On August 9, 2022 Omeros Corporation (Nasdaq: OMER), a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation and immunologic diseases, including complement-mediated diseases and cancers, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2022, which include (Press release, Omeros, AUG 9, 2022, View Source [SID1234617920]):

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!

For the quarter ended June 30, 2022, Omeros earned royalties of $17.2 million on net sales of the company’s former ophthalmology product OMIDRIA. Royalties earned in the quarter represent 50 percent of net sales of OMIDRIA by Rayner Surgical, Inc. (Rayner), which purchased Omeros’ ophthalmology assets in December 2021. Rayner’s reported net sales of $34.5 million for the second quarter of 2022, all of which were in the U.S., establish a new all-time high for quarterly OMIDRIA sales and represent a $5.7 million increase over net sales of $28.8 million reported by Omeros for the second quarter of last year.
Net loss in 2Q 2022 was $30.9 million, or $0.49 per share, which included $3.7 million of non-cash expenses, or $0.06 per share. This compares to a net loss of $28.6 million, or $0.46 per share for the prior year quarter, which included $3.9 million of non-cash expenses, or $0.06 per share.
At June 30, 2022, Omeros had $122.6 million of cash, cash equivalents and short-term investments available for operations, which is a reduction of $19.7 million from March 31, 2022. In addition, Omeros had $14.5 million in net receivables available for operations at June 30, 2022.
In June 2022, Omeros submitted to the United States Food and Drug Administration (FDA) a request for Formal Dispute Resolution regarding the Complete Response Letter (CRL) issued by FDA last year regarding the Company’s biologics license application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Formal dispute resolution is an official pathway that enables a sponsor to appeal a decision by an FDA division to a higher authority within FDA, in this case the Office of New Drugs (OND). Last month, in accordance with the standard dispute resolution procedure, Omeros had a formal meeting with the OND official assigned to decide the dispute. A decision is expected in August 2022.
"We remain confident that approval of narsoplimab in TA-TMA is warranted, and we look forward to OND’s decision later this month," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "In addition to FDA’s decision on narsoplimab approval, a series of value-driving milestones have aligned over the next few quarters: data from our Phase 3 trial of narsoplimab in IgA nephropathy are on track for readout by mid-next year; our MASP-3 inhibitor OMS906 is starting trials in PNH and C3 glomerulopathy with efficacy data targeted by early next year; about that same time, we expect data from our Phase 1 trial of OMS1029, our long-acting MASP-2 inhibitor, which began dosing earlier this week; also in early 2023, our PDE7 inhibitor OMS527 should have data available in a clinically predictive primate model of levodopa-induced dyskinesias; and, with separate payment for OMIDRIA reconfirmed by CMS in its recently released proposed rule for the Outpatient Prospective Payment System, we expect continuing growth in our royalty stream and, should OMIDRIA obtain long-term reimbursement, to secure the $200-million milestone payment."

Second Quarter and Recent Clinical Developments

Recent developments regarding narsoplimab, Omeros’ lead monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in advanced clinical programs for the treatment of TA-TMA, immunoglobulin A (IgA) nephropathy, atypical hemolytic uremic syndrome (aHUS) and severely ill COVID-19 patients, include the following:
Enrollment in Omeros’ Phase 3 ARTEMIS-IGAN trial continues to progress toward an anticipated readout of 9-month data on proteinuria by mid-next year.
The Omeros teams in Cambridge, UK and Seattle recently published two manuscripts detailing the company’s latest COVID-19-related discoveries, the first by Ali et al. in Frontiers in Immunology and the second by Lynch et al. in Clinical and Translational Medicine. Together, these publications describe the findings that:
Patients with severe COVID-19 early in disease show marked complement consumption driven by lectin pathway hyperactivation, causing secondary hypocomplementemia and loss of complement-mediated immune protection against microbial infection. This hypocomplementemia increases the risk of clinically severe infections, a common cause of morbidity and death in COVID-19.
Narsoplimab restores complement function and bactericidal activity, preventing risk of secondary infection.
Narsoplimab is also being evaluated for the treatment of hospitalized COVID-19 patients in the I-SPY COVID-19 platform trial sponsored by Quantum Leap Healthcare Collaborative (QLHC). Omeros looks forward to QLHC’s disclosure of the narsoplimab results.
Recent developments regarding OMS906, Omeros’ lead monoclonal antibody targeting MASP-3, the key activator of the alternative pathway, and OMS1029, the company’s long-acting, next-generation MASP-2 inhibitor, include the following:

