On August 13, 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 cancers and viruses, reported its financial results for the second quarter ended June 30, 2025 (Press release, Moleculin, AUG 13, 2025, View Source [SID1234655205]).

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"The second quarter of 2025 marked a period of momentum for Moleculin. We continued to make meaningful progress in our MIRACLE trial, including a significant expansion into a number of countries, positioning ourselves to achieve our enrollment targets and, importantly, reach an expected pivotal data readout before the end of this year. In addition to progress in our AML program, we reported compelling topline data in our Phase 1B/2 soft tissue sarcoma lung metastases (STS lung mets) trial and continue to build a growing body of preclinical and clinical data showcasing the depth and breadth of the potential Annamycin has to treat a range of cancer indications," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "I am proud of the dedication our team continues to show, and we look forward to building on this progress in the second half of the year and beyond."

Recent Highlights

Annamycin for the Treatment of AML

Expanded its Phase 2B/3 MIRACLE trial with secured approval from the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia;
Four active MIRACLE sites now screening subjects in Ukraine, Georgia, Spain and the US;
Expect to add more than 20 additional sites in the European Union (EU) and the US by the end of Q3 2025; and
Received positive FDA feedback on pediatric study plan for Annamycin in children with R/R AML, setting the start of that trial in 2027.
Other Annamycin

Announced the presentation of encouraging preclinical data for Annamycin, which demonstrated significant efficacy against various primary and metastatic liver cancers;
Bolstered Annamycin intellectual property portfolio with notice of intent to grant a new European patent;
Engaged industry veteran Adriano Treve to explore Annamycin related strategic partnerships; and
Reported positive topline efficacy results from U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of STS lung metastases (MB-107).
Clinical Development Update

Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

The Company is currently evaluating 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") in 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 global, including sites in the US, Europe and the Middle East.

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the Phase 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, and utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) before the end of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

As previously announced regarding the EU, the clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved CTA.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Expected Milestones for Annamycin AML Development Program

Q3 2025 – Site and recruitment updates for MIRACLE trial
Q3 2025 – First patients treated in the US and the EU
Q4 2025 – Announcement of 45th subject being recruited and data readout (n=45) unblinded efficacy/safety review
Q1 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update
1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2027 – Prepare for the launch of an AML pediatric trial
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 – 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
2028 – Primary efficacy data for 2nd line subjects
Soft Tissue Sarcoma (STS) Lung Metastases

As previously announced, the Company reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of STS lung mets (MB-107). The final topline results of MB-107 demonstrated median overall survival (OS) of 13.5 months for subjects as median 7th line therapy (n=36). This compares to OS of 8-12 months for standard of care treatments and 13.4 months for experimental treatments for advanced STS as 2nd line.1

For more information about the MB-107 trial visit clinicaltrials.gov and reference identifier NCT04887298.

Expected Milestones for Annamycin STS Lung Mets Development Program

Q4 2025 – Identify next phase of development / pivotal IIT (investigator-initiated-trial) program
Annamycin, also known by its non-proprietary name of naxtarubicin, 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 EMA.

WP1066 & Brain Tumors

With regard to the Company’s WP1066 oral formula, the Company has an externally funded Phase 1B/2 in combination with radiation treating glioblastoma (GBM), a form of brain cancer, at Northwestern University (Northwestern) that is actively recruiting. This is an investigator-initiated trial where Moleculin’s main cost is supplying drug product. To date Northwestern has recruited 7 subjects, of which 5 have completed treatment and are undergoing follow-up while 2 continue with treatment. No data has been released. Also, the Company has signed an agreement with Emory University enabling Emory to study various WP1066 IV formulations in preclinical studies with the goal of selecting the best molecule to move into a clinical setting towards, most likely, brain cancers such as GBM. Study drug was delivered in April 2025 to Emory with results from such studies expected in the second half of 2025.

Summary of Financial Results for the Second Quarter 2025

Research and development (R&D) expense was $3.6 million and $4.1 million for the three months ended June 30, 2025 and 2024, respectively. The decrease of $0.5 million is mainly related to a reduction in the clinical trials activity.

