Viracta Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

On March 7, 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 fourth quarter and full-year of 2023 and provided a business update (Press release, Viracta Therapeutics, MAR 7, 2024, View Source [SID1234640938]).

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"Nana-val is a first-in-class, all-oral combination treatment regimen that has entered late-stage development to target and treat EBV-associated cancers. Our near-term goal is to address the high unmet medical need of patients living with relapsed or refractory EBV-positive PTCL by advancing Nana-val in this lead indication through regulatory approval as quickly as possible," said Mark Rothera, President and Chief Executive Officer of Viracta. "We are pleased to have successfully completed patient enrollment across both Stage 1 and Stage 2 of the PTCL cohort in our pivotal NAVAL-1 trial. Building on this momentum, we anticipate reporting topline data from Stage 1 in the second quarter of 2024 and engaging with the FDA on a potential accelerated approval pathway in mid-2024."

Clinical Trial Updates and Anticipated Milestones

Pivotal 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:

Completed enrollment of Stage 1 in the R/R EBV+ PTCL cohort (in patients treated with nanatinostat with [n=10] or without [n=10] valganciclovir) in the fourth quarter of 2023.
Completed enrollment of Stage 2 in the R/R EBV+ PTCL cohort of patients treated with Nana-val (n=21, Stage 1 + Stage 2 patients) in the first quarter of 2024.
Amended protocol to additionally enable enrollment of second-line R/R EBV+ DLBCL patients and R/R EBV+ PTLD patients, including pediatric EBV+ PTLD patients ≥ 12 years of age.
Anticipated 2024 Milestones:

Present topline Stage 1 data from both arms of the R/R EBV+ PTCL cohort (in patients treated with nanatinostat with [n=10] or without [n=10] valganciclovir) in the second quarter of 2024, with an aim to clearly delineate the differentiation of Nana-val’s ‘kick and kill’ mechanism of action.
Engage with U.S. Food and Drug Administration (FDA) in mid-2024, to align on requirements for accelerated approval.
Enroll patients into the post-Phase 2 expansion cohort to support potential accelerated approval.
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.
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:

In December 2023, the FDA granted an orphan drug designation (ODD) to Nana-val for the treatment of NPC, the fifth ODD granted to Nana-val by the FDA, the seventh ODD for Nana-val globally, and the first ODD granted to Nana-val in EBV+ solid tumors.
Presented data at ESMO (Free ESMO Whitepaper) Asia Congress 2023 that confirmed partial responses without dose-limiting toxicities through the initial five dose cohorts, supporting continued dose escalation to potentially enhance Nana-val’s antitumor activity.
Completed enrollment of the sixth dose cohort of patients with R/M EBV+ NPC, evaluating the novel split daily dosing (SDD) regimen.
Anticipated 2024 Milestones:

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

Executed an amended license agreement with Day One Biopharmaceuticals
In March 2024, to receive non-dilutive proceeds of $5.0 million related to monetization of a pre-commercialization, event-based milestone from Day One Biopharmaceuticals, Inc.
Executed an amended Royalty Purchase Agreement with XOMA
Amended the Royalty Purchase Agreement with XOMA, modifying the economic value-share under the Royalty Purchase Agreement by which the Company has retained the right, under certain circumstances, to participate in a pre-commercialization event-based milestone up to $5.0 million.
Executed the second amendment under the SVB-Oxford Loan Facility
Amended the SVB-Oxford Loan Facility, providing for a modification of the loan amortization period and a pro-rata reduction in the prospective debt amortization schedule, in exchange for a partial prepayment of the term loan. Pursuant to the terms of the second amendment, the Company has agreed to remit a prepayment of $5.0 million toward the outstanding principal, plus a pro-rata portion of the final payment, by March 15, 2024. Under the terms of the amendment, principal amortization will be deferred between March 2024 and June 2024, totaling approximately $2.9 million. Amortization payments will recommence in July 2024, reflecting the prepayment and reducing prospective amortization payments in 2024 by approximately $3.3 million, in addition to significantly reducing interest owed over the term of the loan. There were no changes to the maturity date of the term loan, which is November 2026.
"We are pleased to have strengthened our balance sheet through the imminent receipt of $5 million in non-dilutive capital at this important time," said Dan Chevallard, Chief Operating Officer and Chief Financial Officer of Viracta. "The proceeds will be used to make a partial prepayment of our outstanding debt balance, while also enabling a concurrent amendment to our credit facility to avail ourselves of an additional interest-only period through June 2024, and further reduce future amortization and interest payments reflecting the prepayment. Pro forma for this prepayment, we will have reduced our debt balance by over 25% since year-end to $18.6M and anticipate ending 2024 with less than $15 million in debt outstanding. The totality of this coordinated set of transactions will extend our cash runway into mid-Q1 2025 and provides a meaningful aggregate cash impact to Viracta well in excess of the proceeds."

