On November 8, 2019 Codiak BioSciences, Inc., a company pioneering exosomes as a new class of biologic medicines, reported new preclinical data for its lead engEx Platform therapeutic candidates, exoIL-12 and exoSTING (Press release, Codiak Biosciences, NOV 8, 2019, View Source [SID1234550825]). Results from multiple studies highlight the potential of Codiak’s precision engineered exosomes to direct pharmacological payloads to specific cells and achieve superior anti-tumor efficacy compared to conventional therapeutic approaches. These data are being presented this week at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in National Harbor, MD.

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"We believe our engEx Platform can fulfill the promise of exosomes, overcoming significant hurdles that have faced the therapeutic development of many promising pathways in the past. We are particularly excited to debut data for exoIL-12 that show how engineered exosomes can direct biological activity to the tumor, re-opening the door for drug development for this long attractive target," said Douglas E. Williams, Ph.D., President and Chief Executive Officer of Codiak. "We are focused on advancing our initial candidates, exoSTING and exoIL-12, through IND-enabling preclinical studies and plan to initiate proof-of-concept clinical trials for both programs in 2020."

Both exoIL-12 and exoSTING were developed via Codiak’s proprietary engEx Platform, which enables the company to design exosomes – nature’s intercellular messengers – with precisely engineered properties, to incorporate various types of biologically active molecules and to be directed to specific cell types and tissues.

exoIL-12 Presentation

exoIL-12 is a precision engineered exosome therapeutic candidate expressing active IL-12 on the surface of the exosome via Codiak’s novel protein scaffold, PTGFRN. The data from exoIL-12 in vivo preclinical studies highlight the unique potential of engEx exosomes to direct and retain IL-12 pharmacology at the tumor injection site, driving significantly greater anti-tumor activity than soluble recombinant IL-12 (rIL-12) and inducing antigen-specific systemic immunity.

Following intratumoral (IT) injection, exoIL-12 was ~100-fold more potent in tumor growth inhibition than rIL-12.
exoIL-12 showed 15-fold improved retention at the injection site and demonstrated four-fold prolonged and improved interferon gamma (IFNγ) production as compared to rIL-12.
exoIL-12 stimulated a dose-dependent reduction in tumor growth in primary IT injected and distal tumors.
Complete responses were observed in ~2/3 of mice treated with exoIL-12 compared to 0% in mice treated with an equivalent dose of rIL-12.
Re-challenge studies of complete responders from the exoIL-12 group showed no tumor regrowth, demonstrating immunologic memory T-cell activity.
Data from the poster titled Exosome surface display of IL-12 results in tumor-retained pharmacology with superior potency and limited systemic exposure compared to recombinant IL-12 (P785) will be presented on Friday, November 8th.

exoSTING Presentation

exoSTING is a precision engineered exosome therapeutic candidate overexpressing the protein PTGFRN, which promotes uptake selectively into antigen presenting cells (APCs) and is loaded with a proprietary synthetic cyclic dinucleotide to stimulate the STING (stimulator of interferon genes) pathway. Consistent with Codiak’s previously reported data, these preclinical studies emphasize exoSTING’s selective activation of the STING pathway in tumor-resident APCs, lack of immune cell ablation in the tumor, generation of antigen-specific T-cell responses and establishment of immunological memory. In addition, preliminary ex vivo studies with human tumor samples confirm the unique biology of exoSTING.

In vitro, exoSTING demonstrated a 100-fold increase in potency of macrophage activation compared to a free STING agonist, as well as selective activation of M2 immunosuppressive macrophages that drive immune responses against tumors.
In vivo in a checkpoint refractory abscopal tumor model, exoSTING preserved the viability of T-cells and APCs, reduced nonspecific tissue damage and recruited T-cells into tumors, whereas free STING agonist resulted in immune cell ablation.
exoSTING increased T-cell infiltration in the tumor microenvironment by four-fold compared to free STING agonist.
exoSTING inhibited tumor growth in both injected and non-injected contralateral tumors, suggesting systemic anti-tumor response.
exoSTING preserved immunological memory response as demonstrated by tumor re-challenge studies. Complete responders showed no tumor regrowth.
Initial ex vivo analyses demonstrated that exoSTING activates the STING pathway in human tumor tissues after IT administration.
Data from the poster titled Selective activation of antigen presenting cells by exoSTING enhances tumor antigen-specific immune response (P666) will be presented on Saturday, November 9th.

