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Clinical data management on-demand firm TrialStat has implemented Pure Storage’s FlashArray and Evergreen Storage subscriptions to expedite research in clinical trials.

The solutions will also enable always-on data management services for TrialStat clients.

Researchers in clinical trials gather and assess large quantities of information on the safety and efficiency of novel vaccines, drugs and medical devices.

The pace with which researchers obtain and work with the data determines the efficiency of the trial.

To address this, TrialStat streamlines data management with an electronic data capture (EDC) platform that includes reporting in real-time, analytics and data extraction.

TrialStat substituted its traditional storage solution with Pure Storage FlashArray//X and could potentially provide researchers with quick and real-time access required to efficiently conclude trials.

The FlashArray’s speed and capacity freed up by TrialStat’s 11:1 data deduplication can expedite trial research and allow the company to devote time for novel innovations for storage maintenance.

Using Pure’s Evergreen Storage subscription, TrialStat can update without interruptions and extend its infrastructure as required.

TrialStat president Chris Hamelin said: “Storage is now a competitive advantage for TrialStat, supporting clinical trials running 24/7 with real-time data access and reporting, and an uptime guarantee.

“We are proud to say that our solutions running on Pure Storage give our customers a strategic advantage by improving the time it takes to run clinical trials while controlling cost.”

Synairgen has completed the recruitment of 610 subjects for its global Phase III SPRINTER trial (SG018) of inhaled interferon beta, SNG001, to treat hospitalised Covid-19 patients.

The company’s double-blind, randomised, placebo-controlled Covid-19 trial is being conducted in 17 countries.

Statistical analysis and quality assurance will be concluded once the last subjects complete the initial trial period of 35 days.

Upon successful completion of the Phase III study, the company plans to file an emergency use authorization application with the US Food and Drug Administration (FDA) to treat Covid-19 patients who require hospitalisation.

“We believe that SNG001, our investigational inhaled formulation of interferon beta, a naturally occurring, broad-spectrum antiviral protein, could offer a compelling new treatment option.”

The company will continue to work together with Parexel division Parexel Biotech on the SPRINTER study.

The FDA granted Fast Track status for SG018, which the company primarily aims to develop as potentially the first broad-spectrum antiviral treatment that would be host-targeted, and directly delivered into the lungs.

In May 2021, Synairgen announced the results from the home cohort of its SG016 Phase II clinical trial of SNG001 in Covid-19 patients. In this trial, the therapy showed a drop in the hospitalisation rate in patients.

The US Food and Drug Administration (FDA) has approved investigational new drug application of Modulation Therapeutics to commence human trial of its drug, MTI-201, to treat uveal melanoma.

Established in 2011, Modulation is a West Virginia University (WVU)-based start-up firm that focuses on the early discovery and development of translational therapies.

The company was jointly founded by WVU Cancer Institute basic research associate director Lori Hazlehurst and WVU Cancer Institute researcher Mark McLaughlin.

The FDA approval to initiate the trial is based on substantial preclinical research. MTI-201 was developed for the treatment of uveal melanoma after tumour spread to other parts of the body.

The drug attains its precision by targeting a particular protein biomarker found in higher concentrations in the uveal melanoma cells.

MTI-201 does not damage healthy cells as the protein is not abundant in these cells.

McLaughlin said: “The current therapy for primary uveal melanoma is irradiation of the eye.

“Because the radiation is coming from the outside, it causes all sorts of collateral damage while it’s going into the site where the tumour actually is.

“Our drug could be injected right into the eye at a very, very low dose. It wouldn’t be circulating throughout the body. It would reach the target immediately.”

In the future, the researchers anticipate that the drug could potentially be utilised for treating localised cases of uveal melanoma before it progresses to other regions.

Although uveal melanoma is a rare illness overall, it is the most common kind of eye cancer.

Biogen and Eisai have reported that their therapy, Aduhelm (aducanumab-avwa), substantially reduced plasma p-tau181 in two Phase III EMERGE and ENGAGE clinical trials.