Fourth-Quarter and Full-Year 2023 Financial Results

Cash position – Cash, cash equivalents, and short-term investments totaled approximately $53.7 million as of December 31, 2023. Pro forma for the aforementioned business transactions, our cash runway to fund operations is extended into mid-Q1 2025.
Research and development expenses – Research and development expenses were approximately $9.4 million and $6.7 million for the three months ended December 31, 2023 and 2022, respectively. Research and development expenses increased to $33.4 million compared to $26.3 million for the years ended December 31, 2023 and 2022, respectively. The increase in research and development expenses in 2023 was primarily driven by increases in costs incurred to support the advancement and expansion of our clinical development programs, including incremental costs to support NAVAL-1, our pivotal trial of Nana-val in patients with R/R EBV+ lymphomas, and our Phase 1b/2 study of Nana-val in patients with advanced EBV+ solid tumors, as well as an increase in personnel-related costs.
General and administrative expenses – General and administrative (G&A) expenses were approximately $4.2 million and $4.9 million for the three months ended December 31, 2023 and 2022, respectively, compared to $17.3 million and $24.3 million for the years ended December 31, 2023 and 2022. The decrease in G&A expenses year over year was largely due to a one-time expense associated with the modification of certain equity awards totaling $5.6 million and $0.8 million in severance-related charges associated with the transition of the former Chief Executive Officer in 2022. The decrease over the comparative three-month period was primarily due to a decrease in share-based compensation expense and corporate liability insurance premiums.
Net loss – Net loss was approximately $13.8 million, or $0.35 per share (basic and diluted), for the quarter ended December 31, 2023, compared to a net loss of $10.3 million or $0.27 per share (basic and diluted), for the same period in 2022. Net loss was approximately $51.1 million, or $1.32 per share (basic and diluted), for the year ended December 31, 2023, compared to a net loss of $49.2 million or $1.30 per share (basic and diluted), for the same period in 2022.
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 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.

Werewolf Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

On March 7, 2024 Werewolf Therapeutics, Inc. (the "Company" or "Werewolf") (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer, reported a business update and announced financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Werewolf Therapeutics, MAR 7, 2024, View Source [SID1234640937]).

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"Werewolf made considerable progress in 2023, setting up 2024 as a year of execution across our pipeline," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "In the first half of this year, we plan to present additional clinical data from our Phase 1/1b clinical trial of WTX-124, including updated monotherapy data and initial combination data, which we anticipate will build on the promising signals of antitumor activity and improved therapeutic index that we observed in the data presented at SITC (Free SITC Whitepaper) last year. In addition, we plan to share data further demonstrating the performance of our platform in our preclinical programs at AACR (Free AACR Whitepaper). We also plan to share initial clinical data from WTX-330, our second clinical candidate, in the second quarter of 2024."

"Additionally, I would like to express my deep appreciation to Cindy Seidel-Dugan, Ph.D., Werewolf’s Chief Scientific Officer, who is retiring effective March 29, 2024, after a long and successful career in the biopharmaceutical industry. Cindy was a founding member of the Werewolf Executive Team and has been instrumental in establishing Werewolf’s innovative science, developing our INDUKINE technology, and shepherding our lead candidates through the discovery and IND-enabling process. We have benefited greatly from Cindy’s significant expertise, and the strong scientific leadership team she has built that will ensure continued success going forward. We wish Cindy the very best in her retirement."
Recent Highlights and Upcoming Milestones

WTX-124: a systemically delivered, conditionally activated Interleukin-2 (IL-2) INDUKINE molecule being developed as monotherapy and in combination with pembrolizumab in multiple solid tumor types.

•In November 2023, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 38th Annual Meeting, Werewolf presented first-in-human monotherapy data from the Phase 1/1b clinical trial of WTX-124. These preliminary data established proof of mechanism for WTX-124 and proof of concept for Werewolf’s INDUKINE design hypothesis.

•In the first half of 2024, Werewolf expects to present additional interim dose-escalation data from the monotherapy dose-escalation arm, nominate a recommended dose for expansion and initiate monotherapy dose expansion arms.
•Additionally, Werewolf continues to progress combination dose escalation cohorts of the Phase 1/1b clinical trial and plans to report initial clinical data from this arm in the first half of 2024.