In July, OMS906 received designation from FDA as an orphan drug for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Orphan-drug designation is granted by FDA to encourage development of a drug that targets a condition affecting fewer than 200,000 U.S. patients annually. The benefits of orphan drug designation include seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and waiver of certain administrative fees.
To accelerate obtaining OMS906 efficacy data, in addition to the Phase 1b trial expected to begin enrolling soon and evaluating OMS906 in patients with PNH who have had an unsatisfactory response to the C5 inhibitor ravulizumab, Omeros is expanding its program of OMS906 clinical trials to include treatment-naïve PNH patients and complement 3 (C3) glomerulopathy patients as well as one or more related indications. Efficacy data in these indications are targeted by early 2023.
Omeros has submitted an abstract describing the results of the OMS906 Phase 1 study for presentation at a major medical congress later this year. Preliminary results from the Phase 1 study were previously reported and no safety signals of concern were noted.
A Phase 1 clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics (PK/PD) of OMS1029 in healthy subjects is underway, with the first dose administered earlier this week. Designed for convenient dosing, OMS1029 is expected to enable Omeros to pursue a range of indications complementary to those for narsoplimab. Based on animal PK/PD data to date, dosing in humans is expected to be once-monthly to once-quarterly by subcutaneous or intravenous administration.
Financial Results

On December 23, 2021, Rayner acquired OMIDRIA and certain related assets and liabilities. The completion of the sale required Omeros to reclassify all revenues and expenses related to OMIDRIA as discontinued operations for fiscal year 2021 in its financial statements.

During the second quarter of 2022, Omeros earned royalties of $17.2 million on sales of OMIDRIA, which were recorded as a reduction from the OMIDRIA contract royalty asset. The company also recorded $10.1 million of income in discontinued operations, primarily representing interest income and remeasurement adjustments to the OMIDRIA contract royalty asset.

Total costs and expenses for the second quarter of 2022 were $37.4 million compared to $45.6 million for the second quarter of 2021. The decrease was primarily due to the timing of narsoplimab manufacturing activities and a reduction in U.S. TA-TMA pre-launch activities.

Net loss was $30.9 million in the second quarter of 2022, or $0.49 per share, which included $3.7 million of non-cash expenses, or $0.06 per share. This compares to a net loss of $28.6 million, or $0.46 per share, including $3.9 million of non-cash expenses, or $0.06 per share, in 2Q 2021.

As of June 30, 2022, the company had $122.2 million of cash, cash equivalents and short-term investments with an additional $14.5 million in receivables, net.

Conference Call Details

To access the live conference call via phone, please dial (833) 634-2592 from the United States and Canada or (412) 902-4100 internationally and ask to be placed into the Omeros earnings call. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (877) 344-7529 from the United States, (412) 317-0088 internationally, and (855) 669-9658 from Canada. The replay access code is 4990130.

For online access to the live or subsequently archived webcast of the conference call, go to Omeros’ website at View Source

On August 9, 2022 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company focused on discovering and developing clinically differentiated small molecule therapeutics targeting fundamental biological pathways of cancers, reported financial results for the second quarter ended June 30, 2022 and highlighted recent corporate accomplishments (Press release, Zentalis Pharmaceuticals, AUG 9, 2022, View Source [SID1234617919]).

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 remain steadfast in our commitment to accelerate the clinical development of our lead candidates, ZN-c3 and ZN-d5, and we took many important steps this quarter – including continuing to strengthen our leadership team and cash position – to help reach this goal," commented Dr. Kimberly Blackwell, Chief Executive Officer of Zentalis. "To that end, we are excited to receive financial and strategic support from Pfizer, whose commitment will help us to realize the full potential of ZN-c3, a selective Wee1 inhibitor designed to induce synthetic lethality in cancer cells. We look forward to sharing updates on our ongoing and planned trials later this year."

