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…
Big Pharma companies trimming pipelines in 2023
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. GSK GSK3915393: GSK said in a quarterly earnings call that it had pulled the plug on GSK3915393, an inhibitor of transglutaminase…
Athira Pharma calls for a fresh approach to treating Alzheimer’s disease
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:…
Noble Leads with its User-Centered Design Strategy
By Tim McLeroy, Executive Director of Marketing and Patient Services, Noble International At Noble, the patient journey drives everything we do. Our patient-centered approach pushes us to fully understand and characterize patient needs, providing data and experiences that help us ease the burden and anxiety of self-administered treatments. While drug delivery devices are well designed…
Ymmunobio gets ownership rights to NPTXR antibodies
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…
Pfizer tops $100B in revenue in 2022, but expects dwindling COVID-19 vaccine sales
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 opens center to study cannabis and cannabinoids
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…
CureVac reports positive outcomes for COVID-19 and flu mRNA vaccines in seniors
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…
MAPS predicts FDA approval for MDMA-assisted therapy in 2024
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)…
New COVID-19 variants prompt FDA to revoke Evusheld’s EUA
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…
An overview of common drug types: biologics, small molecules and cell/gene therapy
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 pandemic waiting to happen: Acute radiation syndrome and the immediate need for nuclear countermeasures
In case of nuclear war, or even a limited nuclear strike, the greatest threat to human health, besides blast and burn injuries, is from acute radiation syndrome or ARS. This ominous and little-known acronym refers to the acute illness that ensues from significant partial- or whole-body irradiation. High turnover tissues such as the skin, bone…
BPIFB4 gene therapy could protect against heart aging
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…
Lilly gets FDA review for empagliflozin in chronic kidney disease
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…
Genascence believes gene therapy can transform the treatment of knee osteoarthritis
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…
FDA hands Lilly’s Alzheimer’s drug donanemab complete response letter
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…
Activist investors pressure MindMed to accelerate clinical development, trim costs
The psychedelic therapeutics market could be worth billions of dollars in the coming years. The FDA has awarded breakthrough therapy designation to psilocybin and MDMA, potentially accelerating its approval of the currently Schedule I substances. Despite the promise, several psychedelic therapeutics companies have seen substantial drops in stock price. For instance, MindMed has seen its…
Takeda and Boehringer Ingelheim selected as top pharma employers
The Top Employers Institute has named Takeda (TSE:4502/NYSE:TAK) and privately-held Boehringer Ingelheim to its list of 15 companies to win global Top Employer certification for 2023. The Top Employers Institute rankings are based on data from its HR Best Practices Survey, which spans 20 topics, including people strategy, work environment, talent acquisition, learning, diversity and inclusion, well-being…
4 top drug discovery innovations of 2022
Late last year, the Galien Foundation highlighted several drug discovery innovations in its annual Prix Galien USA Award Winners, which specifically highlighted drugs from Regeneron and Amgen as well as a platform from Exscientia and the incubators BioLabs and LabCentral. The foundation recently hosted a webinar featuring several executives from the respective winning companies discussing their view…
Moderna’s RSV vaccine mRNA-1345 was more than 80% effective in a Phase 3 study
mRNA vaccine company Moderna (Nasdaq:MRNA) plans on submitting for regulatory approval for its investigational RSV mRNA-1345 vaccine after it met primary efficacy endpoints in the Phase 3 ConquerRSV study (NCT05127434). The RSV data are “a clear positive for MRNA” and represent a good omen for upcoming mRNA Phase 3 influenza data, noted UBS in a…
Add-on Vraylar provides new option for treating major depressive disorder
Traditional antidepressant therapy doesn’t work for all depressed patients. About four out of 10 patients see a noticeable benefit from an antidepressant after six to eight weeks, according to Dr. Arif Khan, a board-certified psychiatrist. For the six out of ten who don’t see a benefit, “the first choice is to switch to a different…
25 potential blockbusters to keep an eye on in 2023
In 1986, the ulcer drug Tagamet from Smith, Kline and French (now GSK) emerged as the first blockbusters, generating more than $1 billion in annual revenue. Fast-forward to 2021, and the COVID-19 vaccine from Pfizer and BioNTech generated close to $60 billion in cumulative revenue. That same year, AbbVie’s Humira (adalimumab) eclipsed $20 billion in sales…
Federated learning pact between Penn Medicine and Intel boosted glioblastoma detection capabilities
A research alliance between Penn Medicine and Intel aggregated data from 6,314 glioblastoma (GBM) patients at 71 sites across six continents over three years. The study made use of federated learning – a distributed machine learning (ML) approach – to facilitate the identification of malignant brain tumors without compromising patient privacy. The research led to…
Genentech on how Lunsumio could shift the treatment landscape for R/R FL
In late 2022, the FDA approved Genentech‘s Lunsumio (mosunetuzumab-axgb), a bispecific antibody for relapsed or refractory follicular lymphoma (R/R FL) patients who have received at least two prior systemic therapies. We recently caught up with Ginna Laport, VP, global head of lymphoma/CLL development franchise at Genentech, to learn more about how the drug could change the…
QurAlis homes in on drug candidates for subset of ALS patients
The Cambridge, Massachusetts–headquartered biotech QurAlis kicked the year off by launching a Phase 1 clinical study of QRL-101 (QRA-244), a potentially novel selective Kv7.2/7.3 ion channel opener to treat hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS). QRL-101 targets Kv7.2/7.3 potassium channels. Kv7 modulation can potentially lower spinal and cortical motor neuron excitability, potentially improving…