Keynote Case Study: Conducting your first clinical trial

Frustrations with preparing for our first Clinical Study – the

case of a small startup company

Overview/History 1990 - 2013

• IP developed • Animal trials (HUJI/Hadassah), Human cases

2014 - 2015

• IP released to the Inventors (from Yissum, Haddasit) • Inventors sign an agreement with Company

2015-today

• Additional animal trials performed (CNS, IBD, T2D, NAFLD) • Additional IP created (2nd generation drugs, combinations, etc.) • Pipeline en route to the FDA – T2D, IBD (PIBD), Psoriasis • Strategy – FDA – 505(b)(2)/”Big Pharma”/Non-Dilutive • FDA – submitted 3 Pre-IND “materials”, received 3 “meeting Granted Letters”, and 3 full answers • FDA agrees with 505(b)(2), opens option for Fast Track with T2D… • Big Pharma – signed CDA’s, Received 1 Term Sheet

Following funding

• Submit 3 IND applications, submit Fast Track (T2D), submit Orphan Designation (PIBD) • Finalize budgets (using CRO, etc.) • Decide on funding for Clinical Trials and continue current activities; raise additional funds as required • Add KOL’s to SAB, Commence Clinical Trials

Major Problem Today (existing and pipeline T2D drugs):

NO drug (pill or injection or other treatment) actually

treats/prevents T2D at its CORE

“Insulin Resistance” rises and

keeps rising…and rising…

Our T2D Drug Restores “Insulin Resistance” to NORMAL

levels

The saddest words in the English language:

“what might have been”.

If that is so, the happiest words are undoubtedly:

“I told you so”.

Why are we here #1?

In God We Trust;

The Plural of Anecdote Is Not Data

Others Must Provide Data

Why are we here #2?

Top 10 Global CROs 2018 A ranking system was developed for the top-10 global clinical research organizations. A score statistic was developed to determine the rank of each organization. Revenues (70%), annual Revenue growth 2016 to 2017 (10%), net income (5%), expenses ratios (5%), revenue per Employee (5%), and scope of service portfolio (5%) were the key input criteria used to calculate the score statistic for and every organization was assigned a score for each of these key input criteria.

Elementary choice…not so fast!

Does this help ME?

Revenues (70%) 5% (over $xx minimum for stability) annual Revenue growth 2016 to 2017 (10%) 0% (unless troubling figure) net income (5%) General Info expenses ratios (5%) ??? revenue per Employee (5%) ??? (maybe of interest for negotiating) scope of service portfolio (5%) 30% Responsiveness during discussions Specific TEAM, leader (experience, commitment, etc.) Proximity/culture PRICE Overhead (time, money) Preparation period “Wholeness” of proposal…no open items not included or not fully quoted…no

“surprises” How do they deal with changes/unexpected….

My rankings would be closer to:

Mature • # of people • Clear roles, redundancy • Hierarchy • Procedures • Beyond “obvious” – sales, marketing, legal, physical space, Brand • Ongoing income

Startup • SMALL # of people • No Clear roles, No

redundancy • No clear Hierarchy • Procedures…always TBD • Beyond “obvious” – 0 sales, 0

marketing, legal, small physical space, No Brand

• Ongoing income – not in the near future.

• External funding only (Investors, ND – grants/Govt. etc.)

• ABCR • Uncertainty • “Burn Rate” • Possible Pivot • URGENCY! Time sensitive

everything!

A startup is NOT a small version of a corporate!!!

MY wish list from a CRO: Consultative sales process No over promises; certainly no under delivery (later) Not yes-men! Demonstrate experience and knowledge

continuously “Startup Special” pricing Turn Key proposal with no surprises (footnoted items) Degrees of freedom for uncertainties and changes – exit

milestones; black swan Strong commitment if the specific team assigned Responsiveness on very timely manner

Dror Chevion CEO and Co-Founder, Concenter BioPharma / Silkim Ltd. www.concenterbiopharma.com| dror@silkim.comemail: |5767050 -54-972Mobile: +

WeChat: DCHEVION

Restoring INSULIN

RESISTANCE to

Normal