Neurocrine Biosciences to Present at the Morgan Stanley 18th Annual Global Healthcare Conference

On September 8, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Morgan Stanley 18th Annual Global Healthcare Conference at 1:30 p.m. ET on Monday, September 14, 2020 (Press release, Neurocrine Biosciences, SEP 8, 2020, View Source [SID1234564742]). Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

GlycoMimetics to Present at Upcoming H.C. Wainwright & Co Annual Global Investment Virtual Conference

On September 8, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that Chief Executive Officer Rachel King will present at the upcoming H.C. Wainwright & Co 22nd Annual Global Investment Virtual Conference taking place September 14-16, 2020 (Press release, GlycoMimetics, SEP 8, 2020, https://ir.glycomimetics.com/news-releases/news-release-details/glycomimetics-present-upcoming-hc-wainwright-co-annual-global [SID1234564741]). The presentation will be available on the company’s website at the "Investors" tab for 30 days, beginning Monday, September 14 at 2:30 p.m. EDT.

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To access the live webcast and subsequent archived recordings for the presentation, please visit the GlycoMimetics website at View Source

Astex Pharmaceuticals and MD Anderson Announce Strategic Collaboration to Accelerate Clinical Evaluation of Therapies for Patients with Leukemia

On September 8, 2020 Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Tokyo, Japan, and The University of Texas MD Anderson Cancer Center reported a strategic collaboration agreement aimed at accelerating the clinical evaluation of Astex’s pipeline of products for patients with certain types of leukemia, including myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) (Press release, Astex Pharmaceuticals, SEP 8, 2020, View Source [SID1234564740]). The collaboration will combine MD Anderson’s clinical trials infrastructure and expertise with Astex’s clinical pipeline products.

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The initial focus will be on evaluating Astex’s oral decitabine and cedazuridine hypomethylating agent (INQOVI) in combinations with other therapies. INQOVI recently was approved by the US FDA and by Health Canada for the treatment of intermediate- and high-risk MDS and CMML.

"MD Anderson is dedicated to providing the best treatment options to our patients, and there is tremendous interest in evaluating how a new generation of oral targeted therapies might work in combination to treat those with leukemia," said Guillermo Garcia-Manero, M.D., Professor and Chief of Section of Myelodysplastic Syndromes, Department of Leukemia at MD Anderson. "This collaboration with Astex will allow us to expand those studies with the ultimate goal of providing patients with oral drug combinations that have the potential of improving clinical outcomes."

Under the collaboration agreement, MD Anderson and Astex will design new clinical studies to be conducted at MD Anderson. Astex will provide funding, test compounds and other support. The collaboration will be overseen by a joint steering committee.

"MD Anderson has been a collaborator with Astex on multiple clinical studies for our pipeline products," said Mohammad Azab, president & chief medical officer of Astex. "We are delighted to be entering into this new collaboration and look forward to continuing to advance this important area of clinical research."

About INQOVI (See View Source)

INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Myelosuppression: Fatal and serious myelosuppression can occur with INQOVI. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%.

Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.

Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors, and anti‑infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

Embryo-Fetal Toxicity: INQOVI can cause fetal harm. Advise pregnant women of the potential risk to a fetus. Advise patients to use effective contraception during treatment with INQOVI and for 6 months (females) or 3 months (males) after last dose.

ADVERSE REACTIONS

Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.

The most common adverse reactions (≥ 20%) were fatigue, constipation, hemorrhage, myalgia, mucositis, arthralgia, nausea, dyspnea, diarrhea, rash, dizziness, febrile neutropenia, edema, headache, cough, decreased appetite, upper respiratory tract infection, pneumonia, and transaminase increased. The most common Grade 3 or 4 laboratory abnormalities (>50%) were leukocytes decreased, platelet count decreased, neutrophil count decreased, and hemoglobin decreased.

USE IN SPECIFIC POPULATIONS

Lactation: Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for at least 2 weeks after the last dose.

Renal Impairment: No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).

Synlogic to Present at Upcoming Virtual Banking Conferences

On September 8, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, reported that Aoife Brennan, M.B. Ch.B., Synlogic’s president and chief executive officer, and other members of the executive team will present at the following virtual banking conferences (Press release, Synlogic, SEP 8, 2020, View Source [SID1234564739]):

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HC Wainwright 22nd Annual Global Investor Conference: The Company will present at 12:00 noon on Tuesday, September 15, 2020.
Oppenheimer Fall Healthcare Life Sciences & Med Tech Summit: The Company will present at 4.10 pm on Wednesday, September 23, 2020
Jefferies IBD Therapeutics Summit: The Company will present at a time to be determined on Wednesday, September 23, 2020.
These are virtual events. A live webcast of the presentations can be accessed under "Event Calendar" in the Investors & Media section of the Company’s website. An archived copy of the webcast will be available on the Synlogic website for approximately 30 days after the event.

