On May 9, 2024 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies for the treatment of cancer and other serious diseases, reported financial results for the first quarter ended March 31, 2024 and provided a review of recent clinical and business highlights (Press release, Xencor, MAY 9, 2024, View Source [SID1234643020]).

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"We have focused our XmAb clinical pipeline and discovery activities on bispecific CD3 and CD28 T cell engagers, which continue to show clinical validation for their potential in treating patients with serious diseases. Our key clinical-stage oncology programs in solid tumors include XmAb819 (ENPP3 x CD3) in clear cell renal cell carcinoma, XmAb808 (B7-H3 x CD28) in prostate cancer and other cancers, and XmAb541 (CLDN6 x CD3) in ovarian cancer and other cancers, which are all now advancing in Phase 1 clinical studies. We plan to select our next T cell engager IND candidate later this year," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "Additionally, Xencor’s best-in-class Xtend antibody half-life extension technology continues to support improved outcomes for patients, with Ultomiris now approved in the U.S. for certain patients with NMOSD, and we are especially delighted by recently published results demonstrating an investigational antibody with Xtend was effective in preventing malaria."

Recent Clinical and Business Highlights
•First Patient Dosed in Phase 1 Study of XmAb541 (CLDN6 x CD3): XmAb541 is a bispecific antibody being developed for patients with CLDN6-positive tumors including advanced ovarian cancer. XmAb541 is designed to engage the immune system, activating T cells for highly potent and targeted killing of tumor cells expressing Claudin-6 (CLDN6), a tumor-associated antigen. Xencor’s XmAb 2+1 multivalent format used in XmAb541 enables greater selectivity for cells expressing CLDN6 over similarly structured Claudin family members, which may be expressed on normal tissue. The first patient was recently dosed in a Phase 1 dose-escalation study.

•FDA Approves Ultomiris (Alexion Pharmaceuticals, Inc.) for Adults with NMOSD: In March 2024, Ultomiris (ravulizumab-cwvz), which incorporates Xencor’s Xtend Fc Domain, was approved in the United States as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). Ultomiris is also approved for certain adults with NMOSD in Japan and the European Union (EU). As part of Xencor’s recent Ultomiris royalty monetization, the Company remains eligible for certain future royalties and milestone payments. Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

•Single Dose of Investigational Antibody with Xtend Confers Protection Against Malaria Infection: Results from a Phase 2 National Institutes of Health (NIH)-sponsored clinical trial published in the New England Journal of Medicine showed that a single dose of L9LS, an experimental monoclonal antibody that incorporates Xencor’s Xtend Fc Domain, was up to 77% effective in preventing malaria in children in Mali for six months, demonstrating the long duration of action that Xtend technology can provide.

•New Chief Financial Officer Appointed: Bart Cornelissen was appointed as Xencor’s senior vice president and chief financial officer. He was most recently vice president, corporate finance at Seagen Inc.
Financial Guidance: Based on current operating plans, Xencor expects to end 2024 with between $475 million and $525 million in cash, cash equivalents and marketable debt securities, and to have cash to fund research and development programs and operations into 2027.
Financial Results for the First Quarter Ended March 31, 2024
Cash, cash equivalents and marketable debt securities totaled $646.7 million as of March 31, 2024, compared to $697.4 million on December 31, 2023.
Revenues for the first quarter ended March 31, 2024 were $12.8 million, compared to $19.0 million for the same period in 2023. Total revenues earned in the first quarter of 2024 included non-cash royalty revenue from Xencor’s Alexion and Morphosys/Incyte agreements, compared to milestone revenue earned from the J&J collaboration and royalties from the Alexion agreement in the first quarter of 2023.
Research and development expenses for the first quarter ended March 31, 2024 were $56.9 million, compared to $65.6 million for the same period in 2023. Decreased research and development spending for the first quarter of 2024 compared to 2023 reflects changes in spending across multiple clinical-stage programs and wind-down costs on terminated programs.
General and administrative expenses for the first quarter ended March 31, 2024 were $13.8 million and were in line with $14.2 million for the same period in 2023.
Other expense, net, for the first quarter ended March 31, 2024 was $10.8 million, compared to $0.02 million for the same period in 2023. Increased other expense for the first quarter of 2024 compared to 2023 reflects impairment charge on equity investments, partially offset by interest income earned on investments and unrealized gain on equity investments.

Non-cash, stock-based compensation expense for the first quarter ended March 31, 2024 was $11.4 million, compared to $12.6 million for the same period in 2023.

Net loss for the first quarter ended March 31, 2024 was $68.0 million, or $(1.11) on a fully diluted per share basis, compared to $60.8 million, or $(1.02) on a fully diluted per share basis, for the same period in 2023.

