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Highlights & Summary
Intro
May was an exciting month for the next-generation therapeutics space, full of new clinical trial and regulatory updates.
Happy reading!
Cell Therapy
- Galapagos and Blood Centers of America announce strategic collaboration to support a decentralized CAR-T manufacturing network | Partnership
- Gameto, a NYC Startup, Raises $33M in Series B for Fertility Treatment Based on George Church’s Research | Financing
- FDA Providers Further Clarity on Safety Testing | Regulatory
Gene Therapy
- PTC Announces Priority Review for Upstaza BLA | Regulatory
- Spark Co-Founder Unveils Latus Bio with $54M Series A | Company Creation
- Pfizer Announces Death in Trial of Duchenne’s Candidate | Clinical Trial
- Freeline Announces Updates on Clinical Programs | Clinical Trial
Oligo Therapy
- Researchers at the University of Pennsylvania Develop an Experimental Avian Flu Vaccine | Clinical Trial
- Strand Therapeutics Announces Programmable mRNA Therapy STX-001 for Solid Tumors Administered on Human Patient | Clinical Trial
Antibody-Drug Conjugates
- SixPeaks Bio AG Debuts With $100 Million in Funding | Company Creation
- Dyne Therapeutics Shares Early Clinical Data for DYNE-101 | Clinical Trial
Cell Therapy
Be Biopharma Announces FDA Clearance of IND Application for BE-101 in Hemophilia B | Regulatory
The FDA has approved Be Bio's B cell therapy, BE-101, for a clinical trial in hemophilia B, expected to begin in the second half of 2024. The Phase 1/2 trial aims to enroll up to 24 patients across four to six U.S. centers. This follows Immusoft's initial human dosing of a similar engineered B cell therapy. BE-101 is designed to be adjustable in dosage and can be redosed as needed. The trial will evaluate the therapy’s safety and its ability to increase factor IX activity levels in patients with moderate to severe hemophilia B, with results expected in 2025.
Galapagos and Blood Centers of America announce strategic collaboration to support a decentralized CAR-T manufacturing network | Partnership
Galapagos has partnered with Blood Centers of America (BCA) to develop a decentralized CAR-T manufacturing network across the U.S. The collaboration utilizes BCA’s numerous community blood centers to support regional hospitals in administering CAR-T therapies, potentially reducing treatment time to seven days. The deal includes Galapagos' existing and future CAR-T therapies, enhancing patient access and maintaining cell integrity by minimizing freeze-thaw cycles. This partnership follows similar agreements with Thermo Fisher and Landmark Bio as Galapagos expands its global decentralized manufacturing strategy.
BMS’ Breyanzi Adds a Third Recent Approval, This Time in Mantle Cell Lymphoma | Regulatory
BMS' CAR-T therapy Breyanzi has received its third recent FDA approval, this time for third-line treatment of adults with relapsed or refractory mantle cell lymphoma. This marks its fourth approval for types of non-Hodgkin lymphoma. Based on the TRANSCEND NHL 001 trial results, 85.3% of patients responded to Breyanzi, with 67.6% achieving a complete response and over half maintaining a response after a year. To support this approval, Bristol Myers has ramped up manufacturing capabilities, reflecting Breyanzi's significant revenue growth. However, the treatment has notable side effects, including cytokine release syndrome and neurological events, and carries a boxed warning for these risks and T cell malignancies.
Gameto, a NYC Startup, Raises $33M in Series B for Fertility Treatment Based on George Church’s Research | Financing
Gameto, a New York-based startup, is pioneering a new IVF technology to potentially eliminate the need for the intense hormone injections currently required for egg maturation. Utilizing research from George Church’s Harvard lab, Gameto has developed a method to mature eggs in the lab using stem cell-derived granulosa cells. Gameto is now preparing for a potential Phase 3 efficacy study in the U.S. and plans commercial launches in Australia and Latin America, where the treatment is regulated differently.
FDA Providers Further Clarity on Safety Testing | Regulatory
The FDA has released a new draft guidance document advising cell therapy sponsors on essential safety testing. The 14-page document details the risks associated with allogeneic cell therapies, such as contamination and genomic changes that could lead to tumor development, and specifies the necessary testing based on the cells’ expansion potential and the number of treatable patients. Public comments on the guidance are invited until July 29.