Positive Initial Data From a Phase 1 Trial With Transgene’s Oncolytic Virus TG6002

On September 8, 2020 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported first positive data from the Phase 1 clinical trial evaluating the intravenous administration of TG6002 in patients with advanced gastrointestinal tumors (Press release, Transgene, SEP 8, 2020, View Source [SID1234564738]).

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The trial aims at confirming the good tolerability of ascending doses of intravenous TG6002 and at providing the first translational data from this novel route of administration.

The independent safety review committee met and recommended that the trial be continued and that the dose level be increased to a 3×109 pfu in the absence of dose-limiting toxicity of TG6002 at the 109 pfu dose.
The first translational data show that, when administered intravenously, TG6002 circulates transiently in the patient’s bloodstream and induces the production of 5-FU at therapeutic doses. The production of 5-FU results from the expression of the FCU1 gene, integrated into the TG6002 genome, as the virus replicates selectively in the tumor cells.
TG6002 has been engineered to combine the oncolytic and immunogenic actions of the virus with the intra-tumoral production of 5-FU, a chemotherapeutic agent. This production is achieved by converting the pro-drug 5-FC (administered orally) into 5-FU, which is expected to reach high concentrations within the tumor. This mechanism of action is based on the expression of the proprietary FCU1 gene that has been integrated with the genome of TG6002.

"This is the first clinical study demonstrating that Transgene’s Vaccinia Virus from the Invir.IO platform is able to reach the tumor when administered intravenously. Importantly, through the production of 5-FU at therapeutic doses, we have also demonstrated the functionality of the FCU1 transgene integrated into the genome of TG6002. These positive initial translational data, combined with a satisfactory tolerability profile, have allowed us to continue this Phase 1 clinical trial and evaluate higher doses of this promising new drug candidate," said Dr. Maud Brandely, MD, PhD, Chief Medical Officer of Transgene.

With this clinical trial, Transgene aims to demonstrate the benefits of intravenous administration of next generation oncolytic viruses such as TG6002 and the candidates derived from the Invir.IO platform, in order to extend the use of these therapies to many solid tumors, including gastrointestinal cancers. This broad utility contrasts with first-generation oncolytic viruses that have only been approved to be given via intra-tumoral administration, which restricts their use to easily accessible tumors.

TG6002 is also being evaluated in another Phase 1/2a clinical trial where it is being given by Intrahepatic Artery Infusion in patients with advanced colorectal cancer with liver metastases (CRLM). In order to respond to the Covid-19 pandemic, the trial’s clinical site had temporarily suspended patient inclusion; recruitment resumed in early September 2020.

About the trial

This trial is a single-arm open-label Phase 1/2 trial evaluating the safety and tolerability of multiple ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug that can be converted in 5-FU, its active metabolite. Based on the safety profile of TG6002, several dose levels have been added to the initial Phase 1 clinical protocol. At the end of this Phase 1 part, Phase 2 patients will receive the recommended dose of TG6002. The trial has safety as primary endpoint for the Phase 1 part and efficacy for the Phase 2 part. The trial also evaluates pharmacokinetic properties and biodistribution of TG6002, along with immune modulation of the tumor micro-environment. This European study will enroll up to 40 patients suffering from advanced gastrointestinal carcinomas who have failed and/or are intolerant to standard therapeutic options in the Phase 1 part. Patients with colon cancer and liver metastases will be enrolled in the phase 2 part.

Dr. Philippe Cassier, M.D., PhD, head of the early-phase trials unit at Centre Léon Bérard (Lyon, France) is the principal investigator of the trial.

About TG6002

TG6002 has been engineered to directly kill cancer cells (oncolysis), to enable the production of a chemotherapy agent (5-FU) within the tumor, and to elicit an immune response by the body against the tumor cells. In preclinical experiments, TG6002 has been shown to induce the shrinkage of the primary tumor as well as the regression of distant metastases1.

The production of 5-FU directly in the tumor aims at achieving a better anti-tumor effect with limited chemotherapy-induced side effects.

­ TG6002 induces the production of 5-FU in the cancer cells it has infected, by enabling the local conversion of the pro-drug 5-FC (administered orally) into 5-FU. 5-FU is a common chemotherapy for patients with CRLM. This mechanism of action is based on the expression of the proprietary FCU1 gene that has been integrated with the genome of TG6002, as the virus replicates selectively in the tumor cells.
­ 5-FU is associated with side effects that can lead to treatment discontinuation. With TG6002, 5-FU is produced within the tumor where it is expected to be present at a high concentration level in contrast to the very low levels anticipated in the rest of the patient’s body.