On November 17, 2025 Shanghai Henlius Biotech, Inc. (2696.HK), and Organon (NYSE: OGN) reported that the US Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for POHERDY (pertuzumab-dpzb) 420 mg/14 mL injection for intravenous use, an interchangeable biosimilar to PERJETA (pertuzumab), for all indications of the reference product.1 POHERDY is the first and only approved pertuzumab biosimilar in the US, representing an important milestone in expanding access to quality and potentially more affordable biologic therapies for patients with certain HER2-positive breast cancers.2

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"Expanding access to treatments for diseases that disproportionately impact women, including breast cancer, the most common cancer among women in the US excluding skin cancer, is at the core of our mission," said Jon Martin, US Commercial Lead, Biosimilars and Established Brands at Organon.3 "Not only is POHERDY the first approved biosimilar to PERJETA in the US, but its approval also builds on Organon’s recent momentum of expanding our biosimilars portfolio in women’s health and oncology. Our collaboration with Henlius is critical to our goal of making health care more sustainable for US patients."

"The FDA approval of POHERDY marks a significant milestone in Henlius’ global expansion and quality biologics development. As the first pertuzumab biosimilar approved in the US, this important achievement demonstrates our core capability to build a sustainable global R&D system grounded in rigorous scientific and regulatory standards. It also reflects Henlius’ steadfast commitment to its patient-centric philosophy and long-term global strategy," said Dr. Jason Zhu, Executive Director and Chief Executive Officer of Henlius. "We will continue accelerating the delivery of quality biologics to benefit more patients worldwide and create greater value for human health."2

"The approval of POHERDY further underscores Henlius’ track record in international registration, together with our strength in quality management and commercialization collaboration," said Ping Cao, Chief Business Development Officer and Senior Vice President of Henlius. "We look forward to working closely with our partner Organon to leverage our complementary strengths in supply chain, market, and distribution networks, jointly enhancing access to quality biologics and providing patients with treatment options that combine quality and affordability."2

POHERDY is a HER2/neu receptor antagonist indicated for use in combination with trastuzumab and docetaxel for the treatment of adults with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease. POHERDY is also indicated for use in combination with trastuzumab and chemotherapy as (i) neoadjuvant treatment of adults with HER2-positive, locally advanced, inflammatory, or early stage breast cancer (either greater than 2 cm in diameter or node positive) as part of a complete treatment regimen for early breast cancer and (ii) adjuvant treatment of adults with HER2-positive early breast cancer at high risk of recurrence. See full indications below.

Pertuzumab products can cause subclinical and clinical cardiac failure manifesting as decreased left ventricular ejection fraction (LVEF) and congestive heart failure (CHF). Evaluate cardiac function prior to and during treatment. Discontinue POHERDY treatment for a confirmed clinically significant decrease in left ventricular function. Exposure to pertuzumab products can cause embryo-fetal death and birth defects. Advise patients of these risks and the need for effective contraception. See additional safety information below.

POHERDY was approved based on the review of a comprehensive data package, which includes analytical similarity, clinical pharmacokinetic studies, and comparative clinical studies demonstrating that POHERDY is highly similar to and interchangeable with the reference product PERJETA in terms of safety, purity, and potency (safety and effectiveness).4,5

In 2022, Henlius entered into a license and supply agreement with Organon, granting Organon the exclusive commercialization rights to multiple biosimilars, including POHERDY. The agreement covers exclusive global commercialization rights except for China.6 The FDA approval of POHERDY will further enhance the partners’ oncology portfolio and their ability to deliver quality biologics to more patients.2

About POHERDY (pertuzumab-dpzb)

POHERDY is a HER2/neu receptor antagonist indicated for:

Metastatic Breast Cancer (MBC): POHERDY is indicated for use in combination with trastuzumab and docetaxel for the treatment of adults with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease.
Early Breast Cancer (EBC): POHERDY is indicated for use in combination with trastuzumab and chemotherapy for:
The neoadjuvant treatment of adults with HER2-positive, locally advanced, inflammatory, or early stage breast cancer (either greater than 2 cm in diameter or node positive) as part of a complete treatment regimen for early breast cancer
The adjuvant treatment of adults with HER2-positive early breast cancer at high risk of recurrence
SELECTED SAFETY INFORMATION

