Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

JULY 7, 2022

While just two gene therapy products have been approved by the FDA, a number of companies are nearing the market with therapies targeting rare diseases, including 14 candidates in Phase III plus three in the pre-registration phase, according to GlobalData. A new frontier in cancer research.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

Pharmaceutical Technology

MARCH 23, 2023

Pfizer recently announced an agreement to acquire Seagen, a biotech company based in the US with four marketed oncology therapeutic agents and a rich pipeline. Seagen specialises in developing antibody-drug conjugates (ADCs) which will complement Pfizer’s oncology portfolio.

Licensing 147

Licensing Licensing Sales Trials 147

pharmaphorum

DECEMBER 16, 2020

AbbVie is to begin clinical development of an antibody designed to neutralise the SARS-CoV-2 coronavirus after licensing the therapy in from Harbour BioMed and Utrecht University. AbbVie has begun a phase 1 clinical trial of the antibody, with clinical development beginning in the US and expanding into Europe.

Antibody 98

Antibody Trials Licensing Licensing 98

pharmaphorum

MAY 13, 2021

There are already several drugs on the market to treat Duchenne, notably from Sarepta Therapeutics which has three FDA-approved drugs that use “exon skipping” technology. These allow the body to produce a correct version of the misfolded dystrophin protein that is the root cause of DMD.

Gene Therapy 102

Gene Therapy Gene Trials Protein 102

pharmaphorum

JANUARY 13, 2022

Clinical trials will be needed to see if doing so can interrupt the neurodegeneration seen in Parkinson’s. Alpha-synuclein is a protein that gets misfolded and clumps together in the brains of patients with Parkinson’s and other diseases collectively known as synucleinopathies.

Antibody 98

Antibody Drugs Protein Licensing 98

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

Pharmaceutical Technology

MAY 31, 2023

Sym-021 is under clinical development by Les Laboratoires Servier and currently in Phase I for Solid Tumor. The therapeutic candidate targets immune checkpoint, programmed cell death protein 1 (PD1). It is developed based on sympress platform technology. It is administered as an intravenous infusion.

Generic Drugs 100

Generic Drugs Hormones Manufacturing Development 100

pharmaphorum

JANUARY 5, 2022

Now, Pfizer and BioNTech are joining forces to try to grab a slice of the market. Pfizer is paying $225 million upfront to its partner to get the ball rolling on the project, including $75 million in cash and $150 million in equity, offset by $25 million from BioNTech in return for an antigen technology that will be used to develop the shot.

Vaccination 105

Vaccination Vaccine Sales Clinical Development 105

Pharmaceutical Technology

FEBRUARY 24, 2023

ACE-1334 is under clinical development by Merck and currently in Phase I for Lung Disease. ACE-1334 overview ACE-1334 is under development for the treatment of pulmonary disease with fibrotic component and systemic sclerosis and its associated interstitial lung disease. It is developed based on protein engineering platform.

Production 100

Production Vaccination Vaccine Engineer 100

Pfizer

SEPTEMBER 16, 2022

Pfizer and BioNTech Receive Positive CHMP Opinion for Conversion of COMIRNATY® Conditional Marketing Authorization to Full Marketing Authorization in the European Union. Standard marketing authorization recommendation applies to all indications and formulations in the European Union, including COMIRNATY® Original/Omicron BA.4-5

Marketing 75

Marketing Vaccination Vaccine Clinical Trials 75

XTalks

DECEMBER 5, 2023

As its active ingredient, repotrectinib inhibits the proto-oncogene tyrosine-protein kinase ROS1 and the tropomyosin receptor tyrosine kinases (TRKs) TRKA, TRKB and TRKC. Other New Treatments for NSCLC on the Market The FDA has recently approved several other medications for various subsets of NSCLC patients.

