On September 4, 2018 TESARO, Inc. (NASDAQ: TSRO), an oncology-focused biopharmaceutical company, reported it has initiated the second stage of the JASPER study that is designed to assess clinical benefit of ZEJULA in combination with an anti-PD-1 antibody in first-line non-small cell lung cancer (NSCLC) patients (Press release, TESARO, SEP 4, 2018, View Source [SID1234529255]). The decision to advance the trial was based on achieving the protocol defined response criteria in the initial cohort of 16 treated patients with high PD-L1 expression, of which 14 were evaluable for a response. Nine of the 14 patients had objective responses by RECIST criteria at the time of the analysis1; with all 14 patients experiencing tumor shrinkage.

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!

"These JASPER data provide preliminary evidence that the combination of ZEJULA and an anti-PD-1 antibody could be active as a first-line treatment for patients with non-small cell lung cancer and high levels of PD-L1 expression," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "In the second stage of the trial, 36 additional patients will be enrolled and treated with ZEJULA in combination with TSR-042, our anti-PD-1 antibody. TSR-042 is the foundation of our lung cancer strategy, and is also being studied as a monotherapy in our GARNET trial in anti-PD-(L)1 naïve patients who have progressed on chemotherapy, and in combination with TSR-022, our anti-TIM-3 antibody, in AMBER, a study in late-line NSCLC patients that have progressed after anti-PD-(L)1 therapy. We look forward to sharing lung cancer data from both GARNET and AMBER at the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November."

About the JASPER Clinical Trial
JASPER is a Phase 2, open-label, single arm trial designed to evaluate the safety and efficacy of ZEJULA in combination with an anti-PD-1 antibody for the treatment of first-line NSCLC. Patients were enrolled in stage 1 of the study and received a starting dose of 200 milligrams of niraparib once per day and 200 milligrams Q3 weeks of an anti-PD-1 antibody. The primary endpoint of stage 1 of the study was objective response rate (ORR) per RECIST. Other endpoints include durability of response, disease control rate, progression free survival (PFS), overall survival (OS) and safety and tolerability.

About ZEJULA (Niraparib)
Niraparib is marketed in the United States and Europe under trade name ZEJULA. ZEJULA (niraparib) is a poly(ADP-ribose) polymerase (PARP) inhibitor indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. In preclinical studies, ZEJULA concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect.

ZEJULA (niraparib) Select Important Safety Information
Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML) was reported in patients treated with ZEJULA in some clinical studies. Discontinue ZEJULA if MDS/AML is confirmed.

Hematologic adverse reactions (thrombocytopenia, anemia and neutropenia) have been reported in patients treated with ZEJULA. Do not start ZEJULA until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1). Monitor complete blood counts weekly for the first month, monthly for the next 11 months of treatment, and periodically after this time.

Hypertension and hypertensive crisis have been reported in patients treated with ZEJULA. Monitor blood pressure and heart rate monthly for the first year and periodically thereafter during treatment with ZEJULA. Closely monitor patients with cardiovascular disorders, especially coronary insufficiency, cardiac arrhythmias, and hypertension.

Based on its mechanism of action, ZEJULA can cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for six months after receiving the final dose. Because of the potential for serious adverse reactions in breastfed infants from ZEJULA, advise a lactating woman not to breastfeed during treatment with ZEJULA and for one month after receiving the final dose.

On September 4, 2018 Dynavax Technologies Corporation (NASDAQ: DVAX) reported publication of a preclinical study demonstrating that inhalation of a TLR9 agonist can stimulate effective immunity against lung tumors and complement the actions of PD-1 blockade to generate durable, systemic anti-tumor immunity (Press release, Dynavax Technologies, SEP 4, 2018, View Source [SID1234530120]). The paper titled Inhaled TLR9 Agonist Renders Lung Tumors Permissive to PD-1 Blockade by Promoting Optimal CD4+ and CD8+ T cell Interplay, by Dynavax scientists M.Gallotta, H. Assi, E. Degagné, S. Kannan, R.Coffman and C. Guiducci was published in the journal Cancer Research. The study demonstrated that combining an inhaled TLR9 agonist with systemic anti-PD-1 led to long-term survival in two different mouse lung tumor models, mediated by systemic immunity that eradicated tumors both in the lung and in distal organs. The study further delineated the distinctive mechanisms of action of these agents in the lung environment.

