On January 13, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported it will share corporate progress and highlights from the Company’s broad and diverse investigational pipeline while presenting at the annual J.P. Morgan Healthcare Conference (Press release, Regeneron, JAN 13, 2025, View Source [SID1234649674]). The presentation is scheduled for 2:15 p.m. Pacific Time (5:15 p.m. Eastern Time) and may be accessed from the "Investors & Media" page of Regeneron’s website.

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"The Regeneron name is synonymous with innovation, brought to life through proprietary technologies and world-class science that produce medicines that make a meaningful impact on patients’ lives," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. "Thanks to our long-term and consistent R&D investment, we have – in addition to our four blockbuster medicines – one of the industry’s largest, most promising and most diverse clinical pipelines. Our therapeutic candidates tackle a myriad of diseases, with the most advanced programs addressing an aggregate commercial market opportunity expected to exceed $220 billion by 2030. We are well positioned for future growth and more confident than ever in the power of Regeneron’s science."

Marketed Products

Dupixent Updates

Dupixent (dupilumab) is now used to treat over a million patients globally. The recent approval and launch in chronic obstructive pulmonary disease (COPD) has had a successful start, with coverage secured from the top commercial and Medicare payers and Dupixent now well positioned to address approximately 300,000 patients in the U.S.
There is continued growth potential in existing and additional indications for diseases in which type 2 inflammation may play a role, including chronic spontaneous urticaria (CSU) with an expected U.S. Food and Drug Administration (FDA) decision by April 18, 2025, and bullous pemphigoid, for which a supplemental Biologics License Application (sBLA) was submitted in the fourth quarter of 2024.
EYLEA HD and EYLEA Updates

On a combined basis, EYLEA HD (aflibercept) Injection 8 mg and EYLEA (aflibercept) Injection 2 mg remained the U.S. anti-VEGF category leader in 2024. Based on preliminary (unaudited) results, the products achieved 1% year-over-year growth by reaching $6 billion in aggregate U.S. net product sales for the year and $1.5 billion in aggregate U.S. net product sales for the fourth quarter of 2024, despite increasing competition. EYLEA HD U.S. net product sales were $305 million in the fourth quarter of 2024. EYLEA U.S. net product sales were $1.19 billion in the fourth quarter of 2024.
Combined EYLEA HD and EYLEA U.S. net product sales for the fourth quarter of 2024 were favorably impacted by approximately $85 million as a result of higher wholesaler inventory levels for EYLEA, partially offset by lower wholesaler inventory levels for EYLEA HD.
The Company filed an application with the FDA for use of the EYLEA HD pre-filled syringe (PFS) with U.S. approval and launch expected by mid-2025.
Longer term data in wet age-related macular degeneration (wAMD) and diabetic macular edema (DME) are under FDA review with a PDUFA date of April 20, 2025 to potentially extend dosing intervals for EYLEA HD up to every-24 weeks.
The Company plans to submit a sBLA for EYLEA HD for every four-week dosing and for retinal vein occlusion (RVO) in the first quarter of 2025 to potentially maximize dosing flexibility and address more retinal diseases.

Libtayo Updates

Libtayo (cemiplimab) exceeded $1 billion in sales for 2024 and remains foundational to Regeneron’s oncology portfolio.
As announced this morning, a Phase 3 study demonstrated that Libtayo is the only immunotherapy to show a statistically significant and clinically meaningful benefit in high-risk cutaneous squamous cell carcinoma (CSCC) in the adjuvant setting; a recent Phase 3 trial with Keytruda failed in the same setting.1 Specifically, adjuvant Libtayo demonstrated a 68% reduction in the risk of disease recurrence or death, compared to placebo (hazard ratio: 0.32; 95% confidence interval: 0.20-0.51; p<0.0001). Grade ≥3 adverse events occurred in 24% (n = 49 of 205) and 14% (n = 29 of 204) of patients in the Libtayo arm and the placebo arm, respectively. Detailed results will be presented at an upcoming medical meeting and will be shared with regulatory authorities with a plan for FDA submission in the first half of 2025.
Phase 3 and Other Major Pipeline Opportunities

