On April 3, 2018 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the full year ended December 31, 2017 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, APR 3, 2018, View Source [SID1234525156]).

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"During 2017 we made meaningful progress advancing both our clinical and corporate objectives, which has positioned us for continued growth throughout 2018 and beyond," stated Peter Nielsen, President and Chief Executive Officer of Bio-Path. "As we move into 2018, we expect to implement the protocol amendments to our Phase 2 clinical trial of prexigebersen for the treatment of acute myeloid leukemia, to prepare for a Phase 1 clinical trial of BP1002 in lymphoma, to begin enrollment of a Phase 1 clinical trial of prexigebersen in solid tumors potentially by year-end, and to start a series of IND-enabling studies for BP1003 in pancreatic cancer. We continue to have confidence in the performance and potential of our DNAbilize platform technology to produce exciting drug candidates to help patients with high unmet medical need."

Recent Corporate Highlights

·Reported Pre-Specified Interim Results from Phase 2 Study of Prexigebersen in Combination with LDAC to Treat AML. Of the 17 evaluable patients, four patients achieved complete responses, one patient achieved a leukemia free, one patient had significantly reduced bone marrow blasts and three patients achieved stable disease. In total, 47% of the evaluable patients showed some form of response to the combination treatment, including four patients with complete remission (23%) and four patients with stable disease.

·Published Data in The Lancet Haematology. In March 2018, the Company announced that data from its Phase 1/1b study of prexigebersen (BP1001) as a treatment for hematological malignancies was published in The Lancet Haematology in an article titled, "Liposomal Grb2 antisense oligodeoxynucleotide (BP1001) in patients with refractory or relapsed haematological malignancies: a single-centre, open-label, dose-escalation, phase 1/1b trial."

·Announced Third Drug Candidate, BP1003, for Treatment of Pancreatic Cancer. In November 2017, Bio-Path announced its third drug candidate, BP1003, entered into preclinical development for the treatment of pancreatic cancer. BP1003 targets the Stat3 protein and is currently being studied in patient-derived tumor models. Previous ex vivo tumor studies have shown BP1003 to successfully penetrate pancreatic tumors and enhance the efficacy of standard frontline treatments. Bio-Path expects to initiate IND-enabling studies for BP1003 in 2018.

Upcoming Events

·Presentation at the 2018 AACR (Free AACR Whitepaper) Annual Meeting. Bio-Path will present preclinical animal model data at the upcoming AACR (Free AACR Whitepaper) Annual Meeting on Wednesday, April 18, 2018, at the Experimental and Molecular Therapeutics Session, Section 36, from 8:00 a.m. – 12:00 p.m. ET in Chicago. The abstract (#5786) is titled: "Grabbing GRB2: The use of liposome-incorporated Grb2 antisense oligonucleotides as a novel therapy in gynecologic malignancies."

Financial Results for the Full Year Ended December 31, 2017

The Company reported a net loss attributable to common stockholders of $8.1 million, or $0.80 per share, for the year ended December 31, 2017, compared to a net loss attributable to common stockholders of $6.8 million, or $0.73 per share, for the year ended December 31, 2016. The increase was primarily due to the deemed dividend related to the warrant conversion in 2017. The per share amounts above have been adjusted to give effect to the 1-for-10 reverse stock split that occurred on February 8, 2018.

Research and development expenses were $5.5 million for both the years ended December 31, 2017 and December 31, 2016.

General and administrative expenses for the year ended December 31, 2017 increased to $3.5 million, compared to $3.0 million for the year ended December 31, 2016. The increase was primarily due to increased legal and audit fees.

As of December 31, 2017, the Company had cash of $6.0 million, compared to $9.4 million at December 31, 2016. Net cash used in operating activities for the year ended December 31, 2017 was $8.0 million compared to $8.1 million for the comparable period in 2016. Net cash used in investing activities for the year ended December 31, 2017 was $0.5 million. Net cash provided by financing activities for the year ended December 31, 2017 was $5.1 million.

Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these full year 2017 financial results and to provide a general update on the Company. To access the conference call please dial 844-815-4963 (domestic) or 210-229-8838 (international) and refer to the conference ID number 5195559. A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On April 3, 2018 AstraZeneca and Merck & Co., Inc., Kenilworth, N.J., US (Merck: known as MSD outside the US and Canada) reported that the European Medicines Agency has validated for review the Marketing Authorisation Application (MAA) for Lynparza (olaparib) for use in patients with deleterious or suspected deleterious BRCA-mutated, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been previously treated with chemotherapy in the neoadjuvant, adjuvant or metastatic setting (Press release, AstraZeneca, APR 3, 2018, View Source [SID1234525155]).

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This is the first regulatory submission for a poly ADP-ribose polymerase (PARP) inhibitor in breast cancer in Europe. If approved, the identification of a patient’s BRCA status could become a critical step in the management of their disease alongside current consideration of their hormone receptor and HER2 status. The MAA includes data from the randomised, open-label, Phase III OlympiAD trial, which investigated Lynparza versus chemotherapy (physician’s choice of capecitabine, eribulin or vinorelbine). In the trial, Lynparza significantly prolonged progression-free survival compared with chemotherapy and reduced the risk of disease progression or death by 42% (HR 0.58; 95% CI 0.43-0.80; P=0.0009 median 7.0 vs. 4.2 months).

In January 2018, Lynparza was approved by the US Food and Drug Administration for use in the treatment of BRCA-mutated HER2-negative metastatic breast cancer, becoming the first PARP inhibitor to be approved beyond ovarian cancer. Lynparza is available in nearly 60 countries and has been used to treat more than 20,000 patients. AstraZeneca and MSD are working together to bring Lynparza to more patients across multiple cancers.

On April 3, 2018 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported that William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, and Gregory K. Chow, Senior Vice President and Chief Financial Officer, will participate at the H.C. Wainwright Annual Global Life Sciences Conference in Monte Carlo, Monaco on Monday, April 9, 2018 at 11:30 a.m. CEST (Press release, Aptose Biosciences, APR 3, 2018, View Source [SID1234525154]):

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Time: 11:30 a.m. CEST
Date: Monday, April 9, 2018
Location: Le Meridien Beach Plaza Hotel, Monte Carlo, Monaco
Live webcast: View Source
The audio webcasts can also be accessed through the Aptose website at www.aptose.com and will be archived shortly after the live event and available for 90 days.

On April 3, 2018 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, reported that it will present new data on Arginase 1 Deficiency patients at the 2018 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) in Charlotte, North Carolina on Thursday, April 12 (Press release, Aeglea BioTherapeutics, APR 3, 2018, View Source [SID1234525152]). The new data will include additional clinical insights on short-term treatment with repeat doses of pegzilarginase, including baseline standardized assessments of neuromotor function. The Company will conduct a clinical update conference call at 8:30 a.m. ET on April 12.

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Title: Weekly Pegzilarginase Produces Marked and Sustained Reductions in Guanidino Compounds in Adults with Arginase 1 Deficiency: Early Phase 1/2 Results

Presenting Author: Roberto Zori, M.D., University of Florida, Gainesville, FL

Poster Number: 803

Presentation Date and Time: Thursday, April 12, 10:00 a.m. to 11:30 a.m. ET

An electronic version of the presentation will be available for download from the Presentations & Events section of the Company’s investor relations website beginning on the day of the their presentation at the 2018 ACMG Annual Clinical Genetics Meeting.

Conference Call & Webcast Details
Aeglea will hold a clinical update conference call on Thursday, April 12, 2018 at 8:30 a.m. ET. To access the live conference call via phone, please dial 1-877-709-8155 (toll free) within the United States, or 1-201-689-8881 internationally. A replay of the call will be available through April 19, 2018 by dialing 1-877-660-6853 within the United States or 1-201-612-7415 internationally. The conference ID is 13678293.

To access the live and archived webcast of the presentation, please visit the Presentations & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary.

About Pegzilarginase (AEB1102) in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a debilitating urea cycle disorder caused by deficiency of a key arginine metabolizing enzyme that leads to severe and progressive hyperargininemia-related neurological abnormalities, hyperammonemia and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. The Company’s Phase 1 data demonstrated that pegzilarginase reduced blood arginine levels into the normal range, supporting its mechanism of action.

