On November 12, 2024 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the third quarter ended September 30, 2024, and recent corporate updates (Press release, Checkpoint Therapeutics, NOV 12, 2024, View Source [SID1234648162]).

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"With the Prescription Drug User Fee Act ("PDUFA") goal date set for next month, we await the decision by the U.S. Food and Drug Administration ("FDA") on our Biologics License Application ("BLA") resubmission for cosibelimab, our anti-programmed death ligand-1 ("PD-L1") antibody," said James Oliviero, President and Chief Executive Officer of Checkpoint. "The $9.2 million in cash proceeds received this month from the exercise of existing warrants has strengthened our balance sheet to extend beyond our PDUFA date and into 2025. We are now fully focused on preparing for the potential approval of cosibelimab and look forward to potentially offering oncologists a new, differentiated treatment option for patients with advanced cutaneous squamous cell carcinoma ("cSCC")."

Recent Corporate Updates:

In July 2024, Checkpoint announced that the FDA accepted for review the resubmission of its BLA for cosibelimab as a complete response to the complete response letter ("CRL") issued in December 2023 and set a PDUFA goal date of December 28, 2024.
Also in July 2024, Checkpoint announced a collaboration to explore the combined therapeutic potential of cosibelimab, its anti-PD-L1 antibody with dual mechanism of action, with GC Cell’s Immuncell-LC, an innovative autologous Cytokine Induced Killer T cell therapy composed of cytotoxic T lymphocytes and natural killer T cells.
Also in July 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 $12.0 million.
In September 2024, Checkpoint presented longer-term data from its pivotal trial of cosibelimab in locally advanced and metastatic cSCC during the European Society for Medical Oncology ("ESMO") Congress 2024. Longer-term results for cosibelimab presented at the ESMO (Free ESMO Whitepaper) Congress demonstrate a deepening of response over time, with higher objective response and complete response rates than initially observed at the primary analyses. A copy of the ESMO (Free ESMO Whitepaper) poster can be found on the Publications page of Checkpoint’s website.
In November 2024, Checkpoint received $9.2 million in cash proceeds through the exercise of existing warrants.
Financial Results:

Cash Position: As of September 30, 2024, Checkpoint’s cash and cash equivalents totaled $4.7 million, compared to $5.0 million at June 30, 2024 and $4.9 million at December 31, 2023, a decrease of $0.3 million for the quarter and a decrease of $0.2 million, year-to-date. Subsequent to the end of the quarter, in November 2024, Checkpoint received $9.2 million in cash proceeds through the exercise of existing warrants.
R&D Expenses: Research and development expenses for the third quarter of 2024 were $6.4 million, compared to $5.5 million for the third quarter of 2023, an increase of $0.9 million. Research and development expenses for the third quarter of 2024 included $0.5 million of non-cash stock expenses, compared to $0.3 million for the third quarter of 2023.

G&A Expenses: General and administrative expenses for the third quarter of 2024 were $3.4 million, compared to $2.2 million for the third quarter of 2023, an increase of $1.2 million. General and administrative expenses for the third quarter of 2024 included $1.4 million of non-cash stock expenses, compared to $0.6 million for the third quarter of 2023.

Net Loss: Net loss attributable to common stockholders for the third quarter of 2024 was $9.7 million, or $0.23 per share, compared to a net loss of $5.7 million, or $0.29 per share, in the third quarter of 2023. Net loss for the third quarter of 2024 included $1.9 million of non-cash stock expenses, compared to $0.9 million for the third quarter of 2023.

On November 12, 2024 mAbxience, a Fresenius Kabi majority-owned group with partial ownership from Insud Pharma, and Egis Pharmaceutical PLC, reported a new strategic license agreement for the commercialization of biosimilar candidates across key Central and Eastern European markets, including Hungary, Poland, Czech Republic, Slovakia, Romania, Bulgaria, Latvia, and Lithuania (Press release, mAbxience, NOV 12, 2024, View Source [SID1234648178]). The partnership also includes the option to expand to additional territories in the future.

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Under the terms of the agreement, mAbxience will hold the marketing authorization for the biosimilars in these regions, while Egis will manage the commercialization and marketing activities. This collaboration underscores the commitment of both companies to broaden access to essential, high-quality therapies in markets where affordability and accessibility are critical.

