On March 16, 2022 Provectus (OTCQB: PVCT) reported that data from an ongoing clinical trial of investigational cancer immunotherapy PV-10 (rose bengal sodium) for the treatment of neuroendocrine tumors (NET) metastatic to the liver (mNET) refractory to somatostatin analogs (SSAs) and peptide receptor radionuclide therapy (PRRT) (NCT02693067) was presented at the annual conference of the European Neuroendocrine Tumor Society (ENETS), held from March 10-11, 2022 in a hybrid setting in Barcelona, Spain and online (Press release, Provectus Biopharmaceuticals, MAR 16, 2022, View Source [SID1234610179]).

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The oral presentation was made by the principal investigator of the clinical trial’s single center at The Queen Elizabeth Hospital (TQEH) in Adelaide, Australia: Tim Price, MBBS, DHlthSc (Medicine), FRACP, Head of Clinical Oncology Research and Chair of the combined Hematology and Medical Oncology Unit at TQEH, and Clinical Professor in the Faculty of Medicine at the University of Adelaide.

Highlights from the 2022 ENETS Presentation:

Patient characteristics
N = 12 patients: 50% male; median age of 66 years (range 47-79)
Primary tumor sites: 7 small bowel (58%), 3 pancreas (25%), 1 caecal (8%), and 1 unknown (8%; likely pancreas)
NET grades: 5 Grade 1 (42%) and 7 Grade 2 (58%)
All patients were refractory to SSA (100%), and 11 received PRRT (92%) as part of their prior treatment
All patients had symptomatic, progressive disease
Baseline chromogranin A (CgA): median 1,585 µg/L (range 35-10,370)
PV-10 treatment
4 patients (33%) received more than 1 dose (range 2-4)
8 patients (67%) received 1 dose
Safety
All treatment-emergent adverse events (TEAEs) were Grade 1 or 2, primarily injection site pain (75%)
Single subjects experienced Grade 3 TEAEs of photosensitivity reaction or transaminases increased
Efficacy
Median progression-free survival (mPFS): 9.4 months (range 1.0-41.8)
Median overall survival (mOS): 22.5 months (range 5.5-42.3); 4 patients alive
Subgroup analysis of primary NET histology, pancreas vs intestine: mPFS 2.7 months vs 19.7 months; mOS 11.8 months vs 25.5 months
Quality of life (QOL)
Stable or improved health status and symptoms after 3 months in most patients
A recording of Dr. Price’s presentation and a copy of his slides are available on Provectus’ website at View Source

Dominic Rodrigues, Vice Chair of the Company’s Board of Directors, said, "Study data continue to show encouraging local and systemic disease control, as well as symptom control, in a heavily pre-treated population and to support a role for monotherapy PV-10 as a treatment for neuroendocrine cancer patients who fail standard therapy."

Mr. Rodrigues added, "PV-10-led combination therapies for neuroendocrine cancer patients may represent opportunities to improve quality of life and clinical outcomes. Front-line approaches may include combining liver-directed PV-10 therapy with systemically-delivered immune checkpoint inhibitors to enhance PV-10’s immune mechanisms, or using PV-10 to enhance the activity of cytotoxic treatments such as peptide receptor radionuclide therapy."

About PV-10

Intralesional (IL) administration of PV-10 for the treatment of solid tumor cancers can yield immunogenic cell death within hours of tumor injection, and induce tumor-specific reactivity in circulating T cells within days. This PV-10-induced functional T cell response may be enhanced and boosted in combination with immune checkpoint blockade (CB). In CB-refractory disease, PV-10 may restore disease-specific T cell function. IL PV-10 has been administered to over 450 patients with melanoma and cancers of the liver in both monotherapy and combination therapy settings. IL PV-10 is administered under visual, tactile, or ultrasound guidance to superficial malignancies, and under CT or ultrasound guidance to visceral hepatic tumors.

Systemic administration of PV-10 is undergoing preclinical study as prophylactic and therapeutic treatments for high-risk and refractory adult solid tumor cancers, and as a therapeutic treatment for relapsed and refractory blood cancers.

