On December 12, 2018 NETRIS Pharma, a clinical-stage company developing novel anticancer therapies targeting dependence receptors, reported successful completion of the first-in-human study on the effects of netrin-1 inhibition in patients with advanced solid tumors (Press release, Netris Pharma, DEC 12, 2018, View Source [SID1234611180]). The Phase 1 dose-escalation study showed that NP137, a first-in-class monoclonal anti-netrin-1 blocking antibody, was safe and well tolerated up to 20mg/kg, with no dose limiting toxicity (DLT), meeting the primary objective. In addition, patients with advanced uterine cancers exhibited encouraging signs of anti-tumor activity, including prolonged stable disease and objective response.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are very pleased with the safety and tolerability profile of NP137 in this study, which enabled evaluation of netrin-1 inhibition in advanced solid tumors," said Philippe Cassier, MD, PhD, Head of Phase 1 unit at Centre Léon Bérard and Principal Investigator of the NP137 study.

"I am really impressed by the preliminary anti-tumor activity observed in this study with patients showing objective response and prolonged one-year disease control," added Professor Jean Yves Blay, MD, Ph.D. General Director of the Centre Léon Bérard and past president of EORTC.

The Phase 1 study was designed to evaluate the maximum tolerated dose, pharmacokinetics and pharmacodynamics, and establish the recommended dose for the Phase 1b/2 expansion study. NP137 was dosed every other week starting from 1 mg/kg up to 20mg/kg in heavily pre-treated patients with advanced solid tumors. Additional study objectives were to evaluate the adverse events (AE), pharmacokinetics (PK), pharmacodynamics (PD) and preliminary anti-tumor activity of NP137. The study enrolled a total of 19 patients in three French centers, the Centre Léon Bérard in Lyon, the Institut Claudius Régaud in Toulouse and the Institut de Cancérologie de l’Ouest in Nantes. A Phase 1 extension study with an additional 24 patients is ongoing to confirm the safety and tolerability of NP137, collect biopsies before and after treatment to allow measurement of potential biomarkers and confirm the preliminary clinical activity seen during dose escalation. The final phase 1a data will be submitted for presentation at an upcoming international medical conference.

"Meeting the primary objective of the dose-escalation part of this study and generating preliminary positive efficacy data, particularly in such chemotherapy-resistant patients, provide an important confirmation of our dependence receptor science and lay the groundwork for larger clinical studies with NP137 as a targeted approach to treat solid tumors in combination with chemotherapy, immune checkpoint inhibitors and tyrosine kinase inhibitors," said Prof. Patrick Mehlen, Chief Executive Officer of NETRIS Pharma. "Additionally, we would like to thank the investigators, patients and caregivers for their participation in this study, and for contributing to our understanding of NP137 as we work to address a significant medical need."

About NP137
Most types of tumors produce an abnormal amount of dependence receptors’ ligands, which prevents cells from dying. Netrin-1 is overexpressed in a large percentage of human cancers, including over two thirds of gynecologic cancers. Expression of netrin-1 often correlates with disease severity and no therapy has ever been tested against this new pathway.

NP137, a humanized monoclonal antibody of isotype IgG1 directed against netrin-1, is the first drug candidate developed by NETRIS Pharma. NP137 prevents the binding of netrin-1 on its dependence receptors, thereby re-inducing cancer cell death and impacting tumor cell plasticity. Pre-clinical studies show NP137 to have an anti-cancer effect as a monotherapy as well as synergistic effects in combination with chemotherapy, immune checkpoint inhibitors or tyrosine kinase inhibitors.

On December 12, 2018 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported that the first patient was treated in a phase 1 dose escalation study combining pelareorep and carfilzomib with Bristol-Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab) to treat relapsed multiple myeloma patients (Press release, Oncolytics Biotech, DEC 12, 2018, View Source [SID1234532025]). This study is based on findings from the NCI 9603 multiple myeloma study that combined pelareorep with carfilzomib that resulted in objective responses, elimination of multiple myeloma cells and most importantly, an inflamed phenotype with PD-L1 overexpression.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Having worked with pelareorep in multiple myeloma and understanding its ability to act as a potentiator of checkpoint blockade, I’m very excited to work with the Oncolytics team on this study," said Dr. Craig Hofmeister, Associate Professor, Department of Hematology and Medical Oncology Emory University School of Medicine. "Pelareorep has proven its ability to create an inflamed phenotype and its potential for upregulation of PD-1 on tumor-infiltrating lymphocytes. My hope is this study leads not only to an effective combination dosing schedule but provides quantitative data describing the expression of PD-1, along with correlative studies that reveal the roles of both immune-mediated and direct cytotoxic myeloma cell killing."

This open-label, phase 1 study, conducted by Dr. Hofmeister at Emory University, will enroll up to 62 patients to examine the side effects and best dosing schedule of pelareorep when given in combination with dexamethasone, carfilzomib, and nivolumab in treating participants with relapsed multiple myeloma. The primary objectives of the study are to determine the maximum tolerated dose of pelareorep in combination with carfilzomib and nivolumab. Secondary outcome measures include time to progression, progression-free survival and overall survival, as well as the characterization of an inflamed phenotype and confirmation of biomarker responses indicative of tumor inflammation.

