On August 6, 2025 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 the presentation of encouraging preclinical data for its lead drug candidate, Annamycin, also known by its non-proprietary name of naxtarubicin, which demonstrated significant efficacy against various primary and metastatic liver cancers, including hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases (Press release, Moleculin, AUG 6, 2025, View Source [SID1234654867]). This is believed to be the result of targeted accumulation in the liver and other organs.

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These findings were highlighted in a poster titled "Liposomal Annamycin (L-ANN) Efficacy Against Primary and Metastatic Liver Cancers," presented by Dr. Waldemar Priebe, Lead Author and Chairman of the Scientific Advisory Board at Moleculin at the recently held Shelby-Lavine Pancreatic Cancer Symposium at MD Anderson Cancer Center.

Key Highlights

Targeted Accumulation in Organs: The preclinical studies confirmed that Annamycin exhibits distinct organotropic properties, leading to significantly higher concentrations in the liver, spleen, lungs, and pancreas when compared to doxorubicin (DOX). This targeted accumulation is critical for effectively treating liver-localized tumors.
Efficacy in Orthotopic Hepatocellular Carcinoma (HCC) Models: Annamycin demonstrated excellent anti-tumor activity in orthotopic HCC models (Hepa 1-6 Luc), showing a marked reduction in tumor progression and improved survival rates in treated animals compared to vehicle controls.
Potent Impact on Colorectal Liver Metastasis: In an experimental liver metastatic model of colorectal carcinoma (CT26 Luc), Annamycin significantly inhibited metastatic growth and extended survival, highlighting its potential for addressing liver metastases in colon cancer that affects close to 50% of patients.
Promising Results in Pancreatic Cancer Liver Metastasis: Annamycin also showed compelling efficacy in liver-implanted human pancreatic ductal adenocarcinoma (MIA PaCa-2), leading to inhibition of tumor growth, suggesting its potential role in managing advanced pancreatic cancer with liver involvement.
Favorable Safety Profile: Consistent with previous preclinical and clinical findings, Annamycin continued to show low or no cardiotoxicity, a significant advantage over traditional anthracyclines like doxorubicin, which are often limited by dose-dependent cardiac side effects. This safety profile was previously observed in clinical trials, where 32 out of 42 subjects reviewed received more than the FDA-established lifetime maximum allowable level of anthracycline without evidence of cardiotoxicity.
"We are incredibly encouraged by these preclinical results, which further validate Annamycin’s potential as a powerful and differentiated therapeutic agent for liver cancers," said Dr. Priebe. "The ability of Annamycin to concentrate effectively in the liver, combined with its demonstrated efficacy across multiple aggressive liver cancer models and its favorable cardiotoxicity profile, positions it as a highly promising candidate for patients facing these devastating diseases. We believe these data provide a strong foundation for advancing Annamycin in clinical development for these indications, offering new hope where treatment options are often limited."

Walter Klemp, Chairman and CEO of Moleculin, added, "We continue to be strongly encouraged by the potential of Annamycin. Beyond our preliminary programs in AML and soft tissue sarcoma lung metastases (STS lung mets), we continue to advance and develop Annamycin through multiple investigator-initiated studies to further unlock its potential as a treatment option for many other types of cancers. We believe this preclinical data highlights that potential and provides additional validation of its unique pharmacological profile and importantly showcases the opportunity for its use across a wide range of cancers."

Moleculin’s novel drug candidate is being positioned to become the first ever non-cardiotoxic anthracycline to be approved and is currently being developed for the treatment of AML and STS lung mets. For more information, please visit moleculin.com.

On August 6, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, reported financial results for the quarter ended June 30, 2025 and provided a business update (Press release, Immunome, AUG 6, 2025, View Source [SID1234654866]).

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"Immunome made substantial progress in the second quarter of 2025, underscored by the continued advancement of our clinical programs towards key milestones," said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. "We look forward to sharing topline data for the RINGSIDE trial of varegacestat before the end of this year, and we are well-positioned to support a new drug application submission for that program as appropriate."

"We recently dosed the third cohort of patients in the dose escalation study of IM-1021, our ROR1 ADC. We are excited by the profile of HC74, the proprietary TOP1 inhibitor payload contained in IM-1021, and are making progress towards IND submission for three additional ADCs utilizing that technology."

