On August 7, 2023 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported financial results for the three and six months ended June 30, 2023, and provided an update on its corporate progress (Press release, BioNTech, AUG 7, 2023, View Source [SID1234633864]).

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"We are progressing our oncology pipeline into late-stage development, having launched a pivotal Phase 3 trial and preparing for additional trials with registrational potential in the coming months," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "Simultaneously, we are enhancing our infectious disease pipeline to address global health needs and are developing an Omicron XBB.1.5-adapted monovalent COVID-19 vaccine to become available for the upcoming fall-winter season, subject to regulatory approvals."

Financial Review for the Second Quarter and First Half of 2023

in millions €, except per share data Second Quarter 2023 Second Quarter 2022 First Half
2023 First Half
2022
Total Revenues2 167.7 3,196.5 1,444.7 9,571.1
Net Profit / (Loss) (190.4) 1,672.0 311.8 5,370.8
Diluted Earnings / (Loss) per Share (0.79) 6.45 1.28 20.69
Total revenues reported were €167.7 million2 for the three months ended June 30, 2023, compared to €3,196.5 million for the comparative prior year period. For the six months ended June 30, 2023, total revenues were €1,444.7 million2, compared to €9,571.1 million for the comparative prior year period. Write-offs by BioNTech’s collaboration partner Pfizer, Inc. ("Pfizer") significantly reduced the Company’s gross profit share in the second quarter and hence negatively influenced its revenues for the three months ended June 30, 2023.

Cost of sales were €162.9 million for the three months ended June 30, 2023, compared to €764.6 million for the comparative prior year period. For the six months ended June 30, 2023, cost of sales were €258.9 million, compared to €2,058.7 million for the comparative prior year period. The change was in line with decreasing COVID-19 vaccine sales.

Research and development expenses were €373.4 million for the three months ended June 30, 2023, compared to €399.6 million for the comparative prior year period. For the six months ended June 30, 2023, research and development expenses were €707.4 million, compared to €685.4 million for the comparative prior year period. Research and Development (R&D) expenses are mainly influenced by progressing clinical studies for pipeline candidates, the development of variant adapted as well as next generation COVID-19 vaccines and expanding R&D headcount.

General and administrative expenses were €122.7 million for the three months ended June 30, 2023, compared to €130.0 million for the comparative prior year period. For the six months ended June 30, 2023, general and administrative expenses were €242.1 million, compared to €220.8 million for the comparative prior year period. G&A expenses were mainly influenced by increased expenses for IT services as well as expanding the G&A headcount.

Income taxes were realized with an amount of €221.8 million tax income for the three months ended June 30, 2023, compared to €647.3 million of tax expenses accrued for the comparative prior year period. For the six months ended June 30, 2023, income taxes were realized with an amount of €16.3 million tax income, compared to €1,966.6 million tax expenses accrued for the comparative prior year period. The derived annual effective income tax rate for the six months ended June 30, 2023, was minus 5.5% which is expected to change over the 2023 financial year to be in line with the updated estimated annual cash effective income tax rate of somewhere around 21% for the BioNTech Group.

Net loss was €190.4 million for the three months ended June 30, 2023, compared to €1,672.0 million net profit for the comparative prior year period. For the six months ended June 30, 2023, net profit was €311.8 million, compared to €5,370.8 million net profit for the comparative prior year period.

Cash and cash equivalents as well as security investments were €14,166.6 million as well as €2,667.0 million, respectively, as of June 30, 2023. Subsequent to the end of the reporting period, the payment settling BioNTech’s gross profit share for the first quarter of 2023 (as defined by the contract with Pfizer) in the amount of €1,059.2 million was received from BioNTech’s collaboration partner as of July 17, 2023. In addition, until early August 2023, €437.7 million was received in connection with the amended COVID-19 Vaccine Purchase Agreement with the European Commission (EC).

Loss per share was €0.79 for the three months ended June 30, 2023, compared to a diluted earnings per share €6.45 for the comparative prior year period. For the six months ended June 30, 2023, diluted earnings per share was €1.28, compared to €20.69 diluted earnings per share for the comparative prior year period.

Shares outstanding as of June 30, 2023, were 239,771,156, excluding 8,781,044 shares in treasury.
In March 2023, BioNTech initiated a new share repurchase program pursuant to which the Company may purchase American Depositary Shares, or ADSs, each representing one ordinary share of the Company, in the amount of up to $0.5 billion during the remainder of 2023. During the three months ended June 30, 2023, 1,532,685 American Depositary Shares were repurchased under the share repurchase program of ADSs at an average price of €100.45 ($108.923), for total consideration of €154.0 million ($166.9 million3).

Cash outflows and share consideration in connection with the acquisition of InstaDeep Ltd. ("InstaDeep") on July 31, BioNTech invested approximately €450 million not including potential future milestones.

"We enter the second half of 2023 with a strong financial position, on track to launch our new variant-adapted COVID-19 vaccine and to conduct multiple clinical trials with registrational potential across our oncology and infectious disease pipeline. The COVID-19 vaccine market remains highly dynamic and difficult to fully predict. Along with our partner Pfizer, the Company continues to focus on supporting successful vaccinations during the autumn respiratory infection season," said Jens Holstein, CFO of BioNTech. "It is our goal to become a multi-product company by investing in our own clinical programs and by complementing them with additional compounds from our partners. With some uncertainty on the revenue line, we are also carefully watching our spending by revisiting our cost base while remaining focused on executing against our strategic goals and providing value to the public and our shareholders."

