On May 10, 2021 Forte Biosciences, Inc. (www.fortebiorx.com) (NASDAQ: FBRX), a clinical-stage biopharmaceutical company reported first quarter 2021 results and provides a general business update on May 10, 2021 (Press release, Tocagen, MAY 10, 2021, View Source [SID1234579578]).

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"Forte has continued to make excellent progress in the fist quarter of 2021. As we have previously highlighted, our randomized clinical trial of FB-401 in atopic dermatitis patients 2 years of age and older, including adolescents and adults, completed enrollment. We expect to report the topline results from that trial in the third quarter of 2021. There is a significant unmet need for safe effective therapies, particularly for children suffering from atopic dermatitis and we are hopeful that FB-401 will address that need," said Paul Wagner, Ph.D., CEO of Forte Biosciences. "In addition to the operational progress, two more patents have now issued, bringing the total number of patents covering FB-401 and our process to eleven as we continue to expand the intellectual property protection."

First Quarter 2021 Results

First Quarter 2021 Business Highlights

In September 2020, Forte initiated a multi-center, placebo-controlled clinical trial of its lead product candidate, FB-401 which was expected to enroll approximately 124 pediatric, adolescent and adult atopic dermatitis subjects aged 2 years of age and older. Enrollment was completed in March 2021 with 154 subjects. Additional information about our Phase 2 trial can be found at ClinicalTrials.gov using the identifier NCT04504279.

Forte ended the first quarter of 2021 with approximately $54.8 million in cash and cash equivalents which Forte believes is sufficient to fund operations for at least the next 12 months. Cash utilization for the first quarter of 2021 was $4.0 million. Forte had approximately 13.5 million shares of common stock outstanding as of March 31, 2021.

First Quarter 2021 Operating Results

Research and development expenses were $3.3 million and $1.4 million for the three months ended March 31, 2021 and 2020, respectively. The increases in 2021 were primarily due to manufacturing, clinical, regulatory and other expenses, including non-cash stock-based compensation, as Forte continues to advance FB-401 through Phase 2 clinical trials.

General and administrative expenses were $1.4 million and $0.7 million for the three months ended March 31, 2021 and 2020, respectively. The increases in 2021 were primarily due to legal, professional, insurance and other expenses as a result of being a public company as well as increases in payroll and related expenses including non-cash stock-based compensation expense as we increased our headcount.

Losses per share were $0.36 and $0.97 for the three months ended March 31, 2021 and 2020, respectively.

Additional detail on our financial results for the first quarter of 2021 can be found in Forte’s Form 10-Q as filed with the SEC on May 10, 2021. You can also find more information in the investor relations section of our website at www.fortebiorx.com.

Conference Call and Webcast Information

Forte management will host a conference call and webcast on Wednesday, May 10th at 4.30 PM Eastern Time. Participants may access the call by dialing 877-705-6003 (Domestic) or 201-493-6725 (International). The conference ID number is: 13719507.

On May 10, 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 first quarter ended March 31, 2021, which include (Press release, Omeros, MAY 10, 2021, View Source [SID1234579577]):

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OMIDRIA revenues for the first quarter of 2021 were $21.1 million compared to $10.6 million in the fourth quarter of 2020. The increase over the prior quarter reflects limited fourth-quarter sales due to delayed confirmation (issued in December) by the Centers for Medicare and Medicaid Services (CMS) that OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% receives separate payment when used in the ambulatory surgery center (ASC) setting.
Net loss in the first quarter of 2021 was $35.1 million, or $0.57 per share, including non-cash expenses of $4.1 million, or $0.07 per share. This compares to a net loss of $37.3 million, or $0.60 per share, which included non-cash expenses of $3.5 million, or $0.07 per share, for the previous quarter.
At March 31, 2021, Omeros had cash, cash equivalents and short-term investments available for operations of $100.5 million.
Dosing of patients with narsoplimab in the I-SPY COVID-19 platform trial began in March 2021. The platform trial, sponsored by Quantum Leap Healthcare Collaborative and partly funded by BARDA, is enrolling patients nationwide to evaluate potential therapies for the treatment of critically ill COVID-19 patients.
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 July 17, 2021 under the Prescription Drug User Fee Act (PDUFA).
In late April, the Centers for Disease Control and Prevention (CDC) and CMS approved a new ICD-10 diagnosis code for TA-TMA, creating a disease-specific code by which facilities will bill for services, and two new ICD-10 procedural codes that allow physicians to bill for the administration of narsoplimab.
"2021 is off to a strong start as we make great progress toward the anticipated launch of narsoplimab for TA-TMA while building momentum with our ophthalmic drug OMIDRIA following CMS’ confirmation of separate payment for OMIDRIA in the ASC setting," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "Narsoplimab dosing is well underway in the I-SPY COVID-19 platform trial, and the need for a therapeutic to treat critically ill COVID-19 patients is receiving increased focus from both U.S. and international agencies. Looking further across our franchise of complement inhibitors, two other narsoplimab Phase 3 programs are running in IgA nephropathy and aHUS, we expect initial data readout next month from the Phase 1 trial of our MASP-3 inhibitor OMS906, and our subcutaneously delivered long-acting MASP-2 inhibitor OMS1029 is slated to enter the clinic in the first half of next year. Our preclinical programs are also progressing, led by our efforts to deliver a GPR174 inhibitor to the clinic as quickly as possible. With the PDUFA date for narsoplimab in TA-TMA rapidly approaching, we remain committed to bringing a long line of important, first-in-class drugs to market."

First Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:
Omeros continued to add new ASC customers in the first quarter of 2021, including seven large ASC chains and private equity groups. The total number of purchasing ASCs increased by 43% in the first quarter over the previous quarter.
A manuscript on pain control and reduction of opioid use intraoperatively with the use of OMIDRIA during cataract surgery has been submitted for publication. Another manuscript on the perioperative use of opioids in cataract surgery pain management and the role of non-opioid alternatives like OMIDRIA has also been submitted for publication.
The Non-Opioids Prevent Addiction in the Nation (NOPAIN) Act has been re-introduced in the Senate. The NOPAIN Act would extend separate payment for non-opioid alternatives like OMIDRIA in both ASCs and hospital outpatient departments on a renewable five-year basis. Currently, OMIDRIA is separately paid in the ASC setting.
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 critically ill COVID-19 patients, include the following:
Data from the pivotal trial of narsoplimab in TA-TMA was featured in a podium presentation at the annual European Society for Blood and Marrow Transplantation (EBMT) meeting in March.
An abstract on narsoplimab treatment in adults with high-risk TA-TMA has also been accepted for oral presentation at the 2021 Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June.
Discussions with the U.S. and foreign governments regarding funding and manufacturing support for narsoplimab are ongoing.
Updates regarding Omeros’ other development programs and platforms include the following:
Omeros has completed all of the intravenous cohorts and the first subcutaneous dosing cohort in the single-ascending-dose study in its Phase 1 clinical trial evaluating OMS906, the company’s inhibitor of MASP-3, the key activator of the alternative pathway of complement. Initial data from the placebo-controlled, double-blind, single-ascending-dose and multiple-ascending-dose trial are expected later this quarter.
A paper detailing the mechanism of action of PDE7 inhibition in nicotine addiction will soon be published in the peer-reviewed Journal of Neuroscience. Omeros has completed a successful Phase 1 trial with OMS527, its PDE7 inhibitor.
Financial Results

For the first quarter of 2021, OMIDRIA revenues were $21.1 million compared to $10.6 million for the fourth quarter of 2020. The uncertainty around OMIDRIA’s reimbursement status affected revenues in the fourth quarter and extending into early February 2021.

Total costs and expenses for the first quarter of 2021 were $51.7 million compared to $47.2 million for the first quarter of 2020. The increase was primarily due to research and development expenses related to narsoplimab manufacturing. Until approval for narsoplimab in TA-TMA is certain, manufacturing costs for narsoplimab are expensed as incurred instead of included as inventory.

For the three months ended March 31, 2021, Omeros reported 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. This compares to a net loss in the previous quarter of $37.3 million, or $0.60 per share, which included non-cash expenses of $3.5 million, or $0.07 per share.

As of March 31, 2021, the company had $100.5 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 85 percent of eligible accounts receivable less certain reserves and $50.0 million.

On March 1, 2021, the company entered into an "at the market" sales agreement which 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 6999269. 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 6999269.

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 May 10, 2021 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage precision oncology company focused on developing targeted drugs to inhibit frontier targets that drive and sustain RAS-addicted cancers, reported its financial results for the first quarter of 2021 and provided a corporate update (Press release, Revolution Medicines, MAY 10, 2021, View Source [SID1234579576]).

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"Revolution Medicines has made excellent progress reinforcing our belief that the company’s cohesive portfolio of innovative clinical and preclinical assets will power compelling rational, mechanism-based combination treatments that provide benefit to patients with RAS-addicted cancers," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines.

"We presented data at the AACR (Free AACR Whitepaper) Annual Meeting 2021 demonstrating the attractive preclinical profiles of two pioneering RAS(ON) inhibitor candidates that are currently undergoing IND-enabling development, RMC-6291 (KRASG12C) and RMC-6236 (RASMULTI). These first examples of RAS(ON) inhibitors intended for human use exhibit differentiated breadth, depth and durability of anti-tumor effects in human cancer models. Further, an important recent scientific paper described multiple genetic mutations causing clinical resistance to leading KRASG12C(OFF) inhibitors but with preserved sensitivity to RAS(ON) inhibitors from our collection. We believe that RMC-6291, RMC-6236 and additional emerging inhibitors in our portfolio hold great promise for use in treating, and overcoming resistance in, patients with a diverse range of RAS-addicted cancers lacking adequate targeted therapeutics.

