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, 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 [SID1234554664]).

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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’s leadership looks forward to participating in dialogue about the Company’s enzyme therapeutics platform during the following industry events, with additional details to be announced.

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 Nymox Pharmaceutical Corporation (NASDAQ: NYMX) is reported that a new peer review report was published in the World Journal of Urology, documenting the successful long-term clinical trial results after Fexapotide Triflutate treatment for early stage prostate cancer (Press release, Nymox, FEB 24, 2020, View Source [SID1234554649]).

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This report represents the first publication in the medical literature of multi-year long-term data from a well-powered prospective randomized multi-center clinical trial for a prostate injectable treatment in men targeted to low grade early prostate cancer and showing statistically significant efficacy for a locally targeted molecular injectable treatment.

The new publication is entitled "Prospective Evaluation of Fexapotide Triflutate Injection ‎Treatment of Grade Group 1 Prostate Cancer: Four Year Results". The authors are Neal Shore, Myrtle Beach, SC; Steven A. Kaplan, New York, NY; Ronald Tutrone, Baltimore, MD; Richard Levin, Towson, MD; James Bailen, Louisville, KY; Alan Hay, Salem, OR; Susan Kalota, Tucson, AZ; Mohamed Bidair, San Diego, CA; Sheldon Freedman, Las Vegas, NV; Kenneth Goldberg, Lewisville, TX; Frederick Snoy, Albuquerque, NM; Jonathan I. Epstein, Baltimore, MD. The article is available online at View Source

The Fexapotide (FT) study was started in 2012 and enrolled 147 men with localized Gleason Grade 6 T1c prostate cancer at 28 U.S. clinical investigation sites. Patients were followed with clinical and laboratory evaluations and regular periodic prostate biopsies for up to 5 years. Patients randomized to FT were treated with a single one-time targeted injection of FT, either 2.5mg or 15mg. Statistical comparisons were made over time of the proportions of subjects and untreated controls who progressed to higher Gleason grade and/or invasive treatments instituted with prostatectomy, radiotherapy, or chemotherapy. Important clinical highlights from the study include: FT treatment reduced cancer progression (-67.7%) compared to controls (3 years, FT 15mg, p<.02, pooled FT p=.0265) and also reduced (-54.7%) the incidence of surgery, radiotherapy or chemotherapy (4 years, FT 15mg p<.02; pooled FT p=.0374). At 4 years the incidence of surgery, radiotherapy or chemotherapy with increased Gleason grade was significantly reduced (FT 15mg -73.3% p=.0059, pooled FT p=.0064). Results for the high dose (FT 15mg) were superior to the lower dose (FT 2.5mg). Safety data showed no serious adverse events related to FT during the study.

The current recommended standard of care for these patients is active surveillance: patients are monitored carefully over time to determine if and when the cancer becomes more advanced and thus will require more aggressive treatment, like radiation therapy or surgery.

Dr. Neal Shore, lead author of the report, said "The study results demonstrate data in a large multi-center clinical trial of men treated with transrectal ultrasound guided FT for early stage, low risk prostate cancers, which suggest an effective result for reducing histopathologic progression, as evidenced by repeated biopsies over time, while also demonstrating a favorable safety profile. This study presents long-term FT data which supports its efficacy for avoidance of biologic progression in an active surveillance prostate cancer population. A therapy to optimize the success of an active surveillance strategy is a welcomed advance for these patients."

Dr Steven Kaplan, Professor of Urology and Director of the Benign Urologic Diseases and The Men’s Health Program at Icahn School of Medicine at Mount Sinai, New York, and a co-author of the report said, "This study reports the long-term data from a prospective study of an injectable for localized Grade Group 1 prostate cancer — which is the first time this has been accomplished for a very common problem in men. Regulatory approval of Fexapotide Triflutate (FT) will be a very important treatment adjunct for countless men with this problem."

The FT treatments were administered in a urology office setting without anesthesia or sedation, consisting of a single relatively painless transrectal injection into the area of the prostate cancer. The percentage of patients with surgery or radiotherapy with Gleason grade progression was reduced by 73.3% in patients treated with a single injection of FT 15mg and by 62.6% in pooled (high and low dose FT groups combined) FT patients compared to controls.