Protein 115

Protein FDA Approval Medicine Trials 115

Pharmaceutical Technology

JUNE 10, 2023

RT-002 is under clinical development by Revance Therapeutics and currently in Phase II for Upper Limb Muscle Spasticity. The drug candidate is an injectable neurotoxin, purified botulinum toxin type A that targets synaptosomal-associated protein 25. It is developed based on TransMTS peptide technology.

Development 100

Development Drugs Clinical Trials Clinical Trials 100

Pharmaceutical Technology

JUNE 10, 2023

RT-002 is under clinical development by Revance Therapeutics and currently in Phase II for Upper Limb Muscle Spasticity. The drug candidate is an injectable neurotoxin, purified botulinum toxin type A that targets synaptosomal-associated protein 25. It is developed based on TransMTS peptide technology.

Development 100

Development Drugs Clinical Trials Clinical Trials 100

Pharmaceutical Technology

JUNE 5, 2023

BI-765063 is under clinical development by Boehringer Ingelheim International and currently in Phase I for Hypopharyngeal Cancer. It acts by targeting cells expressing SIRP-alpha (Signal Regulatory Protein alpha) and CD47. The drug candidate is a second generation check point inhibitor.

Production 100

Production Manufacturing Drugs Clinical Trials 100

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL). It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Head And Neck Cancer Squamous Cell Carcinoma. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL).

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

pharmaphorum

SEPTEMBER 28, 2021

VLA15 targets the outer surface protein A (OspA) of the Borrelia burgdorferi bacteria that causes Lyme disease, a tick-borne infection that causes a characteristic rash that can be accompanied by flu-like symptoms and – in some cases – debilitating tiredness and aches that can last for years.

Vaccination 105

Vaccination Vaccine Bacteria Protein 105

pharmaphorum

SEPTEMBER 18, 2020

A key focus of the three-year agreement with Moderna is to use the LNP platform to deliver gene-editing therapies to lung cells, correcting the defect in the gene and allowing functional CFTR protein to be made. Vertex will be responsible for preclinical and clinical development as well as commercial activities should they reach the market.

Gene Editing 105

Gene Editing Gene Vaccination Vaccine 105

pharmaphorum

FEBRUARY 22, 2021

Sanofi and GlaxoSmithKline are restarting clinical development of their COVID-19 vaccine, which was delayed after hitting a snag late last year. The vaccine is based around an adjuvanted recombinant protein, which will be tested in a new phase 2 study to assess the response including in older adults.

Vaccination 89

Vaccination Vaccine Recombinant DNA Technology Trials 89

Worldwide Clinical Trials

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

pharmaphorum

NOVEMBER 16, 2021

Injectable antibodies targeting PCSK9 have failed to take off as expected in the cholesterol-lowering market, but Merck & Co hopes to do much better with an oral alternative. ” AstraZeneca meanwhile is also working on an oral PCSK9 inhibitor that it acquired from Dogma Therapeutics last year and is in early clinical development.

Trials 98

Trials Drugs Sales Clinical Trials 98

pharmaphorum

NOVEMBER 12, 2020

The Pfizer/BioNTech vaccine is based on mRNA, which instructs the body to produce the “Spike” protein on the surface of the SARS-CoV-2 coronavirus that causes COVID-19. The Independent reported that AstraZeneca’s rival vaccine, which is based on a viral vector and is also nearing the end of clinical development, could be easier to work with.

Vaccination 105

Vaccination Vaccine Manufacturing Regulation 105

pharmaphorum

MARCH 9, 2021

Last year, Belgium’s ExeVir Bio raised 23 million euros ($27 million) to finance development of llama antibodies – and now it is ready to begin clinical development of a potential treatment that could be effective against emerging COVID-19 variants.

Antibody 98

Antibody Life Science Scientist Development 98

pharmaphorum

FEBRUARY 22, 2022

There’s been hardly any change in the use of clot-dissolving (thrombolytic) therapies for conditions like heart attack and stroke for decades, but a small Dutch biotech – TargED Biopharmaceuticals – is hoping to disrupt the market with a new and improved therapy.

