Germany’s Cardior is conducting clinical studies with an antisense oligonucleotide which inhibits a cellular microRNA believed to be involved in the pathology of heart failure, and is attracting attention from other pharma companies interested in the potential of this new mechanism of action.

The Swiss major has been enthusing about its population-based agreement in the UK for the investigational gene silencing cholesterol lower and plans for similar schemes in other countries.

Scrip spoke with R&D head David Reese about improving R&D timelines and success rates, including through the use of genomics and proteomics, as data readouts near for key drugs in big indications.

CEO Vas Narasimhan is confident the Swiss major's gene therapy for spinal muscular atrophy will outperform present and future rivals – Biogen's Spinraza and Roche's risdiplam, respectively – as "we have a better profile simply because we are a one-time therapy."

Two late-stage trials of Austedo have missed their endpoints in the neurological disorder, meaning Teva will miss out on an additional revenue stream from the VMAT2 inhibitor.

Biohaven is still studying its glutamate modulator in three other indications, but its biggest near-term milestone will be approval of the oral CGRP inhibitor rimegepant for acute migraine.

As Biogen moves to file its controversial amyloid drug aducanumab for approval in the US, two similar investigational Alzheimer's drugs – Eli Lilly's solanezumab and Roche's gantenerumab – did not slow memory loss or cognitive decline in a rare, inherited form of the disease.

Despite positive Phase III results in Lennox-Gastaut syndrome, the effect of the company’s low-dose fenfluramine on serious seizures may not be as good as seen with GW Pharma’s Epidiolex. 

The Danish drugmaker's four 'strategic brands' – Brintellix, Abilify Maintena, Northera and Rexulti – are selling well. Now the focus is firmly on breaking into the anti-CGRP market with eptinezumab.

The French drugmaker ended 2019 with a solid Q4 performance and news that its BTK inhibitor SAR442168 will start Phase III testing in progressive and elapsing multiple sclerosis. 

Germany’s family-controlled big pharma, Merck KGaA, is divesting its allergens business, Allergopharma to the fast-growing Munich healthcare company, Dermapharm Holding.

Aimmune has secured approval for the first ever peanut allergy treatment, but safety, cost and administration issues could limit the drug's uptake.

CEO Vas Narasimhan is confident the Swiss major's gene therapy for spinal muscular atrophy will outperform present and future rivals – Biogen's Spinraza and Roche's risdiplam, respectively – as "we have a better profile simply because we are a one-time therapy."

The European Commission has backed J&J's blockbuster to treat children with psoriasis but the drug has fallen foul of NICE for ulcerative colitis.

A strong fourth quarter and full-year 2019 performance by J&J was driven by significant sales growth for Tremfya and Stelara. CFO Wolk said a $4bn opioids settlement remains on track.

Access to branded drugs has remained steady, but there has been less management across brands by commercial payers, according to an analysis of the I&I market access environment by Bernstein.

Switzerland’s Polyphor has redefined its business strategy and is aiming to become a leaner organization, with a potential first-in-class immuno-oncologic, balixafortide, in late-stage clinical development for breast cancer.

Several newly repurposed drugs, including a Bayer antimalarial, have shown potential benefits against the new coronavirus, promoting China to add several to its latest revised treatment guidelines.

After two Indian vaccine makers, Serum Institute of India and Cadila Healthcare joined the fight against COVID-19, Bharat Biotech may follow suit. Others like Hilleman have decided against it for the moment while Panacea Biotech is weighing the pros and cons of developing a SARS-CoV-2 vaccine after a sour experience with H1N1.

While the global health threat from the now officially named SARS-CoV-2 coronavirus has grown since the start of the quarterly earnings season in early February, even many of the companies reporting more recently do not appear overly concerned about potential business impact at this stage.

Genfit will delay unblinding of data from its nearly complete Phase III study of elafibranor to get insight from the FDA on statistical methods in order to optimize its NDA.

Emerging Company Profile: Axcella uses Generally Recognized As Safe (GRAS) amino acids to create drug candidates for hepatic encephalopathy, NASH and other diseases.

Vydaqel saw its uptake take off in the US last year. UK’s NICE to pass judgement on cost effectiveness by June.

The future for glucokinase activation is looking brighter after fresh data on vTv’s oral adjunct to insulin therapy in type 1 diabetes.

With its adrenal insufficiency therapy Alkindi getting established in Europe, the UK biotech is in partnering discussions in the US where approval is expected before the end of the year.

Japan bioventure BioComo was set up around 10 years ago and has since been honing its technology platform. It is now preparing to move into the clinic with a novel vector for immuno-oncology and to raise new funds to support pipeline development.

Deal Snapshot: GSK obtains rights to two of Immatics’ TCR targets, following on the German firm’s similar deals in recent years with Celgene, Genmab and Amgen.

Nanobiotix’s CEO outlines to Scrip how the biotech plans to develop and expand its lead asset into multiple cancer indications, and create a blockbuster.

Switzerland’s SIX exchange-listed drug development company, Basilea, is moving two new oncology candidates along its R&D pipeline, supported by strong revenues from two partnered anti-infective products.

CAR-T therapies are groundbreaking in clinical terms, but have hit barriers in uptake. Can BMS’s challenger go one better than Yescarta and Kymriah?

By combining its expertise in respiratory and vaccines, the UK drugs giant could have a potential blockbuster for chronic obstructive pulmonary disease, which is expected to become the third leading cause of death worldwide by the end of the decade.

Insmed shares got a 40% boost as the company announced plans to move into Phase III this year with its potential first-in-class candidate for the lung disease.

Only nine weeks after US approval, Trikafta, which in theory can treat 90% of all patients with cystic fibrosis, is already Vertex's top-selling medicine.

Any lingering hopes Novartis had that fevipiprant could become a blockbuster have disappeared and the Swiss major has ended development of the asthma drug after two more trials failed, this time for severe disease.

The Italian firm has laid out its ambitious plan to introduce the first carbon minimal pressurized metered dose inhaler for asthma and COPD by 2025 and secured supply of a new environmentally friendly propellant.

Verona CEO tells Scrip Phase III COPD studies for its lead asset will start next year, take up to two years to complete, leading to commercial launch “probably in 2024.”