On May 12, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX) (Viracta or the Company), a precision oncology company targeting virus-associated malignancies, reported financial results for the first quarter of 2021 and provided a clinical and corporate update (Press release, Sunesis, MAY 12, 2021, View Source [SID1234579881]).

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"Viracta has emerged from our first quarter as a publicly traded company well-positioned to make a significant impact on patients and create meaningful value for our shareholders. We are on track to initiate our pivotal NAVAL-1 trial as planned this quarter for the treatment of EBV-associated lymphoma, and we look forward to expanding into our second global clinical program in EBV-associated solid tumors in the second half of 2021," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta.

Dr. Royston continued, "Today, we are excited to provide additional details of the NAVAL-1 trial design, which has been reviewed by the United States Food and Drug Administration. We believe the innovative and adaptive design of this registration-enabling pivotal trial will allow us to simultaneously progress towards potential NDA filings in multiple lymphoma subtypes."

First Quarter 2021 and Recent Highlights

Clinical

Announced design of NAVAL-1, a global pivotal trial in Epstein-Barr virus (EBV)-positive relapsed/refractory (R/R) lymphoma. NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a multinational, multicenter, open-label Phase 2 basket trial. The trial, which will include multiple subtype-specific cohorts of R/R EBV-positive lymphoma patients, is designed to evaluate the anti-tumor activity of the combination treatment of nanatinostat with valganciclovir and is anticipated to enroll up to 140 patients. The primary endpoint of the trial is objective tumor response rate as assessed by an independent review committee. If successful, Viracta believes this trial could support multiple NDA filings across various EBV-positive lymphoma subtypes. Viracta remains on track to initiate NAVAL-1 in Q2 2021.
Completed enrollment in Phase 2 expansion cohort of its Phase 1b/2 R/R EBV-positive lymphoma trial, with updated data expected in H2 2021. VT3996-201, Viracta’s Phase 1b/2 open-label, dose escalation/expansion trial of nanatinostat-valganciclovir combination treatment in R/R EBV-positive lymphoma has generated promising efficacy and safety data to date, as presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020, including preliminary objective response and complete response rates (ORR/CR) of 80%/40% (n=10) and 67%/33% (n=6) in T/NK cell non-Hodgkin’s lymphoma and diffuse large B-cell lymphoma, respectively. The median duration of response was 10.4 months.
Corporate

Closed merger with Sunesis Pharmaceuticals, Inc. Shares of the combined company, Viracta Therapeutics, Inc., commenced trading on the Nasdaq Global Select Market under the ticker symbol "VIRX" on February 25, 2021.
Completed a $65 million private placement. On February 24, 2021, Viracta closed a private financing featuring a premier investor syndicate of biotechnology-focused and institutional accredited investors.
Strengthened balance sheet through a multi-license milestone and royalty monetization transaction with XOMA. In March 2021, Viracta announced that XOMA had purchased the potential future milestones and royalties associated with existing licenses relating to two clinical-stage drug candidates that Viracta obtained in the merger in exchange for an upfront payment of $13.5 million and up to $20 million in a pre-commercialization, event-based milestone. In the merger, Viracta also obtained exclusive and global rights to assets previously held by Sunesis including SNS-510, a PDK-1 inhibitor, and vecabrutinib, a BTK inhibitor. Viracta is evaluating future development and collaboration opportunities for SNS-510 and potential partnering opportunities for vecabrutinib.
Extended intellectual property protection around lead lymphoma program. In March 2021, the U.S. Patent and Trademark Office (USPTO) granted patent 10,953,011, which covers the anticipated dose regimen to be advanced in the NAVAL-1 trial. The patent provides Viracta with intellectual property protection into at least 2040.
Strengthened company leadership with new appointments to the Board of Directors and management team. Throughout the first quarter, Viracta appointed the following life sciences industry veterans to the Board of Directors: Stephen Rubino, Ph.D., MBA, and Barry J. Simon, M.D., who were each appointed following the closing of the merger, Thomas Darcy, who was appointed in connection with the closing of the merger, and Nicole Onetto, M.D., who remained on the combined company’s Board of Directors following the merger. The Company also appointed Cheryl A. Madsen, RAC as Senior Vice President, Regulatory Affairs.
Anticipated 2021 Milestones