On April 2, 2018 Synlogic (Nasdaq:SYBX), a clinical-stage company applying synthetic biology to probiotic bacteria to develop novel living medicines, reported that the first patient was dosed in its Phase 1b/2a clinical trial of SYNB1020 (Press release, Synlogic, APR 2, 2018, View Source [SID1234525474]). SYNB1020 is a Synthetic Biotic medicine being developed for the treatment of hyperammonemia, associated with cirrhosis and urea cycle disorders (UCDs), which can result in severe and life-threatening consequences for patients. This randomized, double-blind, placebo-controlled study is designed to evaluate the safety and tolerability of SYNB1020, as well as its ability to lower blood-ammonia levels in patients with cirrhosis and elevated blood ammonia.

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"Our recently reported Phase 1 trial of SYNB1020 demonstrated that this Synthetic Biotic medicine was well tolerated and provided a dose-dependent proof of mechanism, functioning as designed in healthy volunteers. We look forward to evaluating the safety, tolerability and therapeutic potential of SYNB1020 in patients with liver disease who have developed cirrhosis," said Aoife Brennan, M.B., B.Ch., Synlogic’s chief medical officer. "There is unmet medical need for additional treatment options for patients with chronic liver disease and we are excited by the potential of SYNB1020 in this indication."

Synthetic Biotic therapies are designed to function in the gastrointestinal tract to convert metabolites that can build up to toxic levels in the blood into harmless metabolites that can be excreted from the body. Elevated blood ammonia levels are toxic to the brain and can have severe consequences, including neurologic crises requiring hospitalization and resulting in irreversible cognitive damage and death. SYNB1020 is designed to consume ammonia and convert it to arginine, an amino acid.

About Synlogic’s Phase 1b/2a Trial of SYNB1020 in Patients with Cirrhosis

This Phase 1b/2a study has two parts:

First, an initial sentinel open-label cohort of subjects with cirrhosis and a Model for End-Stage Liver Disease (MELD) score < 12 will receive orally administered SYNB1020 (5 x 1011 CFU TID) for six days. Subjects will be admitted to an inpatient facility for a run-in diet, baseline assessments, safety monitoring, and collection of blood, urine, and fecal samples for the evaluation of safety, tolerability, pharmacokinetics and pharmacodynamics of treatment. Once safety and tolerability have been established in these subjects, enrollment will be opened to subjects in Part 2.

Part 2 of the trial comprises a randomized, double-blinded, placebo-controlled study in patients with cirrhosis and hyperammonemia. Eligible subjects will be admitted to an inpatient facility for a run-in diet and 24-hour ammonia profile, and those with an elevated ammonia level will proceed with randomization and receive either placebo or orally administered SYNB1020 (5 x 1011 CFU TID) for six days. The primary endpoint of the study is safety and tolerability. In addition, the study will evaluate the effect of SYNB1020 administration on plasma ammonia levels as well as other exploratory endpoints.

Synlogic expects to report top-line data from this trial by year-end 2018. More information on this study can be found at View Source under the study ID NCT03447730.

About Hyperammonemia

Hyperammonemia is a metabolic condition characterized by an excess of ammonia in the blood. In healthy individuals, ammonia is primarily produced in the intestine as a byproduct of protein metabolism and microbial degradation of nitrogen-containing compounds. Ammonia is then converted to urea in the liver and is excreted in urine. However, if the liver’s ability to convert ammonia to urea is compromised, either due to a genetic defect such as UCDs or acquired liver disease that leads to cirrhosis, ammonia accumulates in the blood. Elevated blood ammonia levels are toxic to the brain and can have severe consequences, including neurologic crises requiring hospitalization, irreversible cognitive damage and death.

About Synthetic Biotic Medicines

Synlogic’s innovative new class of Synthetic Biotic medicines leverages the tools and principles of synthetic biology to genetically engineer probiotic microbes to perform or deliver critical functions missing or damaged due to disease. The company’s two lead programs, SYNB1020 and SYNB1618, target hyperammonemia as a result of liver damage or genetic disease, and phenylketonuria, respectively. Patients with these diseases are unable to break down commonly occurring by-products of digestion that then accumulate to toxic levels and cause serious health consequences. When delivered orally, Synthetic Biotic medicines can act from the gut to compensate for the dysfunctional metabolic pathway and have a systemic effect, with the potential to significantly improve symptoms of disease for affected patients. Synlogic has earlier-stage programs that apply the broad potential of its Synthetic Biotic platform in other disease areas, from inflammatory and immune disorders to cancer.