PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, announced today that the European Medicines Agency (EMA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of patients with diffuse large B-cell lymphoma (DLBCL).

“The EMA orphan drug designation for PQR309 in DLBCL is another important regulatory milestone, validating the potential therapeutic use of PQR309 in DLBCL,” said Claudia Pluess, Senior Regulatory Affairs Manager at PIQUR. Dr. Vladimir Cmiljanovic, CEO of PIQUR, added, “PIQUR will continue to work with physicians and regulatory agencies to further define the clinical development strategy to bring a potential new treatment option to patients suffering from this disease.”

DLBCL is an aggressive form of lymphoma, and the most common type of non-Hodgkin lymphoma (NHL), accounting for about 30 percent of all NHL cases [1]. The disease occurs primarily in older individuals, though it can also occur in children and young adults in rare cases. 10 to 15 percent of DLBCL patients exhibit refractory disease and an additional 20 to 25 percent relapse after initial response to therapy [2].

In addition to this orphan drug designation by the EMA in DLBCL, PIQUR has also recently received orphan drug designation from the FDA for PQR309 for the treatment of primary CNS lymphoma (PCNSL).

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union must not be more than 5 in 10,000) and allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life threatening or a chronically debilitating rare disease.

References
[1] American Society of Clinical Oncology. Lymphoma – Non-Hodgkin: Subtypes (Dec. 2016 revision). http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed April 2017.
[2] Sehn, L. Paramount prognostic factors that guide therapeutic strategies in diffuse large B-cell lymphoma. Hematology, December 2012; 1; 402-409.

PDF versions of the release German and English.

Dr. Melanie Rolli joins PIQUR as new Chief Medical Officer

PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, announced today that it has strengthened its management team with the appointment of Dr. Melanie Rolli as Chief Medical Officer.

“We are very pleased to welcome Melanie Rolli to PIQUR as our Chief Medical Officer,” said Vladimir Cmiljanovic, CEO of PIQUR Therapeutics. “Melanie’s broad research and drug development expertise and proven leadership skills will be instrumental to the future success of PIQUR.”

Melanie Rolli brings more than a decade of industry experience and senior responsibilities in global development, medical affairs and patient safety. She joins PIQUR from Novartis Pharma, where she held various positions with increasing responsibilities over the last 14 years, most recently as Global Head Medical Safety. Prior to joining Novartis, she worked as a medical doctor at the University Hospital in Mannheim and as a basic researcher in academic settings in Germany and at the Scripps Research Institute, USA.

PIQUR’s management team now consists of the following members:

• Vladimir Cmiljanovic, Chief Executive Officer
• Dr Melanie Rolli, Chief Medical Officer
• Doriano Fabbro, Chief Scientific Officer
• Hervé Girsault, Chief Business Officer

“I am excited to join the talented and passionate team at PIQUR at this stage, and I look forward to helping the team advance the development of new and innovative cancer therapies for patients in need,” said Melanie Rolli.

PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, and Pierre Fabre, the second largest private French pharmaceutical group announced today that they have signed a collaboration agreement for the development of PIQUR’s lead compound PQR309 in dermato-oncology. The exclusive deal will see PIQUR and Pierre Fabre’s R&D teams work together on the evaluation and development of dermatological formulations of PI3K/mTOR inhibitors across multiple therapeutic indications.

Following the first stage of the collaboration, Pierre Fabre will have the option to enter into exclusive negotiations to license global rights for the program in selected skin cancer conditions. Terms and conditions of the collaboration were undisclosed.

Pierre Fabre is strategically positioned in both oncology and dermatology from discovery all the way through commercialization with multiple products at various stages of development and already launched on the market.

“The collaboration with Pierre Fabre, a worldwide leader in dermatology is an important milestone for PIQUR, validating its potential to bring novel solutions to patients for the treatment of dermato-oncology diseases with unmet medical needs,” said Hervé Girsault, Chief Business Officer of PIQUR.

“We are looking forward to working with PIQUR as part of our commitment to external innovation. This collaboration with PIQUR marks an additional milestone in our partnering strategy. Combining our capabilities will accelerate the drug discovery process and bringing forward transformative therapies for patients.” said Laurent Audoly, Head of Research & Development at Pierre Fabre Pharmaceuticals.

Download a PDF version of the press release here (English).

PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, announced today that the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of primary central nervous system lymphoma (PCNSL).

PCNSL is a very rare and aggressive form of lymphoma involving brain and its linings, eyes or spinal cord. The disease affects less than 1 person in 100,000 in the USA with approximately 7,500 new cases reported annually in the USA, Europe and Japan. Treatment options available for PCNSL are limited, and the prognosis with current therapies is poor.

“We are very pleased to receive FDA orphan drug designation for PQR309 in PCNSL. This is an important regulatory milestone for the company and a significant step towards the clinical advancement of PQR309,” said Dr. Ruggero Della Bitta, Chief Medical Officer of PIQUR. “This represents an important step towards addressing a high unmet medical need and bringing a potential treatment to those with this rare and life-threatening disease.”

The Orphan Drug Designation Program, administered by the FDA’s Office of Orphan Products Development, is intended to encourage companies to develop therapeutics for diseases that affect fewer than 200,000 people in the USA. The designation provides the company several benefits and incentives, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, as well as a seven-year period of market exclusivity upon regulatory product approval.

Download PDF versions of the press release here (German) and here (English)

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. Preclinical and Phase 1 studies have shown PQR309 to have a favorable safety, tolerability and pharmacokinetic profile. In addition, PQR309 has shown both preclinical activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in five Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877).

