On August 9, 2021 -Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported financial results for the second quarter 2021, recent business highlights and key catalysts over the next several months (Press release, Atara Biotherapeutics, AUG 9, 2021, View Source [SID1234586116]).

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!

"We are very pleased with the progress achieved across all of our programs and important clinical updates across our full pipeline, which will enable us to deliver on key value drivers through the rest of this year and into 2022," said Pascal Touchon, President and Chief Executive Officer of Atara. "Recent tab-cel regulatory progress and alignment with both FDA and EMA allow us to continue anticipating approvals in 2022, while exciting new ATA188 imaging data reinforces our belief in this potentially transformative therapy for patients with multiple sclerosis."

Tabelecleucel (tab-cel) for Post-Transplant Lymphoproliferative Disease (PTLD)

Atara conducted productive meetings with the U.S. Food and Drug Administration (FDA), gaining clarity on specific next steps required for submission of the Biologics License Application (BLA)
Alignment on key methodologies for evaluating comparability between Atara product used in the pivotal ALLELE study and the intended commercial product. Atara will provide data on substantially all lots made to date through a Type B CMC meeting to enable FDA to make a final determination on the data package to support comparability
FDA decided it cannot make a determination of comparability between material used in non-pivotal and pivotal studies because analytical data is not available and cannot be generated for all lots manufactured at Memorial Sloan Kettering Cancer Center (MSK) and used in non-pivotal studies. Consequently, Atara plans to submit clinical data from the pivotal ALLELE study and the non-pivotal studies as separate, non-pooled analyses in the anticipated BLA submission
FDA has not requested additional assays or manufacturing lots
Atara completed a new data analysis from its Phase 3 ALLELE study, as previously discussed with FDA. Top-line data with additional patients confirm a strong objective response rate (ORR) in line with prior results while demonstrating durability. There were no new safety signals, consistent with previously published findings
These data will be discussed with the FDA through a Type B meeting and are planned to be presented at an appropriate congress in Q4 2021
Based on the outcome of the recent and anticipated interactions with the FDA, Atara expects to complete the BLA submission for tab-cel in Q1 2022. Atara is adapting investment in U.S. commercial readiness toward anticipated approval in H2 2022
Following recent successful Rapporteur/Co-Rapporteur and pre-submission meetings with the European Medicines Agency (EMA), Atara has completed the necessary regulatory and compliance steps needed to submit an EU Marketing Authorization Application (MAA) for tab-cel in patients with EBV+ PTLD, which is on track for November 2021. The Company anticipates a decision regarding approval in H2 2022
Discussions with potential partners for the commercialization of tab-cel in Europe are advancing well and are in line with our expectation to secure a partner by Q4 2021
Atara presented data at the American Transplant Congress (ATC) in June 2021 from a combined long-term overall survival (OS) analysis from three clinical studies of tab-cel demonstrating that patients with EBV+ PTLD following solid organ transplant (SOT) that is relapsed or refractory to initial treatment, derived similar OS benefit of greater than 80 percent at two years whether they achieved complete or partial response with tab-cel
Tab-cel for Potential Additional Indications

Atara continues to pursue development of tab-cel in additional patient populations with an initial focus on immunodeficiency-associated lymphoproliferative diseases (IA-LPDs), given the commonality of their EBV-driven mechanism of disease in immunocompromised patients, high unmet medical need, and positive clinical data to date with tab-cel
Actively enrolling at sites in the Phase 2 multi-cohort study, which is evaluating six patient populations, including four within IA-PLDs and two in other EBV-driven diseases, in the U.S. and EU
Atara joined the Rare Disease Company Coalition, an alliance of innovative life sciences companies engaging policymakers on the unique needs of the rare disease community, the urgency of and support for innovation to address significant unmet patient need and dialogue around policies that advocate for timely and appropriate access to safe and transformational treatments, including tab-cel
ATA188 for Progressive Forms of Multiple Sclerosis (PMS)

Long-term, two-year clinical data from the Phase 1 open-label extension (OLE) and translational data from the Phase 1 study of ATA188 in PMS has been accepted for presentation at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in October
The presentation, titled "Updated open-label extension clinical data and new magnetization transfer ratio imaging data from a Phase I study of ATA188, an off-the-shelf, allogeneic Epstein-Barr virus-targeted T-cell immunotherapy for progressive multiple sclerosis" will feature updated clinical and new imaging biomarker data considered to reflect the state of myelination in the central nervous system, known as magnetization transfer ratio (MTR)
MTR may provide important insights into the mechanism of expanded disability status scale (EDSS) improvement in our clinical assessment of ATA188
Atara is making progress on enrolling the ATA188 Phase 2 randomized, double-blind, placebo-controlled trial (EMBOLD study) evaluating the efficacy and safety of ATA188 in patients with PMS
Planned interim analysis in H1 2022 to assess efficacy and safety
Based on current target, Atara expects to complete enrollment in H1 2022
The Company will present a poster on PMS fatigue and an oral presentation on the ongoing EMBOLD study at the upcoming 2021 Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC) in October
There is growing awareness of the transformative potential of ATA188 in MS among neurologists
CAR T Programs

ATA2271/ATA3271 (Solid Tumors Over-Expressing Mesothelin)

