Regeneron Pharmaceuticals (Nasdaq:REGN) has received an indication for Eylea (aflibercept) injection for preterm infants with retinopathy of prematurity (ROP).  The approval is the first pharmacologic treatment for the condition, which affects roughly 50% to 70% of infants with a weight less than 1250 grams (2.76 pounds) at birth. The condition is more common in infants…

NEJM has published positive results from the KOMFORT Phase 2 trial of oral difelikefalin for nostalgia paresthetica, a condition involving itching, burning or tingly sensations on the back. Cara Therapeutics (Nasdaq:CARA) is the developer of the drug, which won FDA approval in an injectable form for moderate-to-severe pruritus in hemodialysis patients in 2021. That form…

The story of the scleroderma drug candidate FT011 is one of “tenacity and persistence,” said Darren Kelly, CEO and managing director of Certa Therapeutics, which is developing the drug. The drug was discovered around 2006 by Kelly’s laboratory at the University of Melbourne and Spencer Williams of the Bio21 Institute. “It was really [Williams] being the…

South San Francisco–based Neuron23 has started the first-in-human Phase 1 trial of NEU-723 for the treatment of Parkinson’s disease. NEU-723 is a highly potent and selective leucine-rich repeat kinase 2 (LRRK2) inhibitor. This clinical trial will evaluate the safety, tolerability and pharmacokinetics of NEU-723 in healthy volunteers. A targeted approach The company has also collaborated…

Certa Therapeutics, a Melbourne-based biotech company, announced the results of its Phase 2 clinical trial for FT011, a novel oral therapy for chronic fibrosis. The trial showed clinically meaningful improvements in more than 60% of scleroderma patients after 12 weeks of treatment. In the Phase 2 study, 60% of patients treated with a 400 mg…

Johnson & Johnson subsidiary Janssen (NYSE:JNJ) has revealed positive results from the proof-of-concept Phase 2 UNITY clinical trial of nipocalimab for pregnant adults with a high risk of developing severe hemolytic disease of the fetus and newborn (HDFN). HDFN is a rare condition affecting between 3 and 80 out of 100,000 newborns or fetuses each…

In the late 2000s, many companies, particularly Big Pharma, exited the field of central nervous system (CNS) drug development and, particularly psychiatry. “Big Pharma exited the space because they were facing their patent cliff at the time and had to make some critical decisions about where to invest their resources,” said Emer Leahy, CEO of…

Terran Biosciences, a neurology and psychiatry-focused startup, has announced the publication of patent applications under the Patent Cooperation Treaty (PCT) for its novel prodrugs of DMT, 5-MeO-DMT and MDMA. Claim of superior pharmacokinetics The company believes the prodrugs have superior pharmacokinetic profiles compared to the unmodified forms of these Schedule I substances. Additionally, it believes…

A Swiss study found that the monovalent Moderna (Nasdaq:MRNA) COVID-19 booster vaccine may be linked to a higher risk of a type of hives known technically as chronic spontaneous urticaria (CSU). In a cohort of the study based in the Swiss canton of Vaud, 90% of people who received the mRNA-based Moderna COVID-19 booster and developed…

GSK (NYSE:GSK) has received FDA approval for Jesduvroq, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), as a daily treatment for anemia associated with chronic kidney disease in adult dialysis patients. Jesduvroq is the first new medicine for anemia treatment in over 30 years, GSK noted. The drug is the only HIF-PHI to win FDA…

In the U.S., most cancer patients receive care in a community setting, a unique care model that traces its origins back to the original war on cancer. Since then, the National Cancer Institute and other organizations have supported this model of pushing more cancer care to community settings. Community oncology groups are poised to conduct a…

GSK (NYSE:GSK) announced that FDA has granted Orphan Drug Designation (ODD) to Benlysta (belimumab) for systemic sclerosis (SSc), a rare autoimmune disease that can affect multiple organs. Interstitial lung disease (ILD) is the leading cause of death in SSc patients. The monoclonal antibody Benlysta is a B-lymphocyte stimulator (BLyS) specific inhibitor that binds to soluble…

Several companies, including GSK, Janssen, Novartis and Pfizer, have announced that they are cutting their pipelines as they release their full-year 2022 financial results. The following are the drugs removed from these companies’ pipelines. Bristol Myers Squibb Anti-CTLA-4 NF: The oncology monoclonal antibody candidate directed against the human T-cell receptor cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) known as BMS-986218 was…

