On February 18, 2020 Agendia, Inc., a world leader in precision oncology for breast cancer, reported that BMC Cancer published results from the IMPACt trial. The study measured changes in both treatment decisions and physician confidence when using the MammaPrint and BluePrint assays to support medical management for patients with early stage breast cancer (Press release, Agendia, FEB 18, 2020, View Source [SID1234554468]).

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Because breast cancer is not a single disease, outcomes and response to therapy vary greatly from patient to patient based on biology that is not always reflected in standard clinical and pathologic features. Genomic testing has become more widely utilized to help understand these differences with the goal of improving patient outcomes. MammaPrint is a 70-gene signature that offers clarity on a patient’s risk of recurrence, whereas BluePrint is an 80-gene signature that goes beyond the surface of the tumor to identify the functional pathway driving its growth. The IMPACt study aimed to evaluate the role of genomic profiling in treatment planning as well as the degree of physician confidence when utilizing the MammaPrint and BluePrint assays.

"Through Agendia’s comprehensive genomic profiling tools, we are increasing our understanding of an individual’s tumor biology," stated Hatem Soliman, MD. "It is important to evaluate how that information impacts treatment planning in a real world clinical setting."

IMPACt prospectively enrolled 452 patients between November 2015 and August 2017. In the real-world cohort, MammaPrint and BluePrint reclassified 40% of pathologically subtyped tumors. This highlights the utility for a BluePrint molecular subtyping profile in early stage breast cancer and a more personalized approach to treatment for patients. The study also showed that physicians’ treatment plans for patients were consistent with their MammaPrint results in 89% of cases, supporting the use of this signature to inform treatment decisions in clinical practice. For clinically high risk patients for whom chemotherapy was initially recommended, there was a 60% reduction in the use of chemotherapy when patients were classified as Low Risk by MammaPrint. Conversely, when clinically low risk patients had a High Risk genomic profile, chemotherapy was added to the treatment plan in 60% of cases that did not initially include it.

Additionally, physicians reported greater confidence in their treatment decisions for 72% of cases after receiving MammaPrint results, supporting the findings of the 2015 PROMIS trial, which showed increased physician confidence in 79% of patient treatment plans. Both studies demonstrate a high level of certainty that patients are being offered chemotherapy when appropriate and reassurance that Low Risk patients can safely forego chemotherapy and its associated toxicities.

"Physician confidence is an integral component to determining the most effective treatment plan and provides much-needed peace of mind for our patients," says Robert Gabordi, MD.

"We are very encouraged with the results of this important trial confirming the clinical utility of MammaPrint and BluePrint," commented Agendia’s Chief Medical Officer, William Audeh, MD, MS. "This not only underscores the reliability of our genomic assays, but it further reinforces the critical role they play when optimizing a personalized treatment strategy for patients with early stage breast cancer."

On February 18, 2020 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 year ended December 31, 2019 and provided a corporate update (Press release, Arca biopharma, FEB 18, 2020, View Source [SID1234554421]).

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Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, commented, "During 2019, we continued to advance development of Gencaro as a potential genetically-targeted treatment for heart failure patients with atrial fibrillation. In published or submitted for presentation/publication material, we have identified important new effectiveness evidence from the GENETIC-AF trial that have enhanced our understanding of the clinical profile of Gencaro. These data have allowed us to broaden the design of the single, pivotal Phase 3 clinical trial that was developed after consultation with the U.S. Food and Drug Administration via a Special Protocol Assessment agreement. There are currently no FDA approved drug therapies indicated for the treatment of atrial fibrillation in heart failure patients with left ventricular ejection fraction values greater than 40%. Based on our clinical data to date, we believe Gencaro has the potential to help address this substantial unmet medical need. We look forward to further evaluating the pharmacogenetic benefits of Gencaro for patients with our targeted genetic profile in PRECISION-AF, the Phase 3 clinical trial, which, subject to obtaining additional financing, we plan to initiate in the fourth quarter of 2020."

Pipeline Update – Second Half Review

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).

