Arcus Biosciences has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Arcus Biosciences, 2018, MAY 9, 2018, View Source [SID1234527532]).

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On MaY 9, 2018 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today reported its financial results for the first quarter ended March 31, 2018 and provided an update on the company’s recent achievements and anticipated upcoming milestones.

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"During the first quarter of 2018 we continued to make excellent progress across our product portfolio," stated Matthew R. Patterson, Chief Executive Officer of Audentes. "In particular, we are excited by the positive preliminary results we have observed in our XLMTM program and we look forward to reporting additional interim data from the first dose cohort of ASPIRO patients at the upcoming ASGCT (Free ASGCT Whitepaper) annual meeting. We are also pleased by the preliminary data from the first patient treated in our Crigler-Najjar program, which demonstrated an encouraging early safety profile and initial proof-of-concept for efficacy. We look forward to treating our next patient at a higher dose in the coming weeks."

Mr. Patterson continued, "Finally, we are excited by the growing momentum behind our product candidate AT982, which we believe offers a best-in-class approach for the treatment of Pompe disease. Based on recent discussions with regulatory authorities, we have expanded our clinical vision and plan to conduct two separate Phase 1 / 2 clinical studies for the infantile and late onset Pompe disease patient populations in parallel. As such, we have expanded the scope of our IND-enabling preclinical program, and now plan to include both studies in the initial IND filing targeted for the fourth quarter of 2018, which will support the initiation of the studies in the first half of 2019."

Recent Achievements & Upcoming Key Events

AT132 for XLMTM:
Completed dosing of three additional patients (cohort 1 expansion) in the 1×1014 vg/kg cohort of ASPIRO, bringing the total number of patients enrolled to date to seven (six AT132-treated and one delayed-treatment control). No significant treatment-related safety signals have been identified to date in cohort 1 expansion patients.
Plan to report additional interim data during an oral presentation at ASGCT (Free ASGCT Whitepaper) on May 16, 2018. Presentation to include up to 24-week data for the first four subjects enrolled and up to 4-week data in the cohort 1 expansion patients.
Plan to report six-month biopsy data from the first three patients dosed and announce plans with regard to dose escalation in the third quarter of 2018
AT342 for Crigler-Najjar Syndrome:
Initial proof of concept established based on 12-week data from first patient enrolled in VALENS study, a 12-year-old male, at a dose of 1.5×1012 vg/kg
AT342 has been well-tolerated with no significant treatment-related safety signals to date
Treatment resulted in a rapid decline in total bilirubin levels from approximately 11 mg/dL at baseline to 4 mg/dL at week 2 post-dosing, with a gradual return to baseline by week 12. A similar efficacy result was observed with low doses of AT342 in a dose ranging study in the mouse model of Crigler Najjar, while higher doses demonstrated durable bilirubin reduction.
Plan to dose escalate to the 6 x1012 vg/kg and enroll the next patient in VALENS in the coming weeks
Plan to report interim 12-week data in the first patient dosed in VALENS at the 51st Annual Congress of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) on May 10, 2018 and at ASGCT (Free ASGCT Whitepaper) on May 17, 2018
Plan to report the next interim data update from VALENS in the second half of 2018
AT982 for Pompe Disease:
Announced selection of the clinical development candidate, AT982, a novel AAV8 vector designed to express GAA in tissues relevant to Pompe disease, including skeletal muscle, the heart and the nervous system, and in the liver to reduce immunogenicity, thereby addressing the key limitations of existing enzyme replacement therapy for Pompe disease
Plan to present additional data from AT982 in a Pompe mouse model at ASGCT (Free ASGCT Whitepaper) on May 16, 2018
Plan to expand the preclinical program to support the filing of an IND to study AT982 in both infantile and late onset Pompe disease patients
Plan to file the IND in the fourth quarter of 2018 and initiate both Phase 1 / 2 clinical studies in the first half of 2019
AT307 for CASQ2-CPVT:
IND has been submitted. FDA has completed its initial review and provided a short list of questions to be addressed prior to the IND becoming active. We expect to submit responses in the coming weeks.
Received Fast Track designation from the FDA
Continuing patient identification activities to better characterize CASQ2-CPVT prevalence. Results from these efforts will inform clinical plans as they relate to the timing of a potential Phase 1 / 2 study.
Key Corporate Milestones:
In January 2018, Audentes strengthened its balance sheet with the completion of a follow-on financing, issuing 6,612,500 shares of common stock at an offering price of $35.00 per share, resulting in net proceeds of approximately $217.2 million after the deduction of underwriting discounts, commissions and offering expenses
Announced the promotion of Natalie Holles to President and Chief Operating Officer. In this new role, Ms. Holles will oversee the day-to-day operations of the company, including research, development, manufacturing, program management and corporate development.
First Quarter 2018 Financial Results

