On November 9, 2021 Avalo Therapeutics, Inc. (Nasdaq: AVTX), a leading clinical-stage precision medicine company that discovers, develops, and commercializes targeted therapeutics for patients with significant unmet clinical need in immunology, immuno-oncology, and rare genetic diseases reported that business updates and third quarter 2021 financial results (Press release, Avalo Therapeutics, NOV 9, 2021, View Source [SID1234594906]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The third quarter was focused on execution ahead of a number of data readouts in multiple key pipeline programs anticipated in the coming months," said Mike Cola, Chief Executive Officer of Avalo Therapeutics. "We further solidified our balance sheet with a successful public offering in September that puts the Company in a position to support the development of our pipeline through multiple catalysts."

Business Updates:

In July 2021, the Company reported positive initial results for the low-dose cohort (1.0 mg/kg) of its Phase 1b proof-of-concept study of AVTX-002, an investigational first-in-class fully human anti-LIGHT (tumor necrosis factor superfamily member 14 (TNFSF14)) monoclonal antibody (mAb), in adult patients with moderate to severe Crohn’s disease.
Completed rebranding to Avalo Therapeutics, Inc. from Cerecor Inc., accentuating the Company’s transition to developing innovative targeted therapies in immunology, immuno-oncology, and rare genetic diseases.
In September 2021, the Company raised gross proceeds of approximately $31.5 million through a public offering of common stock, which strengthens and extends the Company’s financial resources to advance its clinical pipeline towards key development milestones. Avalo had cash and cash equivalents of $71.5 million as of September 30, 2021.
During the third quarter of 2021, the Company completed its second drawdown of $10 million and third drawdown of $5 million under its previously announced $35 million venture debt financing agreement with Horizon Technology Finance Corporation. With the closing of the second and third tranches, the Company has received the full $35 million of gross proceeds under its debt financing agreement.
Program Updates and Milestones:

AVTX-002: Anti-LIGHT mAb targeting immune-inflammatory diseases including acute respiratory distress syndrome (ARDS) and moderate-to-severe inflammatory bowel disease (Crohn’s disease and ulcerative colitis).
The Company has completed enrollment in Cohort 2 (3.0 mg/kg) of its Phase 1b proof-of-concept trial of AVTX-002 in moderate-to-severe Crohn’s disease and anticipates top-line data in the fourth quarter of 2021.
Based on the positive data from Cohort 1 (1.0 mg/kg) of its Phase 1b trial of AVTX-002 in moderate-to-severe Crohn’s disease, the Company has expanded the IBD program to include patients with moderate-to-severe ulcerative colitis who are refractory to anti-TNF alpha therapies.
The Company remains in dialogue with the FDA and is working through feedback to determine the trial design for a registrational study of AVTX-002 in COVID-19 ARDS and accompanying timelines, including the potential expansion to a larger patient population in broader ARDS.
AVTX-007: Anti-IL-18 mAb targeting immuno-oncology and immune-inflammatory diseases including multiple myeloma (MM) and adult onset Still’s disease (AOSD).
The Company anticipates top-line data from the Phase 1b clinical trial in relapsed or refractory MM patients in the fourth quarter of 2021.
The Company anticipates initial data from the Phase 1b clinical trial in AOSD patients in the first quarter of 2022.

AVTX-006: Dual mTORc1/c2 small molecule inhibitor for complex lymphatic malformations.
The Company anticipates initial data from the Phase 1b proof-of-concept clinical trial in the first quarter of 2022.
AVTX-800 programs (AVTX-801, AVTX-802, and AVTX-803): Therapeutic doses of monosaccharide therapies for congenital disorders of glycosylation (CDGs).
AVTX-801 – In collaboration with the Frontiers in Congenital Disorders of Glycosylation Consortium clinical program, data from the pivotal trial evaluating the safety and efficacy of D-galactose in Phosphoglucomutase-1 deficiency related CDG (PGM1-CDG) patients are anticipated in 2022.
AVTX-802 – Data from the pivotal trial evaluating the safety and efficacy of D-mannose in Mannose phosphate isomerase deficiency related CDG (MPI-CDG) patients are anticipated in 2022.
AVTX-803 – Data from the pivotal trial evaluating the safety and efficacy of L-fucose in Leukocyte Adhesion Deficiency II (LAD II) patients are anticipated in the first half of 2022.
Third Quarter 2021 Financial Update:

As of September 30, 2021, Avalo had $71.5 million in cash and cash equivalents, representing a $52.6 million increase as compared to December 31, 2020. The increase was primarily driven by gross proceeds of $31.5 million from an underwritten public offering completed in September 2021, $35 million from a debt financing agreement entered into in June 2021 ($20 million funded in the second quarter and remaining $15 million funded in the third quarter), and gross proceeds of $40.7 million from an underwritten public offering completed in January 2021. Such increases were partially offset by operating expenditures, the majority of which related to pipeline development.

Net product revenue of the Company’s non-core commercialized product was $1.4 million for the three months ended September 30, 2021, which was largely consistent with the net product revenue for the three months ended September 30, 2020 of $1.1 million.

Total operating expenses increased $3.4 million for the three months ended September 30, 2021 as compared to the three months ended September 30, 2020, which was the largest driver of the increase in net loss period over period. The increased operating expenses were largely driven by a $1.7 million increase in research and development expenses due to Avalo’s continued advancement of its maturing pipeline.

Condensed Consolidated Balance Sheets

(a) The unaudited condensed consolidated statements of operations for the three and nine months ended September 30, 2021 and 2020 have been derived from the reviewed financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.

On November 9, 2021 MannKind Corporation (Nasdaq: MNKD) reported financial results for the quarter and nine months ended September 30, 2021 (Press release, Mannkind, NOV 9, 2021, View Source [SID1234594905]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The MannKind team continues to stay focused and execute our corporate objectives of preparing for the commercial launch of Tyvaso DPI, moving our pipeline forward and growing Afrezza," said Michael Castagna, PharmD, Chief Executive Officer of MannKind Corporation. "The recently completed animal toxicology studies of inhaled clofazimine support our timeline of moving this unique chemical entity into Phase 1 clinical trials by year-end."

Third Quarter 2021 Results
Total revenues were $22.2 million for the third quarter of 2021, an increase of $6.9 million or 45% from the same period in 2020, reflecting Afrezza net revenue of $9.8 million and collaboration and services revenue of $12.5 million. Afrezza net revenue increased $2.5 million, or 34%, compared to $7.3 million in the third quarter of 2020 as a result of higher prescription demand and price, including lower gross-to-net deductions. Collaboration and services revenue for the third quarter of 2021 increased $4.4 million, or 54%, compared to the third quarter of 2020 primarily due to additional development work associated with our collaboration with United Therapeutics ("UT").

Afrezza gross profit for the third quarter of 2021 was $5.9 million compared to $3.7 million in the same period of 2020, a 61% increase that was driven primarily by an increase in Afrezza net revenue. Gross margin in the third quarter of 2021 was 61% compared to 51% for the same period in 2020, reflecting the favorable impact of increased revenue.

Research and development ("R&D") expenses for the third quarter of 2021 were $3.7 million compared to $1.5 million for the third quarter of 2020. This increase of $2.2 million, or 146%, was attributable to research activities on our product pipeline and to the initiation of the Afrezza pediatrics clinical study (INHALE-1).

Selling, general and administrative ("SG&A") expenses for the third quarter of 2021 were $17.2 million compared to $13.9 million for the third quarter of 2020, an increase of $3.3 million, or 24%. The increase was primarily attributable to promotional expenses and patient support services of $2.0 million as well as personnel-related expenses of $2.6 million, which was driven by increased stock-based compensation and additional headcount to support Afrezza growth. The spending in SG&A in the third quarter of 2021 was partially offset by a reduction for the promotional cost for Thyquidity that was recognized as cost of revenue — collaboration and services in 2021.

For the third quarter of 2021, the gain on foreign currency translation for insulin purchase commitments denominated in Euros was $2.1 million compared to a loss of $3.9 million for the third quarter of 2020. The fluctuation was due to the change in the U.S. dollar to Euro foreign currency exchange rate.