Agreement will see eribulin used in combination with novel PI3K/mTOR inhibitor in patients with triple negative breast cancer

Hatfield, UK and Basel, Switzerland, 17 February 2016 – Eisai and PIQUR Therapeutics today announce a landmark agreement to conduct a Phase 1/2b clinical study to investigate PQR309 in combination with Halaven^® (eribulin) in patients with triple-negative breast cancer (TNBC).

A significant number of HER2-negative breast cancer patients are expected to have activated PI3K, de novo or induced by prior chemotherapy administration. PQR309 is currently engaged in multiple Phase 1 and Phase 2 studies as a single agent and has shown promising activity. The combination of a PI3K/mTOR inhibitor with eribulin may prove to be an effective treatment in second line therapy for locally advanced or metastatic TNBC patients.

The Phase 1/2b study is scheduled to begin in early 2016. The initial Phase 1 dose-escalation part of the study will assess the safety and tolerability of PQR309 combined with eribulin in patients with locally advanced or metastatic HER2-negative and triple-negative breast cancer. The Phase 2b expansion part of the study will enroll patients with advanced or metastatic TNBC. The primary objective of this part of the study is to evaluate the efficacy of the PQR309 in combination with eribulin. In total, the Phase1/2b study will enroll approximately 60 patients.

PIQUR will be responsible for conducting the Phase 1/2b clinical trial and the parties may extend the collaboration to include a Phase 3 clinical trial as well as additional trials in new indications of mutual interest.

“We are delighted to enter into this collaboration with PIQUR Therapeutics, which has rapidly established itself as one of the leading experts in the field of PI3K/mTOR inhibitors. Based on convincing pre-clinical data and on the promising results of PIQUR’s front runner compound PQR309 shown in Phase 1 study, we believe that combining eribulin with PQR309  may provide new treatment options for patients with TNBC,” said Dr Takashi Owa, Chief Innovation Officer, Vice President Eisai Co., Ltd.

“We are very pleased to enter into this collaboration with Eisai, one of the worldwide leaders in oncology and to jointly explore new avenues to address an unmet need in breast cancer. We look forward to a fruitful partnership and to pursue the development of this combination treatment following the completion of this first study,” said Hervé Girsault, Chief Business Officer of PIQUR Therapeutics.

Eribulin is the first in the halichondrin class, microtubule dynamics inhibitors with a novel mechanism of action. Structurally eribulin is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Eribulin is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division.¹

Eribulin remains the only single agent chemotherapy to significantly improve overall survival in women with advanced breast cancer after anthracycline and taxane treatment. Metastatic breast cancer is a very difficult condition to treat and only 15% of women will survive beyond five years.²

This collaboration underscores Eisai’s human health care (hhc) mission, the company’s commitment to innovative solutions in disease prevention, cure and care for the health and well-being of people worldwide. Eisai is committed to oncology to address the unmet medical needs of patients and their families.

Download PDF versions of the press release here (German) and here (English)

The European Society for Medical Oncology (ESMO), the leading pan-European organisation in medical oncology, announced that the President for the 2018-2019 period will be Josep Tabernero, Head of the Medical Oncology Department at the Vall d’Hebron University Hospital, and Director of the Vall d’Hebron Institute of Oncology (VHIO) in Barcelona, Spain.

To read the full press release, please visit the ESMO website.

PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company focused on the discovery and development of innovative anti-cancer drugs based on PI3K and mTOR inhibition, today announced the closing of an oversubscribed CHF 18 million (USD 19M) round of Series A2 financing. This round was backed by several existing investors, including Versant Ventures, as well as new, private investors.

The new funding brings the total capital raised by PIQUR to date to over CHF 55 million (USD 60M). The company plans to use the funding to further strengthen its operational basis and to extend the financing horizon for additional Phase 2 studies for its lead compound PQR309, which has shown preclinical activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway, as well as clinical activity in phase 1. In addition, the new funding will support the preclinical safety and first clinical studies for one of its follow-up compounds.

“We greatly appreciate the continued support provided by our existing shareholders and welcome our new investors who bring valuable perspective, experience, and capital resources to the company,” said Vladimir Cmiljanovic, CEO of PIQUR. Gaudenz von Capeller, PIQUR’s CFO, commented: “This financing strengthens our balance sheet as we work to achieve important milestones for our lead candidate as well as the follow-up compound.”

Helping patients to survive cancer  
PIQUR aims to help patients to survive cancer. Two out of three people are now living at least five years after their cancer has been diagnosed. Despite of significant medical innovations in the treatment of cancer, there remains a high unmet medical need for therapies that prolong patients’ survival and improve their quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes involved in malignant disease, such as cell growth, proliferation, survival and metastasis, making them attractive targets in cancer therapy.

About PQR309
PIQUR’s lead compound, PQR309, is a novel, oral, balanced pan- PI3K/mTOR inhibitor with excellent prospects to become a powerful anti-cancer drug. PQR309 compares favorably to current and clinically most advanced pan-PI3K/mTOR inhibitors with respect to the drug-like properties. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to oncologic as well as hematologic malignant diseases involving the brain. PQR309 showed preclinical activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway, as well as clinical activity in phase 1.