Cash Position: At March 31, 2018, Audentes had cash, cash equivalents, and short-term investments of $326.1 million. Current cash, cash equivalents and short-term investments are planned to fund operations into the second half of 2020.
Research and Development Expenses: Research and development expenses were $19.9 million for the first quarter of 2018 compared to $14.6 million for the same period in 2017, an increase of $5.3 million. The increase in research and development expenses was primarily attributable to an increase in development costs related to our AT982 program, increased headcount and related facility costs, increased internal manufacturing costs and higher stock compensation expense, and is partially offset by a decrease in the estimated fair value of the contingent liability associated with the 2015 acquisition of Cardiogen Sciences, resulting in a $2.3 million reduction in research and development expense during the first quarter of 2018. Research and Development expenses included $2.1 million of non-cash stock-based compensation expense.
General and Administrative Expenses: General and administrative expenses were $6.5 million for the first quarter of 2018 compared to $3.6 million for the same period in 2017, an increase of $2.9 million. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs, increased professional service fees, higher stock compensation expense and higher costs driven by continued public company regulatory compliance initiatives. General and administrative expense includes $1.3 million of non-cash stock-based compensation expense.
Net Loss: Net loss was $25.6 million for the first quarter of 2018 compared to $18.1 million for the same period in 2017.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its first quarter 2018 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 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and dial the conference ID 9789828 to access the call.

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

On May 9, 2018 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, today reported first quarter 2018 financial results (Press release, CytomX Therapeutics, MAY 9, 2018, View Source [SID1234527066]).

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As of March 31, 2018, CytomX had cash, cash equivalents and short-term investments of $361.5 million.

"CytomX’s first quarter marked the continued execution across our clinical pipeline, our discovery stage programs and in our partnerships," said Sean McCarthy, D.Phil., president and chief executive officer of CytomX Therapeutics. "We remain on track to share initial Probody clinical data at ASCO (Free ASCO Whitepaper) from PROCLAIM-CX-072, a broad-based study of our lead and wholly-owned anti-PD-L1 Probody therapeutic and we look forward to providing additional pipeline updates as the year progresses."

Business Highlights and Recent Developments

PROCLAIM-CX-072 (PD-L1 Probody Therapeutic) Clinical Program

CX-072 is a Probody therapeutic targeting PD-L1, a clinically- and commercially-validated anti-cancer target.
Initial clinical data from the Phase 1/2 PROCLAIM-CX-072 program will be presented on Monday June 4, from 8:00 – 11:30 a.m. as part of the Developmental Therapeutics—Immunotherapy session at the ASCO (Free ASCO Whitepaper) Annual Meeting.
Abstract 3071 – Preliminary Results of the First-In-Human, Dose-Finding PROCLAIM-072 Trial of the PD-L1 Probody Therapeutic CX-072 as Monotherapy in Patients with Advanced Solid Tumors
Abstract 3072 – Preliminary Interim Results of the First-In-Human, Dose-Finding PROCLAIM-072 Trial of the PD-L1 Probody Therapeutic CX-072 in Combination with Ipilimumab in Patients with Advanced Solid Tumors
The data presented at the meeting will reflect a data cutoff approximately five months later than the abstract submission data cutoff, and therefore, will include longer term follow up as well data from additional patients.
PROCLAIM-CX-2009 (CD166 Probody Drug Conjugate) Clinical Program