"The company also continues broad-based initiatives with our RAS Companion Inhibitor portfolio. For RMC-4630 (SHP2), combination approaches with multiple direct RAS inhibitors remain a high-priority treatment strategy supported by the clinical and preclinical anti-tumor activity, resistance and safety data observed to date across these classes of targeted agents. Amgen’s CodeBreaK 101c study evaluating the combination with sotorasib has demonstrated acceptable tolerability, has cleared early dose levels and is currently dosing patients at the target dose of RMC-4630 (200 mg on a Day 1/Day 2 weekly schedule). We also continue evaluating a second, distinct group of treatment strategies for RMC-4630 in combination with established drugs that potently suppress the RAS signaling pathway, including cobimetinib, a MEK inhibitor and osimertinib, an EGFR inhibitor, to determine whether enhanced pathway inhibition from these drug combinations delivers sufficient anti-tumor activity and tolerability to confer clinical benefit.

"We are also pleased to have begun clinical evaluation of RMC-5552 (mTORC1/4EBP1) in a monotherapy dose-escalation study. In aggregate, these projects with our RAS Companion Inhibitor portfolio, including continued IND-enabling development of RMC-5845 (SOS1), support our long-term goal of combining these assets with RAS(ON) Inhibitors on behalf of patients selected by molecular tumor features.

"To support the expanded and advancing pipeline, Revolution Medicines successfully completed a financing in the first quarter that helped position the company with a strong balance sheet."

R&D Highlights

RAS(ON) Inhibitors – Revolution Medicines continues maturing its first-in-class RAS(ON) Inhibitor platform, including an expansive collection of tri-complex inhibitors targeting diverse oncogenic RAS variants through highly differentiated chemical and pharmacologic profiles.

Potential advantages of RAS(ON) Inhibitors – A recent paper in Cancer Discovery by Dr. Ryan Corcoran’s team at the Massachusetts General Hospital/Harvard Medical School identified multiple resistance mutations that bypass the effects of three first-generation KRASG12C(OFF) inhibitors. Importantly, the researchers found that a KRASG12C-selective RAS(ON) tool compound from the Revolution Medicines portfolio, RM-018, retained potent binding and inhibitory activity against tumor cells harboring an on-target mutation that conferred resistance to all three KRASG12C(OFF) inhibitors tested.

RMC-6291 (KRASG12C)

RMC-6291 is a first-in-class, potent, oral and selective tri-complex inhibitor of KRASG12C(ON) and NRASG12C(ON) with an attractive and differentiated preclinical profile designed to address persistent unmet needs for patients with cancers caused by KRASG12C or NRASG12C.
Data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 showed superior anti-tumor activity for RMC-6291 in preclinical lung and colorectal cancer models driven by a KRASG12C mutation.
The company remains on track to submit an investigational new drug (IND) application in the first half of 2022.

RMC-6236 (RASMULTI)

RMC-6236 is a first-in-class, potent, oral RAS-selective tri-complex, RASMULTI(ON) inhibitor with an attractive preclinical profile and is designed to treat cancers caused by multiple RAS variants for which no targeted treatment is currently available.
Data presented at the recent AACR (Free AACR Whitepaper) meeting demonstrated deep anti-tumor activity of RMC-6236 in preclinical lung, colorectal and pancreatic cancer models driven by various mutations that are common drivers of human cancers, including KRASG12V and KRASG12D.
The company remains on track to submit an IND in the first half of 2022.

Continued expansion of other RAS(ON) inhibitor programs – Revolution Medicines continues to progress an expanding portfolio of potent, cell-active RAS(ON) Inhibitors with the potential to target RAS variants driving the vast majority of RAS-addicted cancers. In particular, the company’s KRASG12D- and KRASG13C-selective programs continue to advance in lead optimization. The company remains on track to nominate a third development candidate from its RAS(ON) inhibitor portfolio in the second half of 2021.
RAS Companion Inhibitors – Revolution Medicines continues to advance and expand multiple clinical studies both as monotherapy and in targeted drug combinations designed to achieve maximum clinical benefit.

RMC-4630 (SHP2 Inhibitor) – RMC-4630 is a potent, oral, selective inhibitor of the SHP2 protein, a central node in the RAS signaling pathway. Its development is being advanced in partnership with, and is primarily funded by, Sanofi, both as monotherapy and in several current and planned combinations.
RMC-4630 and KRASG12C inhibitor sotorasib

To date, the available data from the ongoing Amgen-sponsored CodeBreaK 101c study of the RMC-4630 and sotorasib combination has demonstrated acceptable tolerability and cleared early dose levels.
The CodeBreaK 101c study is currently dosing patients at the target dose of RMC-4630 (200 mg on a Day 1 / Day 2 weekly schedule, the full dose used by the company in monotherapy) in combination with sotorasib. The company looks forward to selection of a combination dose for this study in the second half of 2021.
RMC-4630 and AstraZeneca KRASG12C inhibitor

AstraZeneca plans to evaluate RMC-4630 in combination with an emerging asset targeting KRASG12C(OFF) from AstraZeneca’s portfolio
RMC-4630 and MEK inhibitor cobimetinib (Cotellic)

Phase 1b/2 study of this combination is ongoing, including in expansion cohorts of patients with KRASMUTANT colorectal cancer at the recommended Phase 2 dose and schedule (RP2DS) for this combination. The company continues to expect preliminary safety and clinical activity data from this expansion study in 2022.
RMC-4630 and EGFR inhibitor osimertinib (Tagrisso)

