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TransCode Therapeutics Successfully Completes Initial Dosing of Patients in Cohort 4 of Phase 1a Clinical Trial; No Dose Limiting Toxicities Reported

On May 8, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the third patient in Cohort 4 of its Phase 1a clinical trial has received their initial dose of TTX-MC138 (Press release, TransCode Therapeutics, MAY 8, 2025, View Source [SID1234652781]). All cohorts have enrolled at least three patients who have been dosed with TTX-MC138 at least once. The Safety Review Committee monitoring the clinical trial unanimously approved opening the fourth cohort based on its review of available safety and pharmacokinetic (PK) data. Additionally, the Safety Review Committee approved expanded enrollment in Cohort 3 to obtain additional safety data. To date, 15 patients have received at least one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8 mg/kg to 4.8 mg/kg. Three patients have been treated in the expanded enrollment.

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Ten patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each treatment cycle every 28 days, and may remain on study absent any significant safety observations or disease progression. Two patients who have remained on study the longest have received so far seven doses of TTX-MC138 over approximately seven months and have demonstrated stable disease. The 10 patients currently on study have shown no disease progression. Further, no significant safety or dose limiting toxicities have been reported in any of the trial’s 15 patients. Analysis of PK activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, preliminary PK and pharmacodynamic (PD) data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates miR-10b target engagement at 24 hours post-infusion.

The observed tolerability profile and the available PK/PD results thus far support advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial.

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

About the Trial

TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types and at a certain dose level selected based on preliminary results from the dose-escalation phase.

Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

Whitehawk Reports First Quarter 2025 Financial Results and Recent Highlights

On May 8, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported financial results for the first quarter ended March 31, 2025, and provided recent corporate progress (Press release, Whitehawk Therapeutics, MAY 8, 2025, View Source [SID1234652780]).

"By uniting novel approaches to tumor targeting with next wave technologies, our goal at Whitehawk Therapeutics is to efficiently deliver improved ADC therapies for patients with difficult-to-treat cancers. In the first quarter of 2025, we made meaningful progress in advancing our portfolio toward the clinic and remain on track to bring all three assets to IND by mid-2026, with the first IND filed in Q4 this year," said Dave Lennon, President and CEO of Whitehawk Therapeutics. "Our assets target PTK7, MUC16 and SEZ6 – which we believe have the potential for broad clinical impact comparable to well-established targets like HER2 and TROP2, but with less competition and greater opportunity for differentiation. Coupled with our TOPO1-based leading next wave platform, we are confident in the strength of our strategy and remain committed to advancing with urgency and purpose."

Recent Operational Highlights:

Relaunched as Whitehawk Therapeutics marking evolution into ADC company. Launched with a three-asset portfolio consisting of clinically validated tumor targets that are upregulated in high-potential cancer indications, including lung and ovarian. These assets are engineered to produce minimal off-target toxicity, with a higher therapeutic index and greater stability than first-generation predecessors.
Completed strategic transactions. Closed $100 million PIPE financing and the divestiture of Aadi Subsidiary, Inc. ("Aadi Sub") to Kaken Pharmaceuticals ("Kaken") for $102.4 million, including specified purchase price adjustments. Kaken assumed ownership of Aadi Sub on March 25, 2025, including the Aadi name, trademark and the FYARRO business.
First Quarter 2025 Financial Results:

Cash, cash equivalents and short-term investments as of March 31, 2025, were $231.1 million as compared to $47.2 million as of December 31, 2024.
After completion of transaction-related payments, including the payment of the upfront fees under the Wuxi ADC agreement, we expect to have cash and cash equivalents of approximately $185 million, which we anticipate will fund operations into 2028 based on current plans.
Total revenue for the quarter ended March 31, 2025, was $7.1 million, resulting from sales of FYARRO through March 25, 2025, the closing date of the divestiture to Kaken.
Net income for the three months ended March 31, 2025, was $73.0 million, including a gain of $87.4 million on the sale of Aadi Sub, as compared to a net loss of $18.3 million for the three months ended March 31, 2024. Excluding the gain on the sale of Aadi Sub, net loss for the first quarter of 2025 was $14.4 million.

Aptevo Therapeutics Provides Program Update for Bispecific APVO711, a PD-L1 X CD40 that Combines a Checkpoint Inhibitor and Immune Activation

On May 8, 2025 Aptevo Therapeutics Inc. (Nasdaq:APVO), a clinical-stage biotechnology company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported a program update for APVO711, a dual mechanism bispecific utilizing the PD-L1 arm to block the PD-1/PD-L1 pathway and the CD40 arm to enhance T cell priming through activation of the stimulatory receptor CD40 on antigen presenting cells (Press release, Aptevo Therapeutics, MAY 8, 2025, View Source [SID1234652779]). This update highlights the depth and scientific versatility of Aptevo’s existing pipeline and reinforces the Company’s ability to advance differentiated candidates for solid tumors with significant unmet need. Currently in preclinical development, APVO711 reflects the potential of Aptevo’s platform-based strategy to generate bispecific therapeutics with novel mechanisms and broad clinical relevance.

