On May 8, 2023 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported financial results for the first quarter of 2023 and recent clinical program updates (Press release, Viracta Therapeutics, MAY 8, 2023, View Source [SID1234631183]).

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"With progress from both of our clinical programs accelerating, we have the opportunity to achieve several important milestones this year. NAVAL-1 is enrolling at a rapid pace, and we look forward to readouts across multiple subtypes that could trigger our first advancement into Stage 2 in the months ahead," said Mark Rothera, President and Chief Executive Officer of Viracta. "On the solid tumor front, we are currently seeking to optimize the dose of Nana-val and are pleased to see that its safety profile continues to support further dose escalation into our trial’s fifth dose level. Following the completion of the Phase 1b portion of the trial, we look forward to selecting our recommended Phase 2 dose and advancing our efforts around Nana-val as a potential tumor agnostic therapy for EBV-associated cancers."

Program Highlights and Anticipated Milestones

Pivotal NAVAL-1 trial of Nana-val in relapsed/refractory (R/R) EBV+ lymphoma

NAVAL-1 continues to enroll across each of the trial’s lymphoma subtype cohorts with more than 70 sites open globally.
An update on NAVAL-1’s first lymphoma subtype that may advance from Stage 1 to Stage 2 is anticipated in the second quarter of 2023.
Potential for additional update(s) on other NAVAL-1 lymphoma subtype(s) in the second half of 2023.
Phase 1b/2 trial of Nana-val in patients with recurrent/metastatic (R/M) EBV+ nasopharyngeal carcinoma (NPC) and other advanced EBV+ solid tumors

The trial has advanced into the fifth dose level of the Phase 1b dose escalation portion; no dose-limiting toxicities have been reported to date.
Clinical data previously reported across the first three dose levels (n=10) include one confirmed partial response (PR) at the third dose level and three patients achieving stable disease.
The Company remains on track to report complete Phase 1b dose escalation data and select a recommended Phase 2 dose (RP2D) in the second half of 2023.
Initiation of the trial’s Phase 2 randomized expansion cohort designed to evaluate Nana-val at the RP2D with or without pembrolizumab in patients with R/M EBV+ NPC is expected in the second half of 2023.
Initiation of the trial’s exploratory Phase 1b expansion cohort designed to evaluate Nana-val at the RP2D in patients with other advanced EBV+ solid tumors, including gastric carcinoma, leiomyosarcoma and lymphoepithelioma, is expected in the second half of 2023.
First Quarter 2023 Financial Results

Cash position – Cash, cash equivalents and short-term investments totaled approximately $80.3 million as of March 31, 2023, which Viracta expects will be sufficient to fund its operations into late 2024 excluding any additional borrowing under a $50.0 million credit facility, of which $25.0 million remains available, at the Company’s request, subject to the discretion of the lenders.
Research and development expenses – Research and development expenses were approximately $7.6 million for the three months ended March 31, 2023, compared to approximately $6.1 million for the three months ended March 31, 2022. The increase in research and development expenses 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 in R/R EBV+ lymphoma, and the initiation of our Phase 1b/2 trial for the treatment of EBV+ solid tumors, as well as an increase in personnel-related costs.
General and administrative expenses – General and administrative expenses were approximately $4.6 million for the three months ended March 31, 2023, compared to $4.3 million for the same period in 2022. The increase in general and administrative expenses can be attributed to an increase in personnel-related costs.
Net loss – Net loss was approximately $12.2 million, or $0.32 per share, (basic and diluted) for the quarter ended March 31, 2023, compared to a net loss of $10.5 million, or $0.28 per share (basic and diluted) for the same period in 2022.
About NAVAL-1

NAVAL-1 (NCT05011058) is a global, multicenter trial of Nana-val in R/R EBV+ lymphoma. The trial employs a Simon two-stage design where participants are enrolled into six indication cohorts based on EBV+ lymphoma subtype in Stage 1. If a pre-specified activity threshold is reached within a lymphoma subtype in Stage 1 (n=10), additional patients will be enrolled in Stage 2 for a total of 21 patients. EBV+ lymphoma subtypes demonstrating promising activity in Stage 2 may be further expanded following discussion with regulators to potentially support registration.