LEFT VENTRICULAR DYSFUNCTION and EMBRYO-FETAL TOXICITY

Pertuzumab products can cause subclinical and clinical cardiac failure manifesting as decreased left ventricular ejection fraction (LVEF) and congestive heart failure (CHF). Evaluate cardiac function prior to and during treatment. Discontinue POHERDY treatment for a confirmed clinically significant decrease in left ventricular function.
Exposure to pertuzumab products can cause embryo-fetal death and birth defects. Advise patients of these risks and the need for effective contraception.
CONTRAINDICATIONS

POHERDY is contraindicated in patients with known hypersensitivity to pertuzumab products or to any of its excipients.

WARNINGS AND PRECAUTIONS

Left Ventricular Dysfunction

Pertuzumab products can cause left ventricular dysfunction, including symptomatic heart failure. Decreases in LVEF have been reported with drugs that block HER2 activity, including pertuzumab products.

Assess LVEF prior to initiation of POHERDY and at regular intervals during treatment to ensure that LVEF is within normal limits. If the LVEF declines and has not improved, or has declined further at the subsequent assessment, consider permanent discontinuation of POHERDY and trastuzumab.

In the pertuzumab-treated patients with MBC in CLEOPATRA, left ventricular dysfunction occurred in 4% of patients, and symptomatic left ventricular systolic dysfunction (LVSD) (congestive heart failure) occurred in 1% of patients. Patients who received prior anthracyclines or prior radiotherapy to the chest area may be at higher risk of decreased LVEF or left ventricular dysfunction.

In patients receiving pertuzumab as a neoadjuvant treatment in combination with trastuzumab and docetaxel in NeoSphere, LVEF decline >10% and a drop to <50% occurred in 8% of patients, and left ventricular dysfunction occurred in 3% of patients. LVEF recovered to ≥50% in all of these patients.

In patients receiving neoadjuvant pertuzumab in TRYPHAENA, LVEF decline >10% and a drop to <50% occurred in 7% of patients treated with pertuzumab plus trastuzumab and fluorouracil, epirubicin, and cyclophosphamide (FEC) followed by pertuzumab plus trastuzumab and docetaxel, 16% of patients treated with pertuzumab plus trastuzumab and docetaxel following FEC, and 11% of patients treated with pertuzumab in combination with docetaxel, carboplatin, and trastuzumab (TCH). Left ventricular dysfunction occurred in 6% of patients treated with pertuzumab plus trastuzumab and FEC followed by pertuzumab plus trastuzumab and docetaxel, 4% of patients treated with pertuzumab plus trastuzumab and docetaxel following FEC, and 3% of patients treated with pertuzumab in combination with TCH. Symptomatic LVSD occurred in 4% of patients treated with pertuzumab plus trastuzumab and docetaxel following FEC, 1% of patients treated with pertuzumab in combination with TCH, and none of the patients treated with pertuzumab plus trastuzumab and FEC followed by pertuzumab plus trastuzumab and docetaxel. LVEF recovered to ≥50% in all but 1 patient.

In patients receiving neoadjuvant pertuzumab in BERENICE, in the neoadjuvant period, LVEF decline ≥10% and a drop to <50% as measured by ECHO/MUGA assessment occurred in 7% of patients treated with pertuzumab plus trastuzumab and paclitaxel following dose-dense doxorubicin and cyclophosphamide (ddAC) and 2% of patients treated with pertuzumab plus trastuzumab and docetaxel following FEC. Ejection fraction decreased (asymptomatic LVD) occurred in 7% of patients treated with pertuzumab plus trastuzumab and paclitaxel following ddAC and 4% of the patients treated with pertuzumab plus trastuzumab and docetaxel following FEC in the neoadjuvant period. Symptomatic LVSD (New York Heart Association [NYHA] Class III/IV Congestive Heart Failure) occurred in 2% of patients treated with pertuzumab plus trastuzumab and paclitaxel following ddAC and none of the patients treated with pertuzumab plus trastuzumab and docetaxel following FEC in the neoadjuvant period.

