On May 10, 2024 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) in a late breaking oral presentation at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Baltimore, MD (Press release, Immix Biopharma, MAY 10, 2024, View Source [SID1234643082]). All patients were relapsed/refractory to standards-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone).

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"Relapsed/refractory AL Amyloidosis remains an unmet medical need, with no approved options for treatment," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and NEXICART-1 principal study investigator. "We continue to be encouraged by NXC-201’s response rates and durable effect in patients without significant pre-existing cardiac damage, exemplified by our 28-month longest responder with response ongoing."

"We are excited to present clinical data from the NEXICART-1 clinical study at ASGCT (Free ASGCT Whitepaper). This study has informed the design of NEXICART-2 clinical trial, expected to open in the U.S. mid-2024," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added: "We believe NXC-201’s tolerability profile, including lack of neurotoxicity, makes it uniquely suitable as a potential new treatment option for relapsed/refractory AL Amyloidosis patients."

At the NXC-201 ASGCT (Free ASGCT Whitepaper) 2024 late-breaking oral presentation, data were presented from 13 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2a NEXICART-1 study. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=10).

Patient characteristics:

85% (11/13) had cardiac involvement
38% (5/13) had New York Heart Association (NYHA) stage 3 or 4 heart failure
38% (5/13) had Mayo stage 3 AL amyloidosis disease
Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10)
1 patient, patient 11, was treated and progressed on a BCMA-targeted bispecific antibody before NXC-201 treatment
Safety and efficacy data:

Overall response rate (ORR) of 92% (12/13) for relapsed/refractory AL Amyloidosis patients enrolled in NEXICART-1:
12 out of 12 patients not exposed to prior BCMA-targeted bispecific responded to NXC-201 (100% ORR), of which 9 out of 12 were complete responders (75% CRs)
1 patient with prior exposure to BCMA-targeted bispecific treatment did not respond
Best responder had a duration of response of 28.0 months as of May 10, 2024, with response ongoing
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
Median cytokine release syndrome (CRS) duration was 2 days (range: 1-5):
No grade 4 CRS events
2 experienced no CRS; 3 experienced grade 1 CRS; 6 Experienced grade 2 CRS; 2 experienced grade 3 CRS
The NXC-201 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) late-breaking oral presentation can be accessed on the ImmixBio corporate website at this link: View Source

ASGCT 2024 Presentation Details:

Title
"Academic BCMA-CART cells (HBI0101), a promising approach for the treatment of LC Amyloidosis"
Type
Late Breaking Oral Presentation
Oral Presentation Date/Time
Friday, May 10, 2024, 8:45 am – 9:00 am Eastern Time
Session Title
Late-Breaking Abstracts II
Location
Baltimore Convention Center, Baltimore, MD
About NEXICART-1
NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the recommended Phase 2 dose (RP2D) of NXC-201. The Phase 1b portion has been successfully completed, with a recommended Phase 2 dose (RP2D) of 800 million CAR+T cells.

About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial in relapsed/refractory AL Amyloidosis for CAR-T NXC-201 in the United States. Over a period of approximately 18 months from first patient dosing, NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The objectives are the safety and efficacy of NXC-201. The expected primary endpoints are complete response rate and overall response rate according to consensus recommendations (Palladini et al. 2012).

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, which we believe has the potential to be the only "Single-Day CRS" CAR-T, targeting AL Amyloidosis and other autoimmune diseases. It is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, and expanding into other autoimmune indications. These trials build on a robust NXC-201 clinical dataset initiated in February 2021. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and awarded EU ODD by the EMA in multiple myeloma and AL Amyloidosis.

On May 10, 2024 FORE Biotherapeutics reported that the Company will be presenting at the 2024 RBC Capital Markets Global Healthcare Conference (Press release, Fore Biotherapeutics, MAY 10, 2024, View Source [SID1234643080]). The presentation will take place on Wednesday, May 15, 2024 at 9:00-9:25 AM ET.

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William Hinshaw, Chief Executive Officer, and Jeffrey Sacher, Interim Chief Financial Officer, will host and participate in one-on-one meetings. Please contact your RBC Capital Markets salesperson or Argot Partners to schedule one-on-one meetings with the management team.