About the Phase 1b/2 Trial of Nana-val in R/M EBV+ NPC and Other EBV+ Solid Tumors

This Phase 1b/2 trial (NCT05166577) is an open-label, multinational trial evaluating Nana-val alone and in combination with pembrolizumab. The Phase 1b dose escalation part is designed to evaluate safety and to determine the RP2D of Nana-val in patients with R/M EBV+ NPC. In Phase 2, up to 60 patients with R/M EBV+ NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to evaluate safety, overall response rate, and potential pharmacodynamic markers. 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/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.

On May 8, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultrarare diseases, reported that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, which will be held May 16-20, 2023 at the Los Angeles Convention Center in Los Angeles, California (Press release, Ultragenyx Pharmaceutical, MAY 8, 2023, View Source [SID1234631182]).

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The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.

Clinical and pre-clinical presentations:

Oral presentation: Efficacy and safety at week 52 and up to four years in adults with glycogen storage disease type Ia (GSDIa): Results from a Phase 1/2 clinical trial and long-term follow-up study of DTX401, an AAV-8-mediated, liver-directed gene therapy (Abstract #4)
Date/Time: Thursday, May 18, 8:00 – 8:15 AM PDT
Location: Concourse Hall 152 and 153
First author: Rebecca Riba-Wolman, M.D., University of Connecticut
Oral presentation: Long-term Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #128)
Date/Time: Thursday, May 18, 2:45 PM – 3:00 PM PDT
Location: Room 403 AB
Presenter: Cary Harding, M.D., Oregon Health & Science University
Poster presentation: Immunosuppression to Inhibit Capsid-Specific Humoral Immune Responses in High-dose AAV Gene Therapy in Cynomolgus Macaques (Board No. 1191)
Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Deniz Erturk-Hasdemir, Ph.D., Ultragenyx
Additional presentations including the company’s manufacturing capabilities and Pinnacle PCL (AAV vector producer cell line) platform and technology:

Oral presentation: In Vivo and In Vitro Assessment of Residual DNA Impurity-Derived Transcriptions (Abstract #109)
Date/Time: Thursday, May 18, 1:30 – 1:45 PM PDT
Location: Concourse Hall 150 and 151
Presenter: Hsin-I Jen, Ph.D., Ultragenyx
Poster presentation: Early Development of In Vitro Potency Assays for Rare and Ultrarare Disease Gene Therapy Products (Board No. 782)
Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Elena Balkanska-Sinclair, Ultragenyx

Poster presentation: Global Strategy to Improve the Downstream Manufacturing Process of the Adeno-associated Viral Vector for Glycogen Storage Disease Type Ia Treatment (Board No. 412)

Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Chao Huang, Ph.D., Ultragenyx

Poster presentation: Genome-wide analysis of triple-play plasmid integration in Pinnacle PCL producer cell lines across multiple rAAV programs (Board No. 1162)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Nicholas Richards, M.S., Ultragenyx

Poster presentation: Identification of novel mutations in PCCA and PCCB genes from Propionic Acidemia patient fibroblasts via long-read sequencing (Board No. 972)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Rea Guertler, M.Sc., Ultragenyx

Poster presentation: Development of innovative, scalable, high productivity manufacturing process to enable Ph1/2 clinical supply of UX810, an investigational AAV gene therapy to treat Duchenne’s Muscular Dystrophy (Board No. 1424)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Wei Xue, Ph.D., Ultragenyx

Poster presentation: Development of UX055 AAV Gene Therapy for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD), a Rare Neurological Disorder (Board No. 1340)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Julie Wei, Ph.D., Ultragenyx

Poster presentation: Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy (Board No. 1578)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Kristin Voorhees, Ultragenyx
In addition to the data presentations, Ultragenyx will present at one pre-meeting workshop on Tuesday, May 16:

Pre-meeting workshop: How to Become a Site for AAV Gene Therapy Trials
Time: 8:25-8:50 AM PDT
Location: Concourse Hall 152 and 153
Presenter: Sandra Nino-Siddens, Pharm.D., Ultragenyx

On May 8, 2023 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the quarter ended March 31, 2023 and provided a business update (Press release, Syndax, MAY 8, 2023, View Source [SID1234631181]).

