On March 16, 2023 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the full year ended December 31, 2022 and provided a business update (Press release, Sellas Life Sciences, MAR 16, 2023, View Source [SID1234628929]).

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"Broad success defines the past year, with positive efficacy signals reported across our clinical programs and expansion of our development pipeline," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Early this year we took proactive steps to considerably strengthen our balance sheet and prepare us for the coming year, raising an additional $20 million in gross proceeds from institutional investors, which lays the groundwork for continued advancement of both our GPS and GFH009 programs and carries us through key value-driving milestones in the upcoming year."

Pipeline Program Summary for 2022:

Galinpepimut-S (GPS): Wilms Tumor-1 (WT1) targeting immunotherapeutic

Phase 3 REGAL study in acute myeloid leukemia (AML): Enrollment continued in the global Phase 3 REGAL registrational clinical trial in patients with AML who have achieved complete remission following second-line salvage therapy (CR2 patients). SELLAS announced in November that the REGAL clinical trial protocol and statistical analysis plan were revised to adjust for higher-than-expected overall survival observed in blinded pooled patient data, with interim analysis currently expected to occur in late 2023 or early 2024. SELLAS also announced that 3D Medicines, its development and commercialization partner for Greater China, informed SELLAS that it will participate in the REGAL study with the inclusion of patients from the Greater China territory. This participation will trigger milestone payments totaling $13 million to SELLAS, which are expected during the first half of 2023.

3D Medicines phase 1 clinical trial in China: An investigational new drug (IND) application was approved by China’s National Medical Products Administration for the first clinical trial for GPS in China, which triggered a milestone payment of $1 million to SELLAS. 3D Medicines initiated the open-label, single-arm, multi-center Phase 1 clinical trial in patients with AML, multiple myeloma, non-Hodgkin’s lymphoma, or higher-risk myelodysplastic syndrome.

Phase 1/2 Study in combination with pembrolizumab (Keytruda) in ovarian cancer: SELLAS released top-line data in November showing clinical benefit of GPS in combination with pembrolizumab anti-PD-1 therapy in WT1 positive relapsed or refractory platinum resistant advanced metastatic ovarian cancer patients. The study was conducted under a Clinical Trial Collaboration and Supply Agreement with Merck & Co., Inc., Rahway, N.J., USA (known as MSD outside the United States and Canada), and SELLAS plans to present final data from this study at a medical conference in the first half of 2023.

Phase 1 Study in combination with nivolumab (Opdivo) for malignant pleural mesothelioma (MPM): Updated positive data were announced in June from an investigator sponsored open-label Phase 1 trial for GPS combination therapy with checkpoint inhibitor nivolumab for treatment of MPM in patients who were either refractory to or relapsed after at least one line of the standard of care therapy. Patient enrollment was completed at the end of 2022 and SELLAS expects to report topline data during the first half of 2023.

Manufacturing: Completed and ongoing stability studies confirmed stability of GPS for at least 42 months at -20C and for at least 12 months at 2-8C and room temperatures.

GFH009: highly selective CDK9 inhibitor

License agreement with GenFleet Therapeutics (Shanghai), Inc.: In March, SELLAS entered into a license agreement with GenFleet Therapeutics (Shanghai), Inc. ("GenFleet") for an in-license of global rights outside of Greater China for the highly-selective small molecule cyclin-dependent kinase 9 (CDK9) inhibitor, GFH009.

Phase 1 clinical trial in hematological malignancies: Positive interim data from the ongoing Phase 1 dose-escalating clinical trial showed no dose limiting toxicities; anticancer effects were observed across multiple dose levels in both AML and lymphoma patients, suggesting a broad therapeutic window and high potential for both monotherapy as well as combination treatment. One patient in the AML cohort achieved a confirmed complete response, which SELLAS believes represents the first reported in AML for any CDK9 monotherapy. Initial pharmacodynamics (PD) studies showed clear reductions of CDK9 activity in the two biomarkers, MCL‐1 and MYC.