On March 16, 2022 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the financial and operational results for its third quarter ended January 31, 2022, and provided an overview of recent operational highlights (PharmaCyte’s Fiscal Year begins May 1 and ends April 30) (Press release, PharmaCyte Biotech, MAR 16, 2022, View Source [SID1234610178]).

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Cash Position: PharmaCyte had approximately $87 million in cash on hand as of January 31, 2022.

Recent Q3 Highlights—Corporate:

PharmaCyte established FDIC insured accounts of approximately $50.3 million.
Recent Highlights—Pipeline Products:

In November 2021, PharmaCyte announced that the empty capsule material that makes up its CypCaps pancreatic cancer product does not cause skin irritation.
In November 2021, PharmaCyte launched its malignant ascites program with the commencement of a pivotal study to determine if its treatment for locally advanced, inoperable pancreatic cancer (LAPC)—Cell-in-a-Box (CypCaps) combined with the cancer killing prodrug ifosfamide—can also delay the production and accumulation of malignant ascites.
In December 2021, the Company successfully completed the Cytochrome P450 site of integration DNA sequencing assay and announced the results of an additional, more detailed, analysis of the integration site of the cytochrome P450 2B1 gene from the augmented HEK293 cell clone that PharmaCyte uses in its CypCaps product.
In December 2021, PharmaCyte successfully completed the 36-month time point in its ongoing Master Cell Bank stability study.
In January 2022, PharmaCyte announced that the empty capsule material that makes up PharmaCyte’s CypCaps pancreatic cancer product candidate is not toxic for the encapsulated cells inside the CypCaps.
In February 2022, PharmaCyte provided a comprehensive update of the status of its Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA). Among other things, PharmaCyte reported on: (i) the ongoing stability studies of its clinical trial product CypCaps; (ii) the additional studies it has commenced in response to the FDA’s requests related to the clinical hold; (iii) the exact sequence of the Cytochrome P450 2B1 gene in its clinical trial product; (iv) the biocompatibility studies it has completed and that are underway; (v) micro-compression and swelling assays being conducted on its clinical trial product; (vi) break force and glide testing on its clinical trial product; (vii) studies to show that its clinical trial product is not adversely affected by the catheters interventional radiologists use to deliver the CypCaps to a patient; and (vii) tests to show that its clinical trial product is not affected by the contrast medium used by interventional radiologists to help guide the implantation of the CypCaps into a patient.
Recent Highlights—Financial:

As of January 31, 2022, PharmaCyte’s cash balance and total assets were approximately $87 million.

On January 31, 2022, PharmaCyte’s total stockholder equity was approximately $91 million.

PharmaCyte’s "Other Expenses" decreased by approximately $45,000 and $68,000 for the three and nine months ended January 31, 2022.

Operating expenses increased for the three months ended January 31, 2022, by approximately $99,000 and $297,000 for the nine months ended January 31, 2022, as compared to the prior fiscal year, due to costs associated with: (i) research and development (R&D); (ii) an uplist to Nasdaq Capital Markets; (iii) the closing of two public offerings for approximately $90 million; and (iv) and conducting studies related to lifting the FDA’s clinical hold on PharmaCyte’s proposed treatment for LAPC.

PharmaCyte’s R&D expenses increased from the start of its fiscal year to about $526,000 to date. The two capital raises PharmaCyte conducted in August 2021 allowed for these necessary expenses to be possible.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On March 16, 2022 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported financial results for the fourth quarter and full year 2021 and provided an update on recent corporate activities (Press release, Sunesis, MAR 16, 2022, View Source [SID1234610176]).

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"In 2021, we achieved key corporate and clinical milestones that we believe positioned us for an exciting year ahead," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "We entered the public market while simultaneously completing a successful equity financing, and initiated two clinical studies of our all-oral combination therapy, Nana-val. These included our pivotal NAVAL-1 trial in EBV-positive relapsed/refractory lymphoma and our Phase 1b/2 trial in advanced EBV-positive solid tumors. In addition, we ended the year by presenting final data from Nana-val’s Phase 1b/2 EBV-positive lymphoma trial in an oral presentation at ASH (Free ASH Whitepaper) 2021, which showed complete responses in a heavily pre-treated patient population in need of a new therapeutic option."