In patients receiving adjuvant pertuzumab in APHINITY, the incidence of symptomatic heart failure (NYHA Class III/IV) with a LVEF decline ≥10% and a drop to <50% was 0.6%. Of the patients who experienced symptomatic heart failure, 47% of pertuzumab-treated patients had recovered (defined as 2 consecutive LVEF measurements above 50%) at the data cutoff. The majority of the events (86%) were reported in anthracycline-treated patients. Asymptomatic or mildly symptomatic (NYHA Class II) declines in LVEF ≥10% and a drop to <50% were reported in 3% of pertuzumab-treated patients, of whom 80% recovered at the data cutoff.

Pertuzumab products have not been studied in patients with a pretreatment LVEF value of <50%; a prior history of CHF; decreases in LVEF to <50% during prior trastuzumab therapy; or conditions that could impair left ventricular function such as uncontrolled hypertension, recent myocardial infarction, serious cardiac arrhythmia requiring treatment, or a cumulative prior anthracycline exposure to >360 mg/m2 of doxorubicin or its equivalent.

Embryo-Fetal Toxicity

Based on its mechanism of action and findings in animal studies, pertuzumab products can cause fetal harm when administered to a pregnant woman. Pertuzumab products are HER2/neu receptor antagonists. Cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death have been reported with use of another HER2/neu receptor antagonist (trastuzumab) during pregnancy.

Verify the pregnancy status of females of reproductive potential prior to the initiation of POHERDY. Advise pregnant women and females of reproductive potential that exposure to POHERDY in combination with trastuzumab during pregnancy or within 7 months prior to conception can result in fetal harm, including embryo-fetal death or birth defects. Advise females of reproductive potential to use effective contraception during treatment and for 7 months following the last dose of POHERDY in combination with trastuzumab.

Infusion-Related Reactions

Pertuzumab products can cause serious infusion reactions, including fatal events.

In CLEOPATRA, on the first day, when only pertuzumab was administered, infusion-related reactions occurred in 13% of patients, and <1% were Grade 3 or 4. The most common infusion reactions (≥1%) were pyrexia, chills, fatigue, headache, asthenia, hypersensitivity, and vomiting. During the second cycle when all drugs were administered on the same day, the most common infusion reactions in the pertuzumab-treated group (≥1%) were fatigue, dysgeusia, hypersensitivity, myalgia, and vomiting.

In APHINITY, when pertuzumab was administered in combination with trastuzumab and chemotherapy on the same day, infusion-related reactions occurred in 21% of patients, with <1% of patients experiencing Grade 3-4 events.

Observe patients closely for 60 minutes after the first infusion and for 30 minutes after subsequent infusions of POHERDY. If a significant infusion-related reaction occurs, slow or interrupt the infusion, and administer appropriate medical therapies. Monitor patients carefully until complete resolution of signs and symptoms. Consider permanent discontinuation in patients with severe infusion reactions.

Hypersensitivity Reactions/Anaphylaxis

Pertuzumab products can cause hypersensitivity reactions, including anaphylaxis.

In CLEOPATRA, the overall frequency of hypersensitivity/anaphylaxis reactions was 11% in pertuzumab-treated patients, with Grade 3-4 hypersensitivity reactions and anaphylaxis occurring in 2% of patients.

In NeoSphere, TRYPHAENA, BERENICE, and APHINITY, hypersensitivity/anaphylaxis events were consistent with those observed in CLEOPATRA. In APHINITY, the overall frequency of hypersensitivity/anaphylaxis was 5% in the pertuzumab-treated group. The incidence was highest in the pertuzumab plus TCH–treated group (8%), with 1% Grade 3-4 events.

Observe patients closely for hypersensitivity reactions. Severe hypersensitivity, including anaphylaxis and fatal events, has been observed in patients treated with pertuzumab products. Angioedema has been described in postmarketing reports. Medications to treat such reactions, as well as emergency equipment, should be available for immediate use prior to administration of POHERDY.