José Ramón Millán, Global Partnering & Portfolio Director at mAbxience, commented on the partnership: "We are delighted to partner with Egis to bring our biosimilar candidate to patients in these important Eastern European markets. This agreement strengthens our presence in the region and reaffirms our dedication to ensuring that life-saving treatments are accessible and affordable. We look forward to working closely with Egis to make a significant impact on patient care."

Dr. Klara Marton, Business Development Director from Egis added: "This collaboration with mAbxience enables us to deliver high value treatments to patients across Central and Eastern Europe. We are proud to join forces with a trusted partner like mAbxience, whose commitment to high-quality, affordable healthcare solutions aligns with our mission to improve patient access and patient outcomes in our region."

This agreement marks a significant milestone in mAbxience’s expansion into new markets and further reinforces its commitment to providing accessible, high-quality biosimilar therapies to patients worldwide.

On November 12, 2024 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical stage company focused on developing next generation therapeutics to target difficult-to-treat cancers, reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, Pyxis Oncology, NOV 12, 2024, View Source [SID1234648206]). The Company ended the third quarter of 2024 with $146.3 million in cash, cash equivalents, restricted cash, and short-term investments, which is expected to provide a cash runway into the second half of 2026 and enable the Company to fund the next phase of PYX-201 clinical development.

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"At Pyxis Oncology, our team has been working diligently to deliver preliminary data from our first-in-patient clinical trial of PYX-201. Investigator enthusiasm around our novel mechanism of action, along with our clinical development team’s operational execution, has kept us on track to provide the preliminary clinical findings from our ongoing dose escalation study," said Lara S. Sullivan, M.D., President and CEO of Pyxis Oncology.

Dr. Sullivan continued, "We have now enrolled 80 patients in the PYX-201 trial across multiple tumor types, and I am thrilled to present the Phase 1 dose escalation data next Wednesday in New York City at our first-ever Pyxis Oncology Investor Event."

Recent Clinical Program Updates

PYX-201

PYX-201 is an antibody-drug conjugate (ADC) that is designed to uniquely target Extradomain-B Fibronectin (EDB+FN), which is believed to be a key structural component of the tumor extracellular matrix. PYX-201 is the Company’s lead clinical program being evaluated in an ongoing Phase 1 trial in multiple types of solid tumors.

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To date, 80 subjects have been dosed with PYX-201 in this Phase 1 trial. Dose escalation and safety monitoring remain ongoing for the trial.

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The Company will present preliminary data from the Phase 1 dose escalation trial of PYX-201, including efficacy, safety, and pharmacokinetics (PK). The Company will provide an update on future development plans at a Company hosted investor event in New York City on Wednesday, November 20, 2024, at 4:30 p.m. ET. Additional information about the event can be found here.

PYX-106

PYX-106, a fully human Siglec-15-targeting antibody designed to block suppression of T-cell proliferation and function, is being evaluated in an ongoing Phase 1 clinical study in multiple types of solid tumors.

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Dose escalation of PYX-106 and safety monitoring is ongoing with 45 subjects dosed to date in the Phase 1 trial.

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The Company expects to report preliminary data from the Phase 1 trial of PYX-106, including PK/pharmacodynamic results, by year-end 2024.

Third Quarter 2024 Financial Results

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As of September 30, 2024, Pyxis Oncology had cash and cash equivalents, including restricted cash and short-term investments of $146.3 million. The Company believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2026.

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Research and development expenses were $17.7 million for the quarter ended September 30, 2024, compared to $14.7 million for the quarter ended September 30, 2023. The period-over-period increase was primarily due to increased clinical trial-related expenses, including manufacturing of drug product and drug substance for our ongoing Phase 1 clinical trials of PYX-201 and PYX-106.

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General and administrative expenses were $6.0 million for the quarter ended September 30, 2024, compared to $10.7 million for the quarter ended September 30, 2023. The period-over-period decline was primarily due to lower legal, professional and consulting fees.

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Net loss was $21.2 million, or ($0.35) per common share, for the quarter ended September 30, 2024, compared to $23.0 million, or ($0.56) per common share, for the quarter ended September 30, 2023. Net losses for the quarters ended September 30, 2024, and 2023 included $3.0 million and $5.2 million, respectively, related to non-cash stock-based compensation expense.

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As of November 12, 2024, the outstanding number of shares of common stock of Pyxis Oncology was 59,465,729.

On November 12, 2024 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the third quarter of 2024 and provided an overview of its clinical programs and anticipated milestones (Press release, Vincerx Pharma, NOV 12, 2024, View Source [SID1234648221]).

