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.
- New forms of administration for RUCONEST® for hereditary angioedema (HAE)
- Clinical development of protein replacement products in Pompe and Fabry diseases
- Expansion of recombinant human C1 esterase inhibitor (rhC1INH) clinical development in pre-eclampsia, contrast induced nephropathy (CIN) and other potential large indications
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 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.
Prophylaxis of HAE
In acute HAE, each individual attack is treated. However, with prophylactic therapy, the patient is treated on a regular basis with the aim of preventing attacks or at least reducing the frequency of attacks. In July 2016, Pharming announced positive results from a Phase II clinical study exploring the use of RUCONEST® for prophylaxis of HAE.
Following the positive Phase II study, the US food and Drug Administration (FDA) invited Pharming to submit the Phase II trial early. Subsequently, the FDA issued Pharming with a complete response letter (CRL) in September 2018. As part of Pharming’s continued commitment to HAE patients, the Company is developing new forms of RUCONEST® with new routes of administration to increase convenience, as well as addresses the CRL as part of new prophylaxis studies.
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 and intradermal application systems. And we will be investigating this for both prophylaxis, and acute treatment of HAE and our other indications. Nano-injections could become the future for the treatment of both acute and prophylaxis of HAE attacks and for other indications in our pipeline.
Nano-injections could become the future for the treatment of both acute and prophylaxis of HAE attacks and for other indications in our pipeline.