EMA recommends three medicines for approval
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended three medications for approval during its April meeting.
The treatments proposed include Gilead’s HIV-1 treatment Biktarvy, AcelRx Pharmaceuticals’ hybrid pain therapy Dzuveo (sufentanil), and Obvius Investment’s Carmustine Obvius (carmustine), a generic treatment for brain tumours, non-Hodgkin’s lymphoma and Hodgkin’s disease.
CHMP is to consider a marketing authorisation for all three treatments.
Migraine drug could prove effective against nerve agents
Drug development company Proniras has entered a contract with the US Biomedical Advanced Research and Development Authority (BARDA) to explore Eli Lilly’s investigational migraine drug tezampanel as a potential treatment for nerve agent-induced seizures.
The contract with BARDA is to last five years, and is worth up to $89.5 million. Under its terms, Proniras will be responsible for the preclinical studies, clinical development and manufacture of tezampanel, while BARDA will finance the research. Payments are connected to milestones attained during studies, manufacturing and clinical development under the FDA’s Animal Rule, allowing Proniras to submit efficacy data from animal instead of human models.
“As recent events have clearly demonstrated, the need for medical countermeasures that can effectively treat nerve agent exposure is sadly more than theoretical,” Proniras co-founder Christopher Toombs said.
“Tezampanel holds great potential as a solution to this serious challenge, having shown favourable safety and pharmacokinetic profiles in clinical trials for acute migraine and demonstrating efficacy in preclinical models of nerve agent-induced seizures.”
MS drug could treat side effects of cancer treatment
Researchers from the Saint Louis University School of Medicine have found a multiple sclerosis drug could be used to treat severe pain experienced as an effect of the myeloma treatment drug bortezomib.
Bortezomib is known to cause chemotherapy-induced peripheral neuropathy (CIPN) in around 40% of patients. CIPN is a painful side effect of numerous cancer treatments which often causes patients to discontinue treatment or to report lower quality of life as a result of its effects.
“This growing problem is a major unmet clinical need because the increased efficacy of cancer therapy has resulted in nearly 14 million cancer survivors in the United States, many suffering from the long-term side effects of CIPN,” Saint Louis University School of Medicine professor of pharmacology and physiology Daniela Salvemini said.
New transplant protocol allows treatment of sickle cell disease
Doctors at the University of Illinois Hospital have successfully treated seven sickle cell disease patients using stem cells from donors previously thought to be incompatible, a procedure made possible due to a new transplant treatment protocol.
The new protocol allows family members of patients with aggressive sickle cell disease to donate stem cells if half or more of their human leucocyte (HLA) markers match. Previously, donors had to be ‘fully-matched’, meaning they have completely corresponding HLA markers.
HLA markers are proteins found on the surface of cells, which regulate the immune system by helping identify the cells which do and do not belong in the body. As they are inherited from parents, family members are the most likely donor candidates.
Researchers create GBM tumour models for cancer studies
Scientists at the Salk Institute have generated aggressive glioblastoma multiforme (GBM) tumours in an attempt to discern tumour activity and progression, and investigate new cancer drugs and personalised treatments.
The researchers grew the tumours in a dish by editing two genes in human cerebral organoids. The study was published on 24 April in the journal Cell Reports.
GBM, a form of brain cancer, has previously proven elusive due to the difficulty in observing the tumours’ progression in their natural environment. The recent study provides a model which has the potential to offer new insight into this underexplored area.
Boston doses first patients in two trials of DSP-7888
Boston Biomedical has initiated dosing of the first patient in two of its clinical trials evaluating the DSP-7888 investigational cancer peptide vaccine.
One of the trials is a Phase 1b study of DSP-7888 in combination with checkpoint inhibitors, called WIZARD101CI.
The multi-centre, open-label trial aims to enrol around 84 patients to deliver either DSP-7888 in combination with nivolumab or with atezolizumab.
NovaDigm publishes vulvovaginal candidiasis vaccine study results
NovaDigm Therapeutics has announced the publication of positive data from a Phase IIa study of its NDV-3A vaccine as a treatment for recurrent vulvovaginal candidiasis (RVVC).
NDV-3A is the company’s lead development candidate for the treatment or prevention of diseases caused by fungal or bacterial pathogens, including antimicrobial-resistant strains. It contains a recombinant form of the Candida albicans agglutinin-like sequence 3 (Als3) surface protein, which enables the infection’s invasion of human endothelial cells. Data from the trial found a single dose of NDV-3A with alum adjuvant to be safe, well-tolerated and to effectively reduce instances of RVVC in patients.
Top-line results from the study in August 2016 found it had achieved its primary endpoint of safety and tolerability. No significant differences were found between NDV-3A and placebo for injection site reactions and systemic reactions of grade 3 or greater. In participants under the age of 40, 42% of NDV-3A recipients were recurrence-free at 12 months following vaccination, compared to 22% of placebo recipients.
Novartis launches app for off-site trial participation
Novartis has announced the launch of its app FocalView, an ophthalmic digital research platform designed to allow patient participation in clinical trials from home, improving accessibility and flexibility.
The app was created using the Apple ResearchKit and aims to allow researchers to monitor disease progression using real-time and self-recorded data from patients. Researchers hope that designing clinical trials around patients’ routines will boost participation numbers and lead to more accurate and detailed data, accelerating treatment development.
“Because patients with eye diseases are often not as mobile, FocalView has the potential to offer tremendous benefit for the ophthalmic community and for researchers looking to develop better treatments for these patients,” FocalView medical advisor Dr. Mark Bullimore said.
“Collating validated patient-reported outcomes in clinical trial research is no longer a nice-to-have. This kind of data is fast becoming a critical element of research and development, because it offers a better reflection of real-world patient experiences, fosters better patient compliance and provides researchers with richer and more accurate data points.”
World Malaria Day: drug developers work towards beating malaria
Wednesday 25 April marks the World Health Organization’s (WHO) tenth annual World Malaria Day, with this year taking the theme ‘Ready to Beat Malaria’.
The awareness day represents a time for drug developers and researchers from across the globe to come together and assess the progress made in tackling one of the world’s oldest and deadliest diseases, as well as the challenges still faced. NGOs and commercial businesses take the day as a chance to pledge money towards malaria interventions, while fundraising events are held in support of treatment and prevention programmes.
In celebration of this year’s event, Nigeria-based firm Superior Pharmaceuticals has announced it will be distributing its malaria treatment Sumether for free. The treatment is an artemisinin-based combination therapy (ACT), which sees artemisinin-based compounds combined with a drug from a different class, in this instance the drugs artemether and lumefantrine. Sumether is recommended by WHO as a first-line treatment for malaria patients.
UTHealth receives funding for Phase II trial of MultiStem
University of Texas Health Science Center at Houston (UTHealth) in the US has secured funding to conduct a Phase II trial of MultiStem for use in the early treatment and prevention of complications following severe traumatic injury.
The funding has been provided through a public/private partnership that includes Medical Technology Consortium (MTEC), Memorial Hermann Foundation and Athersys.
MTEC’s funding makes up a sum of $2m, while Memorial Hermann Foundation has provided $1.5m. Athersys will provide its investigational stem cell therapy, MultiStem, in addition to regulatory and operational support for the trial.