2023 Antibody-Drug Conjugates Predictions
We surveyed 41 stakeholders across the ADC space on what lies ahead in 2023.
Highlights & Summary
Intro
February was a month full of new partnerships, with companies teaming up across the field of next-generation therapeutics. Other key announcements include clinical trial updates, regulatory approvals, and new financing deals.
Happy reading!
Cell Therapy
- Sangamo joins others in shelving sickle cell disease plans for now | Clinical Trial
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Gene Therapy
- REGENXBIO’s new internal manufacturing platform providing consistent clinical results | Manufacturing
- Capsida announces expanded partnership with AbbVie | Partnership
- PackGene breaks ground on new production facility | Manufacturing
Oligo Therapy
- Alnylam announces FDA acceptance for ONPATTRO, a treatment for cardiomyopathy for ATTR amyloidosis | Regulatory
- Moderna and Life Edit Therapeutics announce a strategic collaboration to develop mRNA therapies | Partnership
Antibody-Drug Conjugates
- Corbus Pharmaceuticals expands oncology pipeline with the addition of a clinical stage Nectin-4 targeting ADC | Partnership
- ARX517, Ambrx’s Proprietary Anti-PSMA ADC, Shows Encouraging Data in Prostate Cancer Patients | Clinical Trial
- CKD Pharm Licenses Synaffix ADC Technology, Adds Novel ADC Candidate to Oncology Pipeline | Partnership
Cell Therapy
Replay and MD Anderson form new product company Syena to develop TCR-NK cell therapy | Partnership
Replay, a genome writing company, and MD Anderson have co-founded Syena to develop a cord blood-derived T-Cell Receptor Natural Killer (TCR-NK) platform that offers potential for scalable off-the-shelf cell therapy. The platform is based on discoveries of Katy Rezvani, M.D., Ph.D.,, a professor at MD Anderson, and Replay. The promise lies in combining the safety, potency, and scalability of NK cells with the ability of TCRs to target intracellular tumor antigens.
Christi Shaw joined Gilead Sciences' CAR-T-focused Kite Pharma in 2019, when the company had one approved cell therapy, and total cell therapy revenues at around $450M. Now, after more than three years, Shaw is leaving a unit that's responsible for about $1.5 billion in annual revenues and more than 13,000 patients treated.
The Cambridge-based biotech is developing proprietary hematopoietic stem cells that could be the industry’s first off-the-shelf offering. Garuda launched 15 months ago with $72 million in hand and the potential to use its cellular therapy to address and potentially cure over 70 diseases. The team has since identified 50 more targets ripe for stem cell treatment. The Series B funds will funnel into two programs slated to hit the clinic in 2024 - transfusion-dependent beta-thalassemia (TDBT) and bone marrow failure syndrome.
Lineage Cell Therapeutics, a biotech developing allogeneic cell therapies to replace and restore specific cell types of the human body, announced that it has entered into an exclusive agreement with Eterna Therapeutics for the development of novel beta 2 microglobulin (B2M)-deficient iPSC lines, which Lineage will evaluate for development into differentiated cell transplant therapies. The new cell lines to be developed by Eterna will support the potential creation of additional product candidates at Lineage, specifically for the treatment of certain central nervous system (CNS) disorders and other neurology indications.
Sangamo joins others in shelving sickle cell disease plans for now | Clinical Trial
Sickle cell disease (SCD) has been a hot area for cell therapy investment until recently when Sangamo announced that it is stopping its SCD asset development within the same 24-hour window that Novartis announced the end of its partnership with Intelia, a CRISPR company, and GraphiteBio shelved its SCD drug. As for Sanganome, the company said that it will funnel its SCD resources toward its Fabry disease program and autologous renal transplant asset.
Gene Therapy
4D Molecular announces clearance of IND for diabetic macular edema trial | Clinical Trial
The company announced that the FDA has cleared the IND for its investigational drug 4D-150, a vector-based intravitreal genetic medicine for the treatment of DME. The phase 2 SPECTRA clinical trial enrollment is expected to initiate in Q3 2023. The company is also testing this asset in a Ph1/2 trial for wet age-related macular degeneration, which dosed its first patient last year.
REGENXBIO’s new internal manufacturing platform providing consistent clinical results | Manufacturing
The company presented results from the phase 2 trial showing that RGX-314, delivered through a suprachoroidal injection, could be an efficacious treatment for diabetic retinopathy. Data also show that using the company’s proprietary manufacturing platform, NAVXpress, displayed a similar clinical profile to the initial adherent cell culture process.
Capsida announces expanded partnership with AbbVie | Partnership
This new announcement between the two companies is an expansion of the 2021 partnership originally focused on CNS diseases. This expansion broadens the focus to eye diseases, as well. This comes with additional upfront payments to Capsida. Just last month, the company announced a strategic collaboration with Prevail Therapeutics (Eli Lilly), adding to the number of large biopharma partners the company has.