About PIQUR
PIQUR is a Swiss clinical-stage pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel, focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. Both PI3K and mTOR are clinically validated drug targets in oncology. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

Media Relations:
Tatsuo Satoh
Head IT & Communications
T: +41 61 633 29 38
M: +41 78 819 17 44
tatsuo.satoh@piqur.com

Martin Meier-Pfister
Partner IRF Communications
T: +41 43 244 81 40
martin.meier-pfister@irfcom.ch

Investor Relations:
Gaudenz von Capeller
Chief Financial Officer
T:  +41 61 633 29 31
M: +41 79 798 64 43
gaudenz.capeller@piqur.com

The pharmaceutical company wins the 2015 SEF.Award in the “High-tech/Biotech” category

PIQUR has been named the winner of the 2015 Swiss Economic Award in the “High-tech/Biotech” category. Today, at the 17th Swiss Economic Forum in Interlaken, Switzerland’s most prestigious prize for start-up enterprises was awarded in front of an audience of 1,350 representatives from the worlds of business, politics, science and the media. More than 300 start-ups were competing for this year’s Swiss Economic Award, which is subdivided into three categories: “High-tech/Biotech”, “Services” and “Manufacturing/Trade”. The three winning companies were chosen by a high-calibre jury in a three-stage judging process. What most impressed the jury about PIQUR is the speed in bringing together an interdisciplinary and very competent team of experts from industry and academia. Furthermore PIQUR attracted many renowned Swiss and International investors as the company succeeded in reaching key clinical milestones earlier than expected.

Vladimir Cmiljanovic, PIQUR’s CEO, commented on his company’s triumph: “We are delighted to win the Swiss Economic Award, as this is a great tribute to the whole team and everything we have achieved to date. This start-up prize also spurs us on to channel our commitment and energy into further developing our product pipeline. Our goal is to put ourselves in a position to offer another treatment option to as many cancer patients as possible soon.” He then added some words of gratitude: “I would like to take this opportunity to express my sincere thanks to our employees, investors and everyone else who has supported us in achieving our vision since our company was founded.”

About the Swiss Economic Award
Overall around 300 start-ups from all over Switzerland put themselves in the running for this year’s Swiss Economic Award. The preliminary jury analysed the dossiers submitted by the companies based on the criteria specified by the judging process, which is certified by the Swiss Association for Quality Management Systems (SQS). The jurors then short-listed 15 start-ups, which received an on-site visit from a group of 15 experts. The aim of the Swiss Economic Award is to foster an entrepreneurial spirit and help young entrepreneurs make their way in society. The competition is open to all companies that have been established for no more than six years and are based in Switzerland. The winners receive CHF 75,000 in prize money and a comprehensive package of rewards. Further information is available at www.swisseconomic.ch/en/sef-2015.

Helping patients to survive cancer
PIQUR aims to help patients to survive cancer. Two out of three people are now living for at least five years after their cancer has been diagnosed. In spite of significant medical innovations in the field of cancer treatment, there is still a great need for therapies that can prolong patients’ survival and improve their quality of life. PIQUR focuses specifically on PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), key signalling molecules that play a vital role in various biological processes involved in malignant diseases, such as cell proliferation, survival and metastasis, making them attractive prospects for cancer therapy.

About PQR309
PIQUR’s lead compound, PQR309, is a potent and balanced pan-PI3K/mTOR inhibitor with excellent prospects to become a powerful anti-cancer drug. PQR309 compares favorably to current and clinically most advanced pan-PI3K/mTOR inhibitors with respect to the off-target effects and drug-like properties. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use beyond solid tumors and lymphomas and into brain cancers. PQR309 showed activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway.

About PIQUR
PIQUR is a Swiss pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel, focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. Both PI3K and mTOR are clinically validated drug targets in oncology. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com.

Media Relations:
Tatsuo Satoh
Head IT & Communications
T: +41 61 633 29 38
M: +41 78 819 17 44
tatsuo.satoh@piqur.com

Daniel Piller
Partner IRF Communications
T: +41 43 244 81 54
daniel.piller@irfcom.ch

Investor Relations:
Vladimir Cmiljanovic, PhD
Chief Executive Officer
T: +41 61 633 29 30
M: +41 76 516 87 72
vladimir.cmiljanovic@piqur.com

© Swiss Economic Forum 2015

Study confirms that PQR309 is well tolerated and shows promising signs of clinical antitumor activity

Please find PDF versions of the release here (German) and here (English).

PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, today announced positive results from the Phase 1 first-in-human study with PQR309 in advanced solid tumors.

PQR309 is a novel, balanced pan-class 1 PI3K/mTOR inhibitor formulated for oral administration. Preclinical studies demonstrated that PQR309 is a highly selective kinase inhibitor, penetrating blood-brain barrier with potent in vitro as well as in vivo antitumor activity.

The results from the first-in-man Phase 1 trial indicate that PQR309 is safe and well tolerated with expected pharmacokinetic profile, pharmacodynamic effect as well as clinical activity.

In total, 28 patients with advanced solid tumors were treated in cohorts of escalating PQR309 doses. The maximum tolerated dose (MTD) of PQR309 was determined to be 80 mg administered at continuous once-daily dosing schedule. Pharmacokinetic data indicate fast absorption of PQR309 as well as dose proportionality. Pharmacodynamic assessment in tumors shows downregulation of PI3K/mTOR pathway phosphoproteins.

One patient with a dysregulation of the mTOR-pathway demonstrated a confirmed partial response while another patient with a tumor containing a PI3K mutation, showed important tumor reduction and a minor response according to RECIST (Response Evaluation Criteria in Solid Tumors). “Data obtained from this first-in-human clinical study, including the evidence of clinical activity is very encouraging and highlights the clinical potential of PQR309”, commented Dr. Sasa Dimitrijevic, Chief Development Officer of PIQUR.