Dr. Jonathan Epstein a co-author and researcher involved in the study, stated "Although more and more men are electing active surveillance for low grade prostate cancer, approximately 25-35% will show increased grade on follow-up biopsy which typically leads to definitive therapy. In this study, Fexapotide Triflutate decreased the risk of upgrading on follow-up biopsy enabling men to stay on active surveillance. Even if Fexapotide Triflutate enables men to stay on active surveillance longer before they eventually experience upgrading, there would be a significant benefit for men to delay the onset of side effects associated with therapy and in the interim enjoy a better quality of life. Future studies are needed to expand the criteria for study of Fexapotide Triflutate to include men with large volume Grade Group 1 disease and possibly men with low volume, low percent pattern 4 Grade Group 2 cancer. Identifying the ideal candidates for Fexapotide Triflutate also factoring in multiparametric MRI findings remains to be determined."

FT is a pro-apoptotic proprietary drug which promotes natural programmed cell death (apoptosis) in prostatic glandular cells which compose the prostate cancer. FT has been safely administered to men in clinical trials in the U.S. involving over 1700 patients and controls treated for BPH or prostate cancer. FT has completed Phase 3 studies for BPH and further studies of FT for prostate cancer are planned in the near future.

For further detailed information about the published study please refer to the new report online at the World Journal of Urology.

On February 24, 2020 ReadCoor, Inc., a company leading true multi-omic spatial sequencing, reported unveils its first product line, including multi-omic spatial sequencing assays and the RC2 instrument (Press release, ReadCoor, FEB 24, 2020, View Source [SID1234554665]). The platform is powered by ReadCoor’s proprietary FISSEQ (Fluorescent in situ Sequencing) technology, which combines the massive multiplexity of next-generation sequencing (NGS) and high-resolution tissue imaging. The fully integrated platform is now available for use by researchers through ReadCoor’s Select Release Program (SRP), focusing on initial applications in immuno-oncology, neuroscience, and infectious disease, as well as a custom offering. ReadCoor has increased its Series B investment round to $30 million to accelerate commercial efforts.

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The new platform simultaneously detects tens to thousands of RNA and DNA sequences, proteins, and therapeutic molecules at nanoscale 3D resolution in every cell within an intact tissue sample. The key products launched today are:

Automated, turn-key spatial sequencing instrument called RC2 that captures sub-cellular complexity at nanoscale resolution across entire tissue sections.
Application Kits containing all components necessary to prepare tissue samples for spatial sequencing on the RC2 in a single sample processing workflow.
Sample slides provide optimized surfaces compatible with standard sectioning workflows for FFPE, fixed frozen, or fresh frozen tissues and feature markings to guide tissue placement.
Instrument Kits contain all necessary bulk and sequencing reagents to collect spatial sequencing information from up to four tissue samples per run.
Analysis and Visualization Cloudware that fully integrates data management and analysis. Users can manage and analyze sub-cellular molecular information with visualization tools that display molecular and morphological volumes, display locations for all multi-omic targets in 3D, select regions of interest, and employ spatial statistical methods for unbiased discovery.
"We firmly believe our true multi-omic sequencing technology will have a dramatic impact on medicine and healthcare, and this motivates us to provide researchers products that offer unprecedented and meaningful insights into human biology," said Richard Terry, Chief Executive Officer, Chief Technical Officer, and Founder of ReadCoor. "Our Select Release Program serves as the foundation for researchers to develop new therapeutics and treatments to ultimately improve patient care."

Users within the SRP can use ReadCoor’s initial applications to support their specific research interests. The applications offer the following capabilities:

Immuno-oncology
ReadCoor’s immuno-oncology application unifies disparate histological and molecular assays, eliminating the need to allocate precious tumor samples between anatomical and molecular pathology methods. This approach could increase the efficacy of IO treatments for meaningful clinical insights in immuno-oncology, pathology, and drug discovery.
Neuroscience
Comprehensive cell function and phenotyping to atlas the brain using the RC2 platform will have enormous benefits to both basic neuroscience research and biomedical applications such as understanding and treating traumatic brain injury and neurodegenerative diseases.
Infectious Disease
ReadCoor’s initial infectious disease application is designed to advance understanding of HIV biology. This approach has the potential to provide insights into many aspects of HIV’s lifecycle and ultimately inform better treatment options towards eradicating this disease.

On February 24, 2020 SWOG Cancer Research Network reported that many patients with rare, fast-growing neuroendocrine tumors respond well to a common immunotherapy drug combination, according to the first peer-reviewed publication out of DART, short for Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors, a unique rare cancer clinical trial (Press release, SWOG Cancer Research Network, FEB 24, 2020, View Source [SID1234554650]).

