On November 7, 2019 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported third quarter 2019 financial results (Press release, CytomX Therapeutics, NOV 7, 2019, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-third-quarter-2019-financial" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-third-quarter-2019-financial" rel="nofollow">View Source [SID1234550794]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As of September 30, 2019, CytomX had cash, cash equivalents and short-term investments of $325.7 million.

"The CytomX clinical pipeline made excellent progress in Q3 as we continued to advance multiple Probody therapeutic programs across the portfolio," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "The initiation of a Phase 2 trial of our anti-PD-L1 Probody CX-072 in combination with ipilimumab in patients with relapsed refractory melanoma marks our ongoing evolution into a product-focused company seeking to realize the full potential of our novel technology platform. Our first in class Probody Drug Conjugate programs, CX-2009 and CX-2029, also continued to move forward and our pipeline overall is positioned for significant data updates in 2020."

Business Highlights and Recent Developments

CX-072 Anti-PD-L1 Probody Therapeutic Clinical Program

In October, CytomX announced the initiation of the PROCLAIM (Probody Clinical Assessment In Man) CX-072-002 Phase 2 study evaluating the efficacy and tolerability of the anti-PD-L1 Probody CX-072, in combination with the anti-CTLA-4 antibody, ipilimumab, in patients with relapsed or refractory melanoma. The study utilizes a Simon Two-Stage design with approximately 40 patients being enrolled into Stage 1. CytomX anticipates initial data from Stage 1 in 2020. Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03993379.
The Company also announced in October updated clinical data from the Phase 1 PROCLAIM-CX-072-001 dose-finding study of CX-072 in combination with ipilimumab. With enrollment complete, 27 evaluable patients had received ipilimumab (3, 6 or 10 mg/kg) combined with CX-072 (0.3, 1, 3 or 10 mg/kg), with the study achieving a disease control rate (stable disease or better) of 37%. 5 patients achieved confirmed objective responses by RECIST v1.1, including one complete response, for an overall response rate (ORR) of 19% in this heavily pretreated patient population. The median duration of response was 14.6 months (1.9 – 21.2 months) with 4 of the 5 responders still on treatment as of the latest data snapshot. Of the 27 patients treated across all doses, Grade 3/4 treatment related adverse events (TRAEs) were reported in 9 (33%) patients. Grade 3/4 immune-related adverse events (irAEs) were reported in 3 (15%) patients.
Enrollment within the monotherapy cohorts of the PROCLAIM-CX-072-001 study is complete with evaluation of the activity and tolerability of CX-072 monotherapy continuing with ongoing treatment in select cohorts. Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03013491.
CX-2009 Anti-CD166 Probody Drug Conjugate Clinical Program

CytomX anticipates announcing next steps for the PROCLAIM-CX-2009 clinical program by the end of 2019.
BMS-986249 Anti-CTLA-4 Probody Therapeutic Clinical Program

Bristol-Myers Squibb (BMS) is conducting a Phase 1/2a dose escalation clinical study evaluating BMS-986249 alone and in combination with OPDIVO (nivolumab) in advanced solid tumors.
BMS is preparing to initiate the Phase 2 portion of this clinical trial, upon which CytomX is entitled to a $10 million milestone payment. Additional information on this trial is available at ClinicalTrials.gov using the Identifier NCT03369223.
CX-2029 Anti-CD71 Probody Drug Conjugate Clinical Program

CytomX continued enrollment of patients in the PROCLAIM-CX-2029 Phase 1/2 study, which is partnered with AbbVie, evaluating CX-2029 as monotherapy in patients with solid tumors or lymphomas. Additional information on this trial is available at ClinicalTrials.gov using the Identifier NCT003543813.
Additional Corporate Highlights

In October, the Company announced the appointment of Amy C. Peterson, M.D., as executive vice president and chief development officer. In this new role, Dr. Peterson will have oversight of a multi-disciplinary team focused on advancing all aspects of CytomX’s clinical development activities.
In July, the Company announced that its partner AbbVie selected a second target under the companies’ 2016 Discovery Collaboration and Licensing Agreement to discover and develop Probody drug conjugates. The target selection triggered a $10 million payment to CytomX from AbbVie.
Third Quarter 2019 Financial Results
Cash, cash equivalents and short-term investments totaled $325.7 million as of September 30, 2019, compared to $436.1 million as of December 31, 2018.

Revenue was $10.7 million for the three months ended September 30, 2019, compared to $12.5 million for the three months ended September 30, 2018. The decrease in revenue of $1.8 million for the three months ended September 30, 2019 compared to the corresponding period in 2018 was primarily due to a $1.7 million decrease in revenue recognition under the CD71 Agreement with AbbVie due to ongoing dose escalation.

Research and development expenses increased $0.4 million during the three months ended September 30, 2019 compared to the corresponding period in 2018. The increase was attributable to an increase of $1.3 million in personnel-related expenses primarily due to an increase in headcount; an increase of $0.7 million in consulting expenses resulting from increased clinical trial activities and an increase of $0.8 million in the allocation of information technology and facilities related expenses driven partly from an increase in headcount; which amounts were offset by a decrease of $2.4 million in laboratory contracts and services and laboratory supplies as a result of timing of manufacturing activities as well as reduced costs relating to CX-188 which is not presently being advanced.

General and administrative expenses increased by $0.3 million during the three months ended September 30, 2019 compared to the corresponding period in 2018. The increase was attributable to an increase of $0.4 million in consulting expenses primarily related to IT, software implementation and finance services; an increase of $0.5 million in dues and subscriptions primarily related to software and other IT services; an increase of $0.2 million in building maintenance charges; which amounts were partially offset by a decrease of $0.8 million of information technology and facilities-related expenses allocated to the general and administrative functions.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 9449326. An archive of the webcast will be available on the CytomX website from November 7, 2019, until November 14, 2019.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.