Techspec schreef op 6 december 2018 12:06:
www.pharming.com/science/#expanding-b...Pipeline
(accurate November 2018)
Expanding the pipeline beyond Ruconest®: New forms of Ruconest® administration and additional products
With validation secured from the approval of the first product from our transgenic platform, we will now seek to initiate new projects on this platform.
Our transgenic platform remains the only technology that to date can deliver recombinant versions of certain complex human proteins in an economically viable way: this is a result of the low cost of capital investment required to start up a suitable founder herd, the fact that the herd is easily scalable and the high yield of product obtained.
Our strategy:
New forms of administration for RUCONEST® for HAE
Expansion of rhC1INH clinical development in pre-eclampsia, contrast induced nephropathy and other potential large indications
Clinical development of protein replacement products in Pompe and Fabry diseases
rhC1INH (Ruconest)
Recombinant human C1 inhibitor (rhC1INH) is a recombinant human C1 inhibitor protein. Natural C1 inhibitor DNA from a human source is used in Pharming’s protein production technology to ensure expression of the C1 inhibitor protein. Human C1 inhibitor is a human plasma protein involved in the regulation of the complement and contact parts of the immune system. It is a controlling protein in early stages of these cascades and in that way a regulating factor of the immune system.
By inhibiting these systems, the abnormal inflammation can be controlled. This is also the mechanism by which an HAE attack can be treated with C1 inhibitor.
New formulation development of RUCONEST®
The “RUCONEST® liquid” formulation (from 14ml to 3ml) can be used as starting material for the generation of subcutaneous, intra-muscular & intradermal application systems
New proprietary ‘painless’ intradermal delivery applications are being developed. A dissolving point device and a reservoir device.
These painless versions should differentiate RUCONEST® from competitors, all of whom have painful injections
New activities with rhC1INH (RUCONEST®)
Initial Therapeutic Indications selected:
New Potential Indications using existing formulation
Organ damage after contrast media application: Contrast-induced Nephropathy (CIN) recent positive results investigator-sponsored study
Tissue Damage after Toxic Event: Pre-Eclampsia; study to start Q4 2018
Tissue Damage after Hypoxic Event: Delayed Graft Function (new Investigator initiated study)
Shock response after trauma: Hypovolemic Shock – ongoing preclinical research projects with the US Army and US Air Force
Brand New IP:
New Pharming patents filed in 2018 covering the new indications
Patents cover all forms of rhC1INH
Positive Phase II results in Contrast-induced Nephropathy (CIN)
Positive results from a Phase II investigator-initiated study of RUCONEST®
In a DBPC study with 75 patients, RUCONEST® showed a statistically significant effect (p=0.038) in reducing Neutrophil Gelatinase-Associated Lipocalin (NGAL).
Clear results in sub-group of patients (n=30) undergoing PCI.
Median increase in peak urinary NGAL concentration within 48 hours of 1.8 ng/ml compared with an increase of 26.2 ng/ml in the placebo arm (p=0.04).
Median percentage change in the peak urinary NGAL level within 48 hours of 11.3% vs 205.2% in the placebo arm (p=0.001).
Overall patients undergoing invasive procedures requiring high volumes of contrast media experienced a stronger benefit from the RUCONEST® treatment.
Next steps:
Design of additional clinical development to establish efficacy and efficiency of RUCONEST® treatment in patient groups likely to experience the greatest benefit.
…read more in our press release here.
Market size estimation presented at Captial Markets Briefing 21st June 2018
Open de link bovenaan deze post voor de tabellen.
Collaborative Product development: Strategic Collaborations with SIPI
In addition recently we have entered a strategic collaboration for the development, manufacture and commercialisation of new products based on the Pharming technology platform with The Shanghai Institute of Pharmaceutical Industry (SIPI), a Sinopharm Company based in China.
Under the terms of the agreement, Pharming will transfer the Pharming technology platform and manufacturing know- how to SIPI, such that joint global development for new products will take place at SIPI’s facilities in Shanghai and benefit from both the cost advantages of the Pharming platform and the competitive development and manufacturing costs structures at SIPI.
The first project to be jointly developed and manufactured at SIPI will be recombinant C1-inhibitor (conestat alfa).