On November 9, 2019 Bolt Biotherapeutics, Inc., a private biotechnology company focused on using its Immune-Stimulating Antibody Conjugate (ISAC) platform technology to unleash the power of the immune system to treat cancer, reported new preclinical data demonstrating profound antitumor efficacy for its lead HER2 ISAC therapeutic program, when administered as a monotherapy, resulting in the complete eradication of large tumors (Press release, Bolt Biotherapeutics, NOV 9, 2019, View Source [SID1234550814]). The poster presentation entitled "HER2-targeting TLR7/8 immune-stimulating antibody conjugates elicit robust myeloid activation and anti-tumor immune responses in a TLR- and FcR- dependent manner" was presented at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in National Harbor, Maryland.

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"These exciting preclinical data provide a strong rationale for moving our ISAC cancer immunotherapy platform into clinical testing and I’m pleased to report that we expect to initiate our first clinical trial for our HER2 monotherapy in early 2020," stated Randall Schatzman, Ph.D., chief executive officer of Bolt. "While much progress has been made in cancer immunotherapy, there still remains a significant need for single-agent therapies that can impact well-established tumors and provide durable efficacy in tumors that are refractory to standard of care therapies. Our proprietary Boltbody ISACs embody all of these components, and we are highly optimistic about the future of this platform to impact cancer."

"We believe such profound antitumor activity is unprecedented, including complete tumor eradication in large tumors, with an immunotherapeutic systemically administered as a monotherapy," stated David Dornan, Ph.D., senior vice president of research at Bolt Biotherapeutics. "Our data define the details of the mechanism of action by which our Boltbody technology is able to eliminate these hard to treat solid tumors, while generating immunological memory to suppress recurrence."

In the series of studies presented at SITC (Free SITC Whitepaper), key preclinical data show:

ISAC antitumor activity requires tumor target expression, interaction with Fc gamma receptors on immune cells, and TLR7/8 engagement
Single-agent anti-HER2 ISAC treatment led to in vivo tumor regression and clearance in models with large tumor burden and are resistant to anti-HER2 naked antibody treatment
Immunological memory was achieved as measured by protection from subsequent tumor growth. In syngeneic tumor models in which anti-HER2 ISAC treatment led to tumor clearance, hosts that were re-challenged with the parental tumor cell line lacking HER2 antigen expression were resistant to tumor growth. This protection was mediated by T cells as evidenced by the ability to re-establish tumors after the deletion of CD4 and CD8 T cells.
About Bolt Biotherapeutics’ Immune-Stimulating Antibody Conjugate (ISAC) Platform Technology
The Boltbody platform consists of Immune-Stimulating Antibody Conjugates (ISAC) that harness the ability of innate immune agonists to convert cold tumors into immunologically hot tumors thereby illuminating tumors to the immune system and allowing them to be invaded by tumor killing cells. Boltbody ISACs have demonstrated the ability to eliminate tumors following systemic administration in preclinical models and have also led to the development of immunological memory, which is predicted to translate into more durable clinical responses for patients.

On November 9, 2019 Rakuten Medical, Inc. (RMI) a clinical-stage, global biotechnology company developing precision-targeted cancer therapies based on Rakuten Medical’s Illuminox, its proprietary, anti-cancer treatment platform, reported new preclinical data suggesting CD25 photoimmunotherapy (PIT) treatment, combined with an anti-PD1 therapy, may stimulate the immune system, and lead to a synergistic, anti-cancer activity in targeted tumors (Press release, Rakuten Medical, NOV 9, 2019, View Source [SID1234550813]).

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"These exciting data suggests that anti-CD25 photoimmunotherapy may alter the immune tumor environment and unlock the potential of combination immunotherapies for patients living with certain types of cancers for which there are few treatment options," said Miguel Garcia-Guzman, Ph.D., Vice Chairman and Chief Scientific Officer at Rakuten Medical. "We are committed to harnessing the full potential of the immune system through modulation of the cancer tumor environment, and these results support our clinical development program based on our anti-cancer treatment platform, Rakuten Medical’s Illuminox."

