On November 9, 2021 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, immunologic diseases (e.g., complement-mediated diseases and cancers) and central nervous system disorders, reported recent highlights and developments as well as financial results for the third quarter ended September 30, 2021, which include (Press release, Omeros, NOV 9, 2021, View Source [SID1234594904]):

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OMIDRIA revenues for the third quarter of 2021 were $30.0 million compared to $28.8 million in the second quarter. The 4.1 percent increase over the prior quarter primarily reflects growth in sales of OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% in ambulatory surgery centers (ASCs).
Net loss in the third quarter of 2021 was $22.7 million, or $0.36 per share, including non-cash expenses of $6.4 million, or $0.10 per share. This compares to a net loss of $28.6 million, or $0.46 per share, which included non-cash expenses of $3.9 million, or $0.06 per share, for the previous quarter.
At September 30, 2021, Omeros had cash, cash equivalents and short-term investments available for operations of $50.4 million.
In early November, the Centers for Medicare and Medicaid Services (CMS) reconfirmed that OMIDRIA qualifies for separate payment in the ASC setting under CMS’ policy regarding non-opioid pain management surgical drugs.
On October 18, 2021, Omeros announced the receipt of a Complete Response Letter from the U.S. FDA regarding the Company’s biologics license application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Omeros is preparing for a Type A meeting with FDA to determine the most expeditious path forward for approval of narsoplimab in the treatment of HSCT-TMA.
"With CMS reconfirming separate payment for OMIDRIA in the ASC setting, Omeros, together with cataract surgeons and facility administrators, is appreciative and confident that patients will continue to be able to access OMIDRIA, improving surgical outcomes," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "This is reflected in continued sales growth, with an increasing percentage of Medicare Advantage and commercial payers also recognizing the benefits of the drug and appropriately reimbursing for its use. The increasing OMIDRIA revenues are important as we focus our resources on our complement programs, primarily to achieve FDA approval of the narsoplimab BLA in HSCT-TMA and to drive the other high-priority components of our complement franchise – the Phase 3 trial of narsoplimab in IgA nephropathy and our MASP-3 inhibitor OMS906, which we plan to accelerate from a Phase 1 trial in healthy subjects to assessing the drug in PNH patients. We expect that our portfolio of commercial and development programs will continue to advance throughout 2022, and we look forward to capitalizing on the opportunities that the coming year holds."

Third Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:
In early November, CMS released its Outpatient Prospective Payment System (OPPS) and ASC Payment System final rule for calendar year 2022. The final rule reconfirms that OMIDRIA qualifies for separate payment in the ASC setting under CMS’ policy regarding non-opioid pain management surgical drugs.
The NOPAIN Act continues to attract strong bipartisan support in both chambers of Congress, with 34 sponsors in the Senate and 74 in the House of Representatives. If enacted, the bill would mandate, for a renewable period of 5 years, Medicare separate payment in both the ASC and hospital outpatient settings for non-opioid surgical pain management drugs, like OMIDRIA, that have demonstrated in a clinical trial or through data published in a peer-reviewed journal the ability to replace or avoid opioid use or reduce the quantity of opioids prescribed.
A manuscript reporting the results of an independent investigator study demonstrating that the administration of OMIDRIA during cataract surgery is associated with reduced use of intraoperative fentanyl and concurrent pain reduction was published online in the Journal of Cataract and Refractive Surgery. The results of the study are consistent with those of an earlier study published in Clinical Ophthalmology.
A review article discussing the evolution of pain management in cataract surgery, particularly the use and associated risks of opioids in cataract surgery and how the use of non-opioid alternatives, with a focus on OMIDRIA, can help to address the opioid crisis was also accepted for publication in the Journal of Cataract and Refractive Surgery.
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 HSCT-TMA, immunoglobulin A (IgA) nephropathy, atypical hemolytic uremic syndrome (aHUS) and severely ill COVID-19 patients, include the following:
Results from long-term follow-up from the completed Phase 2 clinical trial evaluating narsoplimab in patients with IgA nephropathy were presented by Dr. Richard Lafayette, Professor of Medicine and Director of the Glomerular Disease Center at Stanford University, at the annual congress of the American Society of Nephrology (ASN). Adults with severe IgA nephropathy receiving narsoplimab treatment were followed for up to 35 months and showed that narsoplimab treatment resulted in sustained proteinuria reduction and a markedly slowed rate of decline of estimated glomerular filtration rate (eGFR). Patients received a median of one 12-week course of narsoplimab annually, with 58 percent of patients receiving only one course per year or less. Overall, patients’ renal function, as assessed by eGFR, improved (25 percent of patients) or stabilized versus an external control group matched for proteinuria and eGFR.

Using the same analytical approach adopted by other companies* to determine the impact of proteinuria reduction on long-term risk of need for dialysis, the unprecedented 64.4 percent reduction in proteinuria that was seen in the Phase 2 narsoplimab-treated patients is predicted to delay progression to renal dialysis by more than 41.6 years compared to standard of care, a substantially longer projected delay to need for dialysis than has been reported for any other drug in development for the treatment of renal disease.

