Highlights & Summary
October was a busy month for the next-generation therapeutics field, full of new partnerships, manufacturing announcements and clinical trial updates.
- Cellares Expands Agreement with BMS to Include Second CAR-T Program in Cellares’ Technology Adoption Partnership Program | Partnership
- Beam Therapeutics Reduces Headcount by 20%, and shifts focus on four key programs in sickle cell and AATD, among other | Personnel
- Driven by High Demand, Johnson & Johnson States Plans to Further Ramp Up Manufacturing Capacity for CAR-T Carvykti | Manufacturing
- Bayer Is Stepping Into the Cell Therapy Space By Opening Its First Dedicated Manufacturing Facility in California for $250M | Manufacturing
- Verve Therapeutics Announces Clearance to Start Trials for their Investigational New Drug | Regulatory
- New mRNA Vaccine for Pancreatic Cancer Starts Next Phase | Clinical Trial
- BioNTech Announces $70M Deal with MediLink Therapeutics | Partnership
- Hummingbird Bioscience and Endeavor BioMedicines Enter a License Agreement for HMBD-501 | Partnership
Only six weeks after entering into a partnership with BMS, Cellares is already expanding the deal. BMS will employ Cellares’ Cell Shuttle platform for the automated proof-of-concept manufacturing of a second CAR-T cell therapy. The Cell Shuttle, which is roughly the same size as a small van, combines all cell therapy manufacturing processes in a single unit, and promises to produce the same number of cell therapy batches with 90% less labor and facility space than conventional contract development and manufacturing organizations.
Beam Therapeutics is streamlining its focus by concentrating on four primary base editing programs and laying off around 100 employees, or 20% of its workforce. The company is prioritizing treatments for sickle cell disease (both ex vivo and in vivo), alpha-1 antitrypsin deficiency (AATD), and the ESCAPE program for bone marrow transplant conditioning. They are scaling back on other projects, including the BEAM-201 CAR-T therapy for T-ALL, and halting their hepatitis B research. These changes are aimed at extending Beam’s financial runway into 2026.
Johnson & Johnson is boosting global production of Carvykti, their CAR-T therapy with Legend Biotech, due to high demand, executives announced during Q3 earnings call. Increased capacity is mainly to come from sites in New Jersey, Belgium, Switzerland, and excess capacity from Novartis. A new factory in the Netherlands will open in 2025 to support critical lentivirus production. Despite challenges, including halting its UK launch, Carvykti sales reached $152 million in Q3, with J&J optimistic about future growth and an upcoming FDA decision on its use for multiple myeloma.
CARGO Therapeutics, a CAR-T cell therapy company, is heading to Nasdaq with the symbol CRGX. Formed by Stanford's CAR-T leader Crystal Mackall, CARGO launched nine months ago with $200 million and quickly progressed to a Phase I trial. The company's IPO aims to fund Phase II studies of its CD22-targeted CAR-T therapy. This move makes CARGO the first CAR-T therapy startup to pursue an IPO in 2023, a notable step in a challenging market which has seen several other biotech IPOs falter this year.
Bayer’s new site will support later-stage clinical trials and potentially commercial launches, starting with BlueRock Therapeutics' Parkinson’s disease candidate, bemdaneprocel, set for Phase II trials in early 2024. The facility, spanning 100,000 square feet, boasts advanced capabilities like cell culture and automated filling. Bayer’s cell and gene therapy efforts, consolidated since December 2020, include seven projects, with bemdaneprocel as the flagship. This expansion at the Berkeley campus, where Bayer has invested nearly $500 million over five years, reflects its commitment to cell and gene therapy, propelled by its acquisitions of BlueRock in 2019 and AskBio in 2020.
Regeneron Pharmaceuticals, Inc. and Intellia Therapeutics, Inc. have expanded their partnership to include additional CRISPR-based gene editing therapies, focusing on neurological and muscular diseases. Their initial partnership centered on the use of CRISPR for in vivo use and was subsequently expanded in 2020 to include programs to treat hemophilia A and B. This new agreement will leverage Regeneron's antibody-targeted AAV for delivery with Intellia's Nme2 CRISPR/Cas9 systems adapted for viral vector delivery for gene editing. The expanded agreement involves researching two initial in vivo non-liver targets, with each company leading the design of a specific approach, offering the opportunity for joint development and commercialization.
Freeline Therapeutics, who over time has narrowed their pipeline focus to their clinical program for Gaucher disease, announced having received an unsolicited takeover bid from Syncona. Syncona was an original investor in Freeline and still owns a majority of the company. Freeline also recently announced results from the first two patients dosed in their GALILEO-1 trial, which showed a normalization of cellular GCase levels.