Outlook for the 2023 Financial Year
The Company reiterates its COVID-19 vaccine revenue guidance and updates its previous expense and capex guidance for the 2023 financial year:

BioNTech COVID-19 Vaccine Revenues for the 2023 Financial Year:

Estimated BioNTech COVID-19 vaccine revenues for the full 2023 financial year ~ €5 billion
This estimate reflects expected revenues related to BioNTech’s share of gross profit from COVID-19 vaccine sales in the collaboration partners’ territories, from direct COVID-19 vaccine sales to customers in BioNTech’s territory and expected revenues generated from products manufactured by BioNTech and sold to collaboration partners, which may be influenced by costs such as inventory write-offs once materialized and shared with the collaboration partner Pfizer.

Revenue guidance is based on various assumptions, including, but not limited to, the expected transition from an advanced purchase agreement environment to commercial market ordering starting in some geographies and an expected regulatory recommendation to adapt the COVID-19 vaccines to address newly circulating variants or sublineages of SARS-CoV-2. While vaccine adaptation is expected to lead to increased demand, fewer primary vaccinations and lowered population-wide levels of boosting are anticipated. In addition, seasonal demand is assumed, moving expected revenue generation to the second half of the year 2023 as previously reported. The revenues guidance reflects the revenues as specified by the amendment of the contractual agreement with the EC on behalf of the member states while it largely remains dependent on revenues generated in BioNTech’s collaboration partner’s territories. The market dynamics for COVID-19 vaccines are influenced by various factors which underlie substantial uncertainties and might affect the demand for COVID-19 vaccines in general as well as the Company’s estimated revenues.

Planned 2023 Financial Year Expenses and Capex4:

Previous Guidance Updated Guidance
R&D expenses5 €2,400m – €2,600m €2,000m – €2,200m
SG&A expenses €650m – €700m €600m – €700m
Capital expenditures for operating activities6 €500m – €600m €350m – €450m
Estimated 2023 Financial Year Tax Assumptions:

Previous Guidance Updated Guidance
BioNTech Group estimated annual cash effective income tax rate7 ~ 27% ~ 21%
The full interim unaudited condensed consolidated financial statements can be found in BioNTech’s Report on Form 6-K, filed today with the United States Securities and Exchange Commission ("SEC") and available at View Source

Endnotes
1 Financial information is prepared and presented in Euros and numbers are rounded to millions and billions of Euros in accordance with standard commercial practice.
2 BioNTech’s profit share is estimated based on preliminary data shared between Pfizer and BioNTech as further described in the Annual Report. Any changes in the estimated share of the collaboration partner’s gross profit will be recognized prospectively.
3 Calculated applying the average foreign exchange rate for the three and six months ended June 30, 2023, as published by the German Central Bank (Deutsche Bundesbank).
4 Numbers reflect current base case projections and are calculated based on constant currency rates.
5 Numbers include effects identified from additional collaborations or potential M&A transactions to the extent disclosed and will be updated as needed.
6 Numbers exclude potential effects caused by or driven from collaborations or M&A transactions.
7 Numbers exclude potential effects caused by or driven from share-based payment settlements in the course of 2023.

Operational Review and Pipeline Update for the Second Quarter 2023 and Key Post Period-End Events

COVID-19 Marketed Products

In May, BioNTech and Pfizer announced an agreement with the EC to amend the previous COVID-19 Vaccine Purchase Agreement to deliver COVID-19 vaccines doses to the European Union. The amended agreement reflects BioNTech and Pfizer’s commitment to working collaboratively to help address ongoing public health needs, while respecting the principles of the original agreement. It includes re-phasing of delivery of doses annually through 2026. In addition, the agreement includes an aggregate volume reduction, providing additional flexibility for EU Member States. The EC will maintain access to future adapted COVID-19 vaccines and the ability to donate doses, in alignment with the original agreement.
In June, BioNTech and Pfizer submitted regulatory applications to the EMA and to the U.S. FDA for their Omicron XBB.1.5-adapted monovalent COVID-19 vaccine for individuals 6 months of age and older in line with recommendations from both regulatory agencies. Regulatory submissions in other territories have also been initiated.
BioNTech and Pfizer have manufactured Omicron XBB.1.5-adapted monovalent COVID-19 vaccine doses at risk to ensure readiness ahead of the fall and winter seasons in various regions worldwide. The companies plan to prepare shipments of Omicron XBB.1.5-adapted monovalent COVID-19 vaccine doses for fast delivery following potential regulatory approval.
Oncology Pipeline Highlights – Recent and upcoming trial starts and data readouts

BNT316/ONC-392 (gotistobart) is a next-generation anti-CTLA-4 monoclonal antibody candidate jointly developed by BioNTech and OncoC4, Inc. ("OncoC4"). BNT316/ONC-392 offers a potentially differentiated safety profile that may allow for higher dosing and longer duration of treatment both as a monotherapy and in combination with other therapies.

In June, BioNTech and OncoC4 initiated a Phase 3 clinical trial (NCT05671510) to evaluate BNT316/ONC-392 as monotherapy in non-small cell lung cancer (NSCLC) patients whose disease progressed on anti-PD-1/PD-L1 antibody-based therapy. The program received Fast Track Designation from the FDA in 2022.
Data from a dose escalation and an expansion cohort evaluating BNT316/ONC-392 as monotherapy in NSCLC patients that had progressed on prior immune-checkpoint inhibitor (ICI) therapy as part of the ongoing Phase 1/2 clinical trial (NCT04140526) were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2023. BNT316/ONC-392 was generally well-tolerated with a manageable safety profile. Early readout of the expansion cohort showed encouraging clinical activity in patients with ICI-resistant NSCLC.
BNT323/DB-1303 is a HER2-targeted antibody-drug conjugate (ADC) candidate, being developed in collaboration with Duality Biologics (Suzhou) Co. Ltd. ("DualityBio").