Pipeline Highlights

Varegacestat:

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Immunome expects to report topline data for the Phase 3 RINGSIDE Part B study before the end of 2025.
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Two additional analyses of the Phase 2 RINGSIDE Part A study were presented at 2025 ASCO (Free ASCO Whitepaper) Annual Meeting in June 2025, both of which can be found in the Investors portion of Immunome’s website:
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Subgroup Analysis of the Phase 2 Part of the RINGSIDE Phase 2/3 Trial of Varegacestat for Treatment of Desmoid Tumors
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Change in T2-Weighted Signal Intensity, Change in Tumor Volume, and Exposure-Response Analysis in the RINGSIDE Phase 2 Study of Varegacestat in Patients with Desmoid Tumors
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The European Medicines Agency granted Orphan Drug Designation to varegacestat in July 2025. Varegacestat previously received Orphan Drug Designation from the U.S. Food and Drug Administration in November 2023.
IM-1021: The Phase 1 clinical trial of IM-1021 is ongoing, with patients recently dosed at the third dose level. The trial is an open-label, multicenter dose-escalation and expansion study that is expected to include participants with advanced B-cell lymphomas and advanced solid tumors.

IM-3050: Immunome received IND clearance for IM-3050 in April 2025 and expects to initiate a Phase 1 clinical trial before the end of 2025.

Preclinical Pipeline: Immunome’s three preclinical ADCs against solid tumor targets, IM-1617, IM-1340 and IM-1335, each of which incorporates HC74, are currently undergoing IND-enabling work. Additional undisclosed ADCs are in discovery and lead optimization.

Second Quarter 2025 Financial Results

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As of June 30, 2025, cash, cash equivalents and marketable securities totaled $268.0 million. Immunome expects its current cash position to fund operations into 2027.
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Research and development expenses for the quarter ended June 30, 2025 were $40.5 million, including stock-based compensation costs of $2.2 million.
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General and administrative expenses for the quarter ended June 30, 2025 were $10.0 million, including stock-based compensation expense of $3.1 million.
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Immunome reported a net loss of $43.4 million for the quarter ended June 30, 2025.

On August 6, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported financial results for the second quarter of 2025 and recent business highlights (Press release, Geron, AUG 6, 2025, View Source [SID1234654865]). In a separate press release today, the Company announced the appointment of Harout Semerjian as incoming President and CEO.

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"We are pleased that our sharpened sales strategy is demonstrating signs of commercial success as evidenced by solid U.S. sales and increased demand across a broadening group of treating physicians," said Dawn Carter Bir, Interim President and Chief Executive Officer of Geron. "Last quarter, we set out to increase our commercial sales team by 20% and double our medical science liaisons and I’m pleased to say we have accomplished both of these goals. We believe our investments in commercial and medical affairs will help to bolster awareness and adoption of RYTELO, our first-in-class telomerase inhibitor, and with the leadership experience Harout brings to the company, we look forward to further progress over time."

Recent Business Highlights

RYTELO

Net product revenue of $49.0 million in the second quarter of 2025, an increase of 24% compared to the first quarter.
Quarter-over-quarter demand for RYTELO in the second quarter of 2025 increased by 17%, compared to 1% in the first quarter, driven by increased demand from new patient starts.
Number of ordering accounts is now over 1,000, an increase of approximately 400 year-to-date.
Geron is continuing preparatory activities for the anticipated launch of RYTELO in select EU countries, following approval earlier this year.
IMpactMF Phase 3 Clinical Trial Evaluating imetelstat in relapsed/refractory myelofibrosis

Reached over 95% enrollment as of end of July, with full enrollment expected by year-end 2025.
Interim analysis readout for overall survival expected in the second half of 2026 (when approximately 35% of patient events have occurred), and final analysis expected in the second half of 2028 (when approximately 50% of patient events have occurred).
Recent Medical and Scientific Presentations

Presented multiple presentations at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress.
Together, these presentations support the potential benefits of the first-in-class oligonucleotide telomerase inhibitor RYTELO (imetelstat) for a range of patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia and showcase the progress Geron is making with the ongoing IMpactMF and IMproveMF trials of imetelstat in myelofibrosis.
Second Quarter 2025 Financial Results

Cash and Marketable Securities

As of June 30, 2025, Geron had approximately $432.6 million in cash, cash equivalents, restricted cash and marketable securities, compared to $502.9 million as of December 31, 2024.

Net Loss

For the three and six months ended June 30, 2025, the Company reported a net loss of $16.4 million, or $0.02 per share and $36.2 million, or $0.05 per share, compared to $67.4 million, or $0.10 per share and $122.8 million, or $0.19 per share, for the three and six months ended June 30, 2024.

Revenues

Total product revenue, net for the three and six months ended June 30, 2025, was $49.0 million and $88.4 million, compared to $780,000 for the three and six months ended June 30, 2024, as RYTELO was approved by the FDA in June 2024.

Total net revenue for the three and six months ended June 30, 2025, was $49.0 million and $88.6 million, compared to $882,000 and $1.2 million for the three and six months ended June 30, 2024. Total net revenue includes license fees and royalties in addition to any product revenue, net. The increase in revenue is due to product revenue from U.S. sales of RYTELO, which was approved by the FDA in June 2024.