The U.S. Food and Drug Administration (FDA) issued a Special Protocol Assessment (SPA) agreement for a single Phase 3 clinical trial (PRECISION-AF) to examine Gencaro as a genetically-targeted therapy for the prevention of AF recurrence in certain heart failure patients.
PRECISION-AF is designed as a double-blind, active-controlled, multicenter, international, adaptive study comparing Gencaro with TOPROL-XL (metoprolol succinate) for the prevention of AF recurrence or all-cause mortality in approximately 400 heart failure patients who have left ventricular injection fraction (LVEF) values ≥ 40% and ≤ 55% and the genotype which ARCA believes responds best to Gencaro (ADRB1 Arg389Arg).
Clinical data from the GENETIC-AF Phase 2B clinical trial was published in the Journal of the American College of Cardiology: Heart Failure in May 2019 and was presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in September 2019. These data indicate that the response to Gencaro may 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.
Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the fourth quarter of 2020.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for HF and peripheral arterial disease (PAD).

Subject to securing additional financing, the Company anticipates conducting non-clinical studies to support a potential IND submission and initiation of clinical development in 2021.
2019 Summary Financial Results

Cash and cash equivalents were $8.4 million as of December 31, 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 end of the third quarter of 2020.

Research and development (R&D) expenses for the year ended December 31, 2019 were $1.8 million compared to $4.2 million for 2018. The $2.4 million decrease was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial. If the PRECISION-AF clinical trial is initiated in the second half of 2020, R&D expense in 2020 is expected to be higher than 2019.

General and administrative (G&A) expenses were $4.0 million for 2019 compared to $3.9 million for 2018. The Company expects G&A expenses in 2020 to be consistent with those in 2019 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for 2019 were $5.8 million compared to $8.1 million for 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.

Net loss was $5.5 million, or $4.15 per basic and diluted share, for 2019 compared to $7.9 million, or $10.31 per basic and diluted share, for 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 February 18, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR, reported that Selecta’s Chief Executive Officer, Carsten Brunn, Ph.D., will participate in a fireside chat at the 9th Annual SVB Leerink Global Healthcare Conference in New York on Tuesday, February 25 at 11:00 a.m. Eastern Time (Press release, Selecta Biosciences, FEB 18, 2020, View Source [SID1234554437]). A live webcast and a copy of the presentation will be available on the Investors & Media section of the Selecta website at www.selectabio.com.

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On February 18, 2020 Proscia, a leading provider of artificial intelligence (AI) enabled digital pathology solutions, reported that it has expanded into the international diagnostic pathology market to meet the growing needs of laboratories and cancer patients worldwide (Press release, Proscia, FEB 18, 2020, View Source [SID1234554453]). The company has added the leadership of Arun Ananthapadmanabhan as Executive Vice President, Global Growth, to manage commercial operations for its international market. Proscia’s accelerated global focus follows the company’s November announcement that it received CE Mark for its Concentriq Dx solution for use in primary diagnosis.*

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Digital pathology has experienced broad adoption in international markets, where the regulatory landscape and a severely declining pathologist population have encouraged laboratories to implement image-based workflows. These laboratories are realizing the benefits of digitization, including improved efficiency and productivity. A notable example of successful digitization is Spain’s Granada University Hospitals, where pathologists have signed out, on average, 21% more cases each year since implementing a full digital pathology program for primary diagnosis. Digital laboratories are now poised to adopt AI-based computational applications, which will further enhance efficiency and expand the breadth of critical diagnostic information available to improve patient outcomes.

"The European digital pathology market is demonstrating tremendous potential in the face of critical challenges," said Steve Holloway, Company Director & Principal Analyst at Signify Research. "In a recent workforce census conducted by the Royal College of Pathologists, only three percent of UK pathology departments reported having enough staff to meet clinical demand. Shortages like these are being blamed for ultimately delaying cancer treatment, and the continued adoption of digital pathology and use of artificial intelligence will play a major part in addressing these issues."

Proscia’s Concentriq digital pathology platform operates at the center of the modern, image-based laboratory. Its end-to-end capabilities include automation of time consuming and error-prone manual tasks, streamlined collaboration, and delivery of deeper operational insights into the hands of laboratory managers. Concentriq is an open, flexible platform that works with any scanner and laboratory information system (LIS), offering seamless integrations with Philips, Leica, 3DHISTECH, Roche, and Hamamatsu among other leading whole slide image scanners. It also serves as a launchpad for the company’s suite of AI applications, enabling laboratories to easily deploy AI in practice.