Regeneron is progressing numerous promising drug candidates across diverse disease states, with advanced programs that together have a total addressable commercial market expected to exceed $220 billion by 2030. Some near-term highlights include:

Itepekimab (IL-33) for COPD: Based on genetic data linking IL-33 with increased risk of COPD and Phase 2 results, Regeneron’s next innovation in COPD offers potential for benefit in a broader population, including former smokers, non-cystic fibrosis bronchiectasis and other indications. Results are expected from the Phase 3 AERIFY study in the second half of 2025, with a potential BLA submission to follow.

Fianlimab (LAG3) for melanoma: Combining fianlimab and Libtayo, two potentially best-in-class checkpoint inhibitors, has the potential for differentiated efficacy and safety versus the current standard-of-care. Results from the first Phase 3 study in first-line metastatic melanoma are expected in the second half of 2025, with a potential BLA submission to follow.

Linvoseltamab (BCMAxCD3) for multiple myeloma: Linvoseltamab has potential to be the best-in-class BCMAxCD3 bispecific with its differentiated clinical profile, dosing regimen and administration method. The linvoseltamab BLA has been resubmitted following resolution of third-party manufacturing issues, with launch anticipated in mid-2025. Phase 3 programs in earlier lines of therapy using linvoseltamab monotherapy and novel combinations are also underway.

Odronextamab (CD20xCD3) for lymphoma: Ordspono (odronextamab) has been approved in the European Union for relapsed/refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy, and enrollment is underway for a confirmatory study to support resubmission of the BLA for FL to the FDA in the first quarter of 2025. A broad and differentiated Phase 3 program is also underway to investigate odronextamab in earlier lines of FL and DLBCL. As reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, odronextamab monotherapy showed complete responses in 12 out of 12 evaluable patients with first-line FL in the safety lead-in portion of the Phase 3 program.

Factor XI for anticoagulation: Regeneron’s two-pronged approach to anticoagulation is being evaluated for its potential to control thombosis while minimizing bleeding risk in a variety of patient populations and clinical settings. Two Factor XI antibodies, REGN7508 (catalytic domain) and REGN9933 (A2 domain), will advance to pivotal trials in 2025 on the basis of positive proof-of-concept data announced in December 2024. Current standards of care for thrombosis disorders have challenges including elevated risk of bleeding resulting in underutilization, presenting an unmet need for more specific inhibition of the intrinsic coagulation pathway.

Multiple approaches to obesity: Regeneron is studying various combinations with GLP-based therapies to potentially improve quality of weight loss by preserving lean muscle, as well as improve maintenance of weight loss following GLP-1/GIP discontinuations. A Phase 2 study of trevogrumab and semaglutide with and without garetosmab is now fully enrolled and a Phase 2 study testing combinations of tirzepatide and mibavademab is ongoing, with initial data expected from both in the second half of 2025.

BCMAxCD3/Dupixent in severe allergy: Combining linvoseltamab and Dupixent has the potential to eliminate immunoglobulin E (IgE), the key driver of allergic reactions, and thus potentially reverse severe allergies. A trial in patients with severe food allergies is ongoing, with initial clinical data shared in today’s presentation showing profound reduction of IgE in the first patient treated with this two-drug approach.

C5 Combo (pozelimab and cemdisiran) in complement-mediated diseases: Regeneron’s differentiated siRNA and antibody combination approach has the potential to address multiple complement-mediated diseases, such as generalized myasthenia gravis (Phase 3 results expected in the second half of 2025), paroyxsmal noctural hemoglobinuria (Phase 3 registrational data expected in 2026+) and geographic atrophy, an advanced form of dry AMD (Phase 3 pivotal program underway).