Program Highlights:

The Company is focusing its resources on investigating the full potential of its lead clinical candidates ZN-c3, its Wee1 inhibitor, and ZN-d5, its BCL-2 inhibitor, as monotherapies and in combination across a wide range of cancers. Therefore, Zentalis will discontinue the clinical development of ZN-c5, its oral SERD, and ZN-e4, its EGFR inhibitor, following completion of its existing clinical trials, which are closed to accrual, in these two programs.
In April 2022, Zentalis presented five abstracts at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting and held a webcast event with Key Opinion Leader, Dr. Kathleen Moore, to further discuss the clinical and preclinical data presented at the conference, with additional details available here.
Corporate Highlights:

In April 2022, Zentalis sold 953,834 of its common shares at a price of $26.21 per share to Pfizer for gross proceeds of approximately $25.0 million. Zentalis and Pfizer plan to jointly advance the clinical development of ZN-c3. In addition, Dr. Adam Schayowitz, Vice President & Medicine Team Group Lead for Breast Cancer, Colorectal Cancer and Melanoma, Pfizer, joined Zentalis’ Scientific Advisory Board.
In May 2022, the Company closed an underwritten public offering of 10,330,000 shares of its common stock at a public offering price of $19.38 per share. The total gross proceeds were approximately $200.2 million.
In May 2022, the Company appointed Kimberly Blackwell, M.D., an oncology clinical development veteran, as Chief Executive Officer. Dr. Blackwell has been a member of Zentalis’ Board since 2020 and previously served as Chief Medical Officer of Tempus Labs. Before that, she held clinical development leadership roles at Eli Lilly and Company. Additionally, Board member Dave Johnson was appointed Chairman and Cam Gallagher, MBA, a cofounder of Zentalis, was promoted to President and will remain a Board member.
In July 2022, Zentalis announced the appointment of Andrea Paul, J.D., as General Counsel and Corporate Secretary.
Second Quarter 2022 Financial Results

Cash and Marketable Securities Position: As of June 30, 2022, Zentalis had cash, cash equivalents and marketable securities of $455.2 million. The Company believes that its existing cash, cash equivalents and marketable securities as of June 30, 2022 will be sufficient to fund its operating expenses and capital expenditure requirements into the first quarter of 2025.
Research and Development Expenses: Research and development expenses for the three months ended June 30, 2022 were $43.8 million, compared to $44.8 million for the three months ended June 30, 2021. The decrease was primarily due to licensing milestones and manufacturing expenditures incurred during the three months ended June 30, 2021, which did not recur during the comparable period in 2022.
General and Administrative Expenses: General and administrative expenses for the three months ended June 30, 2022 were $19.6 million, compared to $10.4 million during the three months ended June 30, 2021. The increase in expenses was primarily attributable to an increase in non-recurring, non-cash stock-based compensation and other cash compensation.

On August 9, 2022 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported financial results for the quarter ended June 30, 2022 (Press release, Allogene, AUG 9, 2022, View Source [SID1234617918]).

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 feel confident that we could soon initiate the industry’s first Phase 2 pivotal trial for an allogeneic CAR T product, thereby paving the road not just for ALLO-501A, but our entire portfolio," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "As the use of autologous CAR T therapy increases, we are seeing a greater need for an off-the-shelf, allogeneic CAR T option. We are keenly aware of the devastating consequences patients face when only a minority are able to access the curative potential of CAR T therapy. Clinicians have been forced into the unfathomable position of needing to choose which of their patients will receive this potentially life-saving therapy. As patients face access bottlenecks, we are determined to transform CAR T therapy from a complex individualized procedure to an off-the-shelf, on demand pharmaceutical product."

Pipeline Updates

CD19 Program

In the coming weeks, the Company expects to receive clearance from the U.S. Food and Drug Administration (FDA) to initiate a potential Phase 2 pivotal clinical trial for ALLO-501A in relapsed/refractory (r/r) large B cell lymphoma (LBCL). This includes meeting Chemistry Manufacturing and Controls (CMC) requirements to use ALLO-501A from its manufacturing facility, Cell Forge 1.

In June, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-501A in r/r LBCL. RMAT designation was based on data demonstrating the potential of ALLO-501A to address the unmet need for patients who have failed other therapies. Previously presented data support the potential of ALLO-501A to provide a safe and durable alternative to approved autologous CAR T therapies in CAR T naïve patients, including manageable safety with no dose limiting toxicities (DLTs) or graft-vs-host disease (GvHD) and minimal Grade 3 Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), or Grade 3 cytokine release syndrome (CRS). In the Phase 1 ALPHA2 study, nearly all enrolled patients were able to receive therapy with the median time from enrollment to initiation of treatment of two days.