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"Syndax is at an important inflection point with pivotal data readouts and subsequent U.S. registrational filings from each of our two potential first- and best-in-class product candidates expected by year-end," said Michael A. Metzger, Chief Executive Officer. "As we continue to advance our mission to realize a future in which people with cancer live longer and better than ever before, we look forward to reporting topline data from our AGAVE-201 pivotal trial of axatilimab in chronic graft versus host disease (cGVHD) in mid-2023 as well as topline data from our AUGMENT-101 pivotal trial in patients with KMT2A-rearranged (KMT2Ar) acute leukemia in the third quarter of this year."

Recent Pipeline Progress and Anticipated Milestones

Revumenib

The pivotal Phase 2 portion of AUGMENT-101 is enrolling relapsed/refractory (R/R) patients across distinct trial populations: patients with KMT2Ar acute myeloid leukemia (AML), patients with KMT2Ar acute lymphocytic leukemia (ALL) and patients with nucleophosmin mutant (mNPM1) AML. The Company plans to pool data from the AUGMENT-101 cohorts enrolling adult and pediatric patients with R/R KMT2Ar AML and R/R KMT2Ar ALL to file a single New Drug Application (NDA) for the treatment of adult and pediatric KMT2Ar acute leukemia. The Company expects to share topline data from the KMT2Ar pooled analysis in the third quarter of 2023 and submit an NDA filing by the end of 2023. The Company also expects to complete enrollment of the NPM1 AML cohort in the second half of 2023.
The Company has obtained U.S. Food and Drug Administration (FDA) agreement on a recommended Phase 2 dose (RP2D) of 276 mg of revumenib every 12 hours for patients not receiving a strong CYP3A4 inhibitor.
The Company has several Phase 1 trials of revumenib ongoing that will provide data across the treatment landscape in acute leukemia. These include the following:
BEAT-AML: A combination trial of revumenib with venetoclax and azacitidine in front-line AML patients being conducted as part of the Leukemia & Lymphoma Society’s Beat AML master clinical trial. The Company expects to report initial safety data from the trial by year-end 2023.
AUGMENT-102: A combination trial of revumenib with chemotherapy in adult and pediatric patients with R/R mNPM1 or KMT2Ar acute leukemia. The Company expects to report initial safety data along with an RP2D from the trial by year-end 2023.
INTERCEPT: A trial to test the use of revumenib monotherapy as maintenance therapy in AML patients who are minimal residual disease-positive following initial treatment, being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial. The trial continues to enroll patients.
A proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer is enrolling patients and we expect to have preliminary data by year-end 2023.
Axatilimab

The Company and its partner, Incyte, remain on track to report topline data from the pivotal AGAVE-201 trial evaluating axatilimab in patients with cGVHD following two or more prior lines of therapy in mid-2023, with a Biologics License Application (BLA) filing expected by year-end 2023.
The Company plans to present axatilimab data demonstrating encouraging clinical activity in cGVHD-related bronchiolitis obliterans syndrome at the upcoming 2023 American Thoracic Society (ATS) Conference on May 19-24 in Washington, D.C. The abstract is now available on the ATS website.
Syndax and Incyte expect to initiate a Phase 1 trial assessing axatilimab in combination with ruxolitinib in cGVHD in the second half of 2023.
The Company expects to initiate a randomized, double-blind and placebo-controlled Phase 2 trial to assess the efficacy, safety and tolerability of axatilimab in patients with idiopathic pulmonary fibrosis (IPF) in the second half of 2023.
Corporate Updates