DNA Sequence-Linked Healthcare Database

Regeneron continues to grow its leadership in genetics-driven drug discovery and is building the world’s largest DNA sequence-linked healthcare database, designed to unlock profound insights into how genetics impact health and aid in the development new genetic-based therapies and optimized healthcare services.

The Regeneron Genetics Center has sequenced nearly three million people to date, all with deidentified linked healthcare records.
A newly announced strategic collaboration with Truveta, Inc. is expected to dramatically expand the size of this database, with sequencing and linked Electronic Health Records for up to 10 million additional individuals from Truveta’s network of leading U.S. health systems.
On the basis of its industry-leading capabilities, Regeneron Genetics Center was selected by UK BioBank consortium members to complete proteomic assay data generation for the recently announced UK Biobank Pharma Proteomics Project.

"Regeneron continues to diversify our commercial, clinical and research portfolios by relentlessly pushing the boundaries of innovation and technology," said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron. "In 2025, we will progress dozens of promising new assets and expand the reach of our important established medicines to help even more patients in need. We remain at the forefront of biotechnology’s most remarkable era of drug discovery, striving to change the practice of medicine with approaches spanning antibodies, bispecifics, gene editing, gene silencing, gene therapy and cell therapy supported by DNA sequence- and proteomics-linked healthcare database."

The unapproved uses of EYLEA, EYLEA HD, Dupixent, Libtayo and pozelimab noted here are investigational and have not been fully evaluated by any regulatory authority. Cemdisiran, itepekimab, fianlimab, linvoseltamab, REGN7508, REGN9933, trevogrumab and garetosmab are investigational and have also not been fully evaluated by any regulatory authority. Odronextamab is approved in the European Union as Ordspono to treat R/R FL or DLBCL after two or more lines of systemic therapy, but the safety and efficacy of odronextamab has not been fully evaluated by any other regulatory authority.

On January 13, 2025 Novocure (NASDAQ: NVCR) reported preliminary unaudited financial and operational results for the quarter and full year ended December 31, 2024 (Press release, NovoCure, JAN 13, 2025, View Source [SID1234649690]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields (TTFields).

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"Novocure is at an exciting inflection point as we continue to expand our multi-indication TTFields treatment platform. In 2024, we brought Optune Gio to more than 4,000 glioblastoma patients across the globe, earned FDA approval and launched Optune Lua in non-small cell lung cancer in the U.S., announced two additional successful Phase 3 trial readouts and released our next generation arrays," said Ashley Cordova, Chief Executive Officer. "With two additional indication launches on the horizon, we are well positioned for 2025 and beyond. This progress demonstrates our steadfast commitment to our patient-forward mission: together with our patients, we strive to extend survival in some of the most aggressive forms of cancer."

Financial updates for the year and fourth quarter ended December 31, 2024*:

Total preliminary net revenues for the year were $605.2 million, an increase of 19% compared to the prior year.
2024 growth was primarily driven by our successful launch in France and significantly improved approval rates in the U.S., which are now reflected in our revenue baseline. 2025 net revenue growth is expected to closely reflect growth in Optune Gio active patients.
Total preliminary net revenues for the fourth quarter were $161.3 million, an increase of 21% compared to the same period in 2023.
The U.S., Germany, France and Japan contributed $107.2 million, $17.7 million, $16.0 million and $8.5 million, respectively, with other active markets contributing $9.9 million.
Revenue in Greater China from Novocure’s partnership with Zai Lab totaled $2.0 million.
Improved approval rates in the U.S. resulted in $8.3 million of increased net revenue from prior period claims during the quarter, which we believe should not be considered in our 2025 baseline. This is in addition to the $14.0 million of increased revenue from prior period claims disclosed through the third quarter.
Cash, cash equivalents and short-term investments were $959.9 million as of December 31, 2024.
Operational updates for the fourth quarter ended December 31, 2024:

As of December 31, 2024, there were 4,126 total active patients on TTFields therapy globally.
1,520 Optune Gio prescriptions were received in the quarter, consistent with the same period in 2023. Optune Gio prescriptions from the U.S., Germany, France and Japan contributed 897; 190; 194 and 109 prescriptions, respectively, with the remaining 130 prescriptions received from other active markets.
As of December 31, 2024, there were 4,077 active Optune Gio patients on therapy. Active Optune Gio patients from the U.S., Germany, France and Japan contributed 2,161; 564; 426 and 420 active patients, respectively, with the remaining 506 active patients contributed by other active markets.
On October 15, 2024, Optune Lua was approved by the U.S. Food and Drug Administration (FDA) for the treatment of metastatic non-small cell lung cancer (NSCLC) concurrently with PD-1/PD-L1 inhibitors or docetaxel, in adults who have progressed on or after a platinum-based regimen. As of December 31, 2024, 52 Optune Lua prescriptions were received for NSCLC.
As of December 31, 2024, there were 20 active NSCLC patients on Optune Lua. Additionally, there were 29 active mesothelioma patients on Optune Lua around the globe.
In Q1 2026, Novocure intends to stop reporting new prescriptions and focus on active patients by indication and material market as the key operating statistic.
Fourth quarter and recent updates and achievements:

In October, the FDA granted Breakthrough Device designation for the use of TTFields therapy for brain metastases from non-small cell lung cancer. Breakthrough Device designation provides more frequent, faster and interactive access to the FDA review team and senior management during the review process, priority review of marketing applications upon filing, and expedited review of pre-Premarket Approval Application (PMA) manufacturing and quality systems compliance inspections.
In October, the FDA approved Novocure’s new Head Flexible Electrode (HFE) transducer arrays for use with Optune Gio for the treatment of adult patients with glioblastoma (GBM).
In December, the company announced the Phase 3 PANOVA-3 clinical trial met its primary endpoint, demonstrating a statistically significant improvement in overall survival for patients with unresectable, locally advanced pancreatic cancer. Novocure plans to submit the full data for presentation at an upcoming medical congress.
In December, the FDA granted Breakthrough Device designation for the use of TTFields therapy for the treatment of unresectable, locally advanced pancreatic cancer.
In January 2025, the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) approved Novocure’s new HFE transducer arrays for use with Optune Gio for the treatment of adult patients with GBM.
Anticipated clinical milestones:

Data from Phase 2 PANOVA-4 clinical trial in metastatic pancreatic cancer (2026)
Data from Phase 3 TRIDENT clinical trial in newly diagnosed GBM (2026)
Fourth quarter and full year 2024 financial results conference call:

Novocure will host a conference call and webcast to discuss full year and fourth quarter 2024 financial results at 8:00 a.m. EST on Thursday, February 27, 2025. To access the conference call by phone, use the following conference call registration link and dial-in details will be provided. To access the webcast, use the following webcast registration link.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

*The unaudited results in this press release are preliminary and subject to the completion of the Company’s annual independent audit and, therefore, are subject to adjustment.

On January 13, 2025 Indaptus Therapeutics, Inc. (Nasdaq: INDP) ("Indaptus"), a clinical stage biotechnology company dedicated to pioneering innovative cancer and viral infection treatments, reported that it has entered into securities purchase agreements with investors for the issuance and sale in a private placement priced at-the-market under Nasdaq rules of an aggregate of 2,109,383 of its shares of common stock and accompanying warrants to purchase up to an aggregate of 2,109,383 of its shares of common stock (Press release, Indaptus Therapeutics, JAN 13, 2025, View Source;qmodStoryID=6007485332607308 [SID1234649723]). The combined effective purchase price for each share of common stock and associated warrants is $1.065. The closing of the offering is expected to take place on or about January 15, 2025, subject to the satisfaction of customary closing conditions.

