FDA grants orphan drug designation to CervoMed’s dementia drug
Neflamapimod is an oral investigational small-molecule drug designed to treat frontotemporal dementia.
APIs
AstraZeneca claims phase 3 success with combination therapy for prostate cancer
High-level results from an ongoing clinical trial make Astrazeneca’s combination treatment the first to show benefit in a subtype of prostate cancer.
Eli Lilly announces positive Phase 2 results for Muvalaplin against heart disease
Muvalaplin significantly lowered lipoprotein(a) levels, reducing the risk of cardiovascular events in high-risk adults.
Tirzepatide prevents type 2 diabetes in 99% of prediabetic patients for 3 years
Results from the SURMOUNT-1 trial show that weekly injections of tirzepatide could stop the progression from pre-diabetes to type 2 diabetes in 99% of participants.
Overcoming nonspecific binding challenges in pharmacokinetic assays
Pharmacokinetic (PK) tests form an integral part of the drug development process, helping to ensure the safety and efficacy of new drugs.
Abbisko’s pimicotinib hits primary endpoint in phase 3 TGCT trial
Abbisko’s small molecule drug pimicotinib has smashed the primary goal of a phase 3 trial for the treatment of tenosynovial giant cell tumor (TGCT), taking the asset a step further to the market in China and elsewhere.
Senhwa Biosciences starts trial of silmitasertib in children with difficult to treat cancers
The Taiwanese biotech has launched a phase 1/2 trial to test its small molecule cancer therapy in children and young adults with relapsed or treatment resistant tumors.
FDA grants accelerated approval to Novartis’ Scemblix as a first-line leukemia therapy
Scemblix is the first drug to show superiority over all standard of care therapies for a rare type of leukemia.
Novo Nordisk’s semaglutide shows additional cardiovascular benefits in overweight patients
Clinical trial results have revealed that semaglutide treatment significantly reduced hospital admissions in overweight and obese adults with heart disease.
Axonis raises $115 million to overcome drug resistance in epilepsy
The Boston-based startup Axonis Therapeutics has bagged $115 million in a Series A round to fuel its pipeline of treatments for pain, epilepsy and other conditions.
Competitors race to launch GLP-1 drugs amid soaring weight loss demand
Demand for weight loss drugs targeting GLP-1 has exploded in recent years, leading to disruptions in supply chains. Here are the companies racing for approval of their own versions of these highly coveted drugs.
GSK beefs up US manufacturing site with $800 million investment
The big pharma company GlaxoSmithKline will inject up to $800 million into its manufacturing site in Marietta, Pennsylvania, to double its size and capacity.
Merck acquires cancer startup in a deal worth up to $1.3 billion
Merck’s acquisition of Modifi Biosciences will give it access to preclinical cancer drug candidates that target DNA repair processes.
NICE rejects Donanemab for NHS use despite MHRA approval
Donanemab marks a significant advancement in Alzheimer’s treatment, but NHS patients face a setback with NICE's rejection of its cost-effectiveness.
PTC Therapeutics’ Translarna misses European regulatory hurdle again
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna (ataluren), PTC Therapeutics’ treatment for the genetic disease Duchenne muscular dystrophy (DMD).
Seaport Therapeutics closes $225 million Series B to develop neuropsychiatric drugs
The Boston-based biotech will use the new fundraise to develop its pipeline of drug candidates for conditions such as major depressive disorder and generalized anxiety disorder.
Sanofi abandons phase 2 trial of Denali’s multiple sclerosis candidate
The big pharma company Sanofi has discontinued a phase 2 trial of U.S. partner Denali Therapeutics’ oditrasertib for the treatment of multiple sclerosis after the drug failed to perform.
How a CDMO is staying on top of API and drug product manufacturing trends
As active pharmaceutical ingredients (APIs) and medicines become more complex and the demand for efficiency rises, drug developers and manufacturers must adapt their strategies to keep pace with the evolving landscape.
Women in Science: Sharmeen Roy - 'pharmacy is the perfect blend of all my favourite subjects'
At the forefront of precision dosing, Sharmeen leads DoseMe's efforts to transform personalized medicine from lab research to bedside application.
Antag Therapeutics gets FDA clearance to start clinical trials with new obesity drug
The FDA decision will enable Antag Therapeutics to initiate the clinical development of its lead candidate drug, a first-in-class treatment for obesity.
Drug discovery and bringing new medicine to market - the future of AI in pharma
Artificial intelligence (AI) is now infiltrating core industries and according to Forbes, the market is expected to reach a value of $22.6 billion dollars by next year (2025).
Small molecules in focus as Recipharm bolsters global production facilities
Months after divesting seven European sites, the Swedish contract development and manufacturing organisation (CDMO) Recipharm is expanding its manufacturing capacity with undisclosed investments in its small molecule offerings.
LoQus23 Therapeutics raises £35m to advance therapy for Huntington’s disease
The proceeds of the financing will support the development of LoQus23’s lead candidate, a small molecule inhibitor that is expected to enter the clinic in 2026.
SK pharmteco to strengthen small molecule and peptide capacity with $260 million investment
SK Group’s contract development and manufacturing organization (CDMO) arm SK pharmteco is spending $260 million to expand its small molecule and peptide manufacturing muscle in South Korea.
CPHI 2024
CBD-based formulations from dsm-firmenich at CPHI 2024
At this October’s CPHI event being held in Milan, dsm-firmenich, is inviting stakeholders across the pharmaceutical market to immerse themselves in its patient-inspired innovations.
Women in Science - engineering life’s mysteries: Joaquima Guix Salichs’ journey
Joaquima Guix Salichs is the director of engineering and extraction technology at Bioiberica, where she leads a team of scientists focused on advancing extraction methods and solving large-scale industrial challenges.