Initiation of NAVAL-1, a global Phase 2 pivotal trial for R/R EBV-positive lymphoma: Q2 2021
Clearance of IND for a Phase 1b/2 clinical trial in EBV-positive solid tumors: mid-2021
Initiation of a global Phase 1b/2 clinical trial in EBV-positive solid tumors: H2 2021
Updated data from Phase 1b/2 trial in R/R EBV-positive lymphoma (VT3996-201): H2 2021
First Quarter 2021 Financial Results

Cash Position – Cash and cash equivalents totaled approximately $129.2 million as of March 31, 2021. Viracta expects to end 2021 with greater than $100 million in cash and cash equivalents, which it expects will be sufficient to fund its operations into 2024.
Research and Development Expenses – Research and development expenses were $4.0 million for the quarter ending March 31, 2021, compared to $3.4 million for the same period in 2020. This increase was primarily due to costs incurred to support study initiation activities for NAVAL–1, in addition to an increase in headcount and non-cash share-based compensation.
General and Administrative Expenses – General and administrative expenses were $3.8 million for the quarter ending March 31, 2021, compared to $1.0 million for the same period in 2020. This increase was primarily due to non-recurring, merger-related costs of approximately $2.0 million incurred in the period, in addition to incremental costs associated with being a publicly traded company and non-cash share-based compensation.
Acquired in-process research and development – Non-recurring, non-cash operating expenses of $84.5 million associated with the write-off of in-process research and development acquired in the merger were recorded for the quarter ending March 31, 2021.
Gain on Royalty Purchase Agreement – Gain on Royalty Purchase Agreement for the quarter ended March 31, 2021 was associated with upfront proceeds of $13.5 million received in connection with the multi-license milestone and royalty monetization transaction with XOMA Corporation in March 2021.
Adjusted income or loss from operations – Adjusted income from operations for the quarter ended March 31, 2021, excluding the non-recurring and non-cash operating expenses associated with the write-off of in-process research and development acquired in the merger (a non-GAAP measure) was $5.6 million, compared to an unadjusted loss from operations of $78.8 million. There is not a comparative adjustment to loss from operations for the same period in 2020.
Net loss – Net loss was $79.2 million, or $5.22 per share (basic and diluted) for quarter ended March 31, 2021, compared to $4.4 million, or $1.86 per share (basic and diluted), for the same period in 2020.
Key Opinion Leader Webinar

The design of NAVAL-1, a global pivotal trial in EBV-positive R/R lymphoma, will be discussed during a Key Opinion Leader webinar on May 20, 2021 at 2pm EST. The webinar will feature presentations by Pierluigi Porcu, M.D. (Thomas Jefferson University) and Kristen Cunanan, Ph.D. (Stanford University School of Medicine), who will discuss the current treatment landscape, unmet medical need in EBV-associated lymphoma and the trial design of NAVAL-1. The formal presentations will then be followed by a Q&A session with Drs. Porcu and Cunanan, accompanied by Company management. A link to register for the webcast is here: View Source

About NAVAL-1

NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a registration-enabling multinational, multicenter, open-label Phase 2 basket trial. The trial will include multiple subtype-specific with various EBV-positive relapsed/refractory lymphoma and is anticipated to enroll up to 140 patients. The primary endpoint of the trial is objective tumor response rate, while secondary endpoints include duration of response, survival outcomes, and the safety profile of the combined treatment. Trial eligibility includes patients with EBV-positive R/R lymphoma following two or more prior systemic therapies with no available standard therapies. For ENKTL patients only, eligibility includes patients with R/R disease following one or more prior systemic therapies with no available standard therapies who have failed an asparaginase-containing regimen.

On May 12, 2021 The Bayer Group reported that had a successful start to 2021 (Press release, Bayer, MAY 12, 2021, View Source [SID1234579768]). "We’re seeing a good operational performance overall, but we were impacted by negative currency effects as expected," said CEO Werner Baumann when the quarterly statement for the first quarter of 2021 was published on Wednesday . "Our Crop Science Division achieved encouraging sales growth in an improved market environment. Sales at Pharmaceuticals were steady, and in the Consumer Health Division we performed better than the competition." The company confirmed the full-year outlook it issued in February 2021.

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Group sales in the first quarter rose by 2.8 percent on a currency- and portfolio-adjusted basis (Fx & portfolio adj.) to 12.328 billion euros. EBITDA before special items fell by 6.2 percent to 4.118 billion euros. Negative currency effects impacted sales by 938 million euros and EBITDA before special items by 337 million euros. EBIT increased by 23.4 percent to 3.083 billion euros. This included net special gains of 15 million euros (Q1 2020: net special charges of 639 million euros), with income from a patent dispute related to the blood-clotting medicine Jivi more than offsetting the expenses for the ongoing restructuring program. Net income rose by 40.3 percent to 2.089 billion euros. Core earnings per share from continuing operations fell by 3.0 percent to 2.59 euros.