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The warrants will have an exercise price of $0.94 per share, will be immediately exercisable upon issuance and have a term of five years from the date of issuance.

Paulson Investment Company, LLC is acting as the exclusive placement agent in connection with the offering.

The gross proceeds to Indaptus from the offering are expected to be approximately $2.25 million, before deducting the placement agent’s fees and other offering expenses payable by Indaptus. Indaptus intends to use the net proceeds from the offering to fund its research and development activities and for working capital and general corporate purposes.

The shares of common stock and warrants to be issued in the private placement and shares issuable upon exercise of such warrants were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), and/or Rule 506(b) of Regulation D promulgated thereunder, have not been registered under the Securities Act or applicable state securities laws and may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On January 13, 2025 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported initiation of the GALLOP Phase 1 clinical trial evaluating CB-010 in patients with lupus nephritis (LN) and extrarenal lupus (ERL) (Press release, Caribou Biosciences, JAN 13, 2025, View Source [SID1234649643]). In addition, Caribou highlighted successful execution across its three clinical-stage allogeneic CAR-T cell therapy programs in hematologic malignancies over the past year and provided an outlook on multiple clinical data catalysts planned for 2025.

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"We are excited to share we have initiated the GALLOP Phase 1 trial. This milestone is a testament to the significant momentum we achieved across all four clinical programs throughout 2024," said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer. "In the first half of 2025, we plan to report clinical data from our lead program, CB-010, in second-line and CD19 relapsed large B cell lymphoma patients as well as dose escalation data from our second program, CB-011, in relapsed or refractory multiple myeloma. Pending confirmation of our HLA matching strategy, we plan to initiate a pivotal Phase 3 trial for CB-010 in second-line large B cell lymphoma in the second half of 2025. We are excited to be on the forefront of a new era for allogeneic CAR-T cell therapies, which offer broad access and rapid availability to patients and healthcare systems."

2024 clinical highlights and corporate accomplishments

Clinical highlights
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for B cell non-Hodgkin lymphoma
•At the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, Caribou presented clinical data from the ongoing ANTLER Phase 1 clinical trial that indicated a single dose of CB-010 has the potential to be on par with the safety, efficacy, and durability of approved autologous CAR-T cell therapies.
•A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a donor with ≥4 matching human leukocyte antigen (HLA) alleles showed improved progression free survival (PFS).

•To confirm the HLA matching strategy, Caribou is enrolling approximately 20 additional second-line large B cell lymphoma (2L LBCL) patients in the ongoing ANTLER Phase 1 clinical trial.
•Caribou also began enrolling a proof-of-concept cohort of up to 10 patients who have relapsed following any prior CD19-targeted therapy in this population of unmet need.
•Caribou plans to present data from both the additional 2L and prior CD19 relapsed LBCL patient cohorts in H1 2025. The company plans to initiate a pivotal Phase 3 trial of CB-010 in the second half of 2025, should data from the additional 2L LBCL patients confirm the initial observation that partial HLA matching of patient to the donor results in outcomes on par with autologous CAR-T cell therapies. The Phase 3 trial would be initiated after agreement with the FDA on a pivotal trial design.

CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for lupus
•Caribou expanded the CB-010 program following clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for CB-010 in LN and ERL.
•The FDA granted Fast Track designation to CB-010 for refractory systemic lupus erythematosus (SLE).
•Caribou has initiated the GALLOP Phase 1 clinical trial, an open-label, multicenter clinical trial designed to evaluate a single infusion of CB-010 in adult patients with LN and ERL.