In March 2023, the Company announced the appointment of Neil Gallagher, M.D., Ph.D., as President, Head of Research and Development. Dr. Gallagher brings to Syndax over 20 years of experience as a leading oncology drug developer.
Syndax reported the appointment of Kevin McManus as Chief People Officer. Mr. McManus brings to Syndax over 30 years of experience in human resources and an extensive track record in leadership roles growing organizations and attracting great talent.
First Quarter 2023 Financial Results

As of March 31, 2023, Syndax had cash, cash equivalents, short- and long-term investments of $449.0 million and 69.6 million common shares and prefunded warrants outstanding.

First quarter 2023 research and development expenses increased to $34.1 million from $30.0 million for the comparable prior year period. The increase in research and development expenses was primarily due to increased employee-related expenses and professional fees offset by decreased clinical and manufacturing expenses.

First quarter 2023 general and administrative expenses increased to $12.0 million from $6.8 million for the comparable prior year period. The increase is primarily due to employee-related expenses and professional fees.

For the three months ended March 31, 2023, Syndax reported a net loss attributable to common stockholders of $41.1 million, or $0.59 per share, compared to a net loss attributable to common stockholders of $37.2 million, or $0.63 per share, for the comparable prior year period.

Financial Update and Guidance

For the second quarter of 2023, the Company expects research and development expenses to be $38 to $43 million and total operating expenses to be $53 to $58 million. For the full year of 2023, the Company continues to expect research and development expenses to be $160 to $175 million and total operating expenses to be $225 to $240 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, May 8, 2023.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: SNDX1Q23
Domestic Dial-in Number: 800-579-2543
International Dial-in Number: 785-424-1789
Live webcast: https://www.veracast.com/webcasts/syndax/events/SNDX1Q23.cfm

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

On May 8, 2023 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported that Bill Newell, Chief Executive Officer, will present at the JMP Securities Life Sciences Conference on Monday, May 15th, 2023, at 2:00 p.m. ET / 11:00 a.m. PT in New York City (Press release, Sutro Biopharma, MAY 8, 2023, View Source [SID1234631180]).

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The presentation will be accessible through the News & Events page of the Investor Relations section of the company’s website at www.sutrobio.com. An archived replay will be available for at least 30 days after the event.

On May 8, 2023 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported financial results and business highlights for the first quarter 2023 (Press release, Sana Biotechnology, MAY 8, 2023, View Source [SID1234631177]).

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"Our initial human studies using Sana’s hypoimmune technology remain on track, as we have begun enrolling patients in our SC291 trial and expect to deliver data from two clinical studies in 2023," said Steve Harr, Sana’s President and Chief Executive Officer. "We are also making progress in our earlier-stage pipeline and are on pace to file two additional INDs later this year and potentially three more in 2024. The quality of our key hires, publications in high quality peer-reviewed journals, and presentations at important scientific conferences are recent validations of the science behind Sana’s programs. Our capital position and people give us the resources for multiple data read-outs with our current balance sheet, and we continue to be optimistic about our future, with the opportunity to see the potential of these medicines in patients starting this year."

Recent Corporate Highlights

Opportunity for clinical proof of concept for two different first-in-human studies, each with the potential for initial clinical data this year