Free cash flow came in at minus 3.226 billion euros (Q1 2020: minus 793 million euros), the decline being mainly due to the settlement payments for litigations in the United States. This was also partly the reason for the 13.0 percent increase in net financial debt against the end of 2020, to 33.933 billion euros as of March 31, 2021.

Growth at Crop Science, especially in Latin America and Asia/Pacific

Bayer lifted sales in its agricultural business (Crop Science) by 6.4 percent (Fx & portfolio adj.) to 6.646 billion euros, with particularly strong growth in the Latin America and Asia/Pacific regions. Sales at Herbicides advanced by a significant 13.3 percent – mainly as a result of volume gains in all regions and higher prices, especially for Roundup. Business also improved substantially at Fungicides (plus 22.0 percent) and Vegetable Seeds (plus 13.9 percent). The Fungicides business benefited from volume and price increases in the Latin America region, particularly for the product Fox Xpro. This business also expanded in Asia/Pacific as the market situation normalized following the previous year’s restrictions related to the COVID-19 pandemic. Sales at Vegetable Seeds rose in all regions. The 3.4 percent (Fx & portfolio adj.) increase in sales at Soybean Seed & Traits was driven by volume gains in North America. Sales at Corn Seed & Traits remained at the level of the prior-year quarter (Fx & portfolio adj. minus 0.4 percent). This business developed positively in the Europe/Middle East/Africa region and in Latin America, partly as a result of higher prices, whereas it shrank in North America, in part due to a license expiration.

EBITDA before special items at Crop Science decreased by 6.2 percent to 2.448 billion euros, giving a margin of 36.8 percent. Positive effects from price and volume increases and the contributions from the ongoing efficiency programs did not fully offset the negative currency effects of 252 million euros.

As regards the litigation involving glyphosate-based Roundup products in the United States, approximately 96,000 current claims overall are covered by settlement agreements, or did not meet the settlement program eligibility criteria. The company continues to negotiate with plaintiffs’ counsel to reach agreements in the remainder of current cases. A new proposal for the management of future cases agreed upon with and supported by plaintiffs’ class counsel has been submitted to the responsible judge in California for preliminary approval. For the revised proposal, both parties worked diligently to address the points mentioned by the court.

Pharmaceuticals: significant advances for Xarelto and Eylea

Sales of prescription-only medicines (Pharmaceuticals) came in at 4.365 billion euros and were thus level with the prior-year period on a currency- and portfolio-adjusted basis (Fx & portfolio adj. minus 0.4 percent). In China, sales declines for Glucobay and Avelox as a result of the tender procedures implemented in 2020 were offset by growth in other products, especially the oral anticoagulant Xarelto. In the United States, the division benefited particularly from the launch of the cancer drug Nubeqa.

Bayer raised sales of Xarelto by 6.5 percent (Fx & portfolio adj.). This was mainly due to expanded volumes in China and Russia, while business in Germany declined substantially. A considerable increase of 15.8 percent (Fx & portfolio adj.) in sales of the ophthalmology drug Eylea was driven by positive development in Japan and markedly higher volumes in Europe, China and Canada. The business with the oral contraceptives YAZ/Yasmin/Yasminelle grew by 12.3 percent (Fx & portfolio adj.), with increases especially in China, Japan and the Middle East. However, there was a distinct drop in sales of the Kogenate/Kovaltry/Jivi blood-clotting medicines (Fx & portfolio adj. minus 17.9 percent) and the cancer drug Nexavar (Fx & portfolio adj. minus 21.7 percent) due to competition.

EBITDA before special items at Pharmaceuticals declined by 6.0 percent to 1.498 billion euros, yielding a margin of 34.3 percent. Earnings were diminished by price declines in sales along with negative currency effects of 57 million euros, which were offset to some extent by volume gains and cost reductions. There was also a positive effect on research and development expenses that came partly from the proportionate recognition of proceeds from the sale of a priority review voucher in the United States.
 