The results of this study will be presented at ASCO, May 29 to June 2, 2015:

“A phase 1 first-in-human (FIH) dose-escalation (DE) study of the oral dual PI3K/mTOR inhibitor PQR309 in patients (pts) with advanced solid tumors: Final DE results.” [Poster Session: Developmental Therapeutics—Clinical Pharmacology and Experimental Therapeutics, Sub-category: Signal Transduction, Abstract Number 2592, Mon, May 30, 8:00 – 11:30 AM, Location: S Hall A, Poster Board #308]

PIQUR has also received approval from the health authorities for the initiation of a Phase 2 clinical trial with PQR309, which aims to obtain efficacy data at well-tolerated doses in patients with selected solid tumor and hematological indications with aberrant PI3K/mTOR signaling. “Following recent regulatory approval to start a Phase 2 study of PQR309, we are excited to advance our compound as a potential treatment option for selected cancer indications”, commented Prof. Dr. Richard Herrmann, PIQUR’s Chief Medical Officer.

Helping patients to survive cancer  
PIQUR aims to help patients to survive cancer. Two out of three people are now living at least five years after their cancer has been diagnosed. Despite of significant medical innovations in the treatment of cancer, there remains a high unmet medical need for therapies that prolong patients’ survival and improve their quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes involved in malignant disease, such as cell proliferation, survival and metastasis, making them attractive targets in cancer therapy.

About PQR309
PIQUR’s lead compound, PQR309, is a novel, oral, balanced pan-class 1 PI3K/mTOR inhibitor with excellent prospects to become a powerful anti-cancer drug. PQR309 compares favorably to current and clinically most advanced pan-PI3K/mTOR inhibitors with respect to the drug-like properties. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to oncologic as well as hematologic malignant diseases involving the brain. PQR309 showed activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway.

About PIQUR
PIQUR is a Swiss clinical-stage pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel, focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. Both PI3K and mTOR are clinically validated drug targets in oncology. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

Initial clinical results confirm that PQR309 is well tolerated and shows first signs of clinical antitumor activity

PIQUR Therapeutics AG today announced that the company and its academic collaborators will present five posters highlighting preclinical as well as clinical data on its lead compound, PQR309, at the American Association for Cancer Research (AACR) Annual Meeting, which will take place in Philadelphia, Pennsylvania, April 18-22, 2015. Initial results from the first-in-man phase 1 trial indicate that PQR309 is safe and well-tolerated with no significant side effects. „In addition to the good safety data, PQR309 has also shown the first encouraging signs of clinical activity in solid tumors. With these promising initial clinical results of PQR309, we are making an important step towards our vision to help patients to survive cancer”, commented Dr. Vladimir Cmiljanovic, CEO of PIQUR Therapeutics AG.

Following are the specifics of the abstracts, including session times and locations:

• “PQR309: Structure-based design, synthesis and biological evaluation of a novel, selective, dual pan-PI3K/mTOR inhibitor” [Poster Session: PI3K Pathway and Metabolism Modulators, Abstract Number 2664, Mon, Apr 20, 1:00 – 5:00 PM, Location: Section 32, Poster Board Number 16]
• “PQR309: A potent, brain-penetrant, dual pan-PI3K/mTOR inhibitor with excellent oral bioavailability and tolerability” [Poster Session: Pharmacokinetics and Pharmacodynamics, Abstract Number 4514, Tue, Apr 21, 1:00 – 5:00 PM, Poster Section 32, Poster Board Number 12]
• “First-in-man (FIM) pharmacodynamic (PD) and pharmacokinetic (PK) phase I trial of PQR309 in advanced solid tumors” [Poster Session: Phase I Clinical Trials, Abstract Number CT310, Tue, Apr 21, 8:00 AM – 12:00 PM, Poster Section 24, Poster Board Number 10]
• “Pre-clinical activity and mechanism of action of the novel dual PI3K/mTOR inhibitor PQR309 in B-cell lymphomas” [Poster Session: PI3K Pathway and Metabolism Modulators, Abstract Number 2652, Mon, Apr 20, 1:00 – 5:00 PM, Poster Section 32, Poster Board Number 4]
• “BKM120-mediated G2 arrest: Structural and functional segregation of off-target action and PI3K inhibition” [Poster Session: Signal Transduction Inhibitors, Abstract Number 671, Sun, Apr 19, 1:00 – 5:00 PM, Poster Section 28, Poster Board Number 15]

Helping patients to survive cancer  
PIQUR aims to help patients to survive cancer. Two out of three people are now living at least five years after their cancer has been diagnosed. Despite of significant medical innovations in the treatment of cancer, there remains a high unmet medical need for therapies that not only prolong patients’ survival but also significantly improve quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes, such as cell proliferation, survival and metastasis, making inhibition of the target attractive for cancer therapy.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries, as well as cellular technology platforms, and their excellent products with novel, dual-acting ‘strong PI3K plus fine-tuned mTOR’ inhibitors that address the given challenges, meeting therapeutic, tolerance and galenic needs.

About PQR309
PIQUR’s lead compound, PQR309, is a potent and balanced pan-PI3K/mTOR inhibitor with excellent prospects to become a powerful anti-cancer drug. PQR309 compares favorably to current and clinically most advanced pan-PI3K/mTOR inhibitors with respect to the off-target effects and drug-like properties. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use beyond solid tumors and lymphomas and into brain cancers. PQR309 showed activity in different aggressive cancer cell lines inhibiting the PI3K/mTOR pathway. PIQUR has successfully concluded the first-in-man phase 1 study in Europe; the maximum tolerated dose (MTD) was determined and the results indicated first evidence of clinical antitumor activity. The full results of the phase 1 trial are anticipated in the second quarter of 2015 after completion of the ongoing expansion study in USA.

