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 Oncoceutics, Inc. reported that it will host an investigator meeting and that initial efficacy data of its lead compound ONC201 in patients with H3 K27M-mutant gliomas will be presented at the 2018 Annual Meeting of the Society for Neuro-Oncology in New Orleans, on November 15th to November 18th (Press release, Oncoceutics, NOV 6, 2018, View Source [SID1234558365]).

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The company, along with scientific and medical collaborators, will give 10 presentations that describe several aspects of ONC201, including clinical efficacy and safety, mediated via highly specific interaction with dopamine receptor 2 (DRD2), unique molecular characteristics, and novel insights on dopamine receptor dysregulation in glioma. Moreover, presentations will include the preclinical effectiveness and mechanism of action of ONC206, another molecule from this newly established class of compounds, called imipridones. One of the highlights of the event will be an oral presentation entitled, "Integrated clinical experience with ONC201 in previously-treated H3 K27M-mutant glioma patients," which scored highly in the review process of the SNO conference and will be one of three selected abstracts to be presented by a discussant separate from the oral presentation. The data set that will be disclosed will focus on radiographic and clinical responses in H3 K27M-mutant glioma patients from several clinical programs with ONC201.

"The reported findings are very meaningful for the medical community, and we look forward to sharing our experience with ONC201 in this first group of patients," said Yazmin Odia, MD, Lead Physician of Medical Neuro-Oncology, Miami Cancer Institute. "Patients with H3 K27M-mutant gliomas have a dismal prognosis, and there is currently no known effective drug for these patients."

Additionally, Oncoceutics will hold an investigator meeting for physicians involved in ongoing and future clinical trials with ONC201.

"We are conducting a number of clinical trials to translate these compelling findings into robust clinical data sets," said Wolfgang Oster, MD, PhD, and CEO of Oncoceutics. "We are excited about the progress in our development programs and hope that our findings will make a difference for patients with unmet medical need. We are grateful to the Society for Neuro-Oncology and its members for their interest and collaboration."

More Information: Imipridone-related SNO 2018 Abstracts

ONC201 glioma clinical experience (3 abstracts)

ACTR-34 (Talk): Integrated clinical experience with ONC201 in previously-treated H3 K27M-mutant glioma patients, Chi et al, Sunday November 18th, 8:50-9:00am
ACTR-33 (Talk): Tumor Tissue Penetration and Pharmacodynamics of ONC201 in Adult Recurrent Glioblastoma Patients, Arrillaga-Romany et al, Friday November 16th, 2:40-2:45pm
PDCT-06 (Talk): Phase I clinical trial of ONC201 in pediatric H3 K27M-mutant glioma or newly diagnosed DIPG, Gardner et al, Friday November 16th, 8:22-8:26pm
DRD2 dysregulation in glioma and ONC201 preclinical efficacy (4 abstracts)

DRES-10 (Talk): DRD5 is a modulator of glioma susceptibility to DRD2 antagonism by ONC201, Prabhu et al, Sunday November 18th, 11:25-11:30am
EXTH-26 (Poster): Molecular determinant of clinical response to ONC201, an inhibitor of dopamine receptor 2 (DRD2) signaling, in glioblastoma patients, Li et al, Saturday November 17th
DDIS-12 (Poster): ONC201: The first selective, non-competitive DRD2/3 antagonist for clinical neuro-oncology, Prabhu et al, Saturday November 17th
DDIS-16 (Poster): ONC201 in combination with radiation exhibits synergistic efficacy in high grade gliomas and other advanced cancers, Tarapore et al, Saturday November 17th
Utility of imipridone scaffold in neuro-oncology (3 abstracts)

EXTH-64 (Talk): Imipridones Cause Metabolic Reprogramming and Elicit Unique Vulnerabilities in Glioblastoma, Ishida et al, Friday November 16th, 8:06-8:10pm
EXTH-17 (Talk): Selective, non-competitive DRD2/3 antagonism by imipridone ONC206 is effective in tumors with dopamine receptor dysregulation, Prabhu et al, Friday November 16th, 8:02-8:06pm
EXTH-58 (Poster): ONC206, an imipridone family member, suppresses glioblastoma cells via blocking cancer stemness pathways, Jung et al, Saturday November 17th

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.