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.

On May 10, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the first quarter ended March 31, 2021, which were in line with its previously announced, preliminary first-quarter results (Press release, Veracyte, MAY 10, 2021, View Source [SID1234579573]). The company also provided an update on recent business progress.

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"We delivered strong financial results for the first quarter of 2021, with particular momentum in March, as our business continues to emerge from the COVID-19 pandemic," said Bonnie Anderson, chairman and chief executive officer of Veracyte. "On March 12, we completed our acquisition of Decipher Biosciences which, with its market-leading urologic cancer portfolio, expands our reach into 7 of the top 10 cancers in the U.S. and will accelerate revenue growth.

"Additionally, we are excited to unveil pivotal clinical validation data at ASCO (Free ASCO Whitepaper) for our Percepta Nasal Swab test for early lung cancer detection. We expect to launch it and the Percepta Genomic Atlas as part of our comprehensive lung cancer portfolio later this year. We also anticipate strengthening our global commercial footprint with the introduction of the Envisia classifier, for improved diagnosis of interstitial lung diseases, on the nCounter Analysis System by the end of this year."

First Quarter 2021 Financial Results

For the three months ended March 31, 2021, compared to the prior year (Decipher Biosciences one-time acquisition-related expenses in the first quarter of 2021 increased our operating expenses, net loss and net cash used in operations by $35.1 million and our net loss per share by $0.55):

Total Revenue was $36.7 million, including $3.8 million for urologic cancer testing, an increase of 18%;
Gross Margin was 66%;
Operating Expenses were $82.1 million. Operating Expenses, Excluding Cost of Revenue, were $69.7 million, including $35.1 million related to the acquisition of Decipher Biosciences;
Net Loss and Comprehensive Loss was $41.9 million, including $35.1 million of expenses related to the acquisition of Decipher Biosciences;
Basic and Diluted Net Loss Per Common Share was $0.66, including $0.55 per share attributable to the acquisition related expenses recorded in G&A;
Net Cash Used in Operating Activities was $40.6 million; and
Cash and Cash Equivalents were $324.1 million at March 31, 2021.
First Quarter 2021 and Recent Business Highlights

Commercial Growth:

Total genomic testing and product revenue was $36.1 million, an increase of 19%, compared to the first quarter of 2020.
Total genomic volume was 14,437 tests, an increase of 11%, compared to prior year, including 1,560 tests from Decipher Biosciences.
Continue to expect a mid-year Medicare coverage decision for Decipher Bladder test, which will allow us to leverage our urology sales footprint to accelerate commercial adoption.
Launched General Manager-based global structure, vertically aligning commercial teams within each clinical indication to provide enhanced focus and clinical expertise. Also added key new hires in marketing, managed care and international market access to support multiple new product launches and global expansion later this year.
Evidence Development and Pipeline Advancement:

Six abstracts for our pulmonology products accepted as posters at the American Thoracic Society (ATS) 2021 International Conference this month:
– Percepta Genomic Atlas – New data demonstrating the test’s ability to inform treatment decisions on the same sample used in diagnosis, in anticipation for product launch in Q4 2021.
– Envisia – New data from five abstracts demonstrates the test’s clinical utility and our ability to enable the CLIA lab-based test on the nCounter Analysis System, in anticipation of the test’s international launch later this year.
Six abstracts for our oncology tests accepted for the ASCO (Free ASCO Whitepaper) Annual Meeting in June, including:
– Percepta Nasal Swab – Pivotal, multicenter, double-blind clinical validation data for test to enable early lung cancer detection, setting the stage for anticipated product launch in 2H 2021.
– Decipher Prostate – Two abstracts, including an oral presentation showing test’s ability to identify African American men with higher likelihood of aggressive prostate cancer, will help further distinguish Decipher Prostate from other prostate cancer genomic tests.
– Afirma XA – New data demonstrate the test’s ability to identify patients with gene alterations who may benefit from targeted therapies – at the time of diagnosis.
Two abstracts presented as posters at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Breast Cancer Virtual Congress 2021 meeting this month highlighted potential applications of the PAM50 biomarker on which the Prosigna test is based, as well as initial results from the PROCURE study, which aims to develop consensus around the use of breast cancer genomic tests, including Prosigna.
First patient enrolled and randomized in a study using Veracyte’s LymphMark lymphoma subtyping test to identify and enroll patients with untreated DLBCL who may benefit from Acerta Pharma and AstraZeneca’s Calquence in combination with a traditional chemoimmunotherapy regimen.
Financing:

