On November 7, 2019 NanOlogy LLC, a clinical-stage oncology company, reported that interim data were presented last week at the 2019 ACG annual meeting from two of its clinical trials each evaluating endoscopic ultrasound guided fine needle injection (EUS-FNI) of NanoPac (submicron particle paclitaxel): one for treatment of locally advanced pancreatic cancer (LAPC) and the other for treatment of mucinous cystic neoplasms of the pancreas (MCNs) (Press release, NanOlogy, NOV 7, 2019, View Source [SID1234550711]).

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The pancreatic cancer clinical update of intratumoral NanoPac for treatment of LAPC was presented by Simon K. Lo, MD (Cedars-Sinai) as part of the Pancreatic Cancer/Esophagus plenary session.

The Phase 2a dose-rising and expansion pancreatic cancer trial is evaluating the safety and preliminary efficacy of NanoPac delivered intratumorally by EUS-FNI over 6 months in patients with nonresectable LAPC. After completion of the dose-rising phase, the trial has now enrolled 16 of 22 subjects into the dose expansion phase of the trial in which patients are receiving two intratumoral injections of NanoPac 4 weeks apart.

Highlights from Dr. Lo’s presentation on the dose expansion phase of the trial:

No drug-related local or systemic serious adverse events have been reported to date (n=25) including no reports of acute pancreatitis.
Of 7 subjects who have completed the 6-month study to date, one subject had a partial response with restaging from nonresectable to resectable (see video), 3 had stable disease, 1 had progressive disease, and 2 were withdrawn from the study.
Tumor volume decreases ranging from 7% to 76% have been seen in 7 of 11 subjects upon latest mpMRI to date at either the 3- or 6-month time point.
CA19-9 reductions of greater than 20% have been seen in 5 of 11 subjects upon latest measure to date at either the 3- or 6-month time point.
The pancreatic cyst clinical update on intracystic NanoPac for treatment of MCNs of the pancreas was presented during the poster sessions by Mohamed O. Othman, MD (Baylor College of Medicine). Dr Othman’s poster was recognized as a Presidential Poster, a distinction given to fewer than 5% of accepted abstracts each year, and was ultimately named co-winner for high quality, novel, unique and interesting research.

The Phase 2a dose rising and expansion pancreatic cyst trial is evaluating the safety and preliminary efficacy of NanoPac delivered intracystically by EUS-FNI following aspiration in patients with MCNs. The study is also enrolling in the dose expansion phase of the study and patients are receiving two intracystic injections of NanoPac 12 weeks apart.

Highlights from Dr. Othman’s poster presentation on the trial:

No drug-related local or systemic serious adverse events (SAE) have been reported to date (n=15) including no reports of acute pancreatitis. One case of gastric outlet obstruction that was possibly drug-related occurred in a subject who had a recent unrelated endoscopic procedure for hepatobiliary dysfunction. This condition was subsequently added as an exclusion criterion for the trial.
Plasma paclitaxel levels for all subjects analyzed have not exceed 1ng/mL suggesting that NanoPac particles are retained in the cyst over time.
Cyst volume decreases ranging from 8% to 89% have been seen in 9 of 11 subjects at last available imaging at either the 3- or 6-month time point.
Nanology plans to design follow-on clinical trials for both pancreatic cancer and pancreatic cysts in 2020 to further advance the programs toward regulatory submission.

In 2019, an estimated 57,000 new cases of pancreatic cancer will be diagnosed in the U.S. and 46,000 people will die from the disease. Pancreatic cancer is among the deadliest cancers with 9% survival rate at 5 years. It is also one of the few cancers for which no meaningful improvement in survival has been achieved in the last two decades. MCNs are a subset of pancreatic cysts that risk progression to pancreatic cancer. Patients with high risk MCNs may undergo surgical resection of the pancreas to remove the lesion, a complicated procedure associated with mortality and morbidity rates of 2% and 30% respectively. For both the disease itself and one of its common precursors, Nanology investigational drugs may offer a new way to help prevent or treat pancreatic cancer.

In addition to these trials, NanOlogy is advancing its therapeutic platform in preclinical and clinical programs across genitourinary, peritoneal, lung, and dermal cancers. The NanOlogy therapeutic platform is based on a proprietary submicron particle production technology that reduces the size of taxane API crystals by up to 400 times into stable submicron particles of pure drug with exponentially increased surface area and unique geometry. The characteristics of the particles have recently been granted a composition of matter patent valid in the US (9,814,685) and Australia until 2036, and pending globally.

On November 7, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, today reported its financial results for the third quarter ended September 30, 2019, and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, NOV 7, 2019, View Source [SID1234550791]).