Following completion of the Phase 1 clinical trial and determination of the recommended Phase 2 dose, SELLAS intends to commence a Phase 2a clinical trial of GFH009 in combination with venetoclax and azacitidine in AML patients who failed or did not respond to treatment with venetoclax and azacitidine. The primary endpoint of the Phase 2a clinical trial, which SELLAS expects to initiate during the second quarter of 2023, will likely be complete remission (CR) rate and secondary endpoints will likely include progression free survival, overall survival and proportion of patients proceeding to transplant. The topline data for this study are expected in the fourth quarter of 2023. SELLAS is also planning to potentially commence a Phase 2 clinical trial of GFH009 in certain solid tumors and/or lymphoma in the third quarter of 2023.

Preclinical Pediatric Testing and Results: GFH009 was selected for the National Cancer Institute (NCI) Pediatric Preclinical in Vivo Testing (PIVOT) Program, which involves a two-phase research plan for pharmacokinetics (PK) and efficacy in pediatric tumors. As part of the program, PIVOT principal investigators at eight participating research institutions evaluate GFH009 against pediatric solid tumors and leukemia models, and these studies are supported by NCI cooperative agreement grants. SELLAS also announced results from preclinical in vitro studies of GFH009 in solid tumor and AML cell lines demonstrating significant anti-tumor effects and cancer cell growth inhibition in all selected cell lines.

Financial Results for the Full Year 2022:

Licensing revenue: Licensing revenue for the year ended December 31, 2022 was $1.0 million which related to approval by Chinese regulatory authorities of an IND application by 3D Medicines. This compares to $7.6 million for the year ended December 31, 2021.

R&D Expenses: Research and development expenses for the year ended December 31, 2022 were $20.3 million, as compared to $15.7 million for the year ended December 31, 2021. The increase was primarily due to an increase in clinical trial expenses related to the REGAL clinical trial and personnel related expenses due to increased headcount.

Acquired In-Process Research and Development: Acquired in-process research and development was $10.0 million for the year ended December 31, 2022, resulting from the in-licensing of GFH009. There was no acquired in-process research and development during the year ended December 31, 2021.

G&A Expenses: General and administrative expenses for the year ended December 31, 2022 were $12.6 million, as compared to $11.3 million for the year ended December 31, 2021. The increase was primarily due to personnel related expenses due to increased headcount, which was partially offset by a decrease in amortization expense associated with the capitalized contract acquisition costs of the 3D Medicines license agreement.

Net Loss: Net loss was $41.3 million for the year ended December 31, 2022, or a basic and diluted loss per share of $2.13, as compared to a net loss of $20.7 million for the year ended December 31, 2021, or a basic and diluted loss per share of $1.34.

Cash Position: As of December 31, 2022, cash and cash equivalents totaled approximately $17.1 million. Subsequent to December 31, 2022, the Company consummated an underwritten public offering providing gross proceeds to the Company of $20 million, before deducting underwriting discounts and commissions and offering expenses. An additional $13 million milestone payment from 3D Medicines is expected in the first half of 2023.

Keytruda is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA and is not a trademark of SELLAS. Opdivo is a registered trademark of Bristol-Myers Squibb Company, New York, NY, USA and is not a trademark of SELLAS. The manufacturers of these brands are not affiliated with and do not endorse SELLAS or its products.

On March 16, 2023 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the fourth quarter and year ended December 31, 2022 (Press release, Sana Biotechnology, MAR 16, 2023, View Source [SID1234628928]).

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"2022 began our transformation from a research focused organization to a clinical-stage company, setting the stage for important clinical data in 2023 and 2024 to better define our product candidates and platforms," said Steve Harr, Sana’s President and Chief Executive Officer. "The recent clearance of our first IND – for SC291, a hypoimmune-modified, CD19-targeted allogeneic CAR T therapy for patients with B-cell malignancies – offers the first of several near-term opportunities to understand our hypoimmune technology in patients and its potential to move forward important medicines. We anticipate initial clinical data with this program and hypoimmune islet cells in type 1 diabetic patients in 2023. We also expect to file INDs in oncology for an additional allogeneic CAR T program and our first in vivo fusogen program targeting T cells. Our balance sheet gives us the financial strength to build on our execution in 2022 and push our R&D portfolio forward."