"With APVO711, we’re advancing an innovative dual-mechanism approach to immunotherapy-one that not only boosts the immune system through PD-L1 blockade but also facilitates T-cell priming via CD40 activation. This is important in the development of new therapeutics for cancers that have proven resistant to standard checkpoint therapies. We believe APVO711 has the potential to unlock deeper, more sustained responses, and ultimately expand the reach of immunotherapy to patients who need it most," said Marvin White, President and CEO of Aptevo.

About APVO711

Novel Mechanism: APVO711 is a bispecific antibody targeting PD-L1 × CD40-combining checkpoint inhibition with immune activation in a single molecule.

Designed for Potency: Unlike traditional checkpoint inhibitors, APVO711 simultaneously blocks immune suppression and primes antigen presenting cells to trigger robust T-cell responses.

Targeting the Unmet Need: Developed to address a wide variety of tumor types and augment the anti-tumor responses achieved with PD-1/PD-L1 blockade alone.

Pipeline Synergy: Represents a key part of Aptevo’s novel immuno-oncology platform, with strong potential for combination strategies across solid tumors.

Momentum Building: Preclinical studies demonstrate dual anti-cancer functionality.

"APVO711 represents a novel immunotherapy approach – by targeting both PD-L1 and CD40, this bispecific candidate is designed to block tumor-driven immune suppression while simultaneously priming T cells for a stronger, more durable anti-tumor response. In a landscape where many patients fail to respond to checkpoint inhibitors alone, APVO711 offers a rational strategy to expand the benefits of immunotherapy to a broader range of cancers and patients," said Michelle H. Nelson, Ph.D., Director of Immunobiology at Aptevo Therapeutics.

OmniAb Reports First Quarter 2025 Financial Results and Business Highlights

On May 7, 2025 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three months ended March 31, 2025, and provided operating and partner program updates (Press release, OmniAb, MAY 8, 2025, View Source [SID1234652778]).

"We have started the year with robust deal flow, including both platform and asset-based deals. Our business remains strong as our diversified pipeline of partner programs is progressing with recent and expected new clinical entrants and data readouts," said Matt Foehr, Chief Executive Officer of OmniAb. "Today we announced the xPloration Partner Access Program for OmniAb partners, enhancing the scalability of our technology platforms and creating new business opportunities that we believe will be accretive to both earnings and cash flow in short- and long-term. This initiative furthers our mission to push the frontiers of discovery technologies along with our focus on creating value for our partners and our stakeholders. As we look ahead, our 2025 outlook remains on track with our commitment to running an efficient and leverageable business."

First Quarter 2025 Financial Results

Revenue for the first quarter of 2025 was $4.2 million, compared with $3.8 million for the same period in 2024, with the increase primarily due to the recognition of a $1.0 million Phase 1 milestone payment and higher license fees, partially offset by lower service revenue.

Research and development expense was $12.6 million for the first quarter of 2025, compared with $14.6 million for the same period in 2024, with the decrease primarily due to lower share-based compensation expense and lower external expenses associated with our small-molecule ion channel programs and technology development. General and administrative expense was $7.9 million for the first quarter of 2025, compared with $8.3 million for the same period in 2024, with the decrease primarily due to lower legal fees and share-based compensation expense.

Net loss for the first quarter of 2025 was $18.2 million, or $0.17 per share, compared with a net loss of $19.0 million, or $0.19 per share, for the same period in 2024.

As of March 31, 2025, OmniAb had cash, cash equivalents and short-term investments of $43.6 million.

2025 Financial Guidance

OmniAb affirms guidance for 2025 revenue to be in the range of $20 million to $25 million, and revises operating expense guidance to be in the range of $85 million to $90 million from the previous range of $90 million to $95 million. In addition, OmniAb continues to expect 2025 cash use to be lower than cash use in 2024. Cash use in 2024 was $38.9 million, excluding the 2024 ATM issuance. The 2025 full year effective tax rate is expected to be approximately 0%.

First Quarter 2025 and Recent Business Highlights

During the first quarter of 2025, OmniAb entered into three new platform license agreements including the Wyss Institute at Harvard University, Takis Biotech S.r.l. and Orion Corporation.