About PIQUR
PIQUR is a Swiss pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel, focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. Both PI3K and mTOR are clinically validated drug targets in oncology. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

PIQUR, focused on the research and development of new and innovative cancer treatments, has won the Northwest Switzerland Young Entrepreneurs’ Award.

On 7 November 2014, at the Congress Centre of the Basel Trade Fair, six finalists were presented and recognized for their work in front of an audience of 500 guests. PIQUR stood out from some 60 competitors all vying for the award. The jury was impressed by the company’s professionalism in the field of cancer research. The Northwest Switzerland Young Entrepreneurs’ Award honors innovative companies in the northwest region of Switzerland that have been established or significantly reorganized in the last five years.

Vladimir Cmiljanovic, CEO of PIQUR Therapeutics AG, expressed great pleasure at the award: “The Northwest Switzerland Young Entrepreneurs’ Award is a beautiful tribute to the great commitment shown by the entire team over the last few years. At the same time, the award gives us the incentive to continue the so far successful development of our drug PQR309 with passion and determination. I would like to take this opportunity,” he continued, “to express my sincere thanks to the staff, investors and all the people who have actively supported us since our inception.”

Northwest Switzerland Young Entrepreneurs’ Award
The Northwest Switzerland Young Entrepreneurs’ Award honors innovative companies from the northwest region of Switzerland that have been established or significantly reorganized in the last five years. It is awarded every two years in autumn, alternating with the Prix SVC North Switzerland. The Northwest Switzerland Young Entrepreneurs’ Award is sponsored by the Basel-Stadt Association of Small and Medium-Sized Enterprises and the Junior Chamber International Basel, and the award provides a platform for increasing the regional awareness of young, innovative companies. www.jungunternehmerpreis.ch

Since the start of the company, PIQUR has raised total proceeds of over CHF 37 million

Funds targeted to reach Phase II and proof-of-concept for lead compound PQR309

Please find PDF versions of the release here (German) and here (English).

BASEL, Switzerland / SAN FRANCISCO, USA – PIQUR Therapeutics AG and Versant Ventures jointly announced today the investment of the California-based venture capital firm of CHF 11 million (USD 12M) in PIQUR. The pharmaceutical company focused on the discovery and development of novel, small molecules for the treatment of cancer, has closed a Series A extension round of CHF 18 million (USD 20M). This round brings the total funding of the Series A to CHF 32 million (USD 36M). Versant Ventures led the extension round, which also included new as well as existing private shareholders. Since the start of the company PIQUR has raised total proceeds of over CHF 37 million (USD 42M).

Versant Ventures, specializing in life sciences investments, has more than USD 1.6 billion in assets under management. Versant Ventures has recently established an industry-leading biotech-focused team in Basel, Switzerland. Concurrent with the closing of the transaction, Guido Magni, Venture Partner with Versant Ventures in Basel, has joined PIQUR’s Board of Directors. Guido previously served as a Managing Director of EuroVentures, a Versant incubator, where he was intimately involved in several biotech investments including Synosia (sold to Biotie Therapies), Flexion (IPO) and Okairos (sold to GSK). Prior to that he was the Head of the Medical Science Department of Roche Pharmaceuticals and oversaw the development and the registration of a large number of key oncology drugs.

In addition, Gianni Gromo, Venture Partner with Versant Ventures, will act as an observer in PIQUR’s Board of Directors and will give strategic advice to the company. He was previously the Global Head of the Cardio Metabolic Disease Area at Roche Pharmaceuticals. Currently, he serves on the Board of Novira Therapeutics and on scientific advisory boards of a number of academic institutions and biotechs.

“We would like to thank our existing investors for their strong confidence in PIQUR. We welcome the new private shareholders and Versant Ventures as new investors and look forward to working with Guido Magni and Gianni Gromo,” commented Vladimir Cmiljanovic, CEO of PIQUR Therapeutics AG. “Their experience and network in drug development, profound expertise in oncology as well as in the investment sector, nicely complements PIQUR’s other Board members’ perspectives. We plan to use the funds to advance development of our lead compound PQR309 to reach Phase II and deliver proof-of-concept in the clinic, as well as the discovery of additional drug candidates for oncology and non-oncology indications.”

“This is an exciting time to become involved with PIQUR. The company’s innovative dual-acting PI3K/mTOR inhibitors and highly experienced team made this an attractive investment for Versant. In recent months we have conducted a detailed due diligence of the company and its assets and were impressed by PIQUR’s overall IP and the lead compound PQR309. We are excited to bring in our management and industry expertise into a great management team and to support the development of novel anti-cancer therapeutics,” said Guido Magni, Venture Partner of Versant Ventures.

To turn cancer into a manageable disease
PIQUR aims to help turn cancer into a manageable disease. Despite significant advances in the fight against cancer, there remains a high unmet medical need for therapies that not only prolong patients’ survival but also significantly improve quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes, such as cell proliferation and survival, making inhibition of the target attractive for cancer therapy.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries, as well as cellular technology platforms, and their excellent products with novel, dual-acting ‘strong PI3K plus fine-tuned mTOR’ inhibitors that address the given challenges, meeting therapeutic, tolerance and galenic needs.