ADVERSE REACTIONS

Metastatic Breast Cancer

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and docetaxel were diarrhea, alopecia, neutropenia, nausea, fatigue, rash, and peripheral neuropathy.

Neoadjuvant Treatment of Breast Cancer

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and docetaxel were alopecia, diarrhea, nausea, and neutropenia.

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and docetaxel when given for 3 cycles following 3 cycles of FEC were fatigue, alopecia, diarrhea, nausea, vomiting, and neutropenia.

The most common adverse reactions (>30%) with pertuzumab in combination with TCH were fatigue, alopecia, diarrhea, nausea, vomiting, neutropenia, thrombocytopenia, and anemia.

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and paclitaxel when given for 4 cycles following 4 cycles of ddAC were nausea, diarrhea, alopecia, fatigue, constipation, peripheral neuropathy, and headache.

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and docetaxel when given for 4 cycles following 4 cycles of FEC were diarrhea, nausea, alopecia, asthenia, constipation, fatigue, mucosal inflammation, vomiting, myalgia, and anemia.

Adjuvant Treatment of Breast Cancer

The most common adverse reactions (>30%) with pertuzumab in combination with trastuzumab and chemotherapy were diarrhea, nausea, alopecia, fatigue, peripheral neuropathy, and vomiting.

Before prescribing POHERDY, please read the Prescribing Information, including the Boxed Warning about left ventricular dysfunction and embryo-fetal toxicity.

(Press release, Shanghai Henlius Biotech, NOV 17, 2025, View Source [SID1234660043])

On November 17, 2025 Johnson & Johnson (NYSE: JNJ) reported that it has entered into a definitive agreement to acquire Halda Therapeutics OpCo, Inc. (Halda), a clinical-stage biotechnology company with a proprietary Regulated Induced Proximity TArgeting Chimera (RIPTAC) platform to develop oral, targeted therapies for multiple types of solid tumors, including prostate cancer, for $3.05 billion in cash. The transaction is expected to close within the next few months, subject to antitrust clearance and other customary closing conditions.

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The lead candidate, HLD-0915, is a clinical-stage therapy for prostate cancer, of which new diagnoses are projected to reach 1.7 million globally by 20301. Given the existing unmet need, this once-daily therapy has the potential to transform patient outcomes with its novel precision cancer cell-killing approach that can overcome mechanisms of resistance to treatment. The acquisition also includes several earlier candidates for breast, lung and multiple other tumor types. Halda’s pipeline and platform may also enable the creation of novel targeted therapies beyond oncology.

"This acquisition further strengthens our deep oncology pipeline with an exciting lead asset in prostate cancer and a platform capable of treating multiple cancers and diseases beyond oncology, providing a potential mid- and long-term catalyst for growth," said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. "We look forward to combining Halda’s pipeline, platform and people with our world class R&D, commercial and manufacturing capabilities and advancing our goal of bringing these therapies to patients around the world."

"Many therapies lose effectiveness over time due to resistance. Halda’s innovative technology is designed to work even when cancers no longer respond to standard treatments using a novel mechanism that enables the selective killing of cancer cells," said John C. Reed, M.D., Ph.D., Executive Vice President, Innovative Medicine, R&D, Johnson & Johnson. "Results seen with HLD-0915 demonstrate impressive preliminary efficacy and a strong early safety profile in prostate cancer. We are eager to accelerate the ongoing Phase 1/2 clinical trial of HLD-0915 and progress a pipeline of novel product candidates based on RIPTAC technology​."

The planned acquisition underscores Johnson & Johnson’s longstanding commitment to prostate cancer and industry-leading oncology portfolio, adding new therapies with novel and complementary mechanisms of action. Halda’s pipeline of differentiated assets, if successful, will provide critical new options for patients.

About the Acquisition Agreement

Under the terms of the agreement, Johnson & Johnson will acquire Halda. The transaction will be accounted for as a business combination and is expected to close within the next few months, subject to antitrust clearance and other customary closing conditions. The Company expects dilution in 2026 of $0.15 to Adjusted Earnings Per Share (EPS) due to short-term financing and a non-recurring charge related to the equity awards for Halda employees upon closing. Johnson & Johnson will provide commentary on full year 2026 guidance during the fourth quarter earnings call on Wednesday, January 21, 2026.

