President and CEO of Knopp.

Trials

Drug Target Review

JUNE 12, 2023

With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?

Metabolic Stability

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.,

FDA Approval

The Pharma Data

NOVEMBER 8, 2021

“HER-MES is the first study that directly compared the therapeutic effects of an antibody and a small molecule in migraine prevention,” said Uwe Reuter, M.D., “The positive outcomes strengthen the efficacy and safety profile of erenumab as a migraine prevention treatment for patients with migraine.”

Therapies

The Pharma Data

JANUARY 26, 2021

Food and Drug Administration’s Office of Orphan Products Development has granted Orphan Drug Designation to Uttroside-B , a small molecule chemotherapeutic for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. (OTCQB: QBIO), announced today that the U.S. .

Treatment

New Drug Approvals

DECEMBER 16, 2023

2 On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. 5 Iptacopan , sold under the brand name Fabhalta , is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria. [1] 1] It is a complement factor B inhibitor that was developed by Novartis. [1]

FDA

The Pharma Data

OCTOBER 14, 2020

operating as VBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. About VBL Vascular Biogenics Ltd., Source link.

Treatment

The Pharma Data

OCTOBER 27, 2020

Mirati Therapeutics is a San Diego -based late-stage biotechnology company relentlessly focused on translating drug discovery and research into new treatments for patients by advancing and delivering novel therapeutics that target the genetic and immunologic drivers of cancer. About Mirati Therapeutics. SOURCE Mirati Therapeutics, Inc.

Small Molecule

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. and foreign patents.

Small Molecule

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.

Treatment

The Pharma Data

DECEMBER 13, 2020

Due to the COVID -19 pandemic, the event will be hosted virtually. Senhwa was invited to present positive topline results from their global phase II trial, evaluating the combination of Silmitasertib plus Gemcitabine/Cisplatin compared to Gemcitabine/Cisplatin alone in the frontline treatment of patients with Cholangiocarcinoma.

Small Molecule

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.

Targeted Protein Degradation

The Pharma Data

DECEMBER 10, 2020

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.”. LX9211 is a potent, orally delivered, selective small molecule inhibitor of adapter-associated kinase 1 (AAK1). About LX9211. Source link.

FDA

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.

Small Molecule

The Pharma Data

OCTOBER 6, 2021

The adverse event profile was generally consistent with other clinical safety data for COMIRNATY. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Vaccine

The Pharma Data

OCTOBER 26, 2020

AR-711 was previously shown to be effective in prophylactic as well as therapeutic treatment modes in a SARS-CoV-2 viral challenge study. AR-711 is currently being developed as an inhaled, self-administered treatment for non-hospitalized patients suffering from mild to moderate COVID-19. About Aridis Pharmaceuticals, Inc.

Pharmaceuticals

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. The frequency and severity of adverse events in the telaglenastat-treated population were comparable to that of cabozantinib alone. “We

Trials

Advarra

FEBRUARY 24, 2023

But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? Also known as a clinical event committee (CEC), EAC members evaluate a clinical event in the context of the protocol and the individual participant’s medical information.

Clinical Trials

New Drug Approvals

JANUARY 31, 2024

Sotuletinib (BLZ945) is an experimental drug in development for the treatment of amyotrophic lateral sclerosis (ALS). “Emerging clinical investigational drugs for the treatment of amyotrophic lateral sclerosis” Expert Opinion on Investigational Drugs. 36 (1): 456–467. 32 (2): 141–160.

Small Molecule

The Pharma Data

DECEMBER 9, 2020

About Oncternal Therapeutics Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of cancers with critical unmet medical need. More information is available at. Forward-Looking Information.

Clinical Trials

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. All adverse events (AEs) that were possibly or probably related to LYT-100 were mild and transient and there were no discontinuations. with placebo).

Pharmacokinetics

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.”

Government

The Pharma Data

DECEMBER 14, 2020

The global GBM treatment market is projected to reach $3.3 The ability of a drug candidate to cross the blood brain barrier is of critical importance in treatment outcomes for CNS and brain cancers. It is considered an orphan disease for which there is no cure. representing the largest market.

Clinical Trials

The Pharma Data

DECEMBER 8, 2020

Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced the pricing of an underwritten public offering of 8,110,000 shares of its common stock at a price to the public of $37.00 SAN DIEGO, Dec.

Small Molecule

The Pharma Data

OCTOBER 18, 2020

Despite recent advances in the targeted treatment of AML, the majority of patients will relapse or remain refractory to current therapies and there remains a significant unmet need for new therapies. About CG-806. Forward Looking Statements. This press release contains forward-looking statements within the meaning of Canadian and U.S.

Clinical Trials

The Pharma Data

NOVEMBER 12, 2020

Keros is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet medical need. LEXINGTON, Mass., 12, 2020 (GLOBE NEWSWIRE) — Keros Therapeutics, Inc. About Keros Therapeutics, Inc.

Clinical Trials

The Pharma Data

JANUARY 7, 2021

NASDAQ: GLTO) a NASDAQ listed biotechnology company focused on the development of novel treatments for fibrosis and cancer, today announced the appointment of Dr. Anne Prener to its Board of Directors. “We Actual events or results may differ materially from Galecto’s expectations. BOSTON, Jan. We warmly welcome Anne to our Board.

Small Molecule

The Pharma Data

SEPTEMBER 12, 2020

The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Vaccine

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. The CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions.

Vaccine