Allogene anticipates providing an update on the Phase 1 portion of the ALPHA and ALPHA2 trials toward the end of 2022. This update will include a few additional patients enrolled in ALPHA2 and will focus on longer-term follow up of patients previously treated in the ALPHA and ALPHA2 trials.

The EXPAND trial is expected to begin in 2022 and is planned to support registration of the lymphodepleting agent ALLO-647. This trial is intended to demonstrate the contribution of ALLO-647 to the lymphodepletion regimen.

BCMA Program

Allogene plans to explore its pivotal study approach and timing for its BCMA program by year end. In parallel, the Company intends to work within the framework afforded by its RMAT designation for ALLO-715 to facilitate FDA interactions and determine the best course forward.

Enrollment continues in the Phase 1 UNIVERSAL trial on ALLO-715 in r/r multiple myeloma (MM). Toward the end of 2022, Allogene intends to provide a clinical update that will focus on the longer-term follow up of patients in UNIVERSAL treated with a single dose of ALLO-715. Allogene has made the decision not to advance ALLO-715 in combination with nirogacestat from SpringWorks Therapeutics into dose expansion cohorts. There was no clear indication that the combination would meaningfully improve the benefit-risk profile of ALLO-715 as a monotherapy. Allogene’s Clinical Trial Collaboration Agreement with SpringWorks is expected to remain in effect until the data from the combination study are fully analyzed.

The IGNITE trial on TurboCAR candidate ALLO-605 continues to enroll patients in the dose escalation portion of this Phase 1 study.

Solid Tumor Programs

ALLO-316, which targets CD70, is the Company’s first AlloCAR T candidate for solid tumors. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, anti-tumor efficacy, pharmacokinetics, and pharmacodynamics of ALLO-316 in patients with advanced or metastatic clear cell renal cell carcinoma (RCC).

The FDA previously granted ALLO-316 Fast Track Designation (FTD) based on its potential to address the unmet need for patients with difficult to treat RCC who have failed standard therapies. Metastatic solid tumors have historically been a challenge regardless of treatment modality, and the five-year survival rate for patients with advanced kidney cancer is less than 15%, highlighting the need for innovation.

Corporate Highlights
CF1, Allogene’s commercial scale manufacturing facility located in Newark, California is fully operational and producing GMP material with the intent of supplying ALLO-501A for the planned pivotal study as well as other clinical trials. CF1 is projected to have the ability to manufacture approximately 20,000 ALLO-501A AlloCAR T doses annually at scale. CF1 recently earned a LEED Interior Design and Construction Gold designation from the U.S. Green Building Council (USGBS), a non-profit dedicated to sustainable building design and construction.

In July, Allogene announced the appointment of Stephen L. Mayo, Ph.D., a world-renowned expert in computational protein design, to the company’s Board of Directors. Dr. Mayo is the Bren Professor of Biology and Chemistry and Merkin Institute Professor at the California Institute of Technology in Pasadena, California. Dr. Mayo also serves on the Board of Directors of Merck & Co. and Sarepta Therapeutics, Inc.

Second Quarter Financial Results
•Research and development expenses were $57.2 million for the second quarter of 2022, which includes $13.0 million of non-cash stock-based compensation expense.
•General and administrative expenses were $19.5 million for the second quarter of 2022, which includes $9.9 million of non-cash stock-based compensation expense.
•Net loss for the second quarter of 2022 was $74.8 million, or $0.52 per share, including non-cash stock-based compensation expense of $22.9 million.
•The Company had $686 million in cash, cash equivalents, and investments as of June 30, 2022.

Updated 2022 Financial Guidance
While the Company anticipates spending to increase in the second half relative to the first half of 2022, it now expects full year GAAP Operating Expenses to be at the low end of the previous range of $360 million and $390 million. This includes estimated non-cash stock-based compensation expense of $90 million to $100 million and excluding any impact from potential business development activities. Cash burn for 2022 is now expected to be approximately $250 million.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (800) 715-9871 (U.S.) or 1 (646) 307-1963 (International). The conference ID number for the live call is 7832993. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On August 9, 2022 Neoleukin Therapeutics, Inc., "Neoleukin" (NASDAQ:NLTX), a biopharmaceutical company utilizing sophisticated computational methods to design de novo protein therapeutics, reported financial results for the second quarter ended June 30, 2022 and a midyear corporate update (Press release, Neoleukin Therapeutics, AUG 9, 2022, View Source [SID1234617917]).

