1 (19)

Project for better health 2017–2018

Project description

Instructions

Read the call for applications text containing information on how to apply, the assessment criteria, the aim of the call for applications and the type of projects for which it is intended.

Instructions for this form: the general part of the project description (as far as appendix 1–3) mustnot exceed ten pages (minimum Times New Roman 11pt) in Swedish or English.

Appendix 1 (budget) and 2 (Gantt chart) must be completed by all projects.

Appendix 3 is compulsory for all pharmaceutical projects and is not to exceed six pages (minimum Times New Roman 10pt for all information in tables).

For Mac-users: To tick the boxes in the table, highlight the box and then press the space bar.

Please do not change the formatting of the document. Descriptive text can be cut, but not the questions, as this makes the document hard to assess.

1.ELIGIBILITY AND CLASSIFICATION

Technical Readiness Level (TRL) at start of project TRL definitions are available in Appendix 4. At least TRL3 and at most TRL6 is a requirement to be eligible to apply. For TRL3 all TRL1–3 criteria must be achieved by the start of the project.
3☐ 4☐ 5☐ 6☐
Expected TRL level at the end of the project
3☐ 4☐ 5☐ 6☐ 7☐ 8☐ 9☐
Focus areaTick the area that best describes your project
☐Pharmaceutical project (small molecule drug) (NB if yes, completion of Appendix 2 is compulsory)
☐Pharmaceutical project (biological drug)(NB if yes, completion of Appendix 2 is compulsory) / ☐ MedTech
☐ Diagnostics
☐ E-health/M-health
☐Other………………………………
Hands-on supportThe project consents to being contacted by Swelife for offers of hands-on support / We consent to be contacted by Swelife
Yes ☐ No ☐

Please answer the following questions. Write your replies in the box below each question. PURP

2.AIM OF THE PROJECT

  • Briefly describe your solution/final product
  • List key deliveries to attract further financing to continue development or to enter into collaboration with another partner at the end of the project.

3.CLIENT NEED AND BENEFIT

  • Describe the unmet need you are addressing and the potential effect of your solution on a) the individual patient b) healthcare and c) society.
  • Disease area: Describe the indication or patient group you are focusing on and how extensive it is.
  • Define the target group (“Treatment Patient Population”) for the proposed product or procedure.
  • Describe how the client benefit has been verified against the stakeholders such as a) patients, b) healthcare providers (or other client groups).
  • Describe how the need group (healthcare, patient, client, user …) is connected to the project.

4.BACKGROUND AND STATUS

  • Describe the current status of your project proposal including the research and/or rationale behind the idea. Describe activities and results which have led to the existing TRL, and any other relevant information. See Appendix4 for definition of TRL.

5.MARKET AND COMPETITION

  • Describe the market (including its estimated size) for your product.
  • Describe your marketing strategy including 1st market and subsequent scalability on an international market.
  • Describe competing technologies/projectsand competing methods/productson the market.
  • What are your unique competitive advantages?
  • What is the solution’s potential for enhancing savings, efficiency or quality from a patient perspective?

6.INTELLECTUAL PROPERTY

  • Describe the current IPstatus.
  • Has any professional freedom to operate (FTO)analysisand/or novelty search been carried out?
  • Describe your IPstrategy
  • If relevant, justify your decision not to protect your idea.

7.BUSINESS DEVELOPMENT

  • Describe how the project can increase growth, expertise and/or international investments in Sweden.
  • Describe future plans; will you start a company, take the project to the market yourselves, or apply another business strategy?
  • If relevant, describe any potential partner/s – and indicate whether a potential licensee is already linked to the project

8.REGULATORY PLAN

  • Summarise your regulatory plan (content and timeline) and indicate to what degree the project has been discussed with regulatory authorities.
  • If relevant, describe which ethical permits will be required for continued development and how far you have got in obtaining them.

9.GENDER EQUALITY

  • Describe how the project has taken gender equality into account e.g. with regard to the composition of the project team or the design/implementation of activities and choice of target groups.
  • What is the percentage of women and men among the project’s staff?
  • How is work allocated between women and men in the implementation of the work packages (WP)?

10.CO-FINANSING

  • Describe how the project will achieve the co-finansing

11.PROJECTORGANISATION, MANAGEMENT AND PROJECT PARTNERS

Summarise your project team in the table below

Give a brief description of the project team’s organisation, including the participating industry, clinical partners and/or academic groups (name, affiliation, expertise, project leader and other allocated project responsibilities).