The total shares outstanding were 61,634,685 as of March 31, 2024, compared to 60,381,600 as of March 31, 2023.
Upcoming Investor Conferences
Company management will participate at multiple upcoming investor conferences:
•RBC Capital Markets Global Healthcare Conference
Date: Tuesday, May 14, 2024
Presentation Time: 2:05 p.m. ET / 11:05 a.m. PT
Location: New York City
•BofA Securities Health Care Conference
Date: Wednesday, May 15, 2024
Presentation Time: 4:40 p.m. ET / 1:40 p.m. PT
Location: Las Vegas

Live webcasts of the presentations will be available under "Events & Presentations" in the Investors section of the Company’s website located at www.xencor.com. Replays of the events will be available on the Xencor website for at least 30 days following the presentations.

On May 9, 2024 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended March 31, 2024, and provided a corporate update (Press release, Tempest Therapeutics, MAY 9, 2024, View Source [SID1234643019]).

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"The positive data and mechanistic analysis presented in the first quarter build on the positive preclinical and clinical data package for TPST-1120, further confirming and reinforcing our excitement about the potential of TPST-1120 in liver and kidney cancers, as well as other indications, and our confidence in the program as it moves closer to a pivotal Phase 3 study in first-line HCC," said Stephen Brady, president and chief executive officer of Tempest.

Recent Highlights

TPST-1120 (clinical PPARα antagonist):
Reported new preclinical data at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating that TPST-1120 reduces kidney cancer (RCC) growth as a monotherapy, while also showing increased inhibition when combined with frontline chemotherapy and immunotherapy. These data further support the clinical benefit observed in the TPST-1120 Phase 1 data presented in an oral presentation at ASCO (Free ASCO Whitepaper) 2022.
Published positive data from Phase 1 Trial of TPST-1120 in patients with advanced solid tumors in the Journal of Cancer Research Communications. Data showed that TPST-1120 demonstrated clinical activity, including tumor shrinkage, even in PD-1 inhibitor-refractory and immune-compromised cancers, and was well tolerated both as monotherapy and in combination with nivolumab. These data complement the positive Phase 1b/2 data reported in October 2023 from a global randomized study of TPST-1120 in combination with atezolizumab and bevacizumab in first-line patients with advanced HCC.
Presented new preclinical data showing potent anti-tumor activity in several cancer models treated with TPST-1120 alone or with immune checkpoint inhibitors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2024 Spring Scientific Meeting. The presentation also covered experimental results that corroborated clinical biomarker data from patients with advanced solid tumor cancers treated in the Phase 1 clinical trial of TPST-1120 in multiple solid tumor indications, which showed statistically significant, exposure-dependent elevations in expression levels of multiple immune-related genes, and patients exhibiting objective responses displayed increased circulating free fatty acids (FFA), both of which are in-line with the proposed TPST-1120 mechanism of action.
Potential Future Milestones

TPST-1120 (clinical PPARα antagonist)
Expect to announce updated data from the ongoing randomized study in first-line HCC patients in 2024.
Plan to advance TPST-1120 into a registrational Phase 3 study in first-line HCC patients, subject to obtaining feedback from the FDA.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist)
Plan to advance TPST-1495 into a Phase 2 study in patients with Familial Adenomatous Polyposis ("FAP") in 2024 under the auspices of the Cancer Prevention Clinical Trials Network and funded by the National Cancer Institute ("NCI") Division of Cancer Prevention, subject to final approval of NCI.
Expect to report data from the combination arm at the two highest TPST-1495 doses in patients with advanced endometrial cancer, where prostaglandin signaling is implicated, in 2024.
Financial Results

First Quarter 2024

Tempest ended the quarter with $32.3 million in cash and cash equivalents, compared to $39.2 million on December 31, 2023.
Net loss and net loss per share for the quarter ended March 31, 2024, were $7.9 million and $0.36, respectively, compared to $7.6 million and $0.55, respectively, for the same period in 2023.
Research and development expenses for the quarter were $4.3 million compared to $4.7 million for the same period in 2023. The $0.4 million decrease was primarily due to a decrease in costs incurred from contract research organizations and third-party vendors.
General and administrative expenses for the quarter were $3.6 million compared to $2.9 million for the same period in 2023. The $0.7 million increase was primarily due to share-based compensation expenses and consulting services.
Based on its current cash and operating plan, Tempest expects to have sufficient resources to fund operations into the second quarter of 2025.

On May 9, 2024 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported financial results for the first quarter of 2024 and provided a business update (Press release, Viracta Therapeutics, MAY 9, 2024, View Source [SID1234643018]).