BNT323/DB-1303 is being evaluated in a Phase 1/2 clinical trial (NCT05150691) in patients with advanced/unresectable, recurrent, or metastatic HER2-expressing solid tumors. Data from the ongoing trial were presented at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting suggesting that BNT323/DB-1303 was well tolerated and adverse events (AEs) were manageable. Preliminary antitumor activity was observed in heavily pretreated HER2-expressing patients with a median of seven prior systemic treatment regimens, including other HER2 ADCs.
BNT324/DB-1311 is a topoisomerase-1 inhibitor-based ADC candidate being developed in collaboration with DualityBio.

A first-in-human, open-label Phase 1/2 clinical trial evaluating BNT324/DB-1311 in multiple advanced solid tumors is planned to start this year.
BNT116 is based on BioNTech’s FixVac platform, a wholly owned, systemically administered, off-the-shelf mRNA-based cancer vaccine candidate. This candidate is being evaluated for the treatment of advanced NSCLC.

In July 2023, BioNTech and Regeneron Pharmaceuticals Inc. ("Regeneron") initiated a randomized, controlled, Phase 2 clinical trial (NCT05557591) to evaluate BNT116 in combination with cemiplimab (Regeneron’s Libtayo) and cemiplimab alone as first-line treatment of patients with advanced NSCLC whose tumors express PD-L1 in ≥ 50% of tumor cells.
BNT122 (Autogene cevumeran) is an mRNA cancer vaccine candidate based on an individualized neoantigen-specific immunotherapy (iNeST) approach being developed in collaboration with Genentech, a member of the Roche Group ("Roche").

A randomized Phase 2 clinical trial (NCT05968326) is planned to start in 2H 2023 to further evaluate the safety and efficacy of BNT122 in the adjuvant setting in combination with atezolizumab (Genentech’s Tecentriq) followed by chemotherapy in patients with resected pancreatic ductal adenocarcinoma (PDAC) supported by data from an investigator-initiated Phase 1 trial (NCT04161755). In May 2023, results from the Phase 1 trial were published in the peer-reviewed journal Nature. Trials in other indications are ongoing.
BNT211 is an autologous Claudin-6 (CLDN6)-targeting chimeric antigen receptor (CAR) T cell therapy candidate that is being tested alone and in combination with an investigational CAR-T cell Amplifying RNA Vaccine compound, or CARVac, encoding CLDN6.

A data update from the ongoing Phase 1/2 clinical trial (NCT04503278) was provided at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting detailing the new dose escalation of CLDN6 CAR-T cells with and without a CLDN6-encoding mRNA vaccine for the treatment of CLDN6-positive relapsed/refractory solid tumors using an automated manufacturing process. CLDN6 CAR-T cells ± CLDN6 CARVac showed a moderate safety profile in line with that of manually produced CLDN6 CAR-T cells. Encouraging signs of clinical activity for dose level 1 and 2 were confirmed, including dose-dependent expansion of CAR-T cells demonstrated by an objective response rate (ORR) of 41% in all 17 evaluable patients and an ORR of 75% in patients at dose level 2. Follow-up of treated patients and further enrollment of patients into dose level 2 and 3 are ongoing. After determination of a recommended Phase 2 dose for CLDN6 CAR-T cells, a pivotal trial in germ cell tumors is planned to start in 2024.
Corporate Update for the Second Quarter 2023 and Key Post Period-End Events

In April, BioNTech entered into exclusive license and collaboration agreements with DualityBio to develop, manufacture and commercialize two investigational topoisomerase-1 inhibitor-based ADC assets, BNT323/DB-1303 and BNT324/DB-1311. In August 2023, BioNTech signed another agreement with DualityBio to develop, manufacture and commercialize an additional antibody-drug conjugate, DB-1305.
In July, following on a memorandum of understanding announced in January, BioNTech signed a long-term strategic partnership agreement with the UK Government, NHS England and Genomics England Limited with the aim to provide access to personalized treatments for up to 10,000 patients by 2030, either in clinical trials or as authorized treatment. To execute this, BioNTech plans to set up new laboratories in Cambridge with an expected capacity of more than 70 highly skilled scientists as well as a new regional hub for the United Kingdom.
In July, BioNTech also successfully completed its previously announced acquisition of InstaDeep, following the satisfaction of all customary closing conditions. The acquisition supports the Company’s strategy to build world-leading capabilities in Artificial Intelligence ("AI")-driven drug discovery and development. InstaDeep will operate as a UK-based global subsidiary of BioNTech. The transaction adds approximately 290 highly skilled professionals to BioNTech’s workforce, including teams in AI, machine learning ("ML"), bioengineering, data science, and software development.
Environmental, Social, and Governance (ESG)

BioNTech was founded out of a responsibility to patients and to society and this is still the vision that drives the Company. It gives grounds for BioNTech’s enhanced responsibility: for translating the Company’s science into the health of people worldwide and democratizing access to innovative medicines, for environmental and climate protection, for respecting human rights and for fostering the full potential of all employees.

