On May 9, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported financial results for the first quarter ended March 31, 2023, and provided recent business updates (Press release, Sensei Biotherapeutics, MAY 9, 2023, View Source [SID1234631293]).

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"We are pleased with the progress across our pipeline, underscored by regulatory clearance to proceed with a Phase 1/2 clinical study of our lead candidate, SNS-101, in patients with advanced solid tumors. We look forward to commencing this trial in mid-2023 and expect to move swiftly to a recommended Phase 2 dose," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "We are also maintaining superb progress and promising results from our other pipeline programs, each with breakthrough potential within their target classes, as we advance toward key decision points later this year."

Highlights and Milestones

SNS-101

Sensei continues to advance SNS-101, a conditionally active antibody targeting the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation), which is implicated in resistance to cancer immunotherapy and whose expression correlates with poor survival across numerous cancers. Recent updates for SNS-101 include:

In April 2023, the U.S. Food and Drug Administration (FDA) cleared Sensei’s Investigational New Drug (IND) application for the planned Phase 1/2 clinical trial of SNS-101 in patients with advanced solid tumors. The Phase 1/2 clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SNS-101 as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in advanced solid-tumor cancer patients. Sensei expects to begin clinical trial enrollment and commence patient dosing at a starting dose of 0.3 mg/kg in mid-2023.
In February 2023, Sensei CSO Edward van der Horst presented key preclinical data supporting the mechanism of SNS-101 at the Keystone Symposia on Next Generation Antibody Therapeutics.
Under the recently signed CRADA with the National Cancer Institute (NCI), part of the National Institutes of Health, in February 2023, preclinical studies are in progress with the goal of further elucidating VISTA’s role in immune checkpoint resistance and expanding the potential of SNS-101 as a combination therapy beyond anti-PD-1. In addition, preparations are underway for the NCI to participate as a trial site in the clinical investigation of SNS-101.
In a multi-dose GLP toxicology study, SNS-101 was well tolerated and displayed a favorable multi-dose pharmacokinetic profile, with linear elimination kinetics and an absence of target-mediated drug disposition.
Additional TMAb Platform Updates

Through its Tumor Microenvironment Activated biologics (TMAb) platform, Sensei is also advancing several conditionally active antibody programs, including SNS-102 targeting VSIG4 (V-Set and Immunoglobulin Domain Containing 4), SNS-103 targeting ENTPDase1 (ecto-nucleoside triphosphate diphosphohydrolase-1, also known as CD39) and a recently initiated fourth program.

SNS-102: Eight parental pH-sensitive VSIG4 antibodies have been selected, which have undergone further lead optimization and are currently being characterized.
SNS-103: In April 2023, Sensei presented new preclinical data on SNS-103 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Eight parental pH-sensitive CD39 antibodies have been selected, which have undergone further lead optimization and are currently being characterized.
Sensei remains on track to select product candidates for both SNS-102 and SNS-103 in 2023.
Sensei has initiated early discovery efforts for a fourth TMAB program focused on developing a conditionally active bispecific antibody.
Upon successful candidate selection, Sensei expects to advance one product candidate to IND-enabling studies.
First Quarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $95.5 million as of March 31, 2023, as compared to $107.1 million as of December 31, 2022. Sensei expects its current cash balance to fund operations into the second half of 2025.

Research and Development (R&D) Expenses: R&D expenses were $5.2 million for the quarter ended March 31, 2023, compared to $7.5 million for the quarter ended March 31, 2022. The decrease in R&D expenses was primarily attributable to lower personnel costs due to the restructuring, lower expense relating to lab supply purchases and lower manufacturing related expense partially offset by higher expense associated with clinical trials.

General and Administrative (G&A) Expenses: G&A expenses were $5.8 million for the quarter ended March 31, 2023, compared to $5.0 million for the quarter ended March 31, 2022. The increase in G&A expense was primarily attributable to external professional services, including $1.5 million of non-recurring expenses associated with stockholder activism related to our upcoming 2023 annual meeting of stockholders.