About Versant Ventures
Versant Ventures is a leading venture capital firm that specializes in investments in innovative biopharmaceuticals, medical devices and other life science opportunities. Founded in 1999, the firm consists of an experienced team of managing directors committed to helping entrepreneurs build successful companies that impact healthcare and improve quality of life. The firm’s successful IPOs include Clovis Oncology, Jazz Pharmaceuticals, Genomic Health, Insulet and Kythera. M&A exits include Amira, LenSX, Acclarent, Cameron Health, Ablation Frontiers and Okairos. In addition to an industry-leading biotech-focused team in Basel, Switzerland, Versant is present in San Francisco and two locations in Canada. www.versantventures.com

Media Contact
IRF Communications
Daniel Piller
Rämistrasse 4
8024 Zürich
Switzerland
Tel +41 43 244 81 54
daniel.piller@irfcom.ch
www.irfcom.ch

Contact PIQUR Therapeutics AG
Vladimir Cmiljanovic, PhD
Chief Executive Officer
Mattenstrasse 24A
4058 Basel
Switzerland
Tel +41 61 695 31 40
vladimir.cmiljanovic@piqur.com
www.piqur.com

License agreement with University of Basel extended by additional indications

PIQUR Therapeutics AG, focused on the discovery and development of innovative anti-cancer drugs, has successfully completed the Series A financing round. The private placement was rapidly oversubscribed. Subscribers include existing shareholders as well as new investors from the life science industry.

Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR, commented: “We are delighted by the trust of investors in our company. In particular, we successfully attracted further distinguished individuals from the life science industry as investors. The successful round of funding has encouraged us in our determination to continue pursuing our corporate strategy. The funds that have been procured secure the cost of the first clinical studies with our lead compound PQR309.”

PIQUR has received approval from the authorities in Switzerland and UK to conduct the clinical Phase I study with PQR309. This study will assess the safety in cancer patients, and will help determine the ideal dosage. On January 2, 2014, PQR309 was administered to the first patient at the University Hospital in Basel. “For PIQUR, the very first administration of PQR309 represents an important milestone in clinical development. First results of the study are expected to be released before the end of 2014,” said Prof. Dr. Richard Herrmann, Chief Medical Officer of PIQUR.

Also of crucial importance for the future development of the company is the broadening of the license agreement with the University of Basel. The license agreement with the University of Basel was previously limited to the field of cancer, and has now been extended to a broad range of promising clinical indications. “The expansion of the license agreement is very important for the progress and development of our company. We now have the unlimited freedom to assess the therapeutic benefit of PQR309 and all other research programs in other therapeutic fields, such as immunology, neurology and ophthalmology,” pronounced Prof. Dr. Bernd Giese, Chairman of the Board of Directors.

To turn cancer into a manageable disease
PIQUR aims to help turn cancer into a manageable disease. Despite significant advances in the fight against cancer, there remains a high unmet medical need for therapies that not only prolong patients’ survival but also significantly improve quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes, such as cell proliferation and survival, making inhibition of the target attractive for cancer therapy.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries, as well as cellular technology platforms, and their excellent products with novel, dual-acting ‘strong PI3K plus fine-tuned mTOR’ inhibitors that address the given challenges, meeting therapeutic, tolerance and galenic needs.

Media Contact
IRF Communications
Daniel Piller
Rämistrasse 4
8024 Zürich
Switzerland
Tel +41 43 244 81 54
daniel.piller@irfcom.ch
www.irfcom.ch

Contact PIQUR Therapeutics AG
Vladimir Cmiljanovic, PhD
Chief Executive Officer
Mattenstrasse 24A
4058 Basel
Switzerland
Tel +41 61 695 31 40
vladimir.cmiljanovic@piqur.com
www.piqur.com

PIQUR Therapeutics AG congratulates its Scientific Advisory Board Member, Michael Hall on winning the first Breakthrough Prize in Life Sciences.

Michael N. Hall, Professor of Biochemistry at the Biozentrum of the University of Basel, was awarded the Breakthrough Prize in Life Sciences, 2014, yesterday in the U.S.A. This award honors Hall for his discovery of target of rapamycin (TOR), a central regulator of cell growth and metabolism. This is the first time that this prize, endowed with three million dollars, has been granted to a scientist in Switzerland. The Breakthrough Prize is sponsored, among others, by internet entrepreneurs including Mark Zuckerberg, of Facebook, and Sergey Brin, of Google.

Yesterday, in Mountain View, California, the seven winners of this year’s Breakthrough Awards were presented. Among them was Prof. Michael N. Hall from the Biozentrum, University of Basel, who was honored with a Breakthrough Prize in Life Sciences. The prize is awarded for outstanding research achievements aimed at curing intractable diseases and extending human life.

The Breakthrough Prize was granted this year for the second time and is endowed with three million dollars for each awardee. Among the sponsors of the award are the Google founder Sergey Brin, the biotech entrepreneur Anne Wojcicki and the Facebook founder Mark Zuckerberg.

TOR – A key protein

Michael Hall, who works at the Biozentrum of the University of Basel since 1987, has been awarded this prize for the discovery of the protein «target of rapamycin» (TOR), which regulates both cell growth and cell size. Since the function of this protein is inhibited by the substance rapamycin, Hall named the growth regulator «target of rapamycin» or «TOR» for short. In TOR, Hall found a key protein in cellular communication, which though producing a pharmacological blockade can contain the uncontrolled cell division that is typical for cancer.

Hall’s discovery has contributed to a deeper understanding of the most fundamental life processes such as cell division, growth and death. Insights into TOR signaling pathways and their dysregulation in disease may provide new therapeutic strategies for a wide range of diseases.