WTX-330: a systemically delivered, conditionally activated Interleukin-12 (IL-12) INDUKINE molecule being developed in refractory and/or immunologically unresponsive tumors.

•Werewolf is progressing Study WTX-330×2101, its Phase 1, multi-center, open-label clinical trial evaluating WTX-330 as a monotherapy in patients with immunotherapy insensitive or resistant advanced or metastatic solid tumors or non-Hodgkin lymphoma. Werewolf plans to report initial data from the Phase 1 study in the second quarter of 2024.

Preclinical Portfolio: includes development candidates WTX-712 and WTX-518, INDUKINE molecules targeting IL-21 and IL-18, respectively, for treatment of cancer.

•Werewolf plans to present preclinical data from WTX-712 and WTX-518 at the 2024 AACR (Free AACR Whitepaper) Annual Meeting on antitumor activity in the delivery of IL-21 or IL-18, respectively, to the tumor microenvironment in mouse tumor models. These programs are currently progressing through IND-enabling work.

Abstract Number: 4078
Title: WTX-712, a conditionally active IL-21 INDUKINETM molecule, induces a strong anti-tumor phenotype through a differentiated mechanism
Session Date and Time: Tuesday, April 9, 2024, from 9:00 a.m. – 12:30 p.m.
Abstract Number: 4074
Title: Discovery of WTX-518, an IL-18 pro-drug that is conditionally activated within the tumor microenvironment and induces regressions in mouse tumor models
Session Date and Time: Tuesday, April 9, 2024, from 9:00 a.m. – 12:30 p.m.

Additional Updates:

•In January 2024, Werewolf appointed Michael Atkins, M.D., to its Board of Directors. Dr. Atkins has served as a member of Werewolf’s Scientific Advisory Board since August 2018 and has more than 30 years of experience in translational and clinical research, specializing in melanoma, kidney cancer, and cancer immunotherapy.

Financial Results for the Fourth Quarter and Full Year 2023:

•Cash position: As of December 31, 2023, cash and cash equivalents were $134.3 million, compared to $129.3 million as of December 31, 2022. The Company also had restricted cash and cash equivalents of $21.2 million and $1.2 million as of December 31, 2023 and December 31, 2022, respectively. Based on updated forecasting the Company now expects that its existing cash and cash equivalents at December 31, 2023, and gross proceeds of $17.7 million under the at-the-market sales facility received from January 1, 2024 through March 1, 2024, will be sufficient to fund its operational expenses and capital expenditure requirements through at least the second quarter of 2025.
•Collaboration revenue: Collaboration revenue was $1.5 million for the fourth quarter of 2023, compared to $7.3 million for the same period in 2022, and $19.9 million for the full year 2023, compared to $16.4 million for the same period in 2022. Collaboration revenue consists of revenue recognized from the Company’s licensing agreement with Jazz Pharmaceuticals (Jazz) and includes fixed payments received from Jazz, plus costs incurred for research services to be reimbursed by Jazz.

•Research and development expenses: Research and development expenses were $9.6 million for the fourth quarter of 2023, compared to $15.9 million for the same period in 2022. Research and development expenses were $41.8 million for the full year 2023, compared to $53.8 million for the full year 2022.
•General and administrative expenses: General and administrative expenses were $4.8 million for the fourth quarter of 2023, compared to $4.6 million for the same period in 2022. General and administrative expenses were $18.7 million for the full year 2023, compared to $18.7 million for the full year 2022.
•Net loss: Net loss was $12.0 million for the fourth quarter of 2023, compared to $11.9 million for the same period in 2022. Net loss was $37.4 million for the full year 2023, compared to $53.8 million for the full year 2022.

RAPT Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results

On March 7, 2024 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based therapeutics company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported financial results for the fourth quarter and year ended December 31, 2023 (Press release, RAPT Therapeutics, MAR 7, 2024, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-reports-fourth-quarter-and-full-year-2023 [SID1234640936]).

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"We are working diligently to lift the clinical hold on our Phase 2 trials of zelnecirnon in atopic dermatitis and asthma," said Brian Wong, M.D., Ph.D., President and Chief Executive Officer of RAPT Therapeutics. "Patient safety is our top priority. We expect to provide an update once we have defined a plan to move ahead and appreciate your patience during this process. Separately, our Phase 2 study of tivumecirnon in cancer is ongoing and we will be presenting an update at AACR (Free AACR Whitepaper) in April."