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!

Administration of the TLR9 agonist SD-101 into the lungs of mice with metastatic tumors generated anti-tumor responses that controlled or eliminated tumor growth in the lungs as well as in non-treated organs, including liver. Treatment with SD-101 resulted in ~90% decrease in tumor burden in both the lung and liver. This led to a significant increase in survival time, with a majority of mice surviving beyond 90-100 days. Treatment with SD-101 and anti-PD-1 resulted in a large increase of tumor-reactive T cells, which were required for anti-tumor activity. The durable control of liver metastases shows that local administration of SD-101 to the lung generates an anti-tumor T cell response capable of controlling tumor growth beyond the lung itself.

The TLR9 agonist used in these studies was SD-101, Dynavax’s lead clinical candidate currently being developed as an intratumoral agent in combination with anti-PD-1 therapy in patients with advanced melanoma and head and neck squamous cell carcinoma. Unpublished data demonstrates that another TLR9 agonist, DV281 – optimized for delivery to primary lung tumors and lung metastases – has equivalent activity in these models. These studies provide the preclinical rationale for the Phase 1b dose escalation study of inhaled DV281 currently being conducted by Dynavax in advanced non-small lung cancer patients (NCT03326752). DV281 and SD-101 stimulate potent Type 1 interferon induction along with maturation of dendritic cells into effective antigen-presenting cells. These combined actions lead to the increased numbers of cytotoxic T cells that are critical for the induction of effective systemic anti-tumor immunity.

On September 4, 2018 Santhera Pharmaceuticals (SIX: SANN) reported its first half-year results as of June 30, 2018, outlines the Company’s vision and strategy as it moves through the second half of 2018, and positions itself for future growth (Press release, Santhera Pharmaceuticals, SEP 4, 2018, View Source [SID1234529256]).

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!

Thomas Meier, PhD, Chief Executive Officer of Santhera, said: "Our vision is to be a leader in the development and commercialization of rare disease therapies for neuro-ophthalmology, neuromuscular and pulmonary indications. Our strategy to achieve this vision focuses on three distinct pillars: One, we continue to expand our commercial reach and grow sales of our revenue generating product Raxone for the treatment of LHON. Turnover during the first half-year has been above expectation and we are on track to exceed our 2018 guidance. Two, we are progressing our pipeline assets towards regulatory approval in the EU and the U.S. and, with the inclusion of new data, intend to submit marketing authorization applications for idebenone in DMD in 2019. Three, we are pursuing an active in-licensing strategy for high quality, late-stage rare disease assets with a short time to market."

"We see multiple business development opportunities to leverage our existing development, regulatory and commercial capabilities and our recent in-licensing for POL6014 to treat cystic fibrosis is the first example of Santhera advancing this strategy. With this vision and strategy in mind, we believe Santhera is optimally positioned to create value with its existing and future product portfolio opportunities."

Financial highlights:

1H 2018 sales of CHF 16.0 million, increase of 48% compared to 1H 2017
Operating expenses of CHF 39.9 million (1H 2017: CHF 30.5 million)
Operating result of CHF -26.3 million (1H 2017: CHF -21.4 million) leading to a net result of
CHF -27.4 million (1H 2017: CHF -22.7 million)
Cash, cash equivalents and short-term financial assets of CHF 34.8 million (June 30, 2018)
Full year sales guidance raised to CHF 30-32 million
Operational highlights:

