1. Innovation in Cancer Drug Development Balanced Dual Targeting of PI3K & mTOR BioTrinity 2016

2. Disclaimer
This presentation is being provided for the sole purpose of providing the recipients with background information about PIQUR’s business. This presentation, including the information contained in this disclaimer, does not constitute an offer, invitation or recommendation to acquire or obtain certain rights on PIQUR’s business or assets, nor to subscribe for or purchase any security and neither the presentation, disclaimer nor anything contained in them forms the basis of any contract or commitment. No representation, express or implied, is made as to the fairness, accuracy, completeness or correctness of information, opinions and conclusions contained in this presentation, including the accuracy, likelihood of achievement or reasonableness of any forecasts, prospects, returns or statements in relation to future matters contained in the presentation (“forward-looking statements”). Such forward-looking statements are by their nature subject to significant uncertainties and contingencies and are based on a number of estimates and assumptions that are subject to change at any time (and in many cases are outside the control of PIQUR and its Directors) which may cause the actual results or performance of PIQUR to be materially different from any future results or performance expressed or implied by such forward-looking statements. Forward- looking statements are provided as a general guide only and should not be relied upon as an indication or guarantee of future performance. To the maximum extent permitted by the applicable law, neither PIQUR nor its related corporations, directors, employees or agents, nor any other person, accepts any liability, including, without limitation, any liability arising from fault or negligence, for any loss arising from the use of this presentation or its contents or otherwise arising in connection with it. These terms shall be construed, enforced and performed in accordance with the substantive laws of Switzerland, without reference to the principles of conflicts of laws. The exclusive place of jurisdiction is Basel (City), Switzerland. You represent and confirm by attending and/or retaining this presentation, that you accept the above conditions.

3. Executive Summary PI3K CURE
PI3K/mTOR pathway is deregulated in the majority of cancers
PI3K and mTOR are clinically validated drug targets in oncology
PQR309 has shown encouraging clinical activity & good safety profile in solid tumor patients in phase 1
Phase 2 studies in solid tumors and lymphomas ongoing
PI3K
CURE

4. Cancer ─ A Leading Cause of Death
Leading cause of death worldwide with a high unmet medical need (8.2 million deaths in 2012)
14.1 million new cases in 2012, projected to reach 23.6 million in , a 68% increase
$80 billion global treatment cost in 2012 (indirect cost over $300 bn)
60% overall survival rate with wide disparities by cancer type and by geographies
Source: WHO World Cancer Report 2014

5. PIQUR ─ Company Facts
Founded in 2011 as a spin-off of the University of Basel
Focused on the development of targeted anti-cancer treatments
PIQUR‘s strengths:
Comprehensive know‐how in chemistry and biology of PI3K and mTOR pathways
Evidence of clinical activity in phase 1 with PQR309
Promising pipeline of follow-up and back-up compounds
Team of leading experts with successful track record in the development and launch of oncology blockbusters
PIQUR Therapeutics AG - Corporate Presentation

6. Dr. Vladimir Cmiljanović, CEO Gaudenz von Capeller, CFO
PIQUR Management Team
Dr. Doriano Fabbro, CSO
Dr. Vladimir Cmiljanović, CEO
Gaudenz von Capeller, CFO
Dr. Saša Dimitrijević, CDO
Frances Betts, COO
Hervé Girsault, CBO

7. PQR309 – our «Lead Compound»
PQR309 ─ a novel oral selective dual PI3K/mTOR inhibitor:
Crosses the blood-brain-barrier
Excellent drug-like properties
No off-target activities / highly selective to PI3K/mTOR
Phase 1 first-in-man study in Europe completed
Well-tolerated with no significant side effects
Maximum tolerated dose determined to be 80 mg
Promising signs of clinical anti-tumor activity
Phase 2 proof of concept studies started in August 2015 in selected lymphoma and solid tumor indications
PIQUR Therapeutics AG - Corporate Presentation

8. PQR309 Phase 1 Study Significant tumor reduction in individual patients
Baseline
4 cycles
Corresponding CT slices at baseline (top) and following 4 cycles (bottom) of PQR309 in a patient with relapsed thymic carcinoma and mTOR alteration demonstrating a partial response  42% reduction in target lesions
PIQUR Therapeutics AG - Corporate Presentation

9. PQR309 Phase 1 Study Significant tumor reduction in individual patients
Baseline
2 cycles of PQR309
Corresponding CT slices at baseline (top) and following 2 cycles (bottom) of PQR309 in a patient with sinonasal undifferentiated cancer and PI3K alteration
 24% size reduction in target lesions

10. PQR309 ─ Clinical Development Plan Six ongoing phase 2 studies in solid tumors and lymphomas
2014
2015
2016
2017
2018
2019
Phase 1
Solid tumors
Phase 2
Solid tumors
randomized Phase 2
Solid tumor studies
Phase 3
MTD
PK/PD/ Activity
Efficacy
Filing*
Efficacy
Filing
Phase 1
Lymphoma
studies
Phase 2
Lymphoma studies
Phase 3
MTD
Efficacy
Filing*
Efficacy
Filing
Phase 1 combo
Phase 2
PQR309 combo
randomized Phase 2/3
PQR309 combo vs combo partner
MTD
Efficacy
Filing*
Efficacy
Filing
* Fast Track
CDP PQR309 as of December 14, 2015

11. Pipeline ─ Promising Candidates in Oncology & Other Therapeutic Areas
Target
Product ID
Discovery
Lead Optimization
Preclinical Validation / PoC
Preclinical Safety
Phase 1
Phase 2
Phase 3
PQR309
panPI3K/mTOR
brain-penetrant
PQR530
PQR514
PQR6xx
mTOR
Lymphomas
Solid tumors
Lymphomas
Solid tumors
Lymphomas
Solid tumors
Oncology
CNS / Other indications
R&D collaboration
Pipeline as of December 21, 2015
JBL200=PQR620, AF030=PQR620, FB214=PQR514 and EJ092=PQR692
PIQUR Therapeutics AG - Corporate Presentation

12. PIQUR ─ Strong funding Funding received: Funding plans
USD 60 M raised since inception
Equity from Private Investors in Seed A, B, C, Series A
Series A Extension round led by Versant Ventures
Series A2 backed by Versant Ventures and private investors
Funding fully secured to execute development program up to completion of Phase 2 with PQR309 and Phase 1 for a follow-up compound
Funding plans
Series B financing round in 2017
Alternative licensing deal with pharma partners
Exit Opportunities
Trade sale
IPO
PIQUR Therapeutics AG - Corporate Presentation

13. Contact Details
Vladimir Cmiljanovic, PhD Chief Executive Officer T
Gaudenz von Capeller Chief Financial Officer M  
PIQUR Therapeutics AG  Hochbergerstrasse 60C  CH-4057 Basel Switzerland

14. Thank you !
PIQUR Therapeutics AG - Corporate Presentation