On May 10, 2024 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported that the Company will present updated data from the TakeAim Leukemia study Tuesday, May 14, 2024, at 4:00 p.m. ET (Press release, Curis, MAY 10, 2024, View Source [SID1234643079]). Management will host a conference call on the same day at 4:30 p.m. ET.

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To access the live conference call, please dial 800-836-8184 from the United States or 646-357-8785 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the conference call will be available on the Curis website shortly after completion of the call.

On May 10, 2024 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for life-threatening diseases and conditions, reported that management will be participating in fireside chats and investor meetings at both the JMP Securities Life Sciences Conference being held in New York on May 13 – 14, 2024 and the 2024 RBC Capital Markets Global Healthcare Conference being held in New York on May 14 – 15, 2024. Details for each fireside chat is as follows (Press release, CorMedix, MAY 10, 2024, View Source [SID1234643078]):

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JMP Securities Life Sciences Conference
Date: Tuesday, May 14, 2024
Time: 9:30am EDT
Webcast: Click here

RBC Capital Markets Global Healthcare Conference
Date: Wednesday, May 15, 2024
Time: 1:35pm EDT
Webcast: Click here

A replay of each fireside chat will also be available in the "Events and Presentations" page on the investor relations portion of the Company’s website at: www.cormedix.com

On May 10, 2024 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the first quarter ended March 31, 2024, and recent corporate updates (Press release, Checkpoint Therapeutics, MAY 10, 2024, View Source [SID1234643077]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "Over the past few months, we have worked closely with our third-party contract manufacturing organization ("CMO") for cosibelimab to resolve their inspection deficiencies noted in the complete response letter ("CRL") we received from the U.S. Food and Drug Administration ("FDA") last December. Recently, our CMO submitted to FDA their response to the inspection deficiencies, which we believe could allow for the resubmission of our biologics license application ("BLA"). We plan to meet with the FDA shortly, at which time we will seek to reach alignment for a potential mid-year BLA resubmission."

Recent Corporate Updates:

Checkpoint submitted a BLA to the FDA in January 2023 seeking approval of cosibelimab as a potential new treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC") who are not candidates for curative surgery or curative radiation, and the FDA accepted the BLA for filing in March 2023. In December 2023, the FDA issued a CRL for the cosibelimab BLA. The CRL only cited findings that arose during a multi-sponsor inspection of Checkpoint’s third-party CMO as approvability issues to address in a BLA resubmission. The CRL did not state any concerns about the clinical data package, safety, or labeling for the approvability of cosibelimab. Checkpoint intends to seek to address the feedback in a potential BLA resubmission, which is currently targeted for mid-year.
In January 2024, Checkpoint completed a registered direct offering priced At-the-Market under Nasdaq rules and a concurrent private placement of warrants to purchase Checkpoint common stock, for total gross proceeds of approximately $14.0 million.
In March 2024, Checkpoint announced the appointment of life sciences executive, Amit Sharma, M.D., FACP, FASN, FNKF, currently Vice President of Clinical Development and Therapeutic Head for Nephrology and Hematology at Alexion, AstraZeneca Rare Disease, as a non-executive director to Checkpoint’s Board of Directors.
Financial Results:

Cash Position: As of March 31, 2024, Checkpoint’s cash and cash equivalents totaled $11.2 million, compared to $4.9 million at December 31, 2023, an increase of $6.3 million.
R&D Expenses: Research and development expenses for the first quarter of 2024 were $8.5 million, compared to $15.8 million for the first quarter of 2023, a decrease of $7.3 million. Research and development expenses for the first quarter of 2024 included $0.5 million of non-cash stock expenses, compared to $0.4 million for the first quarter of 2023.
G&A Expenses: General and administrative expenses for the first quarter of 2024 were $2.5 million, compared to $2.3 million for the first quarter of 2023, an increase of $0.2 million. General and administrative expenses for the first quarter of 2024 included $0.6 million of non-cash stock expenses, compared to $0.7 million for the first quarter of 2023.
Net Loss: Net loss attributable to common stockholders for the first quarter of 2024 was $10.9 million, or $0.33 per share, compared to a net loss of $10.5 million, or $0.89 per share, in the first quarter of 2023. Net loss for the first quarter of 2024 and 2023 both included $1.1 million of non-cash stock expenses.