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"Our near-term goal is to address the high unmet medical needs of patients living with relapsed or refractory EBV-positive PTCL by advancing Nana-val through regulatory approval as quickly as possible," said Mark Rothera, President and Chief Executive Officer of Viracta. "Topline results from Stage 1 of the ongoing pivotal Phase 2 NAVAL-1 trial provided a strong signal of efficacy with a generally well-tolerated safety profile that were consistent with outcomes from the prior Phase 1b/2 study of Nana-val. The observed objective and complete response rates of Nana-val far exceeded the nanatinostat monotherapy arm, further validating the combined mechanism of action of Nana-val treatment. We look forward to sharing additional data from Stage 1, which continue to mature, as well as initial results from Stage 2 in the third quarter of 2024. We are encouraged by the growing data from the NAVAL-1 trial that underscore the benefit of Nana-val, our first-in-class, all-oral combination treatment regimen, and plan to engage with the FDA on a potential accelerated approval pathway in mid-2024."

Clinical Trial Updates and Anticipated Milestones

Pivotal Phase 2 NAVAL-1 trial of Nana-val (nanatinostat in combination with valganciclovir) in patients with relapsed or refractory (R/R) Epstein-Barr virus-positive (EBV+) lymphoma

Clinical Trial Updates:

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Presented positive topline Stage 1 data from both arms of the R/R EBV+ peripheral T-cell lymphoma (PTCL) cohort, patients randomized to either nanatinostat monotherapy (n=10) or to nanatinostat in combination with valganciclovir (Nana-val, n=10), at the 2024 Joint Annual Congress of Taiwan Society of Blood and Marrow Transplantation and The Hematology Society of Taiwan.

o
As of the February 7, 2024 data cutoff date, Nana-val (nanatinostat in combination with valganciclovir) demonstrated greater efficacy than nanatinostat alone and was generally well-tolerated. The median duration of response (DoR) continues to mature.
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In the Nana-val arm, the objective response rate (ORR) was 50% and the complete response rate (CRR) was 20% in the intent-to-treat population, while the ORR was 71% and the CRR was 29% in the efficacy-evaluable population.
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In the nanatinostat monotherapy arm, the ORR and CRR were 10% and 0% in the intent-to-treat population, while the ORR was 13% in the efficacy-evaluable population.
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Data also showed that Nana-val was generally well tolerated, with the most common treatment-related adverse events in both treatment arms of thrombocytopenia, anemia, fatigue, decreased appetite, nausea, diarrhea, and weight loss.
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These adverse events were primarily mild to moderate in severity and generally manageable or reversible.
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The type, frequency, and severity of these adverse events were generally consistent with those from over 135 patients with R/R EBV+ lymphomas who have been treated with Nana-val to date.
Anticipated 2024 Milestones:

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Engage with U.S. Food and Drug Administration (FDA) in mid-2024 to align on requirements for accelerated approval.
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Enroll patients into the post-Phase 2 expansion cohort to support potential accelerated approval.
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Present Stage 1 + Stage 2 data (n=21) in the R/R EBV+ PTCL cohort in patients treated with Nana-val in the third quarter of 2024.
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Report Stage 1 data from patients with R/R EBV+ diffuse large B-cell lymphoma (DLBCL) and R/R EBV+ post-transplant lymphoproliferative disorder (PTLD) by year-end 2024.

Phase 1b/2 trial of Nana-val in patients with recurrent/metastatic (R/M) EBV+ nasopharyngeal carcinoma (NPC) and other advanced EBV+ solid tumors (Study 301)

Clinical Trial Updates:

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Completed enrollment of the sixth dose cohort from the Phase 1b dose escalation portion of the trial evaluating the novel split daily dosing (SDD) regimen in patients with R/M EBV+NPC.
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Started enrolling patients into the seventh dose cohort also evaluating the SDD regimen in the Phase 1b dose escalation portion of the trial.
Anticipated 2024 Milestones:

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Determine the recommended Phase 2 dose (RP2D) in the second half of 2024.
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Initiate a dose-optimization cohort to confirm the RP2D as part of the study’s Phase 2 expansion by year-end 2024.

Business Updates

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Engaged with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) on the extension of the NAVAL-1 trial into Japan.
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At a recent meeting, the PMDA endorsed the enrollment of patients in Japan into the NAVAL-1 trial without a preceding Japanese Phase 1 safety/pharmacokinetics study.
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Viracta plans to engage with the PMDA in the second half of 2024 to align on a potential approval pathway for Nana-val in R/R EBV+PTCL.

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The PMDA has granted full approval for R/R PTCL drugs based on a primary ORR endpoint supported by DoR and safety in single-arm studies.