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!

"Our focus at Neoleukin is the advancement of de novo proteins to solve important therapeutic challenges and address unmet medical needs," said Jonathan Drachman, M.D., Chief Executive Officer of Neoleukin. "We are excited to be part of a revolutionary approach to creating therapeutic proteins that are not based on native sequences. Our first programs, including our lead product candidate, NL-201, were developed using sophisticated computational algorithms combined with directed evolution in the laboratory. We have now added machine learning and neural networks to our technological approach, enabling faster, more accurate development of increasingly complex proteins. Using these new processes, we have been able to add two new cytokine mimetics to our discovery pipeline this year."

"At Neoleukin, we are excited to be testing NL-201, which we believe is the first fully de novo protein in clinical trials," said Priti Patel, M.D., Chief Medical Officer. "We are pleased to have begun testing NL-201 in combination with pembrolizumab in mid-May of this year. We believe this is just one of many potential opportunities to harness the immune activating properties of NL-201 in novel combination regimens."

NL-201 Update

Neoleukin is conducting a clinical trial of intravenous NL-201 in patients with advanced solid tumors. The trial is evaluating two different schedules and multiple dose levels in order to determine a recommended Phase 2 dose and schedule. Intermediate dose levels have been added to both schedules, which has extended the timeline to reach the anticipated Phase 2 dose. Dose escalation continues in both schedules, and Neoleukin now anticipates disclosing interim data in 2023.

In April 2022, Neoleukin announced the presentation of preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, highlighting the potential for NL-201 to treat non-Hodgkin lymphoma as well as synergistic antitumor activity when NL-201 is combined with radiation therapy, including significant inhibition of tumor growth and increased survival in preclinical models.

In May 2022, Neoleukin announced treatment of the first patient in a combination arm evaluating the safety and efficacy of Neoleukin’s NL-201 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab), as part of Neoleukin’s ongoing Phase 1 trial. Up to 132 patients will be enrolled in the combination arm of the study, which is being conducted through a clinical collaboration and supply agreement with Merck (known as MSD outside the United States and Canada). The trial is assessing safety, pharmacokinetics, pharmacodynamics, immunogenicity, and antitumor activity. KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

Research Updates

Technology: Neoleukin’s research team is actively engaged in the discovery of additional de novo cytokine mimetics as well as technological advances in order to accelerate the process from concept to proof of preclinical activity. Among these advances, the use of machine learning and neural networks have made it possible to design de novo proteins even when high-resolution structures have not yet been solved, using less compute resources and with a higher percentage of functional sequences. In addition to NL-201, Neoleukin has previously reported preclinical data for two de novo proteins: NL-CVX1, a decoy protein that mimics the human ACE2 binding site of the SARs-CoV2 spike protein and Neo-5171, an inhibitor of both IL-2 and IL-15 signaling.

Pipeline: Neoleukin is exploring an activator of T-regulatory cells for the treatment of inflammation and autoimmune diseases, a next generation IL-2/IL-15 agonist, and two additional undisclosed de novo cytokine mimetics for the treatment of cancer.

Summary of Financial Results

Cash Position: Cash, cash equivalents, and short-term investments totaled $116.5 million as of June 30, 2022, compared to $142.5 million as of December 31, 2021.

Based upon current internal infrastructure and pipeline initiatives, Neoleukin believes it has sufficient cash to fund operations through 2023.

R&D Expenses: Research and development expenses for the second quarter of 2022 increased to $11.0 million from $9.8 million for the second quarter of 2021. The increase was primarily due to increased clinical trial expenses related to NL-201.

G&A Expenses: General and administrative expenses for the second quarter of 2022 decreased to $4.9 million from $5.3 million for the second quarter of 2021. The decrease was primarily attributable to decreases in personnel-related and facility-related costs.