Financial Results for the Fourth Quarter and the Year Ended December 31, 2023

Fourth Quarter Ended December 31, 2023

Net loss for the fourth quarter of 2023 was $30.9 million, compared to $23.0 million for the fourth quarter of 2022.

Research and development expenses for the fourth quarter of 2023 were $26.8 million, compared to $19.5 million for the same period in 2022. The increase in research and development expenses was primarily due to higher development costs related to zelnecirnon, as well as increased expenses for personnel, lab supplies, consultants, facilities and stock-based compensation, partially offset by lower development costs related to tivumecirnon and early-stage programs.

General and administrative expenses for the fourth quarter of 2023 were $6.5 million, compared to $5.0 million for the same period in 2022. The increase in general and administrative expenses was primarily due to increases in expenses for personnel, stock-based compensation and facilities.

Year Ended December 31, 2023

Net loss for the year ended December 31, 2023 was $116.8 million, compared to $83.8 million in 2022.

Research and development expenses for the year ended December 31, 2023 were $101.0 million, compared to $67.1 million in 2022. The increase in research and development expenses was primarily due to higher development costs related to zelnecirnon, as well as increased expenses for personnel, lab supplies, consultants, facilities and stock-based compensation, partially offset by lower development costs related to tivumecirnon and early-stage programs.

General and administrative expenses for the year ended December 31, 2023 were $26.1 million, compared to $20.2 million in 2022. The increase in general and administrative expenses was primarily due to increases in expenses for personnel, stock-based compensation, facilities and professional services.

As of December 31, 2023, the Company had cash and cash equivalents and marketable securities of $158.9 million.

Poseida Therapeutics Provides Updates and Financial Results for the Fourth Quarter and Full Year 2023

On March 7, 2024 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported business updates and financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Poseida Therapeutics, MAR 7, 2024, View Source [SID1234640935]).

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"2024 looks to be a breakout year for Poseida as we build on the recent allogeneic BCMA clinical data presented at ASH (Free ASH Whitepaper), which demonstrated the potential of our high-TSCM allogeneic CAR-T therapies to offer effective, safe, and reliable treatment addressing unmet needs in multiple myeloma. We are able to achieve high levels of CAR-TSCM in our products because of our proprietary, nonviral technologies," said Kristin Yarema, Ph.D., President and Chief Executive Officer. "Over the coming months we are planning data readouts for each of our three clinical-stage CAR-T programs, all of which are manufactured at our own GMP facility and will discuss the latest progress of our gene therapy programs at our R&D Day in April."

Recent 2023 Accomplishments

Cell therapy


Presented positive data from the Phase 1 study of P-BCMA-ALLO1 in relapsed/refractory multiple myeloma (RRMM) at ASH (Free ASH Whitepaper): In December 2023, the Company presented data from its Phase 1 study of P-BCMA-ALLO1 at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. These early data support the Company’s belief that TSCM-rich allogeneic CAR-Ts have the potential to offer promising product response rates, safety profiles, and rapid patient access which if confirmed could provide differentiation from currently available therapies, including autologous CAR-T therapies. The study used product from 6 different CAR-T lots produced from 6 different donors with data demonstrating:

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82% ORR in deep clinical responses, including sCRs and MRD-negative patients from off-the-shelf, allogeneic BCMA-targeted CAR-T in heavily pretreated patients receiving adequate lymphodepletion.
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100% ORR in those patients who were not previously treated with a BCMA-targeted bispecific T-cell engaging antibody.
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Favorable emerging safety and reliability profile, with all (100%) intent-to-treat patients receiving therapy with no use of bridging chemotherapy or other anti-myeloma bridging therapies and low incidences of CRS and neurotoxicity observed (all ≤ Grade 2).
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Preliminary data suggest that allogeneic TSCM-rich CAR-T cells traffic to bone marrow, differentiate to cell-killing effector T cells and persist at least 6 weeks, supporting the hypothesis of cell persistence at tumor-relevant sites.
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8 of 9 responding patients still in response at data cut off.