CB-011, a clinical-stage allogeneic anti-BCMA CAR-T cell therapy for multiple myeloma
•In the dose escalation portion of the CaMMouflage Phase 1 clinical trial for relapsed or refractory multiple myeloma (r/r MM), Caribou implemented a lymphodepletion regimen that includes a deeper dose of cyclophosphamide (increased from 300 to 500 mg/m2/day together with the same fludarabine dose of 30 mg/m2/day for 3 days). Following observations of efficacy, Caribou is enrolling additional patients as backfill at active dose levels with the deeper lymphodepletion regimen.
•No DLTs have been observed in CaMMouflage.
•Caribou plans to present dose escalation data on a minimum of 15 patients at active doses from the ongoing CaMMouflage Phase 1 clinical trial in H1 2025.

CB-012, a clinical-stage allogeneic anti-CLL-1 CAR-T cell therapy for acute myeloid leukemia
•The FDA granted Fast Track and Orphan Drug designations to CB-012 for relapsed or refractory acute myeloid leukemia (r/r AML).
•Caribou is enrolling patients with r/r AML in the dose escalation portion of the ongoing AMpLify Phase 1 clinical trial. Enrollment has concluded for dose level 3 (150×106 CAR-T cells, N=3) and no DLTs were observed. Patients are being enrolled at dose level 4 (300×106 CAR-T cells).

Corporate updates
Appointed experienced executive leaders and expanded the scientific advisory board (SAB)
•In January 2024, Tim Kelly was appointed chief technology officer and he leads Caribou’s process development and manufacturing organizations.
•In August 2024, Tina Albertson, MD, PhD, was appointed chief medical officer and she leads Caribou’s clinical programs and the clinical, regulatory, and medical affairs functions.
•In January 2025, Sri Ryali was appointed chief financial officer and he leads Caribou’s corporate finance, investor relations, and corporate communications functions.

•Terri Laufer, MD, was appointed to Caribou’s SAB. She is a leading rheumatologist known for her extensive research into immune cell regulation and dysfunction that leads to autoimmune diseases.
$281M in cash, cash runway into H2 2026
•Caribou previously reported $281 million in cash, cash equivalents, and marketable securities as of September 30, 2024, which is expected to fund the current operating plan into H2 2026.

2025 Anticipated milestones
•CB-010 ANTLER: Caribou plans to present data from both the additional 2L and prior CD19 relapsed LBCL patient cohorts in H1 2025. Caribou plans to initiate a pivotal Phase 3 clinical trial in H2 2025 should data confirm the initial observation that partial HLA matching drives outcomes that are on par with autologous CAR-T cell therapies.
•CB-010 GALLOP: Caribou plans to provide updates as the GALLOP Phase 1 clinical trial in LN and ERL advances.
•CB-011 CaMMouflage: Caribou plans to present dose escalation data from the ongoing CaMMouflage Phase 1 clinical trial in r/r MM in H1 2025.
•CB-012 AMpLify: Caribou plans to provide updates on dose escalation as the AMpLify Phase 1 clinical trial in r/r AML advances.

Caribou to present at the 43rd Annual J.P. Morgan Healthcare Conference
Dr. Haurwitz is scheduled to present a corporate update at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 at 10:30 am PST.

A live webcast of the presentation will be accessible via Caribou’s website on the Events page. The archived webcast will be available on the Caribou website for 30 days after the event.

About CB-010
CB-010 is the lead clinical-stage product candidate from Caribou’s allogeneic CAR-T cell therapy platform, and it is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 clinical trial and in patients with lupus nephritis (LN) and extrarenal lupus (ERL) in the GALLOP Phase 1 clinical trial. To Caribou’s knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve CAR-T cell activity by limiting premature CAR-T cell exhaustion. The FDA granted CB-010 Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations for B-NHL and Fast Track designations for both B-NHL and refractory systemic lupus erythematosus (SLE). Additional information on the ANTLER trial (NCT04637763) and GALLOP trial (NCT06752876) can be found at clinicaltrials.gov.