Dosing and enrollment continue in the Phase 1b study of this combination and the company continues to expect initial tolerability and pharmacokinetic (PK) data in the second half of 2021.
RMC-4630 and PD-1 inhibitor pembrolizumab (Keytruda)

Sanofi-sponsored Phase 1 study of this combination continues. The RP2DS for this combination is expected in the first half of 2021 and expansion cohorts evaluating this combination in patients with non-small cell lung cancer (NSCLC) are planned.
RMC-4630 monotherapy

Presented dose escalation activity data set from the ongoing Phase 1 study at the recent AACR (Free AACR Whitepaper) meeting, showing anti-tumor activity and safety and tolerability that is consistent with on-pathway inhibition, delivering on a corporate milestone.
Data presented at AACR (Free AACR Whitepaper) meeting showed reduction of variant allele frequency in circulating tumor DNA (ctDNA) samples from patients treated with RMC-4630 for cancers carrying KRASG12C or NF1LOF, further validating the expected clinical mechanism of action of RMC-4630.
RMC-5552 (mTORC1/4EBP1 Inhibitor) – RMC-5552 is a potent, selective bi-steric inhibitor of mTORC1 that suppresses phosphorylation and inactivation of 4EBP1.

Dosing and enrollment are underway in the recently initiated Phase 1 monotherapy dose-escalation study, delivering on a corporate milestone. The company continues to expect initial safety, PK and single agent activity data in 2022.
Preclinical data presented at the recent AACR (Free AACR Whitepaper) meeting demonstrated that bi-steric mTORC1-selective inhibitors drive significant anti-tumor activity as monotherapy and in combination with KRASG12C inhibitors in genetically-defined preclinical models of human cancers.
The company intends to evaluate RMC-5552 in combination with RAS inhibitors for the treatment of tumors driven by co-occurring RAS mutations and genomic activation of the mTORC1 pathway.

RMC-5845 (SOS1 Inhibitor) – RMC-5845 is a potent, selective, oral inhibitor of SOS1, a major switch in the cycling of RAS(OFF) to RAS(ON).

The company remains on track to submit an IND in the second half of 2021 to enable an initial monotherapy dose escalation study and intends to evaluate RMC-5845 for treatment of certain genetically defined RAS-dependent cancers.
Corporate Highlights

Completed upsized financing to strengthen balance sheet and support advancement of expanding pipeline – Public offering of common stock in February 2021 raised net proceeds of $281 million, enabling the company to advance its pipeline, including RAS(ON) Inhibitors RMC-6291 and RMC-6236, through early Phase 1 signal-seeking clinical studies.

Flavia Borellini, Ph.D. joins existing board members Elizabeth McKee Anderson and Neil Exter as Class I director nominee – Dr. Borellini has more than 25 years of executive management experience in the pharmaceutical and biotechnology industry, with a particular focus on global development of targeted oncology drugs, from preclinical to commercial stage.
First Quarter 2021 Financial Highlights

Cash Position: Cash, cash equivalents and marketable securities were $681.6 million as of March 31, 2021, compared to $440.7 million as of December 31, 2020. The increase was primarily due to proceeds from the company’s equity public offering in February 2021.

Revenue: Total revenue, consisting of revenue from the company’s collaboration agreement with Sanofi, was $10.1 million for the quarter ended March 31, 2021, compared to $11.5 million for the quarter ended March 31, 2020. The decrease was due to lower reimbursed research and development services for RMC-4630 resulting from lower manufacturing costs.

R&D Expenses: Research and development expenses were $40.9 million for the quarter ended March 31, 2021, compared to $27.5 million for the quarter ended March 31, 2020. The increase was primarily due to an increase in research expenses associated with the company’s pre-clinical research portfolio, an increase in personnel-related expenses related to additional headcount, and an increase in stock-based compensation.

G&A Expenses: General and administrative expenses were $6.7 million for the quarter ended March 31, 2021, compared to $5.2 million for the quarter ended March 31, 2020. The increase was primarily due to an increase in personnel-related expenses related to additional headcount, and an increase in stock-based compensation.

Net Loss: Net loss was $37.2 million for the quarter ended March 31, 2021, compared to net loss of $19.5 million for the quarter ended March 31, 2020.

2021 Financial Guidance

Revolution Medicines continues to expect full year 2021 GAAP net loss to be between $170 million and $190 million, which includes estimated non-cash stock-based compensation expense of $20 million to $25 million.

On May 10, 2021 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic myeloid malignancies, reported financial results for the first quarter ended March 31, 2021, as well as company highlights and upcoming events (Press release, Geron, MAY 10, 2021, View Source [SID1234579575]). As of March 31, 2021, the Company had $244.7 million in cash and marketable securities, which is expected to fund operations until the end of 2022.

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"We have worked diligently over the past quarter to advance our two Phase 3 clinical trials with registrational intent, and we remain laser focused on improving outcomes for patients and delivering significant value to our shareholders," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.