Another presentation at ASN was the first report of the effects of lectin-pathway inhibition on urinary complement levels in kidney disease. The presentation assessed complement levels in urinary samples collected during the clinical course of a rapidly deteriorating young woman with IgA vasculitis. Narsoplimab treatment was associated with substantial reduction in markers of local complement activation and stabilization of kidney function as measured by eGFR. The work was conducted by a consortium led by Peter Garred, MD, DMSc, Chair and Professor of Clinical Molecular Medicine at the University of Copenhagen.
Last month a manuscript examining the significance of the lectin pathway of complement in the pathogenesis of IgA nephropathy and the role of lectin pathway inhibition with narsoplimab as a potential therapeutic approach was published in the Journal of Clinical Medicine. Dr. Mohamed Daha, Professor Emeritus in the Department of Nephrology at Leiden University, is the senior author on the paper.
Two manuscripts are being prepared for publication based on work conducted at Omeros’ collaborative laboratories at the University of Cambridge. The first is directed to a profile of disease-specific complement-marker abnormalities identified by our team studying hospitalized COVID-19 patients in the two major hospitals affiliated with the University of Cambridge and a large number of sera from the U.K.’s national COVID-response biobank. The data demonstrate that, very early in severe COVID-19, lectin pathway hyperactivation occurs and causes consumption of the complement components shared between the lectin and classical pathways, impairing classical pathway function. Narsoplimab, by blocking lectin pathway activation, has now been shown to restore classical pathway function in COVID-19 patients. Omeros is developing a broad intellectual property position directed to a profile of complement biomarkers – and their associated assays – as a potentially early indicator of severe COVID-19 and as means to assess therapeutic response.

The second manuscript further examines the finding that lectin pathway hyperactivation severely impairs the classical complement activation pathway, which critically supports the infection-fighting adaptive immune response. A substantial incidence of life-threatening secondary infections occur in severe COVID-19. The data suggest that, by blocking the lectin pathway, narsoplimab could allow recovery of classical pathway functional activity and protect against infection by maintaining the complement-dependent antimicrobial defense of adaptive immunity in severe COVID-19 patients.
Updates regarding Omeros’ other development programs and platforms include the following:
Recent data from our Phase 1 clinical trial evaluating the safety, tolerability, pharmacodynamics and pharmacokinetics of our lead MASP-3 inhibitor antibody, OMS906, show high level suppression of alternative complement pathway activity. We have decided to forego the multiple-ascending dose portion of our Phase 1 trial in healthy subjects in favor of moving directly into patients with paroxysmal nocturnal hematuria, or PNH, who have an unsatisfactory response to the C5 inhibitor ravulizumab. We expect this shift to accelerate our overall clinical program evaluating OMS906 in PNH.
Financial Results

For the third quarter of 2021, OMIDRIA revenues were $30.0 million compared to $28.8 million for the second quarter, an increase of $1.2 million or 4.1 percent.

Total costs and expenses for the third quarter of 2021 were $48.3 million compared to $52.8 million for the preceding quarter. The decrease in the third quarter was primarily due to reduced preclinical research and development costs.

For the three months ended September 30, 2021, Omeros reported a net loss of $22.7 million, or $0.36 per share, which included non-cash expenses of $6.4 million, or $0.10 per share. This compares to a net loss in the previous quarter of $28.6 million, or $0.46 per share, which included non-cash expenses of $3.9 million, or $0.06 per share.

As of September 30, 2021, the company had $50.4 million of cash, cash equivalents and short-term investments. The company also has a line of credit, which permits borrowing up to the lesser of $50 million or 85 percent of eligible accounts receivable, less certain reserves. Omeros also has an "at the market" program in place that allows the company to sell, from time to time, up to $150 million of its common stock.

Conference Call Details

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 7744465. 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 7744465.

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

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*Carroll K. et al., Estimating Delay in Time to ESKD for Treatment Effects on Proteinuria in IgA Nephropathy and FSGS. ERA-EDTA 2021, Oral Presentation; and Calliditas Therapeutics AB, April 2019, Investor Day Webinar.

On November 9, 2021 INOVIO (NASDAQ:INO), a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to help protect people from infectious diseases and treat people with cancer, and HPV-associated diseases, reported financial results for the quarter ended September 30, 2021 (Press release, Inovio, NOV 9, 2021, View Source [SID1234594903]). INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Time today to discuss financial results and provide a general business update. The 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, "This morning, INOVIO announced that the U.S. Food and Drug Administration (FDA) provided authorization to proceed for INOVIO’s INNOVATE Phase 3 segment for its COVID-19 vaccine candidate, INO-4800, in the U.S. We’re pleased to have the opportunity for U.S. clinical trial participants to potentially contribute to the enrollment in our INNOVATE Phase 3 segment. Today’s U.S. announcement builds on our intensive global efforts in India, Brazil, Philippines, Mexico, Colombia, and Thailand where we have received authorizations to date.

"With much of the world still requiring broader access to vaccines, INO-4800 is particularly well-positioned to address global vaccine needs, having been shown, in clinical trials to-date, to be well-tolerated and to generate balanced immune responses which comprise both T and B cell engagement. In addition, INO-4800 has demonstrated a favorable thermostability profile that could facilitate global distribution. I am pleased to share that the dosing for INNOVATE Phase 3 segment is underway and we aim to have interim efficacy data in the first half 2022."