Acquisition of worldwide exclusive license to develop and commercialize clinical stage candidate POL6014 for cystic fibrosis (CF) and other pulmonary diseases
Renewal of the Early Access to Medicines Scheme (EAMS) Scientific Opinion by UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for idebenone for patients with Duchenne muscular dystrophy (DMD) in the UK
Launch of Expanded Access Program with idebenone for patients with DMD in the U.S.
Submissions of regulatory dossiers for Raxone in Leber’s hereditary optic neuropathy (LHON) in South Korea and Serbia
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. (planned for 2019)
Progress with clinical development candidates having successfully completed first clinical trial with omigapil in patients with congenital muscular dystrophy (CMD) and advanced preparations for multiple-ascending dose trial for POL6014 in CF
First half-year overview

Strong Raxone sales in 1H 2018
Net sales of Raxone in Europe amounted to CHF 16.0 million (1H 2017: CHF 10.9 million) which corresponds to a strong 48% increase year-on-year. Turnover was mainly driven by increased number of patients receiving the drug in existing markets and new launches in additional EU countries. Santhera’s goal is to provide treatment to LHON patients worldwide and the Company has submitted a new drug application for LHON in South Korea, one of the major markets in Asia. A decision from the South Korean drug regulatory authorities who granted orphan drug designation for Raxone in LHON can be expected by summer 2019. At the end of the first half of 2018, Santhera was marketing Raxone in more than 20 countries.
Broadened product pipeline with licensing agreement
In February, Santhera completed the first step in its strategy to in-license pipeline strengthening, clinical stage product candidates in neuro-ophthalmology, neuromuscular and pulmonary diseases by entering into a license agreement with Polyphor for POL6014. Under the agreement, Santhera obtained the worldwide, exclusive rights to develop and commercialize POL6014, a clinical stage selective inhibitor of human neutrophil elastase with the potential to treat cystic fibrosis and other pulmonary diseases.
UK’s MHRA renewed EAMS positive scientific opinion for Raxone in DMD
In June, the UK’s MHRA renewed the EAMS scientific opinion for Raxone for a further year for patients with DMD in respiratory function decline who are not taking glucocorticoids. Inclusion in EAMS allows eligible patients with DMD, who meet criteria defined under this scheme, to gain free of charge access to Raxone in the UK.
Launch of U.S. Expanded Access Program with idebenone for patients with DMD
Santhera has successfully launched and enrolled the first patients in a U.S. Expanded Access Program (EAP), called BreatheDMD, with idebenone. Through the BreatheDMD program, eligible patients in the U.S. with DMD who are 10 years and older and in respiratory function decline can obtain access to investigational idebenone, at no cost, through a growing network of research centers across the U.S.
New supporting data to be included in submissions for marketing authorization applications for DMD in 2019
In July, Santhera announced results of a comparative analysis of the Phase III DELOS trial outcome with new data from natural history studies. This analysis showed that the treatment effect with idebenone observed in the DELOS trial can be linked to a delay in the initiation of assisted ventilation by three years, which is of high clinical relevance. In coming months, Santhera and its academic partners will prepare for the publication of additional clinical data that demonstrate long-term efficacy of idebenone on respiratory function outcomes in patients with DMD, thereby supporting the positive data from the successful Phase III DELOS trial. The findings will be discussed with regulators in the coming months and will be included in the regulatory dossier in preparation of marketing authorization applications for idebenone in DMD in Europe and the U.S. in 2019.
Omigapil safe and well tolerated in patients with congenital muscular dystrophy (CMD)
The single-center interventional trial to establish the pharmacokinetic profile and to evaluate the safety and tolerability of omigapil in pediatric and adolescent patients with CMD was successfully completed. Santhera plans to seek advice on the clinical development program of omigapil by the TREAT-NMD Advisory Committee for Therapeutics (TACT).
Liquidity base allows for the continuation of the strategy as planned
As of the end of June 2018, Santhera had cash, cash equivalents and short-term financial assets of CHF 34.8 million (December 31, 2017: CHF 58.2 million). These funds will allow the Company to proceed with its clinical trial program and regulatory filings as foreseen.
Revenue Guidance

Santhera will continue to grow its international business, advance its pipeline programs and proceed business development initiatives to expand its late stage product portfolio. Based on its sales performance in the first six months of the current year and the positive outlook, the Company expects to exceed its guidance of CHF 28-30 million and anticipates reaching a higher turnover of CHF 30-32 million in 2018.

