On May 14, 2018 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing investigational drugs for the treatment of orphan oncology indications, reported first quarter 2018 financial results (Press release, Mateon Therapeutics, MAY 14, 2018, View Source [SID1234526565]).

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!

For the three months ended March 31, 2018, Mateon reported a net loss of $0.8 million, a decrease of $3.2 million from the net loss of $4.0 million reported for the three months ended March 31, 2017. R&D expenses decreased to $0.2 million for the first three months of 2018 compared to $2.8 million for the same period in 2017, while general and administrative expenses decreased to $0.6 million for the first three months of 2018 compared to $1.1 million for the same period in 2017.

At March 31, 2018, Mateon had cash and cash equivalents of $0.2 million. In April 2018, Mateon raised net proceeds of approximately $2.4 million in a private placement financing transaction.

"During the first quarter of 2018 our efforts were focused on obtaining capital so that we could advance our product candidates," said William D. Schwieterman, M.D., Chief Executive Officer of Mateon Therapeutics. "After receiving the initial funds from our April financing transaction, we authorized our clinical investigators to resume screening new patients into our clinical study of OXi4503 in relapsed/refractory acute myeloid leukemia and myelodysplastic syndromes. Based on our encouraging preclinical data, we are also planning for a new clinical study of CA4P as an immuno-oncology agent in combination with Opdivo – initially evaluating the combination in advanced melanoma patients who have not responded to currently approved treatments."

On May 14, 2018 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional therapeutics, and Daiichi Sankyo Company, Limited (Daiichi Sankyo) reported that they entered into a new license agreement, building upon their 2016 cross-licensing and collaboration agreement (Press release, Zymeworks, MAY 14, 2018, View Source [SID1234526590]).

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!

"With a successful track record and our first bispecific antibody incorporating the Azymetric and EFECT technology having achieved a key research milestone in 2017, we look forward to adding two more bispecific compounds to our pipeline," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "We are exceptionally impressed with the robust impact that Zymeworks’ technology brings to antibody development."

Under the terms of the second agreement, Daiichi Sankyo will acquire licenses to Zymeworks’ Azymetric and EFECT technology platforms to develop two additional bispecific antibody therapeutics. In exchange, Zymeworks will receive an upfront technology access fee of US$18 million and may receive up to US$466.7 million in potential clinical, regulatory and commercial milestone payments. In addition, Zymeworks will receive up to double-digit tiered royalties on global product sales.

"Expanding our relationship with a leading global pharmaceutical partner like Daiichi Sankyo is extremely satisfying as it underscores the power, versatility, and attractiveness of our technology platforms," said Ali Tehrani, Ph.D., President and CEO of Zymeworks. "Having already used our platforms to discover one bispecific antibody, Daiichi Sankyo now has increased access to our technology to create additional therapeutic candidates. We are pleased to be working with a healthcare pioneer with a proven track record of over 100 years of innovation leading to major breakthroughs in patient care."

Zymeworks and Daiichi Sankyo began working together in September 2016 through an agreement to develop one bispecific antibody therapeutic for which Zymeworks is eligible to receive preclinical, clinical, and commercial milestones payments, as well as up to double-digit tiered royalties on global product sales. Additionally, Zymeworks obtained a license to certain immuno-oncology antibodies from Daiichi Sankyo, with the right to research, develop, and commercialize multiple bispecific products globally in exchange for royalties on global product sales

On May 14, 2018 Immunicom, Inc., a medical technology company developing revolutionary non-pharmaceutical approaches for treating cancer and autoimmune diseases, reported the company has received Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) for its Immunopheresis therapy, which is based on a proprietary technology that selectively removes immune inhibitors from a patient’s bloodstream potentially enabling their natural immune system to more-effectively attack cancer tumors (Press release, Immunicom, MAY 14, 2018, View Source [SID1234637423]).

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!

