On May 8, 2024 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the first quarter of 2024, along with recent product highlights and corporate updates (Press release, Zai Laboratory, MAY 8, 2024, View Source [SID1234642895]).

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"Our first quarter results demonstrate strong commercial execution and pipeline progress across our potential first- and best-in-class product portfolio," said Dr. Samantha Du, Founder, Chairperson, and Chief Executive Officer of Zai Lab. "The launch of VYVGART is off to an impressive start with $13.2 million of sales in the first quarter. Looking ahead, we expect to accelerate commercial performance for the remainder of the year and are preparing for three new potential launches in 2024. We are also excited by the progress of our late-stage pipeline and we are on track to achieve the objectives outlined in our five-year strategic plan, including significant revenue growth and profitability by the end of 2025."

"Our net revenues grew 39% y-o-y or 43% y-o-y at CER in the first quarter, driven by strong execution with the launch of VYVGART and uptake of our existing portfolio," said Josh Smiley, President and Chief Operating Officer of Zai Lab. "With VYVGART’s launch in gMG at the end of last year, and multiple new products and indications expected to launch over the near-term, we are now entering a period of robust growth for Zai Lab. Our significant growth, coupled with our focus on driving efficiencies and productivity across the organization, will drive the evolution of Zai Lab into a profitable, high growth business by the end of 2025. Furthermore, we will continue to focus on expanding our global portfolio through our internal discovery activities and strategic business development," Mr. Smiley concluded.

First-Quarter 2024 Financial Results

•Product revenue was $87.1 million in the first quarter of 2024, compared to $62.8 million for the same period in 2023, representing 39% y-o-y growth and 43% y-o-y growth at CER. This increase was primarily driven by increased sales volumes, including from the launch of VYVGART last September and decreased sales rebates to distributors resulting from price reductions in connection with listings on China’s National Reimbursement Drug List (NRDL) for certain products. This revenue growth included the following:

–ZEJULA: $45.5 million in the first quarter of 2024, an increase of 7% y-o-y from $42.7 million for the same period in 2023, driven by increased hospital sales in first-line ovarian cancer and increased duration of treatment and supported by the renewal of ZEJULA’s NRDL listing for the maintenance treatment of adult patients with first-line and recurrent ovarian cancer, effective January 1, 2024.

–VYVGART: $13.2 million in the first quarter of 2024, compared to nil for the same period in 2023, driven by positive physician and patient reception as well as increased patient access as VYVGART is added to hospital formularies. VYVGART was launched for the treatment of generalized myasthenia gravis (gMG) in September 2023 and was subsequently included for first-time NRDL listing effective, January 1, 2024.

–OPTUNE (Tumor Treating Fields): $12.5 million in the first quarter of 2024, a decrease of 6% y-o-y from $13.3 million for the same period in 2023. Although revenue declined y-o-y for OPTUNE, it increased 49% versus the fourth quarter of 2023, with continued recovery of patient volume expected throughout 2024.

–QINLOCK: $6.1 million in the first quarter of 2024, an increase of 367% y-o-y from $1.3 million for the same period in 2023, driven by its inclusion in the NRDL in the first quarter of 2023 for the fourth-line treatment of advanced gastrointestinal stromal tumors (GIST).

–NUZYRA: $9.9 million in the first quarter of 2024, an increase of 81% y-o-y from $5.5 million for the same period in 2023, driven by the NRDL listings for the IV formulation of NUZYRA for the treatment of adults with community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI) in the first quarter of 2023 and the oral formulation for these indications in the first quarter of 2024.

•Research and Development (R&D) expenses were $54.6 million in the first quarter of 2024, compared to $48.5 million for the same period in 2023. This increase was primarily due to increased clinical trial expenses related to newly initiated studies and progress of existing studies, partially offset by a decrease in milestone fees for our licensed products.

•Selling, General and Administrative expenses were $69.2 million in the first quarter of 2024, compared to $62.5 million for the same period in 2023. This increase was primarily driven by higher general selling expenses and headcount growth associated with the VYVGART launch.

•Net loss was $53.5 million in the first quarter of 2024, or a loss per ordinary share attributable to common stockholders of $0.05 (or loss per American Deposit Share (ADS) of $0.55), compared to a net loss of $49.1 million for the same period in 2023, or a loss per ordinary share of $0.05 (or loss per ADS of $0.51).

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $750.8 million as of March 31, 2024, compared to $806.5 million as of December 31, 2023.