INOVIO Key Updates & Third Quarter 2021 Highlights

Key Updates

INOVIO announced that the U.S. FDA provided authorization to proceed for INOVIO’s INNOVATE Phase 3 segment for INO-4800 in the U.S. INOVIO now has the opportunity for U.S. clinical trial participants to potentially contribute to the enrollment in the INNOVATE Phase 3 segment. Since August 2021, INOVIO has received authorization to conduct its INNOVATE Phase 3 global efficacy segment in: Brazil, Colombia, Mexico, the Philippines, India, the U.S. and, most recently, Thailand. Dosing is underway and the company aims to have interim efficacy data in the first half 2022.
The WHO shared on October 26, 2021 that INO-4800 is one of two vaccine candidates initially selected for its randomized, global Phase 3 clinical trial, Solidarity Trial Vaccines. More information can be found on the WHO’s website.
Subsequent to the quarter end, INOVIO’s Phase 1 clinical data on homologous boosting of INO-4800 was posted as a pre-print in MedRxiv. The paper, titled "SARS-CoV-2 DNA Vaccine INO-4800 Induces Durable Immune Responses Capable of Being Boosted in a Phase 1 Open-Label Trial," found that among the full Phase 1 cohort of 120 participants –99 (82.5%) participants, received an optional booster (or third) dose – INO-4800 produced broad-based immune responses and was well-tolerated as both a two-dose series and as a homologous booster dose in adults of all ages.
INOVIO’s partner Advaccine received regulatory approval to conduct two clinical trials in China investigating boosting with INO-4800. The studies will include prime-boost sequential immunizations using INO-4800 and an inactivated COVID-19 vaccine.
Subsequent to the quarter end, INOVIO completed enrollment (n=220) of its Phase 1B clinical trial for INO-4500, its DNA vaccine candidate for Lassa fever. This trial (LSV-002) is the first vaccine clinical trial for Lassa fever conducted in West Africa, where the viral illness is endemic. INOVIO is advancing INO-4500 with full funding from the Coalition for Epidemic Preparedness Innovations (CEPI).
INOVIO, with Regeneron, continues to evaluate findings from the Phase 1/2 novel combination trial of DNA medicines INO-5401 and INO-9012 in combination with PD-1 inhibitor cemiplimab – which is being jointly developed by Regeneron and Sanofi – for the treatment of newly diagnosed Glioblastoma Multiforme (GBM).
INOVIO Third Quarter 2021 and Subsequent Program Updates

DNA Vaccine Candidates

INO-4800: COVID-19 Vaccine Candidate in Solidarity Trial Vaccines

INOVIO’s INO-4800 is one of two initial vaccine candidates included in the WHO’s Solidarity Trial Vaccines, which is designed to "rapidly evaluate the efficacy and safety of promising new candidate vaccines selected by an independent vaccine prioritization advisory group composed of leading scientists and experts." Recruitment for the trial has begun in Colombia, Mali and the Philippines; enrollment is expected at more than 40 sites across the three countries. According to the WHO, the trial "has the additional potential to uncover second-generation vaccines with greater efficacy, conferring greater protection against variants of concern, offering longer duration of protection, and/or using needle-free routes of administration."

INO-4800 in INNOVATE Phase 3 Trial

With the FDA’s authorization to proceed for INOVIO’s INNOVATE Phase 3 segment for INO-4800 in the U.S., INOVIO now has the opportunity for U.S. clinical trial participants to potentially contribute to the enrollment in this global Phase 3 trial. The FDA has lifted the partial clinical hold following the FDA’s review of additional non-clinical, clinical, and device information provided by INOVIO. U.S. announcement builds on our intensive global efforts in India, Brazil, Philippines, Mexico, Colombia, and Thailand where we have received authorizations to date.

Dosing is underway and the company aims to have interim Phase 3 efficacy data from INNOVATE in the first half of 2022. Pending favorable clinical efficacy data, the company plans to apply for emergency use authorization in the respective countries, where such mechanism is available.

In addition to receiving regulatory approval to proceed with INNOVATE Phase 3 in Colombia, INOVIO also signed a non-binding MOU with Colombia’s Ministry of Health and Social Protection, reflecting the intent to advance efforts to combat the continued threat posed by COVID-19 and to better prepare for future public health emergencies within Colombia. The agreement creates a framework for a collaborative arrangement under which INOVIO and Colombia’s government plan to explore knowledge sharing, technology licensing, and capacity building towards developing and producing vaccines along with biopharmaceuticals in Colombia. The potential results of these efforts include developing local manufacturing capabilities for INOVIO’s DNA medicines and related products and technologies.

Boosting

INOVIO continues to study the boosting capabilities of INO-4800 following an initial primary vaccination series using a different COVID-19 vaccine series (heterologous) or with INO-4800 (homologous). During the third quarter, INOVIO’s partner Advaccine received regulatory authorization to conduct two clinical trials in China investigating boosting with INO-4800. The studies will include prime-boost sequential immunizations using INO-4800 and an inactivated COVID-19 vaccine.

INOVIO’s Phase 1 clinical data on homologous boosting of INO-4800 was posted in pre-print form at MedRxiv.orgafter quarter end. The paper, titled "SARS-CoV-2 DNA Vaccine INO-4800 Induces Durable Immune Responses Capable of Being Boosted in a Phase 1 Open-Label Trial," reports that among the full Phase 1 cohort of 120 participants – of which 82.5%, or 99 participants, received an optional booster (or third) dose – INO-4800 produced balanced immune responses and was well-tolerated as both a two-dose series and as a homologous booster dose in adults of all ages.