A monoclonal antibody, Aducanumab-avwa acts against amyloid-beta.

The latest data from nearly 7,000 plasma samples obtained from more than 1,800 subjects in the Phase III trials showed that there is a statistically significant link between the decline in plasma p-tau, as well as reduced cognitive and functional decline in Alzheimer’s.

Biogen noted that Aduhelm reduced tau pathology as assessed by plasma p-tau18, which is an Alzheimer’s-specific biomarker, versus placebo in the latest assessment.

The statistically significant link between change in plasma p-tau181 and amyloid-beta plaque reduction also demonstrates the impact of Aduhelm treatment on the two crucial pathological features of the disease.

Biogen Research and Development head Alfred Sandrock said: “We now have robust and concordant data that Aduhelm has an effect on two core defining pathologies of Alzheimer’s disease, and substantial evidence of treatment correlation between changes in plasma p-tau181 and the slowing of disease progression.

“We are committed to continuing to generate data, and we believe these new findings can help inform treatment choice and advance Alzheimer’s research including in diagnosis and disease monitoring.”

In July 2021, the companies revealed the design of a real-world observational Phase IV ICARE AD-US study of Aduhelm in Alzheimer’s disease.

This study is intended to obtain real-world data on the long-term efficacy and safety of a 100mg/mL injectable dose of Aduhelm.

The FSSAI has announced a new regulatory framework for foreign facilities that transport food to India in order to ensure the safe import of food products.

Foreign food manufacturing facilities that produce certain kinds of products for export to India will be required to register with the Food Safety and Standards Authority of India and may be subjected to inspection by Indian food safety officials, according to the regulations. From June 1, the Food Safety and Standards (Import) First Amendment Regulations, 2021, will take effect.

International food safety authorities, such as the US Food and Drug Administration, follow similar processes.

Registration Required

According to the FSSAI, depending on its risk assessment, it would designate the types of food items for which multinational factories must register with the regulator from time to time.

“Foreign food production establishments falling under such categories that wish to export such articles of food to India must register with the Food Authority before doing so,” the regulation announcement continued.

Routine vaccination with human papillomavirus vaccines have resulted in an 87% reduction in cervical cancer cases in England, and could even see the need for smear tests phased out in future, according to a new study.

The jabs with have been offered by the NHS to girls between the ages of 12 and 13 since 2008, with more than 10 million doses administered since then, and from September 2019 was also offered to boys in the same age group.

Uptake in girls has been around 85% nationally, although there are variations with lower take-up among less well-off people. The first vaccine used was GlaxoSmithKline’s Cervarix, which was replaced by MSD’s Gardasil in 2012.

The research by King’s College London is the first direct evidence that HPV vaccinationmakes a substantial difference to the risk of developing cervical cancer.

Earlier studies have however demonstrated clearly that the jab can reduce transmission of the virus in the community as well as other consequences of infection such as genital warts.

The study – published in The Lancet – shows that thousands of women have been protected from cervical cancer and the development of precancerous changes to cells in the cervix.

Cases among women who were among the first to receive the vaccine and are now in their 20s – while rare – dropped from around 500 per year to just five over an 11-year period mid-1029.

The vaccine also reduced cervical cancer incidence by 34% in those who received it aged 16 to 18, and by 62% if aged 14 to 16.

Overall, the study estimates that along with 450 avoided cervical cancer cases, around 17,200 pre-malignant lesions called cervical intraepithelial neoplasia (CIN) were prevented by the HPV programme.

It is thought that almost all cases of cervical cancer are caused by HPV, which is also associated with some other forms of cancer affecting the mouth and throat, vagina, vulva, anus and penis.

Cancer Research UK, which funded the research, said the findings were “historic”, but stressed that screening is also imperative to identify cases, particularly among the unvaccinated.