"Completing 75% of the planned enrollment in our MDS trial coupled with the recent dosing of the first patient in our MF trial indicate the continued progress we are making in our two Phase 3 clinical trials for imetelstat," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "We continue to have confidence in imetelstat’s differentiating clinical benefits seen throughout the course of its development, including strong evidence of disease-modifying activity. We’re excited to have another opportunity to highlight imetelstat’s strong data profile through the two abstracts accepted for presentation at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) meeting. We look forward to achieving top-line results from our IMerge Phase 3 study and the promising path ahead for imetelstat."

Dr. Scarlett concluded, "We are excited about the progress we are making to bring this important drug to patients. We are planning for Geron to become a commercial company in 2023 with the potential launch of imetelstat in lower risk MDS. The markets for both lower risk MDS and refractory MF are highly attractive. We continue to make preparations and manage our cash appropriately to support the future buildout of our manufacturing and commercial infrastructure."

Company Highlights and Upcoming Data Presentations

Ongoing IMerge Phase 3 Clinical Trial in Myelodysplastic Syndromes (MDS)

Screening and enrollment for IMerge Phase 3 in MDS continued to progress in the first quarter. In early December 2020, the Company had completed 50% of the planned patient enrollment in IMerge Phase 3. As of the end of April 2021, enrollment has increased to 75%. The Company continues to expect the trial to be fully enrolled in the second half of 2021. Depending on the timing of full enrollment, the Company expects top-line results from IMerge Phase 3 to be available during the time period from the end of 2022 to the first half of 2023.

For further information about IMerge Phase 3, including enrollment criteria, locations, and current status, please visit ClinicalTrials.gov/NCT02598661.

Ongoing IMpactMF Phase 3 Clinical Trial in Refractory Myelofibrosis (MF)

On April 13, the Company announced that the first patient had been dosed in IMpactMF, the only Phase 3 clinical trial in MF with overall survival (OS) as a primary endpoint evaluating imetelstat, a first-in-class telomerase inhibitor. The Company plans to engage over 180 sites to participate in IMpactMF across North America, South America, Europe, Australia, and Asia. The Company continues to expect the interim analysis to occur in 2024 and the final analysis in 2025.

For further information about IMpactMF, including enrollment criteria, locations, and current status, please visit ClinicalTrials.gov/NCT04576156.

Upcoming Data Presentations

Two abstracts reporting new clinical data and analyses from the Phase 2 trials of imetelstat in lower risk MDS and refractory MF have been accepted for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress meeting to be held virtually from June 9 – 17, 2021. Both of the abstracts will be published on May 12, 2021 at 16:00 CEST on ehaweb.org.

First Quarter 2021 Results

For the first quarter of 2021, the Company reported a net loss of $27.8 million, or $0.09 per share, compared to $16.4 million, or $0.08 per share, for the same period in 2020.

Revenues for the first quarter of 2021 were $137,000 compared to $52,000 for the same period in 2020. Royalty revenues in 2021 and 2020 primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets. In connection with the divestiture of Geron’s human embryonic stem cell assets, including intellectual property and proprietary technology, to Lineage Cell Therapeutics, Inc. (formerly BioTime, Inc., which acquired Asterias Biotherapeutics, Inc.) in 2013, Geron is entitled to receive royalties on sales from certain research or commercial products utilizing Geron’s divested intellectual property.

Total operating expenses for the first quarter of 2021 were $28.6 million compared to $16.9 million for the same period in 2020. Research and development expenses for the first quarter of 2021 were $21.1 million compared to $10.8 million for the same period in 2020. The increase in research and development expenses in the first quarter of 2021 compared to the same period in 2020 primarily reflects increased clinical development costs associated with conducting two Phase 3 clinical trials, higher imetelstat manufacturing costs for producing validation batches at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes and higher personnel-related costs for additional headcount. General and administrative expenses for the first quarter of 2021 were $7.5 million compared to $6.1 million for the same period in 2020. The increase in general and administrative expenses in the first quarter of 2021 compared to the same period in 2020 primarily reflects new costs in connection with pre-commercial activities, including modernizing the internal infrastructure to support a commercial launch, and higher legal costs.

Interest income for the first quarter of 2021 was $173,000 compared to $754,000 for the same period in 2020. The decrease in interest income in the first quarter of 2021 compared to the same period in 2020 primarily reflects lower yields on the Company’s reduced marketable securities portfolio.

Interest expense for the first quarter of 2021 was $743,000 and reflects the Company’s debt facility secured in September 2020 for up to $75 million. Currently, $25.0 million has been drawn down under the facility.

Net other income for the first quarter of 2021 was $1.2 million compared to net other expense of $44,000 for the same period in 2020. During the first quarter of 2021, the Company sold all of its holdings in an equity investment resulting in a net realized gain of $1.2 million, including foreign currency translation adjustments.

2021 Financial Guidance Reaffirmed

For fiscal year 2021, the Company continues to expect its operating expense burn to range from $108 to $112 million, which includes costs for the two ongoing Phase 3 clinical trials; producing validation batches of imetelstat at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes; and preparatory activities for regulatory filings to enable drug approval and commercial readiness.

As of March 31, 2021, the Company had 63 employees. The Company plans to grow to a total of approximately 80 to 85 employees by year-end 2021, of which the majority will be development and manufacturing personnel.