Corporate Update

•In April 2024, Andrew Zhu joined Zai Lab as our Chief Commercial Officer in Greater China2. Mr. Zhu’s rich experience in building innovative business models and resource integration will help us further enhance our commercial operations and drive sales and profit growth across Greater China. He joins us from Simcere Zaiming, where he most recently served as Chief Operating Officer responsible for the commercial and pharmaceutical business. He previously served in various operational, sales, and marketing leadership roles at leading global biopharmaceutical companies, including AstraZeneca, Roche, Sanofi, and Bristol Myers Squibb (BMS).

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•Tumor Treating Fields:
–In March 2024, Zai Lab partner Novocure announced positive topline results from the Phase 3 METIS clinical trial for brain metastases from non-small cell lung cancer (NSCLC). The primary endpoint was met with Tumor Treating Fields therapy and supportive care demonstrating a significant improvement in time to intracranial progression versus supportive care alone (21.9 months median versus 11.3 months, respectively). These results will be presented as a late-breaking abstract at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago on June 3. We are participating in the Greater China portion of the METIS trial.

2

•Bemarituzumab (FGFR2b):
–We are enrolling patients in Greater China for the global Phase 3 FORTITUDE-101 and FORTITUDE-102 studies:
◦FORTITUDE-101 is a Phase 3 study of bemarituzumab plus chemotherapy in first-line gastric cancer.
◦FORTITUDE-102 is a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab in first-line gastric cancer.

•Tisotumab Vedotin (Tissue Factor ADC):
–In April 2024, Zai Lab partner Pfizer Inc. and Genmab A/S announced that the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (sBLA) granting full approval for tisotumab vedotin (or TIVDAK) for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. We are participating in the global Phase 3 innovaTV 301 trial and extension study in Greater China.

•Adagrasib (KRASG12C):
–We are evaluating the clinical data of the global Phase 3 KRYSTAL-12 study evaluating adagrasib in previously treated patients with KRASG12C-mutated NSCLC as we decide on next steps in the development of this product across indications.

•ZL-1310 (DLL3 ADC):
–In March 2024, Zai Lab presented findings from preclinical studies highlighting the therapeutic potential of ZL-1310 at the European Lung Cancer Congress (ELCC) 2024.
–We are enrolling patients in the United States and Greater China in the global Phase 1 study in relapsed and refractory second-line+ small cell lung cancer (SCLC) who have progressed after platinum-based treatment.

•ZL-1218 (CCR8):
–We are enrolling patients in the United States, Europe, and Greater China in the global Phase 1 study of ZL-1218 as a single agent and in combination with pembrolizumab in patients with advanced solid tumor malignancies.

Autoimmune Disorders, Infectious Disease, and Neuroscience Pipeline

•Efgartigimod (FcRn):
–In April 2024, Zai Lab submitted an sBLA for efgartigimod SC for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to the National Medical Products Administration (NMPA).

•Xanomeline-Trospium (or KarXT) (M1/M4-agonist):
–In April 2024, Zai Lab partner BMS presented new interim long-term data from the Phase 3 EMERGENT program at the Annual Congress of the Schizophrenia International Research Society (SIRS).
◦In the new interim analysis of long-term efficacy data from the Phase 3 EMERGENT-4 open-label extension trial, KarXT was associated with significant improvement in symptoms of schizophrenia across all efficacy measures at 52 weeks.
◦In the new pooled interim long-term safety and metabolic outcomes from the Phase 3 EMERGENT-4 and EMERGENT-5 trials, KarXT demonstrated a favorable long-term metabolic profile where most patients experienced stability or improvements on metabolic parameters over 52 weeks of treatment.
–We are enrolling patients in a registrational bridging study in mainland China.

Anticipated Major Milestones in 2024

Oncology

Repotrectinib
•Potential NMPA approval of our NDA for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC.

Tumor Treating Fields
•Zai Lab to submit a Marketing Authorization Application (MAA) to the NMPA in second-line+ NSCLC, following progression on or after platinum-based therapy.
•Novocure to provide a topline data readout from the Phase 3 PANOVA-3 clinical trial in locally advanced pancreatic cancer in the fourth quarter of 2024. We are participating in the study in Greater China.

3

ZL-1310 (DLL3 ADC)
•Potential dose escalation data from the global Phase 1 study in relapsed and refractory second-line+ SCLC at the end of 2024 or early 2025.