Operating Expenses

Total operating expenses for the three and six months ended June 30, 2025, were $61.5 million and $117.8 million, compared to $70.2 million and $126.7 million for the three and six months ended June 30, 2024.

Cost of goods sold was approximately $1.2 million and $2.4 million for the three and six months ended June 30, 2025, compared to $17,000 for the three and six months ended June 30, 2024, which consisted of costs to manufacture and distribute RYTELO.

Research and development expenses for the three and six months ended June 30, 2025, were $21.7 million and $36.8 million, compared to $30.8 million and $60.2 million for the same periods in 2024. The decrease in research and development expenses for the three and six months ended June 30, 2025, compared to the same periods in 2024, was primarily due to decreased clinical trial costs associated with a decrease of activity in our Phase 3 IMerge LR-MDS study after FDA approval of RYTELO in 2024, as well as manufacturing and quality costs that were capitalized in the current period now that RYTELO is approved, compared to being expensed in the prior period.

Selling, general and administrative expenses for the three and six months ended June 30, 2025, were $38.6 million and $78.6 million, compared to $39.4 million and $66.5 million for the same periods in 2024. The decrease in selling, general and administrative expenses for the three months ended June 30, 2025, compared to the same period in 2024, is attributed to initial RYTELO launch costs in 2024. The increase in the six months ended June 30, 2025 is primarily due to higher personnel-related expenses from increased headcount to support the commercialization of RYTELO.

2025 Financial Guidance

For fiscal year 2025, the Company maintains its previously announced expectations of total operating expenses to be in the range of approximately $270 million to $285 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

Based on current operating plans and assumptions, the Company believes that existing cash, cash equivalents, and marketable securities, together with anticipated net revenues from U.S. sales of RYTELO, will be sufficient to fund projected operating requirements for the foreseeable future.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Wednesday, August 6, 2025, to discuss business updates and second quarter 2025 financial results.

A live webcast of the conference call and accompanying presentation will be available on the "Investors & Media" page of the Company’s website at www.geron.com. A replay of the webcast will be archived and available on the Company’s website for 30 days.

About RYTELO (imetelstat)

RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with LR-MDS with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.

Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide, available at View Source

About IMpactMF Phase 3

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with intermediate-2 or high-risk myelofibrosis (MF) who are relapsed after or refractory to prior treatment with a JAK inhibitor, also referred to as relapsed/refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete remission, partial remission, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes. IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit clinicaltrials.gov/study/NCT04576156.

On August 6, 2025 Galapagos NV (Euronext & NASDAQ: GLPG) reported that the United States Food and Drug Administration (FDA) has granted RMAT designation to GLPG5101, a second generation anti-CD19/4-1BB CAR-T product candidate for the treatment of relapsed/refractory mantle cell lymphoma (R/R MCL) (Press release, Galapagos, AUG 6, 2025, View Source [SID1234654864]).

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The RMAT designation was established under the U.S. 21st Century Cures Act to accelerate development and review of promising cell and gene therapies for serious or life-threatening conditions. To qualify for RMAT designation, GLPG5101 demonstrated preliminary clinical evidence suggesting it has the potential to treat, modify, reverse, or cure a serious or life-threatening disease.

Clinical data derived from the ongoing ATALANTA-1 study with GLPG5101 in patients with R/R B-cell Non-Hodgkin Lymphoma (B-NHL), including a subset of patients with MCL, supported the RMAT designation. These data1 demonstrated both high objective and high complete response rates, with a manageable safety profile, including low rates of high-grade cytokine release syndrome (CRS), immune effector cell associated neurotoxicity syndrome (ICANS) and low dropout rates.

"This designation reflects the promising clinical activity and safety profile observed in our ongoing Phase 1/2 study and supports our commitment to delivering an effective and timely treatment option to patients in need," said Omotayo Fasan, M.D., Clinical Development Program Head at Galapagos. "With RMAT status allowing for closer collaboration with the FDA, this will enable additional opportunities for accelerated development and assessment timelines."

Benefits of RMAT designation include increased FDA guidance and more frequent interactions during development, eligibility for accelerated approval based on surrogate or intermediate endpoints, all Fast Track and Breakthrough Therapy advantages such as priority review and rolling submissions, and early discussions of potential study endpoints.

Galapagos intends to report updated data from the ATALANTA-1 study at a future medical conference.