SC291 is an ex vivo hypoimmune-modified CD19-directed allogeneic CAR T cell therapy. The goal of the hypoimmune platform is to overcome the immunologic rejection of allogeneic cells, which, if successful with SC291, may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell malignancies.
Received clearance from the Food and Drug Administration (FDA) to initiate a first-in-human Phase 1 study of SC291 in patients with B-cell malignancies (ARDENT).
Began enrollment in the ARDENT Phase 1 study.
Granted Fast Track Designation for SC291 by the FDA for the treatment of relapsed/refractory (r/r) large B-cell lymphoma and r/r chronic lymphocytic leukemia.
SC291 has the potential to serve as clinical proof-of-platform for other hypoimmune-modified CAR T cell candidates using validated CAR constructs in development at Sana for hematological malignancies, such as SC262 (CD22) and SC255 (BCMA). Sana’s goal is to file INDs for SC262 later this year and SC255 in 2024.
Sana is developing SC451, a hypoimmune-modified stem-cell derived islet cell therapy for patients with type 1 diabetes. SC451, which is engineered with Sana’s hypoimmune technology, has the potential to replace missing islet cells without immunosuppression in persons with type 1 diabetes by evading allogeneic and autoimmune responses.
Expect initial data later this year from an investigator-sponsored trial transplanting hypoimmune-modified primary human islet cells into type 1 diabetes patients. The goal of the study is to show cell survival and immune evasion without the need for any immunosuppression.
Sana’s goal is to file an IND for SC451 in 2024.
Published preclinical data in Nature Communications describing immune evasion, persistence, and durable anti-tumor activity of Sana’s hypoimmune-modified CD19-directed CAR T cells

Sana developed hypoimmune-modified CD19 targeted allogeneic CAR T cells and compared them to unmodified CD19-targeted allogeneic CAR T cells in a murine leukemia model with a humanized immune system.
Although both hypoimmune-modified and unmodified CAR T cells showed robust early tumor killing, cell durability was much greater in humanized mice treated with hypoimmune-modified cells. Hypoimmune-modified allogeneic CAR T cells persisted and removed all evidence of tumor for the duration of the study. Hypoimmune-modified CAR T cells also cleared all evidence of tumor after re-injection with cancer cells 90 days into the study. In contrast and consistent with the experience in patients to date, unmodified allogeneic CAR T cells show greatly reduced persistence and a high rate of tumor recurrence in this model.
These studies provide additional insight for SC291 and the allogeneic hypoimmune CAR T platform more broadly, including SC262 and SC255.
Published preclinical data in Science Translational Medicine demonstrating that Sana’s hypoimmune-modified pseudo-islets control type 1 diabetes

Sana developed hypoimmune-modified human islet cells, which cluster into effective endocrine organoids termed "pseudo islets" (p-islets) and studied these p-islets in multiple preclinical models.
Preclinical data showed that p-islets survive, persist, escape allogeneic rejection, and normalize blood glucose in diabetic models with humanized immune systems.
Two different murine models showed that the hypoimmune-modified cells can evade autoimmune rejection and normalize blood glucose. First, these cells were studied in the NOD mouse model, which is the standard model for autoimmunity in diabetes. Second, Sana created a humanized mouse model with immune cells from a diabetic person and transplanted pancreatic islet cells derived from the diabetic person’s stem cells. In both cases, unmodified pancreatic islet cells were rapidly cleared by the immune system. In contrast, hypoimmune-modified pancreatic islet cells survived, persisted, and provided sustained blood glucose control in both models.
These studies provide additional insight for SC451 in persons with type 1 diabetes.
Published preclinical data in Nature Biotechnology demonstrating that Sana’s hypoimmune-modified cells survive allogeneic transplant across several species, including non-human primates (NHPs) with normal immune systems, and remain fully functional

Sana developed hypoimmune-modified NHP induced pluripotent stem cells (iPSCs) and transplanted them into immune-competent NHPs. Results were compared to transplantation of unmodified iPSCs into immune-competent NHPs.
Data showed that hypoimmune-modified iPSCs survived for the duration of the study (16 weeks), while unmodified iPSCs disappeared within two weeks. There was an antibody and T cell response directed toward unmodified cells, but not hypoimmune-modified cells.
Hypoimmune-modified primary NHP pancreatic islet cells survived 40 weeks (duration of the study) after allogeneic transplantation into an immune-competent NHP versus less than one week for unmodified primary islet cells.
Hypoimmune-modified iPSCs were differentiated into pancreatic islet cells. Transplantation of hypoimmune-modified iPSC-derived pancreatic cells into allogeneic diabetic mice with a humanized immune system showed immune evasion after transplantation for the duration of the studies (4 weeks) and amelioration of diabetes and normalization of blood glucose levels.
Presented multiple abstracts at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting highlighting ex vivo hypoimmune-modified allogeneic CAR T cells, as well as in vivo cell-specific delivery of genetic material using Sana’s in vivo fusogen platform