Continued enrollment of P-MUC1C-ALLO1: The Company continued enrollment in the Phase 1 clinical trial of P-MUC1C-ALLO1 for solid tumors. Drawing from insights gained in allogeneic BCMA CAR-T, it is exploring various dosing approaches, including higher lymphodepletion, cell dose, and scheduling in the MUC1C program. Data from the allogeneic BCMA CAR-T program, presented at ASH (Free ASH Whitepaper), underscored the important role of cyclophosphamide conditioning dose in CAR-T cell expansion and persistence. Patients in arms receiving higher doses (500 mg/m2 and 1,000 mg/m2) demonstrated significantly elevated P-BCMA-ALLO1 levels compared to those in the 300 mg/m2 cohort. Consequently, the Company is assessing lymphodepleting conditioning regimens with doses exceeding 300 mg/m2 in the MUC1C program.

Advanced programs within hematologic malignancies at all stages, in partnership with Roche, throughout 2023:
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Previously announced the expected acceleration of achieving milestones, resulting in the receipt of a $30 million payment in the first quarter of 2024.
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Expanded the protocol and continued to enroll patients in the Phase 1 study of P-BCMA-ALLO1, incorporating lymphodepletion learnings from ASH (Free ASH Whitepaper) 2023.
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Continued site activation activities and initiation of enrollment for P-CD19CD20-ALLO1, the Company’s first dual, allogeneic CAR-T for B-cell malignancies.
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Advanced research programs optioned to Roche (P-BCMACD19-ALLO1 for multiple myeloma and P-CD70-ALLO1 for AML).
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Developed and deployed manufacturing platform improvements to all programs that delivered higher and more consistent levels of cell expansion.
Gene therapy


Presented proof-of-principle data supporting P-FVIII-101 as a potential treatment option for Hemophilia A at ASH (Free ASH Whitepaper): New preclinical data presented at ASH (Free ASH Whitepaper) demonstrated the capabilities of the Company’s non-viral approach in providing stable Factor VIII (FVIII) transgene expression through genomic integration. Additionally, the data highlighted re-dosing, or titrating to efficacy, along with favorable tolerability data for this approach, including 52-week durability data in an adult Hemophilia A mouse model supporting sustained and robust expression.

Anticipated Milestones


P-BCMA-ALLO1: The Company plans to present data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in San Diego. At AACR (Free AACR Whitepaper), the Company will present data on a subset of recently enrolled patients refractory to initial BCMA targeting therapy. The poster presentation, titled "Clinical Activity of P-BCMA-ALLO1, a B-cell Maturation Antigen (BCMA) Targeted Allogeneic Chimeric Antigen Receptor T-cell (CAR-T) Therapy, in Relapsed Refractory Multiple Myeloma (RRMM) Patients (pts) Following Progression on Prior BCMA Targeting Therapy," is anticipated to take place on April 8, 2024, 9:00 AM to 12:30 PM PT. Subject to coordination with Roche, the Company plans to provide an additional clinical update on this program at a scientific meeting in the second half of 2024.

P-MUC1C-ALLO1: The Company plans to review initial clinical findings from the Phase 1 clinical trial in solid tumors at AACR (Free AACR Whitepaper) related to cell expansion and lymphodepletion regimens. The poster presentation, titled "Solid Tumor Patients Require Higher Cyclophosphamide Dose than Multiple Myeloma Patients to Achieve Adequate Lymphodepletion (LD) Necessary to Enable Allogeneic CAR-T Expansion," is anticipated to take place on April 8, 2024, 9:00 AM to 12:30 PM PT. In addition to the AACR (Free AACR Whitepaper) presentation, the Company plans to present a more fulsome clinical update at an appropriate forum in the second half of 2024.

P-CD19CD20-ALLO1: Following the initiation of the Phase 1 trial targeting B-cell malignancies in late 2023, the Company expects to provide an interim data update in the second half of 2024, subject to coordination with Roche.

P-PSMA-ALLO1: In January 2024, Poseida announced that it expects to advance its next allogeneic solid tumor program, P-PSMA-ALLO1 in prostate cancer, into IND-enabling studies incorporating the progress and learnings of its previous autologous study and recent advancements in its allogeneic platform in 2024.
The Company believes its TSCM-rich CAR-T platform and associated proprietary technologies have strong potential to deliver new therapeutic approaches in autoimmune disease. The Company is actively developing a strategy in inflammation and autoimmune disease and will provide an update at an appropriate time.

Other Operational Updates and Upcoming Events

Poseida R&D Days

The Company plans to host two R&D Days in 2024 focusing on gene therapy and cell therapy respectively.

The Company plans to host its gene therapy specific R&D Day on April 17, 2024, which will feature presentations from gene therapy management as well as key opinion leaders. The event will highlight the Company’s progress across its proprietary non-viral gene insertion and editing technologies, an update following a strategic review of its portfolio and new preclinical data across in vivo gene therapy programs.