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"2019 has been marked by significant progress across our portfolio, including the recent positive data update from our ASPIRO study at the 24th International Annual Congress of the World Muscle Society," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "AT132 continues to show a promising safety and efficacy profile in patients with XLMTM, with the first seven treated patients now ventilator independent, and we remain on track to submit the BLA for AT132 in mid-2020."

Mr. Patterson continued, "Beyond AT132, we are excited about the significant momentum building across our entire pipeline of development candidates. Importantly, we met a major milestone with the submission of our IND for AT845 for the treatment of Pompe disease and remain on track to submit the first IND in our DMD program in the first quarter of next year. With these and other anticipated milestones in our DMD and DM1 programs, we look forward to 2020 as a year of important catalysts for the company and the advancement of our potentially best and first-in-class genetic medicines for devastating neuromuscular diseases."

Recent Achievements & Upcoming Key Events:

AT132 for X-linked Myotubular Myopathy (XLMTM):

On-track to submit a Biologics Licensing Application (BLA) in the United States in mid-2020 and a Marketing Authorization Application (MAA) in Europe in the second half of 2020.
Presented positive data from the ASPIRO dose escalation cohorts at the 24th International Annual Congress of the World Muscle Society. The first seven treated patients were ventilator independent and all patients were making progress against clinically meaningful developmental milestones with four patients walking independently or with support.
AT132 continues to be generally well-tolerated with a manageable safety profile across both dose cohorts.
AT845 for Pompe Disease:

Submitted a US Investigational New Drug application (IND) for AT845 in the third quarter of 2019; application is currently undergoing review with the U.S. Food & Drug Administration (FDA).
Clinical trial site start-up activities underway; initiating a screening study with US trial sites to accelerate patient identification for enrollment into planned Phase 1/2 study.
Plan to present non-clinical data at WORLD Symposium in February 2020.
AT702/AT753/AT751 for Duchenne Muscular Dystrophy (DMD):

IND-enabling dose ranging and toxicology studies underway for AT702; on-track for first quarter 2020 IND submission and plan to initiate a clinical study in the second quarter of 2020.
Held productive face-to-face meeting with FDA to discuss a platform approach to vectorized exon skipping for DMD, which proposes to streamline nonclinical, chemistry, manufacturing and controls (CMC) and clinical development of a common snRNA backbone combined with unique exon-targeting oligonucleotide sequences to address multiple DMD genotypes.
AT753 exon 53 targeting oligonucleotide sequence selected; manufacturing underway to support IND-enabling preclinical studies to be initiated this quarter.
AT751 exon 51 targeting oligonucleotide screening underway; plan to initiate IND-enabling preclinical studies in the first quarter of 2020.
AT466 for Myotonic Dystrophy (DM1):

In vivo vector screening studies continuing to progress.
Plan to submit IND in 2020.
Manufacturing:

Process performance qualification (PPQ) campaign in progress and facility pre-approval inspection (PAI) readiness on track in support of mid-2020 BLA submission for AT132.
Plasmid manufacturing facility GMP readiness activities complete; GMP plasmid production to begin this quarter with initial runs supporting AT702 clinical supply manufacturing.
Third Quarter 2019 Financial Results

Cash Position: As of September 30, 2019, cash, cash equivalents and marketable securities were $351.5 million.
Research and Development Expenses: Research and development expense was $37.6 million for the three months ended September 30, 2019, compared to $29.9 million for the same period in 2018, an increase of $7.7 million. The increase was primarily attributable to higher program expenses for AT845, new programs AT466 and AT702 initiated in 2019, and additional R&D headcount to advance clinical and pre-clinical programs. Included in R&D expense for the three months ended September 30, 2019 was $3.0 million of non-cash stock-based compensation expense, compared to $2.6 million in the same period in 2018. For the nine months ended September 30, 2019, research and development expense was $114.8 million compared to $76.2 million for the same period in 2018.
General and Administrative Expenses: General and administrative expense was $10.2 million for the three months ended September 30, 2019, compared to $7.8 million for the same period in 2018. The increase was primarily attributable to headcount increases and infrastructure investment to support growth. Included in G&A expense for the three months ended September 30, 2019 was $2.7 million of non-cash stock-based compensation expense, compared to $2.0 million in the same period in 2018. For the nine months ended September 30, 2019, general and administrative expense was $32.0 million compared to $20.6 million for the same period in 2018.
Net Loss: Net loss was $45.7 million for the three months ended September 30, 2019 compared to $36.3 million for the same period in 2018. Basic and diluted net loss per share for the three months ended September 30, 2019, was $1.00 compared with $0.97 for the same period in 2018. For the nine months ended September 30, 2019, net loss was $139.9 million, compared to $93.2 million for the same period in 2018. Basic and diluted net loss per share for the nine months ended September 30, 2019 was $3.14, compared with $2.57 for the same period in 2018.
Conference Call

At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its third quarter 2019 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 8575077 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

On November 7, 2019 Eli Lilly and Company (NYSE: LLY) reported the pricing terms of its previously announced cash tender offer for up to $2,000,006,000 aggregate principal amount of its outstanding debt securities (Press release, Eli Lilly, NOV 7, 2019, View Source [SID1234550846]).