Projectpartner 1:
Name:
Justify participation in the project:
Participation in WP:
Name:
Role in the project:
Expertise:
Name:
Role in the project:
Expertise:
Projectpartner 2
Name:
Justify participation in the project:
Participation in WP:
Name:
Role in the project:
Expertise:
Name:
Role in the project:
Expertise:
Projectpartner 3
Name:
Justify participation in the project:
Participation in WP:
Name:
Role in the project:
Expertise:
Name:
Role in the project:
Expertise:
  • Describe any other key expertise (consultants, etc.) linked to the project who are not project partners.
  • Is there any key expertise that would strengthen the team? What is your plan to link such expertise to the project?
  • Give a brief description of how the project is led and how the collaboration between the parties is coordinated.

12.PROJECTPLAN

The project is to describe at least two work packages (WPs) of which one is to address business development and business intelligence. Each WP is to be specified according to the template below.Add more WPs as necessary.

WP1 title: Business development
Purpose, timeline and content WP1
Activities
Quantitative and qualitative deliveries
Decision point (STOP/GO)
WP2 title:
Purpose, timeline and content WP2
Activities
Quantitative and qualitative deliveries
Decision point (STOP/GO)

13.RISKANALYSIS

Describe the main risks in the project. Reflect on the areas below and account for how you intend to manage them:

Business/market; Technical;Legal/regulatory; Project implementation/financing

Likelihood and consequences are ranked on a scale of 1 to 5, where 1=none, 2=low, 3=medium, 4=high, 5=very high

Risk / Likelihood (1–5) / Consequence (1–5) / How will you manage the risk?

I.Appendix 1 Budget

Complete the budget for each work package and for each project party (if there are several). The total budget for the project is also to be completed.

There are three types of costs:

Salaries, including social security contributions (also note the number of hours)

Other costs

Indirect costs

Other costs are to be specified according to:

1.Equipment/material

2.IPR/consultancy costs

3.Other direct costs (travel, services, etc.)

Copy the table below for each work package. Add or remove project parties as appropriate.

Please note that the budget must also be completed on Vinnova’s portal. Although it is presented in a somewhat different form, you can use the information entered in the table here. It is important that the figures provided in the portal match those given here in the project description.

WP1: / Business development
Period:
(start-end) / 2018 SEK (hours) / 2019 SEK (hours) / Total (SEK) / Vinnova funding (SEK)
Project partner 1
(enter name)
Specify costs for partner 1 / Salaries, including socialsecurity contributions
Equipment, land buildings
Consultancy costs, licensesetc.
Other direct costs including travel
Indirect costs
Project partner 2
(enter name)
Specify costs for partner 2 / Salaries, including social security contributions
Equipment, land, buildings
Consultancy costs, licenses etc.
Other direct costs including travel
Indirect costs
Project partner 3
(enter name)
Specify costs for partner 3 / Salaries, including social security contributions
Equipment, land, buildings
Consultancy costs, licenses etc.
Other direct costs including travel
Indirect costs
Total (SEK)
  • To provide a full picture, indicate whether the project covers further costs beyond the budget above (e.g. non-accountable, non-fundable costs)

II.Appendix 2 Gantt chart

Insert the project’s Gantt chart here

Visualise the various activities and business development, as well as the decision points, in a Gantt chart, to clarify the project plan. Also include relevant activities that lie outside the framework of the application, but clarify which activities are included.

III.Appendix 3for pharmaceutical projects

If you propose a pharmaceutical project, it is compulsory to fill in this section.

Some questions are only applicable to small molecule projects and some are only applicable to large molecule, biological projects, as indicated in the sections below. You only need to fill in the sections that are applicable to your type of project and you may cut out the other sections before submitting your application.

If you propose another type of pharmaceutical project (advanced therapies, proteins, gene therapies for example) you may fill in those parts of appendix 3 that are applicable (use the parts for biological projects) in order to facilitate the assessment of your project.

Use this as a guide for all important blocks in the drug discovery process. Some questions are more relevant for later stage projects; however, we are looking for projects in all phases from LO (lead optimisation) to clinical phase II. Your project will be evaluated based on the stage your project is in (see section I). NB Make sure that you provide enough information so that your project can be evaluated, but do not disclose strictly confidential information in this section, in particular structures, sequences, target identities etc. if not already known in the community. If you are selected for interview, you will be able to present more details about your project.

If needed, put extra information in the comments boxes. It is acceptable if you don’t have answers to all questions.

Appendix 2 should be maximum 6 pages (Georgia 10pt for all information in tables). Remove grey text and non-applicable parts if needed.

Typ av projekt

☐Small molecule project
☐Large molecule (biological) project / ☐ Other……………………..

