Presenting data in partnering discussions
When pursuing collaboration opportunities with healthcare companies it is essential that innovators show the right scientific data to get the other party interested, while keeping the protection of their intellectual property and their competitive position in mind. They should carefully consider what information to share and what to keep confidential.
This visual contains key topics and questions business developers might evaluate to assess scientific quality, feasibility, and the potential to address a significant unmet medical need.
Unmet medical need
A
Which unmet medical need are you addressing? How is the disease diagnosed and how will you define your target patient population? Are biomarkers available to identify the ideal patient population or to measure disease progression? How do you plan to show clinical proof of concept (POC)?
B
Market/referencing: What is the current standard of care? How will your innovation compare to the anticipated best standard of care at the time your product will launch? Why will your innovation succeed where others failed?
C
Competition: Who are your main competitors? Why do you expect to outperform them? What are your Unique Selling Points (USPs)? Is it possible to compare the efficacy of your asset with competing therapeutic solutions in relevant preclinical models?
Mechanism of action (MoA)
A
What is the mechanism of action you are pursuing? What is the modality of your therapeutic solution (small molecule, biologic, oligonucleotide, peptide, vaccine, …)? What is the intended route of administration?
1
Please note: Never reveal the structure or sequence of your asset in a non-confidential deck even if it is publicly available. Under CDA/NDA, only reveal this information if strictly necessary to progress the discussions and the partner has clearly indicated they are willing to view it.
B
What is the drug target?
1
How does the complete target validation package look?
2
What is the expression distribution and the function of the target in healthy tissues?
3
Which target-related side effects might be expected?
4
If the target is novel, why do you believe a new MoA is necessary and why is it the best target to pursue for the unmet medical need? What evidence do you have that it might be disease-driving?
5
If it is not novel, why do you believe your approach will be differentiated to that of others? What are the perceived weaknesses/liabilities you are trying to improve on?
C
Can you show potency values? And full concentration-response curves of the in vitro pharmacodynamics that have been used to calculate them?
D
Do you have evidence that your molecule directly binds to the target? Did you study the nature of target engagement (orthosteric, allosteric, competitive, non-competitive, target-residence time)?
E
Do you have protein crystal structures available that document the relevant binding pocket?
F
What evidence do you have that the observed in vitro pharmacodynamic effects are likely to translate into the patient setting? Can you demonstrate your mechanism of action in human samples? What are the strongest arguments to believe that your innovation will translate to the human condition?
G
Can you demonstrate target engagement in vivo at the anticipated site of action for the required amount of time (PK/PD modeling)?
Presentation of in vivo data
Have you ensured that your data presentation includes:
1
Description of the model
2
Compound formulation (vehicle used in the placebo group)
3
Individual values, averages, and error bars
4
Number of subjects in each group, age
5
Route of administration
6
Doses, dosing regimen
7
Positive control/reference/competitor compound
8
Statistical analysis; and
9
Indication of compound exposure and target coverage at each dose?
Selectivity
Is your data showing the selectivity of the compound against closely related targets? Has the compound been tested in broad selectivity panels (GPCRS, ion channels, cardiac safety panels, transcription factors, …)?
Pharmacokinetics
A
Do you have cross species in vitro (microsomes, hepatocytes) clearance data? Did you document in vivo PK in relevant preclinical species?
B
Can you show a clear in vivopharmacokinetic profile with dose-to-man predictions, based on an understanding of route of metabolism?
Safety screening
A
Can you show results from early in vitro (e.g. hERG, MNT, Ames, cytotox, etc.) and in vivo toxicity (e.g. 7, or 14- days exposure, Irwing test, telemetry, etc.) screening?
B
For biologics, how did you study potential immunogenicity?
Preclinical models
A
Has your compound shown dose-dependent efficacy in well-established and accepted preclinical disease model(s)?
B
Are there any other models that better represent the unmet medical need?
Additional data
A
Which data do you not yet have, but do you intend to generate in the future? What is the timeline, with key inflection points, for the generation of these data?
B
Which data are you not presenting, but is available upon request or under CDA?
C
Do you have any IP protection such as patents? If yes, what is the priority date and have they been published and granted? Have you completed any freedom to operate (FTO) assessment?
D
Are you using any licenses and what is their source?
E
When are you looking to partner, and what are you looking for from a partner? Do you already have other existing partnerships?
F
Who are your current investors and what are your plans for future financing rounds? What are your work plans in relation to financing?
G
Can you give an overview of your company, including corporate information? What is the composition of your team, board and scientific advisory board? If there are any gaps, how do you intend to fill them?