Presented preclinical data demonstrating that hypoimmune-modified CAR T cells provide lasting tumor control in immunocompetent allogeneic humanized mice even with tumor re-challenge.
Presented preclinical data in a late-breaking poster presentation demonstrating that the increased potency of CD8-targeted fusosomes enhances CAR transgene delivery to resting primary T cells.
Presented preclinical data demonstrating the effectiveness of Sana’s fully human CD19 CAR delivered by CD8-targeted fusosomes in tumor killing assays. These fusosomes led to similar levels of tumor control as ex vivo generated CD19 CAR T cells.
Presented preclinical data demonstrating increased potency of CD8-targeted fusosomes delivering a CD19 CAR with pre-treatment of resting T cells with IL-7, rapamycin, or both. Pre-treatment with these molecules led to increased anti-tumor efficacy through increased T cell transduction and greater CAR T cell expansion.
Strengthened Research and Development leadership with the appointment of two seasoned drug developers

Appointed Doug Williams, Ph.D., as President of Research and Development. Dr. Williams has over 30 years of experience leading R&D organizations – including at Biogen, Seattle Genetics (now Seagen), Amgen, and Immunex – and over the course of his career has participated in the development of over a dozen approved drugs including multiple blockbusters.
Appointed Gary Meininger, M.D., as Chief Medical Officer. Dr. Meininger has approximately 20 years of experience in drug development. Most recently, he was at Vertex as Senior Vice President, Head of Clinical Development for Vertex Cell and Genetic Therapies and previously was at Janssen and Merck. Dr. Meininger is currently the industry representative to the FDA’s Endocrine and Metabolic Drug Advisory Committee.
First Quarter 2023 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2023 were $355.1 million compared to $434.0 million as of December 31, 2022. The decrease of $78.9 million was primarily driven by cash used in operations of $79.2 million and cash used for the purchase of property and equipment of $2.2 million. Cash used in operations includes multiple cash payments that will not recur this year. In addition, our cash balance will increase by $6.1 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 months ended March 31, 2023, research and development expenses, inclusive of non-cash expenses, were $67.2 million compared to $72.7 for the same period in 2022. The decrease of $5.5 million was primarily due to a decline in costs to acquire technology, laboratory supplies, third-party manufacturing costs, and other research costs. These decreases were partially offset by increased clinical development costs, personnel-related costs, operating costs for our manufacturing facility in Bothell, Washington, and other allocated costs. Research and development expenses include non-cash stock-based compensation of $6.0 million and $5.7 million, for the three months ended March 31, 2023 and 2022, respectively.

Research and Development Related Success Payments and Contingent Consideration: For the three months ended March 31, 2023, we recognized an expense of $0.1 million and a gain of $55.4 million for the same period in 2022, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. 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 March 31, 2023, inclusive of non-cash expenses, were $16.8 million compared to $14.4 million for the same period in 2022. The increase of $2.4 million was primarily due to operating costs for the previously planned manufacturing facility, formerly in research and development expense, and increased non-cash stock-based compensation and personnel-related expenses. General and administrative expenses include non-cash stock-based compensation of $2.8 million and $2.0 million, for the three months ended March 31, 2023 and 2022, respectively.

Net Loss: Net loss for the three months ended March 31, 2023 was $82.1 million, or $0.43 per share, compared to $31.4 million, or $0.17 per share, for the same period in 2022.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the three months ended March 31, 2023 was $74.8 million compared to $82.0 million for the same period in 2022. 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 Net Loss: Non-GAAP net loss for the three months ended March 31, 2023 was $82.0 million, or $0.43 per share, compared to $86.9 million, or $0.47 per share, for the same period in 2022. Non-GAAP net loss excludes 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."