On May 9, 2024 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported operational highlights and financial results for the first quarter ended March 31, 2024 (Press release, Intellia, MAY 9, 2024, View Source [SID1234642994]).

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"Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies. With one ongoing and two soon-to-be-initiated pivotal Phase 3 trials, Intellia is undoubtedly at the forefront of a new era in medicine," said Intellia President and Chief Executive Officer John Leonard, M.D. "We have been extremely pleased with the speed of enrollment in the Phase 3 MAGNITUDE trial for patients with ATTR amyloidosis with cardiomyopathy, which is tracking ahead of our initial projections. In addition, we now expect to start a pivotal Phase 3 trial of NTLA-2001 for patients with polyneuropathy by year-end, based on productive discussions with the FDA. Moving to NTLA-2002, we expect to report key data readouts from the Phase 1/2 study this year. These data will support the dose selection for the Phase 3 trial and highlight what we believe is the potential for NTLA-2002 to dramatically change the HAE treatment paradigm. Building on our success with in vivo gene inactivation, we are excited to initiate the first-in-human study of NTLA-3001 for AATD this year, positioning us to be the first to clinically validate CRISPR-based gene insertion. We look forward to continuing our strong execution, with many notable milestones to mark the progress against our strategic priorities."

First Quarter 2024 and Recent Operational Highlights

Transthyretin (ATTR) Amyloidosis

NTLA-2001: NTLA-2001 is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in the liver and thereby prevent the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Intellia leads development and commercialization of NTLA-2001 in collaboration with Regeneron.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
The pivotal Phase 3 MAGNITUDE trial is rapidly enrolling. In March, the first patients in the U.S. and globally were dosed. Enrollment is currently tracking well ahead of the Company’s initial projections, with over 30 patients dosed to date and more than 40 additional patients in screening. Many additional sites are expected to open in the weeks and months ahead to further accelerate enrollment.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
Intellia announced today alignment with the U.S. Food and Drug Administration (FDA) on a pivotal Phase 3 trial design to support a biologics license application (BLA) filing for NTLA-2001 as a single-dose treatment for people living with ATTRv-PN, subject to review of its investigational new drug (IND) application. The study is expected to be a small, placebo-controlled trial conducted at ex-U.S. sites with approximately 50 ATTRv-PN patients. The Company plans to initiate the study by year-end.
The Company plans to present updated data from the ongoing Phase 1 study in the second half of 2024.
Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
Intellia plans to initiate the global pivotal Phase 3 study, including U.S. patients, in the second half of 2024, subject to regulatory feedback.
The Company will present updated data from the Phase 1 study at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, taking place May 31 – June 3 in Valencia, Spain. Long-term data from the Phase 1 portion of the Phase 1/2 study will include safety, kallikrein reduction and attack rate data, including number of patients who continue to be completely attack free through the latest follow-up. Additionally, Intellia plans to report topline results from the Phase 2 portion in mid-2024 and present full results at a medical meeting in the second half of 2024.
In January, landmark findings from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published in the New England Journal of Medicine (NEJM).
Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

NTLA-3001: NTLA-3001 is a first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert the wild-type SERPINA1 gene, which encodes the alpha-1 antitrypsin (AAT) protein, with the potential to restore permanent expression of fully functional AAT protein to normal levels after a single dose. This is Intellia’s first wholly owned gene insertion program.
Intellia expects to dose the first patient in a Phase 1 study of NTLA-3001 in 2024.
In Vivo Platform Expansion

Intellia is expanding the range of diseases that can be targeted with its CRISPR-based technologies by deploying new editing and delivery innovations. This includes advancing gene editing programs in five different tissues outside the liver, either independently or in collaboration with partners. These research and preclinical programs are targeting diseases that originate in the bone marrow, brain, muscle, lung and eye, which, if successful, could dramatically expand the opportunities for CRISPR-based treatments.
In February, Intellia and ReCode announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF). The collaboration will leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle delivery platform to precisely correct one or more CF disease-causing gene mutations.
Ex Vivo Program Updates