Dr. Royston continued, "In the year ahead, we anticipate several important advancements and milestones in our clinical programs, including meaningful progress in NAVAL-1, and a preliminary data readout from our ongoing Phase 1b/2 trial in solid tumors. Should we see early efficacy signals for Nana-val in solid tumors, as we did in the Phase 1b/2 EBV-positive lymphoma trial, it could serve as initial support for our pursuit of a tissue agnostic approach to EBV-associated malignancies and expand our addressable patient population. With a cash runway into mid-2024, we are well capitalized to execute on these milestones and our broader corporate strategy."

Fourth Quarter 2021 and Recent Highlights
Clinical

Presented final results from the Phase 1b/2 trial of Nana-val (nanatinostat and valganciclovir) in relapsed/refractory (R/R) EBV+ lymphoma, in an oral presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Data featured in the presentation were from 55 patients with a median of two prior therapies. 75% (41/55) of patients were refractory to their last therapy, and 96% (53/55) had exhausted all standard therapies (per Investigator).
Efficacy data in evaluable patient (n=43):

Across all lymphoma subtypes: overall response rate (ORR) = 40% (17/43); complete response (CR) = 19% (8/43)
T/NK- non-Hodgkin lymphoma: ORR = 60% (9/15); CR = 27% (4/15)
Extranodal NK/T-Cell Lymphoma (ENKTL): ORR = 63% (5/8); CR = 13% (1/8)
Peripheral T-cell lymphoma (PTCL)/ Angioimmunoblastic T-cell lymphoma (AITL): ORR = 67% (4/6); CR = 50% (3/6)
Diffuse large B cell lymphoma (DLBCL): ORR = 67% (4/6); CR = 33% (2/6); Both DLBCL complete responses were in patients refractory to first line R-CHOP
Immunodeficiency-associated lymphoproliferative disorders (IA-LPD): ORR = 50% (3/6); CR = 33% (2/6)
Median duration of response was 10.4 months
Nana-val was generally well tolerated with reversible low-grade toxicities. The most commonly reported treatment emergent adverse events were reversible cytopenias, low grade creatinine elevations, and gastrointestinal symptoms.

Continued enrollment into, and global expansion of, pivotal NAVAL-1 trial of Nana-val for the treatment of R/R EBV+ lymphoma. NAVAL-1 employs a Simon two-stage design where patients are initially enrolled into six cohorts based on lymphoma subtype in Stage 1. If a pre-specified activity threshold is reached, additional patients will be enrolled in Stage 2. Lymphoma subtypes demonstrating promising activity in Stage 2 may be further expanded. If successful, the Company believes NAVAL-1 could potentially support multiple new drug application (NDA) filings across various EBV+ lymphoma subtypes. The Company anticipates providing an update on the initial cohort(s) that expand into Stage 2 in the second half of 2022.
Dosed first patient in the Phase 1b/2 trial of Nana-val for the treatment of EBV+ recurrent or metastatic nasopharyngeal carcinoma (R/M NPC) and other EBV+ solid tumors. The Phase 1b dose escalation portion of the study will evaluate safety and determine the recommended Phase 2 dose (RP2D) of Nana-val in patients with EBV+ R/M NPC. In Phase 2, up to 60 patients with EBV+ R/M NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to evaluate safety and preliminary efficacy. Additionally, patients with other EBV+ solid tumors will be enrolled to receive Nana-val at the RP2D in a Phase 1b dose expansion cohort. Viracta anticipates reporting preliminary Phase 1b safety and efficacy data from the trial in the second half of 2022.
Received orphan drug designation (ODD) from U.S. Food and Drug Administration (FDA) for Nana-val for the treatment of EBV+ Diffuse large B cell lymphoma, not otherwise specified (DLBCL, NOS). This represents the first ODD for EBV+ DLBCL, NOS granted by the FDA, and the fourth ODD granted for Nana-val overall. The FDA previously granted ODD to Nana-val for the treatment of T-cell lymphoma, post-transplant lymphoproliferative disorder and plasmablastic lymphoma.
Preclinical