Neuroscience, Autoimmune Disorders, and Infectious Diseases (NSAiID)

Efgartigimod (FcRn)
•Potential NMPA approval of the BLA for efgartigimod SC for gMG.
•We plan to join in the registrational study of efgartigimod SC in Thyroid Eye Disease (TED) in Greater China in the second half of 2024.

Sulbactam-Durlobactam (SUL-DUR)
•Potential NMPA approval of our NDA for infections caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (ABC).

Xanomeline-Trospium (KarXT) (M1/M4-agonist)
•Zai Lab to complete patient enrollment in the China bridging study in schizophrenia.
•Zai Lab to join the global Phase 3 ADEPT-2 and ADEPT-3 studies in Alzheimer’s disease with psychosis in Greater China in mid-year.
•BMS to report data from the EMERGENT-4 and EMERGENT-5 trials evaluating the long-term safety for treatment of schizophrenia in the second half of 2024.

ZL-1102 (IL-17 Humabody)
•Zai Lab to initiate a global Phase 2 study in mild-to-moderate chronic plaque psoriasis in the second quarter of 2024.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast tomorrow, May 9, 2024, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at View Source Participants must register in advance of the conference call.

Details are as follows:

Registration Link: https://register.vevent.com/register/BIb8622a7cf98e46cd9bc9198a5f105c36

All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering.

A replay will be available shortly after the call and can be accessed by visiting the Company’s website.

On May 8, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the quarter ended March 31, 2024, and provided a business update (Press release, Syndax, MAY 8, 2024, View Source [SID1234642894]).

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"With the potential near-term approvals of revumenib and axatilimab in the third quarter as well as pivotal data from the mNPM1 cohort of the AUGMENT-101 trial in the fourth quarter, the Company is on track to have a historic year punctuated by major value-creating milestones," said Michael A. Metzger, Chief Executive Officer. "Syndax is unparalleled as a SMID cap oncology company with the potential launch of two first- and best-in-class agents into multi-billion-dollar markets with the opportunity for expansion beyond their initial indications. We remain keenly focused on laying the foundation and building an experienced team of experts to ensure our successful transition into a commercial organization."

Recent Pipeline Progress and Anticipated Milestones

Revumenib

In March 2024, the Company announced that the FDA had granted Priority Review for the New Drug Application (NDA) filing for revumenib, a potent, selective small molecule menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia. The NDA filing is being reviewed under the FDA’s Real-Time Oncology Review Program (RTOR) and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2024. RTOR allows for a more efficient review and close engagement between the sponsor and the FDA throughout the submission process, which historically has led to earlier approvals.
In March 2024, the Company also announced the completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth quarter of 2024 and could support a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025.
Positive results from a subset of the pivotal AUGMENT-101 trial in pediatric patients with R/R KMT2Ar AML and acute lymphoid leukemia (ALL) treated with revumenib were featured in a plenary session at the American Society of Pediatric Hematology/Oncology (ASPHO) Annual Meeting in April 2024.
Multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemias are ongoing across the treatment landscape. The trials are expanding to validate recommended Phase 2 doses, with additional data expected in the second half of 2024. These trials include:
BEAT AML: Evaluating the combination of revumenib with venetoclax and Azacytidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial.
SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from the MD Anderson Cancer Center.
AUGMENT-102: Evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias.
A Phase 1 trial of revumenib in combination with 7+3 chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias was initiated during the quarter.
The Company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy by year-end 2024.
Enrollment is ongoing in a Phase 1 proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer. The Company expects to provide an update on the trial in the second quarter of 2024.
Axatilimab

In February, the Company announced that the FDA had accepted the Biologics License Application (BLA) filing for axatilimab, an anti-CSF-1R antibody, in patients with chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The application was granted Priority Review and assigned a PDUFA action date of August 28, 2024.
Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF).
Our partner Incyte plans to initiate two combination trials with axatilimab in chronic GVHD in 2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids.
Corporate Updates

In March 2024, the Company announced the appointment of Steven Closter as Chief Commercial Officer. Mr. Closter brings to Syndax more than 30 years of commercial experience in the biopharmaceutical industry.
First Quarter 2024 Financial Results

As of March 31, 2024, Syndax had cash, cash equivalents, and short and long-term investments of $522.0 million and 85.3 million common shares and prefunded warrants outstanding.

First quarter 2024 research and development expenses increased to $56.5 million from $34.1 million for the comparable prior year period. The increase in research and development expenses was primarily due to increased clinical development and manufacturing costs, increased employee-related expenses and professional fees, and development milestone expense recognized in the current period.