Recent Corporate Highlights

Advancing to the clinic with two opportunities for clinical proof of concept this year for the hypoimmune platform, including ex vivo hypoimmune-modified allogeneic CAR T cells and hypoimmune-modified primary human islet cells:

SC291 is a hypoimmune-modified CD19-targeted allogeneic CAR T for patients with B cell malignancies. The SC291 IND has been cleared, and Sana expects to share initial clinical data later this year. Success unlocks potential value of a broader hypoimmune-modified allogeneic CAR T platform with clinically-validated CD22 and BCMA CAR constructs.
Sana expects an investigator sponsor trial using primary human hypoimmune-modified islet cells transplanted in type 1 diabetes patients to begin later this year. Sana anticipates sharing initial clinical data later this year. The goal is to understand pancreatic islet cell survival without immunosuppression in these autoimmune patients, providing insight into Sana’s ongoing hypoimmune-modified stem cell-derived pancreatic islet cell program (SC451), which has a goal of filing an IND in 2024.
Building pipeline with potential to deliver multiple clinical data readouts over the next several years across three platforms – ex vivo hypoimmune-modified allogeneic CAR T cells, stem-cell derived cell therapies, and the in vivo fusogen platform:

ex vivo hypoimmune-modified allogeneic CAR T platform: Sana has the opportunity to unlock a potentially best-in-class, broadly accessible allogeneic CAR T franchise across multiple patient populations using clinically-validated CAR constructs, including SC291 (CD19), SC262 (CD22), SC255 (BCMA), and beyond.
In 2022, entered into an agreement with the National Institutes of Health (NIH) for worldwide exclusive commercial rights to the NIH’s CD22 chimeric antigen receptor with a fully-human binder for use in certain ex vivo allogeneic CAR T applications. This CAR construct has shown a promising efficacy profile in several clinical studies, including in autologous CD19 CAR T cell therapy failures. SC262 incorporates this clinically-validated CAR with T cells manufactured using hypoimmune technology with a goal of filing an IND in 2023.
In 2022, entered into a non-exclusive agreement with IASO Biotherapeutics and Innovent Biologics for commercial rights to a clinically-validated fully-human B cell maturation antigen (BCMA) CAR construct. SC255 incorporates this clinically-validated CAR with T cells manufactured using the hypoimmune platform with a goal of filing an IND in 2024.
stem-cell derived platform: Sana has several programs using stem-cell derived cell therapies, including SC451 and SC379.
SC451 is a hypoimmune stem-cell derived islet cell program for type 1 diabetes with a goal of a 2024 IND.
SC379 is a stem-cell derived glial progenitor cell therapy targeting multiple central nervous system diseases with a goal of a 2024 IND.
in vivo fusogen platform: The fusogen platform focuses on in vivo cell specific delivery of genetic material. Sana’s lead program using this platform is SG299, previously called SG295, an in vivo CAR T product candidate that utilizes a CD8-targeted fusosome to deliver a CD19 CAR to generate a CD19-targeted CAR T cell. The company continues to progress earlier programs focused on cell-specific delivery of various payloads.
SG299 has the potential to generate CAR T cells in vivo (inside the patient), eliminating the need for conditioning chemotherapy and complex CAR T cell manufacturing. The company’s goal is to file an IND this year to study this drug candidate in patients with B cell malignancies.
In 2022, Sana transitioned to a new manufacturing process for SG295 and renamed the product SG299 in connection with that transition. SG299 has at least a 50X improvement in product potency, which Sana believes has the potential to translate into better efficacy, safety, and long-term manufacturability. The company plans to use this second-generation process for the first-in-human studies in patients with B cell malignancies.
Demonstrated that two additional fusosome candidates eliminated tumors in preclinical models – a CD4+ T cell targeting fusosome delivering a CD19 CAR and a CD8+ T cell targeting fusosome delivering a CD22 CAR.
Advanced Sana’s hypoimmune ex vivo platform and in vivo fusogen platform with presentations at AACR (Free AACR Whitepaper), ASGCT (Free ASGCT Whitepaper), ADA, ISSCR, and ASH (Free ASH Whitepaper):