Presented preclinical data on vecabrutinib, a reversible inhibitor of Bruton’s tyrosine kinase (BTK) and interleukin-2-inducible kinase (ITK), at the 2021 ASH (Free ASH Whitepaper) Annual Meeting. Two presentations were featured, oral and poster, with data that demonstrate the capacity of vecabrutinib to modulate immune responses. Data featured in the oral presentation showed vecabrutinib enhancing the efficacy of chimeric antigen receptor (CAR) T-cells in a murine mantle cell lymphoma model. Vecabrutinib also inhibited secretion of pro-inflammatory cytokines known to cause toxicities associated with CAR T-cell therapy, an observation that was consistent with data from a prior Phase 1 clinical trial evaluating vecabrutinib as a treatment for patients with B-cell malignancies. Data featured in the poster presentation show vecabrutinib significantly reducing signs of sclerodermatous chronic graft versus host disease (cGVHD), including skin irritation, redness, alopecia, and diarrhea, via modulation of pathogenetic B- and T-cell subsets in a murine disease model.
Corporate

Announced addition to the Nasdaq Biotechnology Index (NBI). The NBI is designed to track the performance of a set of securities listed on The Nasdaq Stock Market (Nasdaq) that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark (ICB). The NBI is re-ranked each year and is calculated under a modified capitalization-weighted methodology. Additionally, the NBI forms the basis for a number of Exchange Traded Funds (ETFs).
Secured expanded $50 million credit facility from Silicon Valley Bank (SVB) and Oxford Finance. The credit facility replaces Viracta’s prior $15 million loan and security agreement with SVB and provides the Company with the option to obtain additional non-dilutive funding at a single-digit cost of capital. Through this expanded credit facility, the Company’s existing $5.0 million debt balance was refinanced. The remaining $45.0 million is available to the Company, which is under no obligation to draw funds in the future.
Anticipated 2022 Milestones
Provide preliminary Phase 1b safety and efficacy data from the Phase 1b/2 trial in advanced EBV+ solid tumors: 2H 2022
Update on NAVAL-1 cohort(s) progressing from Stage 1 to Stage 2: 2H 2022
Fourth Quarter and Full Year 2021 Financial Results
Cash Position – Cash and cash equivalents totaled approximately $103.6 million as of December 31, 2021, which Viracta expects will be sufficient to fund its operations into mid-2024, excluding any additional borrowings under the $50.0 million credit facility.
Research and development expenses – Research and development expenses were approximately $7.3 million and $3.6 million for the fourth quarters ended 2021 and 2020, respectively. Research and development expenses were approximately $23.9 million and $13.5 million for years ended December 31, 2021, and December 31, 2020, respectively. The increase in research and development expenses was primarily due to increases in costs incurred to support the initiation of the NAVAL-1 and solid tumor trials as well as an increase in headcount and non-cash share-based compensation.
Purchased and acquired in-process research and development – Purchased and acquired in-process research and development expenses were $88.5 million for the year ended December 31, 2021. The expenses were related to the $4.0 million payment associated with the termination of the collaboration and license agreement with Shenzhen Salubris Pharmaceutical Co. Ltd. and non-cash and non-recurring costs of $84.5 million related to the write-off of in-process research and development acquired in the merger with Sunesis Pharmaceuticals.
General and administrative expenses – General and administrative expenses were approximately $4.0 million and $2.6 million for the fourth quarters ended 2021 and 2020, respectively. General and administrative expenses were approximately $15.4 million and $5.3 million for the years ended December 31, 2021, and December 31, 2020, respectively. The increase was largely due to significant and non-recurring costs associated with the merger, in addition to incremental costs associated with being a publicly traded company, including legal fees, audit fees, consulting expenses, filing fees and increased directors’ and officers’ insurance costs, in addition to an increase in non-cash share-based compensation.
Gain on Royalty Purchase Agreement – Gain on Royalty Purchase Agreement the year ended December 31, 2021, was associated with upfront proceeds of $13.5 million received in connection with the multi-license milestone and royalty monetization transaction with XOMA Corporation in March 2021.
Adjusted loss from operations – Adjusted loss from operations for the year ended December 31, 2021, excluding the non-recurring operating expenses associated with the write-off of in-process research and development acquired in the merger and the termination agreement with Salubris Pharmaceutical Co. Ltd. (a non-GAAP measure) was $25.8 million, compared to a loss from operations of $114.3 million. There is not a comparative adjustment to loss from operations for the same period in 2020.
Net loss – Net loss was approximately $11.4 million, or $0.31 per share (basic and diluted), and approximately $6.3 million, or $13.31 per share (basic and diluted), for the fourth quarters ended 2021 and 2020, respectively. Net loss was approximately $114.8 million, or $3.60 per share (basic and diluted) for the year ended December 31, 2021, compared to a net loss of approximately $19.0 million, or $58.56 per share (basic and diluted) for the year ended December 31, 2020.
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 is key to inducing viral genes that are epigenetically silenced in 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/refractory EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors.

