The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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The Pharma Data

OCTOBER 19, 2020

Transthyretin Amyloidosis (ATTR) is a rare, progressive and fatal disease. But Intellia Therapeutics hopes to change that bleak prognosis with a potentially curative therapy to reverse the gene mutation in ATTR patients. . Previous treatments could only hope to slow progression. hATTR is an inherited DNA mutation of the TTR gene.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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The Pharma Data

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). About Huntington’s disease. Survival ranges from approximately 10-20 years following motor onset of the disease.

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The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. IPF, unclassifiable interstitial lung diseases (uILDs), Long COVID respiratory complications and related sequelae) and lymphedema.

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Drug Target Review

OCTOBER 16, 2024

In reality, our deep understanding of disease pathology and patient needs is instrumental in shaping both basic and clinical research into viable treatments. By fostering the fluid movement between research and clinical practice, we can better contribute to the development of therapies, which has been the most rewarding aspect of my career.

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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

DECEMBER 12, 2023

We use hydrophilic linkers, which prevent ADC aggregation and generate highly stable ADCs, in combination with a unique attachment site on the antibody to create ADCs that retain pharmacokinetic properties similar to the original unconjugated antibody. This helps to maximize the targeted payload delivery to tumor cells.

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The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. About Gilead Sciences in Liver Disease. For more than 20 years, Gilead has sought to address some of the biggest challenges in liver disease. Gilead Lores,Inc.

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Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. There is a great need for a disease-modifying treatment for the 5.3 CLARKSVILLE, Md.,

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Drug Target Review

JUNE 5, 2023

It also displayed favourable pharmacokinetics (PK) and is well tolerated in non-human primates (NHP) at exposure levels above those projected to be efficacious. ZW191 also displayed favourable pharmacokinetics (PK) and is well tolerated in non-human primates (NHP) at exposure levels above those projected to be efficacious.

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The Pharma Data

NOVEMBER 26, 2020

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

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The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A., mL/min/ 1.73m 2 /year.

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The Pharma Data

MARCH 25, 2021

With more than six years of patient experience, a positive benefit-risk profile, strong clinical data and robust real-world data, the totality of evidence solidifies the role of IBRANCE plus endocrine therapy as a treatment for patients with HR+, HER2- metastatic breast cancer.”. The analysis also showed the two-year OS rate was 78.3%

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. UNITY is a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging. SUZHOU, China and ROCKVILLE, Md. , Ascentage Pharma (6855.HK)

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The Pharma Data

APRIL 4, 2023

The approval includes all indications covered by the reference medicine*: rheumatic diseases, Crohn’s disease, ulcerative colitis, plaque psoriasis, uveitis and hidradenitis suppurativa. 1 “Living with a chronic disease can take a significant toll on a patient’s quality of life.

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The Pharma Data

JUNE 7, 2022

Food and Drug Administration (FDA) has approved RIABNI™ (rituximab-arrx), a biosimilar to Rituxan ® , in combination with methotrexate for adults with moderate to severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies.

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Drug Target Review

JULY 12, 2023

These assays provide insights into the molecular mechanisms of disease biology and drug response, enabling the characterisation of gene expression profiles and deviations in diseased cells. Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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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? 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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The Pharma Data

MAY 4, 2021

INDIANAPOLIS , May 4, 2021 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) is donating COVID-19 therapies to Direct Relief, enabling the humanitarian organization to provide COVID-19 therapies at no cost to low- and lower-middle-income countries most heavily impacted by the pandemic.

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The Pharma Data

MAY 5, 2021

It also provided supporting pharmacokinetic data demonstrating the opioid antagonist’s safety and efficacy. . Farxiga use expanded for reducing risks in patients with chronic kidney disease. Farxiga received fast track, breakthrough therapy and priority review designations for the current indication. . New indications.

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The Pharma Data

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ? 12 years of age with severe hemophilia A.

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The Pharma Data

NOVEMBER 6, 2020

The Advisory Committee also voted 0 yes, 7 no and 4 uncertain on the question, “Does Study 103 (PRIME) provide supportive evidence of the effectiveness of aducanumab for the treatment of Alzheimer’s disease?”, Aducanumab (BIIB037) is an investigational human monoclonal antibody studied for the treatment of Alzheimer’s disease.

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The Pharma Data

MARCH 9, 2022

PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) is the first oral therapy specifically designed to combat COVID-19 to be evaluated in a pediatric clinical study PAXLOVID is currently authorized under U.S. million treatment courses delivered thus far and 30 million expected by July to help combat this devastating disease.

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The Pharma Data

APRIL 7, 2022

At Pfizer, we have a strong heritage in, and commitment to, fighting infectious diseases, most recently evidenced by our delivery of the first authorized vaccine and oral therapy to combat COVID-19,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. This press release features multimedia. Sapir, CEO, ReViral.

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Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution.

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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. What are the preclinical characteristics of ISM6331, including its efficacy, safety profile, and drug metabolism and pharmacokinetics (DMPK) properties?

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The Pharma Data

NOVEMBER 11, 2020

“As an orally bioavailable small molecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”. For more information, visit www.selvarx.com.

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The Pharma Data

MARCH 23, 2021

We have designed PF-07321332 as a potential oral therapy that could be prescribed at the first sign of infection, without requiring that patients are hospitalized or in critical care. Together, the two have the potential to create an end to end treatment paradigm that complements vaccination in cases where disease still occurs.”.

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The Pharma Data

OCTOBER 27, 2020

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. Sirnaomics. and China. The company’s $27.4

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The Pharma Data

NOVEMBER 18, 2020

This Phase 1 trial will evaluate the safety, tolerability, and pharmacokinetics of SEFA-1024 in 96 otherwise healthy volunteers with elevated plasma triglyceride levels. SEFA’ stands for Structurally Engineered Fatty Acid, i.e. chemically engineered fatty acids, to generate compounds with differentiated physiochemical properties.

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The Pharma Data

JANUARY 31, 2021

.–( BUSINESS WIRE )– Pinteon Therapeutics, a biotechnology company focused on protecting neuronal health by targeting neurotoxic forms of tau, today announced results from its Phase 1 study of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease.

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The Pharma Data

MARCH 5, 2021

Currently available BTK inhibitors irreversibly inhibit BTK and the long-term efficacy of these therapies can be limited by acquired resistance, most commonly through BTK C481 mutations. In rapidly growing tumors with inherently high rates of BTK turnover, resistance to covalent BTK therapies may be the result of incomplete target inhibition.

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The Pharma Data

FEBRUARY 25, 2022

The study met its primary goal by demonstrating pharmacokinetic equivalence in patients who switched multiple times between treatment with the two medicines. The root cause of many immunological diseases is immuno-inflammation, which requires specifically designed agents.

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