"With the only AI-enabled digital pathology platform, Proscia is uniquely positioned to enter the international market," said David West, CEO of Proscia. "This market is primed for the adoption of digital pathology and artificial intelligence, which we can deliver today. We look forward to building upon our traction in the U.S. and working with our growing partner ecosystem to help laboratories worldwide realize the full promise of digital pathology. We’re pleased to welcome Arun to our leadership team to help us carry out this effort with his deep market expertise."

Arun Ananthapadmanabhan joins Proscia as Executive Vice President, Global Growth, to oversee the company’s international operations. He comes to Proscia from Philips, where he created and led the Computational Pathology business. In addition to strong product and market expertise, Arun brings experience advising on growth strategies from his time as management consultant at Bain. Arun is located in Amsterdam, from where he will be building a team to support his efforts.

"I am excited to join Proscia and advance the company’s mission of perfecting cancer diagnosis with intelligent software," said Arun Ananthapadmanabhan, Proscia’s Executive Vice President, Global Growth. "There are clear synergies between Proscia’s approach to digital and computational pathology and the needs of the international market. Concentriq is a truly open and reliable platform with embedded, future-proof AI workflows. This is a clear unmet need in the market today that can accelerate the adoption of digital pathology. I am passionate about improving the lives of cancer patients and see a real opportunity to do so as part of the Proscia team."

On February 18, 2020 Phosplatin Therapeutics LLC, a clinical stage pharmaceutical company focused on oncology drug development, reported its publication in OncoImmunology of in vitro and in vivo data demonstrating that PT-112 is a bona fide immunogenic cell death (ICD) inducer, and as a result can initiate anticancer immunity, as a monotherapy and in combination with immune checkpoint inhibition (Press release, Phosplatin, FEB 18, 2020, View Source [SID1234554469]).

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PT-112 causes the release of so-called damage associated molecular patterns (DAMPs), a signature of ICD, by dying cancer cells. In addition, PT-112 synergizes with immune checkpoint blockers (ICBs) in the context of superior immune infiltration. These findings are in line with preliminary clinical evidence on the use of PT-112 in patients with solid tumors, either as a monotherapy or in combination with anti-PD-L1 immune checkpoint blockade.

"As a stand-alone agent, PT-112 has been validated as a potent ICD inducer," said Lorenzo Galluzzi, PhD, Assistant Professor of Cell Biology in Radiation Oncology at Weill Cornell Medical College, and senior author of the article. "Our model systems are designed to isolate the immune effects of a given agent, and PT-112 showed positive results across all model systems deployed thus far." "We have been delighted with our collaboration with Dr. Galluzzi and his laboratory team at Weill Cornell," said Matthew R. Price, Executive Vice President & COO at Phosplatin Therapeutics. "This publication underscores PT-112’s role within the emerging field of immunotherapy, and its potential as a best-in-class ICD inducer."

Along with ICD induction, PT-112 possesses a unique combination of factors, including safety in heavily pre-treated patients, single-agent activity in patients with lung cancers, prostate cancer and thymoma. In addition, the pyrophosphate component of the drug is believed to be responsible for bio-distribution that includes high drug concentrations in mineralized bone (osteotropism). PT-112’s features make it a promising candidate for the treatment of cancers that frequently metastasize to bone and/or are not likely to respond to checkpoint inhibitors.

Please refer to the OncoImmunology paper, "PT-112 induces immunogenic cell death and synergizes with immune checkpoint blockers in mouse tumor models," (Issue 9, Vol. 1) for the full description of the design and results of this work. The publication is available online here.

Further information on clinical trials with PT-112 that are currently open can be found at the clinicaltrials.gov registry under NCT 02266745, NCT 03409458, and NCT03288480.

About PT-112

PT-112 is a novel anti-cancer agent, the first cytotoxic small molecule conjugate of pyrophosphate developed in oncology therapeutics. PT-112 promotes immunogenic cell death (ICD), or the release of damage associated molecular patterns (DAMPs) that lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a potential best-in-class small molecule inducer of this immunological form of cancer cell death. The first-in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses and tumor control among heavily pre-treated patients, and was presented at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress, winning "Best Poster" among the Developmental Therapeutics category. The novelty of its pyrophosphate moiety also results in osteotropism, or the propensity of the drug to reach the mineralized bone. This property is of interest in cancer types that originate in bone, or frequently lead to metastatic bone involvement, such as metastatic castrate-resistant prostate cancer mCRPC. The first human clinical results in mCRPC were presented at the 2020 Genitourinary Cancers Symposium on February 13, 2020.