Highlights & Summary
Overall, companies have reported mixed updates throughout November - while AstraZeneca acquired Neogene to enter the Cell Therapy space, GSK continued reducing their Cell Therapy pipeline. Similarly CSL’s approval for its Hemophilia B drug was followed by GSK’s Blenrep withdrawal from the U.S. market.
- Abeona Plans a BLA for EB-101 After Positive Phase 3 Trial Data | Clinical Trial
- Artiva and Affimed Look to Combine an Allogeneic NK Cell Therapy and an Innate Cell Engager for Improved Cell Therapy Outcomes | Partnership
- Adaptimmune Joins a Growing List of Restructuring Companies as They Announce Layoffs of ~30% | Commercial
- CSL Behring’s Gene Therapy for Hemophilia B Approved | Regulatory
- iECURE Nets an Additional $65M in Their Series A | Financing
- Editas Tables Lead Asset after Disappointing Readout | Clinical Trial
- BMS Ends Collaboration with uniQure | Partnership
- RZNOMICS Inc. Starts Clinical Development of Trans-splicing Ribozyme-based RNA Editing Technology Following FDA Approval | Clinical Trial
- Ionis Announces Positive Results from Fesomersen Phase 2B program | Clinical Trial
- GSK Pulls Blenrep from U.S. Market | Commercial
- NextCure and LegoChem Biosciences Enter into Collaboration and Co-Development Agreement | Partnership
- Orum Therapeutics Announces First Patient Dosed with ORM-5029 | Clinical Trial
- ADC Therapeutics Will Not Seek Accelerated Approval for Cami in Hodgkin Lymphoma due to FDA Pushback | Regulatory
AstraZeneca has been the odd one out on cell therapy assets as most other major players have a larger number of assets in development. This acquisition is also in stark contrast as many companies have been reducing their cell therapy pipelines.
This is not the first time GSK has reduced their cell therapy pipeline recently as a saturation in the field has started to produce material effects on businesses.
The application, if approved, would bring cell therapies into new spaces by targeting recessive dystrophic epidermolysis bullosa. Abeona announced they plan to file imminently given the results of their Phase 3 VIITAL trial.
The combination therapy would join a growing trend of combining cell therapies with small molecule or biologic adjuvant therapies (most commonly immunotherapies) to improve efficacy and could have a significant impact on how allogeneic therapies are moved into the clinic.
Combining poor macroeconomic conditions and a saturated cell therapy pipeline has resulted in consistent downsizing and halting asset development as multiple companies have been hit by the difficulties in the space.
CSL Behring’s Gene Therapy for Hemophilia B Approved | Regulatory
CSL Behring announced FDA approval of HEMGENIX, their treatment for Hemophilia B. Originally developed by uniQure, CSL Behring acquired the asset in mid 2020. At launch, the drug will be the most expensive therapy in the world with a list price of $3.5M per dose.
iECURE Nets an Additional $65M in their Series A | Financing
iECURE added to its total Series A haul, originally announced in the fall of 2021. Co-led by Novo Holdings A/S and LYFE Capital, the raise should allow for their investigational product for Ornithine Transcarbamylase to enter the clinic and have readout from a planned Phase I/II trial. Co-founded by Professor James Wilson, the company collaborates closely with the University of Pennsylvania’s Gene Therapy Program.
Editas Tables Lead Asset after Disappointing Readout | Clinical Trial
Editas announced proof-of-concept results for EDIT-101, which saw only 3 of fourteen patients respond to the therapy designed to treat Leber Congenital Amaurosis. The company will now deprioritize the program and look to find a collaboration partner for further development.
BMS Ends Collaboration with uniQure | Partnership
BMS has ended their pact with uniQure that was centered around therapies for congestive heart failure. Penned in 2015, BMS did not give a reason for termination. All of the early-stage assets will continue to be owned by BMS.
Kriya, which has built out a holistic organization with internal manufacturing capabilities, has acquired Redpin and their pipeline of neurology-focused therapies. Redpin has built the capability to selectively activate or silence neurons in a controllable fashion.
As per the agreement between Arcturus and CSL Seqirus, Arcturus will license STARRTM, their proprietary mRNA technology for SARS-CoV-2 vaccine development and other infectious diseases. Arcturus will receive $200 million and will be eligible for additional compensation based on clinical development.
Strand added an additional $45M to their Series A financing with key players participating like Eli Lilly and Playground Global. Strand has a novel platform for programmable mRNA therapeutics and will be starting a Phase 1 clinical trial next year for solid tumor indications.
Rznomics Inc., has received Phase 1/2a approval to start evaluation of RZ-001 for hepatocellular carcinoma. RZ-001 is a gene therapy that utilizes trans-splicing ribozyme-based RNA programming. This approach allows for simultaneous suppression of RNA and expression of therapeutic RNA without any additional proteins or cofactors.
Ionis Announces Positive Results from Fesomersen Phase 2B program | Clinical Trial
Ionis announced positive results for fesomersen for efficacy in reducing Factor XI production in patients with end-stage renal disease on hemodialysis. Fesomersen is an antisense therapy that reduces a primary clotting factor that can increase the risk for thrombosis.
GSK Pulls Blenrep from U.S. Market | Commercial
GSK announced that it will be withdrawing Blenrep from the U.S. market for the treatment of adult patients with R/R multiple myeloma. The decision is based on the DREAMM-3 phase III confirmatory trial, which failed to meet its primary endpoint of PFS. Already-enrolled patients will be able to access the treatment as part of a compassionate use program.
Immunogen received an accelerated approval for ELAHERE™ for the treatment of FRα positive and platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. FRα is expressed in more than ~80% of recurrent ovarian tumors and is associated with poorly differentiated and aggressive tumors. The drug also known as mirvetuximab soravtansine-gynx, is a first-in-class ADC comprising a FRα-binding antibody, cleavable linker, and the maytansinoid payload DM4.
NextCure announced an agreement with LegoChem focused on developing a B7-H4 ADC. The partnership will be based on NextCure’s NC762 and combined with LegoChem’s proprietary conjugation technology (ConjuAIITM). Based on DeciBio’s TheraTrack, there are currently ~5 companies developing ADCs targeting B7-H4 in advanced or metastatic solid tumors.
Orum Therapeutics Announces First Patient Dosed with ORM-5029 | Clinical Trial
Orum Therapeutics has dosed the first patient with its lead asset ORM-5029, in a phase 1 trial for patients with HER2-expressing advanced breast cancer. ORM-5029 is a first-in-class molecule that uses Orum’s GSPT1 platform to deliver catalytic GSPT1 protein degraders to HER2+ tumor cells.
Due to the new strict FDA policy regarding accelerated approval submissions, ADC Therapeutics dropped its plans to file a BLA for Cami next year and paused all investments in its Hodgkin Lymphoma program, but will potentially pursue a partnership to move Cami forward. The FDA announced earlier this month that confirmatory trials need to be “well underway and ideally fully enrolled”, before granting accelerated approvals, which could usually take at least 2 years.