First Quarter 2024 Financial Results

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Cash position – Cash, cash equivalents, and short-term investments totaled approximately $39.6 million as of March 31, 2024, which Viracta expects will be sufficient to fund operations late into the first quarter of 2025.
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Research and development expenses –Research and development expenses were approximately $10.0 million for the three months ended March 31, 2024, compared to approximately $7.6 million for the three months ended March 31, 2023. The increase in research and development expenses was largely due to a non-cash adjustment for insurance costs related to the February 2021 reverse merger with Sunesis Pharmaceuticals of $1.8 million, as well as increases in costs incurred to support the advancement and expansion of our clinical development programs, including incremental costs to support NAVAL-1, our pivotal Phase 2 trial of Nana-val in patients with R/R EBV+ lymphomas.
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General and administrative expenses – General and administrative expenses were approximately $3.9 million for the three months ended March 31, 2024, compared to $4.6 million for the same period in 2023. The decrease in general and administrative expenses was largely due to lower corporate liability insurance premiums and legal costs.
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Net loss – Net loss was approximately $9.1 million, or $0.23 per share (basic and diluted), for the quarter ended March 31, 2024, compared to a net loss of $12.2 million, or $0.32 per share (basic and diluted), for the same period in 2023. This change was primarily the result of $5.0 million of other income received related to the monetization of a pre-commercialization, event-based milestone from Day One Biopharmaceuticals, Inc. in March 2024, partially offset by the non-cash adjustment for insurance costs related to the February 2021 merger of $1.8 million.

About the NAVAL-1 Trial

NAVAL-1 (NCT05011058) is a global, multicenter, clinical trial of Nana-val in patients with relapsed or refractory (R/R) Epstein-Barr virus-positive (EBV+) lymphoma. This Phase 2 trial employs a Simon two-stage design where, in Stage 1, participants are enrolled into one of three indication cohorts based on EBV+ lymphoma subtype. If two objective responses are achieved within a lymphoma subtype in Stage 1 (n=10), then additional patients will be enrolled in Stage 2 for a total of 21 patients. EBV+ lymphoma subtypes demonstrating promising antitumor activity in Stage 2 may be further expanded following discussion with regulators to potentially support registration.

About the Phase 1b/2 Study of Nana-val in Patients with Advanced EBV+ Solid Tumors (Study 301)
This Phase 1b/2 trial (NCT05166577) is an open-label, multinational clinical trial evaluating Nana-val alone and in combination with pembrolizumab. The Phase 1b dose escalation part is designed to evaluate safety and to select the recommended Phase 2 dose (RP2D) of Nana-val in patients with recurrent or metastatic (R/M) Epstein-Barr virus-positive (EBV+) nasopharyngeal carcinoma (NPC). Along with the U.S. Food and Drug Administration’s Project Optimus initiative, at the start of Phase 2, up to 40 patients with R/M EBV+ NPC will be randomized to receive either the RP2D or a dose level below the RP2D in a dose-optimization cohort. Once the RP2D has been confirmed, up to 60 patients with R/M EBV+ NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to further evaluate antitumor activity, safety and tolerability, pharmacokinetics, and potential pharmacodynamic biomarkers. Additionally, patients with other advanced EBV+ solid tumors will be enrolled to receive Nana-val at the RP2D in a Phase 1b dose expansion cohort.

About Nana-val (Nanatinostat and Valganciclovir)

Nanatinostat is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which are key to inducing viral genes that are epigenetically silenced in Epstein-Barr virus (EBV)-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed or refractory (R/R) EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 clinical trial in patients with recurrent or metastatic (R/M) EBV+ NPC and other advanced EBV+ solid tumors.

About Peripheral T-Cell Lymphoma

T-cell lymphomas comprise a heterogeneous group of rare and aggressive malignancies, including peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) and angioimmunoblastic T-cell lymphoma (AITL). There are approximately 5,600 newly diagnosed T-cell lymphoma patients and approximately 2,600 newly diagnosed PTCL-NOS and AITL patients in the U.S. annually. Approximately 70% of these patients are either refractory to first-line therapy, or eventually experience relapse of their disease. Clinical trials are currently recommended for all lines of PTCL therapy, and most patients with R/R PTCL have poor outcomes, with median progression-free survival and median overall survival times reported to be 3.7 and 6.5 months, respectively. Approximately 40% to 65% of PTCL is associated with EBV, the incidence of EBV+ PTCL varies by geography, and reported outcomes for patients with EBV+ PTCL are inferior to those whose disease is EBV-negative. There is no approved targeted treatment specific for EBV+ PTCL, and therefore this represents a high unmet medical need.

About EBV-Associated Cancers

Approximately 90% of the world’s adult population is infected with EBV. Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of cells for the duration of the patient’s life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV-positive (EBV+) lymphomas. EBV is estimated to be associated with approximately 2% of the global cancer burden including lymphoma, nasopharyngeal carcinoma (NPC), and gastric cancer.

On May 9, 2024 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported financial results for the first quarter ended March 31, 2024, and provided corporate updates (Press release, Sensei Biotherapeutics, MAY 9, 2024, View Source [SID1234643017]).