Hall has published his research findings in over 160 scientific articles and currently leads a team of 12 researchers. He is a member of several advisory and editorial boards and professional societies such as the American Association for the Advancement of Science (AAAS). He is the recipient of many awards, including the Cloëtta Prize (2003), the Louis-Jeantet Prize (2009) and the Marcel Benoist Prize (2012). Hall is a SystemsX.ch steering committee member and is Director of the Basel Signaling Alliance. Michael N. Hall is a Swiss-American dual citizen and lives with his family in Basel.

Board of Directors and Management Team strengthened with top experts

Basel, Switzerland – PIQUR Therapeutics AG, focused on the discovery and development of innovative anti-cancer drugs, announces that is has successfully closed a third seed financing round. The private placement was oversubscribed in a very short time frame and included new investors from the life science sector as well as existing shareholders.

Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR commented: “We are delighted to have attracted so much interest to enable us to complete our financing so quickly. The financing gives us the means to start with the clinical development program and to bring our lead compound PQR309 one step closer to become an excellent targeted cancer drug.”

In addition PIQUR was recently awarded a substantial grant from Eurostars for the further development of its front-runner compound PQR309. The Eurostars Programme is a European Joint Programme dedicated to the R&D performing small and mid-sized enterprises, and co-funded by the European Communities and 33 EUREKA member countries. PIQUR applied for the Eurostars project jointly with WIL Research Europe (the Netherlands). In Switzerland the project will be funded by the State Secretariat for Education, Research and Innovation and in the Netherlands by the NL Agency. Cmiljanovic commented: “This is an excellent and encouraging result and another confirmation for our vision to develop PQR309 to an important cancer treatment”.

Additional member of the Board of directors
At the annual Shareholder Meeting on May 27, 2013, Roger Meier was elected as a Non-Executive Member of the Board of Directors. By this appointment the Company broadens the expertise of its Board in the field of management, finance and investor relations. In his earlier career, Roger Meier helped an US investment bank to establish a Swiss client base with institutional investors and consulted mid to large sized multinational companies at Coopers & Lybrand in the field of financial and risk management.

Management team extended with multi-disciplinary and international specialists
During recent months the management team has been extended by the appointment of an experienced Business Development & Licensing industry expert, Mr. Hervé Girsault. PIQUR’s management team now consists of the following members:

• Dr. Vladimir Cmiljanovic, Chief Executive Officer
• Dr. Doriano Fabbro, Chief Scientific Officer
• Frances Betts, Chief Development Officer
• Prof. Dr. Richard Herrmann, Chief Medical Officer
• Gaudenz von Capeller, Chief Financial Officer
• Hervé Girsault, Chief Business Development & Licensing

For more information about the Management team, please see Management.

Cmiljanovic commented: “We have further strengthened our leadership team by top experts with outstanding track records in business and science. Our management team now assembles a multi-disciplinary and international team of specialists from every relevant discipline”.

To turn cancer into a manageable disease
The aim of PIQUR’s activities is to turn cancer into a manageable disease. Given the relatively small number of effective cancer treatments available today, there is an unmet medical need for new therapeutic approaches. One in every two men and one in every three women, living in the developed world, will develop cancer at some stage in their lives. PIQUR targets the PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin) pathways.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries as well as cellular technology platform and their excellent products with novel, dual-acting ‘strong PI3K plus finely-tuned weaker mTOR’ inhibitors that fully address the given challenges, meeting therapeutic, tolerance and galenic needs.

About PIQUR

PIQUR is a Swiss pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. While mTOR is a clinically validated drug target, PI3K inhibition achieved proof of concept status in clinical phase III. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

Media Contact
IRF Communications
Daniel Piller
Rämistrasse 4
8024 Zürich
Switzerland
Tel +41 43 244 81 54
daniel.piller@irfcom.ch
www.irfcom.ch

Contact PIQUR Therapeutics AG
Gaudenz von Capeller

Chief Financial Officer
Mattenstrasse 24A
4058 Basel
Switzerland
Tel +41 79 798 64 43
gaudenz.capeller@piqur.com
www.piqur.com

Basel, Switzerland – PIQUR Therapeutics AG, focusing on the discovery and the development of innovative anti-cancer drugs, announces today that it has successfully completed preclinical profiling and the GLP toxicology studies with its most advanced compound PQR309. The preclinical studies confirm that the compound is a highly selective brain penetrant dual PI3K/mTOR inhibitor, which displays potent anti-proliferative in vitro and in vivo antitumor efficacy. The standard GLP 4-week toxicology studies, which have been successfully completed, will allow PIQUR to start its clinical trials with PQR309 in selected cancer patients.

Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR commented: “This is excellent news for PIQUR. Based on these results no special safety monitoring appears to be necessary during Phase I clinical studies planned to start in Q1/2014. Our compound has a benefit to risk ratio that is anticipated to be superior to other PI3K inhibitor front-runners. This makes PQR309 an excellent candidate to treat cancer patients.” The protocol for Phase I study with PQR309 is being submitted to regulatory authorities and ethics committees for approval to start in January 2014. Phase I will run in four centers in Switzerland, one center in Spain and one center in England. The study focus is on safety and the identification of the maximum tolerated dose.

