On May 6, 2025 Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), reported it has manufactured viral vector with its AAV Curator Platform for Nationwide Children’s Hospital’s Cellular Therapy and Cancer Immunology Program (Press release, Nationwide Children’s Hospital, MAY 6, 2025, View Source [SID1234652610]). The Adeno-Associated Virus (AAV) will be utilized in a novel cell therapy for Universal-Donor CD38KO CD33CAR-NK cells, to be studied in a forthcoming clinical trial. The Phase 1 trial will study the safety of the novel therapy in patients with advanced, high-risk acute myeloid leukemia (AML).

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Within the field of cell therapy, there is a consistent need from researchers to manufacture cGMP virus to support the advancements of these critical therapies. While many AAV manufacturing platforms are specifically designed to be scaled to support large scale indications, Andelyn’s AAV Curator Platform utilizes Optimization-by-Design to be correctly sized for scale and speed to the clinic. The well-established platform allows for the ability to support the exacting viral vector manufacturing needs of the NCH NK cells created on the hospital’s patented universal-donor NK cell platform.

Matt Niloff, Chief Commercial Officer at Andelyn said, "We are excited to be playing a key role in this very promising Universal Donor CD38KO CD33CAR-NK cell therapy technology by NCH. We look forward to the clinical trial and ushering in a new era for patients with AML and other blood cancers."

Andelyn is enabling the progression of life-altering cell and gene therapies for both rare and prevalent diseases with the highest quality standards and scalable end-to-end development and manufacturing capabilities at its Columbus, Ohio facilities.

On May 6, 2025 Intensity Therapeutics, Inc. (Nasdaq: INTS), ("Intensity" or "the Company") a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, and The Swiss Group for Clinical Cancer Research SAKK ("SAKK"), a decentralized academic research institute that has been conducting clinical trials of cancer treatments in all major Swiss hospitals since 1965, reported that the European Medicines Agency ("EMA") has authorized the initiation of the INVINCIBLE-4 (SAKK 66/22) ("INVINCIBLE-4 Study") (NCT06358573) in France in collaboration with Unicancer (Press release, Intensity Therapeutics, MAY 6, 2025, View Source [SID1234652609]).

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The INVINCIBLE-4 Study is a randomized open-label, multicenter study to determine the clinical activity, safety, and tolerability of INT230-6 in patients with early-stage, operable triple-negative breast cancer ("TNBC") who undergo standard of care neoadjuvant immunochemotherapy ("SOC") treatment and SOC alone. The primary endpoint is pathological complete response ("pCR") in the primary tumor and affected lymph nodes. Patients will be randomized one-to-one to receive a regimen of either two doses of INT230-6 followed by SOC, which consists of pembrolizumab, anthracyclines, carboplatin, cyclophosphamide, and paclitaxel (i.e., the Keynote-522 regimen), or the SOC alone. The study is already recruiting patients in Switzerland and is expected to enroll 54 patients.

"We are encouraged to see high levels of tumor necrosis from the MRI scans and evidence of tumor inflammation after two INT230-6 injections and prior to initiation of the SOC in our first patients," said Ursina Zürrer, M.D. Chief Physician for Genetic Counseling, Department of Medical Oncology and Hematology Cantonal Hospital Winterthur, Switzerland, and the Coordinating Investigator for the INVINCIBLE-4 Study. "If the immunological cancer cell death and the ignition of an anti-cancer immune response without increased toxicity in patients receiving INT230-6 shows a meaningful increase in pCR, it would be a major advance for the neoadjuvant treatment of breast cancer and potentially other cancers."

"The acceptance of the INVINCIBLE-4 by the EMA and our expansion of the trial into France is expected to increase our enrollment rate starting in the second quarter of 2025. We should almost double the number of sites actively screening patients. We are excited to work with Unicancer, a group accredited by the French National Cancer Institute with centers of academic excellence and strong operational capability throughout France." Said Lewis H. Bender, President and CEO of Intensity Therapeutics."