Consumer Health: performance ahead of competition cycling over exceptionally strong prior-year quarter

Following an exceptionally strong prior-year quarter with double-digit percentage growth, sales of self-care products (Consumer Health) registered a 4.4 percent decrease (Fx & portfolio adj.) to 1.252 billion euros in a declining market. The performance remained ahead of competition with growth momentum in the Dermatology and Nutritionals categories, with business up by 6.6 percent and 4.7 percent, respectively (Fx & portfolio adj.). However, the continuing high level of protective and hygiene measures and the ongoing lockdowns had a negative impact on sales, particularly on cough and cold products. This led to a decline of 30.1 percent (Fx & portfolio adj.) in the Allergy & Cold category. While sales in Asia/Pacific and Latin America showed continued positive growth, sales in North America and Europe/Middle East/Africa were markedly affected by the extraordinarily mild cough and cold season.

EBITDA before special items at Consumer Health moved back 3.0 percent to 292 million euros. Earnings were diminished by negative currency effects of 26 million euros. At the same time, the EBITDA margin before special items improved by 1.8 percentage points to 23.3 percent, largely on account of lower marketing expenses in the currently volatile market environment as well as one-time gains from the divestment of three non-core brands.

Sustainability efforts further enhanced

Bayer also made good progress in the area of sustainability in the first quarter. As already announced in February, the company is stepping up its efforts to achieve an even more inclusive and diverse workforce and corporate culture, targeting gender parity on all management levels for the year 2030. Also in February, the Consumer Health Division, for example, launched its Nutrient Gap Initiative to help close the gap in the supply of vitamins and minerals to people in underserved regions. The program aims to reach 50 million people annually in these regions by 2030 through direct action and in partnership with major NGOs. The Nutrient Gap Initiative will contribute to the attainment of the Consumer Health Division’s sustainability goal of providing 100 million people in underserved communities with access to everyday health care by 2030.

On May 12, 2021 Incyte (Nasdaq:INCY) reported that multiple abstracts highlighting data from its oncology portfolio will be presented during the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress, held virtually from June 9-17, 2021 (Press release, Incyte, MAY 12, 2021, View Source [SID1234579785]).

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"We are pleased that data highlighting the strength of Incyte’s oncology portfolio and partner-sponsored programs will be presented at this year’s EHA (Free EHA Whitepaper) Virtual Congress," said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapeutics, Incyte. "In particular, data being presented at the congress – including the first presentation of data from our Phase 2 study of parsaclisib, our PI3kδ inhibitor, in autoimmune hemolytic anemia; an oral presentation on real-world data for ruxolitinib, our JAK1/JAK2 inhibitor; and an ePoster from our Phase 2 combination study of ruxolitinib and parsaclisib in patients with myelofibrosis – reinforce our commitment to finding solutions for patients with significant unmet medical needs."

Key abstracts accepted by EHA (Free EHA Whitepaper) include:

Oral Presentations

Ponatinib

OPTIC Primary Analysis: A Dose-Optimization Study of 3 Starting Doses of Ponatinib (PON)1(Abstract #S153. Session: Response, Resistance and Treatment-Free Remission in CML.)

Ruxolitinib

Efficacy and Safety of Ruxolitinib in Patients with Steroid-Refractory Acute Graft-Vs-Host Disease After Crossover in the Phase 3 REACH2 Study2(Abstract #S236. Session: Stem cell transplantation – GvHD.)

Impact of Ruxolitinib on Survival of Patients with Myelofibrosis in Real World – Update of ERNEST (European Registry for Myeloproliferative Neoplasms) Study2(Abstract #S158. Session: Population based studies in myeloid disorders.)

ePosters

Parsaclisib

Efficacy and Safety Results from an Open-Label Phase 2 Study of Parsaclisib for the Treatment of Autoimmune Hemolytic Anemia (AIHA) (Abstract #EP685. Session: Enzymopathies, Membranopathies and Other Anemias.)

Pharmacologic Inhibition of PI3kδ Reduces Autoantibody Formation and is Efficacious in a Preclinical Model of Autoimmune Hemolytic Anemia (Abstract #EP693. Session: Enzymopathies, Membranopathies and Other Anemias.)

FACIT-Fatigue Subscale Outcomes from an Ongoing Phase 2, Open-Label Study of the Phosphatidylinositol 3-Kinase Delta (PI3Kδ) Inhibitor Parsaclisib in Patients with Autoimmune Hemolytic Anemia (AIHA) (Abstract #EP706. Session: Enzymopathies, Membranopathies and Other Anemias.)