To turn cancer into a manageable disease

The aim of PIQUR’s activities is to turn cancer into a manageable disease. Given the relatively small number of effective cancer treatments available today, there is an unmet medical need for new therapeutic approaches. One in every two men and one in every three women, living in the developed world, will develop cancer at some stage in their lives. PIQUR targets the PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin) pathways.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries as well as cellular technology platform and their excellent products with novel, dual-acting ‘strong PI3K plus finely-tuned weaker mTOR’ inhibitors that fully address the given challenges, meeting therapeutic, tolerance and galenic needs.

About PIQUR

PIQUR is a Swiss pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. While mTOR is a clinically validated drug target, PI3K inhibition achieved proof of concept status in clinical phase III. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

Media Contact
IRF Communications
Daniel Piller
Rämistrasse 4
8024 Zürich
Switzerland
Tel +41 43 244 81 54
daniel.piller@irfcom.ch
www.irfcom.ch

Contact PIQUR Therapeutics AG
Vladimir Cmiljanovic, PhD
Chief Executive Officer
Mattenstrasse 22
4002 Basel
Switzerland
Tel +41 61 695 31 40
vladimir.cmiljanovic@piqur.com
www.piqur.com

PIQUR Therapeutics AG is a targeted cancer therapy start-up company focused on the discovery and the development of innovative anti-cancer drugs. PIQUR was incorporated in August 2011 as a spin-off of the University of Basel. PIQUR’s pipeline originates from one of the most promising research areas in oncology.

Financing for the preclinical development has been secured in seed funding in October 2012 from private investors and the Swiss Innovation Promotion Agency KTI/CTI. In addition to that PIQUR is enjoying unique support by its hosting University of Basel, both in terms of IP funding and the use of laboratories and support. These funds will be spent for the preclinical pharmacology and regulatory toxicology studies and the operational set-up of the company.

Exceptional research cluster at Rosental Biopark in Basel

PIQUR is embedded in a broad and very experienced network in Basel and internationally. The exceptional location at the Rosental Biopark enables an exchange between different biotech companies and the neighbouring pharma companies. It builds a unique cluster of young, talented and experienced researchers in the field of oncology. «The team of PIQUR has an outstanding track record in science and business. Selected top-experts from industry have been appointed as executives and advisors», says Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR.

PIQUR is a spin-off of the University of Basel, and there is a licence agreement between the two institutions. The research group of Prof. Dr. Matthias P. Wymann, a professor at the Institute of Biochemistry and Genetics at the University of Basel, has shown how knowledge of the molecular processes involved in the development of disease can be used to develop targeted therapies.

Exemplary cooperation between universities and industry

The University is committed to translational research in which the results of basic research are used to develop commercially viable treatments. PIQUR provides an excellent example of cooperation between universities and industry. “The University is proud of PIQUR, not only for the quality of its basic research but also for the potential benefits that the new drugs offer cancer patients,” said Prof. Dr. Edwin Constable, Deputy Vice Chancellor (Research) at the University of Basel.

In PIQUR’s case, scientists from the fields of organic chemistry, pharmaceutical sciences, molecular biology and biochemistry collaborated to identify new classes of drugs, which are the foundation on which the biotech start-up is based.

To turn cancer into a manageable disease
The aim of PIQUR’s activities is to turn cancer into a manageable disease. Given the relatively small number of cancer treatments available today, there is thus a tremendous need for new therapeutic approaches. One in every 2 men and one in every 3 women, living in the developed world, will develop cancer at some stage in their lives. PIQUR targets the PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin) pathways.

PIQUR’s differentiation against its peers is the outstanding industry expertise, the level of innovation with its unique, proprietary fragment and scaffold libraries as well as cellular technology platform and their best-in-class products with novel, dual-acting ‘strong PI3K plus finely-tuned weaker mTOR’ inhibitors that fully address the given challenges, meeting therapeutic, tolerance and galenic needs.

About PIQUR

PIQUR is a Swiss biotech start-up incorporated in August 2011 as a spin-off of the Basel University focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. While mTOR is a clinically validated drug target, PI3K inhibition achieved proof of concept status in clinical phase IIb. PIQUR has a secured patent space, has protected a large portion of relevant chemical space.

www.piqur.com

University of Basle

The city of Basel is home to the oldest university in Switzerland. Founded upon the initiative of local citizens in 1460, the University of Basel is a modern and attractive centre of teaching, learning, and research situated in the heart of the historic old town. It provides committed individuals from all over the world with a strong academic community and an inspiring work environment. It is home to 3’500 staff. Our mission is to accomplish first-class research, teaching, and public service. We rank among the world’s one hundred best universities and boast a top-ten place among German-speaking universities.

www.unibas.ch

Media Contact:

PIQUR Therapeutics AG
IRF Communications
Stefan Mathys, Daniel Piller
Rämistrasse 4
8024 Zürich
Switzerland

Tel +41 43 244 81 49
Mob +41 79 227 98 31

stefan.mathys@irfcom.ch
daniel.piller@irfcom.ch
www.irfcom.ch

Universität Basel

Matthias Geering
Leiter Kommunikation & Marketing
Petersgraben 35
CH-4003 Basel
Switzerland

Tel +41 61 267 35 75
Mob +41 79 269 70 71
matthias.geering@unibas.ch

PIQUR was incorporated in August 2011 as a spin-off of the Basel University. PIQUR is a targeted cancer therapy start-up company focused on the discovery and the development of innovative anti-cancer drugs. PIQUR is embedded in a huge and very experienced network in Basel and internationally. The exceptional location at the Rosental campus enables an exchange between different biotech companies and builds a unique cluster of young, talented and experienced researchers in the field of oncology. PIQUR is enjoying unique support by its hosting University of Basel, both in terms of IP funding and the use of laboratories and labour.