Intellia is advancing multiple programs, wholly owned and in collaboration with partners, utilizing its allogeneic platform for the treatment of immuno-oncology and autoimmune diseases. The Company’s proprietary allogeneic cell engineering platform avoids both T cell- and NK cell-mediated rejection in preclinical models, a key unsolved challenge with other investigational allogeneic approaches. Cell therapies engineered with Intellia’s allogeneic platform, combined with edits to enhance cell function, offer a new approach to target solid tumors.
Corporate Updates

Corporate Responsibility Report: In April, Intellia published its 2024 Corporate Responsibility Report. The report highlights the Company’s Environmental, Social and Governance (ESG) principles and practices as part of its objective to build a sustainable company, while delivering on its commitments to patients, employees and shareholders.
Upcoming Events

The Company will participate in the following events during the second quarter of 2024:

Bank of America Health Care Conference, May 14, Las Vegas
RBC Capital Markets Global Healthcare Conference, May 14, New York
EAACI Congress 2024, May 31 – June 3, Valencia, Spain
Goldman Sachs 45th Annual Global Healthcare Conference, June 10, Miami
First Quarter 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $953.4 million as of March 31, 2024, compared to $1.0 billion as of December 31, 2023. The decrease was driven by cash used to fund operations of $137.2 million. The decrease was offset in part by $58.0 million of net equity proceeds from the Company’s "At the Market" (ATM) program, $12.6 million of interest income, $5.9 million of reimbursement from its collaborators, and $2.0 million in proceeds from employee-based stock plans. The cash position is expected to fund operations into late 2026.
Collaboration Revenue: Collaboration revenue was $28.9 million during the first quarter of 2024, compared to $12.6 million during the first quarter of 2023. The $16.3 million increase was mainly driven by a $21.0 million non-cash revenue recognition adjustment related to the AvenCell collaboration.
R&D Expenses: Research and development expenses were $111.8 million during the first quarter of 2024, compared to $97.1 million during the first quarter of 2023. The $14.7 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in research and development expenses was $20.2 million for the first quarter of 2024.
G&A Expenses: General and administrative expenses were $31.1 million during the first quarter of 2024, compared to $27.4 million during the first quarter of 2023. The $3.7 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in general and administrative expenses was $14.0 million for the first quarter of 2024.
Net Loss: Net loss was $107.4 million for the first quarter of 2024, compared to $103.1 million during the first quarter of 2023.
Conference Call to Discuss First Quarter 2024 Results

The Company will discuss these results on a conference call today, Thursday, May 9, at 8 a.m. ET.
To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on May 9 at 12 p.m. ET.

On May 9, 2024 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported financial results for the first quarter ended March 31, 2024 and recent corporate highlights (Press release, In8bio, MAY 9, 2024, View Source [SID1234642993]).

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"We continued to make significant progress advancing our gamma-delta T cell programs in the first quarter of 2024," said William Ho, CEO and co-founder of IN8bio. "We presented new preclinical data on our nsCAR platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024 Annual Meeting demonstrating its potential to target and kill various acute myeloid leukemia (AML) cells by targeting CD33 and/or CD123, while preserving healthy bone marrow cells. These findings reinforce our technology’s ability to precisely target "undruggable" cancer targets that have historically been challenging due to on-target, off-tumor toxicity. We will provide an update from our Phase 1 study of INB-100 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting in June, including patient status and survival rate data. We anticipate enrolling ten additional patients in an expansion cohort at the recommended Phase 2 dose, and could potentially submit an investigational new drug (IND) application for a Phase 2 randomized control trial this year. In addition, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, we will provide an update on our Phase 1 INB-200 study in GBM which generated an initial efficacy signal supporting the INB-400 trial."