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"As we direct our efforts and resources toward our ADC technologies and programs, we are committed to advancing VIP943 based on the encouraging safety, efficacy, and tolerability results observed to date. We look forward to presenting additional data from patients at efficacious dose cohorts by early next year," said Ahmed Hamdy, M.D., Chief Executive Officer. "Securing the funding necessary to advance our programs remains a priority. Alongside exploring financing options, we remain focused on strategic partnerships, particularly as pharmaceutical companies intensify their search for truly differentiated and transformative technologies."

THIRD QUARTER 2024 CLINICAL PROGRAM HIGHLIGHTS AND ANTICIPATED MILESTONES

VIP943

VIP943 is a novel CD123-targeted ADC developed with the Company’s next-generation VersAptx platform.
VIP943 has shown promising safety, efficacy, and tolerability in an ongoing Phase 1 dose-escalation study for patients with relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and B-cell acute lymphoblastic leukemia (B-ALL) (NCT06034275). In October, the Company reported two complete responses in this Phase 1 study: one out of four patients with relapsed AML in the 1 mg/kg dose cohort achieved complete remission with incomplete hematologic recovery (CRi), and one out of one patient with higher-risk MDS in the 1.3 mg/kg dose cohort achieved complete remission with limited count recovery (CRL).
VIP943 has shown effective target engagement and elimination of CD123+ malignant cells, with pharmacodynamic data demonstrating decreases in CD123+ blasts after dosing. Preliminary pharmacokinetic data indicates minimal payload release (≤1% in plasma), signifying a stable linker.
Given the favorable safety and tolerability observed for VIP943, the Company continues dose escalation to assess potential for additional efficacy. Enrollment in the once-weekly and twice-weekly (as an induction therapy) dosing schedules is ongoing.
Vincerx expects to share additional Phase 1 study data for VIP943 by early 2025.
Enitociclib

Enitociclib is a highly selective CDK9 inhibitor designed to block the activation of RNA polymerase II, leading to inhibition of oncogenes, including MYC and MCL1.
Enitociclib is currently in a Phase 1 dose-escalation study (NTC05371054) evaluating the combination of enitociclib, venetoclax, and prednisone in diffuse large B-cell lymphoma (DLBCL) and peripheral T-cell lymphoma (PTCL). This study is being conducted in collaboration with the National Institutes of Health (NIH). As of September 2024, the study reported four partial responses (PRs) in seven patients (57% overall response rate), including one patient with double hit lymphoma (DH-DLBCL) and three patients with PTCL.
Enitociclib has successfully completed its Phase 1 dose-escalation study as a monotherapy (NCT02635672), enrolling 63 patients across dose-escalation and expansion cohorts. The treatment demonstrated a favorable safety profile, dose-proportional pharmacokinetics, and on-target pharmacodynamic activity. Clinical benefits included durable complete metabolic remissions in two patients with DH-DLBCL, lasting 3.7 and 2.3 years, with both remissions continuing more than two years after treatment cessation. In addition, a transformed follicular (tFL) patient achieved a PR with a 63% tumor reduction after nearly two years, a meaningful outcome given the historically poor prognosis of tFL. Furthermore, 13 patients with solid tumors achieved stable disease as their best response, including five ovarian cancer patients—indicating a promising path for future combination studies in this indication.
The Company is actively focused on finding a strategic partner to continue the development of this asset.
VIP236

VIP236 is a αVβ3 SMDC conjugated to an optimized camptothecin (CPT) payload developed with the Company’s VersAptx platform.
VIP236 has completed its Phase 1 dose-escalation study (NCT05712889), identifying the maximum tolerated dose that could be utilized in future studies. As reported in October, a total of 29 patients were enrolled in the Phase 1 study, resulting in a 45% disease control rate. The drug demonstrated a favorable safety profile, distinguishing itself from other CPTs by showing no instances of common dose-limiting side effects such as life-threatening diarrhea, severe stomatitis/mucositis, or interstitial lung disease.
The Company intends to identify a partner to champion VIP236 through further development.