The global strategic collaboration for ATA2271 and ATA3271 with Bayer continues to progress well with advancement of the mesothelin-partnered CAR T immunotherapy programs
The Company anticipates presentation of the first update on clinical data from the open-label, single-arm Phase 1 clinical study of ATA2271, an autologous CAR T therapy targeting mesothelin, designed to improve efficacy, persistence, and durability of response for patients with advanced mesothelioma, in an appropriate forum in Q4 2021. Enrollment has been completed for the first cohort and is nearly complete for the second cohort of this study
Atara is continuing to make progress on IND-enabling studies for ATA3271, an off-the-shelf, allogeneic CAR T therapy targeting mesothelin using next-generation PD-1 dominant negative receptor (DNR) and 1XX CAR co-stimulatory signaling domain technologies and expects an IND filing in H2 2022
The Company plans to present preclinical data for ATA3271 at an appropriate forum in Q4 2021
ATA3219 (B-cell Malignancies)

Atara expects to submit an IND for ATA3219, an off-the-shelf, allogeneic CD19 CAR T immunotherapy targeting B-cell malignancies, as a potential best-in-class therapy without the need for T-cell receptor (TCR) gene editing, using our next-generation 1XX CAR co-stimulatory signaling domain and EBV T-cell platform in Q1 2022
Executive and Board Appointments

Cell therapy and oncology expert, Cokey Nguyen, Ph.D., recently joined Atara as Chief Scientific Officer to further our mission of developing transformative therapies for patients with severe diseases. Dr. Nguyen joined Atara from Fate Therapeutics, where, as Vice President, Innovation, Research and Development, he directed strategy for discovery and innovation efforts, and spearheaded the corporate collaboration program with ONO Pharma
Cell therapy and oncology commercialization veteran, Ameet Mallik, was appointed to the Board of Directors. Mr. Mallik is CEO of Rafael Holdings, a late-stage cancer metabolism therapeutic company and has held several leadership roles at Novartis, most recently as EVP & Head of U.S. Oncology. He brings to Atara’s Board a wealth of experience with U.S. payer, access and reimbursement strategies and launches of innovative oncology therapies, including CAR T
Second Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments as of June 30, 2021 totaled $373.4 million, as compared to $435.2 million as of March 31, 2021
Atara believes that its cash as of June 30, 2021 is sufficient to fund planned operations into 2023
License and collaboration revenue was $3.9 million for the second quarter 2021 and consisted of revenue from activities performed under the Bayer Collaboration Agreements. Atara did not recognize any license and collaboration revenue for the same period in 2020
Net cash used in operating activities was $61.6 million for the second quarter 2021, as compared to $56.6 million for the same period in 2020
Atara reported net losses of $83.8 million, or $0.91 per share, for the second quarter 2021, as compared to $77.5 million, or $1.14 per share, for the same period in 2020
Total operating expenses include non-cash expenses of $16.1 million for the second quarter 2021, as compared to $15.9 million for the same period in 2020
Research and development expenses were $68.5 million for the second quarter 2021, as compared to $61.6 million for the same period in 2020
The increase in the second quarter 2021 was primarily due to higher employee-related costs from increased headcount, increased spending on the Company’s ATA188 and CAR T programs and increased facilities and information technology expenses allocated to research and development
Research and development expenses include $8.3 million of non-cash stock-based compensation expenses for the second quarter 2021, as compared to $8.5 million for the same period in 2020
General and administrative expenses were $19.4 million for the second quarter 2021, as compared to $16.4 million for the same period in 2020
General and administrative expenses include $5.5 million of non-cash stock-based compensation expenses for the second quarter 2021, as compared to $5.4 million for the same period in 2020
Conference Call and Webcast Details

Atara will host a live conference call and webcast today, Monday, August 9, 2021, at 4:30 p.m. EDT to discuss the Company’s financial results and recent operational highlights. Analysts and investors can participate in the conference call by dialing 877-407-8291 for domestic callers and 201-689-8345 for international callers, using the conference ID 13720150. A live audio webcast can be accessed by visiting the Investors & Media – News & Events section of atarabio.com. An archived replay will be available on the Company’s website for 30 days.

On August 9, 2021 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the second quarter 2021 and provided an update on the Company’s recent developments (Press release, FibroGen, AUG 9, 2021, View Source [SID1234586115]).

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!

"We continue to be energized by the performance of roxadustat in China, where we are seeing impressive adoption of roxadustat in a rapidly expanding anemia of CKD market. In addition, the positive CHMP opinion in Europe brings roxadustat one step closer to patients in this important region," said Enrique Conterno, Chief Executive Officer, FibroGen. "We look forward to the European Commission decision following the positive CHMP opinion. On the other hand, we are disappointed with the FDA Cardiovascular and Renal Drugs Advisory Committee negative vote, and we will continue to work with our partner AstraZeneca and the FDA on a path forward."

Recent Key Events and Other Developments

Regulatory:

In June, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion relating to the use of roxadustat for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD). A European Commission decision is expected in August 2021.
In July, the U.S. Food and Drug Administration (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted to recommend not approving roxadustat for the treatment of anemia due to CKD. An FDA action on the new drug application is expected in the near future.
_____________________________

1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Clinical:

Pamrevlumab included in the Pancreatic Cancer Action Network’s (PanCAN) Precision PromiseSM adaptive trial platform evaluating pamrevlumab for patients with metastatic pancreatic cancer.
China:

Roxadustat net product revenue in China of $13.4 million, on a US GAAP basis, including revenue generated from our sales to the distribution entity and FibroGen China’s direct sales, compared to $15.4 million last quarter.
Total roxadustat net sales in China of $52.8 million by FibroGen and the distribution entity jointly owned by FibroGen and AstraZeneca, compared to $43.5 million last quarter.
Hospital listings at the end of the second quarter represented approximately 81% of the CKD anemia market opportunity in China versus 74% last quarter.
Clinical Presentations / Publications:

FibroGen and its partners presented three roxadustat oral presentations and ten mini-oral presentations at the recent 58th European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Virtual Congress.
Two additional roxadustat Phase 3 manuscripts on the treatment of anemia of CKD were published in a peer-reviewed medical journal, bringing the total to 7:
Roxadustat for the treatment of anaemia in chronic kidney disease patients not on dialysis: a phase 3, randomised, open-label, active-controlled study (DOLOMITES) Nephrology Dialysis Transplantation.
Efficacy and Cardiovascular Safety of Roxadustat for Treatment of Anemia in Patients with Non–Dialysis-Dependent CKD (NDD Pooled) Clinical Journal of the American Society of Nephrology.
FibroGen presented one oral presentation and one poster presentation of two-year data from a Phase 2 trial of pamrevlumab in non-ambulatory DMD patients at the recent Parent Project Muscular Dystrophy (PPMD) Annual Conference.
Upcoming Data Milestones:

Data from the Phase 2 WHITNEY study of roxadustat in chemotherapy-induced anemia (CIA) expected 3Q 2021 versus prior 2H 2021.
Topline data from the Phase 3 MATTERHORN study of roxadustat in anemia of myelodysplastic syndromes (MDS) now expected 2H 2022 / 1H 2023 versus prior 1H 2022.
Interim analysis of event free survival for potential accelerated approval of Phase 3 LAPIS study of pamrevlumab in locally advanced pancreatic cancer (LAPC) will be completed in 2H 2022.
Topline data from the Phase 3 LELANTOS-1 study of pamrevlumab in Duchenne muscular dystrophy (DMD) now expected 1H 2023 versus prior 2H 2022.
Topline data from the Phase 3 ZEPHYRUS-1 study of pamrevlumab in idiopathic pulmonary fibrosis (IPF) expected mid-2023.
Corporate

Appointed John Hunter, Ph.D. as Chief Scientific Officer.
FibroGen and HiFiBiO announced partnership to advance next-generation therapies for patients with cancer and autoimmune disease. FibroGen exclusively licensed HiFiBiO’s Galectin-9 program, and obtained an exclusive option to their CXCR5 and CCR8 programs.
Eluminex Biosciences exclusively licensed worldwide rights to develop and commercialize FibroGen’s investigational biosynthetic cornea program and the underlying recombinant human collagen Type III technology.
The Company has completed its internal review of the events leading to our April 6, 2021 disclosures in which we clarified that certain cardiovascular safety analyses from the roxadustat Phase 3 program for the treatment of anemia of chronic kidney disease included post-hoc changes to the stratification factors.
Financial:

Total revenue for the second quarter of 2021 was $24.4 million, as compared to $42.9 million for the second quarter of 2020. The current quarter revenue includes $13.4 million net product revenue for roxadustat sales in China, and $19.6 million in development revenue. The current quarter also includes $(8.6) million drug product revenue as a result of the recent unfavorable CRDAC vote.
Operating costs and expenses for the second quarter of 2021 included a one-time charge of $25 million related to our partnership with HiFiBio, and an increase of approximately $20 million driven primarily by pamrevlumab development expenses compared to one year ago.
Net loss for the second quarter of 2021 was $134.0 million, or $1.45 net loss per basic and diluted share, compared to a net loss of $85.3 million, or $0.95 net loss per basic and diluted share one year ago.
Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, August 9, 2021, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update. A live audio webcast of the call may be accessed in the investor section of the Company’s website, www.fibrogen.com. To participate in the conference call by telephone, please dial 1 (877) 658-9081 (U.S. and Canada) or 1 (602) 563-8732 (international), reference the FibroGen second quarter 2021 financial results conference call, and use confirmation number 4951789. A replay of the webcast will be available shortly after the call for a period of four weeks. To access the replay, please dial 1 (855) 859-2056 (domestic) or 1 (404) 537-3406 (international), and use passcode 4951789.

About Roxadustat
Roxadustat, an oral medicine, is the first in a new class of medicines, HIF-PH inhibitors, that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin; improved iron absorption and mobilization; and downregulation of hepcidin. Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in China, Japan, Chile, and South Korea for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). In Europe, the Marketing Authorisation Application is under review by the European Medicines Agency (EMA). In the U.S., the New Drug Application is under review by the U.S. Food and Drug Administration. Several other licensing applications for roxadustat have been submitted by Astellas and AstraZeneca to regulatory authorities across the globe, and are currently in review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, other markets in the Americas, in Australia/New Zealand, and Southeast Asia.

About Pamrevlumab
Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

On August 9, 2021 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, immunologic diseases (e.g., complement-mediated diseases and cancers) and central nervous system disorders, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2021, which include (Press release, Omeros, AUG 9, 2021, View Source [SID1234586114]):

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!

