On October 2, 2023 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a growing pipeline, including Phase II clinical programs, for hard-to-treat cancers and viruses, reported the initial subjects have been treated in the Phase 2 portion of the Company’s Phase Ib/II trial evaluating Annamycin in combination with Cytarabine (Ara-C) for the treatment of subjects with AML (MB-106) (Press release, Moleculin, OCT 2, 2023, View Source [SID1234635563])

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Walter Klemp, Chairman and Chief Executive Officer of Moleculin commented, "This milestone marks an important step in advancing the development of Annamycin for the treatment of AML. Based on the positive preliminary results demonstrated in the Phase 1B portion of this trial, we continue to believe that Annamycin has the potential to be a meaningful treatment option for the treatment of AML. Our team is dedicated to building on the momentum and encouraging results to bring this study across the finish line and towards late-stage development."

The Phase 1B/2 MB-106 trial is an open label trial that builds on the safety and dosage data from the two successfully concluded single agent Annamycin AML Phase 1 trials, MB-104 and MB-105, conducted in the U.S. and Europe, respectively.

As previously announced, Moleculin successfully and safely completed the Phase 1B portion of the trial at 230 mg/m2 of Annamycin in this combination study in August 2023. The total complete response (CR) or complete response with incomplete recovery of peripheral blood count (CRi) demonstrated in the Phase 1B portion of this combination trial was two out of six subjects or 33%. The median age of these subjects was 66 years of age. These data are preliminary and subject to change. In light of the safety and encouraging efficacy seen in the Phase 1B portion of this trial and the final (240 mg/m2) cohort of the MB-105 single agent trial, the safety review committee have concluded (and the Company concurs) that the Phase 2 portion will be conducted at the 230 mg/m2 level of Annamycin dosing.

Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory AML and soft tissue sarcoma (STS) lung metastases and the Company believes it may have the potential to treat additional indications.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of STS lung metastases and the treatment of relapsed or refractory AML. For more information about the MB-106 Phase 1/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.

On October 2, 2023 Immix Biopharma Inc. (Nasdaq: IMMX) ("ImmixBio", "Company", "We" or "Us") reported the presentation of updated data from the ongoing Phase Ib/II NEXICART-1 (NCT04720313) study of its novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201 (Press release, Immix Biopharma, OCT 2, 2023, View Source [SID1234635562]). The updated dataset consists of 63 multiple myeloma patients (including data on 13 new patients and continued follow-up data on 50 previously enrolled patients), at a poster presentation at the 20th International Myeloma Society Annual Meeting in Athens, Greece on September 27-30, 2023 (72 patient count includes 9 AL Amyloidosis patients which will be announced separately).

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"We continue to be very encouraged by NXC-201 clinical results," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator. "We feel these overall response rate data are compelling as 72% was the overall response rate reported for ABECMA from its pivotal 100-patient KarMMa trial in relapsed/refractory multiple myeloma. Additionally, we believe NXC-201 has demonstrated promise in the rapidly growing segment of BCMA-exposed relapsed/refractory multiple myeloma patients who are frequently excluded from CAR-T clinical trials."

Updated clinical data in 63 patients from the ongoing NEXICART-1 (NCT04720313) study of NXC-201 for the treatment of relapsed/refractory multiple myeloma are as of the July 17, 2023 data cut-off. Median follow-up was 11.9 months (range: 0.6-19.0 months). NXC-201 clinical data showed:

95% overall response rate (36 of 38 patients at the therapeutic dose of 800 million CAR+T cells) in relapsed/refractory multiple myeloma who were not exposed to prior BCMA-targeted therapy, producing a median progression free survival (mPFS) of 12.9 months
90% overall response rate (45 of 50 patients at the therapeutic dose of 800 million CAR+T cells) in relapsed/refractory multiple myeloma (including patients with, and without prior BCMA-targeted therapy)
58% complete response rate (29 out of 50 patients at the therapeutic dose of 800 million CAR+T cells) (including patients with, and without prior BCMA-targeted therapy)
Prior NXC-201 dose escalation levels included: 150 x 106 (n=6), 450 x 106 (n=7), and 800 x 106 (n=50) CAR+T cells. The overall response rates for each dose level were: 50%, 86%, and 90%, respectively
Favorable NXC-201 safety data support the potential for NXC-201, if ultimately approved, to reduce hospitalization, with an opportunity for use as outpatient CAR-T cell therapy, potentially reducing related hospitalization costs by up to 80%
"We continue on our path toward developing NXC-201 as a differentiated CAR-T therapy," said Gabriel Morris, Chief Financial Officer of Immix Biopharma. "The waiting lists at major academic medical centers in the United States for multiple myeloma CAR-Ts reflect unmet medical need. We believe that NXC-201 could provide patients, especially those exposed to a prior BCMA-targeted therapy, with an important treatment option and we look forward to next steps in our clinical program, led by our US IND filing, planned for Q4 2023."

