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Highlights & Summary
Intro
January was an exciting month for the next- generation therapeutics space, full of new financing deals, as well as a number of clinical trial and regulatory updates.
Happy reading!
Cell Therapy
- Kite Receives U.S. FDA Approval of Manufacturing Process Change for Yescarta, Reducing Turnaround Time | Manufacturing
- FDA Requests Boxed Warning for CAR-Ts Regarding Secondary Malignancy Risk | Regulatory
- Kyverna Develops Ingenui-T, a Solution for Rapid CAR-T Manufacturing | Manufacturing
Antibody-Drug Conjugates
- OnCusp Therapeutics Raises $100M Series A | Financing
- Dyne Therapeutics Announces Positive Trial Data for DYNE-101 in DM1 | Clinical Trial
- CellmAbs Announces Agreement with BioNTech | Partnership
Gene Therapy
- Canadian Authorities Approve Pfizer’s Beqvez | Regulatory
- Fractyl Health Eyes IPO as Markets Open | Financing
Oligo Therapy
- Strand Therapeutics Receives IND Clearance for Programmable mRNA Therapy STX-001 to Treat Solid Tumors | Clinical Trial
Cell Therapy
Catamaran Bio, a Boston-based biotech specializing in off-the-shelf CAR NK cell therapies, is winding down operations due to challenging financing conditions for early-stage cell therapy companies. CEO Alvin Shih announced the decision, noting the difficulty in securing the necessary resources for meaningful clinical progress. The company, launched in 2020 with $42 million funding, will now seek strategic partners for its technology and therapy candidates. Catamaran's focus was on allogeneic therapies for solid tumor indications, developing the TAILWIND platform and planning tests for therapies targeting renal cell carcinoma and breast/gastric cancers.
Kite Receives U.S. FDA Approval of Manufacturing Process Change for Yescarta, Reducing Turnaround Time | Manufacturing
Gilead and Kite have received FDA approval to reduce the manufacturing time for their cell therapy Yescarta by two days, shortening the median turnaround time from 16 to 14 days. This improvement in the manufacturing process, which includes cell collection, genetic re-engineering, and testing, is seen as an incremental but positive step. The faster process is expected to lower overall costs and increase production capacity at their U.S. facilities. However, this update applies only to Yescarta and not to their other cell therapy, Tecartus.
FDA Releases Guidance on CAR-T Cell Products, Supporting Accelerated Approvals | Regulatory
The FDA has finalized guidance for CAR-T cell products, supporting accelerated approval for genome editing therapies and clarifying recommendations on manufacturing, potency, and clinical study design. The guidance, focusing on cancer indications, also covers considerations for CAR-T cells derived from previously treated patients and may extend to other genetically modified lymphocyte products like CAR-NK cells. This move signals the FDA's commitment to facilitating the development of advanced therapeutics in these rapidly evolving fields.
FDA Requests Boxed Warning for CAR-Ts Regarding Secondary Malignancy Risk | Regulatory
The FDA is adding a class-wide boxed warning for all 6 FDA-approved CAR T-cell therapies due to the potential risk of secondary T-cell malignancies post-treatment. This follows 22 reported cases of T-cell cancers, with genetic evidence linking the therapy to the development of malignancies in some instances. The FDA, emphasizing the overall benefits of these therapies, is conducting 15-year follow-up studies to assess long-term safety. The move has triggered global regulatory reviews. Despite this, experts like Dr. Rahul Banerjee advise continuing CAR T treatments, citing their significant benefits, including the potential for durable remissions and lesser toxicity compared to other therapies.
Kyverna Develops Ingenui-T, a Solution for Rapid CAR-T Manufacturing | Manufacturing
Kyverna Therapeutics has introduced Ingenui-T, a manufacturing process that reduces CAR-T production time from 8-10 days to three days, using less than 300 ml of whole blood instead of traditional leukapheresis. This innovation, enhancing cell activation and potentially reducing costs, is currently employed in trials for their lead candidate, KYV-101, for Lupus nephritis. While Ingenui-T marks a significant advancement in CAR-T therapy, its broader application to other diseases remains unconfirmed by Kyverna.
ADCs
ENHERTU Granted Priority Review in the U.S. for Solid Tumors | Regulatory
ENHERTU has received Priority Review designation from the FDA for the treatment of unresectable or metastatic HER2-positive solid tumors. The application seeks to position ENHERTU as the first HER2-directed treatment with a tumor-agnostic indication. The acknowledgment of Priority Review underscores the potential for significant improvements over existing treatments. If granted approval, ENHERTU has the potential to redefine the treatment landscape for HER2-expressing cancers. The FDA is expected to make its decision by the PDUFA date on May 30, 2024.
OnCusp Therapeutics Raises $100M Series A | Financing
The company has successfully raised $100 million in a Series A financing round, with prominent participation from Novo Holdings, OrbiMed, and F-Prime Capital. This funding will be deployed to advance CUSP06, an ADC targeting CDH6, to the clinical proof-of-concept stage. According to DeciBio’s TheraTrack, there are currently approximately 5 anti-CDH6 ADCs in development.
Corbus Pharmaceuticals Announced Encouraging Trial Data for its Nectin-4 ADC | Clinical Trial
Corbus has reported the results from the first-in-human study of its Nectin-4 ADC, CRB-701, showing promising safety and efficacy. The study revealed a 43% objective response rate (ORR) and a 71% disease control rate (DCR), with no observed dose-limiting toxicities. Nectin-4 stands out as one of the most sought-after ADC targets today, with approximately 15 unique assets currently in development.