OMIDRIA revenues for the second quarter of 2021 were $28.8 million compared to $21.1 million in the first quarter. The 37 percent increase over the prior quarter primarily reflects growth in sales of OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% in ambulatory surgery centers (ASCs).
Net loss in the second quarter of 2021 was $28.6 million, or $0.46 per share, including non-cash expenses of $3.9 million, or $0.06 per share. This compares to a net loss of $35.1 million, or $0.57 per share, which included non-cash expenses of $4.1 million, or $0.07 per share, for the previous quarter.
At June 30, 2021, Omeros had cash, cash equivalents and short-term investments available for operations of $73.7 million.
Omeros’ Biologics License Application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangipathy (HSCT-TMA or TA-TMA) is under priority review by the U.S. FDA with an action date of October 17, 2021 under the Prescription Drug User Fee Act (PDUFA).
Omeros announced preliminary results from the Phase 1 clinical trial of OMS906, the company’s MASP-3 inhibitor, which showed that (i) OMS906 was well tolerated at all doses tested and (ii) human pharmacokinetic and pharmacodynamic data are consistent with once-monthly or less frequent subcutaneous dosing.
"In the second quarter of 2021, Omeros achieved a number of important milestones," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "As OMIDRIA sales continue to grow, CMS in its recent OPPS Proposed Rule reaffirmed its determination that OMIDRIA receive separate payment when used in ASCs, and our MASP-3 inhibitor OMS906 is successfully advancing through its Phase 1 clinical trial. Several other important milestones should reach resolution in the near term – FDA’s decision on our pending BLA for narsoplimab in the treatment of patients with TA-TMA, results from narsoplimab in the COVID-19 I-SPY platform clinical trial, and the outcome of the NOPAIN Act, which, if enacted by Congress, would mandate Medicare separate payment for non-opioid surgical pain management drugs like OMDRIA not only in ASCs but also in hospital outpatient departments. The remainder of our pipeline also pressed ahead, including the GPR174 program, the core of our immuno-oncology platform – a platform that has expanded beyond GPR174 with new CAR-T and adoptive cellular therapy programs. Omeros’ momentum is building, and we look forward to seeing what the rest of the year brings."

Second Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:
In July 2021, the Centers for Medicare and Medicaid Services (CMS) released its Outpatient Prospective Payment System (OPPS) and ASC Payment System proposed rule for calendar year 2022, which reaffirmed its earlier decision that OMIDRIA, when used in the ASC setting, qualifies for separate payment under CMS’ policy regarding non-opioid pain management surgical drugs. This policy has been in effect since 2019.
Recent developments regarding narsoplimab, Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in advanced clinical programs for the treatment of TA-TMA, immunoglobulin A (IgA) nephropathy, atypical hemolytic uremic syndrome (aHUS) and severely ill COVID-19 patients, include the following:
Omeros announced data from the second cohort of 10 critically ill COVID-19 patients treated with narsoplimab under compassionate use in Bergamo, Italy.
All of the patients had comorbidities and/or risk factors for poor outcomes, were mechanically ventilated, and had failed other therapies, including steroids.
90 percent of the patients were intubated prior to initiation of narsoplimab treatment.
80 percent of the patients recovered, survived and were discharged. The two deaths involved a 76-year-old who died of complications related to his pre-existing cardiomyopathy and a 68-year-old who began narsoplimab treatment after 13 days of intubation.
A manuscript from the Omeros-Cambridge Center for Complement and Inflammation Research (OC3IR) on the inhibition of the lectin pathway and MASP-2 as a potential treatment for severe COVID-19 was published in the peer-reviewed journal Frontiers in Immunology.
An abstract on the pharmacokinetic and pharmacodynamic modeling of lectin pathway inhibition by narsoplimab was accepted for presentation at the 16th International Symposium on IgA nephropathy.
Updates regarding Omeros’ other development programs and platforms include the following:
Omeros announced preliminary results from the Phase 1 clinical trial evaluating the pharmacokinetics/pharmacodynamics (PK/PD) and safety of OMS906 in healthy subjects. OMS906 inhibits MASP-3, the key activator of the alternative pathway of complement. MASP-3 is responsible for the conversion of pro-factor D to mature factor D. Data from the first five cohorts of the trial’s single-ascending dose stage show that (i) OMS906 was well tolerated at all doses tested (up to 5 mg/kg) and (ii) a single 3 mg/kg intravenous dose of OMS906 and a single dose of the lowest subcutaneous concentration tested each suppressed mature complement factor D below minimum detectable levels. The human PK/PD data were consistent with once-monthly or less frequent subcutaneous dosing. Following completion of the single- and multiple-ascending dose stages of the Phase 1 trial, Omeros plans to initiate a Phase 2 clinical trial.
A paper detailing the mechanism of action of PDE7 inhibition in nicotine addiction was published in the peer-reviewed Journal of Neuroscience and was selected for inclusion in the journal’s Featured Research page. Omeros has completed a successful Phase 1 trial with the lead compound in its PDE7 inhibitor program.
Financial Results

For the second quarter of 2021, OMIDRIA revenues were $28.8 million compared to $21.1 million for the first quarter.

Total costs and expenses for the second quarter of 2021 were $52.8 million compared to $51.7 million for the first quarter. The increase was primarily due to increased selling, general and administrative expenses in preparation for the anticipated U.S. launch of narsoplimab and additional employee-related costs. Research and development costs decreased quarter over quarter due to the timing of narsoplimab manufacturing-related costs, which are expensed rather than included as inventory until the initial marketing approval for narsoplimab is certain.

For the three months ended June 30, 2021, Omeros reported a net loss of $28.6 million, or $0.46 per share, which included non-cash expenses of $3.9 million, or $0.06 per share. This compares to a net loss in the previous quarter of $35.1 million, or $0.57 per share, which included non-cash expenses of $4.1 million, or $0.07 per share.