Plasmid cell transfection for AAV production induces inflammatory response | Manufacturing
A new study published in Molecular Therapy showed the upregulation of inflammatory and antiviral responses in HEK293 cell lines after transient plasmid transfection to produce AAVs. The authors used RNA-seq to define upregulated pathways, including upregulation of interferon-related cytokine production. This could have implications for manufacturing practices, given the ubiquity of HEK cell lines for AAV production. Suppressing these pathways and the cellular response to the manufactured AAVs may allow for higher production titers and less costly manufacturing.
PackGene breaks ground on new production facility | Manufacturing
PackGene has started construction on its new AAV production facility in Houston, Texas. The planned 25,000 sq.ft. facility will enable both development activities as well as large-volume production. This new facility will operate 500L bioreactors in parallel capable of producing ~1017 viral genomes.
Oligo
Moderna and Merck announced that mRNA-4157/V940, an mRNA cancer vaccine, has been granted Breakthrough Therapy Designation in combination with KEYTRUDA. This designation is based on the promising Phase 2b trial, and is intended to accelerate the development and review of drugs that may be able to treat a severe condition.
Alnylam announces FDA acceptance for ONPATTRO, a treatment for cardiomyopathy of ATTR amyloidosis | Regulatory
Alnylam Pharmaceuticals announced that the FDA has accepted for filing the supplemental New Drug Application for patisiran. Patisiran is an RNAi treatment for cardiomyopathy that is associated with transthyretin-mediated (ATTR) amyloidosis.
Aera Therapeutics launches with $193 million in combined Series A and B financing | Financing
Aera Therapeutics announced its launch with $193 million combined Series A and B financing. Aera Therapeutics has a proprietary delivery platform based on endogenous human proteins. The platform is based on research conducted in the lab of Feng Zhang, Ph.D. of the Broad Institute of MIT and Harvard.
Moderna and Life Edit Therapeutics announce a strategic collaboration to develop mRNA therapies | Partnership
Moderna and Life Edit Therapeutics are starting a collaboration to focus on developing gene editing therapies. This strategic partnership will leverage Life Edit’s gene editing technology which includes a diverse collection of novel RNA-guided nucleases. The collaboration with Moderna will be used to develop novel approaches to target rare genetic diseases.
ADCs
AstraZeneca enters license agreement with KYM Biosciences for CMG901 | Partnership
AstraZeneca and KYM Biosciences have signed an exclusive agreement for a potential first-in-class anti-Claudin18.2 ADC, CMG901. CLDN18.2 is a very hot target in multiple solid tumors, including gastric cancer. AstraZeneca will be responsible for the R&D, manufacturing, and global commercialization of CMG90. According to DeciBio’s Theratrack, there are currently ~12 unique ADCs targeting CLDN18.2 in development.
Pfizer in Talks to Acquire Seagen in Deal Likely Valued at More Than $30 Billion | M&A
Seagen is in talks with Pfizer to be acquired at >$30B, $10B less than the failed talks with Merck. The ADC leader has 3 approved ADCs, and according to DeciBio's TheraTrack, the company has ~11 unique ADCs currently in development. Pfizer projects to lose ~$17B due to patent expirations by 2030, and is working to add significant revenue from BD moves, including acquisitions.
Corbus Pharmaceuticals expands oncology pipeline with the addition of a clinical stage Nectin-4 targeting ADC | Partnership
The company announced that it is expanding its oncology pipeline by entering a licensing agreement with CSPC Megalith Biopharmaceutical, for the development and commercialization of CRB-701 (SYS6002), a novel clinical-stage Nectin-4 targeting ADC. The company is planning to initiate a phase 1 trial in the U.S. in 2024. Padcev is the only nectin-4 targeting ADC on the market, approved for the treatment of bladder cancer.
ARX517, Ambrx’s Proprietary Anti-PSMA ADC, Shows Encouraging Data in Prostate Cancer Patients | Clinical Trial
Preliminary data showed a decrease in PSA across patients enrolled in the dose-escalation trial. While ARX517 is the only anti-PSMA ADC in active clinical trials, 4 companies are developing PSMA-targeting antibody drug conjugates at different stages of development and conjugated to distinct payloads.
CKD Pharm Licenses Synaffix ADC Technology, Adds Novel ADC Candidate to Oncology Pipeline | Partnership
In its 3rd deal of the year, Synaffix is licensing out its proprietary ADC technologies and an undisclosed linker payload. The agreement consists of a single-target access with upfront and milestone payments, plus royalties. CKD will be responsible for the R&D, manufacturing, and commercialization of the ADC. Synaffix will be responsible for the manufacturing of components that are specifically related to its proprietary technologies.
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