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

About Triple Negative Breast Cancer in the Presurgical Setting
Women with aggressive forms of breast cancer, such as TNBC, are often counseled to undergo pre-surgical (neoadjuvant) systemic therapy in advance to reduce the risk of the disease returning. Having a pathological complete response, meaning the absence of live cancer at the time of surgery, has been shown to result in a lower risk of recurrence. Approximately 11-17% of breast cancers test negative for estrogen receptors (ER), progesterone receptors (PR), and overexpression of human epidermal growth factor receptor 2 (HER2) protein, qualifying them as triple negative. There are approximately 56,000 new cases of TNBC in the US and 420,000 Worldwide diagnosed each year, the majority of which are local to the breast. TNBC is considered to be more aggressive and has a poorer prognosis than other types of breast cancer, because there are fewer available targeted medicines. Most patients with local TNBC typically receive immune/chemotherapy before surgery. Since the publication of Keynote-522, the standard neoadjuvant treatment for TNBC includes systemic chemotherapy (anthracyclines, cyclophosphamide, paclitaxel, carboplatin) and the anti-PD-1 monoclonal antibody pembrolizumab. pCR rates are 65%, with rates generally lower in the larger-sized tumors or with lymph node metastasis. The toxicity of the Keynote-522 regimen is high, with 80% of patients experiencing grade 3 or higher treatment-related AEs, including treatment-related adverse events that lead to death in 0.5% of patients.

On May 6, 2025 Viralgen, a leading contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated virus (rAAV) gene therapies, reported to have established a strategic collaboration with Trogenix, a pioneering biotech company dedicated to developing innovative cancer therapies (Press release, Trogenix, MAY 6, 2025, View Source [SID1234652608]). As part of this partnership, Viralgen successfully scaled-up and completed in under 12 months a good manufacturing practice (GMP) clinical trial material batch of Trogenix’s rAAV gene therapy, TGX-007, accelerating the program’s progression toward first-in-human (FIH) clinical trials for glioblastoma, one of the most aggressive and treatment-resistant forms of brain cancer.

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Beyond manufacturing, Viralgen has developed a gene-specific titration method and a custom specific formulation buffer to serve as a diluent for the drug product’s administration. These efforts will support the advancement of Trogenix’s gene therapy.

"Our expertise in rAAV vector manufacturing and ability to scale allows us to support and accelerate critical clinical therapeutic programs" said Jimmy Vanhove, CEO of Viralgen. "We are thrilled to contribute to Trogenix’s pioneering approach in oncology gene therapy, which has potential for curative responses in glioblastoma and other cancers," stated Vanhove.

Trogenix’s proprietary Synthetic Super-Enhancers (SSEs) platform, Odysseus, is designed to develop precision genetic medicines targeting the disease cell state. Following the successful manufacturing of the first GMP batch of Trogenix’s rAAV vector at Viralgen’s state-of-the-art facility, TGX-007 is now advancing toward clinical evaluation, and, ultimately, to patients in need.

"Glioblastoma, the most common form of brain cancer, is a devastating disease with very poor prognosis and few treatment options for patients. At Trogenix, our aim is to transform cancer treatment from chronic disease management to a potentially curative one-time treatment," said Ken Macnamara, PhD, CEO of Trogenix. "By collaborating with Viralgen, we can rapidly scale product supply and bring the therapy to patients as quickly as possible."

This collaboration underscores Viralgen’s commitment to accelerating the development of innovative AAV-based therapies by providing scalable, high-quality manufacturing solutions that help bring treatments to patients faster.

On May 6, 2025 Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company, reported that its product portfolio of FDA-approved brands delivered combined net revenues of $11.7 million during the first quarter of 2025, a 38% increase over the prior year period (Press release, Cumberland Pharmaceuticals, MAY 6, 2025, View Source [SID1234652607]). As a result the Company generated a net profit of $1.3 million for the quarter, adjusted earnings of $2.4 million, and cash flow from operations of $3.9 million.

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Cumberland ended the quarter with approximately $70 million in total assets, $41.6 million in liabilities and $28.7 million of shareholders’ equity.

"We are entering an exciting time for our company, as we continue to build momentum and capitalize on a range of promising opportunities," said Cumberland Pharmaceuticals CEO A.J. Kazimi. "Our optimism is driven by strong performance from our approved brands, the expansion of international partnerships, meaningful progress across our clinical development programs and the potential for strategic acquisitions."

RECENT COMPANY DEVELOPMENTS INCLUDE:

Top-Line DMD Study Results

In February 2025, Cumberland announced positive top-line results from the Phase II study evaluating its ifetroban product candidate in patients with Duchenne muscular dystrophy (DMD). This marks a breakthrough for these patients, as it’s the first successful Phase II study specifically targeting the cardiac complications of their condition.

These study results were selected for a late-breaking presentation in March at the Muscular Dystrophy Association’s Clinical & Scientific Conference. That platform allowed Cumberland to share the promising results with the global DMD community, including leading researchers, clinicians and patient advocates who are working tirelessly to improve outcomes for those affected by this devastating disease.