As of June 30, 2021, the company had $73.7 million of cash, cash equivalents and short-term investments. The company also has a line of credit, which permits borrowing up to the lesser of $50.0 million and 85 percent of eligible accounts receivable less certain reserves. Omeros also has an "at the market" program in place that allows the company to sell, from time to time, up to $150.0 million of its common stock.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 4195376. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 4195376.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at View Source

On August 9, 2021 QIAGEN (NYSE:QGEN; Frankfurt Prime Standard: QIA) and OncXerna Therapeutics, Inc. ("OncXerna"), a precision medicine company using an innovative RNA-expression based biomarker platform to predict patient responses to its targeted oncology therapeutic candidates, reported signing a master companion diagnostics (CDx) agreement to develop a NGS CDx for OncXerna’s product candidate, Navicixizumab, and a non-exclusive license to the Xerna TME panel (Press release, OncXerna Therapeutics, AUG 9, 2021, View Source [SID1234586113]).

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!

QIAGEN and OncXerna have agreed to collaborate to advance the Xerna TME panel towards IVD (in-vitro diagnostic) regulatory approval as a NGS companion diagnostic for Navicixizumab, which is being developed by OncXerna as a treatment for patients with ovarian cancer. The diagnostic will be used to determine if patients with ovarian cancer whose dominant tumor biology is driven by angiogenesis are more likely to benefit from treatment with navicixizumab.

The Xerna TME panel uses proprietary RNA-based gene expression data and a machine learning-based algorithm to classify patients based on the dominant biology of their cancer so that patients can be matched with therapies that directly address these biologies. Pursuant to the non-exclusive license, which is for research use only ("RUO") and clinical development programs, OncXerna has granted QIAGEN the rights to integrate the Xerna TME panel into their NGS workflow solutions, which broadens QIAGEN’s NGS CDx custom panel development capabilities.

"We are very pleased to complete these agreements with QIAGEN, which we see as providing important external validation for the Xerna TME panel and our broader RNA-based biomarker platform," stated Dr. Laura Benjamin, OncXerna Founder and CEO. "As an NGS industry-leader in precision medicine diagnostics with impressive global development, manufacturing and commercial capabilities, we believe QIAGEN is uniquely positioned to help advance the Xerna TME panel towards regulatory approval as an NGS companion diagnostic and, if approved, drive the panel’s adoption. Moreover, integration of the Xerna TME Panel into their workflow solutions could enable QIAGEN to provide a new RNA-based offering to strengthen their overall immune oncology solutions for biopharma customers."

"We believe that the Xerna TME panel that we can now offer our customers will further enhance our strong portfolio in companion diagnostics. Through this agreement, we aim to foster additional NGS-based collaborations with pharmaceutical companies for the development of drug treatments for immune oncology and promote early clinical adoption of precision medicine diagnostics such as our therascreen portfolio and the Xerna TME panel." said Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics at QIAGEN. "We are also thrilled to have the master CDx agreement in place and look forward to working with OncXerna to develop an NGS companion diagnostic for Navicixizumab based on our extensive track record with the development of companion diagnostics for a variety of cancers."

QIAGEN is a pioneer in Precision Medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from next-generation sequencing (NGS) to polymerase chain reaction (PCR) for companion diagnostic development. QIAGEN has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.

Currently, QIAGEN is working under master collaboration agreements with more than 25 companies to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance Precision Medicine for the benefit of patients.

About OncXerna Therapeutics, its Xerna RNA-based Biomarker Platform, and Xerna TME Panel

OncXerna is a precision medicine company using an innovative RNA-expression based biomarker platform to predict patient responses to its targeted oncology therapeutic candidates. OncXerna is working to expand next-generation precision medicine to a larger group of cancer patients by leveraging the company’s Xerna platform to prospectively identify patients based on the dominant biology of their cancer. OncXerna’s approach pairs those patients with OncXerna’s clinical-stage therapeutic candidates and known mechanism of action that directly address these biologies, with the goal to substantially improve patient outcomes. The Xerna TME Panel uses proprietary RNA-based gene expression data and a machine learning-based algorithm to classify patients based on the interplay between angiogenic and immunogenic dominant biologies of the tumor microenvironment (TME), and has been developed as a clinical assay. The Xerna TME Panel is an investigational assay that has not been approved, and has not been demonstrated to be safe or effective for any use.

About Navicixizumab

Navicixizumab is an anti-DLL4/VEGF bispecific antibody product candidate that demonstrated antitumor activity in patients who were previously treated with Avastin (bevacizumab) in a Phase 1b clinical trial. The U.S. Food and Drug Administration granted Fast Track designation to navicixizumab for the treatment of high-grade ovarian, primary peritoneal, or fallopian tube cancer in patients who have received at least three prior therapies and/or prior treatment with Avastin. Navicixizumab is an investigational agent that has not been approved, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced ovarian cancer.

On August 9, 2021 INOVIO (NASDAQ:INO), a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, cancer, and HPV-associated diseases, teported financial results for the quarter ended June 30, 2021 (Press release, Inovio, AUG 9, 2021, View Source [SID1234586112]). INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Time today to discuss financial results and provide a general business update. The business update includes INOVIO’s COVID-19 vaccine development efforts that address both current and future variants of concern ("VOC"), accompanied with recent developments associated with INOVIO’s various therapeutic programs relating to its DNA medicines platform. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

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!

