Neon Therapeutics Submits IPO Filing for NEO-PV-01, a Personalized Neoantigen Vaccine Demonstrating Efficacy Across Melanoma, Non-Small Cell Lung Cancer, and Bladder Cancer

“Life-Saving Device” from the Sky! MSD Completes Delivery of Medications at -70°C via Drone

Direct Relief Announces Successful Proof-of-Concept Study Delivering Temperature-Sensitive Medicines and Vaccines to Remote Areas via Drones, Conducted Jointly by MSD, AT&T, Softbox, and Direct Relief. This Outcome May Represent a Significant Advance in Biopharmaceutical Logistics Innovation and Humanitarian Efforts.

Image source: Provided by Merck & Co., Inc.

For people in remote areas or regions affected by natural disasters, transportation and communication barriers may prevent them from accessing essential medical products. In such situations, drone delivery is one of the most effective solutions to this problem.

Merck & Co. initiated this motion, after which multiple companies joined the project under the coordination of the humanitarian aid organization Direct Relief. Volans-i manufactured all-electric drones and was responsible for their operation. Packaging specialist Softbox designed sample containers capable of maintaining low temperatures. The cold-chain delivery technology on the drones can control temperatures at -70°C, which is required for the storage and transportation of specific vaccines and medications. AT&T’s cloud technology enables researchers to continuously monitor sample temperatures and collect and analyze drone flight data in real time.

Three Birds with One Stone! Neon Cancer Vaccine Extends Progression-Free Survival in Lung Cancer, Bladder Cancer, and Melanoma

Neon Therapeutics Announces That Its Personalized Neoantigen Cancer Vaccine Prolonged Progression-Free Survival in Patients with Melanoma, Non-Small Cell Lung Cancer (NSCLC), and Bladder Cancer in Early-Stage Clinical Trials. These Results Highlight the Potential of Cancer Vaccines as an Innovative Immunotherapy Modality.

NEO-PV-01, developed by Neon Therapeutics, is a “tailor-made” personalized neoantigen vaccine. In the multicenter Phase 1b clinical trial named NT-001, patients with advanced or metastatic melanoma, smoking-related non-small cell lung cancer (NSCLC), and bladder cancer received combination therapy consisting of NEO-PV-01 and Opdivo (nivolumab). Opdivo is a PD-1 monoclonal antibody.

▲Top-line Results of NT-001 Clinical Trial (Image source: Globenewswire.com)

The trial results demonstrated that, compared with historical data for Opdivo monotherapy, the combination therapy prolonged progression-free survival (PFS) in patients with all three cancer types. At a median follow-up of 13.4 months, the median PFS had not yet been reached in patients with melanoma, whereas it was 5.6 months in patients with non-small cell lung cancer (NSCLC) and bladder cancer. Historical data indicate that Opdivo monotherapy yields a PFS of 3–7 months in melanoma patients, 2–4 months in NSCLC patients, and 2–3 months in bladder cancer patients. The combination therapy also exhibited a favorable safety and tolerability profile.

$5 Billion “Mega Deal”! Gilead Sciences and Galapagos Reach Global R&D Collaboration Agreement

Gilead Sciences Announces 10-Year Global R&D Collaboration Agreement with Belgian Biotech Company Galapagos. Gilead Sciences will obtain exclusive rights to develop and commercialize all of Galapagos’s current and future clinical-stage programs outside of Europe. This means that Gilead Sciences will gain access to Galapagos’s six clinical-stage investigational therapies, more than 20 preclinical investigational compounds, and a drug discovery platform.

This collaboration will strengthen the scientific partnership between the two companies. Gilead Sciences will gain access to Galapagos’ established research infrastructure and its unique drug discovery platform. This platform leverages disease-relevant human primary cell assays to identify and validate novel drug targets. By employing adeno-associated virus (AAV) vectors, it enables RNAi-mediated knockdown of mRNA encoding more than 6,000 proteins in cells, facilitating high-throughput screening to uncover key drug targets in diseases such as rheumatoid arthritis (RA), inflammatory bowel disease, and fibrosis. Galapagos is developing small-molecule inhibitors against these targets to actively modify disease progression, rather than merely alleviating symptoms.

