LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

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PerkinElmer

NOVEMBER 9, 2022

While certain patients respond to these therapies, antibody drugs have some natural drawbacks such as poor oral bioavailability, poor permeability of tumor tissues, immune-related adverse events, and high medical costs.

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Drug Target Review

MAY 6, 2024

The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. We found that, indeed, cancer’s earliest defence mechanisms are triggered almost immediately after exposure to a targeted cancer therapy. What if our science can directly impact patients’ lives?”

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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., Additional study treatment-related adverse events reported by ?2% versus 38.9%). .” versus 38.9%). versus 31.2%).

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Therapies FDA Approval Treatment Small Molecule 52

Advarra

FEBRUARY 24, 2023

How do you know your experimental therapy is working? But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? All key questions to consider and address before the therapy even leaves the lab.

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Clinical Trials Trials FDA Therapies 52

LifeSciVC

APRIL 24, 2024

If adverse events are anticipated, it is important to understand gene dosage for such an effect (e.g., and whether a molecule’s pharmacology can help to mitigate safety risk. and whether a molecule’s pharmacology can help to mitigate safety risk. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g.,

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Production Small Molecule Regulations Trials 114

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. Dexpramipexole was well tolerated by patients at all doses tested. President and CEO of Knopp. ABOUT ORAL DEXPRAMIPEXOLE.

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Trials Small Molecule Clinical Trials Treatment 52

The Pharma Data

JANUARY 5, 2021

The presentation will be webcast live and may be accessed from the Investor page of Relay’s website under Events and Presentations. Relay Therapeutics (Nasdaq: RLAY) is a clinical-stage precision medicines company transforming the drug discovery process with the goal of bringing life-changing therapies to patients.

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Small Molecule Therapies Drugs 52

The Pharma Data

AUGUST 30, 2021

The head-to-head study was designed to directly compare the efficacy of abrocitinib 200mg versus dupilumab 300mg, in adult participants on background topical therapy with moderate to severe atopic dermatitis (AD). A larger percentage of patients treated with abrocitinib 200mg experienced adverse events compared to dupilumab 300mg.

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Small Molecule Trials Treatment FDA 52

Drug Target Review

JUNE 12, 2023

TEAD proteins are known to be very important in cancer progression TEAD proteins are known to be very important in cancer progression, and there are a number of therapies in development. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?

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Metabolic Stability Clinical Trials Small Molecule Pharmacokinetics 92

Dark Matter Blog

JULY 16, 2021

My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like small molecules.

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Small Molecule RNA Clinical Trials DNA 52

The Pharma Data

JANUARY 31, 2021

Convergent has exclusive rights to technology related to next generation radiopharmaceutical therapy for prostate cancer that is covered by multiple issued U.S. The key component of Convergent’s PRRT prostate cancer therapy is its proprietary drug, CONV 01-?, and foreign patents. The function of CONV 01-?

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Small Molecule FDA Approval Therapies Trials 52

Drug Target Review

JULY 7, 2023

There is a clear need for new and approved, safe, effective and innovative targeted therapies to address the underlying causes of autoantibody diseases. The power of precision therapy Targeted — or precision — therapy, a technique that has been honed for many years in cancer treatment, is now being applied in a wider context.

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The Pharma Data

APRIL 23, 2021

European Commission approves GSK’s JEMPERLI (dostarlimab), the first anti-PD-1 therapy approved for recurrent or advanced endometrial cancer. The approval makes dostarlimab the first anti-PD-1 therapy available for endometrial cancer in Europe. Hormone replacement therapy for endocrinopathies should be instituted as warranted.

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The Pharma Data

NOVEMBER 29, 2020

1 Meanwhile, these MDM2 inhibitors present some challenges, including dose-limiting hematological toxicities, thus the urgent need to develop new generation of MDM2-targeting therapies in the treatment of cancer. Small-molecule inhibitors of the MDM2-p53 protein-protein interaction (MDM2 inhibitors) in clinical trials for cancer treatment.

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KIF1A

AUGUST 20, 2024

Susannah’s sudden absent spells, reported by the Rosens as seizures, weren’t accompanied by specific seizure-related activity on the EEG, despite epilepsy-related activity between events. Because of this disconnect, these events are described as “spells of behavioral arrest” in the study. Will this _ KAND?

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Science Treatment Therapies Small Molecule 52

New Drug Approvals

JANUARY 31, 2024

It works as a colony-stimulating factor 1 ( CSF1 ) receptor inhibitor. [1] It works as a colony-stimulating factor 1 ( CSF1 ) receptor inhibitor. [1]

Small Molecule 52

Small Molecule Virus Treatment Disease 52

The Pharma Data

JANUARY 17, 2021

The study enrolled 5,050 patients who received either vericiguat or placebo in combination with available heart failure therapies. HFrEF was formerly known as systolic heart failure. It is marked by the heart’s compromised ability to eject blood sufficiently during its contraction phase.

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FDA Small Molecule Immune Response Hospitals 52

The Pharma Data

OCTOBER 27, 2020

Mirati is advancing a novel pipeline to treat large patient populations across multiple programs and tumor types, including two programs, adagrasib and sitravatinib, in registration-enabling studies to treat non-small cell lung cancer (NSCLC). Forward-Looking Statements. View original content to download multimedia: [link]. Source link.

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Small Molecule Immune Response Clinical Development Clinical Trials 52

Elrig

JUNE 4, 2024

The focus used to be more on small molecules, but we’re now starting to see antibody and cell therapeutics. All these ‘worlds’ can learn from each other – especially since the small molecule drug discovery process is much more mature than the antibody which is much more mature than the gene and cell therapy space.