The preclinical data were showcased during a poster presentation during the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th annual meeting:

"Intratumoral depletion of regulatory T-cells using CD25 targeted photoimmunotherapy elicits anti-cancer immune activity and synergizes with PD1 checkpoint blockade in immunocompetent mouse models." (Abstract P774), presented by Jerry J. Fong, Cancer Biology and Pharmacology, Rakuten Medical.

The poster discusses intratumoral depletion of regulatory T-cells (Tregs), a significant source of immune suppression, with an anti-CD25-IR700 conjugate therapy using PIT. Anti-cancer activity and subsequent immune responses following anti-CD25-IR700 PIT treatment, administered alone or in combination with anti-PD1 treatment, were evaluated in immunocompetent mouse models. Key highlights from the studies include:

Rapid and significant reduction of intratumoral Tregs, eliciting significant anti-cancer activity
An increase of non-exhausted T-cells following a single treatment, suggesting systemic activation and intratumoral recruitment of new CD8 T-cells from the periphery
Significant enhancement of anti-cancer activity in vivo, as demonstrated by percentage of mice achieving complete responses (CRs) with combination treatment in comparison to animals receiving one treatment alone
Durable increase of intratumoral CD8 T-cell/Treg ratio
Enhanced systemic adaptive immune responses and induced abscopal anti-cancer effects in a CD8 T-cell dependent manner
Tumor-specific immune memory response as demonstrated by systemic tumor-antigen-specific cytotoxic lymphocytes expansion and prevention of new tumor growth in CR mice
Combination treatment enhanced systemic adaptive immune responses and induced abscopal anti-cancer effects in a CD8 T-cell dependent manner

On November 9, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported the presentation of data from the phase 1 dose-escalation monotherapy study of PRS-343, a 4-1BB/HER2 bispecific for the treatment of HER2-positive solid tumors, in a late-breaking oral presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. PRS-343 demonstrated single-agent anti-tumor activity, including partial responses, in heavily pre-treated patients across multiple HER2-positive tumors (Press release, Pieris Pharmaceuticals, NOV 9, 2019, View Source [SID1234550812]). Beyond demonstrating clinical benefit, PRS-343 showed a potent increase in CD8+ T cell numbers and proliferative index in the tumor microenvironment of responders, indicative of 4-1BB agonism on T cells. PRS-343 was safe and well tolerated at all doses and schedules tested.

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"The data presented today demonstrate PRS-343’s potential to make a meaningful difference for patients with tumors that are difficult to treat with currently-available therapies," said Geoffrey Y. Ku, medical oncologist at Memorial Sloan Kettering and the principal investigator for the PRS-343 monotherapy trial. "Beyond today’s presentation, I look forward to sharing some of these case studies at Pieris’ upcoming R&D day alongside emerging data from the study of PRS-343 in combination with atezolizumab."

The ongoing phase 1 first-in-human, open-label multicenter trial has enrolled 53 patients, including 19 patients with gastric cancer, 14 patients with breast cancer, 6 patients with gynecological cancers, and 14 patients with other tumor types. Eleven dose cohorts have been evaluated at a Q3W dosing schedule, with the 11th dose level (8 mg/kg) also being evaluated at a Q2W dosing schedule. Pre- and post- treatment biopsies were obtained from many of the patients. Trial objectives include evaluation of safety and tolerability, characterizing the pharmacokinetic profile, assessing pharmacodynamic and potential immunogenicity effects, and investigating clinical response.

As of the cut-off date of October 23, 2019, 18 patients were evaluable for a response at active dose levels, which began at cohort 9 (2.5 mg/kg).

At the 8 mg/kg Q2W dose level, one patient with stage 4 gastric adenocarcinoma and one patient with stage 4 gynecological carcinoma achieved confirmed partial responses; the remaining patients experienced stable disease, for an overall disease control rate of 100% in this cohort as best response.
Across the remaining active dose levels and schedules, an additional five patients experienced stable disease.
Biomarker data from post-treatment tumor biopsies in patients receiving active dose levels and showing clinical benefit reflected a pronounced increase in CD8+ T cell numbers.
As of the cutoff date, treatment duration across active dose levels is over 30 weeks.
Treatment-related adverse events (AEs) were primarily grade 1 and 2. The most common AEs were infusion related reactions and fatigue. No patients experienced a dose-limiting toxicity and a maximum tolerated dose has not been reached.
"In addition to being the first 4-1BB bispecific to enter the clinic, PRS-343 is our lead immuno-oncology asset, and we believe this dataset serves as early clinical validation of our 4-1BB-targeting immuno-oncology approach and the Anticalin bispecific platform," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "PRS-343 shows promising signs of efficacy linked to clear biomarker evidence of 4-1BB agonism, in addition to being safe and well tolerated. We look forward to concluding this escalation trial in the near term and initiating an expansion trial informed by the emerging data. We also look forward to initiating clinical development of our 4-1BB/PD-L1 bispecific, PRS-344, with Servier in the first half of next year."