ex vivo hypoimmune platform: Sana’s hypoimmune platform makes multiple genomic modifications to cells with the goal of preventing allogeneic transplant rejection, and importantly includes modifications to prevent both adaptive and innate immune recognition and rejection. Sana’s pipeline includes hypoimmune-modified cells to replace damaged or missing cells in the body in a number of different diseases, including, among others, cancer, type 1 diabetes, and various neurologic conditions.
Presented preclinical data demonstrating that hypoimmune-modified CAR T cells were able to evade both the innate and adaptive arms of the immune system in animal models while retaining their antitumor activity.
Presented preclinical data showing survival of transplanted allogeneic hypoimmune-modified cells of several different types – including pancreatic islet cells, cardiomyocytes, and retinal pigment epithelial cells – in a variety of locations in non-human primates.
Presented preclinical data showing that hypoimmune-modified allogeneic regulatory T cells function and are able to evade immune detection in preclinical models. These cells have the potential to treat a variety of autoimmune disorders.
Presented preclinical data outlining the importance of CD47 overexpression as part of the hypoimmune platform to evade adaptive and innate immune response, the use of CRISPR/Cas12b in scaled hypoimmune-modified allogeneic CAR T manufacturing, the development of assays to evaluate T cell quality from healthy, allogeneic donors, and the generation of a hypoimmune-modified BCMA-directed allogeneic CAR T cell.
hypoimmune-modified pancreatic islet cells: Type 1 diabetes is a disease in which a person’s immune system destroys one’s own pancreatic beta cells, which are a key component in pancreatic islets. Hypoimmune technology is incorporated in SC451, Sana’s ongoing stem cell-derived pancreatic islet cell program, for which Sana has a goal of filing an IND in 2024 for the treatment of type 1 diabetes.
Presented preclinical data showing that transplanted hypoimmune-modified pancreatic islet cells evade allogeneic immune response and autoimmune response in a novel type 1 diabetes mouse model. These data build upon previous in vitro data showing that hypoimmune-modified pancreatic islet cells are not recognized by serum from type 1 diabetic patients, including no T cell or antibody recognition.
Presented preclinical data showing that hypoimmune-modified islet cells transplanted intramuscularly may be capable of persisting and functioning in diabetic patients without immune suppression.
in vivo fusogen platform: Presented additional preclinical data utilizing retargeted fusosomes for in vivo delivery of genetic payloads to various cells, including CD8+ T cells, CD4+ T cells, human hepatocytes, and initial data on our work in hematopoietic stem cells. This technology is the backbone of Sana’s in vivo delivery platform and is incorporated into various product candidates, including SG299.
Announced expected cash runway into 2025 to enable multiple data readouts across the platforms; largest part of cash savings from plans to relocate manufacturing facility to Bothell, Washington

Expect cash runway into 2025 enabling multiple data readouts across the platforms based on current timelines for lead programs.
Announced decision to move Sana’s manufacturing plant from Fremont, CA to Bothell, WA, resulting in approximately $100 million in expected cost savings compared to the initial build-out plan. As part of this decision, Sana signed a lease agreement to develop an approximately 80,000 square foot manufacturing facility in Bothell, WA. The facility will be designed to support the late-stage clinical and early commercial manufacturing of multiple product candidates across the portfolio.
Announced key corporate updates, building on the company’s scientific excellence and operational capabilities

