Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. 2019 Mar 1;55:93–100. 2019 Nov 1;8(6):784–8.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib is being studied in multiple immune-mediated diseases, including psoriasis, rheumatoid arthritis , lupus and inflammatory bowel disease.

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FDA Law Blog: Drug Discovery

OCTOBER 2, 2023

However, the two previous FDA guidance documents, released in May 1998 ( Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products , available here ) and December 2019 ( Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products , available here ), differ from the new guidance.

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The Pharma Data

DECEMBER 13, 2020

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.

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The Pharma Data

JANUARY 17, 2021

The companies announced in January 2019 a collaboration agreement to develop, manufacture and commercialize SPR206 in the Territory. SPR206 is in clinical development as an innovative option for the treatment of multi-drug resistant (MDR) Gram-negative bacterial infections. SHANGHAI , Jan. ” About SPR206.

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The Pharma Data

JANUARY 7, 2021

Cerecor intends to use the net proceeds of the offering for general corporate purposes and working capital, primarily to support the ongoing clinical development of key assets within its pipeline and for general and administrative expenses. ROCKVILLE, Md. and CHESTERBROOK, Pa., 07, 2021 (GLOBE NEWSWIRE) — Cerecor Inc.

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The Pharma Data

NOVEMBER 23, 2020

NYSE American: SYN) is a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need. . ROCKVILLE, Md. , 24, 2020 /PRNewswire/ — Synthetic Biologics, Inc. Synthetic Biologics, Inc.

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The Pharma Data

JANUARY 3, 2021

InspirMed is a newly established subsidiary of TLC specializing in the development of inhalable liposome formulation programs, such as TLC19 for the treatment of Coronavirus disease 2019 (COVID-19) as well as other programs for severe acute and chronic pulmonary diseases. Cautionary Note on Forward-Looking Statements.

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Drug Target Review

AUGUST 24, 2023

These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.

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The Pharma Data

DECEMBER 10, 2020

executive vice president of research and development. “We 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.”. Safe Harbor Statement.

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The Connected Lab

APRIL 8, 2020

The cost to develop a new prescription medicine that gains market approval has gone up 145% to $2.6 billion and takes an average of 10 years to develop 1. To this day, more than 400 million people suffer from rare diseases and 95% of rare diseases lack an FDA approved treatment 3. 2019, Freedman, David H.

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The Pharma Data

MARCH 2, 2021

This new collaboration builds on the positive experience from previous partnerships with Gubra and combines Gubra’s proprietary streaMLine platform and expertise in obesity and peptide chemistry with Boehringer Ingelheim’s expertise in lead optimization and clinical development. population will have obesity. About Boehringer Ingelheim.

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The Pharma Data

OCTOBER 31, 2020

Kuo-Lung Huang , the Chairman and CEO of TaiGen notes, “Influenza is a life threatening disease with significant unmet medical needs and high medical burden. With the IND approval in place, we are ready and eager to extend the clinical study of TG-1000 to the U.S. billion USD in 2019 and is estimated to reach 5.03

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The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Phase1b/2 clinical study to be conducted in the United States and several European countries. Billion by 2025. and Europe.

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The Pharma Data

APRIL 8, 2021

OCREVUS data show its consistent benefit on slowing disease progression in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Data for ENSPRYNG in neuromyelitis optica spectrum disorder (NMOSD) reinforce safety and efficacy, including in patients with concomitant autoimmune diseases (CAIDs). Following U.S.

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The Pharma Data

DECEMBER 12, 2020

Dedicated rare disease unit to be headquartered in Boston. Pascal Soriot , Chief Executive Officer, AstraZeneca , said: “ Alexion has established itself as a leader in complement biology, bringing life-changing benefits to patients with rare diseases. This acquisition allows us to enhance our presence in immunology.

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The Pharma Data

JUNE 2, 2022

Per 2019 agreement, Bristol Myers Squibb to also add one additional autologous TCR-T target where Immatics will receive an upfront payment of $20 million and be eligible for milestone payments and royalties. Our manufacturing process is designed to create hundreds of doses from one single donor leukapheresis. About Bristol Myers Squibb.

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The Pharma Data

JANUARY 27, 2021

.–( BUSINESS WIRE )– PlateletBio , a pioneering biotechnology company developing next-generation allogeneic cell therapies for treatment of human diseases, has named Steven M. Altschuler, M.D., as Chairman of its Board of Directors. “As President and CEO of PlateletBio. The company, based in Watertown, Mass.,

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Alta Sciences

APRIL 17, 2024

Ketamine is a dissociative anesthetic approved in 2019 by the U.S. Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. It is a growing field where new technology, and an increasing understanding of the science, are bringing advancements in drug development.

