Highlights & Summary
Intro
July was a busy month for the next-generation therapeutics field, full of new acquisitions, regulatory updates, and partnership announcements. Other key developments include a number of clinical trial and pipeline updates.
Happy reading!
Cell Therapy
- Kincell Bio Launches to Add More Capacity for Cell Therapy Development and Manufacturing | Manufacturing
- Replay and MD Anderson announce FDA clearance of IND application for first-in-class TCR-NK for multiple myeloma | Regulatory
Gene Therapy
- Astellas Partners with 4DMT on Rare Ophthalmic Targets | Partnership
- LEXEO has IND Approved for PKP2 Cardiomyopathy Candidate | Clinical Trial
Oligo Therapy
- MIT researchers lead a new mRNA manufacturing facility with $82M | Manufacturing
- Sanofi and BioNTech terminate mRNA cytokine candidate | Clinical Trial
- Alnylam reports positive interim Phase 1 Results for RNAi therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy | Clinical Trial
Antibody-Drug Conjugates
- BeiGene teams up with DualityBio | Partnership
- FDA Grants Fast Track Designation for Ambrx’s ARX517 for the Treatment of Prostate Cancer | Regulatory
Cell Therapy
Lilly to Acquire Sigilon Therapeutics for up to $300M | M&A
Lilly and Sigilon have been working together for the last five years to develop encapsulated cell therapies, (e.g. SIG-002) for the treatment of type 1 diabetes, which are meant to free patients from constant disease management by sensing blood glucose levels, restoring insulin production and releasing it over the long term. Lilly’s acquisition will begin by purchasing all outstanding shares by cash (nearly $34.6 million) payable at closing, plus one non-tradeable contingent value right ("CVR") per share which will result in the deal being valued at $309.6 million excluding shares held by Lilly.
Pfizer Decided to Back Caribou Biosciences with a $25M Investment | Financing
The investment deal involved Pfizer purchasing 4.6 million shares of Caribou common stock. The proceeds of the deal will go directly into Caribou’s allogeneic CAR-T cell therapy for multiple myeloma CB-011, currently in a phase 1 clinical trial. Pfizer is encouraged by Caribou’s CRISPR hybrid RNA-DNA guides gene editing tech—dubbed chRDNAs—and the potential Caribou’s therapies have as off-the-shelf cancer treatments.
Tenpoint Therapeutics is pursuing vision-restoring engineered cell-based therapeutics and in vivo reprogramming to address degenerative ocular disease that are both inherited and age-related. Tenpoint’s platform is based on the work of its scientific founders from Moorfields Eye Hospital, University College London Institute of Ophthalmology, Institut de la Vision in Paris and the University of Washington.
Kincell Bio Launches to Add More Capacity for Cell Therapy Development and Manufacturing | Manufacturing
With $36M in funding Kincell Bio emerges as a new CDMO player looking to satisfy the growing need for cell therapy manufacturing. Kincell is a spinout of Inceptor Bio formed to accelerate innovator research and clinical trials. Kincell acquired a facility and a fully staffed team based at its Gainesville, Florida site, and will be focusing on immune cell therapies, including autologous and allogeneic CAR-T, CAR-NK and CAR-M programs.
Replay and MD Anderson announce FDA clearance of IND application for first-in-class TCR-NK for multiple myeloma | Regulatory
Replay and MD Anderson were granted a “safe to proceed” for the IND application for NY-ESO-1 TCR/IL-15 NK, a first-in-class engineered T-Cell Receptor Natural Killer (TCR-NK) cell therapy. NY-ESO-1 is being developed by Syena, a company launched by Replay and MD Anderson that has an exclusive licensing agreement for MD Anderson’s TCR-NK platform.
Gene Therapy
Astellas Partners with 4DMT on Rare Ophthalmic Targets | Partnership
Astellas entered into a strategic license to gain access to 4DMT’s retinotropic R100 vector for a single indication. Originally discovered by 4DMT, the vector efficiently transduces the entire retina. Under the agreement, Astellas will perform all research on the intended target. 4DMT received $20M upfront with future option fees and milestones of < $942.5M.
Capsida Cuts Workforce as Internal Programs Advance | Personnel
Despite multiple partnerships with the likes of AbbVie and Lilly, Capsida confirmed they are cutting personnel to focus on advancing internal candidates. The company previously inked and expanded multiple partnerships with biopharma and focuses on screening for novel serotypes. Capsida’s one publicly-announced internal program is for genetic epilepsy caused by mutations of syntaxin-binding protein 1.