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DART offers the immunotherapy combination of ipilimumab and nivolumab to patients with 53 classes of rare cancers through an innovative "basket" design, which allows a single drug or drug combination to be tested in a variety of tumor types. The trial has rapidly expanded the opportunities for immunotherapy drug development in rare cancers, which make up almost a quarter of all cancers diagnosed worldwide.

DART is managed by SWOG Cancer Research Network, a clinical trials group that is part of the National Cancer Institute’s (NCI) National Clinical Trials Network (NCTN), the oldest and largest publicly-funded cancer research network in the U.S.

The study results appear in Clinical Cancer Research. When presented in March 2019 at the annual meeting of the American Association of Cancer Research, initial findings were met with excitement. That’s because patients with high-grade, or fast-growing, neuroendocrine carcinoma have few treatment options.

"We’re very encouraged by these results," said Sandip Pravin Patel, MD, the DART clinical study chair, an associate professor of medicine at the University of California at San Diego School of Medicine, and a medical oncologist with Moores Cancer Center at UC San Diego Health. "Based on the response, we opened another study enrolling only patients with high-grade neuroendocrine carcinoma to see if we can replicate our results. We’re eager to share those findings later this year."

Patel’s fellow DART chairs are Razelle Kurzrock, MD, of UCSD Moores Cancer Center and Young Kwang Chae, MD, PhD, of Northwestern University.

For this neuroendocrine cohort, 32 eligible patients received the ipilimumab and nivolumab combination. Of the 32, 18 had high-grade cancer, with tumors most commonly appearing in the lungs or gastrointestinal tract. Regardless of where their tumors appeared, eight out of 18 high-grade patients – or 44 percent – saw them shrink partially or completely. By contrast, patients with intermediate or low-grade tumors saw no response.

Since its 2017 launch, physicians at 823 cancer centers, academic medical centers, and community clinics and hospitals have opened DART. As of February 13, 715 patients have enrolled out of a goal of 818 – beating all expectations. Historically, rare cancer trials struggle to find patients, leading many investigators to abandon rare cancer research.

Currently, the trial is enrolling patients with 12 different cancer types. For information, call the NCI’s Cancer Information Service at 1-800-4-CANCER and ask about DART or S1609. More information about the trial, under NCT number NCT02834013, can be found at clinicaltrials.gov.

DART is funded by the National Institutes of Health through NCI grant awards CA180888, CA180819, CA180821, and CA180820 and in part by Bristol-Myers Squibb.

Patel is first author of the Clinical Cancer Research article, and Patel, Chae and Kurzrock all serve as corresponding authors. Other authors include: Megan Othus, PhD, of SWOG and Fred Hutchinson Cancer Research Center; Francis Giles, MD, formerly of Northwestern University and now at NEOMED Therapeutics; Donna Hansel, MD, of UCSD Moores Cancer Center; Preet Singh, MD, of Springfield Clinic; Annette Fontaine, MD, of New Mexico Cancer Center; Manisha Shah, MD, of The Ohio State University Comprehensive Cancer Center; Anup Kasi, MD, MPH, of the University of Kansas Cancer Center; Tareq Al Baghdadi, MD, of IHA Hematology Oncology at St. Joe’s Ann Arbor; Sreekanth Vasireddy, M.D. of Ochsner Health System; Zoran Gatalica, MD, DSc, of Caris Life Sciences; W. Michael Korn, MD, of Caris Life Sciences and the UCSF Helen Diller Family Comprehensive Cancer Center; Jourdain Hayward of the SWOG Statistics and Data Management Center; Christine McLeod of the SWOG Statistics and Data Management Center; Helen X. Chen, MD, of the National Cancer Institute; Elad Sharon, MD, of the National Cancer Institute; Edward Mayerson, MS, of the SWOG Statistics and Data Management Center; Christopher Ryan, MD, of Oregon Health & Science University; Melissa Plets, MS, of the SWOG Statistics and Data Management Center; and Charles D. Blanke, MD, of the SWOG Group Chair’s Office.

On February 24, 2020 Omeros Corporation (NASDAQ: OMER) reported that the company will issue its fourth quarter and year-end 2019 financial results for the period ended December 31, 2019, on Monday, March 2, 2020, after the market closes (Press release, Omeros, FEB 24, 2020, View Source [SID1234554666]). Omeros management will host a conference call and webcast that day at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss the financial results as well as recent developments and highlights.

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Conference Call Details

To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 4870947. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 4870947.

To access the live and subsequently archived webcast of the conference call, go to Omeros’ website at www.omeros.com and select "Events" under the Investors section of the website. Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.