Ponatinib

French Real-Life Observational Study "TOPASE" Evaluating Safety and Efficacy of Ponatinib Confirms Induction of Deep Molecular Responses in 110 Resistant or Intolerant CML Patients (Abstract #EP679. Session: Chronic Myeloid Leukemia – Clinical.)

Ruxolitinib

An Epidemiological Study of the Cardiovascular Health and Thrombotic Risk Profiles of Patients with Myeloproliferative Neoplasms in Primary Care Across the United Kingdom2 (Abstract #EP1090. Session: Myeloproliferative Neoplasms – Clinical.)

Impact of Bone Marrow Fibrosis Grade on Response and Outcome in Patients with Primary Myelofibrosis Treated with Ruxolitinib: A Post-Hoc Analysis of the JUMP Study2 (Abstract #EP1092. Session: Myeloproliferative Neoplasms – Clinical.)

Healthcare Resource Utilization in Patients with Myeloproliferative Neoplasms: A Nationwide Matched Cohort Study2 (Abstract #EP1107. Session: Myeloproliferative Neoplasms – Clinical.)

Ruxolitinib-Parsaclisib Combination Studies

Add-On Parsaclisib (a PI3kδ inhibitor) in Patients with Myelofibrosis and Suboptimal Response to Ruxolitinib: Interim Analysis from a Phase 2 Study (Abstract #EP1075. Session: Myeloproliferative Neoplasms – Clinical.)

Tafasitamab

Estimation of Long-Term Survival with Tafasitamab + Lenalidomide in Relapsed/Refractory Diffuse Large B-Cell Lymphoma3(Abstract #EP553. Session: Aggressive Non-Hodgkin Lymphoma – Clinical.)

First-MIND: A Phase 1b, Open-Label, Randomized Study to Assess Safety of Tafasitamab or Tafasitamab + Lenalidomide in Addition to R-CHOP in Patients with Newly Diagnosed DLBCL3(Abstract #EP496. Session: Aggressive Non-Hodgkin Lymphoma – Clinical.)

Lenalidomide-Induced Effects on Cell Surface Expression of CD19 and CD20 in DLBCL Cell Lines and Functional Impact on Antibody-Mediated Cytotoxicity3(Abstract #EP879 . Session: Lymphoma Biology & Translational Research.)

In addition to the presentations noted above, more than 10 publications highlighting data from Incyte’s portfolio will be made available by EHA (Free EHA Whitepaper) as publications. Notably, these publications include bioequivalence and bioavailability data for ruxolitinib’s once-daily extended release (XR) formulation – Bioequivalence of 50 mg Once-Daily Ruxolitinib Extended Release (XR) Tablets Compared to 25 mg Twice-Daily Ruxolitinib Immediate Release (IR) Tablets (Abstract #PB1706) and Relative Bioavailability and Dose Linearity of Five Strengths of Ruxolitinib Extended Release (XR) Tablets (Abstract #PB1717).

Full listings for oral presentations and ePoster sessions are available on the EHA (Free EHA Whitepaper) website: View Source Oral, poster discussion and poster sessions, as well as track-based clinical science symposia, accepted for presentation at EHA (Free EHA Whitepaper) will be available on demand for registered attendees beginning Friday, June 11, 2021, through Sunday, August 15, 2021.

On May 12, 2021 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi reported the presentation of positive results from the Phase 3 trial investigating the PD-1 inhibitor Libtayo (cemiplimab) in patients with recurrent or metastatic cervical cancer who had previously progressed on chemotherapy (Press release, Regeneron, MAY 12, 2021, View Source [SID1234579801]). The data were shared as part of a European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Plenary and add to previously reported data showing an improvement in overall survival (OS) with Libtayo compared to chemotherapy. The data will form the basis of regulatory submissions in 2021.

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"In this Phase 3 trial, Libtayo demonstrated a significant improvement in overall survival in women with advanced cervical cancer after progression on chemotherapy, reducing the risk of death by 31% compared to chemotherapy in the overall population," said Krishnansu S. Tewari, M.D., Professor and Director of the Division of Gynecologic Oncology at the University of California, Irvine and a trial investigator. "Improvements in progression-free survival and objective response rate were also demonstrated in the overall population compared to chemotherapy. Taken together, this landmark trial – which enrolled patients regardless of PD-L1 expression status – helps support the use of Libtayo as a potential new second-line treatment for women with advanced cervical cancer who face a poor prognosis and limited treatment options."