Notably, a durable anti-SARS-CoV-2 antibody response was observed six months following the second dose, and a homologous booster dose administered between six-to-10.5 months following the second dose also significantly increased humoral and T cell responses. Furthermore, INO-4800 was reported to be well-tolerated, with no treatment-related serious adverse events. Most adverse events were mild in severity and did not increase in frequency with age and subsequent dosing. The newly reported results are consistent with previously shared data from the Phase 2 segment of INOVIO’s INNOVATE Phase 2/3 trial.

INO-4500: Lassa Fever

Subsequent to the quarter end, the company announced full enrollment in its Phase 1B clinical trial for INO-4500, its DNA vaccine candidate for Lassa fever. This trial (LSV-002) is ongoing at the Noguchi Memorial Institute for Medical Research in Accra, Ghana, and is the first vaccine clinical trial for Lassa fever conducted on the African continent. The viral illness is endemic in West Africa. The Phase 1B clinical trial enrolled 220 adult participants who are 18-50 years old, with the primary endpoints of evaluating safety and immunogenicity in an African population. The dosing regimen involves two vaccinations at Days 0 and 28 with either 1.0 mg or 2.0 mg dosing levels. In addition to providing insights on INO-4500’s safety and immunogenicity profile, the trial will inform dose selection for subsequent Phase 2 testing in West Africa. INOVIO is advancing INO-4500 with funding from CEPI, with INOVIO and CEPI planning to establish a stockpile for emergency use after a Phase 2 trial, if successful.

HPV-associated Diseases

VGX-3100: Cervical, Vulvar, and Anal HSIL

REVEAL 1 / REVEAL 2 (Cervical HSIL)

INOVIO has completed follow-up of subjects in REVEAL 1 (Randomized Evaluation of VGX-3100 and Electroporation for the treatment of Cervical HSIL), a Phase 3 pivotal trial evaluating VGX-3100 for the treatment of cervical high-grade squamous intraepithelial lesions caused by HPV-16 and/or HPV-18, for safety and durability of virological clearance for 18 months following the last administration. The company expects to present its findings later this year.

Additionally, INOVIO is advancing its partnership with QIAGEN to co-develop an in-vitro diagnostic tool based on a bio-marker to guide clinical decision-making for the use of VGX-3100 in cervical HSIL.

REVEAL 2 is on track to complete enrollment of 198 adult women with histologically confirmed cervical HSIL before year end. Participants will be evaluated for evidence of cervical HSIL on histology as well as evidence of HPV-16 or HPV-18 in cervical samples by type-specific HPV testing at the Week 36 visit accompanied with a one-month safety follow-up.

Immuno-oncology

INO-5401: Newly Diagnosed Glioblastoma Multiforme (GBM)

INOVIO, along with our collaborator Regeneron, continues to evaluate findings from the Phase 1/2 novel combination trial of DNA medicines INO-5401 (DNA plasmid encoding for HTERT, WT1, and PSMA cancer antigens) and INO-9012 (DNA plasmid encoding IL-12), two of INOVIO’s immunotherapeutic agents, in combination with PD-1 inhibitor cemiplimab – which is being jointly developed by Regeneron and Sanofi – for the treatment of newly diagnosed GBM. Two-year (24 months) overall survival data, including correlative immunology and tissue data, will be presented at a pre-conference workshop of the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting this month.

Third Quarter 2021 Financial Results

Total revenue was $292,000 for the three months ended September 30, 2021, compared to $236,000 for the same period in 2020. Total operating expenses were $60.2 million compared to $36.6 million for the same period in 2020.

INOVIO’s net loss for the quarter ended September 30, 2021 was $60.2 million, or $0.29 per basic and diluted share, compared to net income of $19.2 million, or $0.12 per basic and $0.11 diluted share, for the quarter ended September 30, 2020.

The net income for the 2020 quarter was primarily due to the $35.3 million change in fair value of the derivative liability related to the embedded conversion feature in our August 2019 Convertible Bonds, which was revalued at each reporting period and then immediately prior to the full conversion of these bonds into shares of the Company’s common stock in August 2020. The Company also recorded a gain on investment in affiliated entities of $27.0 million during the quarter, primarily related to the sale of its equity interest in GeneOne. Without the non-cash gain on derivative liability and the gain on investment in affiliated entities, the Company’s net loss for the quarter would have been $43.1 million and basic net loss per share would have been $0.26.

Operating Expenses

Research and development (R&D) expenses for the three months ended September 30, 2021, were $47.1 million compared to $26.5 million for the same period in 2020. The increase in R&D expenses was primarily related to higher drug manufacturing, outside services and clinical study expenses related to INO-4800, an increase in drug manufacturing and clinical study expenses related to INO-4802 and higher employee and contractor compensation. The increase was also due to a decrease in contra-research and development expense recorded from grant agreements of $2.4 million, among other variances

General and administrative (G&A) expenses were $13.2 million for the three months ended September 30, 2021, versus $10.1 million for the same period in 2020. The increase in G&A expenses was primarily related to an increase in employee compensation, including non-cash stock-based compensation, partially offset by lower expenses for work performed related to corporate marketing and communications, among other variances.