Fractyl Health's presentation at the EASD 2023 Annual Meeting introduced the potential of their pancreatic gene therapy candidate (GLP-1 PGTx) for type 2 diabetes (T2D) and obesity, revealing findings that indicated a 24.8% weight loss in a murine obesity model after a single dose of an AAV-based gene therapy delivering a gene for GLP-1 compared to chronic semaglutide's 18.4% weight loss. The study suggests sustained weight loss potential post-single administration. Fractyl Health aims to progress the program through lead optimization and toxicity studies in 2024.
uniQure is undergoing a significant reorganization to cut expenses and will discontinue more than half of its research and technology projects. The firm plans to let go approximately 20% of staff, or 114 positions. They'll focus on high-priority programs like AMT-130 for Huntington's disease and AMT-161 for ALS, the former of which is presently in a Ph1/2 trial. Both utilize miRNA technology to silence their respective targets. The company is also continuing to develop two additional programs aimed at refractory mesial temporal lobe epilepsy and Fabry disease. This reorganization also sees the departure of CSO Ricardo Dolmetsch, with Richard Porter taking over those responsibilities. The restructuring should extend the company runway to the second quarter of 2027.
SpliceBio has entered into a collaboration with Spark Therapeutics to develop a gene therapy targeting an undisclosed inherited retinal disease. The partnership will utilize SpliceBio's Protein Splicing platform, offering a solution for diseases where the necessary gene is too large for current gene therapy delivery methods. Spark gains exclusive rights to develop, manufacture, and commercialize the resulting therapy, providing SpliceBio with upfront and milestone payments of up to $216 million plus potential royalties on net sales. This collaboration aims to address unmet medical needs in inherited retinal diseases, complementing Spark's expertise in gene therapies for these conditions.
Verve Therapeutics has received clearance from the FDA to conduct a clinical trial for VERVE-101, an innovative gene-editing medicine designed to treat heterozygous familial hypercholesterolemia (HeFH). VERVE-101 targets the PCSK9 gene in the liver to reduce LDL cholesterol levels. The company plans to present interim findings from the ongoing heart-1 Phase 1b trial at the American Heart Association’s Scientific Session.
MilliporeSigma, the U.S. and Canada Life Science division of Merck KGaA, Darmstadt, Germany, has inaugurated two state-of-the-art facilities in Darmstadt and Hamburg, Germany, dedicated to manufacturing GMP-grade mRNA drug substances. The €28 million investment in these sites is part of the company's ongoing €1 billion initiative to advance mRNA technologies and fortify its global network and capabilities.
Scientists Katalin Karikó and Drew Weissman from the University of Pennsylvania have been honored with the 2023 Nobel Prize in Physiology or Medicine for their pioneering work on messenger RNA (mRNA). The research, initiated in the late 1990s, unveiled the potential of modifying and effectively delivering mRNA into the body to activate the immune system. This pivotal discovery laid the foundation for the development of mRNA-based vaccines.
New mRNA Vaccine for Pancreatic Cancer Starts Next Phase | Clinical Trial
A phase 2 clinical trial led by Memorial Sloan Kettering Cancer Center is underway to assess the effectiveness of a personalized mRNA vaccine in treating pancreatic cancer. This innovative approach aims to reduce the risk of cancer recurrence following surgery. The mRNA vaccine utilizes neoantigens from pancreatic tumors to activate the immune system, training it to recognize and combat cancer cells.
Eli Lilly and Company is continuing its acquisition spree this year. The move signifies Lilly's continued investment in the ADC sector, building on its previous acquisition of Emergence Therapeutics. The company is set to solidify its position in the competitive cancer modality space, particularly with Mablink Bioscience's PSARLink technology and its upcoming Phase I asset, MBK-103, which targets folate receptor alpha.
The combination exhibited a stunning 53% reduction in the risk of death, leading to a significant increase in median overall survival (OS) compared to chemotherapy in bladder cancer. Furthermore, it demonstrated a substantial 55% reduction in the risk of disease progression or death and a notably higher objective response rate (67.7% versus 44.4% in the chemo arm). These findings emphasize the potential for a revolutionary shift in the standard of care for bladder cancer patients.
Merck has embarked on a significant partnership with Daiichi Sankyo, committing a hefty $4 billion upfront to collaborate on three ADCs targeting HER3, B7-H3, and CDH6. This latest ADC partnership with Daiichi Sankyo exemplifies Merck's dedication to amplifying its pipeline and oncology portfolio, further solidifying its presence in the ADC market.
GSK has struck a deal with Hansoh Pharma, securing global rights to HS-20089, an ADC targeting B7-H4 in clinical trials for gynecologic cancers in China. GSK will pay an initial sum of $85 million, with potential milestone payments reaching $1.4 billion, along with royalties upon commercialization. GSK intends to commence phase 1 trials outside of China in 2024, aiming to enhance its range of treatments for gynecologic cancers.
BioNTech Announces $70M Deal with MediLink Therapeutics | Partnership
Following its recent agreements with DualityBio, BioNTech has now partnered with MediLink Therapeutics which granted the company exclusive global rights for one of its anti-HER2 ADCs. This agreement involves an upfront payment of $70 million and potential milestone payments exceeding $1 billion. HER3 is the target of interest for multiple pharmaceutical companies. DeciBio’s TheraTrack estimates that there are more than 20 HER3-targeting ADCs in development across clinical stages.
Endeavor BioMedicines has acquired exclusive global rights to Hummingbird Bioscience's HMBD-501, an advanced anti-HER3 ADC. HMBD-501 is conjugated to topoisomerase I inhibitor at a DAR of 4, and is designed to have a broader therapeutic index, binds to a unique HER3 receptor epitope, independent of NRG1 levels.