In March 2023, BioNTech published its third ESG report (Sustainability Report 2022). The report highlights the Company’s progress in developing novel medicines and introducing scalable technological innovations. It describes BioNTech’s science-based climate goals (under SBTi review), actions and climate risk management as well as the status of the Companies’ human rights strategy and due diligence. The report addresses diversity, inclusion, equity and belonging, and highlights the importance of BioNTech’s values and culture.

BioNTech recognizes its responsibility as a corporate citizen and is committed to supporting its local communities and beyond through donations, sponsorships and volunteer activities.

Upcoming Investor and Analyst Events

BioNTech’s third quarter 2023 financial results and corporate update are scheduled for Monday, November 6, 2023.
BioNTech will host its 2nd Innovation Day on Tuesday, November 7, 2023, in Boston, USA.

On August 7, 2023 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported its financial and operating results for the second quarter ended June 30, 2023 and provided a corporate update (Press release, Biodesix, AUG 7, 2023, View Source [SID1234633863]).

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"I am very pleased to announce another record-setting quarter from our core lung diagnostics," said Scott Hutton, President and Chief Executive Officer. "In addition to continued sales growth, we had a number of important publications this past quarter that highlighted the benefits to patient care of our lung diagnostic tests in a real-world, clinical setting.

We are also happy to announce the successful completion of a private placement that included some of our largest shareholders and management. All board members, all Section 16 officers, and additional members of the Biodesix leadership team participated in the round raising $27.5 million in equity funding to further support our growth. We also maintained strong cost discipline, with sustainable and improving gross margins to 73% from 65%, and reducing our Operating Expense excluding direct costs and expenses by $2.7 million, all versus first quarter 2023. We continue to focus on projects and initiatives that drive near term-revenue growth, while reducing expenses and cash burn. Overall, the progress and positive trends in our core lung diagnostics tests in the first half of the year solidifies our confidence in reaffirming our 2023 revenue guidance and making progress on our path to profitability."

Business Highlights

Biodesix continues to publish new data supporting the value and utility of their lung diagnostics portfolio. The Company was pleased to announce the following accomplishments:

•
Published the achievement of the primary endpoint of the prospective ORACLE clinical utility study, demonstrating that use of the Nodify XL2 test resulted in a 74% decrease in unnecessary invasive procedures on patients with benign lung nodules compared to the control group;
•
Researchers from Beth Israel Deaconess, Tulane University, and Einstein Medical Center published an independent, multi-center study demonstrating that use of the Nodify XL2 test resulted in a 73% reduction in the number of invasive procedures compared to the control arm;

•
Presented new health economics data on the Nodify XL2, Nodify CDT, and VeriStrat tests at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the International Society for Pharmaceutical and Outcomes Research (ISPOR). These data indicate savings to the US healthcare system with use of the tests;
•
Completed enrollment of 5,000 patients with non-small cell lung cancer (NSCLC) in the large multi-center observational registry study INSIGHT. The study was designed to further validate the utility of the VeriStrat test;
•
Announced that CMS has designated the Nodify CDT Test as an Advanced Diagnostic Laboratory Test (ADLT) effective June 30, 2023. ADLT status is reserved for innovative tests with Medicare coverage that provide new clinical diagnostic information that cannot be obtained from any other test or combination of tests.

Second Quarter 2023 Financial Results

For the three-month period ended June 30, 2023, as compared to the same period of 2022 (where applicable):

•
Total revenue of $11.9 million, an increase of 8% including COVID testing revenue in second quarter 2022, driven primarily by strong year-over-year growth in core lung diagnostics, and a 48% year-over-year increase excluding COVID testing revenues from the prior year comparison.
o
Core lung diagnostic revenue of $11.4 million reflected a year-over-year increase of 58% driven primarily by the continued adoption of Nodify Lung nodule management tests;
o
BioPharma Services revenue of $0.4 million decreased 43% year-over-year. Timelines for existing and new agreements continue to be impacted by delayed enrollment in clinical trials; entered the third quarter of 2023 with continued strong dollars under contract;
o
COVID-19 testing revenue decreased by 100% year-over-year, the Company no longer provides COVID-19 diagnostic testing services commercially;
•
Second quarter 2023 gross profit of $8.6 million, or 73% gross margin as compared to 64% gross margin in the comparable prior year period primarily driven by growth in Lung Diagnostic Testing and optimization of testing workflows that resulted in improvements in costs per test, and the commercial discontinuation of our lower-margin COVID-19 diagnostic testing;
•
Operating expenses (excluding direct costs and expenses) of $19.6 million, an increase of approximately $1.0 million, or 5% as compared to the second quarter 2022 (includes non-cash stock compensation expense of $1.1 million as compared to $1.4 million). This increase is primarily attributable to increased sales and marketing costs to support core lung diagnostic sales growth including increased travel-related costs and marketing programs to enhance product awareness as we actively participate in an increasing number of peer-to-peer physician educational events;
•
Net loss of $13.4 million, a decrease of approximately $2.5 million, or 16%;
•
Cash and cash equivalents of $17.4 million as of June 30, 2023, a decrease of $7.9 million from March 31, 2023. This represented an improvement of approximately 56% in cash utilization versus the cash utilized in the first quarter 2023;
o
Scheduled milestone payment of $2.3 million paid in April 2023 to Integrated Diagnostics;
o
Subsequent to quarter end, raised $27.5 million in gross equity proceeds from a private placement to be used for commercial expansion of sales, research and development, and for general corporate purposes.

2023 Financial Outlook

The Company reaffirms our 2023 financial outlook and expects to generate between $52 million and $55 million in total revenue in 2023.