First quarter 2024 selling, general and administrative expenses increased to $23.0 million from $12.0 million for the comparable prior year period. The increase in selling, general and administrative expenses was primarily due to increased employee-related expenses and professional fees as well as increased commercialization activities for revumenib and axatilimab.

For the three months ended March 31, 2024, Syndax reported a net loss attributable to common stockholders of $72.4 million, or $0.85 per share, compared to a net loss attributable to common stockholders of $41.1 million, or $0.59 per share, for the comparable prior year period.

Financial Guidance

For the second quarter of 2024, the Company expects research and development expenses to be $50 to $55 million and total operating expenses to be $80 to $85 million. For the full year of 2024, the Company continues to expect research and development expenses to be $240 to $260 million and total operating expenses to be $355 to $375 million, which includes an estimated $43 million in non-cash stock compensation expense.

The Company believes that it has sufficient cash runway to fund its research, clinical development and commercial operations through 2026.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 8:00 a.m. ET today, Wednesday, May 8, 2024.

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

Conference ID: Syndax1Q24
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: https://www.veracast.com/webcasts/syndax/events/SNDX1Q24.cfm

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

About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and IPF. Positive topline results from the Phase 2 AGAVE-201 trial showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and Phase 1/2 data of axatilimab in chronic GVHD were published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Syndax has exercised its option under the collaboration agreement to co-commercialize axatilimab in the U.S. and will provide 30% of the commercial effort. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

About the Real-Time Oncology Review Program (RTOR)

RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: View Source

On May 8, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that management will participate in a fireside chat at the BofA Securities 2024 Health Care Conference, taking place in Las Vegas, Nevada on Tuesday, May 14, 2024 at 3:40 p.m. PT / 6:40 p.m. ET (Press release, SpringWorks Therapeutics, MAY 8, 2024, View Source [SID1234642893]).

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To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

On May 8, 2024 Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the "Company"), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported financial results for the first quarter ended March 31, 2024, and recent corporate updates (Press release, Selecta Biosciences, MAY 8, 2024, View Source [SID1234642892]).

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"We continue to make meaningful progress advancing our innovative pipeline of product candidates and remain on track to report topline results from the Phase 2b trial of our lead product candidate, Descartes-08 for MG, in mid-2024," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. "Looking ahead, we also expect to commence dosing in our Phase 2 trial of Descartes-08 in patients with SLE by the end of the second quarter. Descartes-08 is designed with our novel mRNA-engineered CAR-T technology, is not expected to require preconditioning chemotherapy, and is intended to be administered in an outpatient setting. We remain confident Descartes-08 has the potential to expand the reach of cell therapy to patients with autoimmune diseases and serve as the first CAR-T cell therapy for the treatment of autoimmunity."

Dr. Brunn continued, "Additionally, we were excited to announce plans to transition to new corporate headquarters in Frederick, Maryland, that will provide us with the infrastructure to support our next phase of growth. We expect this new facility will allow us to scale our wholly owned, in-house cGMP manufacturing capabilities for late-stage clinical and commercial supply of our mRNA cell therapy product candidates, while continuing to maintain control over product quality and production."

Recent Pipeline Progress and Anticipated Milestones

Descartes-08 for Myasthenia Gravis (MG)

Topline data from randomized Phase 2b trial in patients with MG on track for mid-2024.
Recently granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of MG.
Previously disclosed positive, long-term follow-up results from Phase 2a trial. In April 2024, the Company announced that these data will be featured in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting on May 10, 2024 in Baltimore, Maryland.
Descartes-08, the Company’s lead product candidate, is an autologous anti-B cell maturation antigen (BCMA) mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T).
Descartes-08 for Systemic Lupus Erythematosus (SLE)

Dosing of first patient in Phase 2 trial of Descartes-08 in patients with SLE expected in second quarter of 2024.
The Phase 2 trial is designed to assess the safety and tolerability of outpatient Descartes-08 administration without preconditioning chemotherapy.
SLE is an incurable autoimmune disease marked by systemic inflammation that affects multiple organ systems and impacts approximately 1.5 million people in the United States.
Descartes-15 for Multiple Myeloma

Planning for the first-in-human Phase 1 dose escalation trial is underway to assess the safety and tolerability of outpatient Descartes-15 administration in patients with multiple myeloma.
Descartes-15 is a next-generation autologous anti-BCMA mRNA CAR-T product candidate designed to have predictable and controllable pharmacokinetics, potentially circumventing preconditioning chemotherapy, and avoiding the risk of genomic integration.
The Company expects to subsequently assess Descartes-15 in autoimmune indications.
Corporate Updates