The clinical-stage biopharma Athira Pharma (Nasdaq:ATHA) has published a review paper stressing the need for new treatments for Alzheimer’s disease. The paper also states that one of its small molecule drugs, fosgonimeton, holds potential for treating Alzheimer’s. The Journal of Alzheimer’s Disease published the paper titled “The Case for a Novel Therapeutic Approach to Dementia:…

The biotech Ymmunobio has acquired ownership rights to neuronal pentraxin receptor (NPTXR) antibodies, which could promise in treating several gastrointestinal cancers. Basel, Switzerland–based Ymmunobio reached an agreement with Nagoya University in Japan in 2022 to transfer rights to the antibodies. Professor Mitsuro Kanda of Nagoya University invented the antibodies. A 2020 article published in Molecular…

New York City–based Pfizer (NYSE:PFE) announced that its full-year 2022 revenues were $100.3 billion, a record for the company. Pfizer has more than doubled its revenue compared to 2020 when it generated $42.9 billion in sales. In 2021, its revenue hit $81.3 billion. But the company will likely have trouble sustaining that momentum, considering its revenues,…

Yale School of Medicine has established a research center, the Yale Center for the Science of Cannabis and Cannabinoids, to investigate how cannabis and cannabinoids affect neurodevelopment and mental health. Led by Dr. Deepak Cyril D’Souza, an expert in cannabinoid pharmacology, the center will use a multidisciplinary approach to study the acute and chronic effects…

The biopharma CureVac (Nasdaq:CVAC) and its partner GSK (NYSE:GSK) have announced positive extended preliminary data from two Phase 1 mRNA vaccine trials, one involving COVID-19 and another seasonal flu. The company says the positive data support the next stage of clinical testing for modified mRNA COVID-19 and flu vaccines in 2023. CureVac, which struggled to…

The Multidisciplinary Association for Psychedelic Studies (MAPS) founder and executive director, Rick Doblin, said in an interview with Fox Business Live that the FDA would approve MDMA-assisted therapy for PTSD in April or May of 2024. Last year, MAPS announced the completion of the MAPP2 Phase 3 study, which is the organization’s second to use MDMA (3,4-methylenedioxymethamphetamine)…

The FDA has withdrawn the emergency use authorization (EUA) for AstraZeneca’s Evusheld (tixagevimab with cilgavimab) as a COVID-19 pre-exposure prophylaxis owing to the emergence of new variants. Evusheld has been found to be ineffective against several omicron subvariants, including BQ.1, BQ.1.1, BF.7, BF.11, BA.5.2.6, BA.4.6, BA.2.75.2, XBB, and XBB.1.5.1. The XBB.1.5 subvariant is becoming more…

The most common types of drugs in drug development are small molecules and biologics. Small molecules have a long history in the pharmaceutical industry and remain a pillar of modern medicine.  Conversely, biologics are more complex molecules derived from living organisms.  In this article, we provide a brief overview of common drug types, including biologics, small…

A recently published paper in Cardiovascular Research found that a gene frequently found in centenarians could safeguard heart functionality. In a rodent model, a team of researchers found that the bactericidal/permeability-increasing fold-containing family-B member-4 gene (BPIFB4) gene protected against deterioration of heart function in middle-aged mice (14 months old). The researchers, led by Professor Paolo Madeddu of the University…

The FDA has accepted a supplemental New Drug Application (sNDA) for Jardiance (empagliflozin) tablets as a potential therapy to reduce the risk of kidney disease progression and cardiovascular death in adults with chronic kidney disease (CKD). Some 37 million people in the U.S. have CKD. Lilly (NYSE:LLY) is developing the drug with Boehringer Ingelheim. The…

Osteoarthritis (OA), a degenerative joint disease affecting the knee and other joints, affects hundreds of millions of people worldwide. Genascence (Palo Alto, California) is developing a gene therapy known as GNSC-001 for OA of the knee. A potent inhibitor of interleukin-1 (IL-1) signaling, GNSC-001 is a recombinant adeno-associated virus vector with a coding sequence for…

Last year, Lilly’s (NYSE:LLY) Alzheimer’s monoclonal antibody donanemab appeared to be a superior therapy to aducanumab from Biogen (Nasdaq:BIIB) and Eisai (OTCMKTS:ESALY), the first such antibody to win FDA approval. But facing criticism over its decision to grant accelerated approval to aducanumab, FDA has sent Lilly a complete response letter for the accelerated approval submission of…