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"Building upon our initial data for SNS-101, which we believe support a favorable and potentially best-in-class clinical safety and PK profile, we now look forward to sharing topline dose escalation data at ASCO (Free ASCO Whitepaper) in June and initial dose expansion data in the fourth quarter of this year," said John Celebi, President and Chief Executive Officer. "Looking ahead, we are squarely focused on advancing patient enrollment in the dose expansion cohorts, which are intended to generate additional supportive data and optimize the Phase 2 trial design for SNS-101. With cash runway into the fourth quarter of 2025, we believe we are in a strong position to build on both our clinical momentum on behalf of the patients we aim to serve and our scientific advances, as illustrated by our recent publication in Nature Communications."

Highlights and Milestones

SNS-101

SNS-101 is a conditionally active antibody harnessing the acidic tumor microenvironment to selectively target the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation). VISTA is implicated in numerous cancer indications and its expression correlates with low survival rates. Sensei is conducting a multi-center Phase 1/2 clinical trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SNS-101 as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in patients with advanced solid tumors. Recent updates include:

Sensei has completed enrollment in the dose escalation portion of the Phase 1/2 clinical trial of SNS-101, enrolling 16 patients in the monotherapy arm and 18 patients in the combination arm.
In the monotherapy dose escalation arm, patients have cleared all planned dosing cohorts of 0.3, 1, 3, 10, and 15 mg/kg.
In the combination dose escalation arm, patients have cleared all planned dosing cohorts of 3, 10, and 15 mg/kg of SNS-101 plus Libtayo.
There have been no dose limiting toxicities (DLT) observed across both the monotherapy and the combination arms.
Sensei will present topline clinical data from the monotherapy and combination dose escalation portion of its Phase 1/2 trial of SNS-101 in a poster presentation at the upcoming 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place in Chicago, IL, from May 31 – June 4, 2024.
Title: Initial results from a first-in-human phase 1 study of SNS-101 (pH-selective anti-VISTA antibody) alone or in combination with cemiplimab in patients with advanced solid tumors
Presenter: Dr. Shiraj Sen
Session Type: Poster Session
Session Title: Developmental Therapeutics—Immunotherapy
Date and Time: Saturday, June 1, 2024, at 9:00 a.m. – 12:00 p.m. CDT
Abstract Number: 2600
Patient enrollment is ongoing for both the monotherapy and combination dose expansion arms.
In the monotherapy dose expansion arm, Sensei is enrolling patients with microsatellite stable (MSS) colorectal cancer (CRC) at a dose level of 15 mg/kg.
In the combination dose expansion arm, Sensei is enrolling patients with MSS CRC, Head and Neck (H&N) cancer, non-small cell lung cancer (NSCLC), and melanoma at a dose level of 15 mg/kg plus Libtayo.
The solid tumor types were selected to focus the cancer indications on a basket of more commonly occurring histologies, including both "hot" (NSCLC, H&N, Melanoma) and "cold" (CRC) tumors where the company believes SNS-101 has potential to provide clinical benefit based on VISTA biology and supporting preclinical data. Additional tumor types and doses may be considered for both the monotherapy and combination expansion arms.
The Company expects to report initial data from the dose expansion cohorts and hold an end-of-Phase 1 meeting with the FDA by the end of 2024.
In April 2024, Sensei published a peer-reviewed research paper in Nature Communications describing the mechanism of action of SNS-101 selectively targeting the active form of VISTA within the tumor microenvironment.
In March 2024, the company presented SNS-101 data at Keystone Symposia’s Cancer Immunotherapy: Beyond Immune Checkpoint Blockade and Overcoming Resistance.
In February, Sensei presented at the 10th Annual Immuno-Oncology 360 Conference (IO360).
Corporate Updates

In January 2024, Sensei announced the appointment of Ron Weitzman, M.D., F.A.C.P., as part-time Chief Medical Officer.
In January 2024, Sensei announced a realignment of its resources to fully support the Phase 1/2 clinical trial of SNS-101. As a result, Sensei has paused IND-enabling work on its preclinical-stage TMAb programs, including SNS-102 (VSIG4), SNS-103 (CD39) and SNS-201 (VISTAxCD28). Preclinical work to characterize selected lead antibodies, including their mechanisms of action, and target biology is expected to continue throughout 2024.
As a result of this realignment of resources Sensei’s cash runway now extends into the fourth quarter of 2025.
First Quarter 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $58.1 million as of March 31, 2024, as compared to $65.8 million as of December 31, 2023. The decrease is due to cash used to fund operations. Sensei expects its current cash balance to fund operations into the fourth quarter of 2025.

Research and Development (R&D) Expenses: R&D expenses were $4.9 million for the quarter ended March 31, 2024, compared to $5.2 million for the quarter ended March 31, 2023. The decrease in R&D expenses was primarily attributable to lower manufacturing-related expenses and lower costs related to preclinical research, primarily offset by higher expenses associated with clinical trials.