Preferred Stock Conversion and Reverse Stock Split Approved at Special Meeting of Stockholders

In March 2024, Cartesian announced the approval for the conversion of the Company’s Series A Non-Voting Convertible Preferred Stock into the Company’s common stock and a 1-for-30 reverse stock split of the Company’s common stock.
Following the reverse stock split and the automatic conversion of the Company’s Series A Non-Voting Convertible Preferred Stock into common stock, the number of issued and outstanding shares of the Company’s common stock is approximately 17.8 million shares.
Transitioning Corporate Headquarters to Frederick, Maryland

In March 2024, the Company announced plans to transition its corporate headquarters to Frederick, Maryland. Following this announcement, Cartesian further expanded the footprint of this facility by approximately 30% through an amended agreement.
The Company now has approximately 27,000 square feet of state-of-the-art current good manufacturing practice (cGMP) compliant manufacturing and laboratory space, as well as general and administrative office space to support the Company’s continued growth. This facility reinforces the development of Cartesian’s clinical and preclinical programs through clinical and commercial manufacturing scale capabilities and advanced research and development laboratory space.
By conducting all manufacturing in-house, Cartesian expects to optimize processes more rapidly and iteratively while directly working to ensure adherence to strict quality standards. The Company believes this facility will facilitate production of potent yet safer, cost-effective mRNA cell therapy product candidates for late-stage clinical and commercial supply.
First Quarter 2024 Financial Results

Cash, cash equivalents, and restricted cash of approximately $104.8 million as of March 31, 2024. The Company’s cash, cash equivalents and restricted cash as of March 31, 2024 is expected to support planned operations and the development of Cartesian’s pipeline into the second half of 2026, including the planned Phase 3 trial of Descartes-08 in MG.
Research and development expenses were $9.7 million for the quarter ended March 31, 2024, compared to $18.6 million for the quarter ended March 31, 2023. The decrease in research and development expenses of $8.9 million for the quarter ended March 31, 2024 was primarily the result of reductions in expenses incurred for preclinical and clinical programs due to the strategic reprioritization in the Company’s clinical pipeline.
General and administrative expenses were $9.5 million for the quarter ended March 31, 2024, compared to $5.7 million for the quarter ended March 31, 2023. The increase in expense of $3.8 million for the quarter ended March 31, 2024 was primarily due to an increase in professional fees incurred in connection with the Company’s merger in November 2023.
Net loss was $(56.8) million, or $(10.50) per share (basic/diluted), for the quarter ended March 31, 2024, compared to net loss of $(21.7) million, or $(4.24) per share (basic/diluted), for the quarter ended March 31, 2023.

On May 8, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the first quarter 2024 (Press release, Sana Biotechnology, MAY 8, 2024, View Source [SID1234642891]).

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"We have four ongoing clinical trials in seven indications, and we remain on track to share initial data from each of these studies in 2024," said Steve Harr, Sana’s President and Chief Executive Officer. "We strengthened our capital position with the financing in the first quarter, enabling us to share readouts from multiple clinical studies with our current balance sheet. 2024 is a year of execution at Sana with a goal of better understanding our hypoimmune platform, its potential to treat a number of prevalent diseases, and the impacts of clinical data on our longer-term plans."

Recent Corporate Highlights

Advancing four clinical programs across seven indications, including an allogeneic CAR T program targeting CD19+ cancers, an allogeneic CAR T program for B-cell mediated autoimmune diseases, an allogeneic CAR T program targeting CD22+ cancers, and a primary islet cell therapy in type 1 diabetes:

•
SC291 is a hypoimmune (HIP)-modified CD19-directed allogeneic CAR T for patients with B-cell malignancies and B-cell mediated autoimmune diseases.
•
The ARDENT trial evaluates SC291 in patients with B-cell malignancies. Early SC291 data from the ongoing ARDENT trial suggest the ability to dose safely, the desired immune evasion profile, and early clinical efficacy. Enrollment in this study continues, and Sana expects to share more data in 2024.
•
The GLEAM trial evaluates SC291 in patients with B-cell mediated autoimmune diseases including lupus nephritis, extrarenal lupus, and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. The trial has begun enrollment, and Sana expects to share initial data in 2024.
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The VIVID trial evaluates SC262, a HIP-modified CD22-directed allogeneic CAR T, in patients with relapsed or refractory B-cell malignancies. The VIVID trial initially investigates SC262 in patients who have received prior CD19-directed CAR T therapy. The trial has begun enrollment, and Sana expects to share initial data in 2024.
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UP421 is a primary human HIP-modified islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial is to understand survival and immune evasion of allogeneic islet cells in patients with autoimmunity and without immunosuppression. The Clinical Trial Application (CTA) cleared in 4Q 2023, and Sana expects to share initial data in the first half of 2024.

