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Palleon Pharmaceuticals to Present Preclinical Data on EAGLE and HYDRA Platforms at the Society for Immunotherapy of Cancer’s (SITC) 34th Annual Meeting

On November 7, 2019 Palleon Pharmaceuticals, a leading biotech company developing drugs that target the Siglec-Sialoglycan axis to treat cancer, reported two poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting in National Harbor, MD (Press release, Palleon Pharmaceuticals, NOV 7, 2019, View Source [SID1234550659]).

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The first poster will show recent preclinical data on the company’s EAGLE platform, a first-in-class modality that targets the Siglec-Sialoglycan axis for treating cancer. The Siglec-Sialoglycan axis is very difficult to target by conventional approaches because of the structural diversity of sialylated tumor glycans and the redundancy of the Siglec receptors. The EAGLE platform makes targeting possible by enzymatically removing the critical common immunosuppressive moiety, terminal sialic acids, from tumor glycans regardless of their individual structures or Siglec receptor preferences. New data generated confirm anti-tumor activity and identify predictive biomarkers using EAGLE with engineered human sialidase in both in vitro and in vivo studies. Human sialidase poses fewer immunogenic concerns than bacterial sialidase, which was used in previous EAGLE proof-of-concept studies.

The second poster presentation describes Palleon’s HYDRA platform, an immunohistochemistry-based translational research technology, which allows the identification of a patient’s specific tumor cell surface glycan pattern, or tumor glyco-code. By overcoming the challenge posed by the significant structural heterogeneity of tumor surface glycans, HYDRA has the potential to help select patients who are most likely to respond to treatment.

"The Siglec-Sialoglycan axis, which was a previously underappreciated mechanism of immunosuppression, has recently emerged as a major tumor immune escape pathway," said Jim Broderick, M.D., Chief Executive Officer and Founder of Palleon. "The collective data thus far across our three distinct technology platforms – EAGLE, CONVERGENCE and HYDRA – continue to support that targeting this axis could play an important role in how we develop targeted treatments for cancer and identify the patients who are most likely to respond to treatment."

Full details of the presentations are below.

Poster title: Blockade of glyco-immune checkpoint using EAGLE to potentiate anticancer immunity
Abstract ID: P273
Date & time: Friday, Nov. 8, 12:30 pm – 2:00 pm ET and 6:30 pm – 8:00 pm ET
Location: Prince George’s Exhibition Halls AB

Poster title: HYDRA platform development to investigate Siglec-engaging tumor immunosuppressive glyco-codes
Abstract ID: P118
Date & time: Saturday, Nov. 9, 12:35 pm – 2:05 pm ET and 7:00 pm – 8:30 pm ET
Location: Prince George’s Exhibition Halls AB

AVEO Oncology and Biodesix Announce Initiation of the CyFi-2 Study, a Phase 2 Randomized Study of Ficlatuzumab in Combination with High-Dose Cytarabine vs. High-Dose Cytarabine Alone in Patients with Relapsed and Refractory AML

On November 7, 2019 AVEO Oncology (NASDAQ: AVEO) and Biodesix, Inc. reported the initiation of the CyFi-2 study, a randomized Phase 2 clinical study evaluating ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody product candidate, in combination with high-dose cytarabine vs. high-dose cytarabine alone in patients with relapsed and refractory acute myeloid leukemia (AML) (Press release, AVEO, NOV 7, 2019, View Source [SID1234550658]).

AVEO will sponsor the CyFi-2 study, which is expected to enroll up to a total of 60 patients with AML who failed induction chemotherapy or who achieved a complete response, but relapsed within one year. Patients will be randomized 1:1 to receive either ficlatuzumab in combination with cytarabine or cytarabine alone at the doses and schedules determined in the Phase 1b/2 single-arm CyFi-1 study. CyFi-2’s primary endpoint is complete response rate, with secondary endpoints including safety, disease-free survival, and overall survival. The CyFi-2 study is being conducted as part of the companies’ worldwide partnership to develop and commercialize ficlatuzumab. Under the terms of this agreement, AVEO and Biodesix equally share all development costs.