Issued and sold 8,547,297 shares of common stock in February 2021, including 1,114,864 shares sold upon full exercise of the underwriters’ option to purchase additional shares, at a price to the public of $74.00 per share. The net proceeds to Veracyte from the offering were approximately $593.8 million.
Veracyte used a portion of the net proceeds from the offering, together with its existing cash and cash equivalents, to finance its $600 million cash acquisition of Decipher, which was completed in March 2021. The company intends to use the remaining net proceeds of the offering for working capital and other general corporate purposes, which may include acquiring or investing in complementary businesses, technologies or other assets.
Additionally, in March 2021, the company announced the appointment of Muna Bhanji, a global senior healthcare executive, to its board of directors.

2021 Financial Outlook

Veracyte is reiterating its previous 2021 annual total revenue guidance of approximately $190 million to $200 million, representing growth of approximately 65% over 2020 at the midpoint of the range.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

On May 10, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported that Mardi Dier, the company’s Chief Financial Officer and Camille Bedrosian, M.D., the company’s Chief Medical Officer, will present at the BofA Securities Healthcare Conference on Wednesday, May 12, 2021 at 3:30 PM ET (Press release, Ultragenyx Pharmaceutical, MAY 10, 2021, View Source [SID1234579572]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On May 10, 2021 Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Mersana Therapeutics, MAY 10, 2021, View Source [SID1234579571]).

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

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"The recent initiation of UPLIFT, a single-arm registrational strategy evaluating UpRi in platinum-resistant ovarian cancer, speaks to the team’s commitment to achieving key milestones and dedication to developing therapies for people living with cancer. We believe UpRi has demonstrated promising activity and a differentiated tolerability profile in studies to date in patients with heavily-pretreated ovarian cancer and limited options. UPLIFT has been designed to increase the potential for label differentiation, speed to market, and the probability of success. We also remain on track to initiate the UPGRADE umbrella combination study in the third quarter of 2021 as a first step in bringing UpRi to patients in earlier lines of therapy and establishing UpRi as a foundational medicine in ovarian cancer," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "We are excited to continue this productive year by building out our maturing pipeline of innovative ADC candidates with the potential to address unmet medical needs across multiple different tumor types."

Recent Highlights and Anticipated Milestones

Upifitamab Rilsodotin (UpRi, previously XMT-1536), first-in-class Dolaflexin ADC targeting NaPi2b:

Initiated UPLIFT, a single-arm registration strategy in platinum-resistant ovarian cancer. In April 2021, the Company announced the initiation of patient dosing in UPLIFT, a single-arm registration strategy evaluating the safety and efficacy of UpRi in patients with platinum-resistant ovarian cancer who have received up to four lines of therapy. Consistent with the bevacizumab label, patients previously treated with three or four lines of therapy may enroll without regard to prior bevacizumab treatment. There is no exclusion for patients with baseline peripheral neuropathy. Patients may enroll without regard to NaPi2b expression; however, the role of the biomarker will be evaluated. The primary endpoint will be the objective response rate (ORR) in the high NaPi2b population and the secondary endpoints will be the ORR regardless of NaPi2b expression, as well as duration of response and safety. UPLIFT is an amendment to the ongoing multinational, multi-center, open label study protocol, and the Company expects to enroll approximately 100 patients with high NaPi2b expression and up to 180 patients overall.