About exoIL-12

exoIL-12 is a precision engineered exosome therapeutic candidate expressing IL-12 on the surface of the exosome by molecular fusion to Codiak’s proprietary scaffold protein, PTGRFN. Developed via Codiak’s proprietary engEx Platform, exoIL-12 is designed to target T-cells and Natural Killer cells and induce systemic anti-tumor immunity. Data from Codiak’s preclinical studies suggest that when administered intratumorally, exoIL-12 is retained in the tumor microenvironment, thus resulting in potent anti-tumor immunity and an improved toxicity profile compared to recombinant IL-12. Codiak plans to begin its first Phase 1/2 clinical trial for exoIL-12 initially in patients with Cutaneous T-Cell Lymphoma in the second half of 2020.

About exoSTING

exoSTING is a precision engineered exosome therapeutic candidate that is surface engineered with the protein PTGFRN, and luminally loaded with a proprietary, potent small molecule STING (stimulator of interferon genes) agonist. Developed via Codiak’s proprietary engEx Platform, exoSTING is designed to selectively activate the STING pathway in tumor-resident antigen presenting cells (APCs) and attract and expand immune effector cells in the tumor microenvironment. Data from Codiak’s preclinical studies suggest that when administered intratumorally, exoSTING generates potent, targeted and sustained anti-tumor immunity in numerous preclinical models without systemic elevation of inflammatory cytokines. Codiak plans to initiate its first Phase 1/2 clinical trial for exoSTING in patients with a select group of solid tumors in the first half of 2020.

On November 8, 2019 IMV Inc. (TSX: IMV; NASDAQ: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies, reported its financial and operational results for the third quarter ended September 30, 2019 (Press release, IMV, NOV 8, 2019, View Source [SID1234550824]).

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"In the third quarter and more recently, we have continued to advance our pipeline forward. In particular, we were pleased to report preliminary data at ESMO (Free ESMO Whitepaper) that highlighted DPX-Survivac’s potential to treat numerous solid tumors beyond our lead indications. Additionally, we launched a collaboration with The Wistar Institute, which applies our DPX-based targeted T cell therapy to the common BRAF mutation – another prevalent target across a range of tumor types," said Frederic Ors, President and Chief Executive Officer of IMV. "Importantly, these recent achievements reinforce our belief in the breadth of our platform and its ability to produce cancer-targeted T cells that elicit a more rapid, robust and sustained immune response. Looking ahead, we await key readouts from DPX-Survivac, including updated data from the Phase 2 study in r/r DLBCL at ASH (Free ASH Whitepaper) on December 8, 2019 and topline interim data from our Phase 1b/2 study in advanced ovarian cancer during the first quarter of next year. As we close in on proof-of-concept in these indications of high unmet medical need, we continue to believe both represent fast-to-market opportunities and are therefore preparing to launch potential pivotal studies in 2020."

DPX-Survivac Clinical Program Updates

Phase 1b/2 DeCidE1 Study in Advanced Recurrent Ovarian Cancer

DeCidE1, the Company’s Phase 1b/2 open-label study evaluating the safety and efficacy of DPX-Survivac and intermittent low-dose cyclophosphamide (CPA), with and without Epacadostat, in advanced recurrent ovarian cancer, is ongoing without Epacadostat.

Enrollment is complete in this study and the Company intends to report topline interim data during Q1 2020.

Phase 2 SPiReL Study in Recurrent/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

SPiReL, an investigator-sponsored Phase 2 study evaluating DPX-Survivac and ildCPA in combination with Keytruda (pembrolizumab) in recurrent/refractory DLBCL is ongoing.