Net Loss: Net loss for the second quarter of 2022 was $15.7 million compared to a net loss of $15.1 million in the second quarter of 2021.

About NL-201

NL-201 is a de novo agonist of the IL-2 and IL-15 receptors, designed to expand cancer-fighting CD8 T cells and natural killer (NK) cells without any bias toward cells expressing the alpha receptor subunit (CD25). Previously presented preclinical data has demonstrated the ability of NL-201 to stimulate and expand CD8+ and NK cells at low doses with minimal impact on immunosuppressive regulatory T cells. Furthermore, NL-201 has demonstrated both monotherapy and combination activity across a wide range of preclinical syngeneic tumor models.

Conference Call Information

Neoleukin will host a conference call today to provide a second quarter corporate update and review financials. Details are as follows:

The archived audio webcast with slides will be available on the Investor Relations section of the Neoleukin website approximately two hours after the event and will be available for replay for at least 30 days after the event.

On August 9, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the second quarter of 2022 and provided an update on progress toward achieving key corporate objectives, as well as commercial, clinical and pipeline development milestones (Press release, Exelixis, AUG 9, 2022, View Source [SID1234617916]).

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!

"In the second quarter of 2022, Exelixis continued to execute across each of the core components of our business, highlighted in particular by our commercial and pipeline activities," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer, Exelixis. "The team drove strong commercial performance for CABOMETYX (cabozantinib), resulting in a 22 percent growth in cabozantinib franchise net product revenues year-over-year. In addition, we achieved key cabozantinib milestones, including generating positive data for the progression-free survival primary endpoint of our COSMIC-313 clinical trial and supporting Ipsen as it successfully pursued its CABOMETYX label expansion for a differentiated thyroid cancer indication in the European Union and Canada."

Dr. Morrissey continued: "Exelixis also made significant pipeline advancements during and after the close of the quarter, initiating STELLAR-303, our first phase 3 pivotal study for XL092, as well as the first-in-human phase 1 study for XL114 in non-Hodgkin’s lymphoma. Additionally, we signed new business development agreements with BioInvent and Ryvu Therapeutics to further expand our biologics capabilities and portfolio of biotherapeutics candidates. Moving into the second half of this year, we have much to look forward to, including the potential to further augment the CABOMETYX label through upcoming clinical data readouts expected from the CONTACT-01 and CONTACT-03 pivotal studies in non-small cell lung cancer and renal cell carcinoma, respectively, as well as anticipated clinical updates from our XL092, XB002 and XL102 pipeline programs. I want to thank the Exelixis team for their continued hard work and dedication during the second quarter as we continue to advance our mission on behalf of the patients we serve."

Second Quarter 2022 Financial Results

Total revenues for the quarter ended June 30, 2022 were $419.4 million, compared to $385.2 million for the comparable period in 2021.

Total revenues for the quarter ended June 30, 2022 included net product revenues of $347.0 million, compared to $284.2 million for the comparable period in 2021. The increase in net product revenues was primarily due to an increase in sales volume, which was partially offset by increases in discounts and allowances, primarily from higher utilization in the 340B Drug Pricing Program.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $72.4 million for the quarter ended June 30, 2022, compared to $100.9 million for the comparable period in 2021. The decrease in collaboration revenues was primarily related to a decrease in development cost reimbursements earned, which was partially offset by an increase in the recognition of milestone-related revenues, and higher royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited (Takeda).

Research and development expenses for the quarter ended June 30, 2022 were $199.5 million, compared to $148.8 million for the comparable period in 2021. The increases in research and development expenses were primarily related to increases in personnel expenses, clinical trial costs, license and other collaboration costs, and consulting and outside services expenses, which were partially offset by a decrease in stock-based compensation expense.

Selling, general and administrative expenses for the quarter ended June 30, 2022 were $122.8 million, compared to $98.5 million for the comparable period in 2021. The increases in selling, general and administrative expenses were primarily related to increases in personnel expenses, marketing costs, business technology initiatives and legal costs.

Provision for income taxes for the quarter ended June 30, 2022 was $17.8 million, compared to $28.8 million for the comparable period in 2021, primarily due to a decrease in pre-tax income.

GAAP net income for the quarter ended June 30, 2022 was $70.7 million, or $0.22 per share, basic and diluted, compared to GAAP net income of $96.1 million, or $0.31 per share, basic and $0.30 per share, diluted, for the comparable period in 2021.

