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DeciBio’s 2024 Advanced Therapies Sentiment Survey
We are excited to share the results of our Cell and Gene Therapy 2024 Industry Outlook survey! Based on stakeholder responses across cell therapy, AAV gene therapy, and oligos and mRNA, we highlight the top trends and expectations for what 2024 has in store for advanced therapies. Check it out here!
DeciBio’s 2024 ADC Industry Predictions Survey
Our latest 2024 ADC industry survey, with perspectives from industry leaders such as Merck, AstraZeneca, and Pfizer, paints a positive outlook for the field. Check it out here!
Highlights & Summary
Intro
February was an exciting month for the next-generation therapeutics space, full of new partnerships and clinical trial updates.
Cell Therapy
- BeiGene Ends Pact with Shoreline | Partnership
- Iovance's Tumor-infiltrating Lymphocytes Cell Therapy for Melanoma Receives FDA Approval | Regulatory
- Astellas Enters Deal with Kelonia Worth a Potential $800M for In Vivo Gene Delivery Tech | Partnership
- Neurona Therapeutics Raises $120M to Advance Pipeline of Cell Therapy Candidates for Neurological Disorders | Financing
Antibody-Drug Conjugates
- Firefly Bio Debuts With $94 Million Series A Financing | Company Creation
- Blenrep Nearly Triples Progression-Free Survival in Multiple Myeloma | Clinical Trial
- Samsung Biologics Partners with LegoChem | Partnership
- ProfoundBio Doses First Patient with Its Anti-PTK7 ADC | Clinical Trial
Gene Therapy
- Adverum Announces Preliminary Efficacy for Wet AMD Candidate | Clinical Trial
- 4DMT Announced Preliminary Results for 4D-150 in Wet AMD | Clinical Trial
- Biomarin Reports Modest Launch Numbers for Roctavian | Commercial
Oligo Therapy
- Recode Therapeutics Announces First Participants in a Phase 1 Study for a Cystic Fibrosis mRNA-based Therapy | Clinical Trial
- Moderna Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides Business Updates | Pipeline
Cell Therapy
BeiGene Ends Pact with Shoreline | Partnership
BeiGene has terminated its natural killer cell therapy partnership with Shoreline Biosciences, retracting from what was previously seen as its gateway into cell therapy. Despite a surge in 2023 revenues, the company cited strategic refocusing for the decision. The initial collaboration was marked by a $45 million upfront payment and aimed to combine Shoreline's cell technology with BeiGene's R&D. This shift comes amid a wider industry trend of burgeoning cell therapy ventures tackling complex diseases.
Iovance's Tumor-infiltrating Lymphocytes Cell Therapy for Melanoma Receives FDA Approval | Regulatory
After a protracted regulatory journey, Iovance Biotherapeutics has secured FDA accelerated approval for Amtagvi, the first cell therapy for advanced melanoma and the first based on tumor-infiltrating lymphocytes (TILs). Amtagvi targets a diverse array of cancer antigens, differing from the singular protein target of CAR-T therapies. Priced at $515,000, it's intended for melanoma patients who've exhausted other treatments, including checkpoint inhibitors. Despite potential severe side effects, Amtagvi demonstrated a 31.4% tumor shrinkage rate in Phase II trials. Iovance is conducting further studies to confirm efficacy and exploring TIL applications in other cancers, signaling a potential resurgence for TIL-based therapies.
Astellas Enters Deal with Kelonia Worth a Potential $800M for In Vivo Gene Delivery Tech | Partnership
Astellas has struck a deal with Kelonia Therapeutics, potentially worth nearly $800 million, to create in vivo cell therapies for cancer, featuring an innovative "convertible" CAR-T technology that can be activated or deactivated using an antibody. This collaboration aims to produce off-the-shelf treatments using Kelonia's delivery mechanisms and Xyphos Biosciences' CAR-T platform, bypassing the need for patient-specific tailoring. While still in early research stages, this approach promises to streamline the delivery of gene therapies and enhance safety by controlling CAR-T cell activity. This deal emerges amidst broader industry efforts to refine CAR-T therapies and the FDA's ongoing safety evaluations.