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Holders of notes who tendered, and did not validly withdraw, their notes on or before 5:00 p.m., New York City time, on November 6, 2019 (the early tender date), and which are accepted for purchase by Lilly, are eligible to receive the total consideration. The total consideration for each series of notes is based on the applicable reference yield plus a fixed spread, in each case as set forth in the table below, and is payable to holders of the notes listed in the table below who validly tendered and did not validly withdraw their notes on or before the early tender date and which are accepted for purchase by Lilly. The reference yields listed in the table were determined at 10:00 a.m., New York City time, on November 7, 2019. The total consideration for each series of notes includes an early tender premium of $30 per $1,000 principal amount of notes validly tendered and not validly withdrawn by such holders and accepted for purchase by Lilly.

(1) Per $1,000 principal amount of notes that are tendered and accepted for purchase.

(2) The total consideration includes the early tender premium of $30 per $1,000 principal amount of notes.

(3) The maximum principal amount of 3.950% Notes due 2049 that will be purchased by Lilly is $200,000,000.

(4) The maximum principal amount of 4.150% Notes due 2059 that will be purchased by Lilly is $200,000,000.

(5)The maximum principal amount of 2.350% Notes due 2022 that will be purchased by Lilly is $250,000,000.

All payments for notes purchased in connection with the early tender date will also include accrued and unpaid interest on the principal amount of notes tendered up to, but not including, the initial settlement date, which is currently expected to be November 8, 2019.

Holders of notes who validly tender their notes after the early tender date will, if such notes are accepted by Lilly, receive the tender consideration, which is equal to the total consideration minus $30 per $1,000 principal amount of notes tendered by such holders and accepted for purchase by Lilly. Accrued and unpaid interest up to, but excluding, the applicable settlement date will be paid in cash on all validly tendered notes accepted and purchased by Lilly in the tender offer.

The tender offer is scheduled to expire at 11:59 p.m., New York City time, on November 21, 2019, unless extended or earlier terminated.

In accordance with the terms of the tender offer, the withdrawal date was 5:00 p.m., New York City time, on November 6, 2019. As a result, tendered notes may no longer be withdrawn, except in certain limited circumstances where additional withdrawal rights are required by law.

The tender offer is being conducted upon the terms and subject to the conditions set forth in the Offer to Purchase, dated October 24, 2019, and the related Letter of Transmittal, each as supplemented by the press release dated November 7, 2019 that increased the size of the tender cap.

Lilly has retained Citigroup Global Markets Inc. and Morgan Stanley & Co. LLC to serve as lead dealer managers for the tender offer and BNP Paribas Securities Corp. and J.P. Morgan Securities LLC to serve as co-dealer managers. Lilly has retained Global Bondholder Services Corporation to serve as tender agent and information agent for the tender offer.

Requests for documents relating to the tender offer may be directed to Global Bondholder Services Corporation by telephone at (866) 470-3900, by email at [email protected] or in writing at 65 Broadway, Suite 404, New York, NY 10006. Questions regarding the tender offer may be directed to Citigroup Global Markets Inc. at (212) 723-6106 or to Morgan Stanley & Co. LLC at (800) 624-1808.

On November 7, 2019 Targovax ASA (OSE: TRVX), a clinical stage biotechnology company developing immune activators to target hard-to-treat solid tumors, reported its third quarter 2019 results (Press release, Targovax, NOV 7, 2019, View Source [SID1234550509]).

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A meeting for investors, analysts and press will take place in Oslo today at 10:00 CET (details below).

HIGHLIGHTS FOR THE THIRD QUARTER 2019

Targovax announced data from part 1 of the ONCOS-102 trial in checkpoint inhibitor refractory advanced melanoma, showing validated clinical responses in three out of nine patients (33% ORR), including one patient with a complete response and immune activation in all nine patients
The expansion part of the phase I/II trial of ONCOS-102 in combination with the checkpoint inhibitor Imfinzi in patients with advanced peritoneal malignancies opened for enrollment as the dose escalation part of the trial concluded successfully
Targovax announced the opening of Oslo University Hospital as site for ONCOS-102 trial in melanoma
POST-PERIOD HIGHLIGHTS

In October, Targovax was selected for oral presentation at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019 Annual Meeting. The presentation will be given bv Dr. Alexander Shoushtari, Principal Investigator of ONCOS-102 trial in melanoma, Memorial Sloan Kettering Cancer Center, NYC
Øystein Soug, CEO commented: "Oncolytic viruses are increasingly recognized as an important future class of immune activators, and Targovax is well positioned as one of the leaders in this rapidly evolving field. Currently, our main focus is to deliver the expected data read-outs from our ongoing ONCOS-102 combination trials in 2020, which we hope will solidify Targovax position as a leader in the oncolytic virus space."