(Press release, Johnson & Johnson, NOV 17, 2025, View Source [SID1234660042])

On November 17, 2025 Solve Therapeutics, a clinical-stage biotechnology company developing best-in-class antibody-drug conjugates (ADCs) for solid tumor malignancies, reported it has raised $120 million in an oversubscribed and upsized financing to accelerate the development of its clinical pipeline and proprietary CloakLink linker platform.

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The round was led by Yosemite, with participation from Abingworth, Ally Bridge Group, B Capital, Balyasny Asset Management, Merck & Co., and SymBiosis, and existing investors Alexandria Venture Investments, AyurMaya Capital Management, DC Global Ventures, General Atlantic, and Surveyor Capital (a Citadel company). This latest funding follows a $75 million financing completed in December 2024, bringing Solve’s total capital raised to $321 million.

Solve Therapeutics was founded to develop next-generation ADCs capable of addressing unique challenges presented by solid tumors. Traditional ADCs often face limitations related to payload hydrophobicity, including sub-optimal pharmacokinetics and plasma stability, which can compromise safety and efficacy.

Solve’s proprietary CloakLink technology was engineered to overcome these barriers by increasing ADC stability and decreasing ADC hydrophobicity across a broad range of drug-to-antibody ratios. The result is a class of ADCs with improved pharmacokinetics, enhanced plasma stability, and reduced toxicity, enabling improved therapeutic indices and overall performance.

The company’s lead programs, SLV-154 and SLV-324, are currently in Phase 1 clinical trials in patients with solid tumors. Both utilize the CloakLink platform and targeting antibodies that are specifically engineered for superior ADC performance. The ADCs are paired with novel diagnostic approaches to enable precision patient selection. The new funding will support the completion of Phase 1b studies for both programs and expand the company’s operational capabilities as it advances toward later-stage clinical development.

"We’re thrilled to partner with an outstanding syndicate of investors who share our vision for developing best-in-class ADCs," said Dave Johnson, CEO & Co-Founder, Solve Therapeutics. "Since founding the company, we’ve built a differentiated platform that combines next-generation ADC engineering, a superior hydrophilic linker system, and novel patient-selection diagnostics. This investment syndicate represents a strong endorsement of our science, our team, and our mission to develop more effective and safer targeted therapies for patients with solid tumors."

"Solve is the next wave of ADC innovation," said Dan McHugh, Investor at Yosemite and Solve Therapeutics board member. "By integrating therapeutic development with a novel diagnostic platform, Solve is pushing the boundaries of precision oncology and enabling a more personalized, effective approach to cancer care. Yosemite is excited to support the excellence and innovation demonstrated by this best-in-class team."

Founded by leaders behind VelosBio (acquired by Merck) and Acerta Pharma (acquired by AstraZeneca), Solve Therapeutics is applying decades of combined oncology and ADC expertise to build a pipeline of pioneering therapeutics and diagnostics targeting solid tumors with high unmet need.

(Press release, Solve Therapeutics, NOV 17, 2025, View Source [SID1234660041])

On November 17, 2025 CorriXR Therapeutics, Inc., an oncology-focused biotherapeutics company pioneering a novel gene editing platform to overcome drug resistance in solid tumors, reported the publication of a manuscript in Molecular Therapy Oncology detailing results from a preclinical study evaluating CRISPR-directed gene editing for the treatment of squamous cell lung carcinoma (LUSC). The study was conducted in collaboration with scientists at ChristianaCare’s Gene Editing Institute (GEI).

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"This foundational work strengthens CorriXR’s strategy of disrupting cancer cell survival pathways to restore sensitivity to standard therapies," said Eric B. Kmiec, Ph.D., Founder and Chief Executive Officer of CorriXR Therapeutics and Executive Director of GEI. "These findings build on more than a decade of GEI research into NRF2, a master regulator of cellular stress responses and known driver of treatment resistance. We are encouraged by the consistency of results across in vitro human lung cancer models and our in vivo studies and are actively pursuing IND-enabling work to bring this promising approach to patients."

