MULTIPLE SCLEROSIS: Recovery of UK's strong lead in protecting the MS Brain by securing UK-Based formulation of LIFNano™

PROTECTING THE BRAIN IN PEOPLE WITH MULTIPLE SCLEROSIS (MS)

* **MS** is a demyelinating autoimmune disease that attacks the brain, with year-on-year loss of brain volume, starting late teens and becoming manifest late twenties. There is no cure. Current therapies are immunosuppressive only.
* The COVID-19 pandemic has spotlighted the vulnerable young including the UK's MS-ers (people with MS) who need continued shielding as lockdown eases.
* The microSME **LIFNanoRx (LNT)** is a spin out from the University of Cambridge, founded by scientist Su Metcalfe to treat MS. Recognising the need to protect the brain, she captured the profound neuro-protective properties of LIF - a stem cell growth factor essential for brain health - using nanoparticles of FDA-approved gel to provide a homing device for sustained delivery of LIF cargo to the brain. Collaborator Tarek Fahmy at Yale University contributed remarkable ability to manufacture the LIF-containing nanoparticles and is co-inventor of the technology patents owned by Metcalfe.
* The preclinical data is compelling, confirming efficacy and safety in models of MS, and partially reversing paralysis within 4 days of treatment. Notably the therapeutic nanoparticles "LIFNano(tm)" cross the Blood-Brain Barrier (BBB) - a highly sought after property for treatment of diseases of the central nervous system (CNS).
* By protecting the brain, LIFNano(tm) provides a _low cost, high value, safe solution_ for people suffering from MS. Supported by previous government grants, LIFNanoRx is now a major force - a David amongst Goliaths: unlike the Goliaths - where the MS market is lucrative with high costs to payers and high rewards to Suppliers - LNT brings a uniquely disruptive, low costs high value to patients approach. There are many followers of LNT within the MS community who understand LIF is not only a _drug-free_ natural solution to protect and heal the brain, but also may quench the autoimmune root-cause of MS.

COVID-19

* With a Phase IB clinical trial planned for 2020/2021, to be led by UK's leading MS neurologist Gavin Giovannoni, LNT's progress to clinic was stalled by COVID-19\. Lockdown has stopped our access to the LIFNano(tm) product that is custom made by Yale University.
* This bid to the Sustainable Innovation Fund of I-UK will transfer manufacture of LIFNano(tm) to the UK, to be undertaken by Pharmidex, another UK SME with robust growth providing services to evaluate medicines that treat the brain.
* The Project's budget also accommodates a Clinical Trial Panel - already in place from our previous I-UK BMC award (2017-2019) - bringing added value to this SIF Project within budget and on time. The Panel's experts will oversee IMPD preparation ready for engaging with the MHRA regulatory authorities.

THE SIMPLE CLEAN TECHNOLOGY

* As an entirely synthetic product, LIFNano(tm) vastly reduces impact on the environment compared to cell-based therapies. By exploiting LIF, LNT brings a new generation of treatments for currently untreatable diseases of the CNS, including rare diseases where 30% involve the CNS.

LNT is an innovation leader, with impacts on UK's ability to drive growth. For MS, the immediate need is CTA that will maintain the high trajectory of LNT towards first in human trials.This Project not only brings our simple clean technology to the UK, to overcome the adverse impact of COVID-19 on LNT's progress to treat patients who suffer from MS. It also adds commercial strength to the UK economy as a leader in NanoMedicines.