There has been concern that vaccine hesitancy associated with the COVID-19 pandemic might impact the take-up of other vaccines, including the HPV shot, and the new data should go a long way to reinforcing the benefits of taking it.  All people eligible for the shot can catch-up until their 25th birthday.

“We’ve been eagerly awaiting these results since the introduction of the vaccination programme,” said CRUK’s chief executive Michelle Mitchell.

“Around 850 women die from cervical cancer each year in the UK, so we have the chance to save many lives,” she added.

An oral antiviral drug developed by Pfizer has been shown to dramatically cut the risk of hospitalisation or death from COVID-19 when given in the early stages of infection.

The phase 3 trial found that Paxlovid – which combines new protease inhibitor PF-07321332 with already-approved HIV drug ritonavir – reduced the risk of hospitalisation or death by 89% when given to newly-diagnosed adults who were at risk of developing severe COVID-19 in the EPIC-HR trial.

When the drug was given within three days of symptom onset, 0.8% of patients taking it had died or been hospitalised after 28 days’ follow-up, compared to 7% of a control group who got a placebo pill.

No deaths were reported among people who received Paxlovid, with 10 deaths in the placebo arm, according to Pfizer, which halted the study earlier than expected because the data were so positive.

There are hopes that oral drugs that can easily be administered to people in the community could open up a new front against COVID-19, helping to save lives and reducing the burden on healthcare services by keeping people out of hospital.

The data comes hard on the heels of the UK’s approval of Merck & Co/MSD and Ridgeback Therapeutics’ polymerase inhibitor  Lagevrio (molnupiravir), which is the first oral antiviral for COVID-19 to be authorised for use.

The top-line data for Pfizer’s drug seem favourable at first glance with Merck and Ridgeback’s MOVe-OUT trial, which revealed a 50% reduced risk of hospitalisation or death in compared to placebo with molnupiravir, with all the caveats of trying to compare studies with different designs and endpoints.

There have also been come concerns about the risk of birth defects with molnupiravir due to its mechanism of action that can lead to DNA changes, and Pfizer has been quick to point out that it has found no evidence of “mutagenic DNA interactions” with its protease inhibitor.

The company reckons the data should be enough to secure emergency use authorisations for Paxlovid, and so attention is already turning to access, particularly in lower-income countries.

The company said it plans to adopt a tiered pricing approach to the drug depending on a country’s income, and is “exploring” contract manufacturing deals to for less well-off nations.

Merck meanwhile has already gone a step further, making a deal with the UN-backed Medicines Patent Pool (MPP) to grant royalty-free licenses to generic companies who can manufacture molnupiravir in 105 low- and middle-income countries around the world.

The UK has agreed a supply deal for 250,00 courses of Paxlovid, without revealing the purchase price, along with 480,000 doses of molnupiravir. Pfizer said it is already in advanced purchase agreements with multiple countries and in negotiations with several others for its drug.

Novartis has chalked up another successful trial for its targeted factor B inhibitor iptacopan, an orally-active drug trying to challenge antibody-based therapies for diseases associated with the complement pathway.

A phase 2 trial of iptacopan in patients with the rare kidney disease C3 glomerulopathy (C3G) – which causes progressive kidney failure and has no approved treatments – showed reductions in kidney damage with the drug that Novartis said were “clinically important.”

C3G often affects adolescents and young adults and can lead to kidney failure transplant, with the disease in some cases continuing to attack the donated organ.

In the trial, which included post-transplant patients, 12 weeks of treatment with iptacopan at a dose of 200 mg/day added to background therapy for C3G reduced levels of proteinuria, a marker for kidney damage, by around 45% compared to baseline.

Patients whose C3G returned after a transplant also showed significantly reduced C3 protein deposits in kidney biopsies with iptacopan, which is now in a phase 3 trial for C3G called APPEAR-C3G.

“These results are important for patients with C3G because proteinuria is a key risk predictor for kidney disease progression, and deposits of C3 protein ultimately cause inflammation and kidney damage,” commented lead investigator Edwin Wong of Newcastle University in the UK.

