On November 6, 2019 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, reported financial results for the quarter ended September 30, 2019 and provided a corporate update (Press release, Arca biopharma, NOV 6, 2019, View Source [SID1234552349]).

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"There are currently no FDA approved drug therapies indicated for treating patients with atrial fibrillation and heart failure with left ventricular ejection fraction values greater than 40%," commented Dr. Michael Bristow, ARCA’s President and Chief Executive Officer. "Approximately two-thirds of all heart failure patients have LVEF values greater than 40% and about half of these patients will develop atrial fibrillation. Based on our Phase 2 data, we believe Gencaro has the potential to help address this substantial unmet medical need. We look forward to further evaluating Gencaro in the planned Phase 3 clinical trial, PRECISION-AF, which we plan to initiate in the first quarter of 2020."

Pipeline Update

GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for atrial fibrillation (AF) in patients with heart failure (HF).

In July 2019, the U.S. Food and Drug Administration (FDA) agreed to amend ARCA’s Special Protocol Assessment (SPA) agreement for the Phase 3 PRECISION-AF clinical trial to expand the target trial population to include heart failure patients with left ventricular injection fractions (LVEF) > 40% and < 55%. Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the first quarter of 2020.

In September 2019, Gencaro AF clinical data from the GENETIC-AF Phase 2B trial was presented at the Heart Failure Society of America (HFSA) 2019 Annual Scientific Meeting. The data indicate Gencaro response appears to be greater in HF patients with less severe left ventricular dysfunction, a patient population with no FDA approved drug therapeutic options for AF prevention or heart failure.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for HF and peripheral arterial disease (PAD).

Chemistry, manufacturing and controls (CMC) activities continued in the third quarter.

Subject to securing additional financing, Investigational New Drug (IND)-enabling non-clinical studies are anticipated to begin in the first quarter of 2020, and an IND submission is anticipated in the second half of 2020.
Third Quarter 2019 Summary Financial Results

Cash and cash equivalents were $9.6 million as of September 30, 2019, compared to $6.6 million as of December 31, 2018. ARCA believes that its current cash and cash equivalents will be sufficient to fund its operations, at its current cost structure, after giving effect to potential cost reductions, through the third quarter of 2020.

Research and development (R&D) expenses for the three months ended September 30, 2019 were $0.3 million compared to $0.7 million for the corresponding period of 2018. The decrease in R&D expenses was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial in 2018. The Company does not anticipate having any material clinical trial expense in 2019, consequently R&D expense in 2019 is expected to be lower than in 2018.

General and administrative (G&A) expenses were relatively unchanged at $0.9 million for both the three months ended September 30, 2019 and 2018. The Company expects G&A expenses in 2019 to be consistent with those in 2018 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for the three months ended September 30, 2019 were $1.2 million compared to $1.7 million for the corresponding period of 2018. The decrease in total operating expenses was primarily attributable to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial in 2018.

Net loss was $1.2 million, or $0.76 per share, for the third quarter of 2019 compared to $1.6 million, or $2.06 per share, for the third quarter of 2018.

The Company will need to raise additional capital, and/or complete a partnership or other possible strategic transaction, to fund future operations and develop Gencaro or any other product candidates.

On November 6, 2019 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported the presentation of preclinical data for TransCon Toll-like Receptor (TLR) 7/8 Agonist, an oncology product candidate designed to provide sustained local release of resiquimod, a small molecule with immune-activating and anti-tumor properties, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in National Harbor, Maryland (Press release, Ascendis Pharma, NOV 6, 2019, View Source [SID1234551452]). The poster will be presented on Saturday, November 9, 2019.

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"Our preclinical data showed that TransCon TLR7/8 Agonist, administered as a single intratumoral injection in animal studies, delivered sustained local release of resiquimod over weeks directly to the tumor site and demonstrated potent anti-tumor activity as a monotherapy, as well as in combination with interleukin-2 (IL-2). This highlights the potential for TransCon technologies to improve the safety and efficacy of validated therapeutic candidates in oncology," said Juha Punnonen, M.D., Ph.D., Ascendis Pharma’s Senior Vice President and Head of Oncology. "Presenting these results for the first time at a premier scientific meeting is significant for us, reinforcing the potential applicability of TransCon technologies in the field of oncology."

TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod that is transiently conjugated to a hydrogel carrier via a TransCon linker. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained release of unmodified resiquimod directly to the tumor. In preclinical studies, the effective half-life of resiquimod released from TransCon TLR7/8 Agonist has been found to be approximately 25-fold longer than the parent drug, resiquimod.

Presentation Details

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019
Title Date/Time
P676: Intratumoral delivery of TransCon TLR7/8 Agonist provides potent anti-tumor activity as a monotherapy and in combination with IL-2 while minimizing systemic cytokine induction

Saturday, November 9, 2019

Following the session, the poster will also be available on the company’s website under Selected Publications in the Pipeline section.

About TransCon Oncology Programs
Ascendis Pharma is working to create potentially best-in-class oncology therapies by applying systemic and intratumoral TransCon technologies for clinically validated pathways to improve outcomes currently limited by suboptimal efficacy and systemic toxicity. Three oncology programs are currently in preclinical studies: TransCon TLR7/8 Agonist, TransCon IL-2 b/g and TransCon Vascular Endothelial Growth Factor-Tyrosine Kinase Inhibitor (VEGF-TKI).

About TransCon Technology
TransCon is short for "transient conjugation." The proprietary TransCon platform is an innovative technology to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or a small molecule in multiple therapeutic areas, and can be used systemically or locally.

On November 6, 2019 Ryvu Therapeutics (WSE: RVU), a clinical-stage biopharmaceutical company developing novel small molecule therapies that address emerging targets in oncology, reported that two abstracts have been accepted for presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, FL, taking place from December 7-10, 2019 (Press release, Ryvu Therapeutics, NOV 6, 2019, View Source [SID1234550690]).

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Details of the poster presentations are as follows:

Poster Presentation: SEL120 – a First-in-Class CDK8/19 Inhibitor As a Novel Option for the Treatment of Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome – Data from Preclinical Studies and Introduction to a Phase Ib Clinical Trial

Abstract Number: 2651

Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II

Date: Sunday, December 8, 2019

Time: 6:00 p.m. – 8:00 p.m. PST

Location: Orange County Convention Center, Hall B

Poster Presentation: CDK8 Inhibitors Induce Transcriptional Reprogramming of AML Cells Associated with Differentiation

Abstract Number: 3774

Session: 602. Disordered Gene Expression in Hematologic Malignancy, including Disordered Epigenetic Regulation: Poster III

Date: Monday, December 9, 2019

Time: 6:00 p.m. – 8:00 p.m. PST

Location: Orange County Convention Center, Hall B

To view these abstracts, please visit the American Society of Hematology (ASH) (Free ASH Whitepaper) website located at View Source

On November 6, 2019 Antengene Corporation reported that it has entered into a licensing agreement with AstraZeneca (LSE/STO/NYSE: AZN) under which Antengene has been granted the exclusive global rights to further develop and commercialize AZD0364 (Press release, Antengene, NOV 6, 2019, View Source [SID1234550689]). Antengene Corporation is a China-and-U.S. based clinical-stage biopharmaceutical company focused on discovery and development of novel drugs for treatment of oncology. AstraZeneca is a global, science-led, innovative biopharmaceutical company. AZD0364 is a potent and selective small molecule extracellular signal–regulated kinases 1 and 2 (ERK1/2) inhibitor with an open U.S. investigational new drug (IND) application.

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ERK is a key component of the Ras-Raf-MEK-ERK (RAS/MAPK) pathway, which is frequently altered in cancer and promotes tumour cell growth and survival. AZD0364 is a potent and selective inhibitor of ERK1/2 discovered by AstraZeneca. Any existing agreement regarding the licensing of AZD0364 has been nullified in advance of this agreement between Antengene and AstraZeneca.