Conference Call

The Company will host a conference call today, May 10, 2021 at 4:30 p.m. ET to review its first quarter financial results and provide an update on the ongoing imetelstat Phase 3 clinical trials, IMerge in MDS and IMpactMF in MF.

A live, listen-only webcast will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the conference call live via telephone by pre-registering online using the following link, View Source Upon registration, a phone number, Direct Event Passcode and unique Registrant ID will be sent via email. This information will be needed in order to enter the conference call. Participants are advised to pre-register at least 10 minutes prior to joining the call.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in hematologic myeloid malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

IMerge Phase 3 is currently enrolling patients. For further information about IMerge Phase 3, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT02598661.

About IMpactMF

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete response, partial response, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

On May 10, 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, reported financial results for the quarter ended March 31, 2021 (Press release, Inovio, MAY 10, 2021, View Source [SID1234579574]). INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Standard Time today to discuss financial results and provide a general business update, covering, among other things: the company’s recently reported Phase 2 segment trial data and plans for a global Phase 3 segment for INO-4800’s INNOVATE Phase 2/3 clinical trial; an overall update on the company’s COVID-19 vaccine developments to address current and future variants of concern (VOC) through its INO-4800 and the pan-COVID INO-4802; and a general update on its DNA medicines platform. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

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Dr. J. Joseph Kim, President and CEO of INOVIO, said, "As the global community continues to contend with the COVID-19 pandemic, and as we prepare for endemic considerations to support the continued fight against variants, INOVIO remains well-positioned to address the global demand for COVID-19 vaccines. We recognize that there is an opportunity to have a meaningful impact in the fight against COVID-19 outside the U.S. and, are planning for a global Phase 3 trial for INO-4800. INOVIO is also encouraged by the positive data from the Phase 2 segment of our Phase 2/3 trial of INO-4800, which is being conducted in the United States. INO-4800 continues to be safe and well-tolerated and has been observed to support the body’s ability to generate both robust neutralizing antibodies and T cell responses – which we believe to be essential in protecting against current and emerging variants of concern. Equally important, INO-4800 has a favorable thermostability profile and does not require cold or ultra-cold chain transport."

Dr. Kim added, "INOVIO continues to be pleased with the progress across our DNA medicines platform, and our efforts to address not only infectious disease but also cancer and HPV-associated diseases, and we look forward to sharing additional updates on GBM this summer."

INOVIO Key Updates & First Quarter 2021 Highlights

Key Updates

This morning, INOVIO announced positive preliminary immunogenicity and safety data from the Phase 2 segment of INNOVATE (INOVIO INO-4800 Vaccine Trial for Efficacy), its clinical trial evaluating COVID-19 DNA vaccine candidate, INO-4800. The Phase 2 data showed the vaccine to be safe, well-tolerated and immunogenic in all tested age groups. The Phase 2 results from approximately 400 patients helped determine INOVIO’s selection of a 2.0 mg dose for the global Phase 3 segment of the trial.

INOVIO met primary and secondary efficacy endpoints among all evaluable subjects for REVEAL 1 (Randomized Evaluation of VGX-3100 and Electroporation for the treatment of Cervical HSIL) trial. REVEAL 2, the confirmatory Phase 3 trial for VGX-3100, continues to enroll globally across 48 study sites.

INOVIO and QIAGEN extended their partnership in late February with a new master collaboration agreement to include the co-development of a pre-treatment RNA-based biomarker blood test designed to identify prospective patients who are most likely to benefit from the clinical use of VGX-3100.

In February, INOVIO dosed its first patient in a Phase 1b clinical trial for INO-4500, its DNA vaccine candidate for Lassa fever, in Ghana. INO-4500 is the first vaccine candidate for Lassa fever to enter human trials. As part of a 2018 partnership, INOVIO and CEPI are committed to making INO-4500 available for possible emergency use as a stockpile product after successful completion of the Phase 2 trial.

INOVIO First Quarter 2021 Program Updates

DNA Vaccine Candidates

INO-4800: INNOVATE Phase 2/3 Clinical Trial

The Phase 2 segment of INNOVATE was designed to evaluate the safety, tolerability and immunogenicity of INO-4800 in a two-dose regimen (1.0 mg or 2.0 mg) in a three-to-one-randomization to receive either INO-4800 or placebo for each dose to identify optimal dose(s) for two age groups (18-50 years and 51 years and older) for the subsequent Phase 3 efficacy evaluation. The preliminary Phase 2 results showed that INO-4800 was safe, well-tolerated and immunogenic in all tested age groups. The trial was funded by the Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense, (JPEO-CBRND) in coordination with the Office of the Assistant Secretary of Defense for Health Affairs (OASD(HA)) and the Defense Health Agency. Results from the trial can be found in the paper entitled "Safety and immunogenicity of INO-4800 DNA vaccine against SARS-CoV-2: A Preliminary Report of a Randomized, Blinded, Placebo-controlled, Phase 2 Clinical Trial in Adults at High Risk of Viral Exposure," has been published as a pre-print in MedRxiv (View Source) prior to peer review.