Key findings from the study include:

Restoration of chemosensitivity: Editing 20-40% of LUSC cells to disrupt NRF2 was sufficient to resensitize tumors to chemotherapy, resulting in significant reductions in tumor growth.
Reduced cancer-driving signals: Edited tumors reduced NRF2 expression and downregulation of its downstream markers, demonstrating effective pathway disruption.
No off-target editing above background: Unintended edits remained below 0.2% supporting the specificity and safety of the gene editing approach.
Strong translational potential: The lipid nanoparticle (LNP) delivery system achieved robust editing in both engineered and patient-derived tumor models, reinforcing the feasibility of advancing towards clinical development.
"Treatment resistance remains one of the greatest challenges in oncology, and these data demonstrate that targeting NRF2 can meaningfully resensitize tumors with minimal off-target effects," said Kelly Banas, Ph.D., lead author of the study and Associate Director of Research at GEI. "This approach has the potential to lower chemotherapy doses, reduce toxicity and help patients remain healthier throughout treatment." Kmiec added, "Instead of creating entirely new drugs, we are using gene editing to make existing ones effective again."

The study also highlights that the biology of NRF2 driven resistance extends beyond lung cancer. "While this work focused on LUSC, NRF2 overactivation drives treatment resistance across multiple solid tumors, including head and neck squamous cell carcinoma (HNSCC)," said Kmiec. "These data indicate that CRISPR-enabled targeting of NRF2 may disrupt the tumor microenvironment and address a shared mechanism of therapeutic failure."

LUSC is an aggressive form of non-small cell lung cancer (NSCLC), representing 20-30% of lung cancer cases and affecting an estimated 190,000 people annually in the U.S. Chemotherapy remains a cornerstone of care, but many patients develop resistance, leaving limited options beyond dose escalation, which increases toxicity and typically worsens quality of life. NRF2 overactivation is a well-established driver of this resistance across multiple solid tumors, including HNSCC, esophageal and liver cancers – representing a significant unmet medical need.

These findings provide a compelling foundation to advance CorriXR’s lead program for HNSCC, as well as the Company’s LUSC program, into clinical development. CorriXR and GEI are now independently validating results at commercial CROs, conducting the required safety and regulatory studies to support an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for approval of human trials, and are exploring partnerships to accelerate clinical translation.

The full publication: Functional characterization of tumor-specific CRISPR-directed gene editing as a combinatorial therapy for the treatment of solid tumors – ScienceDirect is available online in Molecular Therapy Oncology.

(Press release, CorriXR Therapeutics, NOV 17, 2025, View Source [SID1234660040])

On November 17, 2025 Acuitas Therapeutics, a global leader in lipid nanoparticle (LNP) delivery systems for the acceleration of partners’ clinical development, reported its Next-Generation LNP advancements, a suite of novel and enhanced technologies that expand the range of diseases treatable with mRNA-LNP medicines, at the 13th International mRNA Health Conference in Berlin. Also at the conference, the company highlighted additional preclinical data on its LNP formulations’ applicability in cancer vaccines, potency, and safety.

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"Commercial validation of our technology has provided both an important milestone of success and the impetus for continued advancement," said Dr. Thomas Madden, CEO of Acuitas Therapeutics. "Our research, presented at the mRNA Health Conference, is focused on two key goals: expanding the application of our technology into new therapeutic areas and enhancing the potency and safety of the platform itself, with novel LNP formulations and various improvement strategies. Underpinning both is our commitment to improving the translatability of preclinical data, which is essential for accelerating the journey of mRNA and personalized therapies from the laboratory bench to the clinic."

Next-Generation LNP for mRNA-based Therapeutics

At the conference, Acuitas’ Chief Scientific Officer, Dr. Ying Tam, showcased the company’s Next-Generation LNP advancements, a comprehensive approach that uses multiple technologies and strategies to improve all aspects of LNP utility and applicability – from potency, safety, extrahepatic targeting, to manufacturing.