Net Loss: Net loss was $10.2 million for the quarter ended March 31, 2023, compared to $12.4 million for the quarter ended March 31, 2022.

On May 9, 2023 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results and corporate updates for the first quarter ended March 31, 2023 (Press release, Scholar Rock, MAY 9, 2023, View Source [SID1234631292]).

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"We are executing across our business and leveraging Scholar Rock’s differentiated platform, which selectively targets latent forms of growth factors, such as myostatin and TGFβ1, to develop highly selective and potentially transformative therapies for patients who have high unmet medical need," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. "We continue to advance our two clinical programs in spinal muscular atrophy and oncology, and we look forward to sharing updates on our progress as we approach several key milestones, including presenting 36-month data on apitegromab from the Phase 2 TOPAZ trial, completing enrollment of our apitegromab Phase 3 SAPPHIRE trial, and providing biomarker and clinical updates from our SRK-181 Phase 1 DRAGON trial."

Recent Company Highlights and Upcoming Milestones

Apitegromab is an investigational antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin and is being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).

Continued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across sites in the U.S. and Europe. Enrollment completion is expected in 2023, with the top-line data readout expected in 2024. If successful and if approved, the company expects to initiate a commercial product launch in 2025.

36-month data from Phase 2 TOPAZ trial to be presented at Cure SMA conference in June. The company announced two upcoming oral presentations at the Cure SMA Research & Clinical Conference, taking place June 28-30 in Orlando, Florida.

24-month data from the TOPAZ trial presented at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March. The company shared data evaluating outcomes after 24 months of treatment with apitegromab, demonstrating sustained improvements in motor function in patients with nonambulatory Types 2 and 3 SMA.

SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Advancing Phase 1 DRAGON proof-of-concept trial. In the second half of 2023, the company expects to provide biomarker and clinical updates from Part B of the DRAGON trial. In March 2023, Scholar Rock presented encore data at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress, which showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of December 2, 2022).
Corporate

Announced the addition of Richard Brudnick to the Board of Directors. In April 2023, Mr. Brudnick joined Scholar Rock’s Board of Directors. As an accomplished biotechnology executive, he brings an extensive background in corporate development and strategy, with over 30 years of industry experience across multiple specialties, stages and therapeutic areas.

First Quarter 2023 Financial Results

For the quarter ended March 31, 2023, net loss was $39.4 million or $0.49 per share compared to a net loss of $8.0 million or $0.21 per share for the quarter ended March 31, 2022.

Revenue was $0 for the quarter ended March 31, 2023, compared to $33.2 million for the quarter ended March 31, 2022. The prior year revenue was related to the Gilead fibrosis-focused research collaboration, which was executed in December 2018 and concluded in December 2021.

Research and development expense was $29.7 million for the quarter ended March 31, 2023, compared to $29.4 million for the quarter ended March 31, 2022. The increase was primarily attributable to costs associated with clinical trials, including the Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, and the DRAGON trial for SRK-181. These increases were offset by decreases in employee compensation and benefits costs, resulting from the restructuring in May 2022.

General and administrative expense was $10.8 million for the quarter ended March 31, 2023, compared to $10.8 million for the quarter ended March 31, 2022.
As of March 31, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $275 million, which is expected to fund the company’s anticipated operating and capital expenditure requirements into 2025.

"Scholar Rock is in a strong financial position, and we are relentlessly focused on achieving our goal of improving the lives of patients who may potentially benefit from our medicines," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We continue to execute on our plan by advancing our clinical programs towards key inflection points, while diligently managing our resources."

On May 9, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2023 (Press release, Repare Therapeutics, MAY 9, 2023, View Source [SID1234631291]).

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"We continue to execute clinically and across our pipeline programs, including presenting initial clinical data of camonsertib in combination with various PARP inhibitors from the ongoing TRESR and ATTACC trials at this year’s AACR (Free AACR Whitepaper) conference," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "These findings are consistent with our preclinical data demonstrating that low, intermittent dosing of the camonsertib and PARP inhibitor combinations appears well tolerated and exhibited antitumor activity, most notably in advanced ovarian cancer. In addition, we are on track to present initial Phase 1 monotherapy data for RP-6306 in June."