Conference call and webcast information

Listeners can register for the webcast via this link. Analysts wishing to participate in the question-and-answer session should use this link. A replay of the webcast will be available via the Company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

For a full list of Biodesix’s press releases and webinars, please visit biodesix.com.

On August 7, 2023 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal third quarter ended June 30, 2023 (Press release, Arrowhead Research Corporation, AUG 7, 2023, View Source [SID1234633862]). The company is hosting a conference call today, Aug. 7, 2023, at 4:30 p.m. ET to discuss the results.

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Webcast and Conference Call and Details

Investors may access a live audio webcast on the Company’s website at View Source A replay of the webcast will be available approximately two hours after the conclusion of the call.

For analysts that wish to participate in the conference call, please register at https://register.vevent.com/register/BIf9cd65655e5444119df91cd4891157f8. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.

Selected Recent Events
•Hosted a Research & Development Day to discuss progress towards the company’s "20 in 25" goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiMTM) platform to a total of 20 clinical stage or marketed products in the year 2025. Key updates included:
•New Phase 1 clinical data on ARO-RAGE showing a continued dose response in healthy volunteers with a single inhaled dose of 184 mg achieving mean knockdown of 90% and a maximum of 95% in bronchoalveolar lavage fluid (BALF)
•Serum sRAGE reductions in asthma patients consistent with effects seen in healthy volunteers at the 44 mg dose, the only dose level with results currently available in asthma patient cohorts

•TRiMTM platform for intrathecal administration for central nervous system (CNS) delivery demonstrates 90-95% dose-dependent mRNA knockdown in disease-relevant spinal cord and cortex brain regions in non-human primates

•TRiMTM platform for adipose tissue delivery achieved up to 98% knockdown and maintained greater than 85% knockdown over 31 weeks

•TRiMTM dimer platform for delivery to hepatocytes achieved equivalent or better knockdown of two separate target genes with longer duration than monomer mixture

•Completed enrollment of the global PALISADE Phase 3 clinical trial evaluating ARO-APOC3 for the treatment of familial chylomicronemia syndrome. The company anticipates that the primary portion of the study will be complete in the second quarter of 2024 with a data readout shortly thereafter

•Presented interim data from the ongoing Phase 2 GATEWAY clinical study of ARO-ANG3 in patients with homozygous familial hypercholesterolemia at the 91st European Atherosclerosis Society Congress showing that ARO-ANG3 achieved 44-48% mean reductions in LDL-C on top of continued standard of care

•Presented updated results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency at the European Association for the Study of the Liver (EASL) Congress 2023

•Filed an application to initiate a Phase 1 clinical trial of ARO-SOD1, an RNAi-based investigational medicine designed to reduce expression of superoxide dismutase 1 (SOD1) in the CNS as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations. ARO-SOD1 is the first therapeutic candidate designed for delivery to the CNS to enter clinical studies that leverages the TRiMTM platform

•Filed an application to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNAi therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). ARO-DUX4 is the first clinical candidate utilizing the TRiMTM platform to target disease associated genes in skeletal muscle

•Earned a $30 million milestone payment from GSK (LSE/NYSE: GSK) following the start of GSK’s Phase 2b trial of GSK4532990, formerly called ARO-HSD, an investigational RNAi therapeutic for the treatment of patients with non-alcoholic steatohepatitis (NASH)

•Earned a $40 million milestone payment from Takeda (TSE:4502/NYSE:TAK) after the first patient was dosed in the Phase 3 REDWOOD clinical study of fazirsiran

On August 7, 2023 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported financial results for the second quarter ended June 30, 2023, and provided a pipeline update on its clinical-stage investigational molecules – targeting TIGIT, the adenosine axis (CD73 and A2a/A2b receptors), HIF-2a and PD-1 – across multiple common cancers (Press release, Arcus Biosciences, AUG 7, 2023, View Source [SID1234633861]).

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"Beyond our broad development program for domvanalimab, our potentially best-in-class Fc-silent anti-TIGIT antibody, in lung and upper GI cancers, our pipeline continues to mature with multiple clinical-stage molecules being evaluated across common cancers of high unmet need. In addition, our discovery platform is expected to continue to generate at least one IND per year, and we expect to advance two new molecules into the clinic prior to the end of 2023," said Terry Rosen, Ph.D., chief executive officer of Arcus. "By early next year, we expect to present several key datasets across our pipeline, including data from our Phase 2 EDGE-Gastric study (ARC-21) for domvanalimab in first-line gastric cancer, overall survival data from ARC-8 for quemliclustat in pancreatic cancer, and data from the Phase 1b ARC-20 study for AB521, our HIF-2a inhibitor."

Pipeline Highlights:

Domvanalimab (Fc-silent anti-TIGIT monoclonal antibody)

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Arcus and Gilead presented interim data from the ongoing ARC-7 study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2023.
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At the time of data cutoff (DCO), February 7, 2023, safety and efficacy were evaluated in all patients treated (n=150).
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With a median follow-up time of approximately 18 months, both domvanalimab-containing study arms demonstrated sustained, clinically meaningful improvements in progression-free survival (PFS) compared to zimberelimab (Z) monotherapy, with a 33% reduction in risk of disease progression or death for the doublet and 28% for the triplet.
•
As of the DCO, approximately twice as many participants remained on study treatment in each of the domvanalimab-containing arms compared to zimberelimab monotherapy. Preliminary duration of response (DoR) analyses also substantially favored the domvanalimab-containing arms.

