COMMERCIALISATION OF A NOVEL PROTEIN VARIANT WITH NEURORESTORATIVE EFFECTS FOR AMYOTROPHIC LATERAL SCLEROSIS

Introduction

REGENERA will assess the technical and commercial feasibility of a novel and optimised C-MANF peptide as a superior treatment for amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease affecting 450,000 people worldwide where motoneurons (MNs) selectively degenerate in the brain and spinal cord.

Disease Characteristics

ALS is characterised by muscle deterioration that rapidly leads to disability and culminates in death 3-5 years after diagnosis. Unfortunately, there is no cure for ALS and current treatments only marginally slow down its progression.

Limitations of Current Treatments

Moreover, promising neurotrophic factors (NTFs) with neuroprotective activity show insufficient efficiency, are unable to reach the brain tissue, and have highly invasive administration routes (i.e. brain injections and intrathecal) and high production costs. As a result, all NTFs clinical trials have failed.

Discovery of C-MANF

Ass. Prof. Voutilainen has discovered C-MANF, a novel peptide which, in contrast to classical NTFs, has several advantages:

1. Protective and restorative effects on motoneurons
2. Ability to penetrate the blood-brain barrier (BBB)
3. Can be subcutaneously administered
4. Can be inexpensively produced

Project Objectives

Within REGENERA, we will assess whether C-MANF is feasible as an early-therapy option for ALS.

Research Methodology

Firstly, we will analyse the pharmacokinetic properties and efficacy of C-MANF in ALS animal models and in human MNs.

Commercial Feasibility

Subsequently, commercial feasibility will be determined by:

1. Verifying IP position and strategy
2. Performing in-depth market and competitor analyses
3. Consolidating these into a business case to establish the best path to commercialisation

Potential Impact

Successful commercialisation of C-MANF could:

• Reduce the profound socioeconomic burden of ALS
• Provide an early-therapy option to delay disease progression and thus extend and improve the patients’ lives
• Provide the pharmaceutical industry with a novel therapeutic that can potentially be used for other neurodegenerative diseases.