Pfizer withdraws sickle cell disease drug Oxbryta amid safety concerns
The US big pharma company Pfizer has voluntarily withdrawn the oral sickle cell disease treatment Oxbryta from the market and halted clinical trials of the drug as data indicate a high risk of severe adverse events.
FDA greenlights first treatment for Niemann-Pick disease type C
The small molecule drug arimoclomol (Miplyffa) has got the green light from the U.S. Food and Drug Administration (FDA) for the treatment of the rare disease Niemann-Pick disease type C (NPC), which until now has lacked FDA-approved therapies.
Biohaven’s real-world trial shows positive results for spinocerebellar ataxia drug
The US biotech’s neurological drug candidate showed a reduction in disease progression of at least 50% and the company will now file with the FDA.
Insilico Medicine claims phase 2 win for AI-designed drug
The Hong Kong headquartered company says its idiopathic pulmonary fibrosis drug performed well in a phase 2a trial and met its primary endpoint.
NuraBio closes $140m fundraising to develop neuroprotective drugs
The series A financing will enable Nura Bio to advance its lead drug candidate into the next stage of clinical trials, shortly after successfully completing a first phase 1 clinical trial.
Boehringer Ingelheim score with phase 3 idiopathic pulmonary fibrosis win
The German big pharma will submit a new drug application to the US FDA after success at phase 3 for nerandomilast.
F2G raises $100 million to combat innovation 'paucity' in antifungals
One year after a rejection by the US Food and Drug Administration (FDA), the UK firm F2G has raised $100 million to push its candidate olorofim to the market for the treatment of rare fungal infections in 2026.
Neurocrine’s schizophrenia drug succeeds at phase 2, but dosing questions remain
Neurocrine Biosciences has achieved late-stage success with muscarinic M4 selective agonist in schizophrenia.
Nasal powder shows promise in boosting immune defense
The INNA-051 formulation is designed to enhance the body’s natural immune response, targeting individuals aged 60 and over who are at increased risk for complications from respiratory viral infections.
Astellas begins phase 3 trial of fezolinetant for VMS in breast cancer
Astellas Pharma Inc. has announced the initiation of its phase 3 pivotal study, Highlight 1, for fezolinetant, an investigational nonhormonal oral treatment for vasomotor symptoms (VMS) in women with breast cancer receiving adjuvant endocrine therapy.
H1 Connect links doctors to global humanitarian missions
H1's decision to partner with GRM for this critical initiative in Ukraine was driven by a strong commitment to harness the power of global healthcare networks to provide humanitarian aid in regions that need it most.
Breaking barriers in science: Thermo Fisher's Melanie Adams-Cioaba on innovation and inclusion
In the rapidly evolving field of drug discovery, Melanie Adams-Cioaba stands at the forefront, leading transformative advancements in cryo-electron microscopy (cryo-EM) at Thermo Fisher Scientific.
Gilead's Livdelzi granted accelerated FDA approval for primary biliary cholangitis
Gilead Sciences, Inc. has announced that the US Food and Drug Administration (FDA) has granted accelerated approval for Livdelzi (seladelpar) for the treatment of primary biliary cholangitis (PBC), a rare and chronic autoimmune disease affecting the bile...
Ayvakit's explosive 185% revenue growth: A game-changer in rare disease treatment
Ayvakit, a groundbreaking therapy developed by Blueprint Medicines, has witnessed a remarkable 185% year-over-year revenue growth in the second quarter of this year (2024).
Lonza boosts clinical services with new bottling capabilities
Lonza, a global leader in the pharmaceutical, biotech, and nutraceutical markets, has announced a significant expansion of its clinical manufacturing services at its small molecules site in Bend, Oregon.
Tonix's trial results showing promise in fibromyalgia treatment
Tonix Pharmaceuticals has announced promising phase 3 trial results for TNX-102 SL (sublingual cyclobenzaprine HCl), a treatment targeting fibromyalgia.
Lykos’ heartache over FDA setback as new trial for groundbreaking PTSD treatment ordered
In a blow to millions of Americans battling post-traumatic stress disorder (PTSD), Lykos Therapeutics announced today that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its new drug application (NDA) for...
Rejuvenate Biomed and SAS unveil AI tool to accelerate age-related drug development
Rejuvenate Biomed and SAS have announced a creative partnership aimed at making big changes to drug discovery through the development of a user-friendly, AI-powered tool.
How CDMOs can optimize operations and balance costs ahead of the 2032 BioSecure Act
As the BioSecure Act approaches its 2032 implementation, Contract Development and Manufacturing Organizations (CDMOs) must prepare for significant industry shifts.
Revolutionary Alzheimer's drug donanemab approved – what does this mean for the future of care?
The approval of donanemab marks a significant breakthrough in Alzheimer's treatment, promising transformative changes in patient care and research.
Biologics driving M&A as Agilent snaps up specialist BIOVECTRA for $925 million
As advanced therapies drive increasing market consolidation, the US giant Agilent Technologies has acquired the Canadian contract development and manufacturing organization (CDMO) BIOVECTRA for $925 million.
Ask the experts: Should pharmacists take on more responsibility or is it a stretch too far?
Back in March this year (2024) under the last UK Conservative government, plans were announced to give greater powers to pharmacy technicians, dental therapists and hygienists after two public consultations.
New drug safety era: Transparency’s portable test kits target every substance
Transparency, a harm reduction organization specializing in at-home drug testing kits, has announced its official launch as the parent organization of the Bunk Police.
ViiV Healthcare's new data shows promising maternal and pregnancy outcomes for Apretude use
ViiV Healthcare has released new data demonstrating the safety and efficacy of Apretude (cabotegravir long-acting for PrEP) during pregnancy.