To achieve Breakthrough Device designation, a technology must demonstrate compelling potential to provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases. In addition, there must be no FDA approved treatments presently available, or the technology must offer significant advantages over existing approved alternatives.
​
The FDA’s intent in granting this specific designation to qualified devices is to collaboratively facilitate expediting the device’s assessment and review processes through more interactive communication and planning with the FDA; ensuring proper data collection, efficient clinical study design, senior management engagement of Agency personnel, and priority review of applicable filings.

In contrast to immunotherapy pharmaceuticals and biologicals that introduce foreign compounds and material into a patient’s body, and which are often accompanied by negative side effects, Immunicom’s patented technology is based on a "subtractive" approach that is intended to potentially limit treatment-associated adverse effects. By removing immune system inhibitors from the blood without introducing new substances, Immunicom’s therapeutic approach has been shown in preliminary preclinical studies to enhance anti-cancer immune system response without causing unwanted side effects.
​
Immunicom’s blood-filtering device technology is the first step in execution of a broader corporate strategic vision. "We are very pleased with FDA’s granting of Breakthrough Device designation for Immunopheresis, our initial immunotherapy product for treating late/end stage IV cancer patients with metastatic solid tumors," said Amir Jafri, CEO of Immunicom, "This significant milestone will enable us to more efficiently pursue device regulatory approval and address critical unmet patient needs sooner, while also continuing our commitment to invest in other exciting treatment options in our product pipeline."

On May 14, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported preclinical data on Toca 521, a retroviral replicating vector (RRV) expressing a single-chain variable fragment targeting PD-L1, will be presented at the 2018 Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), held May 16-19 in Chicago (Press release, Tocagen, MAY 14, 2018, View Source;p=RssLanding&cat=news&id=2348847 [SID1234526566]).

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!

In a preclinical study, Toca 521 reversed PD-1/PD-L1 mediated immune suppression, resulting in robust, durable and highly selective anti-tumor activity that was superior to systemically administered anti-PD-1 or anti-PD-L1 monoclonal antibodies. Tocagen expects to advance Toca 521 into investigational new drug application (IND) enabling studies this year.

In addition, previously disclosed clinical data and new preclinical results from an ovarian cancer model will be presented.

Summaries are provided below for new data sets, for which full posters will be placed on Tocagen’s website following the presentation.

Presentation Type: Poster (Abstract: 111)
Title: PD-L1 Checkpoint Blockade Using a Single-Chain Variable Fragment Targeting PD-L1 Delivered by Retroviral Replicating Vector Enhances Anti-Tumor Effect in Cancer Models
Presenter: Amy Lin, Ph.D., associate director of experimental virology at Tocagen
Date and Time: Wednesday, May 16, 5:30 p.m. CT
Summary:

A RRV expressing a single-chain variable fragment targeting PD-L1, called Toca 521, was developed using Tocagen’s proprietary cancer-selective gene therapy platform technology.
Preclinical results demonstrated Toca 521 reversed PD-1/PD-L1 mediated immune suppression in a human in vitro cell culture system, and conferred robust, durable and highly selective anti-tumor activity compared to systemically administered anti-PD-1 or anti-PD-L1 monoclonal antibodies.
These results indicate further development of Toca 521 is warranted to investigate the potential for improved safety and efficacy profiles compared to systemic monoclonal antibodies against the same checkpoint target. Toca 521 could also be useful in combination with other agents, including immuno-oncology therapies.
Presentation Type: Oral presentation (Abstract: 344)
Title: Treatment of Recurrent HGG Patients with the Retroviral Replicating Vector Toca 511 and Toca FC Resulted in Durable Responses and Survival Lasting 3 Years or Longer: Immune Mechanisms and Molecular Analyses of Tumors
Presenter: Douglas Jolly, Ph.D., executive vice president of research and pharmaceutical development at Tocagen
Date and Time: Thursday, May 17, 9:20-9:40 a.m. CT

Presentation Type: Poster (Abstract: 428)
Title: Toca 511-Mediated Prodrug Activator Gene Therapy: A Promising Therapeutic Strategy for Ovarian Cancer
Presenter: Sara Collins, Ph.D., assistant scientist in the department of cell biology at the University of Miami Miller School of Medicine. These studies were performed in the laboratory of Noriyuki Kasahara, M.D., Ph.D., professor of cell biology and pathology at the University of Miami.
Date and Time: Thursday, May 17, 5:15 p.m. CT
Summary:

