On November 7, 2019 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported financial results for the third quarter ending September 30, 2019 and provided a business update (Press release, Savara, NOV 7, 2019, View Source [SID1234550793]).

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"Based on the totality of evidence, we continue to believe that Molgradex has a clinically meaningful treatment effect on aPAP and we are committed to this program," said Rob Neville, Chief Executive Officer, Savara. "In the near-term, gaining clarity on the best regulatory path forward is our highest corporate priority and we look forward to further collaborating with the FDA and EMA in this regard."

Recent Developments and Upcoming Highlights

Molgradex for aPAP

Announced U.S. Food and Drug Administration (FDA) feedback from Type C meeting that indicated the data included in the briefing package did not provide sufficient evidence of efficacy and safety.
Working in consultation with the FDA to determine the best regulatory path forward for the program.
Presented efficacy and safety results from IMPALA, a Phase 3 study evaluating Molgradex for the treatment of aPAP, at the European Respiratory Society (ERS) International Congress. (Slides from the meeting can be found here.)
Expect to announce data from the open-label follow-up period of IMPALA (weeks 24-48) in Q1 2020.
Plan to initiate discussions with the European Medicines Agency (EMA) regarding the suitability of the IMPALA data for a potential Marketing Authorization Application (MAA).
Molgradex for nontuberculous mycobacterial (NTM) lung infection

Continue to expect top line results from OPTIMA, a Phase 2a clinical study evaluating Molgradex for the treatment of NTM in non-cystic fibrosis (CF) patients, in Q1 2020.
AeroVanc

Anticipate enrollment completion for AVAIL, a pivotal Phase 3 clinical study of AeroVanc for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in CF, in the first half of 2020 with top line results expected in late 2020 or early 2021.
As of November 1, 2019, the study had completed enrollment in the adult population and had enrolled 123 out of a target of 150 patients in the primary analysis population (younger subjects between 6-21 years of age).
Third Quarter Financial Results (Unaudited)

Savara’s net loss attributable to common stockholders for the three months ended September 30, 2019 was $12.4 million, or $(0.30) per share, compared with a net loss attributable to common stockholders of $12.6 million, or $(0.36) per share, for the three months ended September 30, 2018.

Research and development expenses were $9.6 million for the three months ended September 30, 2019, compared with $9.5 million for the three months ended September 30, 2018. The increase was primarily due to approximately $0.6 million in increased costs associated with the development of Molgradex, which was offset by $0.5 million in decreased AeroVanc study costs related to Phase 3 activities.

General and administrative expenses for the three months ended September 30, 2019 were $2.8 million, compared with $3.1 million for the three months ended September 30, 2018. The decrease was primarily due to an approximately $0.6 million noncash stock-based compensation charge incurred in the third quarter of 2018 offset in part by $0.2 million increased personnel costs for the three months ended September 30, 2019.

As of September 30, 2019, Savara had a carrying value of its debt of approximately $25.0 million and had cash, cash equivalents, and short-term investments of approximately $106.3 million. Under the current operating plan, the Company believes this is sufficient capital to fund planned operations well into 2021.

Conference Call/Webcast with Slides

Savara management will host a conference call/webcast with accompanying slides at 4:30 p.m. Eastern Time (ET) / 1:30 p.m. Pacific Time (PT). Shareholders and other interested parties may access the call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and requesting the "Savara Inc." call. A live webcast of the call can be accessed on the Investors page of Savara’s website at View Source

Approximately one hour after the call, a telephone replay will be available and will remain available through November 14, 2019 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10135923. A webcast replay will be available on the Investors page of Savara’s website and will remain available for 30 days.

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.