General and administrative expense was $2.1 million each for the three months ended June 30, 2025 and 2024.

As of June 30, 2025, the Company had cash and cash equivalents of $7.6 million and believes that the cash on hand is sufficient to fund planned operations into the fourth quarter of 2025.

On August 13, 2025 Guided Therapeutics, Inc. (OTC:QB GTHP), the maker of the LuViva Advanced Cervical Scan, a rapid and painless testing platform for cervical cancer detection based on its patented biophotonic technology, reported that it had enrolled enough patients to begin closing out the study and starting data analysis (Press release, Guided Therapeutics, AUG 13, 2025, View Source [SID1234655224]). Data analysis will consist of completing the ongoing external review of all biopsy samples, reviewing and entering data from the study case report forms and performing the statistical analysis pursuant to the study protocol. All four participating clinics have reached their minimum specified quotas resulting in a total of approximately 430 patients enrolled. Part of the pathology analysis is intended to determine the number of patients with and without disease to ensure a representative mix of disease types. Once this part of the analysis has been completed, all clinical sites will be closed out from enrolling additional patients and the clinical report will be filed with the FDA, expected later this year. Additionally, there have been no adverse events linked to the use of the LuViva device, further supporting FDA’s designation of LuViva as a non-significant risk device.

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"We want to thank all of physicians and staff at the clinical sites who made this study possible," said Mark Faupel, CEO of Guided Therapeutics. "As we move from the testing phase to the analysis phase of the study, we have met the Company’s primary 2025 objective. We are optimistic that we have conducted a successful study and look forward to the results of the statistical analyses."

On August 13, 2025 LAVA Therapeutics N.V. (NASDAQ: LVTX, "LAVA," or the "Company"), a clinical-stage immuno-oncology company historically focused on its proprietary Gammabody bispecific gamma delta T cell engagers, reported financial results for the second quarter ended June 30, 2025 and provided a corporate update (Press release, Lava Therapeutics, AUG 13, 2025, View Source [SID1234655305]).

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"We are pleased to announce that LAVA has recently entered into a definitive agreement to be acquired by XOMA Royalty Corporation," said Steve Hurly, Chief Executive Officer of LAVA. "This deal is the outcome of a comprehensive and diligent strategic review process by management and our Board of Directors, conducted under the guidance of our legal and financial advisors, with the objective of maximizing value for our shareholders while supporting the sustained success of LAVA’s business. Our Board of Directors has unanimously determined that the deal is in the best interests of all of our shareholders and has approved the proposed acquisition."

Entry into Share Purchase Agreement; Tender Offer

On August 4, 2025, the Company announced that it has entered a definitive share purchase agreement (the "Purchase Agreement" and the transactions set forth in the Purchase Agreement, the "Transactions") with XOMA Royalty Corporation ("XOMA"), whereby XOMA will acquire all of the issued and outstanding common shares of the Company through a cash tender offer for (i) between $1.16 and $1.24 per share in cash, consisting of (A) $1.16 per share, plus (B) an additional amount of up to $0.08 per share, plus (ii) a non-transferable contingent value right per share representing the right to receive potential contingent cash payments following the closing related to the Company’s two partnered assets as well as its unpartnered programs. Pursuant and subject to the terms of the Purchase Agreement, XOMA will commence a tender offer by August 15, 2025 to acquire all of the Company’s outstanding common shares. The closing of the Transactions is subject to customary closing conditions and is expected to close in the fourth quarter of 2025.

Discontinued LAVA-1266 Program

On August 4, 2025, the Company announced its plans to discontinue its Phase 1 clinical trial of LAVA-1266 for acute myeloid leukemia and myelodysplastic syndrome, and initiate the wind-down of the LAVA-1266 program.

