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 NBE-Therapeutics AG and SOTIO a.s. reported that SOTIO has elected a second target for the development of a next generation antibody-drug conjugate (ADC) under their existing license and collaboration agreement. NBE and SOTIO will collaborate on the discovery, non-clinical development and manufacturing of this second undisclosed target (Press release, NBE Therapeutics, NOV 7, 2019, View Source [SID1234573380]). The development will be based on NBE’s proprietary antibody drug conjugate platform, including NBE’s site-specific SMAC-technologyTM conjugation and its new, highly potent anthracycline toxin platforms. SOTIO will take global responsibility for clinical development, registration and commercialization of the ADC products.

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NBE-Therapeutics’ new iADCTM platform creates highly potent and safe immune-stimulatory ADCs with an anthracycline payload, inducing a long-lasting immunological anti-tumor effect. It has shown unprecedented preclinical data in efficacy, as well as safety, in multiple pipeline programs. Its lead program NBE-002 against the ROR1 target has successfully passed the GLP tox study and is in the final stages of CMC. It is expected to begin its first
in-human study by mid-2020 for triple negative breast cancer and lung cancer, as well asother cancer indications.

NBE is eligible for the option exercise fee, as well as milestone payments and royalties based on global net product sales. In addition, NBE will be reimbursed for its R&D expenses incurred in connection with product development in collaboration with SOTIO.

Dr. Ulf Grawunder, CEO of NBE-Therapeutics commented: "We are very enthusiastic that SOTIO has nominated a second ADC program under the existing collaboration agreement between SOTIO and NBE, confirming the high quality of our ADC platform. We have now created a robust and scalable ADC platform that enables us to leverage iADC development in clinical studies for multiple programs. The collaboration with SOTIO over the past years has further strengthened our technology and we are very proud to have them on board for a second iADC development program."

"Based on the very encouraging data from our first collaboration target with NBE and the SO-N102 program, as well as the data of NBE’s proprietary program NBE-002, we have now exercised the second target option in the collaboration," mentions Dr. Radek Spisek, CEO of SOTIO. "NBE’s product platform addresses the key issues of today’s antibody-drug conjugates and has the potential to provide new and superior treatment options for cancer patients."

On November 7, 2019 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) reported its financial and operating results for the third quarter ended September 30, 2019 (Press release, AEterna Zentaris, NOV 7, 2019, View Source [SID1234553274]).

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All Amounts are in U.S. Dollars

Highlights

Net loss for the third quarter of 2019 was $0.3 million as compared to net loss of $2.5 million for the same period in 2018. Net loss for the nine-months ended September 30, 2019 was $5.0 million as compared to net income of $9.3 million for the same period in 2018.

As of September 30, 2019, we had $10.9 million of unrestricted cash and cash equivalents.

On September 20, 2019, the Company entered into a securities purchase agreement with U.S. institutional investors to purchase $5.0 million (before transaction costs of $0.8 million) of its common shares in a registered direct offering and warrants to purchase common shares in a concurrent private placement. The combined purchase price for one common share and one warrant was $1.50. Under the terms of the securities purchase agreement, the Company sold 3,325,000 common shares. In a concurrent private placement, the Company issued warrants to purchase up to an aggregate of 3,325,000 common shares. The warrants are exercisable commencing six months from the date of issuance, have an exercise price of $1.65 per share and expire 5 years following the date of issuance.

The Company earned royalty income for the nine-month period ending September 30, 2019 of $0.03 million from the license of Macrilen (macimorelin) and we invoiced Novo Nordisk (Novo) $0.8 million for its share of our pediatric clinical study (the "PIP study") costs and $1.1 million for supply chain costs.

During the third quarter of 2019, Novo confirmed that it had initiated a thorough review on support, reimbursement, distribution and marketing arrangements regarding Macrilen (macimorelin) in order to identify improvements to the Macrilen commercialization plans. We continue to work with Novo on addressing the slower than expected U.S. sales to date.

The initial phase of the Macrilen macimorelin PIP study (the "P01 Dose Ranging Study") continued to progress with the first two-thirds of subjects having completed the protocol. We currently expect to complete the P01 Dose Ranging Study in the first quarter of 2020.
Summary of Third Quarter Results and Year-to-date Third Quarter Results

For the three-month period ended September 30, 2019, we reported a consolidated net loss of $0.3 million, or $0.02 loss per common share (basic), as compared with a consolidated net loss of $2.5 million, or $0.15 loss per common share, for the three-month period ended September 30, 2018. The $2.2 million improvement in net results is primarily from a gain in fair value of warrant liability of $2.1 million and a decline in operating expenses of $1.1 million and an increase in gross income of $0.1 million and $0.1 million increase in foreign currency exchange rates, offset by $0.5 million movement in tax recovery and an increase in net finance costs of $0.7 million.

For the nine-month period ended September 30, 2019, we reported a consolidated net loss of $5.0 million, or $0.31 loss per common share, as compared with a consolidated net income of $9.3 million, or $0.57 income per common share (basic), for the nine-month period ended September 30, 2018. The $14.3 million decline in net results is primarily from a reduction of $24.3 million in gross income offset by $6.1 million in tax expense, $3.0 million decline in operating expenses and $0.9 million increase in net finance income.

On June 6, 2019, the Company announced that it was reducing the size of its German workforce and operations to more closely reflect the Company’s ongoing commercial activities in Frankfurt. This restructuring affects 8 employees in Frankfurt, Germany and resulted in $0.8 million of severance costs that are expected to be paid by January 31, 2020.

In July 2019, Michael Ward resigned as managing director of AEZS Germany and Dr. Klaus Paulini assumed this role. In August 2019, Jonathan Pollack resigned as a director of Aeterna Zentaris Inc. and, in September 2019, Brian Garrison, resigned as a Senior Vice President, Global Commercial Operations of Aeterna Zentaris Inc. Subsequent to quarter-end, on October 4, 2019, Dr. Klaus Paulini was announced as President and Chief Executive Officer of the Company, replacing Michael Ward who is entitled to severance of approximately $0.5 million. Dr. Paulini was also appointed as a Director of Aeterna Zentaris Inc. at that time.

Condensed Interim Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the condensed interim consolidated financial statements as at September 30, 2019 and for the three-month and nine-month periods ending September 30, 2019 and 2018 and management’s discussion and analysis of financial condition and results of operations three-month and nine-month periods ending September 30, 2019, will be found at www.zentaris.com in the "Investors" section and at the Company’s profile at www.sedar.com and www.sec.gov.

The following tables set out summary consolidated financial information for the periods indicated. The results of operations for interim periods are not necessarily indicative of the results to be expected for a full year or any future period. The information presented herein does not contain disclosures required by IFRS for consolidated financial statements and should be read in conjunction with the Company’s audited annual consolidated financial statements for the year ended December 31, 2018.

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.