First Quarter 2023 Review and Operational Updates:

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Announced initial clinical data from the Phase 1/2 TRESR and ATTACC trials evaluating camonsertib (RP-3500/RG6526, partnered with Roche), a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), in combination with three poly (ADP-ribose) polymerase (PARP) inhibitors in a Clinical Trials Plenary Session at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
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Camonsertib combinations appear to be well tolerated. Dose limiting toxicity in 68 patients treated with the proposed combination doses were related to myelotoxicity (Grade 3+ anemia 3%, thrombocytopenia 6%, neutropenia 7%, and febrile neutropenia 3%). No prophylactic growth factors were required when administering the PARP inhibitors at evaluated doses.
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Camonsertib combination resulted in durable clinical benefit across tumor types and genomic alterations, regardless of choice of PARP inhibitor and presence of platinum resistance. Overall clinical benefit rate (CBR) for all patients was 48%. Patients with platinum-resistant tumors had an overall response rate (ORR) of 12% and CBR of 49% and benefited similarly to non-platinum-resistant tumors (ORR 13%, CBR 46%).

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Compelling results were observed particularly in patients with advanced ovarian cancer (n = 19). In these patients, overall response was 32%, CBR was 58% and median progression-free survival was approximately 7 months with treatment greater than 16 weeks and ongoing in 9 patients.
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Early circulating tumor DNA molecular responses in 66% (31/47) of evaluable patients confirm antitumor activity of low dose, intermittent PARP inhibitor + ATR inhibitor therapy. The molecular response rate (MRR) was significantly higher in patients with clinical benefit (83%) compared to those without (48%; p=0.015) and significantly higher than camonsertib monotherapy that was also administered at higher doses (43% or 27/63; p=0.02). Molecular responses were also observed in patients with prior PARP inhibitor exposure (57%) and platinum resistance (64%).
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Repare is conducting dose optimization and efficacy assessments in tumor specific expansions in the ATTACC study in collaboration with Roche to support future clinical development plans for camonsertib combinations with PARP inhibitors.
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Evaluating RP-6306, a first-in-class, oral PKMYT1 inhibitor as a monotherapy and in combinations in multiple early clinical studies.
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Repare presented two poster presentations for RP-6306 at the 2023 AACR (Free AACR Whitepaper) Annual Meeting regarding the co-mutation landscape in CCNE1 amplifications and the tumor heterogeneity of copy number in ovarian and uterine cancers. Additionally, several collaborators presented preclinical findings on the potential benefits of combining a Wee1 inhibitor with RP-6306 and the effect of RP-6306 in triple negative breast cancer.
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Repare expects to report initial Phase 1 monotherapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors (MYTHIC) in June 2023. The Company expects to report initial Phase 1 combination therapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors in the fourth quarter of the year.
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Repare is working with clinical investigators to initiate clinical testing, as part of an investigator-sponsored trial (IST), of a fourth RP-6306 combination with carboplatin, with first patient dosing expected this year.
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Repare is collaborating with the Canadian Cancer Trials Group for a basket Phase 2 IST to evaluate RP-6306 in patients with selected, advanced cancers receiving standard agents that is expected to begin this year. A sub-study under the master clinical trial protocol will evaluate RP-6306 in combination with gemcitabine in patients with CD4/6i-resistant ER+/HER2- metastatic breast cancer.
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Advancing preclinical programs into clinical development.
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Repare initiated IND-enabling studies in the first half of this year for a small molecule, now designated RP-1664, against an undisclosed target with potential to enter the clinic in late 2023 or early 2024.
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Repare is also pursuing development of an inhibitor of polymerase theta (Polθ) that is expected to enter the clinic in 2024.
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In April 2023, Repare announced the appointment of Susan Molineaux, Ph.D., to its Board of Directors, effective as of the date of the Company’s upcoming annual meeting of shareholders in June 2023. Concurrent with Dr. Molineaux’s appointment as of the date of the annual meeting, Jerel