About CB-011
CB-011 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the CaMMouflage Phase 1 trial. CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA genome-editing technology. To Caribou’s knowledge, CB-011 is the first allogeneic CAR-T cell therapy in the clinic that is engineered to improve antitumor activity through an immune cloaking strategy with a B2M knockout and insertion of a B2M–HLA-E fusion protein to blunt immune-mediated rejection. CB-011 has been granted Fast Track and Orphan Drug designations by the FDA. Additional information on the CaMMouflage trial (NCT05722418) can be found at clinicaltrials.gov.

About CB-012
CB-012 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in the AMpLify Phase 1 clinical trial in patients with relapsed or refractory acute myeloid leukemia (r/r AML). CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by Caribou’s patented next-generation CRISPR technology platform, which uses Cas12a chRDNA genome editing to significantly improve the specificity of genome edits. To Caribou’s knowledge, CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity. Caribou has exclusively in-licensed from Memorial Sloan Kettering Cancer Center (MSKCC) in the field of allogeneic CLL-1-targeted cell therapy a panel of fully human scFvs targeting CLL-1, from which the company has selected a scFv for the generation of the company’s CAR. CB-012 was granted Fast Track and Orphan Drug designations by the FDA. Additional information on the AMpLify trial (NCT06128044) can be found at clinicaltrials.gov.

On January 13, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported preliminary, unaudited results for the quarter and full year ended December 31, 2024 (Press release, Guardant Health, JAN 13, 2025, View Source [SID1234649659]).

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Fourth quarter 2024 preliminary unaudited financial results

For the three-month period ended December 31, 2024, as compared to the same period of 2023:
•
Total revenue of approximately $200 million, an increase of 29%

•
Reported approximately 57,300 oncology clinical tests (excluding Shield) and approximately 11,050 biopharma tests, an increase of 24% and 16%, respectively

•
Reported approximately 6,400 Shield screening tests, with revenue of approximately $4 million

Full year 2024 preliminary unaudited financial results

For the twelve-month period ended December 31, 2024, as compared to the same period of 2023:

•
Total revenue of approximately $737 million, an increase of 31%

•
Reported approximately 206,700 oncology clinical tests (excluding Shield) and approximately 40,500 biopharma tests, an increase of 20% and 35%, respectively

Preliminary unaudited free cash flow was approximately negative $84 million for the fourth quarter of 2024, and approximately negative $275 million for the full year 2024. Cash, cash equivalents, restricted cash and marketable debt securities were $944 million as of December 31, 2024.

"2024 was an outstanding year for Guardant. We delivered key milestones across the portfolio and completed the upgrade of Guardant360 LDT onto our Smart Liquid Biopsy platform. We finished the year strong with preliminary full year revenue growth of 31%, driven by strong clinical and biopharma revenue and increasing Guardant360 ASPs. Looking ahead, we believe we are well positioned to continue to drive growth in our oncology business," said Helmy Eltoukhy, co-founder and co-CEO.

"We are extremely pleased with the strong traction and support we saw in the first full quarter of commercial launch of Shield. Given this success, we are focused on rapidly growing our commercial infrastructure to capture the massive opportunity ahead. We look forward to achieving additional milestones in 2025 as we continue driving innovation and growth in our screening business," said AmirAli Talasaz, co-founder and co-CEO.

Guardant Health has not completed preparation of its financial statements for the fourth quarter or full year of 2024. The revenue, test volumes and free cash flow presented in this release for the fourth quarter and the year ended December 31, 2024, are preliminary and unaudited and are thus inherently uncertain and subject to change as we complete our financial results. The company is in the process of completing its customary year-end close and review procedures as of and for the year ended December 31, 2024, and there can be no assurance that final results for this period will not differ from these estimates. During the preparation of Guardant Health’s consolidated financial statements and related notes as of and for the year ended December 31, 2024, the company’s independent registered public accountants may identify items that could cause final reported results to be materially different from the preliminary financial estimates presented herein.

Upcoming events

Guardant Health plans to report its fourth quarter and full year audited financial results for the period ended December 31, 2024, during its February 2025 earnings call.