In the overall population, those treated with Libtayo (n=304) experienced significant improvements in OS, progression-free survival (PFS) and objective response rate (ORR), compared to chemotherapy (n=304), including a:

31% reduction in the risk of death (hazard ratio [HR]: 0.69; 95% confidence interval [CI]: 0.56-0.84; one-sided p=0.00011).
25% reduction in the risk of disease progression (HR: 0.75; 95% CI: 0.63-0.89; one-sided p=0.00048).
16% ORR (50 patients; 95% CI: 13-21%; one-sided p=0.00004), compared to 6% with chemotherapy (19 patients). Median duration of response was 16 months with Libtayo (95% CI: 12 months to not yet evaluable) and 7 months with chemotherapy (95% CI: 5-8 months), per Kaplan-Meier estimates.
In the trial, 78% of patients had advanced cervical cancer that was classified as squamous cell carcinoma (SCC). In this patient subgroup, significant improvements were also seen with Libtayo (n=239), compared to chemotherapy (n=238), including a:

27% reduction in the risk of death (HR: 0.73; 95% CI: 0.58-0.91; one-sided p=0.00306).
29% reduction in the risk of disease progression (HR: 0.71; 95% CI: 0.58-0.86; one-sided p=0.00026).
18% ORR (42 patients; 95% CI: 13-23%), compared to 7% with chemotherapy (16 patients; 95% CI: 4-11%).
While assessment of the adenocarcinoma was not a pre-specified endpoint, a post-hoc analysis demonstrated the following outcomes for Libtayo-treated patients (n=65) compared to chemotherapy (n=66), including a:

44% reduction in the risk of death (HR: 0.56; 95% CI: 0.36-0.85; nominal one-sided p<0.005).
9% reduction in the risk of disease progression (HR: 0.91; 95% CI: 0.62-1.34).
12% ORR (8 patients; 95% CI: 6-23%), compared to 5% with chemotherapy (3 patients; 95% CI: 1-13%).
Additionally, the Phase 3 trial found Libtayo-treated patients were able to generally improve or maintain their baseline Global Health Status/Quality of Life (GHS/QOL) over time, while those treated with chemotherapy experienced a deterioration that became clinically meaningful starting at cycle 8, per the EORTC QLQ-C30 (overall estimated mean change [95% CI]: improvement of 1.01 [-2.033, 4.047] for Libtayo, worsening of -6.81 [-10.977, -2.637] for chemotherapy; difference: 7.81; one-sided nominal p=0.00040).

No new Libtayo safety signals were observed. Safety was assessed in patients who received at least 1 dose of study treatment: 300 patients in the Libtayo group (median duration of exposure: 15 weeks; range: 1-101 weeks) and 290 patients in the chemotherapy group (median duration of exposure: 10 weeks; range: 1-82 weeks). Adverse events (AEs) were observed in 88% of Libtayo patients and 91% of chemotherapy patients, with those occurring in 15% or more Libtayo patients being anemia (25% Libtayo, 45% chemotherapy), nausea (18% Libtayo, 33% chemotherapy), fatigue (17% Libtayo, 16% chemotherapy), vomiting (16% Libtayo, 23% chemotherapy), decreased appetite (15% Libtayo, 16% chemotherapy) and constipation (15% Libtayo, 20% chemotherapy). Grade 3 or higher AEs occurred in 45% of Libtayo patients and 53% of chemotherapy patients. Among AEs in 15% or more patients, Grade 3 or higher AEs that occurred more often in the Libtayo group included asthenia (2% Libtayo, 1% chemotherapy) and pyrexia (less than 1% Libtayo, 0% chemotherapy). Immune-related AEs were observed in 16% of Libtayo patients and less than 1% of chemotherapy patients, with 6% and less than 1% being Grade 3 or higher, respectively. Discontinuations due to AEs occurred in 8% of Libtayo patients and 5% of chemotherapy patients.

The use of Libtayo in advanced cervical cancer is investigational and has not been fully reviewed by any regulatory authority.

About the Phase 3 Trial
The Phase 3, open-label, multi-center trial is the largest randomized clinical trial in advanced cervical cancer, and investigated Libtayo monotherapy versus an investigator’s choice of chemotherapy in patients with recurrent or metastatic cervical cancer that has progressed on platinum-based chemotherapy. Patients were allowed to enroll regardless of PD-L1 expression status, with 78% of patients having SCC and 22% having adenocarcinoma or adenosquamous carcinoma. The trial included women from 14 countries: the U.S., Japan, Taiwan, South Korea, Canada, Russia, Poland, Spain, Brazil, Australia, the UK, Italy, Greece and Belgium.