"We now have our second checkpoint inhibitor combination study enrolling, and I’m excited for the potential of the immune and biomarker data to come from it," said Dr. Rita Laeufle, Chief Medical Officer of Oncolytics Biotech. "These studies, along with our soon to be initiated studies combining pelareorep with Merck’s Keytruda, also in multiple myeloma, and Roche’s Tecentriq in neoadjuvant breast cancer, will provide further evidence that pelareorep has the potential to expand the use of checkpoint inhibitors by priming tumors cells. The confirmation of our predictive biomarkers enhances the likelihood of success in registrational studies, thereby reducing both clinical and commercial risk making pelareorep more attractive to potential partners."

For more information about the study, including a comprehensive list of inclusion and exclusion criteria, please visit: www.clinicaltrials.gov (identifier: NCT03605719).

About Pelareorep
Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

On December 12, 2018 Santhera Pharmaceuticals (SIX: SANN) reported its financial results for the nine months ended September 30, 2018, and confirms its guidance and positive outlook (Press release, Santhera Pharmaceuticals, DEC 12, 2018, View Source [SID1234532027]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Santhera is on track to meet its strategic and financial goals for 2018. The Company further delivered on its active in-licensing strategy for high quality, late-stage rare disease assets by acquiring an exclusive sub-license option to vamorolone, the first-in-class dissociative steroid currently in development for the treatment of Duchenne muscular dystrophy (DMD). On the commercial side, the Company has further grown sales of the revenue-generating product Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON) and expects to meet its 2018 full-year sales guidance in the range of CHF 30-32 million.

Santhera continued its business expansion and grew sales to CHF 23.6 million in the first nine months of 2018 which corresponds to a 45% growth rate (compared to the same period in the previous year). Development expenses increased by 49% compared to the same prior-year period, primarily driven by clinical development activities for POL6014 for the treatment of cystic fibrosis (CF) and regulatory work in preparation of filings for idebenone in the indication DMD. Efficient use of resources resulted in lower marketing and sales expenses (-6%) and a slower rise in general and administrative expenses (+18%). Taken together, this contributed to an increase of operating expenses by 20% compared to the same period of last year. In summary, Santhera closed the nine-month period under review with a net result of CHF -39.9 million (Jan.-Sept. 2017: CHF -33.3 million).

Financial highlights:

Sales of CHF 23.6 million (Jan.-Sept. 2018), reflecting an increase by 45% compared to same period last year (Jan.-Sept. 2017: CHF 16.3 million)
Operating expenses of CHF 57.2 million (Jan.-Sept. 2017: CHF 47.7 million)
Operating result of CHF -37.2 million (Jan.-Sept. 2017: CHF -34.2 million), leading to a net result of CHF -39.9 million (Jan.-Sept. 2017: CHF -33.3 million)
Cash, cash equivalents and short-term financial assets of CHF 25.4 million (as of September 30, 2018)
2018 full-year sales guidance of CHF 30-32 million confirmed
Corporate highlights:

Agreement with Idorsia Ltd under which Santhera is to acquire the option to an exclusive sub-license of the first-in-class dissociative steroid vamorolone in all indications and all countries worldwide except Japan and South Korea (November 20, 2018)
Idorsia became largest shareholder in Santhera
Extraordinary General Meeting (EGM) approval of the capital increase in connection with the vamorolone sub-license agreement (December 11, 2018)
Operational highlights (July 2018 to present day):

Analysis of new data linking study findings with idebenone in DMD to clinically relevant patient benefits for inclusion in regulatory submissions in Europe and the U.S.
Positive opinion on orphan drug designation received from European regulators for POL6014 for the treatment of CF
Start of a Phase Ib/IIa multiple ascending dose (MAD) trial with POL6014 in patients with CF
Revenue Guidance:
Santhera will continue to grow its international business, advance its pipeline programs and actively proceed with business development initiatives to expand its late stage product portfolio. Based on its sales performance to date this year, the Company confirms its previous guidance and expects to reach a full-year sales for Raxone in the range of CHF 30-32 million in 2018.

Note
The financial statements as of and for the nine months ended September 30, 2018 have been prepared and are being published exceptionally on the occasion of and in connection with the capital increase proposed to and approved by the EGM held on December 11, 2018. Santhera publishes results in line with the disclosure requirements of the SIX Swiss Exchange. Santhera does not plan on publishing quarterly reports in the future.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On December 12, 2018 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), reported that Yuichi Iwaki, MD, PhD, President and Chief Executive Officer, and Geoffrey O’Brien, JD/MBA, Vice President and Executive Officer, will present a corporate overview at the J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 2:00 pm at the Westin St. Francis Hotel in San Francisco, California (Press release, MediciNova, DEC 12, 2018, View Source;p=irol-newsArticle&ID=2380360 [SID1234532029]). MediciNova’s management team, including Dr. Yuichi Iwaki, Geoffrey O’Brien, and Kazuko Matsuda, MD, PhD, MPH, Chief Medical Officer, will be available for one-on-one meetings at this conference and investors may request a one-on-one meeting through J.P. Morgan.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!