OmniAb entered into a research collaboration and license agreement with Orion Corporation to discover and generate an antibody-based compound for a specific ion-channel target. Under the terms of the agreement, OmniAb will receive an upfront payment of $250,000 and is eligible to receive service payments. OmniAb is also eligible to receive development, regulatory and commercialization milestone payments totaling to over $55 million. OmniAb will receive low- to mid-single digit tiered royalties on net sales, should the program reach commercialization.

As of March 31, 2025, the Company had 95 active partners and 378 active programs, including 33 OmniAb-derived programs in clinical development or being commercialized.

Post-quarter close, OmniAb entered into an asset-based sale with Angelini Pharma for a small molecule Kv7.2 program. OmniAb will receive an upfront payment of $3 million, and potential milestones of over $170 million and royalties.

In addition, OmniAb launched the offering of xPloration to existing partners through a Partner Access Program. xPloration is a high-throughput single B-cell screening instrument that leverages machine learning and artificial intelligence to address challenges in primary B-cell screening with traditional methods, such as limited antibody diversity and lengthy processes. We believe it offers a competitive edge over current market offerings for B-cell screening with unmatched screening throughput, superior hit recovery, exceptional ease-of-use and reliability. OmniAb will showcase xPloration at the 21st Annual PEGS Boston Summit, taking place May 12-16 at the Omni Boston Hotel at the Seaport.

First quarter 2025 and recent partner and business highlights include the following:

IMVT-1402

Immunovant announced that potentially registrational trials for IMVT-1402 are currently enrolling patients in four indications: myasthenia gravis (MG), chronic inflammatory demyelinating polyneuropathy (CIDP), Graves’ disease (GD) and difficult-to-treat rheumatoid arthritis.
A fifth potentially registrational trial for Sjogren’s disease is planned to begin in the summer of 2025. Additionally, a proof-of-concept study has been initiated in a sixth indication, cutaneous lupus erythematosus.
Batoclimab

Immunovant announced positive study results for batoclimab in MG and CIDP. The pivotal study in MG met its primary endpoint, showing a change from baseline in the Myasthenia Gravis Activities of Daily Living score in the acetylcholine receptor antibody positive population at 12 weeks. The 680mg dose arm showed a 5.6-point improvement with a 74% mean immunoglobulin G (IgG) reduction, while the 340mg dose arm showed a 4.7-point improvement with a 64% mean IgG reduction.
Initial CIDP results from Period 1, following standard-of-care washout, demonstrated a mean improvement in the adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) disability score of 1.8 across study arms. An 84% responder rate (with response defined as an aINCAT improvement ≥1) was observed among all patients whose IgG was reduced by ≥70%.
In both batoclimab studies, deeper IgG reductions correlated with better clinical outcomes across a range of assessments and timepoints.
Immunovant expects to announce additional data for batoclimab in GD, including six-month remission data, this summer. Additionally, top-line results for batoclimab from potentially registrational Phase 3 trials in thyroid eye disease are expected in the second half of 2025.
Sugemalimab

CStone Pharmaceuticals announced the submission of a Type II variation application to the European Medicines Agency (EMA) for sugemalimab, seeking approval for the treatment of unresectable stage III non-small cell lung cancer (NSCLC) in patients who have not progressed following platinum-based chemoradiotherapy. This is CStone’s second regulatory submission for sugemalimab to the EMA, following its 2024 approval for metastatic NSCLC.
TEV-53408

Teva Pharmaceuticals initiated a Phase 2 trial of TEV-53408 in adults with celiac disease. The primary efficacy objective is to assess the ability of TEV-53408 to attenuate gluten-induced enteropathy. Additional objectives include the safety assessment of TEV-53408.
RNDO- 564

Rondo Therapeutics published preclinical data for RNDO-564, a CD28 x Nectin-4 bispecific antibody for bladder cancer, in theJournal of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper).In vitrostudies demonstrated that RNDO-564 enhanced T-cell activation and cytotoxicity against Nectin-4 positive tumor cells. The antibody demonstrated significant tumor regression in tumor-bearing mouse models, both alone and with an immune checkpoint inhibitor. Favorable pharmacokinetic and tolerability profiles were observed in non-human primates.
OmniAb recently appointed Philip J. Gotwals, Ph.D., and Steve Crouse to its Board of Directors. Dr. Gotwals, with 30 years of biopharmaceutical experience in R&D, business development, product development, and therapeutic area strategy, along with Mr. Crouse, who brings over 20 years of expertise in life sciences sales and marketing, product development, business development, and general management, will help advance the Company’s strategic initiatives.

OmniAb reported that partners presented data on nine OmniAb derived molecules at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, held April 25-30. These presentations showcased clinical trial designs, as well as data across various preclinical and clinical studies.

The Company also expects that multiple partners will be presenting data from programs developed with OmniAb technology at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 30 – June 3, 2025.