General and Administrative (G&A) Expenses: G&A expenses were $3.8 million for the quarter ended March 31, 2024, compared to $5.8 million for the quarter ended March 31, 2023. The decrease in G&A expense was primarily attributable to decreased cost for external professional services.

Net Loss: Net loss was $8.0 million for the quarter ended March 31, 2024, compared to $10.2 million for the quarter ended March 31, 2023.

On May 9, 2024 Royalty Pharma plc (Nasdaq: RPRX) reported financial results for the first quarter of 2024 and confirmed full year 2024 guidance for Portfolio Receipts (Press release, Royalty Pharma , MAY 9, 2024, View Source [SID1234643016]).

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"We continued to execute on our strategy in the first quarter of 2024 and are on track to deliver our full year guidance" said Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer. "We achieved double-digit growth in Royalty Receipts driven by the strength of our diversified portfolio. With today’s transaction to acquire royalties on Sanofi’s frexalimab, a therapy with multi-blockbuster potential, we’ve increased our development-stage portfolio by five-fold to 15 medicines since our IPO. Looking ahead, 2024 is expected to be an event-rich year for our expanding development-stage pipeline with multiple potential regulatory and clinical events. Furthermore, we continue to be highly active in assessing attractive new royalty opportunities, reflecting the strong demand for capital to fund the ongoing wave of healthcare innovation. Based on the powerful fundamental tailwinds underpinning our business, we remain highly confident in our ability to deliver attractive, long-term compounding growth."

Strong Royalty Receipts growth; Portfolio Receipts growth impacted by a high base of comparison

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Royalty Receipts grew 14% to $705 million, driven by strong portfolio performance, including royalties on Evrysdi, Trelegy and the cystic fibrosis franchise.

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Portfolio Receipts declined to $717 million from $1,131 million, reflecting a $475 million Biohaven-related milestone payment received in the first quarter of 2023.

Capital Deployment of approximately $670 million, including cash expected to be paid for frexalimab

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Announced transaction to acquire royalties on Sanofi’s frexalimab, a potential multi-blockbuster in Phase 3 development for multiple sclerosis, for approximately $525 million including estimated transaction costs.

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Financial capacity of >$3.5 billion (including cash and leverage capacity) to execute on value-creating transactions.

Development-stage portfolio growing to 15 therapies with potential significantly >$1 billion in peak royalties

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11 of 15 therapies are in Phase 3 development or undergoing regulatory review.

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Key events expected over next year: FDA filings for aficamten (cardiovascular), pelabresib (myelofibrosis); pivotal study results for seltorexant (depression), Tremfya (Crohn’s disease); FDA action date for KarXT (schizophrenia).

Financial guidance for full year 2024 (excludes contribution from future transactions)

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Royalty Pharma expects 2024 Portfolio Receipts to be between $2,600 million and $2,700 million.

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2024 Portfolio Receipts guidance includes expected growth in Royalty Receipts of 5% to 9%.Financial & Liquidity Summary

Three Months Ended March 31,
(unaudited)
($ and shares in millions) 2024 2023 Change
Portfolio Receipts

717 1,131 (37 )%
Net cash provided by operating activities

665 1,034 (36 )%
Adjusted EBITDA (non-GAAP)*

656 1,044 (37 )%
Portfolio Cash Flow (non-GAAP)*

584 977 (40 )%
Weighted average Class A ordinary shares outstanding – diluted

597 607 (2 )%

*
See "Liquidity and Capital Resources" section. Adjusted EBITDA and Portfolio Cash Flow are non-GAAP liquidity measures calculated in accordance with the credit agreement.

Portfolio Receipts Highlights

Three Months Ended March 31,
(unaudited)
($ in millions) 2024 2023 Change
Products:

Marketers: Therapeutic Area:
Cystic fibrosis franchise

Vertex Rare disease 218 197 11 %
Trelegy

GSK Respiratory 71 48 46 %
Tysabri

Biogen Neuroscience 69 71 (2 )%
Imbruvica

AbbVie, J&J Cancer 50 57 (12 )%
Evrysdi

Roche Rare disease 45 18 156 %
Promacta

Novartis Hematology 43 41 4 %
Xtandi

Pfizer, Astellas Cancer 41 36 14 %
Tremfya

Johnson & Johnson Immunology 36 32 15 %
Cabometyx/Cometriq

Exelixis, Ipsen, Takeda Cancer 18 16 14 %
Trodelvy

Gilead Cancer 10 7 57 %
Erleada

Johnson & Johnson Cancer 9 6 57 %
Orladeyo

BioCryst Rare disease 9 7 28 %
Spinraza

Biogen Rare disease 7 —  n/a
Nurtec ODT/Zavzpret

Pfizer Neuroscience 6 4 40 %
Other products(5)

73 78 (6 )%

Royalty Receipts

705 616 14 %
Milestones and other contractual receipts

12 515 (98 )%

Portfolio Receipts

717 1,131 (37 )%

Amounts shown in the table may not add due to rounding.