Updates Regarding Partnered Programs

Johnson & Johnson (J&J) Partnered Program (JNJ-89853413) – Phase 1 Trial (NCT06618001)

Designed to target CD33 and gamma delta T cells with a bispecific gamma delta T cell engager

· Key Indications: relapsed or refractory (R/R) acute myeloid leukemia (AML) or R/R higher-risk type of myelodysplastic neoplasms (MDS)

· Current Status: J&J is enrolling patients in a Phase 1, open label, multi-center trial, currently underway in Canada and Spain. The trial includes a dose escalation and dose expansion segment to evaluate JNJ-89853413 in approximately 100 adults with R/R AML or R/R higher risk type of MDS

Pfizer Partnered Program (PF08046052) – Phase 1 Trial (NCT05983133)

Potential first-in-class epidermal growth factor receptor (EGFR) and bispecific gamma delta T cell receptor-targeted therapy

· Key Indications: advanced solid tumors

· Current Status: Pfizer is enrolling patients in a Phase 1 open label, multi-center trial, currently underway in the US and UK. The trial is intended to evaluate PF08046052 in approximately 290 subjects

Second Quarter 2025 Financial Results

· As of June 30, 2025, LAVA had cash, cash equivalents, and short-term investments of $56.2 million, compared to cash, cash equivalents, and short-term investments of $76.6 million as of December 31, 2024.

· Revenue from contracts with customers was zero for the quarters ended June 30, 2025 and 2024, respectively, and zero and $7.0 million for the six months ended June 30, 2025 and 2024, respectively. Revenue of $7.0 million received in the six months ended June 30, 2024 was comprised of a $7.0 million payment from Pfizer related to the achievement of a clinical milestone.

· Research and development expenses were $4.7 million and $6.0 million for the quarters ended June 30, 2025 and 2024, respectively, and $8.9 million and $11.6 million for the six months ended June 30, 2025 and 2024, respectively. The decrease in both periods was primarily due to a reduction in headcount related to restructuring activities and resulting decrease in research and development activity, with lower preclinical and clinical expenses due to the discontinuation of the LAVA-1207 program and a reduction in the estimated remaining clinical trial activities, partially offset by activities for LAVA-1266 occurring in the quarter ended June 30, 2025.

· General and administrative expenses were $2.6 million and $3.4 million for each of the quarters ended June 30, 2025 and 2024, respectively and $6.0 million and $6.8 million for the six months ended June 30, 2025 and 2024, respectively. The decrease reflected in both periods was due to lower headcount and an overall streamlining of administrative and operating costs related to the Company’s restructuring activities, partially offset by increased professional and consultant fees related to the Company’s transition to US GAAP reporting as well as increased severance payments related to the Company’s restructuring action in February 2025.

· Other income (expense), net was a $1.3 million other expense, net and $1.2 million other income, net for the quarters ended June 30, 2025 and 2024, respectively, with other income, net of $3.1 million and $2.7 million for the six months ended June 30, 2025 and 2024, respectively. For the three months ended June 30, 2025 and 2024, the decrease is primarily due to foreign exchange loss, due to fluctuations in the US dollar currency rate compared to the Euro, as well as lower interest rates for cash held in money market accounts, partially offset by lower interest expense incurred as the Company’s outstanding innovation credit from Rijksdienst voor Ondernemend Nederland (RVO) was forgiven in March 2025. Forgiveness of the RVO credit balance also increased other income, net for the six months ended June 30, 2025.

· Net loss was $8.6 million and $8.3 million for the quarters ended June 30, 2025 and 2024, respectively, or $0.32 and $0.31 net loss per share, respectively, and $12.1 million and $8.9 million, or $0.45 or $0.33 net loss per share, for the six months ended June 30, 2025 and 2024, respectively.

On August 13, 2025 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immuno-oncology technologies addressing difficult to treat cancers, reported its financial results for the second quarter 2025 (Press release, Xenetic Biosciences, AUG 13, 2025, View Source [SID1234655164]).

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Recent Highlights

Expanded its collaboration with The Scripps Research Institute ("TSRI") to advance the development of the Company’s development program evaluating the combination of systemic DNase I and CAR T-cell therapies;

Announced advancements from its collaboration partner, PeriNess Ltd. ("PeriNess") including:

Entered into a Clinical Study Agreement to support an exploratory clinical study of DNase I in combination with anti-CD19 CAR T cells in patients with large B cell lymphoma;

Commenced patient dosing in an exploratory clinical study of systemic DNase I in combination with FOLFIRINOX for the first line treatment of unresectable, locally advanced or metastatic pancreatic cancer at Bnei Zion Medical Center; and

Continued pursuit of other strategic collaborations to advance the Company’s technology.