•
Consistent ORR and PFS improvements were observed for the domvanalimab-containing arms in a post-hoc analysis of centrally confirmed PD-L1-high patients.
•
All treatment combinations were generally well tolerated; no new safety signals were observed across the three study arms.
•
Initial ORR data from the ongoing EDGE-Gastric (ARC-21) Phase 2 study of domvanalimab plus zimberelimab and chemotherapy in first-line upper GI cancers are anticipated in the fourth quarter of 2023. These data will be from the cohort that includes the same patient population and dosing regimen as the ongoing Phase 3 study, STAR-221.
•
Domvanalimab has the potential to be first-to-market in this indication, since it is currently the only anti-TIGIT antibody in late-stage clinical development for gastric, esophageal, and gastro-esophageal junction adenocarcinoma. These tumor types represent a potential drug-treatable population of over 25,000 in the US and over 100,000 in G-7 countries.

AB521 (HIF-2a inhibitor)

•
Arcus has initiated the dose-expansion stage of ARC-20, a Phase 1/1b study of AB521 in ccRCC patients, evaluating a 100-mg daily dose, which Arcus believes has the potential to achieve at least 3 times higher levels of the HIF-2a inhibitor than those equivalent to the approved dose of the marketed competitor. No dose-limiting toxicities have been observed to date in ARC-20.
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Initial pharmacokinetic (PK), pharmacodynamic (PD) and safety data, along with any preliminary signs of anti-tumor activity from the dose-escalation phase of ARC-20, are expected in late 2023 or early 2024. Efficacy data from the dose-expansion stage of the ARC-20 study are anticipated later in 2024.
•
A Phase 2 study evaluating AB521 in combination with other agents is anticipated to begin in the fourth quarter of 2023.
Quemliclustat (small-molecule CD73 inhibitor)

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Arcus conducted an analysis of data from the ongoing Phase 1/1b ARC-8 trial evaluating quemliclustat plus chemotherapy with or without zimberelimab in first-line pancreatic cancer.
o
At this analysis, patients had a median follow-up time of 22 months, and the overall survival data were mature.
o
These data were encouraging relative to historical benchmarks for chemotherapy alone and will be submitted for presentation at a future scientific conference.

Etrumadenant (A2a/A2b adenosine receptor antagonist)

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Arcus and Gilead reviewed radiographic PFS (rPFS) data from the randomized cohort of ARC-6, a Phase 1b/2 study evaluating etrumadenant plus zimberelimab and docetaxel versus docetaxel in metastatic castrate-resistant prostate cancer (mCRPC).
o
While the trial will continue to completion, further development of etrumadenant in mCRPC has been deprioritized.
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Data from ARC-9, a Phase 1b/2 study evaluating etrumadenant plus zimberelimab plus chemotherapy in second-line and third-line metastatic colorectal cancer (mCRC), which is fully enrolled, are expected in the first half of 2024, due to a slower-than-anticipated event rate.

Early Clinical and Preclinical Programs

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Arcus initiated ARC-25, a Phase 1 trial in cancer patients for AB598, its anti-CD39 antibody, in the second quarter of 2023.
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Arcus expects to initiate a Phase 1 study in healthy volunteers of AB801, its potent and highly selective Axl inhibitor, in the third quarter of 2023.
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Arcus has identified several chemotypes against KIT, a target involved in multiple allergic and immune-mediated diseases. Advanced molecules, with differentiated chemotypes relative to the previously identified AB375, are undergoing characterization. Arcus expects to select a new development candidate by year end.

Corporate Highlights:

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In May 2023, Arcus and Gilead expanded their previously announced research collaboration focused on oncology to include therapies for the treatment of inflammatory diseases.
o
Under the terms of the expanded collaboration, Arcus received an upfront payment of $35 million and will initiate research programs against up to four targets jointly selected by the parties that are applicable to inflammatory diseases.
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Gilead may exercise an option to license each program at two separate, prespecified time points.
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If Gilead exercises its option at the earlier time point for the first two target programs, Arcus would be eligible to receive up to $420 million in option and milestone payments as well as tiered royalties for each optioned program.
o
For any other option exercise by Gilead for the four target programs, the parties would have rights to co-develop and share global development costs and to co-commercialize and share profits in the United States for optioned programs.
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In June 2023, Arcus sold 1.0 million shares of common stock to Gilead at a purchase price of $19.26 per share, increasing Gilead’s ownership to 19.9%. Gross proceeds to Arcus from the transaction were $19.5 million.

Financial Results for Second Quarter 2023:

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Cash, cash equivalents and marketable securities were $1.0 billion as of June 30, 2023, compared to $1.1 billion as of December 31, 2022. The decrease during the period is primarily due to the use of cash in research and development activities, partially offset by receipts of $35 million in upfront payments from Gilead to initiate Arcus-led discovery and early development activities on two jointly selected inflammation targets and $20 million in proceeds from their purchase of 1.0 million shares of our common stock. Arcus now expects cash utilization between $290 million and $325 million for the year ended December 31, 2023. Arcus continues to expect cash, cash equivalents and marketable securities on-hand to be sufficient to fund operations into 2026.
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Revenues were $29 million for the second quarter 2023, compared to $27 million for the same period in 2022. In the second quarter 2023, Arcus recognized $19 million in license and development service revenues related to the advancement of programs under the Gilead collaboration as well as $10 million in other collaboration revenue primarily related to Gilead’s ongoing rights to access Arcus’s research and development pipeline in accordance with the Gilead collaboration agreement. Revenues were $54 million for the six months ended June 30, 2023, compared to $45 million for the same period in 2022.
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Research and Development (R&D) Expenses were $84 million for the second quarter 2023, compared to $70 million for the same period in 2022. Arcus’s expanding clinical and development activities increased costs by $19 million, partially offset by $5 million in higher reimbursements for shared expenses from Arcus’s collaborations, primarily the Gilead collaboration. The net increase was driven primarily by: increases in clinical costs due to Arcus’s expanding clinical and development activities as Arcus enrolled more patients in its existing and new studies; increases in net employee compensation costs due to our growing headcount; partially offset by a decrease in net drug manufacturing costs due to less manufacturing activity for domvanalimab. Non-cash stock-based compensation expense was $9 million and $7 million for the second quarter 2023 and 2022, respectively. R&D expenses were $165 million for the six months ended June 30, 2023, compared to $131 million for the same period in 2022. For second-quarter 2023 and 2022, Arcus recognized reimbursements of $44 million and $39 million, respectively, for shared expenses from its collaborations, primarily the Gilead collaboration. Reimbursements were $86 million for the six months ended June 30, 2023, compared to $70 million for the same period in 2022.
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General and Administrative (G&A) Expenses were $28 million for the second quarter 2023, compared to $26 million for the same period in 2022. The increase was primarily driven by the increased complexity of supporting Arcus’s expanding clinical pipeline and partnership obligations. Non-cash stock-based compensation expense was $9 million for the second quarter 2023, compared to $8 million for the same period in 2022. G&A expenses were $58 million for the six months ended June 30, 2023, compared to $50 million for the same period in 2022.
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Net Loss was $75 million for the second quarter 2023, compared to $67 million for the same period in 2022. Net loss was $155 million for the six months ended June 30, 2023, compared to $135 million for the same period in 2022.

Arcus Ongoing and Announced Clinical Studies

Trial Name

Arms

Setting

Status

NCT No.

Lung Cancer

ARC-7

zim vs. dom + zim vs. etruma + dom + zim

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Randomized Phase 2

NCT04262856

PACIFIC-8

(Operationalized by AZ)

dom + durva vs. durva

Curative-Intent Stage 3 NSCLC

Ongoing Registrational Phase 3

NCT05211895

ARC-10

dom + zim vs. pembro

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Registrational Phase 3

NCT04736173

STAR-121

(Operationalized by Gilead)

dom + zim + chemo vs. pembro + chemo

1L NSCLC (PD-L1 all-comers)

Ongoing Registrational Phase 3

NCT05502237

EDGE-Lung

dom +/- zim +/- quemli +/- chemo

1L/2L NSCLC (lung cancer platform study)

Ongoing Randomized Phase 2

NCT05676931

VELOCITY-Lung

(Operationalized by Gilead)

dom +/- zim +/- etruma +/- sacituzumab govitecan-hziy or other combos

1L/2L NSCLC (lung cancer platform study)

Ongoing Randomized Phase 2

NCT05633667

Gastrointestinal Cancers

ARC-9

etruma + zim + mFOLFOX vs. SOC

2L/3L/3L+ CRC

Ongoing

Randomized Phase 2

NCT04660812

EDGE-Gastric (ARC-21)

dom +/- zim +/- quemli +/- chemo

1L/2L Upper GI Malignancies

Ongoing

Randomized Phase 2

NCT05329766

STAR-221

dom + zim + chemo vs. nivo + chemo

1L Gastric, Gastroesophageal Junction (GEJ), and Esophageal Adenocarcinoma (EAC)

Ongoing Registrational Phase 3

NCT05568095

Pancreatic Cancer

ARC-8

quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac

1L, 2L PDAC

Ongoing Randomized Phase 1/1b

NCT04104672

Prostate Cancer

ARC-6

etruma + zim + SOC vs. SOC (also enrolling sacituzumab govitecan-hziy combination cohorts)

2L/3L CRPC

Ongoing Randomized Phase 2

NCT04381832

Renal Cancer

ARC-20

AB521

Cancer Patients / ccRCC

Ongoing Phase 1/1b

NCT05536141

Other

ARC-25

AB598

Advanced Malignancies

Ongoing

NCT05891171

dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; nivo: nivolumab; pembro: pembrolizumab; quemli: quemliclustat; SOC: standard of care; zim: zimberelimab

ccRCC: clear-cell renal cell carcinoma; CRC: colorectal cancer; CRPC: castrate-resistant prostate cancer; GI: gastrointestinal; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

On August 7, 2023 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported operating results for the second quarter ended June 30, 2023 and provided a business update (Press release, AnaptysBio, AUG 7, 2023, View Source [SID1234633860]).

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"We have made substantial operating progress including initiating a Phase 2b trial in atopic dermatitis (AD) for ANB032, our BTLA agonist, and approaching initiation of a Phase 2b trial in RA for rosnilimab, our PD-1 agonist," said Daniel Faga, president and chief executive officer of AnaptysBio. "Additionally, we are excited to share that we recently completed enrollment of the GEMINI-1 Phase 3 clinical trial for imsidolimab in GPP and expect to share top-line data in Q4 2023."

"We are excited to appoint Dan Faga to the permanent position of president and CEO," said Jamie Topper, M.D., Ph. D., chairman of the Board of Directors. "Over the last year, Anaptys has completed its strategic portfolio review and Dan led the transition refocusing on the broad development of our differentiated immune cell modulators, including our checkpoint agonist pipeline, in autoimmune and inflammatory diseases. With Dan and his talented team in place, and our strong capital position, the company is well positioned as it enters its next phase of development and growth."