About EBV-Associated Cancers
Approximately 95% of the world’s adult population is infected with Epstein-Barr virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic 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+ lymphomas. EBV is estimated to be associated with approximately 2% of the global cancer burden and is also associated with a variety of solid tumors, including nasopharyngeal carcinoma and gastric cancer.

About Vecabrutinib
Vecabrutinib is a well-tolerated, selective, reversible, non-covalent inhibitor of Bruton’s tyrosine kinase (BTK) and interleukin-2-inducible kinase (ITK). Vecabrutinib is being studied as a potential enhancer of efficacy and safety of CAR T-cell therapy.

On March 16, 2022 Sonnet BioTherapeutics Holdings, Inc. (NASDAQ:SONN) ("Sonnet" or the "Company"), a biopharmaceutical company developing innovative targeted biologic drugs, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for SON-1010, a proprietary version of Interleukin 12 (IL-12) configured using Sonnet’s Fully Human Albumin Binding (FHAB) technology (Press release, Sonnet BioTherapeutics, MAR 16, 2022, View Source [SID1234610175]). This will allow Sonnet to initiate its First-in-Human Phase 1 trial in adult oncology patients in the second quarter of 2022.

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"The FDA’s acceptance of the IND for SON-1010 is an important milestone in the development of our lead FHAB asset, signifying the evolution of Sonnet into a clinical biopharmaceutical company," said Pankaj Mohan, Ph.D., Founder and Chief Executive Officer. "We are excited about the progress we have made with our FHAB platform, which we believe will set the stage for improved efficacy of monospecific and bispecific cytokines, each differentiated by tumor targeting and retention in the tumor microenvironment."

The planned Phase 1 trial will be a multiple ascending dose study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of SON-1010 in adult patients with advanced solid tumors. "We have worked hard to establish a dose range for this extended PK form of IL-12 that can be tested safely and may provide an enhanced therapeutic index," said Richard Kenney, M.D., Sonnet’s Chief Medical Officer. "The goal of this strategy is to carefully adjust the body’s cells and cytokines to enhance the innate immune response to tumors." The study, utilizing a standard 3+3 oncology design in at least 5 cohorts, will establish the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) using monthly subcutaneous injections of SON-1010. The primary endpoint will assess the safety and tolerability of SON-1010, with key secondary endpoints planned to measure PK, PD, immunogenicity, and anti-tumor activity.

On March 16, 2022 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, 2021 (Press release, Sana Biotechnology, MAR 16, 2022, View Source [SID1234610174]).

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"We are pleased with the progress we are making in our pipeline and in building capabilities to execute our vision of exploiting the potential of engineered cells to treat a number of diseases that don’t have effective treatments today," said Steve Harr, Sana’s President and Chief Executive Officer. "In 2021, we meaningfully strengthened our balance sheet, advanced our pipeline giving us the potential for two investigational new drug applications (INDs) in 2022 and multiple INDs per year going forward, built out our supply chain, including commercial access to gene-editing reagents and pluripotent stem cells, and commenced the build-out of our own manufacturing facility. Most importantly, we successfully attracted talent in key business areas, which, combined with the people already inside of the company, give us the capabilities, insights, focus, and dedication to reach our mission for patients."