Non-GAAP net income for the quarter ended June 30, 2022 was $89.7 million, or $0.28 per share, basic and diluted, compared to non-GAAP net income of $117.9 million, or $0.38 per share, basic and $0.37 per share, diluted, for the comparable period in 2021.

Cash, cash equivalents, restricted cash equivalents and investments were $2.0 billion at June 30, 2022, compared to $1.9 billion at December 31, 2021.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (and the related per share measures) stock-based compensation expense, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation expense, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2022 Financial Guidance

(1) Includes $45 million of non-cash stock-based compensation expense.
(2) Includes $50 million of non-cash stock-based compensation expense.

Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $347.0 million during the second quarter of 2022, up 12% over the prior quarter, comprised of net product revenues of $339.2 million from CABOMETYX and $7.9 million from COMETRIQ (cabozantinib). In the second quarter of 2022, global cabozantinib franchise net product revenues generated by Exelixis and its partners were almost $500 million. Exelixis earned $30.2 million in royalty revenues during the quarter ended June 30, 2022, pursuant to collaboration agreements with its partners, Ipsen and Takeda.

Exelixis’ Partner Ipsen Receives European Commission (EC) and Health Canada Approvals for CABOMETYX for Patients with Previously Treated Radioactive Iodine (RAI)-Refractory Differentiated Thyroid Cancer (DTC). In May, Exelixis announced its partner Ipsen received approval from the EC for CABOMETYX as a monotherapy for the treatment of adult patients with locally advanced or metastatic DTC, refractory or not eligible to RAI who have progressed during or after prior systemic therapy. This approval allows for the marketing of CABOMETYX in this indication in all 27 member states of the European Union, Norway, Liechtenstein and Iceland. Similarly, in late April 2022, Ipsen received approval from Health Canada to market CABOMETYX for a similar DTC indication in Canada. As a result of these approvals, Exelixis was eligible to receive $27.0 million in milestone payments from Ipsen, of which $25.7 million was recognized in license revenues and collaboration services revenues in the second quarter of 2022. Both approvals were based on the positive results of the phase 3 COSMIC-311 pivotal trial.

Cabozantinib Data Presentations at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. In June, cabozantinib was the subject of 13 presentations at this year’s ASCO (Free ASCO Whitepaper) Annual Meeting, held from June 3-7 in Chicago. Notable presentations included results from: cohorts 7 and 20 in non-small cell lung cancer; cohorts 3, 4 and 5 in urothelial carcinoma from the ongoing COSMIC-021 study evaluating cabozantinib in combination with atezolizumab across multiple tumor types; and a phase 2 investigator-sponsored trial from the Emory Winship Cancer Institute evaluating the combination of cabozantinib and pembrolizumab in recurrent metastatic head and neck squamous cell carcinoma.

Announcement of Top-line Results from the COSMIC-313 Phase 3 Pivotal Trial Evaluating Cabozantinib in Combination with Nivolumab and Ipilimumab in Previously Untreated Advanced Renal Cell Carcinoma (RCC). In July, Exelixis announced that COSMIC-313 met its primary endpoint, demonstrating significant improvement in progression-free survival (PFS) at the primary analysis. At a prespecified interim analysis for the secondary endpoint of overall survival (OS), the combination of cabozantinib, nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab did not demonstrate a significant benefit. Therefore, the trial will continue to the next analysis of OS. COSMIC-313 is an ongoing phase 3 pivotal trial evaluating the combination of cabozantinib, nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab in patients with previously untreated advanced intermediate- or poor-risk RCC. Exelixis intends to discuss the results with the U.S. Food & Drug Administration (FDA) to determine next steps toward a potential regulatory submission for the combination regimen for patients with previously untreated advanced intermediate- or poor-risk RCC. Detailed findings will be presented at a future medical meeting.