Announced a portfolio prioritization and corporate restructuring designed to optimize the development of programs at or nearing clinical development, continue investments in the core research platforms and innovation, and maintain a strong balance sheet.
Named the top place to work on the BioSpace 2023 Best Places to Work small employer list, based on attributes including compensation, innovation, career growth opportunities, leadership, culture, diversity, equity and inclusion, reputation, and flexibility and remote work.
Strengthened the leadership team with the appointments of Snehal Patel to lead technical operations and Julie Lepin to lead regulatory affairs.
Fourth Quarter 2022 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2022 were $434.0 million compared to $746.9 million as of December 31 2021. The decrease of $312.9 million was primarily driven by cash used in operations of $289.9 million and cash used for the purchase of property and equipment of $20.9 million. Cash used in operations includes $6.2 million of upfront payments related to licensing technology for the company’s CD22 and BCMA programs, $4.3 million of one-time restructuring costs related to the portfolio prioritization and corporate restructuring in the fourth quarter of 2022, and $3.2 million of costs incurred related to the previously planned manufacturing facility in Fremont, CA (the Fremont facility) which will be replaced by the facility in Bothell, WA (the Bothell facility). In addition, our cash balance will increase by $6.7 million in July 2023 as the letter of credit related to the Fremont facility reduces from $6.7 million to $0.6 million in July 2023.
Research and Development Expenses: For the three and twelve months ended December 31, 2022, research and development expenses, inclusive of non-cash expenses, were $63.9 million and $285.9 million, respectively, compared to $108.5 million and $248.6 million for the same periods in 2021. The decrease of $44.6 million for the three months ended December 31, 2022 was primarily due to the one-time upfront payment to Beam Therapeutics Inc. (Beam) in the fourth quarter of 2021 to license its gene editing technology, partially offset by an increase in research, development, and third-party manufacturing costs, and facility and software expenses. The increase of $37.3 million for the twelve months ended December 31, 2022 was largely due to increases in personnel-related expenses, including increased headcount to expand Sana’s research and development capabilities, increased research, laboratory, and third-party manufacturing costs, and allocated personnel costs, depreciation expense, and facility and software costs. These increases were partially offset by the one-time upfront payment to Beam in the fourth quarter of 2021 to license its gene editing technology. Research and development expenses for the three and twelve months ended December 31, 2022 include non-cash stock-based compensation of $6.0 million and $26.6 million, respectively, and $5.3 million and $15.2 million, respectively, for the same periods in 2021.
Research and Development Related Success Payments and Contingent Consideration: For the three and twelve months ended December 31 2022, Sana recognized non-cash gains of $5.5 million and $84.9 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. Sana recognized a non-cash gain of $9.9 million for the three months ended December 31, 2021 and a non-cash expense of $57.9 million for the twelve months ended December 31, 2021. The value of these potential liabilities may fluctuate significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three months ended December 31, 2022, inclusive of non-cash expenses, were $23.3 million compared to $12.7 million for the same period in 2021. The increase of $10.6 million was primarily due to one-time restructuring costs of $8.7 million, including stock-based compensation of $1.9 million, related to the portfolio prioritization and corporate restructuring in the fourth quarter of 2022, operating costs associated with the Fremont facility, business taxes, and legal fees. These increases were offset by a decline in personnel-related expenses. General and administrative expenses for the twelve months ended December 31, 2022 were $71.6 million compared to $50.4 million for the same period in 2021. The increase of $21.2 million was primarily due to one-time restructuring costs of $8.7 million, including stock-based compensation of $1.9 million, related to the portfolio prioritization and corporate restructuring in the fourth quarter of 2022, the write-off of construction in progress costs incurred in connection with the Fremont facility, personnel-related expenses attributable to an increase in headcount to support Sana’s continued research and development activities, and operating costs associated with the Fremont facility. General and administrative expenses for the three and twelve months ended December 31, 2022 include stock-based compensation of $4.6 million and $11.8 million, respectively, and $2.0 million and $7.1 million, respectively, for the same periods in 2021.
Net Loss: Net loss for the three and twelve months ended December 31, 2022 was $80.4 million, or $0.42 per share, and $269.5 million, or $1.43 per share, respectively, compared to $110.7 million, or $0.60 per share, and $355.9 million, or $2.14 per share, respectively, for the same periods in 2021.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the twelve months ended December 31, 2022 was $288.3 million compared to $209.6 million for the same period in 2021. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, cash outflows from business development and non-recurring restructuring activities, and the purchase of property and equipment.
Non-GAAP General and Administrative Expense: Non-GAAP general and administrative expense for the three and twelve months ended December 31, 2022 was $14.6 million and $58.4 million, respectively, compared to $12.7 million and $50.4 million, respectively, for the same periods in 2021. Non-GAAP general and administrative expense excludes one-time restructuring costs, including stock-based compensation, related to the portfolio prioritization and corporate restructuring in the fourth quarter of 2022 and the write-off of construction in progress costs incurred in connection with the Fremont facility.
Non-GAAP Net Loss: Non-GAAP net loss for the three and twelve months ended December 31, 2022 was $77.2 million, or $0.40 per share, and $341.2 million, or $1.81 per share, respectively, compared to $120.6 million, or $0.65 per share, and $298.1 million, or $1.79 per share, respectively, for the same periods in 2021. Non-GAAP net loss excludes non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities, one-time restructuring costs, including stock-based compensation, related to the portfolio prioritization and corporate restructuring in the fourth quarter of 2022, and the write-off of construction in progress costs incurred in connection with the Fremont facility.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."