Conference Call and Webcast

OmniAb management will host a conference call with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 549 8228 using the conference ID 96760. Slides, as well as the live and replay webcast of the call, are available at View Source

Imfinzi regimen demonstrated statistically significant and clinically meaningful improvement in disease-free survival for high-risk non-muscle-invasive bladder cancer in POTOMAC Phase III trial

On May 8, 2025 Astrazeneca reported positive high-level results from the POTOMAC Phase III trial showed one year of treatment with Imfinzi (durvalumab) plus standard-of-care BCG induction and maintenance therapy demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) for patients with high-risk non-muscle-invasive bladder cancer (NMIBC) compared to BCG induction and maintenance therapy alone (Press release, AstraZeneca, MAY 8, 2025, View Source [SID1234652777]).

The trial was not statistically powered to formally test overall survival (OS) however a descriptive analysis demonstrated no detriment.

More than 70% of bladder cancer patients are diagnosed with NMIBC, an early-stage cancer where the tumour is in the tissue that lines the inner surface of the bladder but has not invaded the muscle wall.1-2 About half of patients with NMIBC are classified as high-risk for disease progression or recurrence because of certain characteristics of their cancer, such as tumour grade, stage and specific tumour features.3

Maria De Santis, MD, Head of the Interdisciplinary Uro-Oncology Section at Charité Universitätsmedizin Berlin, Germany, and a principal investigator in the POTOMAC trial, said: "These exciting data show that adding one year of durvalumab to the current standard treatment significantly extends the time patients live without high-risk disease recurrence or progression. While most patients with non-muscle invasive bladder cancer are treated with curative intent, 80 per cent see their disease return and almost half may require life-altering surgery to remove the bladder, underscoring the urgent need to improve treatment."

Cristian Massacesi, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca, said: "The positive results for Imfinzi in the POTOMAC trial represent a significant advance that will potentially allow more patients with early-stage bladder cancer to benefit from this important immunotherapy. Building on the NIAGARA data, this outcome demonstrates our strategy of bringing novel therapies to patients with early-stage disease where there is the greatest potential for long-term benefit."

The safety and tolerability of Imfinzi plus BCG induction and maintenance therapy was consistent with the known safety profiles of the individual medicines, with no new safety concerns identified. The addition of Imfinzi did not compromise patients’ ability to complete BCG induction and maintenance therapy.

The second experimental arm evaluating Imfinzi plus BCG induction-only therapy compared to BCG induction and maintenance therapy alone did not meet the endpoint of DFS.

These data will be presented at a forthcoming medical meeting and shared with global regulatory authorities.

Imfinzi is approved in the US and other countries for patients with muscle-invasive bladder cancer (MIBC) based on results from the NIAGARA Phase III trial and continues to be investigated across early and late-stage bladder cancer in various treatment combinations, including in patients with MIBC who are ineligible or refuse to take cisplatin (VOLGA) and in locally advanced or metastatic disease (NILE).

Notes

Bladder cancer
Bladder cancer is the 9th most common cancer in the world, with more than 614,000 cases diagnosed each year.4 The most common type is urothelial carcinoma, which begins in the urothelial cells of the urinary tract.2

In 2024, an estimated 125,000 patients were treated for high-risk NMIBC, for which the current standard of care is transurethral resection of bladder tumour (TURBT) followed by administration of BCG directly into the bladder.5-6 Up to 80% of patients experience disease recurrence within five years, and rates of progression in high-risk patients can be as high as 45%.2 There is a critical need for treatment options in this curative-intent setting.

POTOMAC
POTOMAC is a randomised, open-label, multi-centre, global Phase III trial evaluating Imfinzi in combination with BCG therapy as a treatment for 1,018 patients with high-risk, BCG-naïve NMIBC who have undergone TURBT prior to randomisation. Patients were randomised 1:1:1 to receive Imfinzi plus BCG induction and maintenance therapy, or Imfinzi plus BCG induction-only therapy, versus standard-of-care BCG induction and maintenance therapy.

The trial was conducted in more than 120 centres across 12 countries including Canada and others across Europe and Asia. The primary endpoint was DFS, defined as time from randomisation to date of first recurrence of high-risk disease or death from any cause, for Imfinzi plus BCG induction and maintenance therapy compared to BCG induction and maintenance therapy alone. Secondary endpoints included DFS for Imfinzi plus BCG induction only therapy versus the comparator arm, as well as OS at five years and safety across both experimental arms of the trial.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its indication in MIBC, Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the European Union (EU).

In March 2025, perioperative Imfinzi added to standard-of-care chemotherapy met the primary endpoint of event-free survival in the MATTERHORN Phase III trial in resectable gastric and gastroesophageal junction cancers.

Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in US and EU). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in EU and Japan.

Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several gastrointestinal cancers.