Published preclinical data in Cell Stem Cell demonstrating that HIP-modified allogeneic islet cells provided lasting endocrine function in a fully immunocompetent non-human primate with type 1 diabetes, enabling the achievement of exogenous insulin independence without immunosuppression for six-month study duration:

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Sana developed HIP-modified allogeneic islet cells, which cluster into effective endocrine organoids termed "pseudo islet grafts" (p-islets). HIP p-islets engrafted and provided stable endocrine function, enabling insulin independence without immunosuppression.

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The allogeneic HIP p-islet graft survived for the six-month duration of the study with no indication of immune recognition of the HIP p-islet engraftment at any time.
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To demonstrate that there was no regeneration or recovery of an endogenous islet cell population in the diabetic NHP, HIP p-islets were eliminated using an anti-CD47 antibody, demonstrating proof of principle of CD47 overexpression and a potential safety switch.

Completed financing with gross proceeds of approximately $189.8 million to further support activities to enable multiple data readouts:

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Closed on an upsized public offering in February 2024 of 21.8 million shares of Sana’s common stock, which includes the full exercise of the underwriter’s option, and pre-funded warrants to purchase 12.7 million shares of Sana’s common stock. The gross proceeds from this offering were approximately $189.8 million before deducting underwriting discounts and commissions and estimated offering expenses.

First Quarter 2024 Financial Results

GAAP Results

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Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2024 were $311.1 million compared to $205.2 million as of December 31, 2023. The increase of $105.9 million was primarily driven by net proceeds from equity financings of $181.5 million during the three months ended March 31, 2024, partially offset by cash used in operations of $65.6 million and cash used for the purchase of property and equipment of $15.8 million.
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Research and Development Expenses: For the three months ended March 31, 2024, research and development expenses, inclusive of non-cash expenses, were $56.4 million compared to $67.2 million for the same period in 2023. The decrease of $10.8 million was primarily due to lower personnel-related costs, including non-cash stock-based compensation, and laboratory costs due to a decrease in headcount related to the strategic repositioning in the fourth quarter of 2023, decreased research costs, and lower costs for third-party manufacturing at contract development and manufacturing organizations. These decreases were partially offset by increased clinical development costs. Research and development expenses include non-cash stock-based compensation of $5.8 million and $6.0 million for the three months ended March 31, 2024 and 2023, respectively.
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Research and Development Related Success Payments and Contingent Consideration: For the three months ended March 31, 2024, Sana recognized a non-cash expense of $38.0 million compared to $0.1 million for the same period in 2023, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. The value of these potential liabilities fluctuate significantly with changes in Sana’s market capitalization and stock price.
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General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2024, inclusive of non-cash expenses, were $16.3 million compared to $16.8 million for the same period in 2023. The decrease of $0.5 million was primarily due to lower personnel-related costs due to a decrease in headcount related to the strategic repositioning in the fourth quarter of 2023, and a decrease in costs related to Sana’s previously planned manufacturing facility in Fremont, California. These decreases were partially offset by an increase in patent and other legal fees. General and administrative expenses include non-cash stock-based compensation of $3.2 million and $2.8 million for the three months ended March 31, 2024 and 2023, respectively.
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Net Loss: Net loss for the three months ended March 31, 2024 was $107.5 million, or $0.49 per share, compared to $82.1 million, or $0.43 per share for the same period in 2023.
Non-GAAP Measures

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Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the three months ended March 31, 2024 was $58.7 million compared to $74.8 million for the same period in 2023. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, cash outflows from business development, non-recurring items, and the purchase of property and equipment.
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Non-GAAP Net Loss: Non-GAAP net loss for the three months ended March 31, 2024 was $69.5 million, or $0.32 per share, compared to $82.0 million, or $0.43 per share, for the same period in 2023. Non-GAAP net loss excludes non-cash expenses and gains related to the change in the estimated fair value of contingent consideration and success payment liabilities.