Neil Berinstein, M.D. FRCPC, ABIM, Hematologist at the Sunnybrook Health Science Centre will deliver a poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 8, 2019, including updated data from the study.

As of November 7, 2019, 17 patients have been enrolled across five different clinical sites in Canada. The non-randomized, open label study is expected to enroll 25 evaluable participants in Canada. Additional patients are being screened and topline data are expected from this study in the first half of 2020.

Phase 2 Basket Trial in Multiple Advanced and Metastatic Solid Tumors

In September 2019, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress in Barcelona, Spain, IMV presented preliminary results from its ongoing Phase 2 basket trial, evaluating DPX-Survivac and intermittent low-dose CPA in combination with pembrolizumab in patients with advanced and metastatic solid tumors.

As of the data cut-off, 23 patients had been treated across all five patient cohorts with bladder, hepatocellular carcinoma (liver), ovarian, and non-small cell lung (NSCLC) cancers, as well as tumors shown to be positive for the microsatellite instability high (MSI-H) biomarker. Preliminary results from the first on-study scan showed signs of clinical activity with tumor regressions observed in patients with ovarian, non-small cell lung and bladder cancers, including two partial responses, and a consistent safety profile including no grade 3-4 immune-related adverse events. The preliminary data are available here.

As of November 7, 2019, 13 clinical sites have been activated, 64 patients are screened, and 31 patients have been enrolled. The study continues to enroll patients towards a total enrollment target of 184 patients across all five cohorts. The Company expects to report topline data in the first half of 2020.

Operational Highlights:

Appointment of Joanne Schindler, M.D., D.V.M. as Chief Medical Officer. Dr. Schindler joined IMV in November 2019 with over 15 years of experience in the biopharmaceutical industry, primarily in early-stage oncology drug development. Most recently, she served as Vice President, Clinical Development and Executive Medical Director at H3 Biomedicine, where she oversaw clinical development strategy and execution.

Research collaboration with Meenhard Herlyn, D.V.M., D.Sc. and The Wistar Institute to develop a targeted T cell therapy against the BRAF mutation. Under this collaboration, IMV will optimize the DPX formulation with Wistar-identified peptides targeting BRAF, one of the most frequently identified cancer-causing mutations in melanoma and various other cancers, including non-Hodgkin’s lymphoma, colorectal cancer, thyroid cancer, and non-small cell lung and ovarian carcinomas. IMV holds an exclusive option to in-license intellectual property related to the program.

Upcoming Milestones:

Over the course of upcoming quarters, the Company expects to deliver the following milestones:

Updated results from Phase 2 SPiReL trial (DLBCL) at ASH (Free ASH Whitepaper) 2019 on December 8, 2019
Topline results from Phase 1b/2 DeCidE1 trial (ovarian) during Q1 2020
Topline results from Phase 2 SPiReL trial (DLBCL) in 1H 2020
Topline results from Phase 2 basket trial (multiple tumors) of DPX-Survivac in 1H 2020.
Overview of Q3 2019 Financial Results (In Canadian dollars)

At September 30, 2019, the Corporation had cash and cash equivalents of $21.4 million and working capital of $21.2 million, compared with $14.9 million and $12.2 million, respectively at December 31, 2018. Management believes that the Corporation’s cash resources of $21.4 million and its additional potential cash resources of $2.3 million will be sufficient to fund operations for the next twelve months.

For the nine-month period ended September 30, 2019, IMV’s cash burn rate (defined as net loss and comprehensive loss adjusted for charges to operations not involving cash as described in the statement of cash flows) was $17.4 million. Based on the current business plan, the Corporation forecasts the quarterly cash burn rate to be between $5 million and $6 million for the fourth quarter of 2019.

The net loss and comprehensive loss of $7.9 million ($0.16 per share) for the three-month period ended September 30, 2019, was $1.9M higher than the net loss and comprehensive loss for three-month period ended September 30, 2018. This relates mainly to an increase in R&D expenses of $1.8M related to the basket trial and pre-clinical preparation for a phase I trial with DPX-SurMAGE.