Corporate Highlights and Recent Developments

Presented data at AACR (Free AACR Whitepaper) 2024, supporting the potential of proprietary constructs targeting CD33 and/or CD123 for in vitro evaluation against various types of leukemia, including AML and chronic myeloid leukemia (CML).
Demonstrated significant differences between cells expressing traditional signaling chimeric antigen receptors (CARs) and those expressing nsCAR constructs, which include a reduction in activation-induced cell death with nsCAR constructs.
Peer-reviewed publication of "Adoptive Cell Therapy for High Grade Gliomas using Simultaneous Temozolomide and Intracranial MGMT-Modified γδ T cells Following Standard Post-Resection Chemotherapy and Radiotherapy: Current Strategy and Future Directions" in Frontiers in Immunology detailing IN8bio’s DeltEx Drug Resistant Immunotherapy (DRI) as a rational therapeutic approach for newly diagnosed GBM.
Announced first patient dosed in the Phase 2 autologous arm of INB-400 in patients with newly diagnosed GBM.
Upcoming Anticipated Pipeline Milestones and Events

American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting (May 10, 2024): Upcoming oral presentation: "Healthy Donor vs Patient Manufactured Autologous DeltEx DRI Product; Immunophenotyping Gene Expression," will unveil new data highlighting the characterization of our clinical manufactured DeltEx DRI product. The presentation will explore the impact of manufacturing on the final cell product from healthy donors and those manufactured from cancer patients, showcasing IN8bio’s robust capabilities and know-how in complex cell therapy process development and manufacturing.
INB-100: Report updated interim results from the ongoing Phase 1 investigator-sponsored trial at the 2024 EHA (Free EHA Whitepaper) Annual Meeting, held June 13-16 in Madrid, Spain. In addition, we will potentially submit an IND application for a Phase 2 registrational trial in 2024 in the AML and myelodysplastic syndrome (MDS) patient setting.
INB-200: Report interim Phase 1 long-term follow up results in GBM at multiple medical meetings in 2024 including at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.
INB-400: Initiated patient dosing in the Phase 2 autologous arm of INB-400 in newly diagnosed GBM. IN8bio expects to treat up to a total of 40 patients in arm A at multiple sites across the United States.
First Quarter 2024 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $4.9 million, compared to $4.4 million for the comparable prior year period. The increase was primarily due to (i) increased personnel-related costs, including salaries and stock-based compensation due to increased headcount and (ii) direct clinical costs for INB-100, INB-200 and INB-400.
General and administrative expenses: General and administrative expenses were $3.7 million, compared to $3.5 million for the comparable prior year period. The increase was primarily due to increased personnel-related costs, including stock-based compensation and rent, offset by cost savings related to directors’ and officers’ insurance premiums and a reduction in professional services.
Net loss: Net loss was $8.6 million, or $0.20 per basic and diluted common share, compared to a net loss of $7.5 million, or $0.30 per basic and diluted common share, for the comparable prior year period.
Cash position: As of March 31, 2024, the Company had cash of $13.0 million, compared to $21.3 million, as of December 31, 2023.

On May 9, 2024 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and recent business highlights for the first quarter of 2024 (Press release, Hookipa Biotech, MAY 9, 2024, View Source [SID1234642992]).

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"The first quarter was about focus at HOOKIPA. We are embarking on a pivotal trial for HB-200 in combination with pembrolizumab and made important decisions to align our organization for late-stage clinical trial execution. We also added to the depth of our executive team with a new Chief Development Officer, Mark Winderlich, who brings crucial experience to help us execute on our clinical development strategy," said Joern Aldag, Chief Executive Officer of HOOKIPA. "We have made great progress and have an FDA-aligned pivotal Phase 2/3 trial design that is patient-centric and we believe has a high probability of success. Our path forward is clear, and the team is excited to take a major step on our path to deliver better outcomes for patients."