CX-2009 is a Probody drug conjugate (PDC) that targets CD166, an antigen that is broadly and highly expressed in many types of cancer.
Preliminary data from Part A of the PROCLAIM-CX-2009 Phase 1/2 clinical program is expected to be presented in the second half of 2018.
CX-2029 (CD71 Probody Drug Conjugate) Preclinical Program

CytomX, in collaboration with AbbVie, is advancing CX-2029, a CD71-directed PDC.
CytomX filed an Investigational New Drug (IND) application for CX-2029 in April 2018.
CytomX expects the initiation of clinical trials in the third quarter of 2018.
CX-188 (PD-1 Probody Therapeutic) Preclinical Program

CytomX is advancing CX-188, a PD-1-directed Probody therapeutic, through IND-enabling studies.
CytomX expects to file an IND application for CX-188 in the second half of 2018.
First Quarter 2018 Financial Results
Cash, cash equivalents and short-term investments totaled $361.5 million as of March 31, 2018, compared to $374.1 million as of December 31, 2017.

Revenue was $14.2 million for the three months ended March 31, 2018, compared to $11.7 million for the three months ended March 31, 2017. The increase was primarily attributable to an increase of $4.9 million relating to the recognition of $6.3 million of the upfront payment received from BMS in connection with an amendment of the Company’s Collaboration and License Agreement entered into in March 2017, offset by $1.2 million in revenue recognized resulting from a reduction in research terms during the three months ended March 31, 2017; an increase of $1.3 million resulting from a change in revenue recognition for the Company’s CD71 Agreement with AbbVie from the straight-line method under the prior revenue recognition standard to the percentage-of-completion method under the new revenue recognition standard, which the Company adopted on January 1, 2018; an increase of $1.3 million related to the Amgen Agreement, which the Company entered into at the end of September 2017; and an increase of $0.9 million resulting primarily from the recognition of the remaining upfront payments resulting from the termination of the Company’s agreement with Pfizer in March 2018. These increases were offset by a decrease of $5.8 million related to ImmunoGen due primarily to the recognition of $6.5 million upon delivery of a Development and Commercialization License to ImmunoGen during the three months ended March 31, 2017.

Research and development expenses increased $7.9 million during the three months ended March 31, 2018 compared to the corresponding period in 2017. The increase was attributable to an increase of $3.5 million in lab contracts and services, consulting expenses and clinical trial startup fees to advance CX-188 and CX-2029 through IND-enabling studies, an increase of $2.2 million in CX-072 resulting from increased clinical trial activities, and an increase of $2.0 million in personnel-related expenses due to an increase in headcount.

General and administrative expense increased $1.7 million during the three months ended March 31, 2018 compared to the corresponding period in 2017. The increase was attributable to an increase of $0.8 million in personnel-related expense due to an increase in headcount, an increase of $0.4 million in stock-based compensation resulting from an increase in headcount and an increase in the average price of the Company’s common stock and an increase of $0.4 million in consulting services primarily due to an increase in tax and accounting compliance activities.

On May 9, 2018 Novavax, Inc., (Nasdaq: NVAX) reported its financial results for the first quarter ended March 31, 2018 (Press release, Novavax, MAY 9, 2018, View Source [SID1234526488]).

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First Quarter and Subsequent Achievements:

RSV F Vaccine

In May 2018, Novavax reached enrollment of approximately 4,600 pregnant women in its Prepare Phase 3 clinical trial of its RSV F Vaccine for infants via maternal immunization. This milestone enables Novavax to initiate a prespecified interim efficacy analysis after approximately six months of follow-up of the last infant born to the approximately 4,600 women enrolled (including 3,000 actively vaccinated women). Completion of this analysis is expected in the first quarter of 2019. Since 2015, the Prepare trial is supported by an $89 million grant from the Bill & Melinda Gates Foundation (BMGF).

In April 2018, Novavax presented at the World Vaccine Congress on the status of its Phase 3 clinical trial of its RSV F Vaccine.

NanoFlu

In April 2018, Novavax presented clinical data at the World Vaccine Congress from the Phase 1/2 clinical trial in older adults comparing trivalent formulations of NanoFlu to the market-leading licensed egg-based, high-dose influenza vaccine for older adults.