Drugs 59

Drugs Protein Clinical Development Antibody 59

pharmaphorum

DECEMBER 7, 2020

China’s Sinovac has raised $515 million to increase its distribution and production capacity as clinical development of its potential coronavirus vaccine nears conclusion. Sinovac has a coronavirus vaccine in late-stage development, which is undergoing large-scale trials in Brazil, Pakistan, Indonesia and Chile.

Vaccination 59

Vaccination Vaccine Production Clinical Development 59

XTalks

OCTOBER 22, 2020

Pharmaceutical development of novel drugs is a complex and tedious process. The information shared between different multidisciplinary teams is critical to reducing the cost and ultimately accelerating the path of bringing a drug to market.

Manufacturing 98

Manufacturing Development Production Gene Therapy 98

pharmaphorum

JUNE 20, 2022

stake in French vaccines developer Valneva at a cost of €90.5 million (around $95 million) and rejigged the terms of their alliance to develop a shot for Lyme disease. The shot targets the outer surface protein A (OspA) of the Borrelia burgdorferi bacteria that causes Lyme disease. Pfizer has acquired an 8.1%

Vaccination 98

Vaccination Vaccine Licensing Licensing 98

pharmaphorum

JANUARY 12, 2021

Dark Antigens represent a new class of cancer-associated antigens that derive from the genomic “dark matter” – the portion of the human genome that is normally not expressed as protein. These sequences are usually silenced in healthy cells but are activated and presented on tumour cells.

Genome 80

Genome Genomics Licensing Licensing 80

pharmaphorum

SEPTEMBER 23, 2020

The Swiss biotech’s Nasdaq-listed shares were down almost 44% in pre-market trading today in the wake of the announcement. AC Immune’s chief executive Prof Andrea Pfeifer said the results were “surprising and disappointing”, given that tau protein has emerged as a promising target for AD therapy.

Protein 64

Protein Trials Drugs Clinical Development 64

The Pharma Data

AUGUST 10, 2020

Novavax is partnering up with the Serum Institute of India Private Limited (SIIPL) in a licensing deal to development and marketing of up to one billion doses of its potential recombinant COVID-19 vaccine candidate, NVX?CoV2373. The candidate is built upon a stable, prefusion protein and includes Novavax’s Matrix?M M adjuvant.

Vaccination 52

Vaccination Vaccine Licensing Licensing 52

XTalks

JULY 17, 2023

In a significant development, Astellas Pharma announced that the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) had accepted their applications for Priority Review and Marketing Authorization respectively for zolbetuximab. Furthermore, atezolizumab is in Phase II clinical development by F.

Clinical Trials 95

Clinical Trials Clinical Trials Trials Antibody 95

pharmaphorum

NOVEMBER 17, 2020

There are 11 potential COVID vaccines in late-stage clinical development and since Pfizer’s announcement two more groups from Russia’s Gamaleya Research Institute and US biotech Moderna have published favourable efficacy results.

Vaccination 77

Vaccination Vaccine RNA Clinical Trials 77

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Roots Analysis

FEBRUARY 27, 2024

The firm also has the right to obtain exclusive licenses to the Affimer proteins for clinical development and commercialization. What are Affimers : Affimers are small proteins that target and bind molecules on cellular surfaces in a manner analogous to monoclonal antibodies.

Antibody 52

Antibody Licensing Licensing Contract Manufacturing 52

pharmaphorum

AUGUST 10, 2020

Evrysdi is designed to treat SMA by increasing production of the survival of the motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

FDA Approval 59

FDA Approval Drugs Gene Therapy Protein 59

pharmaphorum

APRIL 23, 2021

The alliance is based on Obsidian’s cytoDRIVE platform, which uses already approved small-molecule drugs alongside gene-editing medicines to control the expression of protein from a gene sequence. For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52