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The Pharma Data

OCTOBER 9, 2021

“While we did not achieve proof of concept in this study, we are committed to advancing our deucravacitinib clinical program in inflammatory bowel disease, including ulcerative colitis and Crohn’s disease, as well as in psoriatic arthritis, lupus and other immune-mediated diseases. About Ulcerative Colitis.

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The Pharma Data

OCTOBER 15, 2020

ASLAN believes ASLAN003 has the potential to bethe most potent oral inhibitor of DHODH currently in development for autoimmune disease, more than 30 times more potent at inhibiting the DHODH enzyme than teriflunomide. In 2019, ASLAN completed a Phase 2 study testing ASLAN003 in AML. SINGAPORE, Oct.

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The Pharma Data

OCTOBER 28, 2020

SIGNAL Phase 2 trial data continue to support cognitive benefit and reduced brain atrophy with pepinemab treatment in Huntington’s disease. Data provide strong rationale for continued development in Huntington’s and other slowly progressive neurodegenerative diseases, including Alzheimer’s. ROCHESTER, N.Y., Vaccinex, Inc.

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The Pharma Data

MARCH 23, 2021

For those patients with primary central nervous system (CNS) tumors and with CNS metastases, responses and a high disease control rate have been observed. We have the passion and determination to develop innovative medicines that help improve and extend the lives of people living with cancer. It is already approved in the U.S.,

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The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase III clinical trial programme for fenebrutinib, an investigational medicine designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, which may offer novel approach to suppress disease activity and slow disease progression in MS.

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The Pharma Data

OCTOBER 19, 2020

Pneumococcal disease in adults is on the rise globally, in part driven by disease-causing serotypes not targeted by the currently available pneumococcal conjugate vaccine,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories.

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The Pharma Data

AUGUST 7, 2020

Results included global public and private financing from regenerative medicine and advanced therapy developers, and highlighted a 120 per cent increase over the first half of 2019. The ARM report also showcased robust pipelines of therapies targeting indications in cancers, infectious diseases and inherited disorders.

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The Pharma Data

MARCH 16, 2021

Head of Department CNS Diseases Research at Boehringer Ingelheim. “By While peripherally acting COMT inhibitors are currently used as an adjunctive therapy in the treatment of neurological conditions, including Parkinson’s disease, this new collaboration is investigating centrally acting COMT inhibitors in neuropsychiatric disorders.

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The Pharma Data

JUNE 26, 2021

In 2019, Libtayo was approved by the EC as the first treatment for adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation. The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. About Regeneron.

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The Pharma Data

NOVEMBER 18, 2020

Hypertriglyceridemia may cause acute pancreatitis and is often associated with other conditions that increase the risk of heart disease and stroke, including type 2 diabetes, obesity, and metabolic syndrome. A Phase 2b study was initiated in July 2019 (ICONA) to study the efficacy of icosabutate in NASH. NorthSea Therapeutics B.V.(NST)

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The Pharma Data

NOVEMBER 11, 2020

. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S.

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The Pharma Data

MARCH 5, 2021

In the Phase 2, patients are enrolled across various cohorts, depending on disease type and prior therapy. Loxo Oncology at Lilly was created in December 2019, combining the Lilly Research Laboratories oncology organization and Loxo Oncology, which was acquired by Lilly in early 2019. The primary endpoint for Phase 2 is ORR.

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The Pharma Data

DECEMBER 14, 2020

NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases (“the Company”), today announced the pricing of an underwritten public offering of 6,320,000 shares of common stock at a public offering price of $43.50 NEW YORK, Dec. 14, 2020 (GLOBE NEWSWIRE) — TG Therapeutics, Inc.

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The Pharma Data

OCTOBER 18, 2020

PsA is a chronic, immune-mediated, inflammatory disease that most commonly appears in adults aged 30 to 50. Progressive and irreversible, it is characterized by debilitating joint damage, inflammation, enthesitis, dactylitis, axial disease, and skin lesions generally associated with psoriasis. The focus is on cancer.

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The Pharma Data

NOVEMBER 9, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant.

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The Pharma Data

JANUARY 21, 2021

where he developed therapeutics to treat orphan eye diseases. Escolar was the principal medical consultant for Genzyme’s pivotal trial for Myozyme, an enzyme replacement therapy approved to treat adult-onset Pompe disease. Additionally, Viridian named Barrett Katz as its chief medical officer. and Genentech, Inc. Source link.

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