Capital Resources

As of September 30, 2021, cash and cash equivalents and short-term investments were $394.9 million compared to $411.6 million as of December 31, 2020. As of September 30, 2021, the Company had 210.4 million common shares outstanding and 227.0 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 September 30, 2021, which can be accessed at: View Source

Conference Call / Webcast Information

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

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

On November 9, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the third quarter ended September 30, 2021 (Press release, Veracyte, NOV 9, 2021, View Source [SID1234594902]).

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"We are pleased with our third quarter performance as we experienced strong year-over-year revenue growth despite the headwind of the COVID-19 Delta variant," said Marc Stapley, Veracyte’s chief executive officer. "With the completion of our acquisition of HalioDx, the launch of our novel noninvasive Percepta Nasal Swab test for early lung cancer risk assessment and of our Decipher Bladder test to help guide bladder cancer treatment decisions, the pieces are coming together to transform our company into a global cancer diagnostics leader."

Third Quarter 2021 Financial Results

For the third quarter of 2021, as compared with the third quarter of 2020:

Total Revenue was $60.4 million, an increase of 94%, including $4.7 million of HalioDx revenue;
Gross Margin was 64%, a decrease of 300 basis points including the impact of HalioDx and the associated purchase accounting; Gross Margin equaled 68%, an increase of 100 basis points, before the impact of HalioDx;
Operating Expenses, Excluding Cost of Revenue, were $55.4 million, an increase of 123%, including $7.5 million of HalioDx expenses and $5.8 million in acquisition-related expenses;
Net Loss was $14.1 million, an increase of 243%, including $5.8 million of acquisition-related expenses and $6.3 million of HalioDx net loss;
Basic and Diluted Net Loss Per Common Share was $0.20, an increase of 150%;
Net Cash Used in Operating Activities was $1.4 million including $3.9 million of acquisition-related expenses; and
Cash and Cash Equivalents were $164.0 million at September 30, 2021.
For the nine months ended September 30, 2021, compared to the prior year:

Total Revenue was $152.2 million, an increase of 83%, including $4.7 million of HalioDx revenue;
Gross Margin was 66%, an increase of 200 basis points including the impact of HalioDx and the associated purchase accounting; Gross Margin equaled 68%, an increase of 400 basis points, before the impact of HalioDx;
Operating Expenses, Excluding Cost of Revenue, were $170.2 million, an increase of 113%, including $7.5 million of HalioDx expenses and $45.3 million of acquisition-related expenses;
Net Loss was $65.0 million, an increase of 142%, including $45.3 million of acquisition-related expenses and $6.3 million of HalioDx net loss;
Basic and Diluted Net Loss Per Common Share was $0.97, an increase of 87%, including $0.68 per share attributable to acquisition-related expenses recorded in general and administrative expenses; and
Net Cash Used in Operating Activities was $40.1 million, including $43.4 million of acquisition-related expenses.
Third Quarter 2021 and Recent Business Highlights

Commercial Growth:

Grew volume to 20,972 tests, including a small contribution from the Immunoscore Colon Cancer test, an increase of 79% compared to the same period in 2020.
Gained a new Blues coverage policy for Decipher Prostate, making the test a covered benefit for the plan’s 5 million members and bringing the total number of covered lives to over 150 million. Also secured contracts for the test with a large Blues plan and national government payer.
Evidence Development and Guideline Inclusion:

Six abstracts demonstrating the performance and utility of our genomic pulmonology and urology tests were presented at the 2021 American College of Chest Physicians (CHEST) and American Society for Radiation Oncology (ASTRO) annual meetings, respectively.
New long-term clinical utility data for the Afirma Genomic Sequencing Classifier were published in the Journal of the Endocrine Society and showed that the test helped reduce unnecessary surgeries in patients with indeterminate thyroid nodule cytology.
Data published in the Journal of Urology demonstrated that the Decipher Bladder genomic test accurately identifies bladder tumors that are most likely to respond to chemotherapy prior to radical cystectomy.
New NCCN Clinical Practice Guidelines for Oncology were published and include specific treatment recommendations for men with prostate cancer uniquely based on their Decipher Prostate RP genomic classifier score.
New Pan-Asian adapted ESMO (Free ESMO Whitepaper) Clinical Practice Guidelines recommended the Immunoscore Colon Cancer test to refine the prognosis of stage 2 and stage 3 colon cancer patients in conjunction with traditional assessment.
Pipeline Advancement:

Began offering the Percepta Nasal Swab test to a limited number of sites as part of a clinical utility study to build the clinical evidence needed to support reimbursement.
Commenced the commercial launch of the Decipher Bladder test following Medicare coverage.
HalioDx Acquisition:

Completed the acquisition of HalioDx for $321 million on August 2, 2021, bringing to Veracyte IVD manufacturing and development capabilities, deep scientific expertise in immuno-oncology and the Immunoscore Colon Cancer test.
2021 Financial Outlook

Veracyte is updating its 2021 annual total revenue guidance to $210 million to $218 million, from the previous guidance range of $200 million to $208 million, with HalioDx expecting to contribute approximately $10 million. This range represents 79% to 86% total revenue growth for fiscal 2021 compared to fiscal 2020.

