On February 24, 2020 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biopharmaceutical company with a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, reported it has filed a patent application for its drug candidate Namodenoson to be used as a combination therapy with checkpoint inhibitors (Press release, Can-Fite BioPharma, FEB 24, 2020, View Source [SID1234554634]). Titled "Programmed Death 1/Programmed Death Ligand 1 (PD-1/PD-L1) Axis Inhibitor For Use In Combination With An A3 Adenosine Receptor (A3AR) Ligands", the patent application addresses various oncology indications including advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. Namodenoson is currently being developed by Can-Fite as a monotherapy for HCC, with a Phase III study expected to commence in 2020.

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Checkpoint inhibitors, including PD-1 and PD-L1 inhibitors, are a novel class of oncology drugs with a global market value of $10.6 billion in 2017 and estimated to reach $56.5 billion by 2025 according to Allied Market Research. PD-1 and PD-L1 comprise the largest category within the checkpoint inhibitor market, driven by sales of pembrolizumab and nivolumab.

Patients respond very well to checkpoint inhibitors, however a high percentage may develop resistance to the drugs. New studies show that activation of the β-catenin protein in tumor cells helps cancer to escape detection by the body’s natural immune mechanisms and creates resistance to PD-1 and PD-L1 checkpoint inhibitors, including nivolumab and pembrolizumab, two of the most broadly used checkpoint inhibitors on the market today. In pre-clinical studies, Can-Fite’s Namodenoson has shown to significantly inhibit the expression of PD-L1 in liver pathological cells, and therefore has the potential to boost the efficacy of drugs that inhibit PD-1 and PD-L1 by helping patients overcome resistance to the drug.

"The capability of Namodenoson to inhibit the β-catenin pathway in cancer cells has the potential to play a key role in the efficacy of checkpoint inhibitors and may overcome resistance. Based on its mechanism of action, Namodenoson has anti-cancer properties as a monotherapy which may support its use as a powerful combination treatment with checkpoint inhibitors currently on the market," stated Can-Fite CEO Dr. Pnina Fishman.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated as a second line treatment for hepatocellular carcinoma, with a recently completed Phase II trial and planned Phase III trial in this indication. The drug is currently in an ongoing Phase II trial as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On February 24, 2020 Bio-Techne Corporation (NASDAQ: TECH) reported that Jim Hippel, Chief Financial Officer, will present at the Cowen 40th Annual Health Care Conference on Wednesday, March 4, 2020 at 8:00 a.m. EST (Press release, Bio-Techne, FEB 24, 2020, View Sourcenews/detail/177/bio-techne-to-present-at-the-cowen-40th-annual-health-care-conference" target="_blank" title="View Sourcenews/detail/177/bio-techne-to-present-at-the-cowen-40th-annual-health-care-conference" rel="nofollow">View Source [SID1234554633]). The conference will be held at the Boston Marriott Copley Place in Boston, MA. A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link https://wsw.com/webcast/cowen57/tech/.

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On February 24, 2020 Aura Biosciences, a clinical-stage biopharmaceutical company developing a new class of tumor targeted therapies for initial application in primary tumors such as ocular and bladder cancers, reported the presentation of updated clinical data from its ongoing Phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, the Company’s lead product candidate for the first line treatment of primary choroidal melanoma, at the 43rd Annual Macula Society Meeting, which took place February 19-22, 2020 in San Diego, CA (Press release, Aura Biosciences, FEB 24, 2020, View Source [SID1234554632]).

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"The Phase 1b/2 data being recognized at the Macular Society meeting this year show that AU-011 continues to be well tolerated, with a statistically significant reduction in tumor growth rate and vision preservation," said Amy C. Schefler, M.D., Weill Cornell Medical College and Retina Consultants of Houston.

"The high tumor control rates observed to date, together with the high rate of vision preservation, including in patients at high risk for vision loss with sub-macular and peripapillary tumors continue to support AU-011’s significant potential to change the treatment paradigm for patients battling choroidal melanoma," said Cadmus Rich, MD, Chief Medical Officer and Head of Research and Development of Aura Biosciences. "I am proud of the work the company has done to make a significant progress in this ultra-rare disease."

Updated Results from the Phase 1b/2 Study Evaluating AU-011

Dr. Schefler gave an oral presentation titled, "Study Update of an Ongoing Phase 1b/2 Open-label Clinical Trial of AU-011 for the Treatment of Small to Medium Choroidal Melanoma," which highlighted updated data from the ongoing open-label Phase 1b/2 clinical trial, and described the design of the planned Phase 3 trial that Aura expects to initiate during the second half of 2020.

The updated data demonstrate that multiple administrations of light-activated AU-011 were well-tolerated. Among the patients evaluated for safety (n=52), the most common treatment-related adverse events (AEs) were expected and included anterior chamber inflammation, posterior chamber inflammation and increase in intraocular pressure; all were manageable with standard-of-care treatments and the majority resolved without clinical sequelae. Notably, the posterior inflammation appears to originate within and/or around the tumor which is consistent with AU-011’s mechanism of action of acute tumor necrosis. There were two treatment-related serious AEs (vision loss; 3.6%). Tumor control and vision preservation data continue to be supportive of the planned Phase 3 registration trial.