Dr. J. Joseph Kim, President and CEO of INOVIO, said, "With COVID-19 rates surging again globally and an increasing number of breakthrough infections, INOVIO recognizes the need for additional safe and effective first-line vaccines, particularly those which could offer potential boosting capabilities, to combat the spread of the virus and emerging variants, including the rapidly spreading delta variant. INO-4800’s ability to generate CD8 T cells are important to mitigating against variants of concern, including the delta variant. Findings from a study using clinical samples showed that INO-4800 maintained a robust T cell level against the delta variant when compared to T-cell responses from the original wildtype strain. These findings further complement our previously published Phase 1 and 2 trial data for INO-4800. As a reminder, the key advantages of INOVIO’s DNA medicines platform include the ability to generate a balanced immune response that includes engagement of both T cells and B cells, coupled with a favorable transport, thermostability, and tolerability profile. These advantages continue to be integral to our ongoing discussions with countries expected to participate in the global INNOVATE Phase 3 trial for INO-4800, some of which are also considering INO-4800 for both clinical trials and the eventual emergency use authorization (EUA)."

Dr. Kim continued, "In parallel with our global INNOVATE Phase 3 trial, we continue to prepare INOVIO’s next-generation, pan-COVID vaccine candidate, INO-4802, in a Phase 1/2 trial entitled IMPACT (INOVIO INO-4802 Multi-variant Pan-COVID-19 Vaccine Trial), where the goal is to induce cross-reactive immune responses against current and emerging viral variants as either a first-line vaccine, or as a boost."

INOVIO Key Updates & Second Quarter 2021 Highlights

Key Updates

INOVIO expanded its partnership with Advaccine Biopharmaceuticals Suzhou Co., Ltd. ("Advaccine") to jointly conduct the global Phase 3 segment of the ongoing Phase 2/3 trial called INNOVATE (INOVIO INO-4800 Vaccine Trial for Efficacy) in multiple countries.

Subsequent to the quarter end, INOVIO and Advaccine received regulatory allowance to conduct two clinical trials in China investigating heterologous boosting with INO-4800 through partner and trial-sponsor Advaccine, together with Sinovac Biotechnology ("Sinovac"). The studies will evaluate the safety and efficacy of heterologous prime-boost sequential immunizations using INO-4800 and CoronaVac, an inactivated virus COVID-19 vaccine developed by Sinovac and validated by the World Health Organization (WHO) for emergency use.

INO-4800 vaccination maintained a similar level of T cell responses against the delta variant when compared to the T cell responses to the original wildtype strain and showed a similar level of reduced neutralizing antibody activity against the delta variant by the mRNA vaccines. These findings build on previously published results showing that INO-4800 provided broad, cross-reactive immune responses in humans against alpha, beta and gamma VOC.

INOVIO published pre-clinical data as a pre-print for INO-4802 demonstrating cross-reactive immune responses against current and emerging viral variants that shows the potential INOVIO’s next-generation, pan-COVID-19 vaccine candidate, as either a first-line vaccine or potentially as a booster for individuals previously immunized with various wildtype-matched vaccines. INO-4802 induced potent neutralizing antibodies, T cell responses, and protection in a pre-clinical model against the original wildtype strain as well as against the alpha, beta, gamma and, in subsequent research, the delta variant.

Subsequent to the quarter end, INOVIO dosed the first subject in its Phase 2 clinical trial for INO-4700, its DNA vaccine candidate for Middle East Respiratory Syndrome ("MERS"), a disease in the coronavirus family for which there are no approved vaccines. The study, which is sponsored by INOVIO and funded by the Coalition for Epidemic Preparedness Innovations ("CEPI"), is evaluating the safety, tolerability and immunogenicity of INO-4700 in approximately 500 healthy adult volunteers in Jordan and Lebanon.

In 2Q 2021, the University of Pennsylvania enrolled its first patient in a Phase 1b investigator-sponsored trial of INOVIO’s DNA vaccine candidate INO-5401 alone or INO-5401 in combination with INO-9012 delivered with INOVIO’s CELLECTRA smart device, in adult cancer and non-cancer patients with BRCA1 or BRCA2 mutations.

INOVIO Second Quarter 2021 Program Updates

DNA Vaccine Candidates

INO-4800: INNOVATE Phase 3 Trial

INOVIO expanded its partnership with Advaccine to jointly conduct a global Phase 3 trial for INO-4800. Under the terms of the collaboration, INOVIO and Advaccine intend to share equally, subject to specified limitations and conditions, the total cost of the planned global Phase 3 trial, which is estimated to be approximately $100 million. This is an extension of an existing relationship between the two companies, including an exclusive agreement announced in January 2021 under which Advaccine has the exclusive rights to develop, manufacture and commercialize INO-4800 within Greater China, inclusive of mainland China, Hong Kong, Macao and Taiwan. Under the expanded partnership, Advaccine obtained rights to additional Asian countries outside of Greater China. The companies intend to evaluate the safety and efficacy of INO-4800 in a two-dose regimen (2.0 mg), administered one month apart, in a two-to-one randomization in healthy men and non-pregnant women 18 years and older across several countries, with a focus on Latin America, Asia and Africa. The 2.0 mg dose was selected from the Phase 2 segment, where INO-4800 was shown to be generally well-tolerated and immunogenic across all adult age groups. In particular, the geometric mean fold rise of binding and neutralizing antibody levels was statistically significant and greater in the 2.0 mg dose group versus the 1.0 mg dose group. Notably, the T cell immune responses measured by the ELISpot assay were also higher in the 2.0 mg dose group as compared to the 1.0 mg dose group. The primary endpoint of the Phase 3 segment will be virologically confirmed COVID-19 cases, and INOVIO anticipates the first regulatory approval next month.