Patients (median age: 51 years) were randomized to receive Libtayo monotherapy (350 mg every three weeks) or an investigator’s choice of commonly used chemotherapy (pemetrexed, vinorelbine, topotecan, irinotecan or gemcitabine). The primary endpoint for the trial was OS, analyzed first among patients with SCC, then in the total population.

In March, the trial was stopped early based on the highly significant effect of Libtayo on OS among SCC patients and following a unanimous recommendation by the Independent Data Monitoring Committee.

About Cervical Cancer
Cervical cancer is the fourth leading cause of cancer death in women worldwide and is most frequently diagnosed in women between the ages of 35 and 44. Almost all cases are caused by human papillomavirus (HPV) infection, with approximately 80% classified as SCC (arising from cells lining the bottom of the cervix) and the remainder being largely adenocarcinomas (arising from glandular cells in the upper cervix). Cervical cancer is often curable when detected early and effectively managed, but treatment options are more limited in advanced stages.

It is estimated that approximately 570,000 women are diagnosed with cervical cancer worldwide each year, with deaths exceeding 250,000. In the U.S. there are 14,500 new patients diagnosed annually and approximately 4,000 women die each year.

About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.

In the U.S., Libtayo is approved for certain patients with advanced stages of cutaneous squamous cell carcinoma (CSCC), basal cell carcinoma (BCC) and non-small cell lung cancer (NSCLC) with ≥50% PD-L1 expression. Outside of the U.S., Libtayo is approved for certain patients with advanced CSCC in the European Union and six other countries, including Australia, Brazil, the UK and Canada.

The generic name for Libtayo in its approved U.S. indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA). Outside of the U.S., the generic name for Libtayo in its approved indication is cemiplimab.

About the Libtayo Development Program
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. The European Medicines Agency is assessing regulatory submissions for Libtayo monotherapy in advanced NSCLC with ≥50% PD-L1 expression and advanced BCC following treatment with a hedgehog pathway inhibitor, with European Commission decisions expected by mid-2021.

Libtayo monotherapy is being investigated in trials in adjuvant CSCC and neoadjuvant CSCC, as well as in trials combining Libtayo with either conventional or novel therapeutic approaches for both solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately a quarter of all original, FDA-approved fully human monoclonal antibodies currently available. This includes REGEN–COV (casirivimab with imdevimab), Dupixent (dupilumab), Libtayo (cemiplimab-rwlc), Praluent (alirocumab), Kevzara (sarilumab), Evkeeza (evinacumab-dgnb) and Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn).

IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS

What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.

Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI.

Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.

Libtayo is a prescription medicine used to treat people with a type of lung cancer called non-small cell lung cancer (NSCLC). Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR", "ALK "or "ROS1" gene.

It is not known if Libtayo is safe and effective in children.

What is the most important information I should know about Libtayo?
Libtayo is a medicine that may treat certain cancers by working with your immune system. Libtayo can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended.

Call or see your healthcare provider right away if you develop any new or worsening signs or symptoms, including:

Lung problems: cough, shortness of breath, or chest pain
Intestinal problems: diarrhea (loose stools) or more frequent bowel movements than usual, stools that are black, tarry, sticky or have blood or mucus, or severe stomach-area (abdomen) pain or tenderness
Liver problems: yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach area (abdomen), dark urine (tea colored), or bleeding or bruising more easily than normal
Hormone gland problems: headache that will not go away or unusual headaches, eye sensitivity to light, eye problems, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, feeling more hungry or thirsty than usual, urinating more often than usual, hair loss, feeling cold, constipation, your voice gets deeper, dizziness or fainting, or changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness
Kidney problems: decrease in your amount of urine, blood in your urine, swelling of your ankles, or loss of appetite
Skin problems: rash, itching, skin blistering or peeling, painful sores or ulcers in mouth or nose, throat, or genital area, fever or flu-like symptoms, or swollen lymph nodes
Problems can also happen in other organs and tissues. These are not all of the signs and symptoms of immune system problems that can happen with Libtayo. Call or see your healthcare provider right away for any new or worsening signs or symptoms, which may include: chest pain, irregular heartbeat, shortness of breath or swelling of ankles, confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs, double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight, persistent or severe muscle pain or weakness, muscle cramps, low red blood cells, or bruising
Infusion reactions that can sometimes be severe. Signs and symptoms of infusion reactions may include: nausea, chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain, or facial swelling
Rejection of a transplanted organ. Your healthcare provider should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had.
Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with Libtayo. Your healthcare provider will monitor you for these complications.
Getting medical treatment right away may help keep these problems from becoming more serious. Your healthcare provider will check you for these problems during your treatment with Libtayo. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may also need to delay or completely stop treatment with Libtayo if you have severe side effects.