Presentation

The presentation will take place at 10:00 CET at:

Hotel Continental
Stortingsgaten 24/26
0117 Oslo

The presentation will also be webcast live and can be accessed here.

Reporting material

Targovax 3Q presentation

Targovax 3Q report

The quarterly report and presentation are also available at the website www.targovax.com.

On November 7, 2019 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the quarter ended September 30, 2019 and highlighted recent business developments (Press release, GlycoMimetics, NOV 7, 2019, View Source [SID1234550601]). Quarter-end cash and cash equivalents were $170.9 million.

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"In the third quarter of 2019, we continued to progress the late-stage clinical development of our wholly-owned product candidate, uproleselan. Our Company-sponsored Phase 3 trial in relapsed or refractory AML patients and the NCI-sponsored Phase 3 trial for newly diagnosed patients with AML both advanced during the quarter. We are also working with the Duke Cancer Institute towards initiating a single center, proof-of-mechanism Phase 1b trial for GMI-1359, our dual antagonist of E-selectin and CXCR-4, in breast cancer patients with bone metastases," said Rachel King, GlycoMimetics’ Chief Executive Officer.

Ms. King continued, "We are also very much looking forward to the ASH (Free ASH Whitepaper) meeting in December, which has always been an important conference for us, and this year is no different. The key takeaway for us at this year’s ASH (Free ASH Whitepaper) meeting is that data from multiple preclinical and clinical settings show that E-selectin ligand expression on leukemic cells is correlated with poor survival in AML. The data indicate that E-selectin ligand expression is a key driver of environmental-mediated chemoresistance in AML and suggest that uproleselan has the potential to break this chemoresistance, and thereby improve clinical outcomes. Based on this expanding dataset, we are exploring how use of biomarkers may help us in advancing our clinical program.

"Finally, as previously announced, Pfizer reported that its Phase 3 clinical trial evaluating rivipansel in SCD failed to meet its primary endpoint and key secondary endpoints. Of course, this is disappointing, but for some time our operational focus has been on our uproleselan program in AML, and we continue to focus our efforts on diligently and efficiently progressing that exciting clinical program," Ms. King added.

Key Third-Quarter 2019 and Recent Operational Developments:

GlycoMimetics’ pivotal Phase 3 trial of uproleselan in relapsed/refractory AML continued to initiate and activate clinical sites and to enroll patients in the U.S., Australia and now in Europe.
Investigators continued to enroll patients in the NCI-sponsored Phase 3 clinical trial designed to evaluate uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy.
Pfizer announced that the Phase 3 clinical trial evaluating rivipansel in SCD failed to meet the primary endpoint and key secondary endpoints.
As part of a commitment to eliminate certain non-core research and development spending, GlycoMimetics discontinued plans to collaborate with the Haemato Oncology Foundation for Adults in the Netherlands on a Phase 2 trial of uproleselan in newly-diagnosed patients unfit for chemotherapy.
The Company continued to work closely with the Duke Cancer Institute to initiate a Phase 1b proof-of-mechanism clinical trial of GMI-1359 in individuals with breast cancer whose tumors have spread to bone. The trial will evaluate safety and biomarkers of cancer cell mobilization in individuals with hormone receptor positive metastatic breast cancer. The trial will be conducted at Duke University and is expected to initiate during the fourth quarter.
Third Quarter 2019 Financial Results:

Cash position: As of September 30, 2019, GlycoMimetics had cash and cash equivalents of $170.9 million as compared to $209.9 million as of December 31, 2018.
R&D Expenses: The Company’s research and development expenses increased to $10.7 million for the quarter ended September 30, 2019 as compared to $9.7 million for the third quarter of 2018. This increase was primarily the result of expenses relating to the Company’s Phase 3 clinical trial of uproleselan in relapsed or refractory AML patients and supporting the clinical trials of uproleselan conducted by or in collaboration with third parties.
G&A Expenses: The Company’s general and administrative expenses increased to $3.4 million for the quarter ended September 30, 2019 as compared to $2.8 million for the third quarter of 2018. The increase was due to higher patent, legal and non-cash stock-based compensation expenses.
Shares Outstanding: Shares outstanding as of September 30, 2019 were 43,359,949.
The Company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) with passcode 9845948. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. In the fourth quarter of 2019, the Company plans to initiate an exploratory clinical trial in individuals with breast cancer whose tumors have spread to bone.