Business Highlights and Recent Developments

Oncology

HOOKIPA is preparing to start a seamless pivotal Phase 2/3 trial of HB-200 in combination with pembrolizumab for the treatment of patients with Human Papillomavirus 16-positive (HPV16+) recurrent/metastatic PD-L1 CPS ≥ 20 oropharyngeal squamous cell carcinoma (OPSCC) in the first line setting.
The Phase 2/3 trial design and protocol are based on alignment with the FDA following the Company’s Type C meeting.
EMA granted PRIME designation to the investigational product HB-200 in combination with pembrolizumab. PRIME designation is intended to expedite development and review of drug candidates, alone or in combination with other drugs. Eligibility and approval are based on preliminary clinical evidence and indicate that the drug candidate may offer substantial improvement over existing therapies.
The Company anticipates the first patient will be enrolled in the fourth quarter of 2024. HB-200 was accepted for an oral abstract presentation at the ASCO (Free ASCO Whitepaper) 2024 Annual Meeting with data from approximately 40 patients treated with HB-200 in combination with pembrolizumab.
The HB-700 program is a novel arenaviral immunotherapy for KRAS-mutated cancers, including the five mutations that are the primary causes of lung, pancreatic and colon cancers. The Company received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for HB-700 for the treatment of KRAS-mutated cancers. Effective April 25, 2024, HOOKIPA regained full control of the associated intellectual property portfolio and has full collaboration and licensing rights for this program.
Infectious Disease

HB-400 is currently being evaluated in a Phase 1 trial and is one of two independent development programs in HOOKIPA’s collaboration and license agreement with Gilead Sciences, Inc. (Gilead). Gilead is solely responsible for further development and commercialization of the HBV product candidate.
HB-500 is an investigational therapeutic vaccine for the treatment of human immunodeficiency virus (HIV), also partnered with Gilead. HOOKIPA received FDA clearance of its IND application in the fourth quarter of 2023 and expects to initiate a Phase 1 clinical trial of HB-500 in people with HIV in the second quarter of 2024. Under the collaboration agreement with Gilead, HOOKIPA is eligible for a milestone payment upon dosing the first patient in this trial.
Corporate and Financial Updates

Corporate Highlights

On January 29, 2024, HOOKIPA provided an update on its business priorities and oncology partnership programs. The Company announced that it will focus its resources in two strategic areas: (1) the clinical development of a randomized trial for its HB-200 program and (2) its two Gilead-partnered infectious disease cure programs for hepatitis B and HIV.
Mark Winderlich, Ph.D., joined the Company on April 1, 2024, as Chief Development Officer to lead HOOKIPA’s clinical research and development organization.
Financial Highlights

HOOKIPA received a final $10.0 million milestone payment under its now-terminated HB-700 collaboration agreement with Roche. The success-based milestone payment was achieved in connection with HOOKIPA’s submission of an IND application for HB-700 for the treatment of KRAS mutated tumors.
Total revenues of $36.6 million, mainly driven by the recognition of previously received upfront and milestone payments under the now-terminated Roche collaboration, as well as the recent HB-700 milestone achievement, led to a profitable first quarter of 2024.
Anticipated Catalysts & Milestones

Program Indication Upcoming Anticipated Catalysts
Oncology Programs
HB-200 HPV16+ HNSCC
Additional Phase 2 first-line data for HB-200 in combination with pembrolizumab (ASCO 2024)
Pivotal study start (4Q 2024)
HB-700 KRAS
Publication of preclinical data (ASCO 2024)

Infectious Disease Programs: Gilead-Partnered
HB-400 HBV
Gilead-led: Phase 1b actively enrolling
Next milestone: Initiation of Phase 2
(timing determined by Gilead)
HB-500 HIV
Initiation of Phase 1 trial; first patient dosed and associated milestone payment (2Q 2024)

First Quarter 2024 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2024 was $93.0 million compared to $117.5 million as of December 31, 2023. The decrease was primarily attributable to cash used in operating activities.