"We are pleased to report on the consistent positive overall response rate and follow-up data from our 63-patient cohort at IMS," said Ilya Rachman, M.D. Ph.D., Chief Executive Officer of Immix Biopharma. "Industry data suggest that 95% of US medical centers do not offer CAR-T today due to its severe side effect profile. Favorable tolerability observed to date for NXC-201 suggest that, if approved, it could substantially reduce hospitalization from current practice and be largely used in an outpatient setting. This could meaningfully reduce hospital costs and enable NXC-201 CAR-T therapy to be widely adopted."

The 20th IMS multiple myeloma poster can be accessed on the ImmixBio corporate website at this link: View Source

Immix Biopharma Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma.png

Immix Biopharma Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma

Poster Presentation:

Event 20th International Myeloma Society Annual Meeting, Athens, Greece
Title "Safety and efficacy of a locally produced novel anti-BCMA chimeric antigen receptor T-cell (CART) (HBI0101) for the treatment of relapsed and refractory multiple myeloma"
Presentation
Date/Time (EEST)
September 27, 2023 9:00am – 14:30pm;
September 28, 2023 10:00 – 13:30pm;
September 29, 2023 9:30 – 14:15pm
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 was to characterize the safety and confirm the recommended Phase 2 dose (RP2D) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

The Phase 1b portion of the ongoing Phase 1b/2a clinical trial has been successful in determining the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells. The expected primary endpoints for the Phase 2 portion of the ongoing Phase 1b/2a clinical trial of NXC-201 in relapsed/refractory multiple myeloma are overall response rate and duration of response. ImmixBio plans to submit data to the FDA in multiple myeloma once 100 patients are treated with NXC-201. The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. ImmixBio plans to submit data to the FDA in AL amyloidosis once 30-40 patients are treated with NXC-201.

About NXC-201
NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis.

About Multiple Myeloma
Multiple myeloma ("MM") is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 35,730 patients in the United States diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents remains poor. The $13.9 billion Multiple Myeloma market in 2017 is expected to reach $28.7 billion in 2027 according to Wilcock, et al. Nature Reviews.

On October 2, 2023 iBioPharma presents its corporate presentation (Presentation, iBioPharma, OCT 2, 2023, View Source [SID1234635561]).

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On October 2, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported the entry into a definitive agreement for the immediate exercise of certain outstanding (i) Series A warrants to purchase up to an aggregate of 1,734,105 shares of common stock and Series B Series warrants to purchase up to an aggregate of 1,734,105 shares of common stock, each having an exercise price of $4.075 per share, issued by Checkpoint on December 16, 2022 and (ii) Series A warrants to purchase up to an aggregate of 1,428,572 shares of common stock and Series B Series warrants to purchase up to an aggregate of 1,428,572 shares of common stock issued by Checkpoint on February 22, 2023, each having an exercise price of $5.00 per share, at a reduced exercise price of $1.76 per share (Press release, Checkpoint Therapeutics, OCT 2, 2023, View Source [SID1234635560]). The shares of common stock issuable upon exercise of the warrants are registered pursuant to effective registration statements on Form S-3 (File No. 333-251005) and Form S-3 (File No. 333-270474), respectively. The gross proceeds to Checkpoint from the exercise of the warrants are expected to be approximately $11.13 million, prior to deducting placement agent fees and estimated offering expenses. The closing of the offering is expected to occur on or about October 4, 2023, subject to satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

In consideration for the immediate exercise of the warrants for cash, Checkpoint will issue new unregistered Series A warrants to purchase up to 6,325,354 shares of common stock and new unregistered Series B warrants to purchase up to 6,325,354 shares of common stock. The new Series A warrants will have an exercise price of $1.51 per share, will be exercisable immediately upon issuance and have a term equal to five years from the date of issuance. The new Series B warrants will have an exercise price of $1.51 per share, will be exercisable immediately upon issuance and have a term equal to 24 months from the date of issuance.

Checkpoint intends to use the net proceeds of this offering for working capital and general corporate purposes, including the manufacturing of cosibelimab and certain pre-commercial activities in anticipation of potential approval and commercial launch.

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act"), and, along with the shares of common stock issuable upon exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. Checkpoint has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants.

This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in this offering, 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 other jurisdiction.

On October 2, 2023 AskAt reported a Notice of Allowance for a Mexican patent dated September 15, 2023 from the National Institute of Indigenous Peoples (Instituto Nacional de los Pueblos Indígenas, INPI) for a salt and crystal forms patent for its CB2 agonist, AAT-730 (Press release, AskAt, OCT 2, 2023, View Source [SID1234635559]). The notice was issued in connection with Mexican Patent Application No. MX/a/2023/005430 (Filing Date: November 11, 2021). In addition to Mexico, AskAt has received a salt and crystal forms patent in Australia.

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