Roche cuts Two Hemophilia A Programs from Spark Pipeline | Pipeline
Having previously cut a candidate for hemophilia A from Spark’s pipeline, Roche has now cut a second asset for the disease. This comes shortly after BioMarin received approval for their AAV5-based therapy for the disease. Their remaining asset in the space, SPK-8011, just started Ph3 testing. Hemophilia remains a crowded space with the two approved therapies from CSL Behring and BioMarin, and a host of others with clinical-stage assets.
AstraZeneca’s Rare Disease Unit Acquires Preclinical Assets from Pfizer | M&A
AstraZeneca is bolstering their gene therapy unit with the acquisition of preclinical assets from Pfizer. The deal will also see Pfizer employees move over to AZ. The deal has a total value up to $1B plus tiered royalties on any potential future products.
LEXEO has IND Approved for PKP2 Cardiomyopathy Candidate | Clinical Trial
LEXEO had their IND cleared by FDA for LX2020 for the treatment of Arrhythmogenic Cardiomyopathy. This would be the first asset for this indication to reach clinical trials, ahead of Tenaya’s competing TN-401. The trial will look for protein expression, cardiac function, and arrhythmia burden.
Oligo
MIT researchers lead a new mRNA manufacturing facility in an $82M effort | Manufacturing
Faculty at MIT are leading a three year research program to design an integrated mRNA manufacturing platform. The U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and research funded an $82 million effort in order to streamline response to pandemics as well as scale up vaccine production across disease indications.
Roche partners with Alnylam to development RNAi therapeutic to treat hypertension | Partnership
Roche and Alnylam partner to further develop a Phase 2 RNAi therapeutic. Phase 1 data on zilebesiran demonstrated a transformational benefit for patients with hypertension who are at increased risk for cardiovascular disease. This collaboration will leverage Roche’s global reach and Alnylam’s experience with RNA based therapeutics.
Novartis acquires DTx Pharma | M&A
DTx Pharma is a preclinical biotechnology company that has a Fatty Acid Ligand Conjugated Oligo Nucleotide (FALCON) platform. This platform is positioned to help improve delivery of oligonucleotide therapeutics to multiple tissue types with improved targeting and delivery. Novartis will be making an upfront payment of $500M with additional payments on agreed upon milestones.
Sanofi and BioNTech terminate mRNA cytokine candidate | Clinical Trial
Sanofi and BioNTech terminate their clinical program on their intratumoral candidate, SAR441000, a therapeutic made of four mRNAs that encode for different cytokines. The target of the program was to express cytokines in the tumor microenvironment. Sanofi is continuing to prioritize other therapeutic candidates.
Alnylam reports positive interim Phase 1 Results for RNAi therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy | Clinical Trial
Alnylam reports promising interim results from a Phase 1 study of ALN-APP, an RNAi therapeutic targeting amyloid precursor protein. This is the first clinical study of a central nervous system administered RNAi therapeutic that showed a sustained response over 6 months with just one dose. Next steps will include a multiple-dose to learn more about the therapeutic potential.
ADCs
Eli Lilly to Buy Emergence Therapeutics | M&A
Eli Lilly is expanding its oncology business through the acquisition of the European startup Emergence Therapeutics, adding a promising ADC candidate, ETx-22, to its portfolio. ETx-22 targets Nectin-4, a hot target for ADCs, especially following the approval of Astellas' Padcev. According to DeciBio’s TheraTrack, there are more than 10 anti-Nectin-4 ADCs in development.
BeiGene teams up with DualityBio | Partnership
The two companies have announced a partnership for an investigational, preclinical ADC for the treatment of solid tumors. The agreement grants BeiGene an exclusive option for a global clinical and commercial license to the therapy, with potential payments up to $1.3 billion and tiered royalties for DualityBio.
Adcentrx Therapeutics received IND Clearance for its ADRX-0706 | Regulatory
The company has received IND clearance for ADRX-0706, a novel ADC targeting Nectin-4 in advanced solid tumors. A phase 1a/1b clinical trial is expected to start in the second half of 2023. Adcentrx has multiple ADC programs in development, including one in immunology.
Mersana Therapeutics Reduces Workforce Following Lead Asset Failure | Pipeline
Mersana Therapeutics plunges 78%, lays off 50% of its workforce, following lead ADC asset failure in ovarian #cancer. UpRi is an anti-NaPi2b ADC with a high DAR (12 to 15). The company is still pursuing the development of 4 novel ADCs using its Dolasynthen and Immunosynthen platforms.
FDA Grants Fast Track Designation for Ambrx’s ARX517 for the Treatment of Prostate Cancer | Regulatory
Ambrx received the designation for ARX517, an anti-PSMA ADC displaying highly specific tumor cell killing with minimal off-target toxicity. ARX517 is currently in phase 1 of development. According to TheraTrack, there are currently 10 unique ADCs in development for the treatment of prostate cancer, 6 of which target PSMA
.png)
.png)