On March 16, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported a business update and reports financial results for the quarter and full year ended December 31, 2022 (Press release, Panbela Therapeutics, MAR 16, 2023, View Source [SID1234628926]). As previously announced, management is hosting earnings call today at 4:30 p.m. ET.
2022 and early 2023 Highlights:
• Significant progress was made on the conduct of the ASPIRE global clinical trial studying ivospemin (SBP-101) in combination with gemcitabine and nab-paclitaxel in the first-line treatment of metastatic pancreatic ductal adenocarcinoma:
o Initiated ASPIRE trial in January 2022
o Received approval from the Australian Human Research Ethics Committee
(HREC) to expand the ASPIRE global clinical trial to Australia o Enrolled first patient in South Korea o Enrolled first patient in Europe o Enrolled first patient in Australia
o Received approvals to open trial sites in Spain, France and Italy
• An abstract for ivospemin will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper), which will be held April 14-19, 2023. The work reflects the Company’s on-going collaboration with Johns Hopkins University School of Medicine
• Closed a registered public offering yielding gross proceeds of approximately $15 million in Q1 of 2023
• Started Phase II Trial of CPP-1X-T for Recent Onset Type I Diabetes in January 2023, in collaboration with Indiana University and the Juvenile Diabetes Research Foundation
• European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued the Adoption of Commission Implementing Decision relating to the designation of ivospemin as an orphan medicinal product in March 2023
• Closed a registered public offering yielding gross proceeds of approximately $6.0 million in Q4 of 2022
• Hosted an R&D call joined by leading experts for a deep dive on the company’s investigational drug, ivospemin, as a polyamine metabolism modulator in ovarian cancer
• Completed the acquisition of Cancer Prevention Pharmaceuticals, Inc. (CPP)
• Presented a poster highlighting the results for ivospemin as a polyamine metabolism modulator in ovarian cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April 2022
• Presented a poster highlighting the Clinical Data on Phase 1b Clinical Trial of ivospemin in Combination with Gemcitabine and Nab-Paclitaxel in Patients with Metastatic PDA at 2022 ASCO (Free ASCO Whitepaper) GI Meeting
"During Q4 and year to date, we progressed our pipeline, which has been principally funded through collaborations," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "Milestones achieved included first patients enrolled in Australia, Europe and South Korea in our ASPIRE global trial for metastatic pancreatic cancer, and EMA Orphan drug designation. Additionally, we bolstered our balance sheet with gross proceeds from recent public offerings. As we move forward in 2023, we anticipate a consistent stream of milestones to drive shareholder value."
Fourth Quarter ended December 31, 2022 Financial Results
General and administrative expenses were $1.7 million in the fourth quarter of 2022, compared to $1.3 million in the fourth quarter of 2021. The increase primarily is due to severance expenses associated with the acquisition of CPP.
Research and development expenses were $3.5 million in the fourth quarter of 2022, compared to $2.0 million in the fourth quarter of 2021. The change is due primarily to an increase in spending on our clinical studies as we expanded the ASPIRE clinical trial.
Net loss in the fourth quarter of 2022 was $4.7 million, or $5.68 per diluted share, compared to a net loss of $3.5 million, or $10.54 per diluted share, in the fourth quarter of 2021. All share and per-share amounts have been restated to reflect the 40-for-1 reverse split of our common stock, which was effective on January 13, 2023.
Total cash was $1.3 million as of December 31, 2022. Total current assets were $1.8 million and current liabilities were $7.8 million as of the same date. Notes payable, plus accrued interest, on the balance sheet, the result of the acquisition of CPP, totaled approximately $7.2 million. The current portion of the notes payable plus accrued interest totaled approximately $2.0 million.
Subsequent to the end of the year, the Company completed a registered public offering. Gross proceeds from the raise, which closed on January 30, 2023, were approximately $15 million.
Conference Call Information
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Participant Access Code: 116790
Webcast Link: View Source
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On March 16, 2023 Jounce Therapeutics, Inc. (Nasdaq: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that patients from the INNATE Phase 2 trial in the ovarian cancer combination cohort with platinum resistant ovarian cancer are experiencing deep and durable responses based on a pre-planned informal data review (Press release, Jounce Therapeutics, MAR 16, 2023, View Source [SID1234628925]).