The advancements were featured in an oral presentation titled "Next-Generation Lipid Nanoparticles for Clinical Development of mRNA-based Therapeutics." The presentation detailed Acuitas’ efforts in advancing mRNA-LNP beyond current clinical standards and broadening the range of diseases these therapies can treat. Key highlights of the presentation include:

Novel LNP candidates engineered for significantly improved potency, as high as a four-fold increase for some cases, in gene editing and vaccine applications.
Optimized lipid structures reduced liver exposure, leading to increased tolerability, lowered liver enzymes, while preserving therapeutic activity in mice.
DARPin-conjugated LNP candidates that achieved highly targeted delivery to immune cells (T-lymphocytes), with a long-circulating format further enhancing uptake efficiency and expression levels.
Mucous penetrant mRNA-LNP candidates capable of extrahepatic delivery to airway epithelial cells in cystic fibrosis lung models, enabling effective gene editing compared to control LNP.
An alternative LNP manufacturing approach, called pre-formed vesicles (PFV), with equivalent potency to standard benchmark manufacturing methods, offered significant improvements in cost, storage, distribution, and flexibility of LNP manufacturing, especially for personalized mRNA-LNP therapies.
Additional Posters Presented

In addition to its lead presentation, Acuitas also showcased three posters that elucidated the mechanics of LNP delivery and assessed its existing slate of lipids, as well as explored novel options for future formulations.

Applying Clinically Approved ALC-315 in Cancer Vaccines

Acuitas’ ALC-315 ionizable lipid — used in the Pfizer-BioNTech COVID-19 vaccine (COMIRNATY) — was assessed for its cancer vaccine development potential. The research directly compared Acuitas’ LNP to lipoplexes and evaluated modified mRNA against the unmodified mRNA predominantly used in current cancer vaccine trials. Key highlights of this data include:

Using LNP comprised of ALC-315, unmodified RNA induced a stronger antigen-specific CD8 T-cell response compared to a nucleoside-modified mRNA incorporating N1methylpseudouridine-encoding OVA payload as a model tumour antigen, while maintaining strong immunogenicity at one-tenth of the initially tested dose.
Using LNP comprised of ALC-315, mRNA delivered intramuscularly induced equal or superior cellular and humoral immunity compared to intravenously (IV) administered mRNA lipoplexes – an alternative mRNA cancer vaccine format in clinical development – despite mRNA lipoplexes being administered at four-fold higher doses and with more boosts.
Several potent novel proprietary lipids were identified that achieved equivalent cellular responses to ALC-315. Further assessment will be conducted to compare potency and activity using syngeneic neoantigen models.
Novel Lipids with Improved Activity for Prophylactic Vaccine Development

Acuitas identified and validated six novel lipids that induce higher virus-specific immunogenicity compared to ALC-315. Key findings related to these novel lipids include:

The six novel lipid candidates induced equivalent neutralizing antibody titres at a five-fold lower dose than ALC-315.
The lipid candidates demonstrated favorable reactogenicity profiles comparable to ALC-315, while eliciting stronger cellular- and B-cell responses.
Innate immune responses induced by LNP correlated with their reactogenicity, but not with adaptive and innate immune responses.
Several lipid candidates achieved higher in vivo expression in secondary lymphoid organs and reduced liver expression compared to ALC-315.
Impact of Body Weight and Medications on mRNA-LNP Safety in Monkeys

As the industry continues using larger nonhuman primates in translational work, Acuitas sought to understand how body weight, premedications (steroid, H1 and H2 blockers), and concomitant medications (meloxicam) impact LNP activity and tolerability. The study was conducted in monkeys using an IV-administered mRNA-LNP encoding human IgG. Key highlights of this data include:

LNP tolerability is reduced in larger monkeys (>6 kg).
Premedications helped reduce the elevation of liver transaminases.
Premedications improved tolerability but reduced the level of IgG mRNA expression.
Platelet count decreases were greatest in large monkeys and monkeys given meloxicam.
More information on posters presented at the mRNA Health Conference and Vaccine R&D Conference can be found here.

(Press release, Acuitas Therapeutics, NOV 17, 2025, View Source [SID1234660039])