Conference Call
Santhera will host a conference call on September 4, 2018 at 13:00 CEST / 12:00 BST / 07:00 EDT. Thomas Meier, PhD, CEO of Santhera, and Christoph Rentsch, CFO of Santhera, will discuss the half-year 2018 financial results and will provide an update on corporate developments.
Participants are invited to call one of the following numbers 10-15 minutes before the conference call starts (no dial-in code is required):
Europe: +41 58 310 50 00
UK: +44 207 107 0613
USA: +1 631 570 5613

On September 3, 2018 STORM Therapeutics, the drug discovery company focused on the discovery of small molecule therapies modulating RNA epigenetics, and The University of Cambridge reported that they have been granted a Knowledge Transfer Partnership (KTP) (Press release, STORM Therapeutics, SEP 3, 2018, View Source [SID1234561043]). The Grant, of up to £240,000, is to develop an analysis platform using the data warehouse software InterMine to help STORM advance its cancer research.

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!

STORM has established a pipeline of drug discovery programmes to develop novel, first-in-class drugs for the treatment of specific cancers and other diseases with high unmet medical need.

Keith Blundy, Chief Executive of STORM Therapeutics said: "Biology is an extremely fast paced, changing area. STORM is on the cutting edge of cancer research and keeping up with the data that is constantly being generated is vital. Previously, this data has been spread across a number of silos and gathering all of this information, as well as being able to analyse it efficiently, can be costly and prone to error. Through the KTP programme STORM is now partnered with an open-source data warehouse, InterMine, specifically for the integration and analysis of complex biological data, making it easy to query and analyse."

Gos Micklem, Principal Investigator for the InterMine project based at the Department of Genetics at the University of Cambridge said: "We are excited by this KTP award and the opportunity to collaborate with STORM. We see benefits on both sides – STORM gains our experience in large-scale data integration and the freely available open-source InterMine platform, while the adaptations that will be made to accommodate their data and interests will improve InterMine for the benefit of the broader community."

The KTP programme helps businesses in the UK by linking them with an academic organisation, enabling them to bring in new skills and the latest academic thinking to deliver a specific, strategic innovation project.

On September 3, 2018 BeiGene Ltd., a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported that the National Medical Products Administration of China (NMPA, formerly known as CFDA), has accepted its new drug application (NDA) for its anti-PD-1 antibody, tislelizumab, for relapsed/refractory classical Hodgkin’s lymphoma (R/R cHL) (Press release, Boehringer Ingelheim, SEP 3, 2018, View Source [SID1234529412]).

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!

Since BeiGene and Boehringer Ingelheim started their collaboration in 2013, Boehringer Ingelheim’s biopharmaceutical contract manufacturing business, known as Boehringer Ingelheim BioXcellence, has been providing the Chemistry, Manufacturing, and Control (CMC) services for support of the tislelizumab. Boehringer Ingelheim assisted BeiGene with tislelizumab’s development through process and analytical method development, supply of GMP clinical material, and CMC filing support with the NMPA.

Tislelizumab is an investigational anti- PD-1 antibody being studied in a number of malignancies, such as relapsed/refractory classical Hodgkin’s Lymphoma, non-small cell lung cancer, hepatocellular carcinoma, esophageal squamous cell carcinoma, mature T-and NK-cell lymphomas, and urothelial cancer. Boehringer Ingelheim Biopharmaceuticals (China) Ltd. is committed to providing BeiGene with full contract manufacturing support and supply of tislelizumab at the highest global quality standards to serve patients with this important medicine, if approved.