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary intraocular tumor in adults and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes in approximately 50 percent of cases with liver involvement in 80-90% of cases and, unfortunately, metastatic disease is universally fatal (source: OMF). There is a very high unmet need for a new vision sparing targeted therapy that could enable early treatment intervention for this life-threatening rare disease given the lack of approved therapies, and the comorbidities of radioactive treatment options.

About Light-Activated AU-011

AU-011 is a first-in-class targeted therapy in development for the treatment of primary choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs bind selectively to unique receptors on cancer cells in the eye and are derived from technology originally pioneered by Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

On February 24, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as solutions for diseases with high unmet medical need, today reported its fourth-quarter and full-year 2019 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, FEB 24, 2020, View Source [SID1234554631]).

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"Our accomplishments this past year illustrate how we are working to reimagine the potential of human enzymes as transformative solutions for challenging rare genetic disorders," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "In addition to delivering compelling Phase 1/2 data from our lead program for Arginase 1 Deficiency, we’ve made solid progress with our pivotal trial enrollment and started laying the groundwork for the commercial launch. We have also continued to make significant progress in our pipeline, further demonstrating the effectiveness of our platform in advancing programs in multiple therapeutic areas."

"We begin 2020 with a clear vision and heightened conviction of the impact for patients we can create with human enzyme therapeutics. We believe that we are well positioned to achieve a number of important milestones this year, armed with a strong leadership team with deep discovery, development and commercial expertise and driven by our collective goal to address the needs of the communities we serve," concluded Dr Quinn.

Recent Highlights

Pegzilarginase in Arginase 1 Deficiency

The Company expects to complete enrollment of its global, pivotal Phase 3 Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints (PEACE) trial in the third quarter of 2020 and to provide topline data in the first quarter of 2021.

Based on a recent genetic prevalence analysis of Arginase 1 Deficiency (ARG1-D), and with more than 200 patients already identified worldwide, the Company now estimates an addressable patient population of greater than 2,500, up from an estimate of 1,000 patients based solely on initial insights using newborn screening data.

The Company’s patient-identification strategy, informed by critical insights from disease analysis and trial experience, has already identified more than 100 patients in the United States, representing a 40% penetration into the genetic prevalent population.

ACN00177 in Homocystinuria

In January, Aeglea announced the filing of its Clinical Trial Application (CTA) with the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177, a novel engineered human enzyme therapy designed to treat homocystinuria, a serious metabolic disorder that results in elevated levels of plasma homocysteine.

The Company expects to initiate a Phase 1/2 trial for ACN00177 in the second quarter of 2020, with initial human proof of concept in the first quarter of 2021.

The Company estimates an addressable homocystinuria patient population of greater than 5,000.

Upcoming Events

Aeglea will be attending the following investor conferences in the coming quarter. Details of the presentations and webcasts will be announced prior to the events.

19th Annual Needham Healthcare Conference, April 14-15, New York City

H.C. Wainwright 2020 Global Life Sciences Conference, April 20-21, London, United Kingdom

Further, Aeglea leadership looks forward to participating in dialogue about our enzyme therapeutics platform during the following industry events, with additional details forthcoming.

Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), April 26-29, Austin, Texas

World Orphan Drug Congress USA 2020, April 29 to May 1, Oxon Hill, Maryland

Fourth Quarter and Full Year 2019 Financial Results

As of December 31, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $73.4 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $17.6 million for the fourth quarter of 2019 and $11.8 million for the fourth quarter of 2018. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; ramp-up in toxicology, investigational new drug (IND)-enabling studies, and manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses.

Research and development expenses totaled $64.6 million for the year ended December 31, 2019, compared with $36.7 million for the year ended December 31, 2018. The increase was primarily due to investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; a ramp-up in toxicology, IND-enabling studies, and manufacturing activities for ACN00177 in Homocystinuria; and expanded clinical development activity and personnel-related expenses.

General and administrative expenses totaled $4.3 million for the fourth quarter of 2019 and $3.5 million for the fourth quarter of 2018. This increase was primarily due to additional employee headcount, commercial readiness support, and facilities to support company growth.

General and administrative expenses totaled $15.7 million for the year ended December 31, 2019, compared with $12.6 million for the year ended December 31, 2018. This increase was primarily due to additional employee headcount, compensation, and facilities to support company growth.

Net loss totaled $21.5 million and $14.9 million for the fourth quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.4 million for the fourth quarter of 2019 and 2018, respectively. Net loss totaled $78.3 million and $44.3 million for the years ended December 31, 2019 and 2018, respectively, with non-cash stock compensation expense of $4.9 million and $4.3 million for the years ended December 31, 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea’s Phase 1/2 and Phase 2 open-label extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe myopia. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of homocystinuria. The Company has submitted a Clinical Trial Application (CTA) with the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and expects to initiate a Phase 1/2 trial in the second quarter of 2020.

On February 24, 2020 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that Ron Cohen, M.D., Acorda’s President and Chief Executive Officer, will present at the Cowen and Company Annual Health Care Conference on Monday, March 2 at 2:50PM EST (Press release, Acorda Therapeutics, FEB 24, 2020, View Source [SID1234554630]). A live audio webcast of the presentation can be accessed under "Investor Events" in the Investor section of the Acorda website at www.acorda.com, or you may use the link:

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http://wsw.com/webcast/cowen57/acor/