Portfolio Receipts were $717 million in the first quarter of 2024, a decrease of 37% compared to $1,131 million in the same period of 2023. The decrease was attributable to a high base of comparison in the first quarter of 2023 in milestones and other contractual receipts, which reflected a $475 million Zavzpret milestone payment and a $29 million Airsupra payment.

Royalty Receipts increased 14% to $705 million, primarily driven by strong growth in Trelegy and the cystic fibrosis franchise, and incremental royalties acquired on Evrysdi in October 2023.

Liquidity and Capital Resources

Royalty Pharma’s liquidity and capital resources are summarized below:

As of March 31, 2024, Royalty Pharma had cash and cash equivalents of $843 million and total debt with principal value of $6.3 billion.

Liquidity Summary

Three Months Ended March 31,
(unaudited)
($ in millions) 2024 2023
Portfolio Receipts

717 1,131
Payments for operating and professional costs

(61 ) (87 )

Adjusted EBITDA (non-GAAP)

656 1,044
Interest paid, net

(73 ) (67 )

Portfolio Cash Flow (non-GAAP)

584 977
 Amounts shown in the table may not add due to rounding.

•
Adjusted EBITDA (non-GAAP) was $656 million in the first quarter of 2024. Adjusted EBITDA is calculated in accordance with the credit agreement as Portfolio Receipts minus payments for operating and professional costs.

•
Portfolio Cash Flow (non-GAAP) was $584 million in the first quarter of 2024. Portfolio Cash Flow is calculated in accordance with the credit agreement as Adjusted EBITDA minus interest paid or received, net. This measure reflects the cash generated by Royalty Pharma’s business that can be redeployed into value-enhancing royalty acquisitions, used to repay debt, returned to shareholders through dividends or share purchases, or utilized for other discretionary investments.

Royalty Pharma has provided a reconciliation of each non-GAAP measure to the most directly comparable GAAP financial measure, net cash provided by operating activities, in Table 4.

Royalty Pharma announced new transactions of up to $94 million in the first quarter of 2024, which include upfront payments and potential future milestones. Royalty Pharma is also providing an aggregate amount for Capital Deployment, which reflects cash payments during the period for new and previously announced transactions. Capital Deployment was $93 million in the first quarter of 2024, consisting primarily of the $49 million upfront payment for ecopipam and $36 million in research and development funding support for TEV-’749. Additionally, in April 2024, Royalty Pharma made a $50 million milestone payment to Arrowhead related to olpasiran. In May 2024, Royalty Pharma announced a transaction to acquire royalties and milestones on frexalimab owned by ImmuNext for approximately $525 million in cash including estimated transaction costs.

The table below details Capital Deployment by category:

Capital Deployment

Three Months Ended March 31,
($ in millions) 2024 2023
Acquisitions of financial royalty assets

(86 ) (602 )
Development-stage funding payments – ongoing

(1 ) (1 )
Milestone payments

—  (12 )
Investments in equity method investees

(7 ) (4 )
Contributions from legacy non-controlling interests – R&D

0 0

Capital Deployment

(93 ) (618 )
 Amounts may not add due to rounding.

Recent Transactions

During 2024, Royalty Pharma announced new transactions of up to $619 million. Announced transactions amount reflects the entire amount of capital committed for new transactions during the year, including potential future milestones.

Recent transactions include:

•
In January 2024, Royalty Pharma acquired a royalty interest in ecopipam for an upfront payment of $49 million and up to $44 million in milestone payments contingent on the achievement of certain regulatory milestones. Ecopipam is in Phase 3 development by Emalex Biosciences for the treatment of Tourette Syndrome.

•
In May 2024, Royalty Pharma announced a transaction to acquire royalties and milestones on frexalimab owned by ImmuNext for approximately $525 million in cash including estimated transaction costs. ImmuNext is entitled to royalties on frexalimab, which is a first-in-class, second generation anti-CD40 ligand monoclonal antibody in development by Sanofi. Frexalimab is being evaluated in Phase 3 clinical studies for the treatment of multiple sclerosis and is in Phase 2 clinical studies for systemic lupus erythematosus and Type 1 Diabetes.

The information in this section should be read together with Royalty Pharma’s reports and documents filed with the SEC at www.sec.gov and the reader is also encouraged to review all other press releases and information available in the Investors section of Royalty Pharma’s website at www.royaltypharma.com.

Key Developments Relating to the Portfolio

The key developments related to Royalty Pharma’s royalty interests are discussed below based on disclosures from the marketers of the products.