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 November 9, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported it is advancing HB-201 to Phase 2, to be evaluated in combination with pembrolizumab as 1st- or 2nd-line treatment for Human Papillomavirus Positive 16 (HPV16+) squamous cell head and neck cancers (HNSCC) (Press release, Hookipa Biotech, NOV 9, 2021, View Source [SID1234594901]). Interim Phase 1 data in heavily pre-treated patients continue to show HB-200 monotherapy (both HB-201 alone and HB-202/HB-201) is highly effective at expanding T cells, has a favorable tolerability profile and promising, early anti-tumor activity. As of November 1, 2021, among 28 patients dosed intravenously, HB-200 resulted in a 75 percent disease control rate and shrinkage of target lesions in 53 percent of patients. In these patients, HOOKIPA has observed three partial responses (including one confirmed and one unconfirmed in an ongoing patient) and one ongoing patient with a near partial response (29 percent tumor shrinkage). Based on the strength of the HB-200 data, HOOKIPA has prioritized its oncology portfolio and plans further development of its infectious disease programs to be done in partnership with other companies. HOOKIPA will host an investor conference call to review the data at 4:30 p.m. ET.

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"We are incredibly excited about our Phase 1 HB-200 data, especially the demonstrated tumor-specific T cell responses and tumor shrinkage in heavily pre-treated HNSCC patients, which we believe are highly differentiated from other active immunization technologies," said Joern Aldag, Chief Executive Officer at HOOKIPA. "Based on these data, we’re excited to advance our promising HB-200 program into Phase 2, initially with the HB-201 and pembrolizumab combination for head and neck cancer patients, while accelerating the development of our earlier stage immuno-oncology candidates HB-300 and HB-700 in prostate and KRAS-mutated cancers, respectively, and focusing our efforts on exploring the potential of our novel arenaviral technology to address unmet needs in cancer."

HB-200 data update
Interim data from the ongoing Phase 1 dose escalation study (NCT04180215) show that
HB-200 (either as HB-201 or as alternating two-vector HB-202/HB-201) rapidly induces high levels of tumor-specific CD8+ T cells considered to be predictive of response, with a favorable tolerability profile and promising, early anti-tumor activity in heavily pre-treated HPV16+ HNSCC cancer patients.

As of the November 1, 2021 data cut-off, 62 patients (representing 24 new patients since the data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June 2021) with advanced HPV16+ tumors were enrolled and received HB-200 therapy. Forty patients with HNSCC tumors were treated intravenously every three weeks, including 20 patients who received single vector HB-201 and 20 patients who received alternating two-vector HB-202/HB-201. The other 22 patients had either other HPV16+ tumor types (not HNSCC) and/or received different HB-200 regimens. Participants received a median of three prior therapies (ranging from zero to 11), and 87 percent had previously received a checkpoint inhibitor regimen. The following safety and interim efficacy data reflect the November 1 cut-off date.

Safety results
HB-200 continued to demonstrate a favorable tolerability profile in heavily pre-treated patients with HPV16+ cancers, highlighting its potential in possible combination with checkpoint inhibitors and other agents. Treatment-related adverse events were reported in 66 percent of 62 evaluable patients, with only 8 percent experiencing treatment-related adverse events rated grade 3 or higher.

Interim efficacy results
HB-200 demonstrated promising, early anti-tumor activity in the 28 evaluable patients with advanced HNSCC. Specifically:

HB-201 showed a 71 percent disease control rate (10/14 evaluable patients, including one confirmed partial response and one unconfirmed partial response, previously reported in December 2020);
Alternating two-vector HB-202/HB-201 demonstrated a 79 percent disease control rate (11/14 evaluable patients, including one ongoing unconfirmed partial response and one ongoing near partial response with 29 percent tumor shrinkage); and,
HB-200 showed tumor shrinkage in 53 percent of patients (15/28 evaluable patients) and an ongoing median progression-free survival (mPFS) of 3.45 months.
These results compare favorably to the standard of care treatments nivolumab and pembrolizumab used in a 2nd plus-line setting in PD1-inhibitor naïve HNSCC patients. Based on peer-reviewed published data, nivolumab showed a mPFS of 2 months1 whereas pembrolizumab had disease control rates of 35 percent overall and 40 percent in the HPV+ subset in the 2nd plus-line setting.2

T cell data
Interim data continued to show that HB-200 rapidly induces high levels of activated, tumor-specific CD8+ T cells. As of the September 1, 2021 data cut-off, 20 patients were evaluable, including 10 patients who received HB-201 and 10 who received alternating two-vector HB-202/HB-201. The analysis showed:

More than 90 percent of patients showed an increase in tumor-specific CD8+ T cells within 2 weeks of initial HB-200 dose (either HB-201 or HB-202/HB-201),
More than 50 percent of patients had tumor-specific CD8+ T cell levels that exceeded the single-digit percentage threshold of the circulating T cell pool, which is generally considered a strong indicator of response; and,
50 percent of patients with paired biopsies (3/6 patients) showed elevated tumor infiltrating lymphocytes ("TILs"), or an increase in CD8+ T cells in their tumors.
Based on a review of published literature, we believe that no other active immunization approach has demonstrated these types of results, which highlight the magnitude of tumor-specific CD8+ T cells induced by HB-200 therapy as well as the potential for HOOKIPA’s versatile arenaviral platform to enhance anti-tumor activity across tumor killing mechanisms.