▲Galapagos’ Product Pipeline (Image source: Galapagos official website)

Under the terms, Gilead will pay Galapagos a $3.95 billion upfront payment and make an $1.1 billion equity investment, increasing its stake from 13% to 22%.

Ovarian Cancer Receives “Bad News”! GSK’s PARP Inhibitor Shows Strong Performance in Phase 3 Clinical Trial

GlaxoSmithKline (GSK) announced that its PARP inhibitor Zejula (niraparib) achieved positive results in the Phase 3 PRIMA clinical trial. This therapy is indicated for first-line maintenance treatment in patients with ovarian cancer who have responded to platinum-based chemotherapy. The trial results demonstrated a significant improvement in progression-free survival (PFS), regardless of patients’ biomarker status. These findings are expected to support further expansion of Zejula’s indications. The full results of the PRIMA trial will be presented at an upcoming scientific conference.

Zejula is a key PARP inhibitor acquired by GSK following its $5.1 billion purchase of TESARO last year. PARP inhibitors are anticancer drugs developed based on the principle of “synthetic lethality.” By inhibiting the PARP-mediated DNA damage repair mechanism, they cause an accumulation of excessive DNA damage in tumors harboring BRCA gene mutations, thereby inducing cell death.

▲PARP inhibitors may benefit more ovarian cancer patients (Image source: GSK official website)

GSK’s supplemental new drug application (sNDA) to the FDA for expanding the indications of Zejula had previously been granted priority review. In the Phase 2 clinical trial named QUADRA, the therapy demonstrated efficacy not only in ovarian cancer patients with BRCA gene mutations (overall response rate [ORR] of 29%), but also in patients without BRCA gene mutations who exhibited homologous recombination deficiency (HRD) (ORR of 15%). In patients without BRCA gene mutations and without homologous recombination deficiency, Zejula achieved an ORR of 3%.

Stem Cell-Derived CAR-T Therapy on the Horizon? Takeda and Kyoto University’s Breakthrough Therapy Advances Toward Clinical Development

Takeda and Kyoto University Announce That an Innovative CAR-T Therapy Has Transitioned from Academia to Industry, with Clinical Development Entrusted to Takeda. Notably, this CAR-T therapy is developed using the Nobel Prize-winning “induced pluripotent stem cell (iPSC) technology” and holds promise for bringing transformative change to the field of cell therapy.

This therapy was developed by Professor Shin Kaneko’s team. It utilizes an induced pluripotent stem cell (iPSC) bank to create “universal” CAR-T therapies, which can be fine-tuned to meet the needs of different patients. The press release stated that it holds promise for generating large quantities of homogeneous cells for treatment from a single master cell bank. Compared with first-generation CAR-T therapies, it is expected to reduce costs. In preclinical in vivo experiments, this “stem cell-derived CAR-T therapy” targeting CD19 demonstrated potent anti-tumor activity.

“Challenging” PD-1 Therapy in Refractory Bladder Cancer: Astellas’ Innovative ADC Seeks Accelerated Approval

Astellas Pharma and Seattle Genetics jointly announced the submission of a Biologics License Application (BLA) to the U.S. FDA for enfortumab vedotin, an antibody-drug conjugate (ADC) co-developed by the two companies, seeking accelerated approval. This innovative ADC is intended for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have previously received PD-1/L1 inhibitor therapy and platinum-based chemotherapy in the neoadjuvant/adjuvant setting.

Enfortumab vedotin is an antibody-drug conjugate (ADC) that links an anti-Nectin-4 monoclonal antibody to the microtubule-disrupting agent MMAE. It utilizes Seagen’s proprietary antibody conjugation technology. Nectin-4, a cell adhesion molecule expressed on the surface of various solid tumors and highly expressed in urothelial carcinoma, is an ADC target identified by Astellas. This therapy has received Breakthrough Therapy Designation from the FDA.