Next steps for Cumberland’s DMD program include further data analysis and completion of a full study report in preparation for an end-of-Phase-II meeting with the FDA to determine the requirements for the product’s approval.

Vibativ Approval in China

Cumberland’s potent antibiotic, Vibativ, received approval from the regulatory authorities in China. This provides Cumberland access to the world’s second-largest pharmaceutical market. The company expects the product to launch by the end of the year.

FINANCIAL RESULTS:

Net Revenue: For first quarter of 2025, net revenues were $11.7 million and included $3.5 million for Kristalose, $2.3 million for Sancuso, $1.4 million for Vibativ and $1.3 million for Caldolor.

Operating Expenses: Total operating expenses for the quarter were $10.4 million.

Net Income: The net income for the first quarter of 2025 was approximately $1.3 million.

Adjusted Earnings: Adjusted earnings for the quarter were $2.4 million, or $0.16 per share.

Balance Sheet: At March 31, 2025, Cumberland had approximately $70 million in total assets, including $15.1 million in cash and cash equivalents. Liabilities totaled $41.6 million, including $5.2 million on the company’s credit facility. Total shareholders’ equity was $28.7 million on March 31, 2025.

EARNINGS REPORT CALL:

A conference call will be held today, May 06, 2025, at 4:30 p.m. Eastern Time to provide a company update and discuss the financial results.

The link to register is: View Source

Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.

A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting:

View Source

On May 6, 2025 Adcentrx Therapeutics ("Adcentrx"), a clinical-stage biotechnology company redefining Antibody-Drug Conjugate (ADC) therapies for cancer treatment and other life-threatening diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead program, ADRX-0706, for the treatment of patients with locally advanced or metastatic squamous cell cervical cancer (Press release, Adcentrx Therapeutics, MAY 6, 2025, View Source [SID1234652606]).

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ADRX-0706 is a Nectin-4 ADC being evaluated in the Phase 1b portion of an ongoing Phase 1a/b clinical trial (NCT06036121) for the treatment of select advanced solid tumors, including cervical cancer. The company will present interim data from the completed Phase 1a dose escalation portion at the upcoming 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Early findings demonstrated a differentiated safety and pharmacokinetic profile, including a notably lower incidence of adverse events such as peripheral neuropathy, along with preliminary efficacy signals across multiple tumor types.

Cervical cancer represents a significant unmet need, particularly for patients whose disease progresses following initial treatment. Nectin-4 is highly expressed in cervical cancer, making it a compelling tumor type for treatment with a Nectin-4 ADC.

"The Fast Track designation granted by the FDA underscores the significant unmet need in advanced cervical cancer and marks another meaningful milestone for Adcentrx," said Hui Li, Ph.D., Founder and Chief Executive Officer of Adcentrx. "This recognition, together with the early clinical signals observed for ADRX-0706, reinforces the best-in-class potential of our Nectin-4 ADC and provides the opportunity for enhanced regulatory dialogue as we continue advancing this important program through clinical development."

The FDA’s Fast Track program is designed to accelerate the development and review of therapies for serious conditions with unmet medical needs, with the goal of getting important new treatments to patients sooner. The designation enables earlier and more frequent communication with the FDA throughout development and may offer regulatory advantages such as eligibility for Accelerated Approval, Priority Review, and Rolling Review, which allows completed sections of a New Drug Application (NDA) or Biologic License Application (BLA) to be reviewed, rather than waiting for the entire completed application.

About ADRX-0706
ADRX-0706 is a fully proprietary ADC product candidate discovered by Adcentrx. The antibody component is a novel fully human IgG1 targeting Nectin-4, a cell surface adhesion protein with high expression in multiple solid tumors and limited expression in normal tissues. Nectin-4 is associated with poor disease prognosis and is a validated target for ADCs.

The ADRX-0706 antibody is linked to a proprietary tubulin inhibitor payload, AP052, through Adcentrx’s innovative i-Conjugation technology platform – a core component in the design of the company’s ADCs. The platform utilizes a cleavable linker and stable conjugation chemistry to enhance payload delivery. This novel technology enables a highly stable ADC with a drug-antibody ratio of eight (DAR 8) with a substantially expanded therapeutic window as demonstrated in preclinical studies.

ADRX-0706 has a favorable pharmacokinetic and safety profile in preclinical models and has demonstrated significant efficacy across a variety of tumor indications in vitro and in vivo.

For more information about the ongoing ADRX-0706 Phase 1a/b clinical trial, please refer to the Study ID NCT06036121 on ClinicalTrials.gov.