During the second quarter of 2021, INOVIO released as a pre-print results from a study using clinical samples showing that INO-4800 provided broad cross-reactive immune responses in humans against VOC. The study showed the T cell responses induced by INO-4800 vaccination were fully maintained against the alpha, beta, and gamma variants when compared to the T cell responses to the original wildtype strain. Despite recent reports showing a reduction in neutralizing activity against the gamma variant by the mRNA or viral vector vaccines, INO-4800 generated robust neutralizing antibodies at levels against the gamma variant that were comparable to those observed against the wildtype strain. Taken together with the data showing the maintenance of T cell activity, the results reported in this study provide a comprehensive overview of cross-reactive cellular and humoral immune responses against SARS-CoV-2 variants for INO-4800 vaccinated individuals, showing the potential of INO-4800 to combat emerging as well as future variants of concern. The study, entitled, "INO-4800 DNA Vaccine Induces T Cell Activity and Neutralizing Antibodies Against Global SARS-CoV-2 Variants," is available via pre-print in bioRxiv.

Subsequent to this published work, INO-4800 vaccination was also found to maintain a similar level of T cell responses against the delta variant when compared to the T cell responses to the original wildtype strain, while it showed a similar level of reduced neutralizing antibody activity against the delta variant by the mRNA vaccines.

INOVIO continues to evaluate and assess the impact the new circulating strains of SARS-CoV-2 have on the immune profile elicited by INO-4800, as well as assessing boosting capabilities of INO-4800.

INO-4800: Heterologous Prime-Boost Trials

This morning, INOVIO announced the regulatory allowance in China to conduct two clinical trials investigating heterologous boosting with INO-4800 through its partner and trial-sponsor Advaccine, together with Sinovac. The trials will evaluate the safety and efficacy of heterologous prime-boost sequential immunizations using INO-4800 and CoronaVac, an inactivated virus COVID-19 vaccine developed by Sinovac and validated by the WHO for emergency use. China’s Center for Drug Evaluation of the National Medical Products Administration has allowed two Advaccine-sponsored open-label, positive-control trials to evaluate the safety, tolerability and immunogenicity of mixed boosted regimens. Both studies, which will be conducted in China, are anticipated to begin this fall and will involve healthy adult subjects 18 years of age or older.

INOVIO, Advaccine, and Sinovac have completed cross prime-boost pre-clinical animal tests using INO-4800 and CoronaVac, demonstrating that the prime-boost strategy can stimulate high-level of antigen specific binding antibodies, neutralizing antibodies by both live-virus neutralization assay and hACE2 receptor blocking assay, and antigen-specific T cell immune response.

IMPACT (INOVIO INO-4802 Multi-variant Pan-COVID-19 Vaccine Trial):

In parallel with INO-4800, INOVIO is also developing a second generation, pan-COVID vaccine candidate, INO-4802, which is designed to protect against current and future VOC. INO-4802 could potentially offer boosting capabilities in addition to an initial vaccination regimen with INO-4800 and/or other first-generation vaccines, including both adenovirus and mRNA-based platforms.

In 2Q and then updated subsequent to the quarter, INOVIO released a manuscript as a preprint in bioRxiv entitled, "Design and Immunogenicity of a Pan-SARS-CoV-2 Synthetic DNA Vaccine," which demonstrated cross-reactive immune responses against current and emerging viral variants using INOVIO’s next-generation pan-COVID-19 vaccine candidate, INO-4802, as either a first-line vaccine, or potentially as a booster for individuals previously immunized with various wildtype-matched vaccines. Specifically, INO-4802 generated potent neutralizing antibodies, T cell responses, and protection in a preclinical model against the original wildtype strain as well as against the alpha, beta, gamma and delta variants.

Infectious Diseases: Middle East Respiratory Syndrome ("MERS") and Lassa Fever

INOVIO dosed the first subject in its Phase 2 clinical trial for INO-4700, its DNA vaccine candidate for MERS. MERS, which currently has no approved vaccine, is a coronavirus that is about 100 times deadlier than COVID-19 and fatal to approximately 34% of those infected.

The Phase 2 trial is being conducted at sites in Jordan and Lebanon, where MERS cases have been reported. The randomized, double-blinded, placebo-controlled, multi-center trial, which is sponsored by INOVIO and funded by CEPI, evaluates the safety, tolerability and immunogenicity of INO-4700 administered using INOVIO’s CELLECTRA smart device in approximately 500 healthy adult volunteers.

INOVIO’s pursuit of a MERS vaccine is funded by a previously announced $56 million grant from CEPI, under which INOVIO will develop vaccine candidates through Phase 2 against MERS and Lassa fever. INOVIO and CEPI plan to pursue a stockpile of MERS vaccines available for emergency use as soon as possible following Phase 2 testing.

Subsequent to the quarter end, CEPI announced in July 2021 that it is providing $10.3 million in funding to partners in Benin, Guinea, Liberia, and Sierra Leone to participate in the epidemiological research program entitled Enable, which will enroll up to 23,000 participants, including in Nigeria, which began collecting participant data in December 2020. The Enablestudy aims to better understand the rate, location, and spread of Lassa virus across the region. CEPI has supported the development of six Lassa vaccine candidates, including INOVIO’s INO-4500. INO-4500 is the first Lassa vaccine candidate to enter Phase 1 trial in the U.S., and in the first quarter INOVIO dosed the first subject in a Phase 1b clinical trial for INO-4500 in Africa. It remains INOVIO’s and CEPI’s goal to making INO-4500 available for possible emergency use as a stockpile product after successful completion of the Phase 2 trial.

