On November 6, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), reported that the Company will present preclinical data on a new proprietary technology platform called Bolt-On Chimeric Receptor, or BOXR, designed to improve the effectiveness of T cells in solid tumor cancers, at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting taking place November 7-11, 2018 in Washington, D.C (Press release, Unum Therapeutics, NOV 6, 2018, View Source [SID1234530852]).

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Unum has generated a large panel of novel bolt-on transgenes, evaluated using a unique screening strategy, and has identified lead candidates most likely to enhance T cell functions in the solid tumor microenvironment. The bolt-on transgenes enhanced immunometabolism or costimulation of both ACTR- and CAR-expressing T cells and significantly improved their function under stringent in vitro and in vivo conditions.

"We are excited by this new platform, which we believe has the potential to further bolster our ACTR technology pipeline, and highlights our innovative approaches to overcome immunosuppressive challenges in solid tumors," said Seth Ettenberg, Unum’s Chief Scientific Officer. "We look forward to continuing to explore the potential of this novel technology by selecting a lead development candidate to bring to patients in need in the clinical setting."

Details on the presentation are as follows:

Presentation Title: Select metabolic and costimulatory bolt-on transgenes enhance chimeric receptor-bearing T cell activity against solid tumors
Presenter:Luke Barron, PhD, Unum Therapeutics
Session Title: Cell-Based Therapies for Solid Tumors
Poster Number: 216
Date & Time: Saturday, November 10th, 12:20-1:50 and 7-8:30pm
Location:Walter E. Washington Convention Center, Hall E

On November 6, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported three presentations of updated clinical and preclinical data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting & Pre-Conference Programs, to be held Nov. 7-11 in Washington, D.C., the 3rd CNS Anticancer Drug Discovery and Development Conference (CADDDC), to be held Nov. 14-15 in New Orleans and the 23rd Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO), to be held Nov. 15-18 in New Orleans (Press release, Tocagen, NOV 6, 2018, View Source;p=RssLanding&cat=news&id=2375540 [SID1234530869]).

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The clinical data includes details of immunologic trends seen in responding patients with recurrent high grade glioma (HGG) from a Phase 1 study of Toca 511 (vocimagene amiretrorepvec) & Toca FC (5-fluorocytosine, extended-release). Additionally, new preclinical data will be presented demonstrating the enhanced efficacy of Toca 511 & Toca FC regimen when combined with metronomic cyclophosphamide.

Summaries are provided below; full posters or presentations will be placed on Tocagen’s website following the presentation.

Details of the SITC (Free SITC Whitepaper) presentation are as follows:

Presentation Type: Poster (Abstract: P620)
Title: Enhanced Efficacy and Combinability of Low Dose Toca 511 and 5-FC with Metronomic Chemotherapy in Preclinical Models
Presenter: Sophie Viaud, Ph.D.
Date and Time: Saturday, Nov. 10, 7:00-8:30 p.m. ET
Summary:

This study aimed to determine if the addition of metronomic cyclophosphamide would provide therapeutic benefit when combined with Toca 511 and 5-FC (5- fluorocytosine) in a preclinical, subcutaneous glioma model.
The addition of metronomic cyclophosphamide improved tumor control and survival.
Regulatory T cells were significantly reduced in the peripheral blood with the combination with metronomic cyclophosphamide and CD8+ T cells were significantly increased.
Results support the further evaluation of Toca 511 & Toca FC with metronomic dosing of cyclophosphamide and potentially other chemotherapeutics.
Details of the CADDDC presentation are as follows:

Presentation Type: Oral Presentation (Abstract: 0023)
Title: PD-L1 Checkpoint Blockade Using a Single-Chain Variable Fragment Targeting PD-L1 Delivered by Retroviral Replicating Vector (Toca 521) Enhances Anti-Tumor Effect in Cancer Models
Presenter: Amy Lin, Ph.D.
Date and Time: Wednesday, Nov. 14, 1:35-1:45 p.m. CST
Summary:

Toca 521, a retroviral replicating vector (RRV) expressing a single-chain variable fragment (scFv) targeting PD-L1, 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.
Plasma levels of scFv PD-L1 were equivalent to negative control levels and hence very low compared to reported levels in humans given systemic anti-PD1/PD-L1 therapy.
These results warrant further development of Toca 521 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.
Details of the SNO presentation are as follows:

Presentation Type: Poster (Abstract: ATIM-26)
Title: Immunologic trends associated with recurrent high grade glioma patient outcomes in a Phase 1 clinical trial of Toca 511 and Toca FC
Presenter: William Accomando, Ph.D.
Date and Time: Saturday, Nov. 17, 5:00-7:00 p.m. CST
Summary:

Human immune monitoring results from a Phase 1 clinical trial of Toca 511 & Toca FC support an immune-related mechanism of action for the regimen.
Preliminary analyses identified immunologic biomarkers that can potentially predict, with high sensitivity and selectivity, patient outcomes following treatment with Toca 511 & Toca FC.
These results indicate the value of evaluating potential biomarkers of patient outcomes in the ongoing randomized Toca 5 Phase 3 trial in patients with recurrent HGG.
About Toca 511 & Toca FC
Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprising an investigational biologic, Toca 511, and an investigational small molecule, Toca FC. Toca 511 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, only infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered 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 resulting in anti-cancer immune activation and subsequent tumor killing.

On November 6, 2018 Jnana Therapeutics reported that Joanne Kotz, Ph.D., a company co-founder, has been named Chief Executive Officer (Press release, Jnana Therapeutics, NOV 6, 2018, View Source [SID1234530929]). Dr. Kotz has served as Jnana’s president since December 2017.

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"Joanne has demonstrated exceptional leadership in guiding the company through its early stages of growth, and we believe she is the ideal person to bring Jnana through its next phase and execute on the company’s vision to unlock SLC transporters as a therapeutic class," said Dr. Amir Nashat, a member of Jnana’s Board of Directors and a managing partner at Polaris Partners. Dr. Nashat also served previously as the company’s interim Chief Executive Officer. "She has built a talented team and a collaborative, empowering culture, while also leading important work to validate Jnana’s proprietary drug discovery platform. Together these position Jnana for even greater success going forward."

Dr. Kotz has broad life sciences experience in research, business, and operations. She helped formulate the initial scientific strategy for Jnana and has led the business and operations of the company since its launch in January 2017. Prior to founding Jnana, she held a leadership position in the F-Prime Biomedical Research Initiative, whose mission is to advance the discovery of novel therapeutic agents for the treatment of neurodegenerative disease by funding early-stage research in academia and biotech companies. Prior to F-Prime, Dr. Kotz was a director at the Broad Institute, where she played a leadership role in creating and guiding partnerships to advance drug discovery efforts, including the Broad-Bayer collaboration in oncology that resulted in the identification of multiple drug candidates. She received a Ph.D. in chemistry from the University of California, Berkeley and conducted postdoctoral research at the Necker Children’s Hospital in Paris and at Genentech.

"I am very proud of what the Jnana team has accomplished to date," said Dr. Kotz. "We have made significant progress in advancing our technology platform, which is the first to explicitly target the SLC family of metabolite transporters, established a strategic collaboration with Neurocrine Biosciences to discover new medicines for central nervous system disorders, and recruited a strong team committed to making a positive impact on patients and their families. I am honored to have been chosen to serve as CEO, and look forward to guiding Jnana’s efforts to transform our knowledge of SLCs into new therapeutics for patients lacking viable treatment options."

On November 6, 2018 Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage biopharmaceutical company focused on addressing the large unmet need in transthyretin (TTR) amyloidosis (ATTR), reported its financial results for the quarter ended September 30, 2018 and provided an update on the Company’s recent achievements (Press release, Eidos Therapeutics, NOV 6, 2018, View Source [SID1234576272]).

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"We are working to advance the AG10 clinical development program," said Neil Kumar PhD, chief executive officer of Eidos. "The Phase 1 data demonstrated that AG10 was well tolerated at blood concentrations resulting in near-complete TTR stabilization in healthy volunteers. We are announcing the results from the Phase 2 study in ATTR cardiomyopathy patients at the 2018 American Heart Association Annual Scientific Sessions on November 10, 2018."

Recent Achievements and Upcoming Milestones

Eidos presented Phase 1 data at the 2018 Annual Scientific Meeting of the Heart Failure Society of America, demonstrating that AG10 was well tolerated and establishing clinical proof-of-concept in healthy adult volunteers.

The U.S Food & Drug Administration granted Orphan Drug Designation to AG10 for the treatment of ATTR.

The European Medicines Agency adopted a positive opinion for the designation of AG10 as an orphan medicinal product for the treatment of ATTR.

The Journal of Medicinal Chemistry published the design and preclinical characterization of AG10, demonstrating that AG10’s potentially superior stabilizing activity is driven by the unique ability to mimic the disease-protective T119M mutation and its selectivity for TTR.