Revenue: Revenue was $36.6 million for the three months ended March 31, 2024, compared to $3.2 million for the same period in 2023. The increase was primarily due to higher partial revenue recognition under the Roche collaboration as a result of the termination of the agreement, leading to accelerated recognition of the upfront and milestone payments that were initially recorded as deferred revenue.

Research and Development Expenses: HOOKIPA’s research and development expenses were $20.2 million for the three months ended March 31, 2024, compared to $20.9 million for the same period in 2023. The primary drivers of the decrease in research and development expenses were lower personnel-related and laboratory-related expenses, partially offset by higher clinical study expenses for the HB-200 program.

General and Administrative Expenses: General and administrative expenses amounted to $4.1 million for the three months ended March 31, 2024, compared to $4.9 million for the same period in 2023. The primary driver of the decrease in general and administrative expenses was a decrease in personnel-related expenses.

Restructuring Expenses: Restructuring expenses amounted to $1.3 million for the three months ended March 31, 2024, and resulted from severance and other personnel costs as well as consulting costs associated with the Company’s restructuring plan announced in January 2024.

Net Income (Loss): HOOKIPA’s net income was $14.4 million for the three months ended March 31, 2024, compared to a net loss of $19.7 million for the same period in 2023. This increase was primarily due to the accelerated recognition of upfront and milestone payments under the Roche collaboration.

On May 9, 2024 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported financial results for the quarter ended March 31, 2024 (Press release, Guardant Health, MAY 9, 2024, View Source [SID1234642991]).

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First Quarter 2024 Financial Highlights

•Revenue of $168.5 million for the first quarter of 2024, an increase of 31% over the first quarter of 2023
•Reported 46,900 tests to clinical customers and 8,450 tests to biopharmaceutical customers in the first quarter of 2024, representing increases of 20% and 37%, respectively, over the first quarter of 2023
•Reduced free cash flow to $(37.2) million in the first quarter of 2024, compared to $(82.0) million in the prior year
•Raised 2024 annual guidance for revenue to a new range of $675 to $685 million, representing growth of 20% to 21%
•Improved annual free cash flow guidance to $(275) to $(285) million, a reduction of $60 to $70 million compared to 2023
Recent Operating Highlights
•Validated the strength and quality of ECLIPSE clinical data with the study publication in The New England Journal of Medicine
•Launched new service at The Royal Marsden to test advanced NSCLC patients in England through an expanded NHS study
•Presented new data demonstrating the value of epigenomic analysis and methylation sequencing using the Smart Liquid Biopsy platform at the 2024 AACR (Free AACR Whitepaper) Annual Meeting
•Surpassed 500 peer-reviewed publications highlighting Guardant Health technology in scientific literature

"We started the year off very strongly with first quarter revenue growing 31%, driven by both solid volume growth and significant improvements to Guardant360 reimbursement," said Helmy Eltoukhy, co-founder and co-CEO. "In addition to strong topline performance, this was the first quarter of generating positive cash flow in our Therapy Selection business. We also recently surpassed a significant milestone with over 500 peer-reviewed publications highlighting our technology in scientific literature, demonstrating the impact our innovative suite of products have on both patients and the scientific community."
"The publication of ECLIPSE data in The New England Journal of Medicine, one of the world’s leading medical journals, underscores the quality of our clinical data," said AmirAli Talasaz, co-founder and co-CEO. "Our team has worked incredibly hard and is now well-prepared for the upcoming FDA Advisory Committee review on May 23. We are eagerly anticipating the launch of Shield IVD shortly after the expected FDA approval in 2024 and are confident this test is well positioned for rapid adoption."