Before you receive Libtayo, tell your healthcare provider about all your medical conditions, including if you:

— have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus

— have received an organ transplant

— have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)

— have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome

— are pregnant or plan to become pregnant. Libtayo can harm your unborn baby
Females who are able to become pregnant:

Your healthcare provider will give you a pregnancy test before you start treatment.
You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of Libtayo. Talk with your healthcare provider about birth control methods that you can use during this time.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Libtayo.
— are breastfeeding or plan to breastfeed. It is not known if Libtayo passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of Libtayo.

Tell your healthcare provider about all the medicines you take, including prescription and over- the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Libtayo include muscle or bone pain, tiredness, rash, and diarrhea. These are not all the possible side effects of Libtayo. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals and Sanofi at 1-877-542-8296.

On May 12, 2021 Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company that utilizes its human memory B cell discovery engine platform to discover and develop first-in-class antibody therapeutics, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Immunome, MAY 12, 2021, View Source [SID1234579819]).

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First Quarter and Subsequent Highlights

IMM-BCP-001: In February 2021, Immunome announced that it discovered antibodies capable of neutralizing multiple SARS-CoV-2 variants, including the South African Variant, in pseudovirus testing. In April 2021, the company announced antibody selection for its IMM-BCP-01 cocktail and shared data showing that IMM-BCP-01 neutralizes CDC SARS-CoV-2 "Variants of Concern" in preclinical testing. The cocktail was efficacious in Syrian hamsters infected with SARS-CoV-2 (USA-WA1/2020) in both prophylactic and treatment schedules. Further, the cocktail neutralized South Africa and UK variants in live virus testing and Brazil and California variants in pseudovirus testing. An Investigational New Drug (IND) filing for IMM-BCP-001 is planned for late 2Q/early 3Q 2021.
IMM-ONC-001: In March 2021, Immunome announced its proprietary antibody against IL-38 advanced into IND-enabling studies. Based on Immunome’s research findings, IL-38 functions as a novel innate immune checkpoint that inhibits infiltration and pro-inflammatory activity of innate immune cells. The company’s analysis also suggests that IL-38 is over-expressed in certain tumors and is potentially linked to reduced infiltration of innate immune cells. Immunome anticipates filing an IND for this product candidate in 2H 2021.
Appointed Dr. Dennis Giesing as Chief Development Officer and Corleen Roche as Chief Financial Officer. In March 2021, the company announced the appointment of Dennis Giesing, Ph.D. to the role of Chief Development Officer. Dr. Giesing brings more than 35 years of pharmaceutical industry experience to Immunome and will be responsible for driving Immunome’s programs into clinical development. In April 2021, Immunome announced that it appointed Corleen Roche as its Chief Financial Officer. Ms. Roche is a highly accomplished CFO with over 30 years of industry experience to lead the company’s finance and corporate strategy functions.
Completed a $27 million private placement. In April 2021, the company announced a private placement of its common stock for gross proceeds to Immunome of approximately $27 million, before deducting placement agent commissions and other offering expenses. The company currently expects the proceeds from this private placement, together with its cash on hand, will be sufficient to fund its operations through fiscal year 2022.
"I am thrilled with the significant progress that Immunome has made in the development of our product pipeline," said Purnanand Sarma, Ph.D., President and CEO of Immunome. "We are excited to welcome Dr. Giesing and Ms. Roche to the Immunome team as we move towards becoming a clinical stage biopharmaceutical company."

Financial Highlights

Research and development (R&D) expenses: R&D expenses for the three months ended March 31, 2021 were $2.0 million.
General and administrative (G&A) expenses: G&A expenses for the three months ended March 31, 2021 were $1.9 million.
Net loss: Net loss for the three months ended 2021 was $3.9 million.
Cash and cash equivalents: As of March 31, 2021, cash and cash equivalents totaled $36.3 million (excluding $27.0 million in proceeds from the private placement in April 2021).