Gene Therapy
PTC Announces Priority Review for Upstaza BLA | Regulatory
PTC Therapeutics, Inc. announced that the FDA has accepted its BLA for Upstaza™ (eladocagene exuparvovec), a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA has granted Priority Review, with a target decision date of November 13, 2024. Upstaza, designed to treat patients aged 18 months and older with a severe phenotype of AADC deficiency, aims to correct the underlying genetic defect and restore dopamine production. The therapy has shown significant neurological improvements in clinical trials and is administered via a minimally invasive neurosurgical procedure.
Spark Co-Founder Unveils Latus Bio with $54M Series A | Company Creation
Latus Bio, Inc., a biotech company focused on developing gene therapies for CNS disorders, announced its launch and a $54 million Series A financing round led by 8VC and DCVC Bio. The funding will support the development of gene therapies for conditions such as CLN2 and Huntington’s disease, with initial human trials expected by late 2025. The company’s proprietary AAV capsids have demonstrated high gene expression in specific CNS areas in preclinical studies, aiming to improve safety and manufacturing efficiency by lowering the required dose of therapies.
Sarepta Could See FDA Decision on Elevidys Sooner Than Expected | Regulatory
Sarepta Therapeutics indicated that the FDA is ahead of schedule in reviewing the company’s application to expand the label for its Duchenne muscular dystrophy gene therapy, Elevidys. The FDA has informed Sarepta that draft labels will be sent sooner than anticipated, with a decision deadline of June 21. Elevidys, which received accelerated approval last year for boys aged 4 and 5, generated $133.9 million in revenue in Q1. The FDA’s decision is highly anticipated by both patient groups and gene therapy developers.
Pfizer Announces Death in Trial of Duchenne’s Candidate | Clinical Trial
A Duchenne muscular dystrophy patient died of cardiac arrest during a Phase 2 trial of Pfizer’s gene therapy, fordadistrogene movaparvovec, in the DAYLIGHT study for patients aged 2 and 3. Pfizer is pausing dosing in the Phase 3 CIFFREO study for patients aged 4 to 7 as a result. The company is investigating the incident with the trial site investigator and is working with the independent Data Monitoring Committee to review the data. Pfizer emphasized that dosing in other ongoing trials for the gene therapy program has already been completed.
Freeline Announces Updates on Clinical Programs | Clinical Trial
Freeline Therapeutics announced new clinical data from its Phase 1/2 GALILEO-1 trial of FLT201, an AAV gene therapy candidate for Gaucher disease, showing significant reductions in lyso-Gb1 levels and early clinical improvements in bone marrow burden and fatigue. FLT201 demonstrated a favorable safety profile, with no serious adverse events reported. These findings were presented at the ASGCT Annual Meeting, Freeline also announced that FLT201 has received RMAT and PRIME designations to expedite its development.
Oligo
Researchers at the University of Pennsylvania Develop an Experimental Avian Flu Vaccine | Clinical Trial
Researchers at the Perelman School of Medicine have created an experimental mRNA vaccine targeting the avian flu virus H5N1. The vaccine has shown promising results in preclinical trials, producing strong immune responses, and facilitating quick virus elimination with mild symptoms in vaccinated animals. In a comparison between the mRNA vaccine response and the traditional egg-based vaccine response, researchers found that the mRNA vaccine was just as effective, as both vaccines elicited strong antibody responses, regardless of prior seasonal flu exposures.
Twist Bioscience Introduces Multiplexed Gene Fragments | Commercial
Twist Bioscience has introduced Multiplexed Gene Fragments (MGFs), designed for high-throughput screening applications. These customizable double-stranded DNA fragments, ranging from 301 to 500 base pairs, are delivered in pooled formats, significantly enhancing efficiency, and reducing costs for large-scale screening. MGFs are valuable for applications such as CRISPR-based functional screening, antibody discovery, peptide and protein engineering, and mRNA vaccine development.