The Company plans to host an R&D Day focusing on cell therapy in the second half of 2024.

Evaluating Opportunities in CAR-T Beyond Oncology

The Company believes its TSCM-rich CAR-T platform and associated proprietary technologies have strong potential to deliver new therapeutic approaches in autoimmune disease. The Company is developing a strategy in inflammation and autoimmune disease and will provide an update at an appropriate time.

Leadership Updates

Alexander Chapman joined Poseida on February 26, 2024, as Senior Vice President, Investor Relations & Corporate Communications. He has over 20 years’ experience spanning a broad range of strategic roles across the biotech sector. Prior to Poseida, he was the Head of Corporate Communications & Investor Relations at Atara Biotherapeutics, the first company in the world to receive regulatory approval for an allogeneic T-cell immunotherapy. Previously, Mr. Chapman held a series of global and U.S. leadership roles at Amgen, including Corporate Affairs, Value, Access & Pricing, and U.S. Business Operations.

Loren Wagner was promoted to the role of Chief Operations Officer in February 2024. Mr. Wagner joined the Company in March 2021 and most recently served as Senior Vice President, Global Operations. He has more than 30 years of industry experience across a variety of manufacturing, quality, and engineering roles. Prior to the Company, Mr. Wagner was the Head of CAR-T Operations at Bristol Myers Squibb/Celgene. Prior to Celgene, he worked for 12 years at Allergan, where he was responsible for the production and distribution of investigational supplies globally.

Brent Warner, who has served as the Company’s President, Gene Therapy, will depart Poseida to pursue an external career opportunity effective April 1, 2024.

Financial Results for the Fourth Quarter and Full Year 2023

Revenues

Revenues were $25.0 million for the fourth quarter ended December 31, 2023, compared to $10.1 million for the same period in 2022. The increase was primarily due to revenue earned as a result of a milestone achieved in the fourth quarter under the amendment to the collaboration and license agreement with Roche and increased activity under the collaboration, offset by a decrease in revenue related to Takeda due to the termination of its collaboration agreement in the third quarter of 2023.

For the full year ended December 31, 2023, revenues were $64.7 million, compared to $130.5 million for the same period in 2022. The decrease was primarily due to initial license revenue recognized from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, offset by the revenue recognized related to the research services performed under the collaboration and license agreements with Roche and Takeda, including $8.9 million of previously deferred revenue recognized as a result of the termination of its collaboration agreement with Takeda in the third quarter of 2023.

Research and Development Expenses

Research and development expenses were $42.0 million for the fourth quarter ended December 31, 2023, compared to $33.9 million for the same period in 2022. The increase was primarily due to an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, an increase in personnel expenses as a result of increased headcount, and an increase in clinical stage allogeneic programs, partially offset by a decrease in clinical stage autologous programs driven by the wind-down of the Company’s clinical development activities for such programs.

For the full year ended December 31, 2023, research and development expenses were $156.8 million, compared to $152.9 million for the same period in 2022. The increase was primarily due to an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, an increase in personnel expenses as a result of increased headcount, an increase in clinical stage allogeneic programs, and an increase in internal costs related to facilities and other expenses, offset by a decrease in clinical stage autologous programs driven by the wind-down of the Company’s clinical development activities for such programs.

General and Administrative Expenses

General and administrative expenses were $8.9 million for the fourth quarter ended December 31, 2023, compared to $9.4 million for the same period in 2022. The decrease was primarily due to lower insurance costs.

For the full year ended December 31, 2023, general and administrative expenses were $37.4 million, compared to $37.5 million for the same period in 2022. The decrease was primarily due to lower insurance costs, offset by an increase in personnel costs due to an accelerated stock-based compensation expense in the first quarter of 2023 related to a one-time expense associated with the retirement of the Company’s former Executive Chairman.

Net Loss

Net loss was $25.3 million and $123.4 million for the fourth quarter and full year ended December 31, 2023, respectively, compared to net loss of $33.3 million and $64.0 million for the fourth quarter and full year ended December 31, 2022, respectively.

Cash Position

As of December 31, 2023, the Company’s cash, cash equivalents and short-term investments balance was $212.2 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche will be sufficient to fund operations into the second half of 2025. Potential additional payments under the amended Roche collaboration and license agreement and/or potential additional business development could further extend cash runway.

Corporate Overview

On March 7, 2024 Portage Biotech presented its corporate presentation (Presentation, Portage Biotech, MAR 7, 2024, View Source [SID1234640934]).

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