Kenai Therapeutics Announces $82 Million Series A | Financing
Kenai Therapeutics has raised $82 million to explore an off-the-shelf cell therapy for Parkinson's disease. Kenai's Series A funding will support the trial of RNDP-001, an induced pluripotent stem cell-derived therapy. With plans to enter Phase I testing later this year, Kenai aims to offer a scalable and accessible treatment for moderate-to-severe Parkinson's, following developments by companies like BlueRock. The company is also investigating other cell therapies for CNS disorders, leveraging partnerships and expertise in the field. Kenai operates with a small team, supplemented by Fujifilm Cellular Dynamics' manufacturing capabilities.
Neurona Therapeutics Raises $120M to Advance Pipeline of Cell Therapy Candidates for Neurological Disorders | Financing
Neurona Therapeutics has secured $120 million to complete an open-label study and advance its epilepsy cell therapy, NRTX-1001, into Phase II trials by 2025. The company, having raised a total of $285 million, is considering an IPO as its next financing step. Neurona, manufacturing its own stem cell-derived therapy in San Francisco, has reported promising early trial results with significant seizure reduction in patients. With plans to expand the trial to include bilateral disease and possibly Alzheimer's patients with seizures, Neurona is on a hiring spree, gearing up for a potential future in public markets.
ADCs
Firefly Bio Debuts With $94 Million Series A Financing | Company Creation
Firefly Bio, backed by a $94M Series A led by Versant Ventures, is pioneering degrader antibody conjugates (DACs). Founded by Nobel laureate Carolyn Bertozzi and led by CEO Scott Hirsch, Firefly aims to enhance the therapeutic index of ADCs by utilizing degraders and has shown significant reductions in tumor volume in preclinical studies. The company is set to lead in discovering clinically meaningful DACs in oncology and immunology.
Blenrep Nearly Triples Progression-Free Survival in Multiple Myeloma | Clinical Trial
GSK has announced positive results from the DREAMM-7 phase III trial, revealing that the combination of Blenrep with bortezomib and dexamethasone nearly tripled the median PFS compared to the standard daratumumab combination in patients with relapsed/refractory multiple myeloma. The trial showed a 59% reduction in the risk of disease progression or death with the Blenrep combination, along with clinically meaningful improvements in secondary efficacy endpoints. These results will be presented at ASCO.
Samsung Biologics Partners with LegoChem | Partnership
The collaboration focuses on the development and manufacturing of ADC programs, with the aim of treating solid tumors. Samsung Biologics will provide antibody development and drug substance manufacturing services as part of LegoChem Biosciences' ADC program. The collaboration is set to enhance the domestic supply chain for ADCs, and Samsung Biologics plans to support LegoChem Biosciences in submitting an IND application in the first half of 2025.
Datopotamab Deruxtecan BLA accepted in the U.S. for Lung Cancer Patients | Regulatory
If approved, Dato-DXd could be the first TROP2 ADC for lung cancer treatment. The BLA submission is based on positive results from the TROPION-Lung01 phase 3 trial, demonstrating a statistically significant improvement in PFS compared to the standard of care. The PDUFA date is set for December 20, 2024. According to DeciBio’s TheraTrack, there are currently ~30 unique anti-TROP2 ADCs in development, for the treatment of lung cancer.
Nascent Biotech and Manhattan BioSolutions To Explore Novel ADCs | Partnership
The research collaboration will explore ADCs using Nascent's lead clinical candidate, pritumumab, as the tumor-targeting antibody. Pritumumab targets vimentin, a protein linked to cancer growth, with promising safety and efficacy results. Under the partnership, Manhattan BioSolutions will perform PTB conjugations and evaluate resulting ADCs for the treatment of vimentin-positive tumors.