The new results follow positive results in phase 2 trials for iptacopan in other complement-mediated trials including paroxysmal nocturnal hemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS), which have also progressed to late-stage trials.

The first-in-class drug also being tested in another rare kidney disease, idiopathic membranous nephropathy, in phase 2.

Novartis hopes iptacopan can provide an oral alternative to antibodies like AstraZeneca/Alexion’s $4 billion C5 inhibitor Soliris (eculizumab) and follow-up Ultomiris (ravulizumab), which are both approved for PNH and aHUS but not for C3G, for complement diseases.

Analysts at Jefferies think iptacopan could hit $3.6 billion in peak annual sales if it gets approved for all its target indications.

Competition is emerging in C3G however from Apellis Pharmaceuticals – which won FDA approval earlier this year for its injectable C3 inhibitor Empaveli (pegcetacoplan) for PNH earlier this year and is developing the drug for C3G in phase 2 trials – as well as ChemoCentryx’ oral C5a receptor inhibitor Tavneos (avacopan) which is also in mid-stage development.

Another potential oral rival from Alexion – Factor D inhibitor danicopan – was discontinued for C3G last year after a failed phase 2 trial, although follow-up ALXN2050 is still in development.

AbbVie’s experimental subcutaneous Parkinson’s disease drug ABBV-951 hit the mark in a 12-week Phase III study in patients with an advanced form of the disease. The company announced that patients who received daily doses of the drug showed statistically significant increases in “On” time without dyskinesia, the spasms associated with the disease.

AbbVie’s Phase III study compared ABBV-951 to oral treatment with levodopa/carbidopa. In addition to the increases in “On” time hours, AbbVie said the study also showed a significant reduction in “Off” time hours. People diagnosed with Parkinson’s often deal with times classified as “On” and “Off.” The “On” time refers to when patients are responding to medication and symptoms are decreased. The “Off” periods are characterized by the re-emergence of Parkinson’s symptoms. As the disease progresses, “Off” periods in patients can increase in frequency and severity.

With the positive Phase III data in hand, AbbVie is eying global regulatory submissions for ABBV-951, a solution of levodopa and carbidopa prodrugs for continuous subcutaneous infusion. Dr. Michael Severino, vice chairman and president of AbbVie, said the results of the Phase III study highlight the potential for ABBV-951 as a treatment for advanced Parkinson’s disease patients.

Data from the study showed that after 12 weeks of treatment, the “On” time increase was 2.72 hours for ABBV-951 compared to 0.97 hours for oral levodopa/carbidopa, which are standard-of-care treatments for Parkinson’s. According to AbbVie, improvements in “the “On” times were seen in the first week among some of the patients who participated in the Phase III study. The continuous subcutaneous treatment reduced the “Off” times. Phase III data showed that, after 12 weeks of treatment, the“Off” times were reduced by an average of .75 hours for ABBV-951 compared to 0.96 hours for oral levodopa/carbidopa.

Dr. Jason Aldred, a clinical associate professor at Washington State University’s Elson S. Floyd College of Medicine and a principal investigator of the study, called the Phase III data promising. He said the data demonstrate “positive results on a key endpoint” that are used to assess the efficacy of treatments for patients with advanced Parkinson’s. The Phase III study included approximately 130 adult participants with advanced Parkinson’s disease.

AbbVie said full results from the Phase III study would be presented at a future medical meeting or submitted for publication in a peer-reviewed journal.

The continuous subcutaneous treatment maintained a solid safety profile in the study. The majority of the adverse events reported were non-serious and mild to moderate in severity in the ABBV-951 group, the company Parkinson’s disease is a neurodegenerative disorder that affects approximately 10 million people across the globe. It affects dopaminergic neurons in a part of the brain. Disease symptoms typically develop slowly and could begin with slight tremors in the hands, slowness of movement, or limb rigidity. Symptoms include tremor, slowed movement, rigid muscles, impaired posture and balance, loss of automatic movements, and speech changes.