In February 2018, Novavax reported positive top-line results from its Phase 1/2 clinical trial of its trivalent NanoFlu.

Corporate

In April 2018, Novavax completed an underwritten public offering of approximately 34.8 million shares of its common stock, including 4.5 million shares pursuant to the underwriters’ option to purchase additional shares. The shares resulted in net proceeds of $54 million.

Effective on March 14, 2018, John J. Trizzino, former Senior Vice President, Commercial Operations since 2014, was promoted to Senior Vice President, Chief Business Officer and Chief Financial Officer.

Anticipated Events:

Initiation of the Phase 2 clinical trial of quadrivalent formulations of NanoFlu scheduled to begin in the third quarter of 2018.

·Top-line data from the Phase 2 NanoFlu trial and End of Phase 2 meeting with the FDA expected in the first quarter of 2019.

Results of the Prepare Phase 3 interim efficacy analysis for our RSV F Vaccine expected in the first quarter of 2019.

Summary

"We had an extremely productive first quarter, including making important advances in our two lead clinical vaccine programs. We are pleased to have reached the enrollment target for our Prepare Phase 3 RSV F Vaccine trial, which clears the path for following these most recent participants and their babies, and subsequently announcing top-line results of our planned interim efficacy analysis in the first quarter of 2019," said Stanley C. Erck, President and CEO of Novavax, Inc. "We also continue to make significant progress on NanoFlu and plan to initiate a Phase 2 clinical trial in the third quarter of 2018."

Financial Results for the First Quarter Ended March 31, 2018

Novavax reported a net loss of $46.4 million, or $0.14 per share, for the first quarter of 2018, compared to a net loss of $43.9 million, or $0.16 per share, for the first quarter of 2017.

Novavax revenue in the first quarter of 2018 was $9.7 million, compared to $5.7 million in the same period in 2017. This 70% increase was driven by higher revenue recorded under the BMGF grant corresponding to the increased enrollment in the Prepare trial.

Research and development expenses increased 18% to $44.5 million in the first quarter of 2018, compared to $37.7 million for the same period in 2017. The increase was primarily due to increased development activities of the RSV F Vaccine for infants via maternal immunization.

Interest income (expense), net for the first quarter of 2018 was ($2.9) million, compared to ($3.0) million for the same period of 2017.

As of March 31, 2018, Novavax had $164.2 million in cash, cash equivalents, marketable securities and restricted cash, compared to $186.4 million as of December 31, 2017. Net cash used in operating activities for the first quarter of 2018 was $66.1 million, compared to $44.5 million for same period in 2017. The increase in cash usage was primarily due to approximately $16 million of one-time payments, as well as the adoption of a new accounting standard that requires restricted cash to be included in the beginning and ending balances on the statements of cash flows, thus increasing Novavax’ cash usage in the first quarter of 2018 and 2017 by approximately $9 million and $6 million, respectively. We expect our cash used in operating activities to significantly decrease for the subsequent quarters of 2018 as compared to the first quarter of 2018.

Conference Call

Novavax management will host its quarterly conference call today at 4:30 p.m. ET. The dial-in number for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 3687883. A replay of the conference call will be available starting at 7:30 p.m. ET on May 9, 2018 until 7:30 p.m. ET on May 16, 2018. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 3687883.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website at www.novavax.com or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available on the Novavax website until August 9, 2018.

About RSV

RSV is the most common cause of lower respiratory tract infections and the leading viral cause of severe lower respiratory tract disease in infants and young children worldwide, with estimated annual infection and mortality rates of 64 million and 160,000, respectively.1 In the U.S., RSV is the leading cause of hospitalization of infants.2 Despite the induction of post-infection immunity, repeat infection and lifelong susceptibility to RSV is common.3 Currently, there is no approved RSV vaccine available.