MediLink Therapeutics Announces Agreement with Roche for YL211 | Partnership
MediLink Therapeutics has initiated a global collaboration and licensing agreement with Roche to advance the development of YL211, targeting c-Met against solid tumors. Under this agreement, Roche secures exclusive global rights for the development, manufacturing, and commercialization of YL211. MediLink is set to receive upfront and milestone payments totaling $50 million, with the possibility of additional payments reaching nearly $1 billion.
Dyne Therapeutics Announces Positive Trial Data for DYNE-101 in DM1 | Clinical Trial
The company has revealed positive initial clinical data from its ACHIEVE trial in DM1 patients, and DELIVER in DMD patients. DYNE-101, in the ACHIEVE trial, displayed dose-dependent splicing correction, muscle delivery, and DMPK knockdown. Meanwhile, DYNE-251, in the DELIVER trial, exhibited promising results, surpassing dystrophin expression levels of the standard of care for DMD exon 51 with a significantly lower total dose. Non-oncology ADC programs have been steadily increasing, with promising data especially for the delivery of oligonucleotides.
CellmAbs Announces Agreement with BioNTech | Partnership
CellmAbs has entered into a patent assignment and licensing agreement with BioNTech. BioNTech will acquire one of CellmAbs’ pre-clinical ADCs and additional ADC technology. The deal involves upfront and near-term payments to CellmAbs, with the potential for several hundred million US Dollars in milestone payments and tiered royalty payments. This agreement marks a significant achievement for CellmAbs, a company that raised only a single seed investment in June 2019.
Gene Therapy
Akouos Announces Positive Interim Results in Patients with Hearing Loss | Clinical Trial
Akouos, Inc., a subsidiary of Eli Lilly and Company, announced positive outcomes from the Phase 1/2 AK-OTOF-101 study, indicating successful hearing restoration within 30 days of administering AK-OTOF in the first participant—a person with a decade-long history of hearing loss. The clinical trial, focusing on OTOF-mediated hearing loss, involves a single intracochlear administration of AK-OTOF, with the restoration of hearing assessed through behavioral audiometry and auditory brainstem response. In fall of last year, Regeneron shared interim results from their own program in Phase 1/ 2 testing, which demonstrated improved auditory responses in patients.
Voyager and Novartis Enter Collaboration to Develop Novel Capsids | Partnership
Voyager Therapeutics announced a strategic collaboration and capsid license agreement with Novartis to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Novartis gains a target-exclusive license for Voyager’s TRACER™ capsids and other intellectual property for these diseases, with the companies collaborating on a preclinical gene therapy candidate for HD. The collaboration comes with an upfront payment of $100 million, including a $20 million equity purchase, and up to $1.2 billion in milestones and royalties.
Canadian Authorities Approve Pfizer’s Beqvez | Regulatory
Health Canada has granted regulatory approval for Pfizer's gene therapy, Beqvez, designed for treating certain adults with hemophilia B. The approval is based on the positive results from the BENEGENE-2 study, which included 45 patients and demonstrated Beqvez's noninferiority and superiority to recurrent Factor IX treatment. Pfizer, expecting a U.S. regulatory decision in Q2 2023, acquired Beqvez through a 2014 licensing deal with Spark Therapeutics and is on the heels of CSL Behring’s Hemgenix, which received U.S. and Canadian approvals in 2022 and 2023, respectively.
Fractyl Health Eyes IPO as Markets Open | Financing
Fractyl Health, has accelerated its plans for an IPO following recent biotech market trends. The company aims to sell 7.3 million shares, priced between $14 and $16 each, with potential proceeds of $99 million to $114.3 million, earmarked in part for the ongoing preclinical development of the GLP-1-based pancreatic gene therapy Rejuva. The proposed IPO positions Fractyl among recent biotech offerings, although not reaching the scale of GC Oncology and ArriVent. Fractyl is hoping to ride tailwinds in the obesity and diabetes space thanks in part to Novo Nordisk’s and Eli Lilly’s GLP-1 therapies. The company targets initial human studies for 2024.
Kriya Restructures Pipeline to Focus in Three Segments | Pipeline
Kriya Therapeutics provided an update on its gene therapy pipeline, with hopes to advance up to five programs into clinical trials by the end of 2025. The company is targeting prevalent conditions, including geographic atrophy, thyroid eye disease, diabetes, NASH, trigeminal neuralgia, and epilepsy. Kriya's gene therapy platform incorporates an end-to-end development with internal manufacturing.
Oligo
Strand Therapeutics Receives IND Clearance for Programmable mRNA Therapy STX-001 to Treat Solid Tumors | Clinical Trial
Strand Therapeutics has received FDA clearance to begin a Phase 1 trial of STX-001, a novel programmable mRNA therapy designed to treat solid tumors by expressing an IL-12 cytokine directly into the tumor microenvironment. CEO Jake Becraft expressed enthusiasm, noting the potential of STX-001 to be the first of its kind in oncology. STX-001's results suggest it could significantly enhance clinical responses to solid tumors by delivering therapeutic quantities of IL-12 and synergizing with checkpoint inhibitors.
Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug | Regulatory
Vanda Pharmaceuticals' VCA-894A, an antisense oligonucleotide, has been approved for an investigational new drug application to treat Charcot-Marie-Tooth disease, axonal, type 2S (CMT2). This milestone underscores the potential of ASOs in modulating gene expression to target specific genetic variants, offering hope for patients with conditions like CMT2S, characterized by distal muscle weakness and sensory impairment.
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