"We continue to set a strong foundation that we believe positions us for success as we advance our systemic DNase I in combination with immunotherapy, chemotherapy, and radiotherapy across various oncology indications where there remains significant unmet need. We continue to work with our partners and believe the data and information will be invaluable as we look to realize the full potential of our DNase platform technology. Looking ahead, we remain focused on building momentum across all fronts and driving development toward an IND and Phase 1 clinical trial," commented James Parslow, Interim Chief Executive Officer and Chief Financial Officer of Xenetic.

Xenetic continues to advance its DNase-based technology towards Phase 1 clinical development for the treatment of pancreatic carcinoma and other locally advanced or metastatic solid tumors. Preclinical proof-of-concept studies combining DNase I with chemotherapy, immunotherapies, and CAR-T therapy in hematological and solid tumor and metastatic cancer models have been completed. Building on proof-of-concept success, the program has now advanced to mechanism-of-action and translational studies in preparation for a Phase 1 clinical trial.

Additionally, as previously announced in December 2024, Xenetic entered into a Clinical Trial Services Agreement with PeriNess, under which PeriNess will lead in the regulatory approval, operational execution and management of potential exploratory, investigator initiated studies of recombinant DNase as an adjunctive treatment in patients with pancreatic carcinoma and other locally advanced or metastatic solid tumors receiving chemotherapy and immunotherapy in Israeli medical centers.

Summary of Financial Results for Second Quarter 2025

Net loss for the quarter ended June 30, 2025 was approximately $0.7 million. Research & development expenses for the three months ended June 30, 2025 decreased by approximately $277,000, or 29.7%, to approximately $0.7 million from $0.9 million in the comparable quarter in 2024. General and administrative expenses for the three months ended June 30, 2025 decreased by approximately $472,000, or 41.8%, to approximately $0.7 million from approximately $1.1 million in the comparable quarter in 2024. These decreases were primarily due to certain severance and benefits expensed in connection with separation agreements entered into during the second quarter of 2024 with the Company’s former Chief Executive Officer and Chief Scientific Officer.

The Company ended the quarter with approximately $4.8 million in cash.

On August 13, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ), (TSX: BCT) (" BriaCell " or the " Company "), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, reported its acceptance into Memorial Sloan Kettering Cancer Center’s (MSK’s) Therapeutics Accelerator 2025 Cohort program (Press release, BriaCell Therapeutics, AUG 13, 2025, View Source [SID1234655429]). This project aims to accelerate the clinical development of Bria-OTS+, BriaCell’s next generation personalized off-the-shelf immunotherapy, for multiple cancer indications including metastatic breast cancer, prostate cancer, and other cancers.

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As one of the world’s foremost cancer research and treatment institutions, MSK has more than 135 years of leadership in patient care, education and discovery. Through the MSK Therapeutics Accelerator Cohort program, BriaCell will explore access to MSK’s expertise and institutional resources, including GMP manufacturing services, Investigational New Drug (IND) Application preparation and submission and regulatory strategy support to expedite Bria-OTS+ development.

"We are honored to work with the scientific and clinical experts at MSK Therapeutics Accelerator," stated Dr. William V. Williams, BriaCell’s President and CEO. "MSK’s deep expertise in cancer therapy development and state-of-the-art resources make it an ideal partner to accelerate our personalized off-the-shelf immunotherapy platform."

"We are excited to welcome BriaCell into the cohort program," stated Eileen Flowers, PhD, Director, Technology Development & Licensing, in the Office of Entrepreneurship and Commercialization at MSK.

"We believe Bria-OTS+ has the potential to transform cancer care, offering meaningful advances in efficacy and safety for thousands of patients," stated Dr. Miguel Lopez-Lago, BriaCell’s Chief Scientific Officer. "Our participation in this cohort program is yet another step towards achieving that vision."