Dynavax Announces Publication of Preclinical Study of TLR9 Agonist in Lung Cancer

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.

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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 Unpublished data demonstrates that another TLR9 agonist, DV281 – optimized for delivery to primary lung tumors and lung metastases – has equivalent activity in these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.

Santhera Announces Financial Results for the First Half-Year 2018 and Updates on Operational Progress and Growth Strategy

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]).

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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

Biologicals_BeiGene_NDA_China

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]).

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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.

STORM Therapeutics and the University of Cambridge receive Innovate UK funding to help the fight against cancer

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.

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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.

BeiGene Announces Acceptance of New Drug Application for Anti-PD-1 Antibody Tislelizumab in Hodgkin’s Lymphoma in China

On August 31, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), 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 or CDA) has accepted the new drug application (NDA) for tislelizumab, an investigational anti-PD-1 antibody, as a potential treatment for patients with relapsed/refractory classical Hodgkin’s lymphoma (R/R cHL) (Press release, BeiGene, AUG 31, 2018, View Source [SID1234529218]).

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"The acceptance of our first NDA for tislelizumab represents an important milestone for BeiGene and for Chinese patients with Hodgkin’s lymphoma. Patients who relapse or don’t respond to standard treatment with chemotherapy or radiation often have poor prognoses. We are encouraged by the potential for tislelizumab to provide a new treatment option for these patients," said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene.

The NDA is supported by a clinical and non-clinical data package, including the results from a pivotal Phase 2 study of tislelizumab in Chinese patients with R/R cHL. A recent independent review of data from all 70 enrolled patients in an intent-to-treat analysis showed that with a minimum of 24 weeks of follow-up and a median follow-up time of 7.85 months at the data cutoff, overall response rate (ORR) was 85.7 percent, including 61.4 percent complete response (CR). Frequency and severity of adverse events were generally consistent with previously reported Phase 1 safety and tolerability data for tislelizumab, or, in the case of certain immune-related events such as hypothyroidism and fever, consistent with previous reports of other PD-1 antibodies for the treatment of cHL. Full results of the study are planned to be presented at an upcoming medical conference.

"We are excited by the response rates, including high complete response rates we have seen in our pivotal study in R/R cHL, and look forward to working closely with the NMPA to make tislelizumab available to patients in China as quickly as possible," said Wendy Yan, Global Head of Regulatory Affairs at BeiGene.

In addition to the pivotal Phase 2 clinical trial in R/R cHL, tislelizumab is also being studied in global Phase 3 trials in a number of malignancies, including non-small cell lung cancer, hepatocellular carcinoma, and esophageal squamous cell carcinoma; as well as two global Phase 2 trials in patients with previously treated hepatocellular carcinoma or with R/R mature T- and NK-cell lymphomas, and an additional pivotal Phase 2 trial in China in urothelial cancer.

About Classical Hodgkin’s Lymphoma
Hodgkin’s lymphoma is one of the two major types of lymphoma that begin in the lymph nodes and tissues of the lymphatic system. All other lymphomas are classified as non-Hodgkin’s lymphomas. Classical Hodgkin’s lymphoma, the most common form representing about 95 percent of the patients with Hodgkin’s lymphoma, is characterized by the presence of very large cells called Reed-Sternberg cells. There were approximately 2,100 diagnosed cases of Hodgkin’s lymphoma in China in 2012.i Although the cancer can occur in both children and adults, it is most commonly diagnosed in young adults between the ages of 15 and 35 and in older adults over age 50.

About Tislelizumab
Tislelizumab (BGB-A317) is an investigational humanized monoclonal antibody that belongs to a class of immuno-oncology agents known as immune checkpoint inhibitors. Discovered by BeiGene scientists in Beijing, tislelizumab is designed to bind to PD-1, a cell surface receptor that plays an important role in downregulating the immune system by preventing the activation of T-cells. Tislelizumab has demonstrated high affinity and specificity for PD-1. It is potentially differentiated from the currently approved PD-1 antibodies in an engineered Fc region, which is believed to minimize potentially negative interactions with other immune cells, based on preclinical data. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumor cancers outside of Asia (except Japan).