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 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 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with diseases such as diabetes and pulmonary arterial hypertension, reported that it will be featured as a presenting company at the 9th Annual SVB Leerink Global Healthcare Conference on Thursday, February 27, 2020 at 9:30 am (ET) at the Lotte New York Palace Hotel in New York, NY (Press release, Mannkind, FEB 24, 2020, View Source [SID1234554663]). Presenting from the Company will be its Chief Executive Officer, Michael Castagna, PharmD. Interested parties can access a link to the live webcast of the presentation from the News & Events section of the Company’s website at View Source The webcast replay will remain available for 14 days following the live presentation.

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On February 24, 2020 Ascentage Pharma (6855.HK), a globally-focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that the company has received approval from Center of Drug Evaluation (CDE), China NMPA, for the Phase Ib/II clinical trial of APG-1387, Ascentage Pharma’s novel inhibitor of apoptosis proteins (IAP) inhibitor, in combination with chemotherapy (nab-paclitaxel plus gemcitabine) for the treatment of advanced pancreatic cancer.

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This multi-center, open-label clinical trial is comprised of a Phase Ib dose-escalation study and a Phase II efficacy study, and it is designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of APG-1387 in combination with the nab-paclitaxel plus gemcitabine doublet chemotherapy in advanced pancreatic cancer.

APG-1387 is a novel, small molecule IAP inhibitor that induces apoptosis by mimicking the dimeric form of the SMAC protein. In a range of in vivo and in vitro studies in multiple xenograft tumor models, APG-1387, either as a monotherapy or in combination with targeted agents or chemotherapies, demonstrated its ability to effectively inhibit the growth of tumor cells, which provided supporting evidence to the further clinical investigation of APG-1387. APG-1387 is the first IAP inhibitor to enter clinical trials in China.

APG-1387 has completed Phase I clinical trials in advanced solid tumors in China and Australia, and it was shown to be well-tolerated. The preliminary result from the ongoing Phase I trial of APG-1387 in the U.S. was presented at the 2019 Annual Meeting of American Society of Oncology (ASCO) (Free ASCO Whitepaper). The data demonstrated APG-1387’s promising anti-tumor activity in advanced pancreatic cancer patients who had failed multiple prior lines of treatments. Among 10 advanced pancreatic cancer patients treated with APG-1387 monotherapy, 4 patients achieved SD (stable disease), including one patient at 45 mg who has been treated for over 9 cycles with confirmed SD. Overall, APG-1387 was well-tolerated with manageable adverse events.

Pancreatic cancer is a highly aggressive form of gastrointestinal cancer. With a poor prognosis and an incidence rate at par with its mortality rate 1, pancreatic cancer has overtaken liver cancer as the deadliest type of all malignancies. Pancreatic cancer is ranked the ninth largest cancer type in China, with a rising incidence rate year after year. Due to the unique anatomical characteristics of the pancreas, the symptoms of early-stage pancreatic cancer are relatively silent. As a result, pancreatic cancer patients are commonly diagnosed at advanced stages or when they have developed metastasis, disqualifying them for surgery. The current median survival of patients with mPC (metastatic pancreatic cancer) is four to six months, and the 2015 statistics shows a five-year survival rate of just 7.2%, making pancreatic cancer the malignancy with the lowest survival rate in China 2.

In recent years, the rapid advancement in cancer therapies, particularly the introduction of targeted therapies and immunotherapies, has significantly improved the survival of many malignancies, including lung cancer, breast cancer, and liver cancer. However, the treatment standard for pancreatic cancer remains unchanged with very limited options. For patients with unresectable locally advanced or remotely metastasized pancreatic cancer, chemotherapy is still the primary treatment, although it only delivers a median post-treatment survival for shorter than one year. That being the case, there is urgent need to improve the clinical outcomes of pancreatic cancer treatment.

"For pancreatic cancer, in particular those cases that have progressed to advanced stage, there remains an urgent clinical need for more effective treatment options globally. As the first IAP inhibitor to enter clinical trials in China, early clinical data of APG-1387 has demonstrated its great potential for the treatment of advanced pancreatic cancer," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "We will start this Phase Ib/II trial of APG-1387 in China as early as possible, with the hope to bring new treatment options to pancreatic cancer patients."

About APG-1387

APG-1387 is a novel small molecule IAP inhibitor (Inhibitor of Apoptosis Protein), which was discovered and is being developed by Ascentage Pharma. Ascentage is developing APG-1387 globally, and has completed dose escalation Phase I trials in solid tumors in China and Australia, and a Phase Ib/II clinical trial of APG-1387 and pembrolizumab combination is currently ongoing in the U.S. In addition, APG-1387 is also being investigated in a Phase Ib trial for the treatment of patients with Chronic Hepatitis B in China.

On February 24, 2020 Austin-based biotech firm Genprex is reported for more growth after raising about $25.5 million through two recent stock offerings (Press release, Genprex, FEB 24, 2020, View Source [SID1234554661]).

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Genprex announced Jan. 28 that it had sold about 7.62 million shares of its common stock for a total of $8 million. Last week, the company announced it had closed the sale of 5 million shares of its common stock for an additional $17.5 million.

Alliance Global Partners and Joseph Gunnar & Company were the lead placement and co-placement agents, respectively, for both deals, according to the company.

Genprex, which previously operated as Convergen LifeSciences, became a publicly traded company in 2018 through an IPO valued at about $6.4 million.

CEO Rodney Varner said the funding brought in from the two recent stock offerings will help Genprex begin two clinical trials involving the company’s licensed gene therapy technology, known as Oncoprex.

Varner said Genprex, which currently has six full-time employees, is looking to expand its executive team.

"We do plan to add several new executives in the coming months, and I anticipate that they will be extremely accomplished biotech executives who will be able to contribute materially in helping us not only conduct the upcoming clinical trials, but to also manage the growth of the company as we go forward," Varner said.

Oncoprex, which Genprex licensed from the University of Texas MD Anderson Cancer Center, is designed to treat cancers by injecting tumor suppressor genes that are wrapped in nanoparticles directly into a patient’s bloodstream, Varner said.

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The clinical trials planned for this year will involve testing Oncoprex in combination with two other existing therapies to check its effectiveness fighting cancer, Varner said.

In January, the U.S. Food and Drug administration granted a fast track designation for Oncoprex in combination with an existing FDA-approved therapy.

According to the FDA website, the status is "designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need."

"For a very small company like us, that’s an important validation of our technology," Varner said.

Receiving the FDA’s fast track status played a significant role in the company’s recent infusion of funding, Varner said.

The company’s shares closed Monday at $6.03, compared to 36 cents per share in mid January before it announced the fast track status.

"There is a lot of demand right now; people want to invest," Varner said. "But that wasn’t always the case and we have had stops and starts over the last decade."

Varner said Genprex is looking to add more treatments to its research and development pipeline. On Feb. 11, the company announced it had entered into an agreement with the University of Pittsburgh for a gene therapy treatment for Type 1 and Type 2 diabetes.

"Our use of the funding is not limited to any particular technology; we can use it toward all of our technologies," Varner said.