Recent Corporate Highlights

Demonstrating forward progress in moving toward clinical trials for Sana’s multiple platforms including Sana’s ex vivo hypoimmune allogeneic CAR T, in vivo fusogen CAR T, and stem cell-derived programs:

Continued progress in building Sana’s hypoimmune ex vivo platform
Presented data in non-human primates showing survival and immune evasion, without immune suppression, of transplanted stem cells with Sana’s hypoimmune gene modifications.
Entered into a non-exclusive license and development agreement with FUJIFILM Cellular Dynamics, Inc. (FCDI) for access to FCDI induced pluripotent stem cells (iPSCs).
Gained access to gene editing capability to enable programs within Sana’s allogeneic CAR T and pluripotent stem cell portfolio through non-exclusive license for commercial rights to Beam Therapeutics Inc.’s (Beam) CRISPR Cas12b.
Progressed Sana’s hypoimmune allogeneic CD19-targeted CAR T program, SC291; IND expected as early as this year
Continue to progress on key steps required to advance to clinical trials, including contract manufacturing agreement for Phase I clinical supply, gene-editing reagent access through Beam license, Good Laboratory Practices (GLP) toxicology studies, and Good Manufacturing Practices (GMP) manufacturing processes and scale-up.
Presented data showing that hypoimmune CAR T cells evade both innate and adaptive immune systems in murine models, even in animals with pre-existing immunity to CAR T cells.
Presented data showing that CD19-targeted hypoimmune CAR T cells effectively kill tumor cells in mice and functionally evade the innate and adaptive immune system in allogeneic mouse recipients with either a murine or humanized immune system.
Progressed Sana’s in vivo CAR T program, SG295, utilizing a CD8-targeted fusosome to deliver a CD19-targeted CAR; IND expected as early as this year
Continue to progress on key steps to advance to clinical trials, including contract manufacturing agreement for Phase I clinical supply, GLP toxicology studies, and GMP manufacturing processes and scale-up.
Presented data highlighting ability of a single intravenous administration of a CD8-targeted fusosome containing a CD20-targeted CAR to deplete CD20+ B cells in NHPs.
Presented data highlighting ability of a single intravenous administration of SG295 to eliminate CD19+ tumor cells in mouse tumor models.
Expanded Sana’s CAR T capability to potentially develop best-in-class, broadly accessible CAR T cell therapies
Entered into an exclusive agreement with the National Institutes of Health (NIH) for worldwide commercial rights to the NIH’s CD22 chimeric antigen receptor with a fully-human binder. This CAR construct has shown efficacy in several clinical studies, including in CD19 CAR T cell therapy failures. Targeting both CD19 and CD22 with an "off-the-shelf" product, whether in combination with Sana’s hypoimmune platform or fusogen platform, offers the potential of higher and more durable complete response rates in earlier-stage patients as well as in patients that have previously failed an autologous CD19 CAR T cell therapy.
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, which Sana intends to incorporate into both the company’s ex vivo hypoimmune allogeneic and in vivo fusogen platforms for the treatment of multiple myeloma.
Progressed Sana’s stem cell-derived pancreatic beta cell program, SC451, with potential to treat type 1 diabetes
Presented pre-clinical murine data demonstrating the ability to make stem cell-derived hypoimmune pancreatic islet cells with robust function and hypoimmune pancreatic islet cells that evade immune detection and have the ability to regulate glucose levels.
Established necessary agreements to establish GMP grade cell lines, including FCDI and Beam licenses, and secured contract manufacturing partner for cell bank production.
Remain on track for an IND as early as 2023.
Progressed Sana’s stem cell-derived cardiomyocyte program with the goal of treating heart failure
Presented data that demonstrated four edits in ion channels that alter the electrical properties of pluripotent stem cell-derived cardiomyocytes such that they eliminate engraftment arrythmias in a pig transplant model. These results demonstrate important progress in addressing a key risk associated with transplanting cardiomyocytes into the heart.
Strengthened balance sheet and Board leadership; signed lease to add internal manufacturing capability