The Phase 2 study of AG10 in ATTR cardiomyopathy (ATTR-CM) wild-type and mutant patients with symptomatic heart failure (NYHA Class II-III) concluded and eligible subjects entered a long term, open label extension study.

Eidos will present the Phase 2 data for AG10 in ATTR-CM at the Annual Scientific Sessions of the American Heart Association (AHA) in a late-breaking Featured Science oral presentation on November 10, 2018 at 10am EST. Eidos will also host a conference call and webcast on November 12, 2018 at 8am EST to discuss the results of the Phase 2 trial. Details for the conference call can be found at www.eidostx.com.
Financial Results for the Third Quarter 2018

Cash and cash equivalents totaled $166.6 million at September 30, 2018 compared with $5.5 million at December 31, 2017.

Research and development expenses were $7.9 million for the third quarter of 2018, compared to $2.3 million for the same period of 2017, an increase of $5.6 million. The increase was primarily due to increased expenses for contract consultants, contract manufacturing and other activities for AG10 clinical trials and increases in headcount and related salaries and expenses.

General and administrative expenses were $2.6 million for the third quarter of 2018 compared to $0.5 million for the same period in 2017, an increase of $2.1 million. The increase was primarily due to increased salaries and employee-related expenses and increases in professional fees and services in connection with becoming a public company.

Net loss for the quarter ended September 30, 2018 was $10.2 million or $0.29 per common share, compared to a net loss of $2.8 million or $0.74 per common share for the same period in 2017.

About AG10
AG10 is an orally administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 has completed a Phase 2 clinical trial in patients with ATTR cardiomyopathy and symptomatic heart failure. Results from this trial will be presented on November 10, 2018 at the American Heart Association’s Annual Scientific Sessions.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a "rescue mutation" because it has been shown to prevent ATTR in individuals also carrying a pathogenic, or disease-causing, mutation in their other copy of the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the "super-stabilizing" properties of this rescue mutation that have been well described.

With a pedigree stretching back almost a quarter of a century, BIO-Europe is not only Europe’s largest partnering conference but, according to a recent analysis by 1stOncology™ (also covering BIO International and BIO Asia), it is also one of the world’s richest displays of cancer drug development companies under one roof! With over 600 oncology companies from more than thirty different countries present at BIO-Europe 2018, this is truly a global event. Many of these have also just presented their latest scientific/clinical advancements at the freshly completed European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) (ESMO 2018) congress. Now coming together at the BIO-Europe 2018 meeting they represent more than 5,500 cancer drugs, from discovery to marketed, and are responsible for more than 40% of the world’s current output in cancer R&D, see pipeline breakdown below.

These new cancer drug technologies are being developed from a wide array of organizations, from centuries old universities such as Jagiellonian University (Poland) founded in 1364, to startup companies like Cedilla Therapeutics (USA) and Epigene Therapeutics (Canada), both founded in 2018. Regardless of age they are all coming together at BIO-Europe 2018 to engage with global life science partners.

The global nature of this meeting is revealed when looking at the top nations with cancer drug developing companies present at this year BIO-Europe. It is no surprise to see the United States in first place with 190 companies, UK in second with 48 and Japan is in third place with 41 companies. Please see below table for the top ten nations at BIO-Europe 2018.

In today’s fast moving climate where a company can go from an idea to a public company in a blink of an eye, roughly one third of the 600 cancer companies are publicly traded at various stock exchange markets around the world. No less than seven of these have gone through their initial public offering in 2018 alone, namely ARMO BioSciences, ASLAN Pharmaceuticals, Autolus, BeiGene, Forty Seven, MorphoSys and Sutro Biopharma.

The number of cancer startups, founded in the last five years, present at the BIO-Europe meetings amount to almost fifty, see table below for breakdown per year.

The sizeable cancer pipeline of more than five and a half thousand drugs represented at BIO-Europe is a based on a very diverse selection of technologies and discoveries in cancer biology. Almost one third of these are Immune-Oncology (I-O) drugs including Immune Checkpoint drugs, Cancer vaccines, Bispecific immunomodulators, CAR/TCR therapies and Oncolytic virotherapies, see breakdown by type of I-O drugs below.

In the spotlight of this year’s Nobel Prize in Physiology or Medicine, companies at BIO-Europe feature nearly 300 different immune checkpoint drugs. Other hot progress areas in cancer therapeutics include DNA Damage Response (DDR) drugs, epigenetic therapies, Protein Kinase Inhibitors (PKIs) and Antibody-Dug Conjugates (ADCs).

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