Monday 15th April
Roche receives EMA approval for Hemlibra label
The EMA (European Medical Agency) has approved Hemlibra for the use in the majority of patients with haemophilia A, extending its use beyond the minority who has developed resistance to factor Vlll drugs. Roche says that the now ‘extended’ label will be suitable for all age groups and can be used at multiple dosing options eg once weekly, every two weeks etc. This patient friendly dosing schedule is proving popular with patients, who are used to having to inject factor Vlll replacement drugs multiple times per week.
Hemlibra is used to reduce the frequency of bleeding episodes in children and adults with haemophilia A. Hemlibra is a bispecific antibody that mimics the role factor Vlll plays in the blood.
Tuesday 16th April
Sanofi replaced by Regeneron as R&D partner for Alnylam
Regeneron has agreed to pay $800m to gene silencing specialist Alnylam, to buy into its pipeline of drugs for CNS, eye diseases and liver diseases. This deal was only recently announced after Sanofi and Alnylam dissolved a rare disease R&D partnership set up in 2014.
The $800m, will be split into a $400m, upfront payment as well as a $400m on a 5% equity stake in the biotech. There is also the potential added payment of $200m in milestones if the drugs hit certain early clinical development targets.
The specific of the deals does not cover any specific programmes for CNS or eye diseases, as the intention of this collaboration is to combine their respective strengths. Regeneron will take the lead in the eye disease category, as it already has a strong blockbuster drug, with Alnylam already signed up for royalties and milestone payments.
The liver disease alliance will combine both companies existing pipelines to a multi-drug regimen for diseases including non-alcoholic steatohepatitis (NASH), a promising target for the biopharma industry.
This deal is interesting because it is a deal between two biotech companies rather than the common biotech/big pharma model. This further establishes the emergence of RNAI as a new drug class after decades of development.
Wednesday 17th April,
Novartis releases data to further support case for gene therapy Zolgensma in SMA
Novartis released data from its phase 3 trial of the newly acquired gene therapy Zolgensma, with the data reinforcing the drugs already impressive efficacy profile. This data comes ahead of a decision to made by the FDA to evaluate the drug for the treatment of the most severe form of spinal muscular atrophy (SMA).
Zolgensma has been under review since December, after it was given a priority status, and fast tracked by the FDA.
The most promising results from the phase showed that the drug helped those with SMA type 1 sit unassisted for at least 30 seconds. This milestone was achieved for three patients back in phase 1 in September but since an extra five patients have displayed similar results.
With a high chance of approval by the FDA, there has been a focus on the pricing of the treatment. Novartis has already suggested that the one-time treatment be priced at $4m per patient but US medicine pricing watchdog ICER, has urged Novartis for a fair pricing agreement. This comes after current SMA treatment by Biogen, being classed as overpriced, with Spinraza being on the market since 2016 coming in at a price of $750,000 for the first initial year and $375,000 per year thereafter. ICER stated that the price should be slashed by at least 82.76% for the first year and then should cost between $36,000- $65,000 per year thereafter.
ICER has called for Zolgensma to be priced somewhere between $900,000 and $1.5m. Novartis will not release any pricing agreements until the product has been approved.
Thursday 18th April,
Roche announces ‘discovery proteomics’ could be heading for clinical trials
Proteomics is a technology that can take a snapshot of protein expression in tissue samples, this technology is already used in drug discovery and could soon be applied to clinical trials states Roche.
The quantification of proteins in biological samples could be used to identify novel biomarkers and to generate additional information to help explain why clinical intervention is or is not working in a particular patient.
Roche has been working with proteomics specialist Biognosys on a mock trial to explore how it could be used clinical trials. A study has been performed by the two companies looking at cancer samples taken from clinical trials. The method proved to be quick as it uses data analysis to compare the signatures against a database of references. Almost 9,000 proteins were identified in the study with researchers saying that the results had significant statistical power to ‘detect subtle changes in protein expression that may occur in tumor tissue’
Friday 19th April
Novo Nordisk and Gilead Sciences to collaborate to target NASH.
They are too collaborate on new combinations targeting NASH ( Non-alcoholic Steatohepatitis) using Novo’s diabetes drug semaglutide. The drug will be combined with Gilead’s cilofexor and firsocosat in a proof of study concept, as they are both investigational molecules with no approvals of yet.
This is a smart move by both companies in a crowded field of late stage pipeline contenders. Gilead announced its clinical trial results investigating cilofexor with firsocosat in those with NASH, with promising results of out of the 20 patients treated , 74% of them experienced a significant decline of at least 30% in hepatic fat, a biomarker of the disease.
NASH is a chronic liver disease characterized by fat accumulation and inflammation in the liver , which can lead to scarring and fibrosis that impairs liver function.
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