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The INNATE trial evaluates JTX-8064 as a monotherapy and in combination with the PD-1 inhibitor pimivalimab in patients with advanced solid tumors. JTX-8064, is a humanized IgG4 monoclonal antibody designed to specifically bind to the macrophage receptor Leukocyte Immunoglobulin Like Receptor B2 (LILRB2/ILT4), inhibiting LILRB2 binding with its ligands which may result in reprogramming immune-suppressive macrophages to enhance anti-tumor immunity.

A data review was conducted on March 15, 2023 to assess the potential to achieve proof-of-concept (POC) in the ovarian cancer combination cohort of the INNATE trial. In this cohort, 35 patients with third- and fourth-line platinum resistant ovarian cancer were treated with JTX-8064 and Jounce’s PD-1 inhibitor, pimivalimab. There were five RECIST 1.1 responders, four of which are confirmed, with all five patients remaining on study for over six months with continued tumor reduction over time. The remaining 30 patients have discontinued treatment. Upon confirmation of a fifth confirmed partial response (PR), the ovarian combination cohort would meet Jounce internal criteria for POC, based on a statistically meaningful improvement over the benchmark of pembrolizumab alone in the analogous setting. In patients with one to six prior lines of therapy for ovarian cancer, pembrolizumab monotherapy demonstrated a response rate of 8.5%, with duration of response of 10.2 months. Two of the five responders in INNATE have a PD-L1 score of zero, indicating a low likelihood of response to a PD-1 inhibitor. The combination was well tolerated with <10% of patients reporting Grade 3 or greater related adverse events.

Ovarian Cancer Combination Cohort Responders

Subject PD-L1
(CPS) Response
Assessment When was uPR
demonstrated Current status
1 55% cPR Wk 9 -82.8% (wk 45)
2 15% cPR Wk 18 -66.4% (wk 36)
3 5% cPR Wk 18 -87.6% (wk 27)
4 0% cPR Wk 18 -78.26% (wk 27)
5 0% uPR Wk 27 -40% (wk 27)

Ovarian Combination Cohort Target Lesion % Change from Baseline over Time

"Following this analysis, we are pleased to see these results demonstrating deep and durable responses in patients, including those with a PD-L1 score of 0%, on a very well tolerated regimen. These results lead us to believe that there is a potential for meaningful clinical benefit with this combination in patients with few durable therapeutic options," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "Tumor reduction was observed at 9 weeks in all the responding patients in the ovarian cancer cohort of the INNATE trial, but most did not achieve a PR until week 18, which delayed our ability to assess efficacy in this cohort. Platinum resistant ovarian cancer is a patient population with significant unmet need and progressive disease is often associated with debilitating symptoms."

Enrollment has been completed in all cohorts in the INNATE trial. In addition to the patients with ovarian cancer, both durable responses and stable disease have occurred in patients with renal cell carcinoma, biliary tract cancer, and first-line and second/third-line head and neck squamous cell carcinoma, but the ovarian data is the most encouraging with potential to achieve pre-determined POC criteria.

As previously announced, Jounce is seeking to partner JTX-8064. Jounce continues to believe a company with additional resources and a longer value creation timeline could potentially advance this program for the benefit of cancer patients.

On March 16, 2023 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported a clinical development and operational update (Press release, Bio-Path Holdings, MAR 16, 2023, View Source [SID1234628923]).

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"Throughout 2022, we laid the foundation to meaningfully advance our DNAbilize antisense RNAi nanoparticle technology in a number of important oncology indications for which there are limited treatment options," said Peter H. Nielsen, President and Chief Executive Officer of Bio-Path. "In addition to our clinical progress, we made significant investment towards shoring up our manufacturing supply and intellectual property armamentarium. Given these advancements and the proven safety and efficacy profile shown to date across our portfolio, we look forward to several near-term clinical milestones."

Important Near-Term Clinical Milestones

BP1001-A Phase 1/1b Clinical Trial in Solid Tumors

● Important trial with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer with initial cohort completion and data readout expected before mid-year.