Interest expense on debt for the third quarter of 2021 was $2.8 million compared to $2.4 million for the third quarter of 2020. This increase of $0.4 million was the result of interest on the $230.0 million 2.5% senior convertible notes issued in the first quarter of 2021, partially offset by a decrease in interest due to the repayment of $35.1 million of outstanding principal under the Mann Group non-convertible promissory note and the repayment of $10.0 million outstanding principal under the MidCap credit facility in the second quarter of 2021. In addition, we reduced the interest rates on the outstanding principal balances under the MidCap credit facility and the Mann Group convertible note through amendments to the respective agreements in the second quarter of 2021.

Gain on extinguishment of debt, a non-cash item, for the three months ended September 30, 2021 was $4.9 million as a result of the Small Business Association’s (the "SBA") forgiveness of our PPP loan in July 2021.

The net loss for the third quarter of 2021 was $4.4 million, or $0.02 per share, compared to $11.3 million in the third quarter of 2020, or $0.05 per share. The decreased net loss of $6.8 million was primarily due to an increase in Afrezza net revenues and revenues from collaboration and services, as well as a non-cash gain on the extinguishment of the PPP loan, partially offset by an increase in the cost of revenue from collaboration and services and of SG&A expenses. Non-GAAP net loss, adjusted to exclude the PPP loan debt extinguishment was $9.4 million, or $0.04 per share, for the three months ended September 30, 2021 compared to $11.3 million, or $0.05 per share, for the prior year period.

Nine Months September 30, 2021
Total revenues were $62.9 million for the nine months ended September 30, 2021, an increase of $16.2 million or 35% from the same period in 2020, reflecting Afrezza net revenue of $27.8 million and collaboration and services revenue of $35.1 million. Afrezza net revenue increased 25% compared to $22.3 million for the nine months ended September 30, 2020, as a result of higher prescription demand, the negative effects of the COVID-19 pandemic in the prior year period, a more favorable mix of Afrezza cartridges, and price including lower gross-to-net deductions. Collaboration and services revenue for the nine months ended September 30, 2021 increased $10.7 million, or 44%, compared to the nine months ended September 30, 2020, primarily due to additional development work associated with our collaboration with UT.

Afrezza gross profit for the nine months ended September 30, 2021 was $15.3 million compared to $10.8 million in the same period of 2020, a 41% increase that was driven primarily by an increase in Afrezza net revenue. Cost of goods sold increased $1.1 million compared to the same period in 2020, primarily due to a $2.0 million fee for an amendment of our insulin supply agreement and a $1.1 million decrease in manufacturing activities which resulted in a lower amount of costs capitalized to inventory, partially offset by $0.9 million of costs associated with lower manufacturing cost per unit and the termination of the free goods program in December 2020. Gross margin for the nine months ended September 30, 2021 was 55% compared to 49% for the same period in 2020. On a non-GAAP basis, which excludes the $2.0 million insulin supply amendment fee, gross margin was 62% for the nine months ended September 30, 2021 compared to 49% for the same period in 2020, reflecting the favorable impact of increased revenue.

R&D expenses for the nine months ended September 30, 2021 were $8.4 million compared to $4.7 million for the nine months ended September 30, 2020. This increase of $3.7 million, or 79%, was attributable to costs incurred for research activities on the product pipeline and to the initiation of the INHALE-1 study as well as personnel costs associated with additional headcount.

SG&A expenses for the nine months ended September 30, 2021 were $54.7 million compared to $41.9 million for the nine months ended September 30, 2020, an increase of $12.8 million, or 30%. The increase was primarily attributable to promotional expenses and patient support services of $5.8 million and personnel-related expenses of $8.5 million, which reflected increased stock-based compensation, additional headcount to support Afrezza growth and our voluntary reduction in compensation expense in response to the COVID-19 pandemic in the prior year. The spend in selling expenses was partially offset by a reduction related to the co-promotional cost for Thyquidity, which was recognized as cost of revenue – collaborations and services.

An impairment of $1.9 million was recognized for the nine months ended September 30, 2020 for a commitment asset related to the future funding commitments of the MidCap credit facility.

For the nine months ended September 30, 2021, the gain on foreign currency translation for insulin purchase commitments denominated in Euros was $5.0 million compared to a $4.0 million loss for the nine months ended September 30, 2020. The fluctuation was due to the change in the U.S. dollar to Euro foreign currency exchange rate.