▲Mechanism of Action of Enfortumab Vedotin (Image source: Urotoday.com)

This application is based on the results from Cohort 1 of the pivotal Phase 2 clinical trial, EV-201. In this Phase 2 trial, 125 patients received treatment with enfortumab vedotin. The patient population included those who had not responded to PD-1/PD-L1 inhibitors and those who had received more than three prior lines of therapy, some of whom had developed liver metastases. The trial results demonstrated that enfortumab vedotin achieved an objective response rate (ORR) of 44% in these patients. The duration of response was 7.6 months. The median overall survival (OS) was 11.7 months, and the median progression-free survival (PFS) was 5.8 months.

Breaking Bacterial Resistance! Merck’s Innovative Antibiotic Combination Approved for Market Launch

The U.S. FDA Approves Merck & Co.’s (MSD) Innovative Antibiotic Combination Recarbrio for the Treatment of Complicated Urinary Tract Infections (cUTI) and Complicated Intra-abdominal Infections (cIAI) Caused by Specific Susceptible Gram-Negative Bacteria. Recarbrio is an antimicrobial product composed of relebactam, imipenem, and cilastatin. Relebactam is an innovative beta-lactamase inhibitor, imipenem is an approved beta-lactam antibiotic, and cilastatin prevents the renal degradation of imipenem.

Relebactam, developed by Merck & Co., is an intravenously administered inhibitor of Class A and Class C β-lactamases. The combination of relebactam, imipenem, and cilastatin has been granted Qualified Infectious Disease Product (QIDP) designation and Fast Track designation by the FDA for the treatment of complicated urinary tract infections (cUTI), complicated intra-abdominal infections (cIAI), and hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia (HABP/VABP).

▲ Molecular structure of relebactam (Image source: Ed (Edgar181) [Public domain])

This approval is based on the efficacy and safety results of the imipenem-cilastatin combination in the treatment of cUTI and cIAI, as well as findings from in vitro studies and animal infection models for relebactam. The safety of Recarbrio was validated in two clinical trials involving patients with cUTI and cIAI.

"Wake Up" the Ears! Astellas and Frequency Collaborate to Develop Regenerative Therapy

Astellas and Frequency Therapeutics Announce Exclusive Licensing Agreement to Advance the Development and Commercialization of Frequency’s Regenerative Therapy FX-322Astellas and Frequency Therapeutics have announced an exclusive licensing agreement to advance the development and commercialization of Frequency Therapeutics’ regenerative therapy, FX-322. This drug is indicated for the treatment of stable sensorineural hearing loss (SSHL), the most common type of hearing loss.

FX-322 is a small-molecule drug combination designed to activate existing progenitor cells in the inner ear through progenitor cell activation (PCA), promoting hair cell regeneration, with the ultimate goal of reversing biological deficits and restoring tissue health. PCA-based regeneration is a novel therapeutic approach that repairs damaged tissue and restores healthy function in a less complex manner, while potentially offering safety advantages over traditional cell and gene therapies.

Under the collaboration agreement, Astellas will be responsible for the development and commercialization of FX-322 outside the United States, while Frequency will be responsible for its development and commercialization in the United States. The two companies will jointly conduct global clinical trials and commercialization activities. Frequency will receive an upfront payment of $80 million and may be eligible for up to $545 million in subsequent milestone payments based on development and commercialization milestones, as well as future royalties on product sales.

€1.1 Billion to Secure Anti-Fibrotic Therapy! Boehringer Ingelheim Strikes Deal

WuXi AppTec Partner Bridge Biotherapeutics Announces Collaboration and Research License Agreement with Boehringer IngelheimBridge Biotherapeutics, a partner of WuXi AppTec, has announced that it has entered into a collaboration and research license agreement with Boehringer Ingelheim. The two parties will jointly develop BBT-877, an autotaxin inhibitor developed by Bridge, for the treatment of patients with fibrotic interstitial lung diseases, including idiopathic pulmonary fibrosis (IPF). Bridge is a virtual company with only 17 full-time employees. BBT-877 is a clinical-stage investigational therapy developed by the company with “best-in-class” potential.

BBT-877, developed by Bridge Biotherapeutics, is an autotaxin inhibitor. Autotaxin is a protease that mediates key pro-fibrotic events in multiple cell types. BBT-877 has demonstrated promising efficacy and safety in preclinical models. It is currently being evaluated in Phase 1 clinical trials, with Phase 2 trials expected to initiate within the next 12 months.