Findings from the Phase 2 Clinical Trial:

The Phase 2 segment of the trial enrolled approximately 400 participants, 18 years of age or older, at 16 U.S. sites.

Participants received either INO-4800 (1.0 mg or 2.0 mg dose) or placebo at 0 and 4 weeks (randomized 3:3:1:1). Each dose was administered by intradermal injection followed by electroporation using INOVIO’s CELLECTRA, its proprietary smart device.

Safety endpoints included systemic and local administration site reactions through 8 weeks post-dose one (or 4 weeks post-dose 2). Immunology endpoints included antigen-specific binding antibody titers, neutralization titers, and antigen-specific interferon-gamma (IFN-γ) cellular immune responses after two doses of the vaccine.

Vaccine administration was generally safe and well-tolerated. The majority of adverse events (AEs) were Grade 1 and Grade 2 in severity and did not appear to increase in frequency with the second dose. The number of participants experiencing each of the most common AEs did not differ between the two dosing groups.

The geometric mean fold rise of binding and neutralizing antibody levels were statistically significantly greater in the 2.0 mg dose group versus the 1.0 mg dose group.

The T cell immune responses measured by the ELISpot assay were also higher in the 2.0 mg dose group compared to the 1.0 mg dose group.

ClinicalTrials.gov identifier: NCT04642638

Phase 2 results informed INOVIO’s selection of a 2.0 mg dose for the Phase 3 segment of the trial. Given the increasing availability of COVID-19 vaccines authorized for emergency use, in April the Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND), in coordination with the Office of the Assistant Secretary of Defense for Health Affairs (OASD(HA)) and the Defense Health Agency (DHA), informed INOVIO that it will discontinue funding for the planned Phase 3 segment of the INNOVATE trial, while continuing to fund the completion of the ongoing Phase 2 segment. JPEO informed INOVIO that: "The decision results from the changing environment of COVID-19 with the rapid deployment of vaccines. This decision is not a reflection of the awardee or product, rather a fast-moving environment associated with the former Operation Warp Speed on decisions related to future products." The decision by JPEO does not impact other work that INOVIO does with the U.S. government and is neither a result of the current FDA partial clinical hold nor a reflection of the data generated to date for INO-4800.

Recognizing the need to plan for both pandemic and endemic scenarios, as well as the global demand for safe and effective vaccines that are also stable at room temperature and do not require cold-chain or ultra-cold chain transport, INOVIO also announced in April 2021 that it plans to proceed with a global Phase 3 clinical trial for INO-4800 and is working with funders and partners to achieve this plan. The company plans to initiate the global Phase 3 trial this summer.

INO-4800 and INO-4802: Planning for Existing and Future Variants of Concern

INOVIO continues to evaluate the impact of existing and potential new variants of concern for SARS-CoV-2, the virus that causes COVID-19, as well as assessing boosting capabilities for INO-4800. The company is assessing the impact that new circulating strains of the SARS-CoV-2 virus have on the immune profile elicited by INO-4800.

In April, INOVIO published results from a study showing that INO-4800 provides broad cross-reactive immune responses in humans against variants of concern. The study showed the T cell responses induced by INO-4800 vaccination were fully maintained against the UK, South African and Brazilian variants when compared to the T cell responses to the original Wuhan strain. The neutralization levels of INO-4800 against South African and UK variants were reduced to the levels similar to the previous reports of mRNA or viral vector vaccines. Furthermore, despite recent reports showing a reduction in neutralizing activity against the Brazilian variant by the mRNA or viral vector vaccines, INO-4800 generated robust neutralizing antibodies at levels against the Brazilian variant that were comparable to those observed against the Wuhan 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 SARS-CoV-2 variants. The study, entitled "INO-4800 DNA Vaccine Induces T Cell Activity and Neutralizing Antibodies Against Global SARS-CoV-2 Variants," has been submitted for peer review and is available via pre-print in bioRxiv.

In parallel to the late-stage development of INO-4800, the company is also developing a novel, pan-COVID, second-generation vaccine candidate, INO-4802, which is designed to protect against current and potentially future circulating variants. This pan-COVID vaccine could potentially offer boosting capabilities in addition to an initial vaccination regimen with INO-4800 and/or other first-generation vaccines. INOVIO looks forward to sharing additional information on INO-4802 soon.

DNA Immunotherapies: HPV-associated Diseases and Immuno-Oncology

HPV-related Diseases

VGX-3100: Cervical, Vulvar, and Anal HSIL

REVEAL 1 / REVEAL 2 (Cervical HSIL)

In the first quarter, INOVIO announced that it met primary and secondary efficacy endpoints among all evaluable subjects for 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 HSIL caused by HPV-16 and/or HPV-18 using the company’s proprietary CELLECTRA 5PSP device. This trial is one of two ongoing pivotal, randomized, double-blind, multi-center, placebo-controlled, Phase 3 trials (REVEAL 1 and REVEAL 2) designed to assess and confirm the safety, tolerability, immunogenicity, and efficacy of VGX-3100.