Pipeline Highlights

Initiation of First-In-Human Phase 1 Trial Evaluating XL114 Monotherapy in Patients with Non-Hodgkin’s Lymphoma (NHL). In April, Exelixis announced the initiation of the dose-escalation stage of the first-in-human phase 1 trial of XL114, a novel anti-cancer compound that inhibits the CARD11-BCL10-MALT1 complex, as a monotherapy in patients with NHL who have received prior standard therapies. The objectives of the study are to determine the recommended dose and/or the maximum tolerated dose of XL114 and to evaluate the safety and preliminary efficacy of XL114 in patients with NHL. The dose-escalation stage will determine the recommended dose of XL114 in patients with advanced B- and T-cell NHL. In the cohort-expansion stage, the safety and preliminary efficacy of XL114 will be further evaluated in various B-cell NHL-specific expansion cohorts. The primary endpoint of the expansion stage will be objective response rate (ORR) based on lymphoma-specific response criteria as assessed by the investigator.

Initiation of the STELLAR-303 Phase 3 Pivotal Trial Evaluating XL092 in Patients with Metastatic Colorectal Cancer (CRC). In June, Exelixis announced the initiation of STELLAR-303, a phase 3 pivotal trial evaluating XL092 in combination with atezolizumab versus regorafenib in patients with metastatic CRC that is not microsatellite instability-high or mismatch repair-deficient, who have progressed after or are intolerant to the standard of care therapy. STELLAR-303 is a global, multicenter, randomized phase 3 open-label study that will enroll approximately 600 patients with documented RAS status. The primary objective of the study is to evaluate the efficacy of the combination in patients with RAS wild-type disease, and outcomes in patients with RAS-mutated disease will also be evaluated. The primary endpoint is OS, and secondary endpoints include PFS, ORR and duration of response per Response Evaluation Criteria in Solid Tumors version 1.1 as assessed by the investigator. XL092 is Exelixis’ next-generation tyrosine kinase inhibitor in development for multiple advanced tumor types.

Corporate Updates

Exclusive Option and License Agreement with BioInvent International AB (BioInvent) to Develop Novel Antibody-Based Oncology Therapies. In June, Exelixis and BioInvent entered into an option and license agreement focused on the identification and development of novel antibodies for use as oncology therapeutics. The collaboration is intended to expand Exelixis’ portfolio of antibody-based therapies. Target and antibody discovery will be performed using BioInvent’s proprietary n-CoDeR antibody library and patient-centric F.I.R.S.T screening platform, which together allow for parallel target and antibody discovery. Under the terms of the agreement, Exelixis paid BioInvent an upfront fee of $25.0 million in exchange for rights to select three targets identified using BioInvent’s proprietary F.I.R.S.T platform and n-CoDeR library, and will have the right to exercise an option to in-license any of the target programs upon identification of a development candidate directed to that target. Upon option exercise, Exelixis will assume responsibility for all future development and commercialization activities for the development candidate, including potential antibody-drug conjugate (ADC) and bispecific antibody engineering activities.

Exclusive License Agreement with Ryvu Therapeutics S.A. (Ryvu) to Develop Novel STING Agonist-Based Targeted Cancer Therapies. In July, Exelixis and Ryvu announced an exclusive license agreement focused on the development of novel targeted therapies utilizing Ryvu’s STING (STimulator of INterferon Genes) technology. The collaboration is intended to expand Exelixis’ portfolio of biotherapeutics by combining its tumor-specific targeting approaches with Ryvu’s proprietary small molecule STING agonists and STING biology know-how. Under the terms of the agreement, Exelixis is obligated to pay Ryvu an upfront fee of $3.0 million in exchange for certain rights to Ryvu’s STING agonist small molecules, which Exelixis will seek to incorporate into targeted therapies such as ADCs. Exelixis will lead all research activities and, upon selection of each development candidate, will be responsible for all development and commercialization activities. Ryvu will provide expert guidance and know-how during the early research phase of the partnership.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended July 1, 2022, and July 2, 2021, are indicated as being as of and for the periods ended June 30, 2022, and June 30, 2021, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the second quarter of 2022 and provide a general business update during a conference call beginning at 5:00 p.m. ET / 2:00 p.m. PT today, Tuesday, August 9, 2022.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 888-338-9509 (domestic) or 412-902-4281 (international) and ask to be joined into the Exelixis conference call to participate by phone.

A telephone replay will be available until 8:00 p.m. ET on Thursday, August 11, 2022. Access numbers for the telephone replay are: 877-344-7529 (domestic) and 412-317-0088 (international); the passcode is 8698613. A webcast replay will also be archived on www.exelixis.com for one year.