First Quarter 2024 Financial Results

Revenue was $168.5 million for the first quarter of 2024, a 31% increase from $128.7 million for the corresponding prior year period. Precision oncology revenue grew 38%, to $156.2 million for the first quarter of 2024, from $113.4 million for the corresponding prior year period, driven by an increase in clinical and biopharma testing volume, which grew 20% and 37%, respectively, over the prior year period. The increase in precision oncology revenue was also attributable to an increase in reimbursement for our tests, due to an increase in the Medicare reimbursement rate for our Guardant360 LDT test to $5,000, effective January 1, 2024, and an increase in reimbursement received from commercial payers. Development services and other revenue was $12.3 million for the first quarter of 2024, compared to $15.3 million for the corresponding prior year period.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $103.2 million for the first quarter of 2024, an increase of $27.6 million from $75.6 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 61%, as compared to 59% for the corresponding prior year period. Precision oncology gross margin was 62% in the first quarter of 2024, as compared to 60% in the prior year period. Development services and other gross margin was 51% in the first quarter of 2024, as compared to 48% in the prior year period.

Non-GAAP gross profit was $105.3 million for the first quarter of 2024, an increase of $27.6 million, from $77.7 million for the corresponding prior year period. Non-GAAP gross margin was 63% for the first quarter of 2024, as compared to 60% for the corresponding prior year period.
Non-GAAP gross profit excluding screening was $108.0 million for the first quarter of 2024, an increase of $26.9 million, from $81.1 million for the corresponding prior year period. Non-GAAP gross margin excluding screening was 64% for the first quarter of 2024, as compared to 63% for the corresponding prior year period.

Operating expenses were $202.9 million for the first quarter of 2024, as compared to $209.7 million for the corresponding prior year period. Non-GAAP operating expenses were $176.5 million for the first quarter of 2024, as compared to $188.3 million for the corresponding prior year period.
Net loss was $115.0 million for the first quarter of 2024, as compared to $133.5 million for the corresponding prior year period. Net loss per share was $0.94 for the first quarter of 2024, as compared to $1.30 for the corresponding prior year period. The year-over-year decrease in net loss is primarily due to a $34.4 million year over year improvement in loss from operations, and a $11.8 million increase in interest income, partially offset by a $33.9 million increase in unrealized losses recorded for our investment in Lunit, Inc.
Non-GAAP net loss was $56.4 million for the first quarter of 2024, as compared to $108.5 million for the corresponding prior year period. Non-GAAP net loss per share was $0.46 for the first quarter of 2024, as compared to $1.06 for the corresponding prior year period.
Adjusted EBITDA loss was $61.1 million for the first quarter of 2024, as compared to a $101.0 million loss for the corresponding prior year period.
Free cash flow for the first quarter of 2024 was $(37.2) million, as compared to $(82.0) million for the corresponding prior year period. Cash, cash equivalents, and restricted cash were $1.1 billion as of March 31, 2024.
2024 Guidance
Guardant Health now expects full year 2024 revenue excluding screening to be in the range of $675 to $685 million, representing growth of 20% to 21% compared to full year 2023 This compares to the prior range of $655 to $670 million, representing growth of 16% to 19%. Guardant Health now expects full year 2024 non-GAAP gross margin excluding screening to be in the range of 61% to 63%, compared to the prior range of 60% to 62%. Guardant Health now expects total non-GAAP operating expenses to be in the range of $720 to $730 million, representing a flat to 1% decrease compared to 2023, an improvement compared to the prior range of $740 to $750 million. Guardant Health now expects free cash flow to be in the range of $(275) to $(285) million in 2024, an improvement compared to the prior range of $(320) to $(330) million.
Webcast Information
Guardant Health will host a conference call to discuss the first quarter 2024 financial results after market close on Thursday, May 9, 2024 at 1:30 pm Pacific Time / 4:30 pm Eastern Time. A webcast of the conference call can be accessed at View Source The webcast will be archived and available for replay for at least 90 days after the event.

On May 9, 2024 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported financial results for the first quarter ended March 31, 2024 and provided recent corporate and clinical updates (Press release, Gritstone Bio, MAY 9, 2024, View Source [SID1234642990]).