Takara Bio Launches PrimeCap T7 RNA Polymerase for mRNA Vaccine Development | Commercial
Takara Bio is developing GMP-grade enzymes for mRNA synthesis, including PrimeCap T7 RNA polymerase. The PrimeCap T7 RNA polymerase minimized the generation of dsRNA, which is responsible for unwanted immune response. This new polymerase also promotes increased mRNA capping efficiency because of genetic modification to polymerase activity.
Antisense Oligo Therapeutic Viltepso Shows Lack of Efficacy in Phase 3 Trial | Clinical Trial
NS Pharma shared Phase 3 clinical trial results for Viltepso for treatment of Duchenne Muscular Dystrophy caused by a mutation in the dystrophin gene which is amenable to exon 53 skipping. The primary endpoint of time to stand as tested with a treatment and placebo group, and it was found that there was no significant difference in this endpoint between the placebo group and treatment group after 48 weeks. No treatment emergent adverse events were recorded. In a prior Phase 2 trial, subjects receiving Viltepso showed statistically significant improvement in the time to stand primary endpoint.
Strand Therapeutics Announces Programmable mRNA Therapy STX-001 for Solid Tumors Administered on Human Patient | Clinical Trial
Strand Therapeutics announced the first patient in a Phase 1 trial for the first programmable mRNA therapy for solid tumors in the world has been dosed. STX-001 is an investigational synthetic mRNA therapeutic enabling expression of IL-12 cytokine in the solid tumor microenvironment to promote an immune response. The drug is administered directly into the tumor microenvironment. Prior animal studies showed efficacy, with STX-001 administration enabling cancer cell death and recruitment of T cells and NK cells to the tumor microenvironment.
ADCs
Almac Discovery Nominates a First in Class Bispecific ADC | Pipeline
Almac Discovery has announced the nomination of ALM-401, a first-in-class bispecific ROR1/EGFR ADC for refractory lung cancer. Preclinical studies showed superior efficacy to existing ROR1-targeting ADCs and improved therapeutic activity. ALM-401 is half the size of conventional ADCs, allowing better tumor penetration. Almac is looking for a partner to accelerate clinical development.
MediLink Therapeutics Announces Licensing Agreement with BioNTech | Partnership
The two companies have expanded their collaboration, initially started in October 2023. BioNTech will pay $25 million upfront to use MediLink’s TMALIN ADC platform for several undisclosed targets, with potential additional milestone payments up to $1.8 billion. MediLink retains the right of first negotiation for collaboration on ADC candidates in China. This deal builds on their previous agreement, where BioNTech paid $70 million for rights to a HER3-targeted ADC, further solidifying their strategic partnership in developing novel cancer therapies.
SixPeaks Bio AG Debuts With $100 Million in Funding | Company Creation
The company has launched with $110 million in funding to develop advanced obesity therapies. Founded in 2022 by Versant Ventures, the company secured $30 million in Series A financing and up to $80 million through a partnership with AstraZeneca. SixPeaks Bio is developing an innovative antibody-protein conjugate that combines its Activin receptor (IIA/B) antibody with a GLP-1 peptide, aiming to achieve weight loss without muscle loss. This approach, similar to Amgen's AMG 133, could enhance weight loss outcomes and address a major unmet need in obesity treatment, preserving patients' quality of life.
Dyne Therapeutics Shares Early Clinical Data for DYNE-101 | Clinical Trial
The company announced significant progress in developing treatments for DM1 and DMD. DYNE-101 and DYNE-251 demonstrated promising results in Phase 1/2 trials. DYNE-101 showed a dose-dependent improvement in splicing correction and muscle function in DM1 patients, while DYNE-251 increased dystrophin expression in DMD patients more effectively than existing treatments. These advancements led to a significant rise in Dyne's shares and bolstered the company’s plans to seek accelerated approval for both therapies.
IDT Australia to Pioneer National ADC Manufacturing with Government Grant | Manufacturing
IDT Australia has received a grant from the Victorian government to help fund the creation of Australia's first ADC manufacturing facility. This $3.8 million project in Boronia aims to bolster Australia's capacity to produce advanced treatments. The new facility is expected to position Victoria as a leader in advanced drug manufacturing and significantly boost IDT's growth. IDT has already shown strong financial performance and secured key contracts, such as with Sanofi Australia.
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