For the nine-month period ended September 30 2019, the net loss and comprehensive loss of $18.9M ($0.38 per share) was $4.6M higher than the net loss and comprehensive loss for the nine-month period ended September 30 2018. This relates mainly to an increase in R&D expenses of $5.1M related to the basket trial and the preparation for a phase I trial with DPX-SurMAGE.

As of November 7, 2019, the number of issued and outstanding common shares was 50,630,875 and a total of 1,932,080 stock options, warrants, and deferred share units were outstanding.

The Corporation’s unaudited interim condensed consolidated results of operations, financial condition and cash flows for the three and nine-months ended September 30, 2019 and the related management’s discussion and analysis (MD&A) are available on SEDAR at www.sedar.com and on EDGAR at www.sec.gov/edgar.

Conference Call and Webcast Information

Management will host conference call and webcast this morning, November 8, 2019 at 8:00 am ET. Financial analysts are invited to join the conference call by dialing (844) 461-9932 (U.S. and Canada) or (636) 812-6632 (international) using the conference ID# 6590953. Other interested parties will be able to access the live audio webcast at this link: View Source

On November 8, 2019 F-star Therapeutics ("F-star"), a clinical-stage biopharmaceutical company focused on transforming the lives of patients with cancer through the development of innovative tetravalent bispecific (mAb2) antibodies, reported that new preclinical data on FS120, a mAb² product candidate targeting OX40 and CD137, will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019 Annual Meeting in National Harbor, Maryland, United States, being held from 06 – 10 November 2019 (Press release, f-star, NOV 8, 2019, View Source [SID1234550823]).

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FS120 is a potentially best-in-class dual agonist that has the potential to overcome cancer resistance by simultaneously engaging OX40 (CD134, TNFRSF4) and CD137 (4-1BB), two receptors found on the surface of tumor-infiltrating lymphocytes. Targeting this class of receptors using bispecific tetravalent binding mobilizes multiple arms of the immune system, which research shows is essential for eliminating tumors. F-star’s preclinical data demonstrated that FS120’s crosslink-dependent approach has the potential to provide therapeutic benefit, for example in combination with checkpoint antagonists, and reverse T cell exhaustion in immunosuppressive tumor environments.

Neil Brewis, CSO of F-star said "Recent clinical trials involving agonist molecules have reported that a meaningful anti-cancer response is often associated with liver toxicity, in line with preclinical observations. F-star’s OX40/CD137 mAb² antibody is showing preclinical evidence that an effective tumor-killing response can be decoupled from liver inflammation. We look forward to progressing FS120 into the clinic as we aim to potentially improve treatment outcomes for patients with difficult-to-treat cancers."

Some CD137 agonist antibodies have been shown to induce adverse effects either in clinical or in preclinical studies as they constitutively activate T cells and thus release cytotoxic immunity outside of the tumor. In contrast, FS120 is designed to mitigate off-target toxicity through conditional, crosslink-dependent activation upon binding to both OX40 and CD137, which are predominantly present on T cells in the tumor microenvironment.

F-star expects to submit an IND application for FS120 during the fourth quarter of 2019.

Details of the poster are below:

Crosslink-independent CD137 agonism is associated with liver inflammation

Abstract poster number: P775
Poster hall location: Prince George AB
Poster hall hours: 07:00 to 20:00 on Friday 08 November 2019
Poster presentation hours: 12:30 to 14:00 and 18:30 to 20:00 on Friday 08 November 2019

On November 8, 2019 Omeros Corporation (Nasdaq: OMER) reported an upcoming presentation entitled "Phosphatidylserine suppresses T cells through GPR174, and co-inhibition of adenosine receptors and GPR174 synergistically enhances T-cell responses" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Conference on Tumor Immunology and Immunotherapy, which is being held November 17 – 20, 2019 in Boston, Massachusetts (Press release, Omeros, NOV 8, 2019, View Source [SID1234550804]).