"While these T cell data are preliminary, it’s clear that HB-200 induces a rapid and robust vaccine-specific T cell response at magnitudes that we as a field have theorized would result in efficacy, if such levels were ever achieved," said Dmitriy Zamarin, MD, PhD, Translational Research Director in Gynecologic Medical Oncology at Memorial Sloan Kettering Cancer Center (MSK) and co-investigator in this study. "Hookipa’s arenavirus vectors are, for the first time, generating these levels and, with that, we are seeing monotherapy efficacy in patients with advanced heavily-pretreated cancers."

Oncology pipeline expansion
There is considerable unmet need in head and neck cancers, and the HB-200 program represents broad potential for additive benefits in combination with current standard of care and novel agents to improve anti-tumor immune response in these patients. HOOKIPA has initiated the Phase 2 expansion portion of its ongoing HB-200 study to evaluate HB-201 in combination with pembrolizumab in 1st- and 2nd-line HNSCC patients.

The company also plans to initiate a separate, randomized Phase 2 study of HB-200 in combination with pembrolizumab as part of its clinical collaboration with Merck & Co., Inc., Kenilworth, NJ, USA.

Based on the positive HB-200 data to-date, HOOKIPA is focusing future research and development in oncology, advancing efforts in head and neck cancer with HB-200 and prostate cancers with HB-300, as well as expanding its pipeline to include HB-700, a new program targeting KRAS-mutated colorectal, pancreatic and lung cancers.

Infectious disease portfolio update
Updated interim data from the ongoing Phase 2 clinical trial (NCT03629080) of HB-101, a prophylactic Cytomegalovirus (CMV) vaccine candidate, show strong immunogenicity and reduced incidence of CMV viremia in people who received three doses of HB-101, consistent with results previously reported in November 2020. Compared to placebo, participants vaccinated with three HB-101 doses prior to kidney transplant had:

Strong immunogenicity with 86 percent seroconversion and 100 percent CD8+ T cell responses;
a 41 percent reduction in CMV viremia (presence of CMV DNA in the blood);
a 41 percent reduction in the use of antiviral therapy; and,
No change in CMV disease.
While there were two cases of CMV disease reported in the placebo group in November 2020, these cases have since been re-classified as not CMV disease.

Safety and tolerability were evaluated in 80 participants who were enrolled in the trial by the cut-off date of July 30, 2021. HB-101 was generally well tolerated with 21 percent of HB-101 recipients experiencing side effects related to vaccine administration. A total of five cases of human leukocyte antigen (HLA)-sensitization have been reported, four characterized as serious adverse events.

Enrollment closed in June 2021 with 80 patients enrolled, and participants will continue to be monitored for the 12-month observation period following kidney transplantation. Final results are anticipated in 2023. With no approved CMV vaccine, there remains considerable unmet need for people with solid organ transplants. HOOKIPA will explore partnership opportunities for further development of HB-101 in order to focus on advancing its promising oncology portfolio.

HOOKIPA is progressing its research collaboration with Gilead to develop a potential functional cure for Hepatitis B virus (HBV). The HBV program successfully passed Gilead’s Request for Development milestone, and Gilead plans to progress the program into IND-enabling stage in 2022 to support IND filing for the arenavirus vector combination. For the HIV program, after HOOKIPA successfully completed all pre-clinical research obligations in accordance with the mutual Collaboration Agreement, Gilead informed HOOKIPA of their intention not to move forward with this program according to current terms. HOOKIPA is in ongoing discussions with Gilead regarding a revised Collaboration Agreement.

Investor call
HOOKIPA will host an investor conference call to review the data at 4:30 p.m. ET.

A live webcast of the call can be accessed on HOOKIPA’s website at View Source An archived webcast will be available for 30 days on the Events webpage.

About HB-202/HB-201
HB-201 and HB-202 are HOOKIPA’s lead oncology candidates engineered with the company’s proprietary replicating arenaviral vector platform. Each single-vector compound uses a different arenavirus backbone (Lymphocytic Choriomeningitis Virus for HB-201 and Pichinde Virus for HB-202), while expressing the same antigen, an E7E6 fusion protein derived from HPV16. In pre-clinical studies, alternating administration of HB-201 and HB-202 resulted in a ten-fold increase in immune response and better disease control than either compound alone. HB-201 is being tested clinically as a single vector therapy and also in an alternating vector combination with HB-202.

About the HB-200 trial (NCT04180215)
This Phase 1/2 clinical trial is an open-label trial exploring different dose levels and dosing schedules in individuals with treatment-refractory HPV16+ head and neck cancers who progressed on standard of care, including check point inhibitors. The trial is evaluating HB-201 as a monotherapy, as an alternating 2-vector therapy with HB-202, and in combination with a PD-1 inhibitor. The primary endpoint of Phase 1 is a recommended Phase 2 dose. Secondary endpoints include safety and tolerability, as well as preliminary efficacy defined by RECIST 1.1. The study also includes exploratory objectives on immunogenicity and pharmacodynamic biomarkers.

On November 9, 2021 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported financial results for the third quarter ended September 30, 2021 (Press release, NanoString Technologies, NOV 9, 2021, View Source [SID1234594900]).

