Reshma Kewalramani, Vertex CEO (Vertex via YouTube)
Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.
The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.
Protein urine levels, or proteinuria, is a widely accepted biomarker for kidney function and a potential predictor for a patient’s long-term risk of going into kidney failure.
‘As a first result, I think it’s terrific,’ said Jonathan Himmelfarb, director of the Kidney Research Institute at the University of Washington, who was not involved in the study. ‘If it’s sustainable and it reflects improvement in an injury process in the kidneys, it is potentially very significant.’
Vertex sees the study as a proof of concept for its efforts to tackle a larger set of kidney diseases driven by the gene APOL1. Variants of the gene, which plays a role in the immune system, can help boost protection against parasitic infections, including human African trypanosomiasis, also known as African sleeping sickness.
But patients who carry one or especially two variants are also at a higher risk of developing kidney disease. Vertex cites figures that 50% of African Americans carry at least one variant and 13% carry two. It’s one of several factors — alongside access to healthcare and other social determinants of health — that account for the wide disparity in kidney disease between white and Black Americans.
Vertex’s molecule, VX-147, is designed to inhibit APOL1 and slow kidney injury.
‘It’s a very important target from a public health perspective,’ Himmelfarb said.
The data, while early, will likely come as welcome news for analysts, many of whom have remained skeptical of the company’s ability to repeat the success of its cystic fibrosis franchise. In a note Tuesday, Jefferies analyst Michael Yee placed the bar for success at a 15% reduction in proteinuria, but ‘the higher the better.’
The 47.6% reduction came from 13 patients, with Vertex excluding 3 patients who it said did not comply with trial protocols. There were no serious adverse events, the company said. The most common side effects were headache, back pain and nausea, which all occurred in over 15% of patients.
It’s the second successful readout for Vertex this fall after landmark results on a potential cure for type 1 diabetes came in October, albeit for just one patient.
Vertex now plans to move VX-174 into a larger pivotal trial for multiple types of APOL1-mediated kidney diseases. The company did not disclose details for the study, but the FDA has said that, for certain kidney diseases, the agency will grant accelerated approval based on reduction of proteinuria, providing a clear path to market if early results bear out in a larger study.
The company estimates 100,000 people in the US and Europe are currently living with kidney disease potentially addressable by their molecule.
While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.
Stéphane Bancel, Moderna CEO
Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.
Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?
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The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.
While noting the lack of transparency hindered the report’s results, ICER said that the ‘great majority’ of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines ‘fair access.’
Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)
Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.
The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.
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Philip Dormitzer, new GSK global head of vaccines R&D
GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.
Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.
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Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.
As a result, 140 of its staffers will be laid off.
The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a ‘select number of key European markets.’
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In a first, China has featured insulin in its centralized drug procurement program — but the bulk order comes at a sizable cost for multinational pharma players.
Novo Nordisk, Sanofi and Eli Lilly were among eight companies, domestic and foreign, whose insulin products won tenders from the Chinese public hospital system. In exchange, the drugs’ prices were cut, on average, by 48%, saving the medical institutions a collective $1.4 billion on the first batch of 210 million doses, according to state media.
Looking at past test samples, Dutch officials have detected the Omicron variant in cases dating back as far as Nov. 19, adding further evidence that the variant arrived in Europe well before the first cases were detected in South Africa.
The samples in question were taken on Nov. 19 and 23, according to the country’s health institute, RIVM.
‘It is not yet clear whether these people had also visited southern Africa,’ the RIVM said in a statement.
https://endpts.com/vertex-claims-early-win-in-kidney-disease-surpassing-analyst-expectations/