Strengthened balance sheet with net proceeds of $626.6 million from the sale of 27 million shares of common stock in the company’s initial public offering.
Expanded Board of Directors with the addition of Joshua Bilenker, M.D., CEO of Treeline Biosciences, Alise Reicin, M.D., CEO of Tectonic Therapeutic, and Michelle Seitz, CFA, Chairman and CEO of Russell Investments.
Announced a lease agreement to develop a manufacturing facility in Fremont, California to support the manufacture of late-stage clinical development and early commercial product candidates across the multiple technologies in the pipeline.
Fourth Quarter 2021 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2021 were $746.9 million compared to $412.0 million as of December 31, 2020, an increase of $334.9 million. The increase was primarily driven by net proceeds of $626.4 million received in Sana’s initial public offering in February 2021, partially offset by cash used in operations of $201.1 million, a one-time upfront cash payment to Beam of $50.0 million to license its genome editing technology, and cash used for the purchase of property and equipment of $29.9 million.

Research and Development Expenses: For the three and twelve months ended December 31, 2021, research and development expenses, inclusive of non-cash expenses, was $108.5 million and $248.6 million, respectively, compared to $36.5 million and $132.9 million, respectively, for the same periods in 2020. The increases of $72.0 million and $115.7 million, respectively, for the three and twelve months ended December 31, 2021 were due to the one-time upfront payment to Beam to license its genome editing technology, an increase in personnel expenses related to increased headcount to expand Sana’s research and development capabilities, costs for laboratory supplies, costs for preclinical studies and external manufacturing, and facility costs. Research and development expenses include non-cash stock-based compensation of $5.3 million and $15.2 million, respectively, for the three and twelve months ended December 31, 2021 and $2.3 million and $4.9 million, respectively, for the same periods in 2020.

Research and Development Related Success Payments and Contingent Consideration: For the three months ended December 31, 2021, we recognized a non-cash gain of $9.9 million, and for the twelve months ended December 31, 2021, we recognized non-cash expense of $57.9 million, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate, compared to expenses of $67.6 million and $124.9 million, respectively, for the same periods in 2020.

General and Administrative Expenses: General and administrative expenses for the three and twelve months ended December 31, 2021, inclusive of non-cash expenses, were $12.7 million and $50.4 million, respectively, compared to $9.2 million and $28.3 million, respectively, for the same periods in 2020. The increases of $3.5 million and $22.1 million, respectively, in the three and twelve months ended December 31, 2021 were primarily due to increased personnel-related expenses attributable to an increase in headcount to build our infrastructure and support our continued research and development activities, legal fees to support our patent portfolio and license arrangements, insurance associated with being a public company, consulting fees, and facility costs. General and administrative expenses include stock-based compensation of $2.0 million and $7.1 million, respectively, for the three and twelve months ended December 31, 2021 and $0.4 million and $0.9 million, respectively, for the same periods in 2020.

Net Loss: Net loss for the three and twelve months ended December 31, 2021 were $110.7 million, or $0.60 per share, and $355.9 million, or $2.14 per share, respectively, compared to $113.2 million, or $7.40 per share, and $285.3 million, or $21.92 per share, respectively, for the same periods in 2020.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the twelve months ended December 31, 2021 was $209.6 million compared to $125.0 million for the same period in 2020. 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 activities, and the purchase of property and equipment.

Non-GAAP Research and Development Expenses: Non-GAAP research and development expenses for the three and twelve months ended December 31, 2021 were $108.5 million and $248.6 million, respectively, compared to $36.5 million and $123.0 million, respectively, for the same periods in 2020. Non-GAAP research and development expenses excludes certain one-time costs to acquire technology.

Non-GAAP Net Loss: Non-GAAP net loss for the three and twelve months ended December 31, 2021 was $120.6 million, or $0.65 per share, and $298.1 million, or $1.79 per share, respectively, compared to $45.5 million, or $2.98 per share, and $150.4 million, or $11.56 per share, respectively, for the same periods in 2020. Non-GAAP net loss excludes certain one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."