"Supported by promising Phase 1b/2 results from the CyFi-1 study, which showed a 50% complete response rate in primary refractory AML patients (n=18) and an acceptable tolerability profile, the ficlatuzumab-cytarabine combination has the potential to improve outcomes in this historically difficult to treat population," said Michael Bailey, president and chief executive officer of AVEO. "The CyFi-2 study, along with our ongoing randomized Phase 2 study in head and neck cancer, provide two opportunities to potentially pursue registration strategies for ficlatuzumab, assuming favorable study outcomes."

AVEO’s portion of the costs for both the CyFi-2 study and the recently initiated Phase 1b/2 hepatocellular carcinoma study of FOTIVDA (tivozanib) in combination with AstraZeneca’s IMFINZI (durvalumab) is included in the company’s current cash guidance.clatuzumab targets an important pathway which we believe has major clinical significance. We look forward to working with AVEO to enable ficlatuzumab to realize its full potential," said David Brunel, chief executive officer of Biodesix. "At Biodesix, we are committed to supporting biomarker development initiatives to further identify patients who may benefit most from promising therapies."

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in squamous cell carcinoma of the head and neck (SCCHN), metastatic pancreatic ductal cancer (PDAC), and acute myeloid leukemia (AML).

Exicure Reports Third Quarter 2019 Financial Results and Recent Developments

On November 7, 2019 Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the third quarter ended September 30, 2019 and provided an update on corporate progress (Press release, Exicure, NOV 7, 2019, View Source [SID1234550657]).

"Throughout the third quarter we have continued to execute against the strategic goals we set early in the year," said Dr. David Giljohann, Exicure’s chief executive officer. "Our immuno-oncology clinical trial is progressing as anticipated, while we continue to lay the groundwork for expanding our SNA platform in neurology. This scientific progress has been matched with expanded financial resources arising from our recent $63.3 million public offering and Nasdaq listing. In the coming quarters we look forward to building our organization to enhance our strategic execution," concluded Dr. Giljohann.

Third Quarter Corporate Progress and Recent Developments

AST-008 Phase 1b/2 clinical trial on track
As of November 1, 2019, we have dosed 13 patients in the first four cohorts of the Phase 1b stage of the clinical trial;
No treatment related adverse events or dose-limiting toxicity was observed;
Expect to disclose preliminary results from the Phase 1b in December of 2019.
Presented Positive Biomarker Results from Clinical Trial of XCUR17
Presentation at Oligonucleotide Therapeutics Society highlighted work by Dr. James Krueger at The Rockefeller University, in collaboration with Exicure, which examined the effects of XCUR17 on skin biopsies collected from patients in the Phase 1 trial;
Clinical findings in the Phase 1 trial matched reductions in keratin 16 protein and epidermal thickness observed in the patient biopsies;
Clinical findings were also shown to be correlated with downstream psoriasis-related inflammation markers including reductions in beta defensin 4A, interleukin 19, and interleukin 36A versus psoriatic skin at baseline.
Enhanced Financial Resources
Closed oversubscribed underwritten public offering of 31,625,000 shares of common stock at $2.00 per share for gross proceeds of $63.3 million with net proceeds of approximately of $58.8 million;
Up-listed from the OTC to the Nasdaq Stock Market.
Third Quarter 2019 Financial Results and Financial Guidance

Cash Position: Cash and cash equivalents were $70.4 million as of September 30, 2019 compared to $26.3 million as of December 31, 2018.

Research and Development (R&D) Expenses: Research and development expenses were $4.2 million for the quarter ended September 30, 2019, compared to $4.0 million for the quarter ended September 30, 2018. The increase was primarily due to higher employee-related expenses of $0.5 million and higher platform and discovery related expense of $0.1 million, partially offset by lower clinical development program expenses of $0.4 million.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.2 million for the quarter ended September 30, 2019, compared to $1.9 million for the quarter ended September 30, 2018. This increase is primarily due to costs related to the recruitment of two new board members and an open executive position, as well as incurred Nasdaq listing costs.

Net Loss: Net loss was $5.8 million for the quarter ended September 30, 2019, compared to net loss of $5.3 million for the quarter ended September 30, 2018. This increase was due to a $0.4 million reduction in Other income attributable to the (non-cash) fair value adjustment of our common stock warrant liability, the $0.3 million increase in G&A expenses described above, the $0.2 million increase in R&D expenses described above, partially offset by the addition of $0.5 million of revenue associated with the Dermelix transaction.