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"The preliminary Phase 2 data we recently shared are very promising as they suggest that GRANITE is potentially driving benefit in metastatic CRC patients and that our objective of unlocking immunologically ‘cold’ tumors to the benefits of immunotherapy may be within reach," said Andrew Allen, MD, PhD, Co-founder, President & CEO of Gritstone bio. "The emerging trend in progression-free survival, that we anticipate will strengthen as data mature, is particularly encouraging as it puts us in a strong position to potentially engage regulators later this year regarding a Phase 3 study for this common and difficult to treat disease. If successful, we see great potential for GRANITE to expand the scope of immunotherapy and bring meaningful clinical benefit to patients with metastatic CRC as well as other ‘cold’ tumors."

Dr. Allen added, "The progress in, and recognition of our other programs and capabilities is also encouraging. The recent paper in Nature Medicine highlights the scientific rigor with which we built our SLATE platform, describes the discovery of a previously unknown hierarchy of neoantigen immunodominance, and underscores the promise for the ongoing collaboration with Dr. Rosenberg of the NCI to evaluate our SLATE-KRAS vaccine in combination with an autologous T cell therapy. We also continue to push the boundaries of neoantigen identification with EDGE, our powerful AI-driven platform, that can now predict presentation of HLA Class I neoantigens with what we believe to be field-leading accuracy."

Corporate Updates

In April 2024, Gritstone completed an underwritten public offering resulting in gross proceeds of $32.5 million.
In April 2024, Gritstone appointed Stephen Webster to its Board of Directors. A veteran finance executive with over 30 years in the biotechnology industry, Mr. Webster has held several key roles and been involved in multiple strategic transactions. Mr. Webster was the Chief Financial Officer of Spark Therapeutics from July 2014 until its acquisition by Roche for $4.3 billion in December 2019.
Clinical Program Updates
Tumor-Specific Neoantigen Oncology Programs (GRANITE and SLATE)
GRANITE – Personalized neoantigen vaccine program
SLATE – "Off-the-shelf" neoantigen vaccine program

Preliminary results (n = 67) from the randomized Phase 2 study evaluating GRANITE as a front-line maintenance therapy in metastatic microsatellite-stable colorectal cancer (MSS-CRC) demonstrated a favorable trend in progression-free survival (PFS). Long-term circulating tumor DNA (ctDNA) data align with PFS trend and favor GRANITE vs. control patients.
Trend of extended PFS in GRANITE-treated vs. control patients, with greatest difference observed in high-risk group1 where clinical data are more mature.
Hazard ratio of 0.82 (18% relative risk reduction of progression or death with GRANITE vs. control) in the overall population, where clinical data are less mature ([95% CI, 0.34-1.67]; 62% censored)
Hazard ratio of 0.52 (48% relative risk reduction of progression or death with GRANITE vs. control) in a high-risk group1, where clinical data are more mature ([95% CI, 0.15-1.38]; 44% censored)
1High-risk subgroup defined as baseline ctDNA above the median value (2%) for the control group (ctDNA quantified as mean variant allele frequency [VAF] at time of study randomization).

Long-term ctDNA data align with PFS trend and favor GRANITE-treated vs. control patients
Analysis in the high-risk group1 showed that between first blood draw (time of randomization) and last blood draw (most recent study visit), ctDNA shifted from high (>2% VAF) to low (≤2% VAF) in 56% (9/16) of GRANITE patients vs 22% (2/9) of control patients. Progressive disease was observed in 44% (7/16) vs 78% (7/9), respectively, within this group.
Analysis in low-risk group (ctDNA negative group) showed sustained ctDNA negativity was observed in 67% (6/9) GRANITE recipients vs 38% (3/8) control patients. PD observed in 11% (1/9) and 38% (3/8) of these patients, respectively.
Gritstone expects to share mature PFS data and additional long-term ctDNA data in the third quarter of 2024.
In April 2024, Gritstone presented an update on its state-of-the-art neoantigen prediction platform, EDGE, at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in San Diego, CA. EDGE now predicts HLA Class I presentation, associated with CD8+ T cell induction, with >80% accuracy, a performance level that Gritstone believes to be leading the field. Gritstone is also advancing EDGE-II, a new model that has achieved superior predictive performance of HLA Class II presentation and CD4+ immunogenicity over publicly available models. The improvements leverage advances in protein large language models and in-house immunopeptidomics.