Davis, Ph.D., Managing Director at Versant Ventures and a founding member of Repare’s Board of Directors, will step down from the Board. Additionally, Repare has expanded the senior leadership team with the appointment of Daniel Belanger as EVP Human Resources in May 2023.
First Quarter 2023 Financial Results:

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Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of March 31, 2023 were $314.1 million.
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Revenue from collaboration agreements: Revenue from collaboration agreements were $5.7 million and $0.4 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in revenue was due to revenue recognized from our collaboration and license agreement with Roche.
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Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $31.8 million and $26.5 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in Net R&D expenses was primarily due to higher personnel related costs from headcount in support of our development activities, and direct external costs related to the advancement of preclinical programs into IND-enabling studies.
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General and administrative (G&A) expenses: G&A expenses were $8.6 million and $8.8 million for three months ended March 31, 2023 and 2022, respectively. The year-over-year decrease in G&A was primarily due to lower professional fees associated with our collaboration and license agreement with Roche and lower D&O insurance premiums, offset by higher personnel related costs.
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Net loss: Net loss was $34.9 million, or $0.83 per share, in the three months ended March 31, 2023, and $34.8 million, or $0.83 per share in the three months ended March 31, 2022.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

On May 9, 2023 Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultrasensitive biomarker detection, reported financial results for the three months ended March 31, 2023 (Press release, Quanterix, MAY 9, 2023, View Source [SID1234631290]).

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"Our corporate transformation initiated in August 2022 to maximize Quanterix’s full potential is on track. We remain confident that we have taken the right steps to build a strong foundation in preparation for future growth," said Masoud Toloue, President and Chief Executive Officer of Quanterix. "This progress takes on added importance with the FDA’s recent decision to approve tofersen for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis, in large part based on strong surrogate neurofilament light chain biomarker data. This decision adds significant momentum to the use of biomarkers for predicting disease severity and clinical benefit in neurodegenerative diseases, and we believe Quanterix will continue to be at the forefront of these developments."

First Quarter 2023 Financial Highlights

● Q1 total revenue was $28.5 million versus prior year Q1 of $29.6 million, a decrease of 4% driven by a decline in instrument revenue. Q1 total revenue increased 10% from Q4 2022 driven by increased consumable revenue.
● Gross margin, a key success indicator of the strategic realignment, saw strong quarter over quarter improvement. Q1 2023 GAAP gross margin was 59.5% versus Q1 2022 GAAP gross margin of 49.3% and Q4 2022 GAAP gross margin of 48.8%. Q1 2023 non-GAAP gross margin was 53.1% versus Q1 2022 non-GAAP gross margin of 43.2% and Q4 2022 non-GAAP gross margin of 41.3%.
● Q1 net loss was $6.1 million versus prior year Q1 of $18.2 million, a decrease of 66%.
● Cash burn for Q1 was $9.1 million, leaving us with $329.4 million of unrestricted cash as of March 31, 2023.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below.

2023 Business Highlights

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In January 2023, the Company expanded its lab developed test (LDT) menu with the launch of an NfL LDT, which can be used as an aid in the evaluation of individuals for possible neurodegenerative conditions or other causes of neuronal or central nervous system damage. Quanterix’s Simoa NfL is the most widely published NfL test with hundreds of research papers, demonstrating its validity for assessing neuronal damage, and Simoa NfL has become widely adopted in therapeutic clinical trial designs. We believe the recent FDA approval of tofersen, which relied on neurofilament light chain biomarker data, also validates the importance of this test.

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The Company participated in the 2023 Alzheimer’s Disease/Parkinson’s Disease conference in Gothenburg Sweden. Simoa-based biomarkers were highlighted in more than 11 panel discussions and over 30 poster presentations.

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Published discoveries enabled through Quanterix’s Simoa technology continue to illustrate industry reliance on the Company’s ultra-sensitive technology for breakthrough discovery in research and clinical applications. The technology was highlighted in more than 140 new publications in the first quarter 2023, bringing total Simoa-specific inclusions to over 2,200 as of March 31, 2023.