▲Bridge’s R&D Pipeline (Image source: Bridge’s official website)

Under the agreement, Bridge and Boehringer Ingelheim will jointly develop BBT-877 for the treatment of IPF. Bridge will receive an upfront payment of €45 million and milestone payments of up to €1.1 billion.

Targeting “Undruggable” Targets! Orum Expands Cell-Penetrating Antibody Platform Technology

Orum Therapeutics Announces Completion of $30 Million Series B FinancingOrum Therapeutics has announced the completion of a $30 million Series B financing round. The proceeds from this round will be used to expand the company’s proprietary cell-penetrating antibody platform technology, known as Oromab. This platform enables the targeting of “undruggable” targets and the delivery of diverse therapeutic payloads. The company is committed to leveraging this platform to develop innovative therapies for cancer and rare diseases.

▲Mechanism of Action of Orum’s Antibodies (Image source: Orum’s official website)

Orum’s innovative technology platform enables the delivery of antibodies into cells, allowing them to bind intracellular proteins that lack small-molecule binding sites. The company’s Oromab technology platform can engineer antibodies that bind to cell-specific receptors; upon binding to receptors on the cell surface, these antibodies are internalized via endocytosis. Typically, internalized antibodies cannot escape from the resulting endosomes and are ultimately degraded in lysosomes. In contrast, antibodies designed using the Oromab platform can bind to lipid molecules in the mildly acidic endosomal environment, thereby escaping from endosomes into the cytoplasm, where they engage target proteins to exert therapeutic effects.

“Sniping” Colorectal Cancer! Bayer, Bristol Myers Squibb, and Ono Pharmaceutical Join Forces

Bayer, Bristol-Myers Squibb (BMS), and Ono Pharmaceutical Co., Ltd. announced that the three companies have entered into a clinical collaboration agreement to evaluate the efficacy of the combination of Bayer’s kinase inhibitor Stivarga (regorafenib) and BMS/Ono Pharmaceutical’s anti-PD-1 monoclonal antibody Opdivo (nivolumab). This regimen is intended for the treatment of patients with microsatellite stable metastatic colorectal cancer (MSS mCRC), the most common form of metastatic colorectal cancer.

Stivarga is a multi-kinase inhibitor that inhibits various kinases promoting tumor growth, including those mediating the vascular endothelial growth factor (VEGF) signaling pathway. Previously, Stivarga was approved for the treatment of patients with hepatocellular carcinoma who had not responded to existing therapies, patients with metastatic colorectal cancer (CRC), and patients with locally advanced, unresectable, or metastatic gastrointestinal stromal tumors (GIST).

Opdivo is a PD-1 inhibitor designed to help restore anti-tumor immune responses. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option for various types of cancer.

“Illuminating” Tumors! Fluorescent Tumor-Specific Antibody Enters Phase 3 Clinical Trials

SurgiMab Announces That Its Investigational Fluorescent Tumor-Specific Antibody SGM-101 Has Entered Pivotal Phase 3 Clinical Trials. The antibody has been used to improve surgical outcomes in patients with colorectal cancer (CRC) during surgery, yielding positive results, and is poised to become the first tumor-targeted fluorescent probe for fluorescence-guided surgery (FGS).

▲Mechanism of Action of SGM-101 (Image source: Surgimab official website)

SGM-101 is a tumor-specific antibody conjugated with a fluorescent dye, which emits fluorescence upon excitation by near-infrared light. Carcinoembryonic antigen (CEA) is overexpressed on the surface of more than 95% of colorectal cancer cells. This antibody selectively targets CEA, enabling real-time visualization of CEA-overexpressing tumor tissues during surgery. It allows surgeons to clearly delineate the boundary between tumor and healthy tissues, thereby facilitating accurate and complete tumor resection while preventing excessive removal of healthy tissue and better preserving its function. SGM-101 provides a novel intraoperative imaging tool for cancer surgery, a capability not currently achievable with other existing methods.

Original Title: Neon’s New Vaccine “Kills Three Birds with One Stone,” Breaking Through Lung Cancer, Bladder Cancer, and Melanoma; Merck’s Recarbrio “Combats” Bacterial Resistance

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