Jay Mei, Founder and Chief Executive Officer of Antengene Corporation commented, "We are delighted to have the opportunity to work with AstraZeneca. ERK/RAS/MAPK is a key pathway excessively activated in cancers. AZD0364 is a strategic fit to our pipeline and pre-clinical studies demonstrated promising synergistic effects of AZD0364 in combination with other pipeline assets of Antengene targeting critical pathways in cancer treatment. We look forward to a close collaboration with AstraZeneca to bring more novel therapies to patients with unmet medical needs."

Susan Galbraith, Senior Vice President and Head of Research and Early Development, Oncology R&D, AstraZeneca said: "This collaboration will leverage Antengene’s scientific expertise related to ERK biology and sophisticated development capabilities to further explore AZD0364’s potential benefits to meet unmet medical needs."

On November 6, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, reported four oral and two poster presentations related to its AUTO1, AUTO2 and AUTO3 programs at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held December 7-10, 2019 in Orlando, FL (Press release, Autolus, NOV 6, 2019, View Source [SID1234550681]).

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"We are pleased that AUTO1 data will be presented in three oral presentations at ASH (Free ASH Whitepaper). The data form the basis for our decision to move AUTO1 into a pivotal clinical trial in adult ALL, our highest priority program," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "We are also looking forward to presenting data on our other hematological clinical programs at ASH (Free ASH Whitepaper). These presentations will further illustrate the significant progress we have made across our clinical portfolio this year."

The abstracts have been published today and are available on the ASH (Free ASH Whitepaper) website at View Source

The oral presentation details are as follows:

Title: AUTO1 – A novel fast off CD19CAR delivers durable remissions and prolonged CAR T cell persistence with low CRS or neurotoxicity in adult ALL
Presenter: Dr Claire Roddie
Session Date and Time: Saturday, December 7, 2:45 PM Eastern Time

Title: AUTO1 – Therapy of pediatric B-ALL with a lower affinity CD19 CAR leads to enhanced expansion and prolonged CAR T cell persistence in patients with low bone marrow tumor burden, and is associated with a favorable toxicity profile
Presenter: Dr Sara Ghorashian
Session Date and Time: Saturday, December 7, 2:30 PM Eastern Time

Title: AUTO1 – Clonal dynamics of early responder and long-term persisting CAR-T cells in humans
Presenter: Dr Luca Biasco
Session Date and Time: Saturday, December 7, 8:15 AM Eastern Time

Title: AUTO3 – Ongoing Phase 1/2 ALEXANDER clinical trial in patients with relapsed/refractory diffuse large B cell lymphoma (DLBCL)
Presenter: Dr Kirit Ardeshna
Session Date and Time: Saturday, December 7, 3:15 PM Eastern Time

The poster presentation details are as follows:

Title: AUTO2 – Phase 1 First-in-Human study of AUTO2, the first chimeric antigen receptor (CAR) T cell targeting APRIL for patients with relapsed/refractory Multiple Myeloma (RRMM)
Presenter: Dr Rakesh Popat
Session Date and Time: Sunday, December 8, 6:00 PM – 8:00 PM Eastern Time

Title: AUTO3 – Phase 1/2 AMELIA clinical trial of AUTO3 in patients with relapsed/refractory pediatric acute lymphoblastic leukemia (pALL)
Presenter: Professor Persis Amrolia
Session Date and Time: Sunday, December 8, 6:00 PM – 8:00 PM Eastern Time

About AUTO1

AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells’ abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights from UCL Business plc (UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

About AUTO3

AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in pediatric ALL in the AMELIA clinical trial and in diffuse large B cell lymphoma in the ALEXANDER clinical trial.

About AUTO2

AUTO2 is the first dual-targeting programmed T cell product candidate binding to two targets on multiple myeloma cells. AUTO2 uses a human ligand, known as APRIL, which binds to two antigens, B cell Maturation Antigen, or BCMA, and the transmembrane activator and CAML interactor, or TACI, both of which are expressed on the surface of multiple myeloma cancer cells. AUTO2 is designed to address a key escape route used by hematological cancers in response to T cell therapies.