Full Year Business Outlook

We have made a modest increase to our guidance for the full year 2023. We expect revenues to be in the range of $104 to $111 million. We expect GAAP gross margin percentage to be in the high 40s and non-GAAP gross margin percentage to be in the mid 40s. Expected cash burn for 2023 is unchanged, and we expect an approximately 10% improvement over 2022.

Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 9, 2023 at 4:30 p.m. Eastern Time. Individuals interested in listening to the conference call may do so by pre-registering here and obtaining a dial-in number and passcode.

A live webcast will also be available at: View Source You may also access the live webcast by visiting the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. The webcast will be available on the Company’s website for one year following completion of the call.

On May 9, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported financial results for the first quarter ended March 31, 2023 and provided a business update (Press release, Precision Biosciences, MAY 9, 2023, View Source [SID1234631289]).

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"Our progress in the first quarter of 2023 makes it clear that we are focused on the generation of meaningful clinical data from our CAR T programs while working in parallel to advance our broad in vivo portfolio into the clinic as soon as possible," said Michael Amoroso, Chief Executive Officer at Precision BioSciences. "We continue to collect data from our latest patient cohorts for both our azer-cel and PBCAR19B programs and anticipate providing an update once we have sufficient follow up from these cohorts. We expect that these data will inform key next steps for development and potential regulatory interactions."

"From our in vivo portfolio, we continue to leverage the core features of ARCUS that we believe drive high efficiency gene insertion and the ability to make large gene excisions while advancing programs where we see a clear and rapid path to the clinic. Preclinical data presentations at the Global Hepatitis Summit 2023 on hepatitis B virus (HBV) and an upcoming, late breaker oral presentation on Duchenne muscular dystrophy (DMD) at the American Society of Gene & Cell Therapy Meeting highlight key execution steps that have brought ARCUS closer to the clinic. We look forward to providing further updates across our wholly owned and partnered gene editing programs at our upcoming in vivo gene editing R&D Day in the middle of the year," added Mr. Amoroso.

Ex Vivo Allogeneic CAR T Portfolio:

Azer-cel: Precision continues to progress its Phase 1/2a clinical trial of azer-cel, the Company’s lead investigational anti-CD19 allogeneic CAR T candidate, in adult subjects with non-Hodgkin lymphoma (NHL) who have relapsed following CAR T treatment. Precision plans to provide an update on the expansion cohort as well as additional long-term follow up from the previously presented azer-cel cohorts in May 2023. As of May 1, 2023, seven patients have been treated in the expansion/Phase 2a cohort with a lower dose lymphodepletion regimen, in addition to the 12 patients who were treated in previous cohorts.

PBCAR19B: Precision continues to progress the ongoing Phase 1 study of PBCAR19B, its second generation, anti-CD19 targeting allogeneic CAR T candidate designed to evade immune rejection by host T cell and natural killer (NK) cells with a single-gene edit to knock-down beta-2 microglobulin and insert an HLA-E transgene. The Company also expects to provide a program update on seven patients treated at Dose Level 2 in May 2023.

In Vivo Gene Editing Portfolio:

Chronic Hepatitis B Virus (HBV): Precision is prioritizing nomination of a development candidate for its wholly owned PBGENE-HBV in vivo program, with the goal of submitting a Clinical Trial Application (CTA) and/or Investigational New Drug Application (IND) in 2024. An abstract titled "Targeting Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo" was presented in April 2023 at the Global Hepatitis Summit 2023. Data showed that ARCUS demonstrated a high degree of on-target antiviral activity, as measured by substantial reductions of both intracellular cccDNA and secretion of HBsAg, with no detectable translocations in ARCUS-treated primary human hepatocytes. These data support the further development of PBGENE-HBV with the goal of developing a functional cure for HBV. The Company plans to present additional data in 2023.

Novartis Partnered Program: Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic anti-sickling gene as a potential one-time transformative treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, including those targeting an ex vivo gene editing approach.