ProfoundBio Doses First Patient with Its Anti-PTK7 ADC | Clinical Trial
A Phase 1/2 trial was initiated for PRO1107, an anti-PTK7 ADC. PRO1107 incorporates a novel hydrophilic MMAE-based linker-drug platform, aiming for improved safety and efficacy compared to prior PTK7 ADCs. The trial is designed for patients with advanced solid tumors, including non-small cell lung, breast, and ovarian cancer. ProfoundBio aims to demonstrate the potential benefits of its LD343 platform and contribute to innovative ADCs addressing unmet medical needs in cancer treatment.
Gene Therapy
Adverum Announces Preliminary Efficacy for Wet AMD Candidate | Clinical Trial
Adverum Biotechnologies' gene therapy for wet age-related macular degeneration (AMD) showed promising results in a phase 2 study, with reduced treatment burden and maintained visual acuity. The therapy, ixo-vec, demonstrated potential best-in-class clinical activity, with no serious adverse events reported among the 60 patients in the trial. Adverum plans to share a 26-week interim analysis in mid-2024, with discussions for a phase 3 trial design and potential launch in the first half of 2025.
4DMT Announced Preliminary Results for 4D-150 in Wet AMD | Clinical Trial
4DMT similarly announced positive interim data from its Phase II PRISM clinical trial in wet AMD using 4D-150 in patients with severe disease activity and high treatment burden. Using approximately half the dose of Adverum’s trial, the results showed a slightly lower reduction rate in annualized anti-VEGF injections. 4D-150 leverages a transgene encoding for aflibercept, like Adverum’s candidate, in addition to an anti-VEGF-C RNAi. The company plans to expedite discussions with regulators for Phase 3 development. The race in wet AMD is heating up, with REGENXBIO having announced last month interim results for their wet AMD asset partnered with AbbVie.
Biomarin Reports Modest Launch Numbers for Roctavian | Commercial
BioMarin's gene therapy Roctavian faced a sluggish launch trajectory in 2023, treating only three hemophilia A patients despite European and U.S. approvals. Sales of $3.5 million fell below expectations, with challenges including patient motivation, payer support, and physician readiness. Roctavian's high price and complex pre-infusion requirements have hindered uptake, despite patient demand.
Oligo
Recode Therapeutics Announces First Participants in a Phase 1 Study for a Cystic Fibrosis mRNA-based Therapy | Clinical Trial
ReCode Therapeutics launches a Phase 1 clinical trial for RCT2100, an inhaled mRNA therapy targeting cystic fibrosis patients with Class I mutations in the CFTR gene. The trial, conducted in New Zealand, aims to assess safety and tolerability for this subset of patients who do not respond to conventional treatments. RCT2100 utilizes ReCode's novel SORT LNP delivery platform, offering potential hope for those facing profound physical and emotional challenges from the disease.
Moderna Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides Business Updates | Pipeline
Moderna shifts focus by balancing COVID-related wind-down charges with significant recent investments in cancer vaccine production. The company is expanding its Marlborough manufacturing facility to support its partnership with Merck, focusing on personalized cancer therapy, while also streamlining its manufacturing footprint for post-pandemic operations. Despite COVID-related adjustments, Moderna continues to advance its mRNA-4157 cancer vaccine alongside Keytruda in late-stage trials, eyeing expansion into additional tumor types in 2024.
Sirius Therapeutics Begins Phase 1 Trial of Next-Gen siRNA Anticoagulant | Clinical Trial
Sirius Therapeutics has initiated a Phase 1 clinical trial in Australia for SRSD107, its next-generation siRNA therapeutic targeting coagulation Factor XI (FXI) to prevent and treat thromboembolic disorders, with the first subject dosed on January 30th, 2024. The trial aims to evaluate SRSD107's safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers, building on in vivo studies demonstrating significant reduction of FXI levels for up to 6 months after a single subcutaneous dose. Dr. Qunsheng Ji, CEO of Sirius Therapeutics, anticipates this trial will assess SRSD107's potential as a differentiated therapeutic for thromboembolic disorders on a global scale.