HPV-related Diseases

VGX-3100: Cervical, Vulvar, and Anal HSIL

REVEAL 1 / REVEAL 2 (Cervical HSIL)

INOVIO continues to follow subjects in REVEAL 1 (Randomized Evaluation of VGX-3100 and Electroporation for the treatment of Cervical HSIL), a Phase 3 pivotal trial evaluating VGX-3100 for the treatment of cervical high-grade squamous intraepithelial lesions caused by HPV-16 and/or HPV-18, for safety and durability of response for 18 months following the last administration. INOVIO expects to present its findings at a scientific meeting later this year and anticipates full subject-level unblinding for REVEAL 1 in the second half of 2021, which is expected to facilitate better analysis of individual, patient-level data.

Additionally, INOVIO is continuing its partnership with QIAGEN to co-develop an in-vitro diagnostic based on RNA sequencing technology to guide clinical decision-making for the use of VGX-3100 in cervical HSIL. INOVIO expects to report QIAGEN’s findings later this year.

REVEAL 2 continues to enroll across 48 sites globally, with projected total enrollment of approximately 198 adult women with histologically confirmed cervical HSIL. Participants will be evaluated for evidence of cervical HSIL on histology as well as evidence of HPV-16 and/or HPV-18 in cervical samples by type-specific HPV testing at the Week 36 visit accompanied with a one-month safety follow-up.

Immuno-oncology

INO-5401

Glioblastoma Multiforme

INOVIO, along with Regeneron, continues to evaluate its findings from the Phase 1/2 novel combination trial of DNA medicines INO-5401 and INO-9012 in combination with PD-1 inhibitor Libtayo (cemiplimab) – which is being jointly developed by Regeneron and Sanofi – for the treatment of newly diagnosed Glioblastoma Multiforme ("GBM"). The companies anticipate sharing two-year (24 months) overall survival data, including correlative immunology and tissue data, at an oncology conference in the fourth quarter of 2021.

Breast Cancer

Separately, the University of Pennsylvania enrolled its first patient in a Phase 1b investigator-sponsored study of INO-5401 alone or INO-5401 in combination with INO-9012 delivered with INOVIO’s CELLECTRA smart device in adult cancer and non-cancer patients with BRCA1 or BRCA2 mutations. This study, which is being conducted at the University of Pennsylvania, will enroll approximately 44 subjects and will test INO-5401, which contains genes that are active in human cancers (hTERT, PSMA, and WT1) and are believed to be good targets for the immune system for both individuals with cancer or at increased risk of getting cancer. ClinicalTrials.gov identifier: NCT04367675

Manufacturing

Update on Global Manufacturing Consortium

Thermo Fisher, a member of INOVIO’s global manufacturing consortium, announced in July that it opened a new cGMP plasmid DNA manufacturing facility in Carlsbad, California with INOVIO as its first client. The new facility enables Thermo Fisher and its partners to meet anticipated demand for plasmid DNA and other nucleic-acid based therapies.

Second Quarter 2021 Financial Results

Total revenue was $273,000 for the three months ended June 30, 2021, compared to $267,000 for the same period in 2020. Total operating expenses were $83.5 million compared to $33.4 million for the same period in 2020.

INOVIO’s net loss for the three months ended June 30, 2021 was $82.1 million, or $0.39 per basic and diluted share, compared to net loss of $128.7 million, or $0.83 per basic and diluted share, for the three months ended June 30, 2020.

Operating Expenses

Research and development ("R&D") expenses for the three months ended June 30, 2021, were $70.8 million compared to $22.4 million for the same period in 2020. The increase in R&D expenses was primarily attributable to manufacturing scale-up activities for INO-4800. These INO-4800 activities included the acquisition and installation of manufacturing equipment, drug manufacturing, outside services and clinical study expenses. Other increases included engineering services and expensed equipment related to our CELLECTRA 3PSP device array automation project, employee and contractor compensation and drug manufacturing expenses related to our RRP trial. These increases were offset by an increase in contra-research and development expense recorded from grant agreements of $8.1 million, among other variances.

General and administrative ("G&A") expenses were $12.7 million for the three months ended June 30, 2021, compared to $11.1 million for the same period in 2020. The increase in G&A expenses was primarily related to an increase in employee compensation, including non-cash stock-based compensation, partially offset by lower expenses for work performed related to corporate marketing and communications, among other variances.

Capital Resources

As of June 30, 2021, cash and cash equivalents and short-term investments were $443.7 million compared to $411.6 million as of December 31, 2020. As of June 30, 2021, INOVIO had 210.1 million common shares outstanding and 226.7 million common shares outstanding on a fully diluted basis, after giving effect to the exercise, vesting and conversion, as applicable, of its outstanding options, restricted stock units, convertible preferred stock and convertible debt.

INOVIO’s balance sheet and statement of operations are provided below. Additional information is included in INOVIO’s quarterly report on Form 10-Q for the three months ended June 30, 2021, which can be accessed at: View Source

Conference Call / Webcast Information

INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Time today to discuss INOVIO’s financial results and provide a general business update.

The live webcast and a replay may be accessed by visiting INOVIO’s website at View Source

About INOVIO’s DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with COVID-19 and MERS. DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO’s DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO’s proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, which is designed to overcome a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. Administration with the CELLECTRA device is designed to ensure that the DNA medicine is efficiently delivered directly into the body’s cells, where it can go to work to drive an immune response. INOVIO’s DNA medicines do not interfere with or change in any way an individual’s own DNA. The advantages of INOVIO’s DNA medicine platform are how fast DNA medicines can be designed and manufactured; the stability of the products, which do not require freezing in storage and transport; and the robust immune response, safety profile, and tolerability that have been observed in clinical trials.

With more than 3,000 patients receiving INOVIO investigational DNA medicines in more than 7,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.