In March 2024, Nature Medicine published a paper detailing the development of our "off-the-shelf" neoantigen platform, SLATE. The paper described a novel immunodominance hierarchy of tumor neoantigens (including KRAS) that Gritstone discovered in Phase 1 translational studies and leveraged to develop SLATE-KRAS, a "pure" KRAS-directed vaccine candidate that demonstrated superior immunogenicity to the initial version in a subsequent Phase 2 study.

The clinical trial collaboration with the National Cancer Institute (NCI) to evaluate an autologous mutant KRAS-directed TCR-T cell therapy in combination with SLATE-KRAS, Gritstone’s KRAS-directed "off the shelf" vaccine candidate, is ongoing. The study is led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI’s Center for Cancer Research and builds into the growing interest in combining tumor-antigen specific cell therapy with matched vaccines. The IND was cleared by the U.S. Food and Drug Administration (FDA) in October 2023.

Infectious Disease Programs
CORAL – Next-generation SARS-CoV-2 vaccine program that serves as proof-of-concept for Gritstone’s samRNA platform and novel approach in infectious diseases.

In February 2024, Gritstone announced that it plans to incorporate GMP-grade materials in the manufacture of its self-amplifying mRNA (samRNA) candidate, resulting in a delay of the CORAL Phase 2b study (the anticipated 10,000 subject, comparative Phase 2b study contracted by the Biomedical Advanced Research and Development Authority [BARDA]2). This decision is expected to increase the regulatory utility of the study. Gritstone is currently preparing to launch the study and will do so as soon as the company is able.

In April 2024, Gritstone presented a poster highlighting the durability and potential broad utility of its samRNA COVID-19 vaccine at ESCMID Global 2024. The results, which were from the Phase 1 CORAL-CEPI study in South Africa, reinforced previous findings showing induction of broad and durable immune responses through 12 months.
HIV – Collaboration with Gilead under Gilead’s HIV Cure Program to research and develop vaccine-based HIV immunotherapy treatment

The collaboration to research and develop a vaccine-based HIV immunotherapy treatment continues under Gilead’s direction.
First Quarter 2024 Financial Results

Cash, cash equivalents, marketable securities and restricted cash were $52.8 million as of March 31, 2024, compared to $86.9 million as of December 31, 2023.
Research and development expenses were $33.0 million for the three months ended March 31, 2024 compared to $30.5 million for the three months ended March 31, 2023. The increase of $2.5 million was primarily attributable to a one-time severance charge and increases in facilities-related costs, offset by decreases in laboratory supplies, personnel-related costs and outside services.

General and administrative expenses were $8.5 million for the three months ended March 31, 2024 compared to $6.7 million for the three months ended March 31, 2023. The increase of $1.8 million was primarily attributable to increases in personnel-related expenses, facilities-related costs, outside services and a one-time severance charge.

Collaboration, license, and grant revenues were $1.7 million for the three months ended March 31, 2024. During the three months ended March 31, 2024, we recorded $0.4 million in grant revenue from the BARDA Contract, $1.0 million in grant revenue from CEPI, and $0.3 million in grant revenue from the Gates Foundation.
Conference Call & Webcast Details
A conference call and webcast will be held at 4:30pm ET today (May 9):

Conference call: 1-877-407-4018
Conference ID: 13746126
Webcast: View Source;tp_key=d0e680f7aa

An archived replay will be accessible at View Source for 30 days following the event.

2 This project has been supported in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract number 75A50123C00062.