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Marc Gavin, Ph.D., Omeros’ Director of Immunology, will present the company’s new findings that phosphatidylserine, a product of cell stress and death that is abundant in the tumor microenvironment (TME), directly stimulates GPR174 and attenuates T-cell responses.

Adenosine, another product of the TME, is well known to stimulate the same suppressive pathways through different GPCRs, and inhibition of both pathways—with adenosine receptor antagonists and with Omeros’ novel GPR174 inhibitors—results in maximal enhancement of T-cell function, which should translate to effective resistance to the immunosuppressive nature of the TME.

The data will also feature new findings from animal tumor models, which validate GPR174 inhibition for cancer immunotherapy.

Designated B45, the poster will be presented in Poster Session B on Tuesday, November 19, 4:30 p.m. to 7:00 p.m.

On November 8, 2019 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, NOV 8, 2019, View Source [SID1234550803]).

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"As we approach the end of 2019, BioSpecifics is entering its next phase of growth as we seek to unlock new opportunities to generate value for all our stakeholders," said J. Kevin Buchi, chief executive officer of BioSpecifics. "We are excited by the prospect of adding a third marketed XIAFLEX indication to our commercial pipeline with the potential FDA approval of CCH for the treatment of cellulite. BioSpecifics today is a profitable, commercial-stage organization with a diverse development pipeline focused on CCH and the team is eager to build on this trajectory."

Third Quarter 2019 Financial Results

BioSpecifics reported net income of $6.3 million for the third quarter ended September 30, 2019, or $0.86 per basic share and $0.85 per share on a fully diluted basis, compared to net income of $5.0 million, or $0.69 per basic share and $0.69 per share on a fully diluted basis, for the same period in 2018.

Total revenue for the third quarter ended September 30, 2019 was $9.4 million, compared to $8.2 million for the same period in 2018. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Peyronie’s Disease and Dupuytren’s Contracture.

Research and development expenses for the third quarter ended September 30, 2019 were $0.1 million compared to $0.2 million for the same period in 2018.

General and administrative expenses for the third quarter ended September 30, 2019 were $2.0 million, compared to $2.2 million for the same period in 2018.

Provision for income taxes for the third quarter ended September 30, 2019 were $1.6 million, compared to $1.1 million for the same period in 2018.

As of September 30, 2019, BioSpecifics had cash and cash equivalents and investments of $98.4 million, compared to $82.0 million as of December 31, 2018.

As of September 30, 2019, BioSpecifics had 7,343,119 million shares of common stock outstanding.

Commercial & Pipeline Highlights

BioSpecifics’ Royalty Revenues from the XIAFLEX Commercial Franchise Grew by 15% Year-Over-Year for the Third Quarter of 2019: XIAFLEX revenue growth was primarily attributable to royalties associated with higher net sales of XIAFLEX in Dupuytren’s Contracture and Peyronie’s Disease.
BLA filing for CCH for Treatment of Cellulite Submitted in September 2019: On September 6, 2019, Endo announced that it filed its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite.
New XIAFLEX Data Presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America: In October 2019, Endo announced that five new studies of XIAFLEX for the treatment of Peyronie’s Disease were presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America (SMSNA) which continue to support further patient and physician education.
New Data Presented on MRI Evaluation of Cellulite at the American Society for Dermatologic Surgery Annual Meeting: In October 2019, Endo announced the presentation of a case study comparison of the use of a standing MRI versus a prone MRI for the evaluation of cellulite. The comparison results showed a <10° difference between the angle of collagen septae orientation relative to the dermis in the standing and prone MRI. Septae orientation in the prone position was not perpendicular to the skin.
Corporate Highlights

Appointment of New Chief Executive Officer: In October 2019, BioSpecifics announced the appointment of J. Kevin Buchi as Chief Executive Officer, bringing the company diversified leadership experience, coupled with a strong life sciences background.
Appointment of Jennifer Chao as Chairman of Board of Directors: In October 2019, BioSpecifics announced the appointment of Jennifer Chao as chairman of the board of directors, who previously served as an independent director of the board since 2015.