On November 7, 2019 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that management will present at the Credit Suisse Healthcare Conference on Tuesday, November 12, 2019 at 1:30 p.m. ET (Press release, Vertex Pharmaceuticals, NOV 7, 2019, View Source [SID1234550792]).

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Management’s remarks will be available live through Vertex’s website at www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

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 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company committed to giving patients back their innate ability to fight cancer, reported that its Investigational New Drug application (IND) has cleared the required 30-day review by the U.S. Food and Drug Administration (FDA) and is in effect for a Phase 1/2a clinical trial of AFM24, a tetravalent, bispecific epidermal growth factor receptor (EGFR)- and CD16A-binding innate cell engager, in patients with advanced cancers known to express EGFR (Press release, Affimed, NOV 7, 2019, View Source [SID1234550790]).

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"The IND clearance of AFM24 enables us to proceed with our planned Phase 1/2a study aimed at establishing safety and identifying initial signals of efficacy in patients with EGFR-expressing solid tumors," said Dr. Adi Hoess, Chief Executive Officer of Affimed. "There is a tremendous need for novel immuno-oncology approaches and based on its novel mechanism of activating the innate immune system, AFM24 has the potential to address limitations, such as toxicities or resistance, associated with other EGFR-targeted therapies."

The initial goal of the planned Phase 1/2a study is to determine the maximum tolerated dose and recommended Phase 2 dose of AFM24, as well as to evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy. The second part of the study is designed to evaluate the preliminary efficacy of AFM24 in patients with select solid tumor subtypes. The study is planned to initiate in the first half of 2020.

AFM24 has the potential to provide a meaningful benefit to a broad set of patients suffering from EGFR-expressing tumors, including those patients who currently are not being addressed by existing EGFR-targeted therapies. According to internal market research, leading clinical experts across multiple cancer indications see a tremendous need for novel immuno-oncology approaches for the treatment of solid tumors. Preclinical data showed AFM24’s ability to bridge NK cells and macrophages to EGFR-expressing tumor cell lines and induce cell lysis through antibody-dependent cellular cytotoxicity (ADCC), independent of RAS mutational status, and antibody-dependent cellular phagocytosis (ADCP). In addition, AFM24 enhanced tumor infiltration of NK cells and elicited dose-dependent anti-tumor efficacy in in vivo tumor models. Treatment of cynomolgus monkeys with AFM24 showed a favorable safety profile, even when the animals were treated at high dose levels, demonstrating AFM24’s potential to have lower toxicities in humans compared to other EGFR-targeted therapeutics.

About AFM24

AFM24, a tetravalent, bispecific EGFR- and CD16A-binding innate cell engager from Affimed’s fit-for-purpose ROCK platform, is designed to address limitations associated with other EGFR-targeted therapies, such as toxicities or resistance, by using a new mechanism of action to target EGFR-expressing solid tumors through activation of innate immunity rather than inhibition of EGFR-mediated signal transduction.

On November 7, 2019 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company committed to giving patients back their innate ability to fight cancer, reported that Genentech, a member of the Roche Group, has exercised its final option for an exclusive target under the companies’ collaboration agreement to develop and commercialize novel NK cell engager-based immunotherapeutics generated from Affimed’s ROCK platform to treat multiple cancers (Press release, Affimed, NOV 7, 2019, View Source [SID1234550789]). The target selection triggers a milestone payment, in an undisclosed amount, to Affimed from Genentech.

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"The designation of the final target marks another milestone for the collaboration, which brings together Genentech’s deep understanding of cancer immunology with Affimed’s expertise in drug discovery and development of innate cell engagers," said Dr. Adi Hoess, Chief Executive Officer of Affimed. "We look forward to achieving further progress toward generating novel therapies that leverage the full potential of the innate immune system to help people living with cancer."

About the Strategic Collaboration Agreement with Genentech

Affimed’s strategic, multi-target collaboration with Genentech was announced in August 2018. Under this agreement, Affimed applies its fit-for-purpose Redirected Optimized Cell Killing (ROCK) platform and engineering expertise to discover and advance NK cell engager-based immunotherapeutics against certain targets selected by Genentech. Affimed and Genentech collaborate on the discovery, early research and late-stage research phases. Genentech is responsible for clinical development and commercialization worldwide. Affimed received $96 million in upfront and committed funding from Genentech in the fourth quarter of 2018 and may be eligible to receive up to an additional $5 billion in development, regulatory and commercial milestone payments, plus royalties on sales of any developed products.

On November 7, 2019 Phanes Therapeutics and Phanes Biopharmaceuticals ("Phanes") reported an important strategic alliance with Fosun Kite Biotechnology ("Fosun Kite") (Press release, Phanes Therapeutics, NOV 7, 2019, View Source [SID1234550786]). Under the alliance agreement, Phanes will exclusively provide Fosun Kite a collection of all high-affinity humanized antibodies targeting two novel cancer antigens for the development and commercialization of innovative CAR-T products for solid tumor treatments in China (including Hong Kong SAR, Macau SAR and Taiwan). Under the terms of the agreement, Phanes is eligible for upfront and milestone payments as well as royalty fees in China. Fosun Kite also owns the Right of First Negotiation for global commercialization of the antibody use rights outside of China.

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Dr. Ming Wang, CEO of Phanes commented: "This is our second deal of the year following the out-licensing agreement with Hanmi Pharmaceuticals in September. We are very excited about the opportunity to partner with Fosun Kite. Fosun Kite has built tremendous capabilities and expertise in cell therapy while leveraging the CAR-T experience of Kite Pharma, a global pioneer in CAR-T therapy, and they understand what innovation is like. Becoming a partner of Fosun Kite is an important validation of the scientific strength and capability of Phanes Therapeutics in delivering robust biological molecules that are suitable for therapeutic use. Our mission is to become a source of innovation for the biotech and pharma industry in the oncology field, and our efforts since the inception of the company 3 years ago have led to a very impressive pipeline, including monoclonal antibodies (mAbs), bispecific antibodies and single-chain variable fragments (scFvs), among which one mAb and one bispecific antibody have entered the CMC stage. In addition, we have established the PACbody platform, a proprietary technology for building bispecific antibodies. While we are aggressively advancing our internal programs, we are starting to reach out for various types of partnerships to maximize the positive impact of our pipeline in the fight against cancer. Moving forward, deal-making will be one of our major focuses. I’m very confident that we are going to have more deals added to our portfolio."

"We are quite pleased to have Phanes as a partner to advance the discovery and development of our CAR-T pipeline for solid tumor treatment. Phanes has rich experiences in development of humanized therapeutic antibodies as one of the leaders in the industry," said Dr. Richard Wang, Fosun Kite CEO, "In treating patients with hematologic malignancies, CAR-T has already demonstrated its superior efficacies. But in solid tumor treatment the world is still striving for a breakthrough of the technology. With the continuous progress of CAR-T, we believe it has tremendous potential. Fosun Kite has built unparalleled technology platforms and experiences through Yescarta’s commercialization in China, hence we are confident to start building a sustainable R&D pipeline while driving Yescarta’s NDA approval forward in China. With our newly opened R&D Center and the expansion of top R&D talents from China and overseas, we have established a firm foundation to realize this R&D strategy. We look forward to a successful partnership with Phanes, leading to more immuno-cell therapy product development to benefit cancer patients."