INOVIO continues to follow subjects in REVEAL 1 for safety and durability of response for 18 months following the last administration and expects to present its findings at a scientific meeting later this year. The company anticipates subject level full unblinding for REVEAL 1 in the second half of 2021, which will 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. The biomarker blood test could be used to identify prospective VGX-3100 patients who would be most likely to respond to the immunotherapy – an important element of VGX-3100 product and market development. Subject level unblinding for REVEAL 1 will be a key component in enhancing the immune signature of the biomarker, followed by potential confirmatory biomarker data from REVEAL 2.

REVEAL 2 continues to enroll across 48 sites globally. The company continues to assess the impact that the existing pandemic will have on future enrollment in the REVEAL 2 trial. The company believes that it will be in a more suitable position at mid-year to determine if any protocol and/or recruitment adjustments will be necessary.

REVEAL 1 Results

The trial protocol-defined modified intention to treat (mITT) population (N=193) included all subjects with endpoint data. For the primary endpoint of histopathological regression of HSIL combined with virologic clearance of HPV-16 and/or HPV-18 at week 36, the percentage of responders was 23.7% (31/131) in the treatment group, versus 11.3% (7/62) in the placebo group (p=0.022; 12.4% difference in percentage, 95%CI: 0.4,22.5), thus achieving statistical significance. All secondary efficacy endpoints were achieved in the mITT population. These endpoints were: a) regression of cervical HSIL to normal tissue combined with HPV-16 and/or HPV-18 viral clearance, b) regression of cervical HSIL alone, c) regression of cervical HSIL to normal tissue, and d) HPV-16 and/or HPV-18 viral clearance alone. There were no treatment-related serious adverse events and most adverse events were self-resolving and were considered to be mild to moderate, consistent with earlier clinical trials.

Vulvar and Anal HSIL

In January 2021, INOVIO reported positive efficacy results from an open-label Phase 2 trial of VGX-3100 to treat HPV-16 and HPV-18-associated vulvar HSIL. A 25% or more reduction in HPV-16/18-associated vulvar HSIL was observed for 63% of trial participants (12 of 19) treated with VGX-3100 at six months post-treatment. Three out of the 20 participants with histology data (15%) resolved their vulvar HSIL and had no HPV-16/18 virus detectable in the healed area. By comparison, the spontaneous resolution of vulvar HSIL caused by HPV-16/18 is estimated to be 2%. The trial also showed VGX-3100 to be well-tolerated.

The data from the Phase 2 trial of vulvar and anal dysplasia treatments with VGX-3100 were presented at the 2021 ASCCP Virtual Conference. INOVIO continues to evaluate best options for Phase 3 clinical trials for vulvar and anal dysplasia pending further discussions with the FDA.

Immuno-oncology

INO-5401: Newly Diagnosed Glioblastoma Multiforme (GBM)

INOVIO is currently conducting a 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 – in the treatment of newly diagnosed GBM, the deadliest and most aggressive form of brain cancer. The novel combination of INO-5401 + INO-9012 continues to demonstrate a well-tolerated safety profile when given not only with radiation and chemotherapy, but also with PD-1 blockade by Libtayo.

In late 2020, INOVIO shared encouraging interim OS18 data, which also demonstrated immunogenicity and tolerability in a majority of patients. The company anticipates sharing two-year (24 months) overall survival data, including correlative immunology and tissue data, later this year.

First Quarter 2021 Financial Results

Total revenue was $371,000 for the three months ended March 31, 2021, compared to $1.3 million for the same period in 2020. Total operating expenses were $52.9 million compared to $26.6 million for the same period in 2020.

INOVIO’s net loss for the quarter ended March 31, 2021 was $54.4 million, or $0.27 per basic and diluted share, compared to net loss of $32.5 million, or $0.26 per basic and diluted share, for the quarter ended March 31, 2020.

Operating Expenses

Research and development (R&D) expenses for the three months ended March 31, 2021, were $39.0 million compared to $19.1 million for the same period in 2020. The increase in R&D expenses was primarily related to higher drug manufacturing expenses and outside services related to INO-4800 and other clinical trials, higher employee and contractor compensation, including non-cash stock-based compensation, an increase in engineering services related to our CELLECTRA 3PSP device and higher device inventory expense. These increases were offset by an increase in contra-research and development expense recorded from grant agreements of $8.8 million, among other variances.

General and administrative (G&A) expenses were $13.9 million for the three months ended March 31, 2021, versus $7.4 million for the same period in 2020. The increase in G&A expenses was primarily related to an increase in employee and consultant compensation, including non-cash stock-based compensation and legal expenses, among other variances.

Capital Resources

On January 25, 2021, the company closed an underwritten public offering of 20,355,000 shares of common stock at a price of $8.50 per share. The net proceeds to the company, after deducting the underwriters’ discounts and commissions and other offering expenses, were $162.1 million.

As of March 31, 2021, cash and cash equivalents and short-term investments were $518.6 million compared to $411.6 million as of December 31, 2020. As of March 31, 2021, the company had 209.3 million common shares outstanding and 226.5 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 quarter ended March 31, 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, for which programs are being developed with funding support from the U.S. Department of Defense and the Coalition for Epidemic Preparedness Innovations (CEPI). 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, overcoming 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.