THIRD QUARTER 2024 FINANCIAL RESULTS

Vincerx had approximately $10.1 million in cash, cash equivalents, and marketable securities as of September 30, 2024, as compared to approximately $16.3 million as of June 30, 2024. Based on its current business plans and assumptions, Vincerx believes its available capital will be sufficient to meet its operating requirements into early 2025.
Research and development expenses for the third quarter ended September 30, 2024, were approximately $3.9 million, as compared to approximately $6.1 million for the same period in 2023. This decrease is primarily the result of decreases in research services of approximately $2.4 million and personnel-related expenses of approximately $0.8 million, offset by an increase in clinical-related expenses of approximately $0.9 million.
General and administrative expenses for the third quarter ended September 30, 2024, were approximately $3.9 million, as compared to approximately $3.5 million for the same period in 2023. This increase was due to a $0.5 million increase in professional services, partially offset by a decrease in personnel-related expenses of $0.1 million.
For the third quarter ended September 30, 2024, Vincerx reported a net loss of approximately $7.8 million, or $0.17 per share. For the third quarter ended September 30, 2023, Vincerx reported a net loss of approximately $9.0 million, or $0.42 per share.

On November 11, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported its financial results for the quarter ended September 30, 2024 (Press release, Moleculin, NOV 11, 2024, View Source [SID1234648073]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results at 8:30 AM ET on Monday, November 11, 2024 (details below).

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"We are thrilled to have emerged as a late stage company. Our focus remains on the ramp up for and execution of our upcoming MIRACLE trial. We have been extremely active and our recent interactions with potential clinical trial sites globally have been overwhelmingly positive as we prepare for the start of enrollment and dosing early next year. We believe with our recent clinical and regulatory ‘wins,’ we have foundationally set the stage for a transformational year ahead and the opportunity to drive significant value for all stakeholders. With that said, we continue to follow our CRc (complete response composite) preliminary data from our MB-106 Phase 1B/2 AML clinical trial. We are also pleased with the median durability continuing to climb – and is now in excess of 8 months," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Recent Highlights

Appointed Daniel D. Von Hoff, M.D., F.A.C.P., FASCO, FAACR, leading expert in Pancreatic Cancer to its Scientific Advisory Board to Support Development of Annamycin;
Hosted a Virtual Acute Myeloid Leukemia KOL event with internationally renowned Acute Myeloid Leukemia (AML) Key Opinion Leaders to discuss Annamycin, the use of anthracyclines, how Annamycin could significantly change the AML treatment landscape, and the Company’s recently announced global Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (the "MIRACLE" trial);
Presented positive in vivo efficacy data of Annamycin in orthotopic and experimental lung metastatic models of Sarcoma in a poster titled "Annamycin: Opening New Doors for Organotropic Targeting of Primary and Metastatic Lung Cancer," at the IASLC 2024 World Conference on Lung Cancer;
Commenced enrollment and treatment of patients in the Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma; and
Closed a $5.5 million financing upfront with up to an additional $11.0 million of potential aggregate gross proceeds upon the exercise in full of milestone-linked warrants.
Clinical Development Update

Annamycin is currently being evaluated in ongoing clinical trials for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

AML

The Company recently announced the positive discussion and outcome of its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA) supporting the advancement of Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US.

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby approximately the first 75 to 90 subjects will be randomized to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For the second part of the trial, approximately 240 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based not only on the overall balance of safety, tolerability, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Expected Milestones for Annamycin AML Development Program

4Q 2024 – Contracting with MIRACLE trial sites and IRB approval
1Q 2025 – First subject treated in MIRACLE trial
4Q 2025 – Recruitment and overall efficacy rate update (n=45)
2H 2026 – Interim efficacy and safety data (n=90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 2H – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
STS Lung Metastases

As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The study database is locked, and the clinical study report is being written and should be completed in early 2025 and will be released in detail at that time

Expected Milestones for Annamycin STS Lung Mets Development Program

2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal program
Summary of Financial Results for the Third Quarter 2024

Research and development (R&D) expense was $4.9 million and $3.3 million for the three months ended September 30, 2024 and 2023, respectively. The increase over the prior year period is mainly related to costs incurred for clinical trials (clinical research organization and drug production) and sponsored research costs.

General and administrative expense was $2.2 million and $2.6 million for the three months ended September 30, 2024 and 2023, respectively. The decrease of $0.4 million is mainly related to a decrease in regulatory and legal fees.

As of September 30, 2024, the Company had cash and cash equivalents of $9.4 million and believes that the existing cash and cash equivalents as of September 30, 2024, will be sufficient to fund planned operations into the first quarter of 2025.

Conference Call and Webcast

Moleculin management will host its quarterly conference call and webcast for investors, analysts, and other interested parties on Monday, November 11, 2024 at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live audio webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.