2024-13-011 + 2019-06-006 - Hepta4IBD - Heptammune: a first-in-class biologic immunomodulator against autoimmune inflammatory bowel disease

Hepta4IBD

2024-13-011 + 2019-06-006

Injectable human-derived recombinant protein very stable in the circulation and with sustained action to treat autoimmune diseases. Our asset is postulated as an immunomodulator and not as an immunosuppressant

Environmentally speaking the production of Heptammune could be done in E.Coli, representing a well stablished production method that can save production derived goods, as medium for the cell growth and plastic for the incubation. From a Social perspective is takling an unmet medical need that would have outcome in the wellbeing of the society and will reinforce the spanish production ecosystem. From the Governancace point of view, the project is being developed under the Umbrella of the Fundació IDIBELL.

The market arrival of PurPose Biotherapeutics would result in an increase on the wellbeing of IBD non-responders patients. Additionally, it's immunomodulatory effect will restore the immunologic balance, being less aggressive and toxic, derisking also the possible side effects. This effect would result also in the decrease of patient care.

The problem: Inflammatory bowel disease (IBD) refers to 2 chronic inflammatory disorders, Crohn's disease (CD) and Ulcerative Colitis (UC). Both are life long, relapsing disorders of unknown etiology. Current therapies have low safety profiles, low patient adherence to treatment due to toxicity, and can cause immunosuppression, leading to severe treatment-emergent adverse events (TEAE), which results in discontinuation of treatment in a significant number of patients. The solution: The proposed invention is PRP6-HO7, an injectable human-derived recombinant protein very stable in the circulation and with sustained action to treat inflammatory bowel disease. This asset is postulated as an immunomodulator and not as an immunosuppressant, a key differential aspect that would justify its development as an innovative drug with a "first-in-class" character and a unique mechanism of action with a specific receptor (to be discussed under NDA).

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market increase to 2031 is estimated as 10.742 M dolars

Our asset is primarily directed to the global IBD market, expected to generate revenue of €19.30 billion by end of 2025, growing at a CAGR of 4.3% (2019-2025). With UC as the 1st indication for Heptammune, Global market UC in 2021was estimated in €5.8 billion, and €10.4 billion expected for 2031 (5.9% CAGR). And biological therapies hold about 60% of the IBD market share.

Moderate and severe non-responders suffering from IBD

PurPose is planning to secure 2 rounds of funding within the next 4 years. These funds will be instrumental in supporting Heptammune production under cGMP conditions, conducting GLP toxicology/safety studies, and contracting a CRO to prepare and submit the CTA for the Phase I clinical trial. The initial funding round, totaling €5 M (€4 M from VCs + €1 M from grants and loans), will be succeeded by a subsequent round of €20 M dedicated to planning and executing Phase I and II clinical trials.

Currently we have financial needs to cover the last stages of pre-clinical experimental data and we are looking for initial investments (3M €) for the creation of the spin off and cover the path to first in human clinical trials.

Pre-clinical data, toxicology and CMC for spining-out a company

Resets the immune system and resolves inflammation Unique MoA targeting the innate immune system Preclinical PoC achieved The technology will be a first-in-class biologics for non-responder patients in IBD

Mid competence of different alternatives. Scout for the partners of interest and need of feedback from KOL to define the strategy

Roadmap defined in two phase: Pre-clinical POC BEFORE spining-out and a second phase to progress to achieve the First in human clinical trial (spin off)

1- EP11382240 (15/07/11). "Compositions and methods for immunomodulation". 2- EP17382187 (06/04/17). "C4BP-based compounds for treating immunological diseases". 3- EP19382910 (17/10/19). “Compounds for immunomodulation". 4- EP24382150 (15/02/24) "Compounds for treating diseases"

Partnership with regulatory expert Nadina Grosios from EATRIS

Inflammatory bowel disease (IBD) is a chronic immune-inflammatory condition of the gastrointestinal tract with no cure. Worldwide, 6.8 M people suffer from IBD, and its prevalence is expected to rise by ~30% in the next decade. It is managed with expensive inflammatory medications with a high 1-year failure rate. The impact of this is cost to healthcare providers on medication that is not working but, most importantly, bad outcomes for patients who can spend years on the wrong drug, developing irreversible bowel damage necessitating surgery only to relapse after a few years. Standard of care medications for IBD are currently not meeting expectations. Thus, despite more drugs than ever, IBD management is difficult, and outcomes are creeping forward at unsatisfactory rate. Against this scenario and based on our groundbreaking science, we found that a blood protein, the complement regulator C4BP(β-), shows immunomodulatory activity, and have demonstrated its therapeutic efficacy in several murine models of autoimmune diseases. Recently, we have designed a new biologic, Heptammune (PRP6-HO7), a C4BP(β-) analogue holding only immunomodulatory activity, to improve its efficacy, safety, and scalability. Heptammune binds its receptor, “reprogramming” inflammatory myeloid cells toward an anti-inflammatory and tolerogenic phenotype restoring immune homeostasis. Thus, Heptammune acts holistically “resetting” the innate immune system and inducing immune resilience from inflammatory stress.We have demonstrated the superior therapeutic efficacy of Heptammune in murine models of colitis and in myeloid cells from IBD patients, regardless of their medication. Thus, our asset targets the innate immune system, a key differential aspect that justifies its development as an innovative drug with a "first-in-class" character because IBD is mostly driven by innate immune components, which current therapies are not addressing.