Stage of project

Please indicate which stage in drug discovery /development is applicable to your project
☐ LO (Lead optimisation)
☐ Prenomination / CD (clinical drug candidate) selection / ☐ Clinical Phase 1
☐ Clinical Phase 2
☐ Other
Briefly describe:

Target Biology

Knowledge about the target
☐Unknown target
☐ Novel target
☐ Known drug target / family / ☐ Novel target (no known function)
☐ Other
What evidence do you have that the target is relevant to human disease
☐ Hypothetical
☐ Genetic or proteomic data from human
☐ Expression studies in human
☐ Registry clinical studies / ☐In vitro studies
☐In vivo animal models
☐ Exploratory clinical studies
☐ Other
What is the actual/proposed mechanism of action (MoA) on the target
☐ Agonist
☐ Antagonist
☐ Modulator
☐ Allosteric / ☐ Reversible
☐ Irreversible
☐ Other, describe
Comment on Target Biology:

Preclinical Package – NB for small molecules only

Do you have biological assays in place for lead optimisation? / ☐ Yes ☐ No
☐ Not applicable
☐ No assays
☐ Primary assay
☐ Cellular assay
☐ Selectivity assays / ☐In vivo model(s)
☐ Other:
☐ Other:
What kind of in vitro pharmacological data has the project generated?
Describe briefly
Does the project have a chemical series with in vitro DMPK data suitable for the target? / ☐ Yes ☐ No ☐ Not assessed
What parameters have been determined?
☐Microsomal stability Clint
☐Caco-2 permeability / ☐ MDR1-MDCK permeability
☐Log D7.4
☐ Other:
Has the project generated in vitro metabolism data (rodent / non-rodent / human)? / ☐ Yes ☐ No ☐ Not assessed
Is there consistency in the in vitro metabolism rates across non-clinical species and man / ☐ Yes ☐ No ☐ Not assessed
Comments on in vitro metabolism if needed:
Has the project generated in vivo exposure or PK data on an active compound in a rodent species? / ☐ Yes ☐ No ☐ Not assessed
What PK parameters have you measured?
Animal model
☐ Plasma Clearance
☐ %bioavailability (F)
☐ i.v. plasma half life
☐ Plasma protein binding
☐ Other: / Human
☐ Plasma Clearance
☐ %bioavailability (F)
☐ i.v. plasma half life
☐ Plasma protein binding
☐ Other:
Has the project access to relevant disease models or other pharmacodynamics models? / ☐ Yes ☐ No ☐ Not assessed
Has the project generated in vivo efficacy data? / ☐ Yes ☐ No ☐ Not assessed
Has in vivo target engagement been assessed? / ☐ Yes ☐ No ☐ Not assessed
If yes, briefly describe how:
Is there information known about the series with respect to hERG and/or CYP450 inhibition/induction? / ☐ Yes ☐ No ☐ Not assessed
Comment on preclinical package:

Preclinical Package – NB for biologicals only

Do you have biological assays in place for lead optimisation? / ☐ Yes ☐ No
☐ Not applicable
☐ No assays
☐ Biochemical assay
☐ Cellular assay / ☐In vivo model(s)
☐ Other:
☐ Other:
What kind of in vitro pharmacological data has the project generated?
Describe briefly
Has the project generated in vivo exposure or PK data on an active compound in a rodent species? / ☐ Yes ☐ No ☐ Not assessed
What PK parameters have you measured?
Animal model
☐ Clearance
☐ %bioavailability (F)
☐ Serum half-life
☐ Volume of distribution
☐ Other: / Human
☐ Clearance
☐ %bioavailability (F)
☐ Serum half-life
☐ Volume of distribution
☐ Other:
Has the project access to relevant disease models or other pharmacodynamics models? / ☐ Yes ☐ No ☐ Not assessed
Has the project generated in vivo efficacy data? / ☐ Yes ☐ No ☐ Not assessed
Has in vivo target engagement been assessed? / ☐ Yes ☐ No ☐ Not assessed
If yes, briefly describe how:

Chemical Package – NB for small molecules only

What is the origin of your compound / chemical series / candidate drug?
☐Own HTS / screening / chemistry
☐ In-licensing
☐ Repurposing
☐ Natural Ligand
☐ Research tool / ☐Virtual screening hit from docking or pharmacophore model
☐A drug-like compound reported in the patent literature
☐A drug-like compound reported in peer-reviewed literature
☐ Other:
Comment if needed:
Does the project have a compound/chemical series that has activity <1µM (ideally <100nM) in a biochemical assay / ☐ Yes ☐ No ☐ Not assessed
Does the project have a compound/ chemical series that has activity <10μM (ideally <1μM) in a cellular assay? / ☐ Yes ☐ No ☐ Not assessed
What evidence do you have that your compound(s) binds to the desired target?
☐ X-ray crystallography
☐ NMR
☐ ITC / ☐ SPR
☐ Covalent linkage
☐ Other
Have you taken steps to rule out possible artefactual sources of assay activity, i.e. promiscuity, aggregation, fluorescence, protein reactivity, redox or other assay specific processes / ☐ Yes ☐ No ☐ Not assessed
Does the series have a synthetic/isolation method allowing preparation of 1 g sample of an individual compound in >95% purity ? / ☐ Yes ☐ No ☐ Not assessed
Does the series have measured aqueous solubility OR known solubility in an acceptable dosing vehicle consistent with the intended clinical route of administration? / ☐ Yes ☐ No ☐ Not assessed
Comment on Chemical package:

Protein characteristics – NB for biologicals only

What is the origin of your large molecule drug?
☐Own screening
☐ In-licensing
☐ Repurposing
☐ Research tool / ☐A drug-like protein reported in the patent literature
☐A drug-like protein reported in peer-reviewed literature
☐ Other:
Comment if needed:
What expression system is used?
What purification methods are used?
What analytical methods have been used for protein characterization?
☐ Purity by SDS-PAGE
☐ Protein ID by MS
☐ Peptide mapping
☐Bacterial endotoxin assay
☐ Other:
Does the project have a protein that has been tested for activity in a biochemical assay? / ☐ Yes ☐ No ☐ Not assessed
Does the project have a protein that has been tested for activity in a cellular assay? / ☐ Yes ☐ No ☐ Not assessed
Do you have evidence that your protein binds to the desired target (e.g. performed by the Surface plasmon resonance /SPR)? / ☐ Yes ☐ No ☐ Not assessed
Comment on protein characteristics:

Toxicological Package

Is there predictable target related side effects in animals and man modulating this target?
☐ Unknown
☐ Hypothetical
☐ Predictable based on in silico data / ☐ Predictable based on data
☐ Demonstrated
☐ Other
- NB for small molecules only
Has any assessment (computational or experimental) of structural liability for reactive metabolites, genotoxicity or phospholipidosis been carried out? / ☐ Yes ☐ No ☐ Not applicable
- NB for biologicals only
Has any assessment (computational or experimental) of immunogenicity potential been carried out? / ☐ Yes ☐ No ☐ Not applicable
- NB for biologicals only
Has any assessment (computational or experimental) of other immune mediated side effects (e.g. Fc receptor mediated or other) been carried out? / ☐ Yes ☐ No ☐ Not assessed
Do you know if there are potential safety considerations due to likely off-target activities?
☐ Unknown
☐ Hypothetical
☐ Predictable based on data / ☐ Demonstrated
☐ Other
Are thereany overt physical signs observed during in vivo studies performed to date / ☐ Yes ☐ No ☐ Not assessed
Have you performed any GLP Tox? / ☐ Yes ☐ No ☐ Planned ☐ Not applicable
Comment on Toxicological package:

Translational and Clinical Package

Is the proposed new pharmaceutical product to be a first, second or third-line therapy?
☐ 1st
☐ 2nd / ☐ 3rd
☐ Other
Briefly describe your translational science plan
Brief description:
Briefly describe your clinical plan and stratification of patients
Brief description:
Is there an identified, measurable and validated biomarker for clinical efficacy in man?
☐ Activity and PD biomarker known
☐ Effect biomarker only / ☐ Potential suggested
☐ None identified
☐ Other
Does any biomarker measure target engagement in the desired tissue? / ☐ Yes ☐ No ☐ Not assessed
- NB for small molecules only
Is there a method available for production of the active ingredient in 1 kg scale suitable for GMP production? / ☐ Yes ☐ No ☐ Not assessed
- NB for biologicals only
Is there a biopharmaceutical CMC process available for production of the active ingredient in a scale suitable for GMP production? / ☐ Yes ☐ No ☐ Not assessed
Is there stability data available for the active ingredient? / ☐ Yes ☐ No ☐ Not assessed
If so, for how long?
What is the proposed clinical route of administration?
☐ Oral
☐ Intravenous
☐ Subcutaneous
☐ Inhaled / ☐ Intramuscular
☐ Topical
☐ Other:
☐ Not decided
Is there a formulation developed for human use? / ☐ Yes ☐ No ☐ Not assessed
If yes, describe briefly:
What is your expected dose and dosing frequency in man?
Describe briefly:
Comments on the translational and clinical package:

IV.Appendix 4 – guide over TRL levels