About GLPG5101 and ATALANTA-1

GLPG5101 is a second generation anti-CD19/4-1BB CAR-T product candidate, administered as a single fixed intravenous dose. The safety, efficacy and feasibility of decentralized manufactured GLPG5101 are currently being evaluated in the ATALANTA-1 Phase 1/2 study in eight hematological malignancies with high unmet need. The primary objective of the Phase 1 part of the study is to evaluate safety and to determine the recommended dose for the Phase 2 part of the study. Secondary objectives include assessment of efficacy and feasibility of decentralized manufacturing of GLPG5101. The dose levels that were evaluated in Phase 1 are 50×106 (DL1), 110×106 (DL2) and 250×106 (DL3) CAR+ viable T-cells. The primary objective of the Phase 2 part of the study is to evaluate the Objective Response Rate (ORR) while the secondary objectives include Complete Response Rate (CRR), duration of response, progression free survival, overall survival, safety, pharmacokinetic profile, and the feasibility of decentralized manufacturing. Each enrolled patient will be followed for 24 months. The ATALANTA-1 study is currently enrolling patients in the U.S. and Europe.

About relapsed/refractory mantle cell lymphoma

Mantle cell lymphoma is a rare and aggressive subtype of non-Hodgkin lymphoma originating from B cells. Patients with relapsed or refractory disease have progressed after standard therapies and face limited treatment options and reduced survival rates.

On August 6, 2025 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that the Company generated revenue of $811 million for the second quarter ended June 30, 2025, compared to $699 million for the same period of 2024 (Press release, Exact Sciences, AUG 6, 2025, View Source [SID1234654863]).

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"The Exact Sciences team continues to build momentum, advancing our mission through earlier detection," said Kevin Conroy, chairman and CEO. "In the second quarter, we delivered answers to more patients than ever driven by strong momentum behind the successful launch of Cologuard Plus, powerful commercial execution, and exceptional customer satisfaction. This progress strengthens our platform and enables us to deliver better outcomes for patients."

Second quarter 2025 financial results

For the three-month period ended June 30, 2025, as compared to the same period of 2024 (where applicable):

Total revenue was $811 million, an increase of 16% on a reported and core revenue basis
Screening revenue was $628 million, an increase of 18%
Precision Oncology revenue was $183 million, an increase of 9%
Gross margin was 69%, and adjusted gross margin was 72%
Net loss was $1 million, or $0.01 per share, an improvement of $15 million and $0.08 per share, respectively
Adjusted EBITDA was $138 million, an increase of $28 million or 26%, and adjusted EBITDA margin was 17%, an increase of 130 basis points
Operating cash flow was $89 million and free cash flow was $47 million,
Cash, cash equivalents, and marketable securities were $858 million at the end of the quarter
Screening primarily includes laboratory service revenue from Cologuard tests and PreventionGenetics. Precision Oncology includes laboratory service revenue from global Oncotype DX and therapy selection tests.

Platform and pipeline advancements

In April 2025, Exact Sciences launched the Oncodetect test, its molecular residual disease and recurrence monitoring test. Oncodetect identifies residual disease up to two years earlier than imaging, the current standard of care. The Company recently received Medicare coverage through the Centers for Medicare & Medicaid Services’ (CMS) Molecular Diagnostic Services Program (MolDX) for serial use in patients with stage II, III and resectable stage IV colorectal cancer (CRC) in the adjuvant and recurrence monitoring settings over a five-year period.

In September 2025, Exact Sciences plans to launch its Cancerguard multi-cancer screening test as a laboratory-developed test. The Company plans to bring the test to patients through its large commercial organization and unique ExactNexus technology platform.

In August 2025, Exact Sciences acquired exclusive rights to the current and future versions of Freenome’s blood-based colorectal cancer screening tests as well as the underlying technology for colorectal cancer, subject to customary regulatory approvals. Additionally, the Company announced initial study results from the pivotal BLUE-C study for an internal version of its blood-based colorectal cancer screening test.

2025 outlook

The Company has updated its full-year 2025 revenue and adjusted EBITDA guidance:

Prior guidance

August 6 update

Change at midpoint

Y/Y growth rate

Total revenue

$3.070 – $3.120 billion

$3.130 – $3.170 billion

$55.0 million

14%

Screening

$2.390 – $2.425 billion

$2.440 – $2.470 billion

$47.5 million

17%

Precision Oncology

$680 – $695 million

$690 – $700 million

$7.5 million

6%

Adjusted EBITDA

$425 – $455 million

$455 – $475 million

$25.0 million

44%

Second-quarter 2025 conference call & webcast

Company management will host a conference call and webcast on Wednesday, August 6, 2025, at 5 p.m. ET to discuss second-quarter 2025 results. The webcast will be available at exactsciences.com. Domestic callers should dial 888-330-2384 and international callers should dial +1-240-789-2701. The access code for both domestic and international callers is 4437608. A replay of the webcast will be available at exactsciences.com. The webcast, conference call, and replay are open to all interested parties.