Interest expense on debt for the nine months ended September 30, 2021 was $12.4 million compared to $7.1 million for the nine months ended September 30, 2020. This increase of $5.3 million was the result of a $3.7 million milestone obligation achieved in the first quarter of 2021 and interest on the $230.0 million 2.5% senior convertible notes issued in the first quarter of 2021, partially offset by a decrease in interest due to the repayment of $35.1 million of outstanding principal under the Mann Group non-convertible promissory note and the repayment of $10.0 million outstanding principal under the MidCap credit facility in the second quarter of 2021. In addition, we reduced the interest rates on the outstanding principal balances under the MidCap credit facility and the Mann Group convertible note through amendments to the respective agreements in the second quarter of 2021.

Non-cash net loss on extinguishment of debt of $17.2 million for the nine months ended September 30, 2021 consisted of a $22.1 million loss on extinguishment of debt for the amendment to the Mann Group convertible note, which did not result in a change in our financial position, partially offset by a $4.9 million gain on extinguishment of debt as a result of the SBA’s forgiveness of our PPP loan in July 2021.

The net loss for the nine months ended September 30, 2021 was $52.9 million, or $0.21 per share, compared to a $30.8 million net loss in the nine months ended September 30, 2020, or $0.14 per share. The increased net loss of $22.0 million was primarily due to the non-cash loss on extinguishment of the Mann Group convertible note of $22.1 million net of a non-cash gain on extinguishment of the PPP loan of $4.9 million, as well as an increase in SG&A expenses, cost of revenue – collaboration and services, partially offset by an increase in Afrezza net revenues and revenues from collaboration and services. Non-GAAP net loss as adjusted for the $17.2 million net loss on extinguishment of debt and the $2.0 million Amphastar amendment fee was $33.7 million, or $0.14 per share, for the nine months ended September 30, 2021 compared to $30.8 million, or $0.14 per share, for the same period in the prior year.

Cash, cash equivalents, and investments at September 30, 2021 were $181.1 million compared to $67.0 million at December 31, 2020. The increase in cash, cash equivalents and investments was primarily due to the issuance of $230.0 million of 2.5 % senior convertible notes in the first quarter of 2021.

Non-GAAP Measures
To supplement our unaudited condensed consolidated financial statements presented under U.S. generally accepted accounting principles (GAAP), we are presenting certain non-GAAP financial measures. We are providing these non-GAAP financial measures to disclose additional information to facilitate the comparison of past and present operations, and they are among the indicators management uses as a basis for evaluating our financial performance. We believe that these non-GAAP financial measures, when considered together with our GAAP financial results, provide management and investors with an additional understanding of our business operating results, including underlying trends.

These non-GAAP financial measures are not meant to be considered in isolation or as a substitute for comparable GAAP measures; should be read in conjunction with our unaudited condensed consolidated financial statements prepared in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future there may be other items that we may exclude for purposes of our non-GAAP financial measures; and we may in the future cease to exclude items that we have historically excluded for purposes of our non-GAAP financial measures. Likewise, we may determine to modify the nature of its adjustments to arrive at our non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measures as used by us in this report have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following tables reconcile our gross margin financial measure to a non-GAAP presentation as adjusted for the nonrecurring amendment fee related to an amendment to our Insulin Supply Agreement.

The following tables reconcile our financial measure for net loss and EPS as reported in our condensed consolidated statement of operations to a non-GAAP presentation as adjusted for the $4.9 million non-cash gain on extinguishment of the PPP loan for the three months ended September 30, 2021 and the $22.1 million non-cash loss on extinguishment of the Mann Group convertible note and the $4.9 million gain on extinguishment of the PPP loan for the nine months ended September 30, 2021, which did not result in a change in our financial position, as well as the $2.0 million Amphastar amendment fee.

* Not meaningful
(1) There is no provision for income taxes associated with the non-cash net loss on extinguishment of debt or the Amphastar amendment fee as a result of our full valuation allowance.

Conference Call
MannKind will host a conference call and presentation webcast to discuss these results today at 5:00 p.m. Eastern Time. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at www.mannkindcorp.com under Events & Presentations. A replay will be available on MannKind’s website for 14 days.

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]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

OMIDRIA revenues for the 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

_______________
*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

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Dr. J. Joseph Kim, President and CEO of INOVIO, said, "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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are 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