Cash Runway Guidance: We believe that, based on our current operating plans and estimates of expenses, as of the date of this press release, our existing cash and cash equivalents will be sufficient to meet our anticipated cash requirements in excess of twelve months.

Bicycle Therapeutics Reports Third Quarter 2019 Financial Results and Corporate Update

On November 7, 2019 Bicycle Therapeutics plc (NASDAQ:BCYC), a clinical-stage biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycles) technology, reported financial results for the third quarter ended September 30, 2019 and discussed recent corporate updates (Press release, Bicycle Therapeutics, NOV 7, 2019, View Source [SID1234550656]).

"The significant progress we have made in the past few months is instrumental to our goal of advancing our novel pipeline of Bicycle drug candidates through the clinic," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We’ve bolstered all three areas of the pipeline, namely, our wholly-owned Bicycle Toxin Conjugate and immuno-oncology programs, as well as our collaborations to develop new Bicycle-based therapies for indications beyond oncology. We have also continued to strengthen our leadership team both at the corporate and Board levels. These efforts further position the Company to evaluate the potential of Bicycles to enable possible first-in-class medicines that transform the treatment paradigm for people living with a broad range of serious diseases, in ways that existing modalities cannot."

Third Quarter 2019 and Recent Highlights

Presenting New Preclinical Data for Novel, Fully Synthetic Bicycle Tumor-targeted Immune Cell Agonists (TICAs) at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 2019 Annual Meeting. This week, Bicycle is presenting new data showing that its lead immuno-oncology candidate, BT7480, a TICA targeting Nectin-4 and agonizing CD137, rapidly penetrates tumors and effects powerful anti-tumor activity in preclinical models. In addition to data on BT7480, the Company will present preclinical research at SITC (Free SITC Whitepaper) on other emergent Bicycle TICA strategies. Details of the Company’s poster presentations on Saturday, November 9, 2019 can be found in the News section of bicycletherapeutics.com.
Enhanced Executive Team and Board of Directors. In October 2019, Bicycle announced the appointment of Nigel Crockett, Ph.D., as Chief Business Officer and of Veronica Jordan, Ph.D., to the Company’s Board of Directors.
Expanded Neuroscience Collaborations to Include Oxford University’s Oxford Drug Discovery Institute (ODDI). Bicycle announced that it will collaborate with ODDI to use Bicycle technology for the development of novel therapeutics for dementia. This expands upon the collaboration announced in May 2019 between Bicycle and the Dementia Discovery Fund (DDF), a specialized venture capital fund focused on discovering and developing novel therapies for dementia. If promising lead compounds are identified, Bicycle will have rights to the development of the resulting intellectual property and, with DDF, will have the option to jointly establish a new company to develop those compounds.
Presented New Translational Data for Lead Asset BT1718 and Preclinical Data for Other Bicycle Toxin Conjugates at AACR (Free AACR Whitepaper)-NCI-EORTC 2019. Bicycle presented new translational data for BT1718 and preclinical data for BT5528 and BT8009 during poster sessions at the 2019 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). The translational data for BT1718 characterize enrollment criteria for identifying expansion cohorts in the Phase IIa part of the ongoing Phase I/IIa trial.
Presented Updated Data from Phase I/IIa Trial Evaluating BT1718 in Patients with Advanced Solid Tumors at ESMO (Free ESMO Whitepaper) 2019 Annual Congress. Bicycle and Cancer Research UK presented updated data from the ongoing Phase I portion of the Phase I/IIa trial evaluating BT1718 in patients with advanced solid tumors. As of August 7, 2019, 54% of evaluable patients dosed had stable disease at the eight-week timepoint, including a patient who experienced a 45% reduction in a target lesion, and BT1718 appeared tolerable with once-weekly dosing.
Upcoming Investor Presentations

Bicycle will present at the following investor conferences in the fourth quarter of 2019:

Jefferies London Healthcare Conference on Wednesday, November 20, 2019 at 2:40 p.m. GMT (9:40 a.m. ET) in London, England
Piper Jaffray 31st Annual Healthcare Conference on Tuesday, December 3, 2019 at 3:30 p.m. ET in New York, NY
A live webcast of each presentation will be accessible in the Investors & Media section of Bicycle’s website at bicycletherapeutics.com. Archived replays of the webcasts will be available for 90 days following the presentation dates.