About RSV F Vaccine for Infants via Maternal Immunization

Novavax is developing a vaccine that targets the fusion protein, or F protein, of the RSV virus. The F protein has highly conserved amino acid sequences, called antigenic sites, which are the target of neutralizing antibodies and are believed to be ideal vaccine targets. Novavax’ genetically engineered novel F protein antigen exposes a range of these antigenic sites, and can evoke immune responses to them in human vaccine recipients. In a previous Phase 2 clinical trial of the RSV F Vaccine, which assessed the transplacental transfer of maternal antibodies induced by the vaccine, immunized women demonstrated meaningful fold rises in anti-F IgG, palivizumab-competing antibodies and microneutralization titers. In addition, infants’ antibody levels at delivery averaged 90-100% of the mothers’ levels, indicating efficient transplacental transfer of antibodies from mother to infant.

About Influenza

Influenza is a world-wide infectious disease that causes illness in humans with symptoms ranging from mild to life-threatening or even death. Serious illness occurs not only in susceptible populations such as infants, young children and older adults, but also in the general population largely because of infection by continuously evolving strains of influenza which can evade the existing protective antibodies in humans. An estimated one million deaths each year are attributed to influenza.4 Current estimates for seasonal influenza vaccine growth in the top seven markets (U.S., Japan, France, Germany, Italy, Spain and UK), show a potential increase from approximately $3.2 billion in 2015 to $5.3 billion by 2025.5

1 View Source

2 Leader S. Pediatr Infect Dis J. 2002 Jul;21(7):629-32

3 PLOS. "How immunity to respiratory syncytial virus develops in childhood, deteriorates in adults." ScienceDaily. 21 April 2016. View Source

4 Resolution of the World Health Assembly (2003) WHA56.19.28

5 Influenza Vaccines Forecasts. Datamonitor (2013)

About NanoFlu

NanoFlu is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA amino acid protein sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu contains Novavax’ patented saponin-based Matrix-M adjuvant, which has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes.

On May 9, 2018 Lodo Therapeutics Corporation, a drug discovery and development company focused on identifying and producing unique, bioactive natural products directly from the microbial DNA sequence information contained in soil, reported that it has formed a strategic drug discovery collaboration with Genentech, a member of the Roche Group (Press release, Lodo Therapeutics, MAY 9, 2018, View Source [SID1234526409]).

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Under the terms of the agreement, Genentech will utilize Lodo Therapeutics’ proprietary genome mining and biosynthetic cluster assembly platform to identify novel molecules with therapeutic potential against multiple disease-related targets of interest to Genentech. Lodo will receive an undisclosed upfront payment and is eligible to receive research, development and commercialization milestone payments up to $969 million based on achievement of certain predetermined milestones. In addition, Lodo is eligible to receive tiered-royalties on sales of certain products resulting from the collaboration.

"Lodo Therapeutics’ proprietary drug discovery platform is a powerful engine for identifying novel compounds with important therapeutic potential," said Thong Q. Le, chief executive officer at Lodo Therapeutics and Accelerator Life Science Partners. "We are incredibly excited to work with Genentech, and we look forward to demonstrating the power and utility of Lodo’s unique technology for the benefit of global human health."

Compounds derived from natural products comprise a significant proportion of the small molecule drugs used to treat cancer, infections and chronic illnesses such as Type 2 diabetes. Rather than relying on culturing known strains of bacteria, Lodo Therapeutics’ genome-based approach leverages the power of microbial evolution to identify novel, naturally occurring compounds that have therapeutic potential in the treatment of cancer and drug-resistant bacterial infections. This approach is expected to reduce the time and cost of drug discovery.

"Our ability to enter into a strategic collaboration with one of the leaders in innovating wholly new classes of drugs just two years after Lodo Therapeutics was founded reflects the potential of our proprietary platform to be a valuable resource to advance their drug discovery initiatives," said David Pompliano, Ph.D., co-founder and chief scientific officer of Lodo Therapeutics.

"We are excited to work with Genentech in their quest to discover novel, next-generation natural products derived from the microbiome of the soil using this innovative platform developed by Lodo," said Sean Brady, Ph.D., co-founder of Lodo Therapeutics and Associate Professor at The Rockefeller University. James Sabry, M.D., Ph.D., senior vice president and global head of Genentech Partnering, commented,

"Genentech is committed to accessing innovative technologies and we are excited to collaborate with Lodo Therapeutics to apply their Metagenomics Technology Platform to potentially discover therapeutics for difficult drug targets.