Updates on Wholly Owned Immune Cell Modulator Pipeline

ANB032 (BTLA agonist antibody)

Initiated a global Phase 2b trial in moderate-to-severe AD
160-patient placebo-controlled trial assessing three dose levels of subcutaneously administered ANB032 (randomized 1:1:1:1) for a 14-week treatment duration and six-month follow-up period on well established endpoints, including EASI75 and IGA 0/1
Top-line week 14 data anticipated by year-end 2024
Hosted a virtual BTLA Agonist (ANB032) R&D Event in May 2023
Replay of the audio webcast is available here
Rosnilimab (PD-1 agonist antibody)

Anticipate initiation later in Q3 2023 of a global Phase 2b trial in moderate-to-severe RA
Multi-hundred patient placebo-controlled trial assessing three dose levels of subcutaneously administered rosnilimab for up to six months on well-established endpoints including ACR20/50/70 and DAS28
Top-line week 12 data anticipated by mid-year 2025
Plan to initiate a second global Phase 2 trial, in a yet to-be-announced indication, by year-end 2023
Plan to host a virtual PD-1 Agonist (rosnilimab) R&D Event in Q4 2023
ANB033 (anti-CD122 antagonist antibody)

Presented poster on preclinical data for ANB033, an anti-CD122 antagonist for the treatment of inflammatory diseases, at the Federation of Clinical Immunology Societies (FOCIS) Annual Meeting, in June 2023
Poster presentation is available here
Plan to submit an Investigational New Drug (IND) application in H1 2024
Updates on Legacy Clinical-Stage Cytokine Antagonist Programs Available for Out-Licensing

Completed enrollment of the GEMINI-1 Phase 3 trial for imsidolimab (IL-36R) in GPP per the initial target enrollment (n=45)
Top-line data anticipated in Q4 2023
Plan to out-license imsidolimab prior to potential FDA approval
Updates on GSK Immuno-Oncology Financial Collaboration

GSK received U.S FDA approval for Jemperli (dostarlimab) in combination with chemotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer on July 31, 2023
Jemperli is the first immuno-oncology treatment approved in the frontline setting for this patient population in combination with chemotherapy
GSK anticipates top-line data in H1 2024 from the FIRST Phase 3 trial for platinum-based therapy with dostarlimab and niraparib versus platinum-based therapy as first-line treatment of Stage III or IV nonmucinous epithelial ovarian cancer
GSK anticipates top-line data in H2 2024 from COSTAR Lung Phase 3 trial comparing cobolimab plus dostarlimab plus docetaxel to dostarlimab plus docetaxel to docetaxel alone in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and chemotherapy
Organizational Updates

Announced appointment of Daniel Faga to the permanent position of president and chief executive officer of the Company
Mr. Faga will retain his position on the Company’s Board of Directors
Announced appointments of Luisa Salter-Cid, Ph.D., and Dolca Thomas, M.D., to the Company’s Scientific Advisory Board (SAB)
Dr. Salter-Cid is the current chief scientific officer at Pioneering Medicines, a strategic initiative within Flagship Pioneering. She had extensive experience at Bristol-Meyers Squibb where she led teams that advanced more than 20 compounds into clinical development.
Dr. Thomas is currently a venture partner at Samsara BioCapital and serves on the Board of Directors of Allakos Therapeutics, Chinook Therapeutics and Ventus Therapeutics. Dr. Thomas has extensive experience in both large pharma and biotech. Among her prior roles includes serving as Principia’s chief medical officer from 2018 until the Sanofi acquisition in September 2020. Dr. Thomas was also vice president and global head of Translational Medicine for Immunology, Inflammation, and Infectious Disease at Roche, where she was responsible for advancing multiple product candidates through clinical development.
Read their full bios here
Year-End Cash Guidance

Reiterating cash runway through year-end 2026 with updated expected year-end 2023 cash and investments of $380 – $395 million
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $488.7 million as of June 30, 2023, compared to $584.2 million as of December 31, 2022, for a decrease of $95.5 million. The decrease relates primarily to cash used for the $50 million stock repurchase program and operating activities.
Collaboration revenue was $3.5 million and $4.8 million for the three and six months ended June 30, 2023, compared to $1.2 million and $2.2 million for the three and six months ended June 30, 2022. The change is due primarily to increased royalties recognized for sales of Jemperli.
Research and development expenses were $32.9 million and $67.9 million for the three and six months ended June 30, 2023, compared to $20.8 million and $43.4 million for the three and six months ended June 30, 2022. The increase was due primarily to manufacturing and development costs for imsidolimab, rosnilimab, ANB032 and ANB033. The R&D non-cash, stock-based compensation expense was $2.7 million and $5.5 million for the three and six months ended June 30, 2023 as compared to $1.8 million and $3.4 million in the same period in 2022.
General and administrative expenses were $10.7 million and $21.5 million for the three and six months ended June 30, 2023, compared to $8.2 million and $18.4 million for the three and six months ended June 30, 2022. The G&A non-cash, stock-based compensation expense was $5.7 million and $11.8 million for the three and six months ended June 30, 2023 as compared to $4.9 million and $11.0 million in the same period in 2022.
Net loss was $39.8 million and $84.1 million for the three and six months ended June 30, 2023, or a net loss per share of $1.50 and $3.08, compared to a net loss of $32.6 million and $68.8 million for the three and six months ended June 30, 2022, or a net loss per share of $1.15 and $2.46.