BP1002 Phase 1/1b Clinical Trial in Relapsed/Refractory AML

● Focus on patients who relapsed on venetoclax treatment with initial cohort completion and readout expected in the second quarter of 2023.

Prexigebersen (BP1001) Phase 2 Clinical Trial in AML

● Two of the three cohorts in the clinical trial already exceed the minimum efficacy required for enrollment expansion.
● Assess safety and efficacy of each cohort treatment combination therapy with potential to qualify for expedited program status after cohort’s initial interim analysis, which are expected to commence by cohort in the second quarter of 2023.

Clinical Program Overview

Bio-Path’s clinical development program consists of one Phase 2 clinical trial and three Phase 1 or 1/1b clinical trials. There is one additional drug candidate that is in preclinical development, which may be submitted to the FDA later in the year in an Investigational New Drug (IND) application.

Phase 2 Clinical Trial – Bio-Path’s Phase 2 clinical trial is treating Acute Myeloid Leukemia (AML) patients. This trial is comprised of three separate cohorts of patients and treatments, each separately approvable by the FDA as a new drug treatment. The first two cohorts are treating patients with the triple combination of prexigebersen (Bio-Path’s drug candidate BP1001), decitabine and venetoclax. The first cohort includes untreated AML patients, and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort is treating relapsed/refractory AML patients, who are venetoclax-resistant or -intolerant, with the two-drug combination of prexigebersen and decitabine. The interim analysis of each cohort to assess the safety and efficacy of treatment combination therapy is expected to commence by cohort in the second quarter of 2023, with the potential to qualify for expedited regulatory status.

Phase 1/1b Clinical Trial in BP1001-A in Advanced Solid Tumors – Phase 1/1b clinical trial of BP1001-A in patients with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer. BP1001-A is a modified

product candidate that incorporates the same drug substance as prexigebersen but has a slightly modified formulation designed to enhance nanoparticle properties. Completion of the first dose escalation cohort is expected in the coming months.

Phase 1/1b Clinical Trial in BP1002 in Relapsed/Refractory AML. Phase 1/1b clinical trial for BP1002 to treat relapsed/refractory AML patients, including venetoclax-resistant patients. BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. AML patients that fail frontline venetoclax-based therapy have very poor prognosis with median overall survival of less than three months. Completion of the first dose escalation cohort is expected in the coming months.

Phase 1 Clinical Trial in BP1002 in Refractory/Relapsed Lymphoma and Chronic Lymphocytic Leukemia (CLL). Phase 1 clinical trial for refractory/relapsed lymphoma and CLL. The Phase 1 clinical trial is being conducted at several leading cancer centers, including the Georgia Cancer Center, The University of Texas Southwest and New York Medical College. Completion of the current patient cohort is expected in 2023.

Preclinical Work for BP1003. The Company continues to advance its drug candidate, BP1003, for the treatment of advanced solid tumors, including pancreatic cancer. BP1003 is an antisense RNAi nanoparticle targeting the STAT3 protein. Plans are to conduct a Phase 1 study of BP1003 in patients with refractory, metastatic solid tumors (pancreatic, non-small cell lung cancer).

Manufacturing Supply

The COVID-19 pandemic created manufacturing interruptions over the last several years. The Company experienced manufacturing shutdowns in supply chain manufacturing plants and an increase in lost manufactured product batches that created supply disruptions. In addition, the onset of messenger RNA vaccine development for COVID-19 created increased backlog time at oligonucleotide suppliers. These conditions created drug candidate supply shortfalls that caused enrollment challenges for Bio-Path’s clinical trials.

The result of these factors led Bio-Path to double the Company’s supply chain, increase capacity, quality and improve scheduling flexibility. These goals were achieved in 2022 and have resulted in increased drug candidate supply, quality and patient enrollment.

Intellectual Property Protection

Bio-Path’s composition of matter patents protect encroachment from third parties on its proprietary products. This technology is solely owned by Bio-Path. These composition patents allow the Company to apply its core technology to new protein targets and receive new 20-year patents. Bio-Path’s patent portfolio is as follows:

● New composition and methods of use patent issued covers DNAbilize technology, solely owned by Bio-Path.
● Five patents issued in the United States; eight foreign patents issued; one additional foreign patent application allowed.
● Five applications pending in the United States along with 60+ applications pending in foreign jurisdictions.