Preclinical studies evaluating Toca 511 and 5-FC in a mouse model of ovarian cancer were conducted.
Increased survival and reduced tumor burden was observed in mice with pre-existing tumors receiving Toca 511 followed by 5-FC, as compared to untreated controls.
About Toca 511 & Toca FC

Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprised of an investigational biologic, Toca 511 and an investigational small molecule, Toca FC. Toca 511 (vocimagene amiretrorepvec) is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered, extended-release formulation of the prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells in the tumor microenvironment resulting in anti-cancer immune activation and subsequent tumor killing.

On May 14, 2018 Eli Lilly and Company (NYSE: LLY) reported an agreement to acquire AurKa Pharma, Inc., a company established by TVM Capital Life Science to develop oncology compound AK-01, an Aurora kinase A inhibitor that was originally discovered at Lilly (Press release, Eli Lilly, MAY 14, 2018, View Source [SID1234526591]). The compound is a potential first-in-class asset that AurKa Pharma is studying in Phase 1 clinical trials in multiple types of solid tumors.

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!

Aurora kinases are believed to play a crucial role in cellular division by controlling chromosomal segregation. Defects in segregation can cause genetic instability, a condition highly associated with the formation of tumors. Aurora kinases, consisting of Aurora A, Aurora B and Aurora C, are key mitotic regulators required for genome stability and are frequently overexpressed in cancerous tumors. AurKa Pharma’s asset, AK-01, has been shown to be highly selective for Aurora A, with potential clinical benefit observed in Phase 1 studies. Future studies will seek to determine if the selectivity profile of AK-01 can improve efficacy while limiting toxicity risks to a manageable level.

After a review of its clinical pipeline priorities in 2016, Lilly sold the compound to TVM Capital Life Science, which then established AurKa as part of the TVM Life Science Ventures VII fund. The fund is a novel investment model that seeks to develop early-stage pharmaceutical assets in a capital-efficient manner. As part of its innovation strategy, Lilly actively participates with venture capital firms to source early stage opportunities.

"The acquisition of AurKa Pharma supports Lilly’s external innovation strategy, in which we seek to partner with leading life science venture capital firms in order to identify, support and access promising innovation in areas of unmet medical need," said Darren Carroll, senior vice president of corporate business development at Lilly. "We are excited with the value TVM created for this compound through its early-Phase studies, and we look forward to more opportunities in the future."

"Lilly Oncology is focused on the development of innovative cancer therapies that can make a meaningful difference for patients," said Levi Garraway, M.D., Ph.D., senior vice president, global development and medical affairs, Lilly Oncology. "The acquisition of AurKa Pharma expands our pipeline with a promising oncology compound targeting a distinct cell cycle pathway. The work done by AurKa will allow Lilly to leverage emerging data about cancers in which this molecule might be effective, and determine if it can be beneficial to people living with various forms of cancer."

"Through the unique healthcare venture capital model pioneered by TVM Capital Life Science, companies such as AurKa have been established to more quickly and efficiently bring promising compounds to clinical proof-of-concept," said Luc Marengere, Ph.D., Managing Partner at TVM Capital Life Science. "We are pleased that the scientific advances made by AurKa could contribute to the development of AK-01 and hopefully help deliver a potential new medicine for cancer patients."

Under the terms of the agreement, Lilly will acquire all shares of AurKa Pharma. In return, AurKa Pharma shareholders will receive an upfront payment of $110 million. AurKa Pharma shareholders are also eligible to receive up to $465 million in regulatory and sales milestones should AK-01 gain approval in the U.S. and other markets, and achieve certain sales levels.

This transaction will be reflected in Lilly’s reported results and financial guidance according to Generally Accepted Accounting Principles (GAAP), and is subject to customary closing conditions. There will be no change to Lilly’s 2018 non-GAAP earnings per share guidance as a result of this transaction.

Baird is acting as financial advisor to AurKa in this transaction.