On November 8, 2019 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported that the company will participate in the Stifel 2019 Healthcare Conference in November (Press release, Ascendis Pharma, NOV 8, 2019, View Source [SID1234551453]). Company executives will provide a business overview and update on the company’s pipeline programs.

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Details

Event Stifel 2019 Healthcare Conference
Location New York
Date Wednesday, November 20, 2019
Time 3:35 p.m. Eastern Time
A live webcast will be available in the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

On November 8, 2019 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), an immunology company developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the third quarter ended September 30, 2019 and is providing a business update (Press release, INmune Bio, NOV 8, 2019, View Source [SID1234551121]).

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Q3 2019 and Recent Highlights:

·Launched NeuLiv(TM) program for the treatment of NASH
.Presented data related to lead compound XPro1595 at the Society for Neuroscience’s annual meeting
·Announced a potential link between obesity and Alzheimer’s Disease at the Society for Neuroscience 49th annual meeting: Dr. Malú Tansey’s new data was highlighted at the corresponding press conference
·Announced USPTO Allowance of Key Patent Covering the Company’s DN-TNF Platform Technology for Treating Cancer
·Presented XPro1595 for Treating Neuroinflammation in Alzheimer’s Disease at 2019 Alzheimer’s Association International Conference (AAIC) Satellite Symposium
·Invited to present at industry meetings in the US and EU including Targeting Innate Immunity Congress, Inflammasome Therapeutics Summit and Markets&Markets Next Gen Immuno-Oncology Congress
"During the quarter we advanced our existing programs and expanded our pipeline of therapies reprograming the innate immune system to fight disease," stated RJ Tesi, M.D., Chief Executive Officer of INmune Bio. "For our existing pipeline, we are looking to the up-coming Phase II trials for INB03 and Phase 1 trials of INKmune(TM) in Cancer and XPro1595 for Alzheimer’s Disease. Also, we expanded our pipeline with the launch of our NeuLiv(TM) program for the treatment of NASH which we expect to start a Phase 2a trial next year. Looking ahead, the team at INmune Bio is focused on executing against our upcoming milestones to maximize shareholder value."

Upcoming Milestones:

·4Q19 – First patient enrolled XPro1595 Alzheimer’s Disease trial
·1H20 – Initiate INB03 Phase II cancer trial
·2H20 – Initiate NeuLiv(TM) Phase IIa trial in NASH
·2H20 – First patient enrolled INKmune(TM) cancer trial
·2H20 – Phase I data on Alzheimer’s Disease XPro1595 AD data
·Financial Results for the Third Quarter Ended September 30, 2019:
Net loss attributable to common stockholders for the third quarter ended September 30, 2019 was $3.1 million, compared to $1.5 million for the quarter ended September 30, 2018.

Research and development expense totaled approximately $1.2 million for the third quarter ended September 30, 2019, compared with approximately $0.7 million for the quarter ended September 30, 2018. Research and development expenses increased during the three months ended September 30, 2019 as a result of the further advancement of our drug platforms.

General and administrative expense was approximately $1.9 million in the quarter ended September 30, 2019, compared to approximately $0.9 million in the quarter ended September 30, 2018. The $1.0 million increase in general and administrative expense is largely due to costs associated with being a public company.

At September 30, 2019, the Company had cash and cash equivalents of approximately $7.4 million with no debt. During October 2019, the Company received $0.4 million of cash proceeds from Australia pursuant to a research and development tax credit.

As of November 8, 2019, the Company had 10.8 million common and 13.9 million fully diluted shares outstanding.