Financial Results

Cash was $96.0 million as of September 30, 2019, compared with $63.4 million as of December 31, 2018. The September 30, 2019 cash balance excludes a UK research and development incentive payment of approximately $6.0 million received in October 2019.
Research and development expenses were $6.1 million for the three months ended September 30, 2019, compared to $5.6 million for the three months ended September 30, 2018. The increase of $0.4 million is primarily due to $0.3 million of incremental non-cash share-based compensation expense, as well as other personnel related costs.
General and administrative expenses were $4.8 million for the three months ended September 30, 2019, compared to $2.3 million for the three months ended September 30, 2018. The increase of $2.5 million is primarily due to a $0.9 million unfavorable effect of foreign exchange rates, $0.7 million in personnel related costs, including $0.3 million of incremental non-cash share-based compensation expense, as well as increased professional fees related to operations as a public company.
Net loss was $9.5 million, or $(0.53) basic and diluted net loss per share, for the three months ended September 30, 2019, compared to net loss of $7.7 million, or $(17.73) basic and diluted net loss per share, for the quarter ended September 30, 2018.

Triumvira Immunologics Announces Fast Track Designation for Its First TAC T-Cell Therapeutic Product Candidate: TAC01-CD19

On November 7, 2019 Triumvira Immunologics, Inc. (Triumvira), a private, clinical-stage biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, reported that the U.S. Food & Drug Administration (FDA) has granted Fast Track designation for its novel T-cell therapy product TAC01-CD19 in patients with relapsed or refractory Diffuse Large B-Cell Lymphoma (DLBCL) after at least 2 prior systemic therapies (Press release, Triumvira Immunologics, NOV 7, 2019, View Source [SID1234550655]). A Phase 1/2 study (TACTIC-19) conducted in patients with CD19-positive B-cell malignancies, including DLBCL, is expected to be initiated by the end of 2019.

"FDA’s decision to grant Fast Track designation to TAC01-CD19 is an important recognition of both Triumvira’s differentiated cell therapy technology and the critical need to develop new therapies to address the unmet medical need in the treatment of B-cell lymphomas," commented Paul Lammers, MD, MSc., President and CEO of Triumvira. "With our innovative TAC technology, we hope to significantly improve upon the limitations of existing cell therapies, including the risk of cytokine release syndrome (CRS) and neurotoxicity, which would allow us to expand the number of patients eligible to receive this type of treatment."

TAC01-CD19 will be tested at four leading lymphoma clinical study centers in the U.S. and Canada. Based on its preclinical profile, TAC01-CD19 has the potential to represent a significant advancement in T-cell therapy.

About TAC01-CD19

Despite transformational efficacy with existing approved Chimeric Antigen Receptor T-Cells (CAR-T), a significant unmet need remains due to substantial CAR-T toxicities and limited tumor types where CAR-T is effective. Triumvira is developing a proprietary T-Cell Antigen Coupler (TAC) receptor which is structurally and biologically distinct from CAR-T. The first of our pipeline product candidates, TAC01-CD19 is a novel T-cell therapy product targeting CD19, a validated target in lymphomas and leukemias. The product comprises patient-derived T-cells that have been genetically engineered to express the CD19 T-cell Antigen Coupler (TAC). Preclinical data suggest that TAC01-CD19 has the potential for being highly efficacious with minimal side effects in hematological malignancies.

About CD19 and Diffuse Large B-Cell Lymphoma (DLBCL)

CD19 is a B-cell marker and is expressed on the surface of B-cell malignancies such as Diffuse Large B-Cell Lymphoma (DLBCL). DLBCL is a subtype of Non-Hodgkin Lymphomas (NHLs). DLBCL impacted approximately 26,000 patients in the U.S. in 2018. While the objective response rates of 50 – 70% observed for treatment of large B-cell lymphomas with currently marketed patient-derived T-cell products are good, there still exists a significant percentage of patients who either do not benefit from treatment or who are not able to tolerate the serious toxicities associated with these products.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.