TEV-‘749 In May 2024, Teva Pharmaceuticals announced positive efficacy results from its Phase 3 trial evaluating TEV-‘749, a once-monthly subcutaneous long-acting injection of olanzapine, in adult patients with schizophrenia. Results demonstrated that TEV-‘749 met its primary endpoint as measured by a change in the Positive and Negative Syndrome Scale (PANSS) total score from baseline after eight weeks compared to placebo. Additionally, no cases of Post-injection Delirium/Sedation Syndrome (PDSS) have been reported to date, after administration of approximately 80% of the minimal target injection number.
Cystic fibrosis franchise
In May 2024, Vertex announced that it submitted a New Drug Application and Marketing Authorization Application for the new triple combination therapy to the Food & Drug Administration and the European Medicines Agency, respectively, for approval. This follows positive Phase 3 results for the new triple combination therapy in February 2024.

In April 2024, Vertex announced that the European Commission had granted approval for the label expansion of Kalydeco for the treatment of infants down to one month of age with cystic fibrosis (CF) who have certain mutations in the CF transmembrane conductance regulator gene.

Tremfya In May 2024, Johnson and Johnson announced positive Phase 3 results for Tremfya in patients with moderately to severely active Crohn’s disease with inadequate response/intolerance to conventional therapies and/or biologics. Johnson and Johnson submitted applications to the European Medicines Agency seeking to expand the Marketing Authorization Application for Tremfya to include ulcerative colitis and Crohn’s disease.
Xtandi
In April 2024, Astellas Pharma announced the European Commission approved a label extension for Xtandi as monotherapy or in combination with androgen deprivation therapy for the treatment of adult men with high-risk biochemical recurrent non-metastatic hormone-sensitive prostate cancer who are unsuitable for salvage-radiotherapy.

In January 2024, Pfizer announced that the European Commission had approved Talzenna (talazoparib), an oral poly ADP-ribose polymerase inhibitor, in combination with Xtandi, for the treatment of adult patients with metastatic castration-resistant prostate cancer in whom chemotherapy is not clinically indicated.

KarXT In March 2024, Bristol Myers Squibb announced that it completed its acquisition of Karuna. Bristol Myers acquired Karuna for $330 per share, for a total equity value of $14 billion. The New Drug Application for KarXT for the treatment of schizophrenia in adults was accepted for review by the FDA, with a Prescription Drug User Fee Act date of September 26, 2024.
Trontinemab In March 2024, Roche held a neurology update event in which it announced that in people with Alzheimer’s Disease, trontinemab demonstrated rapid and robust amyloid plaque reduction at relatively low doses compared with standard anti-Aß monoclonal antibodies. The sustained low Amyloid Related Imaging Abnormalities incidence and overall favorable safety and tolerability profile support further investigation.

Pelabresib In February 2024, Novartis announced that it had entered into an agreement to make a voluntary public takeover offer to acquire MorphoSys for €68 per share, for a total equity value of €2.7 billion. The closing is expected in the first half of 2024.
Trodelvy In January 2024, Gilead announced that the Phase 3 EVOKE-01 study evaluating Trodelvy compared to docetaxel did not meet its primary endpoint of overall survival in patients with previously treated metastatic non-small cell lung cancer.
2024 Financial Outlook

Royalty Pharma has provided guidance for full year 2024, excluding transactions and borrowings announced after the date of this release, as follows:

Provided May 9, 2024 Previous
Portfolio Receipts

$2,600 million to $2,700 million $2,600 million to $2,700 million
Payments for operating and professional costs

8% to 9% of Portfolio Receipts 8% to 9% of Portfolio Receipts
Interest paid

$160 million $160 million
The above Portfolio Receipts guidance includes expected Royalty Receipts growth of 5% to 9% in 2024.

Royalty Pharma’s full year 2024 guidance reflects a negligible estimated foreign exchange impact to Portfolio Receipts, assuming current foreign exchange rates prevail for 2024.

Total interest paid is based on the semi-annual interest payment schedule of Royalty Pharma’s existing notes and is anticipated to be approximately $160 million in 2024. Interest paid is anticipated to be approximately $79 million in the third quarter of 2024 with de minimis amounts being recorded in the second and fourth quarters of 2024. The projection assumes no additional debt financing in 2024, including no drawdown on the revolving credit facility. In the first quarter of 2024, Royalty Pharma collected interest of $6 million on its cash and cash equivalents, which partially offset interest paid.

Royalty Pharma today provides this guidance based on its most up-to-date view of its prospects. This guidance assumes no major unforeseen adverse events and excludes the contributions from transactions announced subsequent to the date of this press release. Furthermore, Royalty Pharma may amend its guidance in the event it engages in new royalty transactions which have a material near-term financial impact on the company.

Financial Results Call

Royalty Pharma will host a conference call and simultaneous webcast to discuss its first quarter 2024 results today at 8:00 a.m., Eastern Time. Please visit the "Investors" page of the company’s website at View Source to obtain conference call information and to view the live webcast. A replay of the conference call and webcast will be archived on the company’s website for at least 30 days.