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Third Quarter Financial Highlights

Product and service revenue of $36.9 million, 23% year-over-year growth
Instrument revenue of $14.5 million, 13% year-over-year growth. Instrument revenue includes:
$8.5 million of GeoMx Digital Spatial Profiler (DSP) instrument revenue, 14% year-over-year growth
$6.0 million of nCounter Analysis System instrument revenue, 11% year-over-year growth
Consumables revenue of $18.0 million, 32% year-over-year growth. Consumables revenue includes:
$4.5 million GeoMx DSP consumables revenue, 224% year-over-year growth
$13.5 million of nCounter consumables revenue, 10% year-over-year growth
Service revenue of $4.4 million, 24% year-over-year growth
Cash, cash equivalents and short-term investments of $369.9 million at September 30, 2021
"NanoString’s Spatial Biology franchise is having a banner year, as we continue to build on our leadership position in this dynamic market and pursue our mission to map the universe of biology. Our GeoMx DSP instrument orders grew approximately 40%, as our Whole Transcriptome Assays using next generation sequencing readout appealed to customers across both discovery and translational research. We’ve highlighted the power of our new CosMx Spatial Molecular Imager with a newly-released dataset and manuscript that demonstrate in situ imaging of lung cancer samples using both the largest number of RNA targets and currently the only workflow compatible with formalin-fixed paraffin-embedded (FFPE) tissue samples," said Brad Gray, President & CEO of NanoString. "Meanwhile, our nCounter franchise posted strong instrument placements, while consumables revenue continued to be impacted by residual effects of the pandemic."

GeoMx DSP

GeoMx Installed Base: Grew installed base to approximately 225 GeoMx DSP Systems at September 30, 2021, as compared to approximately 100 at September 30, 2020
Spatial Organ Atlas: In October 2021, released an annotated reference dataset of whole transcriptomes from six organs spatially resolved using GeoMx DSP, providing a publicly-accessible database for understanding organ structure and function
GeoMx Publications: Increased cumulative peer-reviewed publications to approximately 70 as of September 30, 2021, with approximately 10 new publications during the quarter
CosMx Spatial Molecular Imager (SMI)

Branding: Introduced the ‘CosMx’ branding for our SMI platform. Together, CosMx SMI and GeoMx DSP represent a compelling portfolio of technologies that span the continuum of applications in spatial biology
First Public Dataset: Released the first high resolution dataset generated using CosMx SMI, including RNA expression from almost 1,000 genes mapped at single cell and subcellular resolution within eight FFPE non-small lung cancer specimens
Technology Publication: Published a manuscript in the online journal BioRxiv describing the CosMx SMI technology, performance specifications and applications of the platform
nCounter

nCounter Installed Base: Grew installed base to approximately 1,030 nCounter Analysis Systems at September 30, 2021, as compared to approximately 915 systems at September 30, 2020
nCounter Antibody Drug Conjugates (ADC) Development Panel Launch: In October 2021, launched the nCounter ADC Development Panel, a specialized gene expression tool that is designed to provide molecular insights into important biological questions and challenges of oncology therapies
nCounter Publications: Surpassed 4,900 cumulative peer-reviewed publications utilizing nCounter technology at September 30, 2021
2021 Outlook

The company updated its revenue outlook for 2021, with results expected as follows:

GeoMx DSP revenue of approximately $49 to $50 million, as compared to previous guidance of $48 to $50 million, driven by instrument orders trending to the top end of the company’s 40% to 50% expected annual growth range
nCounter revenue, inclusive of all service revenue, of $91 to $94 million, as compared to previous guidance of $95 to $97 million, due to reduced consumables utilization caused by the residual effect of the pandemic
Total product and service revenue of $140 to $144 million, as compared to previous guidance of $143 to $147 million
The company reiterated its full-year outlook on gross margin, operating expenses and adjusted EBITDA.

Third Quarter Financial Results

We have elected to present selected non-GAAP, or adjusted, financial measures, including Adjusted EBITDA. These adjusted financial measures are calculated excluding certain items that may make it more challenging to compare our GAAP operating results across periods. Such items may include collaboration revenue, stock-based compensation, depreciation and amortization, or one-time charges such as transaction related fees and expenses or restructuring charges and severance costs. A reconciliation of adjusted financial measures to the nearest comparable GAAP financial measure can be found in the notes and table at the end of this press release.

Supplemental Information

As a supplement to the table above, we have posted to the investor relations section of our website, at www.nanostring.com, supplemental financial data that includes our adjusted financial measures as compared to the nearest comparable GAAP financial measures, for the third quarter and the nine months ended September 30, 2021 and for each quarter of and the full year of 2020.

Conference Call

Management will host a conference call today beginning at 1:30 pm PT / 4:30 pm ET to discuss these results and answer questions. Investors and other interested parties can register for the call in advance by visiting View Source After registering, an email confirmation will be sent, including dial-in details and unique conference call codes for entry. Registration is open throughout the call, but to ensure connection for the full call, registration in advance is recommended. The link to the webcast and audio replay will be made available at the Investor Relations website: www.nanostring.com. A replay of the call will be available beginning November 9, 2021 at 7:30pm ET through midnight ET on November 16, 2021. To access the replay, dial (800) 585-8367 or (416) 621-4642 and reference Conference ID: 2826727. The webcast will also be available on our website for one year following the completion of the call.