Prevail, a wholly-owned subsidiary of Lilly, Partnered Programs: Precision continues its in vivo gene editing collaboration with Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company (Lilly), in applying ARCUS nucleases to three initial targets, including DMD in muscle, a central nervous system directed target, and a liver directed target. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV, that are designed to excise an approximately 500,000 base pair mutation "hot spot" region from the dystrophin gene to generate a variant of the dystrophin protein that is functionally competent.

In April 2023, Precision announced that a late-breaking abstract featuring preclinical data from its PBGENE-DMD program for the potential treatment of DMD was selected for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting being held May 16-20, 2023 in Los Angeles, CA.

Ornithine Transcarbamylase (OTC) Deficiency: Led by iECURE, an ARCUS-mediated gene insertion approach is being pursued as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Non-human primate (NHP) data presented by researchers from the University of Pennsylvania’s Gene Therapy Program demonstrated sustained gene insertion of a therapeutic OTC transgene one-year post-dosing in newborn and infant NHP with high efficiency. iECURE is targeting to submit a CTA and/or IND in the second half of 2023.

Other ARCUS Research: Preclinical data presented at the Keystone Symposium in March 2023 demonstrated the unique potential of ARCUS nucleases to achieve high frequency gene insertion in nondividing primary hepatocytes. These data highlight the potential advantage of ARCUS for use in developing therapeutics for gene addition, for which Precision believes ARCUS is highly differentiated given the need for extremely low levels of off-target editing and high insertion efficiency.

Corporate:

Intellectual Property (IP) Update: In February 2023, the U.S. Patent and Trademark Office issued Precision a Notice of Allowance for an application with composition of matter claims covering a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has been used preclinically for both gene deletion approaches for cardiovascular diseases such as familial hypercholesterolemia as well as a viable and safe site to insert a functional copy of a specific gene to restore function addressing other diseases requiring gene addition. This patent further builds on the Company’s IP portfolio that cover the ARCUS platform and its use developing novel ex vivo and in vivo gene editing therapies.

Quarter Ended March 31, 2023 Financial Results:

Cash and Cash Equivalents: As of March 31, 2023, Precision had approximately $158 million in cash and cash equivalents. In line with the same period for 2022, net cash used in operating activities for the quarter ended March 31, 2023 are expected to be higher in the first fiscal quarter than the remainder of 2023 primarily due to the timing of the Company’s annual compensation cycle. The Company expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements through the first quarter of 2025.

Revenues: Total revenues for the quarter ended March 31, 2023 were $8.8 million, as compared to $3.3 million for the same period in 2022. The increase of $5.5 million in revenue during the quarter ended March 31, 2023 was primarily driven by revenue recognized under the Novartis agreement as work thereunder began in the third quarter of 2022.

Research and Development Expenses: Research and development expenses were $22.2 million for the quarter ended March 31, 2023, as compared to $20.0 million for the same period in 2022. The increase of $2.2 million was primarily due to an increase in direct research and development expenses related to our azer-cel and PBCAR19B product candidates as well as consulting fees related to our increased focus on quality management, partially offset by a decrease in expenses related to manufacture of materials for use in preclinical studies, PBCAR20A external development costs, and share-based compensation expense from recent forfeitures.

General and Administrative Expenses: General and administrative expenses were $11.1 million for the quarter ended March 31, 2023, as compared to $10.7 million for the same period in 2022. The increase of $0.4 million was primarily related to consulting fees related to in vivo program competitive landscape analyses, partially offset by a decrease in insurance expense.

Net Loss: Net loss was $25.1 million, or $(0.23) per share (basic and diluted), for the quarter ended March 31, 2023, as compared to a net loss of $28.2 million, or $(0.46) per share (basic and diluted), for the same period in 2022. Weighted average shares of common stock outstanding were approximately 111.3 million for the quarter ended March 31, 2023, as compared to approximately 61.0 million for the quarter ended March 31, 2022. The increase in weighted average shares of common stock outstanding was primarily due to a $50 million underwritten offering of common stock and Novartis’ $25 million equity investment in 2022.