In April 2021, the Company announced its plans to use Tumor Proportion Score (TPS) greater than or equal to 75% as the predefined threshold for high NaPi2b expression in UPLIFT. In the ovarian cancer expansion study data presented to date, TPS ≥ 75% resulted in an objective response rate (ORR) of 39% in high NaPi2b patients relative to an ORR of 28% in the overall population. As part of its diagnostic development plan, the Company evaluated TPS methodology in the expansion portion of the Phase 1 study in ovarian cancer and demonstrated that TPS captures a broad dynamic range of expression values, enriches for response, and offers reproducibility advantages across commercial labs. The Company also disclosed the steps it has taken to develop a robust, predictive and reproducible commercial diagnostic assay which will be used in a prospectively defined retrospective analysis in UPLIFT.

UPGRADE combination dose escalation umbrella study in ovarian cancer expected to initiate in the third quarter of 2021. The Company plans to initiate the UPGRADE study in the third quarter of 2021 to evaluate the combination of UpRi with other agents, starting with a platinum combination. The Phase 1, open-label, dose-escalation portion of the study will determine the maximum tolerated dose (MTD) and safety and tolerability of a once-every-four-week (Q4W) administration of UpRi in combination with Q4W administration of carboplatin for six cycles followed by UpRi monotherapy in platinum-sensitive patients with high-grade serous ovarian cancer who have received 1-2 prior platinum-based regimens. Patients will not be preselected for NaPi2b expression; however, archival or fresh tissue will be required for retrospective assessment of expression. Upon completion of the dose-escalation portion of the study, the Company plans to initiate the expansion portion in combination with carboplatin for six cycles followed by UpRi monotherapy to assess feasibility as well as efficacy to inform next steps in this broader and less heavily-pretreated patient population.

NSCLC adenocarcinoma cohort of the expansion portion of Phase 1 study continues to enroll patients. The Company is on track to recruit approximately 40 patients in the expansion phase of the study. The Company plans to report interim data in the second half of 2021.
XMT-1592, first Dolasynthen ADC targeting NaPi2b:

Phase 1 dose escalation study of XMT-1592 is ongoing, with interim data anticipated in the second half of 2021. The Company has exceeded the maximum tolerated dose and continues to further explore dose and schedule. The Company plans to disclose interim data in the second half of 2021 and further outline the XMT-1592 development plan in NSCLC in the fourth quarter of 2021.
XMT-1660, first-in-class Dolasynthen ADC targeting B7-H4:

Investigational New Drug (IND)-enabling studies of XMT-1660 ongoing with Phase 1 studies expected to start in early 2022. B7-H4 is expressed in high unmet need tumors such as breast, endometrial and ovarian. B7-H4 is expressed on both tumor cells and immunosuppressive tumor-associated macrophages (TAMs). This provides the potential for both a direct, cytotoxic antitumor effect as well as for additional payload delivery to the tumor microenvironment that can further contribute to immunogenic cell death, dendritic cell activation, and stimulation of an immune response consistent with the features of the Company’s unique DolaLock payload. In a poster presented at the Virtual 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April, the Company presented preclinical data demonstrating XMT-1660’s superior efficacy compared to other potential candidate B7-H4 ADCs, covering a range of drug-to-antibody ratios, in multiple triple-negative breast cancer models as well as a ER+/HER2- breast cancer model. These data further support the clinical development of XMT-1660 for the treatment of B7-H4-expressing tumors.
XMT-2056, first Immunosynthen STING-agonist ADC:

IND-enabling studies of XMT-2056 ongoing with Phase 1 studies expected to start in early 2022. In April 2021, the Company presented two posters at AACR (Free AACR Whitepaper) highlighting preclinical efficacy of XMT-2056 as well as mechanistic data representative of the Immunosynthen platform. These data demonstrated the differentiation of XMT-2056 and its ability to induce robust anti-tumor immune activity, with only minimal increases in systemic cytokine levels, and a significant benefit over the benchmark IV-administered free STING-agonist in mice. Preclinical mechanistic data suggests that the tumor cell-intrinsic STING pathway can be activated in the presence of cues from immune cells, a potential key differentiator from other innate immune activation approaches. Further, in vitro studies showed that blocking Type III interferons (IFNs) inhibits the production of key cytokines and cancer cell killing induced by STING-agonist ADC treatment, pointing to a potentially important role for Type III IFNs in anti-tumor immune responses downstream of STING pathway activation in tumor cells. The Company plans to disclose the target for the XMT-2056 in the fourth quarter of 2021.
Corporate

Appointed Alejandra Carvajal as Senior Vice President and Chief Legal Officer. Ms. Carvajal was most recently the Chief Legal Officer, General Counsel & Secretary at Momenta Pharmaceuticals, where she led the company’s legal operations through both business restructuring and successful execution of an acquisition by Johnson & Johnson for $6.5 billion. She also served as a key strategic legal partner in the company’s financing, business development, and contractual decision-making efforts. Prior to joining Momenta, Ms. Carvajal served as the Vice President, General Counsel at Cerulean Pharma. Previously, she worked at Millennium Pharmaceuticals in several positions of increasing seniority.

Appointed Allene Diaz to Board of Directors. Ms. Diaz has over thirty years of experience in the pharmaceutical industry most recently serving as Senior Vice President of R&D portfolio management and decision sciences at GlaxoSmithKline. She previously held senior strategic product planning and commercial roles at Tesaro and Merck KGaA. She has contributed to the development, launch and commercialization of multiple global cancer therapies. Ms. Diaz is currently on the board of both BCLS Acquisition Corporation and Allena Pharmaceuticals, having previously served as a non-executive director at Erytech for three years before her current board positions.
Upcoming Events

Mersana will participate in a virtual presentation at the Jefferies Healthcare Conference scheduled for June 1-4, 2021.
A Trial in Progress poster detailing the design of the UPLIFT single-arm registration strategy evaluating UpRi in platinum resistant ovarian cancer will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting scheduled for June 4-8, 2021.
First Quarter 2021 Financial Results

Cash and cash equivalents as of March 31, 2021, were $228.4 million, compared to $255.1 million in cash and cash equivalents as of December 31, 2020. In addition, the Company has the option to draw additional funds through its debt financing agreement with Silicon Valley Bank.

Net cash used in operating activities in the first quarter of 2021 was $27.0 million. The Company expects that its available funds will be sufficient to support its operating plan commitments for approximately two years.

Research and development expenses for the first quarter of 2021 were approximately $27.4 million, compared to $12.2 million for the same period in 2020. The difference was primarily due to an increase in UpRi and XMT-1592 manufacturing, clinical and regulatory expenses, an increase in manufacturing activities for preclinical and discovery stage programs and an increase in headcount. Non-cash stock-based compensation expense included in research and development expenses increased by $1.5 million, primarily related to an increase in the valuation of stock-based awards as a result of stock appreciation.
General and administrative expenses for the first quarter of 2021 were approximately $7.2 million, compared to $4.9 million during the same period in 2020 primarily due to an increase in headcount and consulting and professional fees. Non-cash stock-based compensation expense included in general and administrative expenses increased by $0.9 million, primarily related to an increase in the valuation of stock-based awards as a result of stock appreciation.
Net loss for the first quarter of 2021 was $34.7 million, or $0.50 per share, compared to net loss of $16.9 million, or $0.35 per share, for the same period in 2020. Weighted average common shares outstanding for the quarters ended March 31, 2021 and March 31, 2020 were 68,987,857 and 47,988,630, respectively.
Conference Call Details
Mersana Therapeutics will host a conference call and webcast today at 4:30 p.m. ET to report financial results for the first quarter 2021 and provide certain business updates. To access the call, please dial 877-303-9226 (domestic) or 409-981-0870 (international) and provide the Conference ID 5046849. A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com.