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Small Molecule Science Drugs Research 52

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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The Pharma Data

FEBRUARY 25, 2022

In these studies, rimegepant was safe and well-tolerated with rates of adverse events similar to that of placebo. CGRP Receptor Antagonism Small molecule CGRP receptor antagonists represent a novel class of drugs for the treatment of migraine. Chief Executive Officer and Chairman of the Board of Biohaven.

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Treatment Small Molecule FDA Approval Therapies 52

The Pharma Data

OCTOBER 25, 2020

In the study, the drug demonstrated statistically significant and clinically meaningful efficacy and a favorable adverse event and tolerability profile. The study is evaluating the safety and efficacy of CTX110, a wholly-owned allogeneic CAR-T cell therapy targeting CD19+ B-cell cancers. No serious adverse events were observed.

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Clinical Trials Vaccine Trials Virus 52

The Pharma Data

JANUARY 3, 2021

Sixty-two percent of patients were treated with prior PD(L)-1 containing therapy, and the arms were well balanced. The frequency and severity of adverse events in the telaglenastat-treated population were comparable to that of cabozantinib alone. “We SOUTH SAN FRANCISCO, Calif., Median PFS was 9.2 months with cabozantinib and placebo.

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Trials Clinical Trials Small Molecule Therapies 52

The Pharma Data

OCTOBER 18, 2020

Separate from the AML trial, Aptose is conducting a Phase 1 a/b dose escalation study with CG-806 in patients with B-cell malignancies, including chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphomas (NHL), who have failed or are intolerant to current therapies. About CG-806. . . . .

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Codon

APRIL 2, 2023

Read A one-time gene therapy injection for spinal muscular atrophy. Mice and human cells that were given the therapy had normal levels of the survival motor neuron protein, and no symptoms. Gene Therapy. Read A really simple way to isolate AAVs, which are a type of virus often used to deliver gene therapies into cells.

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DNA RNA Engineering Licensing 52

New Drug Approvals

DECEMBER 16, 2023

2 This is of particular importance to PNH, where one of the disease hallmarks is the mutation of the PIGA gene. [1] Research In a clinical study with twelve participants, iptacopan as a single drug led to the normalization of hemolytic markers in most patients, and no serious adverse events occurred during the 12-week study. [5]

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FDA Small Molecule FDA Approval Treatment 62

The Pharma Data

MAY 6, 2022

The adverse event profile was generally consistent with other clinical safety data for the vaccine, with a favorable safety profile observed in individuals with at least 6 months of safety follow-up after the second dose.

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Agency IQ

JUNE 9, 2023

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. The guidance contains recommendations for small molecule drugs, as well as any biological products that fall within CDER’s purview.

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The Pharma Data

JANUARY 7, 2021

ONC201 is an orally administered small molecule dopamine receptor D2 (DRD2) antagonist and caseinolytic protease (ClpP) agonist in late-stage clinical development for recurrent gliomas that harbor the H3 K27M mutation. This acquisition builds upon the vision of my co-founder Wolfgang Oster, M.D., scientific founder Wafik El-Deiry, M.D.,

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Clinical Trials Small Molecule Trials Therapies 52

The Pharma Data

DECEMBER 13, 2020

RLY-1971 has the potential to serve as a backbone for combination therapy across numerous solid tumors and therefore represents an encouraging approach for cancer patients,” said Sanjiv Patel, M.D., In the event of regulatory approval of both RLY-1971 and GDC-6036 in combination, Relay Therapeutics is eligible to receive additional royalties.

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Licensing Licensing Clinical Trials Small Molecule 40

The Pharma Data

OCTOBER 1, 2021

Pfizer today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved CIBINQO ® (abrocitinib), an oral, once-daily, Janus kinase 1 (JAK1) inhibitor, for the treatment of moderate to severe atopic dermatitis (AD) in adults and adolescents aged 12 years and older with inadequate response to existing therapies.

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Clinical Trials Small Molecule Trials Treatment 52

The Pharma Data

JANUARY 14, 2021

We believe that the use of machine learning, genomics and computational methods can help accelerate the revitalization, refocusing and development of small molecule-based therapies. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate.

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Small Molecule Therapies Drug Development Drugs 52

The Pharma Data

SEPTEMBER 12, 2020

The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing. At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. About Pfizer: Breakthroughs That Change Patients’ Lives.

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Vaccine Trials Clinical Trials Small Molecule 52

The Pharma Data

JANUARY 18, 2021

The fireside chat will be available for 90+ days following the event at: [link] 5 69. Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to treat cancers. . A live webcast of this presentation will be available for registered participants at: [link].

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Clinical Trials Small Molecule Trials Therapies 40

The Pharma Data

JANUARY 7, 2021

As CEO of Freeline, a liver-directed gene therapy company, she scaled the company from preclinical stage to a fully integrated biotechnology organization, which included a broad, internally developed pipeline, two programs in clinical development and a commercial-scale, high-quality CMC and manufacturing platform.

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Small Molecule Clinical Development Production Science 52

The Pharma Data

OCTOBER 14, 2020

. “ This new patent is an important milestone achieved by VBL in securing intellectual property protection for our MOSPD2 program and for targeting MOSPD2 for anti-cancer therapy, “ said Erez Feige, Ph.D, MBA, VP Business Operations at VBL. About VBL Vascular Biogenics Ltd., Source link.

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